Proactiveinvestors Australia Pharmaxis Ltd https://www.proactiveinvestors.com.au Proactiveinvestors Australia Pharmaxis Ltd RSS feed en Mon, 22 Jul 2019 03:54:10 +1000 http://blogs.law.harvard.edu/tech/rss Genera CMS action@proactiveinvestors.com (Proactiveinvestors) action@proactiveinvestors.com (Proactiveinvestors) <![CDATA[News - Pharmaxis on track to complete FDA approval process for Bronchitol in Q1 2020 ]]> https://www.proactiveinvestors.com.au/companies/news/222458/pharmaxis-on-track-to-complete-fda-approval-process-for-bronchitol-in-q1-2020-222458.html Pharmaxis Ltd’s (ASX:PXS) US licensee Chiesi Group has received a complete response letter from the US Food and Drug Administration (FDA) detailing the remaining matters to be addressed before Bronchitol® can be approved for adult cystic fibrosis (CF) patients in the US.

Based upon the clear and achievable path to approval communicated in the FDA complete response letter, Pharmaxis believes that the FDA review of the Bronchitol NDA (New Drug Application) will be completed in Q1 2020.

The main requirement included in the FDA complete response letter is that Chiesi revise the product packaging and user instructions; and then conduct a human factor study (HFS) demonstrating that the revised user components enable healthcare professionals to properly administer the mannitol tolerance test.

READ: Pharmaxis welcomes US FDA committee’s recommendation on use of Bronchitol in CF

Pharmaxis CEO Gary Phillips said: “We are confident that the FDA complete response letter provides a clear and achievable path to approval. 

“FDA requested changes to the product packaging and user instructions require that an HFS be conducted. This is targeted for completion by the end of 2019.

“Based on our experience with healthcare professionals in other markets where our training and packaging has supported thousands of mannitol tolerance tests that have been conducted to ensure patients hypersensitive to mannitol are not prescribed Bronchitol, we are very confident the requested FDA changes can be efficiently implemented and will be effective in achieving the desired goal.

“We have been sharing our experiences in other markets with Chiesi and continue to work collaboratively to prepare for a successful introduction to patients in US cystic fibrosis clinics.”   

Background

Bronchitol is an inhaled dry powder for the treatment of cystic fibrosis and has been the subject of three large scale global clinical trials conducted by Pharmaxis.

It is approved and marketed in Europe, Russia, Australia and several other countries.

Chiesi is responsible for the regulatory approval and commercialisation of Bronchitol in the US.

If Bronchitol is approved by the FDA, Pharmaxis will receive a US$10 million milestone payment on the commercial launch of Bronchitol in the US and mid to high teen percentage royalties on in‐market net sales.

Pharmaxis will manufacture and be the exclusive supplier of Bronchitol for the US market.

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Thu, 20 Jun 2019 12:31:00 +1000 https://www.proactiveinvestors.com.au/companies/news/222458/pharmaxis-on-track-to-complete-fda-approval-process-for-bronchitol-in-q1-2020-222458.html
<![CDATA[Media files - Pharmaxis expects an FDA decision on Bronchitol mid-year after positive committee recommendation ]]> https://www.proactiveinvestors.com.au/companies/stocktube/13328/pharmaxis-expects-an-fda-decision-on-bronchitol-mid-year-after-positive-committee-recommendation-13328.html Mon, 13 May 2019 20:23:00 +1000 https://www.proactiveinvestors.com.au/companies/stocktube/13328/pharmaxis-expects-an-fda-decision-on-bronchitol-mid-year-after-positive-committee-recommendation-13328.html <![CDATA[News - Pharmaxis welcomes US FDA committee’s recommendation on use of Bronchitol in CF ]]> https://www.proactiveinvestors.com.au/companies/news/219929/pharmaxis-welcomes-us-fda-committees-recommendation-on-use-of-bronchitol-in-cf-219929.html Pharmaxis Ltd (ASX:PXS) (FRA:UUD) (OTCMKTS:PXSLY) US licensee Chiesi Farmaceutici S.p.A. has received a positive US Food and Drug Administration advisory committee recommendation for use of the inhaled powder Bronchitol in adult patients with cystic fibrosis (CF).

The recommendation is a positive milestone for international commercialisation of the drug the Australian company developed.

Overnight in Washington, nine of the 16-member Pulmonary‐Allergy Drugs Advisory Committee (PADAC) voted yes to the question : “Is the benefit‐risk profile adequate to support approval of DPM (dry powder mannitol) for the proposed indication of the management of cystic fibrosis to improve pulmonary function in patients 18 years of age and older in conjunction with standard therapies?”

PADAC is responsible for reviewing and evaluating safety and effectiveness data for proposed new products for treating pulmonary disease and conditions with allergic and/or immunologic mechanisms and makes recommendations to the US FDA.

READ: Pharmaxis sees progress in Boehringer Ingelheim’s clinical trial in patients with NASH

Pharmaxis chief executive officer Gary Phillips called the committee’s vote for a recommendation “very encouraging” while noting it was not binding on regulators.

Phillips highlighted the company’s next steps and expected timetable in a market release, saying: “Pharmaxis will continue to support Chiesi to work with the FDA to bring Bronchitol to patients in the US.

“We expect the FDA to make its final decision by mid‐year.”

If the FDA approves the drug for marketing, its manufacturer and exclusive supplier Pharmaxis is set to receive a US$10 million payment on the product’s commercial launch in the US market.

US market approval has long been a goal for the dry-powder therapeutic which studies have shown helps CF patients clear mucus from their lungs and improve their lung function.

Three large-scale global clinical trials of Bronchitol have been undertaken by Pharmaxis.

The company’s efforts have resulted in regulatory approvals in a variety of geographic markets, and marketing in Europe, Russia, Australia and a number of other countries.

 

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Wed, 08 May 2019 22:55:00 +1000 https://www.proactiveinvestors.com.au/companies/news/219929/pharmaxis-welcomes-us-fda-committees-recommendation-on-use-of-bronchitol-in-cf-219929.html
<![CDATA[News - Pharmaxis prepares for advisory committee meeting on use of Bronchitol in the US ]]> https://www.proactiveinvestors.com.au/companies/news/217177/pharmaxis-prepares-for-advisory-committee-meeting-on-use-of-bronchitol-in-the-us-217177.html Pharmaxis Ltd (ASX:PXS) is preparing for an important meeting that will make recommendations on the possible use of its Bronchitol® product for adult cystic fibrosis patients in the United States.

The company is supporting its licensee Chiesi Group in preparing for a meeting with the Pulmonary-Allergy Drugs Advisory Committee (PADAC) which will take place in May.

Chiesi, which is responsible for the regulatory approval process, has been advised by the US Food and Drug Administration (FDA) that the PADAC meeting will take place in the US on May 8.

READ: Pharmaxis sees progress in Boehringer Ingelheim’s clinical trial in patients with NASH

Pharmaxis is an Australian pharmaceutical research company focused on inflammation and fibrosis. It has a portfolio of products at various stages of development and approval.

One of these products, Bronchitol, for treatment of cystic fibrosis, is marketed in Europe, Russia and Australia and the company is seeking to market it in the large US market.

As part of this process, Chiesi resubmitted the Bronchitol New Drug Application to the FDA in December of 2018.

Used in treatment of cystic fibrosis patients

Bronchitol is a precision spray-dried form of mannitol, delivered to the lungs by a specially designed, portable inhaler.

It works by rehydrating the airway/lung surface and promoting a productive cough. Bronchitol is used for the treatment of cystic fibrosis patients.

READ: Pharmaxis begins phase one clinical trial of compound targeting pancreatic cancer

PADAC, which is convened by the FDA, reviews and evaluates available data concerning the safety and effectiveness of marketed and investigational human drug products for use in the treatment of pulmonary disease and diseases with allergic and/or immunologic mechanisms.

Through this process, PADAC makes appropriate recommendations to the Commissioner of Food and Drugs in the US.

The committee comprises a core of 11 voting members including the chair.

These members are selected by the Commissioner from among authorities knowledgeable in the fields of pulmonary medicine, allergy, clinical immunology, and epidemiology or statistics.

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Tue, 26 Mar 2019 13:12:00 +1100 https://www.proactiveinvestors.com.au/companies/news/217177/pharmaxis-prepares-for-advisory-committee-meeting-on-use-of-bronchitol-in-the-us-217177.html
<![CDATA[News - Pharmaxis gains new substantial holder with 6.89% interest ]]> https://www.proactiveinvestors.com.au/companies/news/215758/pharmaxis-gains-new-substantial-holder-with-689-interest-215758.html Pharmaxis Ltd (ASX:PXS) has gained a substantial holder in D&A Income Limited which now holds more than 27 million Pharmaxis shares representing a 6.89% interest.

D&A initially held abut 8 million shares but purchased a further 19.1 million late last month for a cash consideration of more than $5 million.

 

Pharmaxis is an Australian pharmaceutical research company focused on inflammation and fibrosis with a portfolio of products at various stages of development and approval.

The company’s development pipeline focuses on its expertise in amine oxidase chemistry and includes:

A series of lysyl oxidase like 2 (LOXL2) inhibitors targeting fibrotic diseases of the liver, heart, kidney and lunch that recently completed phase-one clinical trials;

A lysyl oxidase (LOX) inhibitor in development for pancreatic cancer that recently completed preclinical development; and

A potent inhibitor of semicarbazide-sensitive amine oxidase (SSAO) called BI 1467335 to develop treatment for the liver-related condition non-alcoholic steatohepatitis (NASH) and other inflammatory diseases.

Pharmaxis manufactures and exports its approved products from its facility in Sydney, including Bronchitol for the treatment of cystic fibrosis and Aridol, a lung function test for asthma.

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Mon, 04 Mar 2019 19:57:00 +1100 https://www.proactiveinvestors.com.au/companies/news/215758/pharmaxis-gains-new-substantial-holder-with-689-interest-215758.html
<![CDATA[News - Pharmaxis sees progress in Boehringer Ingelheim’s clinical trial in patients with NASH ]]> https://www.proactiveinvestors.com.au/companies/news/215174/pharmaxis-sees-progress-in-boehringer-ingelheims-clinical-trial-in-patients-with-nash-215174.html Pharmaxis Ltd (ASX:PXS) is looking forward to the results from Boehringer Ingelheim’s Phase 2a clinical trial in patients with non‐alcoholic steatohepatitis (NASH).

Boehringer Ingelheim has completed enrolment of the trial which is assessing BI 1467335 that works by blocking leucocyte adhesion and tissue infiltration in inflammatory processes underlying NASH.

BI 1467335 was acquired from Pharmaxis in May 2015 and is also in an ongoing Boehringer Ingelheim Phase 2a clinical trial for diabetic retinopathy.

Whilst Pharmaxis will not receive any milestone payments for a positive Phase 2a trial result under its contract with Boehringer Ingelheim, a positive result will increase the likelihood of receiving future milestones with the next one being triggered by the start of a phase 3 study.

Phase 2a expected to report in the second half of 2019

The Phase 2a NASH trial is a multi‐centre, double‐blind design in 114 patients with clinical evidence of NASH. The trial is being conducted in nine countries across North America and Europe.

The primary objectives are to establish proof of clinical principle, investigate suitable dosing, and to evaluate the safety of BI 1467335.

Patients have been randomized to either one of four dosages of BI 1467335 or to placebo for a 12‐week treatment period followed by a 4‐week observation period.  

The trial is expected to report in the second half of 2019. A subsequent Phase 2b study will seek to confirm and extend these findings.  

NASH is a major cause of liver fibrosis

Non‐alcoholic fatty liver disease (NAFLD), the most common liver disorder in Western industrialized nations, and its more serious form NASH, is highly prevalent amongst patients with type 2 Diabetes.

NASH is a major cause of liver fibrosis and cirrhosis and is an area of high unmet medical need with no treatments currently available.

The high prevalence of type 2 diabetes and obesity is expected to make NASH one of the most common causes of advanced liver disorders in the coming decades.

25% of the general adult population in the world has NAFLD and the prevalence of NASH has been found to range from 1.5% to 6.45% in current research, a number twice as high as 20 years ago.

READ: Pharmaxis well funded as potential partners assess its anti‐fibrotic mechanisms

Pharmaxis CEO Gary Phillips said: “We are delighted to see the progress made on this study and look forward to this trial reporting in the second half of this year.

“Whilst we do not receive any milestone payments under our contract with Boehringer Ingelheim for a positive trial result, this will still be the first proof of clinical principle of the Pharmaxis drug acquired in 2015.

“A positive result will increase the likelihood of receiving future milestones with the next one being triggered by the start of a phase 3 study.”

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Mon, 25 Feb 2019 14:37:00 +1100 https://www.proactiveinvestors.com.au/companies/news/215174/pharmaxis-sees-progress-in-boehringer-ingelheims-clinical-trial-in-patients-with-nash-215174.html
<![CDATA[News - Pharmaxis begins phase one clinical trial of compound targeting pancreatic cancer ]]> https://www.proactiveinvestors.com.au/companies/news/215089/pharmaxis-begins-phase-one-clinical-trial-of-compound-targeting-pancreatic-cancer-215089.html Pharmaxis Ltd (ASX:PXS) has begun dosing of the first subject in its phase-one clinical trial of an anti-fibrotic Lysyl Oxidase (LOX) inhibitor, focused on treating cancer.

The program compound is an oral once-a-day drug that inhibits all members from the lysyl oxidase enzyme family.

The company successfully cleared pre-clinical safety and toxicity studies in the third quarter of 2018 and has shown significant reductions in in-vivo models of kidney and lung fibrosis, as well as myelofibrosis and pancreatic cancer.

New approach for stromal tumour treatment

Pharmaxis chief executive officer Gary Phillips said: “Moving a new drug into the clinic for the first time is always a significant milestone and this will be the fourth time we have accomplished this during the last 5 years.

“I’m delighted with the productivity of the Pharmaxis team and excited about the potential of this drug to bring a new approach to therapy for hard to treat stromal tumours like pancreatic cancer.

“Our current plan is that a successful phase 1 trial outcome would be the launch pad for a quick transition into cancer patients and to that end we are already in discussion with key leaders and working on potential trial designs.”

READ: Pharmaxis well-funded as potential partners assess its anti-fibrotic mechanisms

The double-blind placebo-controlled study consists of two stages.

The first single ascending dose will be conducted in 40 healthy subjects divided into five groups with each subject taking a single dose or placebo.

The second multiple ascending dose stage will be conducted in 16 healthy subjects and divided into two groups, with each group receiving a different dose or placebo for seven days.

 

 

READ: Pharmaxis LOXL2 program phase 2 ready after completion of 13-week toxicity studies

The company received reports on the 13-week toxicity studies in January 2019 which were conducted for each of its two Lysyl Oxidase Like 2 (LOXL2) inhibitors.

Both were drug compounds were tested at a range of doses in two species over the 13-week period to establish the No Observed Adverse Effect Level (NOAEL).

Doses that resulted in 85% or greater inhibition of the target enzyme in the phase 1 studies were below the human equivalent NOAEL doses and therefore an adequate safety margin to start phase 2 studies up to three months.

The Australian pharmaceutical research company is focused on inflammation and fibrosis with a portfolio of products at various stages of development and approval.Its product Bronchitol for cystic fibrosis is marketed in Europe, Russia and Australia and its product Aridol, for the assessment of asthma is sold in the US, Europe, Australia and Asia.

Its product Bronchitol for cystic fibrosis is marketed in Europe, Russia and Australia and its product Aridol, for the assessment of asthma is sold in the US, Europe, Australia and Asia.

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Fri, 22 Feb 2019 14:57:00 +1100 https://www.proactiveinvestors.com.au/companies/news/215089/pharmaxis-begins-phase-one-clinical-trial-of-compound-targeting-pancreatic-cancer-215089.html
<![CDATA[News - Pharmaxis well funded as potential partners assess its anti‐fibrotic mechanisms ]]> https://www.proactiveinvestors.com.au/companies/news/213663/pharmaxis-well-funded-as-potential-partners-assess-its-antifibrotic-mechanisms-213663.html Pharmaxis Ltd (ASX:PXS) (FRA:UUD) (OTCMKTS:PXSLY) has attracted the attention of large pharmaceutical companies, with several potential partners conducting scientific reviews of the company’s phase 2 study-ready program of two LOXL2 inhibitors with anti‐fibrotic mechanisms.

The well-funded company ended the December quarter with $43 million cash after receiving €28 million ($42 million) of milestone payments in the 2017-18 financial year from the structured deal for SSAO/VAP-1 inhibitor BI 1467335 (formerly PXS‐4728A) it signed with developer-buyer Boehringer Ingelheim in 2015.

READ: Pharmaxis LOXL2 program phase 2 ready after completion of 13-week toxicity studies

Pharmaxis chief executive officer & executive director Gary Phillips confirmed the company had been focusing on its upcoming milestones this month which have been outlined in the company’s quarterly shareholder update.

Mr Phillips, a former Novartis senior executive of many years, acknowledged the company’s two lysyl oxidase like 2 (LOXL2) inhibitors had reached an important development stage.

That turning point involved deciding on whether Pharmaxis should move into phase 2 studies for LOXL2 program which has anti‐fibrotic mechanism or take the partnering track.

The LOXL2 enzyme is fundamental to the fibrotic cascade that follows chronic inflammation in the liver disease non‐alcoholic steatohepatitis (NASH), cardiac fibrosis, kidney fibrosis, and idiopathic pulmonary fibrosis (IPF).

LOXL2 enzyme also plays a role in some cancers.

READ: Pharmaxis partner Chiesi resubmits new drug application for Bronchitol in the US

Pharmaxis’ anti‐fibrotic programs are a drawcard in the pharmaceutical industry, given they are one of a small number in clinical development and demand is strong.

The Sydney-based company has studied its LOXL2 inhibitor compounds in preclinical studies of NASH, IPF, kidney and cardiac fibrosis before putting them through phase 1 studies and three-month toxicity studies.

 

Pharmaxis has previously targeted NASH with BI 1467335 (PXS‐4728A), a Pharmaxis drug discovery that excited Boehringer Ingelheim and is bringing in significant milestone payments for Pharmaxis.

The company’s LOXL2 program is also now attracting attention for its potential for use in NASH but also IPF.

Phillips has been clear about the strategic options for the LOXL2 inhibitors, saying: “We could move directly into phase 2 ourselves and push for the clinical proof of concept that would add further value to the program.

“Alternatively, we could find a large pharma partner to drive the drug discoveries into phase 2.

“It is obvious from the number of licensing and collaboration deals for early stage NASH programs in recent months that demand is strong in this indication, and we also know from our own discussions with pharma that our program is equally attractive as a treatment for idiopathic pulmonary fibrosis (IPF).

“Our healthy cash balance puts us in a strong position to take our time, choose the most appealing option and negotiate the best deal for our shareholders.

“The next step is to commence the commercial partnering discussions and assess the range and type of deal structures on offer.”

READ: Pharmaxis reveals first sales of Aridol in the US after successful relaunch

The Pharmaxis CEO had previously spoken to Proactive Investors’ Stocktube video channel about progress on the LOXL2 program, acknowledging the company had been speaking to potential partners that were following a due diligence process.

Phillips said: “Our team is on stand-by, we’ve been having face-to-face meetings … and conference calls.

“The next stage is commercial discussions once they’re comfortable.”

Revenue generators

Pharmaxis’ $1.7 million in revenues for the December quarter and $3 million revenue for the December half-year came from a variety of sources.

Among these was $232,000 net in revenues in the December quarter from its long-time pipeline product Bronchitol in Australia, Western Europe and Russia and Eastern Europe.

Sales in the three regions were $657,000 in the December 2018 half-year.

READ: Pharmaxis receives positive results from clinical trial of treatment for fibrotic diseases

The inhaled dry powder for the treatment of cystic fibrosis, Bronchitol is approved and marketed in Europe, Russia, Australia and a number of other countries.

Bronchitol was added to the essential drugs list in Russia and was granted national reimbursement status in what is an attractive market on January 1 this year.

The milestones come exactly a year after the Australian Government widened its approval for the drug, positively affecting sales which grossed $294,000 in the December quarter in Australia and $543,000 in the December half-year.

READ: Pharmaxis moving into phase I clinical trial of its small molecule LOX inhibitor

Pharmaxis commercialisation partner Chiesi Farmaceutici SpA is working to secure Bronchitol approval in the US market, resubmitting an application rejected by the US Food and Drug Administration (FDA) in 2013 after an additional Phase 3 study and answering queries raised in a complete response letter from the regulator.

The Australian company is expecting a six to 12-month regulatory approvals process will be followed with the FDA and hopes for an approval that would prompt a US$10 million milestone payment on commercial launch, high-teen royalties and exclusive supply to the US market.

Another income earner for the company in the December quarter was the Aridol lung function test to help doctors diagnose and manage asthma.

Aridol has Australian, major European, US and South Korea approvals.

Sales in the four regions grossed $1.1 million in the December quarter, with December half sales sitting at $1.6 million.

Broader pipeline

Pharmaxis has an oral lysyl oxidase (LOX) program where the drug candidate inhibits all LOX family members and has potential anti‐fibrotic application in severe fibrotic indications.

Studies in animal pre-clinical models have shown promise for use in myelofibrosis and pancreatic cancer.

A phase 1 clinical trial in healthy subjects is expected to start this quarter.

Another pipeline candidate is a dual-acting drug-inhibiting SSAO and myeloperoxidase (MPO) for treating inflammation.

Quarterly results and expected cash outflows

Pharmaxis had $1.3 million of revenues in the quarter, spent $2.7 million on new drug development and $1.3 million on product manufacturing and operating costs.

The company used a total of $4.1 million in cash on operating activities and $229,000 for investing activities with the bulk of that investment going towards property, plant and equipment.

Pharmaxis ended the quarter with $42 million cash with no debt or financing facilities.

Major shareholders

Pharmaxis had 394,291,298 shares on issue on December 31, with the company’s top 20 shareholders holding 68.6% of the company at year’s end.

BVF Partners LP was Pharmaxis’ largest shareholder with a 22% stake.

Arix Bioscience Holdings Limited was the second largest shareholder, with 11%, followed by Australian Ethical Australian Share Fund on 7%.

— with Danielle Doporto

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Wed, 30 Jan 2019 21:15:00 +1100 https://www.proactiveinvestors.com.au/companies/news/213663/pharmaxis-well-funded-as-potential-partners-assess-its-antifibrotic-mechanisms-213663.html
<![CDATA[News - Pharmaxis LOXL2 program phase 2 ready after completion of 13-week toxicity studies ]]> https://www.proactiveinvestors.com.au/companies/news/212738/pharmaxis-loxl2-program-phase-2-ready-after-completion-of-13-week-toxicity-studies-212738.html Pharmaxis Ltd (ASX:PXS) has now received reports on all of the 13‐week toxicity studies conducted for each of its two Lysyl Oxidase Like 2 (LOXL2) inhibitors.

The company is now ready to enter phase 2 clinical studies for fibrotic diseases such as non‐alcoholic steatohepatitis (NASH), cardiac fibrosis and idiopathic pulmonary fibrosis (IPF).

Adequate safety margin to start phase 2 studies

Both the drug compounds were tested at a range of doses in two species over a 13‐week period to establish the No Observed Adverse Effect Level (NOAEL). 

For both compounds, doses that resulted in 85% or greater inhibition of the target enzyme in the phase 1 studies were below the human equivalent NOAEL doses in all toxicity studies and therefore an adequate safety margin to start phase 2 studies of up to three months in length.  

With the data package complete, Pharmaxis is now conducting a final series of scientific briefings to potential partners.

READ: Pharmaxis resubmits new drug application for Bronchitol in the US

Pharmaxis chief executive officer Gary Phillips said: “The results of the toxicity studies complete our scientific package of data for the LOXL2 program. 

“It is a high quality and comprehensive data package that is a testament to the expertise and experience of the Drug Discovery and Clinical Development teams at Pharmaxis.

READ: Pharmaxis reveals first sales of Aridol® in US following successful relaunch

“We have provided the large pharma companies who have been closely monitoring our progress with the latest study results and are now in the process of supporting them to complete their scientific due diligence. 

“We are keen to answer remaining scientific questions of potential partners, discuss their proposed clinical development strategies should they acquire or license the program and progress discussions concerning appropriate commercial terms.”

 

Pharmaxis’ LOXL2 program compounds are highly selective small molecule inhibitors of LOXL2 that can be administered orally and the completed pre‐clinical development program supports the potential of both compounds to treat fibrotic disease in one or more organs.

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Thu, 17 Jan 2019 13:21:00 +1100 https://www.proactiveinvestors.com.au/companies/news/212738/pharmaxis-loxl2-program-phase-2-ready-after-completion-of-13-week-toxicity-studies-212738.html
<![CDATA[News - Pharmaxis resubmits new drug application for Bronchitol in the US ]]> https://www.proactiveinvestors.com.au/companies/news/211533/pharmaxis-resubmits-new-drug-application-for-bronchitol-in-the-us-211533.html Pharmaxis Ltd’s (ASX:PXS) licensee Chiesi Group has resubmitted a new drug application (NDA) for Bronchitol with the US Food and Drug Administration (FDA).

The resubmission responds to matters raised by the FDA in its Complete Response Letter issued in March 2013 and includes the results of the phase 3 clinical trial conducted after consultation with the FDA.

READ: Pharmaxis meets primary endpoint of phase 3 trial of Bronchitol

Bronchitol is a precision spray‐dried form of mannitol, delivered to the lungs by a specially designed, portable inhaler.

It works by rehydrating the airway/lung surface and promoting a productive cough. Bronchitol is used for the treatment of cystic fibrosis patients.

Pharmaxis expects the FDA review process to take between six and 12 months to conclude. Chiesi is responsible for the regulatory approval and commercialisation of Bronchitol in the US.

READ: Pharmaxis reveals first sales of Aridol® in US following successful relaunch

If Bronchitol is approved by the FDA, Pharmaxis will receive a US$10 million milestone payment on the commercial launch of Bronchitol in the US and, mid to high teen percentage royalties on in‐market net sales.

Pharmaxis chief executive officer Gary Phillips said: “The resubmission of the Bronchitol NDA concludes a substantial investment and effort by Pharmaxis and Chiesi over an extended period of time.

“We will continue to support Chiesi in progressing the application with the FDA and look forward to the conclusion of the review in the second half of 2019.”

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Thu, 20 Dec 2018 13:47:00 +1100 https://www.proactiveinvestors.com.au/companies/news/211533/pharmaxis-resubmits-new-drug-application-for-bronchitol-in-the-us-211533.html
<![CDATA[News - Pharmaxis reveals first sales of Aridol® in US following successful relaunch ]]> https://www.proactiveinvestors.com.au/companies/news/211098/pharmaxis-reveals-first-sales-of-aridol-in-us-following-successful-relaunch-211098.html Pharmaxis Ltd (ASX:PXS) has achieved the first sales of its asthma diagnostic test Aridol® in the US following the relaunch of the product.

This follows US FDA approval in August 2018 for its Sydney manufacturing facility to produce and supply Aridol to the US through its exclusive distribution partner Methapharm Inc.

Methapharm has extensive experience in the sales channels and specialist centres that conduct lung function testing.

READ: Pharmaxis receives FDA approval to produce Aridol for US market

Pharmaxis’s CEO Gary Phillips said, “Aridol was approved by the FDA in 2011 to identify bronchial hyperresponsiveness and commercialised by Pharmaxis in the US until its withdrawal from the market in 2013 as part of a corporate restructuring when we closed the facility used to manufacture Aridol for the US.

“Two years ago Pharmaxis partnered with Methapharm to re‐enter the US market and commenced the validation work required to have our remaining manufacturing facility approved by the FDA for Aridol. 

“Both Methapharm and Pharmaxis believe there remains a strong need in the US for objective tests to aid physicians in diagnosing asthma. It’s good to be back.”

Advancing partnership talks for LOXL2

Pharmaxis has a portfolio of drugs ranging from the pre-clinical phase through to the clinical phase and finally drugs in the commercial phase such as Aridol® and Bronchitol®.

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Thu, 13 Dec 2018 11:53:00 +1100 https://www.proactiveinvestors.com.au/companies/news/211098/pharmaxis-reveals-first-sales-of-aridol-in-us-following-successful-relaunch-211098.html
<![CDATA[Media files - Pharmaxis advancing partnership talks for LOXL2, starting pancreatic cancer program ]]> https://www.proactiveinvestors.com.au/companies/stocktube/11300/pharmaxis-advancing-partnership-talks-for-loxl2-starting-pancreatic-cancer-program-11300.html Fri, 23 Nov 2018 14:03:00 +1100 https://www.proactiveinvestors.com.au/companies/stocktube/11300/pharmaxis-advancing-partnership-talks-for-loxl2-starting-pancreatic-cancer-program-11300.html <![CDATA[News - Pharmaxis moving into phase I clinical trial of its small molecule LOX inhibitor ]]> https://www.proactiveinvestors.com.au/companies/news/209514/pharmaxis-moving-into-phase-i-clinical-trial-of-its-small-molecule-lox-inhibitor-209514.html Pharmaxis Ltd (ASX:PXS) has completed the preclinical package on its anti‐fibrotic Lysyl Oxidase (LOX) program focused on pancreatic cancer and will this week file an ethics submission to enable progress into a phase I clinical trial in healthy volunteers.

The trial is planned to commence in the first quarter of 2019.

WATCH: Pharmaxis advancing partnership talks for LOXL2, starting pancreatic cancer program

This is the third program from Pharmaxis’ amine oxidase chemistry platform to reach the important stage of entering human clinical trials.

The company has a successful track record of research, development and commercialisation of human healthcare products for the treatment of fibrotic and inflammatory diseases

The company has a successful track record of research, development and commercialisation of human healthcare products for the treatment of fibrotic and inflammatory diseases

Significantly, Pharmaxis is the first company to progress a small molecule LOX inhibitor into clinical development.

The compound is an oral once‐a‐day drug that inhibits all lysyl oxidase family members (LOX, LOXL1, 2, 3 & 4).

It successfully cleared pre‐clinical safety and toxicity studies in the third quarter of 2018 and has shown significant reductions in fibrosis in in‐vivo models of kidney fibrosis, lung fibrosis, myelofibrosis and pancreatic cancer.

Whereas the Pharmaxis selective LOXL2 inhibitors that are currently completing phase I studies are suited to chronic fibrotic conditions such as IPF and NASH, this all‐encompassing LOX inhibitor is well positioned for the treatment of severe fibrosis as well as cancer with prominent stroma (connective tissue) or fibrotic metastatic niches.

WATCH: The videos of the presentations at today’s onsite Investor Research Briefing READ: Pharmaxis receives positive results from clinical trial of treatment for fibrotic diseases

Pharmaxis is collaborating with Garvan Institute of Medical Research to investigate the therapeutic potential of LOX inhibition in pancreatic cancer.  

Researchers at the Garvan Institute have evidence in mouse models that inhibition of the LOX family alters the tumour microenvironment rendering tumours more susceptible to existing therapies.

The team has also generated positive results in in-vitro and in‐vivo models of pancreatic cancer using the Pharmaxis LOX inhibitor.

READ: Pharmaxis receives positive results from LOXL2 clinical trial

Pharmaxis CEO Gary Phillips said: “Moving another a drug into the clinic further validates the productivity and expertise of our drug discovery and development team.

“This latest drug has shown real promise in pre‐clinical testing and our ambition is to move it into pancreatic cancer patients as soon as possible and demonstrate clinical proof of concept in a disease which has a very high unmet need. 

“The clinical program we envisage in pancreatic cancer is very efficient relative to other fibrotic indications so the opportunity to add significant value to the asset by taking it into phase II clinical trials prior to partnering is very attractive.”

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Tue, 20 Nov 2018 13:11:00 +1100 https://www.proactiveinvestors.com.au/companies/news/209514/pharmaxis-moving-into-phase-i-clinical-trial-of-its-small-molecule-lox-inhibitor-209514.html
<![CDATA[News - Pharmaxis receives positive results from clinical trial of treatment for fibrotic diseases ]]> https://www.proactiveinvestors.com.au/companies/news/209233/pharmaxis-receives-positive-results-from-clinical-trial-of-treatment-for-fibrotic-diseases-209233.html Pharmaxis Ltd (ASX:PXS) has received positive results from a phase I clinical trial for the second of its LOXL2 inhibitor compounds which are being developed to treat fibrotic diseases.

These include the liver disease known as non-alcoholic steatohepatitis (NASH) and the lung disease idiopathic pulmonary fibrosis (IPF).

READ: Pharmaxis receives positive results from LOXL2 clinical trial

Repeating the positive results seen in the phase I trial of the first inhibitor compound, released last month, the excellent drug-like properties demonstrated in earlier pre-clinical testing were confirmed.

There were no adverse safety findings in the first or second stages of the study and the pharmacokinetic profile showed the expected dose-related increases in exposure.

Both inhibitors complete phase I studies

Pharmaxis CEO Gary Phillips said: “Several large pharma companies are interested in the program where both of our LOXL2 inhibitors have now successfully completed phase I studies and demonstrated a best in class profile with 24-hour inhibition of the target enzyme from a single daily dose.

“In a further significant scientific advancement, we have also managed to underline the relevance of the program to potential partners by using our proprietary research tools to confirm that our compounds directly inhibit the activity of the raised levels of LOXL2 seen in diseased tissue from NASH and IPF animal models.”

Two-stage study

The double-blind placebo-controlled study consisted of two stages.

The first single ascending dose stage was conducted in 48 healthy subjects divided into six groups with each taking a single dose ranging from 5mg to 200mg or placebo. 

The second multiple ascending dose stage was conducted in 24 healthy subjects divided into three groups which each received a single daily dose of either 50mg, 100mg, 200mg or placebo for 14 days.

Significant target engagement of the LOXL2 enzyme by both compounds has been demonstrated in blood serum for a full 24 hours from a single dose over a 14-day period.

The second compound achieved more than 85% inhibition over 24 hours from a 100mg daily dose, thus achieving the target for this program.

READ: Pharmaxis gains Arix Bioscience Plc as a new substantial shareholder

Pharmaxis’ LOXL2 program compounds are highly selective small molecule inhibitors of LOXL2 that can be administered orally.

The soon to be completed pre-clinical development program supports the potential of both compounds to treat fibrotic disease in one or more organs.

Remaining elements

Phillips said: “The only remaining elements necessary to finalise the data package that companies are now conducting diligence on, are the 3‐month toxicity studies on both compounds which are due to report later this quarter.

“This is slightly delayed versus initial expectations due to the availability of time slots at the contract organisations we use but successful 3-month studies will significantly enhance the LOXL2 program.

“Following the completion of the data package, Pharmaxis intends to conduct a final series of scientific briefings to potential partners before moving to commercial partnering discussions to secure a comprehensive licensing agreement in 2019,” he added.

Portfolio of products

Pharmaxis is an Australian pharmaceutical research company focused on inflammation and fibrosis with a portfolio of products at various stages of development and approval.

Its product Bronchitol® for cystic fibrosis is marketed in Europe, Russia and Australia while Aridol®, for the assessment of asthma, is sold in Europe, Australia and Asia.

The company’s development pipeline is centred on its expertise in amine oxidase chemistry and includes a series of Lysyl Oxidase Inhibitors under clinical development targeting fibrotic diseases of the heart, kidney, liver and lung.

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Thu, 15 Nov 2018 10:30:00 +1100 https://www.proactiveinvestors.com.au/companies/news/209233/pharmaxis-receives-positive-results-from-clinical-trial-of-treatment-for-fibrotic-diseases-209233.html
<![CDATA[News - Pharmaxis receives positive results from LOXL2 clinical trial ]]> https://www.proactiveinvestors.com.au/companies/news/206839/pharmaxis-receives-positive-results-from-loxl2-clinical-trial-206839.html Pharmaxis Ltd (ASX:PXS) has received positive results from the phase I clinical trial for the first of its LOXL2 inhibitor compounds being developed to treat fibrotic diseases.

These include the liver disease known as non‐alcoholic steatohepatitis (NASH) and the lung disease idiopathic pulmonary fibrosis (IPF).

Importantly, there were no adverse safety findings in the study and the pharmacokinetic profile showed the expected dose-related increases in exposure.

READ: Pharmaxis adds valuable experience with Ed Rayner appointment

Pharmaxis’s CEO Gary Phillips said: “I’m delighted that the excellent pharmacokinetic parameters and the significant and long-lasting inhibition of the target LOXL2 enzyme demonstrated in the single dose stage of the study earlier this year completely translated into the profile we have seen in the multiple dosing study.

“This drug profile has led to increased interest from major pharmaceutical companies looking for good anti-fibrotic programs to acquire. 

“Today’s announcement that enzyme inhibition is further enhanced after daily dosing over 14 days goes a long way to completing the data package on which we will base continuing scientific and commercial discussions with potential partners during the current quarter.” 

Two-stage study

The double‐blind placebo-controlled study consisted of two stages.

The first single ascending dose stage was conducted in 48 healthy subjects divided into six groups with each taking a single dose ranging from 10-400 milligrams or placebo.

The second multiple ascending dose stage was conducted in 24 healthy subjects divided into three groups which each received a single daily dose ranging from 100-400 milligrams or placebo for 14 days.

Early signs show drug inhibits target enzyme

The clinical trial investigated the degree to which the drug can inhibit the target enzyme LOXL2, which is implicated in several different fibrotic diseases.

Importantly, Pharmaxis has been able to demonstrate a large and highly significant inhibition of this enzyme in blood serum for a full 24 hours from a single dose.

Furthermore. daily dosing over a 14‐day period met Pharmaxis’s targeted effect of greater than 80% inhibition at the 400-milligram dose.

Intention to partner the LOXL2 program after phase I

Phillips added: “We believe that the best in class LOXL2 inhibition and the availability of two compounds with differentiated pharmacokinetic profiles make this program very attractive.

“We look forward to concluding a licensing deal with a partner committed to develop the compounds in indications where there remain a lack of treatment options and significant commercial opportunities.”

Additionally, the phase I trial for a second LOXL2 compound being studied has recently completed dosing and will report in the current quarter.

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Thu, 11 Oct 2018 12:20:00 +1100 https://www.proactiveinvestors.com.au/companies/news/206839/pharmaxis-receives-positive-results-from-loxl2-clinical-trial-206839.html
<![CDATA[News - Pharmaxis adds valuable experience with Ed Rayner appointment ]]> https://www.proactiveinvestors.com.au/companies/news/205073/pharmaxis-adds-valuable-experience-with-ed-rayner-appointment-205073.html Pharmaxis Ltd (ASX:PXS) shareholders have elected to appoint biotech investment specialist Edward Rayner as a non‐executive director of the company’s board.

Rayner is an investment director at Arix Bioscience Plc (LSE:ARIX), which will hold an 11% interest in Pharmaxis after the recently approved share placement is completed.

Before joining Arix, Rayner spent 18 years as an equity analyst and portfolio manager in Europe and Australia working for Alliance Bernstein, AMP Capital, UBS Asset Management and JP Morgan Investment Management.

READ: Pharmaxis gains Arix Bioscience Plc as a new substantial shareholder

Pharmaxis Chairman Malcolm McComas said: “We welcome Ed to the board as a non‐executive director.

“His global experience as a specialist biotech investor is very relevant to Pharmaxis as we continue to develop new partnerships and seek out investment opportunities where we can add value. 

“With Ed based in London and fellow Board member Kathleen Metters in New York, our reach and networks are enhanced.  We look forward to Ed’s contribution.” 

Shareholders also approve placement

The extraordinary general meeting (EGM) held yesterday also ratified and approved shares issued in the recent $24 million two-tranche placement.

Notably, the placement price of 32.5 cents, represented a premium to the share price at the time of announcement.

Arix committed to invest $14.2 million in two tranches as part of the placement.

The funding will be used to advance Pharmaxis research into new treatments for a range of fibrotic diseases, including non-alcoholic steatohepatitis (NASH), idiopathic pulmonary fibrosis (IPF), heart and kidney fibrosis.

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Tue, 18 Sep 2018 12:32:00 +1000 https://www.proactiveinvestors.com.au/companies/news/205073/pharmaxis-adds-valuable-experience-with-ed-rayner-appointment-205073.html
<![CDATA[News - Pharmaxis gains Arix Bioscience Plc as a new substantial shareholder ]]> https://www.proactiveinvestors.com.au/companies/news/203235/pharmaxis-gains-arix-bioscience-plc-as-a-new-substantial-shareholder-203235.html Pharmaxis Ltd (ASX:PXS) has raised approximately $6.5 million from UK-based Arix Bioscience Plc (LON:ARIX) via the first tranche of a placement of shares at 32.5 cents each for a 5.6% interest.

In its first Australian investment, Arix has committed to invest a total of $14.2 million as part of Pharmaxis’ two-tranche share placement to sophisticated and institutional investors.

Subject to shareholder approval on September 17, 2018, Arix will emerge with an 11.1% shareholding.

READ: Pharmaxis secures $24 million at premium to support partnering negotiations

The proceeds of the placement will be used to advance Pharmaxis research into new treatments for a range of fibrotic diseases, including non-alcoholic Steatohepatitis (NASH), Idiopathic Pulmonary Fibrosis (IPF), heart and kidney fibrosis.

Pharmaxis’ lead development program is an anti-fibrotic Lysyl Oxidase-Like type 2 (LOXL2) inhibitor that acts at a crucial stage of pathogenic fibrosis.

The final stages of the phase I studies and phase II enabling toxicity studies for LOXL2 are expected to be completed in the third quarter of 2018.

Early LOXL2 results have been attracting interest

As disclosed recently, data received to date from the phase I LOXL2 studies has attracted multiple interested potential partners.

Importantly, Pharmaxis has previously shown it can execute on sale agreements with big pharmaceutical companies after finalising a  2015 deal with Boehringer Ingelheim with significant upfront and milestone payments for its clinical research.

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Tue, 21 Aug 2018 08:00:00 +1000 https://www.proactiveinvestors.com.au/companies/news/203235/pharmaxis-gains-arix-bioscience-plc-as-a-new-substantial-shareholder-203235.html
<![CDATA[News - Pharmaxis receives FDA approval to produce Aridol for US market ]]> https://www.proactiveinvestors.com.au/companies/news/202908/pharmaxis-receives-fda-approval-to-produce-aridol-for-us-market-202908.html Pharmaxis Ltd (ASX:PXS) has received good news for its respiratory business after gaining approval from the US Food and Drug Administration (FDA) for its Sydney manufacturing facility to produce Aridol for the US market.

Aridol is a lung function test designed to help doctors diagnose and manage asthma by detecting active airway inflammation.

Commercial sales of Aridol in the US will commence in the coming December quarter via Pharmaxis’ exclusive distributor in North America, Methapharm Inc.

Notably, Methapharm are experts in the specialist respiratory diagnostic market.

READ: Pharmaxis secures $24 million at premium to support partnering negotiations

Pharmaxis’ CEO Gary Phillips said: “Aridol has proven to be a valuable diagnostic aid in respiratory function laboratories in many global markets. 

“I am delighted that the FDA, having inspected our Sydney manufacturing facility, has given approval to recommence supply of the product to the US market.

“We are also very pleased to have Methapharm, who have many years of experience in the US, to commercialise the product.

“This partnership will bring the benefits of this indirect bronchial challenge test kit to US patients with respiratory symptoms and add a valuable income stream to this business segment of Pharmaxis.”

US market an opportunity to double Aridol sales

Global sales of Aridol excluding the US are currently $2 million per annum and Pharmaxis believes that the US market offers an opportunity to at least double that revenue. 

The company’s Sydney facility in Sydney manufactures Aridol for Europe, Australia and South Korea as well as Bronchitol for Europe, Australia and Russia.

Bronchitol is an inhaled dry powder for the treatment of cystic fibrosis.

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Wed, 15 Aug 2018 12:20:00 +1000 https://www.proactiveinvestors.com.au/companies/news/202908/pharmaxis-receives-fda-approval-to-produce-aridol-for-us-market-202908.html
<![CDATA[News - Pharmaxis secures $24 million at premium to support partnering negotiations ]]> https://www.proactiveinvestors.com.au/companies/news/202252/pharmaxis-secures-24-million-at-premium-to-support-partnering-negotiations-202252.html Pharmaxis Ltd (ASX:PXS) has secured $24 million in funding after receiving commitments from sophisticated and institutional investors for a two-tranche share placement.

Notably, the placement price is 32.5 cents, which is a 3.1% premium to the last closing price.

Funds will strengthen the balance sheet as the company conducts partnering negotiations for its clinical development program into lysyl oxidase 2 inhibitors (LOXL2), expected to occur in 2H18.

Major investment from global healthcare company Arix

The placement received strong support from specialist global healthcare investors including UK-based Arix Bioscience Plc (LON:ARIX), a global healthcare and life science company.

Arix has committed to invest $14.2 million and will emerge with an 11.1% shareholding post-approval.

In conjunction with the investment by Arix, the Pharmaxis board has approved the nomination of Edward Rayner to join the board pending shareholder approval at the upcoming EGM.

Existing shareholder BVF Partners LP has also committed to invest a further $7.1 million to increase its holding to 22.9%, the maximum allowable under ASIC creep provisions.

READ: Pharmaxis powering ahead with phase I LOXL2 studies

Pharmaxis’ CEO Gary Phillips said: “We are delighted with the strong support for the capital raising from Arix and BVF as we head into LOXL2 partnering negotiations.

“This will put the company in a strong position to negotiate the best partnering outcome and continue investing in its preclinical pipeline.

“Pharmaxis will also be strengthened by the global reach of Arix and the addition to the board, subject to shareholder approval, of Ed Rayner. Ed has 18 years of experience as an equity analyst and portfolio manager in Europe and Australia.”

$54 million in cash post-raising

Adjusting the cash balance for the two-tranche placement, the company would have a healthy $54 million pro‐forma cash balance at June 30, 2018, post-raising.

Early LOXL2 results have been attracting interest

As disclosed recently, data received to date from the phase I LOXL2 studies has attracted multiple interested potential partners.

Importantly, Pharmaxis has previously shown it can execute on deals with big pharmaceutical companies through its May 2015 deal with Boehringer Ingelheim.

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Mon, 06 Aug 2018 13:24:00 +1000 https://www.proactiveinvestors.com.au/companies/news/202252/pharmaxis-secures-24-million-at-premium-to-support-partnering-negotiations-202252.html
<![CDATA[News - Pharmaxis enters trading halt ahead of share placement ]]> https://www.proactiveinvestors.com.au/companies/news/202159/pharmaxis-enters-trading-halt-ahead-of-share-placement-202159.html Pharmaxis Ltd (ASX:PXS) has been granted a trading halt by the ASX pending the release of information regarding a share placement.

The company’s shares will remain halted until the earlier of, the release of relevant information, or, the commencement of trading next Tuesday.

READ: Pharmaxis powering ahead with phase I LOXL2 studies

Pharmaxis is an established pharmaceutical research company working on new therapies to treat inflammatory and fibrotic diseases.

This includes liver disease NASH, pulmonary fibrosis, kidney and liver fibrosis, inflammatory bowel diseases and cancer.

The company finished the June quarter with just over $31 million in cash.

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Fri, 03 Aug 2018 11:41:00 +1000 https://www.proactiveinvestors.com.au/companies/news/202159/pharmaxis-enters-trading-halt-ahead-of-share-placement-202159.html
<![CDATA[News - Pharmaxis powering ahead with phase I LOXL2 studies ]]> https://www.proactiveinvestors.com.au/companies/news/201702/pharmaxis-powering-ahead-with-phase-i-loxl2-studies-201702.html Pharmaxis Ltd’s (ASX:PXS) drug discovery team comprises a team of experienced scientists who have been working on two lead candidates that inhibit the fibrotic enzyme LOXL2.

After extensive pre‐clinical testing these compounds were deemed fit to go into human clinical development.

The initial findings from the single ascending dose (SAD) stage were revealed during the June quarter and are noteworthy.

READ: Pharmaxis phase I trial attracts interest from multiple pharmaceutical companies

So far both drugs have good safety profiles, behave in a predictable fashion as the dose is increased and, most importantly, achieved a significant and long-lasting inhibition of the target enzyme which positions them as best in class drugs.

This differentiates them from the competition of other small molecule drugs and antibodies which have failed to demonstrate this same essential feature.  

Progress can be slow but not for Pharmaxis

Pharmaxis’ CEO Gary Phillips said: “There are times in the life of a research-based company when progress can be slow; waiting for late phase clinical studies to recruit patients is but one example.

“However, this last quarter at Pharmaxis has been the exact opposite.

“We have data from the phase I studies in our LOXL2 program coming at us daily, ongoing discussions with multiple interested companies in that program further energised at the June BIO partnering conference, and turning points in some programs within our pre‐clinical pipeline that suggest great things to come.”

Early results attracting interest

The good inhibition of the enzymatic activity for 24 hours after a single dose made for interesting discussions held with potential partners at BIO 2018 in Boston last month.

Pharmaxis has previously shown it can execute on deals with big pharmaceutical companies and has seen an increase in the number of companies and level of interest shown as it progresses its LOXL 2 program.

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Fri, 27 Jul 2018 14:39:00 +1000 https://www.proactiveinvestors.com.au/companies/news/201702/pharmaxis-powering-ahead-with-phase-i-loxl2-studies-201702.html
<![CDATA[News - Pharmaxis phase I trial attracts interest from multiple pharmaceutical companies ]]> https://www.proactiveinvestors.com.au/companies/news/198827/pharmaxis-phase-i-trial-attracts-interest-from-multiple-pharmaceutical-companies-198827.html Pharmaxis Ltd (ASX:PXS) has briefed potential partners attending the BIO18 partnering conference in Boston on data emerging from the phase I clinical studies of its anti‐fibrotic LOXL2 inhibitor program in which two compounds are showing significant inhibition of the enzyme for 24 hours with a single oral dose.  

LOXL2 or lysyl oxidase like 2 has been implicated as a key factor in various fibrotic diseases in organs such as the liver, lung, heart and kidney.

WATCH: Pharmaxis has the "secret sauce" for developing drugs through to commercialisation

Pharmaxis’ two compounds have both cleared the first stage of the studies where single oral doses of different strengths were trialled in healthy volunteers. 

There were no adverse safety findings in this first stage.   Both drugs are now entering the final stage where different fixed doses are given for 14 days.

In addition to studying their safety and pharmacokinetic profiles the two studies are also investigating the degree to which these drugs can inhibit the target enzyme LOXL2.

Importantly, Pharmaxis has been able to demonstrate a large and highly significant inhibition of this enzyme for a full 24 hours with a single oral dose.

Pharmaxis chief executive officer Gary Phillips said: “The data package being reviewed by several multinational pharmaceutical companies under confidentiality agreements is now maturing rapidly as we see the final data being generated by ongoing pre‐clinical and clinical studies. 

“There are a number of key features which have contributed to an increase in the already strong interest amongst these companies.”

The amine oxidase platform at Pharmaxis has generated small molecule enzyme inhibitors to a range of important disease targets that are at various stages of development. 

The SSAO inhibitor acquired by Boehringer is in phase II and the LOXL2 inhibitors are in phase I trials.

Meanwhile a compound inhibiting both myeloperoxidase (MPO) and SSAO and another compound inhibiting all the LOX family of enzymes are both in the final stages of pre‐clinical testing with phase I trials scheduled to commence in the next 6 to 12 months.

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Thu, 14 Jun 2018 11:58:00 +1000 https://www.proactiveinvestors.com.au/companies/news/198827/pharmaxis-phase-i-trial-attracts-interest-from-multiple-pharmaceutical-companies-198827.html
<![CDATA[Media files - Pharmaxis has the "secret sauce" for developing drugs through to commercialisation ]]> https://www.proactiveinvestors.com.au/companies/stocktube/9343/pharmaxis-has-the-secret-sauce-for-developing-drugs-through-to-commercialisation-9343.html Tue, 22 May 2018 09:45:00 +1000 https://www.proactiveinvestors.com.au/companies/stocktube/9343/pharmaxis-has-the-secret-sauce-for-developing-drugs-through-to-commercialisation-9343.html <![CDATA[Media files - Pharmaxis has key drivers in place for short and long term value creation ]]> https://www.proactiveinvestors.com.au/companies/stocktube/9289/pharmaxis-has-key-drivers-in-place-for-short-and-long-term-value-creation-9289.html Tue, 15 May 2018 10:28:00 +1000 https://www.proactiveinvestors.com.au/companies/stocktube/9289/pharmaxis-has-key-drivers-in-place-for-short-and-long-term-value-creation-9289.html <![CDATA[News - Pharmaxis boosts cash position securing sustainable future ]]> https://www.proactiveinvestors.com.au/companies/news/196009/pharmaxis-boosts-cash-position-securing-sustainable-future-196009.html Pharmaxis Ltd (ASX:PXS) finished the March quarter with $34.5 million thanks in part to a $15 million milestone payment from drug licence partner Boehringer Ingelheim.

This strong financial position provides the company with certainty while it continues to extract value from its portfolio of developing drug assets.

 

The company’s goal is to identify unmet needs in human health, discover and develop new solutions and then sell those assets on to large pharma companies.

Pharmaxis demonstrated its capability in this area with the program it sold to Boehringer in 2015 which has potential to be worth up to $625 million.

The next potential payment milestone for Pharmaxis is the commencement of phase III trials by Boehringer which can unlock up to $94 million.

Growth in Europe for Bronchitol

As well as developing drugs and seeking partnerships, Pharmaxis has two respiratory products approved in various world markets that it manufactures at its facility in Australia.

Bronchitol for cystic fibrosis has been the subject of three large-scale clinical trials and is approved and marketed in Europe, Russia and Australia.

Aridol for the diagnosis and assessment of asthma is approved and marketed in Europe, Australia and Asia.

Notably, sales for Bronchitol in Western Europe during the March quarter were $963,000 compared to $887,000 in the March 2017 quarter and $587,000 in the December 2017 quarter.

Russia and US represent market growth opportunities for Pharmaxis.

LOXL2 inhibitor program receiving interest from large pharma

Pharmaxis’ anti fibrotic LOXL2 inhibitor program is reaching its conclusion and attracting strong interest from multiple potential large pharma partners.

Phase I clinical trials that commenced in the second half of 2017 have completed the single ascending dose stage and are proceeding into the final multiple ascending dose stages this quarter before reporting in the September quarter.

Three-month tox studies and the scale-up of manufacturing have commenced so that a potential partner will be able to commence phase II studies without delay.

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Mon, 30 Apr 2018 14:07:00 +1000 https://www.proactiveinvestors.com.au/companies/news/196009/pharmaxis-boosts-cash-position-securing-sustainable-future-196009.html
<![CDATA[Media files - Pharmaxis reveals a quarterly shareholder update full of highlights ]]> https://www.proactiveinvestors.com.au/companies/stocktube/9162/pharmaxis-reveals-a-quarterly-shareholder-update-full-of-highlights-9162.html Mon, 30 Apr 2018 10:01:00 +1000 https://www.proactiveinvestors.com.au/companies/stocktube/9162/pharmaxis-reveals-a-quarterly-shareholder-update-full-of-highlights-9162.html <![CDATA[Media files - Pharmaxis seeing a great deal of interest from potential LOXL2 program partners ]]> https://www.proactiveinvestors.com.au/companies/stocktube/8729/pharmaxis-seeing-a-great-deal-of-interest-from-potential-loxl2-program-partners-8729.html Sun, 11 Feb 2018 20:10:00 +1100 https://www.proactiveinvestors.com.au/companies/stocktube/8729/pharmaxis-seeing-a-great-deal-of-interest-from-potential-loxl2-program-partners-8729.html <![CDATA[Media files - Pharmaxis making strong progress with significant value inflection points ahead ]]> https://www.proactiveinvestors.com.au/companies/stocktube/8711/pharmaxis-making-strong-progress-with-significant-value-inflection-points-ahead-8711.html Wed, 07 Feb 2018 04:09:00 +1100 https://www.proactiveinvestors.com.au/companies/stocktube/8711/pharmaxis-making-strong-progress-with-significant-value-inflection-points-ahead-8711.html <![CDATA[News - Pharmaxis outlines share price catalysts for 2018 ]]> https://www.proactiveinvestors.com.au/companies/news/190782/pharmaxis-outlines-share-price-catalysts-for-2018-190782.html Pharmaxis Ltd (ASX:PXS) has made strong progress in drug development with value inflection points in a number of programs expected in 2018.

The research‐based biotech company has identified novel ways to cure diseases that are poorly treated and has generated new drugs which are then rigorously tested.

After receiving $42 million in milestone payments from Boehringer Ingelheim, the company expects more results later in 2018 which will impact the risk-adjusted valuation of this deal.

READ: Pharmaxis strengthens balance sheet with $15 million milestone payment

Gary Phillips, chief executive officer, said: “The NASH study is due to read out in the September quarter and the diabetic retinopathy study in the December quarter this year.

“Both studies will provide the first clinical proof of concept in humans and, if positive, would represent a huge step forward in their progress towards the clinic and the delivery of more financial rewards for Pharmaxis.

“The second key event will be the mid‐year read out from the phase 1 studies that are underway on the two lead candidates from our anti fibrotic LOXL2 program.”

Company takes control of collaboration

Late last year Pharmaxis decided to take full scientific and commercial control of its collaboration with UK biotech Synairgen.

Phillips said: “This was driven by our increasing confidence in this program and the very high level of interest we are receiving from large pharmaceutical companies that are active in the liver and lung fibrosis markets.

“We have used some of the cash that the BI deal has provided to substantially increase our interest in the commercial returns from the program in exchange for a one‐off payment to Synairgen.

“The end of phase I is not a classic major value inflection point for a new drug given that you have shown safety in healthy volunteers but not yet any efficacy in patients with the disease being targeted.

LOXL2 program a significant valuation catalyst

“Despite this I believe that this point in our LOXL2 program is significant from a valuation perspective.

“There are very few anti fibrotic drugs under development and the number of companies chasing a good asset is high.

“This means that companies are looking earlier and earlier in the development pipeline of biotechs to identify promising drugs to add to their development portfolio.

“I met with many of these companies at the JP Morgan conference in San Francisco earlier this month and several are already investing considerable resources in doing due diligence on our science whilst the phase I studies are ongoing.

“I anticipate that a major deal in the second half of this year will be our reward if the phase I studies are successful.”

Pharmaxis has a portfolio of products at various stages of development and approval.

READ: Pharmaxis secures further Australian government reimbursement for Bronchitol

Its product Bronchitol for cystic fibrosis is marketed in Europe, Russia and Australia.

Another product, Aridol, for the assessment of asthma, is sold in Europe, Australia and Asia.

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Tue, 30 Jan 2018 08:33:00 +1100 https://www.proactiveinvestors.com.au/companies/news/190782/pharmaxis-outlines-share-price-catalysts-for-2018-190782.html
<![CDATA[News - Pharmaxis strengthens balance sheet with $15 million milestone payment ]]> https://www.proactiveinvestors.com.au/companies/news/189839/pharmaxis-strengthens-balance-sheet-with-15-million-milestone-payment-189839.html Pharmaxis Ltd (ASX:PXS) has earned a $15 million payment as Boehringer Ingelheim has expanded its development plan for BI 1467335 to include treatment of blindness caused by diabetes.

Dosing of the first patient in Boehringer Ingelheim’s Phase 2a clinical trial in patients with diabetic retinopathy triggered the payment.

It is the second disease targeted by the drug developed by Pharmaxis, which is being used in a Phase 2a clinical trial for the chronic liver disease NASH (non‐alcoholic steatohepatitis).

The studies into NASH and diabetic retinopathy are due to report in the second half of 2018.

Today’s payment further strengthens the Pharmaxis balance sheet, which held $39 million in cash at the end of September 2017.

WATCH NOW: Pharmaxis' CEO Gary Phillips updates on partnership with Boehringer Ingelheim

Gary Phillips, chief executive officer, said: “This milestone payment marks the expansion of the Boehringer Ingelheim development plan for BI 1467335 into two diseases.

“All the potential development milestones in the $625 million deal would be payable to Pharmaxis should both indications be approved.

“Both development plans attract the same total development payments through to approval, with the second indication milestone payments weighted more towards approval.

“The next milestones from BI are payable if they choose to commence phase 3 trials, at which time the two indications would attract €62 million [circa $94 million] in milestones.”

A leading cause of vision loss

Diabetic retinopathy is the leading cause of vision loss in adults aged 20‐74.

Of an estimated 285 million people with diabetes mellitus worldwide, 33% have signs of the disease.  

The Phase 2a clinical trial of BI 1467335 in diabetic retinopathy will randomise 100 patients to drug or placebo for a 12‐week treatment period with an additional 12‐week follow‐up period.

After the study reports later this year, a subsequent Phase 2b study will seek to confirm and extend the findings.

Two-pronged clinical program

Boehringer Ingelheim’s two‐pronged clinical program with BI 1467335 is focused on tackling the common diseases related to the complications of diabetes which affect millions worldwide.

The drug was acquired by Boehringer Ingelheim in 2015 with an upfront payment of $41 million to initially study NASH.

Additional portfolio

Pharmaxis has a portfolio of products at various stages of development and approval.

Its product Bronchitol for cystic fibrosis is marketed in Europe, Russia and Australia.

READ: Pharmaxis secures further Australian government reimbursement for Bronchitol

Another product, Aridol, for the assessment of asthma, is sold in Europe, Australia and Asia.

The company’s development pipeline is centred on its expertise in amine oxidase chemistry.

It includes a series of Lysyl Oxidase Inhibitors that have entered clinical development targeting fibrotic diseases of the heart, kidney, liver and lung.

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Thu, 11 Jan 2018 11:55:00 +1100 https://www.proactiveinvestors.com.au/companies/news/189839/pharmaxis-strengthens-balance-sheet-with-15-million-milestone-payment-189839.html
<![CDATA[News - Pharmaxis secures further Australian government reimbursement for Bronchitol ]]> https://www.proactiveinvestors.com.au/companies/news/189417/pharmaxis-secures-further-australian-government-reimbursement-for-bronchitol-189417.html Pharmaxis Ltd (ASX:PXS) has achieved extended reimbursement of its drug Bronchitol (mannitol) on the Pharmaceutical Benefits Scheme (PBS).

This means that from the start of 2018, eligible people with cystic fibrosis who are taking Pulmozyme® (dornase alfa), another CF (cystic fibrosis) medication, will be able to add reimbursed Bronchitol to their treatment regime.

Cystic fibrosis is a genetic condition affecting one in every 2500 Australian babies.

People with the condition develop an abnormal amount of excessively thick and sticky mucus within their lungs, airways and the digestive system.

What is Bronchitol

Bronchitol has an active ingredient in mannitol, which is delivered as an inhalable dry powder to restore the airway surface liquid.

Other benefits include enhanced mucus clearance, improved lung function while reducing incidence of lung infections.

Bronchitol has been studied in more than 1000 clinical trial patients4 and is indicated for appropriate CF patients over six years of age.

Pharmaxis plans for a Bronchitol FDA re-submission by Chiesi in 2018.

Chiesi is the distributor for the major EU markets.

Importance of the listing on PBS

This new listing will allow clinicians treating cystic fibrosis to use two agents that work differently, and are proven to work together in the CF lung, to improve mucus clearance.

PBS reimbursement of this combined treatment approach will also significantly reduce paperwork for CF treating teams and improve access for patients.

Gary Phillips, chief executive officer for Pharmaxis, commented:

"We are delighted that more Australians with cystic fibrosis will now have access to treatment with Bronchitol under this expanded listing.

"We are proud that a locally developed and manufactured product is helping people living with this incurable disease."

An expanded PBS listing for Bronchitol was supported by the CF community, healthcare professionals and Cystic Fibrosis Australia in submissions to the government committee advising on medicine reimbursement.

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Wed, 03 Jan 2018 09:07:00 +1100 https://www.proactiveinvestors.com.au/companies/news/189417/pharmaxis-secures-further-australian-government-reimbursement-for-bronchitol-189417.html
<![CDATA[News - Pharmaxis takes full control of collaboration on LOXL2 inhibitors and expands scientific program ]]> https://www.proactiveinvestors.com.au/companies/news/188760/pharmaxis-takes-full-control-of-collaboration-on-loxl2-inhibitors-and-expands-scientific-program-188760.html Pharmaxis Ltd (ASX:PXS) has taken full scientific and commercial control of the collaboration with UK biotech company Synairgen plc (AIM:SNG).

The pharmaceutical research company has expanded the scientific program on LOXL2 inhibitors to maximise its value to potential partners.

Pharmaxis has also substantially increased its interest in the program in return for a payment of £5 million to Synairgen.

Collaboration has progressed pre-clinical stage

The companies have been working on the pre-clinical stage of an antifibrotic Lysyl Oxidase type 2 (LOXL2) inhibitor program.

The LOXL2 enzyme is fundamental to the progression of fibrosis in the liver disease NASH.

This also applies to fibrotic disease in organs such as the heart, kidney, and lung where idiopathic pulmonary fibrosis (IPF) remains a high unmet need.

READ: Pharmaxis boosts its drug discovery ranks

The move to full control has resulted in Pharmaxis doubling the program scope to encompass two lead candidates with unique properties.

It intends to take these to the end of phase I trials with additional toxicology data delivered in parallel to enable them to be phase II ready by mid‐2018.

Full funding responsibility

Pharmaxis has full control and funding responsibility of the ongoing partnering process and aims to conclude a deal after phase I studies have reported.

The company has significantly increased its share of any partnering deal for the LOXL2 program in fibrotic diseases to over 80%.

Synairgen has retained a reduced but fixed percentage share of all future partnering revenues.

The collaboration began in August 2015 with the aim of developing and partnering a small molecule inhibitor of the LOXL2 enzyme at the end of phase I trials.

Two compounds identified

An extensive pre‐clinical program identified two compounds that have all the characteristics of successful once a day, oral drugs.

These show excellent efficacy in several different in vivo fibrosis models.

In regulatory toxicity studies, the two compounds have been well‐tolerated and shown good safety profiles.

WATCH: Pharmaxis' CEO Gary Phillips updates on partnership with Boehringer Ingelheim

Gary Phillips, chief executive officer, said: “The collaboration with Synairgen in the program’s pre‐clinical stage has served its purpose very well.

“However, the increased interest in NASH together with Pharmaxis’ progress over the last few years, its track record of delivering significant partnering deals and enhanced financial position, means we are now in a position to develop this product.

“This will be done under our control with our own resources so that we can maximise shareholder value.”

Discussions with pharma companies

The LOXL2 inhibitor program has been the subject of discussions with large pharma companies since the beginning of 2016.

Pharmaxis expects the interest to intensify as the two compounds progress through phase I clinical trials.

In parallel with these trials, Pharmaxis will enable scientific due diligence of the program by select large pharma companies interested in subsequent partnering discussions.

Phillips said: “To realise the opportunity in a competitive market we need to deliver convincing proof of concept in multiple disease models.

“This includes a phase I toxicology data package that both clears the phase I hurdle and makes this asset phase II ready.”

What is NASH

NASH is a major cause of fibrosis and cirrhosis of the liver and is an area of high unmet medical need with no treatments currently available.

Current research has reported the prevalence of NASH to range from 1.5% to 6.45%, a number twice as high as 20 years ago and the market has been forecast by Deutsche Bank to be worth in excess of $35 billion by 2025.

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Thu, 14 Dec 2017 11:03:00 +1100 https://www.proactiveinvestors.com.au/companies/news/188760/pharmaxis-takes-full-control-of-collaboration-on-loxl2-inhibitors-and-expands-scientific-program-188760.html
<![CDATA[News - Pharmaxis boosts its drug discovery ranks ]]> https://www.proactiveinvestors.com.au/companies/news/185843/pharmaxis-boosts-its-drug-discovery-ranks-185843.html Pharmaxis (ASX:PXS) has made several key appointments to further strengthen its capability in drug discovery and development.

Following a global search, Dr Dieter Hamprecht has been appointed as the head of chemistry in the Pharmaxis drug discovery team.

The company’s drug discovery track record is strong having generated five lead candidates s in fibrosis and inflammation over four years from its amine oxidase chemistry platform.

In addition, Pharmaxis has appointed two new members to its Scientific Advisory Board (SAB), Professor Darren Kelly and Dr Kathleen Metters.

Gary Phillips, CEO, commented: “I am delighted that Pharmaxis has been able to attract high calibre employees and scientific advisors.

“The SSAO inhibitor PXS‐4728A acquired by Boehringer is in two phase 2 trials, two LOXL2 inhibitor candidates are about to enter phase 1 trials whilst a drug inhibiting both yeloperoxidase and SSAO and one inhibiting lysyl oxidases are in the final stages of pre‐candidate profiling before commencing pre‐clinical development with the aim to start phase 1 trials 2018.”

Dr Dieter Hamprecht

Dr Hamprecht was previously the managing director of Boehringer Ingelheim’s research group in Milan.

He has a distinguished career as a medicinal chemist having held senior positions at GSK and Boehringer with 37 publications and 44 patents to his name.

WATCH NOW: Pharmaxis' CEO Gary Phillips updates on partnership with Boehringer Ingelheim ]]>
Thu, 19 Oct 2017 16:35:00 +1100 https://www.proactiveinvestors.com.au/companies/news/185843/pharmaxis-boosts-its-drug-discovery-ranks-185843.html
<![CDATA[Media files - Pharmaxis' CEO Gary Phillips updates on partnership with Boehringer Ingelheim ]]> https://www.proactiveinvestors.com.au/companies/stocktube/8045/pharmaxis-ceo-gary-phillips-updates-on-partnership-with-boehringer-ingelheim-8045.html Fri, 08 Sep 2017 08:25:00 +1000 https://www.proactiveinvestors.com.au/companies/stocktube/8045/pharmaxis-ceo-gary-phillips-updates-on-partnership-with-boehringer-ingelheim-8045.html <![CDATA[Media files - Pharmaxis is set to commence phase 1 trials ]]> https://www.proactiveinvestors.com.au/companies/stocktube/8042/pharmaxis-is-set-to-commence-phase-1-trials-8042.html Thu, 07 Sep 2017 07:55:00 +1000 https://www.proactiveinvestors.com.au/companies/stocktube/8042/pharmaxis-is-set-to-commence-phase-1-trials-8042.html <![CDATA[News - Pharmaxis and collaborator Synairgen advance towards human clinical studies ]]> https://www.proactiveinvestors.com.au/companies/news/183502/pharmaxis-and-collaborator-synairgen-advance-towards-human-clinical-studies-183502.html Pharmaxis Ltd (ASX:PXS) and its collaborator UK biotechnology company Synairgen plc (AIM: SNG) have completed the pre-clinical development stage of their antifibrotic Lysyl Oxidase type 2 (LOXL2) inhibitor program.

This allows the first compound to commence human clinical phase 1 studies in the December quarter 2017.

As part of the collaboration, Synairgen is funding preclinical toxicology work and phase 1, and utilising its BioBank human tissue models technology platform.

Gary Phillips, chief executive officer for Pharmaxis, commented:

"The extensive pre-clinical program performed on our program compounds has confirmed that they have all the characteristics of a successful once a day, oral drug.

"They have shown excellent efficacy in several different in vivo fibrosis models including fibrosis of the liver, lung, kidney and heart.

"These findings have been the subject of presentations at a number of international scientific conferences and more data will be presented at similar upcoming events as the phase 1 studies proceed.

"In regulatory toxicity studies, our compounds have been well tolerated and shown a good safety profile."

Pharmaxis has therefore consolidated its position as a significant competitor in the NASH market as the LOXL2 inhibitor joins its SSAO inhibitor in the clinic.

What is NASH

NASH is a major cause of fibrosis and cirrhosis of the liver and is an area of high unmet medical need with no treatments currently available.

Current research has reported the prevalence of NASH to range from 1.5% to 6.45%, a number twice as high as 20 years ago and the market has been forecast by Deutsche Bank to be worth in excess of $35 billion by 2025.

The potential for the LOXL2 program to be developed as a therapeutic for NASH positions Pharmaxis as a key player in this important and growing disease.

In addition to its anti-fibrotic LOXL2 program, Pharmaxis also has an ongoing interest in the anti-inflammatory SSAO/VAP-1 inhibitor PXS-4728A with which its partner Boehringer has just commenced a NASH phase 2 study.

READ NOW: Pharmaxis to receive cash injection as Boehringer Ingelheim initiates new trials

Phillips added: "NASH is clearly a growing and valuable market for the future.

"It’s a disease that is attracting a variety of well-funded research strategies and it is anticipated that a combination approach that tackles the metabolic, inflammatory and fibrotic drivers of NASH, will be necessary in order to reduce the long term effects of this disease.

"As lysyl oxidase type 2 is the key enzyme in the fibrotic cascade we anticipate that our LOXL2 program will be attractive to many of the multinational pharmaceutical companies that are building a portfolio approach to treating NASH and some other fibrotic diseases such as IPF."

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Wed, 06 Sep 2017 11:46:00 +1000 https://www.proactiveinvestors.com.au/companies/news/183502/pharmaxis-and-collaborator-synairgen-advance-towards-human-clinical-studies-183502.html
<![CDATA[News - Pharmaxis' largest shareholder BVF Partners LP lifts stake ]]> https://www.proactiveinvestors.com.au/companies/news/183128/pharmaxis-largest-shareholder-bvf-partners-lp-lifts-stake-183128.html Pharmaxis Ltd (ASX:PXS) has received notice that its largest shareholder, BVF Partners LP, has now increased its position to 19.9% through on-market purchases.

Pharmaxis has a proven track record of early stage partnering and taking products through to commercialisation.

Earlier in the month the company outlined that it will receive an €18 million milestone payment following Boehringer Ingelheim initiating a European and North American Phase IIa trial.

READ NOW: Pharmaxis to receive cash as Boehringer Ingelheim initiates trials

Pharmaxis has the potential to receive total upfront milestone payments of A$630 million, with A$68 million already received.

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Wed, 30 Aug 2017 11:12:00 +1000 https://www.proactiveinvestors.com.au/companies/news/183128/pharmaxis-largest-shareholder-bvf-partners-lp-lifts-stake-183128.html
<![CDATA[Media files - Pharmaxis gets A$27M injection from Boehringer Ingelheim ]]> https://www.proactiveinvestors.com.au/companies/stocktube/7997/pharmaxis-gets-a27m-injection-from-boehringer-ingelheim-7997.html Mon, 28 Aug 2017 08:19:00 +1000 https://www.proactiveinvestors.com.au/companies/stocktube/7997/pharmaxis-gets-a27m-injection-from-boehringer-ingelheim-7997.html <![CDATA[News - Pharmaxis to receive cash injection as Boehringer Ingelheim initiates new trials ]]> https://www.proactiveinvestors.com.au/companies/news/182963/pharmaxis-to-receive-cash-injection-as-boehringer-ingelheim-initiates-new-trials-182963.html Pharmaxis Ltd (ASX:PXS) will receive an €18 million milestone payment following Boehringer Ingelheim initiating a European and North American Phase IIa trial in NASH with BI 1467335 (formerly known as PXS-4728A).

The Phase II program of investigational drug candidate BI 1467335, which was acquired from Pharmaxis, will start with a 12 week Phase IIa proof of clinical principle study in non-alcoholic steatohepatitis (NASH).

The new study underscores Boehringer’s aspiration to deliver more first in class medicines with breakthrough potential for patients with cardio metabolic diseases.

WATCH NOW: Pharmaxis gets A$27M injection from Boehringer Ingelheim

Gary Phillips, chief executive officer for Pharmaxis, commented: “Pharmaxis selected Boehringer Ingelheim as our partner for PXS-4728A both because of the company’s reputation as a leader in cardio metabolic research and development, and its outstanding track record in advancing external research.

"Today’s announcement of the start of this Phase IIa clinical trial for NASH is excellent news and is very significant for Pharmaxis.

"It triggers the payment of an €18 million (A$27 million) milestone to Pharmaxis and opens the path to a total of €195 million in milestone payments as the drug progresses through development and approval for this indication.

"The initiation of Phase II trials in a second indication later this year by Boehringer Ingelheim can bring the total potential value of the partnership with Boehringer Ingelheim to €418.5 million (A$627 million) plus sales milestones and high single digit earn-out payments on annual net sales."

Liver disease

Non-alcoholic fatty liver disease (NAFLD), the most common liver disorder in Western industrialised nations, and its more serious form NASH, is highly prevalent amongst patients with type 2 Diabetes.

NASH is a major cause of liver fibrosis and cirrhosis and is an area of high unmet medical need with no treatments currently available.

The high prevalence of type 2 diabetes and obesity is expected to make NASH one of the most common causes of advanced liver disorders in coming decades.

25% of the general adult population in the world has NAFLD and the prevalence of NASH has been found to range from 1.5% to 6.45% in current research, a number twice as high as 20 years ago.

Importance of BI 1467335

Dr. Christopher Corsico, chief medical officer for Boehringer, commented: "Advancing BI 1467335 into Phase II clinical research is important news for patients with NASH.

"Boehringer Ingelheim is committed to developing novel therapeutics designed to address unmet medical need and improve public health.

"Boehringer Ingelheim looks forward to further studying this novel compound in NASH patients."

In 2016 Boehringer Ingelheim obtained Fast Track Designation from the US Food and Drug Administration for the development of BI 1467335 in NASH.

Fast track is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions which fill an unmet medical need.

The designation provides opportunities for Boehringer Ingelheim to accelerate the development of this investigational drug candidate in NASH.

This Phase IIa trial is a multi-centre, double-blind design in 150 patients with clinical evidence of NASH.

The primary objectives are to establish proof of clinical principle, investigate suitable dosing, and to evaluate the safety of BI 1467335.

Patients will be randomized to either one of four dosages of BI 1467335 or to placebo for a 12-week treatment period.

A subsequent Phase IIb study will seek to confirm and extend these findings.

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Fri, 25 Aug 2017 12:22:00 +1000 https://www.proactiveinvestors.com.au/companies/news/182963/pharmaxis-to-receive-cash-injection-as-boehringer-ingelheim-initiates-new-trials-182963.html
<![CDATA[News - Pharmaxis receives support for expanded reimbursement of its drug Bronchitol® ]]> https://www.proactiveinvestors.com.au/companies/news/182706/pharmaxis-receives-support-for-expanded-reimbursement-of-its-drug-bronchitol-182706.html Pharmaxis Ltd (ASX:PXS) has received a positive recommendation from the Pharmaceutical Benefits Advisory Committee (PBAC) in Australia for expanded reimbursement of its drug Bronchitol® for the treatment of cystic fibrosis (CF).

Bronchitol® has the active ingredient mannitol delivered as an inhalable dry powder, which restores airway surface liquid.

The PBAC, the government’s independent advisory body on medicine reimbursement, has cleared the way for Pharmaceutical Benefits Scheme (PBS) funding of Bronchitol in combination with reimbursed Pulmozyme® (another CF medication) after considering a submission from Pharmaxis at its most recent meeting.

Gary Phillips, chief executive officer for Pharmaxis, commented:

"The submission for broader access to Bronchitol was strongly supported by the cystic fibrosis community with patients, families, patient organisations and CF clinic teams taking part in the feedback process.

"We look forward to discussing the positive PBAC recommendation with the Australian government to enable the PBS listing that will allow appropriate patients taking Pulmozyme to add reimbursed Bronchitol to their treatment regime."

Nettie Burke, chief executive officer for Cystic Fibrosis Australia (CFA), added:

"Every individual with CF should have access to a variety of treatments which are tailored to their disease and situation.

"The Bronchitol delivery device is portable, and CFA strongly advocates for better, less burdensome treatments that empower people with CF to lead productive lives.

"We are pleased that the PBAC has made this recommendation and anticipate an early listing on the PBS."

A resubmission to the FDA is expected in 2018 seeking approval in the US.

Importance of Bronchitol®

Bronchitol® is an inhaled dry powder delivered via a small handheld inhaler.

It has been studied in more than 1000 patients with CF in clinical trials conducted around the world.

People with CF develop an abnormal amount of excessively thick and sticky mucus within the lungs, airways and the digestive system.

The mucus causes impairment of the digestive functions of the pancreas and traps bacteria in the lungs resulting in recurrent infections which lead to irreversible damage.

Lung failure is the major cause of death for someone with CF.

The recommendation by the PBAC follows successful registration and reimbursement outcomes in a number of other countries.

Financial strength of Pharmaxis

Pharmaxis is well-funded with $21.5 million in cash at the end of June 2017.

The Boehringer NASH phase 2 initiation milestone is expected in in Q3 2017 for €18 million, and adds to this the Boehringer 2nd indication phase 2 milestone for €10 million expected in H2 2017.

The Pharmaxis share register comprises 50% institutional investors.

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Mon, 21 Aug 2017 13:36:00 +1000 https://www.proactiveinvestors.com.au/companies/news/182706/pharmaxis-receives-support-for-expanded-reimbursement-of-its-drug-bronchitol-182706.html
<![CDATA[News - Pharmaxis enters new quarter positioned for growth ]]> https://www.proactiveinvestors.com.au/companies/news/181609/pharmaxis-enters-new-quarter-positioned-for-growth-181609.html Pharmaxis (ASX:PXS) has completed a busy June quarter, which involved three key milestones, recruitment for the phase 2 NASH trial, advancement of the LOXL2 development program and clinical trial results for Bronchitol®.

When the first patient in the phase 2 NASH trial is dosed it will trigger a milestone payment to Pharmaxis of circa A$26 million.

The advancement of the LOXL2 development program remained on track to progress into a phase 1 clinical study in the second half of 2017. 

At the end of June, Pharmaxis had $21.5 million cash at bank.

Gary Phillips, CEO, commented: “Our Drug Discovery team have done outstandingly well to generate five lead candidate small molecule drugs to four different targets in the last two years since we sold the first phase 1 asset to Boehringer.”

Boehringer Ingelheim phase 2 NASH trial

Boehringer acquired PXS-4728A in May 2015 to develop initially as a treatment for non-alcoholic steatohepatitis (NASH).

Under the terms of the agreement, Boehringer has total responsibility for the development program and is required to make milestone payments to Pharmaxis as the drug progresses towards approval as well as other sales related payments post approval. 

Boehringer has advised that a phase 2 NASH trial has commenced recruitment and dosing of the first patient will trigger a milestone payment to Pharmaxis of €18 million (~A$26 million).

Boehringer has now advised that a phase 2 trial in a second indication will commence in the second half of 2017, triggering the payment of a €10 million (~$14 million).

LOXL2 inhibitor program

Pharmaxis is working with its collaborator, UK biotechnology company Synairgen plc (LSE: SNG) to commence a phase 1 study in the second half of 2017.

The Pharmaxis drug discovery group has developed a number of selective inhibitors to the lysyl oxidase type 2 enzyme (LOXL2).

LOXL2 is a key enzyme in the formation of excessive collagen depositions and important in the liver disease NASH, cardiac fibrosis, kidney fibrosis, the fatal lung disease idiopathic pulmonary fibrosis (IPF) and also plays a role in some solid cancers.

Large pharma company interest

The role of LOXL2 in fibrotic diseases such as NASH and pulmonary fibrosis is of significant interest to many large pharma companies.

The annual US BIO conference in June 2017 provided an opportunity to share updates with them on Pharmaxis’s scientific progress and the timing of its partnering process which it plans to initiate in the second half of 2017 and conclude with a partnering deal in 2018. 

Other irons in the fire

Pharmaxis has other drugs in its development pipeline including:

- A LOX inhibitor program which has potential anti-fibrotic application in scarring;
- The SSAO/MPO program which is developing a dual inhibitor with potential anti-inflammatory application in respiratory and cardiovascular disease; and
- The SSAO/MAOB inhibitor program has potential anti-inflammatory application in a number of indications.

Bronchitol for cystic fibrosis

Bronchitol® is an inhaled dry powder for the treatment of cystic fibrosis and has been the subject of three large scale global clinical trials conducted by Pharmaxis.

The product is approved and marketed in Europe, Russia, Australia and several other countries.

The study reported in June paves the way for the company’s partner Chiesi to conclude the US application to market the product for adult cystic fibrosis patients.

This step also completes the change in Pharmaxis’s business model as it reduces exposure to late stage clinical development and commercialisation risk and generates increasing value from early stage clinical development and partnering.

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Fri, 28 Jul 2017 12:23:00 +1000 https://www.proactiveinvestors.com.au/companies/news/181609/pharmaxis-enters-new-quarter-positioned-for-growth-181609.html
<![CDATA[Media files - Pharmaxis Ltd's Gary Phillips reveals completed Phase 3 clinical trial of Bronchitol ]]> https://www.proactiveinvestors.com.au/companies/stocktube/7624/pharmaxis-ltd-s-gary-phillips-reveals-completed-phase-3-clinical-trial-of-bronchitol-7624.html Tue, 13 Jun 2017 10:00:00 +1000 https://www.proactiveinvestors.com.au/companies/stocktube/7624/pharmaxis-ltd-s-gary-phillips-reveals-completed-phase-3-clinical-trial-of-bronchitol-7624.html <![CDATA[News - Pharmaxis appoints highly-experienced Dr Kathleen Metters to the board ]]> https://www.proactiveinvestors.com.au/companies/news/178932/pharmaxis-appoints-highly-experienced-dr-kathleen-metters-to-the-board-178932.html Pharmaxis (ASX:PXS) has appointed Dr Kathleen Metters as a non-executive director on the company, bringing more than 25 years of experience in the discovery and development of novel therapies for the treatment of serious diseases.

Pharmaxis is focused on inflammation and fibrosis with a portfolio of products at various stages of development and approval.

Dr Metters spent nine years as a senior executive with Merck & Co., which included a 4 year period as senior vice president and head of Worldwide Basic Research.

Malcolm McComas, chairman, commented:

"The appointment of Dr Metters to the Pharmaxis board follows a global search for a candidate who could contribute to the Pharmaxis business model of creating value through excellence in drug discovery and development.

"Her distinguished career in drug discovery inside a major multinational Pharma company and more recent role as a CEO of a smaller biotech company gives Dr Metters the experience and insight to make a significant contribution to Pharmaxis.

"We are delighted to welcome her to the board at a time when we are making significant steps forward with our pipeline of anti-inflammatory and anti-fibrotic therapies and progressing our aim to building a globally competitive company."

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Thu, 08 Jun 2017 09:00:00 +1000 https://www.proactiveinvestors.com.au/companies/news/178932/pharmaxis-appoints-highly-experienced-dr-kathleen-metters-to-the-board-178932.html
<![CDATA[News - Pharmaxis shares close higher on new cash payment ]]> https://www.proactiveinvestors.com.au/companies/news/177821/pharmaxis-shares-close-higher-on-new-cash-payment-177821.html Pharmaxis (ASX:PXS) shares performed well yesterday closing up 11.5% to $0.29, a share price high for May.

Strong trading was driven by the company’s drug development partner, Boehringer Ingelheim, confirming that a phase 2 study for a second disease indication from drug PXS-4728A will commence this year.

The second indication study will trigger an additional €10 million payment to Pharmaxis, which is in addition to the already expected €18 million payment for the scheduled mid-year phase 2 trial.

Boehringer acquired PXS‐4728A from Pharmaxis in May 2015 with initial clinical development focused on finding a treatment for the liver disease known as NASH.

This involved an upfront payment of €27.5 million and a total potential deal value of over A$750 million.

Boehringer is responsible for all development, regulatory, manufacturing and commercialisation activities.

Pharmaxis entered the current June quarter with $26.5 million in cash and these two expected payments are looking to add another A$42 million in 2017.

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Tue, 16 May 2017 09:00:00 +1000 https://www.proactiveinvestors.com.au/companies/news/177821/pharmaxis-shares-close-higher-on-new-cash-payment-177821.html
<![CDATA[News - Pharmaxis is now expecting a further cash injection ]]> https://www.proactiveinvestors.com.au/companies/news/177765/pharmaxis-is-now-expecting-a-further-cash-injection-177765.html Pharmaxis (ASX:PXS) is now expecting a new €10 million payment during 2017 from its drug development partner, Boehringer Ingelheim.

The payment is expected due to Boehringer confirming that a phase 2 study for a second disease indication from drug PXS-4728A will commence this year.

The second indication is in addition to the previously scheduled mid-year phase 2 trial, which triggers an €18 million payment.

Gary Phillips, CEO, commented: “We are delighted to see Boehringer looking to exploit that potential and commence clinical development in a fresh indication in the second half of this year.

“The structure of the deal with Boehringer anticipated its potential in more than one disease and the €10 million we expect for the second indication would bring total expected milestones received for starting phase 2 trials in two diseases to approximately A$42 million in this calendar year.”


Background

Pharmaxis is a pharmaceutical research company with a portfolio including two respiratory products approved in various world markets and a research pipeline focused on areas of high unmet clinical need in inflammatory and fibrotic diseases.

The company’s product pipeline is founded on its expertise in the chemistry of amine oxidase inhibitors.


Boehringer Ingelheim: PXS-4728A development

Boehringer acquired PXS‐4728A from Pharmaxis in May 2015 with initial clinical development focused on finding a treatment for the liver disease known as NASH.

This involved an upfront payment of €27.5 million and a total potential deal value of over A$750 million.

In addition to a phase 2 trial in NASH that is scheduled to start mid‐year, Boehringer has confirmed that a phase 2 study for a second disease indication will commence this year. 

Pharmaxis is due to receive milestone payments of €18 million for the start of a phase 2 trial in NASH and €10 million for the second indication.

Boehringer is responsible for all development, regulatory, manufacturing and commercialisation activities.


Analysis

Pharmaxis entered the current June quarter with $26.5 million in cash and these two expected payments are looking to add another A$42 million in 2017.

This significant injection of cash will allow Pharmaxis to further strengthen its drug development pipeline in fibrosis and inflammation and add further scientific expertise. 

Pharmaxis continues to show that it can commercialise early stage research into assets with world class data sets that are highly valued by big pharma companies seeking partnerships.

This means that a substantial amount of the company’s equity value is backed by cash, which places little value on the multiple opportunities in the drug pipeline.

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Mon, 15 May 2017 12:30:00 +1000 https://www.proactiveinvestors.com.au/companies/news/177765/pharmaxis-is-now-expecting-a-further-cash-injection-177765.html
<![CDATA[News - Pharmaxis 'extremely pleased' following supply agreement with Chiesi Farmaceutici SpA ]]> https://www.proactiveinvestors.com.au/companies/news/177467/pharmaxis-extremely-pleased-following-supply-agreement-with-chiesi-farmaceutici-spa-177467.html Pharmaxis (ASX:PXS) has revealed it has entered into an exclusive distribution and supply agreement with global pharmaceutical company Chiesi Farmaceutici SpA.

The agreement is for the commercialisation of Bronchitol® (mannitol) for cystic fibrosis in adults aged 18 years and above in Chiesi’s home market of Italy.

Under the terms of the agreement Chiesi will take over responsibility for the marketing, sales and distribution of Bronchitol with immediate effect.

Italy has approximately 5,000 cystic fibrosis patients and is one of the top 5 EU markets by value.

This new territory has been added as an extension to the agreement signed with Chiesi in 2015 for distribution rights in Germany, Ireland and the United Kingdom.

Pharmaxis continues to manufacture Bronchitol on commercial terms for Chiesi and the two parties are in the final stages of extending the exclusive supply agreement to 2024 for these markets and the United States.

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Tue, 09 May 2017 09:00:00 +1000 https://www.proactiveinvestors.com.au/companies/news/177467/pharmaxis-extremely-pleased-following-supply-agreement-with-chiesi-farmaceutici-spa-177467.html
<![CDATA[News - Pharmaxis approaching three key potential share price drivers ]]> https://www.proactiveinvestors.com.au/companies/news/176557/pharmaxis-approaching-three-key-potential-share-price-drivers-176557.html Pharmaxis (ASX:PXS) has entered the June quarter closing in on three significant and valuable milestones that can potentially drive the company’s share price.

The first is the commencement of a phase 2 NASH trial by Boehringer Ingelheim, triggering the payment of an €18 million or circa A$25 million.

The second is completion of preclinical toxicology studies for the LOXL2 program which is the last step before commencing human phase 1 clinical studies.

Third will be  the top line results from an international clinical trial of Bronchitol® (CF303)  designed to meet the remaining clinical requirements of the Food and Drug Administration (FDA) in order to market the drug in the USA.

Pharmaxis ended the March quarter with $26.5 million cash and during the quarter, two major institutional shareholders increased their holdings to become the company’s largest holders.


Background

Pharmaxis is a pharmaceutical research company with a portfolio including two respiratory products approved in various world markets and a research pipeline focused on areas of high unmet clinical need in inflammatory and fibrotic diseases.

The company’s product pipeline is founded on its expertise in the chemistry of amine oxidase inhibitors.


Catalyst #1: Phase 2 NASH trial

Pharmaxis’ partner Boehringer Ingelheim is on track to commence a phase 2 clinical trial of the drug PXS‐4728A for the liver disease NASH in the second quarter of 2017.

The commencement of the trial will trigger a milestone payment to Pharmaxis of €18 million (~A$25 million).


Catalyst #2: LOXL2 preclinical toxicology studies

Preclinical toxicology studies for the LOXL2 program which are being conducted with U.K. collaborator Synairgen are approaching completion.

This is the last step before commencing human phase 1 clinical studies in the second half of 2017 and move towards a formal partnering process. 


Catalyst #3: Top line results from Bronchitol trial

A third international multicentre clinical trial of cystic fibrosis patients designed to meet the remaining requirements of the US Food and Drug Administration (FDA) was completed in February and is scheduled to report top line results in the current quarter.

Bronchitol is a precision spray‐dried form of mannitol, delivered to the lungs by a specially designed, portable inhaler.

Pharmaxis has partnered its work on Bronchitol for the U.S. with global pharmaceutical company Chiesi Farmaceutici SpA, who has funded US$22 million of the expected US$26 million trial cost.

The U.S. is the largest market for Bronchitol and the pending trial results will potentially make it available in clinics across the U.S.

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Wed, 19 Apr 2017 11:00:00 +1000 https://www.proactiveinvestors.com.au/companies/news/176557/pharmaxis-approaching-three-key-potential-share-price-drivers-176557.html
<![CDATA[News - Pharmaxis’ substantial shareholders increase stakes ]]> https://www.proactiveinvestors.com.au/companies/news/175892/pharmaxis-substantial-shareholders-increase-stakes-175892.html Pharmaxis (ASX:PXS) has witnessed two of its substantial shareholders increase their stakes in the company recently.

Yesterday, National Nominees Ltd ACF Australian Ethical Investment Limited, increased its holding to 10.16% from 8.91%.

More recently, today, BVF Partners LP increased its stake to 18.94% from 12.99% through a number of on-market purchases.

Pharmaxis recently completed the treatment phase in its international clinical trial evaluating Bronchitol® for treatment of cystic fibrosis, a lung disease.

The Phase 3 trial recruited 423 patients across 126 sites in 21 countries in North and South America, Western and Eastern Europe.

The topline results of the trial are expected to be reported in the second quarter of this year.

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Wed, 05 Apr 2017 08:00:00 +1000 https://www.proactiveinvestors.com.au/companies/news/175892/pharmaxis-substantial-shareholders-increase-stakes-175892.html
<![CDATA[News - Pharmaxis completes treatment phase in clinical trial for U.S. market ]]> https://www.proactiveinvestors.com.au/companies/news/173578/pharmaxis-completes-treatment-phase-in-clinical-trial-for-us-market-173578.html Pharmaxis (ASX:PXS) has completed the treatment phase in its international clinical trial evaluating Bronchitol® for treatment of cystic fibrosis, a lung disease.

The Phase 3 trial recruited 423 patients across 126 sites in 21 countries in North and South America, Western and Eastern Europe.

The topline results of the trial are expected to be reported in the second quarter of this year.

The last of 423 has concluded treatment as part of the 26 week investigation of Bronchitol administered twice daily in cystic fibrosis patients aged 18 and over to assess improvements in lung function, pulmonary exacerbations and safety.

Pharmaxis has partnered its work on Bronchitol for the U.S. with global pharmaceutical company Chiesi Farmaceutici SpA, who has funded US$22 million of the expected US$26 million trial cost.

Gary Phillips, CEO, commented: “Having the last of more than 400 participants at 126 sites in 21 countries complete their last clinic visit is a very significant milestone for the Bronchitol business unit.”


Trial details

The Phase 3 trial known as DPM‐CF‐303 has been conducted in accordance with the requirements of the U.S Food and Drug Administration (FDA) to gain marketing approval for Bronchitol in the U.S.

Subject to a positive trial outcome, a response will be submitted to the FDA and a decision on approval can be expected in the second half of 2018.

The trial is a 26 week randomised, double‐blind parallel group investigation of Bronchitol administered twice daily in cystic fibrosis patients.


Bronchitol

Bronchitol is a precision spray‐dried form of mannitol, delivered to the lungs by a specially designed, portable inhaler.

The product is approved for marketing for the treatment of cystic fibrosis patients aged over six years in Australia and Russia and for patients aged 18 years and over throughout the European Union and in Israel.


Chiesi partnership

Chiesi has funded US$22 million of the expected total US$26 million cost of the trial and is also responsible for completing the Bronchitol New Drug Application with the FDA.

Subject to approval in the U.S, Bronchitol will be sold as part of Chiesi’s cystic fibrosis portfolio. 

Milestones totalling up to US$25 million are payable including US$10 million on the launch of Bronchitol. 

Pharmaxis will manufacture Bronchitol in Australia on commercial terms for Chiesi with Pharmaxis retaining a high teens percentage share of sales revenue as its margin.


Analysis

The completion of the treatment phase is another step forward to realising the commercial value of Bronchitol.

The U.S. is the largest market for cystic fibrosis and the pending trial results will potentially make Bronchitol available in clinics across the U.S.

Pharmaxis entered 2017 well-funded with a cash balance of A$29 million.

The company continues to build into a biotech powerhouse focused on fibrosis and inflammation with a number of opportunities and milestones expected in the current calendar year.

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Thu, 23 Feb 2017 10:00:00 +1100 https://www.proactiveinvestors.com.au/companies/news/173578/pharmaxis-completes-treatment-phase-in-clinical-trial-for-us-market-173578.html
<![CDATA[News - Pharmaxis enters 2017 cashed up as shares strengthen ]]> https://www.proactiveinvestors.com.au/companies/news/173287/pharmaxis-enters-2017-cashed-up-as-shares-strengthen-173287.html Pharmaxis (ASX:PXS) has made a solid start to 2017 with the company’s shares trading up 17% over the past three months to $0.305 and a cash balance of A$29 million.

The company continues to build into a biotech powerhouse focused on fibrosis and inflammation with a number of opportunities and milestones expected in the current calendar year.

It has a proven method of progressing from drug discovery to commercialisation supported by its first deal done with Boehringer Ingelheim in 2015 with the acquisition of the Pharmaxis phase 1 investigational drug PXS-4728A.

This involved an upfront payment of €27.5 million and a total potential deal value of over A$750 million.

PXS-4728A is being developed for the treatment of the cardiometabolic liver-related condition NASH.


Upcoming payment from Boehringer

Pharmaxis’s partner Boehringer Ingelheim is on track to commence a phase 2 clinical trial of the drug PXS‐4728A for the liver disease NASH in the second quarter of 2017.

The commencement of the trial will trigger a milestone payment to Pharmaxis of €18 million (~A$25 million).


Other deal opportunities

Pharmaxis is becoming a more enticing target for drug acquisition and partnerships as it continues to develop its fibrosis and inflammation drugs.

The company is working to build on the previous commercialisation successes of its in-house Drug Discovery Team.

Pharmaxis is targeting LOXL2 to reduce liver fibrosis and the liver disease NASH and has received strong interest from large pharmaceutical companies.

Mergers and acquisitions are increasing in the fibrosis and NASH space and there is evidence of structural pressure on big pharma to drive this M&A trend further.

Pharmaxis intends to partner after phase 1 trials and progress to date has been met with great interest by representatives of the large pharmaceutical companies scouting for opportunities.


Background

Pharmaxis is a pharmaceutical research company with a portfolio including two respiratory products approved in various world markets and a research pipeline focused on areas of high unmet clinical need in inflammatory and fibrotic diseases.

The company’s product pipeline is founded on its expertise in the chemistry of amine oxidase inhibitors.


A LOXL2 deal the next catalyst?

Despite a large number of drugs under development to treat the metabolic and inflammatory drivers of fibrosis, Pharmaxis is one of only a few companies developing drugs that directly target fibrosis itself.

Its program should therefore attract significant interest from big pharmaceutical companies who are looking for assets to help build and differentiate their franchises in this large, competitive and valuable market where there is still a high level of unmet need.

Deal values for phase 1 assets in fibrosis remain high with Gilead Sciences, Inc. (NASDAQ:GILD), Allergan and Bristol-Myers Squibb Co (NYSE:BMY) all acquiring anti fibrotic drug programs in the last 6 months with upfront payments for these deals all over US$100 million and total deal values often in excess of US$1 billion.

Mergers and acquisitions are increasing in the fibrosis and NASH space and there is evidence of structural pressure on big pharma to drive this M&A trend further with the number of increasing over the last five years.

With an undiluted market cap of $97.3 million backed by a healthy cash balance and plenty of growth opportunities in 2017, Pharmaxis offers a compelling investment.

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Fri, 17 Feb 2017 08:00:00 +1100 https://www.proactiveinvestors.com.au/companies/news/173287/pharmaxis-enters-2017-cashed-up-as-shares-strengthen-173287.html