Medgenics is a clinical-stage biopharmaceutical company developing an innovative and proprietary platform technology - a biological "Biopump" - which allows patients to produce, within their bodies and on a long-term basis, their own natural human protein therapy for the treatment of a range of chronic diseases, such as anemia and hepatitis C.
“Our technology has promise to provide a major change in medicine. It is not just a product that we have, it’s an industry,” says Pearlman.
Its technology manipulates patients’ own tissue to produce proteins to fight diseases, eliminating the need for hundreds of vaccines.
The company is at an inflection point. Having completed its first clinical trials in anaemia in Israel, Medgenics now has US FDA approval to start its first clinical trials in US patients to treat anaemia in patients undergoing kidney dialysis.
It also has FDA designation of its INFRADURE treatment for Hepatitis D virus as an Orphan Disease and further approval for trials in Israel to treat Hepatitis C being just around the corner.
Additionally, the appointment earlier this week of Dr. Sol Barer as chairman was viewed by investors and industry commentators as a real accolade for the company.
Pearlman adds: “Coming on the heels of recent announcements, we see the appointment of Dr. Barer as a major transformative event at the company.”
As chief executive at Celgene, Dr Barer was a major force in the transformation of the business, which grew from an outfit employing 30 researchers, to a business that’s now worth almost US$30 billion.
The addition to the board of an industry heavyweight of this calibre is a huge endorsement of Medgenics and its technology.
Pearlman adds: “Sol is a highly successful life science innovator and businessman, he knows key people and has extensive deal-making experience.
“This is a potent combination that will be invaluable to ensuring Medgenics’ technology will receive early recognition and appreciation throughout the life science sector.”
Medgenics has more than a hundred patents and patent applications covering its key technology for the removal of tissue, the reinsertion and the processes in between.
The first step in the Biopump treatment is taking a sliver of dermis, the layer sitting directly below the skin, from the patient.
This straightforward process uses their proprietary DermaVac device which extracts a biopsy of dermis tissue, about half the size of a toothpick, with a single puncture of the skin, done under local anaesthetic.
The tissue is maintained in a sealed, disposable processing chamber while outside the body, where a specific gene is inserted into the cells of the Biopump, which produce the desired therapeutic protein needed to treat the illness.
The Biopump is then tested for 10 days to ensure it is stable and producing desired levels of therapeutic protein.
This process is all currently done by hand at the company research facility in Israel but Pearlman says it could all become automated for mass manufacturing.
Medgenics uses similar technology to reinsert the tissue back into the body, one millimetre under the skin.
The number of Biopumps administered depends on the amount of protein each makes and how much the body needs to treat the illness.
Currently Medgenics is focused on anaemia, hepatitis and haemophilia.
However, the huge potential of the Biopump is that the same core technology and techniques may be used in treatments for diabetes, obesity, cancer and many other major diseases.
The company turned a corner when it inaugurated its first Biopump process facility in California for its EPODURE product a few weeks ago.
EPODURE is targeting anaemia associated with kidney failure in dialysis patients.
The new processing facility is a crucial part of the company entering its Phase IIb trials for EPODURE, which plans to see its first testing in US patients in Q4 2012, and first data within six months of launch.
The facility is also important in proving that the Biopump technology can be implemented in standard cell processing facilities and can be transported without losing its function.
Medgenics is also awaiting final approval for Phase I/II studies in Israel for its INFRADURE Biopump to treat Hepatitis C and which could also be used to treat Hepatitis B.
Only last month the company confirmed it had received orphan drug designation (ODD) from the US FDA for its INFRADURE Biopump to treat a rare form of liver disease, Hepatitis D – a crucial landmark which saw the share price jump.
ODD carries multiple benefits including the availability of grant money and seven years of market exclusivity, as well as potential for a fast track regulatory process.
The firm expects it will also have key initial patient data by the end of the year for its Hepatitis C. The INFRADURE Biopumps used for Hepatitis C are essentially the same as those for the other forms of hepatitis as well, so progress on one can help the others.
The next big step for the firm would be a partner or licence agreement, which Pearlman says it is in an increasingly strong position to achieve.
Pearlman adds: “We are the goose that lays the golden egg. We can sell an egg or we can sell a gosling that would be specific for one indication.”
The flexibility of the business model means deals can be adapted to suit the company, the market and the geographical model.
“All the pieces of the puzzle and incredible vision are coming together, we have a great working relationship with the regulators, strong support from our investors, our clinical programmes are consistently producing strong data that the research scientists are really excited about, and we are now joined by Dr. Sol Barer, a major player who has all the right contacts to introduce us to other big players in the field and help drive us towards what we see as a big future.”
The share price has undergone a steady climb since January and is now almost four times its level at the start of the year at 631 pence per share.
“We believe the share price is reflecting a steady delivery of promised milestones and real accomplishments. We said what we were going to do, we have delivered and exceeded.”
The company is funded well into the first quarter of the next financial year, by which time Pearlman expects the company to have achieved several key milestones that will have attracted more investors.
“People are starting to hear the story and see it has real feet. It is not just a wild dream. It’s happening and I think they want to get in on it.”