He said: “The September quarter has been highly productive: we have launched a new clinical study and we have seen important recognition by FDA of the drug’s potential in several forms of brain cancer.
“These achievements leave us ideally positioned as we now transition into the GBM AGILE pivotal study in glioblastoma.
“The remainder of this year, and the year ahead, are likely to be rich in data read-outs and in operational progress, as we see paxalisib move rapidly towards commercialisation.”
FDA special designations
In August 2020, Kazia received Rare Pediatric Disease Designation (RPDD) from the US FDA for paxalisib in the treatment of diffuse intrinsic pontine glioma (DIPG).
The key benefit of it the program is that it allows for the sponsor company to receive a priority review voucher (PRV) at the time of an application for marketing authorization in the paediatric disease.
The PRV can also be sold to another company and used for any new drug, and such transactions have historically commanded prices between US$68-$350 million.
Also in August, the company received Fast Track Designation (FTD) from the US FDA for paxalisib in the treatment of glioblastoma, which is designed to expedite development of pharmaceutical products which demonstrate the potential to address unmet medical needs in serious or life-threatening conditions.
It provides Kazia with the ability to submit a ‘rolling NDA’, in which components of the new drug application can be filed as they become available, increasing efficiency and reducing risk in the approval process.
In addition, the company also received Orphan Drug Designation (ODD) from the US FDA for paxalisib in the treatment of glioma which provides access to certain grant funding, a waiver of PDUFA fees at the time of NDA submission (currently around US$3 million per indication), and a period of data exclusivity which augments the patent protection.
Phase II study in primary CNS Lymphoma
In September 2020, Kazia launched a new clinical collaboration with Dana Farber Cancer Institute where Dr Lakshmi Nayak will lead a phase II investigator-initiated clinical trial in primary CNS lymphoma.
Primary CNS lymphoma is a form of brain cancer that affects approximately 1,500 patients per annum in the United States and is considered a high-potential target for paxalisib, since three of the four FDA-approved PI3K inhibitors are used to treat forms of lymphoma outside the central nervous system.
Paxalisib is being developed to treat glioblastoma - the most common and most aggressive form of primary brain cancer in adults.
Successful capital raise
In October after the quarter ended, Kazia launched a one-for-three accelerated non-renounceable entitlement offer to raise around $25 million, before fees, which was fully underwritten by Bell Potter Securities Limited.
An accelerated institutional component closed on October 2, 2020, raising about $16.4 million from institutional investors, representing around a 70% take-up.
The retail component closed on October 20, 2020, raising a further $8.8 million, with around 32% take-up.
This financing leaves the company well-funded to execute the GBM AGILE pivotal study.
GBM AGILE study
On October 16, 2020, the company executed a definitive agreement with the Global Coalition for Adaptive Research (GCAR) to commence paxalisib’s participation in the GBM AGILE pivotal study in glioblastoma.
The international multi-drug platform study is designed to expedite the approval of new medicines for glioblastoma and runs independently of any individual company, under the leadership of some of the world’s premier experts in the field.
The study is already ongoing, with 30 sites in the United States and Canada participating, and the first drug is Bayer’s Stivarga® (regorafenib).
Paxalisib will be the second drug to enter the study and the first patient into the paxalisib arm is expected to occur in early quarter one of 2021.
Due to the capital raise, the company’s operations are well-funded for the foreseeable future.
The key milestones for the next two quarters are:
- Additional interim data from the ongoing phase II study of paxalisib in glioblastoma (November 2020 – SNO Annual Meeting);
- Initial interim data from the ongoing phase I study of paxalisib in DIPG at St Jude Children’s Research Hospital (November 2020 – SNO Meeting);
- Initial interim data from the ongoing phase II study in breast cancer brain metastases at Dana-Farber Cancer Institute (precise timing remains uncertain due to COVID related disruption of conference schedules);
- Top-line final data from the completed phase I study of Cantrixil in ovarian cancer; and
- First patient in (FPI) to GBM AGILE registration study for paxalisib in glioblastoma (planned for each Q1 2021 to avoid Christmas and New Year period).