On August 7, 2020 the FDA granted the drug paxalisib Rare Pediatric Disease Designation (RPDD) for an aggressive childhood brain cancer. To clarify, this granting of FTD for glioblastoma is not connected to the prior granting of RPDD in DIPG cancers.
- Kazia completed recruitment to its phase II clinical trial of paxalisib in newly diagnosed glioblastoma in February 2020.
- Interim clinical data was presented at the American Association of Cancer Research (AACR).
- Virtual Annual Meeting II in June 2020.
- Overall survival was calculated at 17.7 months compares to a historical figure of 12.7 months for temozolomide, which is the existing FDA-approved standard of care.
- Kazia expects to present further data from this study before the end of 2020 and to conclude the study in early 2021.
- Paxalisib has been selected to join the international GBM AGILE pivotal study in glioblastoma, recruitment is expected to begin this year.
About Kazia Therapeutics Ltd.
Kazia Therapeutics Ltd. is an innovative oncology-focused biotechnology company based in Sydney, Australia. The firm’s pipeline includes two clinical-stage drug development candidates. The company is working toward developing therapies for various cancers.
Kazia Therapeutics Pipeline
Paxalisib is the lead program. It is a small molecule inhibitor of the PI3K / AKT / mTOR pathway, which is being developed to treat glioblastoma. Licensed from Genentech in late 2016, paxalisib entered a phase II clinical trial in 2018. Interim data was reported most recently at AACR in June 2020, further data is expected in 2H 2020. Four additional studies are ongoing in other forms of brain cancer.
The US FDA granted paxalisib Orphan Drug Designation for glioblastoma in February 2018 and granted the drug Fast Track Designation for glioblastoma in August 2020. In addition, paxalisib was granted Rare Pediatric Disease Designation by the US FDA in August 2020.
TRX-E-002-1 (Cantrixil) is a third-generation benzopyran molecule with activity against cancer stem cells and is being developed to treat ovarian cancer. TRX-E-002-1 has completed a phase I clinical trial in Australia and the United States with the final data expected in the second half of the calendar 2020.
Interim data was presented most recently at the AACR conference in June 2020. Cantrixil was granted orphan designation for ovarian cancer by the US FDA in April 2015.
Glioblastoma is one of those cancers which have yet to find a treatment that can stop the fast killing of patients who were unluckily enough to be diagnosed with this brain tumor.
Do we bet on this firm or not?
It is difficult to answer this question as many promising mid-term clinical trial results happen to fail in the large late-phase trials. Among the failed firms is Celldex Therapeutics (CLDX), a company with strong scientific fundamentals whose failure of the late-phase trial to treat glioblastoma killed its stock. It took years for Celldex’s buried stock to resurrect following successful trial results of another product that treats a different cancer.
Kazia Therapeutics Ltd Stock
We are following up on this firm’s clinical trials.
To read yesterday’s article please click here.
Kazia Therapeutics Product Paxalisib Beats Temozolomide in Overall Survival in Mid-Phase Trial
The US FDA Awards Fast Track Designation to Paxalisib for Glioblastoma
Kazia Therapeutics Ltd (KZIA) – an Australian oncology biotechnology company, announced that the United States FDA has granted Fast Track Designation (FTD) to Kazia’s paxalisib for glioblastoma, the most aggressive primary brain cancer. The FTD has been granted for the treatment of newly diagnosed glioblastoma with unmethylated O6-Methylguaninemethyl transferase (MGMT) promotor status who have completed initial radiation with temozolomide.
The indication reflects the patient population studied in an ongoing phase II study and is the primary proposed population for the GBM AGILE pivotal study as well as being the intended indication at commercial launch.