Exciting news this morning for biotech company Kazia Therapeutics Limited ($KZIA), after the American FDA has awarded Fast Track Designation (FTD) to paxalisib for glioblastoma. Glioblastoma is the most common and aggressive brain cancer treated by Kazia with paxalisib – previously called GDC-0084. With the granting of FTD, the development of the drug can be accelerated, in search of the medical need for a better treatment for this form of cancer.

To understand what the FDA approval stands for: “The treatment of patients with newly diagnosed glioblastoma with unmethylated O6-methylguanine methyl transferase (MGMT) promoter status who have completed initial irradiation with concomitant temozolomide.” This is not related to the earlier award of Rare Pediatric Disease Designation (RPDD) in Diffuse Intrinsic Pontine Glioma (DIPG) – an aggressive form of brain cancer in children that is currently also receiving a lot of attention in Kazia.

Paxalisib is gaining momentum

The recognition is very important to Kazia as it confirms the potential of the drug and will significantly accelerate the commercialization of paxalisib. In addition, this results in a close collaboration with the FDA, which means that face-to-face meetings and written consultation will take place during the further development of paxalisib. This makes the pharmaceutical product more likely to qualify for accelerated approval and priority review. With a new drug application (NDA), this can result in faster product approval. For paxalisib, this means that a rolling evaluation is possible, with the advantage that an NDA can already be requested during the process for completed parts and not only in the final phase. Kazia has announced that it will submit the first NDA applications in 2021 to speed up the entire paxalisib process.

Kazia’s CEO – Dr. James Garner – noted that the “rolling review” process allows the company to review substantial portions of the NDA filing before finalizing and submitting. This results in time savings and a decrease in risk of the product. The final phase of paxalisib development can now be anticipated in collaboration with the FDA.

More positive numbers ahead

Where paxalisib differs in numbers is the average survival period. Where temozolomide achieved a mark of 12.7 months – the existing FDA-approved standard of care – the survival time for paxalisib is calculated at 17.7 months. Kazia is expected to present more data in 2H CY2020 and to complete the study around CY2021. The prospect is that paxalisib has been selected to participate in the international GBM AGILE pivotal study in glioblastoma, and recruitment is expected to begin in 2H CY2020.

Due to this development, the stock has jumped 21% in the pre-market to robust volume in response to the FTD status. The more frequent interaction with the FDA will result in an ongoing review of the marketing application. Since Kazia published the preliminary results of the mid-term study in April 2020, the company appears to be in an upward spiral. More data and confirmations are expected further this year with final results in the first half of 2021.

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