Biotech company Kazia Therapeutics (ASX:KZA) has announced positive results from its latest paxalisib study. Paxalisib is a drug whose lead indication is glioblastoma, the most common and most aggressive form of brain cancer, which affects about 250,000 new patients each year.
The prognosis for glioblastoma has not significantly improved in two decades. Following diagnosis, average life expectancy is around 15 months, and fewer than 5 per cent of these patients are alive after 5 years.
The study was conducted with 30 patients in the United States whose genetic profile suggested resistance to temozolomide, which is the only current FDA-approved drug for first-line glioblastoma treatment. Patients were given paxalisib as monotherapy.
60mg once-daily was identified as the maximum tolerated dose. Median overall survival was 15.7 months, compared with a historical 12.7 months for temozolomide; while median progression-free survival was 8.4 months, compared with a figure of 5.3 months associated with temozolomide. The safety profile of paxalisib was consistent with previous clinical studies: hyperglycaemia, oral mucositis, and skin rash were among the most common toxicities.
Kazia CEO, Dr James Garner, comments, “Our task now, as we move rapidly toward a potential marketing authorisation, is to confirm and quantify the benefit associated with paxalisib in glioblastoma patients. This indeed is the focus of our participation in GBM AGILE, which commenced recruiting to the paxalisib arm in January 2021. We are increasingly also exploring additional patient populations for which a brain penetrant PI3K/mTOR inhibitor may provide significant advantages over the standard of care.”
Shares in Kazia Therapeutics (ASX:KZA) are trading 2.9 per cent higher at $1.42.