paxalisib

Sept. 22, 2026: Kazia Therapeutics (NASDAQ: KZIA) said licensee Sovargen dosed the first patient in a Phase 1b/2a trial of paxalisib (SVG103) for mTORopathy-related epilepsies, triggering a $2 million development milestone payment to Kazia under their March 2024 licensing deal.

The study is evaluating paxalisib, an oral brain-penetrant dual PI3K/mTOR inhibitor, in adults with intractable epilepsy associated with focal cortical dysplasia type 2 (FCD T2), tuberous sclerosis complex (TSC), and, per the company background, hemimegalencephaly (HME). The announcement specifically highlights FCD T2 and TSC. No efficacy or safety data were reported; enrollment is ongoing and the companies said they expect the trial to generate data as it advances.

Under the licensing agreement, Sovargen has rights to develop, manufacture, and commercialize paxalisib for mTORopathy-related epilepsies worldwide excluding mainland China, Hong Kong, Macao, and Taiwan. Kazia said it remains eligible for up to $17 million in additional development and regulatory milestones, plus a share of sublicensing revenue and royalties on net sales.

Kazia also noted paxalisib continues in oncology development, with discussions ongoing on a potential registrational study in glioblastoma following a completed Phase 2/3 GBM AGILE study reported in 2024.