Sovargen doses first patient in paxalisib epilepsy trial, triggers $2M payment to Kazia

Kazia Therapeutics announced on September 22, 2026 that its licensing partner Sovargen Co., Ltd. has dosed the first patient in a Phase 1b/2a clinical trial evaluating paxalisib for the treatment of intractable epilepsy associated with focal cortical dysplasia type 2 (FCD T2) and tuberous sclerosis complex (TSC).¹

This dosing event triggers a $2 million development milestone payment to Kazia under the companies' exclusive licensing agreement.¹ That agreement, signed in March 2024, gives Sovargen rights to develop, manufacture, and commercialize paxalisib for mTORopathy-related epilepsies worldwide, though the license excludes mainland China, Hong Kong, Macao, and Taiwan. Beyond the current payment, Kazia stands to receive up to $17 million more in milestone payments tied to future development and regulatory progress, along with a share of sub-licensing revenue and royalties on any resulting product sales.¹

The two conditions under study, FCD T2 and TSC, are rare genetic disorders. According to the release, both involve somatic mutations in the PI3K/Akt/mTOR pathway or mutations in the TSC1 or TSC2 genes, which lead to mTOR pathway overactivation and seizures that resist standard treatment.¹ The release notes that no approved therapies currently exist for FCD T2.¹

Paxalisib itself is described as an oral, brain-penetrant dual inhibitor of PI3K and mTOR.¹ Kazia's CEO, John Friend, said the company's own clinical focus for the drug remains in oncology, while noting interest in its potential in other indications. Sovargen's CEO, Cheolwon Park, said the company remains focused on enrollment and generating data on paxalisib's potential in these disorders.

Kazia's release also recaps paxalisib's broader development history, including a completed Phase 2/3 glioblastoma study (GBM AGILE) reported in 2024, ongoing trials in other cancer types, and multiple prior FDA designations, including Orphan Drug and Fast Track status for glioblastoma and related indications.