Kazia Secures $2M Milestone as Paxalisib Epilepsy Trial Begins

Kazia Therapeutics Limited, an oncology-focused biopharmaceutical company based in Sydney, Australia, has received a $2 million milestone payment as it launches a Phase 1b/2a clinical trial evaluating paxalisib for intractable epilepsy associated with focal cortical dysplasia type 2 (FCD T2) and tuberous sclerosis complex (TSC). These conditions are rare, genetically driven neurological disorders characterized by mTOR pathway overactivation, leading to treatment-resistant seizures. Paxalisib, an oral, brain-penetrant dual inhibitor of PI3K and mTOR, was originally licensed from Genentech in late 2016 and has been the subject of over 15 clinical trials. It holds orphan drug designation for glioblastoma and fast track designation for multiple indications, including epilepsy. Sovargen Co., Ltd., a licensing partner, obtained exclusive rights to develop and commercialize paxalisib for mTORopathy-related epilepsies outside mainland China, Hong Kong, Macao, and Taiwan. The agreement, established in March 2024, includes potential additional milestone payments of up to $17 million, as well as royalties on net sales and sub-licensing revenues. The trial marks a pivotal step in exploring paxalisib’s potential beyond oncology, addressing unmet medical needs in epilepsy.