- Kazia Therapeutics Limited (NASDAQ: KZIA), an oncology-focused biotechnology company developing therapies that selectively reprogram cancer biology, restore anti-tumor immunity and overcome treatment resistance, announced that Sovargen Co., Ltd., a biotechnology company specializing in central nervous system (CNS) diseases that has exclusively licensed paxalisib from Kazia for the development and commercialization of paxalisib as a potential treatment for mTORopathy-related epilepsies, 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). The dosing triggers a $2 million development milestone payment to Kazia under the companies' exclusive licensing agreement.

Kazia and Sovargen entered into the agreement in March 2024, under which Sovargen is responsible for developing, manufacturing and commercializing paxalisib as a potential treatment for the mTORopathy-related epilepsies in all countries worldwide, excluding mainland China, Hong Kong, Macao and Taiwan. Under the agreement, Kazia is eligible to receive additional milestone payments of up to $17 million upon the achievement of further development and regulatory milestones, plus a percentage of sub-licensing revenues and royalties on net sales of any products incorporating paxalisib.

FCD T2 and TSC are rare, genetically driven neurological disorders in which somatic mutations in the PI3K/Akt/mTOR pathway, or mutations in the TSC1 or TSC2 genes, drive overactivation of the mTOR pathway and contribute to treatment-resistant seizures. There are currently no approved therapies for FCD T2. Paxalisib is an oral, brain-penetrant dual inhibitor of PI3K and mTOR.

"This milestone reflects the steady progress Sovargen is making in advancing paxalisib for patients with FCD T2 and TSC, two rare disorders with significant unmet need," said Dr. John Friend II, CEO of Kazia Therapeutics. "While our own clinical focus for paxalisib remains in oncology, we're pleased to see its potential being explored in additional indications where its mechanism of action may address significant unmet medical needs. We look forward to the data generated through this study as the program advances."

"Families living with FCD T2 and TSC have long faced limited treatment options, making the advancement of new therapies crucial," said Cheolwon Park, CEO of Sovargen. "Dosing the first patient is a significant step for our program and reflects the dedication of our team and investigators in bringing this study to life. As enrollment continues, we remain focused on executing a rigorous clinical trial and generating the data needed to better understand paxalisib's potential as a treatment for these rare neurological disorders."