Sovargen Doses First Patient in Phase 1b/2a Trial of Kazia's Paxalisib for FCD T2 and TSC Epilepsy
核心洞察
Sovargen Co. has dosed the first patient in a Phase 1b/2a trial of paxalisib for intractable epilepsy (搜索) associated with focal cortical dysplasia type 2 (搜索) and tuberous sclerosis complex (搜索).
The dosing triggers a $2 million development milestone payment to Kazia Therapeutics (搜索) under the March 2024 exclusive licensing agreement between the two companies.
Paxalisib is an oral, brain-penetrant dual inhibitor of PI3K (搜索) and mTOR (搜索), a mechanism aimed at the mTOR pathway overactivation that drives these treatment-resistant seizures.
Sovargen Co., Ltd. has dosed the first patient in a Phase 1b/2a clinical trial evaluating paxalisib for the treatment of intractable epilepsy (搜索) associated with focal cortical dysplasia type 2 (搜索) (FCD T2) and tuberous sclerosis complex (搜索) (TSC), the company and licensing partner Kazia Therapeutics (搜索) Limited announced. The dosing triggers a $2 million development milestone payment to Kazia under the exclusive licensing agreement between the two companies.
Sovargen, a biotechnology company specializing in central nervous system diseases, holds an exclusive license from Kazia to develop and commercialize paxalisib as a potential treatment for mTORopathy-related epilepsies. The agreement, signed in March 2024, covers all countries worldwide excluding mainland China, Hong Kong, Macao and Taiwan. Under its terms, Sovargen is responsible for development, manufacturing and commercialization, while Kazia is eligible for additional milestone payments of up to $17 million upon achievement of further development and regulatory milestones, plus a percentage of sub-licensing revenues and royalties on net sales of any products incorporating paxalisib.
Targeting mTOR Overactivation in Rare Epilepsies
FCD T2 and TSC are rare, genetically driven neurological disorders. In both, somatic mutations in the PI3K (搜索)/Akt/mTOR (搜索) pathway, or mutations in the TSC1 (搜索) or TSC2 (搜索) 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 (搜索) and mTOR (搜索). The trial is designed to evaluate the drug in patients whose seizures persist despite existing treatment options.
Development Beyond Oncology
Kazia's own clinical focus for paxalisib remains in oncology, where the company describes its programs as developing therapies that selectively reprogram cancer biology, restore anti-tumor immunity and overcome treatment resistance. The epilepsy (搜索) program represents an expansion of the compound into a non-oncology indication.
"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 (搜索). "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."
Sovargen CEO Cheolwon Park framed the first-patient dosing as a programmatic step for a population with few options. "Families living with FCD T2 and TSC have long faced limited treatment options, making the advancement of new therapies crucial," Park said. "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."
Enrollment in the Phase 1b/2a study is ongoing.