The study targets focal cortical dysplasia type 2 and tuberous sclerosis complex, both rare conditions driven by PI3K/Akt/mTOR pathway mutations. Paxalisib, an oral brain-penetrant dual inhibitor, is being tested as a potential remedy for these treatment-resistant seizures, for which no approved therapies currently exist for FCD T2 patients.

Under the terms of their exclusive licensing deal, Kazia remains eligible for up to $17 million in additional regulatory and development milestones, alongside potential royalties. While Kazia continues to prioritize oncology, company leadership noted the importance of exploring the drug's mechanism in addressing significant unmet medical needs within the central nervous system.