Overview

• Study 1 met its primary endpoint with a 4.3-point improvement on the mADL11 scale at Week 8 (p<0.0001), with benefits observed from Week 2 and all key secondary endpoints achieving significance.
• In the randomized-withdrawal Study 2, 55% on ulixacaltamide maintained response versus 33% on placebo (p=0.0369), and the rate of disease improvement reached statistical significance.
• Ulixacaltamide was generally well tolerated with mostly mild to moderate adverse events, including constipation, dizziness, euphoria, brain fog, headache, insomnia and tingling; no deaths or drug-related serious adverse events were reported.
• Praxis has requested a pre-NDA meeting with the FDA and intends to file an NDA by early 2026, with fuller datasets to be shared at medical meetings and in publications.
• Shares more than tripled on the news to roughly $180, as Jefferies raised estimated approval odds to about 70% and projected potential sales above $2.5 billion for what could become only the second ET-specific therapy.