PRAX · Praxis Precision Medicines, Inc.

Praxis Precision Medicines reported significant clinical and regulatory progress across its neuroscience pipeline. The EMBRAVE Part A trial for elsunersen in pediatric SCN2A DEE patients demonstrated robust efficacy, achieving a 77% placebo-adjusted seizure reduction with statistical significance (p=0.015). The safety profile appears favorable with no drug-related serious adverse events or neuroinflammation signals, which is notable for an antisense oligonucleotide in this population. The durability of effect observed in the open-label extension up to one year and the broad functional improvements in 100% of treated patients suggest potential for a differentiated profile in this severe orphan indication. Separately, the FDA acceptance of the relutrigine NDA for priority review marks a critical regulatory milestone. The target action date of September 27, 2026, under PDUFA provides a clear near-term catalyst. The priority review designation indicates the FDA recognizes the potential for relutrigine to address an unmet medical need in SCN2A and SCN8A DEEs. These dual positive developments substantially de-risk the near-term outlook for the company.