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PRAX (Top Pick): Elsunersen Wins FDA Breakthrough Designation in SCN2A-DEE — An Underappreciated Asset Overlooked By the Street
研报英文原文证据摘录
PRAX (Top Pick): Elsunersen Wins FDA Breakthrough Designation in SCN2A-DEE — An Underappreciated Asset Overlooked By the Street
FLASH NOTE
June 22, 2026
Yatin Suneja yatin.suneja@guggenheimpartners.com PRAX (Top Pick): Elsunersen Wins FDA
212 518 9565 Breakthrough Designation in SCN2A-DEE — An
Eddie Hickman, Ph.D.
eddie.hickman@guggenheimpartners.com Underappreciated Asset Overlooked By the Street
212 518 9904
Delma Caiati, M.D., Ph.D. Key Message: PRAX received FDA Breakthrough Therapy Designation (BTD) for
delma.caiati@guggenheimpartners.com elsunersen, its ASO for seizures associated with SCN2A-DEE caused by gain-of-function 617 859 4606
variants. This is the third BTD across the company's late-stage pipeline, which we view
Min Lee, Ph.D. as a meaningful Solidus platform validation event. The designation was supported by
min.lee@guggenheimpartners.com the previously reported EMBRAVE Part A data, which showed a 77% sham-adjusted
212 823 6744 reduction in monthly seizures (p=0.015), with 71% of treated patients achieving >50%
Iris Gao, Ph.D. seizure reduction, 57% achieving at least a 28-day seizure-free period, and benefits
iris.gao@guggenheimpartners.com sustained in the open-label extension for up to one year. Elsunersen now carries BTD,
212 518 9013 Orphan Drug, and Rare Pediatric Disease designations from the FDA, and Orphan
Drug and PRIME designations from the EMA; the Rare Pediatric Disease designation
preserves potential eligibility for a PRV at approval, which carries standalone monetizable
PRAX valuethe FDA(~$200MM).disclosed Thein Decemberpivotal EMBRAVE32025, whichstudyshouldis enrollingacceleratefollowingthe pathalignmentto filing;withwe
Praxis Precision Medicines, Inc. anticipate data in 2027. Assuming ASO pricing applied to the addressable SCN2A-
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