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Will GTX-102 Phase 3 study meet primary endpoint (Bayley-4 cognition) by December 31, 2025?
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Ultragenyx press releases and FDA announcements
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Ultragenyx ($RARE) Announces Successful End-of-Phase 2 Meeting with FDA for GTX-102 Angelman Syndrome
Jul 18, 2024, 01:49 AM
Ultragenyx Pharmaceutical Inc. ($RARE) has announced a successful end-of-Phase 2 meeting with the FDA for its GTX-102 Angelman Syndrome program. The company is set to initiate a Phase 3 study by the end of this year, with primary and secondary endpoints aligned with the FDA. The primary endpoint will focus on Bayley-4 cognition, while the secondary endpoint will be the Multi-Domain Responder Index (MDRI). Ultragenyx's GTX-102 gene therapy has shown early efficacy and targets a market of approximately 60,000 children suffering from Angelman Syndrome. The company projects worldwide revenue for GTX-102 to reach $475 million in FY27, $1,251 million in FY28, and $1,640 million in FY29, valuing the asset at approximately $43 per share. The pivotal study is expected to start in Q4 this year.
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