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Phase 3Data readout

GTX-102

Angelman Syndrome

RAREUltragenyx Pharmaceutical Inc.
Expected catalystDecember 31, 2026Company guidance: Dec 31, 2026

Catalyst overview

Event details

Drug or asset
GTX-102
Development stage
Phase 3
Event type
Data readout
Indication
Angelman Syndrome
Research signalsHigh management interest

Research summary

What is being tracked?

This catalyst tracks a Phase 3 data readout for GTX-102 in Angelman Syndrome. The expected timing is based on the latest available company guidance and may change.

Development notes

ASPIRE, Aurora trials. 7/17/24- Alignment w/ FDA on Ph3 study endpoints. 12/19/24- First patient dosed in ASPIRE trial. 7/31/25- ASPIRE trial enrollment completed. 10/30/25- First patient dosed in Aurora study. Data from Aspire study in H2 2026. 11/04/25:- Reiterated ASPIRE Ph3 fully enrolled; Aspire Ph3 data expected H2 2026. 01/12/26:- Reiterated ASPIRE Ph3 fully enrolled; Aspire Ph3 pivotal data expected H2 2026; Aurora Ph2/3 ongoing. 05/05/26:- New long-term Ph1/2 data show durable multdomain improvement, stable safety; ASPIRE Ph3 data and Aurora enrollment expected H2 2026. 08/04/26:- Aspire Ph3 data expected September or October; Aurora enrollment completion expected H2 2026.

Verification

Primary source

Confirm timing and program details against the company announcement or filing.

Open source

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Company

Ultragenyx Pharmaceutical Inc.

RARE · NASDAQ
Industry
Biotechnology
Pipeline assets
9
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Updated Aug 23, 2026. Dates can change; this is not investment advice.

Disclaimer: Biotech Stock Intel is an independent research platform. Information is provided for informational purposes only and is not investment or medical advice. Always verify catalyst dates against primary sources and conduct your own research.
Biotech Stock Intel© 2026 Biotech Stock Intel
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