Common Stock, $0.001 par value
- Symbol
- RARE
- Exchange
- NASDAQ
- Classification
- COMMON
- Status
- Current
Current Report · Items 8.01 · 8-K
Ultragenyx Pharmaceutical Inc.
RARENASDAQEQUITYCurrent
Other Events
Item 8.01 Other Events. On September 2, 2026, Ultragenyx Pharmaceutical Inc. (“Ultragenyx” or the “Company”) announced results from the Phase 3 Aspire study for apazunersen (GTX-102) in Angelman syndrome. The study did not achieve the primary endpoint of change from Baseline in Bayley-4 cognitive raw score nor the key secondary endpoint of net response in Multidomain Responder Index (“MDRI”).…
Company context
Ultragenyx is a biopharmaceutical company committed to bringing novel therapies to patients for the treatment of serious rare and ultra-rare genetic diseases. The company has built a diverse portfolio of approved medicines and treatment candidates aimed at addressing diseases with high unmet medical need and clear biology, for which there are typically no approved therapies treating the underlying disease.
Current securities
Registered securities in this filing
Ultragenyx Pharmaceutical Inc. · 8-K · Filed 2026-09-02
Disclosure sections
Item 8.01Item 8.01 - Other Events
Item 8.01 Other Events.
On September 2, 2026, Ultragenyx Pharmaceutical Inc. (“Ultragenyx” or the “Company”) announced results from the Phase 3 Aspire study for apazunersen (GTX-102) in Angelman syndrome. The study did not achieve the primary endpoint of change from Baseline in Bayley-4 cognitive raw score nor the key secondary endpoint of net response in Multidomain Responder Index (“MDRI”). The safety profile observed in Aspire was consistent with Phase 1/2.
In Aspire, the randomized groups were comparable at baseline and consistent with the patients studied in Phase 2. There were no differences between the treated and control groups that could support efficacy in the Bayley Cognition raw scores nor in the MDRI when looking at net response or mean changes of the individual five endpoints included in the MDRI.
The Company will evaluate the apazunersen program in light of this outcome and make a decision on its disposition. The Company will also assess its planned operations to define and implement significant expense reductions, while supporting its growing commercial business.