--- type: "Topics" locale: "en" url: "https://longbridge.com/en/topics/100000000883533.md" description: "๐Ÿ“ข ๐—๐—จ๐—ฆ๐—ง ๐—œ๐—ก: $Ultragenyx Pharma(RARE.US) Ultragenyx Phase 3 Aspire Study of Apazunersen Fails Primary and Key Secondary Endpoints๐Ÿ‘‰ ๐—ž๐—ฒ๐˜† ๐—›๐—ถ๐—ด๐—ต๐—น๐—ถ๐—ด๐—ต๐˜๐˜€:โžค ๐—จ๐—น๐˜๐—ฟ๐—ฎ๐—ด๐—ฒ๐—ป๐˜†๐˜…'s Phase 3 ๐—”๐˜€๐—ฝ๐—ถ๐—ฟ๐—ฒ study failed its primary endpoint.โžค Apazunersen showed no efficacy difference in ๐—•๐—ฎ๐˜†๐—น๐—ฒ๐˜†-๐Ÿฐ cognitive raw scores.โžค Study also missed the key secondary ๐— ๐——๐—ฅ๐—œ endpoint.โžค No efficacy support emerged across the ๐—ณ๐—ถ๐˜ƒ๐—ฒ individual MDRI endpoints.โžค Treatment groups were comparable and consistent with prior Phase 2 patients.โžค Safety remained consistent with previous ๐—ฃ๐—ต๐—ฎ๐˜€๐—ฒ ๐Ÿญ/๐Ÿฎ findings.โžค Ultragenyx will evaluate the future of its ๐—ฎ๐—ฝ๐—ฎ๐˜‡๐˜‚๐—ป๐—ฒ๐—ฟ๐˜€๐—ฒ๐—ป program.โžค Company plans ๐˜€๐—ถ๐—ด๐—ป๐—ถ๐—ณ๐—ถ๐—ฐ๐—ฎ๐—ป๐˜ ๐—ฒ๐˜…๐—ฝ๐—ฒ๐—ป๐˜€๐—ฒ ๐—ฟ๐—ฒ๐—ฑ๐˜‚๐—ฐ๐˜๐—ถ๐—ผ๐—ป๐˜€ while maintaining commercial operations.โžค Angelman syndrome affects approximately ๐Ÿฒ๐Ÿฌ,๐Ÿฌ๐Ÿฌ๐Ÿฌ people in accessible markets.๐Ÿ‘‰ ๐—ช๐—ต๐˜† ๐—ง๐—ต๐—ถ๐˜€ ๐— ๐—ฎ๐˜๐˜๐—ฒ๐—ฟ๐˜€:โžค Phase 3 failure creates major uncertainty around ๐—ฎ๐—ฝ๐—ฎ๐˜‡๐˜‚๐—ป๐—ฒ๐—ฟ๐˜€๐—ฒ๐—ป's development future.โžค Missing both major endpoints substantially weakens the therapy's efficacy case.โžค Planned expense reductions could materially reshape ๐—จ๐—น๐˜๐—ฟ๐—ฎ๐—ด๐—ฒ๐—ป๐˜†๐˜…'s operating priorities.โžค Outcome leaves a significant unmet need in ๐—”๐—ป๐—ด๐—ฒ๐—น๐—บ๐—ฎ๐—ป ๐˜€๐˜†๐—ป๐—ฑ๐—ฟ๐—ผ๐—บ๐—ฒ treatment.๐Ÿ‘‰ ๐—˜๐˜…๐—ฝ๐—ฒ๐—ฟ๐˜ ๐—ฆ๐˜๐—ฎ๐˜๐—ฒ๐—บ๐—ฒ๐—ป๐˜:๐—˜๐—บ๐—ถ๐—น ๐—ž๐—ฎ๐—ธ๐—ธ๐—ถ๐˜€, ๐— .๐——., ๐—ฃ๐—ต.๐——., Chief Executive Officer and President of Ultragenyx:"Based on everything we observed in the robust Phase 1/2 clinical development program and long-term extension study, we are disappointed by the Aspire result,"" datetime: "2026-09-02T20:07:20.000Z" locales: - [en](https://longbridge.com/en/topics/100000000883533.md) - [zh-CN](https://longbridge.com/zh-CN/topics/100000000883533.md) - [zh-HK](https://longbridge.com/zh-HK/topics/100000000883533.md) author: "[Hardik Shah](https://longbridge.com/en/profiles/27423834.md)" generator: "portal-rs" --- # ๐Ÿ“ข ๐—๐—จ๐—ฆ๐—ง ๐—œ๐—ก: $Ultragenyx Pharma(RARE.US) Ultragenyx โ€ฆ ### Related Stocks - [RARE.US](https://longbridge.com/en/quote/RARE.US.md) --- > **Disclaimer: This article is for reference only and does not constitute any investment advice.**