Ultragenyx Pharmaceutical Inc.
RARE evidence brief
What we can confirm
- BioSniper Score:
- 5.5 / 10
- Market Cap:
- $1.47B
- Lead Asset:
- Crysvita (Approved)
What it means
What to watch
Prepared research question
What matters most for RARE—catalysts, financial risk and supporting evidence?
Traceable citations · Unknowns marked
BioSniper AI · RARE
Ultragenyx Pharmaceutical Inc. (RARE) is a rare-disease biopharmaceutical company with approved products Crysvita and Dojolvi, a pipeline of gene therapies and antisense oligonucleotides, and a strategic restructuring underway to achieve profitability by 2027.
Key risk: GTX-102 Phase 3 failure
BioSniper Score
Based on 4 of 6 signals
Financial data as of Jun 30, 2026.
Tracking 8 pipeline candidates · 45 clinical trials for Ultragenyx Pharmaceutical Inc..
Ultragenyx Pharmaceutical Inc. (RARE) is a rare-disease biopharmaceutical company with approved products Crysvita and Dojolvi, a pipeline of gene therapies and antisense oligonucleotides, and a strategic restructuring underway to achieve profitability by 2027.
- 2025 total revenue of $673 million, with Crysvita revenue of $481 million and Dojolvi revenue of $96 million [news · published 2026-02-13].
- As of 2026-06-30, the company reported cash & equivalents of $125.0M, total revenue of $214.0M, net income of -$92.0M, and shareholders' equity of -$291.0M [sec].
- The Phase 3 Aspire study for apazunersen (GTX-102) in Angelman syndrome did not achieve the primary endpoint or the key secondary endpoint [news · published 2026-09-02].
- The FDA cleared the IND for UX016, a small molecule prodrug of sialic acid for GNE myopathy [news · published 2026-03-30].
- The company initiated a strategic restructuring plan to reduce expenses and headcount, reiterating a path to profitability in 2027 [news · published 2026-02-13].
What works
- Approved products Crysvita and Dojolvi generate substantial revenue, with 2025 combined revenue of $577 million [news · published 2026-02-13].
- The company has multiple late-stage pipeline candidates, including GTX-102 (Phase 3) and UX111 (BLA resubmitted), targeting large rare-disease markets [news · published 2026-02-16].
- A strategic restructuring plan aims to focus expenses and achieve profitability by 2027 [news · published 2026-02-13].
What to weigh
- The Phase 3 Aspire study for GTX-102 failed to meet its primary and key secondary endpoints [news · published 2026-09-02].
- Shareholders' equity is deeply negative at -$291.0M as of 2026-06-30, indicating accumulated losses [sec].
- Setrusumab (UX143) Phase 2b asteroid study showed no changes in radial trabecula vBMD (p>0.05) [catalyst].
- The company announced a 10% workforce reduction as part of its restructuring, reflecting operational challenges [news · published 2026-02-16].
From the filings
Quoted directly from source documents.
Phase 3 Aspire study for GTX-102 failed primary endpoint
“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").”
News · September 2, 2026
FDA cleared IND for UX016
“the U.S. Food and Drug Administration (the "FDA") has cleared the Investigational New Drug ("IND") application for UX016, an investigational small molecule prodrug of sialic acid ("SA") being evaluated as a substrate replacement therapy for GNE myopathy ("GNEM").”
News · March 30, 2026
2025 total revenue of $673 million
“2025 total revenue of $673 million,Crysvita® revenue of $481 million and Dojolvi® revenue of $96 million”
News · February 13, 2026
Setrusumab Phase 2b asteroid study showed no vBMD change
“There were no changes in radial trabecula vBMD (p>05).”
Catalysts · October 7, 2024
Catalyst timeline
- 2024-10-07 · Trial_Readout: Setrusumab for the treatment of osteogenesis imperfecta: 12-month results from the phase 2b asteroid study — Results showed no changes in radial trabecula vBMD (p>0.05).
- 2024-10-25 · AdCom: AdCom: setrusumab (UX143) — Advisory committee meeting for setrusumab.
- 2024-12-03 · Phase3_Start: Phase3_Start: An Open-label Phase 3 Study of Setrusumab in Pediatric Japanese Subjects With Osteogenesis Imperfecta Type I, III, or IV — Initiation of a Phase 3 study for setrusumab in pediatric Japanese subjects.
- 2025-06-27 · Phase3_Start: Phase3_Start: A Phase 3, Randomized, Double-blind, Sham-controlled Study Investigating the Efficacy and Safety of GTX-102 in Pediatric Subjects With Angelman Syndrome — Initiation of the Phase 3 Aspire study for GTX-102.
- 2025-08-18 · AdCom: AdCom: GTX-102 (apazunersen) — Advisory committee meeting for GTX-102.
- 2025-10-13 · AdCom: AdCom: DTX401 — Advisory committee meeting for DTX401.
- Phase2_Start: Phase2_Start: A Phase 2, Open-label, Basket Study Investigating the Safety and Efficacy of GTX-102 in Adults — Initiation of a Phase 2 basket study for GTX-102 in adults.
Risks & what to watch (4)›
Key risks
- GTX-102 Phase 3 failure — The Aspire study missed primary and key secondary endpoints, jeopardizing the Angelman syndrome program [news · published 2026-09-02].
- Negative shareholders' equity — Shareholders' equity of -$291.0M as of 2026-06-30 signals deep accumulated losses [sec].
- Setrusumab efficacy concerns — Phase 2b asteroid study showed no significant change in radial trabecula vBMD [catalyst].
- Restructuring execution risk — Workforce reduction and expense cuts may impact pipeline progress and morale [news · published 2026-02-16].
What to watch
- PDUFA date for UX111 (Sanfilippo syndrome type A) expected in Q3 2026 [news · published 2026-01-30].
- 2026 total revenue guidance of $730M–$760M from current products [news · published 2026-02-13].
- Potential FDA decisions on DTX401 and GTX-102 [news · published 2026-06-21].
- Progress of the strategic restructuring and path to profitability in 2027 [news · published 2026-02-13].
Sources: Catalysts · News · Pipeline · Generated October 13, 2026 · How we verify sources →
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Evidence & data
Score breakdownBased on 4 of 6 signals · derived from public data
Cash runway data unavailable
Net income -$92M
Lead: Approved
8 candidates
No upcoming catalysts tracked
0 buys · 9 sells (180d)
Pipeline
Regulatory
Financials
Short Interest
as of May 22, 2026- % of float
- 14.4%
- Days to cover
- 7.5
- Shares short
- 13,665,812
- Daily short volume
- -
Short percent of float and days-to-cover reflect reported short positioning. Daily short volume is the exchange-reported short share of a single day's trading volume and is noisier. See the most-shorted biotech ranking →
RARE short interestGovernance
Frequently asked questions
What is the AI investment thesis for Ultragenyx Pharmaceutical Inc.?
Ultragenyx Pharmaceutical Inc. (RARE) is a rare-disease biopharmaceutical company with approved products Crysvita and Dojolvi, a pipeline of gene therapies and antisense oligonucleotides, and a strategic restructuring underway to achieve profitability by 2027.
What is Ultragenyx Pharmaceutical Inc.'s lead drug candidate?
Ultragenyx Pharmaceutical Inc.'s most advanced tracked candidate is Crysvita (Approved) for X-Linked Hypophosphatemia (XLH) and Tumor-Induced Osteomalacia (TIO).
Who is the CEO of Ultragenyx Pharmaceutical Inc.?
Dr. Emil D. Kakkis M.D., Ph.D. is the chief executive officer of Ultragenyx Pharmaceutical Inc. (RARE).
Where is Ultragenyx Pharmaceutical Inc. headquartered?
Ultragenyx Pharmaceutical Inc. (RARE) is headquartered in Novato, CA, United States.
What therapeutic areas does Ultragenyx Pharmaceutical Inc. focus on?
Ultragenyx Pharmaceutical Inc. develops therapies across Oncology, Rare Disease, Gene Therapy, Metabolic, Musculoskeletal.
The AI overview and BioSniper Score are neutral, data-derived signals built from public SEC filings, ClinicalTrials.gov and FDA records — not investment advice.