RARE
Ultragenyx Pharmaceutical Inc.
Nasdaq: RARE · Novato, CA · Healthcare
$24.10-0.96 (-3.83%)Closed
Market Cap$2.33B
Cash$421.0Mmost recent
Runway8 mo$166.5M Q burn
P/E (TTM)—EPS $-5.83
52-Wk Range$18.50 – $41.44
Avg Volume1.9M3-month
Price & Trading Activity
Daily close with catalyst overlays and trading volume
$24.10-44.8%
Pipeline
Drug candidates sponsored by Ultragenyx Pharmaceutical · ClinicalTrials.gov
| Phase | Drug / Candidate | Indications | Status | Next Completion | Trials | |
|---|---|---|---|---|---|---|
| Phase 3 | aceneuramic acid extended-release (Ace-ER) | Hereditary Inclusion Body Myopathy+3 more | Completed | 2017-06-09past | 1 | |
| Phase 3 | Aceneuramic Acid Extended-Release Tablets | Hereditary Inclusion Body Myopathy+4 more | Terminated | 2018-01-10past | 1 | |
| Phase 3 | UX003 | MPS 7+5 more | Completed | 2019-03-26past | 4 | |
| Phase 3 | UX007 | Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)+9 more | Completed | 2020-12-03past | 5 | |
| Phase 3 | DTX401 | Glycogen Storage Disease Type IA+1 more | Completed | 2024-02-20past | 2 | |
| Phase 3 | Bisphosphonate | Osteogenesis Imperfecta | Active, not recruiting | 2025-10-23past | 1 | |
| Phase 3 | GTX-102 | Angelman Syndrome | Recruiting | 2026-07 | 4 | |
| Phase 2 | Setrusumab | Osteogenesis Imperfecta+3 more | Active, not recruiting | 2027-01 | 4 | |
| Phase 2 | UX111 | MPS IIIA+3 more | Recruiting | 2027-07 | 1 | |
| Phase 3 | Triheptanoin | Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)+1 more | Active, not recruiting | 2027-08 | 2 | |
| Phase 3 | Adjuvant Immunomodulatory (IM) Therapy | Mucopolysaccharidosis IIIA+3 more | Enrolling | 2027-08 | 1 | |
| Phase 3 | DTX301 | OTC Deficiency | Active, not recruiting | 2027-09 | 1 | |
| Phase 2 | BPS804 20mg/Kg | Osteopenia+1 more | Completed | 2013-10past | 1 | |
| Phase 2 | Sialic Acid Extended Release (SA-ER) | GNE Myopathy+1 more | Completed | 2013-11past | 1 | |
| Phase 2 | SA-ER 500 mg | GNE Myopathy+1 more | Completed | 2017-02-14past | 1 | |
| Phase 1 | DTX101 | Hemophilia B | Terminated | 2017-10-18past | 1 | |
| Phase 2 | Aceneuramic Acid Extended-Release | Hereditary Inclusion Body Myopathy+5 more | Terminated | 2018-01-10past | 1 | |
| Phase 2 | BPS804 | Hypophosphatasia+2 more | Completed | 2018-11-01past | 4 | |
| Phase 1 | scAAV8OTC | Ornithine Transcarbamylase (OTC) Deficiency | Completed | 2021-12-16past | 1 | |
| Phase 1 | ABO-102 | MPS IIIA+3 more | Terminated | 2022-03-10past | 1 | |
| Phase 1 | UX053 | Glycogen Storage Disease Type III | Terminated | 2023-03-20past | 1 | |
| Phase 1 | UX016 | GNE Myopathy | Not yet recruiting | 2028-12 | 1 | |
| Phase 1 | UX701 | Wilson Disease | Active, not recruiting | 2029-03 | 1 | |
| Phase 1 | Sialic Acid Extended Release (SA-ER) Tablets | Hereditary Inclusion Body Myopathy (HIBM) | Completed | 2012-04past | 1 | |
| N/A | Unnamed | Hereditary Inclusion Body Myopathy+4 more | Completed | 2017-11-30past | 1 | |
| N/A | Unnamed | Ornithine Transcarbamylase Deficiency | Completed | 2020-02-20past | 1 | |
| N/A | Unnamed | Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) | Completed | 2020-06-08past | 1 | |
| N/A | Unnamed | Hemophilia B | Completed | 2021-11-06past | 1 | |
| N/A | Unnamed | Ornithine Transcarbamylase Deficiency | Terminated | 2021-12-15past | 1 | |
| N/A | Unnamed | Glycogen Storage Disease Type IA | Completed | 2022-03-04past | 1 | |
| N/A | Unnamed | Wilson Disease | Completed | 2022-03-25past | 1 | |
| N/A | Unnamed | Glycogen Storage Disease Type III | Completed | 2022-06-30past | 1 | |
| N/A | Unnamed | Creatine Deficiency, X-linked | Terminated | 2022-10-24past | 1 | |
| N/A | Unnamed | Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) | Terminated | 2022-10-27past | 1 | |
| N/A | Unnamed | Ornithine Transcarbamylase Deficiency+2 more | Terminated | 2022-11-17past | 1 | |
| N/A | Unnamed | Glycogen Storage Disease Type III | Terminated | 2023-03-02past | 1 | |
| N/A | Unnamed | Glycogen Storage Disease Type IA+1 more | Completed | 2025-02-25past | 1 | |
| N/A | Unnamed | Ornithine Transcarbamylase (OTC) Deficiency | Active, not recruiting | 2029-12 | 1 | |
| N/A | Unnamed | Tumor-induced Osteomalacia (TIO) | Active, not recruiting | 2032-02-28 | 1 | |
| N/A | Unnamed | X-linked Hypophosphatemia+1 more | Active, not recruiting | 2032-12 | 1 | |
| N/A | Unnamed | Mucopolysaccharidosis VII+3 more | Recruiting | 2033-05 | 1 | |
| N/A | Unnamed | Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) | Active, not recruiting | 2035-12 | 1 | |
| N/A | Unnamed | Glycogen Storage Disease Type Ia | Recruiting | 2036-12 | 1 | |
| N/A | Mepsevii | MPS VII+2 more | Available | — | 1 |
Valuation
Risk-adjusted NPV
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Peer comparison
Cohort check
No peer cohort defined for RARE. Cohorts currently cover gene editing, mRNA, rare-disease gene therapy, and large-cap biotech — more coming soon.