← boardnext 2027-01next 2027-08next 2027-08next 2027-09next 2028-12next 2029-02next 2029-03next 2029-03
RARE
Ultragenyx Pharmaceutical Incdistress 4/10 · elevated risk$27.20-2%today-26% from 52w high
Lead: Sanfilippo syndrome type A (MPS IIIA)
Revenue
$136M
Liquidity
$175M
Runway
0.3 yrs
Shares 1y
+2%
Price & events
+16.2% over 6M
CatalystRating changeDilutionFinancingdashed = today
-1.6%
1D
-10.0%
1W
-5.4%
1M
+12.0%
3M
+12.4%
6M
-2.9%
1Y
-39.8%
2Y
-67.1%
5Y
-80.1%
All
Overview
- Market Cap
- $2.68B
- Volume
- 64.42K
- 52 Week Range
- $18.50 – $36.54
- Total Shares
- 98.49M
- Float Shares
- 94.51M
- Short %
- 17.03%
- % Held by Insiders
- 3.45%
- % Held by Institutions
- 101.71%
- RVOL
- 0.02x
- Beta
- 0.31
Pipeline stages · 9 programs
setrusumab
Preclin›
Ph1›
Ph2›
Ph3›
Filed›
PDUFA›
Approved
Triheptanoin
Preclin›
Ph1›
Ph2›
Ph3›
Filed›
PDUFA›
Approved
Adjuvant Immunomodulatory (IM) Therapy
Preclin›
Ph1›
Ph2›
Ph3›
Filed›
PDUFA›
Approved
DTX301, Oral Corticosteroids, Sodium Acetate
Preclin›
Ph1›
Ph2›
Ph3›
Filed›
PDUFA›
Approved
UX016
Preclin›
Ph1›
Ph2›
Ph3›
Filed›
PDUFA›
Approved
GTX-102
Preclin›
Ph1›
Ph2›
Ph3›
Filed›
PDUFA›
Approved
UX111, Prophylactic Immunomodulatory (IM) Therapy, Optimized Prophylactic IM Therapy
Preclin›
Ph1›
Ph2›
Ph3›
Filed›
PDUFA›
Approved
UX701, Standard of Care (SOC)
Preclin›
Ph1›
Ph2›
Ph3›
Filed›
PDUFA›
Approved
Bisphosphonate, Setrusumab
Preclin›
Ph1›
Ph2›
Ph3›
Filed›
PDUFA›
Approved
Pipeline · 12 trials · 9 upcoming
| Readout | Phase | Drug | Indication | Enroll | Status |
|---|---|---|---|---|---|
| 2027-01 Estimated 50% readout ~2027-03-17 | Phase 3 | setrusumab | Osteogenesis Imperfecta | — | active not recruiting |
| 2027-08 Estimated 50% readout ~2027-10-15 | Phase 3 | Triheptanoin | Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) | — | active not recruiting |
| 2027-08 Estimated 50% readout ~2027-10-15 | Phase 3 | Adjuvant Immunomodulatory (IM) Therapy | Mucopolysaccharidosis IIIA, MPS IIIA, Sanfilippo Syndrome, Sanfilippo A | — | enrolling by invitation |
| 2027-09 Estimated 50% readout ~2027-11-15 | Phase 3 | DTX301, Oral Corticosteroids, Sodium Acetate | OTC Deficiency | — | active not recruiting |
| 2028-12 Estimated 50% readout ~2029-01-15 | Phase 1/2 | UX016 | GNE Myopathy | — | not yet recruiting |
Show all 12 trials · 7 more ▾
| Readout | Phase | Drug | Indication | Enroll | Status |
|---|---|---|---|---|---|
| 2029-02 Estimated 50% readout ~2029-04-17 | Phase 3 | GTX-102 | Angelman Syndrome | — | enrolling by invitation |
| 2029-03 Estimated 50% readout ~2029-05-15 | Phase 2/3 | UX111, Prophylactic Immunomodulatory (IM) Therapy, Optimized Prophylactic IM Therapypriority review | MPS IIIA, Sanfilippo Syndrome, Sanfilippo A, Mucopolysaccharidosis III | — | recruiting |
| 2029-03 Estimated 50% readout ~2029-04-15 | Phase 1/2 | UX701, Standard of Care (SOC) | Wilson Disease | — | active not recruiting |
| 2030-01 Estimated 50% readout ~2030-02-15 | Phase 2 | GTX-102 | Angelman Syndrome | — | recruiting |
| 2026-07 | Phase 3 | GTX-102 | Angelman Syndrome | 129 | active not recruiting |
| 2025-10-23 | Phase 3 | Bisphosphonate, Setrusumab | Osteogenesis Imperfecta | 69 | active not recruiting |
| 2025-10-20 | Phase 2/3 | Setrusumab | Osteogenesis Imperfecta | 183 | active not recruiting |
Catalyst news & presentations · 19
- first half of 2027Guided 55%readoutPhase 3DTX301 · Second primary endpoint data from Phase 3 Enh3ance study evaluating reduction in treatment burden.SEC 8-K
- second half of 2026Guided 55%trial initiationsecondaryPhase 1/2UX016 · first-in-human, Phase 1/2 study evaluating safety and efficacySEC 8-K
- next week at WORLDSymposium 2026Guided 40%conferencedata presentationsecondaryUX111 · Detailed updates from the resubmitted BLA will be presented at WORLDSymposium 2026 in San Diego.SEC 8-K
- second half of 2026Guided 55%readoutPhase 3GTX-102 · Topline data from Phase 3 Aspire study of GTX-102 in patients aged 4-17 with full maternal UBE3A gene deletion.SEC 8-K
- September 19, 2026Confirmed 100%PDUFAPDUFAUX111 · PDUFA action date setSEC 8-K
- August 23, 2026Confirmed 100%PDUFAPDUFADTX401 · PDUFA action date for DTX401 BLASEC 8-K
- 2026-08-04Estimated 45%earningsEarningsEarnings releaseyahoo
- 2026-04-02filing acceptanceFiled (NDA/BLA)UX111 · FDA accepted resubmitted BLA for reviewSEC 8-K
- 2026-03-30fda agreementUX016 · FDA cleared IND for UX016SEC 8-K
- Friday, February 6 at 8 a.m. PSTconferencedata presentationsecondaryUX111 · Oral presentation at WORLDSymposium 2026SEC 8-K
- on February 3, 2026regulatory submissionFiled (NDA/BLA)UX111 · BLA resubmitted to FDASEC 8-K
- fourth quarter of 2025regulatory submissionFiled (NDA/BLA)DTX401 · Initiated rolling BLA submission; non-clinical and clinical modules submitted, CMC module planned for Q4 2025.SEC 8-K
- around the end of the yearreadoutPhase 3UX143 · Final analysis of Phase 3 Orbit studySEC 8-K
- October 30, 2025trial initiationsecondaryGTX-102 · First patient dosed in Aurora study evaluating GTX-102 for Angelman syndrome.SEC 8-K
- July 2025enrollment completesecondaryPhase 3GTX-102 · Phase 3 Aspire study fully enrolled with 129 participantsSEC 8-K
- 2025-07-11crlUX111 · FDA issued Complete Response Letter for BLA; requests additional CMC information and manufacturing facility observations.SEC 8-K
- December 19, 2024regulatory submissionFiled (NDA/BLA)UX111 · Submitted BLA to FDA seeking accelerated approval for UX111 AAV gene therapy based on surrogate endpoint of CSF heparan sulfate reduction.SEC 8-K
- December 19, 2024trial initiationsecondaryPhase 3GTX-102 · First patient dosed in global Phase 3 Aspire study evaluating GTX-102 for Angelman syndromeSEC 8-K
- November 9, 2024conferencedata presentationsecondaryPhase 1/2GTX-102 · Phase 1/2 data presented at FAST Global Science SummitSEC 8-K
Approved drugs (2)
- DOJOLVITRIHEPTANOIN2020 · Prescription
- MEPSEVIIVESTRONIDASE ALFA-VJBK2017 · Prescription
Capital structure — dilution & financing
🔒 Top holders — Pro
Institutional ownership + quarter-over-quarter accumulation/trimming.
Upgrade to ProWall St ratings
strong buy · target $53 (+93% upside) · 19 analysts
- 2024-10-22Cantor FitzgeraldOverweight
- 2024-10-21TD CowenBuy
- 2024-10-01Cantor FitzgeraldOverweight
- 2024-09-26RBC CapitalOutperform
- 2024-09-20Cantor FitzgeraldOverweight
- 2024-09-16Cantor FitzgeraldOverweight
Competitors — Sanfilippo syndrome type A (MPS IIIA)
- none identified
Recent news
- 2026-07-21Ultragenyx Pharmaceutical Stock Extends A 9-Day Losing Streak To A 20% Loss - Trefis
- 2026-07-20(RARE) Movement as an Input in Quant Signal Sets - Stock Traders Daily
- 2026-07-20RARE stock reflects Ultragenyx cash position and recent clinical progress - Ad-hoc-news.de
- 2026-07-18Ultragenyx Pharmaceutical (RARE) On Rare Disease Growth Narrative And Why Valuation Is Back In Focus - Sahm
- 2026-07-17RARE Stock Slumps On Multiple Wall Street Price Target Slashes, Clinical Pipeline Uncertainty - Stocktwits
- 2026-07-17Ultragenyx Pharmaceutical Currently Down Eight Consecutive Days, on Pace for Longest Losing Streak Since December 2024 -- Data Talk - Moomoo
- 2026-07-17Ultragenyx Pharmaceutical Currently Down Eight Consecutive Days, on Pace for Longest Losing Streak Since December 2024 -- Data Talk - Moomoo
- 2026-07-17RARE stock rises after Ultragenyx reports strong Crysvita revenue growth and narrows quarterly loss - Ad-hoc-news.de
Not financial advice. Data: ClinicalTrials.gov, SEC EDGAR, FINRA, Nasdaq, Yahoo.