Ultragenyx Pharmaceutical Inc.
Ultragenyx Pharmaceutical Inc. (RARE): 25 pipeline candidates · 11 upcoming catalysts · ~7 mo cash runway · 44 clinical trials. Updated within the last hour.
BioSniper Score
6-factor signal · derived from public data
7.2 months
D/E -6.46 · 2% dilution
Lead: Approved
25 candidates
11 upcoming, next in ~66d
0 buys · 19 sells (180d)
A transparent, data-derived signal for Ultragenyx Pharmaceutical Inc.across cash runway, pipeline maturity & breadth, catalyst momentum and clinical/regulatory footprint. Each factor traces to tracked public filings — not an investment recommendation.
Key Financials
Market Cap
$2.7B
Cash
$175.0M
Quarterly Burn
$197.0M
Runway
7.2 mo
Revenue
$136.0M
Cash Runway vs Catalysts
A catalyst falls after projected cash runway — potential financing / dilution risk before it hits.
Pipeline (25 candidates)
| Drug | Phase |
|---|---|
| Adjuvant IM Therapy | Phase 2/Phase 3 |
| Adjuvant Immunomodulatory Therapy | Phase 3 |
| Bisphosphonate | Phase 3 |
| Crysvita | Approved |
| Dojolvi | Approved |
| DTX301 | Phase 3 |
| DTX401 | Phase 3 |
| Evinacumab | Phase 3 |
| Evkeeza | Approved |
| GLUT1 DS | Phase 3 |
Showing 10 of 25 candidates. Sign up for full pipeline.
Upcoming Catalysts (11)
Binary FDA approval decision — outcome can sharply revalue the stock.
Clinical data readout — efficacy/safety results are a high-impact catalyst.
36.4% historical Phase 2 primary-endpoint success rate (n=228)
Clinical data readout — efficacy/safety results are a high-impact catalyst.
68.8% historical Phase 3 primary-endpoint success rate (n=362)
Clinical data readout — efficacy/safety results are a high-impact catalyst.
68.8% historical Phase 3 primary-endpoint success rate (n=362)
Clinical data readout — efficacy/safety results are a high-impact catalyst.
68.8% historical Phase 3 primary-endpoint success rate (n=362)
Institutional Holdings
Source: SEC Form 13F · as of Mar 2026Institutional holders
292
Total value held
$1.98B
| Holder | Shares | Value |
|---|---|---|
| vanguard capital management llc | 8,379,662 | $175.6M |
| blackrock, inc. | 7,299,598 | $152.9M |
| vanguard portfolio management llc | 5,385,992 | $112.8M |
| state street corp | 5,021,441 | $105.2M |
| aqr capital management llc | 4,578,024 | $95.9M |
| rtw investments, lp | 3,648,795 | $76.4M |
| sands capital management, llc | 3,214,533 | $67.3M |
| wellington management group llp | 2,640,812 | $55.3M |
| citadel advisors llc | 2,371,391 | $49.7M |
| assenagon asset management s.a. | 2,257,671 | $47.3M |
Showing top 10 of 292 institutional holders of Ultragenyx Pharmaceutical Inc.. Sign up for the full 13F holder list and quarter-by-quarter ownership history.
Frequently asked questions
How many drug candidates are in Ultragenyx Pharmaceutical Inc.'s pipeline?
Ultragenyx Pharmaceutical Inc. (RARE) has 25 pipeline candidates tracked by BioSniper across SEC filings and clinical trial registrations.
What is Ultragenyx Pharmaceutical Inc.'s lead drug candidate?
Ultragenyx Pharmaceutical Inc.'s most advanced tracked candidate is Adjuvant IM Therapy (Phase 2/Phase 3) for MPS IIIA.
What is Ultragenyx Pharmaceutical Inc.'s next catalyst?
Ultragenyx Pharmaceutical Inc.'s next tracked catalyst is a PDUFA event — "PDUFA Date: dtx401 aav gene therapy" — expected August 23, 2026.
What is Ultragenyx Pharmaceutical Inc.'s cash runway?
Based on its most recent reported cash balance and quarterly burn rate, Ultragenyx Pharmaceutical Inc. has approximately 7.2 months of cash runway.
What is Ultragenyx Pharmaceutical Inc.'s market capitalization?
Ultragenyx Pharmaceutical Inc.'s market capitalization is approximately $2.7B.
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