Ultragenyx Pharmaceutical Inc.
RARE · Pharmaceutical Preparations · 98.5M shares · +7%/yr ↑ dilution · EDGAR filings ↗
Reference price $25.05 · est. market cap ~$2.5B — last insider sale, 2026-06-15 (estimated from the dated SEC Form 4 price × shares outstanding — not a live quote)
Grade
C58Sizable rare-disease commercial franchise with four products and 20% revenue growth, but deeply negative free cash flow.
Strengths
- Four approved products, $673M revenue, revenue growth 20% (61st pct) and $737M cash (90th pct)
- Multiple positive readouts (MPS VII vestronidase, DTX401 GSD Ia, LC-FAOD) plus an advancing setrusumab OI program
Risks
- FCF -$472M (2nd pct) and ~$575M net loss; runway 6.2 quarters keeps capital-raise pressure
- Several outright Phase 3 failures (GNE myopathy Ace-ER, Glut1 DS triheptanoin) and a mixed OI Phase 2 primary miss
Notable findings
- Restatement• Provisions in our amended and restated certificate of incorporation and by-laws, as well as provisions of Delaware law, could make it more difficult for a third party to acquire us or increase the c 8-K ↗
- Fund exitedNotable fund(s) exited this quarter: VANGUARD GROUP INC
- Net lossNet loss (net margin -85.4%)
- Cash burnNegative free cash flow (-472.0M)
- Exec changeRecent 8-K item 5.02 8-K ↗
- Trial resultPositive trial readout — primary endpoint met (8-K) 8-K ↗
- Notable holderHeld by 5 notable fund(s): STATE STREET CORP, CITADEL ADVISORS LLC, RENAISSANCE TECHNOLOGIES LLC +2 more (as of 2026-03-31)
- Insider sellingInsider selling — 20 open-market sale(s) by 9 insider(s) (cluster of 9 insiders, ~$3M, last 6mo) Form 4 ↗
- Trial readout soonUpcoming trial readout ~2026-07 — PHASE3 · Angelman Syndrome
- Revenue declineRevenue -34.4% YoY
Quarterly revenue
Last 4 quarters · from SEC filings.
Key metrics
Operating margin
-79.5%
Net margin
-85.4%
Return on equity
718.8%
Return on assets
-37.5%
Revenue growth (QoQ)
-34.4%
Current ratio
2.5×
Free cash flow
-$472M
EBITDA
-$500M
Tangible book value
-$300M
Cash & investments
$737M
Cash runway
6q
Stock comp / revenue
22.7%
Insider transactions · last 18 months
| Date | Insider | Type | Shares | Price |
|---|---|---|---|---|
| 2026-06-15 | Sanders Corazon (Corsee) D. | Sell | 2.0K | $25.05 |
| 2026-06-15 | Parschauer Karah Herdman | Sell | 1.9K | $24.62 |
| 2026-06-01 | Horn Howard | Sell | 4.7K | $23.77 |
| 2026-05-18 | SULIMAN SHEHNAAZ | Sell | 5.7K | $25.12 |
| 2026-05-14 | NARACHI MICHAEL | Grant | 7.8K | — |
| 2026-05-14 | WELCH DANIEL G | Grant | 7.8K | — |
| 2026-05-14 | Fust Matthew K | Grant | 7.8K | — |
| 2026-05-14 | Dunsire Deborah | Grant | 7.8K | — |
| 2026-05-14 | Ray Amrit | Grant | 7.8K | — |
| 2026-05-14 | Sanders Corazon (Corsee) D. | Grant | 7.8K | — |
| 2026-05-14 | SULIMAN SHEHNAAZ | Grant | 7.8K | — |
| 2026-05-05 | Crombez Eric | Sell | 344 | $24.96 |
| 2026-05-01 | Horn Howard | Sell | 4.7K | $24.90 |
| 2026-04-22 | Huizenga Theodore Alan | Grant | 20K | — |
| 2026-04-16 | KAKKIS EMIL D | Grant | 77K | — |
| 2026-04-16 | Huang Dennis Karl | Grant | 23K | — |
| 2026-04-16 | Pinion John Richard | Grant | 23K | — |
| 2026-04-16 | Parschauer Karah Herdman | Grant | 29K | — |
| 2026-04-16 | Harris Erik | Grant | 29K | — |
| 2026-04-16 | Crombez Eric | Grant | 35K | — |
| 2026-04-16 | Horn Howard | Grant | 35K | — |
| 2026-04-01 | Horn Howard | Sell | 4.7K | $20.97 |
| 2026-03-10 | Parschauer Karah Herdman | Sell | 3.5K | $22.67 |
| 2026-03-06 | Parschauer Karah Herdman | Sell | 6.0K | $21.40 |
| 2026-03-03 | Horn Howard | Sell | 1.6K | $22.40 |
| 2026-03-02 | Horn Howard | Sell | 3.1K | $22.54 |
| 2026-03-02 | KAKKIS EMIL D | Sell | 54K | $22.80 |
| 2026-03-02 | Crombez Eric | Sell | 7.0K | $22.80 |
| 2026-03-02 | Harris Erik | Sell | 11K | $22.80 |
| 2026-03-02 | Huang Dennis Karl | Tax withheld | 999 | $23.39 |
| 2026-03-02 | Parschauer Karah Herdman | Sell | 8.1K | $22.80 |
| 2026-03-02 | Huizenga Theodore Alan | Tax withheld | 148 | $23.39 |
| 2026-03-02 | Huizenga Theodore Alan | Sell | 1.6K | $22.80 |
| 2026-03-02 | Horn Howard | Sell | 5.3K | $22.80 |
| 2026-03-02 | Pinion John Richard | Sell | 10K | $22.80 |
| 2026-03-01 | Pinion John Richard | Grant | 11K | — |
| 2026-03-01 | Harris Erik | Grant | 12K | — |
| 2026-03-01 | KAKKIS EMIL D | Grant | 71K | — |
| 2026-03-01 | Parschauer Karah Herdman | Grant | 11K | — |
| 2026-03-01 | Horn Howard | Grant | 6.1K | — |
| 2026-03-01 | Crombez Eric | Grant | 12K | — |
| 2026-03-01 | Huang Dennis Karl | Grant | 11K | — |
| 2026-02-02 | Horn Howard | Sell | 3.1K | $23.56 |
| 2026-01-02 | Horn Howard | Sell | 3.1K | $22.84 |
| 2025-12-29 | Huizenga Theodore Alan | Sell | 85 | $34.38 |
| 2025-12-10 | Horn Howard | Sell | 3.1K | $36.38 |
| 2025-10-13 | Horn Howard | Sell | 7.9K | $31.51 |
| 2025-09-18 | Huizenga Theodore Alan | Sell | 64 | $29.17 |
| 2025-09-12 | Parschauer Karah Herdman | Sell | 2.5K | $31.17 |
| 2025-09-05 | Huizenga Theodore Alan | Sell | 84 | $31.39 |
| 2025-06-20 | Sanders Corazon (Corsee) D. | Sell | 2.4K | $37.39 |
| 2025-05-15 | Dunsire Deborah | Grant | 5.7K | — |
| 2025-05-15 | Ray Amrit | Grant | 5.7K | — |
| 2025-05-15 | Sanders Corazon (Corsee) D. | Grant | 5.7K | — |
| 2025-05-15 | SULIMAN SHEHNAAZ | Grant | 5.7K | — |
| 2025-05-15 | WELCH DANIEL G | Grant | 5.7K | — |
| 2025-05-15 | NARACHI MICHAEL | Grant | 5.7K | — |
| 2025-05-15 | Fust Matthew K | Grant | 5.7K | — |
| 2025-05-05 | Crombez Eric | Sell | 520 | $39.24 |
| 2025-04-21 | Crombez Eric | Sell | 242 | $35.11 |
| 2025-03-06 | Horn Howard | Sell | 1.8K | $40.40 |
| 2025-03-03 | Harris Erik | Sell | 15K | $42.10 |
| 2025-03-03 | Kassberg Thomas Richard | Sell | 6.0K | $42.10 |
| 2025-03-03 | Kassberg Thomas Richard | Tax withheld | 508 | $42.92 |
| 2025-03-03 | Parschauer Karah Herdman | Sell | 13K | $42.10 |
| 2025-03-03 | Crombez Eric | Sell | 8.9K | $42.10 |
| 2025-03-03 | Pinion John Richard | Sell | 14K | $42.10 |
| 2025-03-03 | Pinion John Richard | Tax withheld | 31 | $42.92 |
| 2025-03-03 | KAKKIS EMIL D | Sell | 73K | $42.10 |
| 2025-03-03 | Huang Dennis Karl | Tax withheld | 1.3K | $42.92 |
| 2025-03-03 | Huizenga Theodore Alan | Sell | 967 | $42.10 |
| 2025-03-03 | Huizenga Theodore Alan | Tax withheld | 141 | $42.92 |
| 2025-03-01 | Parschauer Karah Herdman | Grant | 14K | — |
| 2025-03-01 | KAKKIS EMIL D | Grant | 43K | — |
| 2025-03-01 | KAKKIS EMIL D | Grant | 107K | — |
| 2025-03-01 | Horn Howard | Grant | 16K | — |
| 2025-03-01 | Huizenga Theodore Alan | Grant | 9.4K | — |
| 2025-03-01 | Pinion John Richard | Grant | 14K | — |
| 2025-03-01 | Pinion John Richard | Grant | 18K | — |
| 2025-03-01 | Harris Erik | Grant | 17K | — |
| 2025-03-01 | Harris Erik | Grant | 18K | — |
| 2025-03-01 | Kassberg Thomas Richard | Grant | 18K | — |
| 2025-03-01 | Huang Dennis Karl | Grant | 14K | — |
| 2025-03-01 | Huang Dennis Karl | Grant | 18K | — |
| 2025-03-01 | Parschauer Karah Herdman | Grant | 18K | — |
| 2025-03-01 | Crombez Eric | Grant | 17K | — |
| 2025-03-01 | Crombez Eric | Grant | 15K | — |
| 2025-02-28 | KAKKIS EMIL D | Sell | 25K | $42.48 |
| 2025-02-27 | Parschauer Karah Herdman | Sell | 2.2K | $42.93 |
| 2025-02-27 | Parschauer Karah Herdman | Sell | 760 | $42.75 |
All non-derivative Form 4 activity (SEC), newest first.
Clinical trials
| Trial | Title | Phase | Status | Primary completion |
|---|---|---|---|---|
| NCT06636383 ↗ | Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program Glycogen Storage Disease Type Ia | — | Recruiting | 2036-12-01 |
| NCT04632953 ↗ | Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) | — | Active Not Recruiting | 2035-12-01 |
| NCT03651505 ↗ | X-linked Hypophosphatemia Disease Monitoring Program X-linked Hypophosphatemia, Hypophosphatemic Rickets | — | Active Not Recruiting | 2032-12-01 |
| NCT03604835 ↗ | Mucopolysaccharidosis VII Disease Monitoring Program Mucopolysaccharidosis VII, MPS VII | — | Recruiting | 2032-04-01 |
| NCT04783428 ↗ | Tumor-induced Osteomalacia Disease Monitoring Program Tumor-induced Osteomalacia (TIO) | — | Active Not Recruiting | 2032-02-28 |
| NCT07157254 ↗ | A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS) Angelman Syndrome | PHASE2 | Recruiting | 2030-01-01 |
| NCT03636438 ↗ | Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency Ornithine Transcarbamylase (OTC) Deficiency | — | Active Not Recruiting | 2029-12-01 |
| NCT02716246 ↗ | Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH MPS IIIA, Sanfilippo Syndrome | PHASE2/PHASE3 | Recruiting | 2029-03-01 |
| NCT04884815 ↗ | A Phase 1/2/3 Study of UX701 Gene Therapy in Adults With Wilson Disease Wilson Disease | PHASE1/PHASE2 | Active Not Recruiting | 2029-03-01 |
| NCT06415344 ↗ | Long-term Extension of GTX-102 in Angelman Syndrome Angelman Syndrome | PHASE3 | Enrolling By Invitation | 2029-02-01 |
| NCT05345171 ↗ | Clinical Study of DTX301 AAV-Mediated Gene Transfer for Ornithine Transcarbamylase (OTC) Deficiency OTC Deficiency | PHASE3 | Active Not Recruiting | 2027-09-01 |
| NCT05933200 ↗ | A Study to Determine the Effect of Triheptanoin Compared With Even-Chain MCT on MCEs in Pediatric Patients With LC-FAOD Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) | PHASE3 | Active Not Recruiting | 2027-08-01 |
| NCT04360265 ↗ | Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA Mucopolysaccharidosis IIIA, MPS IIIA | PHASE3 | Enrolling By Invitation | 2027-08-01 |
| NCT06636071 ↗ | Setrusumab in Pediatric Japanese Subjects With Osteogenesis Imperfecta Osteogenesis Imperfecta | PHASE3 | Active Not Recruiting | 2027-01-01 |
| NCT06617429 ↗ | Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS) Angelman Syndrome | PHASE3 | Active Not Recruiting | 2026-07-01 |
| NCT05768854 ↗ | Setrusumab vs Bisphosphonates in Pediatric Subjects With Osteogenesis Imperfecta Osteogenesis Imperfecta | PHASE3 | Active Not Recruiting | 2025-10-23 |
| NCT05125809 ↗ | Setrusumab vs Placebo for Osteogenesis Imperfecta Osteogenesis Imperfecta | PHASE2/PHASE3 | Active Not Recruiting | 2025-10-20 |
| NCT07511556 ↗ | First-in-human Study of UX016 in GNEM GNE Myopathy | PHASE1/PHASE2 | Not Yet Recruiting | 2028-12-01 |
| NCT03970278 ↗ | Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia Glycogen Storage Disease Type IA, Von Gierke's Disease (GSD Type Ia) | — | Completed | 2025-02-25 |
| NCT04259281 ↗ | A Study of the Safety and Tolerability of GTX-102 in Children With Angelman Syndrome Angelman Syndrome | PHASE1/PHASE2 | Completed | 2025-01-08 |
Source: ClinicalTrials.gov (U.S. NIH) · active studies first.
Notable events (8-K)
Recent filings
| Form | Filed | Period | Accession |
|---|---|---|---|
| 8-K | 2026-05-18 | 2026-05-14 | 0001193125-26-228639 ↗ |
| 10-Q | 2026-05-06 | 2026-03-31 | 0001193125-26-206960 ↗ |
| 8-K | 2026-05-05 | 2026-05-05 | 0001193125-26-206368 ↗ |
| 8-K | 2026-04-02 | 2026-04-02 | 0001193125-26-139084 ↗ |
| 8-K | 2026-03-30 | 2026-03-30 | 0001193125-26-130498 ↗ |
| 8-K | 2026-03-12 | 2026-03-12 | 0001193125-26-103167 ↗ |
| 8-K | 2026-02-23 | 2026-02-23 | 0001193125-26-062568 ↗ |
| 10-K | 2026-02-18 | 2025-12-31 | 0001193125-26-057329 ↗ |
| 8-K | 2026-02-12 | 2026-02-12 | 0001193125-26-048738 ↗ |
| 8-K | 2026-02-03 | 2026-02-03 | 0001193125-26-034474 ↗ |
| 8-K | 2026-01-30 | 2026-01-30 | 0001193125-26-030590 ↗ |
| 8-K | 2026-01-12 | 2026-01-12 | 0001193125-26-009791 ↗ |