← Ultragenyx Pharmaceutical Inc. (RARE) — catalysts, pipeline & price
Catalyst Dossier · compiled by CeliA 🧫
Ultragenyx Pharmaceutical Inc. (RARE)
Pharmaceutical Preparations · market cap $1.5B · as of 2026-07-15
Research and calibrated estimates, not financial advice — verify before acting.
Next catalyst
2026-11-03 (est.)
Earnings
Pipeline
20
programs
Upcoming events
5
next 90d+
Dossier
Ready
cached
Ultragenyx Pharmaceutical (RARE) is a rare/ultra-rare disease biopharmaceutical with a broad multi-modality pipeline spanning bone, metabolic, CNS, and muscle diseases. At ~$33 per share and a $3.27B market cap, the company carries a 2.7-month cash runway, deeply negative net income, and multiple high-impact Phase 3 catalysts imminent in 2026-2027. The setup is a high-binary-risk story: two near-term Phase 3 readouts (GTX-102 in Angelman Syndrome and setrusumab in Osteogenesis Imperfecta) plus a February 2027 PDUFA date will largely define the stock's trajectory.
The catalyst path
- 2026-11-03 (est.)Earningsimp 5/10
- 2027-01-15 (est.)Phase 3 · setrusumabimp 9/10
- 2027-02-24PDUFAimp 8/10
- 2027-08-15 (est.)Phase 3 · Triheptanoinimp 9/10
- 2027-09-15 (est.)Phase 3 · Oral Corticosteroidsimp 9/10
Pipeline
| Asset | Phase | Indication |
|---|---|---|
| SA-ER 500 mg | Phase 2 | GNE Myopathy |
| aceneuramic acid extended-release (Ace-ER) | Phase 3 | Hereditary Inclusion Body Myopathy |
| UX111 | Phase 3 | MPS IIIA |
| Sialic Acid Extended Release (SA-ER) | Phase 2 | GNE Myopathy |
| UX003 | Phase 3 | MPS 7 |
| BPS804 | Phase 2 | Hypophosphatasia |
| steroid regimen | Phase 2 | GSD1 |
| Oral Corticosteroids | Phase 3 | OTC Deficiency |
| Reactive Corticosteroid Taper Regimen | Phase 2 | Ornithine Transcarbamylase (OTC) Deficiency |
| Bisphosphonate | Phase 3 | Osteogenesis Imperfecta |
| GTX-102 | Phase 3 | Angelman Syndrome |
| Standard of Care (SOC) | Phase 2 | Wilson Disease |
| UX007 | Phase 2 | Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) |
| BPS804 20mg/Kg | Phase 2 | Osteopenia |
| Triheptanoin | Phase 3 | Long-chain Fatty Acid Oxidation Disorders (LC-FAOD) |
| Oral prednisolone | Phase 3 | Glycogen Storage Disease Type IA |
| setrusumab | Phase 3 | Osteogenesis Imperfecta, Type I |
| Burosumab | Phase 3 | Hypophosphatemia |
| [6,6-2H2]glucose | Phase 2 | Glycogen Storage Disease Type IA |
| POST Triheptanoin | Phase 2 | Frontal Lobe Hypometabolism |
Price & technical context
3-month
-55.9%
6-month
-31.1%
12-month
-51.0%
52-wk range
$12.88–$36.54
vs 50-day MA
-31.2%
vs 200-day MA
-39.0%
Realized vol
96.2%
Max drawdown
-78.3%
Daily closes through 2026-10-01. Realized vol annualized; max drawdown over ~2 years.
The full dossier is a Pro feature
Thesis, bull & bear, the if-approved scenario, financials, smart-money signals and risks — compiled by CeliA. The catalyst path & pipeline above are free.
Sources & data
- SEC filings — EDGAR filings for RARE (10-K, 10-Q, S-3, 13D/G, Form 4)
- Clinical trials — 20 registered trials on ClinicalTrials.gov (per-trial NCT links in the pipeline)
- Market & financial data — SEC EDGAR (XBRL filings, Form 4), Yahoo Finance, Nasdaq — prices, statements, key metrics, insider & institutional filings.
- Regulatory — openFDA, FDA advisory & PDUFA calendars.
Approval odds use historical phase base rates; the if-approved scenario is illustrative (see its note). Figures trace to the sources above — verify before acting.
Data from CatalystAlert (ClinicalTrials.gov, SEC EDGAR, openFDA, market-data providers). Compiled by CeliA. Not financial advice.