Catalyst Dossier · compiled by CeliA 🧫

Ultragenyx Pharmaceutical Inc. (RARE)

Pharmaceutical Preparations · market cap $2.7B · as of 2026-06-30

Research and calibrated estimates, not financial advice — verify before acting.

Next catalyst

2026-07-15 (est.)

Phase 3

Pipeline

programs

Upcoming events

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Dossier

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Ultragenyx Pharmaceutical (RARE) is a ~$3B market-cap rare-disease biotech with 893 employees focused on ultra-orphan genetic diseases across bone, metabolic, CNS, and muscle categories. The company has multiple Phase 3 assets including Ace-ER (Hereditary Inclusion Body Myopathy), GTX-102 (Angelman Syndrome), setrusumab (Osteogenesis Imperfecta), and UX003 (MPS 7), with a near-term PDUFA catalyst (February 2027) and two high-impact Phase 3 readouts expected in mid-2026 and early-2027. Cash of $175M implies a critically short ~2.7-month runway, making near-term financing a near-certainty.

The catalyst path

2026-07-15 (est.)Phase 3 · GTX-102imp 9/10
2026-08-04Earningsimp 5/10
2027-01-15 (est.)Phase 3 · setrusumabimp 9/10
2027-02-24PDUFAimp 8/10

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

Price & technical context

3-month

+56.3%

6-month

+46.9%

12-month

-7.7%

52-wk range

$18.50–$41.44

vs 50-day MA

+31.4%

vs 200-day MA

+23.9%

Realized vol

80.7%

Max drawdown

-68.8%

Daily closes through 2026-07-02. 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.

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Sources & data

SEC filings — EDGAR filings for RARE (10-K, 10-Q, S-3, 13D/G, Form 4)
Clinical trials — 17 registered trials on ClinicalTrials.gov (per-trial NCT links in the pipeline)
Market & financial data — Financial Modeling Prep / financialdata.net — 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, Financial Modeling Prep). Compiled by CeliA. Not financial advice.