Ultragenyx Pharmaceutical Inc Dossier - Source-backed chat

Ultragenyx Pharmaceutical Inc

RARENASDAQ

SectorHealth Care

IndustryBiotechnology

Beta (adjusted)0.53

Intrinsic Value

Insufficient data for a value estimateNot enough reliable inputs to publish a fair value for this company yet.

Market Price $15.03Price as of 30 Sep 2026

Data confidenceNot applicable

Market Cap $1.5B

Enterprise Value $2.6B

Shares Outstanding 102.5M diluted

Moat Rating None

Next Earnings Date3 Nov 2026

All prices and values are periodic snapshots, not live quotes. Source dates are shown for reference.

Thesis

Street consensus, scenarios, merits, risks, and invalidation triggers

AI-assisted

Thesis Summary

Ultragenyx Pharmaceutical Inc. represents a compelling buy-side opportunity in the rare disease biotechnology sector. The company's investment thesis is anchored by a robust, growing commercial portfolio (led by Crysvita and Dojolvi) that provides a stable financial foundation, combined with a highly anticipated, late-stage pipeline. The year 2026 serves as a critical inflection point with two near-term gene therapy PDUFA dates (DTX401 in August and UX111 in September) and pivotal Phase 3 data for GTX-102 in Angelman syndrome expected in the second half of the year. Successful execution of these milestones is projected to accelerate top-line growth and solidify the company's guided pathway to profitability by 2027.

This section is the 12-month view: analyst targets and the probability-weighted scenarios built from them. The intrinsic value at the top of this page answers a different question: what the business is worth today.

Street view · analyst 12-month targets19 analysts · as of 18 Aug 2026

Low · most bearish analyst$27.00

Mean target$52.84

High · most bullish analyst$96.00

Our research · scenarios, merits, risks, and invalidation triggersResearch as of 18 Sep 2026

Scenarios · 12-month scenario targetsAnchored at research date

Bear CaseDownside scenario

$27.0015%

The FDA issues Complete Response Letters (CRLs) or demands additional clinical trials for DTX401 or UX111, delaying commercialization. Furthermore, the GTX-102 Phase 3 trial yields ambiguous efficacy data or unexpected safety signals (such as severe lower extremity weakness seen in early development), severely damaging its commercial outlook. Base business growth slows due to payer friction or ordering disruptions in Latin America, forcing the company to raise dilutive capital and push back its 2027 profitability target.

Base CaseCentral scenario

$52.8460%

Matches the consensus mean

Ultragenyx successfully secures FDA approvals for DTX401 and UX111 within the expected regulatory timeframes, and prepares for commercial launches in late 2026/early 2027. The GTX-102 Phase 3 trial meets its primary endpoints with a manageable safety profile, supporting a planned BLA submission. Base commercial revenues for Crysvita and Dojolvi land within the guided ranges of $500M-$520M and $100M-$110M respectively, keeping the company on track for its 2027 profitability target.

Bull CaseUpside scenario

$96.0025%

The FDA approves both DTX401 and UX111 on or before their respective PDUFA dates in Q3 2026, triggering rapid commercial launches. Simultaneously, the GTX-102 Phase 3 Aspire trial delivers highly statistically significant and clinically meaningful results, establishing a clear first-mover advantage in the multi-billion dollar Angelman syndrome market. Commercial execution of Crysvita and Dojolvi exceeds the high end of guidance, accelerating the timeline to positive cash flow and profitability ahead of 2027.

Scenarios are anchored to street consensus at the research date, with our probabilities and rationale.

Key Investment Merits

Diversified commercial base with established rare disease therapies (Crysvita, Dojolvi, Evkeeza, Mepsevii) generating predictable, high-margin revenue.
Two late-stage gene therapy candidates (DTX401 and UX111) with assigned PDUFA dates in Q3 2026, offering near-term commercial catalysts.
GTX-102 represents a potential blockbuster first-in-class disease-modifying therapy for Angelman syndrome, with pivotal Phase 3 data expected in H2 2026.
Clear management commitment to financial discipline, targeting flat-to-down operating expenses in 2026 and a defined path to profitability in 2027.

Key Investment Risks

High clinical and regulatory binary risk associated with the upcoming gene therapy approvals and Phase 3 readouts.
History of substantial cash burn and shareholder dilution to fund the late-stage pipeline.
Commercial execution risks in ultra-rare disease markets, including small patient pools, complex diagnostics, and payer reimbursement friction.
Potential volatility in international revenues, particularly due to variable ordering patterns in Latin America.

Thesis Invalidation Triggers

FDA rejection or significant regulatory delay for either DTX401 or UX111.
Failure of the GTX-102 Phase 3 Aspire study to meet its primary efficacy endpoints or the emergence of unacceptable safety concerns.
A downward revision of the 2026 revenue guidance or failure to achieve the guided 2027 profitability pathway.
Significant unexpected cash burn requiring highly dilutive equity financing.

All scenarios are estimates and subject to change. Past performance is not indicative of future results.

Quality Pillars Members

This section is available to registered members. Create a free account or sign in to unlock the full breakdown.

Explore this dossier

ValuationIntrinsic value, the six-method breakdown, peer medians, and your assumptions sandbox.Financial SnapshotRevenue, profitability, returns, balance sheet, dividends, and the filing-level detail.Qualitative AnalysisBusiness overview, strategic initiatives, and mergers, acquisitions & partnerships.Outlook & Key DatesForward estimates, reporting calendar, and the monitoring framework.