Ultragenyx (RARE) Q2 2026 Earnings Call Transcript
Ultragenyx (RARE) reported Q2 2026 revenue of $214 million, led by Crysvita at $156 million. Other revenue included Dojolvi $27 million, Evkeeza $21 million, and Mepsevii $10 million. Full-year 2026 guidance is $730 million to $760 million. Cash was $436 million; net loss was $92 million. PDUFA dates: DTX401 Aug. 23, 2026 and UX111 Sept. 19, 2026.
How this was made
The 30-second read
Why it matters
This earnings call provides fresh, decision-relevant inputs: quarterly results, full-year revenue guidance, cash position, expense trajectory toward profitability, and specific regulatory and clinical catalyst dates (PDUFAs and Phase 3 top-line timing).
Market read
Traders can map probability-weighted outcomes to a near-term calendar (Aug 23 and Sept 19 PDUFAs) while monitoring guidance credibility and the stated path to 2027 profitability.
What to watch
The guidance excludes potential revenue from upcoming product launches, so upside may be more dependent on successful approvals and launch execution than the current run-rate suggests.
Background
Ultragenyx is a rare-disease biotech with multiple late-stage programs and commercial products (notably Crysvita, plus newer launches like Evkeeza and Dojolvi).
Ticker impact
Ultragenyx reported Q2 2026 revenue of $214M, raised/confirmed 2026 guidance to $730M-$760M, and reiterated PDUFA dates for DTX401 and UX111.
Bias toward volatility and upside skew into Aug 23 and Sept 19 PDUFAs if investors view the guidance and cash runway as de-risking; downside risk if regulatory/clinical outcomes disappoint.
The article contains multiple time-specific, company-specific disclosures (earnings, guidance, PDUFA dates, and expected Phase 3 readout) that can directly reprice probability-weighted outcomes and near-term expectations.
Market effects
Reinforces investor focus on rare-disease commercial execution plus gene-therapy regulatory timelines and priority review voucher monetization.
Highlights non-US commercial momentum (Latin America and Turkey Crysvita sales, Europe early access) that can influence regional biotech sentiment.
PDUFA and Phase 3 timing can affect broader risk appetite for gene-therapy and lysosomal storage disease programs globally.
Counterpoint
Despite strong revenue and cash, the call flags setrusumab regulatory uncertainty in the U.K. and prior phase 3 failures, which can keep valuation discounting high.
Key entities
- companyUltragenyx Pharmaceutical
Reported Q2 2026 results, provided 2026 revenue guidance, and reiterated PDUFA dates and upcoming clinical catalysts.
- programDTX401
GSDIa treatment with FDA PDUFA date Aug. 23, 2026 after BLA acceptance in February.
- programUX111
Sanfilippo syndrome type A treatment with FDA PDUFA date Sept. 19, 2026 after resubmitted BLA for accelerated approval.
- programGTX-102
Angelman syndrome Phase 3 ASPIRE study with expected top-line data in Sept or Oct 2026.
- partnerKyowa Kirin Co., Ltd.
Partnership generating Crysvita North America royalties of $94M in Q2 2026.
