Ultragenyx Pharmaceutical Inc. (RARE) Earnings
Ultragenyx Pharmaceutical Inc. is expected to report next earnings on August 4, 2026 (in NaN days), with a consensus EPS estimate of $-1.24. RARE has beaten EPS estimates in 2 of its last 12 reported quarters (average surprise -20.9% over the last four).
| Report date | EPS est | EPS actual | Surprise | Revenue | Rev. surprise |
|---|---|---|---|---|---|
| May 5, 2026 | $-1.49 | $-1.84 | -23.7% | $136M | -14.0% |
| Feb 12, 2026 | $-1.20 | $-1.29 | -7.5% | $207M | +20.3% |
| Nov 4, 2025 | $-1.23 | $-1.81 | -47.2% | $160M | -19.9% |
| Feb 13, 2025 | $-1.32 | $-1.39 | -5.3% | $165M | +3.9% |
| Aug 1, 2024 | $-1.64 | $-1.52 | +7.3% | $147M | +19.4% |
| May 2, 2024 | $-1.72 | $-2.03 | -18.0% | $109M | -6.2% |
| Feb 15, 2024 | $-1.65 | $-1.52 | +7.9% | $127M | +6.7% |
| Nov 2, 2023 | $-2.08 | $-2.23 | -7.2% | $98M | -10.9% |
| Aug 3, 2023 | $-2.11 | $-2.25 | -6.6% | $108M | +3.3% |
| May 4, 2023 | $-1.97 | $-2.33 | -18.3% | $100M | -4.0% |
| Feb 16, 2023 | $-2.03 | $-2.16 | -6.4% | $103M | -1.7% |
| Nov 2, 2022 | $-2.29 | $-2.43 | -6.1% | $91M | -6.0% |
Source: company filings + earnings calendar. For informational purposes only — not investment advice.
Earnings call summary
Q1 FY2026 · May 5, 2026
AI summary of management’s prepared remarks and analyst Q&A. For informational purposes only — not investment advice.
Management highlights
We're in our 16th year, expected to be transformative with growing revenue and new drug approvals. GTX102 for Angelman syndrome: first patients enrolled in Phase III Aspir study reached day 338 visit, rest completing blinded portion and crossing over. Aurora study expanding GTX102 treatment. Global commercial efforts: generating revenue in over 35 countries, teams commercializing Dolgioli and Metsevi poised to add DTX 401 and US 111. North America, Latin America, Turkey revenue consistent with expectations. DiGiovi study growth continues, over 675 patients in North America on reimbursed therapy, ~300 in Europe. FKESA seeing exceptional growth. Preparing for DTX 401 (PDUFA Aug 23) and UX 111 (PDUFA Sep 19) launches. GTX102 Phase I-II data shows 66 patients on therapy avg 3 years, continuing and improving benefits across domains with favorable safety profile
Guidance
Reaffirm 2026 revenue guidance of $730 - $760 million, CRISFIDA revenue 500 - 520 million, Joel V revenue 100 - 110 million. 2026 combined R&D and SG&A expenses flat to down low single digits vs 2025, 2027 combined R&D and SG&A expenses expected to decrease at least 15% vs 2025
Segment performance
Total revenue for the first quarter of 2026 was $136 million. Chris Vita contributed $93 million, including $39 million from North America, $46 million from Latin America and Turkey, and $8 million from Europe. Dejolbe contributed $18 million. Evkiza contributed $18 million, representing 64% growth over the first quarter of 2025. Mepsevi contributed $7 million. Total operating expenses for the quarter were $305 million, net loss was $185 million, or $1.84 per share. As of March 31, had $534 million in cash, cash equivalents and marketable securities. Net cash used in operations for the quarter was $197 million. 2026 revenue expected to be between $730 and $760 million, CRISFIDA revenue expected to be between 500 and 520 million, Joel V revenue expected to be between 100 and 110 million. 2026 combined R&D and SG&A expenses expected to be flat to down low single digits versus 2025, 2027 combined R&D and SG&A expenses expected to decrease at least 15% versus 2025
Analyst Q&A
Q: Comment on new update for AngelLens, consistency of patients improving on Bayley-4 and MDRI, variability between endpoints for Phase III.
A: Talked about high quality operation for measuring Bayley-4, consistency in Phase II, MDRI being robust.
Q: On GTX102, are you stratifying randomization in Aspire, confidence in protecting against baseline imbalances, timing for Aspire top line data.
A: Stratify by age and cognitive raw score, confident in protecting, not precise on timing but second half.
Q: Clarify on detailed Phase I-II data ahead of Phase III top line, static signal on MDRI, comparison to natural history data set.
A: Probably not see detailed data ahead, MDRI powerful, powered well.
Q: On upcoming gene therapy launches DTX 401 and UX 111, commercial manufacturing and product scale up readiness.
A: Manufacturing going well, building inventory, set up for launch.
Q: Color on timelines for Aurora study data, potential Angelman label ages including adults.
A: Aurora study still enrolling, will collect data, wait to see data.
Q: Color on dose for GTX102, multiple doses, PRV monetization and profitability sustainability.
A: Vast majority on 14 mg, plan to label on that, baked PRV monetization at over $100 million each, plan to continue grow profitability.
Q: Ahead of Phase III Angel Lens data, path forward if MDRI hits but Bayley IV doesn't.
A: Still consider positive study, two ways to succeed.
Q: Latest Angelman cut Phase 1-2 data, cohort A and B vs cohorts 4 and Z.
A: Cohort A and B is primary driver, replicable with phase three.
Q: Contribution of caregiver input to Bayley-4 cognition in Phase I-II, change over time.
A: FDA doesn't want caregiver input in Bayley-4 primary endpoint, phase one, two used caregiver input.
Q: Expectations on sham performance in Aspire study, other surrogate neurodevelopmental indications, profitability assumptions from gene therapy programs.
A: Sham not expected to have effect, not looked at all studies, profitability assumptions include continued growth.
Q: GTX 102 program, reasons for discontinuations, visibility on Chris Vita going forward.
A: Discontinuations due to burden of study participation, confident in Chris Vita due to underlying demand.
Q: Long-term extension data, assessment of metrics for ASPIRE trial Bayley-4 cognition powering, clinically meaningful delta.
A: Long-term shows positive improvements, confident in powering, 10 points well above MID.
Q: UX111 resubmission, requests for information post-acceptance.
A: Routine discussions, excited about program.
Q: Relative conviction on Bailey's four cognition vs MDRI, reallocation of alpha, OI program.
A: MDI more powerful, no need to reallocate more alpha, OI program continues until decision.
Q: Cadence of commercial rollout for PDUFA dates, discontinuation rates in commercial setting.
A: Planning launch, expect to manage reimbursement, discontinuation rates not big issue, will take care of patients