Ultragenyx Pharmaceutical Inc. (RARE) Q4 2025 Earnings Call Transcript
Access earnings results, analyst expectations, report, slides, earnings call, and transcript.
Overview
The earnings call summary and Q&A session reveal several positive indicators: strong revenue guidance for 2025, a clear path to profitability by 2027, and a solid financial position with significant nondilutive capital. Additionally, the company is preparing for potential product launches, and management demonstrates confidence in meeting regulatory criteria for ongoing studies. Although some uncertainties remain, such as the timeline for the IRL resubmission, the overall sentiment is positive, suggesting a likely stock price increase.
Key Financial Performance
Total Revenue for 2025 $673 million, representing 20% growth over 2024. Reasons for growth include strong performance across all products and contributions from over 35 countries.
Crysvita Revenue for 2025 $481 million, representing 17% growth over 2024. Reasons for growth include strong demand in North America, Latin America, Turkey, and Europe.
Dojolvi Revenue for 2025 $96 million, representing 9% growth over 2024. Reasons for growth include steady demand and regulatory wins in regions like Kuwait and the U.K.
Evkeeza Revenue for 2025 $59 million, representing 84% growth over 2024. Reasons for growth include increased demand following launches in territories outside the U.S. and reimbursement approvals in major markets.
Mepsevii Revenue for 2025 $37 million. No year-over-year growth percentage mentioned, but it continues to treat patients in an ultra-rare indication.
Total Operating Expenses for 2025 $1.1 billion for combined R&D and SG&A expenses, with cost of sales at $109 million. Reasons include investments in clinical and manufacturing activities, as well as early-stage research.
Net Loss for 2025 $575 million or $5.83 per share. Reasons include high operating expenses and investments in pipeline development.
Cash and Equivalents as of December 31, 2025 $738 million. No year-over-year change or reasons mentioned.
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Operating Highlights
UX111 for MPS IIIA: Presented updated data showing sustained and significant improvements in neurologic endpoints and biomarkers. Resubmitted BLA to FDA but received an incomplete response letter requiring additional documentation.
DTX401 for glycogen storage disease type Ia: Completed rolling BLA submission; PDUFA action date expected in Q3 2026.
GTX-102 for Angelman syndrome: Phase III Aspire study ongoing; data expected in H2 2026.
UX701 for Wilson disease: Completed enrollment in dosing cohort; data from all cohorts expected later in 2026.
Global commercial reach: Treatments now available in over 35 countries, contributing to 20% year-over-year revenue growth in 2025.
Crysvita: Generated $481 million in revenue, with strong growth in Latin America and Turkey. Variability in revenue due to ordering patterns in Brazil.
Dojolvi: Achieved regulatory wins in Kuwait and the UK; conditional approval in Japan with full approval expected in H2 2026.
Evkeeza: Expanded to 20 countries with 350 patients; registered in Saudi Arabia and launched in Japan.
Strategic restructuring: Implemented a 10% workforce reduction (130 employees) to align resources with high-impact opportunities and target profitability by 2027.
Financial performance: 2025 revenue reached $673 million (20% growth). Operating expenses included $1.1 billion in R&D and SG&A, with a net loss of $575 million.
Profitability target: Aiming for profitability by 2027 through restructuring and focused investments in high-value areas.
Pipeline focus: Prioritizing launches of UX111, DTX401, and GTX-102 while reducing early-stage research and manufacturing costs.
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Risk or Challenges
FDA Incomplete Response Letter for UX111 BLA: The FDA issued an incomplete response letter requiring additional details within supportive documentation on the CMC CRL responses. This could delay the approval and launch of UX111, impacting the company's ability to bring this therapy to market and generate revenue.
Strategic Restructuring Plan: The company announced a 10% workforce reduction, impacting approximately 130 full-time employees. While aimed at cost reduction, this could lead to operational challenges and potential disruptions in ongoing projects.
Revenue Variability in Latin America: The company expects variability in revenue due to uneven ordering patterns, particularly in Brazil. This could lead to financial unpredictability and impact revenue growth projections.
High Operating Expenses: The company reported combined R&D and SG&A expenses of $1.1 billion in 2025, contributing to a net loss of $575 million. High expenses could strain financial resources and delay profitability goals.
Regulatory and Manufacturing Challenges: The need for additional supportive documentation for UX111 and the completion of clinical and manufacturing spend on multiple Phase III studies highlight potential regulatory and operational hurdles.
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Guidance & Outlook
Revenue Expectations for 2026: Total revenue in 2026 is expected to be between $730 million and $760 million, representing 8% to 13% growth over 2025. Crysvita revenue is expected to be between $500 million and $520 million, reflecting growing global demand but partially offset by expected timing of ordering patterns in Brazil. Dojolvi revenue is expected to be between $100 million and $110 million.
Expense Projections for 2026 and 2027: Combined R&D and SG&A expenses for 2026 are expected to be flat to down low single digits versus 2025. For 2027, R&D expenses are expected to decrease by 38% from 2025 levels, driven by the completion of clinical and manufacturing spend on multiple Phase III studies and reduction of early-stage research efforts. SG&A expenses in 2027 are expected to increase to support new product launches and existing approved products. Combined R&D and SG&A expenses are expected to decrease at least 15% in 2027 versus 2025.
Profitability Target: The company aims to achieve profitability by 2027, supported by strategic restructuring, expense reductions, and growth in commercial and new product launches.
Product Launches and Approvals: The company plans to commercialize 3 additional treatments over the next 2 years, including UX111, DTX401, and GTX-102. DTX401 is expected to have a PDUFA action date in Q3 2026. UX111 BLA resubmission is underway following an incomplete response letter from the FDA. Aspire Phase III data for GTX-102 is expected in the second half of 2026.
Market Expansion: The company is expanding its geographic reach for existing products and preparing for broader commercialization opportunities in regions like Japan and APAC. Evkeeza is expected to grow meaningfully, with recent registration in Saudi Arabia and ongoing commercialization in Japan.
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Shareholder Return Plan
The selected topic was not discussed during the call.
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Key Q&A
Q:Why did the company choose Bayley-4 cognition as the primary endpoint for the Phase III Angelman study instead of expressive communication?
A:The company believes Bayley cognition is a core and important function in Angelman patients, demonstrating substantial improvement. Expressive communication, while important, is more heterogeneous and complex to develop. The study also includes a Multi-Domain Responder Index to assess cognition, communication, sleep, behavior, and motor function.
Q:What percentage of patients from Phase III have opted to roll over into the long-term extension portion of the study?
A:The company reported very few dropouts, with a high retention rate similar to Phase I and II, as parents see the treatment as an opportunity for their children to grow and develop.
Q:What is the patient baseline profile for the Phase III Angelman study, and how does it compare to Phase I/II?
A:The baseline data for Phase III is reflective of the Phase I/II expansion trial, which included patients from multiple countries. The company is confident in the comparability of the data.
Q:What are the expectations for cognition changes in the treatment and control arms of the Angelman study?
A:The company expects minimal cognitive change (1 point or less) in the control group based on natural history and randomized studies. They have implemented measures to ensure consistent assessments and believe the study size is appropriate to manage variation.
Q:How does Ultragenyx plan to achieve profitability by 2027?
A:The plan includes double-digit growth from current products, contributions from upcoming launches, cost reductions in R&D and SG&A, and leveraging $735 million in cash and 2 PRVs. Expenses in 2026 are expected to be flat or slightly down, with a 15% reduction in 2027.
Q:What supports the claim that GTX-102 is the most potent ASO in development?
A:The claim is based on preclinical data showing substantial knockdown of the antisense transcript and induction of UBE3A expression in nonhuman primates at lower doses compared to competitors. Clinical data also supports this potency with comparable cognitive improvements at lower doses.
Q:What is the status of the DTX301 program for OTC?
A:The Phase III study is ongoing, and data on the ammonia endpoint is expected to be released this year.
Q:What issues were raised in the IRL for 111, and how is the company addressing them?
A:The issues were known to both the FDA and the company, involving SOP changes and CAPA agreements. The company is providing full documentation to address these issues in a resubmission for the BLA.
Q:How does the restructuring impact the gene therapy pipeline?
A:The restructuring focuses on advancing late-stage programs to approval while maintaining a diversified pipeline. Early-stage gene therapy programs are on hold, but the company plans to continue work in gene therapy alongside other modalities.
Q:Is there evidence supporting the hypothesis that setrusumab-treated patients in the ORBIT study had increased fracture rates due to higher physical activity?
A:The company is evaluating data but has not provided definitive evidence. Treated patients reported improved activity, function, and less bone pain, consistent with the hypothesis.
Q:What is the regulatory bar for the Angelman study, and how confident is the company in meeting it?
A:The regulatory bar involves demonstrating statistically significant, clinically meaningful changes in Bayley cognition and the Multi-Domain Responder Index. The company is confident in meeting these criteria based on prior data and discussions with the FDA.
Q:What is the timeline for monetizing PRVs, and how does it impact profitability?
A:The company plans to monetize PRVs promptly, potentially through pre-monetization agreements. PRVs are modeled at just over $100 million each, contributing to the path to profitability.
Q:What is the status of the UX701 program for Wilson disease?
A:The timeline for data release is less precise, as the company is monitoring patient progress and ensuring sufficient time for standard of care withdrawal. The program is continuing as planned.
Q:What are the expected pricing and launch trajectory for GSDIa and MPS IIIA treatments?
A:GSDIa pricing is expected in the $1-2 million range with a steady demand trajectory. MPS IIIA pricing is expected in the $2-4 million range with more urgent demand.
Q:What measures are in place to ensure consistent Bayley-4 assessments in the Aspire study?
A:The company has a dedicated endpoint development strategy group, comprehensive site training, and centralized testers for Bayley scoring to ensure quality and consistency.
Q:What is the status of the OTC Phase III study, and what are the expectations for success?
A:The study is ongoing, focusing on ammonia control as the primary endpoint. The company views it as an important program but not a top priority.
Q:What is the company's reaction to the FDA's decision on REGENXBIO's CRL and its implications for rare disease biomarkers?
A:The company believes biomarkers are critical for measuring disease and efficacy. While the FDA appears resistant to biomarkers, the company continues to support their use and emphasizes the need for FDA action to support rare disease treatments.
Q:Review of Unclear Management Responses
A:Management avoided providing a clear answer on the correlation between increased physical activity and fracture rates in the setrusumab-treated arm of the ORBIT study, stating that they are still evaluating the data. Additionally, they did not provide a specific timeline for the resubmission of the IRL for 111, only stating that they are working diligently on it.
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Earnings Word Cloud
The most frequently occurring keywords in this quarter's earning call
America Brazil
Aspire
CRL
CSF HS
Corporate
Director
Dojolvi
Executive VP
MPS IIIA
Officer Harris
RD SGA
UX DTX
WORLDSymposium
ability
age stage
area
base
contribution
demand timing
documentation BLA
financials
infrastructure
launch product
life therapy
market patient
measure
product Japan
product launch
reduction
restructuring plan
spend
stage disease
stage research
term value
timing pattern
type Ia
RARE Transcript
Ultragenyx Pharmaceutical Inc. (RARE) Q4 2025 Earnings Call Transcript
Positive2-13
The earnings call summary and Q&A session reveal several positive indicators: strong revenue guidance for 2025, a clear path to profitability by 2027, and a solid financial position with significant nondilutive capital. Additionally, the company is preparing for potential product launches, and management demonstrates confidence in meeting regulatory criteria for ongoing studies. Although some uncertainties remain, such as the timeline for the IRL resubmission, the overall sentiment is positive, suggesting a likely stock price increase.
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