On May 6, 2025 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, reported its financial results for the quarter ended March 31, 2025 and reaffirmed its financial guidance for 2025 (Press release, Ultragenyx Pharmaceutical, MAY 6, 2025, View Source [SID1234652598]).
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"In the first quarter, our commercial team continued expanding our base of revenue around the world, while we also continued to make successful progress for our next potential launch with the review of our first gene therapy BLA for the treatment of Sanfilippo syndrome (MPS IIIA)," said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. "Patients in the UX143 Phase 3 Orbit and Cosmic studies have now been on therapy for at least 12 months and will support the interim analyses in mid-2025. We continue to hear encouraging feedback from investigators in the Phase 2 portion of Orbit, some with patients on therapy for over 2 years, who noted an excellent risk benefit profile during the open-label Phase 2 portion of the study."
First Quarter 2025 Selected Financial Data Tables and Financial Results
Revenues (dollars in thousands), (unaudited)
Three Months Ended March 31,
2025 2024
Crysvita
Product sales – Latin America and Türkiye $ 55,080 $ 36,241
Royalty revenue – U.S. and Canada 40,853 40,402
Royalty revenue – Europe 6,932 5,942
Total Crysvita Revenue 102,865 82,585
Dojolvi 17,009 16,362
Evkeeza 11,031 3,275
Mepsevii 8,387 6,611
Total revenues $ 139,292 $ 108,833
Total Revenues
Ultragenyx reported $139 million in total revenue for the first quarter of 2025, which represents 28% growth compared to the same period in 2024. First quarter 2025 Crysvita revenue was $103 million, which represents 25% growth compared to the same period in 2024. This includes product sales of $55 million from Latin America and Türkiye, which represents 52% growth compared to the same period in 2024. Dojolvi revenue in the first quarter 2025 was $17 million. Evkeeza revenue in the first quarter 2025 was $11 million as we continue to launch in the Ultragenyx territories outside of the United States.
Selected Financial Data (dollars in thousands, except per share amounts), (unaudited)
Three Months Ended March 31,
2025 2024
Total revenues $ 139,292 $ 108,833
Operating expenses:
Cost of sales 28,662 17,533
Research and development 165,772 178,487
Selling, general and administrative 87,797 78,160
Total operating expenses 282,231 274,180
Net loss $ (151,080 ) $ (170,684 )
Net loss per share, basic and diluted $ (1.57 ) $ (2.03 )
Operating Expenses
Total operating expenses for the first quarter of 2025 were $282 million, including non-cash stock-based compensation of $40 million.
Net Loss
For the first quarter of 2025, Ultragenyx reported net loss of $151 million, or $1.57 per share basic and diluted, compared with a net loss for the first quarter of 2024 of $171 million, or $2.03 per share basic and diluted.
Cash Balance and Net Cash Used in Operations
Cash, cash equivalents, and marketable debt securities were $563 million as of March 31, 2025, which reflects cash payments made during the first quarter of 2025 of $30 million for a GTX-102 Phase 3 study milestone and $15 million for an Evkeeza sales milestone, both achieved in the fourth quarter of 2024. Net cash used in operations for the quarter ended March 31, 2025 was $166 million and includes the payment of annual bonuses and a $30 million cash payment for the GTX-102 development milestone noted above.
2025 Financial Guidance
Ultragenyx reaffirmed its financial guidance for 2025. Revenues are expected to grow approximately 14-20% compared to 2024. The company will continue to prioritize expense management, focusing its investments on the execution of multiple upcoming commercial launches and advancing multiple Phase 3 programs. Together, this is expected to lead to a reduction in 2025 net cash used in operations compared to 2024.
For the full year 2025:
Total revenue to be in the range of $640 million to $670 million
Crysvita revenue to be in the range of $460 million to $480 million
Dojolvi revenue to be in the range of $90 million to $100 million
Recent Updates and Clinical Milestones
UX143 (setrusumab) monoclonal antibody for osteogenesis imperfecta (OI): Next interim analysis for Phase 3 Orbit and Cosmic studies is in mid-2025
Patients continue dosing in the ongoing Phase 3 Orbit and Cosmic clinical trials, which evaluate setrusumab in pediatric and young adult patients with OI. The randomized, placebo-controlled Phase 3 portion of the Orbit study is progressing toward a second interim analysis (IA2) in mid-2025 or a final analysis in the fourth quarter 2025. Conduct of the study is going well and patient safety in the Phase 3 is consistent with the Phase 2. Patients in the Cosmic study also are continuing to be treated with either setrusumab or intravenous bisphosphonates (IV-BP) therapy and will be evaluated in parallel with the Orbit interim analysis. If Orbit progresses to full study completion in the fourth quarter of 2025, Cosmic will also continue to a data readout, to align with the Orbit readout without spending alpha at the mid-year interim assessment.
GTX-102 an antisense oligonucleotide for Angelman syndrome: Phase 3 study is enrolling, expect completion in second half of 2025
Enrollment in the global Phase 3 Aspire study began in December 2024 and is expected to enroll approximately 120 children ages four to 17 with Angelman syndrome with a genetically confirmed diagnosis of full maternal UBE3A gene deletion. Phase 3 study site startup and enrollment are going well. Participants are randomized 1:1 to receive GTX-102 by intrathecal injection via lumbar puncture or to the sham comparator group during the 48-week primary efficacy analysis period. The primary endpoint is improvement in cognition assessed by Bayley-4 cognitive raw score, and the key secondary endpoint (with a 10% allocation of alpha) is the Multi-domain Responder Index (MDRI) across the five domains of cognition, receptive communication, behavior, gross motor function, and sleep. Enrollment in the Phase 3 Aspire study is expected to complete in the second half of 2025.
The Phase 2/3 Aurora study, which will evaluate GTX-102 in other Angelman syndrome genotypes and ages, is expected to initiate in 2025.
UX111 AAV gene therapy for Sanfilippo syndrome type A (MPS IIIA): BLA accepted; U.S. Food and Drug Administration (FDA) granted Priority Review with a Prescription Drug User Fee Act (PDUFA) action date of August 18, 2025
In February 2025, the FDA accepted for review the BLA seeking accelerated approval for UX111. The FDA granted the BLA Priority Review with a PDUFA action date of August 18, 2025 and also informed the company that they are not currently planning to hold an advisory committee meeting to discuss this application. The FDA review currently continues to progress as expected with a mid-cycle review recently completed and multiple clinical and manufacturing inspections scheduled and underway. Based on available information, the company continues to expect the FDA to meet its stated timeline with a PDUFA decision on August 18, 2025 that would be followed by a potential launch in the second half of 2025.
DTX401 AAV gene therapy for Glycogen Storage Disease Type Ia (GSDIa): BLA submission expected in mid-2025
As previously disclosed by Ultragenyx in May 2024, the Phase 3 GlucoGene study for the treatment of patients aged eight years and older with GSDIa achieved its primary endpoint, demonstrating that treatment with DTX401 resulted in a statistically significant and clinically meaningful reduction in daily cornstarch intake compared with placebo at Week 48. At Week 48, patients entered a 48-week Crossover Period where patients previously treated with placebo were treated with DTX401. During the Crossover Period, patients demonstrated even greater reductions in total daily cornstarch at their last visit compared to baseline in both the ongoing DTX401 group (-60%) and the Crossover Placebo to DTX401 group (-64%). Glycemic control was maintained in patients treated with DTX401 despite significant reductions in daily cornstarch intake. DTX401 has demonstrated a consistent and acceptable safety profile with no new safety signals identified as of the data cut-off.
Process Performance Qualification (PPQ) runs have been successfully completed in the Company’s U.S. gene therapy manufacturing facility in preparation for upcoming submissions. The latest clinical results and data from PPQ runs will be included as part of a BLA submission expected in mid-2025.
UX701 AAV gene therapy for Wilson Disease: Phase 1/2/3 study ongoing; Cohort 4 enrollment began with completion expected in second half of 2025
Enrollment has begun in the fourth cohort evaluating a 4.0e13 GC/kg dose in the ongoing, dose-finding, stage of the pivotal Cyprus2+ study of UX701 for the treatment of Wilson disease. The company expects to enroll five patients in Cohort 4 who will receive immunomodulation therapy with rituximab and tacrolimus, in addition to the prophylactic oral corticosteroid regimen patients in Cohorts 1 through 3 received, prior to being dosed with UX701. Enrollment in Cohort 4 is expected to complete in the second half of 2025.
The protocol for the pivotal, Stage 2 portion of Cyprus2+ was amended to a 52-week, randomized, open-label, active-controlled design, evaluating the safety and efficacy of UX701 following dose selection in Stage 1. The Stage 2 primary endpoints include comparisons between the UX701 and active control groups of change in 24-hour urinary copper from Baseline at Week 52 and percent reduction in SOC medication by Week 52. Enrollment in this stage is expected following dose selection in Stage 1.
Conference Call and Webcast Information
Ultragenyx will host a conference call today, Tuesday, May 6, 2025, at 2 p.m. PT/5 p.m. ET to discuss the first quarter 2025 financial results and provide a corporate update. The live and replayed webcast of the call will be available through the company’s website at View Source The replay of the call will be available for three months.