Ultragenyx Reports Preliminary 2024 Revenue, Financial Guidance for 2025, Pipeline Updates, and 2025 Milestones
Ultragenyx Pharmaceutical (NASDAQ: RARE) reported preliminary 2024 revenue of $555-560 million, exceeding guidance, with Crysvita revenue of $405-410 million and Dojolvi revenue of $87-89 million. The company projects 2025 total revenue of $640-670 million.
Key pipeline updates include: UX143 Phase 3 Orbit study for osteogenesis imperfecta progressing to second interim analysis in mid-2025; GTX-102 Phase 3 Aspire study for Angelman syndrome expected to complete enrollment in second half of 2025; BLA submission for UX111 gene therapy for Sanfilippo syndrome with PDUFA decision expected in second half of 2025; and DTX401 BLA filing for Glycogen Storage Disease Type Ia planned for mid-2025.
The company ended 2024 with approximately $745 million in cash and investments, expecting 14-20% revenue growth in 2025 with decreased net cash usage in operations.
Ultragenyx Pharmaceutical (NASDAQ: RARE) ha riportato un fatturato preliminare per il 2024 di $555-560 milioni, superando le previsioni, con un fatturato per Crysvita di $405-410 milioni e un fatturato per Dojolvi di $87-89 milioni. L'azienda prevede un fatturato totale per il 2025 di $640-670 milioni.
Le principali novità della pipeline includono: lo studio Orbit di Fase 3 UX143 per l'osteogenesi imperfetta, che sta progredendo verso la seconda analisi intermedia a metà 2025; lo studio Aspire di Fase 3 GTX-102 per la sindrome di Angelman, che si prevede completerà l'arruolamento nella seconda metà del 2025; la presentazione BLA per la terapia genica UX111 per la sindrome di Sanfilippo, con una decisione PDUFA attesa nella seconda metà del 2025; e la presentazione BLA per DTX401 per la malattia di accumulo di glicogeno di tipo Ia, prevista per metà 2025.
L'azienda ha chiuso il 2024 con circa $745 milioni in contante e investimenti, prevedendo una crescita del fatturato del 14-20% nel 2025, con una diminuzione dell'uso netto di contante nelle operazioni.
Ultragenyx Pharmaceutical (NASDAQ: RARE) reportó ingresos preliminares para 2024 de $555-560 millones, superando las expectativas, con ingresos de Crysvita de $405-410 millones y de Dojolvi de $87-89 millones. La compañía proyecta ingresos totales de $640-670 millones para 2025.
Las actualizaciones clave de la pipeline incluyen: estudio Orbit Fase 3 UX143 para osteogénesis imperfecta que avanza hacia el segundo análisis intermedio a mediados de 2025; estudio Aspire Fase 3 GTX-102 para el síndrome de Angelman que se espera complete su inscripción en la segunda mitad de 2025; presentación de BLA para la terapia génica UX111 para el síndrome de Sanfilippo, con decisión PDUFA esperada en la segunda mitad de 2025; y presentación de BLA para DTX401 para la enfermedad de almacenamiento de glucógeno tipo Ia, planificada para mediados de 2025.
La compañía terminó 2024 con aproximadamente $745 millones en efectivo e inversiones, esperando un crecimiento en los ingresos del 14-20% en 2025, con una reducción en el uso de efectivo neto en operaciones.
Ultragenyx Pharmaceutical (NASDAQ: RARE)는 2024년 예비 매출이 $555-560 백만 달러로 가이던스를 초과했다고 보고했습니다. Crysvita 매출은 $405-410 백만 달러, Dojolvi 매출은 $87-89 백만 달러입니다. 회사는 2025년 총 매출을 $640-670 백만 달러로 예상하고 있습니다.
주요 파이프라인 업데이트에는: 2025년 중반에 두 번째 중간 분석으로 진행 중인 골형성부전증에 대한 UX143 3상 Orbit 연구; 2025년 하반기에 등록 완료가 예상되는 앤젤맨 증후군에 대한 GTX-102 3상 Aspire 연구; 산필리포 증후군에 대한 UX111 유전자 치료의 BLA 제출로 2025년 하반기에 PDUFA 결정이 예상됨; 그리고 DTX401의 BLA 제출이 2025년 중반에 계획되어 있습니다.
회사는 2024년을 약 $745 백만 달러의 현금 및 투자 자산과 함께 마감했으며, 2025년에는 14-20%의 매출 성장을 기대하고 있습니다. 운영에서의 순현금 사용이 감소할 것으로 예상됩니다.
Ultragenyx Pharmaceutical (NASDAQ: RARE) a annoncé des revenus provisoires pour 2024 de 555 à 560 millions de dollars, dépassant les prévisions, avec des revenus de Crysvita de 405 à 410 millions de dollars et de Dojolvi de 87 à 89 millions de dollars. La société projette des revenus totaux de 640 à 670 millions de dollars pour 2025.
Les mises à jour clés de la pipeline incluent : l'étude Orbit de Phase 3 UX143 pour l'ostéogenèse imparfaite progressant vers la deuxième analyse intermédiaire à la mi-2025; l'étude Aspire de Phase 3 GTX-102 pour le syndrome d'Angelman, qui devrait achever son recrutement au second semestre 2025; soumission de BLA pour la thérapie génique UX111 pour le syndrome de Sanfilippo, avec une décision PDUFA attendue au second semestre 2025; et dépôt de BLA pour DTX401 pour la maladie de stockage du glycogène de type Ia prévu pour la mi-2025.
La société a terminé l'année 2024 avec environ 745 millions de dollars en espèces et en investissements, prévoyant une croissance des revenus de 14 à 20 % en 2025 avec une diminution de l'utilisation nette de liquidités dans les opérations.
Ultragenyx Pharmaceutical (NASDAQ: RARE) berichtete von ersten Einnahmen für 2024 in Höhe von 555-560 Millionen US-Dollar, was die Erwartungen übertrifft, mit Einnahmen von Crysvita in Höhe von 405-410 Millionen US-Dollar und von Dojolvi in Höhe von 87-89 Millionen US-Dollar. Das Unternehmen erwartet für 2025 einen Gesamtumsatz von 640-670 Millionen US-Dollar.
Wichtige Neuigkeiten zur Pipeline umfassen: die Phase 3 Orbit-Studie UX143 für Osteogenesis imperfecta, die zur zweiten Zwischenanalyse Mitte 2025 fortschreitet; die Phase 3 Aspire-Studie GTX-102 für das Angelman-Syndrom, die voraussichtlich in der zweiten Hälfte von 2025 abgeschlossen wird; die Einreichung einer BLA für die Gentherapie UX111 für das Sanfilippo-Syndrom mit einer PDUFA-Entscheidung, die in der zweiten Hälfte von 2025 erwartet wird; und die BLA-Einreichung für DTX401 zur Glykogenspeicherkrankheit Typ Ia, die für Mitte 2025 geplant ist.
Das Unternehmen schloss das Jahr 2024 mit etwa 745 Millionen US-Dollar in Bar und Investitionen ab und erwartet ein Umsatzwachstum von 14-20% im Jahr 2025 mit einem Rückgang des Netto-Cash-Verbrauchs in den Operationen.
- 2024 revenue exceeded guidance at $555-560M, representing 29% YoY growth
- Crysvita revenue grew 24% YoY to $405-410M
- Dojolvi revenue increased 25% YoY to $87-89M
- Strong cash position of $745M as of December 2024
- Projected 2025 revenue growth of 14-20%
- Multiple late-stage pipeline developments with potential launches of 3-4 new therapies
- Expected decline in net cash from operations in 2025
- Most key clinical trial results and regulatory decisions pending in 2025
Insights
The preliminary 2024 results and 2025 guidance reveal strong commercial execution, with 2024 revenue of
Key commercial products Crysvita and Dojolvi both exceeded guidance, with
The robust late-stage pipeline positions Ultragenyx for significant value creation, with potential for 3-4 new therapy launches in the near term. Critical catalysts in 2025 include:
- UX143 Phase 3 interim analysis for osteogenesis imperfecta
- GTX-102 Phase 3 enrollment completion for Angelman syndrome
- UX111 PDUFA decision for Sanfilippo syndrome
- DTX401 BLA submission for GSDIa
The diversification across multiple rare disease indications and modalities (biologics, gene therapy, antisense oligonucleotides) reduces development risk. The potential to reach 8-9 approved products within a decade demonstrates strong pipeline execution capability.
In the competitive rare disease space, Ultragenyx's commercial performance and pipeline progress stand out. The company's ability to exceed revenue guidance while advancing multiple late-stage programs demonstrates operational excellence. The anticipated launch sequence could transform Ultragenyx from a four-product to an eight-product company, significantly expanding its market presence.
The
Preliminary 2024 total revenue of
2025 expected total revenue guidance of
UX143 (setrusumab) Phase 3 Orbit study for osteogenesis imperfecta progressing to second interim analysis in mid-2025
GTX-102 Phase 3 Aspire study for Angelman syndrome expected to complete enrollment in second half of 2025
NOVATO, Calif., Jan. 12, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultrarare genetic diseases, today reported preliminary unaudited 2024 revenue results, cash and investments at year end 2024, and provided financial guidance for 2025. The company also provided key program updates including that the UX143 (setrusumab) Phase 3 Orbit study is progressing with the second interim analysis in mid-2025.
“In 2024 we grew our business with four products in five indications globally, exceeding the updated revenue guidance we provided in August, and continuing our path toward profitability,” said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. “In 2025, we will continue to expand our commercial base of business while we also prepare for the potential launch of our first gene therapy, in Sanfilippo syndrome, and to file a BLA for our second gene therapy, in Glycogen Storage Disease Type Ia. We are also executing on one of the most valuable late-stage pipelines in rare disease as we anticipate important pivotal Phase 3 results in osteogenesis imperfecta and completion of enrollment in our Phase 3 trial in Angelman syndrome. This progress puts us in the unique position to potentially launch three to four new therapies over the next couple of years, accumulating a total of eight to nine approved products over a 10-year period.”
Ultragenyx will present at the 43ᵗʰ annual J.P. Morgan Healthcare Conference on Monday, January 13, 2025 at 10:30 AM PT. The live and archived webcast of the presentation will be accessible from the company’s website at https://ir.ultragenyx.com/events-presentations.
Financial Update
2024 Preliminary Revenue (unaudited) and 2025 Revenue Guidance
Total revenue for 2024 is estimated to be
In 2025, total revenue is expected to be between
2024 Ending Cash Position (unaudited) and Decreasing 2025 Net Cash Used in Operations
Cash, cash equivalents, and available-for-sale investments were approximately
The 2024 revenues and cash position included in this release are preliminary and are therefore subject to adjustment. The preliminary revenue results are based on management’s initial analysis of operations for the year ended December 31, 2024. The Company expects to issue full financial results for the fourth quarter and fiscal year 2024 in February 2025.
Recent Updates and 2025 Clinical Milestones
UX143 (setrusumab) monoclonal antibody for osteogenesis imperfecta (OI): Phase 3 Orbit study progressing to second interim analysis (IA2) expected in mid-2025
Patients are being dosed 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 towards the second interim analysis in mid-2025 and a potential final analysis in the fourth quarter 2025. 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 and final analyses.
GTX-102 an antisense oligonucleotide for Angelman syndrome: Phase 3 study enrolling; expect enrollment 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. Participants will be 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 will be improvement in cognition assessed by Bayley-4 cognitive raw score, and the key secondary endpoint (with a
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): Biologics license application (BLA) submitted; expect Prescription Drug User Fee Act (PDUFA) decision on the application and launch in second half of 2025
In December 2024, Ultragenyx submitted a BLA to the U.S. Food and Drug Administration for UX111 supported by the available data, including from the ongoing pivotal Transpher A study, demonstrating treatment with UX111 resulted in rapid and sustained decreased levels of heparan sulfate (HS) in cerebral spinal fluid (CSF) in patients with Sanfilippo syndrome type A. The sustained reduction in CSF HS exposure over time was correlated with improved long-term cognitive development compared to the decline observed during the same period of time in natural history data. A PDUFA decision and launch are expected in the second half of 2025.
DTX401 AAV gene therapy for Glycogen Storage Disease Type Ia (GSDIa): BLA filing expected in mid-2025
Ultragenyx previously announced positive topline results from the Phase 3 GlucoGene study for the treatment of patients aged eight years and older. The study 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.
After the 48-week primary efficacy analysis period, crossover patients (previously treated with placebo) were eligible to receive DTX401. These patients were able to titrate cornstarch much more rapidly once they were confirmed to have been treated and had timely direct access to their glucose levels. Patients from the original DTX401 treatment arm who have reached 78 weeks also continued to reduce their daily cornstarch intake, while maintaining glycemic control. DTX401 has demonstrated a consistent and acceptable safety profile with no new safety concerns identified as of the data cut-off.
These results have been discussed with regulatory authorities in a pre-BLA meeting and will be included as part of a BLA submission in mid-2025.
UX701 AAV gene therapy for Wilson Disease: Phase 1/2/3 study ongoing; expect Cohort 4 enrollment completion in second half of 2025
In Stage 1 of the Phase 1/2/3 Cyprus2+ study, 15 patients across three sequential dose cohorts were enrolled and demonstrated clinical activity as well as improvements in copper metabolism. Multiple responders completely tapered off their standard-of-care treatment with responses seen in all three dose cohorts.
The company expects to enroll a fourth cohort in Stage 1 at a moderately increased dose and with an optimized immunomodulation regimen to enhance the efficiency and efficacy of the gene therapy, with the objective of having the majority of patients come off standard-of-care treatment before selecting a dose for the randomized placebo-controlled stage of the study. Enrollment in Cohort 4 is expected to complete in the second half of 2025.
DTX301 AAV gene therapy for Ornithine Transcarbamylase (OTC) Deficiency: Phase 3 study dosing patients; expect enrollment completion in early 2025
Ultragenyx is randomizing and dosing patients in the ongoing Phase 3 study. The pivotal, 64-week study will include up to 50 patients, randomized 1:1 to DTX301 or placebo. The primary endpoints are response as measured by change in 24-hour ammonia levels and removal of ammonia-scavenger medications and protein-restricted diet. Enrollment is expected to be completed in early 2025.
About Ultragenyx
Ultragenyx is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultrarare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease.
The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.
For more information on Ultragenyx, please visit the company's website at: www.ultragenyx.com.
Forward-Looking Statements and Use of Digital Media
Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations and projections regarding its future operating results and financial performance, anticipated cost or expense reductions, the timing, progress and plans for its clinical programs and clinical studies, future regulatory interactions, and the components and timing of regulatory submissions are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause the Company’s clinical development programs, commercial success of its products and product candidates, continued collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainty of clinical drug development and unpredictability and lengthy process for obtaining regulatory approvals, risks related to serious or undesirable side effects of our product candidates, the company’s ability to achieve its projected development goals in its expected timeframes, risks related to reliance on third party partners to conduct certain activities on the company’s behalf, our limited experience in generating revenue from product sales, risks related to product liability lawsuits, our dependence on Kyowa Kirin for the commercial supply of Crysvita, fluctuations in buying or distribution patterns from distributors and specialty pharmacies, the transition back to Kyowa Kirin of our exclusive rights to promote Crysvita in the United States and Canada and unexpected costs, delays, difficulties or adverse impact to revenue related to such transition, smaller than anticipated market opportunities for the company’s products and product candidates, manufacturing risks, competition from other therapies or products, and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations, the company’s future operating results and financial performance, the timing of clinical trial activities and reporting results from same, and the availability or commercial potential of Ultragenyx’s products and drug candidate. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on November 6, 2024, and its subsequent periodic reports filed with the SEC.
In addition to its SEC filings, press releases and public conference calls, Ultragenyx uses its investor relations website and social media outlets to publish important information about the company, including information that may be deemed material to investors, and to comply with its disclosure obligations under Regulation FD. Financial and other information about Ultragenyx is routinely posted and is accessible on Ultragenyx’s Investor Relations website (https://ir.ultragenyx.com/) and LinkedIn website (https://www.linkedin.com/company/ultragenyx-pharmaceutical-inc-/).
Contacts Ultragenyx Pharmaceutical Inc.
Investors
Joshua Higa
ir@ultragenyx.com
Media
Carolyn Wang
media@ultragenyx.com
FAQ
What is Ultragenyx's (RARE) projected revenue guidance for 2025?
How much revenue did Ultragenyx (RARE) generate in 2024?
What is Ultragenyx's (RARE) cash position as of December 2024?
When will Ultragenyx (RARE) complete enrollment for the GTX-102 Phase 3 Aspire study?
What are the key regulatory milestones for Ultragenyx (RARE) in 2025?
