Preliminary 2023 Total Revenue of
2024 expected Total Revenue guidance between
Year-end 2023 cash balance of approximately
“2024 is poised to be a year of significant momentum for Ultragenyx, with clinical catalysts across multiple value-driving programs, meaningful revenue growth from our commercial products, and continued focus on financial discipline,” said
Ultragenyx will present at the 42ᵗʰ annual
Financial Update
2023 Revenue (unaudited) and 2024 Revenue Guidance
2023 | 2024 | ||
($ in millions) | Preliminary Revenue | Revenue Guidance | |
Crysvita | |||
Dojolvi | |||
Total Revenue |
2023 Ending Cash Position (unaudited) and 2024 Cash Used in Operations Guidance
Cash, cash equivalents, and available-for-sale investments were approximately
The 2023 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
Recent Updates and 2024 Clinical Milestones
UX143 (setrusumab) monoclonal antibody for Osteogenesis Imperfecta (OI): Phase 3 portion of Orbit study expected to be fully enrolled in the first quarter of 2024
Patients are being dosed in the late-stage clinical trials, Orbit and Cosmic, which evaluate setrusumab in pediatric and young adult patients with OI. The randomized, placebo-controlled Phase 3 portion of the Orbit study is expected to be fully enrolled around the end of the first quarter of 2024. Additional longer-term Phase 2 data from the Orbit study are expected in 2024. The Phase 3 Cosmic study is an active-controlled study evaluating the effect of setrusumab compared to intravenous bisphosphonate (IV-BP) therapy on annualized total fracture rate in patients aged 2 to <5 years. Cosmic is targeting to enroll approximately 65 patients at more than 20 global sites and is expected to complete enrollment in the first half of 2024.
GTX-102 an antisense oligonucleotide for Angelman Syndrome: Phase 1/2 fully enrolled; expansion data expected in the first half of 2024
Enrollment in the expansion cohorts was completed in
UX701 AAV gene therapy for Wilson Disease: Last patient in Cohort 3 on track to be dosed soon; expect interim Stage 1 data in the first half of 2024
Four of five patients in the third of three dose escalation cohorts in the pivotal study have been dosed, with the fifth patient scheduled to be dosed soon. During Stage 1, the safety and efficacy of UX701 will be evaluated and a dose will be selected for further evaluation in Stage 2, the pivotal, randomized, placebo-controlled stage of the study. Data from Stage 1 are expected in the first half of 2024, which will be followed by dose selection and initiation of Stage 2 in the second half of 2024.
UX111 AAV gene therapy for Sanfilippo syndrome (MPS IIIA): Updated data from pivotal Transfer A study to be presented at WORLDSymposiumTM in
Ultragenyx will present new data from the ongoing pivotal Transfer A study evaluating the efficacy and safety of UX111 in children with MPS IIIA at the 20th Annual WORLDSymposiumTM. The presentation will show that reductions of heparan sulfate exposure in cerebrospinal fluid correlate with improved long-term cognitive function in patients with MPS IIIA following treatment with UX111. Discussions with FDA seeking an accelerated review path are ongoing.
DTX401 AAV gene therapy for Glycogen Storage Disease Type Ia (GSDIa): Dosing in Phase 3 study complete; Phase 3 data readout expected in the first half of 2024
In
DTX301 AAV gene therapy for Ornithine Transcarbamylase (OTC) Deficiency: Phase 3 study dosing patients; expect enrollment to be completed in the first half of 2024
Ultragenyx is randomizing and dosing patients in the ongoing Phase 3 study. The pivotal, 64-week study will include approximately 50 patients, randomized 1:1 to DTX301 or placebo. The primary endpoints are response as measured by removal of ammonia-scavenger medications and protein-restricted diet and change in 24-hour ammonia levels. Enrollment is currently expected to be completed in the first half of 2024.
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
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
In addition to its
Contacts
Investors
ir@ultragenyx.com
Media
media@ultragenyx.com
Source:
2024 GlobeNewswire, Inc., source