Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) announced its third-quarter 2024 financial results, showcasing substantial revenue growth and significant advancements in its clinical programs. The company reported a 42% increase in total revenue compared to the same period in 2023, reaching $139 million. This growth is primarily attributed to the strong performance of Crysvita and Dojolvi, highlighting the increasing demand for Ultragenyx's therapies for rare and ultrarare genetic diseases.
Financial Performance
Total revenue for Q3 2024 reached $139 million, a 42% increase from $98.1 million in Q3 2023. Crysvita revenue grew by 31% to $98 million, driven by product sales in Latin America and Turkey, which saw an 85% increase. Dojolvi revenue increased by 29% to $21 million. Evkeeza revenue reached $11 million, reflecting growing demand outside the United States. The company reaffirmed its full-year 2024 revenue guidance of $530 million to $550 million.
Operating expenses for the third quarter were $271 million, including $42 million in non-cash stock-based compensation. Ultragenyx reported a net loss of $134 million, or $1.40 per share, compared to a net loss of $160 million, or $2.23 per share, for the same period in 2023. Cash, cash equivalents, and marketable debt securities totaled $825 million as of September 30, 2024.
Regulatory and Clinical Updates
Setrusumab (UX143) for Osteogenesis Imperfecta: The FDA granted Breakthrough Therapy Designation (BTD) to setrusumab (UX143) for the treatment of osteogenesis imperfecta (OI). This designation was based on positive results from the Phase 2 portion of the Orbit study, which demonstrated a rapid and clinically meaningful 67% decrease in fracture rate (p=0.0014) in patients. The BTD aims to expedite the development and review of drugs intended to treat serious conditions, where preliminary clinical evidence indicates substantial improvement over existing therapies.
DTX401 for Glycogen Storage Disease Type Ia (GSDIa): Phase 3 follow-up data from the GlucoGene study showed that crossover patients treated with DTX401 experienced a 62% mean reduction in daily cornstarch intake at Week 30 post-treatment. This is compared to a 41% mean reduction at the end of 48 weeks in the original DTX401 treatment arm. Patients were able to titrate cornstarch more rapidly once they were confirmed to have been treated and had timely direct access to their glucose levels. Ultragenyx plans to discuss these results with regulatory authorities to support a BLA submission in mid-2025.
Dojolvi for Long-Chain Fatty Acid Oxidation Disorder (LC-FAOD): Ultragenyx reached an agreement with Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) on conditional filing requirements for Dojolvi. The company expects to file a J-NDA in mid-2025 based on available global clinical data.
GTX-102 for Angelman Syndrome: Longer-term interim data from the Phase 1/2 study of GTX-102 are expected to be presented at upcoming scientific meetings. The company remains on track to initiate the pivotal Phase 3 Aspire study by the end of 2024 and the Aurora study in 2025.
UX111 for Sanfilippo Syndrome Type A (MPS IIIA): Ultragenyx successfully completed a pre-BLA meeting with the FDA and aligned on the details of its BLA, which is expected to be filed around the end of 2024.
UX701 for Wilson Disease: An update from the Phase 1/2/3 Cyprus2+ study demonstrated clinical activity and improvements in copper metabolism for patients treated with UX701. Multiple responders completely tapered off their standard-of-care treatment. The company expects to enroll an additional cohort in Stage 1 with an optimized immunomodulation regimen.
DTX301 for Ornithine Transcarbamylase (OTC) Deficiency: Ultragenyx is actively dosing patients in the ongoing Phase 3 study, with enrollment expected to be completed in the next few months. The 64-week study will include up to 50 patients randomized 1:1 to DTX301 or placebo.
Future Outlook
Ultragenyx is focused on advancing its pipeline of novel therapies for rare and ultrarare genetic diseases. With multiple near-term BLA submissions and approvals anticipated, the company is poised for continued growth and expansion in the rare disease therapeutics market.
