Ultragenyx Announces Q1 2024 Financial Results and Corporate Update

Ultragenyx Pharmaceutical Inc., a biopharmaceutical company dedicated to developing and commercializing novel treatments for rare and ultrarare genetic diseases, has reported its financial results for the first quarter of 2024 and reaffirmed its financial guidance for the year.

In the first quarter of 2024, Ultragenyx achieved a total revenue of $109 million, marking an 8% increase compared to the same period in 2023. Crysvita®, one of its leading products, generated $83 million in revenue, reflecting a 9% growth from the previous year. This growth includes a significant 71% increase in product sales from Latin America and Turkey. Meanwhile, Dojolvi® revenue reached $16 million, a 14% rise over the first quarter of 2023. 

The company's operating expenses for the quarter amounted to $274 million, which includes $37 million in non-cash stock-based compensation. Despite the increase in revenue, Ultragenyx reported a net loss of $171 million for the first quarter of 2024, translating to a loss of $2.03 per share. This compares to a net loss of $164 million, or $2.33 per share, in the first quarter of 2023. The net cash used in operations during this period was $191 million, and as of March 31, 2024, the company held $569 million in cash, cash equivalents, and marketable debt securities.

For the full year 2024, Ultragenyx has reaffirmed its expectation of total revenue between $500 million and $530 million. Specifically, Crysvita revenue is anticipated to be in the range of $375 million to $400 million, while Dojolvi revenue is expected to fall between $75 million and $80 million. The company also envisions net cash used in operations to be less than $400 million.

In terms of clinical milestones, Ultragenyx has made notable progress across several programs:

1. UX143 (setrusumab) for Osteogenesis Imperfecta (OI): The Phase 3 Orbit and Cosmic studies have completed enrollment, with 158 and 66 patients respectively. The Orbit study evaluates the effect of setrusumab on annualized clinical fractures in patients aged 5 to under 25 years, while the Cosmic study compares setrusumab to intravenous bisphosphonate therapy in patients aged 2 to under 7 years. Additional data from the Phase 2 Orbit study is expected in the latter half of 2024.

2. GTX-102 for Angelman Syndrome: Positive interim data from the Phase 1/2 study showed rapid and clinically meaningful improvements in patients. The company plans to hold an End of Phase 2 meeting with the FDA in mid-2024.

3. UX701 for Wilson Disease: All patients in the three dose-escalation cohorts of Stage 1 have been dosed, with interim Stage 1 data expected in the second half of 2024. Stage 2 will commence based on these data, involving a pivotal, randomized, placebo-controlled study.

4. UX111 for Sanfilippo Syndrome (MPS IIIA): Ongoing discussions with the FDA are aimed at seeking an accelerated approval pathway. The company participated in a workshop to discuss relevant biomarkers that could support this expedited review.

5. DTX401 for Glycogen Storage Disease Type Ia (GSDIa): The Phase 3 study completed dosing, with results expected in the second quarter of 2024. The primary focus is on reducing glucose replacement therapy while maintaining glucose control.

6. DTX301 for Ornithine Transcarbamylase (OTC) Deficiency: The Phase 3 study is underway, with patient enrollment expected to conclude in the second half of 2024. The study’s primary outcomes include the removal of ammonia-scavenger medications and changes in 24-hour ammonia levels.

Ultragenyx continues to demonstrate robust progress in both its commercial and clinical endeavors, underscoring its commitment to providing novel therapies for patients with serious rare and ultrarare genetic diseases. The company's diverse portfolio and strategic focus on efficient and urgent drug development highlight its dedication to addressing unmet medical needs.

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