Ultragenyx Q2 2024 Financial Results and Corporate Update

Ultragenyx Pharmaceutical Inc., a biopharmaceutical company specializing in developing and commercializing treatments for rare and ultra-rare genetic diseases, announced its financial results for the second quarter ending June 30, 2024. The company reported total revenue of $147 million, marking a 36% increase from the same period in 2023. Crysvita® generated $114 million in revenue, representing a 37% growth, while Dojolvi® brought in $19 million, a 17% increase from the previous year.

Emil D. Kakkis, M.D., Ph.D., CEO and President of Ultragenyx, attributed the strong financial performance to rising global demand for their commercial therapies. He highlighted the company’s progress in various clinical programs, including positive data from studies in Angelman syndrome, osteogenesis imperfecta, and Glycogen Storage Disease Type Ia (GSDIa). The company is set to achieve more milestones in the latter half of the year, such as initiating a Phase 3 study for Angelman syndrome and seeking accelerated approval for UX111 in Sanfilippo syndrome type A.

The company’s financials for the second quarter of 2024 revealed an increase in operating expenses, which totaled $263 million, including $39 million in non-cash stock-based compensation. Despite this, Ultragenyx managed to reduce its net loss to $132 million, or $1.52 per share, compared to a net loss of $160 million, or $2.25 per share, in the same quarter of the previous year. For the first half of 2024, the net cash used in operations was $268 million, with a cash balance of $874 million as of June 30, 2024.

Ultragenyx has increased its 2024 total revenue guidance to a range of $530 million to $550 million, up from the previous estimate of $500 million to $530 million. Crysvita revenue is expected to be on the higher end of $375 million to $400 million, including sales from Latin America, Turkey, Europe, and North America. Dojolvi revenue is projected to be between $75 million to $80 million. The company anticipates net cash used in operations to be less than $400 million for the year.

In clinical milestones, the company announced significant progress across several programs. The UX143 (setrusumab) monoclonal antibody for osteogenesis imperfecta showed a 67% reduction in the annualized fracture rate after 14 months, alongside consistent improvements in lumbar spine bone mineral density (BMD). GTX-102 for Angelman syndrome is on track to begin a pivotal Phase 3 Aspire study by year-end, following a successful End-of-Phase 2 meeting with the FDA. The study will include approximately 120 patients and focus on cognitive improvement and other key endpoints.

The UX701 AAV gene therapy for Wilson disease completed dosing of the last patient in Cohort 3, with interim Stage 1 data expected in the latter half of 2024. For UX111 in Sanfilippo syndrome type A, Ultragenyx reached an agreement with the FDA on using cerebral spinal fluid heparan sulfate as a surrogate endpoint for accelerated approval. Additionally, positive top-line results from the Phase 3 study of DTX401 gene therapy for GSDIa showed a statistically significant reduction in daily cornstarch intake while maintaining glucose control.

For the DTX301 gene therapy targeting Ornithine Transcarbamylase (OTC) Deficiency, the company is currently dosing patients in a pivotal Phase 3 study, with enrollment expected to be completed in the second half of 2024. The primary goals are to measure the response by the removal of ammonia-scavenger medications and changes in ammonia levels.

Overall, Ultragenyx demonstrated solid growth and significant advancements in its clinical pipeline, positioning itself strongly for continued success in the field of rare and ultra-rare genetic disease treatments.

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