On January 6, 2023 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultrarare genetic diseases, reported preliminary unaudited 2022 revenue results, cash and investments at year end 2022, and provided 2023 guidance on select key financial metrics including net cash used in operations (Press release, Ultragenyx Pharmaceutical, JAN 6, 2023, View Source [SID1234626002]).
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"We have made substantial investments in our pipeline and manufacturing capabilities over the last two years that put us in a special position to advance multiple transformative treatments in late-stage development while continuing to drive revenue growth, delivering approved medicines globally for rare disease patients," said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. "We have also made a substantial effort to focus on key value drivers within the pipeline and reduce cash spending to maintain our strong financial position through 2023."
Ultragenyx will present at the 41ᵗʰ annual J.P. Morgan Healthcare Conference on Tuesday, January 10, 2023 at 9:45 a.m. PT. The live and archived webcast of the presentation will be accessible from the company’s website at View Source
Financial Update
2022 Revenue (unaudited) and 2023 Revenue Guidance ($ in millions)
2022 2023
Preliminary Revenue Revenue Guidance
Crysvita in Ultragenyx Territories1 $257 – $258
Total Crysvita $277 – $279 $325 – $340
Dojolvi $55 – $56 $65 – $75
Total Product Revenue $352 – $356 $425 – $450
In 2023, Crysvita guidance is provided for all regions where Ultragenyx will recognize revenue, including the royalties in Europe, which have been ongoing, and the royalties in North America, which will begin in April 2023. The mid-point of the Total Crysvita guidance range represents a 20% year-over-year growth rate, as compared to the same regions in 2022.
2022 Ending Cash Position (unaudited) and 2023 Cash Used in Operations Guidance
Cash, cash equivalents, and available-for-sale investments were approximately $900 million as of December 31, 2022. Cash uses in 2022 included non-recurring items of approximately $90 million for capital expenses for the gene therapy manufacturing facility, which is nearing completion, $75 million for the acquisition of GeneTx and $30 million upfront fee related to the Evkeeza license and collaboration agreement.
In 2023 with increased focus on key pipeline value drivers, net cash use is projected to be meaningfully reduced. Net Cash Used in Operations for 2023 is expected to be less than $400 million.
The 2022 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, 2022. The Company expects to issue full financial results for the fourth quarter and fiscal year 2022 in February 2023.
Recent Updates and 2023 Clinical Milestones
UX143 (setrusumab) monoclonal antibody for Osteogenesis Imperfecta (OI): Phase 2/3 study dosing patients; Phase 2 data expected in mid-2023
Ultragenyx is currently dosing patients in the Phase 2/3 Orbit study of UX143 in pediatric and adult patients with OI aged five to <26 years. Enrollment completion is anticipated in early 2023 and data from the Phase 2 portion of the study is expected in mid-2023, including two-month changes in bone biomarkers response that will be used to establish the dosing algorithm for the Phase 3 portion of the study. Optimizing the dosing across the age range of the study will support meaningful benefit in the reduction of clinically evident fractures.
In addition, in the first half of 2023, Ultragenyx intends to initiate a randomized study in OI in children under age five with severe bone disease, comparing bisphosphonates to setrusumab. Younger pediatric patients with OI often have a much higher fracture rate than other age groups and a greater medical need, driving clinical urgency for better treatment options. Total fractures is expected to be the primary endpoint in the study.
GTX-102 an antisense oligonucleotide for Angelman Syndrome: Phase 1/2 continues dose exploration
Ultragenyx continues to explore the dose of GTX-102 in cohorts of younger and older patients. Expansion of the dosing cohort population is expected in the first half of 2023.
DTX401 AAV gene therapy for Glycogen Storage Disease Type Ia (GSDIa): Last patient in the Phase 3 study has entered the baseline screening period. Phase 3 data readout expected in the first half of 2024
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The Phase 3 study has a 48-week primary efficacy analysis period, and the company has enrolled approximately 50 patients eight years of age and older, randomized 1:1 to DTX401 or placebo. The primary endpoint is the reduction in oral glucose replacement with cornstarch while maintaining glucose control. The last patient has entered the baseline screening phase of the Phase 3 study of DTX401, with Phase 3 data expected in the first half of 2024.
UX701 AAV gene therapy for Wilson Disease: Stage 1 of pivotal clinical study dosing patients; expect interim Stage 1 enrollment completion in mid-2023
The company is dosing patients in the first stage of the Cyprus2+ study of UX701 under a recently amended protocol that removes placebo from the dose finding stage and enrolls five patients per cohort. During this stage of the study, safety and efficacy of up to three dose levels of UX701 will be evaluated and a dose will be selected for further evaluation in Stage 2. Completion of Stage 1 enrollment is expected in mid-2023 with data on safety and initial signs of clinical activity expected in early 2024.
DTX301 AAV gene therapy for Ornithine Transcarbamylase (OTC) Deficiency: Phase 3 study expected to initiate in the first quarter of 2023
Ultragenyx expects to initiate the Phase 3 study of DTX301 in patients with OTC in the first quarter of 2023. The 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.
1: Ultragenyx territories include the collaboration revenue from the North American profit share territory (U.S. and Canada) and other regions where revenue from product sales is recognized by Ultragenyx (Latin America, Turkey). This excludes the European territory revenue, which is recognized as non-cash royalty revenue since the rights were sold to Royalty Pharma in December 2019.