On May 4, 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 its financial results for the quarter ended March 31, 2023 and reaffirmed its financial guidance for 2023 (Press release, Ultragenyx Pharmaceutical, MAY 4, 2023, View Source [SID1234631059]).
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"In the first quarter we continued the strong growth in product revenue and advanced our clinical programs including completion of the Phase 2 portion of our pivotal study of UX143 in osteogenesis imperfecta. We are analyzing the data and look forward to sharing the results soon. Once completed, we will initiate enrollment of the Phase 3 portion in mid-2023," said Emil D. Kakkis, M.D., Ph.D., chief executive officer and president of Ultragenyx. "We are also making rapid progress in our Phase 1/2 study of GTX-102 in Angelman syndrome, dosing in the expansion cohorts and activating sites in new countries."
First Quarter 2023 Selected Financial Data Tables and Financial Results
Revenues (dollars in thousands), (unaudited)
Three Months Ended March 31,
2023 2022
Crysvita
Revenue in profit-share territory $ 49,906 $ 45,164
Product revenue 21,234 9,394
Non-cash royalty revenue in European territory 4,882 4,838
Total Crysvita revenue 76,022 59,396
Dojolvi 14,303 12,429
Mepsevii 8,480 4,861
Evkeeza 212 —
Daiichi Sankyo 1,479 3,249
Total revenues $ 100,496 $ 79,935
Net Revenues
Ultragenyx reported $100.5 million in total revenue for the first quarter of 2023, which represents 26% growth compared to the first quarter 2022. The growth is primarily driven by increased demand for Crysvita in Latin America and steady growth from the other regions and products. The first quarter 2023 also included $1.5 million of revenue from the technology transfer which was completed during the quarter as part of the collaboration and license agreement with Daiichi Sankyo.
Selected Financial Data (dollars in thousands, except per share amounts), (unaudited)
Three Months Ended March 31,
2023 2022
Total revenues $ 100,496 $ 79,935
Operating expense:
Cost of sales 12,257 6,100
Research and development 165,698 143,155
Selling, general and administrative 76,646 67,312
Total operating expense 254,601 216,567
Net loss $ (163,972 ) $ (152,320 )
Net loss per share, basic and diluted $ (2.33 ) $ (2.19 )
Operating Expenses
Total operating expenses for the first quarter of 2023 were $254.6 million, including non-cash stock-based compensation of $31.9 million. In 2023, annual operating expenses are expected to decrease as the company manages headcount and increases operational leverage while executing on high-value programs.
Net Loss
For the first quarter of 2023, Ultragenyx reported net loss of $164.0 million, or $2.33 per share basic and diluted, compared with a net loss for the first quarter of 2022 of $152.3 million, or $2.19 per share, basic and diluted.
Cash, Cash Equivalents and Marketable Debt Securities
Cash, cash equivalents, and marketable debt securities were approximately $714.6 million as of March 31, 2023.
2023 Financial Guidance
For the full year 2023, the company reaffirms:
Total revenue in the range of $425 million to $450 million
Crysvita revenue in the range of $325 million to $340 million. This includes all regions where Ultragenyx will recognize revenue, including the royalties in Europe, which have been ongoing, and the royalties in North America, which began in April 2023.
Dojolvi revenue in the range of $65 million to $75 million
Net Cash Used in Operations is expected to be less than $400 million
Recent Updates and Clinical Milestones
UX143 (setrusumab) monoclonal antibody for Osteogenesis Imperfecta (OI): Phase 2 enrollment complete; Phase 2 data and Phase 3 initiation expected in mid-2023
Ultragenyx is continuing to dose patients in the Phase 2/3 Orbit study of UX143 in pediatric and adult patients with OI aged five to <26 years. All patients have completed the Phase 2 portion of the study with data expected in mid-2023. These data are expected to include changes in bone biomarkers response and some data on bone mineral density that will be used to establish the dosing algorithm for the Phase 3 portion of the study. The Phase 3 is expected to initiate in mid-2023.
In addition, in the second quarter of 2023, Ultragenyx intends to initiate Cosmic, a randomized study in children under age five with serious bone disease, which compares bisphosphonates to UX143. Younger pediatric patients with OI often have a much higher fracture rate than other age groups driving clinical urgency for better treatment options. The primary endpoint is expected to be the annualized rate of fractures.
GTX-102 antisense oligonucleotide for Angelman syndrome: Phase 1/2 dosing in the expansion cohorts
Outside of the U.S., the dose escalation phase of this study has been completed and the company recently transitioned to dosing patients in the expansion cohorts, which are designed to verify the GTX-102 dose and treatment regimen that will be used in the Phase 3 program. The study has also expanded geographically with multiple new sites activated in Europe and Australia. Approximately 40 patients will be enrolled across Cohort A (ages four to Patients from the dose escalation cohorts continue to exhibit encouraging dose and time-dependent clinical activity following longer-term treatment and maintenance dosing. No additional treatment-related serious adverse events or lower extremity weakness adverse events have occurred since our prior update in January 2023. As of May 2023, 14 patients have had at least six months of exposure to GTX-102 including nine patients with more than one year of continuous therapy. The next data update is expected in the second half of 2023. In the U.S., patients are continuing to receive GTX-102 at the lower loading and maintenance dose. Productive discussions with the FDA are ongoing to harmonize the dosing strategy with the ex-U.S. protocol. DTX401 AAV gene therapy for Glycogen Storage Disease Type Ia (GSDIa): Dosing in Phase 3 study complete DTX301 AAV gene therapy for Ornithine Transcarbamylase (OTC) Deficiency: Phase 3 study dosing patients UX701 AAV gene therapy for Wilson Disease: Stage 1 of pivotal clinical study dosing patients; expect interim Stage 1 enrollment completion in the second half of 2023 UX053 mRNA for glycogen storage disease type III (GSDIII): Phase 1/2 single ascending dose cohort enrolled; data in 1H23 Conference Call and Webcast Information Ultragenyx will host a conference call today, Thursday, May 4, 2023, at 2 p.m. PT/ 5 p.m. ET to discuss the first quarter 2023 financial results and provide a corporate update. The live and replayed webcast of the call will be available through the company’s website at View Source To participate in the live call, please register by clicking on the following link (https://register.vevent.com/register/BI1183803cc36b46baa6367e521e12e605), and you will be provided with dial in details. The replay of the call will be available for one year.
Ultragenyx has randomized and dosed the last patient in the Phase 3 study. The 48-week study has fully enrolled 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. Phase 3 data are expected 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.
Dosing in the first stage of the pivotal study is ongoing under an amended protocol that removes placebo from the dose finding stage and includes 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 the second half of 2023 with data expected in the first half of 2024 that would include safety and potentially initial signs of clinical activity.
Dosing in the single ascending dose stage (SAD) of the Phase 1/2 study of UX053 for the treatment of GSDIII has been completed with no safety issues observed. The company has decided to not enroll patients in the multiple ascending dose cohorts at this time to allow greater focus on other late-stage and larger indication clinical programs. The data from the SAD cohort are being analyzed and are expected in the second quarter of 2023.