Second quarter 2023 total revenue of
Total revenue grew 21% and total Crysvita revenue grew 20% versus the second quarter 2022
Reaffirmed 2023 expected total revenue guidance between
“In the second quarter, our global commercial efforts have resulted in continued meaningful growth of Crysvita, Dojolvi and Mepsevii revenue, including in our key territories outside of the U.S.,” said
Second Quarter 2023 Selected Financial Data Tables and Financial Results
Revenues (dollars in thousands), (unaudited) | |||||||||||||||
Three Months Ended | Six Months Ended | ||||||||||||||
2023 | 2022 | 2023 | 2022 | ||||||||||||
Crysvita | |||||||||||||||
Collaboration revenue in profit-share territory | $ | 19,799 | $ | 51,609 | $ | 69,705 | $ | 96,773 | |||||||
Royalty revenue | 29,061 | — | 29,061 | — | |||||||||||
Non-cash royalty revenue | 17,270 | 5,423 | 22,152 | 10,261 | |||||||||||
Product sales | 16,884 | 12,402 | 38,118 | 21,796 | |||||||||||
Total Crysvita revenue | 83,014 | 69,434 | 159,036 | 128,830 | |||||||||||
Dojolvi | 16,491 | 13,497 | 30,794 | 25,926 | |||||||||||
Mepsevii | 8,439 | 4,933 | 16,919 | 9,794 | |||||||||||
Evkeeza | 365 | — | 577 | — | |||||||||||
Daiichi Sankyo | — | 1,479 | 1,479 | 4,728 | |||||||||||
Total revenues | $ | 108,309 | $ | 89,343 | $ | 208,805 | $ | 169,278 |
Total Revenues
Ultragenyx reported
Selected Financial Data (dollars in thousands, except per share amounts), (unaudited) | |||||||||||||||
Three Months Ended | Six Months Ended | ||||||||||||||
2023 | 2022 | 2023 | 2022 | ||||||||||||
Total revenues | $ | 108,309 | $ | 89,343 | $ | 208,805 | $ | 169,278 | |||||||
Operating expenses: | |||||||||||||||
Cost of sales | 9,914 | 8,270 | 22,171 | 14,370 | |||||||||||
Research and development | 164,949 | 154,529 | 330,647 | 297,684 | |||||||||||
Selling, general and administrative | 81,403 | 68,137 | 158,049 | 135,449 | |||||||||||
Total operating expense | 256,266 | 230,936 | 510,867 | 447,503 | |||||||||||
Net loss | $ | (159,828 | ) | $ | (158,162 | ) | $ | (323,800 | ) | $ | (310,482 | ) | |||
Net loss per share, basic and diluted | $ | (2.25 | ) | $ | (2.26 | ) | $ | (4.58 | ) | $ | (4.45 | ) |
Operating Expenses
Total operating expenses for the second quarter of 2023 were
Net Loss
For the second quarter of 2023, Ultragenyx reported net loss of
Cash,
Cash, cash equivalents, and marketable debt securities were approximately
2023 Financial Guidance
For the full year 2023, the company expects:
- 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 inEurope , which have been ongoing, and the royalties inNorth America , which began inApril 2023 . - Dojolvi revenue in the range of
$65 million to$75 million Net Cash Used in Operations to be around$400 million
Recent Updates and Clinical Milestones
UX143 (setrusumab) monoclonal antibody for Osteogenesis Imperfecta (OI): Two Phase 3 studies enrolling, additional Phase 2 data expected in mid-October
Positive data from the dose-selection Phase 2 portion of the Phase 2/3 Orbit study were announced in
The large increase in BMD observed in the Orbit patient population over the first 3 months was consistent with the rapid increase in serum P1NP levels and was similar to results that took 1 year to achieve in the ASTEROID study in adult OI patients. Treatment with setrusumab for 3 months resulted in an increase in lumbar spine BMD from baseline of 9.4% at 20 mg/kg (n=10), which represents a substantial mean change in Z-score of +0.65 from -2.12 (n=11) at baseline. Treatment with 40 mg/kg (n=7) resulted in a 9.8% BMD increase. Patients on placebo at the 3-month timepoint (n=2) showed no significant change in BMD or change in lumbar spine Z-score.
As of the data cut-off, there had been no treatment-related serious adverse events observed in the study. Reported adverse events were generally consistent with those observed in the ASTEROID study and included infusion associated events, headache and sinusitis. There were no reported hypersensitivity reactions related to setrusumab. There were no safety-related differences observed between dosing groups or age groups.
In July we announced, the first patients were dosed in both of the late-stage clinical trials, Orbit and Cosmic, which evaluate setrusumab in pediatric and young adult patients with OI. Orbit is expected to enroll approximately 195 patients at more than 40 sites across 12 countries. The Phase 3 Cosmic study is an active-controlled study evaluating the effect of setrusumab compared to intravenous bisphosphonate (IV-BP) therapy on annualized total fracture rate in patients aged 2 to <5 years. Cosmic is expected to enroll approximately 65 patients at more than 20 global sites.
Additional data, including fracture frequency information, from the Phase 2 portion of the Orbit study are expected to be shared at an Analyst Day planned for mid-October.
GTX-102 antisense oligonucleotide for Angelman syndrome: Phase 1/2 expansion cohorts enrolling; program update planned for
In
As of
Globally, sites are enrolling patients in the expansion cohorts and will evaluate the same safety, pharmacokinetic, and efficacy measures as the dose escalating cohorts. Enrollment has accelerated in the last two months as additional sites, including the
An interim program update is expected at an Analyst Day in
UX701 AAV gene therapy for Wilson Disease: Stage 1 of pivotal clinical study dosing patients; expect Stage 1 enrollment completion around the end of the year
Dosing in Stage 1 of the pivotal study is ongoing and is expected to enroll five patients per escalating dose cohort. In
DTX401 AAV gene therapy for Glycogen Storage Disease Type Ia (GSDIa): Dosing in Phase 3 study complete
In
DTX301 AAV gene therapy for Ornithine Transcarbamylase (OTC) Deficiency: Phase 3 study dosing patients
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.
Analyst Day planned for mid-October
The company intends to host an Analyst Day in mid-October to highlight additional Phase 2 Orbit clinical data, including fracture frequency information in patients with OI, and provide program updates on the pipeline, including GTX-102 for Angelman syndrome.
Corporate updates
Conference Call and Webcast Information
Ultragenyx will host a conference call today,
About Ultragenyx
Ultragenyx is a biopharmaceutical company committed to bringing novel therapies to patients for the treatment of serious rare and ultrarare genetic diseases. The company has built a diverse portfolio of approved medicines and treatment candidates aimed at addressing diseases with high unmet medical need and clear biology, for which there are typically no approved therapies treating the underlying disease.
The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.
For more information on Ultragenyx, please visit the company's website at: www.ultragenyx.com.
Forward-Looking Statements and Use of Digital Media
Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations and projections regarding its future operating results and financial performance, anticipated cost or expense reductions, the timing, progress and plans for its clinical programs and clinical studies, future regulatory interactions, and the components and timing of regulatory submissions are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainty of clinical drug development and unpredictability and lengthy process for obtaining regulatory approvals, risks related to serious or undesirable side effects of our product candidates, the company’s ability to achieve its projected development goals in its expected timeframes, risks related to reliance on third party partners to conduct certain activities on the company’s behalf, our limited experience in generating revenue from product sales, risks related to product liability lawsuits, our dependence on Kyowa Kirin for the commercial supply of Crysvita, fluctuations in buying or distribution patterns from distributors and specialty pharmacies, the transition back to Kyowa Kirin of our exclusive rights to promote Crysvita in
In addition to its
Selected Statement of Operations Financial Data | |||||||||||||||
(in thousands, except share and per share amounts) | |||||||||||||||
(unaudited) | |||||||||||||||
Three Months Ended | Six Months Ended | ||||||||||||||
2023 | 2022 | 2023 | 2022 | ||||||||||||
Statement of Operations Data: | |||||||||||||||
Revenues: | |||||||||||||||
Collaboration and license | $ | 19,799 | $ | 53,088 | $ | 71,184 | $ | 101,501 | |||||||
Product sales | 42,179 | 30,832 | 86,408 | 57,516 | |||||||||||
Royalty revenue | 46,331 | 5,423 | 51,213 | 10,261 | |||||||||||
Total revenues | 108,309 | 89,343 | 208,805 | 169,278 | |||||||||||
Operating expenses: | |||||||||||||||
Cost of sales | 9,914 | 8,270 | 22,171 | 14,370 | |||||||||||
Research and development | 164,949 | 154,529 | 330,647 | 297,684 | |||||||||||
Selling, general and administrative | 81,403 | 68,137 | 158,049 | 135,449 | |||||||||||
Total operating expenses | 256,266 | 230,936 | 510,867 | 447,503 | |||||||||||
Loss from operations | (147,957 | ) | (141,593 | ) | (302,062 | ) | (278,225 | ) | |||||||
Change in fair value of equity investments | 261 | (10,184 | ) | (73 | ) | (19,513 | ) | ||||||||
Non-cash interest expense on liabilities for sales of future royalties | (15,375 | ) | (6,052 | ) | (31,011 | ) | (12,636 | ) | |||||||
Other income (expense), net | 3,975 | (31 | ) | 10,573 | 752 | ||||||||||
Loss before income taxes | (159,096 | ) | (157,860 | ) | (322,573 | ) | (309,622 | ) | |||||||
Provision for income taxes | (732 | ) | (302 | ) | (1,227 | ) | (860 | ) | |||||||
Net loss | $ | (159,828 | ) | $ | (158,162 | ) | $ | (323,800 | ) | $ | (310,482 | ) | |||
Net loss per share, basic and diluted | $ | (2.25 | ) | $ | (2.26 | ) | $ | (4.58 | ) | $ | (4.45 | ) | |||
Weighted-average shares used in computing net loss per share, basic and diluted | 70,897,991 | 69,925,358 | 70,639,015 | 69,722,141 | |||||||||||
Selected Activity included in Operating Expenses | |||||||||||||||
(in thousands) | |||||||||||||||
(unaudited) | |||||||||||||||
Three Months Ended | Six Months Ended | ||||||||||||||
2023 | 2022 | 2023 | 2022 | ||||||||||||
Non-cash stock-based compensation | $ | 34,653 | $ | 35,865 | $ | 66,592 | $ | 65,252 | |||||||
UX143 clinical milestone | $ | 9,000 | — | $ | 9,000 | — | |||||||||
Selected Balance Sheet Financial Data | |||||||
(in thousands) | |||||||
(unaudited) | |||||||
2023 | 2022 | ||||||
Balance Sheet Data: | |||||||
Cash, cash equivalents, and marketable debt securities | $ | 618,352 | $ | 896,732 | |||
Working capital | 457,758 | 622,689 | |||||
Total assets | 1,311,310 | 1,545,444 | |||||
Total stockholders' equity | 129,030 | 352,494 | |||||
Contacts
Investors
ir@ultragenyx.com
Media
media@ultragenyx.com
Source:
2023 GlobeNewswire, Inc., source