Third quarter 2022 total revenue of
Reaffirm 2022 Crysvita revenue in Ultragenyx territories guidance of
Enrollment completion of DTX401 Phase 3, UX143 Phase 2, and GTX-102 dose escalation cohorts anticipated around the end of the year
“We continue to initiate treatment globally for more patients with Crysvita, Dojolvi and Mepsevii and are further expanding our markets with approvals of Crysvita in
Third Quarter 2022 Financial Results
Net Revenues
For the third quarter of 2022, Ultragenyx reported
Total revenue for the third quarter of 2022 also includes
Operating Expenses
Total operating expenses for the third quarter of 2022 were
Net Loss
For the third quarter of 2022, Ultragenyx reported net loss of
Net cash used in operations for the nine months ended
Cash,
As of
2022 Financial Guidance
The Company continues to expect 2022 revenue for Crysvita in Ultragenyx territories to be between
Third Quarter 2022 Revenue and Selected Financial Data Tables
Revenues (dollars in thousands) | |||||||||||||||
Three Months Ended | Nine Months Ended | ||||||||||||||
2022 | 2021 | 2022 | 2021 | ||||||||||||
Crysvita Collaboration and Product Revenues: | |||||||||||||||
North America Collaboration | $ | 51,348 | $ | 42,971 | $ | 148,121 | $ | 120,987 | |||||||
ROW Product Sales | 13,184 | 7,378 | 34,980 | 16,150 | |||||||||||
Crysvita in Ultragenyx Territories | 64,532 | 50,349 | 183,101 | 137,137 | |||||||||||
EU Royalty Revenue | 5,373 | 4,665 | 15,634 | 13,454 | |||||||||||
Total Crysvita Revenue | 69,905 | 55,014 | 198,735 | 150,591 | |||||||||||
Dojolvi | 13,274 | 10,654 | 39,200 | 27,735 | |||||||||||
Mepsevii | 6,045 | 3,918 | 15,839 | 12,924 | |||||||||||
Daiichi Sankyo | 1,479 | 12,061 | 6,207 | 76,767 | |||||||||||
Total Revenue | $ | 90,703 | $ | 81,647 | $ | 259,981 | $ | 268,017 |
Selected Financial Data (dollars in thousands, except per share amounts) | |||||||||||||||
Three Months Ended | Nine Months Ended | ||||||||||||||
2022 | 2021 | 2022 | 2021 | ||||||||||||
Total revenue | $ | 90,703 | $ | 81,647 | $ | 259,981 | $ | 268,017 | |||||||
Operating expense: | |||||||||||||||
Cost of sales | 8,631 | 4,175 | 23,001 | 12,499 | |||||||||||
Research and development | 237,297 | 113,417 | 534,981 | 374,140 | |||||||||||
Selling, general and administrative | 69,841 | 53,883 | 205,290 | 160,551 | |||||||||||
Total operating expense | 315,769 | 171,475 | 763,272 | 547,190 | |||||||||||
Net loss | $ | (245,106 | ) | $ | (72,998 | ) | $ | (555,588 | ) | $ | (331,567 | ) | |||
Net loss per share, basic and diluted | $ | (3.50 | ) | $ | (1.08 | ) | $ | (7.96 | ) | $ | (4.91 | ) |
Corporate and Program Updates
Crysvita for X-Linked Hypophosphatemia and Tumor Induced Osteomalacia: Amendment to collaboration agreement increases field support after
In
GTX-102 for Angelman Syndrome: Enrollment and dosing in the Phase 1/2 ongoing; dosing in expansion cohorts expected in the first half of 2023
Based on encouraging interim clinical data and scientific confirmation of their technology, we exercised our option to acquire GeneTx and closed on the acquisition for
In
At this time, 13 patients have received cumulative doses of 20 mg or higher, and 14 patients have over 132 days of exposure to treatment, which was the minimum cumulative dose and maximum exposure after the first dose that led to the lower extremity weakness that was seen in the originally treated five patients. There have been no treatment-related SAEs of any type nor adverse events related to lower extremity weakness observed in these patients. No clinically significant or persistent elevations of cerebrospinal fluid protein level have been observed to date.
Redosing of patients originally treated in 2020 under the original protocol has begun outside the
Dosing in the
UX143 (setrusumab) 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 under a recently amended protocol. The updated protocol removes placebo in the dose finding stage to enable more efficient real-time data analyses for the same two dose levels of setrusumab as in the original protocol. We expect to complete enrollment in early 2023 and will have data from the Phase 2 portion of the study in mid-2023, including two-month changes in bone biomarkers response that will be used to establish the optimal dosing algorithm for the Phase 3 portion of the study.
In addition, Ultragenyx intends to initiate an additional randomized study in children with OI under age five in the first half of 2023 with severe bone disease comparing bisphosphates to setrusumab with total fractures as the primary endpoint.
DTX401 for Glycogen Storage Disease Type Ia (GSDIa): Phase 3 study randomizing patients, last patient to be randomized around the end of the year
In
The Phase 3 study has a 48-week primary efficacy analysis period, and the company plans to enroll 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 to be enrolled in the baseline screening phase of the Phase 3 study of DTX401 is expected to occur around the end of the year.
UX701 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 sets 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. The design should allow more real-time assessment of the gene therapy. Completion of Stage 1 enrollment is expected in mid-2023 with data on safety and initial signs of clinical activity expected around the end of 2023 or early 2024.
DTX301 for Ornithine Transcarbamylase (OTC) Deficiency: Phase 3 study expected to initiate around the end of the year
Ultragenyx expects to initiate the Phase 3 study of DTX301 in patients with OTC around the end of 2022. 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 (
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 ultra-rare 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 effects from the COVID-19 pandemic on the company’s clinical and commercial activities and business and operating results, risks related to reliance on third party partners to conduct certain activities on the company’s behalf, including under our collaboration agreement with Kyowa Kirin, 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 | Nine Months Ended | ||||||||||||||
2022 | 2021 | 2022 | 2021 | ||||||||||||
Statement of Operations Data: | |||||||||||||||
Revenues: | |||||||||||||||
Collaboration and license | $ | 52,827 | $ | 55,048 | $ | 154,328 | $ | 197,998 | |||||||
Product sales | 32,503 | 21,950 | 90,019 | 56,809 | |||||||||||
Non-cash collaboration royalty revenue | 5,373 | 4,649 | 15,634 | 13,210 | |||||||||||
Total revenues | 90,703 | 81,647 | 259,981 | 268,017 | |||||||||||
Operating expenses: | |||||||||||||||
Cost of sales | 8,631 | 4,175 | 23,001 | 12,499 | |||||||||||
Research and development | 237,297 | 113,417 | 534,981 | 374,140 | |||||||||||
Selling, general and administrative | 69,841 | 53,883 | 205,290 | 160,551 | |||||||||||
Total operating expenses | 315,769 | 171,475 | 763,272 | 547,190 | |||||||||||
Loss from operations | (225,066 | ) | (89,828 | ) | (503,291 | ) | (279,173 | ) | |||||||
Change in fair value of equity investments | (1,626 | ) | 25,702 | (21,139 | ) | (25,963 | ) | ||||||||
Non-cash interest expense on liabilities related to the sale of future royalties | (14,505 | ) | (8,683 | ) | (27,141 | ) | (25,618 | ) | |||||||
Other income (expense), net | 2,378 | (7 | ) | 3,130 | 211 | ||||||||||
Loss before income taxes | (238,819 | ) | (72,816 | ) | (548,441 | ) | (330,543 | ) | |||||||
Provision for income taxes | (6,287 | ) | (182 | ) | (7,147 | ) | (1,024 | ) | |||||||
Net loss | $ | (245,106 | ) | $ | (72,998 | ) | $ | (555,588 | ) | $ | (331,567 | ) | |||
Net loss per share, basic and diluted | $ | (3.50 | ) | $ | (1.08 | ) | $ | (7.96 | ) | $ | (4.91 | ) | |||
Weighted-average shares used in computing net loss per share, basic and diluted | 70,054,173 | 67,875,363 | 69,834,037 | 67,533,671 |
Selected Activity included in Operating Expenses
(in thousands)
(unaudited)
Three Months Ended | Nine Months Ended | ||||||||||||||
2022 | 2021 | 2022 | 2021 | ||||||||||||
Non-cash stock based compensation | $ | 35,761 | $ | 26,990 | $ | 101,013 | $ | 78,430 | |||||||
Mereo license and collaboration agreement | — | — | — | 50,000 | |||||||||||
In-process research and development expense from | |||||||||||||||
GeneTx acquisition | 75,234 | — | 75,234 | — |
Selected Balance Sheet Financial Data
(in thousands)
(unaudited)
2022 | 2021 | ||||||
Balance Sheet Data: | |||||||
Cash, cash equivalents, and marketable debt securities | $ | 996,238 | $ | 999,129 | |||
Working capital | 785,307 | 675,236 | |||||
Total assets | 1,618,474 | 1,522,397 | |||||
Total stockholders' equity | 468,829 | 922,561 |
Contacts
Investors
ir@ultragenyx.com
Media
media@ultragenyx.com
Source:
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