First quarter 2022 total revenue of $79.9 million and Crysvita® revenue in Ultragenyx territories1 of $54.6 million
Reaffirm 2022 Crysvita revenue in Ultragenyx territories guidance of $250 million to $260 million and Dojolvi revenue of $55 million to $65 million
Cash balance of approximately $814 million as of March 31, 2022
NOVATO, Calif., May 05, 2022 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today reported its financial results for the quarter ended March 31, 2022 and reaffirmed its financial guidance for the year.
“Product based revenue continues to grow driven by increased traction for Crysvita in Latin America,” said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. “In the clinical portfolio, we are now dosing patients in three of our four concurrent pivotal programs and are on track for updated data from the Phase 1/2 for Angelman syndrome mid-year.”
First Quarter 2022 Financial Results
Net RevenuesFor the first quarter of 2022, Ultragenyx reported $79.9 million in total revenue. Ultragenyx recognized $54.6 million in Crysvita (burosumab) revenue in the Ultragenyx territories, which includes $45.2 million in collaboration revenue in the North American profit share territory and net product sales in other regions of $9.4 million. Total royalty revenue related to European Crysvita sales was $4.8 million. Dojolvi (triheptanoin) product sales in the first quarter of 2022 were $12.4 million. Mepsevii (vestronidase alfa) product sales for the first quarter of 2022 were $4.9 million.
Total revenue for the first quarter of 2022 also includes $3.2 million related to technical assistance following the successful completion of technology transfer activities with Daiichi Sankyo. This compares to total revenue in the first quarter of 2021, which includes $42.8 million related to the technology transfer services which were ongoing at the time.
Operating Expenses Total operating expenses for the first quarter of 2022 were $216.6 million, including non-cash stock-based compensation of $29.4 million.
Net Loss For the first quarter of 2022, Ultragenyx reported net loss of $152.3 million, or $2.19 per share basic and diluted, compared with a net loss for the first quarter of 2021 of $136.1 million, or $2.03 per share, basic and diluted. Net cash used in operations for the quarter ended March 31, 2022 was $117.5 million.
Cash, Cash Equivalents and Marketable Debt SecuritiesCash, cash equivalents, and marketable debt securities were $813.8 million as of March 31, 2022.
2022 Financial Guidance
The company continues to expect 2022 revenue for Crysvita in Ultragenyx territories to be between $250 million and $260 million and Dojolvi revenue to be between $55 million and $65 million.
First Quarter 2022 Revenue and Selected Financial Data Tables
| Revenues (dollars in thousands) | |||||||
| Three Months Ended March 31, | |||||||
| 2022 | 2021 | ||||||
| Crysvita Collaboration and Product Revenues: | |||||||
| North America Collaboration | $ | 45,164 | $ | 36,260 | |||
| ROW Product Sales | 9,394 | 5,872 | |||||
| Crysvita in Ultragenyx Territories | 54,558 | 42,132 | |||||
| EU Royalty Revenue | 4,838 | 3,872 | |||||
| Total Crysvita Revenue | 59,396 | 46,004 | |||||
| Dojolvi | 12,429 | 7,034 | |||||
| Mepsevii | 4,861 | 3,607 | |||||
| Daiichi Sankyo | 3,249 | 42,750 | |||||
| Total Revenue | $ | 79,935 | $ | 99,395 | |||
| Selected Financial Data (dollars in thousands, except per share amounts) | |||||||
| Three Months Ended March 31, | |||||||
| 2022 | 2021 | ||||||
| Total revenue | $ | 79,935 | $ | 99,395 | |||
| Operating expense: | |||||||
| Cost of sales | 6,100 | 5,188 | |||||
| Research and development | 143,155 | 147,518 | |||||
| Selling, general and administrative | 67,312 | 53,258 | |||||
| Total operating expense | 216,567 | 205,964 | |||||
| Net loss | $ | (152,320 | ) | $ | (136,141 | ) | |
| Net loss per share, basic and diluted | $ | (2.19 | ) | $ | (2.03 | ) | |
Recent Updates
Evkeeza® (evinacumab) for Homozygous Familial Hypercholesterolemia (HoFH): European marketing authorization transitioned to Ultragenyx, reimbursement processes initiatedThe European Commission has completed transfer of the marketing authorization of Evkeeza from Regeneron. Ultragenyx is preparing reimbursement dossiers for various national health authorities in Europe. Ultragenyx received rights to commercialize and distribute Evkeeza in countries outside the U.S. in January 2022.
UX143 (setrusumab) for Osteogenesis Imperfecta (OI): Dosing has been initiated for the Phase 2/3 Orbit study; Phase 2 study in children under age five planned for second half of 2022Ultragenyx has begun dosing patients in the seamless Phase 2/3 Orbit study of UX143 in pediatric and adult patients with OI ages five to