Second quarter 2022 total revenue of $89.3 million and Crysvita® revenue in Ultragenyx territories1 of $64.0 million
Reaffirm 2022 Crysvita revenue in Ultragenyx territories guidance of $250 million to $260 million and Dojolvi revenue of $55 million to $65 million
Expanded clinical pipeline through acquisition of GeneTx and global rights to UX111 for Sanfilippo syndrome
Completed $500.0 million sale of a capped 30% of royalty interest on the future sales of Crysvita in the United States and Canada
NOVATO, Calif., July 28, 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 June 30, 2022 and provided a corporate update for the year.
“Over the last few months we continued building our commercial footprint, particularly in Latin America, raised $500 million in in a capped royalty financing, and acquired GeneTx, all of which puts us in a strong position to generate meaningful value in the coming years,” said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. “We have one of the most diverse, late-stage pipelines in rare disease, which includes our fourth pivotal gene therapy, UX111 for Sanfilippo syndrome, our pivotal program in osteogenesis imperfecta, and our promising ASO, GTX-102 in Angelman syndrome.”
Second Quarter 2022 Financial Results
Net RevenuesFor the second quarter of 2022, Ultragenyx reported $89.3 million in total revenue. Ultragenyx recognized $64.0 million in Crysvita (burosumab) revenue in the Ultragenyx territories, which includes $51.6 million in collaboration revenue in the North American profit share territory and net product sales in other regions of $12.4 million. Total royalty revenue related to European Crysvita sales was $5.4 million. Dojolvi (triheptanoin) product sales in the second quarter of 2022 were $13.5 million. Mepsevii (vestronidase alfa) product sales for the second quarter of 2022 were $4.9 million.
Total revenue for the second quarter of 2022 also includes $1.5 million related to technical assistance following the successful completion of technology transfer activities with Daiichi Sankyo. This compares to total revenue in the second quarter of 2021, which includes $22.0 million related to the technology transfer services which were ongoing at the time.
Operating Expenses Total operating expenses for the second quarter of 2022 were $230.9 million, including non-cash stock-based compensation of $35.9 million.
Net Loss For the second quarter of 2022, Ultragenyx reported net loss of $158.2 million, or $2.26 per share basic and diluted, compared with a net loss for the second quarter of 2021 of $122.4 million, or $1.81 per share, basic and diluted. Net cash used in operations for the six months ended June 30, 2022 was $194.8 million.
Cash, Cash Equivalents and Marketable Debt SecuritiesAs of June 30, 2022, cash, cash equivalents, and marketable debt securities were $706.1 million.
In July 2022, we raised $500.0 million through the sale of 30% of our royalty interest on the future sales of Crysvita in the United States and Canada, capped at $725.0 million or 1.45x the purchase price, and we exercised our option to acquire GeneTx for $75.0 million.
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.
Second Quarter 2022 Revenue and Selected Financial Data Tables
| Revenues (dollars in thousands) | |||||||||||||||
| Three Months Ended June 30, | Six Months Ended June 30, | ||||||||||||||
| 2022 | 2021 | 2022 | 2021 | ||||||||||||
| Crysvita Collaboration and Product Revenues: | |||||||||||||||
| North America Collaboration | $ | 51,609 | $ | 41,756 | $ | 96,773 | $ | 78,016 | |||||||
| ROW Product Sales | 12,402 | 2,900 | 21,796 | 8,772 | |||||||||||
| Crysvita in Ultragenyx Territories | 64,011 | 44,656 | 118,569 | 86,788 | |||||||||||
| EU Royalty Revenue | 5,423 | 4,917 | 10,261 | 8,789 | |||||||||||
| Total Crysvita Revenue | 69,434 | 49,573 | 128,830 | 95,577 | |||||||||||
| Dojolvi | 13,497 | 10,047 | 25,926 | 17,081 | |||||||||||
| Mepsevii | 4,933 | 5,399 | 9,794 | 9,006 | |||||||||||
| Daiichi Sankyo | 1,479 | 21,956 | 4,728 | 64,706 | |||||||||||
| Total Revenue | $ | 89,343 | $ | 86,975 | $ | 169,278 | $ | 186,370 | |||||||
| Selected Financial Data (dollars in thousands, except per share amounts) | |||||||||||||||
| Three Months Ended June 30, | Six Months Ended June 30, | ||||||||||||||
| 2022 | 2021 | 2022 | 2021 | ||||||||||||
| Total revenue | $ | 89,343 | $ | 86,975 | $ | 169,278 | $ | 186,370 | |||||||
| Operating expense: | |||||||||||||||
| Cost of sales | 8,270 | 3,136 | 14,370 | 8,324 | |||||||||||
| Research and development | 154,529 | 113,205 | 297,684 | 260,723 | |||||||||||
| Selling, general and administrative | 68,137 | 53,410 | 135,449 | 106,668 | |||||||||||
| Total operating expense | 230,936 | 169,751 | 447,503 | 375,715 | |||||||||||
| Net loss | $ | (158,162 | ) | $ | (122,428 | ) | $ | (310,482 | ) | $ | (258,569 | ) | |||
| Net loss per share, basic and diluted | $ | (2.26 | ) | $ | (1.81 | ) | $ | (4.45 | ) | $ | (3.84 | ) | |||
Corporate and Program UpdatesPartial sale of North America Crysvita royaltyIn July 2022, we sold 30% of our royalty interest in Crysvita, in the profit-share territory, to OMERS for $500.0 million, beginning in April 2023. Total payments are capped at $725.0 million, or 1.45x the purchase price, after which payments to OMERS will cease and Ultragenyx will retain the full value of the royalty.
GTX-102 for Angelman Syndrome
In July 2022, we exercised our option to acquire GeneTx and closed on the acquisition for $75.0 million plus future milestone and royalty payments. Additional milestones include $30.0 million upon achievement of the earlier of a Phase 3 clinical study start or product approvals in Canada and the U.K. We will also pay tiered royalties ranging from a mid-single to low double-digit percentage based on worldwide annual net sales.
In July 2022, we also provided an interim data and program update on patients treated in Canada and the U.K. and the U.S. under each region’s amended protocol for the Phase 1/2 study. Further details from this release can be found on the Company website.
We currently expect to provide a program update after we have determined an optimal dose and have gathered substantial data from the expansion cohorts.
UX143 (setrusumab) for Osteogenesis Imperfecta (OI)Ultragenyx is dosing patients in the seamless Phase 2/3 Orbit study of UX143 in pediatric and adult patients with OI ages five to