Ultragenyx Reports Third Quarter 2022 Financial Results and Corporate Update

Third quarter 2022 total revenue of $90.7 million and Crysvita® revenue in Ultragenyx territories1 of $64.5 million

Reaffirm 2022 Crysvita revenue in Ultragenyx territories guidance of $250 million to $260 million and Dojolvi revenue of $55 million to $65 million

Enrollment completion of DTX401 Phase 3, UX143 Phase 2, and GTX-102 dose escalation cohorts anticipated around the end of the year

NOVATO, Calif., Nov. 02, 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 September 30, 2022 and provided a corporate update for 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 Argentina and Dojolvi in Mexico. We are seeing significant enthusiasm for the Evkeeza launch in Europe as we initiate reimbursement steps in member countries and are receiving many urgent calls for access for pediatric cases of HoFH,” said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. “At the same time, we are focusing on our priority clinical programs and in the next few months anticipate completing enrollment in the pivotal study for DTX401 for GSDIa, the important setrusumab Phase 2 stage for osteogenesis imperfecta, and the dose escalation cohorts of the GTX-102 program for Angelman syndrome.”

Third Quarter 2022 Financial Results

Net RevenuesFor the third quarter of 2022, Ultragenyx reported $90.7 million in total revenue. Ultragenyx recognized $64.5 million in Crysvita (burosumab) revenue in the Ultragenyx territories, which includes $51.3 million in collaboration revenue in the North American profit share territory and net product sales in other regions of $13.2 million. Total royalty revenue related to European Crysvita sales was $5.4 million. Dojolvi (triheptanoin) product sales in the third quarter of 2022 were $13.3 million. Mepsevii (vestronidase alfa) product sales for the third quarter of 2022 were $6.0 million.

Total revenue for the third 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 third quarter of 2021, which includes $12.1 million related to the technology transfer services which were ongoing at the time.

Operating Expenses Total operating expenses for the third quarter of 2022 were $315.8 million, which includes research and development expenses of $237.3 million, selling, general and administrative expenses of $69.8 million, and cost of sales of $8.6 million. Operating expenses for the quarter include $75.2 million in research and development expense to acquire GeneTx and non-cash stock-based compensation of $35.8 million.

Net Loss For the third quarter of 2022, Ultragenyx reported net loss of $245.1 million, or $3.50 per share basic and diluted, which includes $75.2 million in research and development expense related to the acquisition of GeneTx. This compares with a net loss for the third quarter of 2021 of $73.0 million, or $1.08 per share, basic and diluted.

Net Cash Used in Operations Net cash used in operations for the nine months ended September 30, 2022 was $290.1 million.

Cash, Cash Equivalents and Marketable Debt SecuritiesAs of September 30, 2022, cash, cash equivalents, and marketable debt securities were $996.2 million, which includes $491.0 million in net proceeds that was received in July 2022 from OMERS through the sale of a portion of our Crysvita royalty in the profit share territory. Total payments to OMERS are capped at $725 million, which is 1.45 times the purchase price.

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.

Third Quarter 2022 Revenue and Selected Financial Data Tables

Corporate and Program Updates

Crysvita for X-Linked Hypophosphatemia and Tumor Induced Osteomalacia: Amendment to collaboration agreement increases field support after April 2023

In September 2022, we entered into an amendment to our collaboration agreement with Kyowa Kirin Co. Ltd. (KKC) related to the transition of commercialization responsibilities from Ultragenyx to KKC on the transition date in April 2023. Under the terms of the amendment, Kyowa Kirin North America (KKNA) recently began field operations in the U.S. to further support the transition between the two companies and provide additional resources to support commercialization activities ahead of the transition. In addition, the amendment allows Ultragenyx to continue promoting and supporting the Crysvita program in the field in the U.S. until April 2024, which is one additional year after the transition date. During this period, Ultragenyx will continue to share costs of the Crysvita commercial operating expenses with KKC. The amendment strengthens our partnership in driving Crysvita forward with a substantial increase in the total commercial effort after the transition date to optimally support Crysvita’s growth while also allowing more overlap time to assure a smooth and productive transition of responsibilities.

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 $75.4 million, net of cash acquired from GeneTx, in July 2022. We are required to make milestone payments based on certain achievements and royalty payments on net sales.

In July 2022, we also provided an interim data update on patients treated in Canada, the U.K, and the U.S. under each region's amended protocol for the Phase 1/2 study of GTX-102. As of the data cut-off for that update, a total of 11 patients had reached at least the Day 128 evaluation, with three patients reaching the Day 170 Pre-Maintenance Dose (PMD) evaluation. We evaluated patients across various clinical measurements and found encouraging signs of clinical activity including some changes in quantitative measures such as the Bayley-4 expressive and receptive communication that were statistically significantly different from normal variation, even starting at the lowest doses of the titration ramp. The quantitative data also appear to show a dose-dependent effect and that some patients did improve further with longer follow up.

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 U.S. Two of the original five patients treated in the U.S. have now restarted treatment at a Canadian site and, since restarting, have received two or more doses with no treatment-related SAEs of any type nor adverse events related to lower extremity weakness observed in these patients to date.

Dosing in the U.K. and Canada is ongoing with patients continuing to initiate treatment at higher loading doses and to receive treatment in the maintenance phase. Discussions with the FDA to harmonize the U.S. with the Canada/U.K. protocol are ongoing. We expect to begin enrollment of the expansion cohorts in the first half of 2023. We also expect to provide the next data update when we have substantive data on a larger number of patients in the program.

UX143 (setrusumab) for Osteogenesis Imperfecta (OI): Phase 2/3 study dosing patients; Phase 2 data expected in mid-2023Ultragenyx is currently dosing patients in the Phase 2/3 Orbit study of UX143 in pediatric and adult patients with OI aged five to