On August 2, 2021 Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, reported its financial results for the second quarter 2021 and reaffirmed its financial guidance for 2021 (Press release, Ultragenyx Pharmaceutical, AUG 2, 2021, View Source [SID1234585538]).
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"In the first half of 2021 we have advanced all our commercial, clinical, and regulatory priorities. This has led to meaningful revenue growth in the second quarter and has positioned us well as we enter a period of significant execution on our clinical programs in the second half of this year," said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. "We have one of the most robust late-stage pipelines in both gene therapy and other biologics for rare diseases and are on track to initiate four pivotal clinical trials over the next six months."
Second Quarter 2021 Financials
In the second quarter 2021, Crysvita revenue in Ultragenyx territories1 increased 38% versus the second quarter 2020. This increase is driven by demand from pediatric and adult patients with X-linked hypophosphatemia (XLH) and patients with tumor induced osteomalacia (TIO), which became Crysvita’s second approved indication in the United States in June 2020.
Dojolvi revenue in the second quarter 2021 continued to build on the launch momentum after the U.S. Food and Drug Administration (FDA) approval in June 2020. As of the end of the second quarter 2021, the company has received approximately 270 completed start forms from approximately 130 unique prescribers. This has led to approximately 220 patients on reimbursed therapy as of the end of June 2021.
Second quarter 2021 revenue included $22.0 million related to the technology transfer as part of the Daiichi Sankyo strategic manufacturing partnership around the HeLa PCL and HEK293 technologies. This revenue is expected to taper significantly through the end of this year as these activities come to a close.
Total operating expenses of $169.8 million in the second quarter 2021 increased 36% or $45.0 million versus the second quarter 2020, primarily driven by pipeline advancements including four registrational studies. For the year, total operating expenses are expected to increase modestly as the company continues the commercial launch of Dojolvi and supports six clinical programs, including four pivotal studies.
Net cash used in operations for the six months ended June 30, 2021 was $224.7 million, compared to net cash used of $7.8 million for the same period in 2020 which included approximately $135 million of operating cash received in 2020 from Daiichi Sankyo related to the collaboration and license agreement. Cash, cash equivalents, and marketable debt securities were $973.8 million as of June 30, 2021.
2021 Financial Guidance
Crysvita Guidance in Ultragenyx Territories
The company reaffirms the 2021 guidance range for Crysvita that was provided at the beginning of the year. This range is $180 million to $190 million and includes the North American profit share region and the other regions where product sales are recognized.
Second Quarter 2021 Revenue and Selected Financial Data Tables
Program Updates and Upcoming Milestones
Crysvita for the treatment of X-Linked Hypophosphatemia, or XLH
The European Commission (EC) approved Crysvita for self-administration as a treatment option when recommended by the treating physician, for pediatric and adult patients with XLH.
GTX-102 for the treatment of Angelman Syndrome, partnered with GeneTx
Health Canada (HC) cleared a protocol amendment and the U.K. Medicines and Healthcare Products Regulatory Agency (MHRA) approved a Clinical Trial Application to initiate the Phase 1/2 study in Canada and the U.K, respectively.
Following a productive meeting with the FDA, GeneTx submitted an amended protocol. The companies have received feedback on the amended protocol and will make additional revisions in order to resume the study in the U.S.
The Phase 1/2 study is expected to begin in the U.K. and Canada in the second half of 2021, with early clinical data from some patients in the study expected before the end of the year.
DTX401 for the treatment of Glycogen Storage Disease Type Ia, or GSDIa
Longer-term Phase 1/2 data were presented at the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 2021 Annual Meeting demonstrating a durable response from all nine patients lasting up to more than 2.5 years following treatment with DTX401.
Operations are underway to initiate a Phase 3 study with a 48-week primary efficacy analysis period with the plan to enroll approximately 50 patients 8 years of age and older, randomized 1:1 to DTX401 (1.0 x 10^13 GC/kg dose) or placebo. The coprimary endpoints are the reduction in oral glucose replacement with cornstarch while maintaining or improving glucose control assessed by continuous glucose monitoring.
The first patient in the Phase 3 study is expected to be dosed in the second half of 2021.
DTX301 for the treatment of Ornithine Transcarbamylase, or OTC, Deficiency
Longer-term Phase 1/2 data were presented at the ASGCT (Free ASGCT Whitepaper) 2021 Annual Meeting showing a response from all three patients at the Phase 3 dose and a total of 6 of 9 responders in the first three cohorts of patients enrolled. This response included durable metabolic control lasting up to more than three years following treatment with DTX301 in patients with OTC deficiency.
Operations are underway to initiate a Phase 3 study that will include a 64-week primary efficacy analysis period and enroll approximately 50 patients 12 years of age and older, randomized 1:1 to DTX301 (1.7 x 10^13 GC/kg dose) or placebo. The co-primary endpoints are the percentage of patients who achieve a response as measured by discontinuation or reduction in baseline disease management and 24-hour plasma ammonia levels.
The first patient in the Phase 3 study is expected to be dosed in the second half of 2021.
UX701 for the treatment of Wilson Disease
Operations are underway to initiate a seamless, single-protocol Phase 1/2/3 study. The first part of the study will enroll approximately 27 patients (nine per cohort), randomized 2:1 to DTX701, manufactured using the company’s proprietary HeLa 2.0 producer cell line (PCL) process at the 2,000 liter scale, or placebo. The dose cohorts will be enrolled sequentially using ascending doses. The patients will be followed for 52 weeks before transitioning to long-term follow-up and selecting a pivotal dose. The dose will be determined based on the safety profile, changes in biomarkers of copper metabolism, and the reduction in the use of the current standard of care.
The first patient in the Phase 1/2/3 study is expected to be dosed in the second half of 2021.
UX053 for the treatment of Glycogen Storage Disease Type III, or GSDIII
UX053 was granted Orphan Drug Designation (ODD) by the FDA and European Medicines Agency (EMA), highlighting the significant unmet need for patients with GSDIII.
The first patient in the Phase 1/2 study is expected to be dosed in the second half of 2021.
1: Ultragenyx territories include the collaboration revenue from the North American profit share territory and other regions where revenue from product sales are recognized by Ultragenyx. This excludes the European territory revenue, which is recognized as non-cash royalty revenue since the rights were sold to Royalty Pharma in December 2019.
Conference Call and Webcast Information
Ultragenyx will host a conference call today, Monday, August 2, 2021, at 2 p.m. PT/ 5 p.m. ET to discuss the second quarter 2021 financial results and provide a corporate update. The live and replayed webcast of the call will be available through the company’s website at View Source To participate in the live call by phone, dial (855) 797-6910 (USA) or (262) 912-6260 (international) and enter the passcode 3654725. The replay of the call will be available for one year.