Ultragenyx Pharmaceutical(RARE) - 2024 Q2 - Earnings Call Transcript

Financial Data and Key Metrics - Total revenue for Q2 2024 was $147 million, driven by strong performance across the commercial portfolio [12][13] - Crysvita contributed$114 million, with $67 million from North America,$40 million from Latin America and Turkey, and $6 million from Europe [13] - Dojolvi revenue was$19 million, Evkeeza revenue was $8 million, and Mepsevii revenue was$6 million in Q2 2024 [13] - Operating expenses were 263 million, including R&D expenses of 162 million, SG&A expenses of $81 million, and cost of sales of$21 million [13] - Net loss for Q2 2024 was $132 million or$1.52 per share [14] - Cash, cash equivalents, and marketable securities totaled $874 million as of June 30, 2024 [14] Business Line Performance - Crysvita demand in the US remained strong, with 60$530 million - $550 million, reflecting strong performance across all products [14][15] - The company expects to file two BLAs, provide Phase III data for UX143, and advance the GTX-102 Phase III study over the next 12-18 months [25] - Management highlighted the importance of patient urgency in driving commercial success for gene therapies, particularly for GSDIa [58][59] Other Important Information - The company completed a successful end of Phase II meeting with the FDA for GTX-102, aligning on Phase III study design and key endpoints [6][19] - The company is evaluating life cycle management strategies for GTX-102, including the potential use of lumbar catheter-type devices to improve patient experience [33] - The company is monitoring the potential expiration of the rare pediatric disease voucher program, which could impact future rare disease drug development [74][75] Q&A Session Summary Question: Competitive landscape for Angelman syndrome and trial design for GTX-102 [26] - The company is confident in GTX-102's competitive position, citing superior long-term data and potency compared to competitors [46] - The Phase III trial design includes a reduction in loading doses from four to three, with cognition raw scores as the primary endpoint and expressive communication as a secondary endpoint [27] Question: Wilson disease data update and next steps [36] - The delay in the Wilson disease data update was due to longer-than-expected patient enrollment and the need for at least six months of data from the last patient [36] - The company is focused on biochemical markers, including copper levels, to assess the efficacy of the gene therapy [28] Question: Setrusumab's impact on pain and fracture rates in Osteogenesis Imperfecta (OI) patients [38][40] - Setrusumab has shown significant improvements in bone mineral density and fracture rates, with patients reporting reduced pain and increased activity levels [38][39] - The company expects that increased activity levels will further enhance bone strength, despite the potential for higher fracture risk [41] Question: GTX-102's Phase III trial design and FDA requirements [51] - The FDA agreed to a continuous variable analysis for the Bayley-4 endpoint, with no set threshold for success [52] - The company aims to replicate the Phase II results, which showed significant improvements in multiple domains, including cognition, communication, and behavior [53] Question: Setrusumab's Phase III enrollment and patient types [69][72] - The Phase III Orbit study enrolled more severe OI patients (Type 3 and 4) compared to the Phase II study, with a focus on capturing data across different age groups and disease types [70][72] - The study is well-powered to detect significant fracture reductions, with a target enrollment of 150 patients [72] Question: Rare pediatric disease voucher program and its impact on drug development [74] - The company has benefited from the rare pediatric disease voucher program, generating significant cash from the sale of two vouchers [75] - The company supports the reauthorization of the program, which has been instrumental in advancing rare disease drug development [76] Question: Potential competition from Amgen's romosozumab in OI [78] - The company believes its data for setrusumab is superior to Amgen's Phase II data for romosozumab, citing higher bone mineral density improvements and fracture reductions [79] - The company is confident in its competitive position, given the optimized dosing and superior efficacy of setrusumab [80]