Release Date: August 01, 2024
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
Positive Points
- Ultragenyx Pharmaceutical Inc (RARE, Financial) reported strong revenue performance, leading to an increase in total revenue guidance for the year.
- Positive Phase III results from the DTX401 gene therapy for glycosidase Type 1a and long-term positive Phase II results from UX 143 antibody for Osteogenesis Imperfecta.
- Successful completion of an end of Phase II meeting with the FDA for GTX-102 for Angelman syndrome, aligning on Phase III study design and key endpoints.
- Strong commercial performance of Crysvita in the U.S. and Latin America, with significant patient additions and new prescribers.
- Ultragenyx Pharmaceutical Inc (RARE) has a robust cash position with $874 million in cash, cash equivalents, and marketable securities as of June 30, 2024.
Negative Points
- Net loss for the second quarter was $132 million, or $1.52 per share, indicating ongoing financial challenges.
- Total operating expenses were high at $263 million, including significant R&D and SG&A expenses.
- Revenue from Dojolvi and Mepsevii remains relatively low compared to Crysvita, indicating potential reliance on a single product for revenue growth.
- Quarter-to-quarter variability in Latin America revenue due to uneven ordering patterns, which could affect financial stability.
- The company is still in the process of finalizing details for multiple regulatory submissions, which could pose risks if there are delays or issues with the FDA.
Q & A Highlights
Q: Angelman syndrome data update. We also saw quite a few other data updates from both Ionis and Roche. What are your latest thoughts regarding the competitive landscape and the read-through to your trial design, especially if we're looking at the loading dose frequency and endpoints such as expressive communication for Bailey?
A: We've been following the important data updates closely. We had a successful end of Phase II meeting with the FDA and have locked in our plans for Phase III. We have reduced the loading doses from 4 to 3, allowing us to increase our dose and drive to higher doses in a shorter period. We are moving forward with cognition raw scores for our primary endpoint, but expressive communication remains an important secondary endpoint. (Eric Crombez, Chief Medical Officer)
Q: Regarding the Wilson disease data expected in the second half of this year, how many more patients and what kind of data on biomarkers do you expect?
A: There will be data from 15 patients, 5 in each cohort. The focus will be primarily on biochemical data, such as copper levels and distribution. This will help us understand if the gene therapy is working well. (Emil Kakkis, CEO)
Q: Can you help us understand how you look at Osteogenesis Imperfecta (OI) with regard to the Phase II data translating to Phase III? Particularly, how do improvements impact the rate of fractures for the population?
A: The 14-month data showed continued improvements in bone mineral density and a significant reduction in fractures. We expect these improvements to translate well into Phase III. Patients feeling better and becoming more active should support the clinical data and the value of the product. (Emil Kakkis, CEO)
Q: What is your strategy for maximizing GTX-102's product differentiation given the competitive landscape with other ASOs? Have you considered using an Ommaya reservoir for GTX-102?
A: Our data shows long-term improvements and higher potency compared to competitors. We believe we will differentiate on superior efficacy. Regarding the Ommaya reservoir, our goal is to get the drug approved first and then look at ways to make it easier for patients, including potential life cycle management strategies. (Emil Kakkis, CEO)
Q: On Wilson disease, what are the next steps after the interim Stage 1 readout?
A: The interim readout took longer due to patient enrollment and the nature of the transporter for copper. We need at least 6 months of data from the last patient, followed by data cleaning and analysis. We are encouraged by the early data and will continue to plan for Phase III. (Emil Kakkis, CEO)
Q: For setrusumab, do you think there are any benefits this drug could potentially show as it relates to the pain these patients experience?
A: Yes, stronger bones should reduce micro fractures and improve pain. We are evaluating pain, quality of life, and other measures in the study, and we believe setrusumab will be an important therapy for OI. (Emil Kakkis, CEO)
Q: For UX-111, are there any gating factors for the BLA filing later this year or early next year?
A: The main factor is ensuring the CMC is in line. We are working closely with our contract manufacturer and the agency to finalize the details. We expect to get there this year. (Emil Kakkis, CEO)
Q: How do you view the potential impact of Amgen's romosozumab in OI on setrusumab?
A: We believe setrusumab is superior in terms of bone mineral density improvement and fracture reduction. Amgen's dosing is not optimized for OI, and we feel confident in our position. (Emil Kakkis, CEO)
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
