Release Date: May 02, 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 positive interim data from the Phase 1/2 study of GTX-102 in Angelman Syndrome, showing significant improvement in patient development.
- The company completed enrollment for the Phase 2/3 Orbit study and Phase 3 Cosmic study in Osteogenesis Imperfecta, indicating accelerated interest and support from the study investigators.
- Ultragenyx Pharmaceutical Inc (RARE) demonstrated a robust safety profile for GTX-102, with manageable lower extremity weakness observed in only a small fraction of patients.
- Crysvita's performance in North America remains strong with a significant increase in new prescribers and patient start forms, indicating growing market penetration and demand.
- Ultragenyx Pharmaceutical Inc (RARE) reaffirmed its 2024 revenue guidance, expecting significant growth and projecting total revenue to be between $500 million and $530 million.
Negative Points
- The company reported a net loss of $171 million for the first quarter of 2024, highlighting ongoing financial challenges.
- Operating expenses for Ultragenyx Pharmaceutical Inc (RARE) were high, totaling $274 million for the quarter, which included substantial R&D and SG&A expenses.
- The company experienced a larger use of cash in operations during the first quarter, amounting to $191 million, primarily due to annual bonuses and a high accounts receivable balance.
- Ultragenyx Pharmaceutical Inc (RARE) faces uncertainties regarding the finalization of the Phase 3 trial design and endpoint selection for GTX-102, which could impact regulatory approval timelines.
- Despite strong growth projections, Ultragenyx Pharmaceutical Inc (RARE) continues to navigate the complexities of transitioning manufacturing processes in-house, which could affect the timing of future product filings and launches.
Q & A Highlights
Q: Could you remind everyone what is required in terms of the primary endpoint to reach a positive study for the GSDIa program?
A: Emil D. Kakkis, CEO of Ultragenyx, explained that the primary endpoint for the GSDIa program is the reduction in cornstarch utilization, comparing the treated group to the placebo group, with the requirement of maintaining good glucose control. He also discussed the transition of manufacturing to their plant and the upcoming discussions with the FDA to finalize the filing strategy.
Q: Regarding the GTX-102 update, what are the most misunderstood or divergent points of feedback from the Street compared to physician or KOL feedback?
A: Emil D. Kakkis noted that there is a misunderstanding about the significance of changes in the Bayley scores and the safety concerns related to lower extremity weakness. He emphasized that the changes are meaningful and the safety issues are manageable, which is supported by feedback from treating physicians and regulatory bodies.
Q: For the Orbit and Cosmic study for OI, will you submit for different age groups as part of a single application?
A: Emil D. Kakkis confirmed that both studies are expected to read out around the same time and that the plan is to file for the full age range based on that data, as it would be challenging to submit for one part of the age range and not the youngest.
Q: Regarding the GTX-102 Phase 3 trial design, what will be your single domain for the primary endpoint?
A: Emil D. Kakkis indicated that the primary endpoint for the GTX-102 Phase 3 trial is likely to be the Bayley cognition score, which integrates multiple improvements, is rapidly improving, and is a validated tool known to the FDA.
Q: Can you provide more details on how you're assessing changes in global copper metabolism for Wilson's disease?
A: Emil D. Kakkis and Eric Crombez discussed measuring free copper in urine and ceruloplasmin oxidase activity as main ways to assess copper metabolism. They also mentioned conducting a liver biopsy sub-study to examine copper concentration and histopathology.
Q: What would you view as a positive outcome from the end of Phase 2 meeting with the FDA for Angelman?
A: Emil D. Kakkis expressed that a positive outcome would involve finalizing the endpoint, palette of endpoints, and statistical approach, ensuring the study is positioned for approval. He mentioned that the primary focus would likely be on finalizing endpoints with the FDA.
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
