Release Date: November 12, 2024
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
Positive Points
- Sangamo Therapeutics Inc (SGMO, Financial) has transformed from a Phase 1/2 company to a pre-BLA company due to significant regulatory developments in its Fabry disease program.
- The company has signed a neurology epigenetic regulation and capsid delivery license agreement with Genentech, receiving $50 million in upfront license fees and milestone payments.
- Sangamo has submitted its first-ever IND application for a neurology indication, marking a significant milestone in its pipeline development.
- The Fabry disease program has a clear regulatory pathway to accelerated approval, potentially reducing the time to approval by three years.
- Pfizer's Phase 3 AFFINE trial for hemophilia A met its primary endpoint, potentially unlocking up to $220 million in regulatory and commercial milestones for Sangamo.
Negative Points
- Sangamo's cash runway is only sufficient to fund planned operations into the first quarter of 2025, absent additional funding from partnerships or milestone payments.
- The company is still in the process of securing partnerships for its Fabry program, which could delay potential revenue streams.
- There is uncertainty regarding the FDA's requirements for the Fabry program, particularly concerning the need for continued statistical significance at 104 weeks.
- The commercial success of the hemophilia A program is uncertain, especially in light of BioMarin's challenges with a similar product.
- Sangamo's financial stability is heavily reliant on successful BLA submissions and potential partnerships, which are not guaranteed.
Q & A Highlights
Q: Regarding the FDA comments on the Fabry program, does the FDA want to see statistical significance of the eGFR slope at 52 weeks compared to baseline? And what does the assessment at 104 weeks mean?
A: (Alexander Macrae, CEO) The FDA has agreed that the eGFR slope at 52 weeks can serve as the primary basis for accelerated approval. There is no requirement for a confirmatory study. The data at 104 weeks will be used to confirm clinical benefits, and we anticipate presenting the two-year data for full approval in 2026.
Q: When can we expect the next data update from the Fabry program?
A: (Nathalie Dubois-Stringfellow, Chief Development Officer) The Phase 1/2 study has become a registrational study, and we expect to have the top-line data in the second quarter of 2025. The last patient visit will be in April 2025, and data cleaning will follow.
Q: Can you comment on the FDA's approach to the Fabry program compared to previous requirements for other companies?
A: (Alexander Macrae, CEO) The FDA's approach has evolved, particularly under Peter Marks at CBER, who is focused on finding paths forward for rare diseases. Our positive eGFR slope data was compelling enough for the FDA to agree to an accelerated approval pathway without requiring a head-to-head trial or additional studies.
Q: How confident are you that Pfizer will support the approval and commercial launch of the hemophilia A therapy?
A: (Alexander Macrae, CEO) Pfizer is in discussions with regulatory authorities, which suggests their continued enthusiasm for the product. We are pleased with the data they will present at the ASH Annual Meeting.
Q: Is the FDA requiring any specific data from female Fabry patients or those with cardiac variants for the endpoint revolving around renal function?
A: (Alexander Macrae, CEO) The FDA discussions have been about Fabry patients in general, not specifically about gender or cardiac involvement. The data set includes a broad range of patients, and improvements have been observed across various measures, including renal function and patient-reported outcomes.
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
