Ionis Pharmaceuticals, Inc. CEO Brett Monia said the company remains confident in the Phase III trial design for Wainua (eplontersen) for the treatment of hereditary transthyretin mediated amyloidosis (ATTR) cardiomyopathy, days after rival Alnylam Pharmaceuticals Inc. made changes to the endpoint and timeline of an ongoing Phase III trial testing Amvuttra (vutrisiran) for the same indication.
Key Takeaways
- Ionis responded to investor questions about the Phase III development program for Wainua in ATTR cardiomyopathy, after Alnylam announced changes to the study of a rival drug.
- CEO Brett Monia said the company remains confident in the Phase III CARDIO-TTRansform trial design and the potential of an early data read out in 2025
Alnylam’s announcement on 15 February during the company’s fourth quarter sales and earnings call startled some investors, as the Phase III study, HELIOS-B, had been on track to read out in March or April and will now be delayed until June or July. (Also see "Alnylam Surprises Investors With Last-Minute HELIOS-B Revisions" - Scrip, 15 February, 2024
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