Release Date: February 13, 2025
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
Q: What is Agios Pharmaceuticals' strategy for updating the investment community on the safety profile of Mitapivat, especially concerning hepatocellular injury? A: Sarah Gheuens, Chief Medical Officer, stated that if there are any changes to the safety profile of Mitapivat, the company will update the investment community as they have done in the past when new safety information became available.
Q: How does Agios Pharmaceuticals view the peak sales potential for Mitapivat in thalassemia and sickle cell disease? A: Brian Goff, CEO, expressed confidence in the multi-billion-dollar potential of Pyrukynd, citing the significant unmet need in thalassemia, where two-thirds of the U.S. patient population has no approved therapy, and the high unmet need in sickle cell disease, which has increased due to limitations in available therapeutic options.
Q: What changes were made to the sickle cell trial protocol following the liver toxicity disclosure? A: Sarah Gheuens explained that monitoring for liver enzymes was already part of the core period of trials. The open-label extension (OLE) portion was adjusted to align with the core period's monthly monitoring for the first six months of exposure.
Q: How does Agios Pharmaceuticals plan to approach the development of Tepapivat in sickle cell disease? A: Brian Goff and Tsveta Milanova, Chief Commercial Officer, indicated that they are building a sickle cell disease franchise with both Pyrukynd and Tepapivat. The development path will be guided by data from ongoing studies, and they aim to provide multiple treatment options for this complex disease.
Q: How should investors model the launch trajectory for Mitapivat in thalassemia, considering potential pent-up demand? A: Tsveta Milanova noted that while there is a focus on educating and increasing urgency among physicians, they do not expect an initial bolus of patients. The launch will target about 65% of the U.S. thalassemia patient population, capturing patients as they visit their doctors.
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
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