Agios Pharmaceuticals Inc at JPMorgan Healthcare Conference Transcript

The following is excerpted from the question-and-answer section of the transcript.

Question: Tessa Romero - JPMorgan - Analyst : Great. Brian, thank you for the presentation and welcome, everyone. So I thought I would start the conversation in thalassemia. Can you walk us through the framework for how you're thinking about the cadence for potential regulatory regulatory filing acceptances and approvals and timelines to launch? I know we had an acceptance of your filing in the US, but can you just walk us through that?

Question: Tessa Romero - JPMorgan - Analyst : When you think about the use of the product in VAL, what are the key opportunities and potential challenges as they relate to uptake?

Question: Tessa Romero - JPMorgan - Analyst : We have fielded a number of questions since the ASH conference in December about whether hepatocellular injury as a potential risk will or will not hold back use in patients with thalassemia. What have you heard in the early days here from the physician community on the risk benefit trade off? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. JANUARY 15, 2025 / 3:30PM, AGIO.OQ - Agios Pharmaceuticals Inc at JPMorgan Healthcare Conference

Question: Tessa Romero - JPMorgan - Analyst : Okay. I'm just being mindful of the time here. Okay. Let's switch gears to sickle cell, maybe. So as we think about the Phase III portion of RISE UP, were there any protocol changes, statistical plan adjustments that you made on the heels of the Phase II? Or is the, like, statistical test that you're using all kind of remaining the same? And then as a follow up to the question here. Do you need to hit on both of these endpoints to support sNDA at this point?

Question: Tessa Romero - JPMorgan - Analyst : Okay. Why do you think you can hit on annualized rate of SCPCs, which has historically been a really challenging end point?

Question: Tessa Romero - JPMorgan - Analyst : Okay. Last question for me. Is business development a priority for Agios in 2025?

Question: Tessa Romero - JPMorgan - Analyst : Great. Thank you.