Agios Pharmaceuticals Inc at Barclays Global Healthcare Conference Transcript

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

Question: Gena Wang - Barclays - Analyst : Sounds great. So maybe starting with your PKD program there. How has the interaction with the FDA proceeding in recent weeks since your supplementary NDA just was accepted and a PDUFA was assigned?

Question: Gena Wang - Barclays - Analyst : Any comments regarding potential ADCOM?

Question: Gena Wang - Barclays - Analyst : What could be the latest time that FDA -- that you know that there would not be any ADCOM? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. MARCH 12, 2025 / 4:30PM, AGIO.OQ - Agios Pharmaceuticals Inc at Barclays Global Healthcare Conference

Question: Gena Wang - Barclays - Analyst : Okay. And so maybe regarding the label change, what is in the community, with the increased monitoring, what is the feedback there?

Question: Gena Wang - Barclays - Analyst : Okay. And then regarding the potential timing for approvals in thalassemia in the US, EU, and other parts of the world.

Question: Gena Wang - Barclays - Analyst : Okay. And then for the commercialization, what kind of maybe preparation you have done and then regarding the sales outreach?

Question: Gena Wang - Barclays - Analyst : Okay. And regarding the safety profile, do you think beta-thalassemia and the sickle cell have a different -- quite like a threshold that what patients are willing to take?

Question: Gena Wang - Barclays - Analyst : Okay. So I think that that's a good transition to sickle cell. So maybe the Phase 3 trial, remind us the primary endpoint and the -- what is your goal for the Phase 3 trial?

Question: Gena Wang - Barclays - Analyst : So regarding the primary endpoint, what is the alpha allocation? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. MARCH 12, 2025 / 4:30PM, AGIO.OQ - Agios Pharmaceuticals Inc at Barclays Global Healthcare Conference

Question: Gena Wang - Barclays - Analyst : Okay. And what is your assumption for the control arm?

Question: Gena Wang - Barclays - Analyst : Okay. So basically, 30% reduction?

Question: Gena Wang - Barclays - Analyst : And then for the hemoglobin level, the percentage --

Question: Gena Wang - Barclays - Analyst : Okay. And for the VOE also is 90% power?

Question: Gena Wang - Barclays - Analyst : Okay. And then going to the secondary endpoint. So maybe remind us what you show so far and why you designed the study this way? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. MARCH 12, 2025 / 4:30PM, AGIO.OQ - Agios Pharmaceuticals Inc at Barclays Global Healthcare Conference

Question: Gena Wang - Barclays - Analyst : Okay. And then secondary endpoint, is there any hierarchy there or it will be just even split of the alpha?

Question: Gena Wang - Barclays - Analyst : Okay. That's very good. And then maybe status Phase 3, like the timing --

Question: Gena Wang - Barclays - Analyst : Okay. So maybe going back to the primary endpoint, it seems you have a co-primary endpoint?

Question: Gena Wang - Barclays - Analyst : I see. So as long as you're hitting one, that will be enough for approval.

Question: Gena Wang - Barclays - Analyst : Right. But then if the -- what you define as a positive study as long as you hit one of the primary endpoint, that will be enough or if they want you to hit both?

Question: Gena Wang - Barclays - Analyst : Okay. Just trying to understand, so have you agreed with the FDA like -- did the FDA say anything that one is enough or you want to --

Question: Gena Wang - Barclays - Analyst : Right. Okay, that makes sense. And then was the study design was based on the feedback from the FDA?

Question: Gena Wang - Barclays - Analyst : Okay. Quickly on your AG-946, what kind of data will we see later this year? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. MARCH 12, 2025 / 4:30PM, AGIO.OQ - Agios Pharmaceuticals Inc at Barclays Global Healthcare Conference

Question: Gena Wang - Barclays - Analyst : Okay. Good. And then for your PK franchise, you do have several assets there, and how do you see this coexist in the marketplace?

Question: Gena Wang - Barclays - Analyst : Okay. Good. And Cecilia, you did mention you have the $1.5 billion, that's a lot of cash for biotech company. So how do you, say, from the BD perspective, how would you use this cash?

Question: Gena Wang - Barclays - Analyst : Very good. I think we have one minute left. So maybe which are the key topics you wanted to emphasize and that you think investors were misunderstood? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. MARCH 12, 2025 / 4:30PM, AGIO.OQ - Agios Pharmaceuticals Inc at Barclays Global Healthcare Conference

Question: Gena Wang - Barclays - Analyst : Thank you very much.