Agios Pharmaceuticals Inc AQVESME FDA Approval Transcript

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

Question: Samantha Semenkow - Citi Infrastructure Investments LLC - Analyst : Happy holidays. Congratulations to the team for the approval. I just wanted to follow up a little bit. Can you speak a little bit more about the physician target list that you've generated? I'm wondering how many physicians are in that list? And what proportion of those 4,000 addressable patients are managed by these physicians?

Question: Gregory Renza - Truist Securities - Analyst : Brian and team, happy holidays as well, and congrats on the great news. Brian, maybe just with respect to the outcome here on the label and also the details on the REMS program, in light of just the extra time that was utilized by the FDA and for you, your reference of being highly engaged with the agency over the last several weeks and of course, over this filing. Could you just comment a bit about how you're LSEG STREETEVENTS | www.lseg.com | Contact Us consent of LSEG. 'LSEG' and the LSEG logo are registered trademarks of LSEG and its affiliated companies. DECEMBER 24, 2025 / 1:00PM, AGIO.OQ - Agios Pharmaceuticals Inc AQVESME FDA Approval viewing where you all landed up on the label and the REMS program, how it sort of fits your expectations? And what maybe has -- had evolved over the last several weeks to the last few months with the extension that brings you to where you are today?

Question: Eric Schmidt - Cantor Fitzgerald LP - Analyst : My congratulations as well, quite the achievement, many firsts for patients. Maybe first, could you -- once the REMS program is in place in late January, Tsveta, I was hoping you could just sort of walk us through what actually has to happen from the time a start form is in place to shipping the drug to patients, specifically with regard to the steps that the REMS provider and pharmacist and patient need to go through. And then the second question would be on pricing. I noticed there's a modest price discrepancy relative to the price of mitapivat in PKD. How do you police that or ensure no contamination across price utilization?

Question: Alec Stranahan - Bofa Merrill Lynch Asset Holdings Inc - Analyst : Congrats from us on the approval, really great to see. Maybe two quick questions on just the launch dynamic piece. And then I've got a follow-up. I guess as we're modeling the launch, should we assume initial sales will likely happen in 2Q with little to none in 1Q, just given the lead time on the prescription to dose? And I guess, would you say the normalization on the new start to dosing could happen this year? Or is this more of a 2027 aspect for the launch?

Question: Alec Stranahan - Bofa Merrill Lynch Asset Holdings Inc - Analyst : Okay. That's helpful. And then maybe one quick one on sickle cell. I guess given you haven't seen the similar levels of liver AEs in RISE UP, I'm curious how you think about potential labeling if you were to see, say, a conditional approval here. Do you think there's a path to getting sickle cell added to the PYRUKYND label where there's no black box?

Question: Alec Stranahan - Bofa Merrill Lynch Asset Holdings Inc - Analyst : All right. Congrats again on the approval.

Question: Marc Frahm - TD Cowen - Analyst : Congrats on the approval. Maybe just following up on a few of the other questions. Just given there's maybe a bit of a disconnect between prescription demand and the ability to book revenue at least in the early stages, just will you be providing the TRx volumes as part of your kind of quarterly reporting? Is that expectations? And are there other metrics kind of along the certification process that you think are particularly key to -- for investors to be tracking the progress of the launch? And then related on the pricing question, just if you are successful in getting the FDA to ultimately agree to a sickle cell label based on the data, would this branding allow you, you think, also to keep a differentiated price for sickle cell that could potentially be even a bigger gap than the PYRUKYND pricing gap right now?

Question: Marc Frahm - TD Cowen - Analyst : Okay. Congrats again.

Question: Emily Bodnar - HC Wainwright & Co - Analyst : Congrats on the approval. I guess, first one, if you could comment on how long that process for the REMS certification takes with the three elements that you discussed? And also curious on how common liver issues such as cirrhosis are in thalassemia patients. And then maybe if you can comment on ex-US launch process with some of your partners and how we should be thinking about that ramp-up?

Question: Serena Zheng - Goldman Sachs - Analyst : This is [Serena Zheng] for Salveen. Congratulations on the approval. Could you comment on the path of getting from this 4,000 addressable population at launch to the 6,000 target TAM in the longer term and the expected drivers of growth that you believe will contribute to this?

Question: Cassie Yuan - RBC Capital Markets Inc - Analyst : Team, this is Cassie on for Luca. Congratulations on the approval. We have a question for the launch as well. On luspatercept, it generated $117 million and $224 million in revenue in year one and year two post launch in thalassemia alone according to Evaluate Pharma. Is that the right comp for us to think about as we think about the revenue for AQVESME in 2026 and 2027? Or would you advise us against that comp? And if you can also maybe speak to the alpha versus beta thalassemia breakdown in your 4,000 initial targeted population, that would be much appreciated. LSEG STREETEVENTS | www.lseg.com | Contact Us consent of LSEG. 'LSEG' and the LSEG logo are registered trademarks of LSEG and its affiliated companies. DECEMBER 24, 2025 / 1:00PM, AGIO.OQ - Agios Pharmaceuticals Inc AQVESME FDA Approval