Agios Pharmaceuticals Inc Investor Breakout Session Transcript

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

Question: Gregory Renza - RBC Capital Markets - Analyst : Great. Thanks. Good day, Brian and team. Congrats again on the update set today, certainly, over the course of this year and thanks for taking my question. Brian, and maybe for you there, of course, and for the doctors on the line, just curious if you could comment a bit on how the related to quality-of-life measures and the FACIT fatigue, of course, correlate to hemoglobin improvement. And do we think that those quality-of-life improvements are driven by the hemoglobin increase or are there other aspects of maybe mitapivat's biology that could also contribute? Thanks. Thanks very much.

Question: Gregory Renza - RBC Capital Markets - Analyst : Got it. Thank you. And maybe just more -- I'll use my follow-up and just with respect to the primary endpoint and the secondary endpoint results, including that quality-of-life data, maybe for the doctors, what percentage of thalassemia patients would you believe could benefit from mitapivat in light of these data? Thanks, again. Ali Taher - Naef K. Basile Cancer Institute, American University of Beirut Medical Center - Professor of Medicine, Hematology & Oncology and Director (multiple speakers) In this data, in non-transfusion dependent thalassemia, these are a group of patients that do not require transfusion regularly. And they might require transfusion every now and then, maybe not all the life, may be few times through the life. But the major issue that we have clearly shown that if they have a low hemoglobin, they will develop most -- at least three morbidities and more during the life of it from adolescence to adulthood. So increasing the hemoglobin is really something which is very important, not only to improve the quality of life, which you could see directly as a key secondary endpoint, but waiting for few more years, you would see maybe that older motor ability it is will decrease and will not be there anymore. So I'm sure that the majority of the NTDT patients were requiring increasing their hemoglobin. And this is not for sure, not those that have a hemoglobin of 10, we're talking about does that with a hemoglobin less than 10. But it remains to be that treating these patients should be a personalized treatment. Each patient is a different patient.

Question: Eric Schmidt - Cantor Fitzgerald - Analyst : Yeah, thanks. And let me ask -- let me add my congratulations as well. It's great to see some real innovation in the field of thalassemia. Maybe a first question for Sarah or the company, it is around the open label expansion, do you have a sense of the number of patients who enrolled and are continuing on therapy in that OLE? without the prior written consent of Thomson Reuters. 'Thomson Reuters' and the Thomson Reuters logo are registered trademarks of Thomson Reuters and its affiliated companies. JUNE 16, 2024 / 2:00PM, AGIO.OQ - Agios Pharmaceuticals Inc Investor Breakout Session

Question: Eric Schmidt - Cantor Fitzgerald - Analyst : That's great to hear. Maybe as a follow-up for doctors Taher and Kuo. it sounds like mitapivat is probably going to get a pretty broad label for Alpha and beta-thalassemia as well transfusion dependent and independent patients. So maybe just can you give us a sense of which of your patients are going to be offered the drug? Which are kind of the best candidates? And conversely, is there a subgroup of patients who you don't think are candidates? Thank you.

Question: Eric Schmidt - Cantor Fitzgerald - Analyst : Thanks, Kevin. Glad to hear that. Ali Taher - Naef K. Basile Cancer Institute, American University of Beirut Medical Center - Professor of Medicine, Hematology & Oncology and Director I follow a large number of NTDT. patient around the, let's say, 200 patients that I've been following over a long period of time, 20 to 22 years. As Kevin said, the majority of this especially, those that are moving into adulthood the majority have at least three to four morbidities. Some of these morbidities are really hard morbidities like pulmonary hypertension and other. So it is really my job to give such a product or such a novel therapy to most of the patients in order to reduce these morbidities or abolish them hopefully, when we do a pediatric study.

Question: Tessa Romero - JP Morgan - Analyst : Hi. Good morning. Thank you so much for taking our questions. So first one from us is what were the reasons for that for [TEAE] from the mitapivat arm that led to treatment discontinuation? Ali Taher - Naef K. Basile Cancer Institute, American University of Beirut Medical Center - Professor of Medicine, Hematology & Oncology and Director (multiple speakers) thrombocytopenia, mild thrombocytopenia increase in liver function tests, abdominal distention, and arthralgia. I think these are the four. So I mean, these are four -- to me minor events, but these were the causes of the reasons for stopping the drug.

Question: Tessa Romero - JP Morgan - Analyst : Okay. And then, the second line or the follow up from us is you have talked about the objective for a broad label and certainly these were adult studies. What is your strategy in pediatrics? Thanks.

Question: Tessa Romero - JP Morgan - Analyst : Okay. Thanks so much for taking our question. without the prior written consent of Thomson Reuters. 'Thomson Reuters' and the Thomson Reuters logo are registered trademarks of Thomson Reuters and its affiliated companies. JUNE 16, 2024 / 2:00PM, AGIO.OQ - Agios Pharmaceuticals Inc Investor Breakout Session

Question: Alec Stranahan - Bank of America - Analyst : Hey, guys. Thanks for taking our questions, and want to just offer my congrats as well on the breadth of the positive data to date come from the thalassemia program. First question, maybe one for Dr. Taher and Dr. Kuo. How do you view the overall convenience of mitapivat in regards to currently available treatments? I guess how would a broad approval of an oral therapy change the way patients are managed from a logistical perspective in either

Question: Alec Stranahan - Bank of America - Analyst : Great. Thanks a lot. All right, great. One more quick follow-up, if I may. Just a regulatory question. How do you think the FDA will assess the totality of the data? Do you think they'll look at each study ENERGIZE and ENERGIZE-T on its own? Or is there maybe some read-across they'll make for, say, biomarkers or the pro end points? Thank you.

Question: Alec Stranahan - Bank of America - Analyst : Great. Thank you.

Question: Divya Rao - Cowen Inc. - Analyst : Hi, guys. This is Divya on for Mark. I will also add my congrats on kind of all the progress and really great data. I just have two quick questions. One is when can we expect a filing in the EU for thalassemia? And my second question is on the data itself. I know competitors have used like PROs tiredness and weakness. I think which is obviously a different endpoint from that of the (inaudible) Can you give us a little bit of color on kind of the difference in criteria and like what it kind of goes into each of those PROs?

Question: Divya Rao - Cowen Inc. - Analyst : And maybe I can ask for that comment as well.

Question: Divya Rao - Cowen Inc. - Analyst : Yeah, that was helpful. So are your competitors have used a PRO, I think if I tiredness and weakness, which is different from the FACIT-fatigue score. Can you give us a little bit of color on the difference in criteria and how each of those are evaluated?

Question: Divya Rao - Cowen Inc. - Analyst : In terms of the evaluation of how each of those are done, what are the criteria and how are they kind of different between the FACIT-fatigue score and the tiredness and weakness PRO?

Question: Divya Rao - Cowen Inc. - Analyst : That's helpful. Thank you.