The following is excerpted from the question-and-answer section of the transcript.
(Questions from industry analysts are provided in full, but answers are omitted - download the transcript to see the full question-and-answer session)
Question: Eric Schmidt - Cantor Fitzgerald - Analyst
: Thank you for the opportunity to ask questions. Maybe first just on the thalassemia review process, it's been almost two months now since you've
got the PDUFA extension. Do you have a better sense of what type of a REMS program the FDA is interested in here? I know there's a spectrum of
maybe more or less onerous REMS programs.
And then just a second follow-up question. I think both Cecilia and Brian mentioned actively looking for external assets. What type of BD makes
sense for the company at this stage? And would you wait until the sickle cell readout to transact?
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OCTOBER 30, 2025 / 12:00PM, AGIO.OQ - Q3 2025 Agios Pharmaceuticals Inc Earnings Call
Question: Eric Schmidt - Cantor Fitzgerald - Analyst
: Thanks. Good luck with the upcoming readouts.
Question: Alec Stranahan - BofA Securities - Analyst
: Hey, guys. Thanks for taking our questions. Just a couple from us. It's been interesting to see how the different geographies have approached the
risk of liver injury for mitapivat in thalassemia. Maybe could you just remind us what sort of liver monitoring requirements are so far being required
in Saudi and potentially in EU if it's approved as well? And do you think this could change depending on the US label? And then maybe second, as
a follow-up, curious how this monitoring requirement is changing maybe your commercial approach in these different areas. Thank you.
Question: Marc Frahm - TD Cowen - Analyst
: Hi. Thanks for taking my questions. Just back on the liver events, can you maybe just as you've continued to dose people and follow them for longer
and longer, just kind of comment on if any additional events have been observed kind of across the PKR trials? And to the extent any have, have
they continued to conform to the kind of description before of occurring within six months and importantly, the patient returning to baseline
whenever they do stop mitapivat as a result?
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OCTOBER 30, 2025 / 12:00PM, AGIO.OQ - Q3 2025 Agios Pharmaceuticals Inc Earnings Call
And then maybe on the commercial side, I recognize it will take quite a while to work through case-by-case access and actually get to revenue for
any of those patients, let alone the reimbursement negotiations. But maybe can you comment on just any level of demand you're already seeing
in the Gulf to kind of start that process?
Question: Tessa Romero - JPMorgan - Analyst
: Hey, Brian and team. Thanks so much for taking our questions. So for the Phase 3 portion of RISE UP, has the last patient exited the trial yet? And
what are any considerations in terms of the steps and timing to get to your top line? And then I have a follow-up.
Question: Tessa Romero - JPMorgan - Analyst
: Okay. So you can't tell us if your top line will be before or after your PDUFA date?
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