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: Gregory Renza - RBC Capital Market - Analyst
: Congrats on the progress. Brian, it's really helpful to hear your confirmation about no advisory committee for the September PDUFA. I'm just curious
if you can comment on, first, has that mid-cycle review happened? I think it should have already occurred. Just wanted to get clarity on that.
And secondly, what are the next steps? As you mentioned, the engagement with FDA has been strong as you think about labeling and the next
steps of the late cycle. Thanks so much.
Question: Gregory Renza - RBC Capital Market - Analyst
: And maybe a quick follow-up. Just broadly with the sickle cell community and as you think about especially tebapivat and the enrollment there,
how has the Oxbryta wean off the current market conditions? How is that, in your view, impacted the recruitment of trials, the sickle cell community
as they sort of transition and await for new options in the marketplace?
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MAY 01, 2025 / 12:00PM, AGIO.OQ - Q1 2025 Agios Pharmaceuticals Inc Earnings Call
Question: Alec Stranahan - Bank of America - Analyst
: Congrats on the progress in the quarter. Maybe first, since you alluded to it a couple of times, maybe you could just remind us about your plans
for launching mitapivat ex-US. When could these approvals come through for that?
And do you think it would make sense to try and keep the economics in-house by leaving the launches yourself given the strong balance sheet?
And then I've got a follow-up.
Question: Alec Stranahan - Bank of America - Analyst
: And then maybe one, great to see Krishnan joining the team. Curious if bringing him on board to lead corporate strategy represents any shifts in
kind of the way you're thinking or allocating resources going forward, either through increased BD or areas of pipeline focus?
Question: Divya Rao - TD Cowen - Analyst
: This is Divya on for Mark. I'll add my congrats on all the progress. Just one question from us. Have you seen any changes to your communication
frequency with the FDA given the recent reshuffling that's been happening at the agency? And then, is there a deadline for when the FDA needs
to inform you of a potential Ad Comm?
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MAY 01, 2025 / 12:00PM, AGIO.OQ - Q1 2025 Agios Pharmaceuticals Inc Earnings Call
Question: Hiromichi Nagayumi - Cantor Fitzgerald - Analyst
: This is Hiro on behalf of Eric Schmidt here at Cantor. I wanted to ask a bit about the rationale and conviction for starting the Phase 2 tebapivat study
in sickle cell in mid-2025 prior to the Phase 3 readout of PYRUKYND in late 2025.
Question: Emily Bodnar - H.C. Wainwright & Co., LLC - Analyst
: I guess for thalassemia, maybe if you can touch a bit on your plans for marketing for non patients compared to
transfusion-dependent patients, particularly on the non-transfusion side since those patients currently don't have any treatments?
Question: Amanda Acosta-Ruiz - Leerink Partners - Analyst
: This is Amanda on for Andy. It seems that you're starting to discuss more tebapivat more frequently in sickle cell disease and are slated to start the
Phase 2. Is there any color that you can provide on kind of differences on how you're thinking of like patients going into that study and the tebapivat
study versus RISE UP or any endpoints, differences there that might be a focus?
Any learnings that you're taking into this new trial? And also, have tebapivat shown any signs of impacting the liver in these early studies? Or how
are you thinking about that?
Question: Tessa Romero - JP Morgan - Analyst
: Double-clicking back to a prior question, can you confirm or not if you have completed the mid-cycle meeting? And if so, can you comment on
any high-level discussion you have had around labeling? And is there a scenario where REMS is needed? Or can you rule this out at this point?
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MAY 01, 2025 / 12:00PM, AGIO.OQ - Q1 2025 Agios Pharmaceuticals Inc Earnings Call
Question: Lydia Erdman - Goldman Sachs - Analyst
: This is Lydia on for Salveen. Congrats on all the progress. Maybe just another one on the potential thalassemia launch. Could you just comment
on any anticipated evolution of PYRUKYND pricing in the context of payer feedback, the existing price in PKD, and the patient population here?
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