Agios Pharmaceuticals Inc Q4 2024 Earnings Call Transcript

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

Question: Eric Schmidt - Cantor Fitzgerald - Analyst : Congrats on all the progress looking forward to another remarkable year in 2025. Maybe first for Sarah, what's the company's plan or strategy for updating the investment community on the safety profile of mitapivat should there be any further cases of hepatocellular injury or any signals whatsoever there? And then for Brian, now for probably the past year or so, I think you've been talking about the multibillion-dollar potential of mitapivat in these additional indications. How do you think about peak sales potential in either thalassemia or sickle cell disease as you construct that multibillion-dollar estimate?

Question: Divya Rao - TD Cowen - Analyst : This is Divya on for Mark. Congrats on all the progress. Just two, one on sickle cell. So after the liver tox disclosure in Dow last year, can you provide any details on changes to the sickle cell trial protocol? Is it mostly in the OLE portion? Or were there any changes to the core portion of the trial? And then I have a follow-up.

Question: Divya Rao - TD Cowen - Analyst : That's helpful. And then with tebapivat being explored in sickle cell, how should we think about the development path there? Is this more of like a proof of concept before moving into other diseases? Or if the trial is successful, do you actually plan to move forward into pivotal development in sickle cell with this program?

Question: Gregory Renza - RBC Capital Market - Analyst : Great. Brian and team, congrats on the progress in the year. Brian, maybe just dipping into the pipeline and as you get your hand on the PK activation portfolio, I just wanted to ask about just your enthusiasm on how you're characterizing your level of enthusiasm with respect to AG-181 that the PAH stabilizer. Maybe just remind us of that specific mechanism, how it stabilizes PAH and perhaps why it should work well with patients who don't respond to that co-factor treatment?

Question: Gregory Renza - RBC Capital Market - Analyst : That's helpful. And maybe just a broader question, taking a step back. As you look at your potential cash deployment internally and of course, externally, how are you seeing the market for external assets, especially in light of what is a rather active market, not just domestically, but also globally.

Question: Greg Harrison - Scotiabank - Analyst : Congrats on the progress. Thinking about how you think investors should be modeling the launch trajectory in mitapivat in thalassemia in maybe in 2026, understanding there will be some time to get access and lag time before initiating therapy in 4Q. But would you expect there to be pent-up demand or an initial bolus in some of these patient groups that you discussed as the initial launch focus?

Question: Greg Harrison - Scotiabank - Analyst : That's helpful. I also wanted to ask about the Gulf region and the launch there. Are you getting a better handle on the commercial potential in the region? And how should investors be thinking about that opportunity?

Question: Chris Raymond - Piper Sandler - Analyst : Two questions. First, I guess, on the PD studies in PKD, I think you've characterized ACTIVATE Kids and ACTIVATE Kids T as giving insights into future pediatric work in thal and sickle cell. Can you maybe elaborate a little bit on this benefit? Would you expect some perhaps short circuiting. I guess, of a pediatric program there? Or would there potentially be labeling benefits sort of out of the gate? -- just from this work? And then the second question, I know you've had this question a number of times, but just on the PDUFA and a potential panel. Just with all this focus on liver injury, are you -- companies rarely hope for a panel, but it would seem that with all this attention that's on this, this actually might be beneficial just to sort of put some light on this. Any thoughts there on your desire there on a panel or not?

Question: Alex Shanahan - Bank of America - Analyst : On the updates from us as well and look forward to a busy 2025 ahead. Just two from us. Thinking about the process of scaling your sales force for thal, given the ENERGIZE studies have both been presented at major medical meetings, what is your sense of the level of awareness amongst physicians as well as patient advocacy groups? Is this kind of tracking with your hopes? Or is there maybe more work to do there? And has this fed into the number of reps you've hired for commercialization or not really?

Question: Alex Shanahan - Bank of America - Analyst : And then just a quick one on pediatric PKD. Curious if you -- just to put a finer point, if you intend to seek approval in both transfusion populations, the regularly and not regularly transfused. And whether you think approval across the patient groups could be warranted, maybe given the totality of the data across the two studies.

Question: Tessa Romero - JP Morgan - Analyst : So just a brief financial question from us to start. Cecilia, in terms of your OpEx, what is the right way to think about the evolution of your SG&A expenses and the cadence of a ramp-up there over 2025 and into 2026. And on the R&D side, any additional color you can give us there? And then I have one follow-up.

Question: Tessa Romero - JP Morgan - Analyst : Okay. And can you quantify what growth means?

Question: Tessa Romero - JP Morgan - Analyst : Okay. And then within that from a timeline perspective can you lay out for region beyond the US how you are thinking about timing of approval? So I'm thinking about the EU, Saudi Arabia and the UAE? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. FEBRUARY 13, 2025 / 1:00PM, AGIO.OQ - Q4 2024 Agios Pharmaceuticals Inc Earnings Call