Agios Pharmaceuticals at Bank of America 2025 Healthcare Conference: Strategic Pipeline Advances

On Wednesday, 14 May 2025, Agios Pharmaceuticals (NASDAQ:AGIO) presented at the Bank of America 2025 Healthcare Conference, providing insights into its strategic initiatives and pipeline advancements. The company shared positive developments, including promising Phase 3 results for thalassemia, while also addressing challenges like hepatocellular injury in trials. Agios is poised for growth with a strong financial foundation and a focus on expanding its PyraKine franchise.

Key Takeaways

• Agios announced positive Phase 3 results for thalassemia, with regulatory filings underway and an FDA PDUFA date set for September 7th.
• The company is financially robust, ending Q1 with $1.4 billion, supporting its growth and expansion plans.
• Agios is preparing for the potential launch of PyraKine in thalassemia, expanding its sales force and focusing on disease education.
• The Rise Up Phase 3 study for sickle cell disease is fully enrolled, with data expected by year-end.
• Agios is prioritizing US market entry for thalassemia, with partnerships planned for international expansion.

Financial Results

• Cash Position: Agios concluded the first quarter with approximately $1.4 billion, providing a solid base for disciplined capital allocation.
• Revenue & Growth: While specific revenue figures were not disclosed, the company emphasized the potential for PyraKine to become a multibillion-dollar franchise.
• Capital Allocation: Agios is focused on successful product launches, advancing its mid-stage pipeline, and exploring business development opportunities.

Operational Updates

• Regulatory Progress: The FDA has accepted Agios’ application for thalassemia, with a PDUFA date of September 7th. Filings in Europe, Saudi Arabia, and the UAE are progressing as expected.
• Commercial Readiness: The commercial team is prepared for the thalassemia launch, with the sales force expanded from 20 to 40 representatives.
• Clinical Trial Updates: The Rise Up Phase 3 study for sickle cell disease is fully enrolled, with results anticipated by year-end. A new trial for tebipivat is set to begin mid-year.
• Geographical Expansion: Agios is prioritizing the US market for thalassemia and has partnered with Newbridge for commercialization in the Gulf region.

Future Outlook

• Regulatory Milestones: Agios is awaiting an FDA decision on thalassemia by September 7th and regulatory outcomes in Europe, Saudi Arabia, and the UAE.
• Clinical Milestones: Data from the Rise Up Phase 3 study in sickle cell disease is expected by the end of the year, with further trials for tebipivat planned.
• Strategic Focus: The company is committed to building a sustainable franchise in red blood cell disorders, balancing capital allocation between commercialization and pipeline development.

Q&A Highlights

• Thalassemia Approval: The FDA review process is proceeding smoothly, with no significant issues reported. The absence of an advisory committee is viewed positively.
• Sickle Cell Disease Strategy: Agios aims to address the significant unmet needs in sickle cell disease, advocating for multiple treatment options in the market.
• Competitive Landscape: Mitapivat’s oral therapy is positioned as an accessible alternative to gene therapy, with Agios welcoming increased investment in sickle cell disease treatment.

For further details, readers are encouraged to refer to the full transcript below.

Full transcript - Bank of America 2025 Healthcare Conference:

Alex Stranahan, Senior Biotech Analyst, Bank of America: Everyone. Thanks for joining this session with Agios Pharmaceuticals, and welcome to the first session of day two of the twenty twenty five Bank of America Healthcare Conference. My name is Alex Stranahan. I’m senior biotech analyst covering Agios at Bank of America, and pleased to be joined today by Brian Goff, chief executive officer, and Sarah Gruens, chief medical officer of Agios. Thanks for being here, guys.

Brian Goff, Chief Executive Officer, Agios Pharmaceuticals: Thanks a lot, Al. It’s great to be back with you again. I can’t believe it’s just been a year.

Alex Stranahan, Senior Biotech Analyst, Bank of America: I know. It feels like we’ve had a year’s worth of news in four months already.

Brian Goff, Chief Executive Officer, Agios Pharmaceuticals: Yes.

Alex Stranahan, Senior Biotech Analyst, Bank of America: So it’ll be interesting to see what the second half has in store. But, you know, Brian, maybe maybe just we can start off, you know, Adios, what’s going on at the company? What are the main focus areas for you guys? And what’s sort of the, you know, the second half outlook in terms of things to be Yeah.

Brian Goff, Chief Executive Officer, Agios Pharmaceuticals: Happy to. And and, again, thanks for hosting us, and thanks a lot to everybody who has joined us to hear the Agios story. It’s a really exciting time. And if you’re new to Agios, I’ll just start by saying our mission is to develop and deliver medicines that have transformative potential for patients who are living with rare diseases. And and the real focus of that effort are rare diseases that involve the destruction or the dysfunction of red blood cells.

Namely pyruvate kinase deficiency or PKD, thalassemia, sickle cell disease, and low risk MDS. And and the real highlight of our pipeline is a product called PyraKine or Mitapivat. And mitapivat involves a very novel mechanism of action targeted towards improving the metabolism of red blood cells, which ultimately leads to more energy available and a healthier cell. And then pyrokind is currently commercialized for PKD or, again, pyruvate kinase deficiency, but we are pursuing indications in thalassemia and sickle cell disease, and I’m sure we’ll have a great discussion about those pursuits today. We had a really transformative year in 2024, and and that’s why I I can’t imagine how how fast this year has gone by and how much we’ve accomplished.

Last year, we were delighted to announce the results of two pivotal phase three studies for thalassemia. They’re known as ENERGIZE and ENERGIZE T, And, we delivered statistical significance on all of the primary and all key secondary endpoints. That put us in a great position where, December of last year, we announced that we had simultaneously filed for regulatory approval in four different markets, in The US, in Europe, and in The Gulf in both Saudi Arabia and United Arab Emirates. And then we had a great start to this year where, we announced in January that the FDA had accepted our application and granted a PDUFA goal date of September seventh of this year, which is a side note, is less than four months away now. So we’re very excited about that opportunity.

Our commercial team has really geared up, and I would say at this point is fully ready for this exciting launch opportunity of pyrokinin thalassemia. And then the other big event from last year is the focuses on sickle cell disease. And in October, we were really pleased to announce that we had fully enrolled our rise up phase three study in sickle cell disease. It’s a fifty two week study. So fast forward, that’s another really important catalyst for us later this year.

We expect to read out that data by year end. The other thing I’ll just mention that that happened last year is we significantly strengthened our balance sheet, and we’re in a great position, particularly in this macroeconomic environment. We ended the first quarter of this year with approximately $1,400,000,000 on the balance sheet. So it’s been a it’s been a great year. And Sarah and I would be delighted to talk about all of that progress and more.

The last two things I’ll just say is, again, on the on the balance sheet perspective, in this environment, we really think we’re in a position of strength, and we see the the strength of our balance sheet as an opportunity for us to remain disciplined in capital allocation, but really create significant value for shareholders, most important of which is our journey to build a multibillion dollar franchise with PyroKine. And then one point of pride for the organization is we talk a lot internally about being fueled by connections. That really, means that we stay very close to the patient communities. We’re very passionate about the work that we’re doing. And I think, frankly, that’s contributed to our performance, and it also certainly fuels the culture internally at Agios.

So plenty more to say, but I’ll stop there and would welcome your questions.

Alex Stranahan, Senior Biotech Analyst, Bank of America: Okay. Fantastic. So definitely, several irons in the fire. Maybe first we can talk about thalassemia. Sure.

PDUFA date, September 7, like you said. Every everything’s still on on track here or any feedback from the agency you received on on timing? Yeah.

Brian Goff, Chief Executive Officer, Agios Pharmaceuticals: I’ll let Sarah start on that one. It’s been, so far a good journey.

Sarah Gruens, Chief Medical Officer, Agios Pharmaceuticals: So far so good. Feels like a very normal procedure for us with questions coming in and us providing the answers. It’s our PDUFA date is September 7, so we’re very anchored to that date and very excited about that getting closer.

Brian Goff, Chief Executive Officer, Agios Pharmaceuticals: And we maybe I’ll just add too that one of the updates that we offered on our earnings call is the first quarter of the year, our messaging was that the FDA has been silent. We haven’t heard anything with respect to an advisory committee. The update we provided a couple weeks ago is the FDA has now communicated that at this time, they’re not planning an advisory committee, but, of course, the review is ongoing for thalassemia. I guess given

Alex Stranahan, Senior Biotech Analyst, Bank of America: that this would be maybe potentially the first broad approval across thalassemia subtypes, was an AdCom maybe expected or was that kind of factored into your review thought process?

Sarah Gruens, Chief Medical Officer, Agios Pharmaceuticals: We did not expect an AdCom or had a wish

Alex Stranahan, Senior Biotech Analyst, Bank of America: for an AdCom I

Sarah Gruens, Chief Medical Officer, Agios Pharmaceuticals: think the story Brian highlighted it, Yes, it’s the first indication for a broad population of thalassemia that was submitted, but the clinical trial data is clear. We have on the efficacy side a very clear and clean story with statistical significance on all primary and key secondary endpoints across the two trials. The subgroup analyses highlight that everybody stands to potentially respond to the drug. From that side, it’s pretty straightforward story. Then on the safety side as well, we have a clear safety profile.

As you know, we’ve updated our safety profile with hepatocellular injury, that as well is an event that you can monitor for and then it’s manageable because you can discontinue the drug. The labeling language that we’ve proposed around that is also clear. And the benefit risk profile therefore in the overall thalassemia population is from our perspective, favorable. So in that context, I think in AdCom, I don’t know. It seems like a like a lot.

Alex Stranahan, Senior Biotech Analyst, Bank of America: Yeah. And especially having the approval in in PK PKU. Helps.

Brian Goff, Chief Executive Officer, Agios Pharmaceuticals: Yeah. I mean, this this is an sNDA, and we have now even commercialization wise three years plus of experience with pyrokin in pyruvate kinase deficiency and multiple late stage trials across multiple hemolytic anemias. So we really feel like we’re in a position of strength. Okay.

Alex Stranahan, Senior Biotech Analyst, Bank of America: Very good. And appreciate this is dynamic and influx almost daily, but any high level thoughts on FDA staffing, ability to meet approval deadlines? We haven’t seen too many myths, maybe one or two, but those could be one offs. And I know that Makary has reiterated that they feel that they’re fully staffed. I don’t think they’ve let any of the reviewers really go in this transition, but any changes or color from your recent interaction, making sure?

Sarah Gruens, Chief Medical Officer, Agios Pharmaceuticals: No. Not that we have noticed. I think this really, from our perspective, feels like a normal procedure with, you know, back and forth across the whole spectrum of the filing. So there is not a topic that seems to be forgotten or neglected. Or so from our perspective, it feels really like a business as usual.

And so therefore, we are continuing with that same mindset and are very much looking forward to September 7.

Brian Goff, Chief Executive Officer, Agios Pharmaceuticals: Yeah. And and maybe I’ll just add. We’re talking about FDA, right now, understandably. But again, we filed in four markets simultaneously, and you could add the same sentence to all of those, regulatory engagements. They’ve all continued at pace and as expected.

And again, we’re just really excited about having the chance for not just one having approval for one segment of thalassemia, but this is a high unmet need population with very limited choices. And so what we’re what we’re pursuing is alpha thalassemia as well as beta thalassemia and both nontransfusion dependent and transfusion dependent, all subtypes. And that’s across all the geography. Correct. Yep.

Maybe

Alex Stranahan, Senior Biotech Analyst, Bank of America: on that point, Brian, just remind us of your plans for the ex US launch, when these approvals could come, how you’re balancing driving successful launches, international expansion, retaining economics.

Brian Goff, Chief Executive Officer, Agios Pharmaceuticals: Yeah. It’s a little tough to say when the approvals will come. But as I say, we’re at pace and very encouraged by the progress that we’re making. With respect to different geographies around the world, we’re really clear on the priorities, particularly for thalassemia. It’s US is our number one priority.

And then outside The US, the clear number two is Saudi Arabia, and you could even say the Gulf region proper. And last last year, we announced a partnership that we put in place with you could think of a full service distributor by the name of Newbridge to help with the commercialization in The Gulf. And the reason we did that is, this goes back to capital allocation efficiency, it would take quite an effort for us, Agios, to scale up to capture the potential that exists. So we sought out in a very competitive process a partner who knows the region very well, is already scaled up and ready to go, and we have that in place. Looking beyond The Middle East, then Europe as an example, we expect to pursue the same option where we’re not planning on going direct.

We would look for and are having engaged discussions now on partnerships, again, to make sure that we protect our priorities of direct in The US, partnership ex US.

Alex Stranahan, Senior Biotech Analyst, Bank of America: Yeah. And that’s probably the right balance, right, in terms of if we were to go it alone, you would be maybe sacrificing the the volume side. You’d have to build out the capabilities. It’s a big investment. So you maybe come out ahead through that kind

Brian Goff, Chief Executive Officer, Agios Pharmaceuticals: of It’s clear to us that for quite a distance, next dollar invested will have a better return in The US, through direct investment than, versus the partnerships, you know, outside The US.

Alex Stranahan, Senior Biotech Analyst, Bank of America: Very good. And I guess for the FCC countries, is expansion or or, I guess, approval here dependent on getting that US approval? Is there any linking between these these different geographies?

Brian Goff, Chief Executive Officer, Agios Pharmaceuticals: Sarah can speak to that. One thing I’ll just say is that we did get and it’s very new, but we got breakthrough designation in Saudi Arabia. So that’s part of the backdrop. But yeah.

Sarah Gruens, Chief Medical Officer, Agios Pharmaceuticals: Exactly. So it’s because of that that we are able to file at like, as a new a new entity there directly to the SFDA. So this was a new procedure, and so we are going through that new procedure. But it feels also very much like it’s almost like an in between the the European and The US procedures. That’s that’s what it feels like for the team.

So we’re getting questions, answering them, you know, with deadlines, prespecified deadlines, and everything is just moving along there as well.

Brian Goff, Chief Executive Officer, Agios Pharmaceuticals: And we we haven’t talked about so far, but just to put some numbers on this, in The US for thalassemia, we’re talking about six thousand, adult patients. In Saudi Arabia, in The Middle East, we’re talking about seventy thousand. And on a per capita basis, the prevalence is roughly eight to nine times greater than The US. So there is there is a lot of focus by the regulators, by the minister of health on this particular disease, which we’re quite proud to be a part of.

Alex Stranahan, Senior Biotech Analyst, Bank of America: Makes sense. We touched on briefly kind of the overlap with having the approval already in hand for PKD. You have a Salesforce for PKD. There’s also been some label updates to that PKD label. Maybe walk us through sort of where you stand on the Salesforce, if you could leverage that also for a thalassemia launch, and then whether you think all of sort of the known profile of mitapivat is kind of reflected on the label today.

Brian Goff, Chief Executive Officer, Agios Pharmaceuticals: Yeah. I’ll let I’ll speak first to about the how we’ve set up our commercialization model, and then Sarah can comment on the label update, which was really important and we were quite pleased to have early this year. As you noted so we launched in PKD, in February of twenty twenty two, and it was a rather small team. This is an ultra rare disease. So for sales folks, they’re about 20.

And there’s more than that because what’s important about rare disease commercialization is not just frontline salespeople, but patient services, clinical nurse educators. There’s a there’s a whole support team. But to dimensionalize it, about 20 for PKD and in for thalassemia, and we waited until we saw this great data last year to to make this change, but we scaled up to about 40 for thalassemia salespeople. That is fully recruited, fully trained, and what the team is focused on right now are really two different things in preparation for launch. First, very importantly, is disease state education.

This is a disease where there have been very limited choices. And what tends to happen classically in a rare disease like that is the sense of urgency is more diminished. So we’re really putting emphasis on the fact that non transfusion dependent thalassemia patients, for example, are not less at risk. They might be more at risk because they’re undertreated and they’re suffering from chronic daily hemolysis where the patient may pay a price down the road. So disease state education is one part and the other is account profiling.

So we have a really good map through claims database analysis and interaction with these different sites of care to know where we wanna prioritize our efforts. So we are fully ready to go. And again, now we’re just waiting for the PDUFA date, But from a label perspective, maybe Sarah can talk about that Yeah,

Sarah Gruens, Chief Medical Officer, Agios Pharmaceuticals: so the hepatocellular injury that we observed in thalassemia, that warning and precaution language that we have proposed is in the PKD label, so it describes what we have seen in thalassaemia. So it says in patients with another condition being thalassaemia because it’s not a labeled condition to date for pyruvate kind. And then it basically describes the events that we’ve observed and then what physicians should be on the lookout for. Very straightforward language there. And from our perspective, when that was implemented in the PKD label, or maybe you should actually speak to that about the we see continued demand for for pyruvate kinase deficiency patients, basically.

Brian Goff, Chief Executive Officer, Agios Pharmaceuticals: We I know we talked to a lot of investors who have done their own market checks and and have talked to, clinicians who have treated either PKD patients or thalassemia or sickle cell disease. And the general consensus that we we certainly see this ourselves is that testing on a monthly basis when you’re onboarding a patient to a new treatment for hemolytic anemia is standard of care. And so it’s it’s been a kind of nonevent. And in our labeling recommendations, our recommendation from the FDA, they align with is test everybody. Don’t try to be too fancy about certain sub segments because that’s, again, the normal course of treatment.

And in this last earnings update, we were really pleased to be able to show that PKD continues to be very slow ramp because it’s ultra, ultra rare, but we actually had increase in patient demand with the backdrop of this label change. So we felt very comfortable in that regard that both patients and clinicians have certainly accepted this just fine. Okay.

Alex Stranahan, Senior Biotech Analyst, Bank of America: Okay. Makes sense. Maybe shifting gears now to sickle cell. You’ve got data update in 4Q. Maybe walk us through the trial designs or the powering assumptions on the two primary endpoints, which it sounds like if either one is met, that could maybe be a path forward for submission for approval.

But maybe just walk us through sort of the design and the thought process there.

Sarah Gruens, Chief Medical Officer, Agios Pharmaceuticals: Yeah. Sure. So it’s the Rise Up trial. It’s an ongoing phase three placebo controlled randomized trial for patients with sickle cell disease. And what we’re trying to do with this trial is highlight that mitapivat can potentially provide benefit for the totality of the disease.

Because sickle cell disease is a very complex disease, but it’s really the features are hemolytic anemia on one end and then vaso occlusion on the other end. So the trial is designed in such a way that we could speak to both of those components of the disease. So we have two primary endpoints. So the one primary endpoint hemoglobin response is tackling that hemolytic anemia component and then analyzed rate of sickle cell pain crisis is tackling the vaso occlusion component of the disease. And so there is an alpha split on those two endpoints and that allows us then if one or both are positive to transfer alpha to secondary endpoint testing, has other ways of looking at how patients feel, which is important for from a regulatory perspective that you demonstrate that people feel and or function or survive better.

So that is actually then in that bucket we’re looking at how patients feel via fatigue. So we’re with this drug, just like what we’ve done for thalassaemia and pyruvate kinase deficiency, trying to highlight that when you have that improvement in hemoglobin improvement in hemolytic anemia, people also feel better, feel less fatigued. That less fatigue that fatigue component. I mean, people are very focused on vaso occlusion in the context of sickle cell disease because it’s a dramatic event, right? It’s very.

It’s it’s horrific. It’s painful. People can die in the context of having an episode like that. But what patients when you talk to patients when they highlight that the fatigue is extremely devastating for them as part of their disease and that there’s not really anything they can do about it. So that’s what we are trying to highlight with this trial design that we can tackle all of these components of the disease.

Of course, on the hemolytic anemia side, we are building upon the evidence that we have generated for thalassemia and for pyruvate kinase deficiency. Vaso occlusion that’s purely in sickle cell disease, a component that is important and that we’re build that evidence or why we think we have a possibility to actually highlight benefit there is build upon our mechanistically on a lot of preclinical work, but then also on some of our investigator sponsored trials in which we have followed sickle cell disease patients. But then most importantly, our own phase two, which was another blinded randomized controlled trial in which we were able to select the dose for phase three, but also saw when we were testing fifty milligrams BID and one hundred milligrams BID against placebo that there was this trend on improvement on vaso occlusion which was seventy percent in the one hundred milligram BID dose reduction. So therefore we’re excited about our phase three. We’re very much looking forward to the end of the year, and we’re hoping that the the cards will turn favorably.

Brian Goff, Chief Executive Officer, Agios Pharmaceuticals: I’d like, if I could, just to add one point to a big picture about sickle cell disease is that sometimes what gets lost in these discussions is this is a lethal disease. This is where the lifespan of someone with sickle cell disease is in the forties, which is, you know, shocking to say out loud and and shocking to hear. But what it tells us is one, all of these endpoints matter. And we feel really good about the careful and thoughtful design that we’ve put into our Rise Up trial. Two is there is room for and a need for multiple different types of drugs and treatment options.

And frankly, that’s one of the motivators that we’ve had with having mitapivat. We’re talking right now about the Rise Up trial, but we also announced recently that we’ll be starting mid this year with our other PK activator, tebipivat, a Rise Up phase two like trial for sickle cell disease. And the reason is we want patients to have optionality and we’re looking to build a lasting franchise, but we know there’s no one drug that everybody responds to. And so that’s really the backdrop of our strategy, but it’s also a mindset of how to think about, quote, competition in this space. There is room for plenty.

Yeah.

Alex Stranahan, Senior Biotech Analyst, Bank of America: Okay. Maybe we can double click on that point, Brian. You know, how do you see tebipipat? You know, we’ve gotten questions like, why start the study when we haven’t seen the mitapivat data? Like, I mean, the timing component is whatever.

It’s like whenever it’s ready to go, like, let’s get it to patients. But how do you see these as maybe coexisting? And is there any mechanistic rationale why one would work better in a certain patient than the other?

Brian Goff, Chief Executive Officer, Agios Pharmaceuticals: Well, there are plenty of examples. I mean, really compelling examples across the industry, particularly in rare diseases where there are multiple drugs, even more than two within the same therapeutic application, same disease. And so to answer your question, what we’re doing is putting our foot forward now on phase two for tebipivat, but we know we will learn a lot as we go forward. By year end, we’ll get the readout of the rise up bitapivat phase three data. At a point, we will learn the results of the tepapivat phase two data.

And of course, are other peak there’s another PK activator, etabopivat, in development as well and we expect at a certain point to learn what that profile looks like and other modalities. And we’ll make smart, efficient decisions about how to allocate our capital and how to further develop tebipivat accordingly. But again, there’s a lot on the table. There is so much unmet need, and we have enough runway in front of us to be able to design the trial post phase two for tebipivat according to what the highest needs are.

Alex Stranahan, Senior Biotech Analyst, Bank of America: Okay. Maybe we can talk about how you guys are thinking about the competitive landscape. We’ve had some withdrawals. We’ve got cell therapy, gene therapy options coming up the pipe or recently approved. How do you see mitapivat and maybe timbipivat sort of slotting in to the evolving treatment paradigm?

Brian Goff, Chief Executive Officer, Agios Pharmaceuticals: This is oral therapy as a starting point, which makes it, you know, very accessible for ideally a broad population and also ideally beyond the borders of The US as we have geographic expansion. I think to have gene therapy available, if you’re a patient, that is really exciting hope. But from a pragmatic perspective, is on a whole different side of the spectrum in terms of availability and ease of onboarding and the conditioning that comes with it and so on. But I think it creates a really important inspiration. And from our view too, the more different types of companies that are investing in sickle cell disease, the better, because everybody will invest in education around the world on the unmet need, how patients need to be given different therapeutic options.

And I think we’re just exceptionally well positioned pending our data, not just for PyroKine, but again, to build this real lasting franchise.

Alex Stranahan, Senior Biotech Analyst, Bank of America: Okay, makes sense. And you guys put out a press release this morning highlighting your EHA presentations next month. Maybe walk us through sort of the scope of what we should expect there. I think there was maybe a couple on sickle cell as well.

Sarah Gruens, Chief Medical Officer, Agios Pharmaceuticals: Sure. I’ll just has again a really great presence at a conference. So the team has done an amazing job and we’re very pleased because we have a couple of oral presentations that are either our collaborators or ourselves that have data that will be presented. We’re very proud of our pediatric PKD data. So we had a data announcement on the regularly transfused pediatric pyruvate kinase deficiency patients.

A while ago, I’m liking out of the timing, but we were able to submit that to EHA and it’s going to be presented there. What we have there is a couple of children became transfusion free, which of course is very meaningful for those specific patients to be. You know, when you’re dependent on transfusions and then you don’t need them anymore, that’s very clinically meaningful. So we’re very, very happy that that data will get a spotlight. And then we have one of the investigator sponsored trials that I mentioned earlier for sickle cell disease will be presenting data as well estimate.

So that’s a trial that is run-in The Netherlands by doctor Van Bier. So that data will be presented. And then we have also some work preclinical work in MDS patients highlighting that PKM two is reduced in MDS, which, of course, is important for our mechanism of action. So we have a very nice spread of early mechanistic work to clinical data in pivotal trials that is going to be presented, so we can’t wait to to be there.

Brian Goff, Chief Executive Officer, Agios Pharmaceuticals: And this is like EHA and ASH. These are our Super Bowls. And so we’re we’re really pleased to have 14 different abstracts at EHA and a lot to share across our entire pipeline. Yeah. Mulan’s a

Alex Stranahan, Senior Biotech Analyst, Bank of America: little bit better than Orlando. I’ll say. Yeah. Sure. Brian, may maybe circling back just in the last minute or two that we have, $1,400,000,000 in cash is a great position to work from.

Is there a scenario where you get approval in thal, the launch is going well, sickle cell looks like it could be approvable as well, that this cash balance could actually carry you through to profitability. Like, what’s sort of the runway here, and how are you thinking about capital allocation over the next

Brian Goff, Chief Executive Officer, Agios Pharmaceuticals: Well, I love that scenario. If we’re gonna close down here, that’s that’s a good way to start. Yeah. I think first of all, 1,400,000,000.0, we know that that is a very unique position to be in in this macroeconomic environment. We don’t take that lightly.

We’re very serious about efficient capital allocation, very disciplined. Our priorities are clear, and you mentioned the first one, which is get the launches right, create maximum value. Our ambition is a multibillion dollar potential for this Pyrokine franchise. Number two is we do have a kind of middle part of our pipeline. We talked about tebipivat a little bit that we’re looking to advance as well, and that would be our second priority.

And third is expand the pipeline, and that can come in two different directions, internal organic opportunities, which we constantly pursue. Sarah’s team does a great job from a translational perspective and and research. We also are, of course, looking externally. And in fact, we we recently added a key member of our team, Krishnan Viswanathan, who’s tasked with business development, corporate strategy, just to make sure that we’re constantly assessing the landscape externally. And that just positions us really well as a company to be sustainable and continue to grow and deliver value for shareholders.

Fantastic.

Alex Stranahan, Senior Biotech Analyst, Bank of America: Well, with that, I think we’re right at time. So please join me in thanking Brian and Zara for the great conversation. Thanks for being here at the conference.

Brian Goff, Chief Executive Officer, Agios Pharmaceuticals: Thanks a lot, Alan. Thanks, everyone.

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