Agios Pharm at Leerink’s Global Healthcare Conference: Strategic Insights

On Tuesday, 11 March 2025, Agios Pharmaceuticals (NASDAQ: AGIO) presented at Leerink’s Global Healthcare Conference 2025, offering a strategic overview of its pipeline and commercialization efforts. The company highlighted both the challenges and opportunities in its lead assets, particularly focusing on Pyrukynd and tepavivat. While the launch of Pyrukynd for pyruvate kinase deficiency (PKD) has been slower than anticipated, Agios remains optimistic about future prospects, including potential approvals for thalassemia and sickle cell disease.

Key Takeaways

• Agios holds a strong financial position with $1.5 billion in cash and equivalents.
• Pyrukynd’s launch for PKD is slower than expected due to the disease’s ultra-rare nature.
• The company is preparing for a thalassemia launch, with regulatory submissions in multiple regions.
• Agios is optimistic about Phase 3 trial results for sickle cell disease expected by year-end.
• The company plans strategic partnerships for distribution in the Gulf countries and Europe.

Financial Results

Agios ended 2024 with a robust cash position of $1.5 billion, strengthened by monetizing the Tibsovo royalty through a transaction with Servier in 2021. Capital allocation priorities include:

• Maximizing potential launches to create a multibillion-dollar franchise for Pyrukynd.
• Advancing the pipeline with tepavivat and earlier-stage programs.
• Expanding the pipeline through internal organic opportunities and external business development.

Operational Updates

PKD (Pyruvate Kinase Deficiency):

• Pyrukynd is approved and commercialized in the US for adults with PKD.
• The launch is slower than expected due to the ultra-rare nature of the disease and low diagnosis rates.
• Patients report improved quality of life and drug persistency is high.

Thalassemia:

• Two Phase 3 studies, Energize and Energize-T, were completed.
• Regulatory submissions were made in the US, EMA, Kingdom of Saudi Arabia, and UAE.
• A PDUFA date is set by the FDA for September.
• Potential liver toxicity in thalassemia patients was discussed, with emphasis on reversibility upon drug discontinuation.

Sickle Cell Disease:

• Enrollment in the Phase 3 study, Rise Up, was completed in October last year.
• Topline data readout is expected towards the end of this year.
• Potential US launch is anticipated in 2026, following thalassemia.

Lower Risk MDS (Myelodysplastic Syndromes):

• A Phase 2b study is underway with tepavivat, with enrollment expected to complete by year-end.
• Data is expected next year.

Future Outlook

Thalassemia:

• Focus on US launch preparation with disease education and profiling for physicians.
• Plans to partner for commercialization in Europe, similar to the distributor agreement with Newbridge for the GCC.

Sickle Cell Disease:

• Awaiting Phase 3 topline data by the end of the year.
• Exploring both mitapivat and tepavivat to build a franchise in sickle cell disease.
• Pricing decisions will be data-driven, considering the competitive landscape.

Expansion Strategy:

• Continuously assessing internal and external opportunities to expand the pipeline through business development.

Q&A Highlights

PKD Commercialization:

• The ultra-rare nature of PKD leads to a slower launch with lower diagnosis rates.
• Patients on Pyrukynd generally report feeling better, improving activities of daily life.

Thalassemia Commercial Opportunity:

• First focus is the US for commercialization.
• Newbridge will commercialize in the Gulf countries, particularly Saudi Arabia.
• Europe plans to pursue a similar partnership model for commercialization.

Sickle Cell Disease Trial Design:

• The Rise Up trial is designed with optionality, with an alpha split on two primary endpoints (VOC improvement and hemoglobin).
• The trial has operational safeguards, like an adjudication committee, to manage the accuracy of data.

Readers are encouraged to refer to the full transcript for a detailed understanding of Agios’s strategic plans and insights.

Full transcript - Leerink’s Global Healthcare Conference 2025:

Andy Behrens, Senior Biotech Analyst, Target Oncology: Good morning, everyone. Day two of the Layering Partners Global Healthcare Conference. I’m Andy Behrens, Senior Biotech Analyst at Target Oncology. We’re really excited to have Agios join us for this session. We have Sarah and Cecilia.

Thank you for spending some time with us to walk us through the story. Maybe for those that are not familiar with Agios, you could tell us a little bit about the company before we jump into the Q and A.

Sarah, Unidentified role, Agios: Great. Thank you for having us, Sandy, and thank you for everybody listening in. So for those that are not familiar with the story, Agios is focused on developing therapies that hope to elevate and improve the lives of patients with rare diseases. Our focus today is in diseases that result in the dysfunction or destruction of red blood cells, particularly in pyruvate kinase deficiency or PKD, thalassemia, sickle cell, and lower risk MDS. Our lead asset is called pyrokindometapivat, and its unique mechanism of action is intended to improve the metabolism of the red blood cells, improving hydration, longevity, and overall energy.

And pyrokin today is approved in The US and is commercialized in The US for adults with pyrolytic kinase deficiency or PKD, and we’re also hoping to expand the indications and potentially launch in thalassemia and sickle cell disease. We had a tremendous year in 2024. We had a lot of corporate goals set up at the beginning of the year, which we met on or ahead of time, and very excited with the progress we’ve made with our pipeline. I’d say a particular note was that a senior program for us. We had two phase three studies that read out last year.

The first one is Energize that was in non transfusion dependent patients with thalassemia. The second one is energized t with patients with transfusion dependent thalassemia. And together with the data for both studies, we were able to find by the end of the year simultaneously in four regions, The US, EMA, Europe, Kingdom Of Saudi Arabia and United Arab Emirates. For The US particularly, we have a PDUFA date set by the FDA for target profiling for the physicians that may have patients, target profiling for the physicians that may have patients as we gear to kind of a larger indication. We started with PKD, which is ultra rare.

Thalassemia is still rare, but but a bigger indication. On the sickle cell front, we were also very excited to announce in October of last year that we completed the enrollment of our Phase three study call rights up. And for that one, the next milestone will be towards the end of this year, the readout of the top line for that study. And if successful, hopefully have a launch in The US right after thalassemia in 2026. We also made progress with our second PK activator called tepaper pivot or 89 for six before that.

And that one has an ongoing study in lower risk MDS, which is also a very exciting area with fast growth. And we have a phase two b study currently underway there with completion enrollment expected to be completed by the end of this year with data coming next year. A second path, whichever pivot we’re exploring is in sickle cell disease as we plan to start a Phase two study by mid of this year. And that would allow us to build a franchise in sickle cell, which is a disease with very high unmet need. And last but not least, we also have a very strong balance sheet, which we bolstered last year as we monetize the royalty for Horace Idenib as part of our transaction with Servier back in 2021.

We ended up the year with 1,500,000,000.0 in cash and equivalents. And the way we’re thinking about that for capital allocation is in three key areas of priorities. One is obviously maximizing these potential launches and creating a multibillion franchise for Pirate Kind. Second would be advancing the pipeline with Teva Pivot and some of the earlier stage programs we have. And a third bucket would be to expand the pipeline, both from an internal organic opportunities we’re looking at as well as looking externally through business development.

So a very exciting year ahead of us as well for us at Algiers and looking forward to your questions, Andy.

Andy Behrens, Senior Biotech Analyst, Target Oncology: Great. And anybody in the audience has questions, feel free to let me know. It’s why don’t we start with with pirocad commercially now? It’s it’s done done fairly well, but it’s obviously a small indication. What what have been the takeaways, from patients, staying on the drug?

I mean, what’s been the tolerability of of the the profile of the drug? Yeah.

Sarah, Unidentified role, Agios: So for context, viral PKD is an ultra rare disease. It’s very low diagnosis rates and not an urgency to treat. So it’s a slower launch in a way than we expect, than we do for thalassemia. But to your point, we’ve learned a lot of things in terms of how patients feel after taking the drug. We’ve had very good persistency on the drug, and it also allowed us to build foundation in capabilities in things like market access and patient services that we plan to expand in Tallacinha.

We have not seen any pushback from an access perspective in PKD. But as we pivot a little bit to Tallahassee, it’s a little different because we know that there’s six thousand adult patients. They are diagnosed. They’re known to the system. So from a launch perspective, we can do target profiling.

We can do disease education more and more dedicated there. And the other thing I’d say we also learn in PKD is my wide inch deep in a way. It’s like one physician will have one patient in their whole career. So you don’t have, like, a patient have a sorry. A physician have a bolus of patient.

So that’s a little different in thalassemia as well. We have a little bit more depth. And also the patient community is more organized in thalassemia. So very different launches, but PKD served as a really good foundation for us.

Andy Behrens, Senior Biotech Analyst, Target Oncology: Right. And in general, the patients that I mean, I guess the feedback we got is patients like the drug, it makes them feel better, they stay on the drug. The ones that do come off of treatment for PKD, is it generally for lack of efficacy or it’s safety tolerability type?

Cecilia, Unidentified role, Agios: Yeah. So for PKD, the underlying, the pathophysiology is a different mutation for each patient. There’s a ton of different mutations, so we can’t predict treatment response based on the underlying genotype. So the way to go about this is for a patient to try and then in within six months, if they don’t have, you know, an improvement on hemolytic parameters or hemoglobin or they don’t feel better, they just tend to come off. The vast majority of patients, though, do feel better and do have some improvement on hemoglobin and on their hemolytic parameters.

So even if they don’t reach a clinical trial bar of one point five grams per deciliter, but they reach a hemoglobin improvement of one gram per deciliter, for instance, they choose to stay on the drug. Okay.

Andy Behrens, Senior Biotech Analyst, Target Oncology: And they do really feel better. Like when

Cecilia, Unidentified role, Agios: you talk to patients, it’s and those are things you can’t really measure in a clinical trial, but the way they activities in their life, going back to a full time job, going to school, being able to complete a shower. I always think back with this one patient who described to us that

Andy Behrens, Senior Biotech Analyst, Target Oncology: she was never able in her life to go

Cecilia, Unidentified role, Agios: through one shower without having to take breaks, things that we can’t even imagine as like having an impact. Now she’s able to lift her arms, wash her hair, finish her shower. And those are things that are really meaningful to people.

Andy Behrens, Senior Biotech Analyst, Target Oncology: Right. And I think it’s I mean, the reason I ask the question is I think when we talk about sickle cell, the drugs that were approved and or are no longer approved, I think that the feedback we got was patients didn’t necessarily feel better. Some of them felt worse.

Cecilia, Unidentified role, Agios: Right. And so I think this is something that we have seen in PKD and now also in thalassemia that the way we improve hemoglobin actually, also makes people feel better. And these are the first programs that actually were able to deliver on a statistical significant result, for fatigue. So we’ve measured fatigue both on the PKD program and in the thalassemia program. And for the thalassemia program, it was actually our key secondary endpoint.

And we hit statistically on an improvement in fatigue.

Andy Behrens, Senior Biotech Analyst, Target Oncology: Okay. No, I think that’s definitely important. When we talk about thalassemia and then we’ll go to sickle cell, you do have the PDUFA date in September. And there’s some speculation about whether or not there’ll be an adcom or not. Some people think that could be a good thing.

And I think you said on your earnings call, you’re not welcoming an adcom. Obviously, they’re unpredictable and a lot of work and expense to prepare for. But what are your thoughts now about the potential for an adcom ahead of that September PDUFA day?

Cecilia, Unidentified role, Agios: So the same as our earnings call still, like we are in the process of filing an active engagement with the regulators to date, we have not been informed that we will have an advisory committee. Of course, if that would change, we would let you all know. And to the point of we’re not welcoming it, we are. Like, if they tell us that we have to have an outcome, we will, you know, obviously be fully ready to engage with the regulators that way and then would welcome that opportunity. Needless to say, I like from a filing perspective and a workload perspective and indeed the resources, I would prefer that we just progress as is versus actually having one.

Andy Behrens, Senior Biotech Analyst, Target Oncology: Right. Okay. I mean, I think one of the reasons people are questioning the need for Adcom was was your disclosure at the ASH meeting about, the potential for liver toxicity. I guess what struck me was, you know, we have a lot of companies that have small molecules that have LFT elevations and they usually classify them as transit LFT elevations. And so I guess you’re taking that next step.

People are questioning whether, you know, that’s conservatism on the company or is there something that makes you more concerned than the companies that are seeing these trends in LFT elevations?

Cecilia, Unidentified role, Agios: Well, I think it’s all about context of the disease and the risk profile observed. Right? So thalassemia patients are patients who have a lot of liver disease as part of their condition. So if you don’t treat thalassemia, for sure people end up with bad livers over time. And actually now that people are better managed, like one of the big causes of death in thalassemia is liver disease.

So it makes it very hard to actually examine and look at data because there is a lot of confounders in this patient population. That being said, why we actually called it an important potential risk was because we had observed, you know, cases in our program that had a similar pattern. And the fact of the matter was that despite confounders or no confounders, they all improved on discontinuing the drug and returned back to their baseline. While we don’t call it just a regular, like, transaminase fluctuation, on a drug is because two of the two out of the five patients ended up having a hospitalization, which to me is like that’s not just a random lab fluctuation, it’s more serious than that. And while we feel it’s appropriate to actually have a warning and precaution around it to draw extra attention of the physicians to this phenomenon.

Andy Behrens, Senior Biotech Analyst, Target Oncology: Okay. And the liver disease in thalassemia, and we’ll talk, I guess, in sickle cell whether it’s also seen there. You said even in patients that are not treated, meaning if you didn’t give transfusions or chelators or anything, you still there is like an innate liver disease. It’s just from the is it the hemolytic aspect that causes iron overload?

Cecilia, Unidentified role, Agios: Yeah. They have a dysregulated iron metabolism and they, you know, ongoing hemolysis releases free heme and iron into your system, which basically it does accumulate in your organs even if you’re not transfused. Obviously, if you get transfusions on top, it typically iron overload is higher and people are on chelation, but not all non transfusion dependent patients are on chelation.

Andy Behrens, Senior Biotech Analyst, Target Oncology: Okay. And then sickle cell is also hemolytic disease with, you know, iron overload and, and it has also ischemic aspect to it. But, do those patients also have a similar background rate of liver disease?

Cecilia, Unidentified role, Agios: Liver is one of the target or like is one of the organs that really gets damaged in the context of sickle cell disease. But to your point, the pathophysiology is a little bit different between thal and sickle. So in sickle cell disease, you have this acute ischemic component that adds to it. And then for both diseases, if you get transfusions, there’s always risk with transfusions by by itself. If you already have some baseline abnormalities, you tend to respond more severely to whatever insult comes next.

So there’s a multitude of reasons.

Andy Behrens, Senior Biotech Analyst, Target Oncology: Right. I think, you know, obviously, the benign profile of the drug and PKD and patients staying on the drug and the feedback we got was very positive. So it caught a lot of us off guard when you guys had that disclosure at ASH. That was on the heels of, you know, of the decision of Broxperto, which was also another surprise, I guess, that, you know, made people question, you know, the VFC need in that trial. So I think it’s, you know, I understand, you know, why the market reacted like it did and, you know, we’ll see how things

Cecilia, Unidentified role, Agios: Yes. And I think that’s where there is a difference between how the market reacted and how the physicians in our clinical trials and the community has reacted to this. I think when you speak to the providers who take care of thalassemia patients, they are routinely monitoring for liver disease. We just discussed it like from thalassemia, but also iron collation by itself. You need to watch for liver disease, so they are very much in tune with this.

So for us to propose a monitoring on a monthly basis for the first six months, and then when you observe something to actually act upon, it was almost for them routine. And so we didn’t have that type of reaction speaking to physicians.

Andy Behrens, Senior Biotech Analyst, Target Oncology: Okay. And in terms of your next generation tepidivat, and then I think there was a prior drug, there was a PK drug developed in around twenty fifteen, twenty sixteen. Is there anything about is it a direct consequence of mechanism potentially or is it something with the scaffold or metabolite? Do we know, you know, if there’s something related to the drugs?

Cecilia, Unidentified role, Agios: So the three drugs are different from the scaffold thalassemia. I think if we talk about drug induced liver injury, it’s extremely complex, but typically almost any drug that gets metabolized in the liver at some point will have a signal for liver injury, being it transaminase fluctuations versus more severe events. And sometimes that is linked to the way it gets metabolized. Sometimes that is linked to the dose. Sometimes there may be risk factors within a specific patient population.

For us, this is not possible to really determine that at this point in time because it’s a very few number of patients in the context of a rare disease that is a very confounded patient population. So for us, we took an approach of just highlighting when you start taking this drug for the first six months, just watch out for these liver enzymes and then take appropriate action as you see fit, which is a very clear and clean guidance, which is typically what you like from a post marketing perspective to be simple and direct.

Andy Behrens, Senior Biotech Analyst, Target Oncology: Right. Okay. And when the patients, I think you said this, when they, if it’s identified by the monitoring, it is reversible by the patients have to come off the drug permanently or do they just dose reduced so they can go back on it?

Cecilia, Unidentified role, Agios: So it depends because what if you see something like this, we what we put in our PKD label is to just discontinue the drug and then if you think there is another cause and you want to restart the drug, you can do a rechallenge, but that is when you think it’s something else causing the event, right? Like, typically, when you have a pattern like this, physicians tend to do a workup in the context of the individual in front of them. So maybe they have a viral infection or maybe maybe it is the drug. Like, they all will make their independent assessment. And then if they find another cause, they can restart.

Andy Behrens, Senior Biotech Analyst, Target Oncology: Right. Okay. In terms of the commercial opportunity, what are the plans for the company? A lot of the thalassemia patients are not in The U. S.

And the Mediterranean region. So walk us through kind of how you guys see the commercial opportunity if the drug

Sarah, Unidentified role, Agios: is Yeah. So our first focus obviously is The US, which we spoke a little earlier, and the team is obviously gearing up for for that. For Axios, we’re not directly commercializing. What we’ve decided is to partner with, it’s a distributor agreement, but it’s a full service provider. And the biggest area outside of The US is the Gulf countries with Saudi being about 75% of that region.

As I mentioned, we filed in Saudi and United Arab Emirates in parallel with, The US and Europe, and we have breakthrough medicine designation from a regulatory perspective. So I can speak to that in a second. So from a commercial perspective, GCC is about seventy thousand prevalent patients in in Tallahasseea in that region. From a dynamics, it’s more similar to a European type of markets, both from a pricing dynamics perspective and and in general, how it works. They have Ministry of Health.

They have private institutions. They have academic institutions. So you, go through the regulatory approval and pricing initial discussion at a national level first, and each nation does their own review. And then you still have to go through formulary access discussion. So it takes longer, like, different in The US where you get your approval pricing, you go.

So this will it’s a bigger opportunity, of course. It will take longer to get there. There’s a generally slower pricing than The US, but given the volume, obviously, there would be a lot of discussions with them in terms of budgetary impact. But we’re very excited. We partnered with a company named Newbridge.

They’re very well known in the region. This is a very common model. We’ve all had experience in prior companies doing this, and and they work very close together with us. We still have full strategic control in terms of of the decision making, But for me, it’s also a very, efficient way of doing capital allocation or just having to build, you know, to a new infrastructure outside of The US. So that’s for GCC.

And then for Europe, we’re looking to to build a similar model.

Andy Behrens, Senior Biotech Analyst, Target Oncology: Okay. And I guess, you know, there is an approved drug in this indication. So what, I guess, what do we learn about the reimbursement? I mean, you guys, I think you suggested that the pricing for thalassemia at least could be maintained at the PKD pricing, but it sounds sickle cell and that might be tougher. But, is this a price sensitive market?

Sarah, Unidentified role, Agios: So the way we think about it, we usually don’t really discuss pricing until you have full data and a label in general. So when we think about so PKD today is three hundred and thirty five thousand annual WACC in The US. When and like I said before, we haven’t seen any pushback. It’s not a managed category. It’s an ultra rare disease.

Thalassemia is bigger in the context for allergies, but it’s still a rare disease. It’s still in the neighborhood from a payer perspective, so we think we’re very well positioned there, from a pricing perspective. As you migrate, yeah, you know, to sickle cell, we’re hopeful to be in that position to have that indication as well. It’s a much bigger indication, it’s a hundred thousand patients. We will guide our pricing decision based on the data when we see it as well as a competitive landscape around it as well.

Andy Behrens, Senior Biotech Analyst, Target Oncology: Okay. I mean, some of us have always thought, you know, even before Brian took the helm that TAVR could be, you know, at least it would solve the pricing problem to have a separate drug and sickle cell, but the company has always been committed to mitapivat and parikine.

Sarah, Unidentified role, Agios: Yeah. And we’re still looking at so temapivat is is something we will explore. So sickle cell is a disease that’s gonna need many, many therapies. These patients, it’s a it’s a complex disease. Not everybody responds to drugs.

So we are and that’s what we are excited to see the phase one data we presented last year and entering into a phase two to build a franchise from a commercial perspective to be able to take more of the branded, you know, patients on on branded drugs. And it also gives us a different we also have different patent lies and pricing could be a way to differentiate. We’ll be guided. Similar thing would be guided by the data once we have both right with the period and tele period data. But we’re very excited to have two options for these patients that are in dire need of course.

Andy Behrens, Senior Biotech Analyst, Target Oncology: Right. And so let’s talk a little bit about we have under ten minutes left. I mean sickle cell for a lot of investors is the real big opportunity. There’s obviously MDS, which is also big that you guys are pursuing, but I think that’s considered more of an option. Sickle cell seems most tangible, I guess, especially if we get approved and patients feel better.

Now that OXBryta is no longer on the market, the competition is less. What do you think? I mean, the question, I guess, you know, is a VOC, is a clinically meaningful endpoint going to be important? Is the trend going to be okay? And, you know, we did see another PK drug actually in a small trial show a VOC benefit.

So, I guess, you know, do do you think, I guess, first that FDA wants to see a definitive VOC benefit or is a trend gonna be enough?

Cecilia, Unidentified role, Agios: I think it always depends on the totality of the data you present. Obviously, we’re shooting with a primary endpoint to show improvement on a VOC, but the way we’ve designed the trial is if we wouldn’t, if we would only have a trend, so wouldn’t reach statistical significance because we’ve done an alpha split on the two primary endpoints, the other primary endpoint being hemoglobin, we can move on to secondary endpoint testing, which has other ways to look at how patients feel and function and also other ways to look at VOC. So the way the trial is designed, this is a lot of optionality. Obviously, we’re hoping to hit on both primary endpoints.

Andy Behrens, Senior Biotech Analyst, Target Oncology: Right. Okay. What, in terms of the background VOC rates going in, I think that it’s sometimes difficult because patients when they enter a trial are monitored more regularly than they are maybe before when they were just being treated by their primary care physician or their hematologist. How does that tend to trend in trials? Is it that once you end up with more severe patients or less severe patients when you use the background rate as entry criteria?

Cecilia, Unidentified role, Agios: Yeah. So I think, so we look back for one year, at the patients that enroll into the trial, and one of the requirements is to have had at least two sickle cell pain crisis in the year prior. And so we have a definition that is applied to what is a sickle cell pain crisis. And then the investigator highlights effect that we that they meet or don’t meet that criteria. So if they meet the criteria, then they can move on into the trial.

And then each sickle cell pain crisis event that would occur gets captured per that definition, and then it goes on to an adjudication committee. So that’s the difference, right? We have an adjudication committee while the trial is ongoing. We are not adjudicating historical events. Same definition, but we don’t make all, I mean, it wouldn’t be possible to do everything in a screening period, basically.

So, so you do rely on that. That being said, the way the investigators assessed the events and the way the adjudication committee assessed the events in our phase two was very similar. So that’s always a good good sign. And on top of that, our phase two data, the way we’ve captured it, we had projected that people would have around the baseline sickle cell pain crisis rate of about three, and that is exactly what occurred in the Phase two. Then in the Phase two, we had a little bit of a lower rate, in the patient population once they were in the trial, which speaks to the fluctuation, in the patient population, but it still allowed us to measure a dose response on sickle cell pain crises.

So we feel very confident with the way we’ve applied the definition, the way we’ve set up our inclusion criteria and the adjudication committee, and then the data collected in the trial, that that is a really good setup for phase three. For phase three, we have the same type of assessment, so same inclusion criteria, same type of patient population, which is typically where sickle cell disease, I think, sometimes had some issues that the criteria from Phase two changed quite a bit towards the Phase three patient population. So we’re not doing that. Similar patient population, same adjudication committee, a lot of the same, sites and investigators that contributed to phase two, they’re all still in phase three. So, you know, we try to make it as protected as possible.

Andy Behrens, Senior Biotech Analyst, Target Oncology: Right. I think one of the the KOLs highlighted to us, I think, with one of the DACVIA, which which didn’t demonstrate the VOC on the confirmatory basis. But, then I guess it’s that in the background rate, in the baseline rate for the previous year, patients may not go to the hospital and a lot of the definitions of VOC require some sort of definitive intervention either hospital visit or written change in medication or something. And so and then in the trial when you’re watching a patient like they’re definitely going to go like if they have pain they’re told to go. So it actually inflates the amount of VSC.

So it’s very hard to demonstrate a VSC benefit in a trial. Is that what happened with ADACCO?

Cecilia, Unidentified role, Agios: I don’t know what exactly happened with Adacchio. There were like a couple of chain differences between the phase two and phase three. I don’t I don’t know ultimately what drove this. Right? So I that may be part of it, but I think there is potentially additional reasons, and maybe all of it compounds.

So I think from our perspective, though, we are, like I said, we have a lot of safeguards, like operational safeguards along the way, trying to keep things as similar as possible, to be able to replicate. And then the other thing that we do is, actually measure, there is a pain diarrhea as well for patients to fill out so that we can capture events that occur that maybe did not trigger the patient to go to the hospital. So we have other things in the trial to try to collect all of that data.

Andy Behrens, Senior Biotech Analyst, Target Oncology: Right. Okay. And then the other, I guess, concern with OXPRITO was the infection rate from malaria, which I guess you’re decreasing the sickling. So, you know, I mean, is that how are you I mean, you’re going to have patients, I guess, enrolled from the same regions. How do you how have you, taken that risk?

Cecilia, Unidentified role, Agios: Yes. So we do have patients enrolled, out of regions where malaria is present, which I think is the right thing to do in the sense of that’s where sickle cell disease is. So typically, it’s a good thing to try to enroll a patient, a population that is representative of the overall patient population, also because people that live in Western Europe or in The U. S, they go back and forth and can come home with malaria, so it’s good to understand what happens typically in the context of a clinical trial. Hydroxyurea did not have that phenomenon, right, and so this was something that was observed on Angsparita.

I don’t know if that was a random chance versus something really linked to potentially mechanism of action. What we have done is we try to go for really balanced enrollments, across all of the different regions in the world. You could enroll a sickle cell disease trial really much faster if you go to certain regions, but we try to really make sure we have a good representation of The US, Western Europe, and The Middle East as well, avoiding some of that.

Andy Behrens, Senior Biotech Analyst, Target Oncology: Okay. Do you get the sense? I mean, it seems like if you guys can succeed on the trial and get it approved, there’s there’s a huge need now, especially for a drug that improves hemoglobin, but also makes patients feel better and lessens the amount of time they spend in the hospital. So it really is, you know, it’s it’s it’s a big opportunity if you can get it get the drug there.

Cecilia, Unidentified role, Agios: Yeah. We’re very excited. Very excited. And we’re, like, shooting to have the data by the end of this year. Okay.

Andy Behrens, Senior Biotech Analyst, Target Oncology: Well, great. Any questions from the audience? Rajesh? Okay. Thank you.

Appreciate it. We look forward to the updates and all the progress you guys have made.

Sarah, Unidentified role, Agios: Thank you. Thank you for having us, Sandeep.

Andy Behrens, Senior Biotech Analyst, Target Oncology: Thank you, everyone.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.