Agios Pharmaceuticals Q1 2024 Earnings Call Transcript

Quartr
Provided by Quartr
May 2, 2024

There are 11 speakers on the call.

Operator

Good morning, and welcome to Agios First Quarter 2024 Conference Call. At this time, all participants are in

Speaker 1

a listen only mode.

Operator

There will be a question and answer session at the end. Please be advised that today's conference is being recorded at Agios' request. I would now like to turn the call over to Chris Taylor, Vice President, Investor Relations and Corporate Communications for Agios. Please begin.

Speaker 2

Thank you, operator. Good morning, everyone, and welcome to Agios' conference call and webcast to discuss Q1 2024 financial results and recent business highlights. You can access slides for today's call by going to the Investors section of our website, agios.com. On today's call, I'm joined by our Chief Executive Officer, Brian Goff Doctor. Sarah Huyns, Chief Medical Officer and Head of R and D Sveta Milanova, our Chief Commercial Officer and Cecilia Jones, Chief Financial Officer.

Speaker 2

Before we get started, I would like to remind everyone that some of the statements we make on this call will include forward looking statements. Actual results and events could differ materially from those expressed or implied by any forward looking statements as a result of various risks, uncertainties and other factors, including those set forth in our most recent filings with the SEC and any other future filings that we may make with the SEC. And with that, I'll turn the call over to Brian.

Speaker 3

Good morning, everyone, and thank you for joining us. Our mission at Agios is to develop and deliver transformative medicines that elevate and extend the lives of patients living with rare diseases, and we are off to a fast start in 2024. Our foundation today leverages mitapivat's novel mechanism of action, which focuses on overall red blood cell health and has been a key driver for our recent clinical results. On January 3, we reported positive data from the Phase 3 Energize study of our lead PK activator mitapivat marketed as pyrokine in patients with non transfusion dependent thalassemia. This study met both the primary endpoint of hemoglobin response rate as well as both key secondary endpoints associated with change from baseline in facet fatigue score and average hemoglobin concentration and we look forward to presenting these data at an upcoming medical meeting.

Speaker 3

As a reminder, non transfusion dependent thalassemia accounts for approximately 2 thirds of thalassemia in the U. S. And has no FDA approved treatment options. Despite not requiring regular transfusions, it is increasingly understood that these patients experience a significant impact on their quality of life, a wide range of serious morbidities and an elevated risk of premature death due to chronic hemolysis and ineffective erythropoiesis. Based on these data, our team is actively preparing for a potential launch in thalassemia in the U.

Speaker 3

S. Complementing the Energize study in non transfusion dependent thalassemia, we continue to advance the Phase 3 Energize T study of mitapivat in transfusion dependent thalassemia. We expect to report data from this study in the second quarter, a slightly more refined timeframe than previously communicated. And we plan to submit a single regulatory filing encompassing data from both energize and energize T to the FDA by the end of the year. In parallel, we look forward to near term milestones across several additional clinical programs in our pipeline, including completing enrollment in the Phase 3 portion of the RISE UP study of mitapivat in sickle cell disease by the end of this year and reporting data from 4 additional Phase 3 studies by the end of 2025.

Speaker 3

Sarah will provide a detailed update on our progress and upcoming milestones across R and D in just a few minutes. Given the consistent positive data we've generated across the mitapivat development program and the high unmet need in our target disease areas, we believe mitapivat has the potential to transform the course of multiple hemolytic anemias by improving red blood cell health and to become a multibillion dollar franchise. To help realize the full commercial potential of mitapivat and based on the strength of the energized data, our commercial organization is laser focused on building upon the infrastructure established through our current launch in pyruvate kinase deficiency or PKD to prepare for potential U. S. Launches of mitapivat in thalassemia in 2025 and in sickle cell disease in 2026.

Speaker 3

Sveta will provide greater detail on the market opportunity in thalassemia and the team's robust preparation for launch as well as an update on our current launch in PKD in just a bit. Finally, as you'll hear from Cecilia, we ended the Q1 with a strong cash position with approximately $714,000,000 in cash and investments on the balance sheet. Importantly, we have the potential to further bolster our cash position in the near term as Servier announced FDA filing acceptance and priority review for a new drug application for vorasidenib for the treatment of certain IDH mutant diffuse glioma. You'll recall that as part of the divestiture of Agios' oncology business to Servier, Agios retains rights to a potential $200,000,000 milestone upon FDA approval of vorasidenib and 15% royalties on potential U. S.

Speaker 3

Net sales. If approved, vorasidenib would become a 1st in class targeted therapy for patients with IDH mutant gliomas and we look forward to the PDUFA action date of August 20, 2024. With that, I'll turn the call over to Sarah.

Speaker 1

Thanks, Brian. As we approach top line data readout for the Phase 3 Energized T study of mitapivat in transfusion dependent thalassemia, I would like to highlight a few key elements of the mitapivat development program in thalassemia. As a reminder, the Phase 3 program of mitapivat in thalassemia, namely Energize and Energize T was designed to deliver data across all subpopulations of thalassemia, including alpha and beta thalassemia in populations with different transfusion needs. As Brian mentioned, only patients with transfusion dependent beta thalassemia, which represents 1 third of U. S.

Speaker 1

Patients have an SBA approved treatment option. The other 2 thirds of patients in the U. S, including all patients with alpha thalassemia and those patients with beta thalassemia who are non transfusion dependent have no approved treatment. It is a common misperception that non transfusion dependent or NTD thalassemia patients are less sick when the reality is that these patients suffer from a poor quality of life and a high rate of serious morbidities, including thrombosis and premature death. This population, which represents approximately 2 thirds of total thalassemia patients in the U.

Speaker 1

S. Has a high unmet need for any treatment. This unmet need was strongly reinforced through our Phase 3 Energize study by the speed of enrollment, the total number of patients enrolled and the high completion and rollover rates we observed. We were very pleased to announce positive results from this study in January and are eagerly awaiting the opportunity to present more complete results at an upcoming medical meeting. Turning to those results.

Speaker 1

Our goal was to build upon our Phase 2 findings with a more rigorous way to measure immunoglobulin response in the Phase 3 ENERGISE trial, which we defined in the primary endpoint as an increase of equal or more than 1 gram per deciliter in average hemoglobin concentrations from week 12 through week 24 compared to baseline, but in a much larger trial. We were excited to be able to announce success in this trial as treatments with 100 milligrams mitopivab BID demonstrated a highly statistically significant result on the primary endpoint of hemoglobin response rate with 42.3% of patients in the treatment arm achieving a hemoglobin response versus 1.6% of patients in the placebo arm. In addition, treatment with mitapac also resulted in statistically significant improvements in both key secondary endpoints, including a change from baseline in average facetic score, an important patient reported measure of how patients feel. In line with its novel mechanism of action that improves overall red blood cell health, mitapacab is the first molecule that has shown in a randomized controlled trial that it does not only improve hemoglobin, but actually makes people with thalassemia feel less fatigued. In addition, across the primary and secondary endpoints, all prespecified subgroup analyses favored lizapivab compared to placebo, suggesting that no single subgroup was responsible for driving the results, which supports our aim to file for a broad label covering all thalassemia subtypes.

Speaker 1

Turning to Energize P. Let me highlight 3 key reasons why we are excited about the upcoming readout in transfusion dependent thalassemia. First, it is important to recall that regardless of a patient's transfusion needs, thalassemia is a hemolytic anemia. By up regulating PK activity and improving overall red blood cell health, mitapicap's novel mechanism of action directly addresses the underlying pathophysiology of hemolysis in all thalassemia subtypes. 2nd, this is a similar approach to the one we took for our PK deficiency program.

Speaker 1

In that case, in the Phase 3 ACTIVATE T study of mitapivat in regularly transfused adults with pyrotechnine deficiency, mitapivat demonstrated a statistically significant and clinically meaningful reduction in transfusion burden. These data together with positive data from the Phase III ACTIVATE study in patients with PK deficiency who are not regularly transfused led to FDA approval of mitapivat for adults with PK deficiency regardless of transfusion status. And we look forward to the potential to achieve the same in thalassemia. And 3rd, in line with mitapivat's mechanism of action, we have seen consistency in the data with improvements in hemolysis and ineffective erythropoiesis in clinical studies across 3 hemolytic anemia, namely CK deficiency, sickle cell disease and non transfusion dependent thalassemia. As a reminder, the primary endpoint of Energize C is a transfusion reduction response defined as a 60% or greater reduction in trans red blood cell units with a reduction of equal or more than 2 units of transfused red blood cells in any consecutive 12 week period through week 48 compared with baseline.

Speaker 1

This definition allows 2 ways for patients to achieve a reduction in transfusion burden. First, via an increased time interval between transfusions or second, through the use of fewer units or both. In order to standardize as much as possible the standard of care in this large global trial, each patient had a specific hemoglobin threshold value that was calculated based on their individual transfusion history prior to enrolling in the trial. Each patient's individual hemoglobin threshold value determines the hemoglobin value by which they will receive a transfusion in the trial. As the mechanism of action of mitapivat focuses on increasing red blood cell health and decreasing hemolysis, we are hoping to maintain hemoglobin levels above this threshold and reduce the need for transfusions.

Speaker 1

We designed this study by incorporating learnings from prior studies as well as agency feedback and believe the primary endpoint dynamic assessment period reflects what matters to patients and physicians as well as regulators. We now look forward to the readout of this study in the Q2 and are planning to submit a single regulatory filing to the FDA and compacting data from both energized and energized T by the end of this year, seeking a label that covers people living with all subtypes of thalassemia. Turning to sickle cell disease. Enrollment in the Phase 3 portion of the RISE UP study of mitapivat continues to progress and we are on track to complete enrollment by the end of the year. We have increasing conviction about the role of mitapivat in sickle cell disease, where we believe we have the potential to be both best in class and 1st in class.

Speaker 1

We look forward to reporting top line data from this 52 week study next year and believe firmly in mitapivat's potential to address the high unmet need in this disease by improving anemia, making patients feel better and reducing sickle cell pain crisis. We also remain on track to deliver on all milestones across the rest of our advancing pipeline. This quarter, we commenced dosing in the Phase 1 study of AG-one hundred and eighty one, an oral phenylalanine hydroxylase or PAH stabilizer for phenylketonuria abbreviated as PKU, a patient's population with limited treatment options. We are excited about the potential to introduce a novel mechanism of action for PKU treatment and look forward to providing an update on next steps as enrollment progresses. Based on the data generated in the Phase 2a study of our novel PK activator AG946 in lower risk MDS, we plan to increase the doses evaluated in the upcoming Phase 2b study, which we expect to initiate in mid-twenty 24.

Speaker 1

And in pediatric PKD deficiency, we expect to complete enrollment of the Phase 3 ACTIVATE KIT study in the middle of this year and we also now expect to report top line data from the Phase 3 ACTIVATE KIT study in mid-twenty 24 sooner than our original projection of year end. This is a very exciting time at Agios and we look forward to providing additional readouts and progress as we proceed through the year. In particular, we are looking forward to sharing with you the status of submissions for EHA when they are made public in a couple of weeks. With that, I will now turn the call over to Sarah. Thanks, Sarah.

Speaker 1

Today, a diagnosis of thalassemia can be daunting for patients and their families. Regardless of the disease subtype, treatment options are limited and the burden of disease as well as the associated cost of care is significant. All forms of thalassemia, including non transfusion dependent thalassemia brings higher rates of serious morbidities, reduced quality of life and a heightened risk of premature death. There are approximately 6,000 diagnosed adults living with leukemia in the U. S.

Speaker 1

Approximately 4,000 of whom are non transfusion dependent and have no available treatment options today. As Sarah mentioned, this patient population was studied in our energized clinical trial, which demonstrated the benefit of mitapivat in non transfusion dependent thalassemia patients. The remaining 2,000 are transfusion dependent and have no oral treatment options. We are eagerly awaiting the data from our Energize C study in the Q2, which is focused on these transfusion dependent patients. Our goal with mitotiva is to transform the treatment of thalassemia by becoming the 3rd therapy approved for all subtypes of the disease.

Speaker 1

Galvanized by the positive data from the Energize study and the potential for positive data from Energize C, our commercial organization is actively preparing to address this high unmet need with a potential launch in thalassemia next year in the U. S. As we have highlighted, the thalassemia market in the U. S. Has more favorable commercial dynamics than CK deficiency.

Speaker 1

In addition to the data we are generating through the mitapivac clinical development program, we believe there are 3 key factors that have the potential to support adoption of MYCAPIVA in thalassemia in the U. S. 1st, driven by the availability of newborn screening and well established ICD-ten codes, the diagnosis rate in thalassemia is high with many patients diagnosed before adulthood. 2nd, both patients and providers are concentrated in limited number of centers with approximately 50% of all diagnosed patients treated at fewer than 150 affiliated practices, providing a clear focus for our initial launch. And third, our clinical trial sites in the U.

Speaker 1

S. Are in some of the main centers of excellence. So many treating physicians will have firsthand experience with mitapivat. Given this data and mitapivat target profile, we believe we are well positioned to provide a potential foundational treatment option for patients with thalassemia regardless of subtype. Our team is focused on 4 areas of thalassemia U.

Speaker 1

S. Launch preparation. 1st, we continue to advance extensive market research and claim data analysis to further refine our market insights and our HCP targeting. 2nd, we are rolling out a disease education campaign in the coming weeks designed for both patients and clinicians, highlighting the long term complications and burden of disease across all thalassemia subtypes, particularly non transfusion dependent patients who suffer from the misconceptions that they are at less risk. 3rd, we are executing a disciplined expansion of our commercial and medical teams to right size the organization for a successful launch in this larger, but still rare market.

Speaker 1

And 4th, our market access team is already engaging with payers on disease state education. I'm proud that our team has obtained broad market access for cytokines in PK deficiency and we look forward to the same strong outcome in thalassemia. In addition to the U. S, we aim to maximize the potential of markets outside the U. S.

Speaker 1

Through coordinated regulatory filings, which we intend to pursue with 1 or more partners. These markets include the Gulf region, which is home to approximately 70,000 thalassemia patients and some of the leading treatment centers in our clinical trials. Let me now provide an update on the current launch of Thyrokines in PK deficiency. In the Q1 of 2024, we generated $8,200,000 in net Thyrokines revenue compared to 7 point $1,000,000 in the Q4 of 2023. In the U.

Speaker 1

S, a total of 188 patients have completed a prescription enrollment form including 10 in the Q1 of 2024, a 6% increase versus the prior quarter. This has translated into net 120 patients on therapy, a 10% increase versus the prior quarter. Patients on therapy continue to spend from a growing and diverse prescriber base of 162 physicians and represent a broad demographic and disease manifestation range that is consistent with adult PK deficiency population in the U. S. We continue to be encouraged by the persistency of patients on treatment and remain focused on efficiently identifying providers likely to treat patients with PK deficiency.

Speaker 1

We believe the capabilities we continue to strengthen through the current launch, including efficient targeting analytics, patient and ACP awareness and education and patient access will provide a firm foundation to maximize potential future U. S. Launches of mitapivat in thalassemia in 2025 and in sickle cell disease in 2026. Above all, Agios is once again incredibly proud to be pioneering a potential new therapy for these 2 underserved patient populations. With that, I'll turn the call over to Cecilia.

Speaker 1

Thanks, Vera. Our Q1 2024 financial results can be found in the press release we issued this morning and more detail will be included in our 10 Q, which will be filed later today. Let me now take a moment to provide some context and highlight a few key points. Q1 2024 net Pyrocand revenue was $8,200,000 an increase of $2,600,000 compared to the Q1 of 2023. Consistent with other rare disease launches, growth to net has been and is expected to be in the 10% to 20% range on an annual basis.

Speaker 1

Cost of sales for the quarter was $600,000 R and D expenses were $68,600,000 for the Q1, an increase of $1,300,000 compared to the Q1 of 2023. This increase was primarily driven by an increase in process development offset by a decrease in workforce related expenditures. SG and A expenses were $31,000,000 for the Q1, an increase of $2,600,000 compared to the prior year quarter. This was primarily driven by an increase in commercial related activities as we prepare for the potential approval of Pyrogant in thalassemia. As a reminder, as part of the divestiture of our oncology business to Servier, we retain rights to a potential $200,000,000 milestone upon FDA approval of 15% royalties on potential U.

Speaker 1

S. Net sales. We ended the quarter with cash, cash equivalents and marketable securities of approximately $714,300,000 We expect that this balance together with anticipated product revenue, interest income and the potential for acitinib milestone will enable the company to fund our operating expenditures and capital expenditures through several value creating milestones and at least into 2026. This guidance does not include cash inflows that could extend the runway beyond 2026, including the potential royalties or royalty monetization from vorasidenib, commercializing Mifephibir outside of the U. S.

Speaker 1

Through 1 or more partnerships or other potential strategic business or financial agreements. We remain focused on creating shareholder value, including by proactively managing our cost base and deploying a disciplined cash allocation approach as we prepare to support potential future launches of Paragard. As we move toward additional potential value creating milestones in the near term, I am confident that our strong balance sheet will enable us to execute from a position of strength.

Speaker 4

I will now turn the

Speaker 1

call back over to Brian for his closing remarks.

Speaker 3

Thanks Cecilia. Driven by a novel and differentiated mechanism of action that improves red blood cell health and a clinical data package that includes positive data spanning 3 hemolytic anemia, we believe mitapivat is poised to become a 1st in class and best in class treatment option for multiple indications and with potential to become a multibillion dollar franchise. I'm very proud of our team for steadily delivering significant progress and looking ahead, I'm truly excited about the catalyst rich 24 months in front of us. As we continue to take steps toward realizing our vision of becoming a leading rare disease company, we'll continue to strive to be responsible stewards of our balance sheet and evaluate meaningful opportunities for value creation. Finally, I'd like to thank all of our employees for their hard work and dedication to our mission of developing and delivering transformative medicines that elevate and extend the lives of patients living with rare diseases and all of our partners, including the patients, physicians, caregivers and participants in our clinical development programs.

Speaker 3

With that, we'll now open the call for questions.

Operator

Thank you. And our first question will come from the line of Gregory Renza with RBC Capital Markets. Your line is now open.

Speaker 5

Great, thanks. Hey, good morning, Brian and team. Congratulations on the progress and thanks for taking my questions.

Speaker 2

Sure. Thanks, Craig.

Speaker 5

Thank you. Brian, we certainly appreciate the more precision and timing for the Energize T data. I'm just curious that just narrowing it at least from midyear to Q2, can you just comment a bit on sort of the relevance factors now, what needs to be done and maybe some of the influences around just getting it to that narrower earlier timeframe on the Q2? And maybe related to that, just remind us just the mechanics on patients rolling over to the open label extension? Thanks so much.

Speaker 3

Sure. Thanks, Greg. I'm going to let Sarah talk about the mechanics. But I will say that we are very much looking forward to the data. It's a very special time at Agios because everyone who's followed us and I know Greg you certainly have been there knows that we were delighted to have the energized data back in January.

Speaker 3

And this study Energize T is twice the length. That was a Energize was a 24 week study. This is 48 weeks. And we know there's a lot of interest and so we were delighted to give, as you said, a little more granularity on the timing. But Sarah, you want to talk about some of the mechanics?

Speaker 1

Sure. And so the granularity of the timing is driven by the patient. The last couple of patients in the study, their decisions to roll over into open label extension or not. Because as you know, if a patient would have decided to not go into open label, then they would need to taper down the drug and then have a safety follow-up visit, which could which really drive the timeframe that creates basically a 7 week difference between potential readouts. So now we have much more precision around that type of information and so we can narrow our timeframe.

Speaker 1

So that's why we've updated our guidance today. We're very excited about where we are right now and the team is very focused on delivering the next steps for this specific trial.

Speaker 3

Yes. And so this was the exact same scenario that we faced last year when we read out the RISE UP Phase 2 data for sickle cell disease and of course the same for Energize in January. The big picture that I'll just end with here is that, our intent as we noted in our prepared comments is to file both studies together to target a label that encompasses all thalassemia patients, all subtypes. And all that said, of course, we look forward to the data, but we're already in a significant position of strength given the Energize data and the fact that as you heard from Sveta, that addresses already 2 thirds of the population in the U. S.

Speaker 3

With non transfusion dependent thalassemia.

Speaker 5

That's great, Brian. Thank you very much. Maybe just a follow-up just broadly on the landscape, certainly with PK activation competition with the tavapivin from Novo maybe having some timelines now established. What do you make of that? How do you think about the landscape longer term?

Speaker 5

You're certainly moving fast, but as you think about the jockeying between the two offerings for patients ultimately? Thanks again.

Speaker 3

Yes. Thanks for the question. I mean, certainly, we take a lot of pride in the fact that we're the worldwide leader in PK activation. And I think given some of the recent competitive updates that have been made public, specifically with the other PK activator, Itavo Pivot from Novo Nordisk. We took note of the most recent updated timelines and that's why you heard in our prepared comments that we've often said we feel very confident in our best in class positioning.

Speaker 3

We feel increasingly confident now across both thalassemia and certainly sickle cell disease with 1st in class potential. And that all leads to this franchise having multibillion dollar potential with mitapizat. We're in a real significant position of strength.

Speaker 1

Yes, absolutely. And when we think commercially for thalassemia, we are definitely very much ahead. What we've heard from Norway is that they'll be reading out data in 2020 from their Phase 2 study and we don't know about their commitment to Phase 3. So for thalassemia, we're very excited to pioneer the way and having a treatment option for all thalassemia patients. In sickle cell disease, equally exciting on our part, again, giving us an opportunity to basically bring the first gene of this product with the potential to hit all the clinically meaningful and commercially meaningful endpoints of hemoglobin improvement, reductions in VOC and improvement in quality of life cells.

Speaker 1

We're excited to be moving forward.

Speaker 3

And this is where we're taking full advantage of SPETA's expertise, deep expertise in rare disease launches. This is what you live for is a chance to change the lives of patients and thalassemia is certainly that scenario and this launch has the potential to be next year, so coming soon.

Operator

Thank you. Our next question will come from the line of Divya Rao with TD Cowen. Your line is now open.

Speaker 6

Good morning. This is Divya on for Mark. Thanks for taking our question. One on the thalassemia, the thalassemia, the transfusion dependent thalassemia trial coming up. Can you walk us through the powering assumptions for the primary endpoint?

Speaker 6

And then I have a follow-up.

Speaker 3

Sure. Thanks, Divya. Sarah, you want to take that?

Speaker 1

Yes. So we haven't spoken in detail about the powering assumption underlying the number, but we of course have leveraged all information available to make sure that we have very good statistical justification for this trial and feel confident in the primary endpoint and the sample size that we have selected. The primary endpoint by itself, as you know, is a 50% reduction in any 12 week rolling period, which we believe can provide very meaningful assessment of a reduction in transfusion burden for thalassemia patients.

Speaker 6

Got it. And then just quickly as a follow-up, for AD-nine forty six, do we still expect to see the full data from the Phase IIa trial in sometime this year?

Speaker 1

So we haven't spoken about when we are going to release the Phase IIa data as the team is assessing the best opportunity on when to do so and the best conference, also in line of when we are getting Phase 2b up and running. So very focused on that, very excited. We have a milestone around Phase 2b for this year and so we're

Speaker 4

on track to deliver that.

Speaker 7

Okay. Thank you.

Operator

Thank you. And our next question comes from the line of Eric Schmidt with Cantor. Your line is now open.

Speaker 8

Good morning, everyone. Thanks for taking my question and congrats on all the progress. Maybe just two quick ones. First for Brian and Sarah on this thalassemia filing. 1, I guess, can you confirm whether it's going to be an NDA filing?

Speaker 8

It sounds like you're already preparing that filing. Just to be very specific, if for some reason the TDP study falls short, you'd still be filing for non transfusion dependent patients. Is that correct?

Speaker 1

So thanks for the question. So our goal is to file the 2 studies together. As you know, we have already the first part of the data. So indeed, our filing preparations are completely underway and team is on track to deliver. And now we're waiting for that second study.

Speaker 1

So the fact is that we already have great data for 2 thirds of the patient population. So we, of course, want the 2nd study to deliver and make it a great story. If the study would fall short, then it truly depends on what the data would show, how we would approach that. But our intent is to file for all thalassemia patients and get that broad indication.

Speaker 8

Okay. Thanks for the clarification, Sarah. And just a second question on the sickle cell Phase 3 study that's enrolling. Can you provide any kind of color on how that's going and what percent of the study is already on board or maybe a finer insight into your timelines for next year? Thanks.

Speaker 1

Sure. So we are very excited about the RISE UP study. Everything is going well and the team is doing an amazing job getting that study completely up and running and we are on track with our enrollments and so are completely expecting to be able to deliver towards full enrollment towards the end of this year. We do not guide to specific numbers expected in specific weeks or months. These things are very rapidly changing in big Phase III trials.

Speaker 1

So we guide towards bigger milestones.

Speaker 8

Thanks very much. Congrats again.

Speaker 3

Thanks a lot, Eric.

Operator

Thank you. Our next question will come from the line of Alec Stranahan with Bank of America. Your line is now open.

Speaker 9

Hey guys, thanks for taking our questions. Just a couple from us. First, a commercial question. Assuming both transfusion and non transfusion end up on the label, any reason to think you'd need to maybe tailor your sales strategy differently between these two patient groups? Or will it be more or less the same, given they're likely seeing the same prescribers?

Speaker 9

And then I've got a follow-up.

Speaker 3

Sure. Thanks, Alex, and welcome to the Agios call. And Stedi, do you want to start off?

Speaker 1

Absolutely. We are super excited with seeing the positive data from the Energize study. And as I said, we are eagerly awaiting the data from the Energize tea as well. In terms of launch preparation, we are actively in a lab launch preparation mode. We believe that there is a high unmet need across best BOS patient populations, transfusion dependent and non transfusion dependent.

Speaker 1

We have a lot of data that we are basically our launch preparation strategy on, including claims data, availability at 5G10 codes and we have basically planned to approach in the same way both patient populations because of the same underlying pathophysiology of the disease, the connection between the two patient populations and as you said, the overlap in the prescriber base. Of course, you'll see a little bit more concentration in the non transfusion dependent patients in the KOL and centers of excellence setting and a bit more of the non transfusion dependent in the community setting, but that would not change the approach of deployment and communication and education.

Speaker 3

The only thing I would add is that with the transfusion dependent patients, there's obviously more regularity of clinician visits than you tend to see with the non transfusion dependent patients. But I'm really proud of the fact that Sveta and the team are prepared for all scenarios.

Speaker 9

Okay, perfect. And then just one question on the ex U. S. Opportunity. I know you've said that the plan would likely be to partner in ex U.

Speaker 9

S. Geographies. But any additional color you can provide around timing or scope of the ex U. S. Opportunity, just from a modeling perspective for us?

Speaker 9

Thank you.

Speaker 1

Absolutely. So in addition to actively preparing for launch in the U. S, we are also actively searching for the best possible partner for us to maximize the ex U. S. Opportunity.

Speaker 1

We've mentioned on several occasions that the Gulf region is a high priority for us given that there are 70,000 patients with a listemia in that region. Sarah and the team are preparing for regulatory submissions in these regions actively as well to submit as quickly as possible that that will allow us. And we'll provide updates as soon as we have progressed this discussion and identify the partner, but we are in a very strong position, especially now with a positive data from Energize to have a very high quality discussions with partners who have strong expertise in the region. We are excited about this because our clinical studies are also done in the region and there is a lot of careful advocacy and experience with the leukaemia ad mitativa, so equally an exciting place as the U. S.

Speaker 1

Launch as well.

Speaker 9

Great. Thanks again for the question.

Speaker 3

Thank you, Al.

Operator

Thank you. Our next question comes from the line of Tess Romero with JPMorgan. Your line is now open.

Speaker 7

Hi, good morning, Brian and team. Thanks so much for taking our question.

Speaker 3

Sure.

Speaker 7

Question for Cecilia here. I mean, just curious, can you walk us through the pushes and pulls on your balance sheet and how you think about means to potentially extend that runway and what your latest thinking is there? We noticed you included in your release today, how interested specifically are you in potentially a royalty monetization for vorasidenib? And then on the partnering side outside of the U. S, just to clarify that as comments here, could that occur this year?

Speaker 7

Thanks.

Speaker 1

Thanks, Tessa. It's Dara and we can add on that as you asked too.

Speaker 4

So yes, we have guided that we have our cash at least into 2026. We purposely left out a couple of things that

Speaker 1

you mentioned, so I'll cover some of those.

Speaker 4

But there's other things that we obviously continue to manage our cost base thoughtfully and in a disciplined way. We now are starting to prepare for the launch, but we waited to make sure we had the data before we went ahead with that. On the vora side, that's another option that we looked as we evaluate to extend our runway. We as we had in the prepared remarks, so Servier publicly announced that the FDA accepted their filing and they have a PDUFA date on August 20. And as a reminder, we have a $200,000,000 milestone on FDA approval and 15% royalties on U.

Speaker 4

S. Net sales for that. And we will evaluate opportunities to sell that, the whole amount or a portion of that as part of our way to extend the runway as we would do with other things. The other option or the other piece that we left out, as you mentioned, is the ex U. S.

Speaker 4

Portion. That can come in different shapes and forms and that's part of the reason we left it out. The timing, obviously, the teams are working to make sure we have the best partners and that is reflective of where the patients are like Freda described and we want to try to obviously do that as fast as possible to get access to those patients.

Operator

Thank you. And our next question comes from the line of Salveen Richter with Goldman Sachs. Your line is now open.

Speaker 4

Hi. This is Lydia on

Speaker 10

for Salveen. Thanks so much for taking our question. Just on the upcoming Energize T readout, could you just discuss the clinical bar for success here and what you're hoping to see in terms of that percentage of patients with a reduction in transfusion burden? And then should we expect this readout to kind of take a similar form to the earlier Energize readout? Thanks so much.

Speaker 1

Thanks for the question. So for in regards to the energized T readout, of course, we're hoping to hit on our primary end points with a reduction 50% reduction in any 12 week rolling period. It's we have not spoken about the exact difference we are shooting for between the placebo or and mitapibat. But in regards to the trial itself, it is of course well designed to be able to hit on a primary endpoint with the assumptions that are underpinning that design. What we know though is there is the clinical trial bar of what we're shooting for that 50% reduction.

Speaker 1

But in the real world, we know that the bar actually may be lower, because the what the compound the product would do is basically remove some of the clinic visits for patients if they can skip transfusions and that would have a big impact on quality of life for transfusion dependent thalassemia patients. I think Sarah can maybe add a little bit on what we expect for the real world. Absolutely. You mentioned it's our the clinical bar is obviously very important from a regulatory perspective. But what we hear both from patients and clinicians in terms of utilization, they'll be looking to the refusal transfusion burden even at the lower rate and they'll kind of frequency by a week on a patient's basis, it is very meaningful on actually healthcare system basis as well and clinicians visits is very meaningful.

Speaker 1

So we're super excited to and wait to see the data and getting ready actively for launch.

Speaker 3

And I'll just add maybe one last comment, which is that obviously we're going for statistical significance in the Energized T study. From past experience, we know that there could be the temptation for cross study comparisons as well. And I just want to emphasize that the compelling totality of the profile of mitapivat means we don't necessarily need numerical equivalence. What we need is statistical significance from the endpoint. That's what we're targeting.

Speaker 3

But the fact that this is an oral therapy means the patients are already in a way free of the clinic relative to other alternatives, I. E. Luspatercept. And we see that is on top of all else. That's another real advantage at the patient level.

Speaker 10

Thanks so much.

Speaker 3

You're welcome.

Operator

Thank you. And our last question will come from the line of Danielle Brill with Raymond James. Your line is now open.

Speaker 6

Hi, guys. Good morning. Thanks so much for taking my questions. First, I wanted to ask a question about the expected placebo response for energized tea. In the Patercept's trial, I believe the placebo response rate, as you defined it for your primary, was around 6%.

Speaker 6

Curious your thoughts on how the placebo population is able to achieve that level of reduction? And are you expecting a similar outcome in Energized Tea? And then as a quick follow-up, I'm just curious how you plan to announce the data. Will this be a top line PR or is it possible that you'll present it at EHA? Thank you.

Speaker 1

Thanks. Yes, so there is always fluctuation when you measure transfusion. So 6% for the placebo in regards to the primary endpoint definition is a very reasonable observation. And that is also because in clinical practice, sometimes patients get transfused based on symptom presence and things like that. So in regards to how we are going to announce the data, yes, we typically do a top line release via press release.

Speaker 1

We will not be able to present data at EHA because the timeframe for submission of data there is long gone. So that will that

Speaker 6

Understood. Thanks so much.

Speaker 3

Thank you.

Operator

Thank you. And this concludes today's question and answer session. I'll now turn the call back to Brian Gull for closing remarks.

Speaker 3

Thanks a lot, Norma, and thank you very much everyone for participating in today's call. I think it's hope it's clear. It's a very exciting time at Agios. We believe that we're well poised to deliver transformative new therapies for patients as well as creating significant long term value to shareholders. So thanks again and we look forward to speaking with all of you again soon.

Operator

This concludes today's conference call. Thank you for your participation. You may now disconnect. Everyone have a wonderful day.

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Earnings Conference Call
Agios Pharmaceuticals Q1 2024
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