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spk09: Good morning and welcome to OJEO's first quarter 2024 conference call. At this time, all participants are in a listen-only mode. There will be a question and answer session at the end. Please be advised that today's conference is being recorded at OJEO's request. I would now like to turn the call over to Chris Taylor, Vice President, Investor Relations, and Corporate Communications for OJEO's. Please begin.
spk02: Thank you, Operator. Good morning everyone and welcome to OJEO's conference call and webcast to discuss first quarter 2024 financial results and recent business highlights. You can access slides for today's call by going to the Investor section of our website ojios.com. On today's call, I'm joined by our Chief Executive Officer Brian Goff, Dr. Sarah Hewens, Chief Medical Officer and Head of R&D, Svetta Milanova, our Chief Commercial Officer, and Cecilia Jones, Chief Financial Officer. 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,
spk04: and events
spk02: 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
spk08: to Brian. Good morning everyone and thank you for joining us. Our mission at OJEO's 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 Minipivet'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 3rd, we reported positive data from the Phase III Energized Study of our EPK activator, Minipivet, marketed as pyrachine in patients with -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. As a reminder, -transfusion-dependent thalassemia accounts for approximately two-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.S. Complementing the Energized Study in -transfusion-dependent thalassemia, we continue to advance the Phase III Energized T-Study of Minipivet 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 Energized and Energized 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 III portion of the Rise Up Study of Minipivet in Sickle Cell Disease by the end of this year
spk00: and
spk08: reporting data from four additional Phase III studies by the end of 2025. Sarah will provide a detailed update on our progress and upcoming milestones across R&D in just a few minutes. Given the consistent positive data we've generated across the Minipivet Development Program and the high unmet need in our target disease areas, we believe Minipivet has the potential to transform the course of multiple hemolytic anemias by improving red blood cell health and to become a -billion-dollar franchise. To help realize the full commercial potential of Minipivet, 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 Minipivet in Thalassemia in 2025 and in Sickle Cell Disease in 2026. 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 first quarter with a strong cash position with approximately $714 million 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 voracidinib for the treatment of certain IDH mutant-diffused glioma. You'll recall that as part of the divestiture of Adios' oncology business to Servier, Adios retains rights to a potential $200 million milestone upon FDA approval of voracidinib and 15% royalties on potential U.S. net sales. If approved, voracidinib would become a first 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.
spk12: Thanks, Brian. As we approach the top line data readout for the Phase 3 Energized T study of Mitapifac in transfusion dependent thalassemia, I would like to highlight a few key elements of the Mitapifac development program in thalassemia. As a reminder, the Phase 3 program of Mitapifac in thalassemia, namely Energize and Energize T, was designed to deliver data across all subpopulations of thalassemia, including alpha and beta thalassemia and populations with different transfusion needs. As Brian mentioned, only patients with transfusion dependent beta thalassemia, which represents one third of U.S. patients, have an FDA approved treatment option. The other two 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 treatments. It is a common misperception that non-transfusion dependent or NPD 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 two thirds of total thalassemia patients in the U.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, our goal was to build upon our Phase 2 findings with a more rigorous way to measure a hemoglobin response in the Phase 3 Energize trial, which we defined in the primary endpoints as an increase of equal or more than one 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 mg mL mitopifaz BID demonstrated a highly statistically significant result on the primary endpoints of hemoglobin response rate, with .3% of patients in the treatment arm achieving a hemoglobin response versus .6% of patients in the placebo arm. In addition, treatment with mitopifaz also resulted in statistically significant improvements in both key secondary endpoints, including a change from baseline in average rapid fatigue score and important patient report as measure of how patients feel. In line with its novel mechanism of action that improves overall red blood cell health, mitopifaz 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 pre-specified subgroup analyses favoritly talk about 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. Turning to energize B, let me highlight three 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, mitopifaz's novel mechanism of action directly addresses the underlying pathophysiology of hemolysis in all thalassemia subtypes. Second, this is a similar approach to the one we took for our PK deficiency program. In that case, in the phase three active AC study of mitopifaz in thalassemia, which regularly transfuse adults with pyruvate kinase deficiency, mitopifaz demonstrated a statistically significant and clinically meaningful reduction in transfusion burden. These data, together with positive data from the phase three active AC study in patients with PK deficiency who are not regularly transfused, led to FDA approval of mitopifaz for adults with PK deficiency regardless of transfusion status, and we look forward to the potential to achieve the same in thalassemia. And third, in line with mitopifaz's mechanism of action, we have seen consistency in the data with improvements in hemolysis and ineffective erythropoiesis in clinical studies across three hemolytic anemias, namely PK deficiency, sickle cell disease, and non-transfusion dependent thalassemia. As a reminder, the primary endpoint of energized C is the transfusion reduction response defined as a 60 percent or greater reduction in transfused red blood cell units with a reduction of equal or more than two units of transfused red blood cells in any consecutive 12-week period through week 48 compared with baseline. This definition allows two 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 mitopifaz 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. We designed this study by incorporating learnings from prior studies as well as agency feedback and believe the primary endpoint's 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 second quarter and are planning to submit a single regulatory filing to the FDA and composing data from both Energize and Energize-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 three portion of the RISUPS study of mitopifaz 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 mitopifaz in sickle cell disease where we believe have the potential to be both best in class and first in class. We look forward to reporting top line data from this 52-week study next year and believe firmly in mitopifaz 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 commence dosing in the phase one study of AG181, 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 two A study of our novel PK activator AG946 in lower risk MDS, we plan to increase the dose of evaluators in the upcoming phase two B study, which we expect to initiate in mid 2024. And in pediatric PK deficiency, we expect to complete enrollment of the phase three Activate Kits study in the middle of this year, and we also now expect to report stop line data from the phase three Activate Kits T study in mid 2024, sooner than our original projection of year end. This is a very exciting time at AGEOs, and we look forward to providing additional readouts and progress as we proceed through the year. In particular, we're looking forward to share 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.
spk13: Thanks, Sarah. 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, bring high rates of serious morbidities, reduced quality of life, and a heightened risk of premature death. There are approximately 6,000 diagnosed adults living with thalassemia in the U.S., 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 Energize clinical trial, which demonstrated the benefits of metativates 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 second quarter, which is focused on these transfusion dependent patients. Our goal with metativates is to transform the treatment of thalassemia by becoming the first therapy approved for all subtypes of the disease. 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 these high unmet needs 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 TK deficiency. In addition to the data we are generating through the Metativates clinical development program, we believe there are three key factors that have the potential to support adoption of metativates in thalassemia in the U.S. First, driven by the availability of newborn screening and well established ICD-10 codes, the diagnosis rate in thalassemia is high with many patients diagnosed before adulthood. Second, 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 trials sites in the U.S. are in some of the main centers of excellence, so many treating physicians will have first-hand experience with metativates. Given this data and Metativates target product 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 four areas of thalassemia U.S. launch preparation. First, we continue to advance extensive market research and claim data analysis to further refine our market insights and our HEP targeting. Second, 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 misconception that they are at less risk. Third, 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. And fourth, 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 pyrokin 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. through coordinated regulatory filing, which we intend to pursue with one 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 pyrokin in PK deficiency. In the first quarter of 2024, we generated $8.2 million in net pyrokin revenue compared to $7.1 million in the fourth quarter of 2023. In the U.S., a total of 188 patients have completed a prescription enrollment form, including 10 in the first quarter 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 the 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. We believe the capabilities we continue to strengthen through the current launch, including efficient targeting analytics, patient and HCP awareness and education, and patient access, will provide a firm foundation to maximize potential future U.S. launches of metapivot in thalassemia in 2025 and in sickle disease in 2026. Above all, Agios is once again incredibly proud to be pioneering a potential new therapy for these two underserved patient populations. With that, I will turn the call over to Cecilia.
spk11: Thanks, Vera. Our first quarter 2024 financial results can be found in the press release we released this morning and more detail will be included in our 10 queue which will be fired later today. Let me now take a moment to provide some context and highlight a few key points. First quarter 2024 net pyroclin revenue was $8.2 million, an increase of $2.6 million compared to the first quarter of 2023. Consistent with other rare disease launches, growth to net has been and our basis. Cost of sales for the quarter was $0.6 million. R&D expenses were $68.6 million for the first quarter, an increase of $1.3 million compared to the first quarter of 2023. This increase was primarily driven by an increase in process development expenses offset by a decrease in workforce related expenditures. ST&A expenses were $31 million for the first quarter, an increase of $1.6 million 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 pyroclin in Thalassemia. As a reminder, as part of the divestiture of our oncology business to Serbia, we retain rights to a potential $200 million milestone upon FDA approval of oracidinib and 15% royalties on potential US net sales. We ended the quarter with cash, cash equivalents and marketable securities of approximately $714.3 million.
spk12: We expect
spk11: that this balance, together with anticipated product revenue, interest income and the potential for oracidinib 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 a runway beyond 2026, including the potential royalties or royalty monetization from oracidinib, commercializing metapayment outside of the US through one 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, and we prepare to support potential future launches of pyroclin. 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. I will now turn the call back over to Brian for his closing remarks.
spk08: 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 three hemolytic anemias, we believe Metapivet is poised to become a first in class and best in class treatment option for multiple indications and with potential to become a multi-billion 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. With that, we'll now open the call for questions.
spk09: Thank you. To ask your question, you'll need to press star 1-1 on your telephone. To assure your question, please press star 1-1 again. We ask that you please limit your questions to one question and one follow-up. Please stand by, we'll compile the Q&A roster. One moment for our first question. Our first question will come from the line of Gregory Renzza with RBC Capital Markets. Your line is now open.
spk05: Great, thanks. Hey, good morning Brian and team. Congratulations on the progress and thanks for taking my questions. Sure. Thanks, Greg. Thank you. Brian, we certainly appreciate the more precision and timing for the Energize-T data. I'm just curious, just narrowing it at least from mid-year to second quarter, can you just comment a bit on sort of the relevant factors now, what needs to be done and maybe some of the influences around just getting it to that narrower earlier time frame on the second quarter and maybe related to that, just remind us just the mechanics on patients rolling over to the open label extension. Thanks so much.
spk08: 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 Energize data back in January and this study Energize-T is twice the length. 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 mechanics?
spk12: 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 drives the time frame. That creates basically a seven-week difference between potential readouts. So now we have much more precision around that type of information and so we can narrow our time frame 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.
spk08: Yeah, 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 two-thirds of the population of the US with non-transfusion dependent thalassemia.
spk05: 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 Tata Piven from NOVO maybe having some timelines now established, what do you make of that? How do you think about the landscape longer term? You're certainly moving fast, but as you think about the jockeying between the two offerings for patients ultimately. Thanks again. Yeah, thanks
spk08: 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, Etavo 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 -in-class positioning. We feel increasingly confident now across both thalassemia and certainly sickle cell disease with -in-class potential and that all leads to this franchise having multi-billion dollar potential with Mitapivad. We're in a real significant position of strength.
spk13: Yeah, absolutely and when we think commercially for thalassemia, we are definitely very much ahead. What we've heard from NOVO is that they'll be reading out data in 2026 from their phase two study and we don't know about their commitment to phase three. So for thalassemia, we're very excited to pioneer the way and having a treatment option for all thalassemia patients. In sickle cell disease, we are progressing with the recruitment of our clinic phase three clinical trial, everything going on plan. What we've heard with Etavo Pivot is they are expecting sickle cell disease data in 2027 which will be put them behind our anticipated launch in 2026, again giving us an opportunity to basically bring the first in NOVO this product with the immobilization improvement, reductions in VOC and improvement in quality of life. So we're excited to be moving forward.
spk08: And this is where we're taking full advantage of SPEDA'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.
spk09: Thank you. Our next question will come from the line of Divya Rao with TD Cowan. Your line is now open.
spk10: Good morning, this is Divya on her mark. Thanks for taking our question. One on the thalassemia, the transfusion dependent thalassemia trial coming up, can you walk us through the powering assumptions for the primary endpoint and then I have a follow up.
spk08: Sure, thanks Divya. Sorry, you want to take that?
spk12: 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.
spk10: And then just quickly as a follow-up, for the for 8946, can we still expect to see the full data from the phase 2A trial sometime this year?
spk12: So we haven't spoken about when we are going to release the phase 2A 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 on track to deliver that.
spk10: Okay, thank you.
spk09: Thank you. And our next question comes from the line of Eric Smith with Cantor. Your line is now open.
spk03: Good morning everyone. Thanks for taking my question and congrats on all the progress. Maybe just two quick ones, the first for Brian and Sarah on this thalassemia filing. One, I guess can you confirm whether it's going to be an NDA filing? 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?
spk12: So thanks for the question. So our goal is to file the two studies together. As you know, we have already the first part of the data so indeed our filing preparations are completely under way and STEAM is on track to deliver. And now we're waiting for that second study. So the fact is that we already have great data for two thirds of the patient population so we of course want the second study to deliver and make it a great story. If the study would fall short, then it's really 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.
spk03: Thanks for the clarification Sarah. Just a second question on the sickle cell phase three study that's enrolling. Can you provide any kind of color on how that's going? And 4% of the study is already on board or maybe a timeline for next year?
spk12: 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 three trials so we guide towards bigger milestones.
spk03: Thanks very much. Congrats again. Thanks a lot Eric.
spk09: Thank you. Our next question will come from the line of Alec Stranahan with Bank of America. The line is now open.
spk14: 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? And then I've got a follow-up.
spk08: Sure, thanks Alec and welcome to the audio call and study. You want to start off?
spk13: 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 T as well. In terms of launch preparation, we are actively in a LEMP launch preparation mode. We believe that there is a high and met need across best both patient populations, transfusion dependent and non-transfusion dependent. We have a lot of data that we are basing our launch preparation strategy on including claims availability of 5 to 10 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 see a little bit more concentration in the non-transfusion dependent patients, in the care well 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.
spk08: The only thing I would add is that with the transfusion dependent patients there is obviously more regularity of clinician visits than you tend to see with the non-transfusion dependent patients but I am really proud of the fact that Sfeda and the team are prepared for all scenarios.
spk14: Okay, perfect. Then just one question on the XUS opportunity. I know you said that the plan would likely be to partner in XUS geographies but any additional color you can provide around timing or scope of the XUS opportunity just from a modeling perspective for us. Thank you.
spk13: Absolutely. In addition to actively preparing for launching the US, we are also actively searching for the best possible partner for to maximize the XUS opportunity. We've mentioned on several occasions that the Gulf region is a high priority for us given that there are 70,000 patients with the leukemia 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 will provide updates as soon as we have a discussion and identify the partner but we are in a very strong position especially now with the positive data from Energize to have a very high quality discussion with partners who have strong expertise in the region. We are excited about it because our clinical studies are also done in the region and there is a lot of care well advocacy and experience with the leukemia and the negative as well. Equally an exciting place as the US launch as well.
spk14: Great. Thanks again for the question. Thank you.
spk09: Thank you. Our next question comes from the line of Tess Romero with JP Morgan. Your line is now open.
spk01: Hi. Good morning Brian and team. Thanks so much for taking our question.
spk03: Sure.
spk01: A 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 and potentially a royalty monetization for voracidinib and then on the partnering side outside of the US. Just to clarify that as comments here. Could that occur this year. Thanks.
spk11: Thanks Tess. I'll just start and we can add on the CS too. So yes we have guided that we have cash at least into 2026 and we purposely left out a couple of things that you mentioned so I'll cover some of those but there's other things like we obviously continue to manage our cost spaces 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 had in the prepared remarks the survey publicly announced that the FDA accepted your filing and they have a pitiful date on August 20th and as a reminder we have a 200 million dollar milestone on FDA approval and 15% royalties on US 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 wish to with other things. The other option or the other piece that we left out as you mentioned is the ex-US 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.
spk00: Thank you.
spk09: Thank you. And our next question comes from Lillian of Salveen Richter with Goldman Sachs. The line is now open.
spk07: Hi this is Lydia. 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 transition burden and then should we expect this readout to kind of take a similar form to the earlier Energize readout? Thanks so much.
spk12: Thanks for the question. So in regards to the Energize-T readout of course we're hoping to hit on our primary end point with a reduction 50% reduction in any 12-week rolling period. We have not spoken about the exact difference we are shooting for between placebo or end mitopin valve but in regards to the trial itself it is of course well designed to be able to hit on a primary end point 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 but in the real world we know that the bar actually may be lower because what 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 Seda can maybe add a little bit on what we expect for the real world.
spk13: Absolutely. You mentioned it Sarah. 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 reduced transfusion burden even at the lower rate and they'll consider that clinically meaningful because even extending the transfusion kind of frequency by a week on a patient's basis it is very meaningful on a actually healthcare system basis as well and clinicians visit is very meaningful. So we're super excited too and we're just to see the data and getting ready actively for launch.
spk08: And I'll just add maybe one last comment which is that obviously we're going for statistical significance in the Energize-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 Mitipivac means we don't necessarily need numerical equivalence. What we need is statistical significance from the end point that's what we're targeting 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. loose-pattern sept and we see that is on top of all else that's another real advantage at the patient level.
spk07: Thanks so much.
spk08: You're welcome.
spk09: Thank you and our last question will come from the line of Danielle Brill with Raymond James. Your line is now open.
spk06: Hi guys, good morning. Thanks so much for taking my questions. First I wanted to ask a question about the expected placebo response for Energize-T. In the Patterthup trial I believe the placebo response rate as you defined it for your primary was around six percent. Curious your thoughts on how the placebo population is able to achieve that level of reduction and are you expecting similar outcome in Energize-T? 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.
spk12: Thanks. Yeah so there's always fluctuation when you measure transfusions so six percent for the placebo in regards to the primary end point definition is a very reasonable observation. 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. We will not be able to present data at EHA because the time frame for submission of data there is long gone. So that is not in the plan.
spk06: Understood. Thanks so much. Thank you.
spk09: Thank you. And this concludes today's question and answer session. I'll now turn the call back to Brian Gough for closing remarks.
spk08: Thanks a lot Norma and thank you very much everyone for participating in today's call. I 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.
spk09: This concludes today's conference call. Thank you for your participation. You may now disconnect. Everyone have a wonderful day. Good morning and welcome to AGIOS first quarter 2024 conference call. At this time all participants are in a listen-only mode. There'll 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.
spk02: Thank you operator. Good morning everyone and welcome to AGIOS conference call and webcast to discuss first quarter 2024 financial results and recent business highlights. You can access slides for today's call by going to the investor section of our website agios.com. On today's call I'm joined by our chief executive officer Brian Goff, Dr. Sarah Hewins, chief medical officer and head of R&D, Svetta Milanova, our chief commercial officer and Cecilia Jones, chief financial officer. 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
spk04: events
spk02: 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
spk08: the call over to Brian. 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 Minnipivet'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 3rd we reported positive data from the phase three energized study of our lead PK activator Minnipivet marketed as pyrachine in patients with non-current
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