Q3 2024 Agios Pharmaceuticals Inc Earnings Call
The End
Speaker Change: Good morning and welcome to Agios 3rd Quarter 2024 Conference call. At this time, our participants are under this and only mode. They will be a question after session at the end.
Speaker Change: Please be a advice that today's call is being recorded at Agios Request. I would like to turn the call over to Chris Taylor, Vice President and Vice Relations and Corporate Communications for Agios.
Chris Taylor: Thank you, operator. Good morning, everyone and welcome to Agios Conference Call and Webcast. To discuss our third quarter, 2024, financial results and recent business highlights. You can access the slides for today's call by going to the investor section of our website agios.com.
Chris Taylor: On today's call, I'm joined by our chief executive officer Brian Goff, Dr. Sarah Heuens, Chief Medical Officer and Head of R&D, Svetta Milanova, Chief Commercial Officer and Cecilia Jones, Chief Financial Officer.
Chris Taylor: 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.
Chris Taylor: Actually, events and results could differ materially from those expressed or implied by any forward-looking statements.
Chris Taylor: As a result of various risks on certainties and other factors, including those we set for, and are most recent filings with the SEC and any other future filings that we may make with the SEC. With that, please turn the call over to Brian.
Brian Goff: Thanks, Chris. 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.
Brian Goff: We are especially focused on rare diseases that result in the dysfunction and destruction of red blood cells, including paravay kinestheticic and CERPKD, Thalusemia, sickle cell disease and low risk, mild dysplastics syndrome, or MDS.
Brian Goff: and Red Blood cells comprise over 80% of all cells in the human body, optimizing Red Cell Health represents an important path to improved individual health.
Brian Goff: Our lead product, Mitapivet, a Paravite Kines activator, has a novel mechanism of action that improves red blood cell metabolism and increases the amount of energy or ATP available to support red blood cell health.
Brian Goff: We are proud to have delivered positive data in phase three programs in PKD and Tallahassemia and we are excited about the prospects of Minipivad for sickle cell disease.
Brian Goff: Complimenting those data readouts and the continued progress across our current pipeline, I am delighted to confirm today that Agios has received $1.1 billion in milestone payments.
Brian Goff: Following FDA approval in August of Worcesterside Nym, which originated at Agios.
Brian Goff: These payments include a $95 million payment from royalty's pharma. In connection with the Vora Cydnib royalty purchase agreement, Agios announced in May 2024.
Brian Goff: and a $200 million payment from Serbia in connection with Agios' is the best of it's oncology business in 2021.
Brian Goff: Given the positive phase 3 data readouts of Minipivat in Fallosemia accomplished this year, and the recently announced achievement of full enrollment of the phase 3 rise up sickle cell disease study.
Brian Goff: We aim to deploy this strong cash base to prepare for the potential launches of Minute Pivot in Thalicemia in 2025 and in sickle cell disease in 2026.
Brian Goff: Beyond this significant infusion of capital, we also made important progress across our advancing clinical pipeline.
Brian Goff: First, we announced top line data from the Phase 3 Activate Kids T Study of Mitapivat in children with PK deficiency who are regularly transfused. Our first pediatric study of Mitapivat.
Brian Goff: and second, we achieved two milestones in our Clinical Development Program for Lower Risk MDS.
Brian Goff: We announced that the FDA granted orphan drug designation to our potent PK activator Teva Pivot. Formerly known as AG946 for the treatment of Milo-Despostics Syndrome's or MDS.
Brian Goff: and we have initiated enrollment and begun patient dosing in the phase 2B study of Teva Kivat in lower risk MDS.
Brian Goff: and Area with Profound on that need and significant market growth.
Brian Goff: Importantly, as we round out a very productive and successful 2024, we are on track to achieve our key remaining milestone for this year.
Brian Goff: Submission of an S&DA for Mitipivet in Fallosemia by the end of the year, seeking a broad label that includes adults living with all subtypes of Fallosemia.
Brian Goff: Sarah will provide a detailed update on our progress and upcoming milestones across R&D in just a few minutes.
Brian Goff: Leveraging the positive data observed in the energized T phase 3 studies of mid-apivat are expanding commercial organization is actively preparing for a potential U.S. launch of mid-apivat in Palacemia in 2025.
Brian Goff: and Svetta will provide greater detail on the market opportunity in Thalassimia and the team's robust preparation as well as an update on our current launch in PKD in just a bit.
Brian Goff: Finally, as you'll hear from Cecilia, we ended the third quarter with a very strong cash position, with approximately $1.7 billion in cash and investments on the balance sheet.
Brian Goff: which includes the $1.1 billion in milestone payments we receive following FDA approval of more
Brian Goff: and before handing off to Sarah I want to emphasize how incredibly proud we are for all that's been achieved thus far in 2024. How intensely focused we remain on finishing the year strongly and on enthusiasm for continued progress.
Brian Goff: in 2025 and beyond. With that, I'll now turn the call over to Sarah.
Sarah Heuens: Thanks Brian. Just recently we were able to attend two significant meetings where we continued engaging with the community.
Sarah Heuens: At both the Academy for Sickle Cell and Tala Cine at Conference in the SCDAA Annual Convention, we were reminded once again about the difficult journey for Sickle Cell patients. And it reinforces our conviction for what we are aiming to bring to patients.
Sarah Heuens: Turning to sickle cell disease, we are saddened by the recent events impacting this community and continue to be step-fast in our commitment to deliver a novel oral therapy that potentially improves the neemia, COC and how patients feel and function, particularly with respect to fatigue.
Sarah Heuens: Given the positive data we generated in the phase 2 portion of the rise of the study, as well as the positive data we have generated in other humolithic inemia that share a common path of physiology.
Sarah Heuens: We are excited about the potential for Mithapivat to become a convenient first-in-class and best-in-class treatment option that improves red blood cell health for patients living with sick-cell disease.
Sarah Heuens: As announced last week, we are very pleased that we met our goal of completing enrollment in the safe free rise up study by the end of the year. Just over a year after we began recruiting this cohort.
Sarah Heuens: As a reminder, the Phase 3 study had a planned sample size of 198 subjects, randomized 2-1 to meet WVB's first placebo across approximately 100 trial sites globally.
Sarah Heuens: There are two primary and points. He McLoban response defined as an increase of at least one gram per deserber leader in average he McLoban concentrations over weeks 24 to 52 compared with day's line and annualized rate of sickle self pain crises.
Sarah Heuens: Driven by a differentiated mechanism of action, we believe that the top of us has potential to address a high-unmet need in this disease by improving anemia, reducing sickle-cell pain crisis and making patients feel better. We look forward to reporting top-line data from the Phase 3 next year.
Sarah Heuens: For Thalasemia, we aim to deliver the first therapy of Bruce for all Thalasemia subtypes.
Sarah Heuens: Based on the positive data generated in both the Phase 3 Energized Study of Metapyvac in adults with non-transdusion dependent alpha or vasatolosemia
Sarah Heuens: and the 6th 3 Energized C study of Metapyvata in adults with transfusion dependent Alpha or Beta Talsemia. We believe Metapyvata has the potential to become a foundational and convenient oral treatment options for all subtype of Talsemia, including all genotypes and all levels of transfusion burden.
Sarah Heuens: We are working diligently to progress the filing and continue to expect to file an S&DA by the end of this year, seeking a label that includes adults living with all subtypes of Palestinian.
Sarah Heuens: In pediatric BK deficiency, we were pleased to announce top-line data from the Phase 3 Activate Kids Study of Metapurov's and Children with BK deficiency who are regularly transfused.
Sarah Heuens: We are proud of this trial because first this is the first phase 3 study to report data in this population and importantly it's the first completed Pediatric Study of Mittapivat for Agios
Sarah Heuens: While the pre-specified statistical criteria in front of Brian Goff and Port was not met, we believe these results were clinically meaningful.
Sarah Heuens: Given the percentage of patients that achieved the transfusion reduction response, including those who had transfusion free response with no red blood cell transfusions, and those who achieved a normal hemoglobin response defined as hemoglobin concentrations within normal limits at least once in eight weeks or more after transfusion.
Sarah Heuens: And second, 60 results from a WS working systems with the safety profile for a WS previously observed in adults with PK deficiency who are regularly transfused.
Sarah Heuens: With enrollment completed in the phase 3 activate, kids shrug will look forward to an anticipated data readout next year.
Sarah Heuens: In parallel with the progress we continue to make across the mid-topic of development program, this quarter we also made important advancements in our Zepapyphot program
Sarah Heuens: First, we initiated the Phase 2 study of the Bhaktivat in Lower Risk MDS
Sarah Heuens: This open-level study will enroll at sortle of 60 individuals for transfusion dependent lower risk MDS across three dose levels Then milligrams, 50 milligrams, and 20 milligrams, each of which is greater than the five milligrams dose that was evaluated in the previous phase to a study
Sarah Heuens: Each those-level cohort will be opened for enrolments sequentially without pauses between the cohorts, meaning that we will first enroll 20 patients in the 10-milligram dos cohort, and once those slots are filled, we will begin enrolling 20 patients in the 15-milligram dos cohort, and then we will do the same for the third cohort of 20 patients at the 20-milligram dos.
Sarah Heuens: There is a 24-week-court period for each cohort followed by a 156-week extension period.
Sarah Heuens: The primary endpoint of this study will be transfusion in the pendants, defined as remaining transfusion free for at least 8 consecutive weeks during the poor period.
Sarah Heuens: Secondary and Points will include safety, change in hemoglobin and additional measures of anemia.
Sarah Heuens: Similar to Tala Simia, with this program, we aim to deliver the first oral therapy that addresses an Indian due to ineffective aerophoys in lower risk MDS, which affects approximately 75,000 to 80,000 patients in the US and EU-5 and accounts for approximately 70% of all MDS cases.
Sarah Heuens: Also this quarter, we were delighted to receive FDA Orphan drug designation for the Vapiva for the treatment of MDS
Sarah Heuens: This underscores the importance of bringing new oral treatment options to patient suffering from dysverid disease and we expect to provide additional milestone timelines once enrollment is further along.
Sarah Heuens: Looking forward, we are excited to be attending the upcoming annual meeting of the American Society of Himadology, Ash, and look forward to the publication of accepted abstract on November 6th. With that, I will now turn the call over to SETA.
SETA: Thanks, Sarah. As we prepare for a potential launch in Tala Simia, we are reminded of the significant and met need in this disease. And also, the value-pire-okine may bring to Tala Simia patients a crock-all sub-tides.
SETA: We continue to validate this through the powerful patient connections with experience at conferences like AskCut and in local community meetings at selected patient centers.
SETA: Stalosimia is much more prevalent than big-a-deficiency, with approximately 6000 adults diagnosed in the U.S.
SETA: Approximately 4,000 have come are non-transfusion dependent.
SETA: The diagnosis rate in the last 28 high, with many patients diagnosed before adulthood.
SETA: Given the availability of claims data and ICD-tank holds, these patients are known to the healthcare system which provides importance clarity for our launch preparation.
SETA: Threatment options are limited, especially for patients with non-transfusion dependent disease, with most patients relying on support if therapy.
SETA: All forms of the La Cemia, bring high rates of serious morbidities, reduced quality of life and a heightened risk of premature death.
SETA: The burden of disease is high and the associated cost of care is significant.
Speaker Change: Galvanite by the positive data from the Energise and Energise T-studies. Our commercial organization is actively preparing to address this high and met need with a potential US launch of Fire O'Kines in Tala Simia next year.
Speaker Change: We're especially enthusiastic about the potential for pyrokind to become the first therapy approved for all Thalassinius subtypes.
Speaker Change: Our team continues to make progress across four areas of long preparation.
Speaker Change: First, we continue to conduct extensive market research and claim data-based analyses to further refine our market insights and inform our launch targeting and execution.
Speaker Change: Based on the work we've done, we estimate that prior times initial launch focus will address approximately 65% of the doubt-stallysimian patient population.
Speaker Change: We expect patients who already have regular contact with a healthcare system due to symptoms of their disease to be considered for therapy first.
Speaker Change: Dispaceschions segment includes transfusion dependentations, as well as non-transfusion dependentations who are already experiencing complications or are living with the billi-tating fatigue.
Speaker Change: Our team is actively engaged in the field, deepening our insights across the different patient segments and the diverse multicultural aspects of the lasimia.
Speaker Change: Second, our ongoing disease state education campaign has been focused on three areas.
Speaker Change: First, we are highlighting the pathophysiology of the disease, including ineffective or as a disease and hemolids.
Speaker Change: Second, we believe it's important for patients and physicians to understand the long-term complications and burden of disease across all the leukemia subtitles.
Speaker Change: Particularly, for patients with non-transfusion-dependent disease, who suffer from the misconception that they are at less risk.
Speaker Change: and Third, as we continue to engage with both patients and physicians, we highlight the importance of frequent disease monitoring and management. So treatment plans can be tailored to the individual patients need.
Speaker Change: To bring all of this together, we are pleased to share another example of the powerful, re-think-stellacemia campaign, which we have rolled out in the recent months.
Speaker Change: The striking image and headline invites closer infection of the words, depicting the hidden risks of the lasimia.
Speaker Change: The feedback from this campaign has been very positive from both patients and physicians.
Speaker Change: They highlight how relevant and valuable they find the disease education content, not only when it comes to understanding the disease pathophysiology and complications, but also to connect that to the importance of ongoing monitoring and management.
Speaker Change: Our third area of focus in launch preparation is the discipline expansion of our commercial and medical teams to right-side their organization for a successful launch in this larger but still rare market.
Speaker Change: We're steadily building the team and adding additional capabilities as we approach a potential launch with the keen focus on being a capital efficient as possible.
Speaker Change: and Ford, our market-passes team is already engaging with players on this state education.
Speaker Change: I'm proud of the broad access our team has achieved for a by-row kind in big a deficiency and we are aiming for the same strong outcome in Tala Simia.
Speaker Change: Outside the US, the Goff Corporation Council, or GCC Region, is home to approximately 70,000 patients with the LaSimea and some of the leading treatment centers in the region were part of our clinical trials.
Speaker Change: As we announced last quarter, we're pleased to have entered into a distribution agreement with New British from the Suity Coast, to prepare for a potential commercialization of pyrokind in the GCC region.
Speaker Change: Complementing this partnership, we are delighted to have secured the breakthrough medicine designation for Pio-Cantinthalisemia, granted by the Saudi FDA or SFDA.
Speaker Change: We're actively collaborating with new bridge to develop an optimal regulatory and commercial strategy for the GCC region.
Speaker Change: As a reminder, the access button in the video GCCC countries usually begins with a price bet at the regulatory level, followed by access negotiations with health authorities, local institutions and a private sector.
Speaker Change: We look forward to navigating each of these elements in collaboration with Newbridge.
Speaker Change: Next, let me provide a brief update on the current launch of pyrochines in PK Decision C. In the third quarter of 2024, we generated 9 million dollars in net pyrochines revenue compared to 8.6 million dollars in the second quarter of 2024.
Speaker Change: In the US, a total of 211 patients have completed a prescription enrollment form, including 10 in the third quarter of 2024. A 5% increase versus the prior quarter.
Speaker Change: This has translated into 127 net patients on therapy
Speaker Change: We believe the capabilities we continue to strengthen through the current launch.
Speaker Change: will provide a firm foundation for which to maximize potential future US launches of kind in Della, Kenya, in 2025 and in sickle cell disease in 2020-6.
Speaker Change: In closing, we are inspired and energized by the potential to bring a new therapy to this underserved patient populations around the world. With that, I will turn the call over to Cecilia.
Cecilia Jones: Thanks, Feta. I served for 2024 financial results. Can be found in the press release we issued this morning. And more detail will be included in our 10Q which will be filed later today. Let me now take a moment to provide some context and hide at a few key points.
Cecilia Jones: 3rd order 2024 Net Pirate and revenue was $9 million, an increase of 22% compared to the 3rd order of 2023.
Cecilia Jones: Consistent with other rare disease launches, Grosston and it has been an inspected to be in the 10-20% range on an annual basis.
Cecilia Jones: Going forward, aligned with our strategic shift towards preparing for the potential launch in Tala Simia. For Piquity Revenue, we expect to see new discurals and all of our quarter-variability in line with what we have seen in recent quarters.
Cecilia Jones: Cost of sales for the quarter over 0.8 million dollars.
Cecilia Jones: R&D expenses were $72.5 million for the third quarter, a decrease of $9.4 million compared to the third quarter of 2020.
Cecilia Jones: The secret was primarily driven by the 17.5 million dollar upfront payment associated with the licensed agreement with the Nileum, which was recorded in the prior year.
Cecilia Jones: It's DNA expenses were 38.5 million dollars for the third quarter, an increase of $12.7 million compared to the prior year quarter.
Cecilia Jones: This was primarily driven by an increase in commercial related activities as we prepare for the potential approval of PyroCind in Talesemia in 2025.
Cecilia Jones: Importantly, this quarter we received a total of $1.1 billion in milestone payments following the FDA approval of our side in the next.
Cecilia Jones: The statements include a $905 million payment from Royal Teefarma, in connection with the Royal Teef Project's Agreement as is announced in May 2024 As well as a $200 million payment from Serbia, in connection with Ajuse Vester of a top quality business in 2021.
Cecilia Jones: Both of which will record and their other income in the PMF.
Cecilia Jones: As a reminder, as it will be changed 3% royalty on annual US-Nets sales of both hydrogen and if greater than $1 billion.
Cecilia Jones: Including this milestone payments, we ended the border with cash, cash equivalent and marketable securities of approximately $1.7 billion.
Cecilia Jones: We expect the respondents to gather with anticipated product revenue and interest income We provide the financial independence to prepare for potentials pyrrkin launchers in Tala Simia and Circo Felicis
Cecilia Jones: Advances in this programme, an opportunistically expanded pipelines through both internally and externally discovered assets.
Cecilia Jones: We remain focused on creating shareholders' value, including by proactive limiting or cost-based and deploying a disciplined cash-alocation approach, as we prepare to support potential future launches of pirate-kind.
Speaker Change: As we move toward additional potential value creating milestones in the near term, I am confident that our balance sheet would continue to enable us to execute from a position of strength. I will now turn the call back over to Brian.
Brian Goff: Thanks Cecilia, before we open the line for questions, I want to conclude with a few words. 2024 has been marked by exceptional progress at Agios.
Brian Goff: We continue to deliver on our key priorities and have a very strong financial position, allowing us to maintain this great momentum in the final months of the year and beyond.
Brian Goff: In addition to its approved indication in PKD, Medipivat represents a significant opportunity to meaningfully elevate the standard of care for patients with valicemia and sickle cell disease.
Brian Goff: We consistently hear firsthand from these communities that there is an immediate critical need for new regimens to treat these diseases.
Brian Goff: With this, we believe, Middipivet is poised to become a first and best in class treatment option for multiple indications, translating into a franchise with multi-billion dollar potential.
Brian Goff: In addition to Minitivat, we continue to drive our other promising clinical programs forward, focusing on areas where there is a high patient need and providing us with additional opportunities to create significant long-term value and impact for all stakeholders.
Brian Goff: We look forward to the future as we strive to change the trajectory of these rare diseases.
Brian Goff: with that and like to open the call for questions. Operator, please open the line.
Speaker Change: Thank you, and at this time we will conduct a question and answer session as a reminder to ask a question you need to press star 11 on your telephone and wait for need to be announced. To withdraw your question please press star 11 again.
Speaker Change: Police and Brian, what we can power the Q&A roster. One moment for our first question.
Speaker Change: Our first question, conflant of Greg Harrison from Scrocher Bank, you line is open.
Speaker Change: Hey, good morning and thanks for taking the question and great to see all the progress.
Greg Harrison: To For Me, how are you thinking about capital allocation and appetite for business development now that?
Greg Harrison: or a side in it that's been largely monetized. Maybe there's a particular area, you guys find attractive or a complementary. And then on sickle cell, maybe you can speak to expectations for the phase three portion of the trial, and if you can comment on powering.
Greg Harrison: with your spectative VLCD.
Speaker Change: Sure and Greg, welcome back, great to hear you again, I'm glad to be here.
Speaker Change: Two parts will, Cecilia can handle the capital allocation question that Sarah can take over for rise up phase three expectations.
Cecilia Jones: Great, thanks, correct. So in terms of capital allocation, we're of course very proud of the strong balance sheet we have now and in terms of how we think about that, our priorities of course will be the potential launches for the Islander Bopoli Cycle Cells.
Cecilia Jones: and Poutine to Advice our pipeline, which you hear a little bit more about MDS today as well. And then expanding that pipeline both internally and externally will put it discipline and the amount of cash we have in the baggonship doesn't change that.
Cecilia Jones: We look at things, you know, we're the same criteria we have in the past in terms of being rare, transformative, have a period of regulatory bias and obviously create value for a shareholder. So that hasn't...
Cecilia Jones: Change, we continue to look at that and different areas we said we were not in terms of modality and we'll keep looking at it to help we want to expand that.
Speaker Change: and then I'll turn it over to Sarah but I do want to just lead in by saying how proud I am of Sarah and the team for this incredible consistent executional excellence who are really pleased with the fact that
Sarah Heuens: Even before today's call we were able to announce complete enrollment of the phase three study for rise up. And if you're keeping track, it's been about 12 months. And I think that says a lot about the profile, the trust the community has enough.
Sarah Heuens: and again very importantly the execution likes once from the team. So with that Sarah, how about that as a set of them? Thank you Brian. Thanks for a question.
Sarah Heuens: So, yes, so we are very proud of the rise of team, we indeed accomplished.
Sarah Heuens: and Complete Enrollment, which was one of our milestones for this year. So, I'm very excited about that and are now looking forward to YouTube.
Sarah Heuens: To finish the trial, to get it to the end.
Sarah Heuens: So the expectations for phase 3, so it's a one-year duration. We have two primary and once as you know, one is seem of global response. The other one is sickle-self pain crisis reduction.
Sarah Heuens: and that is of course with the goal to try to deliver a drug that can hit on the total of your sickle cell disease, meeting both in Momente, Kenya and Vesa Occlusion improvements. Both endpoints are powered to have a 9% or slightly more.
Sarah Heuens: to be able to detect that difference. So we're very excited about this and are looking forward to the end of the trial.
Speaker Change: Great, thanks so much. Sure, on moment for our next question.
Speaker Change: Our ex-question will confine out Danielle Brill from Raymond James, who line his open.
Speaker Change: Hey guys, this is Alex on for Danielle. Thanks for taking our question. Another one on Rise Up. Can you talk a little bit about your primary end points in Rise Up studies? Do you think you need to hit on both end points or will success on just one support or regulatory filing? For example, is it possible that hitting on hemoglobin response and another secondary clinical endpoint like fatigue?
Speaker Change: and the Coold support of regulatory filing. Thanks so much.
Speaker Change: Well, thanks for the question. So yes, we have two primary and points. One being himoglobin, I've just mentioned, and then we had one forks from the OC. We did.
Speaker Change: We did choose a two primary end points because they can provide relevant information for the totality of the disease. So in a situation in which we would not be able to hit on one and point the only way to down the other and point we still have an opportunity to transfer alpha.
Speaker Change: to secondary endpoint testing, which would allow us to further look for clinical benefits.
Speaker Change: One of those influences in D50, we always try to deliver with our drug across the different people in the caninas that we have studied, the hemoglobin plus story meeting that.
Speaker Change: We improve him with the technique in Nemia, but then also try to hit on the field and function and as you know, we've been able to deliver to that on TKD and Tala Scenia, highlighting an improvement in fatigue.
Speaker Change: are Stella Simia, they are also highlighted that there was an improvement in the six-minute walk-up test. And so our rise-up studies set up in a similar way to...
Speaker Change: to provide, you know, most always that we can speak to clinical benefit for sick of all these patients.
Speaker Change: In regards to what is needed for regulatory approval, there is the regulators who look at the clinical trial data at the end of the package, of course, when we submit, we always...
Speaker Change: We believe that there is a benefit to this profile that supports approval, but it's ultimately the regulator that due to review and decide on approval. I would like to add, and this is, again, echoing not just the point of execution, like excellence, but also the scientific expertise from the team that
Speaker Change: Everything that Sarah just went through was actually outlined in a poster in 2021.
Speaker Change: In other words, we didn't change the design from day one. It's been set up this way.
Speaker Change: and I think that's important particularly in the context of the recent evolution in sickle cell disease with other therapeutic options. And we're very proud of the fact that from day one, we had a design that was intended to be a so-called hemoglobin plus design.
Speaker Change: Thank you, one moment for our next question.
Speaker Change: Our next question of confiliant of Greg Renzel from RBC Capital Markets, Elon is open.
Greg Renzel: Greg, good morning, Brian and team congrats on the progress and thanks for taking my questions.
Greg Renzel: and the next episode of the
Greg Renzel: Brian, you've commented on just the trust that you've earned with him.
Greg Renzel: respect to the community.
Greg Renzel: on the execution and the value that you're providing in this development program for a permittive pivot with sickle cell.
Greg Renzel: Just curious to hear your comments on that general feedback, the potential for mistrust, from the community, just given the oxbrider withdrawal and the happenings that you alluded to there. What is odd you have doing to at least track that, detect that and sort of incorporate that into your go forward with the trial and also how you are touching patients.
Greg Renzel: and just secondarily, have you had any communications or plan to with regular tour just on the potential risks that have been sort of highlighted about running trials in the Africa site?
Speaker Change: Thanks for watching.
Speaker Change: and the
Speaker Change: Yeah, thanks a lot, Greg. It's an important question that you ask because...
Speaker Change: The Sickle-Seld disease community, I think.
Speaker Change: Not surprisingly to anybody has been through unlocked. It's always been limited treatment options that have been available.
Speaker Change: The community of large is certainly going through a journey of drugs available and then not available in certain regions and it's a real challenge. An example of what we do is Sarah and I and others from the team.
Speaker Change: Just attended the annual Sickle Cell Disease Association of America Meeting in Atlanta.
Speaker Change: and it was a chance for us to listen most importantly and hear what's on their mind, what their concerns are, how they think about treatment options in the future. And again, just to echo what I'm very proud of is...
Speaker Change: We have always looked to maintain high touch with the community and an example of that goes all the way back to when we designed the rise up trial.
Speaker Change: We very deliberately involved members of the Sequel cell warrior community.
Speaker Change: and the input that we got was not just in the trial design, inclusion, exclusion criteria, but very importantly, how to appropriately recruit for the trial. And now you've seen the results of that. It recruited beautifully.
Speaker Change: Hopefully when we get to the point of commercialization, we're leaning on that community to guide us in that regard as well. So I think the main message about trust is...
Speaker Change: We try to be very visible. We listen without bias. And we make sure that the community sees their fingerprint on what we do based on the input that they've provided.
Speaker Change: Yes, and then to add to that in regards to going forward with the trial, so we continue to, you know, it's a phase three trial. So obviously we do monitoring of data as we go along. We do all of our standards.
Speaker Change: procedures to make sure that we continue to execute well on this trial and we'll continue to watch carefully as this trial evolves. And in regards to potential risk for sickle cell disease, specifically with running clinical trials.
Speaker Change: It's a tough, devastating disease in which patients are suffering from a ton of comorbidities and it's a deadly disease.
Speaker Change: So there is always challenges when you run a trial like this.
Speaker Change: but it speaks to the unmet need in this space, but relations are wildly strong, challenging and difficult.
Speaker Change: There's such a met-need in this patient population that we really hope to be able to deliver a drug that can be meaningful for the patient community and treat the mortality of the disease.
Speaker Change: Just maybe one more point on this to drive it home is Sarah and I also had a call with
Speaker Change: with members of the sickle cell disease warrior community, actually part of the advocacy group that I just talked about. And on that call there were seven members.
Speaker Change: of the community and among that three of them were in crisis or recently had a crisis and we walked away with sobering reminder about
Speaker Change: Just how serious this disease is and how desperate the community is for new therapeutic options. And it should be plural for, we always cheer for progress, remember, everybody, because this is a disease that needs multiple options.
Speaker Change: Thanks so much, Karl.
Karl: You're welcome.
Speaker Change: One moment for our next question.
Speaker Change: Our next question of confline of Eric Schmidt from Counterfeit's Gerald, your line is open.
Eric Schmidt: Thanks for all the updates and for taking my question. Just sticking with this topic of the unfortunate withdrawal of VoxBright out about a month or so ago.
Eric Schmidt: What have you learned scientifically or what kind of thoughts do you have scientifically with regard to the mechanism of that drug and how it relates or doesn't relate to the P-bad and how it's sort of...
Eric Schmidt: and maybe influences your chances of success in the ongoing study. Thank you.
Speaker Change: So thanks for the question. Well, as you know, Brian, there has a very different mechanism of action and we feel, you know, it should start like we really feel sad about what happened.
Speaker Change: The Brian's earlier pointed to the suit.
Speaker Change: It's a sap circumstance, right? Because this patient community really has...
Speaker Change: Fairly any options, so we're sat to see this.
Speaker Change: It is a very different mechanism of action. The clinical trials were set up differently as well, so I think that is...
Speaker Change: We don't know what happens beyond what has been publicly declared.
Speaker Change: but then focusing on our trial, our drug works very different. We and have that multi-modal approach to treating
Speaker Change: potentially treating sick of cell disease.
Speaker Change: So we do reduce to 3DTG and increase ATT and we think both of these components.
Speaker Change: can interplay and be important to avoid sickling and to improve them all within their body and theemia of co-opulment. So, very different mechanism of action than of Sprite that, which can also, of course, translate into the clinical data that we have seen so far with a very, with a face to clinical trial data set that was very robust.
Speaker Change: and a placebo-controlled trial and that mechanism of action has translated into us.
Speaker Change: Demonstrating and Improvement on Himo Goff, and then also observing that positive transferring mouse and VOC reductions. So we are excited to continue to progress this, the drop for in this A3.
Speaker Change: Thank you, one moment for our next question.
Speaker Change: An ex-question of Coffin line of Chris Raymond from Piper's Handler, Elias Owen.
Speaker Change: Thanks for taking the question and congrats to you guys. You may be just another question.
Speaker Change: with regard to the expiry to situation. I know you talked about engaging in the sickle cell community, but this is a question that's come up with investors a lot and I'm not sure if there's a real kind of answer here.
Speaker Change: Do you have a sense as to how the regulatory perspective has changed on this, you know, there's some debate that perhaps is made it, you know, raise the bar.
Speaker Change: and then another debate that perhaps it's actually lowered the bar in terms of them. You know, another Sarah began the market. Just any sense there for how.
Speaker Change: and regulators might be reacting to this. And I think so thanks for that question because I think Brian actually touched upon it like with the both of that he presented.
Speaker Change: In 2021, which was a trial design or faith III, from the get-go, we had a very different...
Speaker Change: Clinical Development Approach plus a regulatory approach, then the pathway that OBSBRIGHTA follow.
Speaker Change: accelerated approval by demonstrating him a global improvement at the time of submission and then had to follow on studies to further demonstrate clinical benefits, our goal from when we first designed this trial is to deliver.
Speaker Change: A treatment at the end of the clinical trial can speak to clinical meaningful benefit, meaning hemoglobin plus and sickle-solupting crises reduction. So based on what happened, we have not changed our...
Speaker Change: A proge to our clinical design or our proge to the regulators. As I mentioned earlier, for any data package you submit, it's always a collaboration with the regulators after they review the first.
Speaker Change: The first package and then they'll ask us more questions and ultimately they get to decide on the ultimate benefit risk.
Speaker Change: Thank you.
Speaker Change: One moment for our next question.
Speaker Change: Our next question, Confident of Tesla Romero from Jake Morgan, you line is open.
Speaker Change: Good morning, Brian and Dean, thanks so much for taking our question. So switching gear a little bit to Faulusina.
Speaker Change: You outlined this morning several patient groups and your slidesack that are part of your initial launch focus for prior kind.
Speaker Change: Are you able to provide a little bit more specific?
Speaker Change: Quantitative Detail around how the epidemiology breaks out in terms of patient numbers and each of these three key target populations you mentioned. And how often are these patients generally seeing their healthcare provider across these three key groups? Thanks so much.
Speaker Change: Thanks, Tessa. I'm just going to start by saying that you're making sped a smile, because we love the questions on sickle cell disease. We're very excited about the potential.
Speaker Change: with a potential launch in area in 2026, but even closer.
Speaker Change: is Thalusemia, where we've consistently messaged that we're looking forward to this potential launch in 2025. So I'm going to let's try to take over our questions.
Speaker Change: Absolutely, thanks for the question. We are very excited with the potential of having an approval for Tala Simeon X-ER and actively preparing for launch.
Speaker Change: When we think about the La Semia, truly it's an indication, it's a beautiful area, this is indications when it comes to commercialization for three main reasons.
Speaker Change: The first thing is that we know that there are 6,000 adults diagnosed with telesymeans the U.S. and I'm going to underline diagnosed here because these patients are known and engaged to the healthcare system and we know that that's the case because they're ICD-10 codes available which allows us to effectively and efficiently deploy our resources when it comes to launch preparations.
Speaker Change: We talked a lot about sickle cell disease in the high-end metneed there, but that's also very, very, through Fort La Semia as well. The metneed is known and we continue to engage both with patients and physicians in our pretty long efforts to continue to educate and fund the burden of disease.
Speaker Change: When it comes to which patients will be considered for our initial launch of its crisis you mentioned, we believe that about 65% of the 6000 patients will be our, we will be our focus for initial launch.
Speaker Change: and these patients are three groups of patients, these are patients that are very actively engaged with the healthcare system.
Speaker Change: The first one is the transfusion dependent patients who are going frequently for their transfusions and have an engagement with their hematologists. That depends on their transfusion frequency, but more or less you can say that they are seeing an engagement with the healthcare system as probably on a monthly basis.
Speaker Change: The second group of patients are the non-transvision dependents who have already developed comorbilities, they require frequent monitoring and management.
Speaker Change: It depends on the specific patient population and the frequency there, but we know both from claims data and our initial accounts profiling that these patients are relatively regularly seen.
Speaker Change: and they will be considered for management and further monitoring as well. And the third part of the patient population which comprises the 65% are the non-transfusion dependent patients.
Speaker Change: who are experiencing the beauty dancing for the, it's impacting their daily lives and they'll be sticking for a potential treatment option as well.
Speaker Change: We haven't provided specific details on this patient population, but as the launch progresses, we will be providing additional information as well. And I'll just add too, I mean, Sveta with all of her rare disease launch experiences, the first to remind us that...
Speaker Change: Watch sometimes is a sort of odd term because it's not a point in time, it's a continuum. So even on this guidance with the sequencing, if we could say it that way, it's segments.
Speaker Change: Will continue instead of constantly working on keeping the team ahead of second one, segment two, etc. And we expect to be progressing for the long term in Falle Semia and then again hopefully in sickle cell disease as well.
Speaker Change: Okay, great. Thanks so much for taking me a question. You're welcome.
Speaker Change: Thank you, one more one for our next question.
Speaker Change: An ex-question of Coffeline, the Divier Rau from T.D. Kelland, you'll end us open.
Speaker Change: Good morning, team. This is Divia Onfer, Mark. Thanks for taking our question. Want to stay on Galaxy Mia? Would you be able to discuss how you're thinking about...
Speaker Change: Timing and Design of the Pediatric Trial, now that we have at least initial data or the top line data from.
Speaker Change: The Kid's trial in P.K.D. And then our second question is, the initial date of the PIVA in MDS.
Speaker Change: and obviously she's a little bit of signal, but it was relatively modest for the five-megdose. Could you tell us what you think the bar for success is for the face-to-be now that you're looking at, you know, the doubles that are higher than the initial dose tested? Thank you.
Speaker Change: Thanks, Lydia. So for Salosemia, the Pediatric Trial, yes, so indeed we are very, very proud of the first PKD Pediatric Trial, in which we were able to demonstrate.
Speaker Change: that some children experienced transfusion in dependence over very police defeat out, because that's not really going to be meaningful.
Speaker Change: For the timing and the design of the trial, so we are now, we also now have the benefit risk profile in the Taoistimia adult patient population, so you can expect a similar approach from us as what we have taken for PKZ. We have not gone into further detail on the timing of when we would initiate, so that more to come at a later time point.
Speaker Change: in regards to MDS. Yes, we did have, or we did, we are pre-specified.
Speaker Change: Criterium for the face to A in which we observe 40% of transusion independence in the face and population we have enrolled.
Speaker Change: and to your point we did learn there that the PK in the S-Stations was different than what we had observed in healthy volunteers. So that of course let me change it for our face to be. And as you see, we now have more forwards with the face to be testing higher those, three higher those, to be able to hopefully strengthen the observations that we have observed.
Speaker Change: and our focused on transfusion independence in that trial and then eight-week or more transfusion independence based on clinical advisory feedback that we receive.
Speaker Change: So, are excited to progress that trial and more to come.
Speaker Change: and we are very excited about the potential in MDS. I know folks pay attention to others who are in this market and we took note this morning of the continued progress.
Speaker Change: and we see with Rebels on.
Speaker Change: and it's a rapidly growing market. There is very high on that need. It's similar in a way to sickle cell disease and that multiple therapeutic options will be the answer. And we're very proud of the fact that we have a very differentiated mechanism. So this is an important step for us to be able to start the phase 2B.
Speaker Change: Thank you, one moment for our next question.
Speaker Change: A Rex question of a couple of lines of Alex Tranahan from Bank of America. He line is open.
Speaker Change: Hey guys, thanks for taking our questions. To, to from me.
Alex Tranahan: First on the Valecimia Launch Prep, given it is a continuum as Brian said, wonder if you have a sense as to how quickly pay or access.
Alex Tranahan: Come online and...
Alex Tranahan: Would you say pairs are fairly aware of mid-apid vats clinical profile and is unmet need or
Alex Tranahan: maybe some more work to do there.
Alex Tranahan: and then on the upcoming full readout for Energize P. Should we expect any additional efficacy data from the readout beyond what was shown in the top line? I guess we'll do direct people's attention.
Alex Tranahan: and the next update and it seems like Ash is probably the next most likely venue. Thank you.
Speaker Change: Thanks, Aliken. So, Svetka can start with the question on the payers and the tell see me and watching them, Sarah can take over with energy, ST. Absolutely. Thanks, Aliken. It's a very good and relevant question. So, from a therapist that he's probably as you've seen from the slides and what we can communicate to get to today, the market access is a corpivory in our lunch preparation.
Speaker Change: Our market-activity team is already engaging with pairs on disease-state education. When you look at the Lithiumia, you put a pair lens on it. It's a rare disease. There is a relatively lower awareness of the disease so we are definitely educating on the disease.
Speaker Change: but on the positive side of it is that it's a rare disease and we don't expect that to be high on the payer rate or for management as well.
Speaker Change: So the team is preparing for launch. I must say we've had an excellent outcome in our access efforts from P.K. deficiency. We have a very strong team and we continue to see the same success and progress moving forward with the Lassimian.
Speaker Change: Great, and then Sarah for Energize T. Yes, for Energize T. So we, in our press release, you know that we've declared our primary endpoint plus one of the secondary endpoint. So you can...
Speaker Change: Thanks for most difficult to how we do all of our phase 3 readouts and intent to treat analysis with the details around key secondary endpoints as well in the safety overview. Indeed, we're hoping to be able to present on upcoming medical meetings. Ash is currently not...
Speaker Change: Navajo Olnick, so you know abstracts will be released on November 5th, so we are eagerly waiting for that as well.
Speaker Change: Thank you, one moment for an expression.
Speaker Change: or Exquash from Confeline or Salvey and Richter from Goldman Sachs, you'll end this open.
Speaker Change: Hi, this is Lydia on Fris Alvien. Thanks so much for kicking our question and congrats on all the progress. I'm just two quick wins from us.
Speaker Change: So on sickle cell, now that enrollment is complete, are able to provide any details on the number of baseline VOCs and then just on Tabipovat. Can you discuss when you plan to share data from the phase 2B and to remind us what we'll be included with this update? Thank you so much.
Speaker Change: So for sickle cell disease, we do not present baseline characteristics. We typically tend to present everything when we release the sub-blind results.
Speaker Change: Nothing to come before we provided the full data readout and then in regards to Deb Affirat and the MDSS2B, MHS.
Speaker Change: and the other one.
Speaker Change: Thank you. One moment for our next question.
Speaker Change: Our next question of offline of Andrew Barrens from Leerwick Partners, Galena Zopin.
Speaker Change: I think, two for me, on 946, I think he does some patience in the phase 2-h-y-o-l-high, than the 5-millivans.
Speaker Change: If that's correct, we see those data presented somewhere and can you remind me of how much pleasure and you think you have on Dersing before you might hit a DLT and then I think this was touched on a bit but we've heard that there was a problem of steps to the similarity in the African trial sites.
Speaker Change: and some investors think that this was related to problems and conduct of their sites. I think your trial is at some of the sites and you know, can give us some color on what's being done to ensure that a similar problem doesn't occur. Thanks.
Speaker Change: Thanks, Andy. I'm going to let Sarah go, but I did want to first say you asked in terms of 946 and we're practicing on our end to say, Peppa Peppa, we're very proud of the new INN that we have for our second more potent PK activator. And Sarah, you want to start with the two way.
Sarah Heuens: So the face to A had the state to the five milligrams of those, and so we did not test higher those in that to A. So right now the higher doses that are being tested is in the two.
Sarah Heuens: and the former former president of the trial, which is then 15 and 20 milligrams.
Sarah Heuens: in regards to when I expect to hit the DLT, I don't know if but I think we haven't gathered the data yet, so the data will occur over time and then we'll see if we observe anything that makes us not want to do.
Sarah Heuens: and further explore a certain dose. So, more to come on that, in regards to what you ask for, me tapi-vap and sepsis and Milanova, and Milanova observed in the Oksbrite at Trials.
Sarah Heuens: So...
Sarah Heuens: Malaria isn't the making certain regions of the world. We have part of our trials and our needs conducted in Africa as well. But we have a very good geographical distribution across the board within Rise Up.
Sarah Heuens: We are excited about that. The observations that have happened on OXBRIZA are not observations that have happened on hydroxyurephor instances that have been observed in Africa. So I think our data will accrue over time and will will observe when we have the final trial.
Speaker Change: Okay, I'll take care of you.
Speaker Change: Thank you.
Speaker Change: Thank you and this includes RQ and A, and I'll turn it over to Brian for any final remarks.
Brian Goff: Alright, thanks a lot Victor and thank you very much everyone for participating in today's call. It is clear this is a very exciting time in Agios. We truly believe that we're poised to deliver transformative new therapies for patients.
Brian Goff: and to create significant long-term value for shareholders. So, thanks again, and we look forward to speaking with you again soon.
Speaker Change: Thank you for your participation in today's conference. This does conclude the program you may now disconnect everyone have a great day.
Speaker Change: The The