Q1 2025 Agios Pharmaceuticals Inc Earnings Call
[music].
Good morning, and welcome to <unk> first quarter 2025 conference call. At this time all participants are in a listen only mode there'll be a question answer session. At the end. Please be advised that this call is being recorded at <unk> request I would like to turn the call over to Christina Vice President of Investor.
Our relations and corporate communications for <unk>.
Christina: Thank you operator, good morning, everyone and welcome to <unk> Conference call and webcast to discuss our first quarter 2025 financial results and recent business highlights you can access the slides for today's call by going to the investors section of our website <unk> Dot com.
Brian Goff: On today's call, we'll hear from our Chief Executive Officer, Brian Goff, Dr. Sarah Hewins, Chief Medical Officer, and head of research and development, So that a melanoma chief commercial officer, and Cecilia Jones, Chief Financial Officer.
Brian Goff: 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 events and results 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 or.
Brian Goff: Our most recent filings with the SEC and any other future filings that we may make with the SEC.
Brian Goff: And with that I'm pleased to turn the call over to Brian.
Brian Goff: Thanks, Chris Good morning, everyone and thank you for joining us our mission at <unk> is to develop and deliver transformative medicines to elevate and extend the lives of patients living with rare diseases.
Brian Goff: Today, we are fortunate to have multiple late stage programs nearing critical readouts for potential commercialization and have a very strong balance sheet.
Brian Goff: We are maintaining our focus on executing against the objectives, we've laid out including the ongoing regulatory reviews of our thalassemia program for which we have seen continued and consistent FDA engagement.
Brian Goff: And with approximately $1 $4 billion of cash on hand, and a disciplined approach to capital allocation. We believe we will have financial independence to fund the company through new approvals and product launches.
Brian Goff: Advancing our pipeline all of which is especially notable in today's market environment.
Brian Goff: Our lead product <unk> or mid of Tibet pyruvate kinase 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 have the exciting prospect of two additional commercial launches to support our potential multibillion dollar opportunity for private guy.
Brian Goff: We are planning for a potential approval and launch in thalassemia in the U S. In September of this year, followed by sickle cell disease in 2026.
Brian Goff: Beyond fiber kind of early and mid stage pipeline is robust and poised for clinical advancement offering a strong foundation for innovation and long term growth.
Brian Goff: And finally supporting it all is our highly experienced team and a strong balance sheet I noted earlier.
Brian Goff: We look at 2025 as a breakout year for high Geos as we focus on three key priorities first.
Brian Goff: Maximizing the potential of the pirate kind franchise, including the pursuit of new indications.
Brian Goff: Second advancing and diversifying our key pipeline programs and third strategically focusing our capital deployment to sustain and drive our growth.
Brian Goff: We have begun the year with a strong start executing toward important milestones earlier. This year, we announced positive topline results from the activate kids phase III trial of <unk> in pediatric.
Brian Goff: Patients with PK deficiency, who are not regularly transfused.
Brian Goff: We also anticipate some exciting developments for our mid and early stage pipeline programs.
Brian Goff: For <unk>, our novel PK activator, we expect to complete enrollment in the ongoing phase <unk> study in lower risk Mds by year end.
Brian Goff: And to initiate enrollment in a phase II study in sickle cell disease by mid 2025.
Brian Goff: Additionally, we expect to file an investigational new drug application for AG 203 six.
Brian Goff: Sigh RNA targeting Tempur six inhibition intended for the treatment of polycythemia Vera in mid 2025.
Brian Goff: And the most significant expected events for 2025 include the September 7th to do with the goal date for.
Brian Goff: Our SMB E filing of pirate kind in thalassemia now only four months away.
Brian Goff: The phase III readout of the rise up study of <unk> in sickle cell disease by year end.
Brian Goff: As you can see this year promises to be exciting with multiple catalysts across our pipeline that hold significant value for shareholders and have transformative potential for patients.
Speaker Change: Before I turn it over to Sarah I'd like to formally welcome Chris <unk>, who joined US in March as Chief Corporate development and strategy Officer, having previously served as the President and Chief operating officer of <unk> Biopharma and in various senior roles at both Bristol Myers Squibb and Celgene.
Sarah: He has diverse experience and strategic vision will be instrumental in maximizing the potential of our current assets. While also exploring potential expansion opportunities with that let me now turn it over to Sarah.
Sarah: Thanks, Brian.
Sarah: Pipeline includes a well rounded mix of late stage programs nearing market entry and promising mid and early stage opportunities.
Sarah: <unk> is a rare lifelong inherited blood disorder that causes chronic anemia in patients with does senior often experience a range of debilitating competitions, such as organ damage stroke and other serious health issue.
Sarah: One of the most commonly cited patient concerns is chronic fatigue, which is unaddressed by the currently available therapies.
Sarah: Goldman management strategies for thalassemia, such as blood transfusions, and Iron Chelation therapy can also lead to significant secondary effects compounding the health challenges patient states.
Sarah: Today patients have limited or no effective treatment options with 67% of diagnosed patients in the U S. Having no approved therapies.
Sarah: In 2024, we announced positive results from the Energizer and our J D phase III trials evaluating <unk> versus placebo in adults with non transfusion dependent and transfusion dependence all thought Rebecca policy and yet respectively.
Sarah: Based on the compelling clinical profile observed in both the Energizer and our J D. Phase III studies, we believe we suffered loss has the potential to become a foundational and convenient oral medication for thalassemia patients regardless of their genotype or transfusion needs.
In December we announced the simultaneous filing for regulatory approval of pilot.
Sarah: Indication in the U S European Union Kingdom of Saudi Arabia, and the United Arab Emirates.
Sarah: As Brian mentioned in the U S. We are not just four months away from our produce our goal date of September 7th and our interactions with the FDA have continued as expected.
Sarah: The FDA has communicated that at this time No Advisory Committee meeting is planned in the review is ongoing.
Sarah: Moving onto sickle cell disease. This inherited lifestyle blood disorder is estimated to affect approximately 120000 to 135000 individuals across the U S. <unk> five with a global prevalence exceeding $3 million.
Sarah: Clinical features of sickle cell disease, or chronic hemolytic anemia, and visa occlusion, which can lead to poor quality of life organ damage and early mortality.
Sarah: As an urgent need for novel therapeutic options and our data to date indicate that <unk> may be a transformational therapy for these patients with the ability to address multiple aspects of the disease.
Sarah: The phase III study completed enrollment in October 2024, with over 200 patients enrolled globally, achieving this milestone just over a year after recruitment again.
Sarah: In this study we have two independent primary endpoints hemoglobin response, an analyzed rate of sickle cell pain crises Danny.
Sarah: Danny either primary endpoint allows us to apply I'll hop to the testing of the trough key secondary endpoints with our secondary endpoints. We are using a variety of measures to assist with talking about the potential in improving how patients feel and function.
Sarah: We expect to report top line results from this phase III study in late 2025 with a potential regulatory filing in U S approval in 2026.
Sarah: We believe we talked about has the potential to emerge as a best in class therapy aimed at addressing the high unmet need in this disease by improving anemia, reducing sickle cell pain crisis, and making patients feel better.
Sarah: Beyond this we aim to further address the broad range of disease manifestations in diverse areas of unmet need in sickle cell disease. So it's not every patient will respond to a given therapy, we see an opportunity to expand the patient population with complementary approaches.
Sarah: So with that in the <unk>, which is a potent <unk> activator currently being explored as a potential treatment option for low risk Mds may also holds promise in sickle cell disease.
Sarah: We are planning to begin enrollment in our phase two study of <unk> in sickle cell disease in the coming months. This will be a randomized placebo controlled dose finding study, including a total of 66 patients.
Sarah: Patients will be randomized to either 255 or seven five milligrams of <unk> or placebo.
Sarah: The primary endpoint will be a hemoglobin response as defined by an increase of at least one gram per deciliter from week 12, compared to baseline and secondary endpoints will include the hemolysis and patient reported outcomes to evaluating how patients feel and function. We expect data from this study will provide proof of concept for <unk> and <unk>.
Sarah: US to select the dose for phase III.
Sarah: As I mentioned <unk> is also being evaluated in a phase II B study in lower risk Mds, where we aim to deliver the first oral therapy that addresses anemia due to ineffective erythropoiesis in the disease.
Sarah: <unk> affects approximately 75% to 80000 patients in the U S and EU five with lower risk Mds accounting for approximately 70% of all Mds cases.
Sarah: We are on track to complete enrollment in this study later this year with data Readouts planned for early next year.
Sarah: And finally, two items to note in our early stage pipeline programs for <unk> targeting spinel kitting area. We are progressing to studying multiple ascending doses in our ongoing healthy volunteer study mid year. We are also on track to file an IND in mid 2025 for 80 236, RSR R&D targeting.
Speaker Change: Six intended for the treatment of Polycythemia Vera So we're quite excited about the progress plans across the entire portfolio. This year with that I will now turn the call over to setup.
Sarah: Sarah.
Sarah: Our commercial organization is driven by the potential to expand <unk> indications to include bulk lithemia and sickle cell disease by 2026.
Sarah: Lithemia, we're aiming to deliver the first therapy indicated to treat all subtypes of the disease and with <unk>. Our goal is to deliver a novel oral therapy that improve anemia reduces vasal occlusive crises or voc's and improves fatigue.
Sarah: <unk>.
Sarah: Across these indications we believe <unk> represents a multibillion dollar opportunity.
Sarah: Looking at the upcoming potential launch of the lithium in the U S. The underlying market dynamics into leukemia support a significant opportunity for <unk>.
Sarah: The lithemia patients are diagnosed and non to the healthcare system.
Sarah: Gordon of disease is well characterized and there are well established kols and patient advocacy groups.
Sarah: All of these elements will help drive adoption.
Sarah: We are now just four months away from a potential U S approval and our team is working diligently to prepare for a potential launch.
Sarah: First we are executing a robust disease state education campaign focused on both patients and healthcare providers.
Sarah: Our campaign highlights the Lithemia disease, pathophysiology long term complications and burden.
Sarah: The importance of frequent monitoring and management.
Additionally, it embraces the cultural diversity of that the Lithemia patient community.
Sarah: Im proud to report that our team has organized several highly attended patient program in Cantonese Mandarin and Arabic.
Sarah: Feedback from the community has been overwhelmingly positive and we are planning additional programs as we prepare for launch.
Sarah: Second we have right sized our cross functional team to ensure a successful launch in this larger yet still router market.
Sarah: For example for PK deficiency, we have had a sales team of 20 professional and for the leukemia, we have strategically growing the sales organization to approximately twice that size.
Sarah: This team is fully on board focusing on disease State education, and detailed account profiling to enable a focused and effective launch shortly after approval.
And third.
Sarah: Our market access team is actively engaging and educating payers on the lithemia through pre approval information exchange meetings.
Dan: This is Dan.
Speaker Change: Adding and support patient access.
Speaker Change: Feedback from Bayer research and these interactions has been positive with recognition of the unmet need and the strength of the product profile.
Speaker Change: We expect the majority of patients to be on commercial plans.
Speaker Change: A reminder, the initial coverage a final kind of in the Lithemia will be through medical exception process, while policies are still being established.
Speaker Change: Given our experience and strong track record in PK deficiency.
Speaker Change: We're well positioned to navigate the medical exception process and replicate the success, we have had with begin deficiency.
Speaker Change: There are approximately 6000 adults diagnosed with the lithium in the U S.
Speaker Change: Most patients diagnosed before adulthood.
Speaker Change: With the availability of claims data, we can identify where these patients are managed within the health care system offering valuable clarity for our launch preparations.
Speaker Change: We think that population we estimate that final guidance initial launch focus will address approximately 65% of the doubtful lithemia patient population.
Speaker Change: We expect patients with more frequent contact with the health care system due to their disease symptoms to be considered for therapy first.
Speaker Change: These patients include those who are transfusion dependent.
Speaker Change: As well as those were non transfusion dependent but already are experiencing complication or debilitating fatigue.
Speaker Change: We conducted market research to identify top clinical characteristics healthcare providers will consider when prescribing viral kind.
Speaker Change: Four key attributes were identified as most important.
Speaker Change: The impact on hemoglobin levels reduction in transfusion burden improvement of fatigue and iron overload.
Speaker Change: Taking into account these element final guidance profile is well position for each of these important criteria.
Speaker Change: Central to our messaging is the transformative profile of pyracantha into leukemia characterized by a number of for it.
Speaker Change: This is potentially the first therapy for alpha and beta thalassemia patients.
Speaker Change: The first oral therapy for the disease.
Speaker Change: The first treatment to demonstrate quality of life improvements for non transfusion dependent patients.
Speaker Change: And the first treatment to demonstrate 36 week durability of effect in reducing transfusion burden.
Speaker Change: This is what motivates us to deliver <unk> as quickly as possible so people suffering from thalassemia.
Speaker Change: Finally, let me provide a brief update on the revenue for the first quarter.
Speaker Change: In the first quarter of 'twenty Blankly, five we generated $8 7 million in net <unk> revenue compared to $8 $2 million in the first quarter of last year.
Speaker Change: In the U S. A total of 234 patients have completed a prescription enrollment forum, including 11 in the first quarter of FY 'twenty five.
Speaker Change: A 5% increase versus the prior quarter.
Speaker Change: This has translated into 136 net patients on therapy also an increase of 5% versus the prior quarter and we continue to see strong persistence.
Speaker Change: We believe the capabilities, we continued to strengthen towards the current launch will provide a foundation, helping us to maximize potential U S launches in the Lithemia in 2025.
Speaker Change: In sickle cell disease in 2026.
Cecilia: 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: Thanks, Brett.
Speaker Change: First quarter 2025 financial results can be found in the press release, we issued this morning and more detail will be included in our 10-Q, which will be filed later today.
Speaker Change: Let me now take a moment to provide some context and highlight a few key points first.
Speaker Change: First quarter 2025, net <unk> revenue was $8 7 million.
Speaker Change: An increase of 6% compared to $8 $2 million in the first quarter of 2024.
Speaker Change: Compared to the fourth quarter of 2024 revenues decreased by 19% primarily due to the benefit of year end stocking and adjustments to certain revenue reserves that we previously noted for Q4.
Speaker Change: Importantly, a further detail we saw an increase in both new prescriptions and new patient starts in the first quarter since the January label update, which we see as a strong testament to the product's profile.
Speaker Change: Gross to net has generally been and is expected to be in the 10% to 20% range on an annual basis consistent with other rare disease launches and will also experience quarter to quarter variability.
Speaker Change: As a reminder, with our focus on palace senior disease State education as we prepare for a September seven <unk> eight we continue to expect 2025 revenues for PK deficiency to be relatively flat compared to 2024.
Speaker Change: Regarding further asemia it is worth noting that it can take several weeks, particularly at launch between a prescription enrollment for patients initiating therapy.
Speaker Change: Combined with the expected 10% to setup payer access we're looking at a more of a partial quarter in Q4, which should be factored into modeling revenue expectations for 2025.
Speaker Change: Mostly we are eager for the September 7th could do for data to arrive and the team is well prepared for it.
Speaker Change: Looking forward to 2026 and beyond we are optimistic about the team's ability to translate the favorable market dynamics. That's further described earlier into significant revenue trajectory for thalassemia.
Speaker Change: Returning to the first quarter results cost of sales for the quarter was $1 $1 million.
Speaker Change: R&D expenses were $72 $7 million for the first quarter, an increase of $4 $1 million compared to the first quarter of 2024.
Speaker Change: This was primarily attributed to an increase in workforce related expenses and costs associated with our clinical trials of Teva pivot in lower risk Mds in sickle cell disease, partially offset by lower costs associated with the clinical trials of Mr. <unk> in pediatric PK PD.
SG&A expenses were $41 $5 million for the first quarter, an increase of $10 5 million compared to the prior year quarter.
Speaker Change: This was primarily driven by an increase in commercial related activities, including head count as we prepare for the potential approval of <unk> later this year.
Speaker Change: We are closely monitoring the potential for new targets to increase our operating expenses, but at this time, we do not anticipate a material impact. Please see our 10-Q filing later today for additional related disclosures.
Speaker Change: We ended the first quarter with cash cash equivalents in marketable securities of approximately $1 $4 billion.
Speaker Change: As Brian mentioned, we expect this balance together with anticipated product revenue and interest income will provide the financial independence for potential Paragon launches in Palestinian sickle cell disease, advancing existing programs and opportunistically expanding our pipeline through both internally and externally discovered.
Speaker Change: Sets.
Speaker Change: In closing, we remain focused on creating shareholder value, including by proactively managing our cost base and deploying a disciplined cash location approach as we prepare to support potential future launches of <unk>.
Speaker Change: As we move towards additional potential value, creating milestones. This year, we are confident that our balance sheet will continue to enable us to execute from a position of strength.
Brian Goff: I will now turn the call back over to Brian.
Brian Goff: Thanks, Cecilia we believe the remainder of 2025 will be incredibly exciting for <unk> based on the potential approval and launch of <unk> in thalassemia.
Brian Goff: A critical phase III readout in sickle cell disease, and important anticipated progress across our mid and early stage pipeline.
Brian Goff: In closing I'd like to briefly reinforce our fortunate position of having a very strong balance sheet, which provides us with the ability to independently execute across our key priorities.
Brian Goff: We remain committed to disciplined cash allocation and long term shareholder value creation as we all navigate the current market environment.
Brian Goff: With that I'd like to now open the call for questions. Operator, Please open the line.
Brian Goff: Thank you and as a reminder to ask a question you will need to press star one one on your telephone and wait for your name to be announced to withdraw. Your question. Please press star one again please.
Brian Goff: Please limit yourself to one question one follow up please standby with the Q&A roster.
Brian Goff: One moment for our first question.
Speaker Change: Our first question will come from the line of Gregory <unk> from RBC capital markets. Your line is open.
Gregory: Great Hey, Brian and team congrats.
Speaker Change: Congrats on the progress thanks for taking my questions.
Speaker Change: Brian It's really helpful to hear your confirmation about no advisory committee for the September.
Speaker Change: So I'm just curious if you can comment on that first has that mid cycle review.
Speaker Change: Happened I think you should have already occurred just wanted to get clarity on that and secondly, what are the next steps that you've mentioned the engagement with FDA has been has been strong as you think about.
Speaker Change: Labeling and the next steps of the late cycle. Thanks, So much yes.
Speaker Change: Yes sure.
Speaker Change: As you noted we're really pleased with how we're progressing its been very consistent.
Speaker Change: With the FDA and the fact that we were able to say.
Speaker Change: At this time, it's been communicated no AD com, but of course, it's an ongoing regulatory review sorry, do you want to add.
Speaker Change: Color from your perspective, no. It's exactly what you just said I think we are.
Speaker Change: Pleased with the process.
Speaker Change: We are engaged with the agencies because we filed in multiple regions and everything is progressing we're very much looking forward to our producer date of September seven.
Speaker Change: I will just take the opportunity Greg to reinforce the fact that I think you heard the excitement instead as voice, where we're now four months away from that to do and I must say the commercial team is very prepared and we're very much looking forward to getting to that date, and then hopefully having the opportunity to serve the patients who count on us.
Speaker Change: That's really helpful. And then maybe a quick follow up just just broadly with the sickle cell community and as you think about.
Speaker Change: Especially type of pivot and the enrollment there how has the spread.
Speaker Change: Wean off the current market conditions, how is that in your view impacted the recruitment of trials.
Speaker Change: The sickle cell community as they sort of transitioning in a wait for for new options in the marketplace.
Speaker Change: Congrats.
Speaker Change: Sure you bet, Sarah can start and then set it might want to add a little color to one sickle cell in general and the opportunity in front of us.
Speaker Change: And so.
Speaker Change: From our perspective of course, we were disappointed when.
Speaker Change: When Fox was withdrawn because we really are looking for.
Speaker Change: All drugs that can potentially provide benefits for patients and hope that sickle cell disease patients ultimately will have many options to choose from.
Speaker Change: From our perspective on the clinical trial conduct we have not observed any any changes as it relates to our programs.
Speaker Change: Yes.
Speaker Change: Just to start off of course from us commercially sickle cell disease is a large market. There are over 100000 patients in the U S. The withdrawal lovely.
Speaker Change: Brian devastating for the community and that just strengthened the unmet medical need.
Speaker Change: We are excited about the potential opportunity to have two products on the market to serve the community and for us to continue to grow the number of patients who can benefit from from innovative therapy.
Speaker Change: When it comes to kind of the sentiment of course, we're going to take that into account as part of our launch preparations and the engagement with the community they deserve the trust and respect.
Speaker Change: We will continue to do so.
Speaker Change: Okay.
Speaker Change: Thank you one moment our next question.
Speaker Change: Our next question will come from the line of Alec Stranahan from Bank of America. Your line is open.
Alec Stranahan: Okay, great yeah. Thanks for the questions guys and congrats on the progress.
Speaker Change: Quarter.
Speaker Change: Maybe first one for you alluded to it a couple of times.
Speaker Change: Maybe you could just remind us about your plans for launching that I'd say that ex U S.
Speaker Change: When could these approvals come through for <unk> and <unk>.
Speaker Change: Or you think it would make sense to try and keep the economics in house by leaving the launches here yourself given the strong balance sheet and then I've got a follow up.
Alec Stranahan: Yes, Thanks, Alex.
Alec Stranahan: I'm going to let Scott take over on that question I will just start by reinforcing our by far the two most important geographies for thalassemia is first the U S and secondly, the Gulf region, GCC and we are very well prepared with regard to both of those yes absolutely.
Alec Stranahan: When it comes to ex U S. As Sarah noted, we have submitted to regulatory authorities UAE, Saudi Arabia in Europe from a commercial launch preparation and Embraer IP, Saudi is the salary or the GCC region is that is the next priority for us when we look at that.
Alec Stranahan: Ex U S opportunity.
Alec Stranahan: When you think about launch timing and uptake I would say that especially the Gulf countries. There look more similar to the European market. So it takes some time from approval to actually get on formulary and get access to patients and see the uptake.
Speaker Change: We have a very strong partner in the golf with Newbridge, which we believe that there'll be very well positioned to combine our strong expertise and knowledge into lithemia with their strong expertise and knowledge.
Speaker Change: In the region to execute on our behalf successfully and on economics may be Cecilia can touch on that yeah. I think on the economics as we announced last year. We have the partnership with <unk>. This is a revenue split which allows us to leverage the best of both wired setup that new great knowledge of the region.
Speaker Change: Our team support knowledge of the product and efficient way for capital deployment for Us for Europe, We plan to do something very similar in terms of the structure and will provide an update on what we do so.
Speaker Change: Okay, Great and then maybe one.
Speaker Change: Great to see Christian on joining the team.
Speaker Change: Curious if it's bringing him on board until the corporate strategy represents any shifts in terms of the way youre thinking or allocating resources going forward, either through increased BB or better yet.
Speaker Change: Areas of pipeline focus thank you.
Speaker Change: Yes, Thanks, I am really happy to have Kristian onboard he's a very experienced leader across as I noted in my earlier comments multiple companies senior roles.
Speaker Change: Very well conducted individual and.
Speaker Change: We're just delighted to have him on the team.
Speaker Change: Answer your question, it's not a shift.
Speaker Change: Building capabilities and a reinforcement of our real focus for corporate strategy capital allocation specifically.
Speaker Change: In order of priority first it's getting these launches right. We're really excited as we've noted about <unk> now with the producer just four months away.
Speaker Change: We are.
Speaker Change: Equally excited about the rise update that will play out at the end of this year and that could present a back to back once scenario.
Speaker Change: With sickle cell launch towards the end of 2026.
Speaker Change: We also are not a one product company, we're really proud to talk about the middle in earlier part of our pipeline today and so that's a key priority and third as BD any healthy Biopharma biotech company should always be looking at.
Speaker Change: Got to continue to build out the pipeline.
Speaker Change: And we have a very strong balance sheet as I noted, we will be extremely disciplined because we have internal opportunities that sets a very high bar, but Chris not really adds to that capability and again, we're just delighted to have him on the team.
Speaker Change: Excellent thanks for the color.
Speaker Change: Youre very welcome.
Speaker Change: Thank you one moment our next question.
Speaker Change: Our next question comes from the line of David.
Speaker Change: ROE from TV Cowen Your line is open.
Speaker Change: Yes.
Speaker Change: Hi, Brian This is David on for Mark I'll add my congrats on all the progress.
Speaker Change: Just one question from us.
Speaker Change: Have you seen any changes to your communication frequency.
Speaker Change: With the FDA given the recent reshuffling thats been happening at the agency and then is there a deadline for when the update is inform you of a potential outcome.
Speaker Change: Yeah.
Speaker Change: Thanks, Michelle for further questions. So no our communication with the agency on our programs have been the same as before so I think it is.
Speaker Change: Hum.
Speaker Change: Normal back and forth in the context of especially for the filing it's the normal back and forth commuter.
Speaker Change: Communication with questions and answers. So as you know that the <unk> date is September 7th and <unk> as the review is ongoing and agency always has done.
Speaker Change: Opportunity to request for an advisory committee to date, though they have informed US that there is no advisory committee planned and that the review is still on call.
Speaker Change: And <unk> I'll, just take the opportunity to give credit to the FDA, because we know that there've been a lot of dynamics, but I think this also reinforces that this is a high unmet need area, where we have two stellar studies that read out last year and the data.
Speaker Change: We've put into our file really aligns beautifully with what we hear consistently is the unmet need that we're trying to fulfill so.
Speaker Change: We're appreciative that continued focus and really pleased with how we're progressing.
Speaker Change: Thank you one moment our next question.
Speaker Change: Yeah.
Hero Naomi: Our next question comes from the line of Hero Naomi from Cantor. Your line is open.
Speaker Change: Hi, This is here on behalf of Eric Schmidt Youre at Cantor <unk>. Thanks, So much for taking my question.
Speaker Change: Ask a bit about the rationale and conviction for starting the phase two study in sickle cell in mid 2025 prior to the phase III readout of <unk>.
Speaker Change: 25.
Speaker Change: Yes, I think again, that's going to be a two parter, Sarah should start with Teva pivot itself and why.
Speaker Change: While we're excited about it and instead I can.
Speaker Change: Add more about what we're trying to achieve in sickle cell for the patient community in general, which clearly needs.
Speaker Change: More not fewer options ahead.
Speaker Change: So you are all.
Speaker Change: Like we were very pleased with the phase one data that we have generated in sort of a pickup in sickle cell disease.
Speaker Change: And when you look at drug development that that program, specifically that allows us to move forward to phase two which we're very excited about starting midyear and then of course within drug development. This is we're set up the organization in the R&D organization. We work very closely together, because we will always design our trials two words.
Speaker Change: The profiles that we believe can make meaningful change and with that I'll hand, it over to Sam absolutely still the way to think about it is really interest steps.
Sam: First of all I mean, it's 100000 patient population with a very large unmet medical need and when you think about this market.
Speaker Change: Easily absorb any sort of needs of multiple therapies.
Speaker Change: The second one is the fact that we hear it loud and clear from Kols and physicians and experts in the field not every patient will respond to every single therapy. So they would like to have many options. So they can choose from and find the best options for their therapy and when it comes to timing in the car.
Speaker Change: Positioning of the two products.
Speaker Change: We are actually looking to get all of the patient population with our portfolio of products, we'll actually more specifically srs at position tab up even after we have done the Mississippi. The dates of the rise up data. So starting now will allow us to actually have the dataset and make the best informed decisions for us how to move forward with phase.
Speaker Change: Three.
Speaker Change: And I think folks listening in may noticed that his background too but.
Speaker Change: Prior life. So that it has a lot of experience building lasting franchises that have a very similar dynamic of serving that.
Speaker Change: Even greater patient population. So we're we're looking to leverage your expertise in that regard.
Speaker Change: Thank you so much.
Speaker Change: One moment for our next question.
Speaker Change: Our next question comes from the line of Emily Bodnar from H C. Wainwright. Your line is open.
Emily Bodnar: Hi, Good morning, Thanks for taking my question I guess for both EMEA, maybe if you could touch a bit on your plans for marketing for non transfusion dependent patients compared to transfusion dependence dependent patients.
Speaker Change: Clearly on the non transfusion side.
Emily Bodnar: <unk> is currently don't have any treatment.
Speaker Change: Yes, perfect setup.
Speaker Change: So first of all our outside with how excited we are about the potential launch into Lithemia, which is just four months away. We have deployed to the team and I can say that we are ready for launch.
Speaker Change: That's really really exciting and energizing.
Speaker Change: When if and when it comes to the market itself I just want to mention very quickly it's a really.
Speaker Change: Attractive rare disease market patients are diagnosed and known to the healthcare system. There is a good understanding and characterization of the unmet need of growth, both transfusion dependent and non transfusion dependent patients our disease State education actually primarily focuses on the non transfusion dependent patients with <unk>.
Speaker Change: Actually yes.
Speaker Change: It relates to your question and of course, they are well established kols and patient advocacy groups that will help us drive adoption. When it comes in when it comes in terms of prioritization and different approaches for both patient populations. We believe <unk> has a strong value proposition across both transfusion dependent and non transfusion dependent.
Speaker Change: <unk>.
Speaker Change: Our initial launch focus will actually be equally deployed against the transfusion dependent patients, but also on the non transfusion dependent patients, which have hemoglobin levels less than 10 already have developed complication or experiencing debilitating fatigue and the reasons for that is that these patients are already.
Speaker Change: In an active engagement and communications with their health care providers, they are likely to hear about the therapy for us.
Speaker Change: We see them as a good starting point for our commercial uptake.
Speaker Change: Okay. Thank you.
Speaker Change: Thank you one moment our next question.
Speaker Change: Our next.
Speaker Change: <unk> comes from the line of Andrew Berens from Leerink Partners. Your line is open.
Speaker Change: Hi, everyone other than the Amanda on for Andy Thanks for taking my question it.
Speaker Change: It seems that you are starting to discuss more to the pivot more frequently in sickle cell disease and are slated to start the phase III is there any color that you can provide on kind of differences on how youre thinking like patients going into that study and then to that study versus rise up or any endpoints differences there that might be a focus.
Speaker Change: Any learnings that you are taking into this new trial and also have started to that shown any signs of impacting deliver in these early studies or how are you thinking about that thanks.
Speaker Change: Sure Amanda I'll, just start by making a comment that we will follow a very staged process to guide us on how we continue to differentiate but perhaps Eric can just add a little bit on the design itself for the phase two within thoughts ahead. So the phase two for <unk> at this sort of.
Speaker Change: Classic dose finding study in which we're looking for proof of concept of the update on by looking at a hemoglobin response. So it's a very standard development in sickle cell disease I would say.
Speaker Change: As we have said that I discussed earlier I think this is really about we're in a phase that the drug has shown promise in a phase one so now we're bringing it forward to a phase III.
Speaker Change: And you can continue to establish its benefit risk profile and so far we have not observed a liver signal.
Speaker Change: So that's.
Speaker Change: The team will continue to accrue safety data in that program and then a set of highlighted earlier me between each development phase III work very closely with our commercial team to make sure that the trials redesign can meet the product profile that commercial.
Speaker Change: <unk> requesting to be met at the end of the trial and that will be driven by how the market evolves.
Speaker Change: And how the population is growing and the unmet need within that patient population and so that is a little bit too early now to discuss because.
Speaker Change: The phase III comes after Facebook.
Speaker Change: And again this is.
Speaker Change: Our disciplined approach with capital allocation, we don't want to get ahead of ourselves.
Speaker Change: Just as last year, we waited for the energized data readout before we started building our commercial team very similar approach here as we look to build a sickle cell franchise, we will learn more from the phase two and then we'll make the right decisions at that time.
Speaker Change: Q1 moment, our next question.
Tess Romero: Our next question comes from the line of Tess Romero from Jpmorgan. Your line is open.
Tess Romero: Hey, guys. Thanks, so much for taking our question sure.
Tess Romero: Double clicking back to a prior question can you confirm or not if you have completed the mid cycle meeting and if so can you comment on any high level discussion you have had around labeling and.
Tess Romero: Is there a scenario where rins as needed or can you roll. This out at this point. Thanks, so much.
Tess Romero: Thanks for the question so.
Speaker Change: The process with the SBA they will announce the date of the review, which is the <unk> goal date of September seven so we're really working towards that with them and along the way you have different touch points, which may or may not be meeting questions et cetera, it's less it's less defined.
Speaker Change: Process than for instance, the EMEA, which certain point you can submit a question.
Speaker Change: Your filing in your key question, that's very specific dates et cetera, well. We have mentioned before is that we have a collaborative engagements with the FDA, we're receiving questions back and forth.
Speaker Change: This is part of the standard process that we will be.
Speaker Change: Feel it's a very normal engagement at this point in time with the agency.
Speaker Change: <unk>.
Speaker Change: Again like I think the only thing that I would really of anchor towards too is the September 7th.
Speaker Change: At this point in time the labeling negotiations.
Speaker Change: As we've mentioned to review is ongoing rate to date, they have not informed us that there will be an advisory committee, but that review is ongoing as we've mentioned label.
Speaker Change: Labeling negotiations typically go later in the process of a review cycle. So it's too too early for that in regards to <unk>.
Speaker Change: Rents or not.
Speaker Change: I think you can only regionally.
Speaker Change: Fully be certain when you have reached your could do faculty at all on what the ultimate label shelf and what will be required, but I think right now we're very pleased with where we are and it's from our perspective.
Speaker Change: Just a normal process.
Speaker Change: Thank you.
Thank you.
Speaker Change: As a reminder, that star one for questions or.
Speaker Change: Our next question comes from the line of solving Richter from Goldman Sachs. Your line is open.
Lidia: Hi, Good morning. This is lidia on for Sylvia and thanks, So much for taking my question and congrats on all the progress maybe.
Lidia: Maybe just another one on the potential of thalassemia launch could you just comment on any anticipated evolution of pirate Hines pricing in the context of payer feedback the existing price <unk> and the patient population here. Thanks, so much.
Speaker Change: Thank you for the question I can't wait for the September seven for <unk> and for Us to talk a little bit more specifics about pricing then.
Lidia: But of course.
Lidia: Any pricing decision will be anchored in the value proposition of the product the label that we get based on where we stated today.
Lidia: <unk> is a rare disease and from a payer perspective, we don't expect that category to be managed all of the interactions and the bay area that we've done indicates that there is a good understanding of the unmet medical need and a very positive feedback on the product profile, we have a very strong market access team.
Lidia: I am very confident we can navigate the pricing opportunity with Dallas EMEA.
Lidia: Very well.
Speaker Change: Thanks, so much.
Speaker Change: Thank you I'm not showing any further questions at this time I would now like to call turn the call over back to Brian for any closing remarks.
Speaker Change: Alright, Thanks, a lot Victor and thank you very much everybody for participating in today's call. We are as we've noted.
Speaker Change: Four months into another busy year.
Speaker Change: We're four months away from the <unk> date for thalassemia, which is very exciting. So we really believe that it ought to use we're poised to deliver transformative new therapies for patients and create significant long term value to our shareholders. So thanks again, and we look forward to speaking with all of you again real 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: Okay.
Speaker Change: [music].
Speaker Change: Okay.
Speaker Change: Good.
Speaker Change: Yes.
Speaker Change: Yes.
Speaker Change: [music].
Speaker Change: Okay.