Agios Pharmaceuticals Q1 2023 Earnings Call Transcript

Quartr
Provided by Quartr
May 4, 2023

There are 9 speakers on the call.

Operator

Good morning, and welcome to Agios' First Quarter 2023 Conference Call. At this time, all participants are in a listen only mode. There will be a question and answer session at the end. Please be advised this call is being recorded at Agios' request. I would now like to turn the call over to Agios' Chief Financial Officer, Cecilia Jones.

Operator

Please go ahead.

Speaker 1

Thank you, operator. Good morning, everyone, and welcome to Agios' Q1 2023 conference call. You can access slides for today's call by going to the Investors section of our website, adios.com. On today's call, I am joined by our Chief Executive Officer, Brian Boff Doctor. Sarah Humes, our Chief Medical Officer and Head of Research and Development and Sveta Milanova, our Chief Commercial Officer.

Speaker 1

Before we get started, I would like to remind everyone that some of the statements including those set forth in our most recent filings with the SEC and any other future filings that we may make with the SEC. With that, I will turn the call over to Brian.

Speaker 2

Thanks, Cecilia. Good morning, everyone, and thank you for joining us. Agios is the pioneering leader in PK activation and is dedicated to developing and delivering transformative therapies for patients living with rare diseases. We continue to generate consistent and compelling data with our PK activators across multiple disease areas, Highlighting the potential of this differentiated mechanism of action to transform patient function, quality of life And long term outcomes in PK deficiency, thalassemia, sickle cell disease and lower risk MDS. Driven by the enthusiasm of our clinical investigators and the operational excellence of our research and development team, I'm pleased to report that we have closed screening in our Phase 3 studies in thalassemia, which together encompass the entire thalassemia population and expect to complete enrollment of these studies later this month.

Speaker 2

On behalf of the Agios team, I'd like to thank the patients, Caregivers and physicians for their continued participation in these trials. As Sarah will describe in more detail, the rapid enrollment of these studies Highlights the significant unmet need in these areas and bolsters our conviction in our broader clinical development strategy. In this context, we continue to advance our robust clinical stage pipeline and are on track to announce the data readout From the Phase 2 portion of the operationally seamless Phase 2, 3 RISE UP study of pyrokin in sickle cell disease And the gono go decision at Phase 3 in the middle of this year. Echoing the positive reception of both pyrokind and AG-nine forty six and clinicians on the impact that Pyrakine is having on individuals living with PK deficiency in the real world. We're encouraged to see that the efficacy of pyrokind observed in the clinical trial experience is translating to persistency on therapy in the real world, And we believe this has positive implications for the potential long term impact of PK activation on how patients feel and function In not just PK deficiency, but also thalassemia, sickle cell disease and lower risk MDS.

Speaker 2

Seda will provide a detailed update on our commercial performance in just a few minutes. As you'll hear from Cecilia, we We ended the Q1 of 2023 in an enviable cash position with approximately $1,000,000,000 on the balance sheet. As a reminder, as part of the divestiture of our oncology business to Servier in April of 2021, We retain rights to a potential milestone upon FDA approval of vorasidenib and royalties on potential U. S. Net sales.

Speaker 2

We were therefore pleased to see that Servier's Phase 3 trial of oracitinib in patients with residual or recurrent IDH Low grade glioma met both its primary endpoint and key secondary endpoints. These data underscore Agios' Strong track record in the discovery and development of therapeutics for disease areas with high unmet need, which to date spans 5 approved indications across 3 separate products. In addition to the upcoming Phase 2 data readout of by the end of the year, including completing enrollment of the Phase 3 studies of pyrotine and thalassemia, Enrolling more than half of the patients in the Phase 3 ACTIVATE Kids and ACTIVATE Kids T studies of pyrokinin pediatric PK deficiency, Completing enrollment of the Phase 2a study of AG-nine forty six in lower risk MDS and Filing the IND for our PAH stabilizer for the treatment of PKU. Finally, looking forward to what we expect to be a catalyst rich next few years. We anticipate readouts from the Phase 3 studies of pyrokine in thalassemia in 2024 And readouts from the Phase 3 studies of pyrokinin sickle cell disease and pediatric PK deficiency in 2025.

Speaker 2

With that, I will now turn the call over to Sarah. Sarah?

Speaker 1

Thanks, Brian. In the Q1 of the year, our team made tremendous progress Executing across our industry leading pipeline of PK activators, and we were pleased to receive the 2023 Sideline Award for Excellence in Rare Disease Drug Development. This award recognizes SpiroKine's approval in PK deficiency and its significant potential impact in other hemolytic anemia, including sickle cell disease and Let me now provide a brief update on each of our programs, beginning with thalassemia, where Pyrites has the potential to The first oral therapy to improve hemolytic anemia and ineffective erythropoiesis across all thalassemia subtypes. Specifically, the Phase 3 thalassemia program comprises 2 randomized placebo controlled trials, each of which is enrolling patients with both alpha and beta thalassemia. Energize is enrolling patients who are not regularly transfused with a primary endpoint of hemoglobin response and Energize P is enrolling patients who are regularly transfused with the primary endpoint of transfusion reduction response.

Speaker 1

Reflecting our operational In clinical development and our investigators on to the extent for the potential of spirochetes in this indication, we have closed screening in both Energize and Energize T and we expect to complete enrollment later this month and report top line data next year. Turning to Our goal is to address the high unmet patients' need with an oral therapy that improves anemia, reduces sickle cell pain crisis and provides a durable improvement in how patients To meet this differentiated target product profile, we are advancing the operationally seamless Phase IIIII rise up The primary endpoints of the fully enrolled Phase 2 portion of the RISE UP studies are thermoglobin response and safety and we are on track to announce the Phase 2 data readout and the go no go decision to Phase 3 in the middle of this year. This will be a data driven decision informed by the protocol defined go no go criteria in the Phase 2 portion of Ryzhov, including any additional data from the Phase 2 secondary endpoints And longer term data from the ongoing extension studies of the investigator sponsored trials at the NIH and the University of Utrecht. Finally, we continue to advance the Phase 3 ACTIVATE KITS and ACTIVATE KITS T studies of pyracamcin pediatric PK efficiency as we aim to deliver the 1st approved therapy for children living with this disease.

Speaker 1

We expect to enroll at least half of the patients in each of these studies by the end of the year. Turning to the development of AG-nine forty six, our novel PK activator that has the potential to strengthen our PK As a reminder, low risk MDS accounts for approximately 70 Based on the data we have generated in the Phase 1 study of AG-nine forty six in healthy adults. We look forward to progressing this study and expect to complete enrollment of the Phase 2a portion by the end of this year. As we continue to advance the development of our PK activators, we also aim to expand our portfolio beyond PK through both business development and advancements of our earlier stage pipeline. In this context, we continue to progress our small molecule Phenylalanine hydroxylase or Our PAH Stabilizer program to directly address the underlying cause of phenylketonuria and we are targeting an IND filing for this program by the end of this year.

Speaker 1

Overall, I'm excited by the tremendous progress the team made executing across our portfolio this quarter and look forward to a number of important milestones over the next several months. With that, I will now turn the call over to Sarah. Thank you, Sarah. Our commercial organization Is laser focused on executing a comprehensive strategy that addresses each phase of the patient journey From disease awareness to reimbursement, adherence and persistency. By doing so, we aim to maximize the opportunity In the current launch in PK's efficiency and build the capabilities needed to fully realize the potential of anticipated future launches in thalassemia, Sickle cell disease and lower risk MDS.

Speaker 1

A foundational element of any successful launch Is a clear benefit risk profile consistent with the positive feedback we received from patients, providers, We are pleased that the compelling data we observed with pyrokines in the CKD deficiency clinical trial program It continues to translate into persistent treatment use in the real world. Specifically, our market research data indicate That nearly 100% of our target healthcare providers are likely to recommend Biokind So they are adult patients with PK deficiency. For clinicians, key drivers of these recommendations include Improvement in hemoglobin levels, reductions in transfusion frequency and impact on long term disease complications and comorbidities. While we remain focused on patient identification and improving disease awareness, Launch to date, discontinuations have remained low overall, reauthorizations have not been a barrier and persistency of treatment has remained strong beyond 6 months from treatment initiation. Together, we believe this reflects both The positive impact of Pyrokines on how patients feel and function and the strength of our commercial capabilities, including market access and patient services.

Speaker 1

In the Q1 of 2023, Which represented the 4th full quarter of launch, we generated $5,600,000 in net SpiroKine revenue, A total of 127 patients have now completed a prescription enrollment form or PES, including 22 in the Q4 of 2023, a 21% increase versus the Q4 of 2022. This has translated into net 89 patients on therapy, A 14% increase over last quarter. Patients on therapy continue to stem from a growing and diverse provider base I have 113 physicians and represent a broad demographic and disease manifestation range that is consistent with the adult PK deficiency population. I am confident that strengthening the commercial capabilities We are prioritizing today, including our ability to identify providers likely to treat patients appropriate for pyrokind, Enhance diagnostic efficiency, activate a broad range of prescribers and maintain long term adherence and reimbursement We'll help maximize the potential of the current launch and lay the foundation for potential launches in a meaningfully larger patient populations. Our next potential launch is in thalassemia, where approximately 60% of patients Do not have an approved therapy in the U.

Speaker 1

S. Importantly, Virokhyme has the potential to become the 1st oral therapy To improve hemolytic anemia and ineffective erythropoiesis across the full range of thalassemia patients, including both alpha and beta thalassemia. Alpha thalassemia currently has no approved treatment options and we will therefore require a particular focus on patient identification and disease education, leveraging the capabilities we are building in the current launch in PK deficiency. Beta thalassemia, on the other hand, is the more common form of the disease, Constituting approximately 2 thirds of the thalassemia cases in the U. S.

Speaker 1

Given its greater prevalence and more competitive landscape, We believe adoption in beta thalassemia will require a greater emphasis on market access and Viralkind's overall product profile, including efficacy, safety and route of administration. Taken together, each of these potential launches We will require a tailored commercial strategy and we aim to leverage capability from our current launch to address the needs of each of these populations. The full range of thalassemia patients is comprised of approximately 18,000 to 23,000 patients in the U. S. And EU5.

Speaker 1

In a meaningful addressable market outside these geographies, such as the Gulf Cooperation Council Countries, or GCC, where the prevalence of thalassemia is 8 to 9 times higher than the U. S. We look forward to evolving our commercial capabilities to meet the unique needs of our target diseases and deliver transformative new treatment options to these areas of profound unmet patient need. With that, I will now turn the call over to Cecilia. Thanks, Sarah.

Speaker 1

Our Q1 2023 financial results can be found in the press release we issued this morning, which I will summarize. More detail will be included in our 10 Q, which will be filed later today. Q1 2023 net Firocan revenue was $5,600,000 an increase of $1,300,000 compared to Q4 2022. As Biogen is the 1st therapy for this ultra rare patient population, we continue to gather data and insights on the launch trajectory and will not be providing guidance At this time, consistent with other rare disease launches, gross to net continues to be in the 10% to 20% range. Cost of sales for the quarter was $600,000 Moving to expenses and the balance sheet.

Speaker 1

R and D expenses were $67,300,000 for the Q1, a decrease of $2,800,000 compared to the Q1 of 2022. This decrease was primarily driven by the $1,500,000 of reimbursable transition related expenses we provided to Servier In the Q1 of 2022 related to the sale of the oncology business, SG and A Expenses were $28,400,000 for the Q1, a decrease of $3,100,000 compared to the Q1 of 2022 That was primarily driven by a reduction in workforce related expenses. As a reminder, TIBSOVO Royalty, Which was recorded under royalty income from gain on sale of oncology business on our income statement has ceased Given the sale of our rights to 5% royalties on U. S. Net sales of TIBSOVO to CAGARD for a one time payment of $131,800,000 in October 2022.

Speaker 1

As part of the divestiture of our oncology business to Servier, We retain rights to a potential $200,000,000 milestone upon FDA approval of oracitanib and 15% royalties on potential U. S. Net sales. We ended the quarter with cash, cash equivalents and marketable securities of approximately $1,000,000,000 We expect our cash, cash equivalents and marketable securities, together with anticipated product revenue and Interest income will enable us to execute our operating plan, including funding the currently planned development program for mitapivat, AG-nine forty six and our PAH stabilizer and commercialize mitotepal outside of the U. S.

Speaker 1

Through 1 or more partnership. To maintain our strong cash position, we will remain focused on proactively managing our cost base and deploying a disciplined cash allocation approach. I'll now turn the call back over to Brian for his closing remarks.

Speaker 2

Thanks, Cecilia. Given the rapid enrollment Of our ongoing studies of pyrokine in thalassemia, the persistence of real world pyrokine treatment in our current launch in PK deficiency In our strong balance sheet, our team is energized and focused on advancing our industry leading pipeline of PK activators To address the profound unmet needs of patients suffering from rare hematologic diseases. As we look ahead to the clinical and regulatory milestones We expect in the balance of the year, we will continue to strive to be responsible stewards of our balance sheet and evaluate meaningful opportunities for value creation. Finally, I'd like to thank all of our employees for their hard work and dedication to our mission of transforming the lives of patients Living with rare diseases and all of our partners, including the physicians, patients, caregivers and participants in our clinical development programs. With that, we will now open the call for questions.

Operator

Thank Our first question comes from Gregory Renza with RBC Capital Markets. Your line is open.

Speaker 3

Great. Thanks, Brian and team. Congrats on the progress and thank you for taking my question. Brian, maybe just more on the near term, If I may, I know there's little you and the team can perhaps say about the sickle cell regut that's upcoming. But perhaps if you could Just remind us or provide some color around some of the scenarios that you are accounting for when it comes to factoring in Your no go decision, what is perhaps nested in there as you think about some of the potential outcomes of that data and how you think about The new information that's forthcoming.

Speaker 3

And also as it relates to Brian, just the consistency of the data, as you have mentioned, you've been very pleased with that Over time and how that factors into the outcome as well. Thanks so much.

Speaker 2

Sure. Good morning, Greg, and thanks Yes, with respect to sickle cell disease, first off, I just want to again commend Sarah and her team for the excellent progress That she's made not just with sickle cell and getting us to the point of a data readout at the midpoint of the year, but As we noted on the call, I'm especially proud of the fact that thalassemia now with Screening completed puts us in a position for a readout in that domain in 2024, and that's equally important as well. For sickle cell, we have, of course, a number of scenarios that we're planning for. We are hoping and planning for success, of course, and that's why The trial was designed to be operationally seamless Phase twothree. So with success in the Phase 2 readout, The great news there is that Sarah and the team would be ready to go quickly and leverage the same Clinical site setup that we have in Phase 2 and obviously this is a very high unmet Patient need arena, and so we're eager on their behalf in that scenario to move forward.

Speaker 2

Specifically, with respect to what we're looking for, for the data, We've talked about this many times that we're going to take a holistic approach as we look at the data. The primary endpoint, Of course, in the Phase 2 rise up readout, we'll be focused on hemoglobin improvement. We have secondary endpoints, markers of hemolysis and the like. And then, of course, safety will be a key element and that will also guide us on the dose that we would pursue with success. At a very high level, the way we're thinking about sickle cell disease is that we'll be looking for a hemoglobin plus Dynamic.

Speaker 2

And so that could be hemoglobin plus a trend in VOCs, though it's a 12 week study, it's not designed for VOCs per se. It could also be patient feel and function. And we know that having studied the sickle cell disease arena For quite some time, that is a very important dynamic. It's not just success for the patient in the short term, it's the ability to stay on a therapy chronically, Which by the way is why we're so encouraged by the persistency that we're seeing so far in our PKD launch. So maybe I'll stop there and just see, Sarah, if you want to add anything With respect to the scenarios?

Speaker 1

No, I think it's covered. I think we're very excited about the way the study is designed because it does allow and gives us Flexibility for our different scenarios.

Speaker 2

And I'll just say again, Greg, we're excited about the period to come. We have Sickle cell disease readout at the midpoint of the year. We have thalassemia where we're going to be looking at the readout in 2024. We have AG-nine forty six where we're looking for proof of concept in our Phase 2a study in low intermediate risk MDS at year end. And then earlier in our research pipeline, we have the PA stabilizer that we're pursuing for IND at year end.

Speaker 2

So, it's a pretty busy time to come, but thanks again for the question.

Speaker 3

Thanks, Brian.

Speaker 4

You bet.

Operator

Our next question comes from Tess Romero from JPM. Your line is open.

Speaker 5

Good morning, guys. Thanks so much for taking the question. So you've laid out a number of factors that will contribute to the gono go for mitapivat Can you just remind us of the status of the Phase 1 sickle cell disease cohort for AG-nine forty six? And do any of the results there in patients on 946 factor into this decision, right? So in other words, could there be a scenario in which you elect to move 946 forward instead of mid to that?

Speaker 5

Thanks so much.

Speaker 2

Thanks a lot, Tessa. I'm going to have Sarah weigh in on this one.

Speaker 1

Hi, Tessa. So for 946, The Phase 1 is ongoing and we have not disclosed the time points at which we will be presenting data. In regards to the second part of the question, 946 and mitapivat are the totally different phase of development And 348 has built Somitapivat has built a huge Safety profile has much longer exposure and is really past the Phase 1 Development, so to substitute the one compounds for the other is not something that we believe is doable, just in the context of each Product needs to build up its own safety profile and 946 in our view is less advanced at this point.

Speaker 2

And Tess, again, I'll just reiterate that I started the call this way that with Agios as the leader in PKR activators, we are Pleased to have not one, but 2 that we're pursuing in clinical trials. And that gives us, as you noted, quite a lot of Optionality, the near term readout that we're quite eager to see would be in the low intermediate risk MDS patient population, again, at the year end. But it is quite an advantage to even be able to stratify across different therapeutic areas with 2 PKR activators When we get into the data readout, if positive, preparing for launch and then the economics that could surround that to have 2 is particularly advantageous versus having all therapies lined up within 1. So,

Operator

Thank you. And our next question is coming from Mark Breidenbach From Oppenheimer, your line is open.

Speaker 4

Hey, good morning, guys. Thanks for taking the questions. Two quick ones from me. First, with respect to the Energize and Energize T trials, which are nearly completing enrollment. I'm curious if you've seen any trends in specific geographies that are enrolling patients fastest, Given that these trials have so many ex U.

Speaker 4

S. Clinical sites, and is that trend potentially going to influence Your plans for regulatory filings, which territories to file in the first? And then the second question is just with respect to vorasidenib and the maybe Serdier's plans For a future NDA filing, do you have any clarity on when such a filing might happen? Thanks for taking the questions. Sure.

Speaker 2

Thanks a lot, Mark. So we'll take those in sequence. Sarah, you want to start off with Energize, Energize T in the clinical trial geographies?

Speaker 1

Sure. So, and we've presented in the past our clinical trial distribution globally. And so this is really in the context of reflecting where thalassemia is present and also reflecting How the distribution of thalassemia is between different genotypes because the program is developed to deliver a product for All thalassemia, meaning alpha and beta thalassemia regularly and non regularly transfused. And so the clinical development program It's designed that way, but also designed in such a way that it is feasible to enroll these trials that way. So far we have been very pleased with how that has worked out.

Speaker 1

Our clinical trial enrollment is really reflective of the distribution of thalassemia globally. And so we do not see any changes needed to any of our regulatory

Speaker 2

And I'll just add before we transition to Cecilia for the question for vorasidenib that Having Smeda Milunova on board as our Chief Commercial Officer is really advantageous. Mark, when we think about with successful data, To your point, your question about launch sequencing, as Seda spoke about in her remarks, this is Thalassemia is a uniquely concentrated addressable market geographically, and so we will be particularly thoughtful about The sequencing in part, well, of course, to address access for the most patients in those geographies as possible, But also with an eye towards the value maximizing economics with respect to pricing and so on. And again, this all comes obviously with Positive data, but it puts us in a position of strength with respect to how concentrated that population is. And Having Svet on board as a world class expert in globalization of rare disease products is a real advantage. Maybe we'll have Cecilia comment on vorasidenin.

Speaker 2

Yes.

Speaker 1

So we're very excited obviously to hear the Phase III results. It's a great huge unmet need for patients. From our side, we retained the rights for a $200,000,000 milestone on FDA approval and the 15,000,000 15% Royalty and net U. S. Sales, we don't have clarity on timing yet and we'll provide an update when we have it.

Speaker 2

And obviously, this is in Servier shop, But the original science and progress came out of Agios. And so I know we've got a lot of Agios employees listening in as well. And then I just want to say we are off the charts proud of what that could mean for glioma patients where they haven't had any progress in the last 20 plus years. And This only adds to the track record that Agios has had across a number of fronts, and now we're leveraging all that great science into our progress in PKR activation.

Speaker 4

Thanks so much. You bet.

Operator

We have a question from Greg Harrison with Bank of America. Your line is open.

Speaker 6

Hey, good morning. Thanks for taking the question. How would you say your PAH stabilizer Is different from a drug like Kuvan? And where would you see it fitting in the PKU treatment landscape with enzyme replacement and Gene therapy approved or under development?

Speaker 2

So, Greg, first off, good morning. I will start by saying too early to give Too much on that front. However, we are keen to progress our PA Stabilizer to IND, but I'm going to let Sarah

Speaker 1

And I think our program is really designed like all of our programs are really designed to target unmet need in patient And for this one specifically as well, we will be designing the program to meet a product profile that we believe is Can provide meaningful difference for patients living with PKU. The current therapies available each have their limitations. And so that is what we are hoping to address with this product to create a differentiation there. And that can be on the safety profile, but also on the amount of product people need to take to get to some meaningful difference And to actually also shoot for further reduction of A better improvement basically of the phenylketonuria overall. And so more to come on that as we progress the molecule.

Speaker 1

It's early now and as with any phase moving to IND and then Phase 1, we will be learning as we develop the program.

Speaker 2

And it's another great example of why we're so passionate about rare diseases because every single therapeutic area that we pursue is desperate For innovation and disruption and whether that's PKD or sickle cells, we've talked about already, thalassemia, NDS And PAH, that's the common thread of our focus.

Speaker 6

Got it. That's helpful. If I could sneak one quick one in on the sickle cell. What forum will you use to announce that data? Would it be Press release or a conference and then will you be announcing the gono go decision at the same time or could there be a delay?

Speaker 1

So in regards to how and when we will announce, it will depend on when we receive the data, but we are Planning to do a press release, which will not include all of the data because of course We do want to present at a medical meeting and so it's always a balancing act between those 2. But in typical Agile's fashion, I think what we've done for our past Programs like PKD, you can expect something in that similar vein?

Speaker 2

Yes. I mean, our target will be To give our stakeholders enough clarity as to the why behind our decision, whichever way that goes based on the data.

Speaker 6

Got it. Thanks again.

Speaker 2

You bet, Greg. Thanks.

Operator

Thank you. One moment. We have a question from Divya Rao from TD Cowen. Your line is open.

Speaker 7

Hi, guys. Thanks for taking our questions. This is Divya on for Mark. Given the commentary on needing to see this hemoglobin Plus response, if the VOC trend isn't the view is it seen, is the view that the PRO analysis would be predictive Of ultimately showing a VOC benefit or do you think the PROs themselves support this hemoglobin plus commercial profile? And then I guess Specifically, are there any PROs that are particularly important?

Speaker 1

So this is the Phase To read out, right. So the PRO analysis and VOC benefit, all of that will really come at the end of Phase 3 in line with our product profile. What we are doing now with the Phase 2, it has primary endpoints of hemoglobin response and safety and then indeed secondary endpoints That can capture PROs, VOCs, hemolytic parameters, But those will be looking we will be looking for trends in those data sets. We will, of course, be looking at the totality of the data generated to date Across the different studies that are using mitapivat in sickle cell disease and that is being taken into account.

Speaker 7

That's helpful. And then if I could sneak another one also. On PKD, I guess one is, was there any stocking effect seen in Q1? And then With this being the 2nd month of reauthorization, are you seeing any trends or I guess changes in the way payers are evaluating what would be considered a response or benefit?

Speaker 2

Thanks, Divya. I'm going to have Cecilia comment on stocking and then Sveta is always eager to talk about launch dynamics. So,

Speaker 4

Thanks, Jillian.

Speaker 1

Thanks, Brad. So as a reminder, we only have we have a limited distribution model. We have 1 specialty pharmacy and 1 specialty distributor. So our inventory levels are Inventories on hand are limited. So you see there might be some fluctuations quarter over quarter.

Speaker 1

We're still talking about small numbers.

Speaker 2

Nothing meaningful. Yes. And Sarah?

Speaker 1

Absolutely. So as I noted in my remarks, we are really encouraged with the positive reception of Biokarn's profile From patients, physicians and on the payer front as well. But when it comes to treatment initiations, but also we see that we reauthorizations as well. Until today, the reauthorizations have not been a barrier. Patients do utilize the inclusion Excluding criteria as well as the primary endpoint, defining in the clinical trial to assess reauthorization.

Speaker 1

However, the totality of the product profile is taken into account and we see that both in the success of the reauthorization and the strong We see even though early in the launch with small patient numbers, 4 patients will continue post 6 months of reauthorization as well.

Speaker 2

Yes. And we hope that on today's discussion, we hope that message really comes through that pyrokine in PKD is And we certainly have heard that anecdotally from patients. We now are starting to see that in persistency. And then our market research, as That I had commented on earlier has certainly indicated that the clinicians who are on our target list, which is inclusive of those who have actually prescribed Pyrokine Share in that enthusiasm. So the launch dynamic really is not so much around access Or enthusiasm for the product.

Speaker 2

It's really about education, diagnostic efficiency and activating Patients especially to begin therapy. That's really where Sveta's emphasis commercially is focused.

Speaker 7

That's helpful. Thank you.

Speaker 2

You're welcome.

Operator

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

Speaker 8

Hi, good morning. This is Anna Mig on for Salveen. Just one on the PKD launch. Could you just provide an update on Patient discontinuation rates and whether what you're seeing in the real world is consistent with what was observed in the clinical trials? And then just on sickle cell, I guess you've walked us through the what you're going to be looking at in terms of the data.

Speaker 8

But I guess are there specific deal breakers from the of So the factors that you're looking at that would lead to a no go decision, if you could just frame that scenario? Thank you.

Speaker 2

Sure. And we'll do that again in sequence. So, Sveta can start with TKD discontinuations and then sero on sickle cell.

Speaker 1

Absolutely. So, as we mentioned, we are really encouraged to see that the efficacy of biopharma, which we observed In the clinical trials and experience there is very much translating into persistency to therapy in the real world setting. With that in mind, the discontinuations have remained low overall. And so far, as we mentioned, reauthorization But it really gives us also the encouragement that the importance of consistency, which is associated with pyrokine Could potentially translate in other indications, future indications we are looking to expand into. And that can be a critical differentiator knowing how important persistence

Speaker 2

Super. Sarah?

Speaker 1

Yes. And I think to that point of the clinical trial, that Persistency and adherence we've seen in the clinical trials, which has allowed us to continue to follow patients in the long term extensions, which has allowed us to continue to study Maintenance of effect, impact on how patients feel and function, and then we're also very excited about our iron overload data that we continue to present. And so it's that compelling data across the board. As Sarah mentioned, it's very important to us because we also look at that in the context of the other hemolytic anemia. And then turning to the other hemolytic anemia of sickle cell disease.

Speaker 1

In regards to the no go scenarios there, there are some obvious No go scenarios as what would be on the clinical trial as expected for a clinical trial is when a clinical trial would fail. So if you Do not see hemoglobin response, for instance, in the primary endpoint or we have a safety signal that tips the benefit risk profile the wrong way, Things like that are all proactively defined in our go, no go scenarios. And then we have, As for any other clinical development program, clear goals and then scenarios in which more data will need to be assessed.

Operator

Thank you. And I'm showing no other questions in the queue. I'd like to turn the call back to Mr. Brian Gough for any closing remarks.

Speaker 2

Well, thanks a lot and thanks everyone for participating in today's call and of course for your continued interest in Agios. So as you heard this morning, we are making great progress across our portfolio. I am incredibly proud of the team For what they're doing on behalf of patients who count on us, and I'm confident in our potential to continue to deliver significant value

Operator

This concludes today's conference call. Thank you for participating. You may now disconnect.

Powered by Quartr
Earnings Conference Call
Agios Pharmaceuticals Q1 2023
00:00 / 00:00