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spk05: Good morning, and welcome to AGO's second quarter 2021 conference call. At this time, all participants are in a listen-only mode. There will be a question-and-answer session at the end. Please be advised that this call is being recorded at AGO's request. I would now like to turn the call over to Jessica Renningkamp, Director of Corporate Communications.
spk13: Thank you, Operator. Good morning, everyone, and welcome to AGO's second quarter 2021 conference call. You can access slides for today's call by going to the Investors section of our website, adios.com. With me on the call today with prepared remarks are Dr. Jackie Faust, our Chief Executive Officer, Dr. Chris Bowden, our Chief Medical Officer, Darren Miles, our Chief Commercial Officer, Jonathan Biller, our Chief Financial Officer and Head of Legal and Corporate Affairs, and Dr. Bruce Carr, our Chief Scientific Officer, who will join for Q&A. 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 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 Jackie.
spk07: Thanks, Jessie. Good morning, everyone, and thanks for joining our second quarter 2021 financial results call. We are pleased to report another quarter of strong progress at AGIOS, the first full quarter since the close of the sale of our oncology business to Servier. The team is focused, energized, and excited for the future as we continue to advance our pipeline in genetically defined diseases led by Medipivet across its three initial indications in hemolytic anemias. We achieved significant recent milestones with the submission of our NDA for Medipivet in adults with pyruvate kinase deficiency in June, as well as with the submission of our MAA to the EMA the same month. We look forward to working with the FDA and EMA as they complete their reviews. With these two filings, we are one step closer to delivering the first potentially disease-modifying therapy for people with pyruvate kinase deficiency. These regulatory submissions are supported by strong Phase III data from the ACTIVATE and ACTIVATE-T studies. Full results from both were recently presented at the European Hematology Association, or EHA, virtual congress in June. Our preparations for the commercial launch of MediPIVAT in PK deficiency continue to advance, and we remain on track for anticipated approval and launch in 2022. In addition, we would like to extend our congratulations to the Pyruvate Kinase Deficiency Foundation and the Thrive with Pyruvate Kinase Deficiency Organization both of which recently launched as the first two U.S.-based advocacy organizations dedicated to PK deficiency. Both organizations are patient-led, and it's very exciting to see how this community has coalesced and the momentum they are gaining in raising awareness and advocating for those living with this disease. Beyond PK deficiency, we are also making excellent progress with the late stage development of midipivate and thalassemia and sickle cell disease. At EHA, we presented positive results from our phase two open label study of midipivate in adults with non-transfusion dependent alpha or beta thalassemia. These data continue to validate the potential of PK activation as an entirely new mechanism for treating thalassemia and represent the first clinical data generated in alpha thalassemia patients. We remain on track to initiate our two registrational phase three trials, ENERGYZE and ENERGYZE-T, in not regularly transfused and regularly transfused adults with thalassemia, respectively, this year. In sickle cell disease, we also remain on track to initiate a pivotal phase two, three trial of Minipivet by the end of the year. Beyond MediPIVAT, we are building on our expertise in PK activation and cellular metabolism and are advancing our earlier stage research programs in genetically defined diseases to position us for sustainable growth and momentum. In the fourth quarter, we plan to host an investor day to share more information about our commercial launch planning for MediPIVAT and PK deficiency and talk more in detail about our research and development pipeline. Before I turn the call over to Chris to provide an update on our clinical development programs, I wanted to take a moment to share that Chris has decided to retire from his role as Chief Medical Officer at Adios after a terrific seven years on our leadership team. After a transition period to his successor over the coming months, I'm very pleased that Chris will remain part of our team as a strategic advisor through at least the end of 2022. We are excited for him as he enters this new chapter of his life and want to thank him for his leadership and many years of service at Agios. Among his many accomplishments and contributions, he oversaw the build-out of our medical and clinical development organizations, acted as Agios' lead medical representative internally and externally, navigated complex regulatory interactions in order to bring important drugs to patients, and played a key role in the evolution of our organization and culture. He's been an integral part of our team, and his work will continue impacting the lives of patients for many years to come. On behalf of everyone at AGIOS, I wish Chris all the best in the future. I'm equally excited to announce that Dr. Sarah Guins, who has up to now served as our head of clinical development for genetically defined diseases, will assume the role of Chief Medical Officer effective September 1st. Sarah has been with AGIOS for almost two years and has been an impactful and energizing leader across the organization during her tenure. Among Sarah's most significant accomplishments at AGIOS, she played an integral role in the work associated with the PK deficiency filings and the design and strategy for our sickle cell disease pivotal programs. She knows all aspects of our programs inside and out and has a genuine passion for serving people with genetically defined diseases. Prior to Agios, Sarah held roles of increasing responsibility at Biogen in safety and risk management, medical affairs, and clinical development, and she is a neurologist by training. We look forward to continuing to see great things from her as she takes on this new level of responsibility. With that, I will now turn the call over to Chris.
spk12: Thanks, Jackie. As Jackie mentioned, we are excited about the potential we have to impact the lives of individuals with genetically defined diseases. We achieved important milestones for both AGIOS and the PK deficiency community this past quarter with the submission of our global regulatory filings for Mitipivac in the U.S. and E.U. We're pleased to share that our MAA has passed validation, which triggered the start of the MAA review procedure. For the NDA, the FDA has 60 days from submission to determine whether the application is accepted, so we expect to hear from them in mid-August. Information about whether the application is accepted, whether MediPivot has been granted priority review, and the PDUFA date will all be communicated by the agency at that time. Overall, we are very pleased with our progress and believe we have significant momentum as we look to a busy second half of 2021. At EHA, we presented full data from the ACTIVATE and ACTIVATE-T Phase III studies evaluating mid-PIVAP in adults with PK deficiency who are not regularly transfused and those who are regularly transfused, respectively. Both studies met their primary and secondary endpoints, including patient-reported outcomes that addressed symptom burden and quality-of-life impact of PK deficiency. Medipivap was generally well-tolerated, and the safety profile observed in both studies was consistent with previously published data. During our EHA investor event, we also heard from Dr. Andreas Glentos, who highlighted the significant impact PK deficiency has on patients' quality of life the limitations of the current PK deficiency treatment landscape, and the potential for midipivap to serve as the first disease-modifying therapy for these individuals. Moving to thalassemia, we also presented data at EHA on all 20 patients in the core period of our Phase II study of midipivap and non-transfusion-dependent alpha and beta thalassemia. Consistent with previously announced proof-of-concept data, The study met its primary endpoint with 16 of the 20 patients achieving a hemoglobin increase of greater than or equal to one gram per deciliter from baseline at one or more assessments during weeks four through 12. In addition, a sustained hemoglobin response and improvements in hemolysis and ineffective erythropoiesis were observed in both alpha and beta thalassemia patients treated with midipivac. Midipivac was also well tolerated with a safety profile consistent with previous studies. We believe these results underscore the potential of midipivac to meaningfully improve hallmarks of thalassemia, including hemolysis and ineffective erythropoiesis. We are also excited to see data generated for the first time in alpha-thalassemia, demonstrating an increase in hemoglobin from baseline in all five patients in this subgroup. Our two global placebo-controlled pivotal trials of midipivap and thalassemia, Energize and Energize-T, are on track to initiate this year as we continue the process of submitting these trial protocols globally and prepare sites for enrollment. Now turning to sickle cell disease. We remain on track to initiate our pivotal Phase 2-3 clinical trial by the end of this year. The Phase 2-3 study of midipivap and sickle cell disease will include patients who are 16 years of age or older, have had between 2 to 10 sickle cell crises in the past 12 months and have hemoglobin within the range of 5.5 to 10.5 grams per deciliter during screening. Phase 2 will randomize 69 patients 1 to 1 to 1 to 50 milligrams midipivac twice daily, 100 milligrams midipivac twice daily, or match placebo. The primary endpoint is a hemoglobin response defined as greater than or equal to one gram per deciliter change from baseline to week 12, and the data will be used to establish a clear dosing paradigm for the phase three portion. Phase three, which will commence after the phase two analysis, will randomize 198 patients two to one to the selected phase two dose of midipivac or matched placebo. The study will have two primary endpoints. hemoglobin response defined as greater than or equal to one gram per deciliter change from baseline to week 52, and a reduction in annualized rate of sickle cell pain crises. We believe the design of this Phase 2-3 study minimizes risks to the approval path for mid-PIVAT in this challenging disease and maximizes the likelihood of a label with a broad indication. In addition, we continue to work with our collaborators at the NIH and the University of Utrecht on their studies of mediprivat and sickle cell disease. Data from both studies are expected to be submitted for presentation at ASH in December. At the NIH, Dr. Chen has completed enrollment in the core study with 17 patients and continues to enroll the extension study. We anticipate the datasets at ASH will provide additional efficacy, safety, and translational data that supports the clinical development of midipivate in people with sickle cell disease. Beyond midipivate, we're also advancing our next generation PKR activator, AG946, in a phase one healthy volunteer study. The trial began enrolling last year, and we expect to submit data for presentation at ASH. Our analysis of the totality of the AG946 healthy volunteer data will inform next steps for the clinical development of this molecule. As part of our investor event this fall, we will talk in more detail about our research and development pipeline. Before I turn it over to Darren to discuss our commercial activities, I want to thank all of my AGIOS colleagues for the privilege of working with you over the last seven years. It has been so rewarding and fun, and the accomplishments speak for themselves. My successor, Sarah Glenz, is poised to lead our team to more great accomplishments, and I look forward to watching the AGIOS story continue to unfold. Darren?
spk09: Thank you, Chris. We continue to make strong progress on commercial launch preparations for mid-PIVAD and PK deficiency. All customer-facing and patient support infrastructure and talent are hired and fully trained. Our field team, comprised of both sales representatives and credentialed nurse clinical educators, are now fully engaged with physicians potentially managing patients with PK deficiency across the U.S. and are focused on profiling accounts, raising disease awareness, and executing patient support and profiling plans through disease education. An important feature of their work is educating physicians on the availability of the AGO-sponsored genetic testing service, AnemiaID. With now well over 1,000 test kits requested, we expect AnemiaID to become an increasingly important program to help support patients seeking a definitive diagnosis of their hemolytic anemia and an aid in accelerating our PK deficiency patient profiling efforts. Our market research now shows that 70% of responding physicians routinely order additional diagnostic tests for their patients with hemolytic anemias of unknown etiology. This is an important insight, which favorably pretends future adoption of genetic testing for accurate diagnosis of hemolytic anemias, including PK deficiency. Response to the anemia ID program has been very enthusiastic. And in our research, responding physicians indicated they are inclined to test a significant portion of their patients with hemolytic anemia of unknown etiology based on the breadth of mutations included in the panel and its relative ease of use. In June, we also launched the reimagined MyoGeo's patient support service dedicated to people living with PK deficiency and their caregivers, leveraging our learnings and much of the technical infrastructure from the oncology MyoGeo's program while incorporating insights from PK deficiency patients and clinicians create a customized program designed to meet the specific needs of this community. Enrolled patients and caregivers are connected with a dedicated and clinically trained patient support manager to provide ongoing tailored support, educational resources, and opportunities to connect with other patients and caregivers in the PK deficiency community. Amongst other educational efforts, our field team is educating physicians and their staff on the availability of this service and how they can support patients with enrolling. Following the disclosure of the ACTIVATE and ACTIVATE-T findings at EHA in June, we fielded new market research to update our insights into general physician awareness and understanding of PK deficiency and the potential implications for disease management, assuming MediPIVET's approval next year. We found that PK deficiency disease awareness metrics are improved over the same time period in 2020, with 85% of academic and community physician respondents now familiar with PK deficiency. and more than half indicating that they manage one to two previously diagnosed PK deficiency patients. This is encouraging because it indicates that physicians are increasingly recognizing and diagnosing PK deficiency, which we intend to continue to support with additional educational activities in the months leading up to approval in 2022. Our research also suggests that based on their understanding of the Phase III data, Many physicians will consider prescribing Medipiva to PK deficiency patients with different levels of disease severity, including with respect to transfusion history and hemoglobin level. While prescribing will be based on individual patient history, presentation, and parameters of the approved label for Medipiva, we are encouraged by these responses and the overall increase in physician understanding of the burden of disease. Overall, We're very pleased by the progress we've made with launch preparations across each function at AGIOS and the response from the treatment community to the broad disease education efforts. We expect this to accelerate in the coming months now that we have our full complement in the field and as excitement about what we'll be able to do for patients' growth related to the activate disclosures. Now I'll turn it over to Jonathan to review second quarter financials.
spk11: Thanks, Darren. Our second quarter 2021 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 filing later today. As a reminder, our second quarter financial results discussion only includes our continuing operations on a comparative basis, which excludes results from our divested oncology business. Research and development for the second quarter was $62 million, an increase of $7.9 million compared to the second quarter of 2020. The year-over-year increase in R&D was driven primarily by startup costs associated with the phase three studies of mitopivet and thalassemia and sickle cell disease, and the filing of our NDA and MAA for mitopivet and PK deficiency, as well as our launch preparation efforts. Selling general and administrative expenses were $29.2 million for the second quarter, essentially flat compared to SG&A expenses for the second quarter of 2020. We also recorded $2 million in TIPSOBO income from royalties in the second quarter of 2021, which is included within the gain on sale of oncology business line item in our income statement. We ended the quarter with cash, cash equivalents, and marketable securities of approximately $1.7 billion. With this cash balance, we expect to be able to execute our current operating plan through major catalysts and to cash flow positivity without the need to raise additional equity. In Q2, we executed on 529 million of the up to 1.2 billion of share repurchases authorized by our Board of Directors, inclusive of the shares acquired from Bristol-Myers Squibb. Specifically, we repurchased just under 10.5 million shares at an average price of $50.41, reducing our total shares outstanding by approximately 15% to 59.9 million shares at quarter end. As previously disclosed, the 10b-5-1 plan we put in place in early Q2 provides for maximum additional share repurchases of approximately $415 million through year end. The ultimate amount of additional share repurchases through year end will depend upon the volatility of our share price over this time period. Operator, please open the line for questions.
spk05: Thank you. And as a reminder, if you would like to ask a question, press the star and the one key on your touchtone telephone. Our first question comes from Alicia Young with Cantor Fitzgerald. Your line is open.
spk06: Hey, guys. Thanks for taking my question, and congrats on all the progress. I wanted to talk a little bit about MediPivot and maybe just, you know, as you're doing some of the education for patient support, you know, how frequently do these people kind of see their physicians and, like, What kind of outreach and effort needs to be done there, or is it more just kind of like a structural kind of warehousing effect, or is there actually work that needs to be done to bring these people back under care? Thanks.
spk09: Hey, Alethea, this is Darren. The issue is not going to be necessarily getting patients to come back in because they're usually under, for those who have been diagnosed and obviously attached to a physician, they're under some sort of monitoring plan, right? Those with more severe disease are going to see their physicians more regularly. Those with less severe disease will have a longer, typically will have a longer time span between physician visits. But remember, they're continuing to have their iron load monitored, hemoglobin monitored. So the frequency of physician visits isn't necessarily where we would focus. I think what we're doing now is focusing on the patient profiling, identification, and physician education and patient education on the disease. And I think that's where we need to continue, even post-launch, to focus much of our efforts. The physician-patient relationship will be fine, and there's nothing there that we need to delve into. We just need to make sure that they're aware. And once that's done, I think everything else falls from there.
spk06: Great. Thank you.
spk05: Thank you. Our next question comes from Kenan McKay with RBC Capital Markets. Your line is open.
spk01: Hi. Thanks for taking the question. First off, Chris, I'm sorry to hear about your departure, but looking forward to meeting your successor, Sarah, and glad to hear that you're staying on into next year with the transition. Maybe one quick question on the regulatory filings here. From , I'm wondering which branch of the FDA you are interacting with with those regulatory submissions in PKD. Is that hematology or the rare disease division?
spk12: Hey, Ken. It's Chris. The FDA team that we're working with are on the cardiology, renal, and the oncology, hematology division. The submission itself is in the cardiology, renal. So it's an interesting setup, actually, because the people who have been most involved with us and are most active on the submission are on the hematology division.
spk01: That is just what I was going to sort of follow up with and just wonder around some of the interactions so far. Is that division sort of up to speed on rare diseases and PKD, or what have the interactions to date been like? Thank you.
spk12: The group, the team that we've been working with, with FDA, has been with the program for, has been advising us and interacting with us since I joined the company. So they're very well acquainted with the disease and the special nature of what it takes when you're in a rare disease population. So I don't have any concerns there. And I think the consistency and I think importantly over the years that you've heard us talking about the guidance and why we've designed the trials we have in an effort to provide them as much data as they're going to need to make a decision for us to demonstrate clinical benefit. It's been a consistent group of people.
spk05: Thank you. Our next question comes from Anupam Rama with JP Morgan. Your line is open.
spk08: Hi, guys. Thanks so much for taking the question. Perhaps a broader strategic question. I think many of us on the street, we model a price cut from a PIVAT longer term to account for the SCD indication. What threshold of peak sales in SCD does pivot have to meet, in your view, to justify a price cut versus, say, maximizing the value of the product by keeping ultra-orphan pricing? Thanks so much.
spk09: It would be premature to talk about what peak sales would be in sickle cell. What I can tell you is that it would require, assuming you just want it to at least break even, right, as you move from the first two indications into sickle cell and then you account for a cut, what we think would be required would only require less than 4%, 3% share in that market to be able to at least break even across the whole program. So it doesn't take a whole lot because the size of sickle cell is significant for you to be able to accommodate that cut. That's why we're so optimistic about the incremental value that we'll be able to gain once we move into sickle cell despite competition.
spk07: And it's Jackie. I'm just going to jump in real quick. As we've said many times, but I think both Chris and Darren have said this often, we'll make those pricing decisions based on the totality of the clinical data that we have at that point in time and the product profile, of course. So you can imagine there's a lot going on when you think about that with respect to hemoglobin increase, VOC reduction, all the secondary endpoints and the patient-reported outcomes and all of that. So lots of things to think about when we make that pricing decision at some point in the future.
spk08: Thanks so much for taking our questions.
spk05: Thank you. Our next question comes from Selvine Richter with Goldman Sachs. Your line is open.
spk03: Great. Good morning, and thank you for taking our question. This is Elizabeth on for Selvine. Could you just remind us if you intend to launch simultaneously in both the U.S. and EU for MediPivot and PKD? And then, you know, knowing that you're sort of discussing this at your investor day in 4Q, maybe if you could just comment on the structure and size of the launch team for and your progress in terms of identifying more patients, and then how many patients with PKD have been identified to date. Thank you.
spk09: Gotcha. All right, Sylvain, this is Darren. A lot in there, so let me see if I can unpack that. Let me start with the structure question first. So as I mentioned in our prepared comments earlier, we fully hired our internal and external teams, right? That's about just under 20 representatives, sales representatives, and then a small contingent of nurse educators that will collaborate with them. And that's for the U.S. team. For the EU, as we've shared previously, we've got boots on the ground there already, right? So we have an MSL team that's established, and then we have a small group of of experts that have been dedicated to building awareness and extending or advancing our patient profiling efforts in each of the major markets in the EU. And we continue to do the work, particularly on the critical path activities, to ensure that we will be able to launch a bit of PIVAT in the EU shortly after approval. But we've also shared that we're evaluating a number of options, right, considering the opportunity to be able to engage with a partner who's got a footprint that already exists in the EU and hopefully outside and beyond the EU, given the amount of value for Medipivet that extends, patient value that extends beyond the major markets, that can overlay Medipivet or include Medipivet in their existing infrastructure. And that's where we've been focusing our efforts. So the idea is, keep things moving to enable a launch, particularly in Germany, where you can launch immediately and have free pricing, but also be able to then transition that work to a potential partner once we find the sort of deal that we believe fairly reflects the value that a pivot will bring.
spk03: Great. Thank you. And then maybe if you could just comment on the the patient identification efforts and the patients identified today.
spk09: Yeah, yeah. So we continue to do the work behind what we refer to as patient profiling efforts, right, being able to identify more patients. We previously shared that we've identified about 1,000 patients between the U.S. and the and the EU, and that's inclusive of pediatric patients, patients that are enrolled in our various clinical programs, as well as patients, incidental patients that we come across as we're engaging with physicians. And it's an important number that I know everyone wants to hear more and more about, and we do keep on top of that. That's not where I would focus though, because particularly as we move closer to the launch here in the US, what you really need to do is be able to then attach each of those patients who are de-identified to us, right? So we don't know their personal, their identified information. But you want to be able to attach it to an individual physician, you want to ensure that they're getting tested, see if the physician is inclined to use an EMEID to confirm the diagnosis even further. And then we've also made our patient support program available in the last month And ideally, those physicians would then see value in referring their patients to the programs. And that helps you to get a better sense of the absolute confirmed patient population here in the U.S. So that's where we're focusing our efforts, right? Continuing to advance the diagnostic differential and get those patients the appropriate support that they need, get them the education that myogenes can provide which then will lead us up to and through the approval.
spk03: Thank you. That's very helpful. No problem.
spk05: Thank you. Our next question comes from Michael Schmidt with Guggenheim Securities. Your line is open.
spk04: Hey, this is Kelsey Goodwin on for Michael. Thanks for taking our question. Maybe actually building off of that one a bit, I guess going forward, how do you kind of see the rate and slope of the PKD diagnosis kind of evolving as you continue these efforts? I guess, what kind of assumptions do you kind of build in over the next, you know, near-term, long-term of this launch?
spk09: Yeah, the, you know, we have some indication from, you know, what we were observing with anemia ID, which is still in its early days, and some indication from the Spanish IST, screening IST, that reported about 20% of those hemolytic anemia patients that were screened that ultimately were diagnosed with PKD. You get a good sense for what your diagnosis rate may end up being amongst those patients who are tested, particularly those patients who are diagnosed with hemolytic anemia, but hemolytic anemia of unknown etiology. I'm not concerned about that in terms of the ramp for the launch, because I think we're doing everything that we need to do to facilitate testing. I expect we'll see that accelerate now that we have our full contingent in the field to help educate practices and our MyoGeo support to educate patients. The thing that's outstanding for me, and I think we're focused on better understanding, is what the access profile will look like, particularly in those first six to nine months of launch, where we know that for number of payers, you're going to have a new-to-market block that will require physicians seeking medical exception, and that will then extend the time between when you're diagnosed and when you're ultimately diagnosed. when treatment's ultimately made available to the patient. So the demand, and we have this confirmed in our recent quantitative work, the demand is gonna be there, so that you have the desire to be able to treat broadly for these patients. We've got all the programs in place, like an anemia ID, the work that we shared with you last time on 23andMe, to help increase overall awareness drive support patients in having better testing options available to them. So the opportunity for the patient to receive the treatment I think is going to be high. The question is the amount of time in those first months for the physicians to be able to work through the access challenges.
spk04: Got it. Okay, super helpful. Thank you so much. No problem.
spk05: Thank you. Our next question comes from Mark Breenbach with Oppenheimer. Your line is open.
spk10: Hey, good morning, guys. Congrats on the forward progress. And Chris, of course, we're sorry to see you go as well. You know, I'm just wondering, with regard to the pivotal trial in sickle cell disease, if you can comment on what the main gating items are that remain before this trial can be initiated. And in terms of the academic sickle cell trials with data that we might see later this year, can you maybe give us a sense for how many patients to expect from the UTREX trial and just remind us the main differences between this study and the NIH study? Thanks so much.
spk12: Okay. It's Chris here. launching any of the Phase 3 in thalassemia in the Phase 2-3 study, the global studies. So the protocols are written and finalized, and now we're in that heavy lift of operationalizing the studies. And that's a whole list of activities that involve regulatory agencies, sites, CROs, And in addition to the protocol, there's all the logistics that come with setting up your IVRS, that is your randomization systems, setting up all the translational medicine laboratory assays, how those things are going to flow. So it's a very complicated series of activities that are, some of them can run, many of them run in parallel. But in each individual country and within each individual country at individual sites, you're contracting and going through just a number of activities. So there are too many to name, but it's a well-described and well-acknowledged that it's a complicated series of activities that our team has done several times now successfully. And it takes some time to get everything up and running. The key piece that we're focused on in all of that is identifying sites that have patients throughout the world. These will be global studies. And to really look to everything we can do to make the trials accessible for patients and have maximum terminal velocity. So the first patient in is very important. And the last patient out is perhaps even more important. So that's that piece, and that's one of the fun things about doing this job. And then the second piece in terms of the sickle cell, the Utrecht study will enroll up to 10 patients. That group is very skilled and well-known in terms of their ability to look at both red cell metabolism, as well as using the oxygen scanner to look at point of sickling and other components that are important biomarkers in the sickle cell area. That trial starts patients at 20, and then they can go to 50 and then go up to 100 milligrams. And then they're on study for at least a year. So that's different from the NIH study, where the NIH study was the first study in adults with sickle cell disease where they started at 5, 20, 50, and then they made the amendment to 100. So it's a total of eight weeks of treatment. And then there's this taper. And then patients who are willing and able can then go into an extension. So I think there's a lot of similarities in terms of understanding the important endpoints that we've talked about. And you get a sense of efficacy and safety from both. The Utrecht study, I think, gives you immediately over time some further understanding of chronic dosing that the NIH study doesn't give you quite immediately because those patients are on eight weeks of therapy. And then we'll definitely get more long-term data from patients who go into the extension, but we don't know yet how many of those patients that's at the end of the day going to be. So I'll stop there.
spk10: Okay, super helpful. And just a very quick follow-up, is the Phase 2-3 going to be inclusive of African trial sites? Yes. All right, thank you.
spk05: Thank you. We have a question from Danielle Brill with Raymond James. Your line is open.
spk02: Hi, good morning. Thanks so much for the questions. I guess first, can you remind us what your expectations are for an adcom meeting, and maybe what your internal expectations are for priority review. And then, Darren, I wanted to clarify something from your prepared remarks. Excuse me. Did you say that your market research indicates 70% of providers already order genetic testing for anemias of unknown etiologies or that they would like to? Thank you.
spk09: Let me answer the second one, and then I'll leave the room for Chris to be able to answer you on the first. So the 70% number refers to the proportion of physicians who are routinely ordering more tests to try to be able to get to a more definitive diagnosis for those patients who are diagnosed with hematolytic anemia but have unknown etiology, which means that they're inclined to either order enzyme tests or a genetic panel or both. which some may do reflexively. And the reason why I mentioned I provided that insight is because then it means that we have a highly receptive audience then to anemia ID and gives us an indication of what further uptake will look like, particularly now that we've got more folks out there educating the community about it. So with that, let me turn it over to Chris.
spk12: Hi, your question was with regards to priority review for the private kinase deficiency submission, right?
spk02: Yes.
spk12: Yeah. So we'll find out the FDA's view on that, their decision, 60 days from when we file. So that should be sometime in the August timeframe, middle of August or so.
spk02: Okay. I guess...
spk12: Sorry, we lost you.
spk02: What are your thoughts on advisory committee meeting?
spk12: Oh, an ad comm. Too early to tell. Sometimes you get some signals early on, but we're early in the stage yet, so it's too early to know.
spk02: Understood. Thank you.
spk05: Thank you, and there are no other questions in the queue. I'd like to turn the call back to Jackie Faust for closing remarks.
spk07: Thank you, operator, and thank you, everyone, for the questions this morning. As we move into our very bright future as a transformed AGEOS, we look forward to making a meaningful difference in the lives of patients with genetically defined diseases, starting with our potential launch in PK deficiency next year. I would like to thank my OCHOS colleagues for their dedication and passion for making a difference for our patients. I also would like to thank all of the patients, caregivers, and physicians who partner with us in so many ways. And today, a special thanks to Chris. We will have more interactions with him, I'm sure, with you all over the next couple of months. We're very, very happy and pleased that he's willing to stay here engage with us, Agios, through the end of 2022, at least as a strategic advisor, because we'll continue to benefit from his wisdom and insights. So thank you very much, Chris, and we look forward to a few parties over the next couple of months to celebrate your next chapter. Thank you, everybody, for joining us today. You may now disconnect. Take care.
spk05: this concludes today's conference call thank you for participating you may now disconnect
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