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Agios Pharmaceuticals, inc (NASDAQ:AGIO)
Q2 2021 Earnings Call
Jul 29, 2021, 8:00 a.m. ET

Contents:

  • Prepared Remarks
  • Questions and Answers
  • Call Participants

Prepared Remarks:

Operator

Good morning and welcome to Agios Second Quarter 2021 Conference Call. [Operator Instructions] There will be a question-and-answer session at the end. [Operator Instructions]

I would now like to turn the call over to Jessica Rennekamp, Director of Corporate Communications.

Jessica Rennekamp -- Director of Corporate Communications

Thank you, operator. Good morning, everyone, and welcome to Agios second quarter 2021 conference call. You can access Slides for today's call by going to the investors section of our website agios.com.

With me on the call today with prepared remarks are Dr. Jackie Fouse, our Chief Executive Officer; Dr. Chris Bowden, our Chief Medical Officer; Darrin Miles, our Chief Commercial Officer; Jonathan Biller, our Chief Financial Officer and Head of Legal and Corporate Affairs; and Dr. Bruce Car, 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.

Jacqualyn Fouse -- Chief Executive Officer

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 mitapivat across its three initial indications in hemolytic anemias.

We achieved significant recent milestones with the submission of our NDA for mitapivat 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 3 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 mitapivat in PK deficiency continued 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 US-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 in advocating for those living with this disease.

Beyond PK deficiency, we are also making excellent progress with the late-stage development of mitapivat in thalassemia and sickle cell disease. At EHA, we presented positive results from our Phase 2 open label study of mitapivat 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 3 trials, ENERGIZE and ENERGIZE-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 2/3 trial on mitapivat by the end of the year. Beyond mitapivat, we are building on our expertise in PK activation and cellular metabolism and are advancing our earlier stage research programs in genetically redefined 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 mitapivat in 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 Agios 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 has 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 Gheuens, 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.

Chris Bowden -- Chief Medical Officer

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 filing for mitapivat in the US and EU.

We are 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 mitapivat 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 3 studies evaluating mitapivat 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 address symptom burden and quality of life impact of PK deficiency. Mitapivat 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 Glenthoj, who highlighted the significant impact of PK deficiency has on patient's quality of life, the limitations of the current PK deficiency treatment landscape, and the potential for mitapivat 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 2 study of mitapivat 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 1 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 mitapivat. Mitapivat was also well tolerated with a safety profile consistent with previous studies. We believe these results underscore the potential of mitapivat 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 trial of mitapivat in 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 mitapivat in sickle cell disease will include patients who are 16-years of age or older, have had between two to 10 sickle cell crisis in the past 12-months, and have hemoglobin within the range of 5.5 to 10.5 grams per deciliter during the screening. The Phase 2 will randomize 69 patients one to one to one to 50 milligrams mitapivat twice daily, 100 milligrams mitapivat twice daily, or match placebo. The primary endpoint in the hemoglobin response defined as greater than or equal to 1 gram per deciliter change from baseline to week 12 and the data will be used to establish a clear dosing paradigm for the Phase 3 portion.

Phase 3, which will commence after the Phase 2 analysis, will randomize 198 patients two to one with the selected Phase 2 dose of mitapivat or matched placebo. The study will have two primary endpoints, hemoglobin response defined as greater than or equal to 1 gram per deciliter change from baseline to week 52 and a reduction in annualized rate of sickle cell pain crisis. We believe the design of this Phase 2/3 study minimizes risks to the approval path for mitapivat 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 Food Drug on their studies of mitapivat in sickle cell disease. Data from both studies are expected to be submitted for presentation at ASH in December. At the NIH, Dr. Chan 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 mitapivat in people with sickle cell disease.

Beyond mitapivat, we're also advancing our next generation PKR activator AG946 in a Phase 1 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 Darrin to discuss our commercial activity, 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 Gheuens, is poised to lead our team to more great accomplishments and I look forward to watching the Agios story continue to unfold. Darrin?

Darrin Miles -- Chief Commercial Officer

Thank you, Chris. We continue to make strong progress on commercial launch preparations for mitapivat in PK deficiency. Our 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 engage with physicians potentially managing patients with PK deficiency across the US 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 Agios sponsored genetic testing service Anemia ID with now well over 1,000 test kits requested. We expect Anemia ID to become an increasingly important program to help support patients seeking a definitive diagnosis of their hemolytic anemia and in aid and accelerating our PK Deficiency patient profiling efforts. Our market research now shows that 70% of responding physicians routinely order additional a diagnostic test for their patients with hemolytic anemias of unknown etiology. This important insight which favorably portends 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 My Agios 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 My Agios program, while incorporating insights from PK deficiency patients and clinicians to 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 the service and how they can support patients with enrollment. 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 mitapivat'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 3 data, many physicians will consider prescribing mitapivat 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 mitapivat, 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 of 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 grows related to the ACTIVATE disclosures.

Now, I will turn it over to Jonathan to review second quarter financials.

Jonathan Biller -- Chief Financial Officer and Head of Legal and Corporate Affairs

Thanks, Darrin. 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 start-up costs associated with the Phase 3 studies mitapivat in thalassemia and sickle cell disease and the filing of our NDA and MAA for mitapivat in 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 TIBSOVO 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 10b5-1 plan we put in place in early Q2 provides for maximum additional share repurchases of approximately $415 million at 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.

Questions and Answers:

Operator

Thank you. [Operator Instructions] Our first question comes from Alethia Young with Cantor Fitzgerald. Your line is open.

Alethia Young -- Cantor Fitzgerald -- Analyst

Hey guys. Thanks for taking my question and congrats on all the progress. I want to talk a little bit about mitapivat and maybe just as you're doing some of the education for patient support. 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.

Darrin Miles -- Chief Commercial Officer

Hey Alethia, this is Darrin. Yes, the issue is not going to be necessarily getting patients to come back in because they're usually under -- for those who've been diagnosed and obviously attached to a physician, they are under some, sort of, monitoring plan, right? Those with more severe disease are going to see their physicians more regularly and those with less severe disease will have a longer -- typically have a longer time span between physician visits. But remember, they're continuing to have the iron level monitored, hemoglobin monitored.

So, the frequency of physician visits isn't necessarily where we want to -- 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 should be fine and we don't need to -- 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 follows from there.

Alethia Young -- Cantor Fitzgerald -- Analyst

Great. Thank you.

Operator

Thank you. Our next question comes from Kennen MacKay with RBC Capital Markets. Your line is open.

Kennen MacKay -- RBC Capital Markets -- Analyst

Hi. Thanks for taking the question. First off Chris, I'm sorry to hear about your departure, but looking forward to meet 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 for mitapivat. Wondering, which branch of the FDA you are interacting with, with those regulatory submissions in PKD. Is that hematology or the rare disease division?

Chris Bowden -- Chief Medical Officer

Hey Kennen, it's Chris. The FDA team we're working with are in the cardiology, renal and the oncology, hematology divisions. The submission itself is in the cardiology, renal. So, it's an interesting setup, because the people who have been most involved with us and are most active on the submission are in the hematology division.

Kennen MacKay -- RBC Capital Markets -- Analyst

That is just what I was going to sort of follow-up with and just wondered around some of the interactions so far. You've got division sort of up to speed on rare diseases and PKD or what have the interactions to-date been like? Thank you.

Chris Bowden -- Chief Medical Officer

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 are 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 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.

Kennen MacKay -- RBC Capital Markets -- Analyst

Thank you.

Operator

Thank you. Our next question comes from Anupam Rama with JPMorgan. Your line is open.

Anupam Rama -- JPMorgan -- Analyst

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 for mitapivat longer term to account for the SCD indication. What threshold of peak sales in SCD does mitapivat 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.

Darrin Miles -- Chief Commercial Officer

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 wanted to at least breakeven, right? As you move from the first two indications and into sickle cell then you account for cut. What we think would be required would only require less than 4%, 3% share in that market to be able to at least breakeven 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. That's why we're so optimistic about the incremental value that we will be able to gain once we move into sickle cell despite competition.

Jacqualyn Fouse -- Chief Executive Officer

And it's Jackie, I'm just going to jump in real quick. As we said many times, I think both Chris and Darrin 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 is 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.

Anupam Rama -- JPMorgan -- Analyst

Thanks so much for taking our questions.

Operator

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

Elizabeth Webster -- Goldman Sachs -- Analyst

Hey, good morning and thank you for taking our question. This is Elizabeth on for Salveen. Could you just remind us if you intend to launch simultaneously in both the US and EU for mitapivat in PKD? And then 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 and your progress in terms of identifying more patients? And then how many patients with PKD have been identified to-date? Thank you.

Darrin Miles -- Chief Commercial Officer

Got you. Alright Salveen, this is Darrin. There's 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 my prepared comments earlier, we fully hired our internal and external teams and 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 US team.

For the EU, as we've shared previously, we got boots on the ground there already. So, we have an MSL team that's established and then we have a small group 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 mitapivat 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's already -- that already exists in the EU and hopefully outside or beyond the EU given the amount of value for mitapivat that extends -- patient value that extends beyond the major markets that can overlay mitapivat or include mitapivat in the 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 repricing. And then -- but also be able to then transition network to a potential partner once we find the sort of deal that we believe fairly reflects the value that mitapivat will bring.

Elizabeth Webster -- Goldman Sachs -- Analyst

Great. Thank you. And then maybe if you could just comment on the patient identification efforts and patients identified today?

Darrin Miles -- Chief Commercial Officer

Yes. So, we continue to do the work behind what we referred to as patient profiling efforts, being able to identify more patients. We previously shared that we've identified about 1,000 patients between the US and the EU and that's inclusive of pediatric patients, patients that are enrolled in our various clinical programs, as well as patients -- selective 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, but -- 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, we really need to do is be able to then attach each of those physicians -- patients already identified to us.

So we don't know their personal -- their identified information, but you want to be attached to an individual physician, you want to ensure that they're getting tested, see if the physician is inclined to use Anemia ID 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 US. 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 they can -- that My Agios can provide, which then will lead us up to and through the approval.

Elizabeth Webster -- Goldman Sachs -- Analyst

Thank you. That's very helpful.

Darrin Miles -- Chief Commercial Officer

No problem.

Operator

Thank you. Our next question comes from Michael Schmidt with Guggenheim Securities. Your line is open.

Kelsey Goodwin -- Guggenheim Securities -- Analyst

Hey, this is Kelsey Goodwin on for Michael. Thanks for taking our question. Maybe actually building off of that a bit, I guess going forward how do you kind of see the rate and slope of the PKD diagnoses, kind of, evolving as you continue these efforts? I guess what kind of assumptions do you kind of build in over the next near-term, long-term of this launch?

Darrin Miles -- Chief Commercial Officer

We have some indication from what we're observing with Anemia ID, which is still in its early days and some indication from the Spanish IST -- screening IST that reported about -- I think about 20% of those hemolytic anemia patients that were screened ultimately were diagnosed with PKD. We get a good sense for what your diagnosis rate may end up being among those patients who are tested, particularly those patients, who have hemolytic anemia, but hemolytic anemia of unknown etiology. That I'm less -- I'm not concerned about that in terms of the ramp for the launch, because I think we continue to -- we're doing everything that we need to do to facilitate testing. I expect we'll see that accelerate now that we have a full contingent in the field to help educate practices and our Agios 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, right? Where we know that for a number of payers, you're going to have a new to market block that will require physician seeking medical exception and that will then extend the time between when you're diagnosed and when you're ultimately -- when treatment is ultimately made available to the patient.

So, the demand -- and we have this confirmed in our recent quantitative work. The demand is going to be there so we have the desire to be able to treat broadly for these patients. We've got all the programs in place like Anemia ID, the work that we shared with you last time on 23 & Me to help increase the awareness drive, support patients in having better testing options available to them. And then so the script -- the opportunity for the patients to receive the treatment I think it's going to be high. The question is the amount of time in those first months for the physicians to be put to work through the access challenges.

Kelsey Goodwin -- Guggenheim Securities -- Analyst

Got it. Okay. Super helpful. Thank you so much.

Darrin Miles -- Chief Commercial Officer

No problem.

Operator

Thank you. Our next question comes from Mark Breidenbach with Oppenheimer. Your line is open.

Mark Breidenbach -- Oppenheimer -- Analyst

Hey, good morning guys. Congrats on the forward progress and Chris, of course we're sorry to see you go as well. 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 ACTIVATE 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 Utrecht trial and just remind us the main differences between this study and the NIH study? Thanks so much.

Chris Bowden -- Chief Medical Officer

Okay. It's Chris here. Launching any of the Phase III in thalassemia and the Phase II/III study -- the global studies, 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 of 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 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 are focused on in all of that is identifying sites that have patients throughout the world including global studies and to really look to everything we can do and 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.

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 plain sickling and other components that are important biomarkers in the sickle cell area. That trial starts patients at 20 milligrams and then they can go to 50 milligrams and go to 100 milligrams and then they are on study for at least a year.

So, that's different from the NIH study where that was -- the NIH study with the first study in adults with sickle cell disease where they started at 5 milligrams, 20 milligrams, 50 milligrams and then they may the amendment to 100 milligrams. 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 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.

Mark Breidenbach -- Oppenheimer -- Analyst

Okay. Super helpful. And then just a very quick follow-up. Is the Phase 2/3 going to be inclusive of African trial sites?

Chris Bowden -- Chief Medical Officer

Yes.

Mark Breidenbach -- Oppenheimer -- Analyst

All right, thank you.

Operator

Thank you. We have a question from Danielle Brill with Raymond James. Your line is open.

Danielle Brill -- Raymond James -- Analyst

Hi, good morning. Thanks so much for the questions. I guess first can you remind us where your expectations are for an AdCom meeting and maybe what your internal expectations are for priority review? And then Darrin, I wanted to clarify something from your prepared remarks. Did you say that your market research indicates 70% of providers already ordered genetic testing for anemias of unknown etiologies or that they would like to? Thank you.

Darrin Miles -- Chief Commercial Officer

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 hemolytic anemia but of unknown etiology, which means that they are inclined to either order enzyme tests or a genetic panel or both, which some they do reflexively. And the reason why I mentioned -- I provided that insight is because then it means then we have a highly receptive audience then to Anemia ID and give us an indication of what further uptake would 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.

Chris Bowden -- Chief Medical Officer

Hi. Your question was with regards to priority review for the pyruvate kinase deficiency submission, right?

Danielle Brill -- Raymond James -- Analyst

Yes.

Chris Bowden -- Chief Medical Officer

Yes. So, we'll find out the FDA's view on that and their decision 60 days from when we filed so that should be sometime in the August timeframe, middle of August or so.

Danielle Brill -- Raymond James -- Analyst

Okay. I guess [Technical Issues]

Chris Bowden -- Chief Medical Officer

Sorry, we lost you.

Danielle Brill -- Raymond James -- Analyst

What are your thoughts on Advisory Committee meeting?

Chris Bowden -- Chief Medical Officer

An AdCom, too early to tell. You -- sometimes you get some signals early on, but we're early in the stage yet so it's too early to know.

Danielle Brill -- Raymond James -- Analyst

Understood. Thank you.

Operator

Thank you. And there are no other questions in the queue. I'd like to turn the call back to Jackie Fouse for closing remarks.

Jacqualyn Fouse -- Chief Executive Officer

Thank you, operator, and thank you, everyone, for the questions this morning. As we move into our very bright future as a transformed Agios, 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 Agios colleagues for their dedication and passion for making a difference for our patients. I also would like to thank all 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 is willing to stay engaged with us Agios through the end of 2022 at least as a strategic advisor because we'll continue to benefit from his wisdom and insight. So, thank you very much, Chris, and we look forward to a few parties over the next couple of months for your next chapter. Thank you everybody for joining us today. You may now disconnect. Take care.

Operator

[Operator Closing Remarks]

Duration: 43 minutes

Call participants:

Jessica Rennekamp -- Director of Corporate Communications

Jacqualyn Fouse -- Chief Executive Officer

Chris Bowden -- Chief Medical Officer

Darrin Miles -- Chief Commercial Officer

Jonathan Biller -- Chief Financial Officer and Head of Legal and Corporate Affairs

Alethia Young -- Cantor Fitzgerald -- Analyst

Kennen MacKay -- RBC Capital Markets -- Analyst

Anupam Rama -- JPMorgan -- Analyst

Elizabeth Webster -- Goldman Sachs -- Analyst

Kelsey Goodwin -- Guggenheim Securities -- Analyst

Mark Breidenbach -- Oppenheimer -- Analyst

Danielle Brill -- Raymond James -- Analyst

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