Agios Pharmaceuticals Inc (AGIO) 2016 Q3 法說會逐字稿

Good morning and welcome to Agios' third-quarter 2016 conference call. (Operator Instructions) Please be advised that this call is being recorded at Agios' request.

I would now like to turn the call over to Kendra Adams, Senior Director Investor and Public Relations of Agios. Please go ahead.

Thank you, Charlotte. Good morning, everyone, and welcome to Agios' third-quarter 2016 conference call. You can listen to a live webcast with slides or a replay of today's call by going to the investors and media section of our website, agios.com.

With me on the call today with prepared remarks are Dr. David Schenkein, our Chief Executive Officer, who will review highlights for the third quarter; Dr. Chris Bowden, our Chief Medical Officer, who will discuss our clinical development activities; Andrew Hirsch, our Chief Financial Officer, who will summarize will Agios' third-quarter 2016 financial results. And Dr. Scott Biller, our Chief Scientific Officer, will also be joining for Q&A.

Before we begin, I would like to remind you that today's discussion will include statements about the Company's future expectations, plans, and prospects that constitute forward-looking statements for purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including those discussed in the risk factors section of our annual report on Form 10-K, which is on file with the SEC.

In addition, any forward-looking statements represent our views only as of today and should not be relied upon as representing our views as of any subsequent date. While we may elect to update these forward-looking statements at some point in the future, we specifically disclaim any obligation to do so, even if our views change.

With that, I'll turn the call over to David.

Thanks, Kendra. Good morning, everybody, and thanks for joining us today. The driving force behind the work we do here at Agios and what personally motivates me as a physician is the desire to create medicines with the potential to transform patients' lives.

This past quarter, we've made meaningful progress across the business that helps advance our groundbreaking science and begins to transition Agios from a pure R&D company to a global commercial stage company with worldwide rights to two late-stage medicines.

During the third quarter, we executed against our late-stage development goals for our IDH mutant inhibitors, advanced our PKR activator programs towards molecule selection, and strengthened our balance sheet through a recent financing.

In September, we and Celgene announced our plan to submit an NDA for enasidenib, formally AG-221, in relapsed-refractory AML by the end of 2016, just three years since we dosed our first patient. The NDA will be based on data from the ongoing Phase 1/2 trial in patients with advanced hematologic malignancies with an IDH2 mutation.

Transforming Agios from a blank piece of paper to our first NDA filing in seven years is a reflection of the quality of our research and the focus of our execution. We plan to follow a similar path for our second IDH inhibitor AG-120, which could result in an NDA submission in 2017. These are milestones that will enable us to make an extraordinary impact on patients' lives with our first approved medicines.

Our PKR activators, which showed impressive data earlier this year at EHA, have the potential to deliver the first disease-modifying therapy to patients with pyruvate kinase deficiency, a rare and debilitating anemia. These are two areas where we are leading the science and have the potential to deliver the first targeted medicines to patients.

Our development milestones, including AG-120 regulatory timeline, PKR molecule selection, and PKR pivotal trial design, are on track. And we expect to provide an update on these milestones in early 2017. In addition, we remain on track to initiate preclinical work for the MTAP program and we'll update you early next year.

Looking ahead to ASH, total of seven abstracts have been accepted and three have been selected for oral presentations. These abstracts, which we expect ASH to post online this morning, feature an array of clinical and early scientific data that demonstrates our continued leadership in the field of cellular metabolism and the potential for our PKR and IDH programs to have a meaningful impact on patients' lives.

I'm also excited to have our new Chief Financial Officer Andrew Hirsch on the call with us today. Andrew's 20 years of experience in a range of strategic and operating roles will be invaluable as we prepare to launch our first medicines and build a sustainable long-term global biopharmaceutical company. I'm looking forward to his contributions to the team as we build Agios for the future.

I'll now turn the call over to Chris to discuss our clinical activities.

Thanks, David. We have a robust data set at ASH this year across both our IDH and PKR portfolios that demonstrate the potential for these molecules to improve patient outcomes. Updated data from both our AG-120 and PKR lead programs highlight the advances we continue to make in the clinic, while new scientific findings keep us at the forefront of these fields.

Starting with our IDH1 inhibitor, our AG-120 abstract has been selected for an oral presentation. The dose escalation data in this abstract shows that AG-120 continues to demonstrate impressive single-agent activity and durable responses in a very sick patient population.

We will also be presenting molecular data for the first time measuring mutant IDH1 levels in a subset of patients treated with AG-120. Some patients with a complete remission also had mutational clearance where no detectable IDH1 mutant clone was found in their bone marrow or peripheral blood.

This suggests some patients on AG-120 are experiencing a deeper response at a molecular level, highlighting the profound activity of our drug. We will continue to push the science in this field and are excited to present these data, which will include a longer follow-up, in San Diego. As we have previously noted, data from the expansion cohorts will not be shown at this year's ASH.

Enasidenib data that Celgene submitted in myelodysplastic syndrome has also been accepted for an oral presentation at ASH. Agios and Celgene plan to support an investigator-initiated trial in MDS that is expected to begin in 2017.

We have a clear goal with our IDH inhibitors to one day provide a therapy for every patient who has an IDH mutation, regardless of their tumor type or stage of disease. The clinical activity, unique mechanism, and safety profile of our IDH inhibitors, combined with the convenience of once-a-day pill, gives our medicines the potential to change the treatment paradigm for AML patients with these mutations.

We are working hard to get our medicines to waiting patients by supporting Celgene's NDA submission for enasidenib. And are exploring a similar expedited regulatory strategy for AG-120, which could result in an NDA submission in 2017.

Moving our drug to the frontline setting is an important component of our development strategy and will enable us to help more patients earlier in their diagnosis. We have two trials underway combining enasidenib and AG-120 with both VIDAZA and standard of care intensive chemotherapy. We are also planning to begin a Phase 3 frontline study for AG-1200 in the first half of 2017.

For patients with IDH mutated solid tumors, we continue to explore the potential for our IDH inhibitors, where the use of a differentiation therapy is an opportunity help a large number of patients with difficult-to-treat diseases.

We are evaluating four expansion cohorts in different tumor types to build our findings from the AG-120 Phase 1 dose escalation. We'll have the first look at expansion cohort data in glioma patients at the Society for Neuro-oncology's Annual Meeting in Scottsdale, Arizona, later this month.

Low-grade glioma is a disease where patients have limited or no treatment options and are in need of new therapies. Drug development in this space will need to explore novel endpoints and imaging techniques. The data at SNO is the first safety and efficacy readout for AG-120 in defined cohorts of patients with low-grade glioma treated with a 500-milligram daily dose of AG-120.

Importantly, we are assessing tumor growth using MRI volumetric assessments. And this cohort is critical to establishing standard imaging protocols across collaborating institutions as we explore novel imaging approaches to assess drug activity.

In chondrosarcoma, we will present expansion cohort data at the Connective Tissue Oncology Society Annual Meeting in Lisbon next week. This cohort is now closed for enrollment and we are considering a variety of clinical development approaches from Company-sponsored studies to cooperative group collaborations.

In cholangiocarcinoma, we remain on track to initiate a randomized study by the end of the year. As they evolved over time, these data will inform future studies of AG-120 and AG-881, our brain-penetrant pan-IDH inhibitor.

Our development strategies for glioma and other solid tumors will be executed on a disease-by-disease basis, as data continues to emerge from our expansion cohorts and in discussion with investigators and regulatory authorities where appropriate.

Moving to our PKR activator program, our AG-348 Phase 2 study in patients with pyruvate kinase deficiency, also known as DRIVE PK, has also been accepted for an oral presentation at ASH. The first data we presented from this study at EHA in June demonstrated a profound sustained change in the hemoglobin levels of 9 out of 18 patients with pyruvate kinase deficiency. Data in the abstract continue to demonstrate robust activity and support pivotal development of our PKR activators in this rare anemia.

At ASH, we will present updated data with additional patients and longer follow-up and analysis of hemoglobin response by genotype. We will also be presenting updated Phase 1 data from the AG-519 healthy volunteer study. Data in the abstract continue to demonstrate a favorable safety profile and robust dose-dependent changes in ATP and 2,3-DPG levels consistent with PKR enzyme activation.

We are also excited to present the first metabolic data linking AG-348's impact on hemoglobin levels with activation of the PKR pathway in patients with pyruvate kinase deficiency. In order to increase our understanding of the underlying biology of pyruvate kinase deficiency, we conducted additional studies on the red blood cells of a small subset of DRIVE PK patients.

In this subset, patients who had hemoglobin level increases greater than 1 gram per deciliter on AG-348 experience a greater than 50% average increase in the rate of metabolism of the PKR pathway. While those with less than 1 gram per deciliter increase did not experience this change.

These preliminary findings are the first demonstration of a direct link between increased pathway function and increases in hemoglobin levels in patients with AG-348. Updated analyses, including additional patients, will be presented next month in San Diego.

Our work evaluating the role of iron overload is helping shape our understanding of the significant disease burden these patients face. Updated data covering this topic from the ongoing natural history study sponsored by Boston Children's Hospital will be updated at ASH.

Data available in the abstract continue to demonstrate that life-threatening iron accumulation into vital organs can be found in patients regardless of disease severity or transfusion status and is potentially associated with liver fibrosis, endochronological abnormalities, and cardiac events. These data also identify splenectomy as a potential cause of iron overload, highlighting the need for a therapy outside of this intervention.

We remain on track to select a PKR molecule based on the totality of data from both the AG-348 and AG-519 programs and finalize pivotal trial design by the end of this year and expect to provide an update in early 2017. Our goal is to provide a treatment for every patient with pyruvate kinase deficiency, and we are developing a global strategy aimed at the adult and pediatric populations.

The excitement around our molecules makes building the clinical organization here at Agios a real pleasure. As we continue to broaden and advance our programs, our team is also expanding to include medical science liaisons and other individuals who will be instrumental to delivering the potential benefit our medicines can bring to patients.

I'm now going to turn the call over to Andrew to review our financials.

Thanks, Chris. Before I dive into the financial results for the quarter, I want to take this opportunity to say how excited I am to join David and the team here at Agios at such an important time in the Company's development as we began to build out our commercial and global capabilities to bring our groundbreaking science to patients.

We are in a strong financial position today and are well capitalized as we continue to advance multiple late-stage clinical programs, maintain our investment in what has been an extremely productive research engine, and begin the buildout of our commercial capabilities to support anticipated product launches to achieve our vision of an independent, multiproduct, biopharmaceutical company.

We ended the third quarter with cash, cash equivalents, and marketable securities of $623 million compared to $376 million at the end of 2015. This increase in cash was driven by cash received from Celgene totaling $252 million, which includes the $200 million upfront payment from the May 2016 collaboration agreement, a $25 million milestone from the initiation of enasidenib Phase 3 IDHENTIFY study, and $27 million of program funding related to our collaboration agreements.

The September 30 cash balance also includes net proceeds of $162 million from our September follow-on offering, in which we sold approximately 3.9 million shares of common stock at $44.50 per share. These cash inflows were offset by cash expenses to fund operating and capital expenditures of approximately $166 million during the first 9 months of the year.

As a result, we now expect to end the year with more than $550 million in cash, cash equivalents, and marketable securities. Based on our current operating plans, we expect that this amount, together with the anticipated expense reimbursements under our collaboration agreements with Celgene, will enable us to fund our operating expenses and capital requirements through at least the end of 2018.

Turning now to the third-quarter P&L, we recognized $9 million of collaboration revenue for the third quarter of 2016 compared to $7 million last quarter and $5 million for the third quarter last year. Research and development expenses were $61 million compared to $51 million for the second quarter of 2016 and $36 million for the third quarter of 2015.

The increase in year-over-year R&D expense for the quarter was primarily due to increased enrollment of our clinical trials with AG-120 and AG-519 as well as increased manufacturing activities for both programs. The increase in R&D expense compared to Q2 of this year was primarily driven by the increased manufacturing activity during the quarter.

Furthermore, the increases in both year-over-year and quarter-over-quarter R&D expense were also driven by the modification of the Celgene agreement. If you recall, as of August 15 of this year, we are responsible for 100% of the development costs for AG-120. So halfway through Q3, we picked up 100% of these costs.

It's also important to note that the third-quarter 2016 Celgene reimbursement for AG-120 expenses through August 15 is now primarily recognized in the revenue line as compared to a contraexpense in previous periods. This detail will be outlined in the 10-Q that we plan to file after market today.

General and administrative expenses were $12 million for the third quarter compared to $13 million last quarter and $10 million for the third quarter last year. The year-over-year increase was largely due to increased headcount and other professional expenses to support our growing global operations.

I will now turn it back to David to wrap up.

Thanks, Andrew. I'm incredibly proud of our progress to date in 2016. As I mentioned earlier in the call, we made important progress this quarter across the business as we move beyond the clinic to become a global commercial-stage company.

The data we expect to present over the next month represent the foundation on which our future plans for our IDH and PKR programs will be developed. And we look forward to updating you on those plans early next year.

For the remainder of the year and into 2017, we are focused on continuing to execute to get our medicines to patients we know are waiting. Keeping the patient at the center of everything we do is our driving focus and ultimately creates value for everyone in the Agios community.

I'd like to thank all the people at Agios who made these accomplishments possible. Also a big thank you to all the patients, doctors, nurses, and caregivers who have participated in our clinical trials. Thank you for all your continued support and we look forward to seeing many of you at ASH.

And with that, operator, we'll turn it over to you for questions.

(Operator Instructions) Eric Schmidt, Cowen and Company.

Half of me wants to run out the clock until 9 o'clock and ask you about your ASH abstracts, but I guess you won't permit that. Either Chris or David, it sounds like on the PKR activator program that you're going to make the decision on which compound to move forward. And also decide on the protocol for Phase 3, but you're not going to communicate that decision before year end. That will slip into next year? Is that right?

Eric, this is David. Yes, we can't keep you on the line until nine. Sorry. So thanks for your question. So we are on track, as Chris mentioned and as I mentioned as well, to get all the activities done for both the PKR program, and I would also mention for the AG-120, to make some of the really important milestones decisions that we communicated earlier, including the regulatory strategy, timing for 120, and molecule selection pivotal trial design.

But we think given that some of the work we still need to get done, including some regulatory meetings, will occur later in December, that is most appropriate to do that in a venue very early in the year. And we'll communicate that, obviously, to you.

So there's no delay. Everything remains on track for those important decisions for us internally by the end of the year, and we'll communicate them very early in the year in 2017.

And are the late December FDA meetings for both programs, David?

I think it's fair to say that we have a series of regulatory meetings throughout December that will impact both those programs.

Okay. And then just a quick one on 881. We haven't heard much about the compound. Are things still on track?

I'm going to let Chris answer that one.

Hi, Eric. Chris Bowden here. Things are going well in terms of accrual on the Phase 1 trials for 881 in both heme and solid tumors and glioma. And as per usual, when we have that critical mass of data, it allows us to tell a story. We look forward to getting that data in the public domain.

Okay. And very last one. Is the NDA on Celgene's part on 221 associated with a milestone payment?

It is not associated with a milestone payment. And Andrew can articulate a little bit more.

Yes. So all the regulatory and approval milestones for 221 are actually based on X-US activities.

Thank you.

Anupam Rama, JPMorgan.

Thanks so much for taking the question. Looking to next week, help us understand what we should be looking for here in the AG-120 chondrosarcoma data that's going to be presented. Maybe you could outline the scope of data that we'll be getting here next week. Thanks so much.

Thanks, Anupam, for your question. This is David. I'm going to turn it over to Chris.

So that data comes from patients with chondrosarcoma who were treated on in the dose escalation as well as the dose expansion component of the AG-120 study. And so that data will get into the safety and efficacy in that cohort of patients. Think it will certainly give people a sense of how the drug behaves from a safety perspective. And it will be the first efficacy data that we get out there.

And then I think we've been in discussions with the investigators working with us on the trial as well as discussing internally what the next steps are. And as I pointed out in my remarks, there's a range of options that we can pursue. And what we ultimately decide to do will depend on further discussions, both internally and with investigators as well.

Great. Thanks so much for taking our question.

Kennen MacKay, Credit Suisse.

Thanks for taking my questions. Maybe one for David. I was wondering if you could just sort of walk us through the division not to present the updated 221 data ahead of the filing here.

Thanks for your question. We communicated this, I think, in the second quarter when we talked a little bit about this. While there's a difference in practice in different companies, I think Celgene and Agios are pretty aligned that in general, we prefer not to present public -- into the public domain data that we know is going to be sitting in a regulatory file until we've had the opportunity to really share that with the regulators and have them had an opportunity to review it and hopefully accept those files. That's general practice.

I mean, sometimes you will break that rule. But in this case, we felt pretty strongly, as did Celgene, that it made sense to keep that data internal until it's been vetted and reviewed by the FDA.

And David, also on the IDH inhibitors, you'd always mentioned sort of the focus isn't necessarily just on the CR rate or even the ORR or duration of therapy. And these agents have a very clean safety profile and enable the patients to have a recovery in their blood counts, which could stave off infections, which could be linked to mortality.

Should we sort of be thinking that there could be enough data to maybe show some kind of survival benefit in this population?

Yes, Kennen. So obviously as we've said before, and Chris and I have both said, that it's really going to be the totality of the data. And that's what makes us so excited by the data that we've generated to date. It's the entire profile of the drug.

It's a -- as you said, knowing the right patients is the first really truly targeted therapy, certainly the first targeted against IDH that will be at this stage. It's the safety profile; it's that profound efficacy, the CRs, but everything else we're seeing as well. And we think that will potentially provide real clinical benefit for patients.

Obviously, we'll be looking at survival data for both enasidenib and for AG-120. Remember, there's no control arm in those studies, but we do have very good historical controls. So again, we continue to remain incredibly excited by the data we've seen and look forward to sharing that obviously with the regulators very shortly and then with the rest of you.

Got you. And then maybe just a final question on upcoming catalysts. I was wondering if you could give us any guidance as to when we might see combination data from the combo trials with 7+3 in HMAs and VIDAZA?

Chris?

Kennen, it's Chris here. Those trials are ongoing. And as per our usual policy is that when we've got an adequate data set for these trials, which are really about feasibility, safety, and tolerability, we will publish those data and provide some guidance as to when that will be. We're not able to do that at this time, but the trials are accruing well.

Fair enough. Thanks guys. Appreciate it.

[Cavin Dahl], Needham & Company.

Thanks for taking my call. So a couple of my questions have already been answered. But I was wondering if you could expand again on the molecular data that's being presented on AG-120. You talked about the immune IDH1 levels. So just wondering if you could expand on that a little bit more?

Well, before I turn it over to Chris, thanks for your question. This is David. We can't expand too much. Remember, we can just maybe clarify Chris's comments. The abstracts will be out later this morning and then obviously there'll be a lot more data at the meeting. So Chris?

As we've been doing in our AML trials is that we make collection of tissue. It's a really important component of what we are doing in order to try to gain a greater understanding of what's happening at the molecular level. Last year's ASH, we presented some data looking at co-occurring mutations in patients that were treated on our trials.

And this [miayapp] data that's going to be presented at ASH this year is part of that overall molecular -- that series of molecular investigations that we're looking at. And what we've been doing is looking at what happens to the IDH1 mutant clone in patients with AML who are treated on our trials as they are treated over time.

And what we've demonstrated is that some patients have -- with a complete remission also have mutational clearance. And so we are looking forward at San Diego to providing some further details around that, including the types of assays that we are conducting, more quantitative data around that, what that profile looks like over time, and those types of issues.

Okay, [thanks].

This is David. I'll just add that I think as Chris has already mentioned. But just to remind you what I think makes this so important data is remember, this with a single agent in the relapsed-refractory setting. Go ahead with your next question?

Just a quick follow-up on that, and then I do have one other quick question. Is it safe to assume that these type of studies are also being applied for 221 and 881 as well to understand how things are changing upon treatment?

Yes, we look at these types of -- we conduct these types of investigations in our IDH inhibitor program in patients with AML.

Okay. And so then the last question. You talked about MRI imaging technologies that you are developing for AG-120 in gliomas. Are these type of technologies going to be applied to 881, where Celgene has some partial control over those studies?

Yes. So the MRI volumetric methodology that I was talking about and that we've been looking at in our studies is really important radiographic techniques for us to try to get as deep an understanding as we can of treatment-effective AG-120 and potentially AG-881 in patients with glioma.

So a lot -- a really important component of what we are doing in the AG-120 expansion cohort in patients with glioma is to really build those foundations, get all of the centers aligned in terms of how we're going to conduct these techniques to try to reduce institutional variations in how they assess MRI scans, how they actually acquire the MRI scans.

Okay, great. Thanks. That's all the questions I had.

Yatin Suneja, SunTrust.

Congrats on all the progress, and thank you for taking my question. Just a couple questions on the PKD. Could you maybe give us an update on how many additional healthy volunteers you have treated with 519? And any updated comment on the safety profile, specifically with regard to the thrombocytopenia. And then I have a follow-up.

So before I turn it over to Chris, Yatin, thanks for your question. We don't give out those kind of details. I mean, the abstracts will be out, so there will be some information there. But let me turn it over to Chris.

So the trial continues and we're putting healthy volunteers onto the study. It's a really important part of this aspect of understanding which molecule we're going to select so that we have the totality of data around both AG-348 and AG-519 to guide us into a molecule selection decision by the end of the year.

Okay. Just a question on the pivotal programs. So recently, we saw FDA agreeing to a fairly unique two-stage pivotal trial in sickle cell disease space, where the first part is trying to figure out the dose. And then you evaluate the dose in the second part.

I mean, obviously, you can't comment on the pivotal program that you're going to run with your PKD molecules, but could you adopt a similar trial design for -- let's say you move 519 ahead, since you don't know the dose in patients?

We're working really hard on putting our pivotal trial design together internally. And we are discussing that with regulators. And I'm not able to disclose details around what that trial looks like today.

I would point out and acknowledge it, as you pointed out, that sickle cell is a different disease from pyruvate kinase deficiency. And so the types of things that we've demonstrated with dry PK -- robust increases in hemoglobin. We've discussed what we think is a clinically meaningful increase in hemoglobin that's somewhere between 1 to 2 grams based on transfusions, outcomes with transfusions, splenectomy, et cetera.

Those are the things that we are incorporating into our thinking around our pivotal trial and what we are discussing with regulators as well as investigators and experts who treat patients with this disease.

Got it. Thank you.

Terence Flynn, Goldman Sachs.

Thanks for taking the question. Maybe just a couple on PKD for me as well. Can you just remind us if there's any correlation between genotype and transfusion status? And then, if not, why not.

And then the second question at this point, just if you're confident will be able to include transfusion patients in the pivotal trial program. Or is there a possibility that you could have additional work you need to do. Thanks.

Thanks, Terence. David here. Thanks for your questions. Chris?

So with regards to the second part of your question, we are not able to disclose yet -- we're not providing guidance on what that patient population looks like. And specifically transfusion dependent or regularly transfused in or out.

The second piece is the -- I'll approach it from the perspective of can one determine disease severity on the basis of genotype. And the published data to date hasn't been too clear on that. And in fact, when you see patients with identical genotypes, they have varying severities of disease.

So why that is, your question, we don't know. And it's certainly something that we're hoping that we'll be able to find out or learn more about as we continue to study this disease, both with our drugs as well as through the natural history study.

Terence, David here. Let me just add, and as you know, we've talked about this. Our commitment in pyruvate kinase deficiency is to develop our molecule through all patient populations and all age groups. That's the goal here.

Great. Thanks a lot, guys.

Michael Schmidt, Leerink Partners.

Thanks for taking my questions. I had another one on the PKD program. And I know you've presented data looking at correlation with hemoglobin increases with genotype. But I was wondering if there was a dependency on baseline hemoglobin levels in your response to the drug. And in this context, how you think about the opportunity in beta thalassemia?

Michael, thanks for your question. David here. So we've talked about this in the past that it's just too -- the patient numbers from our first presentation at EHA was limited to 18 patients. And so it was just a little bit too early to draw any strong conclusions.

As Chris just articulated to the last question, we think probably genotype is probably the most likely predictor of response to a PKR activator as opposed to baseline hemoglobin. Because baseline hemoglobin is affected by many different factors, including whether or not the patients had their spleen removed when they were a child, how often they are getting transfused, et cetera.

So I just think we are focused right now on genotype more than anything else. And again, as I mentioned earlier, our commitment is to create a medicine that will help all of these patients.

And second part of your question was around beta thal. Very different disease, obviously. We presented, as you know, some preclinical data and we are hoping to be able to update you by the end of this year or early in the year in terms of what our plans are in other hemolytic anemias.

Okay, great. Thanks. And maybe one more on the MTAP program. I know it's still early, but how do you think about the opportunity there, more broadly speaking? Are you initially focusing on one drug, one target there, or multiple? And are there certain tumor types that could be the low-hanging fruit for those agents initially. Thanks.

Yes, Michael, thanks for your question. Again, it's a very exciting program for us. We haven't given out a lot of details.

But Scott Biller, our Chief Scientific Officer, is with us this morning and let me let him give you a little bit more on that.

Yes, happy to comment. This is Scott. As you know, this deletion is present across 15% of all human tumors. And it's prevalent to a high percentage in some tumors, and in other tumors, a small percentage. But they have happened to be very big populations. So think about ALK translocations and lung cancer.

So we're not guiding as to what our clinical strategy would be; we're focusing on meeting our milestone of getting a molecule into preclinical development. But we are also focused on the MTAP-deleted vulnerabilities as a pathway. There's multiple targets there. We think there's lots of opportunity and are very excited to bring our molecule forward.

Great. Thank you.

Kennen MacKay, Credit Suisse.

Thanks for taking the follow-up. I just had an additional question on the drug-induced thrombocytopenia that we've seen with 519. And wondering if there was any additional data you could present to sort of confirm that mechanism. And then on a population basis, if there was anything else you could do to sort of confirm how rare this would be expected to be in a broader population?

Kennen, it's Chris here. If you look at some pretty extensive literature on drug-induced immune thrombocytopenia, there's several different mechanisms that are presented for why this occurs. And sorting all that out can be challenging.

I think from our perspective, the way we look at this is that it's an effect that we expect to be rare based on what you see with other drugs where this has been reported on. In this patient population of individuals with pyruvate kinase deficiency, they have their blood monitored on a regular basis. So this is something that can be monitored and addressed if it were to occur.

Overall, in terms of doing population-based studies to try to get a better handle on what the frequency is, we've looked at a lot of that literature that's published now on a large number of drugs. And it's consistent with the fact that this occurs rarely.

And for the most part, when you stop drug, it goes away. And I think we're comfortable with those things at this point. And what our goals are in our forward-looking clinical trials is to monitor carefully and have good directions in place if in fact this were to be observed again.

Thanks, Chris. That super helpful. And I guess from our perspective as analysts is that aside from looking at correlates with other drugs that are out there, is there anything else we should sort of be focusing on in terms of gaining comfort around this as a rare event? Or is it just going to be sort of seeing additional patients and not seeing this occur again?

I think from our perspective, I think you have the literature experience with a plethora of drugs that have been developed and are on the market. And yes, I think we'll -- it's something that we'll need to continue to monitor for. And as we continue to expand the programs.

Okay. Thanks, Chris. I really appreciate it.

Thank you. And I would now like to turn the call back over to David Schenkein for closing remarks.

I'd like to thank everybody for participating on the call today. Again, thank all the patients and caregivers and nurses who participate in our clinical trials. And all the Agios employees for all of your tremendous work and all of you for your support. Enjoy the rest of your day and thanks again.

Ladies and gentlemen, thank you for participating in today's conference. This does conclude the program and you may all disconnect. Everyone, have a great day.