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Forty Seven, Inc. (FTSV) Q2 Earnings Call Transcript

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Forty Seven, Inc. (NASDAQ: FTSV)
Q2 2019 Earnings Call
Aug. 13, 2019, 4:30 p.m. ET

Contents:

  • Prepared Remarks
  • Questions and Answers
  • Call Participants

Prepared Remarks:

Operator

Good afternoon and welcome to the Forty Seven Second Quarter 2019 Financial Results Conference Call. At this time, all participants are in a listen-only mode. This call is being webcast live on the Investors section of Forty Seven's website at www.fortyseveninc.com. Please be advised that this call is being recorded. Following the formal remarks, we will open up the call for your questions.

At this time, I would like to turn the call over to Michael Horowicz with Stern Investor Relations.

Michael Horowicz-- Stern Investor Relations

Thank you. This afternoon, Forty Seven issued a press release detailing its second quarter 2019 financial results along with anticipated future milestones and recent accomplishments. This release is available on the Investors section of Forty Seven's website at www.fortyseveninc.com.

Today's call will being with prepared remarks by Dr. Mark McCamish, Chief Executive Officer of Forty Seven; Dr. Chris Takimoto, Chief Medical Officer; and Ann Rhoads, Chief Financial Officer of Forty Seven. Then we will open the call up for questions. Dr. Craig Gibbs, Chief Business Officer, Dr. Mark Chao, Founder and Vice President of Clinical Development, and Dr. Jens-Peter Volkmer, Founder and Vice President of Research and Early Development are also on the call and will be available for questions.


Before we begin, I would like to remind everyone that statements we make on this call will include forward-looking statements. These statements are subject to risks and uncertainties that could cause actual results to differ. A description of these risks can be found in our most recent periodic reports we file with the SEC. Any forward-looking statements are made only as of today's date and we undertake no obligation to update these forward-looking statements to reflect subsequent events or circumstances.

I would now like to turn the call over to Mark.

Mark McCamish -- Chief Executive Officer

Thanks, Michael. Good afternoon, everyone. Thank you for joining us today to review our second quarter 2019 financial results and recent business highlights which we announced earlier today.

In the second quarter, we continued to build and strengthen our clinical pipeline with important progress across multiple programs. This afternoon, our objective is to provide a recap of the promising 5F9 data we presented in the second quarter and to update you on our plans to advance into registration-enabling trials in myelodysplastic syndrome, or MDS, and diffuse large B-cell lymphoma, or DLBCL. We will also provide some commentary around the market opportunity in both these indications and how we think 5F9 might complement existing therapies and address outstanding areas of unmet need.

First, however, I want to provide perspective on the growing strength of our business. At Forty Seven, we are focused on developing novel checkpoint therapies to activate macrophages in the fight against cancer. Based on our scientific founders' discovery of the fundamental role that CD47 plays in cancer invasion, we believe we can introduce a new therapeutic strategy for treating a range of oncology indications. We aim to block the CD47 "do not eat me" signal and, as a result, restore ability of the macrophages to attack and destroy cancer.

Today, our development program for 5F9, our lead anti-CD47 antibody, includes over 10 different clinical trials in a range of solid and liquid tumors and many of which are co-funded or funded by our clinical trial collaborators. Our broader pipeline includes additional programs focused on related or synergistic targets. Based on recent interactions with the FDA, we believe we have potential fast-to-market opportunities in both MDF and DLBCL and we plan to initiate our first registration-enabling trials in the first quarter of 2020. If successful, we would be in a position to file our first BOA for 5F9 in the fourth quarter of 2021.

Now, let me turn to our recent achievements. Over the last quarter, we made tremendous progress toward our vision of helping patients worldwide defeat their cancer. In June, we presented exciting new data in MDF, acute myeloid leukemia, or AML, DLBCL, and follicular lymphoma, or FL, at ASCO, EHA, and Lugano meetings. In each population, the data showed meaningful clinical activity and importantly demonstrated in patients treated thus far with 5F9 that it offers a safe and well-tolerated option, even for patients who are too frail to tolerate standard-of-care therapy.

These results, combined with promising feedback from the FDA, strengthen our confidence in 5F9 as a centerpiece of our corporate strategy and provide a strong foundation upon which we can continue to execute. In a few moments, Chris Takimoto will walk you through these data in greater detail and also provide color around the registration-enabling trials in MDF and DLBCL, which we expect to initiate early next year.

Now, I want to take a moment to highlight our strategic collaboration with Ono Pharmaceutical, which we announced in July. We believe this partnership is important for several reasons. First, it enables us to expand the development of 5F9 into key global regions, namely Japan, South Korea, Taiwan, and the Asian countries, while still allowing us to focus our internal efforts on our path to U.S. registration and potentially to pursue other global partnerships.

Second, it allows us to work with a proven leader in the immuno-oncology space who has the unique regional expertise and capabilities to successfully develop and launch a targeted therapy in East Asian markets. In addition to end licensing and commercializing therapies like Kyprolis, Braftovi, and Mektovi, Ono secured the first regulatory approval for Opdivo worldwide, which makes us confident that they will be able to advance 5F9 rapidly and efficiently.

Finally, it provides additional non-diluted capital to Forty Seven through both an approximately $15.8 million upfront payment and the potential for up to an additional approximately $104 million in future development and commercial milestones, plus tiered percentage royalties on future net sales.

Also in July, we significantly strengthened our balance sheet with a successful follow-on offering that raised approximately $86.3 million in gross proceeds. With this financing, we have sufficient capital to advance our ongoing efforts through the first quarter of 2021 and well beyond critical data readouts from nearly all of our planned and ongoing clinical trials.

Before turning the call over to Chris to provide his perspective on the new 5F9 data, as well as next steps in both MDS and diffuse large b-cell, I wanted to highlight one participant in this equity raise. As we announced in August, the Leukemia and Lymphoma Society has recently committed to provide up to $6 million of additional funding to Forty Seven, $3 million of which came as an equity investment in our July offering and $3 million of which will be granted upon the achievement of select milestones in our MDS program. LLS has been a longtime supporter of Forty Seven, and we are humbled by their continued trust in our execution capabilities.

We are also encouraged by their commitment to MDS, in particular. LLS has made MDS a key strategic priority for the foundation, which speaks to the tremendous unmet need in this patient population and reflects the lack of new options approved in this indication, even as other hematologic cancers have seen significant advancements. We are excited to partner with them on the development of 5F9 in MDS, where we believe we can provide the first disease-modifying treatment in nearly 15 years.

With that, I'd like to turn the call over to Chris Takimoto.

Chris Takimoto -- Chief Medical Officer

Thanks, Mark, and good afternoon, everyone. As Mark described, we've made important progress across our clinical stage pipeline. We're working hard to advance our ongoing trials and to expand our clinical activities across our portfolio with the goal of quickly and efficiently delivering new medicines to patients.

In June, we were pleased to share important updates from our Phase 1b clinical trial in patients with MDS and AML and from our Phase 1b and Phase 2 non-Hodgkin lymphoma clinical trial in patients with DLBCL and follicular lymphoma. While I will focus my comments today on MDS and DLBCL populations, I do want to highlight that we're very encouraged by the new data in both AML and follicular lymphoma, and we continue to view both as meaningful long-term opportunities for 5F9.

Turning now to MDS, at ASCO, we presented an initial update on our Phase 1b trial of 5F9 in patients with MDS and AML. In addition to demonstrating an excellent tolerability profile, we saw outstanding clinical activity in a broad population of patients. Notably, in the higher risk MDS patients treated with 5F9 and azacitidine, we saw an overall response rate of 100% with 55% of the patients achieving a CR. Importantly, no responding patients relapsed or progressed on the combination regimen and multiple patients showed further improvement in their responses over time.

We approached the agency with these data in hand and we received guidance suggesting that a single-arm trial evaluating 5F9 in combination with azacitidine could be sufficient to support an approval in first-line intermediate to very high-risk MDS, with primary endpoints of CR plus PR in durability of response. We anticipate needing a sample size of 91 patients with six-month efficacy data and 12-month safety follow-up and we plan to work with the FDA under a special protocol assessment, or SPA, to finalize key parameters for the study.

As of now, we expect to take a two-part approach: we'll expand our current trial, which uses a weekly dosing regimen, to 91 patients to accelerate that acquisition of 12 months of safety data, and we'll work with the FDA under a SPA to design a second new trial using a more convenient two-week dosing regimen to evaluate the combination in an additional 91 MDS patients. Importantly, either study could be registration-enabling, increasing the probability of a successful BLA filing.

We expect to expand and complete enrollment in our existing MDS trial in the third quarter of 2020, to initiate our second MDS trial in the first quarter of 2020, and to complete enrollment in the first quarter of 2021. This will allow us to file a BLA using combined efficacy and safety data in the fourth quarter of 2021.

We're particularly excited about our path forward in MDS. Unlike other hematologic malignancies, which have seen a number of new approvals in recent years, the treatment landscape in MDS has not changed in over a decade. There are just three approved therapies available to patients: azacitidine, decitabine, and lenalidomide. Of these, only azacitidine and decitabine are approved for higher risk patients, where they offer a combined CR plus PR rate of just 16% to 17%. As a result, the vast majority of MDS patients choose to forego therapy and receive only supportive care, such as blood transfusions and growth factors.

For this reason, we believe that a disease-modifying therapy such as 5F9 plus azacitidine could have tremendous potential in MDS. We plan to focus our initial efforts on first-line, intermediate to very high-risk patients, as defined by the IPSSR, and to expand over time into the relapse, refractory, and lower risk populations.

Let me now turn to our diffuse large b-cell lymphoma program. At EHA and Lugano in June, we presented new data for 5F9 in combination with rituximab in heavily pretreated patients for whom recently approved therapies are frequently not an option. This includes patients with advanced forms of DLBCL who have failed multiple lines of treatment or who are ineligible for existing options, such as CAR-T cell therapy, due to advanced age, significant co-morbidities, or the diagnosis of rapidly progressive disease.

As we discussed in June, this was a very different population from that which we treated in our Phase 1b study. On average, the Phase 2 patients were quite a bit older and 89% of these patients were CAR-T therapy ineligible, meaning that they were generally sicker and more difficult to treat. Even so, we were encouraged by the efficacy data we reported out, which showed an overall response rate of 45% across all patients treated in the Phase 1b and Phase 2 portions of our trial, a 29% overall response rate for patients treated in the Phase 2 portion of the trial, and an overall response rate of 38% in the subgroup analysis of the combined Phase 1b and Phase 2 patients with three or more prior lines of therapy.

Importantly, our data also showed similar response rates across multiple DLBCL sub-types, including patients with primary refractory disease and irrespective of the number of prior lines of therapy.

Based on these data, as in MDS, we met with the FDA in the second quarter of this year and we were pleased to receive guidance suggesting that a single-arm trial of 5F9 in combination with rituximab would support an initial filing in heavily pretreated DLBCL with primary endpoints evaluating overall response rate, including PRs and CRs, and the durability of response. We expect this trial to enroll approximately 100 patients beginning in the first quarter of 2020 and we expect that we'll be in a position to report interim efficacy results by the fourth quarter of 2020.

Now, let me comment briefly on the target patient population for this study. Based upon FDA feedback, we focused on heavily pretreated DLBCL patients, defined as those who have received at least two prior lines of therapy, and those who are CAR-T ineligible. We're now working to refine the eligibility criteria for our trial based on clinical and translational data from our Phase 1b and Phase 2 trials. Our goal is to identify the patient population where we can have the greatest impact based on clinical and laboratory profiles as well as patient medical histories.

As you know, despite recent approvals, there is still substantial unmet medical needs in the refractory DLBCL population. As we previously described, we believe that there are approximately 40,000 to 50,000 DLBCL patients on drug therapy in the U.S. at any given time. Amongst these patients, between 10% and 20% are on later lines of therapy.

However, once patients reach the third line setting, there is a substantial drop-off in available treatment options. A majority of patients are ineligible for newly approved CAR-T cell therapies due to medical limitations, rapidly progressive disease, and/or an inability to gain access to therapy. Furthermore, even for those who have options available, response rates are typically low, especially in the elderly population.

There remains a clear and compelling need for new therapies that can better serve older, more heavily pretreated DLBCL patients, and it is here that 5F9 has the greatest potential as a highly effective, safe, and extremely well-tolerated regimen.

Before turning the call over to Ann, let me remind you that we have a number of other milestones expected before year's end, including updated clinical data in MDS and AML, Phase 1b safety and efficacy data in colorectal cancer, and Phase 1b safety and efficacy data in ovarian cancer. Together, these upcoming readouts provide opportunities for us to characterize further 5F9 and better define those patient populations in which it offers the greatest benefit. We look forward to providing further updates as we work to accelerate the development of 5F9.

We are also pleased with our continued progress on the FSI-174, which is our anti-cKIT antibody. As a reminder, we are exploring the potential to use this antibody as a novel conditioning regimen for bone marrow transplantation. We are on track for an IND filing by the end of this year. In addition, we plan to present proof of concept, non-human primate data from this program at a major medical meeting in the fourth quarter of this year.

So, with that, I will turn the call over to Ann to review our financial results for the second quarter of 2019.

Ann Rhoads -- Chief Financial Officer

Thanks, Chris. In July, we fortified our financial position with a successful follow-on equity financing, resulting in gross proceeds of approximately $86.3 million. With this capital, we believe we are sufficiently funded to support our planned operations and capital expenditures through the end of the first quarter of 2021, which is beyond anticipated interim data readouts for almost all of our ongoing clinical trials, while also investing in our earlier stage pipeline. We're humbled by the continued support of our existing shareholders and excited to welcome new investors to Forty Seven as well.

We ended the second quarter of 2019 with cash, cash equivalents, and short-term investments of $99 million, compared to $139 million as of December 31, 2018. This does not include the gross proceeds of $86.3 million from our follow-on financing that closed in July 2019 or the upfront payment of approximately $15.8 million we will receive as part of our collaboration with Ono.

Turning to our second quarter 2019 income statement, during the second quarter, we incurred $18.8 million in research and development expenses, compared to $13.6 million for the same period last year. This increase in R&D expenses was primarily driven by a $3.2 million increase in third-party costs related to advancing our current clinical programs for 5F9 and associated contract manufacturing costs, as well as a $1.1 million increase in our pre-clinical and discovery program costs related to our immuno-oncology efforts.

General and administrative expenses were $5.1 million for the second quarter of 2019, compared to $3.4 million for the second quarter of 2018. This increase in G&A spending was largely due to an $800,000 increase in personnel-related costs, driven by an increase in headcount, a $600,000 increase in accounting and consulting expenses incurred in connection with our operating as a public company, and a $200,000 increase in directors' and officers' insurance expense.

Our net loss for the quarter was $23.2 million, or $0.74 per share, compared to a net loss of $16.7 million, or $2.52 per share, for the second quarter of 2018.

With that, I'll now turn the call over to the operator for questions.

Questions and Answers:

Operator

Thank you. Ladies and gentlemen, if you have a question at this time, please press "*1" on your touchtone telephone. And if your question has been answered or you wish to remove yourself from the queue, please press "#". To prevent any background noise, we ask that you please place your line on mute once your question has been stated.

And our first question comes from the line of Matthew Harrison with Morgan Stanley. Your line is now open.

Matthew Harrison -- Morgan Stanley -- Analyst

Great. Good afternoon. Thanks for taking the question. Two from me. I guess, first, Chris, maybe you could just comment on the ovarian and colorectal combo studies and just how we should be thinking about your ability there to be to a cohort of patients where you think you're at an effective dose and we can get a good idea of what kind of efficacy you might be able to achieve with that combination. And then the second question is just around patient population. And maybe you could talk a little bit in more detail for the pivotal DLBCL study, what sort of work you're doing right now and when you'll be able to give us clarity on what sort of a final patient population looks like. Thanks.

Operator

Speakers, have you muted your phone? Ladies and gentlemen, please stand by. And we are live.

Mark McCamish -- Chief Executive Officer

Hello, everyone. Sorry. We somehow got cut off. This is Mark McCamish. We were just trying to interface with Matthew Harrison on two questions. We got the first question, which Chris elegantly answered and you didn't hear anything of it. So, let me go back and make sure that Matthew is still there. And the first question, Matthew, was on the ovarian and CRC. The second question we did not hear.

Matthew Harrison -- Morgan Stanley -- Analyst

Yeah. Can you hear me?

Mark McCamish -- Chief Executive Officer

Yeah.

Chris Takimoto -- Chief Medical Officer

Yeah.

Matthew Harrison -- Morgan Stanley -- Analyst

Okay. Great. So, the second question, I was just asking broadly on -- can you talk about the work that you're doing related to patient populations for the pivotal DLBCL study and what sort of the timeline for that work is and when you think you'll be able to tell everybody what the patient population you've settled on and the criteria you used to establish that?

Chris Takimoto -- Chief Medical Officer

Sure. So, Matt, let me address your question about the ovarian and colorectal cancer programs. So, as we've mentioned previously, we anticipate sharing results from our colorectal cancer combination trial with cetuximab and our ovarian cancer trial with avelumab in the fourth quarter of this year. We have mentioned in these programs that we have seen some degree of activity but I think the key message that we have today is that the company is really focused on the two indications that we updated you on earlier -- that is MDS and DLBCL -- and that's really where the major focus of our company is at the present time.

Mark McCamish -- Chief Executive Officer

And DLBCL patient population.

Chris Takimoto -- Chief Medical Officer

Yeah. And then to your second question about the DLBCL patient population, as you're likely aware, we have a very extensive translational component to the study and we are looking at these patients in our DLBCL population, specifically to look at both clinical and laboratory markers that may help us select a patient population for further focus. And at the present time, these activities are under way and we anticipate -- you asked about when we would likely be able to share more information there. So, we would anticipate that some point in 2020 we would actually be able to share more specific details about that program.

Mark McCamish -- Chief Executive Officer

And, Matt, this is Mark. We're encouraged by the initial translational work that we believe will inform us on patient selection. And as Chris mentioned, we're in that process now. We're going to be starting that trial in the first quarter so, obviously, we'll have to have the inclusion criteria developed and laid out prior to that. So, that'll be in the next few months this year.

Matthew Harrison -- Morgan Stanley -- Analyst

Great. Thanks very much.

Mark McCamish -- Chief Executive Officer

Thanks, Matt.

Operator

Thank you. And our next question comes from the line of Michael Schmidt with Guggenheim. Your line is now open.

Michael Schmidt -- Guggenheim -- Analyst

Hey, guys. Thanks for taking my questions. Maybe just a follow-up, maybe a bigger picture follow-up question on sort of the general positioning in DLBCL. And it sounds like you're still finalizing the exact patient selection criteria but just how should we think about the market in the interim in terms of annual patients that might be treatment candidates in DLBCL initially?

Mark McCamish -- Chief Executive Officer

Right. Michael, thanks. This is Mark McCamish. Let me just start with that and maybe Craig can refine it in terms of patient numbers. As you know, DLBCL is a fairly large patient population overall. And then as you begin to have the treatment and the failures, that population is being more defined as we go forward. What we see is a high unmet medical need.

And just to reflect back, recall, as we were moving in this area, what we were trying to search for is a quicker strategy for early registration. Now, FDA is always guiding companies toward randomized control trials and if we have a randomized control trial, we can go to earlier line patients and go with combinations for comparison. For a single arm trial, what we got was guidance from them to avoid some of the medications which will either receive approval or recently received approval in this patient population.

And so for using a single arm trial for earlier approval, they guided us to heavily pretreated, second line, including CAR-T ineligible. So, we explored the CAR-T ineligibility in our Phase 2 portion, learned a lot about that in terms of the types of patients that are there, and there's clearly a group of patients that are progressing very rapidly, that actually progress before even getting the therapeutic dose. Remember, we have a priming does of 5F9 and then the first therapeutic dose and some of those progress quite rapidly. So, obviously, those patients do not add to the overall response rate that's there.

So, we're using the clinical criteria as well as the translational criteria that we're finalizing now to identify that patient population. And we shared with you in slides previously, and these slides are still on the web, where we looked at Phase 1b patients, Phase 2 patients, and then a group that we called "greater than 3 prior lines," and we're kind of guiding you all toward that response rate we saw in the greater than three prior lines and that's what we've focused on, in terms of the inclusion criteria, as well as the translational medicine data to have that patient population.

And recall that this, again, is our first step in and we've got multiple opportunities to advance in earlier lines of treatment with combinations. As you know, we're working with Acerta with acalabrutinib. We're working with Genentech with atezolizumab. We're also doing ongoing studies with combinations of chemo that will get us to earlier lines of treatment. So, this first indication is just to get in the door.

And let me turn it over to Craig for other thoughts.

Craig Gibbs -- Chief Business Officer

Sure, Michael. Michael, I'm sure you're aware, there's probably about 90,000 to 95,000 DLBCL patients in the U.S. And at any given point in time, about half of them, about 48,000, are on treatment. And if you start getting into the treatment experienced patients, over 9,000 are on second line and a little over 7,000 are on third line and beyond. And we're really focused on that second line and beyond population.

The real unmet needs in that patient population includes patients that are ineligible for CAR-T therapy. This could be because they're elderly, because they have co-morbidities that mean they wouldn't be able to withstand some of the side effects and toxicities associated with CAR-T. It also includes patients who have rapidly progressive disease who can't wait around for an adoptive cell therapy, which takes several weeks to implement. And, finally, patients who don't have access, whether it's for geographical or economic reasons.

The other unmet need in this population, particularly patients in third and later lines of therapy, is with the current therapies, even some of the new, emerging therapies, there's a big drop-off in response rate when you go from beyond second line to beyond third line. So, those are the patient populations we're targeting.

Michael Schmidt -- Guggenheim -- Analyst

Understood. And then I think you did mention a potential interim efficacy analysis in the design of the study. I'm just wondering if you could share, I guess, if you prefer a utility analysis or a certain verbal rank to head in that interim analysis? Thanks.

Chris Takimoto -- Chief Medical Officer

So, in terms of the study, both when it initiates and when we would have data to present, again, we're targeting the first quarter of next year for starting the DLBCL population under the new eligibility criteria. And then we would anticipate actually having data to present from that study toward the end of 2020, in terms of an early public presentation of the data.

Michael Schmidt -- Guggenheim -- Analyst

Okay. And then following up on Matt's question, just the combination studies that you're doing in ovarian and colorectal in combination with avelumab and cetuximab, respectively, I guess how should we think about those? It sounded like potentially the solid tumor indications may be a lower priority at this point or did I understand it correctly?

Mark McCamish -- Chief Executive Officer

Michael, we have to really focus on where we're at. We did go through this fundraising component to get to secondary. We have pathways forward with MDS and diffuse large b-cell. We elect to prioritize in that area. That being said, we're still continuing to bring those trials to fruition and will be able to share that data with you by the end of the year, and that will then color how we look at it for additional approaches. Recall, we've talked about the initial regional partnership with Ono, which we're very pleased with. We'll continue to have discussions with a global partner and that global partner can inform us about how much we can expand our efforts beyond the single arm trials that we're focused on now.

Michael Schmidt -- Guggenheim -- Analyst

Makes sense. Great. Thank you.

Mark McCamish -- Chief Executive Officer

Thank you.

Operator

Thank you. And our next question comes from the line of Martin Auster with Credit Suisse. Your line is now open.

EK -- Credit Suisse -- Analyst

Good afternoon, everyone. This is EK speaking on behalf of Marty. Thanks for taking the question. I just have two questions. The first question has to do around the CAR-T ineligible patient population that FDA guided toward you studying 5F9 in. Is this something that could potentially actually make it to the label that will definitely say this drug is for CAR-T ineligible patients, therefore precluding competitors, such as ApaLI and MorphoSys, from treating this patient population? Maybe perhaps is it, from a reimbursement perspective, though a doctor can select for one of these drugs but it won't be reimbursed? Something to that degree?

Mark McCamish -- Chief Executive Officer

Thanks, EK Let me start on that and see. In that situation, the earlier discussions with FDA were focusing on us pushing to a single arm trial approach. They then suggested that the CAR-T ineligible patient population would be of interest to them, although it was ill-defined. No one had used CAR-T ineligible previously. So, they asked us to have that as an inclusion criteria for our Phase 2 and then define how physicians classify them as CAR-T ineligible. Now, we did that experiment and found a variety of responses within that CAR-T ineligible population.

But in our follow-up meeting with FDA, wherein we were discussing the trial design and the numbers of patients, we basically defaulted to greater than second line, heavily pretreated, including CAR-T ineligible. So, since it's including but not exclusively CAR-T ineligible, it's unlikely it would be on the label as only for CAR-T ineligible. So, in that sense, what we're trying to do is use that term but also then say how are they ineligible but not restricted to CAR-T ineligible. Because based on the Phase 2 data, we feel that that would be more restrictive. So, it is more inclusive than CAR-T ineligible and we'll define how physicians describe CAR-T ineligible patients.

EK -- Credit Suisse -- Analyst

Okay. Thank you. And then just one last question. Since you're going to be expanding a couple of the cohorts into Q2 weekly dosing as opposed to the weekly dosing that you already updated on, can you kind of elaborate on what's giving you guys comfort around that you're not gonna be compromising any type of efficacy by moving to this more prolonged dosing regimen? Is there any PK/PD models that's established that maybe you can provide further color on or anything to that degree?

Mark McCamish -- Chief Executive Officer

Sure. So, this is MDS specifically. We're already with Q2 week dosing with lymphoma. And with MDS and AML, we do have access to bone marrow and actually blast cells. So, we're able to quantitate the receptor occupancy in those cells so it is actually fairly straightforward, particularly compared to any solid tumor component where access is an issue. And we've done various models around that in terms of the receptor occupancy based on PK.

So, we're pretty confident that going to every two weeks will meet the receptor occupancy necessary for efficacy and we're using the weekly dosing that we're continuing right now, as Chris mentioned, to accumulate patients for longer term safety follow-up. And if you're dosing every week, that safety follow-up is very applicable to dosing half as much every other week. And so we're continuing that, primarily to speed up the potential for the application when we're using the second part, which is the 91 patients, and that'll be treated every two weeks. So, it's a nice way to ensure we've got complete data. We're quite confident from the PK receptor occupancy data that the every two week dosing will be adequate but we're using the weekly dosing to accumulate the 12-month safety data necessary for the file.

EK -- Credit Suisse -- Analyst

All right. Thank you for that, Mark.

Mark McCamish -- Chief Executive Officer

Sure. Thank you.

Operator

Thank you. And our next question comes from the line of Mark Breidenbach with Oppenheimer. Your line is now open.

Mark Breidenbach -- Oppenheimer -- Analyst

Hey, guys. Thanks for taking the questions. Just a couple, both really focused on the DLBCL program. Maybe you can comment on what you're seeing as the benchmark you would need to beat in the population you're targeting. Thinking back to the Scholar 1 retrospective analysis, the response rates weren't that far out of line with the response rates you've seen in your Phase 2 cohort. So, I'm wondering if you have a benchmark that you're internally thinking you need to clear for product registration.

Mark McCamish -- Chief Executive Officer

So, first, let me take a shot and then I'm looking at Chris. He'll kick me under the table on this. So, basically, as you quote, the Scholar 1 is important. But, as a reminder, the duration in that was very, very short. In fact, the overall survival was something like six months. So, the duration of response for Scholar 1 in these patient populations is quite short. So, the hurdle is not just ORR, which is a combination of PR and CR, but it's durable ORR. And we have had discussions with the agency around that. We tried to clarify statistically what the lower margin would be and we've had those discussions.

And, again, we feel that the data that we've provided to you previously on the enrolled patients, when we looked at the greater than three prior lines -- and I know that we're not going to be using greater than three as our inclusion criteria -- but in all of the evaluation for this, that greater than three prior lines had an overall response rate of 38% and we think that that's in the ballpark of what we'll be using in this patient population. And duration is gonna be key. So, I think the Scholar 1 gives you a good baseline characteristic with very short duration and then you've seen from the polatuzumab data, as they go to beyond second line, their overall response rate falls off quite a bit as well. So, it just gives you some guidance but, recall, it's going to be a combination of response rate and durability.

Chris, do you want to refine that?

Chris Takimoto -- Chief Medical Officer

Yeah, no. I think that's really the key point. I think the only thing I would add is that if you actually look at the polatuzumab data and actually look at the subset analysis of patients that have had two or more prior lines of therapy before going on that study, the overall response rate there was 35%. So, down from their overall population. And we are seeing better than that in our patients with similar lines of therapy. So, I think those set up for good benchmarks going forward.

Mark Breidenbach -- Oppenheimer -- Analyst

Okay. That's helpful. And just not to harp on this point, but I was wondering, in the definition of CAR-T ineligible patients, would the considerations solely focus on medical reasons for CAR-T ineligibility or would you also include socioeconomic factors that would prevent a patient from being eligible for CAR-T treatment?

Mark McCamish -- Chief Executive Officer

I think that both are key in this situation. Again, we're providing the data to back up what CAR-T ineligibility is. From our experience in that CAR-T ineligible population, there is a subgroup that are progressing quite rapidly. And those are patients who, by the time you get them enrolled and then you get your first priming dose, they can have already progressed. And those are the patients that are very challenging to deal with. And so one of the things that we're looking at, of course, in the inclusion criteria and the protocol itself, is wash-out time. We were pretty liberal with this before. With our first study, it was two weeks. So, they could have been on a prior protocol and two weeks later be enrolled. And we're looking at that as well. And it's not just the CAR-T ineligible, it's that rapidly progressing -- that, within a week, they're already progressed. So, those are just to give you an idea of the patients that we're looking at and we think that we've got a reasonable handle on.

Mark Breidenbach -- Oppenheimer -- Analyst

Okay. That's helpful. Thanks for taking the questions.

Mark McCamish -- Chief Executive Officer

Thanks, Mark.

Operator

Thank you. And our next question comes from the line of Tony Butler with ROTH Capital. Your line is now open.

Tony Butler -- ROTH Capital -- Analyst

Yes. Thanks very much. Just really two questions. One is the sites that you open up for the expanded studies or the new studies, if you will, for either MDS or DLBCL, are those only in the U.S.? That is, do they include the EU? That's No. 1. And No. 2 is what's your thought about AML going forward? You said you're still interested. I get that. But does that just place AML on the back burner for a period of time? And then if I may sneak in, again, solid tumor data that we'll see later this year, does that, regardless of whatever the outcomes are, given that your plates are pretty full with these two registration studies, do those also move to the back burner? Thanks very much.

Mark McCamish -- Chief Executive Officer

All right. Mark Chao will step in here and help on the MDS and then we'll go from there.

Mark Chao -- Founder and Vice President of Clinical Development

Yeah. So, I'll take two of the three questions and then, again, reiterate back to Chris for the third. So, for the first question, in terms of the sites for both expansion for MDS and DLBCL, currently, our ongoing studies are primarily in the U.S. We do have a site in the United Kingdom. I think for the expansions, we're primarily focused on U.S.-based accrual but there is a possibility, given the fact that we want to accelerate timelines, to go ex-U.S. And, again, we're looking at which countries to go into. But I think, primarily, it's been a U.S.-based focus. And we want to ensure that physicians in the U.S., because we anticipate that's where approval is going to be first, to have the maximum amount of experience, but certainly want to expand to ex-U.S. markets.

I think your second question, in regards to AML, again, I'd reiterate what Chris mentioned. We're highly encouraged by that data. Again, the ASCO/EHA data, again, with 14 patients shared around a 60% response rate with some initial untreated duration. I think, obviously, when we look at AML, and we're looking at rapid paths to approval, we do know that the venetoclax has been approved and, certainly, that's been used, which is a great option for patients. And we think -- looking at strategies of how we can impact the AML landscape, just given that agent and others.

So, I think we're still looking. In our program, we're certainly looking at sub-populations. Certainly, an unmet need are patients that don't respond to venetoclax and there's some emerging data coming out there in terms of certain mutational subsets. And also patients who fail venetoclax. We know it's not a curative regimen so patients ultimately relapse. And in that relapse refractory setting, there is no treatment option. So, that, again, could represent a fast path for approval. So, I think we're still interrogating that data. Again, looking at where we potentially develop, again, with a core focus in MDS and DLBCL but certainly there are definitely upside opportunities, particularly in AML. And then I'll defer your solid tumor question back to Chris as well.

Mark McCamish -- Chief Executive Officer

And just maybe just to add on to what Mark is saying, we're still enrolling AML patients and MDS patients in that trial so it gives you an idea that we're still quite interested in the AML side. Chris?

Chris Takimoto -- Chief Medical Officer

And then, Tony, your third question was how we prioritize the solid tumor programs. And, again, these are ongoing studies and we are going to be data driven. But very clearly, I think when you look at the strong signals that we're seeing of activity in diseases like MDS as well as the clear registration path forward, those are very clearly the highest priorities for the company and so that's really where our major focus, we anticipate, will remain -- on DLBCL and MDS.

Mark McCamish -- Chief Executive Officer

And, again, it gets back to the partnering strategies as well. Always, from the beginning, these are large populations, some of whom which we wouldn't be able to commercialize ourselves, and a global commercial partner would be able to weigh in here. Obviously, there are real needs in the colorectal area. Not much available. So, obviously, the data coming out will be important to do that but we also have to be respectful of our resources and be able to plan appropriately and make sure we're successful in our first filing. And that would be, of course, the priority.

Tony Butler -- ROTH Capital -- Analyst

Thanks very much, guys.

Mark McCamish -- Chief Executive Officer

Thanks, Tony.

Operator

Thank you. And as a reminder, ladies and gentlemen, if you would like to ask a question, please press "*1" on your touchtone telephone. And our next question comes from the line of Swayumpakula Ramakanth with H.C. Wainwright. Your line is now open.

Swayumpakula Ramakanth -- H.C. Wainwright -- Analyst

Thank you. This is RK from H.C. Wainwright. Most of my questions have been asked but I just have a question on one of your partnerships. That is with Acerta. How should we think about that triplet investigation in DLBCL? How does that program go along with what you are doing with the doublet in the CAR-T ineligible population?

Mark McCamish -- Chief Executive Officer

Let me take a shot and then Craig can jump in. So, in this situation, RK, acalabrutinib has shown some efficacy but relatively short duration. So, AstraZeneca are very interested in whether the combination would be of benefit based on the science surrounding that. We are quite interested in the fact that we want to go to early lines of treatment as well. And how do you drive that forward? It's going to be a key component because, again, the initial approval is just the foot in the door. How do we begin to expand that? And so we're evaluating various partnerships to do so. And in this sense, this is fully funded by Acerta/AstraZeneca as well. So, it's a nice way for us to evaluate potential for getting to earlier lines of treatment with that combination. And that's pretty much the focus.

Mark Chao -- Founder and Vice President of Clinical Development

I might also -- this is Mark here -- add that one of our core strategies in combinations, as we've mentioned, has been combination with either chemotherapy or small molecules that seem to have synergies with up-regulating "eat me" signals. So, we think this may be part of that strategy that extends beyond our azacitidine experience and other types of chemo into certain small molecules. And, again, we're quite excited to test that clinically.

Swayumpakula Ramakanth -- H.C. Wainwright -- Analyst

Thank you. Thank you both.

Mark McCamish -- Chief Executive Officer

Thanks, RK.

Operator

Thank you. And I'm not showing any further questions on the phone line so I'd now like to turn the call back to Mark McCamish.

Mark McCamish -- Chief Executive Officer

Thanks, Chris, and thank you all for participating in this and taking the time to join us today despite our technical difficulties. We also really want to express our thanks for your continued support for Forty Seven. We look forward to updating you again soon and at this point we'll end the call. Thanks, everybody.

Operator

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

Duration: 49 minutes

Call participants:

Michael Horowicz-- Stern Investor Relations

Mark McCamish -- Chief Executive Officer

Chris Takimoto -- Chief Medical Officer

Ann Rhoads -- Chief Financial Officer

Craig Gibbs -- Chief Business Officer

Mark Chao -- Founder and Vice President of Clinical Development

Matthew Harrison -- Morgan Stanley -- Analyst

Michael Schmidt -- Guggenheim -- Analyst

EK -- Credit Suisse -- Analyst

Mark Breidenbach -- Oppenheimer -- Analyst

Tony Butler -- ROTH Capital -- Analyst

Swayumpakula Ramakanth -- H.C. Wainwright -- Analyst

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