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Arqule Inc (NASDAQ:ARQL)
Q2 2019 Earnings Call
Aug 7, 2019, 9:00 a.m. ET

Contents:

  • Prepared Remarks
  • Questions and Answers
  • Call Participants

Prepared Remarks:

Operator

Good morning, ladies and gentlemen, and welcome to the ArQule Second Quarter Year 2019 Earnings Conference Call. [Operator Instructions]

I would now like to turn the conference over to your host, Mr. Peter Lawrence, President and COO.

Peter S. Lawrence -- President and Chief Operating Officer

Good morning, everyone. Welcome to the ArQule investor conference call reviewing operational and financial results for the second quarter of 2019. I'm Pete Lawrence, the President and COO of ArQule. This morning, we issued a press release that reported results for the second fiscal quarter ended June 30, 2019. This release is available on our website at arqule.com. Leading the call today will be Paolo Pucci, our Chief Executive Officer.

Also present from the company are Dr. Brian Schwartz, Head of R&D and Dr. Marc Schegerin, CFO and Head of Strategy. Marc is traveling today but is on the call via cell and should be available for Q&A. As a result, I'll present our financial results. Before we begin, please note that we will be making forward-looking statements as defined in the Private Securities Litigation Act of 1995. These statements will include, among other things, projections regarding the timing of key events related to ArQule's proprietary pipeline and financial guidance for 2019.

Actual results may differ materially from those projected in the forward-looking statements due to numerous risks and uncertainties that exist in ArQule's operations, development efforts and the business environment, including those factors discussed in our reports on Forms 10-Q and 10-K and other documents filed with the Securities and Exchange Commission. The forward-looking statements contained in this call represent the judgment of ArQule as of today. ArQule disclaims any intent or obligation to update any forward-looking statement, except to the extent required by law. We will provide an opportunity for questions and answers at the end of the call.

I'd now like to introduce the CEO of ArQule, Paolo Pucci.

Paolo Pucci -- Director and Chief Executive Officer

Peter, thank you very much for the introduction, and thank you all on the call for joining us today. I hope everybody is having an eventful summer. Allow me to give first a brief overview of some of the progress we have made recently as a company. Then, I will turn the call to Brian for a deeper analysis of the clinical programs. And then, Peter will come back with financial results. And then, we'll be ready to take your questions. I would say -- I will start by saying that Q2 was a watershed period for ArQule in several ways. And we have delivered across the whole spectrum of our commitments both clinically and financially.

First, what is mostly focused this year is the BTK inhibitor. So in mutated B-cell malignancies, with ARQ 531, we presented first-in-kind dual inhibitor wild-type and C481-mutated BTK clinical activity data in refractory CLL as well as one responding Richter's Transformation at the EHA conference. Since then, we have continued to obtain data, and we have continued to analyze it. And we have come to the decision, which I'm happy to report today, that after assessing the totality of the data that is currently available to us, we have selected 65 milligram once-a-day as the recommended starting Phase II dose for ARQ 531. This kind of concludes a journey that has lasted almost two years since we filed an IND for this drug and opens the door to a very exciting next stage for this first and best-in-class molecule.

What is important to note also, in conjunction to this decision, is that we have observed no additional dose-limiting toxicity and that a maximum tolerated dose has not been reached for this study. So this concludes the brief overview of where we are with ARQ 531 with the most important development we have to report since we last discussed this program in EHA's conference call with Brian and Dr. Woyach, who's our PI at Ohio State. In rare overgrowth Spectrum disorder with miransertib, our first-generation AKT inhibitor, we have continued our plan to initiate sites around the world, and we expect to begin dosing Proteus and PROS patients in this -- in the 2 registrational cohorts of the 3 cohort studies still this summer. We see considerable interest and considerable enthusiasm from the investigators who have been following this patient for a long time without much therapeutic options other than maybe some off-label use of other drugs available.

And we are ever more determined to try to bring a viable therapy to this patient. In addition to following the process of opening the sites, we have also presented further updates for both Proteus syndrome and PROS at the European Society of Human Genetics. So we continue to build the data that underpins the registrational effort that we are currently engaged in. And we will continue to provide, where necessary, drug for compassionate use as we have done in the past, in parallel to conducting the registrational trial. We do not want to leave anybody behind if we can help it. Then for the corporate update. I would focus just on one item, and the one item is financial. We secured additional runway for all our clinical programs by raising proceeds -- gross proceeds of about $100 million in a very well received and well oversubscribed common stock offering lately.

And that we can discuss further in the Q&A what would that finance, although much of it is available publicly already in the prospectus that govern that transaction. I would like to express my thanks to our current investors that participated. And I would express my warm welcome to a number of savvy, large and highly specialized investors that have joined ArQule through this transaction. As the filings proceed, we will be more evident who the kind of core investors that participated in the transaction was.

Now let me pass to Brian for a brief clinical update of the core programs at this time. Thank you.

Jonathan Chang -- Analyst

Thank you, Paolo. Let me start with ARQ 531, which is our potent and reversible dual inhibitor of both wild-type and C481-mutant BTK. As Paolo mentioned, we recently presented a poster at the European Society of Hematology for ARQ 531 in refractory B-cell malignancies. The results were highly encouraging for us and the investigators. Of the 6 evaluable C481S CLL patients who have started at 65 milligrams once-a-day, 4 reported a partial response at the first scan. Of the 4, we also reported that 2 had already received the second scan at cycle 5, and we'll confirm those responders. In addition, the first Richter's Transformation patients in this study also reported a partial response at the first scan.

We are happy to report that we have now seen partial responses in 4 distinct B-cell malignancies: follicular lymphoma, CLL hovering the C481S mutation, Richter's Transformation and DLBCL. Preliminary results from the ongoing Phase I dose escalation study suggests that ARQ 531 is well tolerated at 65 milligrams once-a-day and has a manageable safety profile in multiple B-cell malignancies. Pharmacokinetic data showed that subjects who received 65 milligrams exhibited steady-state Cmin concentrations above the 1 micromolar level that we had predicted preclinically, and the plasma half-life range from 30 -- from 20 to 30 hours, suggesting a sustained and complete BTK inhibition, which can be achieved at 65 milligrams once-a-day. Maintaining a sustained Cmin concentration above 1 micromolar at the 65 milligram dose is predicted to be a critical factor in achieving antitumor response.

In summary, antitumor activity was observed at 65 milligram once-a-day in Cohort 7 in heavily pretreated subjects with an ORR, or objective response rate, of 62.5% in the 4 relapse and refractory CLL patients who harbored the BTK C481s mutation. In addition, one Richter's Transformation patient, as mentioned, also responded out of the 8 evaluable patients in this cohort presented at EHA. Subjects were also enrolled in the next dose level of 65 milligrams QD cohort 8. Though no DLTs were observed, some patients reported grade 2 adverse events, which either led to treatment discontinuation in one subject or dose reduction to 65 milligrams in 3 subjects. A maximum tolerated dose was not reached per study as per protocol. We also reported the first patient evaluated for clinical activity in this cohort.

A DLBCL patient had a partial response. Based on these promising preliminary results, we determined that the 65 milligram once-a-day dose will be our recommended starting dose for Phase II studies for ARQ 531 in subjects with B-cell malignancies. Please remember that ARQ 531 was chosen for its unique preclinical profile. And now that we are at the necessary exposure in humans, these attributes are becoming apparent in the clinic as well. Attributes such as domain selective kinase inhibition profile, long residence time in the BTK binding pockets, good pharmacokinetic profile for once-daily dosing and clear activity in a wide range of B-cell malignancies are all coming into focus as the study matures. In summary, we couldn't be more pleased with the performance of this drug so far, and we look forward to initiating multiple expansion cohorts at 65 milligrams this summer and presenting a comprehensive update at a major medical conference by the end of this year.

Let me now move on to miransertib in rare overgrowth spectrum disorders. Miransertib is a potent and selective AKT inhibitor. Our objective is to be the first and best-in-class AKT inhibitor in Proteus and PROS family of rare overgrowth diseases. This family of diseases is ultra rare, very heterogeneous and the patients currently suffer from dismal quality of life and early mortality. No systemic therapies have been approved for this patient population, and the only current treatment option is surgery. As a reminder, our registration program, the MOSAIC trial, will consist of one protocol divided into multiple cohorts. The first cohort will focus on Proteus syndrome and will enroll at least 10 patients. The second cohort will focus on the PROS family of overgrowth disorders and will enroll at least 20 patients.

The third cohort will be a signal generation arm that includes patients from either group who did not qualify for Cohort 1 and 2 but may otherwise benefit from treatment. These cohorts will be open-label, and the primary endpoint will be response rate driven based on objective, measurable criteria. We are currently enrolling additional sites globally and expect to dose the first patient very soon. Our other programs. For ARQ 751, the next-generation AKT inhibitor, the signal generation work in oncology continues in the Phase Ib study, and we plan to present data from this study in the second half of the year. Finally, for derazantinib, our FGFR inhibitor, our partners, Basilea and Sinovant, continued to implement their plans respectively for registrational Phase II trial in ICCA and toward Phase I initiation in China.

With that update, I would now like to turn it over to Pete.

Peter S. Lawrence -- President and Chief Operating Officer

Thanks, Brian. Turning to the financials. The company reported a net loss of $9.1 million or $0.08 per basic share for the quarter ended June 30, 2019, compared with net income of $5.2 million or $0.05 per diluted share for the quarter ended June 30, 2018. As of June 30 this year, the company had a total of approximately $182.8 million in cash, cash equivalents and marketable securities. Revenues in Q2 2019 were $0.3 million compared with revenues of $13.7 million in Q2 2018. Research and development revenue this quarter was comprised of $254,000 of reimbursable clinical trial costs from our Sinovant licensing agreement and $27,000 of revenue from reimbursable costs associated with our Basilea licensing agreement. Research and development expense for Q2 2019 was $6.3 million compared with $6.8 million for Q2 of 2018.

General and administrative expense was $3.2 million in Q2 2019 compared with $2.2 million in Q2 2018. The $1 million increase was primarily due to higher labor-related costs and stock-based compensation costs. For 2019, ArQule expects revenue to range between $3 million and $5 million. Net loss is expected to range between $40 million and $43 million and net loss per share to range between $0.35 and $0.37 for the year. As a result of our common stock offering in June, we're updating our cash guidance. ArQule now expects to end 2019 with approximately $160 million in cash and marketable securities.

With that, I'd like to turn the call over for Q&A. Operator, please feel free to open the line for questions.

Questions and Answers:

Operator

[Operator Instructions] Your first question comes from Gregory Renza with RBC Capital Markets.

Gregory Renza -- RBC Capital Markets -- Analyst

Hey guys congratulations on all the developments and thanks for taking my question.

Paolo Pucci -- Director and Chief Executive Officer

Thank you, Greg.

Gregory Renza -- RBC Capital Markets -- Analyst

Perhaps a question for Brian. I'm just curious if you could perhaps touch a little bit more on some of your thinking and logic around landing on the recommended dose. And just perhaps a commentary on some of that incremental data that has been mentioned that has perhaps helped to secure your confidence around the selection and the path forward.

Paolo Pucci -- Director and Chief Executive Officer

So let me take a first stab, and then I'll let Brian chime in. The time that there has a -- our last meeting from EHA finalization of the poster and us taking the decision, it's a relatively short time. However, there is -- however, there would be additional data that has come. That data has come from either side of the 65 milligram cohort, which is cohort 7, meaning we have been able to see data coming from patients that were initiated at 75, both for safety and efficacy, and would then be escalated to 65. So we have had some basic understanding of the space between 65 and 75. Also, remember, we had a number of patients that had been dose escalated from lower doses all the way to 45 and then on to 65. Those patients, also we -- also, we have seen some data from those patients being dosed at 65.

That's what we talk -- when we talk about the general understanding of the data, that's what we really talk about. A very solid understanding of the cohort of patients that started at 65 from the efficacy point of view and from the safety point of view, and then an understanding of what -- of the 45 to 65 and an understanding of 75 to 65. Now you're still not talking a tremendous amount of patients, but our -- for the 2 ends, right, 75 to 65, 45 to 65. But the core knowledge that we have acquired with 65 is now quite robust in terms of the number of patients and length of follow-up. That's what I would -- that's all I would comment in general. Brian, do you want to add anything?

Dr. Brian Schwartz -- Chief Medical Officer

So thanks for the question. I think Paolo covered most of the data. I think our criteria for selecting the dose was based on a number of criteria all being met. One is total BTK inhibition, a comfort level that the Cmin concentration for patients is above 1 micromolar. The safety profile, obviously, is very paramount and in the level of activity. As Paolo mentioned, since EHA, we have received data that we will disclose at an upcoming conference around additional pharmacokinetics, additional safety data and both increasing dose and decreasing doses of patients in the trial. With that information, it's very clear that if you're looking for a drug in CLL, where safety is very important together with efficacy the 65 milligram fulfills that criteria. We would also like to mention that we didn't officially declare a maximum tolerated dose, so do preserve the option to increase the dose at a later stage in difficult-to-treat tumor types should you require additional drug.

Paolo Pucci -- Director and Chief Executive Officer

So this is -- Greg, consistent with our strategy. We wanted a drug with the characteristic that Brian has highlighted as a single agent drug, right? We wanted the drug that would be safe. We wanted the drug that would be effective in the target population. We wanted a drug that would give us the exposure, but we also wanted a drug that preserves the ability to play well in combinations, which is, in large part, where many of these market segments are moving, and that was also factored in the consideration. Because when you are looking at combination, you want the cleanest possible profile -- safety profile. We also now know that this drug was designed in a way that strikes for now a good balance between having a domain selective profile and an acceptable safety profile. So we knew that it had very little supply ability, also an important factor when you think about combination. And now I think we have a dose that is viable both as a single agent and will probably be more so as combination. Remember also when you think about combination down the road is that this is once-a-day, so the minimum burden -- the posology burden that one would hope for.

Gregory Renza -- RBC Capital Markets -- Analyst

That's great, Paolo and Brian. And combination is exactly where I wanted to go with my next question. Paolo, how does combination planning factor into your next phase with the planned regulatory interactions and your design of the upcoming expansion cohorts?

Dr. Brian Schwartz -- Chief Medical Officer

I think, Greg, combination would really come into play with regards to our bigger trial or the next phase of development. So we've always commented and always made our initial trial an uncontrolled response rate driven trial in a selected population, the C481s-mutant population. We know the field is moving, and our confirmatory trial is highly likely to have a combination on with one of the most commonly used drugs in the space. So the next step is to prepare, make sure that our drug is combinable and be ready to move into the confirmatory trial setting in combination.

Paolo Pucci -- Director and Chief Executive Officer

And there are a number of combinations we're going to assess as well. Some might turn out to be created. We are running -- starting to kicking off, as a matter of fact, a second round of life cycle planning for the drug now that we can better specify its profile. And we'll assess those potential combinations in there. But we believe that we have a drug here that demonstrates to be able to stand on its own, single agent, which is always important, particularly when that is demonstrated so early in the process of a life of a drug. And now we're going to test if all the elements that we've built in the drug make it also an outstanding drug in combination, and we're hopeful for that.

Operator

Your next question comes from Jonathan Chang with SVB Leerink.

Jonathan Chang -- SVB Leerink -- Analyst

Hi guys. Thanks for taking my questions and congrats on the progress.

Paolo Pucci -- Director and Chief Executive Officer

Thank you.

Jonathan Chang -- SVB Leerink -- Analyst

To clarify, is cohort 8 still open? Are you still enrolling and/or treating patients at the 75 mg dose?

Paolo Pucci -- Director and Chief Executive Officer

No, we're not. We are treating all patients at this point in time at 65 milligrams. All the patients that were started on 75 have been de-escalated to 65. All the patients that were at 45 have been escalated to 65. And so all the patients before are going now at 65 milligrams, and the new patients are likely going to be enrolled in the expansion cohort now because the Phase I part of this Phase I/II has served its purpose with the dose selection. So the data coming up here will be largely out of this bolus of patients that are now at 65 regardless of what dose they started at.

But as Brian said it, as we understand the side effect profile better over time, we understand durability, as we assess the broad spectrum of indications we can consider, then now having reached an MTD, we still have the possibility to disclose further dose escalation. In CLL, which is the target indication, it didn't appear that, that was necessary. And remember that we weren't -- for those that are joining this call fresh, remember that we went into cohort 8 without the benefit of having been able to assess thoroughly efficacy from cohort 6 or cohort 7. So by the time we were initiating cohort 8, we started to get a compelling efficacy from cohort 7. So things needs to be put in their historical prospects in order to rationalize them. And for those of you on the call that are new to the story, that's the historical context you have to consider.

Jonathan Chang -- SVB Leerink -- Analyst

Got it. And also, just to clarify, when you mentioned seeing additional data from patients who went from 45 to 65, does that include additional efficacy data? Or are we talking safety from across the data?

Paolo Pucci -- Director and Chief Executive Officer

Yes, it includes efficacy as well.

Jonathan Chang -- SVB Leerink -- Analyst

I see. So there have been additional scans post the transition since patients gotten the 65 mg dose?

Paolo Pucci -- Director and Chief Executive Officer

All I can say is it includes efficacy data, Jonathan. And we have said that some of the patients at 45 were due to be scanned at some point in time during the summer. It's common knowledge -- it's public knowledge, the frequency of scanning, so one could kind of get it. We will give the details at ASH.

Jonathan Chang -- SVB Leerink -- Analyst

Understood. And then just last question, if you could elaborate how you're thinking about next development steps for 531 ahead of the planned regulatory interaction.

Dr. Brian Schwartz -- Chief Medical Officer

So Jonathan in terms of the current -- the regulatory interactions, we'll hope to get in front of the FDA at a pre -- at a meeting that's sponsored of and taken advantage of the end of Phase I meeting. We will discuss on full review of the data our decision for the recommended Phase II dose as well as the future developments in CLL. The focus will be on the C481S-mutant development plan moving forward. In parallel, our phase -- the trial that we currently have open now, as Paolo mentioned, will expand into multiple different cohorts. There are 7 cohorts that have been identified, which include a cohort which is BTK intolerant -- without the -- BTK intolerant or without the mutation, a cohort of follicular Richter's, mantle cell, marginal zone, Waldenstrom and double-hit high-grade DLBCL. So we've got a number of different sort of phase evaluate -- efficacy evaluations both into this trial, but we'll be moving forward with -- at the agency with CLL first.

Jonathan Chang -- SVB Leerink -- Analyst

Got it. Thank you very much.

Dr. Brian Schwartz -- Chief Medical Officer

You are welcome.

Operator

Your next question comes from Chad Messer with Needham & Company.

Chad Messer -- Needham & Company -- Analyst

Great. Thanks. Good morning and thanks for taking my question.

Dr. Brian Schwartz -- Chief Medical Officer

You are welcome Chad.

Chad Messer -- Needham & Company -- Analyst

Yes. Great. So it seems like you guys have really threaded the needle here between safety and efficacy with 531. And in fact, I was quite impressed by some comments from Dr. Woyach on the EHA call about how well tolerated the drug is. Just wondering if you guys have any thoughts about how you kind of accomplish that. And the whole the BTK class, most of the side effects are pretty much on target, but you guys seemed to have a very potent molecule, not without side effects but with a really good tolerability profile. Is that something to do with reversible versus irreversible or some other PK characteristics? Or are we just lucky here?

Paolo Pucci -- Director and Chief Executive Officer

Luck is always important, Chad, in everything we do. What I would say for fairness is that our data set is a Phase I data set, so it could not be compared for safety with the mountain of data, for example, ibrutinib zone, particularly as far as cardiovascular side effects go, which generally emerge with a much larger numbers of patients treated. That said, we have observed in and predicted through the pre-clinical experiments that the drug should be tolerable. We had no red flags really coming out of our preclinical work done so far. And to me, the surprise was more -- was not the side effect profile overall but how we keep a manageable side effect profile in such a heavily treated population. That was -- that surprised me on the upside until now at least. Brian, I don't know if you want to make any comments.

Dr. Brian Schwartz -- Chief Medical Officer

I think, Chad, we often view things in a binary way: it's a responder, it's not a responder. If you look at the totality of the data, there's clear and increasing degree of both clinical objective and un-objective measures of response from when we started to get full BTK inhibition at around 20 to 30 milligrams. So even though we didn't have much responses, it appeared as if clinical efficacy was clearly coming. Once we got to the 65 milligram cohort, it was very clear the efficacy was there. And then the question was, do you go higher or not? And we know this is a kinase inhibitor that is selected for a bunch of different families of kinase targets. So the question was, do you go higher or do you take that efficacy and just expand? We haven't disclosed here, but we'll disclose at the next opportunity the full PK profile. We've got a big jump between 30 and 45, a jump between 45 and 65. And then we'll disclose the full PK -- long-term PK. And I think it will be much clearer for everyone why the 65 milligram dose is really the dose we should take forward.

Chad Messer -- Needham & Company -- Analyst

All right. Understood. I look forward to those data updates.

Paolo Pucci -- Director and Chief Executive Officer

Okay. And then, again, 65 was at the higher -- at the higher end of the range of predicted effective and say, hopefully, dose based on the preclinical experiments. So pretty much in line with what the expectations were based on preclinical. And we have done, as you know, Chad, because you followed the story probably longer than everybody here, we did quite a bit of preclinical. And I would like to remind the people here on the call that they can find all that preclinical work in CLL on our website that has the cancer discovery publication where it is all revealed.

Operator

Your next question comes from Hartaj Singh with Oppenheimer & Company.

Hartaj Singh -- Oppenheimer & Company -- Analyst

Great. Thank you all for the two questions. One I have is just -- and maybe just focus on something else, on ARQ 751. I know, Brian, you've got a readout later this year. Roche has made a really big deal of ipatasertib, their AKT inhibitor. They presented some data at AACR. And it seems like if you and them sort of in the lead in this area, the AKT inhibition area seems to be pretty interesting. How -- can you just sort of frame for us how that -- those readouts should look? What are the solid tumors that are interesting to you in that study? I think Roche has focused on triple-negative breast cancer. And then also the potential for combining with various immuno-oncology approaches with the AKT. And then I just got a quick follow-up on miransertib.

Paolo Pucci -- Director and Chief Executive Officer

Maybe I'll try to take the first one. For us, we're still a relatively small company, and we have several programs ongoing. 751 for us is a program that is potentially valuable in many ways. But unlike ARQ 531 and miransertib in rare diseases, it's dependent a little bit on what the development with the Genentech program is going to be. If the Genentech program proves to be a phenomenal success, as we hope for the patient always, then that program will validate the target in -- and this is in answer to your question, in a variety of hormone-sensitive tumors and in a variety of combinations. So we are really talking of a combination strategy, which we will need to follow on. Very large studies. Not terribly likely to do, we'll be able to chase after Genentech independently, so we will be open to considering partnerships at that point because these are not relatively manageable-sized studies that we are planning to do with 531 to cross the finish line, and then we are doing miransertib to cross the finish line there as well.

So that's the way we see right now 751 strategically. So in the meantime then, we wait for Genentech to declare the target being successful or otherwise. We are preparing so that we'll be able to actually use it alone for what we can and in partnership probably broader a fast forward strategy there. So nothing has changed in our strategy for 751. The capital raise that has been executed recently goes largely to fund the fairly expensive program that Brian is beginning to detail for 531. Brian also has some thoughts that are out-of-the-box for 751 that will bring it in a synergistic format with 531, but we'd rather keep those thoughts to ourselves for now, if you forgive us for that for competitive reasons.

Hartaj Singh -- Oppenheimer & Company -- Analyst

Great. And Paolo, that's a great option to have. I'm really looking forward to more insights there. On miransertib, we had done a physician call with the KOL who's been really more than...

Paolo Pucci -- Director and Chief Executive Officer

An excellent call, an excellent call, because I think even patient associations -- it has made its way to those patient associations. And they were very grateful that you brought attention to such a rare and devastating disease like that on their behalf. Thanks for that.

Hartaj Singh -- Oppenheimer & Company -- Analyst

I appreciate it. It was just great to hear the passion of this KOL. One question I had was that as you're kind of thinking about miransertib, I know that the trial has to be very focused from a structural perspective on the patients that you're looking at. You've got 3 cohorts. But this overgrowth syndrome is a very wide variation of diseases. In totality, can -- it's very big, but the individual parts can be smaller. Can you just think a little bit -- help us through that? As you see the data, four in your 3 cohorts, how in the future could you see you the program broadening out from the more focused approach that you have now if it could be broadened out in the future?

Dr. Brian Schwartz -- Chief Medical Officer

Thanks, Hartaj. Let me just talk a little bit about the philosophy that we have and in discussions primarily with the FDA what our picture is. So because we're dealing with such a small patient population and so heterogeneous, we need to target a hard endpoint for initial approval. So the concept was for Proteus syndrome, our hard endpoint because of the work -- wonderful work done by the NIH, with regards to the longitudinal effect of the CCTN lesions, we could use that as a hard endpoint for Proteus disease. PROS is much more complex in that, as you mentioned, they're very different, But we're trying to find 20 patients with similar type lesions that we can measure using our specific MRI volumetric technique to show the change. The reason why we put in cohort 3 was to capture all those other heterogeneous groups so that our label potentially could read all PROS and all Proteus and not just the ones with a measurable lesion that we'll use in the Phase I -- so in cohort 1 and cohort 2. So it was designed in such a way that we would get hard endpoints, but we would also get a much bigger pool where we would have other endpoints as well. The other reason for including more patients was to get the effect on other instruments like quality of life on the whole group, which we feel will be very important for the label as well.

Paolo Pucci -- Director and Chief Executive Officer

And for miransertib as well. Now that it's gaining notoriety, I would say, in the scientific community, we are starting to receive inbound proposals for diseases that are probably of interest for the drug that we had not thought about. And that's the advantage of a drug gaining controversy and scientific notoriety through the work we're publishing through activities like your excellent conference call, etc. So here as well, there might be additional opportunities we have not contemplated until now. And those as well we'll keep covered for competitive reasons. But at the moment, we went through the fact to pursue them dependently or through investing -- initiating investigator-initiated activities, which is something we have done in the past and we plan to continue doing, then we'll be able to discuss them too.

Hartaj Singh -- Oppenheimer & Company -- Analyst

Great Paolo. That's great that you have all this going on. Really appreciate the questions.

Paolo Pucci -- Director and Chief Executive Officer

You're welcome.

Operator

Your next question comes from Matthew Cross with JonesTrading.

Matthew Cross -- JonesTrading -- Analyst

Hey guys. Congrats on the momentous quarter and the exciting new results. And just a couple of questions from me here. So among those out there who are still somewhat skeptical about the reversible BTK proposition and maybe believe that a covalent bonding is really necessary long term to ibrutinib's efficacy, effect durability seems to be the thing they really want to see. And as you've alluded to the FL patient who achieved a PR has been on drug for over two years now, which I think speaks to that somewhat, but we see now moving on with a Phase II dose. I was wondering if you could help guide us toward the duration of treatment we may see updated at ASH. Maybe the range of treatment duration expected by that time and median time on treatment we should expect.

Paolo Pucci -- Director and Chief Executive Officer

I think we can all calculate backwards for the patients that were present at ASH -- sorry, at EHA. Those patients had about 5 months of therapy. Some have received the second scan, some have not. Some have received a second scan after EHA, so probably they will receive another scan. So you're talking about nine to 12 months for those patients -- for the patients that were up-dosed from cohort 45. Some have been on therapy a long, long time. Once they were to move to 65, so probably longer than that. The lymphoma patient, for example, will be going at that point for 2.5 years, I think. And for the patients that were down-dosed from 75 to 65, then running the math, you have the most -- the most you can observe is 6 months. That's as much as I can say. And then the ASH poster will contain all the details.

Matthew Cross -- JonesTrading -- Analyst

Perfect. Yes, that's all the detail I needed. And then just following up a little bit on Hartaj's question related to 751. Are you expecting to present the full results of this dose escalation by that time? I wouldn't expect so, but maybe you could update us on where in that process for the monotherapy signal finding you are currently? And additionally, should we anticipate an update on combination efforts at that time?

Paolo Pucci -- Director and Chief Executive Officer

Yes. Yes. I think the plan is to present as much as we will have [Indecipherable] at that time as we have done for all the previous presentations. And we should be done with the preclinical work for at least one combination, the more -- the one that everybody would expect. Everybody is combining with the venetoclax, so we're probably starting there as well. But it'd be mostly preclinical work. I don't -- for 531. For 751, we're doing some combination as well. I thought you were asking 531. Sorry.

Matthew Cross -- JonesTrading -- Analyst

Sorry, on 751.

Dr. Brian Schwartz -- Chief Medical Officer

So 751, there's 4 groups. We'll present on all 4. The one group is in combination with fulvestrant. We have a group in combination with paclitaxel. Then there's one group with AKT 1, 2 and 3 mutations and amplifications, and one group with PI3K mutation. So you're correct. It will be very focused on female malignancies, and we hope to have that wrapped up by the end of the year.

Matthew Cross -- JonesTrading -- Analyst

Okay. Great. Thank you for the clarification. Really appreciate it and looking forward to seeing the results of the discussions with the FDA as far as 531. Thanks guys, and looking forward to the updates by ASH.

Paolo Pucci -- Director and Chief Executive Officer

Thank you.

Operator

[Operator Instructions] Your next question comes from Tony Butler with Roth Capital Partners.

Tony Butler -- Roth Capital Partners -- Analyst

Yes good morning. Paolo or Brian, the question relates to outside of CLL for 531. And I understand in the expansion study, you are looking at mantle cell and Waldenstrom's. So the question would be, would you predict that because I would assume you'd like indications there outside of CLL, which is -- clearly, there are broad labels better than a narrow one. Would you consider running either parallel trials? Or would it be a larger study? And I realized you may not have the answer, I'm more interested in how you're thinking about it as you walk into that conversation with the FDA a little bit later. And again, the rationale is indications outside of CLL in those NHL subpopulations.

Dr. Brian Schwartz -- Chief Medical Officer

So thank you so much for that question. I think it's really dependent on the population, and we have some internal ideas that we need to solidify. But just from a big strategic perspective, in -- for example, indications such as Richter's, one could easily expand the same study. In other indications, which are much more complicated. You would have to move it into another study. So we -- the way that our protocol is written now, you look at 10 patients for at least 3 responses. At 24 -- you add another 14 patients to get a total of 24 for the cohort if you meet the first part of that study. And then we would make a decision whether to go into a new study or continue that study as a potential registration path. We've decided to take the CLL C481S-mutant population out in a new type develop -- to discuss with the FDA as a stand-alone because that will really be our first indication. And you would want that as clean and as well thought through as possible.

Paolo Pucci -- Director and Chief Executive Officer

And therefore, we have the bulk of the data right now. And then as data accumulates in the expansion cohorts, then we will look into the other options. And Tony, we'll be able to discuss more thoroughly in the future as soon as we finished this second round of life cycle management for the drug.

Tony Butler -- Roth Capital Partners -- Analyst

Thank you Paolo. Thank you Brian.

Paolo Pucci -- Director and Chief Executive Officer

You are welcome.

Operator

[Operator Instructions] I am showing no further questions at this time. I would now turn the conference back to Paolo Pucci.

Paolo Pucci -- Director and Chief Executive Officer

Thank you, everybody, for your participation today, and enjoy the rest of the day. Bye-bye.

Dr. Brian Schwartz -- Chief Medical Officer

Bye-bye.

Operator

[Operator Closing Remarks]

Duration: 53 minutes

Call participants:

Peter S. Lawrence -- President and Chief Operating Officer

Paolo Pucci -- Director and Chief Executive Officer

Dr. Brian Schwartz -- Chief Medical Officer

Jonathan Chang -- SVB Leerink -- Analyst

Gregory Renza -- RBC Capital Markets -- Analyst

Chad Messer -- Needham & Company -- Analyst

Hartaj Singh -- Oppenheimer & Company -- Analyst

Matthew Cross -- JonesTrading -- Analyst

Tony Butler -- Roth Capital Partners -- Analyst

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