Earnings Call Transcript - FULC Q3 2025

Operator, Operator

Good morning, and welcome to the Fulcrum Therapeutics Third Quarter 2025 Financial Results and Business Update Conference Call. This call is being webcast live and can be accessed on the Investors section of Fulcrum's website at www.fulcrumtx.com and is being recorded. Please be reminded that remarks during this call may contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 and may include statements about the company's future expectations and plans, clinical development timelines, and financial projections. While these forward-looking statements represent Fulcrum's views as of today, this should not be relied upon as representing the company's views in the future. Fulcrum may update these statements in the future, but is not taking on any obligation to do so. Please refer to Fulcrum's most recent filings with the Securities and Exchange Commission for discussions of certain risks and uncertainties associated with the company's business. Leading the call today will be Alex Sapir, CEO and President of Fulcrum. Joining Alex on the call are Alan Musso, Chief Financial Officer; and Dr. Iain Fraser, Senior Vice President, Clinical Development. After providing updates on the company's key programs, there will be a brief Q&A in which the Fulcrum management team will be available for questions. With that, it's my pleasure to turn the call over to Alex.

Alex Sapir, CEO

That's great. Thanks, Shannon, and good morning, everybody, and thank you all for joining us today. The past several months have certainly been a busy, but more importantly, a very exciting time for Fulcrum marked by significant progress with our lead program, pociredir, for the treatment of sickle cell disease, which is an inherited blood disorder with a high unmet need, afflicting approximately 100,000 people in the U.S. and approximately 7.7 million people worldwide. There is an ever-increasing need for better treatment options for sickle cell disease patients who face not only an impaired quality of life due to chronic pain, fatigue, and acute complications like vaso-occlusive crises, but also very high rates of mortality. Patients with sickle cell disease face a greater than 20-year reduction in life expectancy, with a mortality rate that is 9 times higher than the general population. So as we continue on our journey to find better treatment options for these patients, we were very encouraged by the data presented this past July from the 12-milligram dose cohort of the Phase Ib PIONEER trial, which demonstrated the potential for pociredir to meaningfully improve outcomes for people with sickle cell disease. Now digging into that data a little bit more at a high level, pociredir demonstrated a dose-dependent and clinically meaningful increase in fetal hemoglobin, near pancellular induction of that fetal hemoglobin or HbF, improvement in key biomarkers of hemolysis, resulting in a subsequent increase in total hemoglobin and finally, encouraging reduction in vaso-occlusive crises or VOCs. Equally as important, pociredir continued to be well-tolerated, with all treatment adverse events being grade 1 in severity and all resolving during the treatment period without any disruption in study drug. These encouraging results achieved our target product profile criteria and position pociredir as a potentially best-in-class once-daily oral therapy for sickle cell disease. In August of this year, we submitted a protocol to the FDA to initiate an open-label extension or OLE trial, which will allow patients to continue receiving pociredir after completing the PIONEER trial, thus enabling longer-term evaluation of safety and durability of response. We're also pleased to share today that we have completed enrollment in the 20-milligram dose cohort with a total of 12 evaluable patients, and we will present data from this cohort at the American Society of Hematology or ASH conference in early December. The over-enrollment seen in the 12- and 20-milligram cohorts is a testament to the enthusiasm from the physicians involved in the study. Now with a number of these 12 patients starting drug in September, we expect approximately half of the 12 patients will have completed their day 84 visit and all patients will have completed their day 42 visit at the time of our data cutoff for the ASH meeting. Approximately 60% of the patients enrolled in this 20-milligram cohort have come from the U.S., with the remainder coming primarily from sites in Nigeria, which are newer sites that were not yet activated in time to participate in the 12-milligram cohort. The mean and median HbF levels at the start of the study for this cohort were 7.1% and 7.3%, respectively. We're also pleased to see patients remaining in the study with a greater than 90% adherence to the once-daily oral drug regimen. We continue to believe that inducing fetal hemoglobin is the optimal strategy for treating sickle cell disease. Evidence for this approach continues to grow as highlighted in our recent presentation earlier this month at the Annual Conference for the Academy for Sickle Cell and Thalassemia or ASCAT, where we demonstrated a quantitative correlation between increased fetal hemoglobin levels and reduced vaso-occlusive crises in sickle cell disease. This data, together with the 12-milligram cohort data that we shared in July, gives us confidence that pociredir has the potential to provide a differentiated therapeutic option for people living with sickle cell disease. We look forward to sharing additional results from the PIONEER trial at the upcoming ASH conference in December, and we plan to engage with the FDA for an end of Phase I meeting in Q1 of 2026 to align on the next stage of our clinical development for pociredir. Outside of pociredir, we continue to advance our program for the potential treatment of bone marrow failure syndromes such as Diamond Blackfan anemia, 5q deletion syndrome, Shwachman-Diamond syndrome, and Fanconi anemia, and we plan to submit an IND for these benign hematological conditions in the fourth quarter of 2025. Additionally, we recently presented preclinical data at ESMO this month for FTX-6274, an oral EED inhibitor, which demonstrated robust efficacy in castration-resistant prostate cancer models. We are encouraged by these findings, which highlight the potential of EED inhibition beyond our current hematology programs. With that overview, let me now turn it over to Alan Musso, our Chief Financial Officer, to run through the numbers for the quarter. Alan, over to you.

Alan Musso, CFO

Thank you, Alex. I'll now go over our results for the third quarter ended September 30, 2025. Our research and development expenses were $14.3 million for the third quarter of 2025 compared to $14.6 million for the third quarter of 2024. The decrease of $0.3 million was primarily due to decreased employee compensation costs as a result of the workforce reduction we implemented in September of last year, as well as a decrease in costs associated with our discontinued losmapimod program, partially offset by increased costs relating to advancing our pociredir program. The general and administrative expenses were $7.6 million for the third quarter of 2025 compared to $8.4 million for the third quarter of 2024. The decrease of $0.8 million was primarily associated with decreased professional services costs. The net loss was $19.6 million for the third quarter of 2025 compared to a net loss of $21.7 million in the third quarter of 2024. Now turning to the balance sheet. We ended the third quarter of 2025 with cash, cash equivalents, and marketable securities of $200.6 million compared to $241 million as of December 31, 2024. The decrease of $40.4 million is primarily due to the cash used to fund our operating activities. Finally, turning to cash guidance. Based on our current operating plans, we expect our existing cash, cash equivalents, and marketable securities will be sufficient to fund our current operating requirements into 2028, providing sufficient runway to substantially progress the clinical development of pociredir. With that, Alex, let me turn the call back over to you.

Alex Sapir, CEO

Great. Thanks so much, Alan. So before opening it up to Q&A with all of you, let me just conclude with a couple of final comments. Fulcrum has achieved meaningful milestones in the first three quarters of this year, with one of two planned data readouts for our lead program, pociredir, yielding very encouraging results in sickle cell disease. We are excited about the upcoming data readout at ASH and the opportunities ahead. We are fortunate to have a committed and talented team, coupled with a strong balance sheet, which positions us well to achieve our goal of delivering transformative therapies. With the opening remarks concluded, Shannon, let's go ahead and open up the line for questions.

Operator, Operator

Our first question comes from Kristen Kluska with Cantor Fitzgerald.

Kristen Kluska, Analyst

So I appreciate you disclosing the baseline characteristics for this cohort, and it looks pretty similar to what we saw for Cohort 3. So I'm wondering now that we know the baseline characteristics are relatively similar, now that we know the inclusion/exclusion criteria is more appropriate to compare apples-to-apples to these. How are you internally thinking about what is a win here and whether there are ways to measure if there's a dose response?

Alex Sapir, CEO

Yes, Kristen, it's a great question. We're going to handle that in two parts. I'll start, and then I'll turn it over to Iain because I do think there is a nuance for the approximately 50% of the patients for which we have full day 84 data. In terms of your question about what is a win, I would argue that we've already won. If you go back and you look at the 12-milligram cohort, we essentially achieved that target product profile that we felt we needed to achieve. We saw robust and rapid increases in fetal hemoglobin. We were nearing levels of pancellularity. We saw all of the markers of hemolysis going down as we would have expected and a subsequent increase in total hemoglobin nearing one gram per deciliter at the end of that 12 weeks. We saw a trend toward a reduction in VOCs, which obviously we need to confirm in a registrational study. The drug was extremely well tolerated, with all of the treatment-emergent adverse events being grade 1 in severity. So I would argue that we think we have won with the 12-milligram cohort. Now do we expect to see an increase in efficacy for the 20 relative to the 12? I think to answer that question, we really have to look at the healthy volunteer data in which we did show a dose response, again, in healthy volunteers at day 14 when measuring the fold induction of not the protein, not fetal hemoglobin, but the HBG mRNA. So I think based on that healthy volunteer data alone, we would expect 20 to outperform the 12, but we will know that in just a matter of weeks when we present this data at ASH. Iain, I do think it is probably important to talk a little bit about what we've seen with the first half of patients that started on the study and what we plan to present at ASH coming up in December.

Iain Fraser, Senior Vice President, Clinical Development

Yes, absolutely, Alex. Happy to address that question from Kristen. Based on when the last patients enrolled in the study, which was really towards the end of September, we expect that about half of the cohort will have completed the day 84 treatment visit at the time of the data cutoff for the ASH meeting data presentation. We expect that we should have 42-day visit available for all the patients, all 12 patients in that 20-milligram cohort. Interestingly, for those patients that have completed the full 84-day treatment period, and these were the earliest patients enrolled in the cohort, their baseline HbF have tended to skew on the lower end of the range. And so when we present that data, once we have the exact number of patients for that cutoff, I think it will be very important to accommodate the baseline for those 50% or so of the patients. Obviously, when we release the full day 84 data, once everybody has completed the full treatment period, that will reflect the 7.1%. So I just wanted to highlight that piece for the partial cohort data that we'll be sharing later this year.

Kristen Kluska, Analyst

Okay. And then the OLE study, I know in the past, you've noted it was something you're looking into, but I think this is the first time you've officially announced plans to start that. So I'm curious if there was interest from the patients that were in the trial, the physicians that have been investigators in the study, and also how this study may help with some of your future FDA discussions.

Alex Sapir, CEO

Yes. Great question, Kristen. And you're absolutely right. I think that the timing of starting the OLE study was quite frankly, really borne out of discussions that I had with several of the investigators in which they were expressing to me some of their patients' anxiety as those patients were getting closer and closer to day 84, and that we really didn't have anything at the time to offer them once the 12 weeks of dosing was up. In light of that, we decided to kick off this OLE study earlier than we had initially planned in order to allow some of those patients in the PIONEER trial to come off drug and to be able to roll into an open-label extension. I will tell you that in the conversations I've had with the investigators, they were quite happy that their voice was heard. And as a result of their voice being heard, we reacted accordingly. Iain, maybe you want to comment a little bit on how this data may help inform future discussions with the agency?

Iain Fraser, Senior Vice President, Clinical Development

Yes. Happy to do that, Alex. So that study is now being operationalized at the moment. One of the key aspects of that allowing us to continue treatment in those patients who were previously restricted to the three months contained within the PIONEER study allows us to generate additional safety data in that patient population, which I think overall for the program is going to be an important piece.

Operator, Operator

Our next question comes from the line of Joseph P. Schwartz with Leerink Partners.

Joseph Schwartz, Analyst

Congrats on the progress. I was wondering if you can give us any insight into the baseline level of HbF for the patients in the first half of the 20-milligram cohort that will get data on first and how it compares to the second half of patients who were enrolled?

Alex Sapir, CEO

Yes. Great question, Joe. I think to answer that, let me turn that one over to Iain. He's obviously been very close to each of these patients and very close to the study as well.

Iain Fraser, Senior Vice President, Clinical Development

Yes. As we said, Joe, the initial patients enrolled have trended lower than that 7.1% mean that reflects the entire 12 patients in the cohort. Because we don't know exactly where the cut is going to be with the 50%, depending on which samples get to the lab in times of the data cutoff, we haven't given the precise number there. We will obviously have that when we have those exact data, and we'll reveal that at the time of the data disclosure. But I think it is important just thinking ahead that overall, the trend there was just by chance, the initial patients enrolled in the cohort who on average had lower baseline than the 7.1% mean.

Alex Sapir, CEO

Yes. And maybe just to sort of add a little bit on what Kristen said earlier. Now we can look at sort of apples-to-apples. The fact that we now have a lower baseline for the roughly 50% of patients that will have completed day 84 by the time of ASH, we're looking really more like apples to oranges. The way to account for differences in baselines is to really look at that fold induction curve. That's one of the slides that we've included in our investor presentation comparing the 6 milligram to the 12 milligram. I encourage everybody to look at that because the fact that these patients—the first half of the patients had tended to be on the lower end of that average—we'll certainly look at that sort of full deduction because that is one way to normalize for differences in the baseline. Suffice it to say, Joe, clearly, the first half of the patients had a lower baseline. Then, obviously, the second half of those patients tended to be a bit higher than the 7.1% and 7.3% that we mentioned in our opening remarks.

Joseph Schwartz, Analyst

Okay. Great. And then how are you currently thinking about the addressable market following Oxbryta withdrawal? And can you give us some insight into your assumptions that go into your current estimate?

Alex Sapir, CEO

Yes, it's a great question. We have evaluated the market based on the data that exists in terms of what percent of patients have what number of VOCs in a given year. Our belief is that about 25% of patients have either four VOCs during a 12-month period or two VOCs during a 6-month period. But obviously, because our drug at present cannot be concomitantly used with hydroxyurea, some of those patients are currently on hydroxyurea. So we've taken a bit of a haircut. We estimate right now that roughly about 20% of the 100,000 patients in the U.S. currently meet the inclusion/exclusion criteria as defined in the PIONEER trial. Obviously, when we engage those conversations with the agency, one of the questions we will be asking them is the potential to expand to a broader set of patients. But I will say, overarchingly, our overarching goal with this program is to get this drug to market as quickly as possible because, as you mentioned, patients don't have the treatment options that they did several years ago with the withdrawal of Oxbryta, the cell and gene therapy market has not been commercially successful due to costs, risks, and complexities. Our overarching goal and something that I continue to stress to this team is to make sure that we get this drug to market as quickly as possible to help as many patients as we can—not just in the U.S. but globally for the 7.7 million patients who have sickle cell disease outside of the U.S.

Operator, Operator

Our next question comes from the line of Corinne Johnson with Goldman Sachs.

Corinne Johnson, Analyst

Maybe two from us. As you think about the 20 mg dose cohort and consider the full data set that you get later, what do you need to see from that in order for that to be the go-forward Phase III dose considering your point earlier that you've kind of already won with 12 mg? Also, what's included in the cash runway guidance with respect to the scope of the Phase III program? Could you speak about duration and endpoints that would be included in that guidance?

Alex Sapir, CEO

Yes. Two great questions, Corinne. Maybe I'll turn it over to Iain for the first half of that question and then turn it over to Alan for your second question.

Iain Fraser, Senior Vice President, Clinical Development

Thanks, Corinne. We'll be looking across the totality of the data in the 20 mg cohort. We would be delighted to see that the efficacy endpoints in the study indicate improvement, with an ongoing favorable tolerability and safety profile and good adherence to the study drug. We'll be paying close attention to HbF levels with particular emphasis on the dose response as measured by a fold induction of HbF. Based on the induction of HBG mRNA that we observed at 14 days in the prior healthy volunteer study, we expect the 20 mg dose cohort could outperform that observed at the 12 mg cohort. One key learning from the 12 mg data readout as we compare it to the previous 6 mg readout is that it was important to evaluate that as a fold induction because the baselines across those cohorts were different, which plays a big role. We'll also be looking to ensure we see a response in HbF across all patients, focusing on the extent to which we achieve pancellularity and improvements in markers of hemolysis, reductions in anemia, and trends towards reductions in VOCs, while recognizing that the study isn't powered specifically around the VOCs. Overall, we expect to see continued safety and tolerability as we've observed in both healthy volunteers and patients treated to date.

Alan Musso, CFO

On your question on cash runway and guidance, that is a full success sort of forecast for the organic program. We anticipate moving forward with pociredir to the next trial. We've talked to a number of CROs who have mapped out what we think that could look like and feel very good that that is fully accounted for as we move forward with the cash guidance. We also anticipate moving forward with the program for DBA and other bone marrow failure syndromes as well as continued progression of work in the preclinical phase. It's sort of an all-in full success view of what's going on in the pipeline at this time.

Operator, Operator

Our next question comes from the line of Edward Tenthoff with Piper Sandler.

Edward Tenthoff, Analyst

Thanks for all the detail that you provided. Really excited to see the results down in Orlando in just a couple of weeks. My first question really has to do with what do you think is actually going to be published in the ASH abstract release? It probably won't be the full data set, but I want to get a sense of what might actually be in the abstract versus what you laid out in terms of what you'll be presenting at ASH. Do you think that will be a poster or an oral presentation?

Alex Sapir, CEO

Yes. Two great questions, Ted. Yes. So the abstract that will be made public by ASH next Monday, November 3, does not include any of the 20 mg data. It does include data from the 12 mg cohort, but there's no data from the 20 mg cohort. It was really a placeholder to ensure that we could get a spot at the ASH conference. In terms of whether it's a poster or oral, that will be released by ASH on Monday. We think it's most appropriate and prudent to let ASH share all the abstracts in sickle cell and hematology that have been accepted and those accepted for poster or oral presentations. So stay tuned; we'll know more on Monday.

Edward Tenthoff, Analyst

Great. And then one quick follow-up, if I may. I appreciate sort of the extra color with respect to the early patients maybe being on the lower baseline side, and we saw that the higher the baseline, the more HbF, the more response. When you look at all of these factors, what really is most important to KOLs, regulators, and yourself in terms of defining the activity of pociredir? Is it the percent HbF? Is it the percentage of patients above 20? Is it the total HBG? What is the most important, or is it the totality of all that data?

Alex Sapir, CEO

It's a great question. I’ll start, but Iain will probably have a better answer than I will. I've had a lot of conversations with investigators, not only in the U.S. but outside of the U.S., since the data we released in July around the 12 mg. When I pressure tested your question with them, asking which of the five parameters—fetal hemoglobin levels, pancellularity, decrease in markers of hemolysis, subsequent increase in total hemoglobin, and a trend towards a reduction in VOCs—they find most important, they essentially came back, stating it's really about the totality. You can't pinpoint one versus another. We know fetal hemoglobin will reduce VOCs; we know an increase in total hemoglobin will reduce fatigue that patients feel, which is very important for them. We know the increase in fetal hemoglobin is imperative that that happens at a pancellular level. So I really think it's each one of those criteria that we shared in July that impresses them with the overall profile of the product. Iain, anything to add?

Iain Fraser, Senior Vice President, Clinical Development

The only thing I would add is that getting patients into that 20% plus range is associated with very dramatic benefits in the outcomes of the disease. That's important. We showed in the 12 mg dose that about half of the patients were able to achieve that, which I think is particularly encouraging. I do want to also recognize that there are some patients who have very low baseline HbF in the 2%, 3%, 4% range, often associated with very severe disease. For those patients to get to the 20s is a much bigger climb. However, providing them with a threefold induction over their baseline is considered a significant benefit.

Edward Tenthoff, Analyst

When would we get the final PIONEER data set? Do you think that would be all the way at EHA next year?

Alex Sapir, CEO

It's a great question, Ted. Most patients should be wrapped up with their dosing by the very end of this year. We expect to have the full data set to share with everybody sometime in the first quarter of next year.

Operator, Operator

Our next question comes from the line of Matthew Biegler with OPCO.

Matthew Biegler, Analyst

I had a follow-up on the demographics. I know we've talked about it a bit, but it sounds like if I'm reading between the lines from Iain's comments earlier that the Nigerian patients might be a bit sicker? Or would you say overall, the two cohorts are largely similar in terms of baseline severity, and the heterogeneity in baseline hemoglobin is just random variation?

Alex Sapir, CEO

Yes, it's a great question.

Iain Fraser, Senior Vice President, Clinical Development

Yes, Matt, thanks. I wouldn't assume that it's the Nigerian patients specifically that are more severe. It's really just the way in which patients came into this cohort that affects their baseline. I think it's by chance that has led to lower averages among some of the earlier patients enrolled in the cohort. I don’t think it's a geographical aspect.

Matthew Biegler, Analyst

Got you. Maybe a bigger picture question for me is then, assuming the 20 mg looks good or maybe marginally better than the 12, do you keep dose escalating? Or do you think that 20 is your recommended Phase II dose?

Alex Sapir, CEO

Yes. Iain, do you want to take that?

Iain Fraser, Senior Vice President, Clinical Development

What we've indicated previously is that the current protocol allows us to dose escalate up to 30 mg. Based on what we saw at the healthy volunteer level, we did not see much increment at the 14-day mark when escalating from 20 mg to 30 mg. Based on that, along with the promising data we've already seen at the 12 mg cohort and expectations around the 20 mg cohort, we're not planning to proceed with that 30 mg dose.

Operator, Operator

Our next question comes from the line of Andres Maldonado with H.C. Wainwright.

Andres Maldonado, Analyst

Congrats on the progress. One quick one for me. When you have the 20 mg data in hand, how do you feel pociredir's efficacy will generalize in less severe patients? Can you walk us through the mechanistic argument for why that will be?

Alex Sapir, CEO

Yes, it's a great question, Andres. Iain, do you want to take that?

Iain Fraser, Senior Vice President, Clinical Development

Thanks, Andres. We do have some data from the early part of the study in a less severe patient population or at least in a patient population that didn't have the same severity criteria that the 12 and the 20 mg cohorts have had. In the early part of the study, we observed, albeit at the lower end of the dosing scale of 6 mg and 2 mg, some robust effects on HbF induction in that cohort, including some patients who were on concomitant hydroxyurea at the time. So we don't expect a difference in pociredir's ability to induce HbF based on the patient's disease severity. We also see induction in healthy volunteers and individuals with sickle trait who are heterozygous for the sickle mutation.

Operator, Operator

Our next question comes from Luca Issi with RBC.

Luca Issi, Analyst

Congrats on all the progress. Maybe Iain, a quick one actually on safety. Obviously, it is really hard to prove a negative here. What is the FDA telling you about how many patients and maybe how long of a follow-up are they hoping to see in order to feel comfortable about safety here? Any context would be much appreciated. And then maybe just a quick one on the plan going forward here. Can you talk about the clinical plan beyond this Phase Ib? Should we assume that you start directly a registrational trial after this? Or is it still possible that you'll need an intermediate Phase II before going into a registrational trial? Any color would be appreciated.

Alex Sapir, CEO

That's great. Yes, Luca, two great questions. Iain, maybe I'll turn that one over to you.

Iain Fraser, Senior Vice President, Clinical Development

As we've indicated previously in our discussions with the agency, there's obviously a context of risk and benefit. In our discussions with them, the plan was to complete the PIONEER study as we approach the end of that now and to bring those data, along with all the data from our Phase I program, back to the agency to discuss next steps with them. There was no specific numerical criteria provided around that; obviously, we expect the ongoing favorable safety and tolerability profile will be important, but that also needs to be contextualized with the potential benefit that the therapy provides. There are no specific criteria outlined there, but certainly the plan is to bring that back to the agency for that discussion. The next study design will be based on our discussions with regulators and influenced by the data emerging from the PIONEER study and our other Phase I studies. We believe that there's potential for the next study to be a registrational study in the context of what's previously been used for agents inducing HbF. We expect that a clinical endpoint such as VOC reduction could likely be the primary clinical endpoint in that study. However, there's also potential for an earlier look at the study and interim look where HbF could be evaluated as a surrogate endpoint for potential accelerated approval in sickle cell disease. But of course, all this will be discussed with the regulators with the context of the full PIONEER data set before initiating the next study.

Operator, Operator

Our next question comes from the line of Tazeen Ahmad with Bank of America.

Tazeen Ahmad, Analyst

I have a couple. Just in terms of the rules regarding embargo for ASH, when the abstracts get released on Monday, is there a requirement that you would not be allowed to talk about the additional data that would be shown at the meeting itself? Would you be in a position to potentially release the new data before the actual presentation, whether it be a poster or an oral at the meeting? And then just to clarify a couple of points from earlier, what additional data from prior cohorts are you expecting to show, if any, at your presentation at ASH? Basically, I want to get a sense of what metrics to expect beyond what we saw for Cohort 3.

Alex Sapir, CEO

Yes. Two great questions, Tazeen. I'll start and then I'll turn it over to Iain for maybe additional color. So as I mentioned in the question that Ted asked, the abstracts when they get released by ASH on or specifically the PIONEER pociredir abstract released on Monday will not have any of the 20 mg data. We won't share any of that data until it gets presented in the poster session or in the oral session as you mentioned. We'll find out on Monday whether that data has been accepted. However, we may have an opportunity to do a call after that data is presented over the weekend or shortly thereafter, to provide a bit more color on that information, so stay tuned for that. Regarding additional data, I'll have Iain answer that question.

Iain Fraser, Senior Vice President, Clinical Development

As you will recall, for the 16 patients in the 12 mg cohort we previously presented at the end of July, we did not have all the follow-up data at that time. There is a 4-week safety follow-up where those patients are no longer on the drug. That follow-up data will be presented at the ASH presentation along with the full safety data set. We provided all the safety data available at the time of our previous release, including some AEs that occurred during that safety follow-up period. We will round that out to make that a more complete data set around the 12 mg cohort.

Alex Sapir, CEO

Shannon, are there any more questions in the queue?

Operator, Operator

I'm currently showing no further questions at this time. This does conclude today's conference call. Thank you all for your participation. You may now disconnect.