Forma Therapeutics Holdings, Inc.

Welcome to the Forma Therapeutics First Quarter 2022 Financial Results and Business Update Conference Call. All participants are currently in listen-only mode. Following management's prepared remarks, we will hold a Q&A session. To ask a question at that time, please press the star key followed by 1 on your touchtone phone. If anyone has difficulty hearing the conference, please press star 0 for operator assistance. As a reminder, this call is being recorded. Today, May 6, 2022. I would now like to turn the conference over to Mario Corso. Please go ahead.

Thank you, Gigi. This is Mario Corso, Senior Director of Investor Relations at FORMA. Good morning to our listeners, and welcome to today's call to review first quarter 2022 financial results and business update. On this call, I'm joined by Frank Lee, our President and Chief Executive Officer, and Patrick Kelly, our Chief Medical Officer, Dave Cook, our Chief Scientific Officer, and Todd Shigog, our Chief Financial Officer. Before we begin, I'd like to caution listeners that comments made and financial information provided during this conference call include certain statements that are estimates, beliefs, forward-looking, and are subject to various risks and uncertainties. Any statements made during this call that are not statements of historical or current facts are intended to be forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. We want to emphasize that such forward-looking statements reflect our current expectations and assumptions regarding timing, enrollment, success and data announcements of our current ongoing clinical trials, therapeutic potential, hypotheses of mechanisms of action and clinical benefits, and safety of our product candidates, planned regulatory submissions, our financial condition and capital requirements, our business operations, development plans, the potential impact of COVID-19 on our business, and clinical development and relationships with third parties and collaborators, and are neither predictions nor guarantees of future events or performance. Actual results could differ materially from those stated or implied by these forward-looking statements, due to risks and uncertainties associated with our business, including those under the heading titled Risk Factors in our annual report on Form 10-K for the year ended December 31st, 2021, and in our quarterly report on Form 10-Q for the quarter ended March 31st, 2022, that will be filed with the SEC today, and in subsequent reports, including our current reports on Form 8K. The company disclaims any obligation to update or revise any forward-looking statements, except as required by applicable law. Before turning the call over to Frank, I'd like to mention our upcoming R&D Day with investors that was outlined in today's release. This meeting will take place virtually on May 26th from 8 to 10 a.m. Eastern Time. Further information will be made available on our website, www.formatherapeutics.com. With that, I will now turn the call over to Frank, our President and Chief Executive Officer.

Thank you, Mario. Good morning, everyone, and thank you for joining us today. The first quarter of this year marked strong progress in our ongoing clinical programs. We continued enrollment in our Phase 2-3 trial of atavlopivap in sickle cell disease, the Hibiscus Study, and are pleased to say we're on track for the first interim analysis in the latter part of this year. We're also on track with enrollment in the Phase 1 trial of FT7051 in metastatic castration-resistant prostate cancer. We expect to soon be able to identify a dose for further expansion. We're initiating two Etabo PVAP Phase II trials this year. The first is underway in transfusion-dependent and non-transfusion-dependent thalassemia, as well as sickle cell disease patients who are transfusion-dependent. Sickle cell disease patients who are transfusion-dependent represent approximately 20% of the overall sickle cell disease patient population and represent an area of substantial unmet need. The second trial will begin in the second half of this year in lower risk myelodysplastic syndrome, MDS. Low risk MDS represents another area of substantial unmet need. Chronic transfusions to treat the symptomatic anemia associated with MDS is common practice. Whether a patient is requiring blood transfusions to treat their hemolytic anemia as in the case of thalassemia or sickle cell disease, or to treat their anemia due to poor production, as in the case of MDS, iron overload remains a substantial and significant risk. We believe Etabopivac's unique mechanism of action may improve the symptomatic anemia in all these indications, reducing the need for transfusion therapy and reducing the risk for iron overload. As we look forward, we have some important upcoming events. On May 26th, we plan to host our first Research and Development Day. At this event, we will provide the first look into our research portfolio, including a compound which is currently in IND-enabling studies. In addition, we'll provide additional details on the non-sickle cell disease at TAVLA-PVEP programs and discuss the emerging science of red blood cell health. We'll also share a trial update on our FT7051 program. And towards the end of the year, we're on track to reach interim analysis one from the hibiscus study and also to show early data from the transfusion study. Being a trusted partner to the patient communities we serve is an important corporate goal for FORMA. Among other initiatives, we launched our FORMA Bridge program at the end of last year. The transition from pediatric to adult care can be very challenging. FORMA Bridge was designed to support this important transition, and I'm very pleased to report that there have been a high level of interest in the program, and we received a number of proposals from a mix of healthcare institutions, patient organizations, and community-based organizations. Grant awards are expected to be announced in the coming months. We've had a strong start to the year, and we're well-positioned to deliver on our goals. We are well-capitalized with over $441 million in cash. We have a differentiated and growing clinical stage pipeline, and we have a highly engaged group of employees who embody the idea of the science of giving a damn. In closing, I'd like to recognize the remarkable efforts of our investigators and patients for their support and contributions in advancing our purpose to transform the lives of patients living with rare hematologic disorders and cancers. I'll now turn over the call to Pat to provide a brief update on our development programs.

Thank you, Frank, and good morning, everyone. I will provide a brief overview of our development programs for Atavo PVET and FT7051. Etabopivac is our once-daily selective PKR activator with a distinct multimodal mechanism of action to improve oxygen binding and reduce hemoglobin S polymerization via a decrease in 2,3-TPG, and also to repair the membrane damage of red cells via an increase in ATP. We have completed our Phase I trial of Etabopivac in sickle cell disease, and the results were presented in December at the ASH annual meeting. We have generated a compelling data set based on measures of response in hemoglobin, reticulocytes, and hemolysis, and a comprehensive data set demonstrating an improvement in measures of red blood cell health, such as deformability and hydration. We believe the data also suggests that daily etabopivac can have important systemic effects, such as reducing inflammation, hypercoagulability, and hypoxia. We are very encouraged by the VOC trend analysis from the phase one trial, demonstrating with greater than three years of Etabo PVAST exposure, there was a 68% reduction in the annualized rate of VOCs resulting in hospitalization compared to the patient-reported VOCs in the 12 months prior to trial enrollment. We have also observed improvement in the paid events that do not qualify as VOCs. Looking ahead, we have initiated a Phase II trial of Atavo PVET to explore the potential in transfusion-dependent sickle cell disease and also both transfusion-dependent and non-transfusion-dependent thalassemia. As Frank noted earlier, up to 20% of patients with sickle cell disease are on a chronic blood transfusion program, most commonly for the prevention of stroke, a devastating and far too common consequence of their sickle cell disease. This trial will enroll a total of 60 patients across the three treatment arms with response measured by the proportion of patients with a reduction in red blood cell transfusion requirements compared to prior history or a hemoglobin increase over baseline from those patients with thalassemia not receiving chronic transfusions. We expect to have some initial results from this trial in late 2022. For our FT7051, program or our CBP-P300 inhibitor in development for the treatment of metastatic castration-resistant prostate cancer, the ongoing Phase I trial has continued dose escalation. We intend to provide a trial update later this month during our R&D day with more comprehensive results for the trial expected later this year. With that, I will now turn the call over to our Chief Financial Officer, Todd.

Thank you, Pat, and thank you, everyone, for joining us on today's call. I will first spend a few minutes discussing our financial results for the quarter ended March 31, 2022, and then discuss our cash position and outlook. Our net loss for the quarter ended March 31, 2022 was $41.1 million, which compares with a net loss of $36.0 million for the quarter ending March March 31, 2021. The increased net loss was driven by increased spending in support of our preclinical and clinical development programs, as well as employee hiring to support our operations. Research and development expenses were $31.3 million for the quarter ended March 31, 2022, compared to $26.3 million for the quarter ended March 31, 2021. The increase was primarily attributable to the increase in research and development staff to support the advancement of Atavo PVAT and other programs, an increase in equity-based compensation, the conduct of our Phase 2-3 trial in sickle cell disease patients, and study startup costs related to our Phase 2 trial in thalassemia. General and administrative expenses were $13.1 million for the quarter ended March 31, 2022, compared to $9.9 million for the quarter ended March 31, 2021. The increase was primarily attributable to equity-based compensation, costs due to executive and staff hiring, and other related general and administrative costs. Our cash, cash equivalents, and marketable securities balance as of March 31, 2022 was $441.3 million compared to $490.3 million at year-end 2021. Cash use reflects operating expenses and working capital requirements to support our operations. Overall, we continue to be in a strong financial position with funding through the third quarter of 2024. Gigi, we may now take questions.

As a reminder, to ask a question, you will need to press star 1 on your telephone. To withdraw your question, press the found key. Please stand by while we compile the Q&A roster. Our first question comes from the line of Maury Raycroft from Jefferies. Your line is now open.

Hi, good morning. Congrats on the progress, and thanks for taking my questions. I did want to check in on just the regulatory front. for sickle cell disease and see if you can comment on just any discussions to support the 24-week hemoglobin response as a surrogate endpoint for an accelerated approval path. And also if you can clarify if you need to wait for the first interim to be completed before you can finalize those plans.

Thanks for the question, Mari. It's Frank Lee here. We continue to have discussions and working through the regulatory path And so no further updates at this time. It's very consistent with what we've communicated before.

Got it. Okay. And for the R&D day coming up, looking forward to the 7051 data there, I'm wondering if you can talk a little bit more about where you're at with the dose escalation. And just wanted to clarify if the expansion cohort, if that has started yet. or will that be – if you can put a finer point on when that could happen.

Yeah, so we'll provide some additional details, Mari, on the progress there. And, you know, I think this will be more of a trial update on where we are, and we are making good progress. And so I'd say stay tuned for the May 26th R&D day. We'll provide additional details there.

Okay. Maybe last question, just we've talked about the ARV7 patient population in the past, and just wondering if you can say if we should expect an update in that population at the R&D day, and how many ARV7 patients that you could have data in?

Yeah, maybe, Pat, you could speak to this a little bit. I know we've had good success in enrolling patients overall in ARV7, so maybe you can speak to this.

Yeah, just briefly, you know, more detail at the end of the month, but yeah, not surprisingly, with the understanding that this targeted therapy should have activity in that population, you know, we continue to monitor patients as they enroll, looking for that mutation, and we're seeing a fairly robust enrollment into this phase one. of patients with detectable ARV7 disease.

Great. Okay, thanks for taking my questions. Thanks, Mari.

Thank you. Our next question comes from the line Mark from Oppenheimer. Your line is now open.

Hey, good morning, guys, and thanks for taking the questions. Just a couple quick ones from me. First of all, I wanted to confirm with the upcoming interim data from a TAVO pivot, the IA1 interim readout. This is where you'll be announcing which dose between the 200 and 400 milligram once daily dosing you'll be advancing into the Phase III portion of the trial. Is that correct?

Yes, it's dose selection. Pat, you can speak a little bit more about this one.

Yeah, that's right. As a reminder, the phase two portion is looking at 200 milligrams versus 400 milligrams versus placebo on a daily basis. And that analysis will be out at the end of the year.

Okay. And at any point during phase one dose escalation, Did you encounter any evidence of thrombocytopenia? I'm just asking because we recently learned of another PPR activator that hit thrombocytopenia and DLT during dose escalation.

Yeah, no, in healthy volunteers as well as sickle cell patients, you know, the only hematologic effects were the hemoglobin improvement and the, you know, improvement in absolute reticulocyte counts. Some effects on improving white cell counts as well, but no effects on platelets. And this is the second PKR activator with that. As you might recall, another company had a molecule that in healthy volunteers there was problems with thrombocytopenia as well as liver toxicity that made that program discontinued.

Okay, that's good color. And maybe just a quick one for Todd. There was a pretty sharp reduction in R&D expense from the previous quarter. Just, you know, how should we be thinking about this line item going forward in 2022?

Yeah, no, thanks, Mark. Yeah, the fourth quarter was a bit higher, as you pointed out, versus what we saw in the first quarter. And that's just timing of development activities. You know, manufacturing in particular can be a little bit lumpy depending upon when actual work occurs. But, you know, as we, you know, trend towards plan, you know, I would expect to see it, you know, gradually increase with the advancement of the programs, with the addition of staffing and so forth to execute on the program. So, you know, I think the fourth quarter was a little bit of a blip, but I think you can look at, you know, the trend increasing from Q1.

All right, super helpful. Thanks for taking the questions, and congrats on the forward progress. Thanks, Mark.

Thank you. Our next question comes from the line of Prakhar Agrawal from Cancer. Your line is now open.

Hi, congrats on the call, and thanks for taking my questions. So firstly, on the new molecule that you plan to disclose during the R&D day, I know there will be more details there, but as a teaser, can you maybe provide more details on whether this is focused on oncology or hematology, and why are you excited about this molecule?

So, Prakash, thanks for the question. It's in oncology, and, you know, we're going to go through the program in a lot more detail, but we'll just keep it that way. It's in oncology, and that's the broad guidance we've been providing so far.

Thanks. And lastly, on FT7051, Any updates on potential combination therapies here and maybe even moving on to earlier lines? What combos do you think might make sense and when can we expect to hear more? Will it be after the full data disclosure at the end of the year or could it be sooner? Thank you for taking my questions.

And maybe Dave, you take that one. Sure. You know, I think there's always an interest in moving into earlier line patients. Patients in the phase one thus far have had a long history of pretreatment, and many of them experienced chemotherapy, as we illustrated from the cohort last fall. As we move into earlier lines, we will consider combinations, but I don't think we'll have anything definitive to say until the full data set at the end of the year.

Thank you. As a reminder, to ask a question, you will need to press star 1 on your telephone. To withdraw your question, press the pound key. Our next question comes from the line of Robin Jarner from Craig Hallam. Your line is now open.

Congratulations on the progress thus far. I wanted to ask you about the data update for the transfusion-dependent sickle cell disease and the thalassemias. What kind of initial results might we expect for you to report by the end of the year?

Thanks for the question, Robin. These will be early results, and so I wouldn't expect anything definitive.

Okay, thank you for that. Yeah. Okay. And then also for the pediatric sickle cell disease and also MDS, what are the timelines for those new studies? When can we expect to see data and the nature of those readouts?

So we'll be, you know, we're initiating the pediatric studies. And so, you know, that'll take some time, as you know, to recruit. So I don't think we've put definitive timelines out there yet, but we've initiated those studies.

Thank you.

Thank you. At this time, I'm showing no further questions. I would like to turn the call back over to Frank Lee for closing remarks.

All right. Well, thank you everybody for participating on today's call. I'd like to really thank our employees, our investigators, our patients who continue to support our research and development pipeline progress. And, again, I want to thank all of you for participating on today's call. Thank you very much.

This concludes today's conference call. Thank you for participating. You may now disconnect.