Forma Therapeutics Holdings, Inc.

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Good morning.

My name is Josh, and I will be your conference operator today. At this time, I would like to welcome everyone to the Forma Therapeutic Second 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. Thank you. I would now like to turn the conference over to Dr. Adam Biro, Senior Vice President at Kendall Investor Relations. Please go ahead.

Good morning, and welcome to Forma's second quarter 2022 financial results and business update conference call. Before we begin, I encourage everyone to go to the news and investors section of formatherapeutics.com to find the press release detailing the company's second quarter 2022 performance that we will discuss today. I would like to remind you that any statements we will be making that are not statements of historical or current facts are based on our current expectations and beliefs. and are intended to be forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements are neither predictions nor guarantees of future events or performance and include those regarding timing, enrollment, success and data announcements of our current ongoing clinical trials, therapeutic and market potential, hypotheses of mechanisms of action, clinical benefits, safety of our product candidates, expansion and development of clinical programs, and our financial condition and capital requirements. Our actual results may differ materially from those expressed or implied by any forward-looking statements as a result of various risks and uncertainties associated with our business, including those under the heading Risk Factors in our quarterly report on Form 10-Q for the quarter ended June 30, 2022 that will be filed with the SEC today and in subsequent reports, including our current reports on Form 8-K. On today's call, I am joined by our President and Chief Executive Officer, Frank Lee, Dr. Tina Melian, Head of Research and Development, Dr. Cameron Trenner, Executive Director, Clinical Development, Dr. Dave Cook, our Chief Scientific Officer, and our CFO, Todd Chagall.

I will now turn the call over to Frank.

Thank you, Adam. Good morning, everyone, and thank you for joining us today. Forum Run aims to become a recognized leader in rare hematologic diseases and cancers and a trusted partner to the communities we serve. We're advancing a robust pipeline led by TABWPVAT, are well capitalized, and last month we're pleased to announce the appointment of two new members of our executive team. Dr. Tina Melian joins us as Executive Vice President, Head of Research and Development with over 20 years' experience developing patient-centric therapies for rare and orphan disease indications, spanning multiple modalities in all phases of development. And Linnea Spezi joins us as Senior Vice President, Chief Human Resources Officer, with over 25 years of HR leadership experience in life sciences and healthcare services. I'm confident that Tino and Linnea will help drive our next phase of growth into late-stage clinical development and commercialization as we prepare to deliver meaningful medicines to patients we serve. We also recently announced that Dr. Selwyn Vickers, a member of our board, and Dr. Patrick Kelly, our chief medical officer, have left FORMA. I'd like to congratulate Dr. Vickers on his new position as president and CEO of Memorial Sloan Kettering Cancer Center. As part of this transition, he is stepping down from public company boards. Pat joined FORMA approximately seven years ago, and FORMA is primarily a research and early development company. Pat's passion and expertise are in translational medicine and early clinical development. We're grateful to Pat for his contributions to our programs and for establishing a world-class hematology development team. comprised of six MDs who have deep experience leading centers that treat patients with sickle cell disease, thalassemia, and MDS. We're now moving toward global late-stage development and ultimately commercialization. Tino Melian, our head of research and development, has a lot of expertise in this area, and we're very pleased to have him with us. Pat will continue to serve as a strategic advisor to FORMA and T&L will assume direct responsibilities for research, development, and medical teams. Pat has no immediate plans beyond spending time with his family and continuing to support FORMA through a consulting capacity. It's been my privilege to work alongside Dr. Vickers and Dr. Kelly, and I'm deeply thankful for the important contribution that they've made to our organization. We wish them all the best in the future, and will continue to maintain their patient-centric focus in all that we do. We now have in place an executive team with experienced leading organizations through global late-stage development and commercial launch. We brought on board John Bishop, our head of technical operations earlier this year. As you know, we recently brought on board Tino and Linnea as well. Other members of the executive team have similar experience. And I'm very proud of the executive team we've assembled for the next chapter of our growth. I'd like to now invite Tino, our Executive Vice President of Research and Development, to provide a bit more detail on his background and plans to drive our R&D organization forward. Tino?

Thanks, Frank. I'm delighted to be here.

In my 20 plus years of experience in the biopharmaceutical industry, including experience leading global late-stage rare disease trials, it is rare that I see a company as poised for growth and expansion as FORMA is today. Since joining last month, I've been impressed by the level of scientific rigor across the organization, the compelling data generated to date across our clinical programs, and the potential of the Tableau PVET, to truly transform the treatment of simple cell disease, thalassemia, and lower risk MDS. The quality of these programs rests on the shoulders of strong leadership and deep scientific expertise. I want to thank Pat for helping to build a world-class hematology development team and for advancing the atypical PVET program through the generation of robust, clinically meaningful data that has positioned FORMA for global, late-stage development and commercialization. Looking forward, my priorities are to drive operational excellence across the atypical PVET development program as we advance towards a potential approval in sickle cell disease, and data readouts in Phase II studies expanding its therapeutic potential to additional populations, including transfused populations in sickle cell disease, thalassemia, and lower-risk MDS. In parallel, I aim to selectively expand our preclinical portfolio, recognizing the particularly strong foundation that FORMA has built in hematology and oncology.

With that, let me now turn the call back over to Frank.

Thanks, Tino. Earlier this week, we were pleased to announce that we've entered into an exclusive license agreement with Rigel Pharmaceuticals to develop, manufacture, and commercialize Lutacidinib, our mutant IDH1 inhibitor for the potential treatment of mutant IDH1 positive relapsed or refractory acute myeloid leukemia. Based on the strength of the Registrational Phase II dataset, we believe elutisidinib has the potential to represent an impactful differentiated therapy for relapsed refractory AML. The FDA has accepted our NDA for elutisidinib with a PDUFA action date of February 15, 2023. Under the terms of the agreement, formal receive an upfront payment of $2 million and is eligible to receive an additional $17.5 million upon the achievement of certain near-term regulatory approval and first commercial sale milestones. A total of up to an additional $215.5 million in connection with the achievement of certain development and commercial milestones and potential tiered royalties in the low teens to mid-30s. Given Rigel's focus on hematologic diseases and cancers and the strength of their commercial infrastructure, we are confident that they are the right partner to deliver elutisidinib to patients in need. More broadly, this agreement highlights our R&D capabilities and will enable us to further sharpen our focus on advancing our pipeline, which we highlighted in detail at our inaugural R&D day in May. This event included a presentation of encouraging analyses from our phase one study of atavipivet and sickle cell disease, indicating a reduction in reported pain-related adverse events, which we believe further underscores the potential of atavipivet to reduce vaso-occlusive crises in ongoing phase two, three hibiscus study. Enrollment in hibiscus study is on track with interim analysis one expected near the end of this year. As a reminder, this analysis will enable dose selection for the Phase III portion of the study. Based on continuing discussions with the FDA, we continue to believe that accelerated approval remains a viable path with the clinical data package we expect at the time of submission. As we outlined at R&D Day, we believe that a TAPA-PVAS multimodal mechanism of action has the potential to improve symptomatic anemia across multiple indications beyond sickle cell disease. We continue to advance the Phase II Gladiola study of atavipivac and transfusion-dependent and non-transfusion-dependent thalassemia, as well as sickle cell disease patients who are receiving chronic transfusions. As a reminder, approximately 20% of the overall sickle cell disease patient population is transfusion-dependent. and represents an area of substantial unmet need. We expect initial results from the study by the end of the year. Also by the end of the year, we expect to further expand the ATTAVA PVAD development program to include phase two studies in pediatric sickle cell disease and lower risk myelodysplastic syndrome, or MDS. Similar to hemolytic anemias, chronic transfusions are the common practice in MDS treatment. paradigm and can result in iron overload. We look forward to evaluate the potential of the tablet PVAT to target the underlying pathophysiology of low-risk MDS and reduce transfusion burden in this area of high IMET need. Moving to our Phase 1 study of FT7051 and metastatic castration-resistant prostate cancer. As discussed at R&D Day, we plan to evaluate an alternative dosing schedule in a less heavily pretreated patient population and are currently processing the protocol amendment. Based on this protocol amendment, we now expect to share additional data from the study in the first half of next year. At FORMA, being a trusted partner to the patient communities we serve is central to our long-term corporate vision. For patients with sickle cell disease, the transition from pediatric to adult care can be very challenging. To help patients navigate this important transition, we launched our Form a Bridge program at the end of last year. And I'm pleased to report that there's been a high level of interest in the program. We've received a number of proposals from a mix of healthcare institutions, patient organizations, and community-based organizations. And we expect to announce grant awards in the coming months. With $395 million in cash, we are well positioned to continue to advance our portfolio and deliver on our goals. And we're actively managing expenses to potentially extend our cash runaway beyond the third quarter of 2024. In closing, I'd like to thank our employees and recognize the remarkable efforts of the 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 Cameron, Executive Director and Head of the ETAVA PVAP Program, to provide a brief update on ETAVA PVAP. Cameron.

Thank you, Frank, and good morning, everyone. Let me begin by providing more detail on the data update from the phase one study of Atavo PVAT that we announced at R&D Day. Atavo PVAT is a once-daily selective PKR activator with a distinct multimodal mechanism of action designed to improve oxygen binding and delivery and to reduce hemoglobin S polymerization by decreasing 2,3-DPG and to repair red cell membrane damage by increasing ATP. The now-completed Phase I program for Etabopivac was quite robust. We studied Etabopivac in 90 healthy volunteers at up to 1,000 milligrams in a single ascending dose study, followed by multiple ascending dose studies. We then evaluated Etabopivac in 42 patients with sickle cell disease, 15 of whom participated in a 12-week study. open-label extension arm in which all patients received 400 milligrams Etabopivad once a day. Data from this open-label extension study demonstrated that Etabopivad is well tolerated, resulted in a significant and sustained increase in hemoglobin, and significant reductions in reticulocytes, bilirubin, LDH, and systemic markers of sickle cell pathophysiology. In addition to these multiple improvements in measures of sickle cell biology, patients also experienced a 68% reduction in VOCs compared to prior history. For patients with sickle cell disease, meso-occlusive crises represent only the tip of the iceberg as it relates to pain events. To better understand all pain events in our patients, We analyzed our open-label extension data and found that administration of a type of PVAT resulted in a time-dependent reduction in total pain events, culminating in an 80% decrease in all pain-related adverse events in the final four weeks of treatment. Taken together, we believe the concordance of these data across measures of sickle cell pathophysiology, vaso-occlusive crises, and other pain events provide further support for the potential of Etabo PVAT to provide benefits to patients with sickle cell disease, including reduction of vaso-occlusive crises in the ongoing Phase 2-3 hibiscus study. Further, these data and a well-tolerated safety profile provide a strong rationale for the broader Etabo PVAT development program in thalassemia and lower-risk MDS. Let me now turn the call over to Dave for an update on FT7051 and our research pipeline.

Thanks, Cameron. As we described at our recent R&D day, pharmacokinetic and target engagement biomarker data from the ongoing Phase I study of FT7051, our CBP P300 inhibitor in Phase I for metastatic castration-resistant prostate cancer, demonstrate that current dose levels deliver drug exposure in the predicted efficacious range. Based on the PKPD and safety profiles, going forward, we plan to evaluate an alternative dosing schedule in a less heavily pre-treated population. As Frank mentioned, we now expect to share additional data from this study in the first half of next year. As we continue to advance our clinical portfolio, we are also building a deep preclinical pipeline focused on three key areas. First, developing transformative combination therapies for sickle cell disease by validating novel mechanisms that may be complementary to Atavo PVET. Second, by unlocking the therapeutic potential of red blood cell health in areas beyond traditional hemoglobinopathies and anemias. And third, by developing molecules in our pipeline for targeted oncology with an emphasis on synthetic lethality. With that, I will now turn the call over to Todd.

Thank you, Dave, and thank you, everyone, for joining us today. I will spend the first few minutes discussing our financial results for the quarter ended June 30th, 2022, and then discuss our cash position and outlook. Our net loss for the quarter ended June 30th, 2022 was $52.6 million, which compares with a net loss of $43.6 million for the quarter ended June 30th, 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 $39.1 million for the quarter ended June 30th, 2022, compared to 31.6 million for the quarter ended June 30th, 2021. This increase was primarily attributable to the increase in research and development staff to support our advancement of Atavo PVET and other programs, including the conduct of our Phase 2-3 hibiscus trial in sickle cell disease patients and the Phase 2 trial of Atavo PVET in thalassemia. General and administrative expenses were $13.9 million for the quarter ended June 30, 2022, compared to $12.5 million for the quarter ended June 30, 2021. The increase was primarily attributable to professional services, costs due to executive and staff hiring, and other related general and administrative costs. Cash equivalents and marketable securities balance as of June 30, 2022 was $395.9 million compared to $490.3 million as of December 31, 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.

We will now open the call for questions.

Thank you.

As a reminder, to ask a question, you will need to press star 1 1 on your telephone. Please stand by while we compile the Q&A roster.

Our first question comes from Mark Breidenbach with Oppenheimer.

You may proceed.

Hey, good morning, guys, and thanks for taking our questions. Congrats on finding a home for a ludicide nib. I guess I'll start by asking if you'd consider selling rights to the royalty stream from Rigel, similar to what we saw Agios do with IDFA, or is the plan to kind of hold on to the royalty stream for the foreseeable future? And then a second question for me is, with respect to next steps for 7051, You know, I'm wondering if we can assume the next clinical update in the first half of next year will include patients enrolled after the protocol amendment. And if you expect the overall size of the phase one trial to stay more or less the same despite the planned amendments. Thanks for taking that question.

Hey, thanks, Mark, for the question. It's Frank here. with the deal for elutisidinib. And let me just say that we're really pleased with the deal, and it's consistent with our commitment to patients, and they're a fantastic partner. They've got a strong presence in hematology and oncology and a good commercial infrastructure, and it's a bright time. This is going to be a meaningful asset for Rigel. And on our end, there's some near-term milestones, as we've talked about, The PDUPA data is coming up in February, so these are near-term milestones. And overall, what I'm really happy about is that this is, I think, the R&D capabilities to bring a molecule from discovery all the way through now potentially an approval. So that said, with respect to the relative milestones ahead, we haven't made any decisions about that going forward, but we'll certainly consider it, but no decisions have have been made. Secondly, with respect to 751, I'll let Dave comment further on this. But it'll take us a little while to process the amendment to the protocol. And then I would suspect that it'd be reasonably the same in terms of the number of patients to expect sometime in the first half next year. But Dave, let me turn it over to you.

Sure. Mark, thanks for the question. Just to remind everybody, In the patients enrolled so far, these were really advanced patients. Over half of them had experienced chemotherapy, and the median number of prior lines of therapy was three. So we know this is a very advanced population. So a key thing about this amendment is to go into an earlier population and exclude those who have had the chemotherapy. In addition, we're exploring other schedules, we've seen good target engagement, predicted pharmacologically effective dose levels, and so we feel like we've got the biomarker to guide that future under the amendment. I'll leave it at that. Thank you.

All right. Terrific. Thanks for filling the questions, and congrats on the progress. Thanks, Mark.

Thank you. One moment for questions.

Our next question comes from Maury Raycroft with Jefferies.

You may proceed.

Hi. Good morning. Thanks for taking my questions, and congrats on the progress of the quarter. For hibiscus, for the first interim analyses, just want to check in on the latest you're saying on expectations in terms of number of patients and the extent of data you plan to report. And in addition to dose selection, what would be viewed as a positive update in this first interim?

Just to back up a little bit, overall, we're tracking well to plan. And so, what we expect, again, is that we'll be in a position to have the IA1 done, you know, sometime into the year. I'd like to, you know, turn it over to Cameron to ask him to speak about this, but it's largely dose selection. and in terms of the number of patients that we're expecting. So, Cameron, let me turn it over to you.

Yeah, thanks, Frank, and thanks for the question. Yeah, I think we share the enthusiasm for what's to come from hibiscus and just a level set. The minimum bar for data to make decisions at IA1 is the 60th patient in reaching 12 weeks of therapy. So that's the bar at which we'll consider IA1. As Frank just said, we're on track and excited about that around the end of the year. And yeah, the readout from that will be dose selection in this blinded study as recommended by our DSMB.

Sorry.

Go ahead.

Go ahead, Mark.

Yeah. Yeah, that's helpful. The other question I had was for hibiscus for segmenting severe versus moderate baseline VOCs for the patients. Can you provide more info on how many patients are going to fall into each category? And do you plan on providing more information on overall baseline data ahead of the interim?

So, Mari, we're not going to provide any additional data until the interim. But Cameron can speak to how we're generally thinking about stratifying the patients based on VOCs. So, Cameron?

Yeah, thanks. So, the stratification is in the analysis, not in randomization. So, I can't make any predictions about how many patients will fall where. But we'll analyze based on who had fewer VOCs and more VOCs.

Got it. Okay. Thanks for taking my questions.

Thanks, Bahrain.

Thank you. And as a reminder, to ask a question, you will need to press star 1-1 on your telephone.

Our next question comes from Andrew Behrens with SVB. You may proceed.

Hey, how are you? This is Chris on for Andrew. Two questions. So for the first one, for the partnership that you guys have for alutacitinib, in terms of the costs, what are they responsible for and what are you guys responsible for? And for the second question, what sort of commentary has given you more confidence about an accelerated approval pathway?

Thanks for the question, Chris. First, with respect to a little side nib and I've taught here to provide some additional color. But overall, what's really important here is not only that we found a very good home for this asset in support of our overall mission in terms of doing right by patients, but it also allows us to now really focus on our key programs. And those are related to Italo PBAT, as you know, going forward. So this is an important step for the company, and I'm really pleased with the way it all transpired. So Pat, or I'm sorry, Todd, you can speak to a little bit more color here.

Yeah, sure. Hi, Chris. Good to connect with you. So just in terms of ongoing responsibilities, it's largely tied around completion of the support required to get it through the final reviews with the FDA and approval. That includes, you know, completing, you know, the ongoing study, which is nearly final, so it's in sort of the wrap-up stages, as well as some, you know, validation batches and manufacturing, which have been initiated and just need to stop. So, it's very near-term activities leading up to approval. And, you know, at that point and forward, any additional activities would be, you know, taken on by the partner RIGEL. So, fairly minimal amount of ongoing expenditures and internal effort beyond getting it over the finish line.

And let's see, Chris, you had a second question. Remind me of the second question.

Yeah, sure. So the second question was really around, you know, what sort of commentary has given you more confidence about an accelerated approval pathway for a topic like that? Sure.

First, let me just say that we've been having ongoing dialogue with the FDA and it's been constructive. And so we have that not only for but also for our other programs as well. And so we've certainly had discussions around two points. One is what will be at the time of accelerated approval submission. And it's important we not only have hibiscus, but all of the other programs that we've talked about in our portfolio. So that's an important concept. And secondly, as we've discussed at the R&D day, we think that an important study to move forward with would be a TCD study to look at stroke risk. And so those things combined, we think, along with all of our discussions, I think point to our ability, we think, number one, accelerator approval door is still open, and secondly, that at the time of submission, for one, the accelerator approval door is still open, and secondly, that at the time of submission, we anticipate having the data we need, and we'll just have to see what the data look like and make a determination as to whether we go for accelerator approval or not. And finally, let me just say that, as we talked about before, Hibiscus provides for not only an accelerated approval pathway potentially, but also the traditional approval via VOC. So hopefully that helps.

Got it. Thanks. And so I just want to gain some more clarity if possible. So discussions with the FDA have kind of led to this discussion idea that accelerated approval is still available? Is that it? The SAA has given you commentary around that?

Well, we can't go into particulars about discussions with the FDA, but we can tell you we've been having them, and we believe that based on those discussions, the accelerated approval path remains open.

Got it. Thank you very much.

Thanks, Chris.

Thank you. One moment for questions. And as a reminder, to ask a question, you will need to press star 11 on your telephone.

Our next question comes from Prakhar Agarwal with Cantor.

You may proceed.

Hi, good morning, and thanks for taking my questions. Congrats on executing the deal this week. So maybe first question for the Phase 2 Hibiscus trial. At the interim analysis one, will you disclose any VOC data recognizing that it may not be mature, but still 12 weeks could start giving directional signal, and it's probably the most important endpoint for chaos right now. And second question, on the low-risk MDS trial start, I think you mentioned trial start by end of the year. So just curious as to what are the steps that you are working through that is taking some time? Are you waiting for the interim analysis one in sickle cell before making a decision on the low-risk MDS trial and the dose to be tested there? or 400 milligrams is the go-to dose for MDS? And if the trial gets initiated end of the year, would you still expect data for low-risk MDS by mid-2023? Thank you.

Yeah, so thanks, Ricard. Good morning. First on the question of analysis one, we don't have any plans to provide any data beyond selection of the dose and any safety commentary. So we think it's going to be too early at that point in time to comment on that. So that's our plans for Hibiscus Interim Analysis 1 at present. Secondly, with respect to the low-risk MDS, and I'll ask Cameron here to comment broadly But we are tracking overall to provide some initial data as we've got it to middle of next year. So, Cameron, maybe you can speak to where we are with getting the study up and running. Yeah, absolutely. Thank you.

One comment on On where we'll be at IA1 real quick for hibiscus, just as a reminder, 12 weeks of data is essentially exactly what we had in our phase one study, which we've talked broadly about, and we've shown the trends both in BOC and total pain, in addition to being blinded, so we won't have the data to show you at IA1. I think we pretty well know what that signal looks like early, and we're all more interested in what it's going to look like with more data. As in regards to MDS, no, we're not waiting on dose selection for hibiscus. It's an independent study. And essentially, it just happens to overlap in time. And what we're doing right now is what you'd normally do in site startup with selection of sites, contracting, budgeting, and approvals. We're making good progress, and as I said, I think we'll be getting it started in Q4, such that we'll have some early data to talk about mid-next year. Thank you.

Thank you.

Thank you.

And I'm not showing any further questions at this time. I would now like to turn the call back over to Frank Lee for any further remarks.

Thank you. Well, first of all, let me thank our participants for logging on this morning. And I especially want to thank our patients and investigators for their contributions for a very strong second quarter for FORMA. And we look forward to having further discussions in the future. So this concludes our call, and thank you for logging on.

Thank you. This concludes today's conference call. Thank you for participating.

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Good morning.

My name is Josh, and I will be your conference operator today. At this time, I would like to welcome everyone to the Forma Therapeutic Second 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. Thank you. I would now like to turn the conference over to Dr. Adam Biro, Senior Vice President at Kendall Investor Relations.

Please go ahead. Good morning, and welcome to FORMA's second quarter 2022 financial results and business update conference call.

Before we begin, I encourage everyone to go to the news and investors section of formatherapeutics.com to find the press release detailing the company's second quarter 2022 performance that we will discuss today. I would like to remind you that any statements we will be making that are not statements of historical or current facts are based on our current expectations and beliefs. and are intended to be forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements are neither predictions nor guarantees of future events or performance and include those regarding timing, enrollment, success and data announcements of our current ongoing clinical trials, therapeutic and market potential, hypotheses of mechanisms of action, clinical benefits, safety of our product candidates, expansion and development of clinical programs, and our financial condition and capital requirements. Our actual results may differ materially from those expressed or implied by any forward-looking statements as a result of various risks and uncertainties associated with our business, including those under the heading Risk Factors in our quarterly report on Form 10-Q for the quarter ended June 30, 2022 that will be filed with the SEC today and in subsequent reports, including our current reports on Form 8-K. On today's call, I am joined by our President and Chief Executive Officer, Frank Lee, Dr. Tina Melian, Head of Research and Development, Dr. Cameron Trenner, Executive Director, Clinical Development, Dr. Dave Cook, our Chief Scientific Officer, and our CFO, Todd Chagat.

I will now turn the call over to Frank.

Thank you, Adam. Good morning, everyone, and thank you for joining us today. former names to become a recognized leader in rare hematologic diseases and cancers, and a trusted partner to the communities we serve. We're advancing a robust pipeline led by TABWPVAT, are well capitalized. And last month, we're pleased to announce the appointment of two new members of our executive team. Dr. Tina Melian joins us as Executive Vice President, Head of Research and Development with over 20 years experience developing patient-centric therapies for rare and orphan disease indications spanning multiple modalities in all phases of development. And Linnea Spezi joins us as Senior Vice President Chief Human Resources Officer with over 25 years of HR leadership experience in life sciences and healthcare services. I'm confident that Tino and Linnea will help drive our next phase of growth into late-stage clinical development and commercialization as we prepare to deliver meaningful medicines to patients we serve. We also recently announced that Dr. Selwyn Vickers, a member of our board, and Dr. Patrick Kelly, our chief medical officer, have left FORMA. I'd like to congratulate Dr. Vickers on his new position as president and CEO of Memorial Sloan Kettering Cancer Center. As part of this transition, he is stepping down from public company boards. Pat joined FORMA approximately seven years ago, and FORMA is primarily a research and early development company. Pat's passion and expertise are in translational medicine and early clinical development. We're grateful to Pat for his contributions to our programs and for establishing a world-class hematology development team. comprised of six MDs who have deep experience leading centers that treat patients with sickle cell disease, thalassemia, and MDS. We're now moving toward global late-stage development and ultimately commercialization. Tina Millian, our head of research and development, has a lot of expertise in this area, and we're very pleased to have him with us. Pat will continue to serve as a strategic advisor to FORMA And Tino will assume direct responsibilities for research, development, and medical teams. Pat has no immediate plans beyond spending time with his family and continuing to support FORMA through a consulting capacity. It's been my privilege to work alongside Dr. Vickers and Dr. Kelly, and I'm deeply thankful for the important contribution that they've made to our organization. We wish them all the best in the future. and will continue to maintain their patient-centric focus in all that we do. We now have in place an executive team with experienced leading organizations through global late-stage development and commercial launch. We brought on board John Bishop, our head of technical operations earlier this year. As you know, we recently brought on board Tino and Linnea as well. Other members of the executive team have similar experiences. And I'm very proud of the executive team we've assembled for the next chapter of our group. I'd like to now invite Tino, our Executive Vice President of Research and Development, to provide a bit more detail on his background and plans to drive our R&D organization forward. Tino?

Thanks, Frank. I'm delighted to be here.

In my 20 plus years of experience in the biopharmaceutical industry, including experience leading global late-stage rare disease trials, it is rare that I see a company as poised for growth and expansion as FORA is today. Since joining last month, I've been impressed by the level of scientific rigor across the organization, the compelling data generated to date across our clinical programs, and the potential of a TABLE PBAT to truly transform the treatment of simple cell disease, thalassemia, and lower risk MDS. The quality of these programs rests on the shoulders of strong leadership and deep scientific expertise. I want to thank Pat for helping to build a world-class hematology development team and for advancing the atypical PPAP program through the generation of robust, clinically meaningful data that has positioned FORMA for global, late-stage development and commercialization. Looking forward, my priorities are to drive operational excellence across the atypical PPAP development program as we advance towards a potential approval in sickle cell disease, and data readouts in Phase II studies expanding its therapeutic potential to additional populations, including transfused populations in sickle cell disease, thalassemia, and lower-risk MDS. In parallel, I aim to selectively expand our preclinical portfolio, recognizing the particularly strong foundation that FORMA has built in hematology and oncology.

With that, let me now turn the call back over to Frank.

Thanks, Tino. Earlier this week, we were pleased to announce that we've entered into an exclusive license agreement with Rigel Pharmaceuticals to develop, manufacture, and commercialize Lutacidinib, our mutant IDH1 inhibitor for the potential treatment of mutant IDH1 positive relapsed or refractory acute myeloid leukemia. Based on the strength of the Registrational Phase II dataset, we believe elutisidinib has the potential to represent an impactful differentiated therapy for relapsed refractory AML. The FDA has accepted our NDA for elutisidinib with a PDUFA action date of February 15, 2023. Under the terms of the agreement, formal receive an upfront payment of $2 million and is eligible to receive an additional $17.5 million upon the achievement of certain near-term regulatory approval and first commercial sale milestones. A total of up to an additional $215.5 million in connection with the achievement of certain development and commercial milestones and potential tiered royalties in the low teens to mid-30s. Given Rigel's focus on hematologic diseases and cancers and the strength of their commercial infrastructure, we are confident that they are the right partner to deliver the ludicide net to patients in need. More broadly, this agreement highlights our R&D capabilities and will enable us to further sharpen our focus on advancing our pipeline, which we highlighted in detail at our inaugural R&D day in May. This event included a presentation of encouraging analyses from our phase one study of atavipivet and sickle cell disease, indicating a reduction in reported pain-related adverse events, which we believe further underscores the potential of atavipivet to reduce vaso-occlusive crises in ongoing phase two, three hibiscus study. Enrollment in hibiscus study is on track, with interim analysis one expected near the end of this year. As a reminder, this analysis will enable dose selection for the Phase III portion of the study. Based on continuing discussions with the FDA, we continue to believe that accelerated approval remains a viable path with the clinical data package we expect at the time of submission. As we outlined at R&D Day, we believe that a TAPA-PVAS multimodal mechanism of action has the potential to improve symptomatic anemia across multiple indications beyond sickle cell disease. We continue to advance the Phase II Gladiola study of atavipivac and transfusion-dependent and non-transfusion-dependent thalassemia, as well as sickle cell disease patients who are receiving chronic transfusions. As a reminder, approximately 20% of the overall sickle cell disease patient population is transfusion-dependent. and represents an area of substantial unmet need. We expect initial results from the study by the end of the year. Also by the end of the year, we expect to further expand the ATTAVA PVAD Development Program to include Phase II studies in pediatric sickle cell disease and lower-risk myelodysplastic syndrome, or MDS. Similar to hemolytic anemias, chronic transfusions are the common practice in MDS treatment paradigm and can result in iron overload. We look forward to evaluate the potential of EtablaPVAT to target the underlying pathophysiology of low-risk MDS and reduce transfusion burden in this area of high IMET need. Moving to our Phase 1 study of FT7051 and metastatic castration-resistant prostate cancer. As discussed at R&D Day, We plan to evaluate an alternative dosing schedule in a less heavily pretreated patient population and are currently processing the protocol amendment. Based on this protocol amendment, we now expect to share additional data from the study in the first half of next year. At FORMA, being a trusted partner to the patient communities we serve is central to our long-term corporate vision. For patients with sickle cell disease, the transition from pediatric to adult care can be very challenging. To help patients navigate this important transition, we launched our Form a Bridge program at the end of last year. And I'm pleased to report that there's been a high level of interest in the program. We've received a number of proposals from a mix of healthcare institutions, patient organizations, and community-based organizations. and we expect to announce grant awards in the coming months. With $395 million in cash, we are well positioned to continue to advance our portfolio and deliver on our goals. And we're actively managing expenses to potentially extend our cash runway beyond the third quarter of 2024. In closing, I'd like to thank our employees and recognize the remarkable efforts of the 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 Cameron, Executive Director and Head of the Atava PVAP Program, to provide a brief update on Atava PVAP. Cameron.

Thank you, Frank, and good morning, everyone.

Let me begin by providing more detail on the data update from the phase one study of Atavo PVAT that we announced at R&D Day. Atavo PVAT is a once daily selective PKR activator with a distinct multimodal mechanism of action designed to improve oxygen binding and delivery and to reduce hemoglobin S polymerization by decreasing 2,3-DPG and to repair red cell membrane damage by increasing ATP. The now-completed phase one program for Atabu PVAT was quite robust. We studied Atabu PVAT in 90 healthy volunteers at up to 1,000 milligrams in a single ascending dose study, followed by multiple ascending dose studies. We then evaluated Atabu PVAT in 42 patients with sickle cell disease 15 of whom participated in a 12-week open-label extension arm in which all patients received 400 milligrams Etabopivac once a day. Data from this open-label extension study demonstrated that Etabopivac is well-tolerated, resulted in a significant and sustained increase in hemoglobin, and significant reductions in reticulocytes, bilirubin, LDH, and systemic markers of sickle cell pathophysiology. Importantly, in addition to these multiple improvements in measures of sickle cell biology, patients also experienced a 68% reduction in VOCs compared to prior history. For patients with sickle cell disease, vaso-occlusive crises represent only the tip of the iceberg as it relates to pain events. To better understand all pain events in our patients, we analyzed our open-label extension data and found that administration of a type of PVAT resulted in a time-dependent reduction in total pain events, culminating in an 80% decrease in all pain-related adverse events in the final four weeks of treatment. Taken together, we believe the concordance of these data across measures of sickle cell pathophysiology, vaso-occlusive crises, and other pain events provide further support for the potential of Etabo-PVAT to provide benefits to patients with sickle cell disease, including reduction of vaso-occlusive crises in the ongoing Phase 2-3 hibiscus study. Further, these data and a well-tolerated safety profile provide a strong rationale for the broader Etabo-PVAT development program in thalassemia and lower-risk MDS. Let me now turn the call over to Dave for an update on FT7051 and our research pipeline.

Thanks, Cameron. As we described at our recent R&D day, pharmacokinetic and target engagement biomarker data from the ongoing Phase I study of FT7051, our CBP P300 inhibitor in Phase I for metastatic castration-resistant prostate cancer, that current dose levels deliver drug exposure in the predicted efficacious range. Based on the PKPD and safety profiles, going forward, we plan to evaluate an alternative dosing schedule in a less heavily pre-treated population. As Frank mentioned, we now expect to share additional data from this study in the first half of next year. As we continue to advance our clinical portfolio, we are also building a deep preclinical pipeline focused on three key areas. First, developing transformative combination therapies for sickle cell disease by validating novel mechanisms that may be complementary to Atavo PVET. Second, by unlocking the therapeutic potential of red blood cell health in areas beyond traditional hemoglobinopathies and anemias. And third, by developing molecules in our pipeline for targeted oncology with an emphasis on synthetic lethality. With that, I will now turn the call over to Todd.

Thank you, Dave, and thank you, everyone, for joining us today. I will spend the first few minutes discussing our financial results for the quarter ended June 30th, 2022, and then discuss our cash position and outlook. Our net loss for the quarter ended June 30th, 2022 was $52.6 million, which compares with a net loss of $43.6 million for the quarter ended June 30th, 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 $39.1 million for the quarter ended June 30th, 2022, compared to 31.6 million for the quarter ended June 30th, 2021. This increase was primarily attributable to the increase in research and development staff to support our advancement of Atavo PVET and other programs, including the conduct of our Phase 2-3 hibiscus trial in sickle cell disease patients and the Phase 2 trial of Atavo PVET in thalassemia. General and administrative expenses were $13.9 million for the quarter ended June 30, 2022, compared to $12.5 million for the quarter ended June 30, 2021. The increase was primarily attributable to professional services, costs due to executive and staff hiring, and other related general and administrative costs. Cash equivalents and marketable securities balance as of June 30, 2022 was $395.9 million compared to $490.3 million as of December 31, 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.

We will now open the call for questions.

Thank you.

As a reminder, to ask a question, you'll need to press star 1 1 on your telephone. Please stand by.

We compile the Q&A roster. Our first question comes from Mark Bredenbach with Oppenheimer.

You may proceed.

Hey, good morning, guys, and thanks for taking our questions. Congrats on finding a home for a ludicide nib. I guess I'll start by asking if you'd consider selling rights to the royalty stream from Rigel, similar to what we saw Agios do with IDFA, or is the plan to kind of hold on to the royalty stream for the foreseeable future? And then a second question for me is, with respect to next steps for 7051, You know, I'm wondering if we can assume the next clinical update in the first half of next year will include patients enrolled after the protocol amendment, and if you expect the overall size of the Phase 1 trial to stay more or less the same despite the planned amendments. Thanks for taking that question.

Hey, thanks, Mark, for the question. It's Frank here. with the deal for elutisidinib. And let me just say that we're really pleased with the deal, and it's consistent with our commitment to patients, and they're a fantastic partner. They've got a strong presence in hematology and oncology and a good commercial infrastructure, and it's a bright time. This is going to be a meaningful asset for Rajo. And on our end, there's some near-term milestones, as we've talked about, The PDUPA data is coming up in February, so these are near-term milestones. And overall, what I'm really happy about is that this is, I think, the R&D capabilities to bring a molecule from discovery all the way through now potentially an approval. So that said, with respect to the relative milestones ahead, we haven't made any decisions about that going forward, but we'll certainly consider it, but no decisions have been made. Secondly, with respect to 751, I'll let Dave comment further on this. But it'll take us a little while to process the amendment to the protocol. And then I would suspect that it'd be reasonably the same in terms of the number of patients to expect sometime in the first half next year. But Dave, let me turn it over to you.

Sure. Mark, thanks for the question. Just to remind everybody, In the patients enrolled so far, these were really advanced patients. Over half of them had experienced chemotherapy, and the median number of prior lines of therapy was three. So we know this is a very advanced population. So a key thing about this amendment is to go into an earlier population and exclude those who have had the chemotherapy. In addition, we're exploring other schedules, we've seen good target engagement, predicted pharmacologically effective dose levels. And so, we feel like we've got the biomarker to guide that future under the amendment. And I'll leave it at that. Thank you.

All right. Terrific. Thanks for filling the questions and congrats on the progress. Thanks, Mark.

Thank you. One moment for questions.

Our next question comes from Maury Raycroft with Jefferies.

You may proceed.

Hi. Good morning. Thanks for taking my questions, and congrats on the progress of the quarter. For hibiscus, for the first interim analyses, just want to check in on the latest you're saying on expectations in terms of number of patients and the extent of data you plan to report. And in addition to dose selection, what would be viewed as a positive update in this first interim?

Just to back up a little bit, overall, we're tracking well to plan. And so what we expect, again, is that we'll be in a position to have the IA1 done, you know, sometime into the year. I'd like to, you know, turn it over to Cameron to ask him to speak about this, but it's largely dose selection. and in terms of the number of patients that we're expecting. So, Cameron, let me turn it over to you.

Yeah, thanks, Frank, and thanks for the question. Yeah, I think we share the enthusiasm for what's to come from hibiscus and just a level set. The minimum bar for data to make decisions at IA1 is the 60th patient in reaching 12 weeks of therapy. So, that's the bar at which we'll consider IA1. As Frank just said, we're on track and excited about that around the end of the year. And yeah, the readout from that will be dose selection in this blinded study as recommended by our DSMB.

Sorry.

Go ahead.

Go ahead, Mari.

Yeah. Yeah, that's helpful. The other question I had was for hibiscus for segmenting severe versus moderate baseline VOCs for the patients. Can you provide more info on how many patients are going to fall into each category? And do you plan on providing more information on overall baseline data ahead of the interim?

So, Mari, we're not going to provide any additional data until the interim. But Cameron can speak to how we're generally thinking about stratifying the patients based on VOCs. So, Cameron?

Yeah, thanks. So, the stratification is in the analysis, not in randomization. So, I can't make any predictions about how many patients will fall where. But we'll analyze based on who had fewer VOCs and more VOCs. Got it.

Okay. Thanks for taking my questions.

Thanks, Warren.

Thank you. And as a reminder, to ask a question, you will need to press star 1-1 on your telephone.

Our next question comes from Andrew Behrens with SVB. You may proceed.

Hey, how are you? This is Chris on for Andrew. Just a Two questions. So for the first one, for the partnership that you guys have for alutacitinib, in terms of the costs, what are they responsible for and what are you guys responsible for? And for the second question, what sort of commentary has given you more confidence about an accelerated approval pathway?

Thanks for the question, Chris. First, with respect to a little side nib and Todd here to provide some additional color. But overall, what's really important here is not only that we found a very good home for this asset in support of our overall mission in terms of doing right by patients, but it also allows us to now really focus on our key programs. And those are related to Etabo PBAT, as you know, going forward. So this is an important step for the company, and I'm really pleased with the way it all transpired. So Pat, or I'm sorry, Todd, you can speak to a little bit more color here.

Yeah, sure. Hi, Chris. Good to connect with you. So just in terms of, you know, ongoing responsibilities, it's largely tied around, you know, completion of the support required, you know, to get it, you know, through the final reviews with the FDA and approval. That includes, you know, completing, you know, the ongoing study, which is nearly final, so it's in sort of the wrap-up stages, as well as some, you know, validation batches and manufacturing, which have been initiated and just need to stop. So, it's very near-term activities leading up to approval. And, you know, at that point and forward, any additional activities would be, you know, taken on by the partner RIGEL. So, fairly minimal amount of ongoing expenditures and internal effort beyond getting it over the finish line.

And let's see, Chris, you had a second question. Remind me of the second question.

Yeah, sure. So the second question was really around, you know, what sort of commentary has given you more confidence about an accelerated approval pathway for a topic like that? Sure.

First, let me just say that we've been having ongoing dialogue with the FDA and it's been constructive. And so we have that not only for but also for our other programs as well. And so we've certainly had discussions around two points. One is what will be at the time of accelerated approval submission. And it's important We not only have Hibiscus, but all of the other programs that we've talked about in our portfolio. So that's an important concept. And secondly, as we've discussed at the R&D day, we think that an important study to move forward with would be a TCD to look at stroke risk. And so those things combined, we think, along with all of our discussions, I think point to our ability, we think, number one, accelerator approval door is still open, and secondly, that at the time of submission, for one, the accelerator approval door is still open, and secondly, that at the time of submission, we anticipate having the data we need, and we'll just have to see what the data look like and make a determination as to whether we go for accelerator approval or not. And finally, let me just say that, as we talked about before, Hibiscus provides for not only an accelerated approval pathway potentially, but also the traditional approval via VOC. So hopefully that helps.

Got it. Thanks. And so I just want to gain some more clarity if possible. So discussions with the FDA have kind of led to this discussion idea that accelerated approval is still available? Is that it? The FDA has given you commentary around that?

Well, we can't go into particulars about discussions with the FDA, but we can tell you we've been having them, and we believe that based on those discussions, the accelerated approval path remains open.

Got it. Thank you very much.

Thanks, Chris.

Thank you. One moment for questions. And as a reminder, to ask a question, you will need to press star 11 on your telephone.

Our next question comes from Prakhar Agarwal with Cantor.

You may proceed.

Hi, good morning, and thanks for taking my questions. Congrats on executing the deal this week. So maybe first question for the Phase 2 Hibiscus trial. At the interim analysis one, will you disclose any VOC data recognizing that it may not be mature, but still 12 weeks could start giving directional signal, and it's probably the most important endpoint for chaos right now. And second question, on the low-risk MDS trial start, I think you mentioned trial start by end of the year. So just curious as to what are the steps that you are working through that is taking some time? Are you waiting for the interim analysis one in sickle cell before making a decision on the low-risk MDS trial and the dose to be tested there? or 400 milligrams is the go-to dose for MDS? And if the trial gets initiated end of the year, would you still expect data for low-risk MDS by mid-2023? Thank you.

Yeah, so thanks, Ricard. Good morning. First on the question of analysis one, we don't have any plans to provide any data beyond selection of the dose and any safety commentary. So we think it's going to be too early at that point in time to comment on that. So that's our plans for hibiscus interim analysis one at present. Secondly, with respect to the low-risk MDS, and I'll ask Cameron here to comment broadly But we are tracking overall to provide some initial data as we've got it to the middle of next year. So, Cameron, maybe you can speak to where we are with getting the study up and running. Yeah, absolutely. Thank you.

One comment on On where we'll be at IA1 real quick for hibiscus, just as a reminder, 12 weeks of data is essentially exactly what we had in our phase one study, which we've talked broadly about, and we've shown the trends both in BOC and total pain, in addition to being blinded, so we won't have the data to show you at IA1. I think we pretty well know what that signal looks like early, and we're all more interested in what it's going to look like with more data. As in regards to MDS, no, we're not waiting on dose selection for hibiscus. It's an independent study. And essentially, it just happens to overlap in time. And what we're doing right now is what you'd normally do in site startup with selection of sites, contracting, budgeting, and approvals. We're making good progress, and as I said, I think we'll be getting it started in Q4, such that we'll have some early data to talk about mid-next year. Thank you.

Thank you.

Thank you.

And I'm not showing any further questions at this time. I would now like to turn the call back over to Frank Lee for any further remarks.

Thank you. Well, first of all, let me thank our participants for logging on this morning. And I especially want to thank our patients, investigators for their contributions for a very strong second quarter for FORMA. And we look forward to having further discussions in the future. So this concludes our call, and thank you for logging on.

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