FMTX - Forma Therapeutics Holdings Inc. (FMTX) CEO Frank Lee on Q2 2022 Results - Earnings Call Transcript
Forma Therapeutics Holdings Inc. (FMTX)
Q2 2022 Earnings Conference Call
August 5, 2022 8:00 AM ET
Company Participants
Adam Bero - Investor Relations
Frank Lee - President and Chief Executive Officer
Agustin Melian - Executive Vice President, Research and Development
Cameron Trenor - Executive Director, Clinical Development
Dave Cook - Chief Scientific Officer
Todd Shegog - Chief Financial Officer
Conference Call Participants
Mark Breidenbach - Oppenheimer
Maury Raycroft - Jefferies
Andrew Berens - SVB
Prakhar Agrawal - Cantor
Presentation
Operator
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 Therapeutics 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 Bureau, Senior Vice President at Kendall Investor Relations. Please go ahead.
Adam Bero
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 through 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 in the 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'm joined by our President and Chief Executive Officer, Frank Lee; Dr. Tino Melian, Head of Research and Development. Dr. Cameron Trenor, Executive Director, Clinical Development; Dr. Dave Cook, our Chief Scientific Officer; and our CFO, Todd Shegog.
I will now turn the call over to Frank.
Frank Lee
Thank you, Adam. Good morning everyone and thank you for joining us today. Forma 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 etavopivat are well capitalized, and last month we're pleased to announce the appointment of two new members of our executive team. Dr. Tino 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 Linea Aspesi 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 the Tino and Linea Lineo 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 SloanKettering Cancer Center. As part of this transition, he is stepping down from public company boards. Pat joined Forma approximately seven years ago when Forma is primarily a research and early development company. Pat’s passion and expertise are in translational medicine, early clinical develop. 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, Tino will assume direct responsibilities for research, development and medical teams. Pat has no immediate plans beyond spending time with this 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 we'll 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've brought on board John Bishop, our Head of Technical Operations earlier this year. As you know, we recently brought on board Tino and Lineo 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 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.
Agustin Melian
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 trial, it is rare that I see a company that’s 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 and across our clinical programs and the potential of etavopivat to truly transform the treatment of sickle cell disease, thalassemia and more risk MDS.
The quality of these programs rest on the shoulders of strong leadership and deep scientific expertise. I wanted to thank Pat for helping to build a world-class hematology development team and for advancing the etavopivat 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 etavopivat development program as we advance toward a potential approval in sickle cell disease and data readout in Phase II studies, expanding its therapeutic potential to additional populations, including transfuse populations in sickle cell disease, thalassemia and lower-risk MDS.
In parallel aim to selectively expand our preclinical portfolio, recognizing particularly strong foundation that Forma has built in hematology and on [indiscernible].
With that, let me now turn the call back over to Frank.
Frank Lee
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 side olutasidenib, 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 olutasidenib has the potential to represent an impactful and differentiated therapy for relapsed refractory AML. The FDA has accepted our NDA for olutasidenib with a PDUFA action date of February 15, 2023. Under the terms of the agreement Forma revived 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 sales 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 olutasidenib 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 in our Inaugural R&D Day in May. This event included a presentation of encouraging analysis from our Phase I study of etavopivat in sickle cell disease, indicating a reduction in reported pain related adverse events, which we believe further underscores the potential of etavopivat to reduce vaso-occlusive crises in ongoing Phase II/III 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 etavopivat’s 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 Gladiolus study of etavopivat in transfusion dependent and non-transfusion dependent thalassemia, as well as sickle cell disease patients who are receiving chronic transfusion.
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 this study by the end of the year. Also, by the end of the year we expect to further expand the etavopivat development program to include Phase II studies in pediatric sickle cell disease and lower risk myelodysplastic syndrome or MDS. Similar to hemolytic anemia’s chronic transfusions are the common practice in MDS treatment paradigm and can result in iron overload. We look forward to evaluate the potential of etavopivat to target the underlying pathophysiology of lower risk MDS and reduced transfusion burden in this area of high unmet need.
Moving to our Phase I study of FT-7051 in metastatic castration-resistant prostate cancer. As discussed at R&D Day, we plan to evaluate an alternative dosing schedule in a less heavily pre-treated patient population and are currently processing the protocol amendment. Based on this protocol amendment, we now expect to share additional data from this study in the first half of next year.
As 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 Forma Bridge program at the end of last year. And I'm pleased to report that there has been a high level of interest in the program. We 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 are 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 etavopivat program to provide a brief update on etavopivat. Cameron?
Cameron Trenor
Thank you, Frank, and good morning everyone. Let me begin by providing more detail on the data update from the Phase I study of etavopivat that we announced at R&D Day. The etavopivat 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 a decrease in two, three DPG and to repair red cell membrane damage by increase in ATP. The now completed Phase I program for etavopivat was quite robust. We studied etavopivat 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 the etavopivat 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 of etavopivat once a day.
Data from this open label extension study demonstrated that etavopivat is well tolerated, resulted in a significant and sustained increase in hemoglobin and significant reductions in reticulocyte, 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 etavopivat 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 etavopivat to provide benefits to patients with sickle cell disease, including reduction of vaso occlusive crises in the ongoing Phase II/III Hibiscus Study. Further, these data and a well-tolerated safety profile provide a strong rationale for the broader etavopivat development program in thalassemia and lower risk MDS.
Let me now turn the call over to Dave for an update on FT-7051 and our research pipeline.
Dave Cook
Thanks, Cameron. As we described at our recent R&D Day, pharmacokinetic and target engagement biomarker data from the ongoing Phase I study of FT-7051 or 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 PK/PD 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 a etavopivat. Second, by unlocking the therapeutic potential of red blood cell health in areas beyond traditional hemoglobinopathies and anemia. 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.
Todd Shegog
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 30, 2022 and then discuss our cash position and outlook. Our net loss for the quarter ended June 30, 2022 was $52.6 million, which compares with a net loss of $43.6 million for the quarter ended June 30, 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 30, 2022, compared to $31.6 million for the quarter ended June 30, 2021. This increase was primarily attributable to the increase in research and development staff to support our advancement of etavopivat and other programs, including the conduct of our Phase II/III Hibiscus trial in sickle cell disease patients and the Phase III trial of etavopivat 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, cost due to executive and staff hiring and other related general and administrative costs. Our cash, 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.
Question-and-Answer Session
Operator
Thank you. [Operator Instructions] Our first question comes from Mark rating Buck with Oppenheimer. You may proceed.
Mark Breidenbach
Hey, good morning, guys and thanks for taking our questions. Congrats on finding a home for olutasidenib. I guess I'll start by asking if you'd consider selling rights to the royalty stream from Rigel similar to what we saw [indiscernible] kind of hold onto the royalty stream for the foreseeable future? And then second question for me is, with respect to next steps for 7051. 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 I trial to stay more or less the same despite the planned amendments? Thanks for taking my questions.
Frank Lee
Hey. Thanks, Mark for the question, it's Frank here. First off, [Technical Difficulty] deal for olutasidenib and I would 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, oncology and a good commercial infrastructure. And it's [Technical Difficulty] right time this is going to be a meaningful asset for Rigel. And on our end, there is some near-term milestones as we've talked about, the PDUFA date is coming up in February. So these are near-term milestones. And overall, what I'm really happy with is, this is -- I think it [Technical Difficulty] R&D capabilities to bring a molecule from discovery all the way through now potentially an approval. So with respect to the royalty 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 7051. I'll let Dave comment further on this, but it will take us a little while to process the amendment to the protocol. And then I would suspect that it would 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.
Dave Cook
Sure. Mark, thanks for the question. Just to remind everybody, in the patients enrolled so far -- and these were really advanced patients, over half of them have experienced chemotherapy and the median number of prior lines of therapy with [indiscernible]. 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 as 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.
Mark Breidenbach
All right, terrific. Thanks for filling the questions and congrats on the progress.
Frank Lee
Thanks, Mark.
Operator
Thank you. One moment for questions. Our next question comes from Maury Raycroft with Jefferies. You may proceed.
Maury Raycroft
Hi, good morning. Thanks for taking my questions and congrats on the progress for the quarter. For Hibiscus, for the first interim analysis just wanted 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?
Frank Lee
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 sometime end of the year. I'd like to turn it over to Cameron to ask him to speak about this, but it’s largely dose selection and in terms of number of patients that we're expecting. So Cameron, let me turn it over to you.
Cameron Trenor
Thanks, Frank. And thanks for the question. Yeah, I think we share the enthusiasm for what's to come from Hibiscus. And just to level set, the minimum bar data to make decisions at IA1 is the 60 of the patients again reaching 12 weeks of therapy. So that's the bar, which will consider IA1. As Frank just said, we're on track and excited about that around the end of the year. And the other -- the readout from that will be dose selection in this blinded study as recommended by our DSMB.
Frank Lee
[Multiple Speaker] Sorry. Go ahead, Maury.
Maury Raycroft
Yeah. That's helpful. And then one of 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?
Frank Lee
So Maury, 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.
Cameron Trenor
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 will analyze based on who had fewer VOCs and more VOCs.
Maury Raycroft
Got it, okay. Thanks for taking my questions.
Frank Lee
Thanks, Maury.
Operator
Thank you. [Operator Instructions] Our next question comes from Andrew Berens with SVB. You may proceed.
Unidentified Participant
Hi. How are you? This is Chris on for Andrew. Just two questions. So for the first one for the partnership that you guys have for olutasidenib. In terms of the costs, what are they responsible for and what are you guys are responsible for? And for the second question, what sort of commentary has given you more confidence about an accelerated approval pathway?
Frank Lee
Thanks for the question, Chris. First, with respect to olutasidenib and -- I’ll let 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 etavopivat 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 -- I'm sorry, Todd, you can speak to a little bit more color here.
Todd Shegog
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 for the FDA an approval, that includes completing the ongoing study, which is nearly final [indiscernible] the wrap-up stages, as well as some validation batches manufacturing, which have been initiated and we just stopped. So it's very near term activities leading up to approval. And at that point and forward any additional activities would be taken on by the partner Rigel. So fairly minimal amount of ongoing expenditures and internal effort beyond getting it over the finish line.
Frank Lee
And let's see, Chris, you had a second question. Remind me of the second question.
Unidentified Participant
Yeah, sure. So the second question was really around what sort of commentary has given you more confidence about an accelerated approval pathway for etavopivat?
Frank Lee
Sure. First, let me just say that we've been having ongoing dialog 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 [Technical Difficulty] at the time of accelerated approval submission and [Technical Difficulty] 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 [TCD study] (ph) to look at stroke risk. And so those things combined, we think along with all of our discussions. I think point two our ability we think -- number one, the accelerated approval door is still open. And secondly, that at the time of submission we anticipate having the data we need. We'll just have to see what the data look like and make a determination as to whether we go for accelerated approval or not.
And finally, let me just say that, as we’ve talked about before, Hibiscus provides not only an accelerated approval pathway potentially, but also the traditional approval by VOC. So hopefully that helps.
Unidentified Participant
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 idea that -- I thought approval is still available, [indiscernible] your commentary around that.
Frank Lee
Well, we can't go into the 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.
Unidentified Participant
Got it. Thank you very much.
Frank Lee
Thanks, Chris.
Operator
Thank you. On moment for questions. [Operator Instructions] our next question comes from Prakhar Agrawal with Cantor. You may proceed.
Prakhar Agrawal
Hi, good morning and thanks for taking my questions. Congrats on executing the deal this week. So, maybe first question for the Phase II 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 KOLs right now? And second question, on the lower risk MDS trial start, I think you mentioned trial started by end of the year. So just curious as to what are the steps that you're 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 dose to be tested there or 400 milligram 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.
Frank Lee
Yes. So thanks, Prakhar. Good morning. First, on the question [Technical Difficulty] 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, 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 run.
Cameron Trenor
Yeah, absolutely. Thank you. One comment on where it will be at IA1 real quick for Hibiscus, just as a reminder, 12 weeks of data is essentially exactly what we had in our Phase I study, which we've talked broadly about and we've shown the trends both in BSE and total pain in addition to be in blinded, so we won't have the data to show you with IA1. I think we pretty well know what that signal looks like early. I mean, 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 we're -- essentially it just happens to overlap in time and what we're doing right now is what you normally do when the site start up with selection of sites, contracting, budgeting and approvals. We're making good progress. And as I said, I think we'll be getting started in Q4, such that we'll have some early data to talk about a bit next year. Thank you.
Prakhar Agrawal
Thank you.
Operator
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.
Frank Lee
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 pro forma and we look forward to having further discussions in the future. So this concludes our call and thank you for logging on.
Operator
Thank you. This concludes today's conference call. Thank you for participating. You may now disconnect.
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Forma Therapeutics Holdings Inc. (FMTX) CEO Frank Lee on Q2 2022 Results - Earnings Call Transcript