Q2 2021 Forma Therapeutics Holdings Inc Earnings Call
[music].
Welcome to the formal therapeutics second quarter 2021 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 one on your Touchtone phone. If anyone has difficulty hearing the conference. Please press star zero for operator assistance Azure.
As a reminder, this call is being recorded today August 13, 2021, 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 former.
Good morning to our listeners and welcome to today's call to review second quarter 2021 financial results and business update.
On this call Im joined by frankly, our President and Chief Executive Officer.
Kelly, our Chief Medical Officer.
Dave Cooke, our Chief Scientific officer.
And Todd <unk>, our Chief Financial Officer.
Before we begin I'd.
Like to caution listeners that comments made and financial information provided during the conference call includes certain statements that are estimates beliefs forward looking and are subject to various risks and uncertainties.
<unk> 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 1095.
We want to emphasize that such forward looking statements reflect our current expectations and assumptions regarding timing success in data announcements of our current and ongoing clinical trials.
Therapeutic potential in clinical benefit and safety of our product candidates planned regulatory submissions.
Our financial condition, and our business operations development efforts and the potential impact of COVID-19 on our business and clinical development and relationships with third parties and collaborators.
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 entitled Risk factors in our quarterly report on Form 10-Q for the quarter ended June 32021 that will be filed with the SEC today and in subsequent reports, including our current reports on form 8-K.
The company disclaims any obligation to update or revise any forward looking statements, except those required by applicable law.
I will now turn the call over to Frank our President and Chief Executive Officer.
Thank you Mario.
Hey, good morning, everyone and thank you for joining us on today's call.
Second quarter, Mark form his one year anniversary as a public company and I am extremely proud of all that we've accomplished while at the same time recognizing that we have much work left to do on our mission to improve the lives of patients living with rare hematologic diseases and cancers.
Before turning the call over to patent day for more detailed review of our pipeline I'd like to provide some high level thoughts on our second quarter achievements.
First our lead development program once daily PK R activator in alcohol and tablet feedback.
Formally ft 42 O two.
We believe that the new phase one results in sickle cell disease presented at the <unk> meeting in June provide growing evidence that a tab of vivat.
Highly differentiated profile.
The multimodal mechanism of action may be able to modify the course of this complex and multifactorial disease.
More specifically initially that would be that results show sustained hemoglobin increase in nearly all patients and.
An improvement in Red blood cell health and lifespan as measured by metabolic and systemic markers.
Altogether. These data support the potential of a tablet feedback to improve important clinical outcomes, such as data <unk> occlusive crisis or Voc's and.
And our goal is to have a tablet that approved as the first sickle cell treatment that can improve the hemoglobin <unk>.
As we advance our ongoing pivotal phase II III sickle cell trial, the Hibiscus study.
We're also partnering closely with the sickle cell community to improve patient access and care.
And I am very proud to say that we've assembled a talented group of people who are all in and make a difference in the lives of patients living with sickle cell disease.
Looking forward, we plan to start the.
Phase II thalassemia trial later this year.
And a sickle cell pediatric trial in the first half of next year.
Furthermore, we are evaluating that would be that for studying other populations were improving red blood cell health and lifespan could benefit patients.
Turning to our prostate cancer program, which began enrolling a phase one trial earlier this year of the oral CVP P 300 inhibitor ft 751 in men with metastatic castration resistant prostate cancer.
This is a population of manage cancer has either progressed, while on current standard of care anti androgen treatment and door for whom chemotherapy has not been successful.
So the unmet need is significant.
CVT 300 is a novel pathway and we're very interested in the potential to address a broad range of AI driven cancers and in particular patients with <unk> for which there are no approved therapies.
David will shortly provide an update on our ft 751 development program.
With respect to our third development compound the IH one inhibitor <unk> for relapsed refractory AML. The Registrational results presented at <unk> showed that we believe it's a very.
<unk> profile in terms of response rate duration of response.
And survival.
And based on these results we are preparing a new drug application for regulatory submission.
In addition, I'm very pleased to welcome John Bishop.
Our new Chief Technology officer to the executive team.
John will lead chemistry manufacturing control CMC related functions and quality encompassing <unk> early pipeline through commercial.
John's background includes extensive experience in CMC development in oncology and hematology and we're very excited to have him on board.
Before turning the call over to Pat for a more in depth review of the tablet P that kind of a side note.
I'd like to once again this quarter to recognize the contributions made by patients investigators health care workers and our employees as.
As they navigate the challenges posed by the COVID-19 pandemic.
And help us pursue our purpose to transform the lives of patients living with rare hematologic diseases and cancers.
I'll now turn it over the call to Pat.
Thank you Frank and good morning, everyone.
Im delighted to provide an update on our <unk> development program and outlined the Alere decided in our results that were recently presented at scientific conferences.
<unk> is a once daily highly selective PK R activator with a distinct multimodal mechanism of action.
This multimodal mechanism is important and that decreased to three D. P. G improves oxygen binding to hemoglobin S, which decreases polymerization, thereby reducing the cycling and the hemolysis of sickle rbcs.
And those by the richest structures formed by the oxygenated hemoglobin Nash.
The increase in ATP allows the cells to repair damage to the membrane that has incurred through multiple cycles of cell signaling.
Together the effects of decreased to three D. P G and increase ATP improve as the sickle RBC membrane function, which can lead to improved cellular hydration membrane repair and ultimately to formability.
From the beginning of our type of P that clinical development. Our goal has been to generate clinical data that would support an improvement in both the anemia and the base of occlusive events characterized sickle cell disease we.
We've also sought to elucidate the effects of the top will be back on the sickle RBC as measured by numerous markers of Red blood cell health.
We designed a rigorous and comprehensive phase one program and we are very pleased with the updated data that was presented during the <unk> meeting in June.
We believe atop a pea that has the potential to reduce the hemolysis and anemia sickle cell disease by improving RBC health and lifespan piece.
These direct effects on the sickle RBC made then reduce the inflammation and hyper coagulate ability risk that's associated with sickle cell disease, and ultimately through chronic daily dosing of Tahoe peanut may significantly reduce the debilitating vasal occlusive events that characterized sickle cell disease.
Yes.
In terms of our phase one results thus far the following key data were observed from the multiple ascending dose cohorts that administered at Tahoe P that for two weeks and.
And the initial open label extension results administrating at tableau keyed up for up to 12 weeks.
Sustained increases in hemoglobin levels were observed 73% or 11 out of 15 patients treated for two weeks achieved an increase in hemoglobin of one gram per deciliter or greater.
And data from the open label extension showed that 88% of patients or seven out of eight experienced a greater than one gram per deciliter increase that was sustained for up to 12 weeks.
We also observed improvements in RBC oxygenation and affordability.
Red blood cells from sickle cell patients treated with the tablet P that.
For two weeks had increased oxygen affinity with a significant shift in the point of cyclic and improved or formability, when compared to the pre dosing level functions.
We also saw a significant reduction in smaller cities with markers approaching normal levels in some patients.
With just two weeks of dosing, 100% of patients showed a reduction in their particular site counts with some patients normalizing by the end of treatment.
A majority of patients also demonstrated a marked decrease in LDH and indirect bilirubin levels as compared to their baseline levels.
Reduction in systemic biomarkers related to inflammation and hyper collateral ability.
The initial results from patients receiving up to 12 weeks at the top of Pea that treatment indicated that in addition to the improvements in the metabolic markers of Red cell Red blood cell health.
There were improvements in systemic markers of inflammation and coagulation and these can be observed with chronic daily dosing of the top of that.
Finally atop a pea that was well tolerated even at doses up to 600 milligrams daily, which is 100 and 150% of the maximum dose currently being evaluated in the Hibiscus study.
And that.
With this dosing schedule and had a safety profile that was consistent with underlying sickle cell disease.
I have summarized for you today the key results presented during Ehealth and more detailed information can be found in the poster available on our website.
Later this year, we expect to present at a scientific conference updated results from the open label extension trial, and which up to 20 patients will be dosed with 400 milligrams of Otago P that daily for 12 weeks.
With respect to our planned trials, we are on track to begin enrollment prior to the end of this year for thalassemia in the first half of 2022 and patient in pediatric patients with sickle cell disease.
Now turning to allude decided there are mutant <unk> inhibitor being evaluated in patients with relapsed or refractory acute myeloid leukemia or AML.
In the fourth quarter of last year, we announced that the interim analysis successfully met the primary endpoint of a durable complete response rate in a registrational phase II trial.
These data were presented at both the annual <unk> and <unk> meetings in June.
The primary efficacy Evaluable population was comprised of 123 relapsed or refractory AML patients, who received 150 milligrams at the Luna side and it twice a day.
Or at least six months prior to the interim analysis last year.
The key data points were as follows the primary endpoint was achieved with a complete remission or complete remission with partial hematologic recovery at a rate of 33, 3% with the majority of patients having a complete response.
The duration of CR for CRH for people on treatment was greater than 13 eight months. The median overall survival was 10 five months.
For all treated the median overall survival for the for the non CR or CRH responders was 15 months.
And the median overall survival has not been reached for those patients with a CR or a CRH within 18 months survival estimate of 87%.
Also among patients with a complete remission.
Who are transfusion dependent at baseline.
56 day transfusion independence was achieved in 100% of patients.
Who required platelet transfusions at study entry and 88% of patients who require red blood cell transfusions at study entry.
Moreover, our Luna side, and it was well tolerated with adverse events consistent with late stage disease in the heavily pretreated population.
At present, our work continues preparing an <unk>, new drug application or NDA for regulatory filing.
With that I'm going to now turn the call over to Dave who will provide an update on the ft. Seven O five one for <unk>.
Allstate cancer.
Thanks Pat.
I'm going to spend the next few minutes discussing our clinical stage C. V. P. P 300 inhibitor FTE $70.51.
751 targeted targets the androgen receptor pathway by a novel mechanism that we believe has the potential to address many of the resistance mechanism scene for standard of care are signaling inhibitors.
And tumor cell lines ft, $7.51 has been shown to decrease expression, who they are and also decrease the expression of <unk> dependent genes.
Additionally, ft $7.51 has been observed to inhibit prostate cancer cell proliferation in vitro.
And in a patient derived xenograft mouse models in vivo.
We believe the mechanism of action of Ft, $7.51 is applicable to a broad range of resistance mechanisms, including the <unk> splice variant, which lacks the air hormone binding domain and for which there are no approved treatments.
Published data suggest that <unk> may be detectable and approximately 20% to 40% of men. After third line treatment and its presence correlates with substantially shorter overall survival.
Activity in <unk> expressing cancer cells could express could represent an accelerated path to market.
Additionally.
We believe ft 751 has potential for use in earlier lines of prostate cancer therapy, as well as other AAR dependent tumors, such as triple negative breast cancer.
We began enrolling a phase one trial of <unk> 751 in the first quarter of this year and up to 45 men with metastatic castrate resistant prostate cancer.
These men had failed at least one line of therapy and are typically heavily pretreated, receiving one or both of abiraterone and <unk> and in many instances also chemotherapy prior to experiencing disease progression.
The trial utilizes an open label adaptive design, starting with a dose of 25 milligram with titration to as many as six higher doses on a three week on one week off cycle based upon pre defined safety and Tolerability criteria.
Circulating tumor cells or profile, including <unk>, seven expression and genetic markers of resistance.
Clinical assessment will include PSA levels, and radiographic measurements of tumor burden.
This trial was presently in the dose titration phase and we are pleased to announce today that an abstract has been accepted for presentation at the ACR NCI.
RPC virtual International conference on molecular targets and cancer Therapeutics, taking place October 7th attack.
The results will be presented at the conference that will include data from a small number of patients encompassing primarily PK PD and Tolerability and safety information, although some may remain on therapy for a duration sufficient to assess preliminary clinical response.
More comprehensive results from the trial are anticipated in 2022, including tumor response rates and underlying genetic characteristics of responders. We look forward to results from this study to clarify our next steps in development given the high unmet need in this population of men as well as the compounds novel mechanisms of action and positive.
<unk> clinical data.
I will now turn the call over to our Chief Financial Officer Todd.
Thank you, Dave and thank you everyone for joining us on today's call I will now spend a few minutes discussing our financial results for the second quarter of 2021, and then discuss our cash position and outlook.
Our net loss for the quarter ended June 32021 was $43.6 million, which compares with a net loss of $25.4 million for the quarter ending June 30th 2020.
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 $31.6 million for the quarter ended June 30th 2021, compared to $25 million for the quarter ended June 32020 the.
The increase was primarily attributable to a tablet P that development the ongoing phase one and phase two three trials.
D for sickle cell patients and startup costs for the thalassemia trial as well as increases in staff and stock based compensation.
General and administrative expenses were $12.5 million for the quarter ended June 30th 2021, compared to $6.4 million for the quarter ended June 30th 2020.
The increase was primarily attributable to increased equity based compensation executive and staff hiring professional fees and insurance.
Our cash cash equivalents in marketable securities balance of June as of June 30th 2021 was $570.8 million compared to $645.6 million at the year end 2020.
Cash use in the second quarter reflects operating expenses and working capital requirements to support operations.
Overall, we continue to be in a very strong financial position with funding through the third quarter of 2024.
<unk>, we can now take on questions.
Thank you as a reminder, task a question you will need to press star one on your telephone to withdraw your question press the pound King please standby, while we compile the Q&A roster.
Our first question comes from the line of Ameren Nieland from Cantor Fitzgerald. Your line is now open.
Hi, Thanks for taking my question.
Our study and I guess, just curious what the rationale is for including sickle cell patient tourists transfer.
Transitions.
And I'm not recruiting stoppage listeners.
Yeah.
Right.
Good morning. Thanks for the question, let me turn this over to Pat.
Yeah. So the.
Dallas EMEA charter all the pilot 201 trial that we are initiating includes both transfusion dependent and non transfusion dependent thalassemia as well as a cohort of sickle cell patients on chronic transfusions as well.
Got it Okay, and then just a quick follow up color, what youre trying to learn.
First of all throughout Europe.
The study.
So the the endpoints of that we'll look at hemoglobin response in both the transfusion dependent populations as well as the.
Pre transfusion as well as non transfusion dependent thalassemia patients and those patients receiving chronic transfusion look at.
The impact on reducing the transfusion burden in those patients.
Great excellent.
Ooh.
Thank you. Our next question comes from the line of Thiago <unk> from Credit Suisse. Your line is now open.
Hey, Thanks for taking the question. So one follow up on the 751. So again just to set expectations correctly for the October update I'm, assuming we're probably not going to get sufficient biomarker data across across the patients.
So just wanted to clarify if that is the case again, we're generally going to try something that disrupts pay from an efficacy perspective.
But regardless of the data coming in October or perhaps next year. What are you looking for given the trial design.
The novel mechanism of action.
As early PSA response at a certain level indicative of.
What you're looking for I'm, just trying to understand how to interpret that data given the novelty of the approach. Thank you.
Yes, thanks for the question Thiago.
Just a high level thought and let me turn it over to David and Pat for further comment.
We've got it before we are on track to deliver safety and PK PD.
<unk> for the Triple meeting later this year and the full results. The final results will be sometime mid next year. So that's the broad sort of I would say expectation.
And certainly depending on how the trial goes we may have some other endpoints, but Dave why don't I turn it over to you.
Sure Hi, Thiago.
Yes, Youre correct will be.
A limited number of patients that will be at a dose.
This year.
We expect to be in the therapeutic range and remember that.
The way PSA responses defined that's a 90 day PSA response so.
Not only has to be at a therapeutic dose.
But had been on treatment for 90 days in order to assess that so we think that we'll have a much more robust dataset.
The sort of middle of next year timeframe.
In addition to looking at PSA response, which is really a very typical.
Parameter that slipped out in these early patients we will be looking at.
Radiographic response.
So we can correlate the two of those.
Got it appreciate the details there thank you.
Thank you. Our next question comes from the line of Maury Raycroft from Jefferies. Your line is now open.
Hi, good morning, everyone and thanks for taking my questions.
First one is on top of your update at <unk> showed it can improve overall circle RBC functional health and increase hemoglobin production, but we're just wondering if you can help set expectations for ash on what to be looking for on a POC use if it if.
The update will be robust enough to get some perspective, there or at what point should we be able to learn more about that.
Hey, Laurie good morning. Thanks, Thanks for the question.
Just at a high level.
What we presented at <unk> just recently in June.
Basically if you.
Look at what we're planning to present at Ash and it's basically that.
Those endpoints, albeit with more patients.
That have completed now the 12 week dosing regimen. So that's what to expect so when you take a look at the <unk> data.
Types of data that we presented that would just think about that.
With a full 12 week course of therapy for more patients.
With respect to V O sees.
Definitively have the VLC data until we conduct a hibiscus trial, but in the meantime, there are some.
I'd say markers that we're looking at that maybe.
Maybe associated with Boc and certainly Dave you might wanted to talk a little bit more about that.
Sure Hi, Morey.
So we know that the vlcc's represented complex process.
That starts with cycling, but translates into things like.
Coagulation at the site of vascular damage.
And inflammatory responses.
So the study is actually carefully looking at a number of innate immune cytokines. We're looking at factors the activation of the coagulation pathway, which actually is quite activated typically at baseline for these patients so actually looking at the ability to reduce that activation.
And.
And then some other more systemic Merck Biomarkers for instance.
The.
<unk>.
The reduction in the drive that particular site.
Drive so those are the kinds of systemic biomarkers that I think will build confidence that we're going to see.
Difference in DLC and the Hibiscus trial I'll just pointed out.
But if you look at for instance, the one approved drug that we have the reduces vlccs well too now with.
With Chris Moser map patients still experience Vlccs and that's why it's very challenging and the absence of a placebo.
In a blinded study to positively.
Say that you are seeing a reduction of <unk> in a small sample open label study and Thats, where theres a high need.
<unk> study, but we think these precursors of coagulation immune activation red cell deformability are all.
Very <unk>.
Strong precursors that we will.
Have a benefit for <unk>.
Got it that's all really helpful perspective, and then.
Also just wanted to check on you mentioned during the prepared remarks, the potential to explore tableau with other indications and so I was just wondering if you can talk a little bit more about the plan there and when we could learn more about that strategy.
Yes, MRI, we're hard at work looking.
At the possibility of some other indications in populations.
Outside of the ones, we've already talked about that as the valve program and Pete sickle cell, we are considering a number of other <unk>.
Indications in populations, we won't be ready to share that until likely towards the end of the year. So that's the timeline that we're operating under.
Got it and last question for me just for the Triple meeting update is there anything else you can say on baseline characteristics and just wanted to clarify if I'm. If youll have perspective into whether patients are every seven or not at baseline and at that point.
Sure, Dave do you want to or Pat do you want to speak to that.
Sure I'll just say.
Obviously this is a heavily pretreated population, while they only have to have failed one line of therapy typically.
<unk> had multiple lines may affect had chemotherapy.
We are doing.
Circulating tumor cell in Cte DNA analysis, so we will have.
Both the <unk> status and mutational data however that data is batched. So if we don't get the data for instance, with each patient, but we batch. It so the amount of data that would be available will depend on it.
Some sense on timing of when patients enter the study.
That's they end up and so that's why it's I think it's really.
Worth keeping in perspective that this will be early data, we will share what we can as we always do but by no means should.
Should we think this is being comprehensive since we're still in the dose escalation phase.
Got it that's helpful. Thanks for taking my questions.
Okay.
Our next question comes from the line of Mark Breidenbach from Oppenheimer. Your line is now open.
Hey, good morning, guys. Congrats on the progress just wondering.
If the early success of surveys age Io trial.
<unk> in a newly diagnosed AML setting.
That has had any positive impact on your partnering discussions.
For all of two.
Although poseidon.
And I'm wondering if it's inspired plans for four months of run rate.
A randomized trial in a similar setting.
Hey, Mark Good morning, Yes, we're very excited about the sudden program as we've talked about before.
The data presented are actually pretty remarkable.
That said, we are committed to partnering strategy that we've talked about before and we are in partnering discussions and so I can't comment on those and what the partnership will entail, but what I can say is we're very encouraged by.
The program and also the interest in the area.
Okay.
Just a quick follow up on the sickle cell program it might be a little early but maybe you can give us a sense for what the pediatric sickle cell trial might look like in terms of size and if we can expect any pediatric specific efficacy endpoints things like trans cranial.
Doppler flow velocity things like that.
Yeah, Let me just say at a high level, we're still in a course discussions with the regulatory authorities. So nothing definitive but certainly we have some thoughts.
Pat maybe you can share some initial thoughts.
Yes, I think we're looking at all opportunities in this setting.
Of this study.
The intent is to look at the.
The pharmacology, the pharmacodynamics and the safety.
And that's a careful.
<unk> of the top O P that young patients.
The additional efficacy or clinical benefit endpoints are part of that discussion as Frank mentioned.
Got it thanks for taking the questions.
Okay.
Thank you. Our next question comes from the line of Robin Garner from Craig Hallum. Your line is now open.
Congratulation on the advances in the quarter I have a question for you on the top al looking at the upcoming studies in thalassemia in pediatric patients how could we expect the hemoglobin response to tableau to differ given underlying differences in these populations versus.
Adults with sickle cell disease.
Hey, Robyn thanks for the question.
Pat you want to you want to take this one.
Yes, I think it's a it's part of the.
Questions, we're asking to characterize the activity of the type of people that in these particular patient populations.
<unk>.
The activity of it's hover should.
It will be active in all red cells.
And as such the activity that we're seeing in sickle patients red cells that.
Including patients under 18 already is giving us confidence that.
This mechanism should benefit younger patients with sickle cell disease, as well as thalassemia patients with unstable hemoglobin.
Would you expect to see more of an effect in pediatric patients, who typically have very different responses than adults to sickle cell.
<unk> therapeutics.
Oh well.
We're very pleased with the results we've seen to date in the sickle cell adult patients with a very robust hemoglobin response generally across the entire patient population.
It'll be hard to improve on that I suspect.
And we'll see I think youre right.
<unk> younger have done better with standard of care in terms of those therapies.
Generally healthier without.
The accumulated morbidity so.
That gives us confidence that.
What we see in adult patients will translate to better or nearly equivalent to earn better responses.
Young population.
Okay. Thank you for answering my question.
Great.
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.
Alright, well.
We thank everyone for taking the time to participate in today's call.
The second quarter of 2021 brought important new data for our lead.
Compound of tableau and sickle cell as well as our progress on ft 751 in prostate cancer as well as the Luna side didn't AML.
And it's really continuing our mission to bring new therapies to patients with rare hematologic disorders and cancers.
We look forward to sharing more progress on our development programs prior to the year end.
This concludes today's call. Thank you all have a great day.
This concludes today's conference call. Thank you for participating you may now disconnect.
[music].
[music].
[music].
[music].