Q1 2021 Forma Therapeutics Holdings Inc Earnings Call
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Welcome to the foremost therapeutics first quarter 2021 financial results and business update conference call. All participants are currently in a listen only mode. Following management prepared remarks, we will hold a question and answer session to ask a question at this time.
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This call is being recorded today May 14, 2021, I would now like to turn the conference of the true Mario Corso. Please go ahead.
Thank you operator, this is Mario Corso senior director of Investor Relations at forma.
Good morning to our listeners and welcome to today's call to review first quarter 2021 financial results and business update.
On this call I'm joined by frankly, our President and Chief Executive Officer.
Kelly, our Chief Medical Officer.
Dave Cooke, our Chief Scientific officer.
And Todd Shegog, Chief Financial Officer.
Before we begin I'd like to caution listeners that comments made and financial information provided during this conference call includes certain statements that are estimates beliefs forward looking and are subject to various risks and uncertainties.
Statements made during this call that are not statements of historical or current facts are intended to be forward looking statements pursuant to the safe Harbor provisions of the private Securities Litigation Reform Act of 1090 fives.
We want to emphasize that such forward looking statements reflect our current expectations and assumptions regarding timing success and data announcements of our current ongoing clinical trials.
Therapeutics potential and clinical benefits and safety thereof planned regulatory submissions, our financial condition, our business operations development efforts and the potential impact of COVID-19 on our business from clinical development and relationships with third parties and collaborators.
And on 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 annual report on form 10-K for the year ended December 31, 2020, and our quarterly report on form 10-Q.
For the quarter ended March 31, 2021 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 as required by applicable law.
I will now turn the call over to Frank our President and Chief Executive Officer.
Thank you Mario.
Morning, everyone.
Thank you for joining us on today's call.
I'm pleased to say, we continue to make strong progress in the first quarter with our R&D pipeline.
And I will share some high level thoughts with you before turning it over to Anne day for more detailed remarks.
I'd like to first knowledge of time effort and contributions made by patients investigators health care workers and our employees.
Navigating challenges posed by the COVID-19 pandemic.
And make it possible for us to pursue our purpose and that is to transform the lives of patients living with rare hematologic disorders cancers.
I'm proud of our talented employees and how they live on our guiding principles and values every day and per.
A suite of our purpose I believe.
Our patient focused culture is a key strength, which differentiates Columbia.
During the first quarter, we achieved important milestones for our two development programs Ft 42, two our oral PK R activator being developed for sickle cell disease.
And F. T 751 on oral CVP P 300 inhibitor for metastatic castrate resistant prostate cancer.
Starting with Ft 42, two.
The potential foundational therapy with the potential to provide benefits from both hemoglobin and basal occlusive crisis in a once daily pill.
We disclosed that the phase II III Hibiscus study began enrolling patients in the first quarter.
In addition, we disclosed initial blinded results from the two week from.
Second two week cohort in a randomized multicenter placebo controlled phase one trial.
Successfully completed the mad portion of the trial.
The key takeaways were as follows number one.
No dose limiting toxicity or significant treatment related adverse events have been identified number two hematologic <unk> hemolytic responses indicated a consistent biologic effect an improvement in red blood cell health.
And responses were typically burst at the end of the 14 day treatment period.
Suggesting patients may benefit from longer duration of treatment beyond the 14 days.
Importantly completion of the Mad cohorts allowed us to continue moving forward with the next phase of this trial.
The ongoing 400 milligram 12 week open label extension.
And finally results from the Mad cohorts together support the 200 milligram and 400 milligram doses being evaluated in the ongoing phase III trial to Hibiscus study.
The next steps on this program will be the presentation of the combined unblinded Mad cohorts as well as initial open label extension results to date.
At the European Hematology Association or <unk> meeting, taking place virtually June 9th through the <unk>.
17.
Building on the ongoing sickle cell program on the thalassemia trial getting underway later this year.
We see potential for F. T 42, two to benefit us.
Their patient populations with high unmet medical needs.
Including hemolytic anemias and rare diseases.
We're taking a review internally and expect to provide more specific.
During the second half of the year.
With respect to Ft 751, our oral CVP P 300 inhibitor from metastatic castrate resistant prostate cancer, we reported the first patient dosed in the ongoing phase one trial early in the first quarter.
And are pleased with the initial interest in the trial among both the centers and patients.
We look forward to the initial results from this trial in the fourth quarter of this year.
And in particular, the data for patients with air B seven spice periods for which there are no currently available treatments.
I'll now turn over the call to Pat to discuss at day 42, two as well as a little side name in more detail.
Thank you Frank and good morning, everyone.
Im delighted to provide updates on two of our programs F. T 42 O two for treating patients with sickle cell disease.
We decided that for treating patients with relapsed or refractory AML with an <unk> mutation.
As Frank mentioned, we are very pleased with the completion of the two week Mad dosing cohorts for the F. T 40, chiller to program demonstrating at 42 or two was well tolerated with a favorable safety profile.
We also saw a hemoglobin increase on one gram or greater at the end of the 14 day treatment period.
<unk> 71 per cent of the patients.
And nearly all patients experienced benefit and other important surrogate markers. These include the particular site counts LDH <unk> bilirubin, indicating improvement in Red blood cell health.
Looking forward, we will disclose a complete analysis of the unblinded two week Mad cohorts and for the first time, we will be providing an analysis of the initial patients receiving F. T 42 O two beyond the two weeks of dosing from the open label 12 week extension cohort.
These results will be presented at the <unk> annual meeting taking place channel nights through the 17th.
We expect to have additional data on RBC health, which combined with the improved red cell functional studies supports the potential for reduction in the rate of the ocs.
Altogether, we are pleased with the emerging profile of F. T 42, O two indicating potential benefits beyond hemoglobin improvement.
We would also like to take this opportunity to reiterate that our ongoing phase two three trial the Hibiscus study.
It's designed to be consistent with FDA guidance and reflects regulatory feedback.
This study design includes two separate primary endpoints hemoglobin change following 24 weeks of treatment.
And supportive and accelerated regulatory approval.
And a 52 week rate of the seas in support of our whole regulatory approval if positive.
Finally, with respect to Ft, 42, O to our planned trials and policymakers and pediatric sickle cell disease are progressing well with enrollment anticipated to begin in the second half of this year for thalassemia in the first half of 2022 in pediatric patients with sickle cell disease.
Now turning to allude decided there are mutant on each one inhibitor being evaluated in patients with relapsed or refractory acute myeloid leukemia or AML.
In October of last year, we announced that the interim analysis successfully met the primary endpoint of a durable complete response rate in this registrational phase II trial.
We are pleased to announce that these data have been accepted for oral presentations at both the annual <unk> and <unk> meetings in June.
In addition, our work continues on preparing a filing of a new drug application or an NDA to the U S. FDA.
With that I'm going to now turn the call over to Dave who will provide an update on the F. T 705, one program for prostate cancer.
Thanks Pat.
I'm going to spend the next few minutes discussing our clinical stage CVP P 300 inhibitor ft $7 51.
Ft 751 target the androgen receptor pathway by a novel mechanism that has the potential to address many of the resistance mechanism seen per standard of care.
Signaling inhibitors.
And tumor cell lines Ft, 751 has been shown to decrease our expression and also decrease our dependent gene expression.
In addition, ft 751 has been observed to inhibit prostate cancer cell proliferation in vitro and in a patient derived xenograft model in vivo.
We believe the mechanism of action of Ft, $7 51 is applicable to a broad range of resistance mechanisms, including the ARV seven spice strength, which lacks the AAR hormone binding domain and from which there are no approved treatments.
Published data suggests that <unk> may be detectable and approximately 20% to 40% of men after third line treatment and it correlates with substantially shorter overall survival.
Activity in <unk> expressing cancer could represent an accelerated path to market.
In addition, 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.
Our phase one trial began enrolling early in the first quarter of this year.
This trial is planned to enroll up to 45 men with metastatic castrate resistant prostate cancer, who failed one or more lines of standard of care utilizes an adaptive design, starting with a dose of 25 milligrams 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 will be profiled, including <unk> seven expression and genetic markers of resistance and clinical assessments will include PSA levels and radiographic measurements of tumor burden.
We expect to have initial clinical results in the fourth quarter of this year and a subset of patients.
These results May include safety Tolerability PK PD.
And preliminary biomarker data.
A more complete results from the trial are anticipated in 2022, including an assessment of tumor response rates and underlying genetic characteristics of responders.
The high unmet need in this population of men and the novel mechanism of our molecule. We are enthusiastic regarding the promise of ft, $7 51, and look forward to sharing data as they become available I will now turn the call over to Todd <unk>, Our Chief Financial Officer.
Thank you, Dave and thank you everyone for joining us on today's call I will first spend a few minutes discussing our financial results for the first quarter of 2021, and then discuss our cash position and outlook on.
Our net loss for the first quarter of 2021 was 36.0 million, which compares with net income of $11 2 million for the quarter ending March 31 2020.
The year over year change in net loss was attributable largely attributable to a onetime gain on the divestiture of our hit discovery capabilities and income tax benefits in the first quarter of 2020.
Research and development expenses were $26 3 million in the quarter versus $23 2 million for the quarter ending March 31 2020.
This increased spending supports progress with our two compounds in development.
Including the phase one and phase two three trials for F. T 42 O to sickle cell disease and the start of the phase one trial for ft, $7 51 in metastatic prostate cancer.
General and administrative expense in the quarter was $9 9 million as compared to $8 9 million in the quarter ending March 31 2020.
This increase reflects the costs related to being a public company.
Stock compensation expense professional fees and insurance and increased compensation expense.
Our cash cash equivalence and marketable securities balance as of March 31, 2021 was $603 7 million compared to $645 6 million at year end 2020.
Cash used in the first quarter reflects the net loss as well as changes in working capital.
Our current cash run rate continues to extend through the third quarter of 2024, which includes ongoing clinical trials as well as planned commencement of ft 42 O two development in thalassemia and the pediatric sickle cell population.
Overall, we continue to be in a strong financial position and are well financed through important upcoming clinical milestones.
Operator, we can now take questions.
Thank you and as a reminder to ask a question simply press star one on your telephone to withdraw your question press the pound or hash key.
On mom and while we compile the Q&A roster.
Our first question comes from Anthony line with Cantor Fitzgerald. Your question. Please.
Hi, good morning, so for fortunate on account can you just speak to the trend toward increasing hemoglobin response that you saw for the 14 day treatment periods for the first two cohorts, whether you'd expect to see potentially a better response. So far this walk on treatment period, and the open label day that'd be hot.
Hi, and thanks for the questions frankly here, let me just.
Provide some high level remarks, and turn it over to Pat for some additional thoughts.
Certainly the data we have now.
Primarily from the first two weeks of treatment and were certainly looking forward to the open label extension data, which will provide a look at various cuts on patients some will have completed.
<unk> weeks and some old completed to anywhere from two to 12 weeks and so we'll have a spectrum of patients.
And certainly we're encouraged by what we saw in the early data from the two Mad studies. So let me turn it over to Pat for some additional thoughts there.
Great. Thanks, Brian Yeah, I think he said it well.
On a key here is as people are aware from the 600 milligram or Matt to cohort patients participating in that group were allowed to rollover into the 12 week open label.
And as we previously disclosed six out of the first nine patients enrolled in the mat to did rollover into the 12 weeks. So we will be able to have.
On a comparative comparison, if you will.
In that population.
Based on their two week experience and the Mad two versus their two week or greater exposure experience in the in the 12 week cohort.
Great that's helpful.
Yeah.
Thank you.
Our next question comes from Andrew Berens with SBB Leerink. Your question. Please.
Hi, Good morning, guys a couple from me please.
I was wondering if you could give us some color on the decision to go into the hemolytic anemia is with.
Would these be acquired or inherited chronic or acute treatment and I guess, how many do you think are addressable by 40 to go to on the peak share class and then I do have a quick question on the prostate cancer program. Afterwards, that's okay.
Sure. Thanks for the question Andy.
So we're looking through.
And going through our analysis, and we will share more of that in the second half of the year.
Certainly there are other PK or agonists one in particular.
It is looking at PK PD.
Certainly that's one of the areas, we'll look at but we haven't made any decisions yet and so at the current time, we're doing are.
<unk> so to speak and we'll have more information on share in the second half of the year, but we certainly think there is more potential than just be just to be focus on sickle cell disease and thalassemia.
Okay.
More than potentially PK D also because a very broad area.
Yes, it's potentially so I don't want to make any comments at this point until we complete our analysis, but certainly theres a theres potential beyond the two initial indications that we talked about <unk>.
Okay.
And then on the prostate cancer program will be upcoming data set. That's presented include <unk> seven patients one way or all comers and then just what's the pathophysiological rationale for targeting <unk> seven with $7 51.
Sure, Let me answer that and then turn it over to day for some additional comments on so.
We will share what data we have around about the fourth quarter of this year. It will be the initial cut of the data and certainly right now we're enrolling all comers late line patients.
Some of whom will be RMB seven and so we'll have we will have those cuts on the data.
Albeit the initial analysis now let me turn it over to Dave for some additional comments.
Yeah, Hi, Andy.
I think as Frank said, we're really kind of doing in an all comers study in phase one we don't want to make assumptions about where the.
On the drug may have efficacy or not on that being said given the what we know about AB seven we expect to have a number of patients with the AB seven phenotype. The rationale there is twofold. One we know that these spice variance what they do is they tend to.
Delete the protein domains that include the ligand binding domain, so meaning the C terminal is where you have truncation and the N terminal where.
We're CVP and P 300 interact is intact. So we think the mechanism of our drug by disrupting that.
Should continue to be useful the second really is empirical is that we can show for us.
Instance, that AB seven expressing cell lines, which are resistant to <unk> for instance are sensitive to ft $7 51.
Okay. Thanks appreciate the color.
Our.
Question comes from Thiago <unk>.
With credit Suisse. Your question please.
Alright, Thanks for taking the question. So just a follow up on RBC health data that we might see a we got some questions on the osmose scan data.
So just want to understand a little bit better.
What day trained or patient variability for that specific measurement and how well establish it is in sickle cell. It's not something that you see for every single program. So any color there would be helpful. Thanks.
Thanks for the question Thiago, Let me first ask.
Dave to give a little bit on color on the osmose scan and maybe perhaps Pat can talk a little bit further after that so okay.
Sure.
Well first of all that measurement has been around for 40 years or so was developed in the early eighties and it's been well validated as to what it indicates although it is a complex measurement.
It's not typically used in clinical labs, because our commercial devices have only been available fairly recently.
What it indicates our several things.
It indicates critical hemolytic volume, meaning it has to do to the surface to volume ratio of red cells and it also indicates elements of membrane function for instance on their high osmotic pressure when items are leaking in water water also 10 or what items leaking out water tends to leak out and the <unk>.
Moving to restore iron gradients is.
Key element of having appropriate ATP levels and so we're able to show that we can handle the vs. Osmotic radiance, we think it demonstrates the health of the red cell and its ability to maintain.
Dynamic balance.
Pat.
Yes, I think the other piece of that is yes. These are complex assays that.
It can be a bit challenging.
As each lab, we've learned has different parameters, but what's important for what we've done is we've centralized the analysis of all samples are sent to one lab. So theres a consistency in that analysis.
And based on that work in the Mad cohorts will.
Come to appreciate that yes between the patients there might be variability just based on the phenotypes of the individual patient, but there's consistency within the patients themselves. So having a patient and certainly our experience with patients who are enrolled in both the bad and the open label that we.
See a very consistent.
Phenotype for the individual patient over a period of time, where they're not receiving treatment. So.
We'll have as part of our complete analysis of the bad cohorts is all of that information in those groups in total.
Great very helpful. Thank you.
Thank you and as a reminder, if you have a question simply press star one on your telephone.
We have a question from the line of Mark Breidenbach with Oppenheimer. Your question. Please.
Hey, good morning, guys and congrats on getting enrollment started in biscuits.
We've seen a little bit of data, suggesting there was a substantial dip in healthcare provider visits by sickle cell patients during the COVID-19 pandemic and I'm just wondering if your clinical sites are seeing traffic starting to normalize in terms of sickle cell patients coming back to the clinic and I'm also wondering.
If you have any plans to open clinical sites in ex U S territories, especially in Africa to potentially help accelerate enrollment and hibiscus.
Thanks for the question Mark.
I guess on me.
Talk about the impact of the pandemic broadly and then turn it over to Pat in terms of.
On the steps, we've been taking to navigate the pandemic.
Sure.
First I'll say that certainly the pandemic has on an impact on our efforts.
So we've had really double down in.
And think about not only on the traditional ways, but.
Non traditional and innovative ways to.
Enrolled patients and so on based on that and the team's efforts we've been able to deliver the data that we've been talking about so far and so.
We will see how this plays out.
Moving forward, but so far we've been able to navigate.
COVID-19 and the pandemic.
Pat why don't you talk a little bit more about specifically, what we're seeing at the science.
Sure.
I think you know.
Really the activities globally have been in parallel not just U S focus, but also ex U S in terms of getting.
Countries up and running.
But like what you can read in the papers.
The activity at various countries and centers has really been impacted based on how the severity of COVID-19 is.
Hitting or impacting those hospitals and countries.
So what we're seeing really is.
More activity in the U S. As you can imagine that the vaccines have rolled out and.
Sites are starting to.
Catch up in terms of clinical research. These are coming on line certainly.
And thats for our benefits in the sense that we're already there we've already.
Have the approvals in contracts so as.
As COVID-19 and the vaccine in the U S is kind of diminished in the vaccine is approved.
Health care activity.
This is starting to open up so we're looking for.
We're hopeful I guess, that's how we would put it.
Okay in terms of plans too.
To open sites in Africa, where there are high concentrations in sickle cell patients.
Yes, we're looking we have a number of countries that we have been.
And working on and it's really.
Dependent on availability.
And resources.
It's from our own internal activities, our CFO as well as the countries themselves at this point, but we're not we're not disclosing specific countries, but we're looking everywhere right.
Okay got it.
On one follow up on the prostate cancer program.
Data were ex.
<unk> in fourth quarter.
Just to be clear that will include some of the CTC profiling data. So so ARV seven status in <unk>.
Specific point mutations confer resistance day are targeting therapies that sort of thing or is that is that going to come later in 2022.
Go ahead and take that one.
Yes, Mark I don't think we've been specific yet.
And part of that is going to depend on our strategy for.
We're doing the analysis on how it gets batch so I don't want to commit for.
The genotype data.
At the end of the year, but obviously that's really important.
Interpret the results globally, so we're going to be working hard at that but I don't think were yet.
Have a specific commitment around whether that day it'll be available by the end of the year.
Got it okay. Thanks for the clarification and congrats on the progress.
Thank you and this concludes our Q&A session I would like to turn the call back to Frank Lee for his final remarks.
Well. Thank you everybody, we just want to thank everyone for taking the time to participate in today's call. The first quarter brought some important progress in our R&D pipeline.
<unk> focused on rare hematologic stores in cancers.
We're executing our strategy to bring these new medicines to patients and look forward to sharing more progress during the course of the year. So have a great day everybody. Thank you.
And this concludes today's conference. Thank you for your participation and you may now disconnect.
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