Q4 2021 Forma Therapeutics Holdings Inc Earnings Call
Welcome to the former Therapeutics year end 2021 financial results and business update conference call. All participants are currently in listen only mode.
Management's prepared remarks, we will hold a Q&A session.
To ask a question at that time. Please press the Starkey followed by one on you touched on phone.
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As a reminder, this call is being recorded today March <unk> 2022, I would now like to turn the conference over to Mario Corso. Please go ahead.
Thank you Michelle.
Mario Corso senior director of Investor Relations at Formula.
Good morning to our listeners and welcome to today's call to discuss <unk>.
Year end 2021 financial results and business update.
On this call Im joined by frankly.
<unk> Chief Executive Officer.
Patrick Kelly, our Chief Medical Officer.
Dave Cooke, our Chief Scientific officer.
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 any statements made during this call that are not statements of historical or current facts are intended to be forward looking.
<unk> pursuant to the Safe Harbor provisions of the private Securities Litigation Reform Act of 1095.
Want to emphasize that such forward looking statements reflect our current expectations and assumptions regarding timing enrollment.
<unk> data announcements of our current and ongoing clinical trials therapeutic potential in clinical benefits and safety of our product candidates planned regulatory submissions, our financial condition and capital requirements, our business operation development plans the potential impact of COVID-19 on our business and.
Clinical development and relationships with third parties and collaborators and are neither predictions nor guarantees of future events or performance.
Actual results could differ materially from those stated or implied by these forward looking statements due to risks and uncertainties associated with our business, including those under the heading entitled risk factors in our.
Reports on Form 10-K for the year ended December 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 law.
Before turning the call over to Frank I'd like to mention two upcoming activities as outlined in today's press release.
We'll be participating virtually in the Oppenheimer Healthcare conference March 15 to 16.
And we will be holding our first R&D pipeline review in May 2022.
Further information on these events will be made available on our website www dot pharma therapeutics dot com with that I will now turn the call over to Frank our President and Chief Executive Officer.
Thank you Mario.
Good morning, everyone.
My pleasure to review form his significant accomplishments in 2021.
And outline our plans for 2022 as well as the longer term vision for the company.
As you know <unk> purpose is to transform the lives of patients living with rare hematologic diseases and cancers.
In support of our mission.
2021 was a year to generate early stage clinical trial results on our two active clinical development programs.
Have a pea that for people living with sickle cell disease.
And ft, $7 51 for men living with metastatic castration resistant prostate cancer.
These clinical data form the building blocks for expanded development in 2022 and beyond.
In the latter part of 2021, we completed the phase one trial of our once daily <unk> activator atop a payback.
This molecule has the potential to be a foundational therapy for sickle cell disease.
<unk> multimodal mechanism of action.
Results were presented from the open label extension of the trial at the Ash annual meeting in December .
And we believe this was the most rigorous and comprehensive phase one program conducted in people living with sickle cell disease.
Not only assessing the typical measures of hemoglobin and hemolysis, but also analyzing numerous biomarkers of red blood cell health.
Pat will review the impressive results here shortly.
While we completed the phase one trial and continue to enroll the phase II III Hibiscus study, we also furthered our commitments to improving access to care with the recent announcement of our former bridge program.
Aid in pediatric to adult transition as well as the additional.
The addition of a renowned leader in sickle cell disease Doctor EAP assumed co.
In the newly created role of Chief patient Officer.
Unlike similar orphan diseases, such as hemophilia cystic fibrosis sickle cell disease remains under funded under resource and underappreciated.
As a result, sickle cell patients Smiths endure barriers to quality of care like no other disease.
We believe every sickle cell patient deserves high quality care.
And we're working closely with the community to make this happen.
Being a trusted partner to the communities. We serve is important for rare orphan diseases.
But it is particularly the case with sickle cell disease.
Another important 2021 milestones achieved with our CVP 300 inhibitor ft $7 51.
With the presentation in October at the Triple meeting of the first in human results from our ongoing phase one trial in metastatic castration resistant prostate cancer.
We're encouraged by the safety profile and the early data, suggesting that CBP 300 inhibition may provide a novel approach for patients who have failed standard of care.
Dave will detail the results shortly.
Now looking ahead 2022 is a year of advancing the clinical development of a tablet P that and expanding <unk> pipeline to position the company for longer term success.
We continue to enroll the global phase II III Hibiscus study and expect the interim analysis, one dose selection decision towards the end of this year.
We're also broadening our tablet <unk> development in sickle cell disease to include the transfusion dependent and pediatric populations.
We are also progressing in new areas, such as thalassemia, and low risk Myelodysplastic syndrome or Mds.
While also evaluating other disease areas in order to bring all of that we have learned about PK, our mechanism and red blood cell health two additional areas with substantial unmet patient need.
We expect to provide updated data for <unk> 751 in metastatic castration resistant prostate cancer later in the year, including safety Tolerability profile and activity.
This data will enable us to discuss the clinical development approach for ft, 751, and positioning in the current treatment landscape.
Finally, this year with respect to our third development compound the IH one inhibitor the Luna side.
For relapsed refractory AML, where.
We're progressing our new drug application as we continue a partner strategy for this molecule.
I am proud of all that we accomplished it format last year and enthused about our plans for this year and beyond for.
We're building a company that is positioned for success with multiple clinical stage assets in development.
Research capabilities to sustain our strategy.
Substantial capital to achieve our near term and long term objectives.
As we discussed before the COVID-19 pandemic has created substantial ongoing challenges for both the clinical trial sites and for patients.
We're observing conditions improving over time and are working very closely with the clinical sites.
As well as the sickle cell community.
With that I'd like to acknowledge those who help navigate the ongoing challenges posed by the COVID-19 pandemic, including patients investigators.
Their workers and our employees.
As we pursue our purpose to transform the lives of patients living with rare hematologic disorders cancers.
I'll now turn over the call to Pat.
Kelly to discuss recent tablet P that and <unk> results in more detail.
Yes.
Thank you Frank and good morning, everyone.
I will provide a brief overview of our development programs for <unk>, and <unk> decided nib, including clinical results that were presented at the Ash annual meeting in December .
Tom will be that is our once daily selected PK R activator with a distinct multimodal mechanism of action that improves oxygen binding and reduces hemoglobin polymerization by decreasing to <unk>.
And also repairing the damage sickle RBC membrane by increasing ATP.
We believe that the effects of decreased two three dbg and increased ATP can improve the sickle RBC health and lifespan.
Potentially modifying the course of sickle cell disease.
In December at the Ash annual meeting complete results were presented from the 12 week open label extension of our Phase one <unk> study, which we believe is the most comprehensive characterization of any novel agent in sickle cell disease.
We presented conventional measures of response.
Including hemoglobin particular sites and hemolysis and also presented critical RBC parameters, such as the formability oxygen binding and hydration.
We also looked at systemic markers of inflammation coagulation and hypoxia.
Annualized voc's prior to during and following <unk> administration.
Key findings from the 15 patients with sickle cell disease treated up to 12 weeks included the following.
A topic that dosing of 400 milligrams daily was well tolerated with a safety profile that was consistent with the underlying sickle cell disease.
All patients experienced an improvement in hemoglobin levels with 11 out of 15 or 73% of patients achieving a greater than one gram per deciliter hemoglobin improvement with a maximum increase of one five grams per deciliter.
An increase in the lifespan of Rbcs was observed based on the increase in the hemoglobin in a concomitant reduction in the absolute ridiculous site count.
There was reduced Intervascular hemolysis, a key driver of sickle cell pathology, which was shown by decreases in indirect bilirubin and lactate dehydrogenase.
Improved sickle RBC. The formability was demonstrated as measured by the oxygen scan with a shift in the point of cycling to lower oxygen pressures.
Improved RBC hydration with observed with a decreased intracellular hemoglobin concentration and a decrease in the number of dead cells.
There was a reduction in systemic markers of inflammation coagulation and hypoxia.
Knowing the potential to reduce the downstream measures of sickle cell disease pathophysiology.
In terms of visa occlusive crises in the 15 patients dose for up to 12 weeks. There was only one VLCC requiring hospitalization reported while on treatment, which was precipitated by a COVID-19 infection.
Notably that patient remained on a title that ended well.
In the four weeks post the type of people that treatment. There were three reports of DSC, which we believe is consistent with that seen in other sickle cell disease trials and largely reflects the removal of a therapy that was having a positive clinical impact.
We also conducted an analysis of patient reported DLC rates in the prior 12 months prior to the trial enrollment and compared it to the boc rate we observed during the treatment period, which totaled over three years of patient exposure.
There was a 68% reduction in the annualized rate of the ocs, resulting in hospitalization.
<unk> finding in the context of the benefits we observed in the April eight aforementioned measures of sickle cell disease and RBC health.
Overall, we are very pleased with the phase one of the top IP that results gaining significant insights into the consistent PK PD properties at the top of Peanuts, and the importance of RBC health.
The results strongly support our ongoing phase III <unk> III, Hibiscus study and provide a rationale for our ongoing and planned.
Expansion into other areas, including transfusion dependent sickle cell disease.
Lets EMEA myelodysplastic syndrome, and pulmonary diseases.
Now turning to allude deciding that.
Each one inhibitor being in valley evaluated in patients with relapsed or refractory acute myeloid leukemia or AML.
Following the presentation of the positive Registrational phase II results at the <unk> annual meeting last June new results from the trial were presented at the Ash annual meeting in December .
Patients were enrolled into <unk>, plus <unk> combination cohorts based on their disease status and prior therapy and.
While you're waiting for their best response with a primary endpoint of a composite complete remission plus complete remission with partial hematologic recovery or ACR CRH rate.
Okay.
The group of patients who had not yet received therapy for AML, where candidates and more candidates for <unk> as a single first line treatment.
I see our CR rate of 45%.
The other groups enroll relapsed refractory patients who have had prior hypothetical I think agent or.
<unk> inhibitor or candidates for any society.
CRH rates in these groups ranged from 30% to 47%.
We believe these findings support the potential of Alere decided that as the basis of combination therapy in patients with AML, who have not achieved a durable response from prior therapies.
With that I'm going to now turn the call over to Dave who will provide an update on the ft 705, one program for prostate cancer.
Thanks, Pat Im going to spend the next few minutes discussing our CVP P 300 inhibitor ft, $7 51, which is in development for the treatment of metastatic castrate resistant prostate cancer.
Because <unk> hundred modulates AAR via the N terminal domain, we believe ft, $7 51 may be able to address a broad range of resistance mechanisms that limited the utility of standard of care are signaling inhibitors.
In addition, tumors expressing the <unk> splice variant, which lacks the C terminal.
Oman binding domain.
For which there are no approved treatments as a potential therapeutic target, perhaps $7 51.
We began enrolling a phase one trial last year and up to 45 men with metastatic castrate resistant prostate cancer, who have failed at least one line of therapy and are typically heavily pre treated.
Receiving one or both of abrogate around and <unk> and in many instances also chemotherapy.
Trial utilizes an open label adaptive design, starting with a dose of 25 milligrams given on a three week one week off cycle.
And dose escalation.
Based on pre defined safety and Tolerability criteria.
Initial results in eight patients were presented at the Triple meeting in October showing an encouraging safety profile and biologic effects consistent with the inhibition of the <unk> 300 pathway.
Importantly, the 150 milligram dose achieved drug concentrations that approached the predictive efficacious dose based on modeling with preclinical data and.
In addition, skin biopsies demonstrated a reduction in histone acetylation, a marker of inhibition of this pathway.
All but one of the treatment emergent events were grade two or lower with no events leading to treatment discontinuation.
One patient in the highest 150 milligram dose cohort experienced a great III hyperglycemia, which was medically managed and the dose of ft 751 was reduced.
This patient was the first in trial and the trial, who has a valuable at 12 weeks for the standard surrogate marker of disease progression.
Prostate specific antigen level, which declined by greater than 50%.
And then greater than 80% at 16 weeks.
<unk> tumor, which is progressing prior to enrollment with classified as stable disease and confirmed by radiography.
We are encouraged by these initial results and dose escalation in the trial has continued since the October update we.
We look forward to providing updated trial results towards the middle of this year.
Actual results from the trial will inform our next steps with respect to potential combinations lines of therapy, and mutational status of patients with that 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 first spend a few minutes discussing our financial results for the quarter and year ended December 31, 2021, and then discuss our cash position and outlook.
Our net loss for the quarter and year ended December 31, 2021 was $50 1 million and 173.0 million, which compares to a net loss of $28 6 million and $70 4 million for the quarter and year ended December 31 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 $37.0 million and $125 7 million for the quarter and year ended December 31, 2021, compared to $24 9 million and $93 4 million for the quarter and year ended December 31 2020.
The increase was primarily attributable to costs of our clinical and preclinical development programs manufacturing activities, R&D staffing and equity based compensation.
General and administrative expenses were $13 2 million and $48 3 million for the quarter and year ended December 31, 2021, compared to $7 9 million and $30 8 million for the quarter and year ended December 31 2020.
The increase was primarily attributed to equity based compensation staffing costs and professional fees and insurance.
Our cash cash equivalents in marketable securities balance as of December 31, 2021 was $493 million.
Compared to $645 6 million at year end 2020.
Cash use reflects operating expenses and working capital requirements to support our operations.
For all we continue to be in a very strong financial position with funding through the third quarter of 2024.
I'd like to thank you and we can now take questions.
Okay.
If you'd like to ask a question. Please press Star then one.
Austin has been answered and you'd like to remove yourself from the queue press the pound key.
Our first question from Santiago <unk> with <unk>.
Your line is open.
Hey, Thanks for taking my question.
So I wanted to get more detail on rethink discussions with FDA on the potential regulatory path forward right. So we've discussed within this path on the potential approval.
Approval.
And it sounds like you would have to provide.
Some additional evidence.
That correlates to increasing hemoglobin with the relevant clinical endpoints I'm curious of what that could look like.
And perhaps just taking a step back.
What would be the difference in timeline containing a potential accelerated approval based on the hemoglobin response at 24 weeks then you have the final POC.
Endpoint at 52, plus a few months of.
Additional regulatory review.
So our estimate is about a nine to 12 month difference between the two options potentially but are we missing something there. Thank you.
Yes.
Hi, Thiago good morning, Thanks for the question.
So let me take a step back.
What's important to note here is that our hibiscus trial, which is who we are studying which is the phase III.
<unk> has always included the ability to pursue accelerated and also the traditional path and so as we've mentioned before we've been working very closely.
With the FDA.
Make sure that we can provide the data required and that specifically is.
Lincoln.
Being able to demonstrate that because this is a unique mechanism of action PK R. <unk>.
We're able to.
Correlate hemoglobin response to a clinically meaningful.
Endpoint.
So what we plan on doing what we are doing is collecting data mechanistic data epidemiologic data as well as our own clinical data, which much of which we shared at our ash presentation at the end of last year.
And so that's the overall plan and the key thing here is that we are generating this information because the PK or mechanism is and in fact are unique and different.
So that's an important point.
With respect to timelines I'd say broadly speaking we are in alignment with you that to the extent that we can pursue accelerated approval that would likely mean, an approval about a year earlier than the traditional path.
Got it perfect. Thank you so much.
Okay. Thanks.
Thanks for the questions.
Our next question comes from Alicia Yap with Cantor Fitzgerald. Your line is open.
Hey, guys. Thanks for taking my question and congrats on the progress so far I guess for me. One can you just talk a little bit maybe furthering on the Tiago question kind of from a commercial setting, let's say you are able to get approval without the hemoglobin. How do you think that you love, but do you think.
Wanted to <unk>. Nonetheless evidence you have may be able to bridge that and then just on the prostate cancer program.
Frame, a little bit more detail like what we should expect as far as like kind of.
Having data and how what are the gating factors for success there as you read out more data.
Thanks for the question.
First on the question of.
The approval what that might look like to the extent that we can get accelerated approval versus let's say a traditional path that will be a year later.
Certainly launching with hemoglobin only.
Isn't as optimal as launching with both hemoglobin and let's say other clinically meaningful endpoints like voc's.
That said, we'd be launching a year ahead of time, roughly speaking and so I think there's a trade off there and just depends on what we see in the data as we unwind the results at IAA too. So I think it really depends but to your point I think there are the tradeoffs of launching early with let's say not the complete data set versus launch.
A year later with a complete data set so it will all eventually depend on the data.
With respect to the prostate cancer program.
What do you expect roughly mid year is just an update to what we presented at the Triple meeting last year. So more patients followed for a longer period of time.
And I would expect that we wouldn't see the complete dataset until towards the end of this year early next year and we're aiming to recruit enroll 45 patients in total.
We continue to have.
Actually a very strong demand for the clinical trial.
And it's progressing nicely.
We are dose escalating and so we should have a reasonable update mid year.
Great. Thank you.
Our next question comes from Maury Raycroft with Jefferies. Your line is open.
Hi, good morning, and thanks for taking my questions ill start off with prostate cancer for $7 51.
Just wondering if you could talk a little bit more on your latest thinking on combo options and mutation status and how that's going to factor into the mid year update and then also just wanted to know if you can say if you've submitted an abstract to <unk> at this point.
So Dave why don't you, maybe you talked about 751.
Sure. Thanks Maury.
Relative to the combo options were clearly evaluating multiple possibilities in the setting of phase two.
And I think Theres, a case to be made for looking at combos early but also.
Continuing to evaluate as monotherapy, depending on the data that we see.
So we were looking at the broad range of options.
Regarding mutation status.
We have done in the past, we will will be fairly transparent about the data that we get and we are doing both genetic analysis.
They are.
As well as looking at <unk> expression variant and I'll remind you that our.
One patient who had a PSA response in the first.
Cohort.
We actually have an <unk> mutation associated with the <unk> resistance. So we will continue to.
Build that dataset.
<unk> reported as the data comes out.
And Mark to your question about submission of abstracts.
We typically don't comment on that and we'll comment on that once an abstract is accepted or presentation accepted so.
Right now, we're not commenting on any sort of submissions on any any chunks.
Okay. Okay. Thanks, and maybe just a quick question you probably can't say too much but just with the impact of Covid recently, just wondering if you can comment on enrollment status for Hibiscus and also for the phase two.
Transfusion dependent and sickle cell disease study than the one in beta thal.
Enrollment is going for that study as well if you can provide any more granularity.
Alright, Thanks for that question, it's an important one and as we said before I mean, we've been working very very closely with the sites.
Both here and abroad.
And.
What I can tell you is that we maintain our guidance.
As for Hibiscus to have the IAA, one interim analysis, one towards the end of this year.
And with respect to the.
<unk> and also the transfusion dependent trial, that's on track as well for early results at the end of this year so no change in guidance.
As I mentioned earlier, we have been working very very closely with the sites. The team is.
Really Ben.
Yes.
Making sure that we're trying to.
Help the sites as much as possible help to patients as much as possible. It is a difficult situation. We do see it I would say improving overtime and so fingers crossed that we continue to see that trend.
But right now I can again confirm the guidance that we provided before with respect to how we're progressing on both of those trials.
Got it okay. Thanks for taking my questions.
Our next question comes from Mark Breidenbach with Oppenheimer. Your line is open.
Hey, good morning, and thanks for taking the questions.
Just following up on $70 51, it sounds like we shouldn't be expecting a recommended phase two dose to be announced mid.
Mid year.
If I'm understanding correctly and also is it correct to assume we shouldnt expect.
Dose expansion cohorts to.
To be initiated later this year or is that more of a 2000 22023.
Joel.
Yes.
So we don't expect to make an RP, Judy midyear, but thats really an objective for the end of the year. The team is actively planning for.
Expansion cohort.
And we will announce that at the right time, but some of the early investments that one makes at risk.
Relative to drug supply and so the company's got planned timing for making those investments as the data mature. So we should have we expect no gap between the end of phase, one and a phase II dose expansion.
Okay fair enough.
Maybe one for Paul.
Can you just remind us.
Protocol allows for re sizing of the phase III component based on the magnitude of splitting into what Youre seeing in phase two is there any any chance for for altering the size.
Maybe to go after.
The boc endpoint with statistical significance.
Yes, thanks Mark.
But.
Protocol is.
<unk>.
It is fairly SaaS, but this can change based on conversations with the FDA if there's.
<unk> to.
To take learnings, we will do that but remember that this is blinded analysis, so it'll be difficult for us to do anything in the current design.
Other than.
So all of the data as it as it gets unblinded.
Yes.
Okay understood alright, thanks for taking my questions.
Sure. Thanks Mark.
As a reminder, cask a question. Please press Star then one.
Our next question comes from Andrew Burd with SBB. Your line is open.
Hi.
My questions are on the redeployment.
You guys are moving their portfolios when the regulatory process I was wondering if the plan is still to divest so how should we think about the ability to monetize it.
It looks like it's probably not a good apples to apples pulls from Servier.
At Antelope.
As a corollary, we announced the hiring of Chief patient officer is that really more for the sickle cell program or is this assignment you're considering going forward with the Ibs program alone.
Andy you are breaking up on me a little bit there, but I think I caught your questions.
So first on <unk>.
Our partnering strategy has not changed so we remain committed to the partner strategy with program the NDA.
So that's number one with respect to let's say comps relative to the Luna side Nib and the <unk> deal I think these are very different kinds of deals.
As you might recall the deal involving survey evolve not only Tim.
So over but it also involve abroad.
Ray of other pipeline molecules in addition to the infrastructure and people.
And so this is a very different kind of a deal.
That said I mean this is a differentiated molecule. If you look at the small scale, we believe that.
It does have the potential to be.
Best in class based on the duration of response that we've seen which is an impressive 18 months for the CR CRH responders. So.
Hopefully that answers your question about comps.
And finally with respect to the Chief patient Officer.
Let me just say that we're absolutely delighted.
Dr Hippie onboard soon.
She is a remarkable.
<unk> investigators leader and a champion for patients.
And for primary mandate is to make sure that we bring the patient voice into everything that we do here internally and also helped to champion access reimbursement.
Ending.
And care for patients broadly speaking.
So this includes sickle cell patients.
<unk> patients.
Other therapeutic areas that we're involved so hopefully that answers your question I'm really proud of the fact that.
In many ways, we are leading the way here with regard to a position like this reporting directly into the Ceo's office.
She has been onboard just a few short weeks and I'm already seeing the impact that cheesecake.
No that does answer my questions. Thanks.
Thanks, Andy.
There are no further questions at this time I'd like to turn the call back over to Frank Li for any closing remarks.
Well, we'd like to thank everyone for taking the time to participate in today's call.
As we outlined today 2021 was a year of significant progress and accomplishments our forma.
And in 2022, we have plans to do even more in pursuit of our purpose to transform the lives of people living with rare hematologic diseases and cancers.
In closing I'd like to thank our patients investigators and employees for their substantial contributions in 2021.
And so I'd like to say, thank you again and this concludes today's call have a great day.
This concludes the program you may now disconnect.
This concludes the program you may now disconnect.
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