Q2 2022 Forma Therapeutics Holdings Inc Earnings Call

August 5, 2022

The conference will begin shortly to raise your hand during Q&A you can dial star one one.

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Good morning, My name is Josh and I will be your conference operator today at this time I would like to welcome everyone to the former 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.

Good morning, and welcome performance second quarter, 2022 financial results and business update conference call.

Before we begin I encourage everyone to go to the news and investors section a former therapeutics dot 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 995.

These statements are neither a prediction nor guarantees of future events or performance and include those regarding the timing enrollment success in data announcements of our current ongoing clinical trial therapeutic and market potential hypotheses of mechanisms of action clinical.

Clinical benefits.

Product candidates expansion and development of clinical programs.

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 32022 that will be filed with the SEC today and in subsequent reports.

Our current reports on form 8-K.

On today's call I'm joined by our President and Chief Executive Officer frankly.

Tina <unk> head of research and development.

Dr. Cameron Turner Executive director clinical development Dr.

Dr. Dave Cooke, our Chief Scientific officer, and our CFO .

I will now turn the call over to Frank.

Thank you Adam good morning, everyone and thank you for joining us today.

Barbara aims to become a recognized leader in rare hematologic diseases and cancers and a trusted partner to the communities we serve.

We are advancing a robust pipeline led by type of payback.

We're well capitalized and last month, we're pleased to announce the appointment of two new members of our executive team.

Dr. Tina <unk> joins us as executive Vice President.

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 when they asked Betsy 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 am confident Latino and linear will help drive our next phase of growth until late stage clinical development and commercialization as we prepare to deliver meaningful medicines to patients for sure.

We also recently announced that Dr. Selwyn Vickers and member of our Board and Dr. Patrick Kelly, Our Chief Medical Officer at platform.

I'd like to congratulate Dr. Vickers on his new position as President and CEO of Memorial Sloan Kettering Cancer Center.

As part of this transition he is stepping down from public company boards.

Pat joined form of approximately seven years ago, and foremost primarily of research and early development company.

Pat's passion and expertise in translational medicine early clinical development.

We're grateful to Pat for his contributions to our programs and for establishing a world class Hematology development team comprised of six Mds, who have deep experience, leading centers that treat patients with sickle cell disease thalassemia and Mds.

We're now moving toward global late stage development, and ultimately commercialization <unk> million, our head of research and development has a lot of expertise in this area and we're very pleased to have with us.

That will continue to serve as a strategic advisor to pharma.

Tina will assume direct responsibility for research development and medical teams.

Pat has no immediate plans beyond spending time with his family and continuing to support pharma through our 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 will continue to maintain their patient centric focus and all that we do.

We now have in place an executive team with experienced leading organizations through global late stage development and commercial launch.

We brought onboard John Bishop our head of technical operations earlier. This year as you know, we recently brought onboard chino in linear 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 plan to drive our R&D organization.

Tim.

Thanks, Brad 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.

Is poised for growth and expansion at format is today.

Since joining last month.

I have been impressed by the level of scientific rigor across the organization.

Telling data generated to date across our clinical programs and the potential of the tablet P that.

To truly transform the treatment of sickle cell disease that was EMEA and lower risk Mds.

The quality of these program rests on the shoulders of strong leadership and deep scientific expertise.

I want to thank Pat for helping to build a world class hematology development team and for advancing the.

Program through the generation of robot clinically meaningful data the disposition format mobile route stage development and commercialization.

Looking forward my priorities are to drive operational excellence across the temple developed.

<unk> development program.

As we advance toward a potential approval in sickle cell disease, and data Readouts and phase II studies in expanding its therapeutic potential two additional populations <unk>.

Including transfused populations in sickle cell disease power ischemia and lower risk Mds.

In parallel I aim to selectively expand our preclinical portfolio recognizing.

Recognizing the particularly strong foundation that format has built in hematology and oncology.

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

Thanks Gino.

Earlier. This week, we were pleased to announce that we've entered into an exclusive license agreement with <unk> pharmaceuticals to develop manufacture and commercialize a low side of it.

Our mutant <unk> inhibitor for the potential treatment of mutant <unk> positive relapsed or refractory acute myeloid leukemia.

Based on the strength of the Registrational phase III dataset, we believe that <unk> has the potential to represent an impactful and differentiated therapy for relapsed refractory AML.

The FDA has accepted our NDA for <unk> with a <unk> action date of February 15th 2023.

Under the terms of the agreement formal receive an upfront payment of $2 million and is eligible to receive an additional $17 $5 million. Upon the achievement of certain near term regulatory approval and first commercial sale of milestones.

Total of up to an additional $215 5 million in connection with the achievement of certain development and commercial milestones and.

And potential tiered royalties in the low teens to mid Thirty's.

Given <unk> focus on hematologic diseases, and cancers, and the strength of the commercial infrastructure.

We are confident that they are the right partner to deliver <unk> to patients in need.

More broadly this agreement highlights our R&D capabilities.

<unk> will enable us to further sharpen our focus on advancing our pipeline, which we highlighted in detail at our inaugural R&D day in May.

This event included a presentation of encouraging analysis from our phase one study of a tablet P that in sickle cell disease.

Indicating a reduction in reported pain related adverse events, which we believe further underscores the potential of a tablet P that to reduce basal occlusive crises and ongoing phase II III Hibiscus study.

Enrollment and Hibiscus study is on track with interim analysis, one expected near the end of this year.

As a reminder, this analysis will enable dose selection for the phase III portion of the study.

Based on continuing discussions with the FDA, we continue to believe that accelerated approval remains a viable path.

With the clinical data package, we expect at the time of submission.

As we outlined at R&D day, we believe that a tap of Pvs multimodal mechanism of action has the potential to improve symptomatic anemia across multiple indications beyond sickle cell disease.

We continue to advance the phase III Gladiolus study of a tablet P VAT and transfusion dependent and non transfusion dependent thalassemia as well as sickle cell disease patients who are receiving chronic transfusions.

As a reminder, approximately 20% of the overall sickle cell disease patient population is transfusion dependent and represents an area of substantial unmet need.

We expect initial results from this study by the end of the year.

Also by the end of the year, we expect to further expand the tablet P that development program to include Phase two studies in pediatric sickle cell disease, and lower risk Myelodysplastic syndrome or Mds.

Similar to hemolytic anemia is chronic transfusions are there common practice in Mds treatment paradigm and can result in iron overload.

We look forward to evaluate the potential of a tablet P that to target the underlying pathophysiology of low risk Mds.

And reduce transfusion burden in this area of high unmet need.

Moving to our phase one study of Ft, 751 in metastatic castration resistant prostate cancer.

As discussed at R&D day, we plan to evaluate an alternative dosing schedule.

Less heavily pretreated patient population and are currently processing the protocol amendment.

Based on this protocol amendment, we now expect to share additional data from this study in the first half of next year.

At pharma 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 am pleased to report that there has been a high level of interest in the program we.

We've received a number of proposals from a mix of health care 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 cancers.

I'll now turn it over the call to Cameron Executive director and head of the tablet P that program to provide a brief update on a tablet P that Cameron.

Thank you Frank and good morning, everyone.

Let me begin by providing more detail on the data update from the phase one study of <unk> that we announced at R&D day.

The title is a once daily selective PK activator with a distinct multimodal mechanism of action designed to improve oxygen binding and delivery and to reduce hemoglobin S polymerization by decreasing $2 <unk>.

And to repair red cell membrane damage by increasing the ATP.

The now completed phase one program for <unk> was quite robust.

We studied the tableau <unk> added 90 healthy volunteers at up to 1000 milligrams in a single ascending dose study.

By multiple ascending dose studies.

We then evaluated the tableau <unk> added 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 <unk> once a day.

Data from this open label extension study demonstrated that <unk> is well tolerated.

<unk> and a significant and sustained increase in hemoglobin and significant reductions in particular sites bilirubin, LDH and systemic markers of sickle cell pathophysiology.

Importantly in addition to these multiple improvements in measures of sickle cell biology patients also experience a 68% reduction in <unk> compared to prior history.

For patients with sickle cell disease.

Basal occlusive crises represent only the tip of the iceberg as it relates to paid events.

To better understand all pain events in our patients we analyzed our open label extension data and found that administration of <unk> <unk>.

<unk> and a time dependent reduction in total paid amounts, 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 basal occlusive crises and other payment apps provide further support for the potential of the type of payback to provide benefits to patients with sickle cell disease.

Including reduction the basal occlusive crises in the ongoing phase II three hibiscus study.

Further these data and a well tolerated safety profile provide a strong rationale for the broader a title P that development program in thalassemia in lower risk Mds.

Let me now turn the call over to Dave for an update on FTE 70, 51, and our research pipeline.

Thanks Cameron.

As we described at our recent R&D day.

Pharmacokinetics target engagement biomarker data from the ongoing phase one study of Ft, $7 51, our CVP P 300 inhibitor in phase one for metastatic castrate castration resistant prostate cancer demonstrate the current dose levels delivered drug exposure and the predicted efficacious.

This range.

Based on the PK PD and safety profiles.

Going forward, we plan to evaluate an alternative dosing schedule and a less heavily pretreated 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 <unk>.

<unk> transformative combination therapies for sickle cell disease.

Validating novel mechanisms that may be complementary to a top of vivat.

Second.

By unlocking the therapeutic potential of Red blood cell health in areas beyond traditional traditional haemoglobinopathy and anemia.

And third by developing.

Molecules in our pipeline for targeted oncology with an emphasis on synthetic modality.

With that I will now turn the call over to Todd.

Thank you, Dave and thank you everyone for joining us today.

I'll spend the first few minutes discussing our financial results for the quarter ended June 32022, and then discuss our cash position and outlook.

Our net loss for the quarter ended June 32022 was $52 6 million, which compares with a net loss of $43 6 million for the quarter ended June 32021.

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 32022, compared to $31 6 million for the quarter ended June 32021.

This increase was primarily attributable to the increase in research and development staff to support our advancement of <unk> and other programs, including the conduct of our phase two three hibiscus trial in sickle cell disease patients in the phase III trial of the tablet P that palace EMEA.

General and administrative expenses were $13 9 million for the quarter ended June 32022, compared to $12 5 million for the quarter ended June 32021.

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 32022, with $395 9 million compared to 493 million as of December 31, 2021.

Cash use reflects operating expenses and working capital requirements to support our operations.

Overall, we continue to be in a strong financial position with funding through the third quarter of 2024.

We will now open the call for questions.

Thank you as a reminder to ask a question you will need to press star one on your telephone please standby, while we compile the Q&A roster.

Our first question comes from Mark Breidenbach with Oppenheimer You May proceed.

Hey, good morning, guys and thanks for taking our question.

Congrats on finding a home for elite tightened.

I guess I'll start by asking if you'd consider selling rights to the royalty stream from Rigel similar to what we saw as you do with I D.

Or kind of hold on to the royalty stream for the foreseeable future.

Then second question for me is.

With respect to next steps for $70 51.

I'm wondering if we can assume the next clinical update in the <unk>.

First half of next year.

Crude patients enrolled after the protocol amendment.

And.

If you expect the overall size of the phase one trial.

Are they more or less the same despite the planned amendment. Thanks, Thanks for taking my questions.

Hey, Thanks, Marc for the question.

Thank you.

First off.

Liberty deal for Luna side Nib.

And let me just say that we're really pleased with the deal and it's consistent with our commitment to patients.

They're a fantastic partner they have got a strong presence in hematology oncology and a good commercial infrastructure.

Hi, Todd this is going to be a meaningful asset for rigel.

On our end, there's some near term milestones as we've talked about.

The data is coming up in February . So these are near term miles.

And overall, what I'm really happy about is that this is I think it will.

R&D capabilities to bring a molecule from discovery all the way through now potentially an approval.

So that said with respect to the royalty milestones ahead, we haven't made any decisions about.

That going forward.

But we will certainly consider it but no decisions.

And then secondly, with respect to 751.

I'll, let <unk>.

Dave comment further on this but.

It'll take us a little while to process.

The amendment to the protocol.

And then.

I would suspect that it.

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.

Sure Mark Thanks for the question just to remind everybody.

In the patients enrolled so far on these where it really advanced patients over half of them had experienced chemotherapy.

And the median number of prior lines of therapy with three so we know this is a very advanced population. So a key thing about this amendment.

Go into an earlier population and exclude those who've had to chemotherapy. In addition, we're exploring other regimens other schedules.

<unk> seen good target engagement predicted pharmacologically effective dose.

Dose levels until we feel like we've got the biomarker to guide that that future.

Under under the under the Amendment and I'll leave it at that thank you.

Alright terrific. Thanks for filling our questions and congrats on the progress. Thanks Bart.

Thank you one moment for questions.

Our next.

Comes from Maury Raycroft with Jefferies. You May proceed.

Hi, good morning, Thanks for taking my questions and congrats on the progress for the quarter.

For a viscous.

The first interim analyses just wanted to check in on the latest youre, 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.

Okay.

<unk>.

Back up a little bit overall, we're tracking well to plan.

And so what we expect again as that.

We'll be in a position.

To have one done sometime end of the year.

Okay.

I'd like to turn it over to Cameron to ask him to speak about this but it's largely done.

Our selection.

In terms of the number of patients that we're expecting so kamran, let me turn to David.

Yes, Thanks, Brian and thanks for the question.

Yes, I think we share the enthusiasm.

For what's to come from Hibiscus.

Just to level set the.

The minimum bar for data.

To make decisions at IAA, one is with 60 patients again, reaching 12 weeks of therapy.

So the bar, which will consider either one as Frank just said were on track and excited about that around the end of the year.

And the other the readout from that will be dose selection at this blinded study as recommended by our DSP.

Sorry.

Go ahead go ahead Mark.

Yes, that's helpful. And then the other question I had was.

Or hibiscus for segmenting severe versus moderate baseline biopsies.

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.

Similarly, we're not going to provide any additional data until the interim.

Cameron can speak to how we're generally thinking about strategy, but just trying to find the patients based on vlccs. So okay alright.

Yes. Thanks.

So the stratification is in the analysis, not and not and randomization. So I can't make any predictions about how many patients will fall where.

But we will analyze based on who had fewer boc isn't more abuses.

Got it okay. Thanks for taking my questions.

Thanks Laurie.

Thank you and as a reminder to ask a question you will need to press star one one on your telephone.

Our next question comes from Andrew Berens with SBB you May proceed.

Hey, how are you this is Chris on for Andrew.

Just two questions for so for the first one for the partnership that you guys have for Olympic.

In terms of the cost.

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.

Thanks for the question Chris.

First with respect to <unk> Ben.

Todd here to provide some additional color, but overall, what's really important here is not not only that we found.

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 tableau feedback as you know going forward.

Important step for the company.

And we're really pleased with weight.

It all transpired, so Pat or I'm, sorry, Todd you can speak to a little bit more color here, yes, sure hi, Chris good to connect with you. So just in terms of.

Ongoing responsibilities, it's largely tied around completion of the support required to get it through the final reviews with the FDA and approval.

That includes <unk>.

Pleading.

Ongoing study, which is nearly final such in sort of the ramp up stages as well as some validation batches in manufacturing, which have been initiated and just need to stop 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 by the partner.

Rachel so fairly minimal amount of ongoing expenditures and internal effort beyond getting it over the finish line.

And let's.

Let's say, Chris you had a second question remind me the second question.

Yes sure. So the second question was really around.

Whats sort of commentary has given you more confidence about an accelerated approval pathway for <unk>.

Sure.

First let me just say that.

We've been having ongoing dialogue with the FDA has 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.

The table at the time.

Accelerated approval submission and simple.

We not only have like biscuits, but all of the other programs that we've talked about in our portfolio. So that's an important concept.

And secondly, as we discussed at the R&D day, we think that.

But an important study to move forward with would be <unk> to look at stroke risk. So those things combined we think along with all of our discussions I think point to.

Our ability we think number one the accelerated approval door is still open and secondly.

At the time of submission.

But one the accelerated approval door is still open and secondly that at the time of submission.

We anticipate having the data we need yes, we will just have to see what the data look like.

Determination as to whether we go for accelerated approval or not.

And finally, let me just say that as we go.

Talked about before Hibiscus provides for not only an accelerated approval.

Pathway potentially but also the traditional approval by <unk>.

Boc.

So hopefully that helps.

Got it thanks.

One group.

Weight gain some more clarity if possible.

Discussions with the FDA.

We have kind of led to this.

Idea that I saw an approval is still available is that is that given your commentary around that.

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

Elevated approval path remains open.

Got it thank you very much.

Thanks, Chris.

Thank you one moment for questions.

And as a reminder to ask a question you will need to press star one on your telephone.

Our next question comes from <unk> Agarwal with Cantor you May proceed.

Hi, good morning, and thanks for taking my questions Congrats on executing the Adobe suite.

So maybe first question for the phase two hibiscus trout.

The interim analysis, one when do you disclose any <unk> data.

Is there anything that it may not be mature 12 weeks could start giving directional signal and it's probably the most important endpoint for us right now.

And second question on the lower risk Mds trial, Scott I think you mentioned, Scott I will start by end of the year. So.

So just curious as to what are the steps that you are working through that it's taking some time are you waiting for the interim analysis, one and sickle cell.

Before making a decision on the lower risk Mds trial, and the dose to be tested there.

Our 400 milligram dose for MBS.

If the trial gets initiated ended the year and what do you still expect data for the risk Mds by mid 2023.

Do you.

Yeah. So thanks <unk> good morning.

First on the question.

So now I'm 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.

Plans for Hibiscus interim analysis one.

At present.

Secondly, with respect to the low risk Mds.

Ill ask Cameron here to comment broadly, but we are tracking overall to providing some initial data.

As we've got it to middle of next year. So camera, maybe you can speak to where we are with getting.

Getting the study up and running.

Yeah, absolutely. Thank you.

One comment on.

On where we'll be at one real quick for Hibiscus just as a reminder, 12 weeks of data is essentially exactly what we had in our phase one study, which we've talked broadly about and we've shown the trends both in POC in total pain.

In addition to being blinded so we won't have the data to show you what I wanted.

I think we pretty well know what that signal looks like early.

There are more interested in what it is going to look like with more data.

As a as in regards to Mds.

No we're not waiting on dose selection for Hibiscus, It's an independent study.

And.

Actually it just happens to overlap.

Time.

And what we're doing right now is what you would normally do inside startup.

The selection of sites contracting.

Budgeting that approvals, we're making good progress.

I think we will be getting started in Q4, such that we'll have some early data to talk about a bit next year. Thank you.

Thank you.

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

Thank you well first of all let me thank our participants for logging on this morning.

I, especially want to thank our patients investigators.

So their contributions for a very strong second quarter performance.

And we look forward to having further discussions.

The future. So this concludes our call and thank you for logging on.

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

The conference will begin shortly to raise Johan during Q&A you can dial one one.

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Good morning, and welcome performance second quarter, 2022 financial results and business update conference call.

Before we begin I encourage everyone to go to the news and investors section a form of therapeutic dot 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 thickness 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 995.

These statements are neither a prediction nor guarantees of future events or performance and include those regarding the timing enrollment success in data announcements of our current ongoing clinical trials therapeutic and market potential hypotheses of mechanisms of action clinical.

Clinical benefits of our product candidates expansion and development of clinical programs and our financial condition and capital requirements.

And our current reports on form 8-K.

On today's call I am joined by our President and Chief Executive Officer frankly, Dr.

Dr. Tina <unk> head of research and development Dr.

Dr. Cameron Turner Executive director clinical development Dr.

Dr. Dave Cooke, our Chief Scientific officer, and our CFO Patrick.

I will now turn the call over to Frank.

Thank you Adam good morning, everyone and thank you for joining us today.

<unk> aims to become a recognized leader in rare hematologic diseases and cancers and a trusted partner to the communities we serve.

We are advancing a robust pipeline led by tablet P that.

Our well capitalized and last month, we're pleased to announce the appointment of two new members of our executive team.

Dr. Tina <unk> 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 when asked Betsy 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 am confident Latino and linear will help drive our next phase of growth into late stage clinical development and commercialization as we prepare to deliver meaningful medicines to patients for sure.

We also recently announced that Dr. Selwyn Vickers a member of our board and Dr. Patrick Kelly, Our Chief Medical Officer at last form.

I'd like to congratulate Dr. Vickers on his new position as President and CEO of Memorial Sloan Kettering Cancer Center as.

As part of this transition is stepping down from public company boards.

Pat joined pharma, approximately seven years ago, and foremost primarily of research and early development company.

<unk> passion and expertise in translational medicine early clinical development.

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 <unk>, our head of research and development has a lot of expertise in this area and we're very pleased to have with us.

Pat will continue to serve as a strategic advisor to pharma and P&L, assuming direct responsibilities for research development and medical teams.

That has no immediate plans beyond spending time with his family and continuing to support pharma through our 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 will continue to maintain their patient centric focus and all that we do.

We now have in place an executive team with experienced leading organizations through global late stage development and commercial launch.

We brought onboard John Bishop our head of technical operations earlier. This year as you know, we recently brought onboard chino in linear 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 plan to drive our R&D organization Paul Tim.

Tim.

Thanks, Brad.

Related 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 is poised for growth and expansion at format is today.

Since joining last month I've been impressed by the level of scientific rigor across the organization.

The compelling data generated to date across our clinical programs and the potential of the tablet P that to truly transform the treatment of sickle cell disease, Alex EMEA and lower risk Mds.

The quality of these program rests on the shoulders of a strong leadership and deep scientific expertise.

I want to thank Pat for helping to build a world class hematology development team and for advancing the.

Program through the generation of robust clinically meaningful data that disposition format mobile route stage development and commercialization.

Looking forward my priorities are to drive operational excellence across the temple developed.

<unk> development program.

As we advance toward a potential approval in sickle cell disease, and data Readouts and phase II studies to expand its therapeutic potential for additional populations.

<unk> transfused populations in sickle cell disease, now ischemia and lower risk Mds.

In parallel I aim to selectively expand our preclinical portfolio.

Recognizing particularly strong foundation that format has built.

Rajiv handle oncology.

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

Thanks Dino.

Earlier. This week, we were pleased to announce that we've entered into an exclusive license agreement with <unk> pharmaceuticals to develop manufacture and commercialize Lotus sizes.

Our mutant <unk> inhibitor for the potential treatment of mutant <unk> positive relapsed or refractory acute myeloid leukemia.

Based on the strength of the Registrational phase III dataset, we believe that <unk> has the potential to represent an impactful and differentiated therapy for relapsed refractory AML.

The FDA has accepted our NDA for <unk> for the <unk> action date of February 15th 2023.

Under the terms of the agreement formal receive an upfront payment of $2 million and is eligible to receive an additional $17 5 million.

Upon the achievement of certain near term regulatory approval and first commercial sale of milestones.

A total of up to an additional $215 $5 million in connection with the achievement of certain development and commercial milestones and.

And potential tiered royalties in the low teens to mid Thirty's.

Given right gels focus on hematologic diseases, and cancers, and the strength of the commercial infrastructure.

We are confident that they are the right partner to deliver <unk> to patients in need.

More broadly this agreement highlights our R&D capabilities.

And will enable us to further sharpen our focus on advancing our pipeline, which we highlighted in detail at our inaugural R&D day in May.

This event included a presentation of encouraging analysis from our phase one study of a tablet P that in sickle cell disease.

Indicating a reduction in reported pain related adverse events, which we believe further underscores the potential of the tablet P that to reduce basal occlusive crises.

Ongoing phase II III Hibiscus study.

Enrollment and Hibiscus study is on track with interim analysis, one expected near the end of this year.

As a reminder, this analysis will enable dose selection for the phase III portion of the study.

Based on continuing discussions with the FDA, we continue to believe that accelerated approval remains a viable path.

With the clinical data package, we expect at the time of submission.

As we outlined at R&D day, we believe that a tap of Pvs multimodal mechanism of action has the potential to improve symptomatic anemia across multiple indications beyond sickle cell disease.

As a reminder, approximately 20% of the overall sickle cell disease patient population is transfusion dependent and.

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 tablet P that development program to include Phase II studies in pediatric sickle cell disease, and lower risk Myelodysplastic syndrome or Mds.

Similar to hemolytic anemia of chronic transfusions other common practice in NPS treatment paradigm and can result in iron overload.

We look forward to evaluate the potential of the tablet P that to target the underlying pathophysiology of low risk Mds and reduce transfusion burden in this area of high unmet need.

Moving to our phase one study of Ft, $7 51 in metastatic castration resistant prostate cancer.

As discussed at R&D day, we plan to evaluate an alternative dosing schedule.

Less heavily pretreated patient population and are currently processing the protocol amendment.

Based on this protocol amendment, we now expect to share additional data from this study in the first half of next year.

At pharma 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 am 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 health care 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.

I'll now turn it over the call to camera executive director and head of the tablet P that program to provide a brief update on a tablet P that Cameron.

Thank you Frank and good morning, everyone.

Let me begin by providing more detail on the data update from the phase one study of <unk> that we announced at R&D day.

The title is a once daily selective PK activator with a distinct multimodal mechanism of action designed to improve oxygen binding and delivery and to reduce hemoglobin polymerization by a decrease in <unk>.

And to repair red cell membrane damage by increasing the ATP.

The now completed phase one program for <unk> was quite robust.

We started to tableau added 90 healthy volunteers at up to 1000 milligrams in a single ascending dose study.

Claude by multiple ascending dose studies.

Data from this open label extension study demonstrated that <unk> is well tolerated.

<unk> and a significant and sustained increase in hemoglobin and significant reductions in particular sites bilirubin, LDH and systemic markers of sickle cell pathophysiology.

Importantly in addition to these multiple improvements in measures of sickle cell biology patients also experience a 68% reduction in <unk> compared to prior history.

For patients with sickle cell disease phase.

Basal occlusive crises represent only the tip of the iceberg as it relates to paid events.

To better understand all pain events in our patients we analyzed our open label extension data and found that administration of the title <unk> resulted in a time dependent reduction in total paid amounts, 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 basal occlusive crises and other paint events provide further support for the potential of the type of payback to provide benefits to patients with sickle cell disease.

Including reduction of <unk> occlusive crises in the ongoing phase III three Hibiscus study.

Further these data and a well tolerated safety profile provide a strong rationale for the broader <unk> development program in thalassemia in lower risk Mds.

Let me now turn the call over to Dave for an update on FTE 70, 51, and our research pipeline.

Thanks Cameron.

As we described at our recent R&D day.

Pharmacokinetic and target engagement biomarker data from the ongoing phase one study of ft, $7 51, our CVP P 300 inhibitor in phase one for metastatic castrate castration resistant prostate cancer demonstrate the current dose levels delivered drug exposure and the predicted efficacious.

This range.

Based on the PK PD and safety profiles.

Going forward, we plan to evaluate an alternative dosing schedule and a less heavily pretreated 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.

Validating novel mechanisms that may be complementary to a top of vivat.

Second.

By unlocking the therapeutic potential of Red blood cell health in areas beyond traditional traditional haemoglobinopathy 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.

Thank you, Dave and thank you everyone for joining us today I'll spend the first few minutes discussing our financial results for the quarter ended June 32022, and then discuss our cash position and outlook.

Our net loss for the quarter ended June 32022 was $52 6 million, which compares with a net loss of $43 6 million for the quarter ended June 32021.

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 32022, compared to $31 6 million for the quarter ended June 32021.

General and administrative expenses were $13 9 million for the quarter ended June 32022, compared to $12 5 million for the quarter ended June 32021.

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 32022, with $395 9 million compared to $493 million as of December 31, 2021.

Cash use reflects operating expenses and working capital requirements to support our operations.

Overall, we continue to be in a strong financial position with funding through the third quarter of 2024.

We will now open the call for questions.

Thank you as a reminder to ask a question you will need to press star one on your telephone please standby, while we compile the Q&A roster.

Our first question comes from Mark Breidenbach with Oppenheimer You May proceed.

Hey, good morning, guys and thanks for taking our question.

Congrats on finding a home for aluminum siding.

I guess I'll start by asking if you would consider selling rights to the royalty stream from Rigel similar to what we saw.

With I D.

Or kind of hold on to the royalty stream for the foreseeable future.

And then second question for me as well.

With respect to next steps for $7 51.

I'm wondering if we can assume the next clinical update in the first half of next year will include patients enrolled after the protocol Amendment.

And.

If you expect the overall size of the phase one trial to stay more or less the same despite the planned amendment. Thanks, Thanks for taking my questions.

Hey, Thanks, Marc for the question, it's Frank here.

First off.

Liberty deal for Luna side Nib.

And let me just say that we're really pleased with the deal and it's consistent with our commitment to patients.

And they're a fantastic partner they have got a strong presence in hematology oncology and a good commercial infrastructure.

Brian This is going to be a meaningful asset for rigel and on our end, there's some near term milestones as we've talked about.

Paducah date is coming up in February . So these are near term miles.

And overall, what I'm really happy about is that this is.

<unk>.

R&D capabilities to bring the molecule from discovery all the way through now potentially an approval.

So that said with respect to the royalty milestones ahead, we haven't made any decisions.

That going forward.

But we will certainly consider it but no decisions.

Secondly.

Secondly, with respect to 751.

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.

Sure Mark Thanks for the question just to remind everybody.

And the patients enrolled so far on these really advanced patients over half of them had experienced chemotherapy.

And the median number of prior lines of therapy with three so we know this is a very advanced population. So a key thing about this amendment.

Go into an earlier population and exclude those who have had to chemotherapy. In addition, we're exploring other regimens other schedules.

Seeing good target engagement predicted pharmacologically effective dose.

Dose levels and so we feel like we've got the biomarker the guide that that future.

Under under the under the Amendment and I'll leave it at that thank you.

Alright terrific. Thanks for filling our questions and congrats on the progress. Thanks Bart.

Thank you one moment for questions.

Our next question comes from Maury Raycroft with Jefferies. You May proceed.

Hi, good morning, Thanks for taking my questions and congrats on the progress for the quarter.

For <unk> for.

So the first interim analysis, just wanted to check in on the latest youre, saying on expectations in terms of number of patients and the extent of data you plan to report and in addition to dose selection and what would be viewed as a positive update in this first interim.

Okay.

Just.

Back up a little bit overall, we're tracking well to plan.

And so what we expect again as that.

We'll be in a position to.

<unk> done some time into the year.

Okay.

I'd like to turn it over to Cameron to ask him to speak about this but it's largely dose selection.

In terms of number of patients that we're expecting so kamran, let me turn it over to Steve.

Yes, Thanks, Brian and thanks for the question.

Yes, I think we share the enthusiasm.

For what's to come from Hibiscus.

Just to level set.

The minimum bar for data.

To make decisions with I E. One is with 60 patients again, reaching 12 weeks of therapy.

So that's the Barra, which will consider I won as Frank just said were on track and excited about that around the end of the year.

And the other the readout from that will be dose selection. This blinded study as recommended by our <unk>.

Alright.

Go ahead go ahead Mark.

Yes, that's helpful and then one day.

Q2 2022 Forma Therapeutics Holdings Inc Earnings Call

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