Syros Pharmaceuticals Inc. Q1 2023 Earnings Call

The recording is 17.

And 53 seconds.

[music].

Okay.

Those pharmaceuticals first quarter 2023 financial results conference call.

All participants are in a listen only mode.

This call is being webcast live.

Investors and media section of cirrhosis website at Www Dot cereals dotcom.

Please be advised that today's call is being recorded.

At this time I would like to turn the call over to Karen <unk>.

Director of Investor Relations and corporate communications at <unk>.

Thank you. This morning, we issued a press release announcing our first quarter 2023 financial results and business update.

The release is available on the Investor and media section of <unk> website at Www.

Dot com.

We will begin the call with prepared remarks by Doctor Nancy Simonian, Our Chief Executive Officer, Dr. David Roth, Our Chief Medical Officer.

Jason Hart, our Chief Financial Officer, We will then open the call for questions Christian Stephens, Our Chief Development Officer, Dr. Eric Olson, our Chief Scientific Officer, and Kyle <unk>, Our Chief Commercial officer are also on the call and will be available for Q&A.

Before we begin I would like to remind everyone that the statements. We make on this conference call will include forward looking statements.

Actual events or results could differ materially from those expressed or implied by any forward looking statements. As a result of various risks uncertainties and other factors, including those set forth in the risk factors section of our annual report on Form 10-K, and our quarterly report on Form 10-Q that we.

Filed this morning, and any other filings that we may make with the SEC in the future any forward looking statements represent our views only as of today and should not be relied upon as representing our views as of any subsequent date, we specifically disclaim any obligation to update or revise.

Any forward looking statements.

I would now like to turn the call over to Nancy.

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

2023 is off to a strong start as we focus on clinical execution.

And move closer to our goal of delivering new standards of care for the frontline treatment of hematologic malignancies.

Today, we will be sharing progress with you across our programs in Mds AML.

<unk> and <unk>.

Oh.

We are excited about our continued progress on enrollment in our select Mds, one trial and we remain on track to complete enrollment of the 190 patients in the fourth quarter of this year and to announce pivotal complete response data from select Mds one trial in the third quarter of 2024.

We recently amended the protocol for the select Mds one trial to include overall survival as a key secondary endpoint.

The primary endpoint of the trial remains complete response and 190 patients.

By including overall survival as a key secondary endpoint, we have the potential to convert an accelerated approval based on CR two.

To a full approval based on OS if warranted using an efficient one trial design.

Importantly, this amendment does not impact the timing of our pivotal CR data from select Mds one.

David will provide additional details on the amended protocol shortly.

Turning to our efforts at AML.

Showing promising data from the safety lead in we initiated the randomized portion of the phase II trial last quarter.

We also remain on track to provide an update on the dose confirmation study up 'twenty 101 in APL and what's the next steps on our registration pathway in the second half of this year.

Taken together these upcoming milestones laid the groundwork for multiple catalysts over the upcoming 12 to 18 months.

And which we will progress and turnover cards on each of our lead programs and move closer to our ultimate goal of delivering effective well tolerated and convenient therapies to targeted patient populations in need of better options.

We believe Mds.

AML and APL each represent significant market opportunities in the frontline setting, which we are well positioned to address through our own commercial efforts in the United States.

With that I'd like to turn the call over to David to provide a more detailed update on our ongoing clinical programs.

David.

Thank you Nancy.

Today I will focus on the amended protocol for the select Mds, one phase III trial, which is evaluating the combination of <unk> and Asa sided team.

Julie diagnosed higher risk Mds patients with RARA over expression, which we believe represents approximately 50% of patients with higher risk Mds.

As a reminder, this study is a double blind placebo controlled trial evaluating <unk>, plus decitabine versus placebo plus <unk>.

Hi.

As Nancy mentioned, we recently amended the study protocol to include overall survival as a key secondary endpoint, while continuing to use complete response as the primary endpoint for potential approval.

Recent FDA feedback continues to support our use of complete response rate as an appropriate primary efficacy endpoint for either full or accelerated approval with supporting data on durability of complete response.

And CR rate will remain the primary endpoint in our study.

That said, if <unk> received accelerated approval.

Additionally, overall survival as a key secondary endpoint in the screening study could allow select MBS one to also confirm clinical benefit to support full approval in the future.

Potentially avoiding the need for separate confirmatory study.

For use of a single randomized controlled trial to support potential accelerated approval and subsequent conversion to full approval. If needed is also consistent with recently issued draft FDA guidance on the clinical shortened considerations to support accelerated approval of oncology therapeutics.

This one trial approach offers many advantages over a separate confirmatory trial.

Wowing US to include the 190 patients enrolled to support the primary C are important to also support the confirmatory endpoint.

This ensures consistency of the patient population.

Fort the primary and secondary analyses.

Theyre all enrolled in the same protocol at the same sites by the same investigators and with identical eligibility criteria.

It also provides the FDA assurances that we are well underway with the delivery of our confirmatory data to provide them with increased confidence when evaluating our primary endpoint data for regulatory decision making.

In addition to this being a more efficient way to confirm clinical benefit a one trial design has additional advantages will potentially speeding the delivery of confirmatory data and limiting trial related costs and expenses.

Under the amended protocol select Mds, one will enroll a total of 550 newly diagnosed higher risk Mds patients, including the initial 190 patients supporting the primary endpoint to power the study for the key secondary endpoint.

Importantly, and as Nancy mentioned, we continue to expect that we will complete enrollment of the initial 190 patients necessary to support approval either accelerated or.

Using a CR and police and the fourth quarter of this year and then report pivotal <unk> data in the third quarter of 2024.

As we discussed last quarter in January 2023, the FDA granted fast track designation to <unk> for the treatment of higher risk Mds.

This designation provides us with several advantages, including priority review timelines and further underscores the critical need for new treatment options for this patient population.

A group that has been underserved by available therapies.

Again, the existing standard of care Hyperventilating agents offer only a 17% complete response rate and a median survival of $18 six months normally therapies beyond each of these have been approved since 2006.

Turning to our study of <unk> in AML.

Previously announced that we had initiated the randomized portion of the study in newly diagnosed unfit AML patients with water over expression.

Like higher risk Mds AML represents a significant unmet medical need.

Of the patients diagnosed with unfit AML approximately a third do not respond to upfront treatment with <unk> and.

And a majority of those with initial responses ultimately relapse.

These patients have a very poor prognosis with median overall survival rates of only two four months.

As we announced late last year.

Safety lead in portion of select one.

A triplet combination of Tammy by our team and the venues demonstrated an 83% CR cri rate with a rapid onset of action and no evidence of increased toxicity relative to historical data we have been.

Rates of myeloid suppression.

These data underscore the potential of <unk> and <unk>.

Combination with existing standard of care to deliver improved outcomes to be approximately 30% of AML patients who are positive for <unk> overexpression.

As a reminder, the randomized portion of the trial is designed to evaluate the safety and efficacy of <unk>.

In combination with venues.

Compared to venues and approximately 80 newly diagnosed unfit AML patients with RARA overexpression randomized one to one with.

With a composite CR rate or the CRC or high rate as the primary endpoint.

We continue to remain on track to report initial data in the fourth quarter of this year.

Additional data expected in 2024.

Lastly, I'll turn to 'twenty 101 hour.

Our novel oral form of course, we are trying oxide ore.

<unk>, which is currently being evaluated in an ongoing dose confirmation study for the frontline treatment of acute myeloid leukemia.

The current standard treatment regimen with intravenous Ato creates a significant burden for patients involving up to 140 treatment infusions each over two to four hours for nearly a year.

We believe <unk> 101 has the potential to offer a reduced treatment burden by providing patients an all oral regimen and as you said.

Active while also increasing access and reducing health care costs and utilization.

We look forward to providing an update on the dose confirmation study in the second half of this year to describe the planned development path and timing for further evaluation of <unk> 101 in a registration enabling study.

Study and PPL.

With that I would like to turn the call over to Jason to review, our first quarter financial results.

Jason.

Thank you David now turning to our first quarter financial results, we recognized $3 million in revenue in the first quarter of 2023, consisting entirely of revenue recognized under our collaboration with Pfizer.

<unk> recognized $5 5 million of revenue in the first quarter of 2022, consisting of $5 $1 million in revenue recognized under our collaboration with Pfizer and $400000 under our collaboration with insight.

R&D expenses were $28 8 million in the first quarter of 2023 as compared to $25 2 million for the first quarter of 2022.

This increase was primarily due to the advancement of the Companys late stage clinical programs.

Based on our current operating plans, we expect that our future R&D expenses relating to our drug discovery stage programs will continue to be reimbursed by our collaboration partners.

G&A expenses were $7 4 million in the first quarter of 2023 as compared to $6 $9 million for the first quarter of 2022.

The increase relates to supporting the advancement of our late stage clinical programs.

We reported a net loss for the first quarter of $23 8 million or <unk> 85 per share compared to a net loss of $25 1 million or $3 99 per share for the same period in 2022.

Cash cash equivalents in marketable securities as of March 31, 2023, or $166 million as compared with $202 million on December 31 2022.

We believe our current cash position will be sufficient to fund our anticipated operating expenses and capital expenditure requirements into 2025, which is beyond phase III data from the select Mds, one trial and initial data from the randomized portion of the select AML one trials.

With that I will turn the call over to the operator for questions.

Thank you ladies and gentlemen, we will now begin the question and answer session should you have a question. Please press star followed by the one on your Touchtone phone you will hear a suite on prompt acknowledging your request question.

Questions will be taken in the order received.

Should you wish to cancel your request. Please press the star followed by the tail.

The first question is from Phil Nadeau from TD Cowen. Please ask your question.

Good morning, Thanks for taking my questions and congrats on the continued progress first on the addition of the OS endpoint to select Mds.

I'm curious if you'd be willing to disclose the powering on the OS endpoint. It seems like there's quite a few people on that that you're going to enroll. Additionally in the trial. So it seems like it's likely to be well powered.

Curious if you've disclosed exactly what the partner will be.

Hey, Bill Thanks for the question David will answer that yes. Thanks Bill.

So just to remind everyone. The study remains.

Ah study focused on our primary endpoint based on the complete response rate and as we've previously shared that power assumption.

As over 90% powered to demonstrate a difference in the CR rates between the two arms with respect to the key secondary endpoint the powering assumptions are 80%.

And 550 patients.

Provides enough power to detect a difference in median overall survival between the two arms.

And then in terms of the increment.

Is the assumption for the control arm the $18 six months that you referenced in the prepared remarks.

And if so what is the assumption for the year.

The testing on.

Yes so.

So we haven't specifically stated what the actual.

Statistical assumptions are but.

You are correct that.

Those are the tried and true.

Data for <unk> as a single agent in high risk Mds you are in the high teens, So I think thats a reasonable assumption.

And then.

Are there interim analysis, along the way.

So we haven't stated that there are interim analyses, but obviously the.

The initial analysis for the primary endpoint is CR.

It could be interpreted as an interim analysis, along the way to the <unk>.

<unk> analysis when the study is completed and the secondary key secondary is reported out.

Subsequently.

Great and then last question on this.

I appreciate that it's really early.

Any estimates on when the overall survival data could be available.

So yes, we haven't.

<unk> guided to win when that data would be available as you know survival is an event driven endpoint. So.

That's certainly something that we're going to have to.

Rejected in the future.

But right now we're focused on delivering the key data, which is the pivotal data upon which we anticipate the drug.

We hopefully will be approved and thats, the CR rate and just to remind everybody we remain on track for <unk>.

Fully enrolling the 190 patients needed.

For the CR primary analysis by the end of this year and we expect to have that data available in the third quarter of 2024.

So these are the things about which were really greatly excited.

Obviously, the key secondary endpoint based on survival will come after that.

Great and then last question from US on 'twenty 101 have you had preliminary FDA discussions about.

Recent FDA discussions about the trial redesign.

And.

If not when will.

Would you expect to hold those discussions.

So let me I'll answer the question by saying that we've been guiding and we continue to say that in the second half of this year, we will provide an update on the.

The.

Those confirmation study and at that point, we will also share.

Information about our plans for.

Moving into the Registrational trial, the phase III trial for <unk>.

Which the approval will be sought and I think it would be best to reserve all of the details around that and things around regulatory interactions et cetera for that update.

Perfect. Thanks for taking my questions.

Thanks Bill.

Yes.

Thank you. The next one is from Ted any Hoff from Piper Sandler. Please ask your question.

Great. Thank you.

Bill ahead is leading to this morning.

So only thing really left to ask about is.

Select AML and with respect to the phase two data homeland is what expectations are and what would really define it going thanks.

Thanks Ted.

David to address that right so far the select AML one study.

Again, just to make sure everyone's up to speed, we reported at the end of last year.

Great progress on this study we.

Sure the data coming from the safety lead in.

Demonstrated tolerability of the drug we would not see increased toxicity, when adding Jamie Baird team onto the backbone of <unk>. So we're really excited about that and that enabled us to move into the randomized portion of the trial that we initiated in the first quarter. We also were pleased to see an 83% CR Cri.

Reminding everyone that is the.

Primary endpoint for that study so it's randomized trial.

It's currently enrolling two arms, the triplet versus a doublet Tommy <unk> versus <unk>.

And we expect to have an update on that.

In the fourth quarter of this year.

Thank you.

Our next question is from Jason Butler from JMP Securities. Please ask your question.

Hi, Thanks for taking my question guys I guess I just have a follow up on the AML study.

Can you give us a sense of the number of patients and maturity of data will get later this year and I guess ultimately the question is do you think you can.

Come to some kind of go no go decision based on this first cut of data or will it be based on ultimately the final data set.

Yes so.

It's a follow up as well on the prior question as well on this one.

We haven't specifically guided to the numbers of patients that would be.

Part of our update at the end of the year and also in terms of the data maturity. So we reported initiating the randomized portion in the first quarter. So that can give you a sense as to the potential for how mature the data might be.

At the end of this year.

And then specifically the go no go criteria again, we havent.

Specifically guided to.

What the bar would be for triggering the next phase of investment.

Moving into a.

Registrational trial. This is again a randomized phase II.

And it will be the very first prospectively collected data on the performance of Zen plus Asia in RARA positive patients compared to our triplet. So again, we're very excited about it but we'll have to.

See the totality of the data in order to really answer that question.

More specifically.

Great. Thank you.

Thanks, Jason.

Yeah.

Thank you once again, ladies and gentlemen, as a reminder, should you have a question. Please press star followed by the one.

The next question is from Mark Breidenbach from Oppenheimer. Please ask your question.

Hey, good morning, guys. Thanks for taking our questions just a couple from me.

First in the prepared remarks, I think I heard that there is still a possibility.

<unk> could be approved in Mds.

Fully approved based on the CR endpoint alone.

Or it could be an accelerated approval I was hoping maybe you could just expand on what would be the gating factors that would influence.

Our full versus accelerated approval on that endpoint and then second question I noticed that you had a couple of poster presentations.

At <unk>.

We're coming up at <unk>, four or 500 609.

Just wondering are these going to include new clinical data or trial still being enrolled around.

Testing testing this.

Drug maybe just give us a quick status update and what's your expected ESCO front from six to nine thank you.

Great Mark I'm going to have David take the first question and I'll take the 56 to nine months.

Sure so.

The FDA has.

Really reiterated.

Even more recently to us that the complete response rate as an appropriate primary efficacy endpoint that could support either the full approval or the accelerated approval, obviously in the context of the duration of the remission.

And as always with regulatory evaluation, the totality of the data.

We will also inform their decision making so.

The specifics around what is going to sway the pendulum in favor of a full approval versus an accelerated approval.

<unk> remains.

Depending on what our what our data package.

<unk> stem to give devaluation, but it's pretty clear that <unk>.

<unk> is an excellent surrogate for.

There's multiple studies that are correlated.

We had overall survival.

And so I think that it's really going to depend on the totality of our data and it really just to remind everyone that the.

Acknowledgements at.

The potential for an accelerated approval exists has always meant that there might be a need for a confirmatory trial. If we got the accelerated approval. So we've really known all along that one.

One might need to do that and encouraged now by really the great progress we've been making in the enrollment the expansion of our trial to a global footprint that includes 13 countries and over 100 actively enrolling sites.

Coupled with the.

The increasing awareness that FDA really likes to understand that a sponsor is committed to delivering the confirmatory data.

As they make their decisions. It just provides them with reassurance when theyre thinking about how to award the approval.

So we thought that it was.

Very sensible to take advantage of.

The ongoing progress in <unk> one study.

And use that to potentially fulfill both objectives.

It was needed of course, if it's just a full approval.

It wasn't required but certainly it's an important thing to do and we will continue to do that anyway.

Hey, Mark and then on the $56 nine a question that you have yes, we do have to add.

Abstracts are posters at <unk> on $56 nine just as a reminder.

We are.

Actively looking for a partner for that program.

We are presenting these data because we think they're very supportive of this being a best in class CDK <unk> inhibitor.

Where we have a great dose and schedule and we see clinical activity and very difficult to treat patient populations and I think these two presentations one on our experience.

Breast cancer with <unk> and the other kind of focusing more on our data and <unk> in combination I think will be.

Yes, it really will be compelling to show the potential for this agent, but as you know we are.

Ourselves, putting further capital into the development of this program and then start looking for a partnership.

Great. Thank you.

Thank you there are no further questions at this time I will now hand, the call back to Nancy for closing remarks.

Thank you operator, and thank you everyone for joining us today and for your continued support of Cerus. Please reach out to us with any further questions and have a great day.

Thank you ladies and gentlemen, the conference has now ended thank you all for joining you may all disconnect.