Q4 2022 Syndax Pharmaceuticals Inc Earnings Call
Okay.
Good day, everyone and welcome to this index fourth quarter 2022 conference call.
Today's call is being recorded at this time I would like to turn the call over to Sharon <unk> head of investment relations that's indexed pharmaceuticals.
Thank you operator, welcome and thank you all for joining us today for <unk>.
This fourth quarter and full year 2022 financial and operating results I'm Sharon.
And with me this afternoon to provide an update on the company's progress.
No.
Michael Metzger, Chief Executive Officer, Dr. Briggs, Morrison, President and head of R&D and he Goldman.
Goldman Chief Financial Officer also joining us on the call today for our question and answer session. Our Doctor Arrogantly, Chief Scientific Officer, Becky Angelique Engel Chief business Officer.
I was accompanied by a slide deck that has been posted on the investor page of the company's website.
Now turning to our forward looking statements on slide two before we begin I'd like to remind you that any statements made during this call that are not.
Historical are considered to be forward looking statements within the meaning of the private Securities Litigation Reform Act with 1995 actual results may differ materially from those indicated by these.
As a result of various important factors.
As discussed in the risk factors section in the company's most recent annual report on Form 10-K, as well as other reports filed with the SEC.
Any forward looking statement made represent our views as of today February 28 2023 only.
A replay of this call will be available on the company's website.
<unk> dot com on its completion.
With that I'm pleased to turn the call over to Michael Metzger, Chief Executive Officer.
Thank you Sharon and thank you to all of you joining on the webcast today.
Now turning to slide three 2022, it was a year of growth and strong execution pursuing that.
Good great progress working towards our long term goals, all of which put us firmly on the path to becoming a fully integrated commercial stage biopharmaceutical company delivering two best in class products to patients in areas of high unmet medical need.
On revenue minute, we made significant progress enrolling patients in the augment 101 pivotal trials announced breakthrough therapy designation for patients with relapsed refractory <unk> <unk>.
<unk> raised acute leukemia and presented robust updated phase one data that further supported recommended best in class clinical profile.
Oral sessions at the 2022 American Society of Hematologic Hematology annual meeting.
We initiated additional phase one trials designed to test recommended as a variety of settings, including combinations with standard of care regimen.
Correct. The tiller, Matt we completed enrollment in the pivotal Agave 201 trial published the initial phase <unk> chronic graft versus host disease dataset.
The journal of clinical oncology and made progress expanding the exits on that development program in collaboration with our partner insight.
We are fortunate to have insight is a strong collaborator to help us successfully advanced acetone app developers.
Additionally in December we bolstered our balance sheet with a $172 $5 million follow on offering our cash runway is now expected to extend into the second half of 2025, allowing us to appropriately invest to maximize the value pipeline prepare for potential U S commercial launches in 2000.
Four and pursue potential business development opportunities.
Our focus determination and.
Ending commitment to patients has propelled us forward to this critical year a year that we expect will be marked by pivotal readouts and registration filings for our two lead drug candidates both of which are first and potentially best in class treatments.
'twenty three is an incredibly significant year versus index and I am confident that we have the expertise and resources to execute on our goals along with the determination to realize a future in which people with cancer longer and better than ever before.
Turning to slide four we provide a high level summary of our current corporate priorities.
Starting with revenue, our highly selective <unk> inhibitor or.
Our pivotal phase III augment 101 trial evaluating <unk> in patients with relapsed refractory NPM, one four Kmt <unk> rearranged acute leukemia is progressing well.
We are happy to report that we have completed enrollment of the <unk>.
Of a sufficient number of patients with <unk> rearrangement to support a registration filing and we are on track to report topline data from the trial in the third quarter of 2023.
This data will provide the basis for an NDA filing by year end 2023, I will provide more details on augment 101 later in the call.
In addition to the augment 101 trial, we have ongoing trials testing recommended in earlier lines of therapy and in combination with standard of care medicine to treat these forms of acute leukemia. We are committed to unlocking the full potential of this important therapy.
With that goal in mind, we expect to have several additional data readouts in 2023 for every minute. These include initial topline data from augment 100 to chemo combination trial in relapsed refractory acute leukemia patients safety data from the dose escalation phase of the beat AML cooperative trial of revenue management.
In combination with <unk>.
In newly diagnosed AML patients and initial topline data from the first solid tumor proof of concept trial in metastatic colorectal cancer.
Moving to <unk>, our antibody against CSF, one or.
We remain on track to report topline data in mid 2023 from the pivotal phase III agave to a one trial evaluating <unk> in patients with chronic graft versus host disease, or gvhd and plan to submit a BLA filing by the end of 2023.
To maximize the value of the <unk> program, we anticipate initiate initiating additional trials in 2023, including a combination of <unk> and <unk> in frontline <unk> HD that we expect insight to begin later this year as well as the phase II trial of <unk> in idiopathic pulmonary fibrosis.
Or Ips that we anticipate kicking off in the first half of 2023.
We continue to evaluate potential opportunities to in license earlier stage targeted oncology compounds that we believe could become high value differentiated assets. We believe that we have sufficient capital to bring in new early stage assets, but our bar for doing so is quite high as any compound would need to complement our current pipeline.
And fit in with our long term corporate strategy.
Let's now turn to slide five and I'll provide further details on the <unk> program.
As you recall the phase II portion of augment 101 is designed as three single arm pivotal trials with distinct patient populations that enroll independently. These patient populations are <unk> rearranged ALLL A&P to re raise AML and NPM one.
The primary endpoint of each pivotal trial as a percentage of patients achieving CR CRH with secondary endpoints, including durability of CRC Ara H response transfusion independence overall survival and safety.
In December we received breakthrough therapy designation or <unk> for Revlimid for the treatment of all adult and pediatric patients with relapsed or refractory <unk> rearranged acute leukemia, whether presenting as acute myeloid or employed phenotype.
Our BCD underscores the potential if approved for revenue managed to be the first drug to address the rapid unmet medical needs.
<unk> R leukemia accounting for up to 10% of all acute leukemias.
Based on the broad breakthrough therapy designation and our ongoing conversations with regulators, we will pull the data generated from both the AML and <unk>.
Cohorts into a single NDA filing aimed at an indication to treat all relapsed refractory acute leukemia patients with <unk> rearrangement.
Thanks to the enthusiastic support of our investigators we have already enrolled a sufficient number of patients with <unk> rearrangement in the phase II portion of the augment 101 trial to enable enabled this filing strategy.
We expect to share top line data from this population in the third quarter and filing the NDA for the treatment of relapsed refractory <unk> acute leukemia in adults and pediatric patients by the end of 2023.
Separately, we continue to enroll relapsed refractory NPM, one mutant AML patients and expect to report completion of enrollment for these patients in the second half of 2023.
These trials are enrolling well and there was significant excitement for <unk> by investigators at the Ash annual meeting in December where we presented updated phase one data that Bruce will take you through on slide six Briggs.
Alright, thanks, so much Michael.
So as many of you know at the Ash annual meeting in December of last year. Dr. <unk> from the MD Anderson Cancer Center presented updated results from displays one portion of the augment 101 trial and two oral presentations.
Of the 60 patients evaluable for efficacy in the phase one portion 30% attained CR cri.
And notably in patients treated at the recommended phase II dose CR CRH was the same in both <unk> and <unk> subsets at 27%.
The median duration of CR CRH response was impressive in this very advanced patient population at nine one months.
Hi, MRV negativity rate of 78% among patients achieving a CR CRH, which is very meaningful in this patient population and it enables patients to proceed to potentially curative bone marrow transplant.
We also reported compelling data on 12 patients who achieved a response.
On revenue met of treatment and then went on to receive a stem cell transplant.
And Additionally, three transplant patients who are treated with revenue added maintenance therapy and the capacity you setting following stem cell transplant or non mild latest stem cell boost two of whom remained in remission for over a year as of the data cutoff.
As a reminder, in the pivotal trial patients can be restarted on revenue met of after bone marrow transplant design features that provide the data on the potential role of <unk> in the post transplant maintenance setting.
Revenue made up continues to be well tolerated and there were no discontinuation due to treatment related adverse events.
As you look across the development landscape. We believe the data continue to support revenue minutes compelling clinical profile and position. It has not only first in class best in class treatments.
I'll now turn it back over to Michael.
Great. Thanks Briggs.
Turning to slide seven given the compelling clinical and safety profile, we have observed with every minute our clinical development plan seeks to expand its use beyond relapsed or refractory setting into earlier setting and in combination with approved therapies here, we highlight our ongoing trials that revenue minutes across the full treatment landscape.
Starting with the phase one beat AML umbrella trial.
As part of our collaboration with the leukemia and lymphoma Society revenue <unk> is being combined with <unk> and they decided it needed to treat newly diagnosed AML patients with an MTM, one mutation or KFC to a rearrangement who are unfit for induction chemotherapy.
<unk> is the first man an inhibitor to be included in the trial, which will assess safety as well as initial efficacy enrollment is ongoing and we expect initial safety data from this trial to be available in 2023.
Longer term, we expect that positive AML trial results could lead to a phase three trial, which could serve as the basis for our future regulatory filings we.
We are also currently enrolling patients in the augment 102 trial designed to assess <unk> in combination with standard salvage chemotherapy for patients with relapsed or refractory <unk> mutant <unk> rearranged acute leukemia. We also expect to have initial data available from this trial in 2023.
And finally, the intercept trial. This trial was recently initiated as part of the intercept Master clinical trial led by the Australia and Asian.
Cana in lymphoma group. The trial. This trial is a creative approach to treating patients early in their disease course.
Is focused on investigating novel therapies that target early relapse and clonal evolution as preemptive therapy in AML.
It is designed to explore the activity of <unk>. The first <unk> inhibitor to be included as monotherapy in patients with AML, who have <unk> positive disease. Following initial treatment a group of patients at very high risk of relapse.
We're also in the process of planning a trial in combination with standard of care intensive chemotherapy used to treat patients in the frontline setting we plan to initiate that study in the second half of this year.
As is detailed on slide eight unlocking the full potential of <unk> beyond the relapsed refractory acute leukemia setting by moving it earlier in treatment and in combination with other approved agents has the potential to add meaningful value both for our shareholders and even more importantly for patients in need.
With these expansion opportunities, we see the potential to address upwards of 12000, NPM, one and Kmt <unk> acute leukemia patients across various settings. These two forms of acute leukemia together represent up to 40% of the overall AML population, which to our knowledge will be the largest subpopulation of AML can be addressed.
By new targeted therapies. There are currently no FDA approved therapies targeting <unk> or NPM, one acute leukemia.
We anticipate that revenue minutes could become the backbone of choice for patients with <unk> and NPM one in acute leukemia and were also looking to explore revenue met him as a treatment in solid tumors based on the compelling preclinical science supporting the role of the Menin <unk> interaction and data continue driven tumors.
Proof of concept signal seeking phase one clinical trial in colorectal cancer is open for enrollment and we expect to report topline data by year end 2023.
But let me now turn to <unk>, our potentially best in class monoclonal antibody therapy targeting the CSF one receptor that has the potential to serve as an effective practice changing intervention in this underserved population.
Slide nine provides an overview of the pivotal agave 201 dose ranging trial evaluating <unk> in patients with gvhd.
While has completed enrollment of 240 patients whose disease has progressed after two prior therapies.
We are at least two years of age and at net.
Overall entry criteria.
Patients were randomized to one of three treatment groups each investigating.
Zinc dose of telematics given everyone given either every once or every two weeks or once every four weeks.
The primary endpoint is overall response rate using the 2014 NIH consensus criteria for <unk>, while secondary endpoints include duration of response and validated quality of life assessments using the modified lease since its scale. We continue to expect to report topline data from the Gaba a 201 trial in mid 2020.
Great.
First the terms of our collaboration agreement insight will be leading the regulatory filings and see Gvhd, which we expect will occur in the later part of 2023.
Slide 10.
Details of the results from the phase one two trial, which showed <unk> ability to provide meaningful clinical responses in chronic gvhd patients that had been refractory to multiple prior therapies. This data was presented at the 2021 Ash annual meeting and subsequently published in the journal of clinical oncology in December These data.
Were well received amongst thought leaders who recognize <unk>.
Allomap is having a clinical profile that would benefit will be a benefit in the treatment of these heavily pre treated patients.
The overall response rate in the trial was 68% and the median time on treatment was over six months.
Overall response rate in the phase II portion with 82% in the median time to response was rapid at four weeks importantly, clinical responses were accompanied by a reduction in the sea gvhd symptom burden and improvements and all see gvhd involved organ systems. Additionally, more than half of the responders reduce their use of sterne.
Alright.
<unk> demonstrated a favorable safety profile in this refractory population with the most common adverse events consistent with on target effects of G. CSF.
CSF one hour in addition.
Slide 11 highlights the broad clinical and commercial opportunity for <unk>.
Gvhd represents a high unmet medical need and an important commercial opportunity with approximately 14000 patients suffering from Cte ph D. In the U S. Today.
And insights to leave the data generated to date with active <unk> suggested has the potential to play an important role in the treatment of CGP HD, both as monotherapy and given the safety profile in combination with complementary medicine.
Thus in collaboration with insight we are enthusiastic about expanding the exits on that program into frontline see gvhd in combination with rux.
And we expect a phase one combination trial in patients with newly diagnosed gvhd begin later in 2023.
The successful commercial launches of insights Jakafi and <unk> reservoir rock are encouraging both posted meaningful early revenues that begin to speak to the commercial opportunity in CGP HD.
<unk> has a differentiated mechanism of action from these products and could potentially offer a unique set of benefits to patients once approved.
Beyond CVA C. Gvhd, we are excited about the opportunity to expand access to allomap into fibrotic diseases, where the monocyte macrophage.
Lineage plays a key role we expect to initiate a robust phase II trial in IPF in the first half of 2023.
If successful we believe this trial along with one additional phase III trial could form the basis for FDA approval for the treatment of <unk>, yes.
Through combinations in the frontline setting as well as the opportunities to expand to ex U S markets, we envisioned the <unk> opportunity growing meaningfully.
I will now turn the call over to Keith to review our financial results.
Thank you Michael let me take a few minutes to discuss our financial results for the fourth quarter and full year 2022.
Turning to slide 12.
The results of our operations for the fourth quarter of 2022, and the comparison to the prior year's quarter are included in our press release, So I will not repeat them in these remarks.
Additional financial details are available in our fourth quarter and full year report, which was filed earlier this afternoon on Form 10-K.
I'd like to point out that our net loss for the fourth quarter was $39 2 million or.
Or <unk> 62 per share.
<unk> to a net gain of $96 $2 million.
Or $1 81 per share for the same period last year.
This difference is primarily attributed to the recognition of the upfront payment from insight related to the exit till about collaborations which we entered into in December of 2021.
For the full year of 2022, our net loss was $149 3 million or.
Or $2 46 per share.
Compared to a net gain of $24 9 million or <unk> 48 per share for the same period last year.
The difference is largely due to the upfront payment in December 2021 from insight.
We ended the fourth quarter with $481 $3 million in cash equivalents in marketable securities.
Including the net proceeds of $162 million.
From our follow on offering completed in December of 2022.
And $69 3 million shares and pre funded warrants outstanding.
Our current cash is expected to provide runway into the second half of 2025.
And covers our aggressive development and pre commercialization plans.
For both the <unk> and Thats until that programs as well as provides us flexibility should we decide to selectively engages early stage business development.
Looking ahead I'd like to provide financial guidance for the first quarter and full year 2023.
For the first quarter of 2023, we expect research and development expenses.
To be $30 million to $35 million and total operating expenses to be $40 million to $45 million.
For the full year of 2023.
Company expects research and development expenses.
To be between $160 million to $175 million.
In total operating expenses to be 225.
To $240 million.
Including approximately $30 million of noncash stock compensation expense.
With that let me now turn the call back over to Michael.
Thank you Keith.
And as you have heard during the call 2022 was an exceptional year of growth and execution.
It represents only the beginning of Whats index aims to accomplish in the near future.
With two pivotal trial Readouts and potential registration filings in 2023 for our two lead drug candidates both of which are first and potentially best in class treatments index.
The index is at the beginning of a significant transition into a fully integrated biopharmaceutical company, serving multiple patient populations of high unmet medical need.
In addition, both programs offer the potential for broad franchise opportunities beyond their initial registration indications, adding this index is long term growth potential.
<unk> ambitious goals and milestones that I've set out for 2023, and I am confident that we have the right plan and team in place to execute on them.
Further we are in a strong financial position with a balance sheet that allows us to deliver on key near term milestones.
As always I want to express our deep appreciation to this index team collaborators and most importantly, the patients trial sites and investigators involved with our clinical programs.
As all of you who help us to execute on our mission of realizing a future, which people with cancer live longer and better than ever before.
And I'd also like to thank our committed long term investors, who continue to share our and our vision and support us in buildings index.
And with that I'll turn it back over to the operator.
And open the call for questions. Thank you.
At this time, if you would like to ask a question. Please press the star and one on your Touchtone phone you may remove yourself from the queue by pressing star and two.
Once again to ask a question. Please press the Star then one.
And we'll take our first question.
From Madhu Kumar with Goldman Sachs. Your line is open.
Hi, This is omar.
Yes.
First are our first question is does the <unk>.
Now consider <unk> rearranged AML.
Finally purposes, and then second what are you looking to see from the two different combo studies of revenue.
Alright. Thank you for the question, Yes, I think the answer the first question, which is does the FDA as far as I heard the FDA consider the.
One cohort or the filing purposes for KFC and the answer is yes, we will be combining.
Both AML and ALLL cohorts pool.
Pulling the patients and filing them together.
Yes.
That's the plan.
And the second question I'm, sorry, you cut out in the second half of it so I couldnt hear it could you repeat that please.
Sure. So the second question what are you looking to see from two different combo studies that revenue.
Okay.
Once again it cut out I think you said, where we're looking to see from this from the two studies is that correct.
From the two combination studies for revenue.
Gotcha. So the combination studies, we have multiple combination studies ongoing.
Augment 102, which is the chemo combo.
We'll have data on we expect later in the year.
And we have.
Frontline trials going with then Asia with beat AML.
And we will be designing other trials in addition to that in the frontline setting.
I think we haven't necessarily set out guidance on what we're looking to we're looking to achieve I think these are early studies, where we're going to be looking at first of course safety and early efficacy and trying to establish a recommended phase II dose and I think that would be that would be the main objective of those trials, which will open.
Hopefully achieve by the end of this year.
Great sounds good thanks for taking my questions.
Yes.
Our next question comes from Phil Nadeau with Cowen Cowen and company. Your line is open.
Good afternoon, congrats on the progress and thanks for taking our questions first a follow up on the Kmt to a population.
So for the readout from the 101 trial.
It sounds like both AML and <unk> patients will be will.
We will be co mingled.
I guess now it's a combined cohort.
Well.
Well, the <unk> annualized regardless of myeloid versus not folks citic.
Algae or will there be any stratification for the.
The underlying disease.
Yes, thanks for the question Phil.
And maybe I'll, let Briggs answer that question.
Yeah, So Phil there.
You may recall that when we announced the breakthrough therapy designation and come back to.
Hi, My first question.
It did except that came to <unk>.
We arrange leukemia is one disease, whether it presents as AML, our ALLL adults or kids.
And part of the.
Support for that was that the data we presented was a combination of AML and ALLL adults and peds. So we did break it out and we will break it out for the agency to further support.
That designation.
But the analysis.
We'll be looking at that integrated population altogether.
Got it okay and in terms of the patient numbers necessary I know you've said that you've.
You've already recruited them.
Because the number of patients equivalent to what we thought you were going to roll in cohort two and to be in the study or because theyre not being combining now being combined.
It required number of patients actually equivalent to just.
Really one one cohort of patients.
Yes, Bill Thanks for the follow up I think we're going to what we we haven't really guided and we're not planning to guide on that.
Specific number of patients in.
And the combined cohorts I think what we're what we've said is that with the sufficient number based on.
Both BCD and agency feedback we feel that we are aligned on.
Our strategy to have a sufficient number of patients that will constitute a filing.
By the end of the year, we'll have that data of course in the third quarter, but we're at this point, we're not guiding specifically to how many patients are in the pooled analysis.
Got it Okay and then.
Last question on revenue before one question on <unk>.
NPM one pivotal data.
Sorry, the enrollment completion in the second half of the year.
It seems to suggest that a first half 'twenty for maybe mid 'twenty four.
Based on your prior guidance for the <unk> population is that a fair assessment or is there. Some reason why there could be a longer interval between enrollment completion and data and the NPM one patients.
Thanks, Phil I think the same rules apply I think we say, we followed patients for six months.
We're not being specific as to exactly when in the second half we will have the NPM one enrolled so.
I'm going to be a little bit.
<unk> not project out when we when we will have top line data yet in 2024.
But I think we.
<unk>.
Great and then last question from Us on X tone that would you be willing to provide any additional details on the IPF phase two now.
Well, we have to wait until the trials underway.
Yes. Thanks for thanks for the question. So we were excited to obviously get that trial started and we'll probably share more details on the trial design.
Yes.
We're underway I think thats.
I'll say for now.
Okay, great. Thank you.
Thanks, Phil.
Our next.
<unk> comes from <unk> Rama with Jpmorgan. Your line is open.
Hi, This is priyanka on for Annabel Rama just a quick question from us.
Between the two Kmt a cohort could you provide any color or clarity on enrollment breakdown.
Yes. Thank you for the question as I said previously and we're not going to.
Specific guidance on how many patients were contributed from the either of the two cohorts, but we're guiding to is that we have a sufficient number that we can complete the analysis and we will provide that topline data in the in the third quarter.
So I think it will have to be satisfied with that for now I think thats the.
Interesting.
Thank you.
Two questions.
Our next question comes from Yigal <unk> with Citi. Your line is open.
Yeah.
Hi, Jamie this is Oscar.
You can work on for Yigal, Thanks for taking my questions.
On the NPM one do you have clarity on the number of empty on NPM, one patients you need to enroll.
To hit your timeline guidance have you spoken with the FDA.
NPM one.
Yes. Thanks, Yeah. Thanks for the question.
As previously disclosed 64 patients NPM one patient.
As we had laid out the three cohorts 64 patients.
The number that we will look to enroll.
And we have of course spoken with the agency about not only in one became each way.
Had a fair amount of interaction with them over the several months since we started this program.
And we've aligned on our not only our design, but I'll have a statistics.
Go into India.
Each of the cohorts so.
Yes, nothing really new there it's still it's still the same it's still the same number of patients that we had guided to.
Okay are you able to provide any clarity on where you are in terms of fitting.
For patient number.
Only that yeah. Thanks for the follow up only that.
We've guided to the second half of the year, sometimes in second half of the year, we'll be able team, we announced that we've enrolled 64 and.
Youll be able to figure out from there exactly when or.
Whereabouts, we are with.
With presenting top line data.
Okay got it and maybe one more on ox until them up for the frontline combo trial with <unk>, which you are studying later this quarter.
Should we be thinking about maybe the combinability of <unk>.
Maybe in terms of.
The potential for overlapping toxicities, maybe such as pneumonia or liver enzymes for example.
Is there any color you can share on compatibility. Thanks.
Yes. Thanks, Thanks for the question I'll start maybe I'll have.
Briggs address the overlapping toxicity just to point out I think the the trial. This is insights combination trial there'll be there'll be kicking off and I think the guidance. Later this year, we didn't specify first quarter in particular, but I think it's.
Trial, we're all looking forward to and maybe Briggs.
You can address the overlapping tox, yes look I think that was part of a keen interest of insight and wanted to partner with US next tell them that because at least if you look at the mono therapy.
Toxicities of each agent pays looks like today with combined quite well, but that of course is that is the purpose of the event.
Initial phase of the combinations as to characterize that.
Going into that trial will be quite optimistic that we can get full doses of both agents.
Okay got it thanks for taking my questions.
Thank you.
Our next question comes from Brad <unk> with Stifel. Your line is open.
Good afternoon, and thanks for taking the questions. This is Brad.
Doesn't sound like I can get more out of you on augment 101, so maybe I'll ask about the status of the phase one <unk> pharm work youre doing around the multiple doses of revenue for the <unk> inhibitor strength should we expect the data disclosure from that this year.
Brad Thanks for the question.
As you I think pointed out in your question. These are supportive.
These are supportive trials or this particular trial is supportive.
Piece of our filing potential filing for <unk>.
And that work is ongoing.
I don't think we guided to.
We would necessarily disclose data from that trial.
Related to certain pieces of this there may be there will be data that comes out in the.
And the ultimate the ultimate filing when we disclose data, but I don't think theres any particular timeline related to when we might disc.
Disclose.
That particular study.
But thanks for the question.
Okay, and then maybe one on on the safety data for the beat AML, because it's exciting to get.
Get some combo data this year I guess to set expectations for that can you discuss the revenue dose being used there is it all of the 163 on a strong sip or is there any dose escalation of revenue taking part in that combo and then for the vent as a component.
What is the schedule for Ven does it require continuous dosing or physicians use the shortened schedule thats often used in clinical practice now thank you.
Yes, Brad very good question, and maybe I'll, let Briggs address both of those yeah. So.
Starting is the.
The standard venues are labeled regimen.
Obviously there are.
On protocol specified.
Dose interruptions if needed but.
The request was to start with it.
Labeled regimen.
And then there is a dose escalation component to the <unk>.
But everybody is on a strong inhibitor.
Great. Thank you.
Thank you Brad.
Our next question comes from Peter Lawson with Barclays. Your line is open.
Great. Thanks for taking the questions.
Just around the NPM one just any.
Any update around how enrollment is proceeding.
And then I've got a question on exiting them up.
Thanks, Peter Thanks for the question <unk>.
<unk> enrollment is going well.
You can see <unk> is going well as well so I think they are both with great enthusiasm.
Certainly ash I would say that investigators.
Really engaged with us and have.
It's really related to the data that they saw at the meeting and so MTN when enrollment is going well.
So there is I think that relates to our <unk>.
<unk> ability to complete enrollment in the second half of this year.
And then you had a follow up.
Yes, just around actually until about just with the Sanofi is reservoir rock just with that launch kind of how do you look at that.
Gage your own potential launches.
How should we think about peak revenues.
Whether you think this is ultimately a single agent or a combination agent regimen.
Thanks for the questions Peter.
So yes look I think you heard in my remarks, <unk> has done well first year sales.
Or is that two to two.
$250 million in it.
Impressive.
This is our drug has a different mechanism of action and we think impressive results thus far in clinical trials.
And I think the opportunity here in chronic graft versus host disease is to access.
Patients in the relapsed refractory setting I think these patients tend to over there.
Of course of their treatment journey, they tend to get multiple therapies they cycle through the different therapies and so.
Our opportunity. We think is is what reservoir rock and ore.
Jakob <unk> experienced in the third line and the ability to pick up meaningful share.
Ultimately this and that the mono therapy, but ultimately.
The opportunity here extends itself well beyond just monotherapy into potential combinations reservoir rock.
Is.
It has its own opportunity for combination potentially but the mechanism of action of our drug is non overlapping with jackup fee and so thats. The Briggs pointed out that's the.
Reason why I think we're so excited about being able to combine it in the frontline setting and potentially reduce the use of steroids, which could really expand the use of this drug beyond what.
What reservoir has even shown in relapsed refractory first year.
Think about that.
A little harder.
First year sales of two to $2 50, that's showing to be a potentially very large opportunity and again thats adjusted relapsed refractory third line plus.
Disease, and so the opportunity to go in combination and get the patients earlier in their treatment journey, I think really pretends well for who are our drug.
And I think.
Or is there something else to your question Peter.
That was great. Thank you so much.
Thank you.
And our next question comes from <unk> Patel with B Riley Securities. Your line is open.
Yeah, Hey, good afternoon, thanks for taking the questions.
You mentioned that the enrollment for the NPM, one cohort is going well. So I'm just curious about the timing differences versus the <unk>.
Cohort is that is that just a function of when these trials started enrolling.
Just trying to understand the gap there.
Yes, Thanks, Scott good question.
Look as I said, we are pleased with the enrollment or.
And the physician enthusiasm for both NPM one in <unk>.
Do think that.
BTG is it sort of advantage to our filing strategy for <unk> and the ability to combine cohorts.
They have something to do with our ability to kind of move.
Slightly more quickly.
With <unk>, So I think that's as part of it the other part of it is at KMT <unk>, there's probably less competition for patients with our competitors.
And.
NPM, one maybe drawing more patient pay.
Patients in the various studies so I do think that there is there are some competing factors here.
Reverting factors here, but ultimately I think we're doing quite well with MTM, one and we feel that.
Getting to market as quickly as we can with <unk> will advantage us greatly as we bring data behind for MTM, one hopefully get that approved as well.
Okay.
And then maybe one question on the combo studies has the dosing combined commenced.
The beat AML study I think you stated that we're just expecting safety. This year. So I was just wondering if dosing started yet.
Yes. Thank you for the question. So yes, the combination studies have initiated.
So there is the again the augment 100 to chemo combination that has been enrolling patients as well as the beat AML trial that is also enrolling patients with April .
Okay, Alright fantastic thanks for taking the questions.
Thank you.
And our next question comes from Bert Hazlett with <unk>. Your line is open.
Thanks, Thanks for taking the question just one or two for me could you just remind with the.
The consideration of revenue met a post transplant is there a.
Regulatory.
An indication strategy, there or are you thinking about that.
Companionable this thing our publication strategy.
Could you just frame, how youre thinking about that from a regulatory perspective.
Sure Chris do you want to take that question from Barclays.
Yeah, So bert.
Ongoing pivotal trials.
Allow patients to go back on to <unk>.
<unk> after their if they have response go to transplant. They can go back on.
<unk> it.
That.
Work in the pivotal trial, we don't anticipate a label indication for that.
Best case scenario is we might be able to describe in the label that how the trial was conducted so I think.
That information will be mostly.
And to the community through publications it does.
Set us up for potentially made formal maintenance trial down the road, but the way we are handling it in the pivotal trial.
It won't be a claim regarding.
Sure.
Okay.
Helpful. Thank you.
And then just shifting gears from a corporate perspective.
<unk>.
I believe you have a co promote option there.
<unk> hired a head of commercial relatively recently.
How are you thinking about that decision and is there a particular timing.
That needs to be.
Executed on your part.
Alright, Thanks for the question good question.
You are correct.
Our agreement with insight does include a co promotion option on extra telenet.
That election is made in proximity to finally, so it is something that.
We will have to.
Kind of.
Let people understand a little bit better in the coming months as we get closer and closer to that time point, obviously, we will have a finally as we said.
Before the end of the year so.
We're thinking actively about it I would just say that we have two programs that.
We've described today and as you know that are really on top of one another in terms of timing both core.
For filing and approval and so.
The commercial opportunities, while not the same or.
May have some synergy in terms of the doctors that are being called on.
How we promote these products so the thought.
Essentially that there could be some very nice synergy between what we do with <unk> and so we're thinking actively about whether whether the co promotion option makes sense for us.
Okay. Thanks, and I know it may be early but any thoughts on sales force sizing infrastructure things like that.
Sure its a longer conversation I would say that this is a focused commercial effort meaning that.
These are not.
Very large patient populations more importantly, they are covered by reasonably focused number of physicians.
So.
So the field.
<unk> for us to cover it is somewhere in the order of 40 to 50 position supporting 50 reps.
We'll refine those estimates and start to give some more color to what.
What the organization looks like that we are building, but we are actively engaged in bringing in leadership.
To the company in order to be ready in time for launch for both of these funds.
Terrific look forward to more thanks.
Thank you Barry.
Our next question comes from Joel Beatty with Baird. Your line is open.
Great. Thanks for taking my questions first one is on the pools PMT to a cohort.
Anticipating the efficacy will be presented just for the full cohort on the label or could there also be a breakdown of AML and <unk> on the label.
Yes, Joe Great question I think.
That was sort of alluded to or part of another question that was asked earlier.
This strategy for pulling it out.
We'll be presenting a.
Total number of patients and there'll be analyzed I think of course, the agency will look at all the subsets and.
And we will have a breakdown of that and I think that'll be part of our package.
Essentially the the analysis the primary analysis that will be done as on a pooled patient.
Okay. Thanks, and then.
Question on R&D, because it looks like it'll ramp up over the course of 2023.
Any priorities that you anticipate will be a part of that increased spending.
Yes, Joe this is Keith I'll take that one so.
A lot of the.
The increase in 2023 will go towards the initiatives that Mike laid out in his comments.
We have a pretty expensive.
Development program for <unk> to realize the full potential of the current pivotal programs our focus in relapsed refractory, obviously, but going forward you heard Mike will expand upon.
All of the work needed to do.
To again recognize our full potential looking at frontline combos and therapy et cetera. Additionally, in anticipation of a filing by year end.
And to be ready for a launch in 2024 there'll be an increased increase in CMC slush pick up pick up expenses.
To get our registration batches manufactured remember, we do have not only.
Solid tablet formulation that will be bringing to market, but also a liquid formulation.
As well.
That's the majority.
The increase.
Great. Thank you.
Thank you Joe.
It appears we have no further questions at this time I'll turn the program back to Michael Metzger for any additional or closing remarks.
Great. Thank you operator, and thank you all we look forward to seeing many of you at the Cowen and Barclays healthcare conferences in the next few weeks in March and with that I wish you all good night. Thank you.
This does conclude today's program. Thank you for your participation and you may disconnect at any time.
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