Q2 2022 aTyr Pharma Inc Earnings Call
Okay.
Good afternoon, ladies and gentlemen, and welcome to the a tier pharma second quarter 2022 conference call. At this time all participants are in a listen only mode. Later, we will conduct a question and answer session and instructions will be given at that time to ask a question during.
Session, you'll need to press star one one on your telephone as a reminder, this conference is being recorded for replay purposes.
It is now my pleasure to hand, the conference over.
Call over to Ashley Dunston, eight tires director of Investor Relations and corporate Communications. Mr. Johnson you may begin.
Thank you and good afternoon, everyone and thank you for joining us today to discuss our second quarter 2022 operating results and corporate update.
Joined today by Dr. Sanjay Shukla, our president and CEO , Ms Jill Broadfoot, our CFO and Dr.
I V. P of research on the call Sanjay will provide an update on our corporate strategy, including our clinical program for <unk>.
And I think it will go over our research and discovery programs and not doing too and I journey.
Joe will review the financial results and our current financial position of course, when they get back to Sanjay to open up the call for any questions.
Before we begin I would like to remind everyone that except for statements of historical facts the.
Statements made by management and responses to questions. On this conference call are forward looking statements under the Safe Harbor provision of the private Securities Litigation Reform Act of 1995.
These statements involve risks and uncertainties that can cause actual results to differ materially from those in such forward looking statements. Please see the forward looking statement disclaimer in the company's press release issued this afternoon as well as the risk factors in the company's SEC filings and included in our most recent annual report on Form 10-K subsequently filed quarterly reports on Form 10-Q and a.
And our other SEC filings and you reliance should not be placed on forward looking statements, which speak only as of the date. They are made as facts and circumstances underlying these forward looking statements may change.
As required by law <unk> pharma disclaims any obligation to update these forward looking statements to reflect future information events or circumstances, I will now turn the call over to Sanjay.
Thank you Ashley.
Good afternoon, everyone and thank you for joining us for our second quarter 2022 results conference call.
We're pleased with the progress we've made in the second quarter as we advance our phase III study.
And patients.
Sure.
This global pivotal study is a major milestone for a tire and the sarcoidosis community.
As it is projected to be the largest interventional study for patients with.
To date.
I'm happy to report that the study is underway with several centers initiated in the U S.
And importantly, we remain on track to enroll a patient this quarter.
As we began I'll summarize a few highlights since we last spoke in May.
We announced plans to initiate a global pivotal phase III study to evaluate the efficacy and safety of vessels.
<unk>.
Yeah.
We announced fibroblast growth factor receptor or MTR part for <unk>.
As the target receptor for a fragment of the alanyl trna synthetase, which is also known as ours.
And we were recently very pleased to receive FDA fast track designation for <unk> for the treatment of pulmonary sarcoidosis.
Since the announcement of the <unk> study in May 2022.
Rapidly executed a number of operational milestones to advance the study start.
Multiple interactions with regulatory authorities in the U S EU and Japan have occurred.
Along with the submission of the study protocol.
Clinical trial applications to regulatory authorities ethics committees and institutional review boards.
Site selection qualification and initiation for several sites have occurred as well as an investigator meeting.
Four U S sites.
It's been a highly productive period for <unk>.
From the point of receiving our FDA Green light on the design of the study just last quarter to now and we eagerly anticipate enrolling of patients.
I'd like to acknowledge our fantastic clinical operations team that has moved at light speed to get our pivotal trial and launch so expeditiously.
With that said, let's discuss our clinical program for Repsol.
A bit more detail.
As a reminder, <unk> is a first in class immuno modulator for fibrotic lung disease.
<unk> is a novel FC fusion protein based on the naturally occurring splice variant of the lung enriched trna synthetase hardest fragment.
Downregulates aberrant immune responses in inflammatory disease states.
<unk> has been shown pre clinically to down regulate inflammatory cytokine and chemokine signaling and reduce inflammation and fibrosis.
The <unk> two or <unk> receptor.
Regulated on key immune cells during active inflammation.
And it is enriched and flame lung tissue.
So for demand by selectively to MRP too.
Therefore has the potential to normalize the immune system.
Serving to resolve inflammation and prevent progressive fibrosis, thereby stabilizing lung function and alleviating morbidity and mortality.
We're developing <unk> as a potential treatment for patients with interstitial lung disease or ILD, a group of rare immune mediated fibrotic lung disorders.
Our initial ILD indication rest of the day modest pulmonary sarcoidosis.
Sarcoidosis is the most prevalent ILD and it's characterized by the formation of granulomas or comps of immune cells and garner more organs of the body.
Sarcoidosis that affects the lungs is called pulmonary sarcoidosis and occurs in more than 90% of cases.
If left untreated persistent granulomatous inflammation can lead to irreversible scarring and fibrosis may lead to respiratory failure and death.
We estimate that there are close to 200000 patients with pulmonary sarcoidosis in the U S.
Around 150000 in major European markets.
And another 20000 in Japan.
Up to 75% of patients require treatment for their disease.
Approximately half of these will progressively.
Progressive disease, despite treatment and around one in five of all patients.
We will undergo.
We'll go on to develop lung fibrosis.
Indication for treatment of sarcoidosis is twofold to avoid danger to in Oregon.
Or to improve quality of life.
First of all three minutes, typically corticosteroids, which may effectively control symptoms, but are associated with severe debilitating side effects, particularly with chronic treatment.
Second and third line treatments are anti metabolite immunosuppressants, such as methotrexate and biologic immuno modulators such as Infliximab.
These drugs are also known to cause serious side effects.
Outside of Prednisone and other glucocorticoid approved in the 19 fifties.
None of these therapies are approved for the treatment of sarcoidosis and all are used based on limited clinical evidence.
We believe the initial target population for <unk> will be patients, whose diseases progressing despite steroid treatment.
However, even patients who are able to control their symptoms with steroids often experience such debilitating side effects.
You're forced to choose between living with the burden of disease or.
Or the toxic effects of steroid treatment.
Therefore, we also see an upside opportunity for <unk> as a steroid sparing agent in patients who are responsive, but unable to tolerate their steroid treatment.
Combined these populations represent an addressable market of roughly 150 to 200000 patients from major market.
Even with conservative market penetration and pricing assumptions. This represents a significant peak sales opportunity in sarcoidosis alone.
Because this is an orphan disease and treatment options are limited the FDA granted orphan drug designation for <unk> for the treatment of sarcoidosis.
Additionally, we recently announced that FDA.
Has also granted fast track designation.
Perhaps look at a month for the treatment of pulmonary sarcoidosis.
The Fda's fast track designation helps facilitate development and.
Extra data review of drugs to treat serious or life threatening diseases with unmet medical need.
Fast track designation provides certain benefits, including more frequent interactions with the FDA throughout the development program as well as eligibility for accelerated approval priority review and Rolling review.
This fast track designation underscores the significant need for new therapy that provides clinically meaningful outcomes for patients living with pulmonary sarcoidosis and reinforces the potential vessel for demand to be a transformative disease modifying therapy.
And address a major unmet medical need.
We see further upside potential perhaps of Piedmont and other forms of ILD.
Immuno modulator treatment is the current standard of care.
This includes indications such as scleroderma related ILD.
Other connective tissue disease related audis, and chronic hypersensitivity pneumonitis among others.
These diseases share overlapping immune pathology with sarcoidosis and are currently treated with similar drugs.
Additionally, as tophet Ahmad.
Has been shown to be effective in animal models of these diseases.
Taken collectively the opportunity for <unk>, and sarcoidosis and other ILD represent 2% to $3 billion in peak sales.
Let's take a moment to go over the data, we generated perhaps to Piedmont and some of the details around the current <unk> study.
As a reminder, last September we reported clinical proof of concept for <unk> based on positive results from my Phase <unk> study in pulmonary sarcoidosis.
Study, which included a forced steroid taper demonstrated safety tolerability and a consistent dose response for our Sofia tomorrow on key efficacy endpoints and improvements compared to placebo, including measures of steroid reduction.
Function sarcoidosis symptom measures and inflammatory biomarkers.
According to medical experts and this is the first randomized placebo controlled trial of any therapy for pulmonary sarcoidosis that.
That demonstrates effect on physiologic and quality of life measures concurrent with steroid reduction.
Based on findings from the Phase <unk> study and feedback from the FDA.
May of this year, we announced plans to initiate the <unk> study.
This is a global pivotal phase III randomized double blind placebo controlled study to evaluate the efficacy and safety of <unk> and.
In patients with pulmonary sarcoidosis.
Is the 52 week study consisting of three parallel cohorts randomized equally to either three milligrams per kilogram or five milligrams per kilogram up absolute good Ahmad.
Placebo.
Dosed intravenously once a month for a total of 12 doses.
The study intense enrolled 264 patients with pulmonary sarcoidosis at multiple centers in North America, Europe and Japan.
The trial design incorporates a forced steroid taper design with the primary endpoint of the study.
<unk> steroid production.
Secondary endpoints include measures of lung function and sarcoidosis symptoms.
The trial design for our Sofia is based on key learnings from the phase <unk> trial that we believe sets up this study for clinical and regulatory success.
This includes takeaways from the forced steroid taper and results for steroid reduction in the post taper period.
And the <unk> II study, which was a six month trial pace.
Patients underwent enforced tariffs.
Steroid taper to five milligrams with the option to be titrated to zero at week 16 based on symptoms.
We learned that patients could generally handled a forced taper.
And even though an eight week taper was aggressive we found that those patients on <unk>, we're able to taper more successfully.
To be able to evaluate the efficacy of <unk> compared to placebo in <unk> fit patients will be forced.
To fully tapered their steroids zero milligrams.
Though over 12 weeks instead of eight.
We believe that by providing more time for the taper.
And tapering steroids completely.
Finally by following patients for an additional 24 weeks compared to the prior studies, we expect more patients receiving placebo experienced worsening symptoms requiring increased steroids compared to patients receiving <unk>.
We also have adjusted the entry criteria for background steroids from a minimum of 10 milligrams in the <unk> study to a minimum of seven five milligrams of prednisone per day in the phase III.
This aligns with feedback from physicians.
But there are many patients who require a bit less than 10 milligrams of daily steroids to maintain their symptoms.
And could really benefit from a reduction.
A reduction of two or two five milligrams of <unk> daily steroids for these patients over the course of time could be very impactful.
On the whole we believe these adjustments will enrich the study and build upon the positive findings from the phase <unk> trial.
Again, so if it is the largest interventional study for patients with target doses to date with this study <unk>.
<unk> is positioned to be the first disease modifying therapy to market for patients with this debilitating disease and based on data to date, we believe one that could reduce steroid burden.
Maintain lung function and improved symptoms.
I'll now turn the call over to Leslie Nagel, our VP of research to discuss our preclinical and discovery program.
Our trna synthetase platform.
Thank you Sanjay.
Our focus is on advancing our clinical program for <unk>. We also have a number of exciting opportunities emerging in research.
Our lead preclinical candidate in HR 28 or 28.
A fully humanized monoclonal antibody that selectively and functionally blocks the interaction between anarchy Xu a cell surface receptor that is up regulated.
Variety of solid tumors, and particularly expressed in many aggressive cancer.
Jeff one of its primary layer.
Jeff is a validated mediator of tumor survival in gorilla and correlates with tumor Invasiveness and Mcafee.
Current therapies that directly target the classic veg F that Jeff receptor signaling do not block the anarchy, two bedroom receptor Shanghai.
Preclinical data suggests that by blocking the interaction between digest and anarchy too.
Tim maybe an effective novel therapeutics that combat resistance and reduces invasion and metastasis by down regulating master drivers of lineage participants such as that one.
To serve as a differentiated approach targeting aggressive cancer.
Our current work for 2010 is focused on designing and refining our clinical strategy, we are focusing on resistance and highly aggressive solid tumors, where anarchy jayden from keybanc.
We are prioritizing tumors based on scientific rationale that's by our preclinical data and support from external literature, and then incorporating the clinical development path for each.
To generate a list of top target tumor types.
This approach provides us with data driven methodology to select indications to target our first clinical study for 2018.
Neuro endocrine tumors, including pancreatic neuroendocrine tumors.
Intergroup prostate cancer renal cell carcinoma, and triple negative breast cancer, all scoring high using this algorithm.
We continue to hone in on the target indications are 2008 time may be the most effective we are on track to initiate a phase one study in cancer patients in the fourth quarter of 2022.
At this point I wanted to take some time to highlight our trna synthetase platform.
Is the size of the HR is founded upon and we expect to be a defining part of our future our approach to drug discovery and development is rooted in our deep understanding of this family approach.
Extracellular pathway. They regulate it is our mission to generate therapeutics targeting these pathways in order to improve patient outcomes.
We have built an intellectual property estate of over 200 issued patents to date that creates a strategic boundary amount our proprietary library of extracellular trna synthetase protein fragment. This library covers fragrance from fall 2010 in some cases, placing particular emphasis on those that have the highest likelihood of being.
Therapeutically viable.
Our experience with Bernie <unk> from discovery, all the way to clinical proof of concept has paved the way to develop additional molecules for machine Valley and has educated us on the most efficient ways to translate these discovery findings into therapeutic potential.
Innovation, enabling rapid target cell receptor identification and addition to phenotypic screening efforts have become hallmarks of our research program.
Currently accelerating research efforts to mine this library or potential new therapy.
As an example in June and a poster presented at the Keystone symposia on tissue fibrosis, and repair we announced the discovery of a target receptor for one Carrington fragment from Allen <unk> Caron.
Ours to be FGF are for this.
This receptor is involved in many cellular processes, including cell proliferation differentiation and tissue repair.
<unk> is known to play a role in diseases related to inflammation and fibrosis, including conditions, where unchurch fibrosis can proceed the development of certain cancers.
We look forward to further interrogating this interaction between this fragment of ours and of GFR for exploring the implications for this synthetase fragment as a potential new therapeutic.
Moreover, the method utilized to identify this receptor can be further employed to identify and validate new molecular target from RTI. Since this platform, which we believe holds great value that we aim to continue to unlock.
I will now turn it over to our Chief Financial Officer, Jill Broadfoot to review our financial results.
Thank you Leslie I'm happy to report that we ended the second quarter 2022, with $89 3 million in cash restricted cash cash equivalents and investments.
Search and development expenses were $9 1 million for the second quarter, 2022, which consisted of product development and manufacturing costs for the outlook for demand in 2810 programs as well as startup costs for the phase III <unk>.
General and.
And administrative expenses were $3 4 million for the second quarter 2022.
Common shares outstanding were approximately $28 1 million.
Fully diluted shares were $33 1 million as of June 32022.
While we are taking important steps to advance our clinical preclinical and discovery programs. We believe we are tracking well with our capital utilization, we continue to implement a data driven approach to chairman capital allocation program.
Warrant further exploration, we believe our current cash position along with the opportunity for additional milestone payments from our partner Kieran pharmaceutical.
This approach now I'd like to turn the call back over to Sanjay before we open it up to Q&A.
Thank you Jill.
As we've discussed here today, we've really had a highly productive quarter, particularly for our <unk> clinical program.
And while we focus on advancing a sofa demand we've continued to capitalize on our intellectual property portfolio by unlocking new ways to create therapeutics from this novel biology through the discovery.
<unk> targets for trna synthetase.
And the expiration of their role in disease.
We believe we are in a strong position financially and with the launch of the <unk> study. We are looking forward to entering the next phase for the company.
We appreciate your support and look forward to providing additional updates.
This quarter.
And now open it up 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 Gregory Renzo with RBC capital markets. You May proceed.
Hey, good afternoon, Sanjay and team congratulations on all the progress to date and thanks for taking my questions.
Maybe I can just start.
With respect to the regulatory progress outside of the U S. I know you had some some points at the top there.
Just to build on that maybe a little further I'm just curious how we would think about the level of regulatory alignment and ex U S countries disregarding steroid sparing as the primary endpoint and maybe just touching on.
The strategy for ex U S approval and launch and then maybe I'll just ask more more broadly if I may.
As you speak to getting set.
<unk> set up and getting a patient.
On drug for third quarter, but I'm just curious if you could comment a bit on what you see is just some of the broader challenges and risks to that.
The timeline is as you build alignment with the centers and get patients on trial. Thank you so much.
Thanks for the question Gregg So first off with regard to regulatory interactions what I can say is <unk>.
They've been very productive and frankly, the alignment has been 100% regarding the primary endpoint I think it speaks to the fact that Stuart reduction now.
Universally is being viewed quite favorably.
As an approvable endpoint in you've seen other companies start to of course in other indications receive approvals based on CRE production.
Our plan is.
<unk> really Chris and well thought out plan.
Not only FDA not only Japanese regulators, but the larger European regulators continue to support and frankly.
The back and forwards have been.
Very easy I think it also helps that we have buy in from probably upwards to 60 experts worldwide.
Folks have been waiting a long time for a circuit OS program to get into phase III to.
To my knowledge, we're the only one effort to kind of get.
To this point.
So I do think that there is really strong desire.
To get to to get a drug approved.
I think we have smartly advance the program and now it is really set up well for success.
When you think about some operational challenges you always have to think about.
Planning ahead of time.
Two really mobilized sites and get things moving.
Having some of the launch activities at Etfs, where we started to bring people together show them our data.
This has been rather fast.
So the fact that we've been able to basically from one quarter.
Flipped a switch here and are close to enrolling a patient I think it speaks to not only our clinical operations team, which is I would say two standard deviations better than anybody out there in industry.
They've demonstrated that they can get a trial done through COVID-19 respiratory pandemic and we finished a respiratory trial in a very great population here. So I think.
We have a great team, but also the patient and provider community have been so excited to get involved with this trial, we want to keep that excitement in that.
Sort of acceleration.
It's occurred from our phase two data and continue to build upon that as we go into.
This trial, so that we can move as quickly as we can patients don't want to wait we don't want to wait to get the results out we know it's a longer trial, but does start thus far I would say we're in a fast start here.
That's great Sanjay I appreciate the color and congratulations again.
Thanks, Greg.
Thank you one moment for questions.
Our next question comes from Joe <unk> with HC Wainwright you May proceed.
Hey, everybody. Good afternoon. Thanks for taking the question Sanjay first a logistical question on <unk>.
Why do we need to see to be able to get cure and into enrolling patients in the study.
Yes, I think it's just a matter of.
I can't.
Curing itself being.
One company they are operationalized in Japan, but thus far I can tell you that we have no hiccups from a regulatory point of view.
With Japan, and I think it's just a matter of now.
Opening sites and getting patients dosed over there.
We are focusing on the U S first and I would expect the first patient to be enrolled in the U S.
And then and then I think.
In the following months, we will look for Japan.
Japan, and Europe to start to enroll as well so.
What I can tell you is.
To guide to exactly when a patient might be enrolled in Japan, but we're tracking really well over there.
And thus far there is there is nothing in the timelines that is slowing us down.
Got it got it and then.
You talked about upsell said in looking at certain inclusion criteria I was wondering if you can describe a little bit.
Exclusion criteria, because if im looking at some of the differences between the earlier study in <unk>.
Look at some differences with regard to the initial fibrosis level as well as what appears to be differences in the imaging that.
I think could potentially broaden the population.
Yes, Im glad you brought that up so let's first address the imaging the field has moved.
Back into <unk> as being the appropriate imaging.
Metric now it is not an endpoint is not a validated endpoint that.
The regulators would improve but we are using it to enrich.
I'd say two two day rich in a way patients that are two fibrotic.
Our drug works best when there's active inflammation. Therefore, we want to make sure that we have patients that are not to fibrotic now in previous studies I think we have the opportunity to.
Exclude based on pet scan, but as I said <unk> now.
The sort of more acceptable way of delineating fibrosis and inflammation I do think our cutoff being at 20% aligns with some of the other therapies you've seen Tuscaloosa Madden intended in the scleroderma trials have that as a cutoff.
I expect to happen.
The toughest patients to treat with <unk> I expect that we will be able to frankly excludes some of those patients. We may end up with more patients with some mixed disease, but still enough inflammation that we can move their disease and modify it. So I think it does two things for us number one.
Those patients that may be too far gone to do well with <unk>, we think we will exclude those patients but.
But we also think we will capture some patients and maybe even this has relevance to a larger market opportunity.
Patients, who have mixed findings, but still enough inflammation, so that we can impact and.
Reduced their steroids.
Remember these are patients that also if you start to have fibrosis steroids can make your fibrosis worse, so theyre in a tricky situation.
I think this points to our ability to enroll better enroll smarter and probably long term.
Have a much larger footprint of <unk>.
Market potential here with patients with mixed disease.
Got it that's helpful. Thanks, and then just real quick if you don't mind.
With regard to 2810 are you willing to share either.
Let's call it an initial communication strategy.
Subject to change obviously about how we how would you look to.
Released news from the upcoming Phase <unk> study do you have a.
Proverbial Magic Magic number of patients that you would want to see a couple of responses first or a critical mass of patients before you release news because obviously these are going to be.
<unk> patient population.
Yes, So I think the first step and then Leslie alluded to it we have a number of.
Top target tumor types.
We've outlined.
Just on I think smartly looking at literature looking at our data talking to experts.
We're going to be able to enrich right off the bat. So I think first understanding what the trial exactly looks like.
The expectation here is I will come back to you.
And the rest of the public to basically outlined what that clinical protocol looks like.
Daniel Youre going to be able to get into when are you going to have batches of data available is it going to be at the end or iteratively.
Given that it's an early trial its a safety trial.
Expectation here as well.
We could probably have a more steady flow of information first starting with safety. But then also looking at pockets of response in these early trials similar to what most cancer studies are set up where you at least want to check the box. There early on some safety work, but then you can start to dig into okay from these enriched <unk>.
Types, where you're seeing the best effects.
That's going to further define what the next steps for that program are going to be but I'm really proud of the work. The research team has done here to again really be data driven and I would say, we're one of the most data driven biotech companies out there. So we're not.
Our goal is to set up these experiments for success.
Learned along the way.
The valves are things that arent really working well, but then really focus on what's working well so stay tuned for that Joe Im anxious to get those details out soon as well.
Got it thanks, a lot Sanjay.
Yes.
Yeah.
Thank you one moment for questions.
Our next question comes from hard Taj Singh with Oppenheimer You May proceed.
Great. Thank you. Thanks for the couple of questions and really nice update from JMP.
I was hoping.
You described the paper.
So steady.
I believe going from like you start sterile tape out about two weeks and going to about 12 weeks.
Recruiting these patients can you.
That's pretty.
Maybe aggressive relative to the previous study right I mean is that a good sign that youre feeling confident you can take these patients off more quickly on steroids and try to get them to have low steroid level as possible.
The first question and then how does that impact enrollment.
And then you put the absolute fit study.
That paper and then secondly, you talked about how your clinical team is now starting to really kind of come together.
Do you have an R&D for 2010 in the fourth quarter.
<unk> project in the clinic.
What are the main differences youre seeing developing your targeted therapies between oncology and inflammation and thanks for the question.
So.
All up break this into parts versus the taper itself and I'll start maybe with enrollment.
Fact that we saw patients.
Get to zero in the last trial was really an outstanding findings and really unexpected in such a fast manner. So in a six month trial to see.
And that last cohort.
Three out of nine patients get to zero.
That was really unexpected but it also has I think lit a fire around.
The patients to providers the internal team here to say, let's let's really try to actually see if this drug could have even more spirit steroid sparing effects may be a steroid replacement type type of effect for certain patients.
That's actually helping us with enrollment and some early projections because patients now look with that data that we produced and say this I might have an opportunity here to get off steroids, if I participate in this trial.
So I think in many ways. The design is going to help us enroll better.
Now with that in mind, we have to be a little bit more careful about how we taper we had an eight week taper that brought folks down from anywhere at a 10% to 25 milligrams of prednisone down to five over eight weeks.
We have to we have to go now we're trying to go to zero, which essentially a similar population seven 5% to 25.
So we got to give it a little bit more time, so we've added four weeks.
So I think.
In my mind and the experts mine. We've designed this very closely with people like by Boston, Dan cover some of the experts that were involved in the last trial.
We feel.
Bullish with the goal here to get to zero patients want to actually try to go to zero and we're allowing for a little bit more time for more weeks. That's also not going to hurt us in our analysis because you really are now we're looking at basically.
Basically a year trial, so we're adding.
Another 2024 weeks of therapy here.
So I think all in all this is going to provide us I think.
A lift from enrollment it is something that I think we're going to challenge. The enormous here. It's why I think the market opportunity is closer to $3 billion. When you talked about what <unk> could do for patients not only sarcoidosis, but in a number of interstitial lung diseases, and we think as time goes on.
Hopefully the investment community picks up on the fact that.
What's staring you in the face and hitting you in the mouth here is a transformative multibillion dollars of therapy, we hope people start to pay attention to that the patients and providers already have the regulators have.
I think I think this is something that people need to wake up and see the kinds of impact we can make here.
Yes, no that makes sense.
And just the broader question Sanjay on as Youre going from developing.
In inflammation to oncology.
And I think this is one of the actually very unique things for me for <unk> is that you've got some pathway is that you don't really comment with a lot of other companies I imagine scarcity value, but then what does that do to your discussions with regulators you guys Youre looking at RP to synthetase.
Yes, okay. So thats.
That's important follow up here I think first and foremost validating the platform is what a sofa to Monday. So that's also given we've always had comfort in that but externally. There is a lot better comfort. We produced some data already from a safety perspective now understand the modality. There is different it's an antibody so it's.
A bit more straightforward.
But the approach is still for us to basically say, yes, we are playing outside of the norms of most drugs in oncology and we are breaking ground on a new area in oncology.
Again, it's really guided around understanding of inflammation and fibrosis and we've used that term quite a bit both are mechanisms that play a huge role in oncology as well.
So I think what we've done here is.
Smartly designed again a plan.
Regulators I think are becoming more understanding around our biology, it's making those kind of interactions easier it's speeding things up.
But at the same time, we're going to really.
Create real cogent clinical strategies.
Because again I think I think our goal here is to run smart experiments that we believe will succeed and we will do that even early on in oncology you have to do that really really early on because in many ways. The market expects to see response, even in those early patient trials. You don't you don't do healthy volunteer work eco strategy straight into.
Patients for example, so we will we will incorporate I would say a more accelerated way of demonstrating effects.
For 2018.
And lastly, I will just say, even the FTF. Our findings again, we're now anchoring and we feel really really good around the process that we have here and that is a receptor that is better understood the nor appealing to and clearly has more implications with regard to liver fibrosis liver cancer kidney fibrosis.
A lot of literature, there, we're going to be hitting it from a different angle and our angle has already produced the most really fantastic findings can sarcoidosis.
Since the <unk>. So I think I think there is something that will be set around the process that Leslie has also developed here with the research teams. So that we create a steady stream of derisked and potentially really transformative pipeline candidates.
Yes. Thanks, Thank you for all for the questions and all the color really appreciate it.
Thank you and as a reminder to ask a question you will need to press star one on your telephone.
Our next question comes from Yale Jen with Laidlaw <unk> co you May proceed.
Good afternoon.
Thanks for taking the questions.
My first question is that.
Alright. Thank you you have mentioned that the last time that the.
Enrollment until data released at the roughly 200 to.
Two two and half years I guess.
And whether this timeline is still holds.
Yes, hi yield absolutely I mean, we're just getting started.
Allow me to update guidance, depending on how enrollment goes give me, let's get let's get the 70 centers up let's start to see some data, but I think it's fair to say that.
This data will be looking at that.
That timeline that you've laid out there I mean, as we get closer to.
Into 'twenty three 'twenty, we're going to know them once that last patient gets enrolled.
But I think based on our assumptions and current projections.
That that timeline.
That's correct.
Okay, Great that's very helpful.
Actually mentioned earlier.
And you answered that you could have.
Hi, bulk patients other than the.
Cycle, those Hello, Liza in the lung.
My question to you is that if so would you said would you also start to annualize the symptom relief will other aspect of this.
Mixed patients.
There are several doses.
Other tissues.
As a gift.
Insight into whether.
Let me expand it to other indications.
So I think what you're getting at is the extent of fibrosis and lets understand all of these patients have some fibrosis.
Some might have just one or 2% others may have 15% or 16%, but they all may have cough shortness of breath and be on a significant amount of prednisone.
To your point do we have any baked in stratification to look at.
More than 10% more than 15% not necessarily I mean, these are going to be tertiary endpoints in the end what do we do know is nor Poland targeting nor plan seems to be a potent anti inflammatory and anti fibrotic.
We are focused on the anti inflammatory effects and a longer trial, we may start to see that were bending things from a fibrotic perspective.
So we're just going to be prepared to look at that.
When you think about.
<unk> is an anti fibrotic in the end that's really what we're trying to do here, we try to prevent fibrosis from taking hold in these patients.
It is also why I think experts in other areas like even Ips are really intrigued about wanting to try a sofia demand over there because nothing can really change and disease modify we've seen therapies.
In development now that $1 billion valuation.
And not show dose response for example.
And there's a lot of hope I think in IPF to see anti fibrotic effects.
Going to interrogate that in our trial, because sarcoidosis and certainly in the same family as IPF.
But being more inflammatory that's what we're prioritizing that's why steroid reduction as our primary endpoint.
But to your point.
I do think that the larger market opportunity again, I'm going to stress here is several billion dollars because we have a unique offering that.
Frankly sits outside those approved therapies.
The fact that we shall see symptom improvement is something that no therapy in fibrotic lung disease has really shown.
So I think this is where there's a lot of excitement we're going to keep our eyes on the prize.
Execute well on the sarcoidosis trial, but there is significant interest, yes, and even some of those other fibrotic conditions.
Okay, Great and maybe last one here is that while in 2010.
Yes.
You're going to start a phase one study.
Fourth quarter should we just assume a somewhat typical cancer study so a dose ranging study design, maybe such as the three plus three maybe a multiple single dose ascending and.
What might be the PD readout.
You may have so what kind of biomarker you might also look for and thanks.
I'm going to hold off on the biomarker part because thats going to have a lot to do also with tumor types that we pick.
What I can tell you is it will have some elements.
We pride ourselves of being.
Real cutting edge drug developers here.
So I think we're going to have an expedited adaptive type of approach, but yes, I think we know in cancer. There are certain boxes, you have to check with regards to.
Modular safety.
Cohorts and then potentially then looking at efficacy in certain tumor types. So it will have some classic elements because we don't we want to get the IND approved.
But I also think there's going to be some opportunities here to again enrich the risk and try to get an early signal.
Once I actually know for example, if it's triple negative breast that we're focusing a little bit more on her neuroendocrine. We can then kind of get into a.
A cadre of PD biomarkers to start to look at.
We think we have a very differentiated opportunity mechanistically.
We are quite bullish on that program as well.
As we set it up.
Okay, great. Thanks.
Thanks, a lot and best of luck and congrats on all the progress this fall.
Thanks Neil.
Thank you and I'm not showing any further questions. At this time I would now like to turn the call back over to Sanjay Shukla for any closing remarks.
So thanks, everybody for tuning in today, obviously, a lot of operational progress maybe.
Not the Sexiest updates as we've had in the past, where we are we're putting out clinical data, but certainly a quick turnaround here from last quarter and I'm really proud of the work. The team has done to really get this study launched basically in a quarter here.
From the Green light.
I appreciate everyone's interest continued support and we look forward to talking to you in the future. Thank you.
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Okay.
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