Q4 2023 aTyr Pharma Inc Earnings Call
Okay.
Operator: Good afternoon, ladies and gentlemen, and welcome to the aTyr Pharma fourth quarter and full year 2023 conference call. At this time, all participants are in a listen-only mode.
Good afternoon, ladies and gentlemen, and welcome to the a tier pharma fourth quarter and full year 2023 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.
Operator: Later, we will conduct a question and answer session, and instructions will be given at that time. To ask a question during the session, please press star 1 1 on your telephone and wait for your name to be announced. To withdraw your question, please press star 1 1 again. As a reminder, this conference is being recorded for replay purposes. It is now my pleasure to hand the conference call over to Ashley Dunston, ATyr's Director of Investor Relations and Public Affairs. Ms. Dunston, you may begin. Thank you, and good afternoon everyone.
Ask a question during this session. Please press star one one on your telephone and wait for your name to be announced to withdraw your question. Please press star one again.
Minder. This conference is being recorded for replay purposes. It is now my pleasure to hand, the conference call over to Ashley Dunston, eight tires director of Investor Relations and public Affairs. Mr. Johnson you may begin.
Okay.
Thank you and good afternoon, everyone. Thank you for joining us today to discuss a tire fourth quarter and full year 2023 operating results and corporate update we are joined today by Dr. Sanjay Shukla, our president and CEO and Ms. Jill Broadfoot our CFO.
Ashley Dunston: Thank you for joining us today to discuss aTyr's fourth quarter and full year 2023 operating results and corporate update. We are joined today by Dr. Sanjay Shukla, our President and CEO, and Ms. Jill Broadfoot, our CFO. On the call, Sanjay will provide an update on our corporate strategy, including our clinical program for exofitamide and research and discovery programs. Jill will review our financial results and our current financial position before handing it back to Sanjay to open the call to 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 Security 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.
On the call Sanjay will provide an update on our corporate strategy, including our clinical program for X linked to the Mod and research and discovery programs.
Joe will review, our financial results and our current financial position before handing it back to Sanjay to open the call up for any questions.
Before we begin I would like to remind everyone that except for statements of historical facts.
<unk> 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.
The statements involve risks and uncertainties that can cause actual results to differ materially from those in such forward looking statements.
Ashley Dunston: 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 in our other SEC filings. Undue reliance should not be placed on forward-looking statements that speak only as of the day they are made, as facts and circumstances underlying these forward-looking statements may change. Except as required by law, aTyr 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 Sunshine. Thank you, Ashley.
These forward looking statements 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 in our other accuracy filings.
Reliance should not be placed on forward looking statements, which speak only as of the day. They are made are 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.
Sanjay Shukla: Good afternoon everyone, and thank you for joining us for our fourth quarter and full year 2023 results conference call. At aTyr, we are leveraging evolutionary intelligence to translate tRNA-synthetase biology into new therapies for fibrosis and inflammation. Our lead therapeutic candidate, Efsel Fittemad, is a first-in-class biologic immunomodulator based on a naturally-occurring, lung-enriched splice variant of the tRNA synthetase heart. F-sulfitamide selectively modulates activated myeloid cells via Neuropilin-2 or Nrp2, to resolve inflammation without immune suppression and potentially prevent the progression of fibros
Good afternoon, everyone and thank you for joining us for our fourth quarter and full year 2023 results conference call.
At <unk>, we are leveraging evolutionary intelligence the tree.
Late trna synthetase biology into new therapies for fibrosis and inflammation.
Our lead therapeutic candidate so Piedmont.
As a first in class biologic immuno modulator based on a naturally occurring long enriched splice variant of the trna synthetase cars.
A sofa demand selectively modulate activated myeloid cells via <unk> or an RFP too.
To resolve inflammation without immune suppression.
And potentially prevent the progression of fibrosis.
Sanjay Shukla: We're developing efsofetamide as a treatment for patients with interstitial lung disease, or ILD, a group of rare immune-mediated disorders that can cause chronic inflammation and fibrosis of the lungs. 2023 was an important year for aTyr as we progressed and expanded our F-sulfidamide clinical development program, which now includes two ongoing clinical studies. The Phase 3 FSOFIP study in patients with pulmonary sarcoidosis, a major form of ILD, and the Phase II EPSO-Connect study in patients with ILD related to systemic sclerosis, which is known as SSC or more commonly scleroderma. Throughout the past year, we have also greatly enhanced our mechanistic understanding of the way in which epsilicidomide is conferring its anti Nrp2 is highly expressed on activated immune cells undergoing an inflammatory response, notably myeloid cells, including monocytes and macrophages.
We're developing <unk> as a treatment for patients with interstitial lung disease or ILD, a group of rare immune mediated disorders that can cause chronic inflammation and fibrosis of the lungs.
2023 was an important year for <unk> as we progressed and expanded our absolute <unk> clinical development program, which now includes two ongoing clinical studies.
Phase III <unk> study in patients with pulmonary sarcoidosis, a major form of Iot.
And the phase III <unk> connect study in patients with ILD related to systemic sclerosis.
Which is known as SFC or more commonly scleroderma.
Throughout the past year, we have also greatly enhanced our mechanistic understanding of the way in which absolutely demand is confirming its anti inflammatory effects.
<unk> is highly expressed on activated immune cells doing inflammatory response.
<unk> myeloid cells, including monocytes in macrophages.
Sanjay Shukla: By binding NRP2, epsilcitamide guides the differentiation of monocytes at the site of inflammation into a macrophage subtype that is less pro-inflammatory to resolve aberrant inflammation. Dysregulated inflammation is a hallmark of myeloid-driven diseases such as IOD, where persistent, uncontrolled inflammation can lead to the progression of fibrosis. With this new understanding, we now have even greater clarity and confidence as to why Ef Our lead indication for F-sulfonamide is molar sarcoidosis, the most prevalent form of ILD, where approximately 70% of patients will have symptomatic disease and nearly 20% will develop lung fibrosis. Current standard of care is primarily oral corticosteroids, a highly toxic treatment that has limited clinical evidence, is broadly immunosuppressive and comes with side effects, resulting in a high disease burden for patients. F-sulfet is a global pivotal phase 3 study evaluating F-sulfenamide compared to placebo in the context of a forced steroid taper in patients with pulmonary sarcoidosis.
By binding <unk> absolute cinemark guides, the differentiation of monocytes at the site of inflammation into a macro funds subtype.
That is less pro inflammatory.
To resolve aberrant inflammation.
This regulated in inflammation.
A hallmark of myeloid driven diseases, such as Iot, where persistent uncontrolled inflammation can lead to the progression of fibrosis.
With this new understanding we now have even greater clarity and confidence as to why a softer demand may represent a breakthrough in treatment for Iot.
Our lead indication, perhaps so could a modest sarcoidosis, the most prevalent form of ILD.
We are approximately 70% of patients will have symptomatic disease, and nearly 20% will develop lung fibrosis.
Current standard of care, it's primarily oral corticosteroids are highly toxic treatment.
That has limited preliminary clinical evidence is broadly immunosuppressive and comes with side effects.
Resulting in a high disease burden for patients.
Absolutely fit is a global pivotal phase III study evaluating absolute cinemark.
Third to placebo in the context of a forced steroid taper in patients with pulmonary sarcoidosis.
This study is currently enrolling at more than 90 centers in nine countries.
We're pleased with the progress we've made thus far with this study which is expected to be the largest interventional study ever conducted in sarcoidosis.
Sanjay Shukla: This study is currently enrolling at more than 90 centers in nine countries. We're pleased with the progress we've made thus far with this study, which is expected to be the largest interventional study ever conducted in sarcoidosis. Completing enrollment in Epsilon Fit is our primary focus, and we anticipate doing so in the second quarter of this year.
Completing enrollment in absolute crude is our primary focus.
And we anticipate doing so in the second quarter of this year.
In the past few months as patients have completed the 52 week of Sophia study.
We have received multiple inquiries from study principles study principal investigators pis.
Those patients are requested to continue treatment once they completed the trial.
While <unk> and let's see patients are all blinded.
Sanjay Shukla: In the past few months, as patients have completed the 52-week FSOFIT study, We've received multiple inquiries from study principals, study principal investigators, or PIs, whose patients requested to continue treatment once they completed the trial, while ATyr PIs and patients are all blinded to what treatment patients received as part of the study, either esophageal or placebo. The feedback we've received suggests that some patients have performed well and want to continue on study drugs rather than return to the treatment regimen they had prior to the study. For some patients, that may entail resuming or increasing steroid doses, which many patients are reluctant to do.
So what treatment patients receive as part of the study.
Either <unk> or placebo.
Feedback. We've received has suggested that some patients have performed well and work to continue on study drug.
Rather than returning to the treatment regimen.
Meyer to the study.
For some patients that may entail resuming our increasing steroid dose, which many patients are reluctant to do.
Based on this feedback we decided to implement an individual patient expanded access program or EAP.
For patients who complete a sofa.
This individual patient EAP is designed to allow access to a softer demand for patients who have or are in the process of completing our software.
Beyond the duration of the clinical trial.
The company Pis and patients will remain blinded to treatment that occurred as part of their success.
Safety is a key component of any EAP.
We were able to implement this program based on the existing safety database from prior <unk> clinical studies.
And additional safety and Tolerability data from a data safety monitoring board or DSM be review of data from a sofa.
Sanjay Shukla: Based on this feedback, we decided to implement an individual patient expanded access program, or EAP, for patients who complete FSOFET. This individual patient EAP is designed to allow access to esophetamide for patients who have or are in the process of completing esophetamide beyond the duration of the clinical trial. The company, PIs, and patients will remain blinded to the treatment that occurred as part of F-CELT
Which included the evaluation of patients that completed 52 weeks of treatment.
The DSM V review recommended the study proceed without modification, suggesting no major safety concerns.
And while many types of EAP, they're typically implemented after data from a study has been unblinded we decided.
To implement this individual patient EAP early.
Not only based on feedback and demand.
But in part to continue to support those patients who have dedicated their time and entrusted us with their help by participating in this important study.
Sanjay Shukla: Safety is a key component of any EAP. We were able to implement this program based on the existing safety database from prior FSOFET and Mod Clinical studies and additional safety and tolerability data from a Data Safety and Monitoring Board, or DSMB, review of data from FSOFET, which included the evaluation of patients that completed 52 weeks of treatment. The DSMB review recommended the study proceed without modifications, suggesting no major safety concerns.
This program reflects our ongoing commitment to the sarcoidosis community as we work to develop a safe and effective treatment for that.
And me.
Our second indication, perhaps so could a modest SSC ILD.
SSC is a form of connective tissue disease, where ILD, commonly occurs and is a leading cause of mortality.
Current treatment options are limited and like sarcoidosis do not treat the underlying disease or improved quality of life.
So connect is a phase II proof of concept study evaluating a softer demand.
Compared to placebo in patients with SSC ILD.
This study, which dose the first patient last quarter is currently open for enrolment at multiple centers in the U S.
Sanjay Shukla: And while many types of EAPs are typically implemented after data from a study has been unblinded, we decided to implement this individual patient EAP early, not only based on feedback and demand, but in part to continue to support those patients who have dedicated their time and entrusted us with their health by participating in this important study. This program reflects our ongoing commitment to the sarcoidosis community as we work to develop safe and effective treatments for those in need. Our second indication for afsocudimod is SSC ILD. SSE is a form of connective tissue disease where ILD commonly occurs and is a leading cause of mortality.
We're focused on generating data from this study in.
In 2024, and we expect to provide an update on this study later this year.
We estimate that the two indications.
That comprise our current clinical program, perhaps so kind of model.
I would like to target doses and SSC ILD collectively represent a potential $2 billion to $3 billion global market opportunity.
This does not include any upside potential in other forms of the more than 200, ild's, whereas <unk> unique mechanism of action.
To address complex immune pathology.
And desirable safety profile may be able to disrupt standard of care.
While our primary focus is our clinical program, perhaps to put a month, we continue to leverage our intellectual property or IP estate covering domains from all 20, human trna synthetase and utilize our platform as an engine to generate new pipeline candidates.
Sanjay Shukla: Current treatment options are limited, and like sarcoidosis, they do not treat the underlying disease or improve quality of life. EFSO-Connect is a Phase II proof-of-concept study evaluating EFSO-Firmon compared to placebo in patients with SSCILD. This study, which did its first patient last quarter, is currently open for enrollment at multiple centers in the US. We're focused on generating data from this study in 2024, and we expect to provide an update on the study later this year. We estimate that the two indications that comprise our current clinical program for apsophenamide, OMICS Argyrosis, and SSE ILD collectively represent a potential $2-3 billion global market opportunity. This does not include any upside potential in other forms of the more than 200 ILDs, where FSOFIDMOB's unique mechanism of action to address complex immune pathology and desirable safety profile may be able to disrupt standard of care. While our primary focus is our clinical program for epsifidamone, we continue to leverage our intellectual property, or IP, at State, covering domains from all 20 human tRNA synthetases and utilize DNA synthetases are ancient essential proteins that have evolved novel domains to regulate diverse pathways extracellularly in humans.
Trna synthetase or ancient essential proteins that have evolved novel domains to regulate diverse pathways extracellularly and humans.
By identifying extra cellular receptors signaling pathways for these domains.
Can elucidate the role these proteins play in cellular response and explore disease areas, where they may have therapeutic benefit.
Our two most advanced trna synthetase candidates, a preclinical development or <unk> of 101 and <unk> 750.
Both of which have specific interactions with targets.
The implications and fibrosis.
These targets include latent transforming growth factor beta binding protein, one or <unk>, one and.
And fiberglass growth factor receptor for or F. G. F. G F R four respectively.
If you are a 101, which is derived from a domain of deteriorate synthetase dark.
And in fact fibrotic effects by selectively inducing.
Doses of mile fiberglass targeting.
Targeting a key hallmark of fibrosis pathology.
Which is the persistence of activated micro fiber glass.
This mechanism may support broad therapeutic applications in indications like lung liver and kidney fibrosis.
The hidden biology that we've been able to unlock from our platform continues to inspire us including the weigh in with some of these appended domains like absolute <unk> interact extracellularly with previously under the radar targets like <unk> and in particular.
Sanjay Shukla: By identifying extracellular receptors and signaling pathways for these domains, we can elucidate the role these proteins play in cellular responses and explore disease areas where they may have therapeutic benefits. Our two most advanced tRNA synthetase candidates in preclinical development are ATYR0101 and ATYR0750, both of which have specific interactions with targets that have implications for fibrosis. These targets include Latent Transforming Growth Factor Beta Binding Protein 1, or LTBP1, and Fibroblast Growth Factor Receptor 4, or FGFR4, respectively. ATYR-0101, which is derived from a domain of the tRNA synthetase DARS, exerts its antifibrotic effects by selectively inducing apoptosis of myofibroblasts, targeting a key hallmark of fibrosis pathology, which is the persistence of activated myofibroblasts.
Its role as an immune regulator.
Or buying more well known targets in unique ways like <unk> 750.
This platform, which is based on signaling pathways that have evolved over billions of years to maintain homeostasis is an excellent example of bio innovation and has the potential to disrupt traditional drug discovery.
That is increasingly rely on on exploiting existing signaling pathways to generate mid to therapies.
Our conviction.
As to the potential of trna synthetase biology to lead to transformative medicines continues to grow stronger as our research advances.
I'll now turn it over to our Chief Financial Officer, Jill Broadfoot to review our financial results.
Thank you Sanjay.
2023, with 101 7 million in cash restricted cash cash equivalents and investments.
And in license revenue related to the clearing agreement with <unk> 4 million for the year ended 2023, which consisted of drug product material sold to Karen that the Japan question of external debt.
As a reminder, Karen is our partner for the development and commercialization of <unk> in Japan.
They are responsible for cost related to <unk>.
Jill Broadfoot: This mechanism may support broad therapeutic applications in indications like lung, liver, and kidney fibrosis. The hidden biology that we have been able to unlock from our platform continues to inspire us, including the way in which some of these appended domains, like F-sulfitamide, interact exocellularly with previously under-the-radar targets, like NRP2, and in particular, its role as an immune regulator, or buy more well-known targets in This platform, which is based on signaling pathways that have evolved over billions of years to maintain homeostasis, is an excellent example of bioinnovation and has the potential to disrupt traditional drug discovery, a process that is increasingly reliant on exploiting existing signaling pathways to generate ME2 therapy. Our conviction as to the potential of tRNA-synthetase biology to lead to transformative medicines continues to grow stronger as our research advances Thank you, Sanjay. We ended 2023 with $101.7 million in cash, restricted cash, cash equivalents, and investments.
In Japan, and purchase drug product material from that with a small market.
Under this agreement we have received $20 million in upfront and milestone payments from this partnership to date and we are eligible to receive up to an additional $155 million, which is primarily related to certain development and regulatory milestones.
Research and development expenses were $42 3 million for the year ended 2023, which consisted primarily of clinical trial with <unk>.
<unk> and <unk> connect studies manufacturing costs for the ESCO program and research and development costs.
And Matt and discovery programs.
General and administrative expenses were $13 million for the year ended 2023.
Based on our current operational finance and existing cash we maintain our prior financial guidance and believe our cash runway is expected to be sufficient to fund the company through the filing of a biologics license application or a subset of mod and pulmonary sarcoidosis.
Takes into account the continued allocation of the majority of our resources.
Our efforts.
Clinical development program, which is our main value driver for the company, while also committing to gesture Judith resources to our trna synthetase pipeline candidates to maintain an active discovery program and enhance our IPSA.
The more our forecast for cash guidance does not include any potential future milestone payments from Kieran or any proceeds that may result from additional potential partnerships or sources of non dilutive funding. So it does consider proceeds from the prudent use of our aftermarket facility.
Jill Broadfoot: Collaboration and licensed revenue related to the Kirin Agreement was $0.4 million for the year ended 2023, which consisted of drug product material sold to Kirin for the Japan portion of FSOFIT. As a reminder, Kirin is our partner for the development and commercialization of FSOFIT mods for ILD in Japan, where they are responsible for costs related to FSOFIT in Japan and purchase drug product material from us with a small markup. Under this agreement, we have received $20 million in upfront and milestone payments from this partnership to date, and we are eligible to receive up to $155 million, which is primarily related to certain development and regulatory milestones. Research and development expenses were $42.3 million for the year-ended 2023, which consisted primarily of clinical trial costs for the EFSO-Fit and EFSO-Connect studies, manufacturing costs for the EFSO-Fit-a-Mod General and administrative expenses were $13 million for the year ended 2023.
We implemented this operational plan more than a year ago, driven by our emphasis on maximizing the efficiency and adapting to prevailing macroeconomic conditions.
And continues to be an effective way to meet our primary corporate objectives relative to optimal capital utilization now.
Now I'd like to turn the call back over to Sanjay before we open it up to Q&A.
Thanks Jill.
As we look back on the past year were filled with optimism for the future.
Our scientific expertise and understanding trna biology.
His unparalleled across the industry.
We're leveraging this unique biology and harnessing ancient genetic code developed throughout the course of billions of years.
And a trillion species.
While other companies are just beginning to explore.
The untapped potential of <unk> biology.
We are leading the way with our grades of understanding the science, which we have leveraged to progress a phase III therapeutic candidates.
It's not just our understanding of the analyst frontier of trna synthetase biology that sets us apart.
That's still a fair amount represents the reality not just the concept of a potential near term meaningful Q&A based therapy that can change the lives of patients.
Jill Broadfoot: Based on our current operational plans and existing cash, we maintain our prior financial guidance and believe our cash runway is expected to be sufficient to fund the company through the filing of a biologics license application for efsofetimod and pulmonary sarcoidosis. This takes into account the continued allocation of the majority of our resources to our efsofetimod clinical development program, which is our main value driver for the company, while also committing judiciously to our tRNA synthetase pipeline candidates to maintain an active discovery program and advance our IP estate. Furthermore, our forecast for our cash guidance does not include any potential future milestone payments from Curin or any proceeds that may result from additional potential partnerships or sources of non-dilutive funding, though it does consider proceeds from the prudent use of our at-the-market facility.
We're on the cusp of a once in a generation therapy for sarcoidosis engineer.
Engineered from our natural physiology and are marching towards the channels to transform the lives of ILD patients.
So let me talk a dose this is only the starting point for how <unk> may be able to help millions of ILD patients.
As the only biotech company in phase III development for this indication.
We note the accelerating pipeline of candidates not just for sarcoidosis, but also for ILD more broadly.
Other biopharmaceutical companies, who are following our lead also see the multibillion dollar market opportunity in this space.
While these companies have yet to show proof of concept <unk> well beyond that.
With patients requesting to remain on treatment as we complete our current trial sarcoidosis due to the benefits they are experiencing.
So we believe it's just the beginning for us here at a time.
New validated targets are emerging from our <unk>.
Evolutionary intelligence or <unk> driven.
Drug discovery platform.
Jill Broadfoot: We implemented this operational plan more than a year ago, driven by our emphasis on maximizing efficiency and adapting to prevailing macroeconomic conditions, and this plan continues to be an effective way to meet our primary corporate objectives relative to optimal capital utilization. Now, I'd like to turn the call back over to Sanjay before we open it up to Q&A. Thank you, Kiel.
Alternatively, artificial intelligence or AI, driven drug discovery is truly in its infancy.
And these unproven hyped approaches do not have the advantage of our drug discovery engine.
Hey, tires, uncovering hidden functions embedded in our genetic code.
That were developed throughout evolution encompassing.
Infinite real life biological experiments and over a trillion species over billions of years.
Just think about that.
The synthetase domains, we have mapped have developed over billions of years since the beginning of the tree of life.
Sanjay Shukla: As we look back on the past year, we're filled with optimism for the future. Our scientific expertise in understanding tRNA biology is unparalleled across the industry for leveraging this unique biology and harnessing ancient genetic codes developed throughout the course of billions of years and a trillion species, while other companies are just beginning to explore the untapped potential of pRNA biology. We're leading the way with our understanding of this fire, which we have leveraged to progress a Phase III therapeutic candidate. But it's not just our understanding of the endless frontier of tRNA synthetase biology that sets us apart. Essofetamide represents the reality, not just the concept, of a potential near-term meaningful tRNA-based therapy that can change the lives of patients. We're on the cusp of a once-in-a-generation therapy for sarcoidosis, engineered from our natural physiology, and are marching toward the chance to transform the lives of ILD patients. Ulnar sarcoidosis is only the starting point for how efsafetamide may be able to help millions of ILD patients, as the only biotech company in phase 3 development for this indication. However, we note the accelerating pipeline of candidates, not just for sarcoidosis, but also for ILD more broadly.
Since the evolution of complex systems, all species, including humans share the same genetic domains.
That were created over billions of years of biological stress and strain.
<unk> all species to thrive and.
And overcome disease and dysfunction.
As companies embark on computational models and approaches.
Critical to find new and novel targets a tire. Meanwhile, has an IP library of hundreds of potentially applications proteins.
Just waiting to be unleashed from our own genetic programming.
Leveraging novel domains that have been tested and validated since the beginnings of life.
That we now look to target towards current day disease and dysfunction.
I Hope you can now better understand why.
We're still optimistic here at <unk> not only in the short term with absolutely no demand.
But also in the long term potential.
Great power to become the next transformative biotech company of our time.
We appreciate your interest at this time.
Joe and I will be happy to take your questions.
Thank you as a reminder to ask a question. Please press star one on your telephone and wait for your name to be announced to withdraw. Your question. Please press star one again, one moment for questions.
Our first question comes from Gregory <unk> with RBC capital markets. You May proceed.
Great Good afternoon, Sanjay and Jill Congrats on the progress. Thanks, Thanks for the update and thanks for taking my questions.
Sanjay maybe just going to the expanded access program, it's great to hear.
Sanjay Shukla: Other biopharmaceutical companies who are following our lead also see the multi-billion dollar market opportunity in this space. However, while these companies have yet to show proof of concept, EpsilCinemaD is well beyond that, with patients requesting to remain on treatment as they complete our current trial in sarcoidosis due to the benefits they are experiencing. So we believe it's just the beginning for us here at aTyr; new validated targets are emerging from our evolutionary intelligence or EI-driven drug discovery platform. Alternatively, AI-driven drug discovery is really in its infancy. And these unproven, hyped approaches do not have the advantage of our EI drug discovery yet; aTyr is uncovering hidden functions embedded in our genetic code that were developed throughout evolution, encompassing infinite real-life biological experiments in over a trillion species over billions of years. Just think about that. The synthetase domains that we have mapped have developed over billions of years since the beginning of the tree of life.
The color you've provided just wanted to ask if you could maybe put into context. The significance of this to you anticipate amount, especially as these blinded patients are undergoing the considerable taper floor for the trial.
And then secondly, just any other details.
You are seeing whether it's the patterns from from the centers, where that or what that demand is in order to.
To have these patients in these trial participants to continue.
With respect to that to the program and then I have a follow up as well thanks.
Sure Greg Thanks for the question so.
Really over the last I would say.
Six months, if you will.
We have been hearing I've been hearing directly from.
So I would say.
At this point dozens of <unk> around the world about how happy they are with the performance.
Although this study drug other study Jonathan will highlight say that over and over again, because we don't know if its absorption of moderate placebo and how their patients are performing quite well in the trial.
It had reached the point, however that patients now rolling off the trial.
Frankly don't want to go back on steroids.
If they have been able to to taper off or.
Or increase their steroids.
Operator: Since the evolution of complex systems, all species, including humans, share these same genetic domains that were created over billions of years of biological stress and strain, allowing all species to thrive and overcome disease and dysfunction. As companies embark on computational models and approaches to quote unquote find new and novel targets aTyr, meanwhile, has an IP library of hundreds of potentially efficacious proteins just waiting to be unleashed from our own genetic programming, leveraging novel domains that have been tested and validated since the beginnings of life and that we now look to target toward current day disease and dysfunction. I hope you can now better understand why we're so optimistic here at aTyr, not only in the short term with EpsilCodamon but also in the long-term potential to become the next transformative biotech company of our time. We appreciate your interest at this time. Jill and I will be happy to take your questions. Thank you. As a reminder, to ask a question, please press star 1 1 on your telephone and wait for your name to be announced. To withdraw your question, please press star 1 1 again.
Many of these patients have been on steroids for 510, sometimes 20 years. So this is off a day or <unk>.
Real unique.
Opportunity.
In this trial to live their lives with a lot less or no steroids. So.
They've gotten to the point, where I thought.
It's best for us to even think about EMEA AP really early on.
Most of these programs are put in place after UN blind and you finished the trial.
But with data.
Obviously, we're looking at data next year.
We wanted to be able to really fulfill our mission to really be patient oriented patient first.
And look to implement this program.
Since we've put out the note I think it's been received even even with more interest.
This is certainly something that is a relief to a lot of these patients.
And I do think that.
It's a typical for a company to.
To do this sort of thing ahead of data release.
I would much rather have patients wanting to remain in the trial.
Happy to get out of the truck put it that way so I think.
For me clinically.
Think about this clinically I think we're doing the right thing here ethically as well and I think it's a.
It's probably the best biomarker you could ask for.
In my opinion.
Yeah. That's helpful. I appreciate the color and then just maybe shifting to connect just thinking about that.
Gregory Renzo: One moment for questions. Our first question comes from Gregory Renzo with RBC Capital Markets. He may proceed. Great, good afternoon. Progress, maybe just going, and I just wanted to ask. I'll, and then. Any other details?
That trial open for enrollment across these multiple centers and I think I heard you mentioned.
Date should come later this year, just with respect to that uptick, but what is your objective. There of course, we're all interested in that.
In the 12 week scan assessment as well, but just curious how we should be thinking about that is it more procedural nature or is it something where I'd like to at least look and see some details of that program. Thanks again and congrats.
Sanjay Shukla: that. Greg, thanks for the question. So really, over the last few weeks, I would say, you know, at this point, dozens of PIs around the world have said how happy they are with the performance of the study drug. I'm going to highlight say that over and over again because we don't know if it's F-sulfidamide or placebo and how their patients are performing quite well in the trial. It has reached the point, however, that patients now rolling off the trial Frankly don't want to go back on steroids, you know, if they have been able to taper off or increase their steroids. Many of these patients have been on steroids for 5, 10, sometimes 20 years, so this has offered a real unique opportunity in this trial to live their lives with a lot less or no steroids. It had gotten to the point where I thought...
Sure so.
We're just in that phase now, where I think you'll you'll see.
Six 810 centers up and.
And ready to enroll at this point getting that first patient in last quarter was important but now we're getting into the meat of.
These sites being able to enroll and like I said, we will have a good idea later this year around what kind of data we will put out here there are opportunities with that dataset to look at things.
Even a three month end point, which is some of the skin.
Skin, scoring that you pointed out there.
Like I said I wanted to see how we do here in the first half of the year with regards to enrolling in that trial now that we are approaching.
And more or less.
Activation or nearing full activation of the centers that we want to get up.
Enrolling in screening for that trial.
Got it thanks again.
Thanks, Thank you.
Sanjay Shukla: It's best for us to even think about an EAP really early on, because most of these programs are put in place after you unblind and you finish the trial. But, you know, with data. Obviously, we're looking at data next year. We wanted to be able to really fulfill our mission to really be patient-oriented, patient-first, and look to implement this program. Since we've put out the note, I think it's been received even with more interest. This is certainly something that is a relief to a lot of these patients, and I do think that it's atypical for a company to do this sort of thing ahead of data release. I would much rather have patients wanting to remain in the trial and happy to get out of the trial, put it that way. So I think for me clinically, when I think about this clinically, I think we're doing the right thing here ethically as well. And I think it's probably the best biomarker you could ask for, in my opinion. That's helpful; I appreciate the color.
One moment for questions.
Our next question comes from Joe <unk> with H C. Wainwright you May proceed.
Hey, everybody good afternoon, thanks for taking the questions.
So I just wanted to start more start first two also with the EAP. So.
Pretty intriguing like you said to be able to have an EAP ahead of data and if I heard you correctly, both physicians and patients remain blinded even after.
Exiting so fit as to what they had been previously on so first.
Was curious if you could provide any more color.
Or anecdotes that the physicians are giving you with regard to wanting to be into the EAP you alluded to.
Maybe quote that they were patients are feeling better but are there any more details you can share around that.
What are the general logistics of setting up an EAP and related to that what are the cost implications.
Yes.
Great question, Joe So I think just at a high level.
Without sort of getting into specific endpoints, what it really comes down to us.
Sanjay Shukla: And then maybe shifting to, that trial open for enrollment across, make it hurt you, date that update, what is your objective there? Sure, so we're just in that phase now where I think you'll see 6, 8, 10 centers up and ready to enroll at this point. Getting that first patient in last quarter was important, but now we're getting into the meat of, you know, these sites being able to enroll. And like I said, we'll have a good idea later this year around what kind of data we'll put out here. There are opportunities with that dataset to look at things, even a three-month endpoint, which is some of the skin scoring that you pointed out there. But like I said, I want to see how we do here in the first half of the year with regard to enrolling in that trial now that we are approaching more or less full activation or nearing full activation of the centers that we want to get up and running and enrolling and screening for that trial. Thanks.
These physicians.
Have to put these patients back on something.
Which was presumably steroids as they entered the trial and now the patients are refusing to.
To want to do that so there is it speaks to the lack of options that we have here for circuit doses. These patients of course are not.
So fibrotic that they need the anti fibrotic.
So now there is a quandary patients.
Patients are doing well and they tasted kind of the best.
Experience.
Recently.
And they're in their recent cure.
Whether it's removing steroids.
Partially or completely.
So I think.
We have to sort of step up here.
Many of them many of the experts, we're we're sort of in a quandary now.
So I think it speaks to the fact that as I said. This is this is a really strong biomarker in my mind to have this kind of demand.
From centers and from patients.
With regards to how would this logistically be administered.
Here in the U S.
Typically as a direct submission from the Pi I would think of this as more like classical compassionate use.
Operator: Thank you. One moment for questions. Our next question comes from Joe Panguinis with H.G. Wainwright. You may proceed. Good afternoon, everybody. Good afternoon. Sanjay, I want to start more, start first also with the...
As opposed to the some of the EAP, we know of.
When drugs are approved and we're waiting for them to get to the market. So this would be an individual.
At their center cross referencing RMB and some bidding.
The FTA.
Because we have some of those positive.
Risk evaluations from <unk> perspective.
Joe Panguinis: It's pretty intriguing, like you said, to be able to have an EAP ahead of date. I hope I heard you correctly, but after first, I was curious if you could provide any more color or anecdotes, giving you with regard to better, aTyr Pharma setting. Subs by www.zeoranger.co.uk, What are the causes?
We feel as though we're also in good shape.
To support this.
This vertical.
We may provide.
Small grants to kind of help patients with some of their travel or maybe even some infusion costs, that's going to be on a case by case level.
Sanjay Shukla: Yeah, great questions, Joe. So, I think just at a high level, without sort of getting into specific endpoints, what it really comes down to is that these physicians have to put these patients back on something, which was presumably steroids, you know, as they entered the trial. And now the patients are refusing to want to do that. So it speaks to the lack of options that we have here for sarcoidosis. These patients, of course, are not so fibrotic that they need antifibrotics. So now there's a quandary.
But this will be something that the centers themselves will bear a little bit more of the financial burden. If you will will provide drug for free and we're in good shape from a drug supply perspective, but I do not expect nor should any.
The investors expect that this will materially impact.
Really our cash position, we have the drug we have drug supply will help out with some administrative hub.
Templates and things like that and maybe perhaps some small stipend to help patients when you look at Europe.
Look at other places around the world, that's going to be really a little bit more specific each country. For example in Europe. Some of them have more formal pathways that are more streamlined similar to here in the U S.
Sanjay Shukla: Patients are doing well, and they've had kind of the best experience recently in their recent care, whether it's removing steroids partially or completely, so I think. We have to sort of step up here because many of the experts are sort of in a quandary now. So I think it speaks to the fact that, as I said, this is a really strong biomarker in my mind to have this kind of demand from centers and from patients. With regard to how this would logistically be administered, here in the U.S., it typically is a direct submission from the PI.
Other other regions, we're working more closely with individual hospitals and.
And determining how best to.
Provide drug.
To those patients that want it so.
It's.
It's an atypical program, but I also think it speaks to our commitment.
To really respond to this demand from patients in <unk>.
Very very helpful and then.
Yes, when you consider being in a pivotal study right now that will go into call. It roughly mid next year and the phase II <unk> study.
Sanjay Shukla: I would think of this as more like classical compassionate use as opposed to some of the EAPs we know of when drugs are approved, and we're waiting for them to get to the market. So this would be an individual PI at their center cross-referencing and submitting to the FDA. Because we have some of those positive risk evaluations from a DSMB perspective, we feel as though we're also in good shape to support this vertical. And we may provide, you know, small grants to kind of help patients with some of their travel or maybe even some infusion costs. That's going to be on a case-by-case basis, but this will be something that the centers themselves will bear a little bit more of the financial burden, if you will.
How would you sort of characterize at this moment.
Our manufacturing needs to get beyond that and to potential commercialization, perhaps though.
Yes, so we had transitioned a few years ago.
A more commercially.
Oriented and scalable type of partnership.
So we're in really good shape here to essentially have drug manufactured clear all the hurdles also with the FDA to support a launch.
With some of those initial batch.
Batches and runs more or less mapped out here.
Okay.
Of course, there is.
If you go out five five years seven years from now I wouldn't say, we have we have that type of capacity, but in the short term here to get through our clinical program and the early commercialization work.
Sanjay Shukla: We'll provide drugs for free, and we're in good shape from a drug supply perspective. But I do not expect, nor should any of the investors expect, that this will materially impact our cash position. We have the drug. We have the drug supply. We'll help out with some administrative templates and things like that, and maybe perhaps some small stipends to help patients. But when you look at Europe, and look at other places around the world, that's going to be a little bit more specific. Each country, for example, in Europe, some of them have more formal pathways that are more streamlined, similar to here in the U.S. and other, you know, other regions.
We're in good shape here.
And we're also fortunate that.
The manufacturers here in the U S. So we have good control. Good line of sight I know things are happening worldwide with manufacturers right now where.
There's a lot of controversy if it's U S based but we are de risked there as well by having a good manufacturing plan that we also spend a lot of time talking to regulators with to make sure. They want to make sure that we have that drug product ready for patients because theyre going to there's going to be a.
A high amount of of demand assuming this drug gets approved these patients have been as I said waiting for a therapy their whole lives.
Sanjay Shukla: We're working, you know, more closely with individual hospitals in determining how best to provide this drug to those patients that want it. It's an atypical program, but I also think it speaks to our commitment to really respond to this demand from patients and PIs. Very, very helpful, and then uh... when you consider you know being in a pivotal study right now, all, uh... how would you sort of characterize it as manufacturing? Get beyond, Yeah, so we had transitioned a few years ago to a more commercially oriented and So we're in really good shape here to essentially have the drug manufactured, and clear all the hurdles also with the FDA to support a launch with some of those initial batches and runs, you know, more or less mapped out here.
Well that five to seven year comment would be a great problem to have but thanks for all the comments Sanjay.
Sure.
Thank you.
One moment for questions.
Our next question comes from Yasmin Rahimi with Piper Sandler you May proceed.
Hey, Jim This is Jim John for you guys and congrats on the continued progress and thanks for taking my questions.
As Joe said.
So any additional guidance to bring enrollment to the finish line and is there actually an opportunity for additional enrollment beyond the 264 patients.
And second could you provide more color on the percentage or number of patients continuing from associate to the EAP program.
Yes. So 264 is kind of our goal that's a lot of that is based on our power calculations it assumes that.
That you can have a nominal.
Sanjay Shukla: But, you know, of course, if you go out five, five, seven years from now, I wouldn't say we have that type of capacity, but in the short term, to get through our clinical program and the early commercialization work, we're in good shape here. And we're also fortunate that, you know, the manufacturers here in the US, so we have good control, and good line of sight.
Dropout rate.
So we're well powered.
Actually overpowered in many ways compared to other phase III trials.
92% powered here.
264 is.
As our goal, we still view that as.
A solid number there.
It allows for also.
A buffer that if you had 10% of the patients discontinue for any reason you would still be able to hit your power calculation. So we're.
Sanjay Shukla: I know things are happening worldwide with manufacturers right now where there's a lot of controversy if it's not US-based, but we're de-risked there as well by having a good manufacturing plan that we also spend a lot of time talking to regulators about to make sure they want to make sure that we have that drug product ready for patients, because there's going to be a high amount of demand, assuming this drug gets approved These patients have been, as I said, waiting for therapy their whole lives. 5 to 7, a problem to have, but thanks for all.
Still set with that number sometimes you can see trials that might be a few.
New patients over a few patients under a lot of that has to do with patient screening right. There at the yen and our last trial, which was the 36 patient trial, we had eligible patients the day, we stop screening.
We ended up having a few more patients or one more patient enrollment of that trial, because they were screen screw.
Screen eligible and we don't want to sort of shut down enrollment if someone is.
As already screen and is eligible.
So I would say that we're still going to be right in that ballpark there.
Sanjay Shukla: Sure. Thank you. One moment for questions. Our next question comes from Yasmin Rahimi with Piper Sandler. You may proceed. Hey team, this is Junggu on behalf of Yaz.
<unk> hundred 64 with regards to how many are going to move into the EAP.
We don't know yet.
We are continuing to generate requests as you can imagine as more and more patients finish the trial.
Junggu: Congratulations on the continued progress and thanks for taking our questions. For SOFIT, do you still need additional sites to bring enrollment to the finish line? And second, could you provide more color on the percentage or number of patients continuing from FSOFIT to the EAP program? Yeah, so 264 is kind of our goal, and a lot of that is based on our power calculations. It assumes that you can't have a nominal dropout rate. So we're well powered, actually overpowered in many ways compared to other phase three trials. 92% of the power is generated here.
We may get more patients interested my early read on it is.
We are well built here to have.
A substantial number of patients if they want the drug we're ready we're ready to provide it.
But I don't have an exact number.
But we are prepared.
Potentially.
Give the EAP program, how does therefore.
Very very large number of patients will have to wait and see what kind of demand. It continues early on right now I'll say since we put the announcement.
We probably had even more demand.
Since we put the NFL announcement out.
Thank you so much for the color.
Thank you.
One moment for questions.
Our next question comes from Yale Jen with Laidlaw <unk> co you May proceed.
Good afternoon, and thanks for taking the questions.
Sanjay Shukla: 264 is our goal. We still view that as a solid number there. It also allows for a buffer that if you had 10% of the patients discontinue for any reason, you'd still be able to hit your power calculation. So we're still set with that number. Sometimes you can see trials that might be a few patients over, a few patients under. A lot of that has to do with patient screening right there at the end in our last trial, which was the 36 patient trial. We had eligible patients the day we stopped screening. We ended up having a few more patients, one more patient, enrolled in that trial because they were screened, and were screening eligible, and we don't want to sort of shut down enrollment if someone is already screened and is eligible. So I would say that we're still going to be right in that ballpark at 264.
I came in late so maybe you can reiterate a little bit in terms of the enrollment.
Enrollment I know the press release indicated.
Is on track.
Was there any other colors of that didn't have another questions.
No very much on track.
Very much feeling good.
About Q2 this year, so very much on track.
Okay, Great Thats, good and you.
You mentioned that.
Two preclinical programs and David.
Interesting.
Juncture was those.
The programs that you intend to partner out at some point or what will be the path you are thinking about moving forward with those.
Preclinical programs.
Well I think it's I mean.
Everything is a biotech.
Kind of where we are you're always you always have to look at partnering in those sorts of opportunities. There's no shortage of interest obviously, we have.
Sanjay Shukla: With regard to how many are going to move into the EAP, we don't know yet, but we are continuing to generate requests, as you can imagine, as more and more patients finish the trial. We may get, you know, more patients interested. My early read on it is, You know, we're well built here to have a substantial number of patients, if they want the drug, we're ready, we're ready to provide it. But I don't have an exact number, but we are prepared, you know, to potentially give the AP program and have it available for, you know, a very, very large number of patients. We'll have to wait and see what kind of demand continues. Early on, right now, I'll say since we made the announcement, we probably have had even more demand since we put the announcement out. I thank you so much.
Rather novel platform here and the platform now is generating or or validated opportunities.
So that will be something that we'll look at we always look at this.
Potential arrangements at the same time, we also are potentially sitting on assets here that.
Really are producing signals really never seen before when you talk about being able to induce apoptotic this amount of fiber glass.
Don't think Youll hear too many companies.
Really seeing those kinds of early signals. So that's where this biology I think it's important for everyone to understand theres hidden, but theres hidden potential here theres hidden potential efficacy here.
Operator: Thank you. One moment for questions. Our next question comes from Yale Jin with Laidlaw & Co. You may proceed. Good afternoon, and thanks for taking the question.
And I think what <unk> is doing is really looking at these genetic codes, saying, where it can be best be applied where there is disease and dysfunction.
No.
Unlike generations of Mi two therapies that have been out there that are not disease modifying I think all of our approaches <unk> and even some of the pipeline approaches we have here are.
Yale Jin: Uh, just, I came in late, so... reiterate a little bit in terms of EPSIL FIT enrollment. The press release indicated it's on track. Was there any other color of that?
Yale Jin: Didn't have enough. No, Yale, very much on track, very much feeling good about Q2 this year, so very much on track. Okay, great. That's good. And you mentioned a few, two preclinical programs, and they look very interesting at this juncture. Are those the programs you intend to partner out at some point, or what will be?
We have a very very high high bar here they have to be disease modifying they have to really be showing act.
Activity here that has two standard deviations better.
Then most opportunities out there.
And I think we want to be careful about.
Yes.
Giving away these gems.
Too early but.
We are open to talk to.
Big players at all times.
Sanjay Shukla: path you are thinking about moving forward with those preclinical programs. Well, I think it's a, I mean, for everything as a biotech, like, you know, kind of where we are, you always, you always have to look at partnering and those sorts of opportunities; there's no shortage of interest. Obviously, we have, you know, a rather novel platform here.
Okay, Great and maybe just sneaking in one more question here, which is that the you said the trial is on track to complete to complete enrollment.
Second quarter.
Just curious in terms of the kirin in Japan, given they're smaller.
Have they already completed any colors on that.
Yes.
Yes.
Can't really get into.
Who is completed.
More or less as competitive enrollment there arent hard caps per se.
Sanjay Shukla: And the platform now is generating more and more validated opportunities, so that will be something that we'll look at. We always look at these sorts of potential arrangements.
Country by country, we have our goals on what we want where we want to get too.
So so I wouldn't think of it as a separate trial.
Sanjay Shukla: At the same time, we also are potentially, you know, sitting on assets here that really are producing signals never seen before. When you talk about being able to induce apoptosis in myofibroblasts, I don't think you'll hear too many companies really seeing those kinds of early signals. So that's why this biology, I think, it's important for everyone to understand.
We've said in the past that.
Regions like Japan, you might expect 25% to 30 patients there so.
So Japan like the rest of the world.
Working in right now are very much on track.
To meet our projections to get this trial wrapped up here in Q2.
Enrollment.
Okay, great. Thanks, a lot and congrats on all the progress.
Thank you Yale.
Sanjay Shukla: There's hidden potential here; there's hidden potential efficacy here. And I think what aTyr is doing is really looking at these genetic codes and saying where they can best be applied where there is disease and dysfunction. So, unlike, you know, generations of Me Too therapies that have been out there that are not disease modifying, I think all of our approaches, Epsilfitimod, and even some of the pipeline approaches we have here are, you know, we have a very, very high bar here. They have to be disease modifying, and they have to really be showing activity here that is two standard deviations better than most opportunities out there.
Thank you and as a reminder to ask a question. Please press star one on your telephone and wait for your name to be announced.
One moment for questions.
Our next question comes from Robert Leboyer with Noble capital markets. You May proceed.
Good afternoon.
Gratulation on all the progress.
I also have a question on the expanded access program and was wondering whether you are going to be compiling endpoints and other data that can be used in the BLA.
Or whether you can present some of the extension study data.
At medical meetings either before.
<unk>.
The data is announced or afterwards.
Sanjay Shukla: And I think we want to be careful about, you know, giving away these gems too early, but we're open to talking to big players at all times. Okay, great. Maybe just speaking one more question here, which is that, as you said, the trial is on track to complete enrollment in the second quarter, and just curious in terms of happening in Japan given they have a smaller site. Have they already completed any colors on that?
Yes, it's a great question Robert So just wanted to highlight one thing here.
This is being conducted outside of our protocol and that's been done for a few reasons number one we want to hit our BLA timelines and submit finished the primary.
Trial, if you will the primary portion of the trial and submit that the secondary piece of the trial is sitting outside of our protocol.
Why are we doing it that way well first of all health authorities didn't mandate that we do any kind of open label extension. There was no known safety risks observed early in the program and our last program.
Sanjay Shukla: Yeah, I can't really get into who's completed or not. I mean, it more or less is competitive enrollment. There aren't hard caps per se, country by country.
And we had long term.
Safety data already from.
Sanjay Shukla: We have our goals for what we want, where we want to get to, so I wouldn't think of it as a separate trial. We've said in the past that in a region like Japan, you might expect 25 to 30 patients there.
From this trial. So there was really no need to have a a more formal open label extension or anything like that so this trial will be done outside of the boundaries of our protocol now.
Already I would say.
Many academics are really interested in looking at patients 18 24 months out.
Sanjay Shukla: So Japan, like the rest of the world that we're working in right now, is very much on track to meet our projections to get this trial wrapped up here in Q2, at least enrollment. Okay, great. Thanks a lot and congratulations on all the problems.
Perhaps with or without <unk>.
Absolute amount long term.
So I can't say that there would not be an academic consortium of some of our experts that would want to put out this data.
Yale Jin: Thank you, Gail. Thank you. And as a reminder, to ask a question, please press star one on your telephone and wait for your name to be announced. One moment for a question. Our next question comes from Robert Laboyer with Noble Capital Markets. He may proceed. Good afternoon, and congratulations on all the progress.
Support that.
They can look at different things that within the confines of our own protocol, we haven't been able to look at.
To look at long term.
Steroid reduction effects.
Some of them some of the early promising things, we see with regards to.
Things like weight loss.
It was always theoretical you get off steroids, you could help with weight.
Operator: I also have a Bandit Act and was wondering whether you're going to be compiling endpoints and other data that can be used in the BLA or whether you can present the extension, study data, at medical meetings either before the day that is announced or after. Yeah, it's a great question, Robert. So what I just want to highlight one thing here is that this is being conducted outside of our protocol, and that's being done for a few reasons. Number one, we want to hit our BLA timelines and finish the primary trial, if you will, the primary portion of the trial, and submit that. This secondary piece of the trial is sitting outside of our protocol. Why are we doing it that way?
We're seeing some.
Rather rather.
Remarkable signals there. It's early it's anecdotal this might be something that we could look at those investigators could look at.
In some sort of academic survey they may want to look at some sort of imaging data two or three years later, but right now we're focused on getting this drug approved getting the BLA out those trick.
Trickle of that kind of interesting data could almost be a head start to phase four.
The way I look at it we also don't want to spend money on that sort of downstream side of the fence right now, but I think theres going to be some intriguing.
Robert Laboyer: Well, first of all, health authorities didn't mandate that we do any kind of open label extension. There were no known safety risks observed early in the program, in our last program, and we had long-term safety data already from this trial. So there was really no need to have a more formal open label extension or anything like that.
Potential there to collect data.
Not only for the patients that continue in the trial, but also those that.
Might have to revert back to.
Steroids.
And see what happens with those patients but.
I think it's a great question, Robert really nice how youre thinking about that and it's already things that experts are are noodling over.
Sanjay Shukla: So this trial will be done outside of the boundaries of our protocol. Now, already, I would say, many academics are really interested in looking at patients 18-24 months out, perhaps with or without F-sulfidamide long term. So, I can't say that there would not be an academic consortium of some of our experts that would want to put out this data. But I would support that. I think they could look at different things that, within the confines of our own protocol, we haven't been able to look at, to look at long-term, you know, steroid reduction effects. You know, some of the early promising things we see with regard to things like weight loss, you know, it was always theoretical: you get off steroids, you can help with weight. You know, we're seeing some, you know, rather, you know, remarkable signals there. It's early, it's anecdotal.
Potentially do something outside of the trial.
Okay, great. Thank you very much.
Thank you I would now like to turn the call back over to Sanjay for any closing remarks.
Great well, we thank everybody interest great questions today, I know theres a lot of expectation it was really looking forward to.
Getting this trial.
Wrapped up here, we are two <unk>.
Appreciate everyone's interest here. Thank you for following us and look forward to talking to you in the future. Thanks again.
Thank you for your participation you may now disconnect.
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Sanjay Shukla: This might be something that we could look at, those investigators could look at in some sort of academic survey. They may want to look at some sort of imaging data two or three years later. But right now, we're focused on getting this drug approved, getting the BLA out. A trickle of that kind of interesting data could almost be a head start to phase four. That's the way I look at it. We also don't want to spend money on that sort of, you know, downstream side of the fence right now.
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Sanjay Shukla: But I think there's going to be some intriguing potential there to collect data, not only for the patients that continue in the trial but also for those that might have to revert back to steroids and see what happens with those patients. But I think it's a great question, Robert. Really nice how you're thinking about that. And it's already things that, you know, experts are noodling over potentially doing something outside of the trial.
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Sanjay Shukla: Okay, great. Thank you very much. Thank you. I would now like to turn the call back over to Sanjay for any closing remarks. Great. Well, we thank everybody for their interest. Great questions today.
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Sanjay Shukla: I know there's a lot of expectation and everyone's really looking forward to getting this trial, you know, wrapped up here. We should appreciate everyone's interest here. Thank you for following us, and I look forward to talking to you in the future. Thanks again. Thank you for your participation. You may now disconnect. Everyone, thanks for watching! Thank you for watching!
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