Q2 2021 aTyr Pharma Inc Earnings Call
[music].
Good afternoon, ladies and gentlemen, and welcome to the I T tire pharma second quarter 2 it sounds from 'twenty 1 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 the session you will need the press star 1 on your telephone.
If you require any further assistance please press star zero.
The reminder, this conference is being recorded for replay purposes. It is now my pleasure to hand, the conference call over to Ashleigh Dunston, 8 tires director of Investor Relations and corporate Communications. Mr. Johnson you may begin.
Thank you operator, and good afternoon, everyone. Thank you for joining us today to discuss a tire of second quarter 2021 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.
The including our clinical program for a T Y our 1923, and our research and discovery programs.
The 2 including a preclinical program for a T Y R 28 pump Jill will review the financial results and our current financial positioning before handing it 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 day.
These statements involve risks and uncertainties that can cause actual results to differ materially from those of such forward looking statements.
Please see the 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, and quarterly reports on form 10-Q.
Undue reliance should not be placed on forward looking statements, which speak only as of the date. They are made a facts and circumstances underlying these forward looking statements may change expected, except as required by law a tire pharma disclaims any obligation to update these forward looking statements to reflect future information events or circumstances I will now turn the call.
Call over to Sanjay.
Thank you Ashley.
Good afternoon, everyone and thank you for joining us for our second quarter 2021 results conference call.
As we make our way through 2021, we continue to make significant progress in our mission to translate a novel biological pathways and the innovative therapeutics.
Improved outcomes for patients.
Notably, we recently completed the last subject in our phase 1 of the 2 a proof of concept study of our lead therapeutic candidate <unk> 1923 or $19.23.
Pulmonary sarcoidosis are lead interstitial lung disease or ILD indication.
We expect to report results from this important study in mid September of this year.
This is a significant milestone for <unk> and the upcoming readout represents the key inflection point for 1923 clinical program.
And trna synthetase biology platform.
The clinical proof of mechanism for 1923 established from our Phase III study in COVID-19 patients and the favorable safety profile demonstrated to date, along with the preclinical efficacy observed in multiple translational ILD model support the potential for $19.23, as a new therapeutic approach for pulp.
And any sarcoidosis and possibly other forms of ILD.
We believe 1923 could potentially offer an alternate the.
The current treatments, such as steroids with improved efficacy and fewer side effects.
In addition to finishing up the important work for 19 of 23 clinical program.
We've continued to generate new data and published research related to our preclinical program further strengthening our pipeline.
As we begin today I will summarize a few additional highlights since we last spoke in may.
We hosted a key opinion leader of Kols event on current treatment options for pulmonary sarcoidosis, featuring Dr. Daniel Culver share of pulmonary medicine, and director of diffused principle of lung disease at the Cleveland clinic.
Sure in the pharmaceutical company, our partner for the development and commercialization of $19.23 for ILD in Japan.
<unk> phase 1 study in healthy Japanese volunteers.
We had 2 abstracts from 19 to 23 accepted for presentation of the upcoming European respiratory Society or IRS International Congress.
We expanded our research collaboration with the Iowa State University or OSU to deepen the understanding of the immune mechanisms of Sarcode Granuloma formation.
And identify potential biomarkers of efficacy for 1923 first of all.
Pulmonary sarcoidosis.
Dr. Elliott crowds of professor of Pulmonology critical care and sleep medicine, net OSU will serve as the principal investigator.
We received the patent grant from the U S patent and trademark office covering methods for the use of Histidyl trna synthetase or hard FC fusion proteins, which includes the use of 19 to 23.4 of reducing inflammatory response in the lung.
We appointed Dr. Sarah that go in the highly accomplished drug development and clinical research executive to our board of directors. Dr. <unk> that go into the Hematologist Oncologist, who has previously held chief Medical officer positions at several biotech company.
We presented a poster of the Keystone symposia cancer stem cells advances in biology, and clinical translation meeting related to preclinical research highlighting mechanistic insights into the tumor inhibitory effects of AP Y R 28, 10, or 28.10 of.
Our lead anti <unk>, 2 or <unk> <unk>.
Jeff antibody candidate in preclinical development for cancer.
And finally, we presented a poster at the antibody engineering and Therapeutics Europe Virtual conference related to a second anti <unk>, 2 antibody, which demonstrated the selective blocking.
Of that antibody to some of <unk> 3 F signaling.
We've made significant progress during the first half of this year and we see the second half of this year is shaping up to be as equally productive.
Let's begin with our clinical program for 1923.
We're developing 1923 as a potential treatment for patients with ILD a group of rare immune mediated disorders that can cause progressive fibrosis of alone.
There are more than 200 types of ILD.
The 80% of these patients fall into 4 main <unk> main disease categories pulmonary sarcoidosis, chronic hypersensitivity pneumonitis connective tissue disease, ILD and idiopathic pulmonary fibrosis.
All of these diseases have limited standard of care with the substantial morbidity and mortality.
19 of 23 has the potential to address this unmet need by targeting the aberrant immune responses central to ILD pathology and preventing progression of fibrosis. The key driver of poor outcomes in these patients.
We estimate there are over 500000 ILD patients in the U S alone and over 3 million patients globally.
While our initial focus for 19 of 23 is pulmonary sarcoidosis the.
The mechanism of action or MAA day.
Data from preclinical models and demonstrated effects on key inflammatory biomarkers in patients with COVID-19 pneumonia.
Suggests that 1923 could have potential in other ILD indications as well.
Our initial ILD indication for 19 of 23 is pulmonary sarcoidosis.
A hallmark disease characteristic of pulmonary sarcoidosis as the formation of granulomas or clumps of immune cells in the lungs.
The formation of these granulomas, it's driven by persistent aberrant inflammation.
If left untreated it can lead to irreversible scarring of fibrosis, and diminished lung function, which may lead the respiratory failure or the need for of lung transplant.
We estimate the patient population for pulmonary sarcoidosis to be approximately 200000 patients in the U S. Although estimate to do vary.
About half of all patients will require some form of systemic therapy.
And unfortunately, 30% of all patients will have chronic progressive disease despite available treatments.
The current standard of care for pulmonary sarcoidosis typically includes treating the inflammation with corticosteroids and other immunosuppressive therapies.
These treatments can help manage inflammation in the alleviate symptoms such as cough and shortness of breath.
They have no demonstrated efficacy on disease progression.
Can result in serious long term toxicity.
Additionally, many patients do not respond to currently available treatments.
So there is a substantial need for a safer more effective treatment that could reduce or replace the requirement for chronic corticosteroids or other immunosuppressive therapy.
This need was recently reinforced by 1 of the leading experts in the field Dr. Daniel cover at the Cleveland Clinic.
In June we hosted of Kols event with Dr. Culver, who discussed the limitations with the current standard of care and unmet medical need for treating patients with pulmonary sarcoidosis, including the toxicity burden of chronic steroid use and the need for better steroid sparing agents.
Through this event, we heard firsthand from Dr. Culver about some of the side effects related to steroid use and sarcoidosis, including some staggering statistics related to metabolic complications quality of life economic burden and risk of mortality.
Leading him to believe that there is no safe maintenance dose of corticosteroids in patients with sarcoidosis.
We were unable to attend the event I encourage you to listen to the replay which can be found on our website.
Now, let's talk a bit more of about 1923 and why we believe.
It has the potential first in class immuno modulator.
For some of the inflammatory lung diseases, we've been discussing including pulmonary sarcoidosis.
19 of 23, the novel FC fusion protein based on the naturally occurring splice variant.
Of the lung enriched trna synthetase hardest fragment that downregulates average of immune responses and inflammatory disease states.
1923 has been shown pre clinically to downregulation of inflammatory cytokines, chemo kind of signaling and reduce inflammation and fibrosis.
And our <unk> is up regulated on key immune cells known to play a role in inflammation and is enriched in inflamed lung tissue.
1923 selectively binds to an RFP to and 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.
As a reminder, our ongoing trial in pulmonary sarcoidosis as a phase <unk> randomized double blind placebo controlled multiple ascending dose clinical trial in 37 pulmonary sarcoidosis patients.
The trial consists of 3 cohorts of testing doses of 1.3 and 5 milligrams per kilogram of <unk> 1923, or placebo dose intravenously every month for 6 months the.
The primary objective of the study is to evaluate the safety and tolerability of multiple ascending doses of 1923.
Secondary objectives include assessment of the potential steroid sparing effects of 19 to 23. In addition to other exploratory assessments of efficacy such as lung imaging lung function of tests by pulmonary function tests and relevant serum biomarkers.
Based on our trial design, which includes a forced steroid taper an integral element of the study is to assess steroid burden in the 1923 treatment groups compared to placebo.
As we have discussed here a bit today and reinforced by Dr. Culver Theres, the crucial need for alternatives to existing treatment options, including steroids. We look forward to the results of this study, which we expect to report in mid September of this year.
While we have advanced our clinical program for 1923, we continue to conduct research to deepen our understanding of 19 to 23, the MLA and advance our understanding of sarcoidosis disease pathology.
We are pleased to have announced that we have 2 abstracts for 1923 that have been accepted for presentation at <unk> in September.
1 of these abstracts will present, the biomarker data from our phase II study in patients with COVID-19 pneumonia.
Earlier this year, we released some findings from this data which showed substantial anti inflammatory effects in patients consistent with findings from our animal models. This.
Of this data provides the first inpatient mechanistic proof of concept for 1923, and we look forward to sharing more details about the data at <unk>.
The second abstract will present data from a pilot proof of concept study conducted in collaboration with Dr. Elliott crowds are a leader in sarcoidosis research and treatment at OSU.
Which demonstrated the ability of a splice variant of cars the active component and portion of 19.1923 to disrupt Sarcoid granuloma formation in vitro.
Based on the successful pilot study findings, we announced just earlier today that we are expanding our research collaboration with <unk> and Dr. Krauser to continue this important work the.
The collaboration is intended to deepen our understanding of the immune mechanisms of sarcoma Granuloma formation Andi.
And identify potential biomarkers of efficacy for 19 of 23 of.
The study will assess the effect of $19.23 on Sarcoid Granuloma formation in vitro using blood samples taken from sarcoidosis patients.
We will also focus on identifying the relevant immune mechanisms triggered and granuloma formation and analyze the promising biomarkers predictive of strong granuloma formation in order to assess whether they can be use of a predictive biomarker for treatment selection for treatment response to 1923.
We're very excited to continue our work with Dr. Crowder ended <unk>.
The research generated from this collaboration May help direct us the biomarkers indicative of a population that may be sensitive to treatment with 19 of 23.
Which could present the opportunity to take a much needed step forward in managing this disease.
And lead to improved patient outcomes.
To wrap up our discussion on the $19.23, we are pleased to inform you that Huron pharmaceutical our partner for the development and commercialization of $19.23 for ILD in Japan has completed its phase 1 study, which investigated the safety pharmacokinetics or PK and Immunogenicity of 1923 known as.
<unk> dash or $1.20 in Japan, and 32 healthy Japanese volunteers. In this study 1923 was observed to be generally safe and well tolerated with no drug related serious adverse events and PK findings were consistent with previous studies of $19.23.
Before we turn to our preclinical program I wanted to take a minute to highlight an important business update that occurred in the second quarter.
In May we announced the appointment of doctor's share of that going to <unk> board of directors.
Factors that Glenn of Hematology oncologist by training is an experienced pharmaceutical drug development and clinical research executive.
It was previously held chief Medical officer positions at several biotech company.
She also has a wealth of experience working at large pharmaceutical companies, including Novartis and Schering Plough now Merck, where she was involved in supporting the development of a number of important marketed therapies include.
Including Gleevec, the Affinia ex Jade and Temodar we.
We believe Dr <unk> experience and the advancing programs at both biotech and large pharma companies is ideally situated to support and guidance retire as we prepare for the next clinical stage program to emerge from our trna synthetase biology platform.
Now I'd like to take a few minutes to discuss our preclinical program, which includes the development of the anti <unk> antibodies for cancer and inflammation.
And our <unk> is a compelling therapeutic target in the number of disease areas, including oncology and inflammation.
When it comes to cancer <unk> is up regulated on a variety of solid tumors, and it's particularly enriched in highly aggressive tumors with the expression linked to worsened patient outcomes in several cancers.
Which may include drug resistance to current therapies, such as chemotherapy or targeted agents.
And our B..2 is also highly expressed on key immune cells implicating regulating cancer progression, including tumor associated macrophages and myeloid derived suppressor cells among others.
The antibodies that can selectively block different aspects of <unk> 2 signaling pathways may of the therapeutic potential and these aggressive cancers, where an RFP to is implicated.
Our lead anti <unk> antibody in IND candidate is 28.10, a fully humanized monoclonal antibody that specifically and functionally blocks the interaction between <unk> and VEGF.
The role of NR, PTO and veg F signaling in the tumor microenvironment and its potential importance in the progression of certain aggressive cancers is becoming increasingly validated we.
We have generated a body of compelling preclinical data in both human derived and animal model of demonstrating 28 tenths of blocking ability and tumor inhibitory effects.
Notably we have continued to strengthen our mechanistic understanding of the link between <unk> and the critical process of epithelium, mesenchymoma transition or empty, which is of great importance and regulating tumor growth progression and metastatic cascade as well as being implicated in tumor of Asian.
Of the immune system.
At the recent Keystone symposia of cancer stem cells advances in biology, and clinical translation, we presented a poster demonstrating that in preclinical studies 2000, Eighteen's sensitize certain patient derived xenograft models of triple negative breast cancer to chemotherapy.
We are actively working to understand the underlying gene signatures that confer a responsiveness. These.
These findings build upon our mechanistic understanding of the 2810 and demonstrate the molecular basis for the selected selectivity by directly obstructing the verge of binding site of MRP too.
28 turns of ability to affect the E&P and cancer stem cell properties, maybe 1 mechanism by which it mediates the anti tumor effects we have observed.
This work moves us closer to identify the underlying characteristics within the tumor that may confer of responsiveness the treatment with 28%.
<unk>, enabling activities were 28% to support advancement of clinical trials in cancer in the future are ongoing.
I'll now turn it over to our Chief Financial Officer, Jill Broadfoot to review our financial results.
Thank you Sanjay cash cash equivalents and investments as of June 32021 were $44.1 million range.
Research and development expenses were $7.7 million for the second quarter of 2021, which consisted primarily of $19.23, and 28.10 program costs, including increased manufacturing costs.
General and administrative expenses were $2.8 million for the second quarter of 2021.
We do expect expenses to continue to increase throughout 2021 as research and development of $19.23, and 28 <unk> progressed.
Including additional manufacturing cost for both 1923, and 28.10 to prepare for future clinical trials.
However, we have sufficient capital to take us through the readout of our trial in pulmonary sarcoidosis and initiate a patient trial in cancer for 28.10.
Common shares outstanding were $16.9 million and fully diluted shares were $18.7 million as of August 19, 2021 now.
Now I'd like to turn the call back over to Sanjay before we open it up to Q&A.
Thank you Jill.
We're highly encouraged by what we've been able to accomplish in the first half of 2021 and.
And we are looking forward to the upcoming readout for our proof of concept study for 1923.
We've taken a very.
<unk> approach to the development for 1923.
When you look at $19.20, threes MLA, we've shown that 1923, downregulates innate and adaptive immune responses during active inflammation.
19, <unk> bind selectively to neuropil into a receptor the.
What we've shown is up regulated on key immune cells doing inflammation, including Sarcoid granulomas.
And now based on data, we will present at Urs.
We've shown that the active portion of 19 to 23 is the ability to disrupt sarcoid granuloma formation in vitro.
We've establish a library of data demonstrating 1922, each favorable clinical safety profile, which has consistently shown the 1923 of safe and generally well tolerated with no serious drug related adverse events.
This now includes data from 2 phase 1 studies in healthy volunteers the.
A phase II study in COVID-19 patients and.
And 2 independent data safety monitoring board reviews from our current study in pulmonary sarcoidosis patients.
And finally.
We've demonstrated potential efficacy, which includes a robust portfolio of translational work, demonstrating 1920, three's anti inflammatory and anti fibrotic effects in multiple preclinical models of ILD.
And clinical proof of mechanism from our phase II study in COVID-19, with biomarker data showing that 1923 reduced inflammatory cytokine levels in patients consistent with preclinical models, including cytokines that are directly implicated in sarcoidosis and other forms of ILD.
Based on the totality of this data we believe 1923 has the potential to be a transformative alternative to steroids and other available treatments with improved outcomes in patients with pulmonary sarcoidosis.
This upcoming readout represents years of hard work and dedication to developing and advancing our trna synthetase biology platform to 1 that can generate new therapeutic targets.
And from which we can develop a new class of medicines we.
We look forward to sharing the results from this important study with you in mid September.
We appreciate your interest continued support at this time, Jill and I will be happy to take your questions.
Yeah.
As a reminder to ask a question you will need to press star 1 on your telephone to withdraw your question press the county, please standby, while we compile the Q&A roster.
Your first question comes from the line of the color I've got a wall of <unk>.
Jones trading your line is open.
Hi, Thanks for taking my questions on 19, 2 and 3 I had a couple of.
On the healthy volunteer trial updates from Kieran could.
Could you expand on what has been seen in the safety database I understand there were no drug related SAE split on.
And then the diverse events, how did that compare with what has been seen with 90% or any of you see wasn't anything new.
Secondly, as we think about the September Readouts, what data will be disclosed at the time of price or the.
What exploratory efficacy endpoints, we might see.
Or what endpoints you might need to reach and then I had a follow up.
Sure Thanks per car.
With respect to our partners trial as I mentioned this trial is complete.
The 1923 tracked well.
Very consistent of what we saw in our trial.
No serious adverse events related to drug effect.
And the generally safe and well tolerated so very much consistent with the findings we saw several years ago with our phase 1 trial.
Those of the details that I can share with you.
About that trial.
With regard to your second question around the readout in September we expect to not only report the primary.
Objective, which is safety and tolerability of the trial, but we will be focusing on those.
Key.
Exploratory endpoints, namely around steroids steroid burden.
Looking at pulmonary function tests imaging.
Clamatorial Biomarkers.
So really we're looking at the total totality of all of that data.
In an attempt to really get to get it all out really at once I do not expect thinks the trickle in the after that we're looking to.
Look at all of the data here and read out all of the primary and secondary objectives of the trial.
Yeah.
Got it.
Thank you same question on the NLP to 14, the antibody that the second 1 antibody that blocks and other pizza interaction that the muscle of the Dixon.
Could you expand on how this could play a disciplined drilled on 20 of 10 and 1 of the potential next steps for this.
Thank you.
Yes. This is this is an early opportunity for US 28.10 day.
Emerged out of the suite of specific antibodies that block different.
Epitopes of <unk>.
Our 2008 to 10 blocks VEGF signaling. This is this is the next.
<unk> that we're looking at blocking <unk> signaling.
With regards to how.
Tuning Northland biology could be different it could be useful.
In some instances in cancer. We of course are seeing some effects rather early on with 2018 with regard to some of forums, there broadly implicated a bit more with inflammation.
We now want to look at how that signaling could be useful either in cancer or inflammation of.
The idea here is by tuning nor of Poland biology.
Our suite of antibodies could be useful.
Not only in cancer, but also potentially as a as a <unk>.
Even a more targeted anti inflammatory.
When you think about some of foreign biology, but again the first thing we did there was to make sure of that its a good strong blocking antibody. We have observed that now we will start to tease out a little bit more of a mechanistic understanding of that next generation antibody.
Your next question comes from the line of an umbrella of Britain Chatty of H C. Wainwright. Your line is open.
And thank you for taking my questions.
A couple of for me. So we've got the the upcoming phase <unk>. Obviously, we are excited to see the potential push the repopulating with the tire 1922.
And does the guidance do you expect this of some level of variability in patient responses based for example on the length of treatment with steroids.
And also I guess, what would be the bank that either of these value.
The ADT.
Okay. So so your question is really about duration of of prior steroid therapy.
Yes, correct.
Of note that the inclusion criteria would at least 4 weeks on staples steroid doses if I'm correct.
That's right that's right and most of these patients.
Can sometimes be on steroids for years. So this is Todd.
This will be an important.
Element for us to look at closely in our trial.
What what we understand from the experts. However is once you progressed of needing at least 10 milligrams of <unk> zone of day upwards of 25, that's really what we've enrolled in our trial you now have moderate disease.
In many ways. These patients are similar whether they receive therapy for 6 months or sometimes for several years, but it is going to be an important.
Characteristic for us to look at to determine whether or not duration of steroid.
Burden contributed to any of the responsiveness, but.
I think thats an open question.
I will say, though 1 of the things we really look to do is to eliminate folks who self resolved.
This is why we focused in on a population receiving a moderate to high doses of steroids in our trial to create a bit more homogeneity.
Amongst the the patients enrolled but that will be important for us to look at for sure.
Got it got it. Thank you that's very helpful and it was the it's nice to see and the update on coatings baseline.
The guidance that I was wondering if you can provide more color on the strategy for the development of Lafayette of 1923 with coating.
Good day.
1920 team of 2 pivotal should we expect Johnny to participate in a parallel development in subsequent doses.
Would they talk with some other of the IMD.
So the plan at this moment is for sure and as our partner.
Potentially join our next trial.
We are advancing this program forward. In addition, the results of the ability to move into other.
Interstitial lung diseases.
With Kieran.
Something that obviously.
Obviously, we will sit down and map out that strategy current has the ability to look at interstitial lung disease in Japan independently or together with us.
So I think these readouts are going to solidify a little bit more of the strategy, but at this point our aim here is to conduct.
Potentially a worldwide trial net.
<unk> in which cure and can hopefully support us with the Japanese expert in the trial sites.
Got it got it thank you very much.
Your next question comes from the line of hard such King of Oppenheimer. Your line is open.
Great. Thank you for the questions.
Looking forward to September.
Just a couple of questions from my part of my end..1 is you know when I look at the secondary outcome measures.
Sanjay.
Yes.
I will talk all of their list of the total cumulative steroid dose.
We've got some number of patients who achieve and maintain the cheaper dose.
Would you be would you consider more important is it trying to get the patient down.
So all of that targeted type of dose.
As of the 2 cumulative dose.
Also during the call. This presentation he had some.
Of the opinions there just your thoughts there and then I just have a couple of follow.
Yes, great question of our Taj I think the way to think about this is we really are looking at the steroid burden a couple of ways.
First off we want to be able to evaluate.
Whether or not folks were able to maintain of response and response being.
Being able to be on 5 milligrams or less in our trial.
That is a sub therapeutic dose.
And if the patients do well on our therapy and are able to maintain that kind of dose.
Remember these are patients that have had very.
A difficult time, even dropping 1 milligram they've sort of landed somewhere between 10% to 25 milligrams, sometimes for several years. So that's number 1 number 2 looking at the total steroid dose over time over the 6 months.
With regard to what Dr. Culver talked about this could be obviously.
Have a big impact on that sort of toxic toxic burden over time, and if we can show some differences between placebo and treated populations there.
That could also be a nice win for us So I think those of the.
The kind of ways. We're looking at this trial of course, we have the ability that <unk>.
Patient was able to even taper to zero I think Dr. Culver highlighted that that would be of real outstanding finding of who were able to do that even in the short trial.
These patients at the end of the day, 1 of get offer on their way of getting off steroids and if we're able to observe anything like that I think that would be a real tremendous finding.
With regard to some of the secondary endpoints.
<unk>.
Right now we really are looking closely at some of those inflammatory Biomarkers. In addition to some of those other endpoints that you'd see on control of Dot Gov biomarkers of become really important.
In particular after we demonstrated some nice.
The improvement in COVID-19 patients.
Yeah, No that's very helpful. Thank you.
It would be great like you said in the 24 week trial to show these effects.
The very long.
What is.
The other question of habits.
Are you assuming you had a positive readout in mid September 1 what could be the next step with regulators and the timing on that.
And then.
Association, the how important the view of whats you collaboration of looking at.
What could be of potential diagnosis from Doug.
Diagnostic strategy, the Hong Kong of adapting to the interaction with regulators and thanks for the.
Question.
Sure. So obviously, we would want to move really quick here. These are working in rare disease. These patients can't wait. So after these readouts, we would look to get aggressive.
The have an aggressive timeline to get back to the FDA.
Some of that will also involve us sitting down with the investigators of our trial.
Really mapping out their thoughts, but we would want to quickly get to the FDA. So that we could start.
A what is hopefully a.
A single pivotal trial next year again, that's pending some of that FDA dialogue, but our approach here would be that in rare disease. These patients can't wait.
And our approach would be to really move forward.
With an aggressive regulatory strategy with regard to your question around the OSU collaboration absolutely. This could be a avenue for us to develop an ex vivo assay, which can ifs.
Effectively identify.
Patient sensitive to 2008 to 1923.
Prior to even dosing.
These patients so I think as a as you point out of kind.
Kind of companion predictive.
Diagnostic this would be something that we would work towards.
And potentially used to.
At a minimum enrich our target population of patient population for the next trial. So I think it sets us up really nicely to create even more of homogeneity around those patients that respond to 1923.
Great. Thank you Sanjay.
Your next question comes from the line of Yale Jen of.
Laidlaw and company your line is open.
Good afternoon, and thanks for taking the questions.
My first question is that sort.
I'll follow up with the previous 1 which is the in terms of the Japan study versus the 30 day you have done in healthy volunteers.
With the PK differences between the GAAP niece, and I get the America or there is not much.
Out of flow.
Yes, Hi, yes, no. We saw consistent findings are asked life of our drug is somewhere between 8 or 9 days and those those PK findings tracked.
In the Japanese population as well.
So we've more or less are in the same ballpark, which is 1 of the reasons you conduct this trial.
It gets there sometimes can be differences in PK between the populations.
Okay great.
The second question is that the.
In terms of the readout in mid September.
Well Daniel you think you will communicate to the street would that be of analysts.
The investor call or other means.
Or just the press release.
We would we would plan to have a call certainly the successes.
This is an important inflection point for us. So we will certainly want to get everybody on the line.
And go through the data with each of you.
Okay, and then maybe.
2 quick ones first 1 is that the in the kind of the desktop in terms of the secondary outcome measure 1 thing called the incident in the.
Either anti <unk> antibodies.
Would you give a little more color what that day.
Sure. So as we are working with the class of of medicines coming from the trna synthetase World.
The ultra rare condition.
Called the anti synthetase syndrome.
In this in this example.
The anti synthetase patients, sometimes develop antibodies to a.
A.
Protein fragment of full length of Histidyl trna synthetase. So as we are working in the same class part of our safety is to ensure that we do not develop any autoimmunity.
Joe 1 of the antibodies are something that we're going to be required to watch for.
However, I will tell you that anti sensitive patients who develop antibodies to histidyl trna synthetase day developed interstitial lung disease. So in many ways.
Dealing with patients that already have interstitial lung disease and another way to think about this is.
Those patients that were you knock out this this.
This clinical pathway of round trna biology.
And in some ways to demonstrate proof of <unk>.
Concept, if you will that.
This is an important pathway the.
The protein fragment is involved in regulating the development of interstitial lung disease, but as we are working with this class the FDA from a safety perspective, we have to monitor Joe 1 of antibodies.
Because we are a trna synthetase platform company.
Okay, Great and maybe the last question use of housekeeping 1.
In terms of the licensing in the collaboration.
Collaboration agreement that the use.
For the remaining of 2021 do you see any potential revenue insight in this timeframe.
Or how should we think about that.
The piece of land.
Well, we we haven't really disclosed when each of those milestones are the the majority of the remaining $165 million is geared towards development, but we did see the $2 million.
Milestone from received upon the last patient in for their trial. So you can basically kind of think of that as what we've received from the phase 1 perspective. So the next milestone would.
You can think of that is coming in that next stage of development.
Which would not begin this year.
Okay, so but the lag.
Patients in the list.
So it's not the 1 you completed completing the enrollment rate.
The complete the treatment.
The already.
Yes.
Right. They have the last patient in in December of last year, and we meet the right.
And the first quarter of this year.
Okay, great. Thanks, a lot.
And thanks for taking the questions.
Your next question comes from the line of Jed The dollar of Roth Capital Partners. Your line is open.
Hi, Thanks for taking my question just have a few of the first 1.
Just on how many patients.
You plan to have by the biomarker data on COVID-19, and then there'll be presented before the pulmonary star data readout, how would you be comfortable with index, Jason Zhang from that data readout.
Sure. Thanks, Doug. So this was again we presented this data earlier this year there were 32 patients in that Covid.
19, mechanistic proof of concept trial, we ran.
Previously presented that over 80%, 82% of those inflammatory markers substantially declined when 1923 with views on on top of dexamethasone. So this is the.
This is the poster, which which essentially brings all of that data together. So there isn't exactly new information coming from that we've previously released at the the street.
We thought it would be useful and the community.
I would like to see this in a pan of medical conference. So that's really what that poster is about previously.
The highlight of this data.
The interferon gamma IL 6 MCP 1.
Significant knockdown in the Covid pneumonia patients.
These are the same biomarkers that we saw really move significantly in our animal studies. So this poster basically gets its arms around all of that information that we presented back in Q1.
And is being presented at European respiratory Society.
Which happens to be.
As it turns out.
Just.
In the short period right before we were about to announce data from sarcoidosis.
Thank you Sanjay and then just a follow up regarding the pulmonary Nissan that'll be coming out next month.
The highlights or would it be full patient by patient detail.
We'd like to be as detailed as possible. There is no topline interim we're aiming for full results being able to.
Look at not only all of the safety end points, but even those exploratory efficacy endpoints that we've highlighted.
Thank you and then just a follow up to that 1 actually was just also wondering with the small sample size of adding data points, where you would expect to see some day had already and how do you plan to tackle that 1.
With the other data points of kind of determining the goldfarb strategy.
Yes, I think Dr. Culver highlighted this that pulmonary function tests, sometimes can be perhaps the most variable in this population.
The way we look at it is we may be able to look at point of function tests and stratify based on response, we may learn something there our current trial did not.
Create a stratification or inclusion criteria around forced vital capacity.
We were advised by the experts too.
As the standpoint is pretty variable too.
Leave it alone.
I think with regards to.
Some of the lung imaging of course, that's a on validated.
Pet scans are part of our new biomarker being used out there. So we're going to learn the that's why these are exploratory endpoint I think the way the what we are really prioritizing the safety.
Looking at steroid sparing effect and looking at those Biomarkers.
Patient reported outcomes are also going to be really important here cost fatigue shortness of breath are all areas that it's per.
<unk> are doing well and we were able to also see improvement.
Knocking on their steroids, well, then I think those 2 PD markers going the same direction as Dr. Culver pointed out it could be really really impactful.
Thank you Sanjay and looking forward to the data readout.
Thank you.
There are no further questions at this time I will now turn the call over the Sanjay Shukla for closing remarks.
Great well, we thank everyone for their interest great questions today.
Looking forward to getting back to everyone.
The very soon here in the near future. So thank you everybody have a great day.
This concludes today's conference call. Thank you for participating you may now disconnect presenters. Please stay on the line.
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