Q3 2020 Aptinyx Inc Earnings Call
<unk> conference call at this time, all participants are on listen only mode. Following the formal remarks, we will open up the call for your questions. Please be advised that the call is being recorded at the company's request.
At this time I would like to turn the call over to Nick Smith Senior director of corporate development.
<unk> and Investor relations at at the Knicks Nick Please proceed.
Thank you operator, good afternoon, everyone and thank you for joining us on todays conference call to discuss after Nexus third quarter 2020 financial and operating results.
A press release, describing financial results and recent highlights is now available on.
Well upside.
Before we begin we'd like to extend a warm welcome to Chris Raymond of Piper Sandler who recently initiated coverage on that kind of after tax welcome Chris We're glad to have you join US this afternoon I'd.
On today's call Robert Riedl, our President and Chief Executive Officer will discuss our business and clinical progress then Ashish gone up our Chief Financial Officer.
And Chief Business Officer will review the financial results.
Additionally for the Q and a portion of the call. We're joined by Andy Kidd, Our Chief operating officer trafficking, our senior Vice President of clinical development and Rolando good year as senior Vice President of medical and Pharmacovigilance I'd.
I'd like to remind everyone that statements made.
Made during this conference call will include forward looking statements within the meaning of the private Securities Litigation Reform Act of 1995, which involve risks and uncertainties that can cause actual results to differ materially any forward looking statements are made only as of today and we disclaim any obligation to update these forward looking statements.
We see the forward looking.
Survey this disclaimer that financial results release issued this afternoon and the risk factors in the company's current and subsequent filings with the FTC if.
It's now my pleasure to turn the call over to Norbert.
Thank you Nick and good afternoon, everyone.
We appreciate you joining us on today's call and I hope all of you.
As well as your loved ones.
And colleagues safe and in good health.
Clearly the COVID-19 pandemic has been challenging for all of us.
On both personal and professional level throughout 2020.
Like many in our industry, our focus adept at Nics was affected.
But.
I'm pleased to convey to you that I believe our company is turning a corner.
And heading in a very positive direction with each of our development program.
In response to the pen debate, we have taken measures to support the Doolittle advancement of our pipeline.
Ensure patient safety.
And the integrity of the data from our clinical studies.
And manage our finances in other responsible manner.
All of these internal efforts and the relentless dedication of our team have collectively resulted in key positive developments across our pipeline and company.
Over the past few months.
Among the most notable of these developments was the announcement of positive results from our phase two exploratory study.
Of NYSE 73 in post traumatic stress disorder.
Which demonstrated clinically meaningful improvements in treating patients living with.
PTSD.
These results surpassed the expectations, we had for this sickness seeking study.
Validating the potential of our mechanistic approach to fixing.
The data from this study are highly informative for several reasons.
They provide us with a pass.
For advancing and why it's definitely in development for patients because pts fee.
And.
In addition.
We have now validated our novel approaches and then be able to accept a modulation in.
Please therapeutic indications.
Well my algebra.
Painfully TPN and PC.
PST.
With respect to anyone else to nine to five.
In September we are pleased to we commenced our phase Twob study opened likes to nine to five.
In patients because probably mileage, though.
In which we had previously talked enrollment due to COVID-19 related challenge.
Yes.
Thanks to our teams extensive efforts during the enrollment costs.
We are also on track to resume our phase Twob study you have in place to nine to five in painful DPN.
Before the end of the year.
And in.
In the coming months, we anticipated.
The resumption of our clinical investigation of NYSE 458 inch.
In patients with cognitive impairment.
As we bring these studies take online.
We expect to report results from each of them in Twentytwenty two.
Accordingly last month.
We bolstered our financial strength with the close of a $48 million common stock offering.
To fund our operations, who these multiple catalytic data readout.
All of this progress leaves us well positioned and equipped choice.
To responsibly advance our novel and EMEA receptor modulator.
Two with patients who may benefit from them.
Let's begin by discussing and mice to nine to five.
Our clinical stage development candidates under evaluation into chronic pain indications.
Fibromyalgia and painful diabetic peripheral neuropathy.
Or TPN.
Due to the escalation of the COVID-19 pandemic in the U.S.
We have to suspend enrollment in both of our phase Twob studies in chronic pain in late March of this year.
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Fortunately.
In September we announced that we were able to her comments.
A phase to be fibromyalgia study and.
And we are presently actively recruiting and enrolling patients.
While it is still early into the commencement pace.
Patient recruitment screening and enrollment are tracking well.
And I am confident that we have taken necessary precautions.
Our.
To ensure the safety of all those involved in the study.
Against the backdrop of the continuing COVID-19 pandemic in the U.S.
We look forward to providing future updates on this study as patient screening and enrollment progresses.
Hello.
Outside of our major study.
We are taking final steps, Chile commenced our phase Twob study of invites to nine to five.
In painful DPN.
We have taken necessary steps to mitigate the elevated risks posed by profit 19 to the DPN population.
And we have inside.
He talked at certain political changes.
Intended to streamline patient enrollment.
We are confident that the adjustments made to the study's design.
Will enable us to effectively clean enrolling new patients following the studies the initiation.
Even in the face of increasing.
This is of COVID-19.
Without any compromise to the rigor and quality of the study.
We still anticipate that this study will we start before the end of this year.
Our current expectation is that both chronic pain studies should treat out.
In the first half of Twentytwenty two.
Let's move now to end why Seveneight suite and the positive results from our phase two study in patients with post traumatic stress disorder.
The study represents the first time and like 73 was evaluated in patients with PT.
Yes, Steve.
And tested daily dosing of 10 milligrams.
And 50 milligrams of NYSE seveneight sleep against placebo.
We enrolled 153 patients in the study.
Who were evaluated over four weeks of treatment.
No patient.
We see more than four weeks of treatment and by accident its sweet.
Heading into the study we are seeking to understand where the end like 73 could demonstrate efficacy signals on the symptoms associated with PTC.
We looked at various efficacy endpoints.
With the cats, five total EPS Corp, and cement and trust us cause serving as our primary measures of efficacy.
As this was an initial exploratory study it was not powered to achieve statistical significance.
But rather to determine whether end by 73.
As a therapeutic effect in this patient population.
And to guide future development.
However, we were pleased to find that and why its 73 had clinically meaningful.
Statistically significant and mechanistically relevant effect.
On.
Numerous efficacy endpoints with only four weeks of treatment.
We observed a clear dose response.
With the 50 milligram dose performing better than the 10 milligram dose, although both demonstrated statistically significant effect compared to placebo on certain measures.
On the caps five total score we saw a clinically meaningful mean reduction, which the 50 milligram dose.
In addition.
We saw very strong results or cause responder analysis.
Showing a significantly greater proportion of patients on end like 73.
Achieving clinically meaningful improvements.
On the caps five total score.
This is Tom that analysis demonstrate that anyway, sevenx, we can provide meaningful therapeutic benefit.
For a substantial portion of these highly diverse patients.
An important factor when clinicians are making prescribing decisions.
Potentially most notably.
And why its 73 demonstrated a clinically meaningful and statistically significant improvement on the caps five of our sales entry activity score in.
Treatment arm.
This symptom trust us core evaluates many of the hallmarks of PTSD.
Including et cetera rated startle response.
Hyper vigilance.
Irritability or aggression.
Self destructive behavior and disruptions in.
Concentration in sleep.
The effects observed in this domain of symptoms aligned with our understanding of the mechanism of unlike 73, and therefore enhance our confidence in the reliability.
And real quick usability of these effects.
Hi.
Importantly.
And why is 73 exhibited a favorable safety and Tolerability profile.
Very few treatment related adverse events.
The most common and why its 73 related AG in the study was headache.
Just four cases total cost.
Stages.
Which was comparable to placebo.
No dissociative or hallucinogenic adverse events were observed.
Which further underscores the differentiated safety profile with our novel.
And then the receptor modulators.
We believe these are critical design.
As we consider the tremendous medical goodnough PTSD.
And mental health more broadly.
And we are looking forward to advancing this novel therapeutic option for patients.
Given the clear evidence of treatment effect and Tolerability observed in this study.
As well as painful.
Formation. This design provides to get the next steps in development of and why its 73.
We are now preparing for interaction with the FDA to gather feedback on the design of the next study.
It is our intention to design this study such that it could be a registration supportive.
If it's because as a positive.
We expect to get regulatory feedback in the first half of 2021.
And anticipate commencing the next study in the second half of 2021.
It is also noteworthy to highlight that along with our preview.
Studies in fibromyalgia and CPM.
This successful outcome in PTSD.
Represents the third phase two study.
That has demonstrated demonstrated the therapeutic potential of product candidates from our novel LMCA receptor modulator platform.
And supports our continued miss.
Mission of developing numerous novel therapies for CNS disorders.
Finally, let's touch on in my 458.
Which is in development for the treatment of cognitive impairment.
Following the escalation of the COVID-19 pandemic.
We suspend.
Ended our exploratory study in patients with Parkinson's disease related copies of impairment.
Based on our compelling preclinical primates data.
We are eager to get back into the clinic was and why it for.
For five days.
We are finalizing a revised study designed to evaluate and my forfeited.
Third in patients whose cognitive impairment.
And expect to be able to resume this study in the first quarter of next year.
Altogether these.
These activities of the past several months.
Further validated our technology platform and.
Accelerated momentum across.
Our clinical pipeline.
I believe we are well positioned to advance our clinical candidates across chronic pain.
PST and cognitive impairment with multiple milestones expected over the next 18 months.
With that I.
I'll now hand, the call over to Ashish to discuss out.
Third quarter financial results.
Thank you Norbert.
With respect to our third quarter Twentytwenty financials, starting with the balance sheet. We ended the quarter with $103.8 million in cash cash equivalents compared to $98.8 million on December 31, 2019.
As previously discussed this cash balance was bolstered by the $48 million in gross proceeds collected from a common stock offering in October 2020.
And we expect our current cash will fund our anticipated operations into 2023 and enable multiple phase two clinical data readouts.
Revenues for the third quarter of Twentytwenty was zero point $3 million compared to zero point $9 million for the same period in 2019.
These revenues were related to our research collaboration with Allergan.
Which is now a wholly owned subsidiary of EFI.
We do not rely.
Upon these revenues to fund our operations and consistent with the terms of the research collaboration agreement.
Joint research activities and associated payments by Allergan to after next came to their contractual conclusion.
During the third quarter.
R&D expenses were six.
$6.6 million for the third quarter Twentytwenty compared to $11.7 million for the same period in 2019.
With the Recommencement of our fibromyalgia study in.
And the plans to restart our phase two DPN study by the end of this year.
We expect to see some ramp up in R&D expenses coming quarters.
Hi.
We reported DNA expenses of $5 million for the third quarter of Twentytwenty compared to $4.5 million for the same period in 2019.
The slight increase was primarily driven by noncash stock based compensation expenses.
Our net loss for the third quarter.
But your 20 was $11.3 million compared to a net loss of $14.8 million for the same period in 2019.
With that I will turn the call back over to Mark.
Thank you Ashish.
As we prepare to turn the page to 2021.
We are excited to be moving out.
Our clinical pipeline of novel.
I didn't see it in India receptor modulators forwards across multiple indications of high unmet patient need and substantial commercial opportunity.
We will be happy to begin taking your questions now.
[noise].
As a reminder, if you like to ask a question you need to press star one on your telephone.
And your first question comes from the line of Chris Raymond with Piper Sandler.
Hi, Thanks for.
Taking the question thanks, and thanks for all the detail so just on the PTSD study.
That's commencing next year.
Detecting just a little bit of a change in the language.
I think when you guys had the data you referred to it as pivotal but.
But it looks like you're it's now describes registration supportive.
I Wonder if you could just maybe give any any color is there.
Her additional data required are you looking to fill out the package.
With with something else or is or is that just a stylists to change. Thanks.
Yeah, Great question, Chris. Thank you look we have to meet with FDA and we have to discuss the data and discuss with the design of that.
The design of the next study.
But I think we just want to clarify that the next study will be one.
That will be aligned with what's typically a registration study should look like.
Namely with respect to duration of treatment in particular as well as endpoint and so.
And that has not changed from being be communicated to our results and our next steps last time, we talked and that continues to be the plan, but I think it is clear right that we have to make sure that we have a meeting with the FDA get the appropriate feedback and guidance and then be should be off to the races. In the second half of next year.
And next study.
Great. Thank you very much.
Your next question.
Your next question comes from the line of Mark Goodman with SVB Leerink.
Hey, Mark.
Hey.
Since the two questions first.
For 58 cognitive impairment in Parkinson's seems to be a lot of companies that are early stages, well trying to figure. This opportunity out I was wondering if you could talk about some of the other mechanisms the competitive environment out there just you know with respect to what you've seen out there.
And it makes us a sick.
Sigma one that they're trying to.
Forward there.
In that area just curious what your thoughts were.
And then second.
Just mentioned on the call a little bit of a.
R&D thoughts little bit of a pick up in the fourth quarter, but trying to give us a sense of really what's going on with next year and how how much we should have R&D.
Really ramping up thanks.
Yes, so mark thanks for the questions as always.
I stopped at 458, and then see if maybe.
One of my colleagues asked for about comments on it I think we of course, all know that the population of Parkinsons patients is particularly.
Vulnerable.
To echo.
COVID-19 environment that has not in any way subsided.
And so it is our responsibility to make sure that we provide an environment.
Where we have a.
In a way of conducting that study that takes into account all the necessary precautions.
So not to expose patients on necessarily to anyways.
And to make sure the data integrity is Stephen I think we have learned from the Pts T study.
We are learning from the problem Al just studies that we have a pretty good handle on what that needs to look like which is why we have guided to in my call.
Since two Q1 as our starting point to kick that study off again.
And we are tweaking, the protocol and making necessary adjustments and changes.
Largely in line with providing that safer environment and.
I feel pretty confident that once we kick it.
It off we should be able to conducted all the way to its completion.
And we have you mentioned, what those changes I ask you basically get to the restart and.
Let you know that at the right time, Yeah. It's Andy here I think just to add there you know there is a.
A few other companies out there.
I think it is all relative.
When you say a lot.
You know it's it's.
Despite a handful and this is an area of huge unmet needs.
Really as more.
Striking that probably on there just hasn't been a lot of activity and the Parkinson cognition Harriet before.
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And ER and there's a lot of opportunity and I think an increasing recognition of how important symptoms are so you mentioned you know the Anavex Sigma one agonist, we know that and Lilly has a domain one agonist.
We know that I think stage has talked about Parkinson's and.
And also hunting them and so forth I fear and M.D.A.M.. So there's a few mechanisms out there.
They're all like you said in relatively early stages, I think thats actually in some ways refreshing to see activity in this area because there's so much unmet need and I don't think we I don't think we view two or three other programs in early stages.
As anything other than you know, it's great for patients and a great recognition of the unmet need.
And then high markets Ashish with regard to burn in R&D spend.
We've talked about our existing cash balance funding or operations, which are primarily centered on R&D.
And expenditures.
Twentytwenty, one, particularly due to the 2023 and we look at that that time period.
And the ramp up of studies that we've discussed.
Relatively speaking Twentytwenty, one we'll have to higher weight.
For for expenditures, certainly and I think my.
Substantial margin.
As we seek to complete those studies the 2022 timeframe.
You'd see that ramp down rationally.
So that's sort of how I describe the apportionment on what.
That expenditure over that period of runway.
Thanks.
Your next question comes from a line of return Baral with Cowen.
Hi, Good afternoon, guys. Thanks, Hi, Thanks, guys for taking the question I'm glad to hear you guys still well two questions for me.
One is a very simple question, but the.
Certainly the expenses.
Can you describe the geographic distribution of the trial that you will be starting and if that's changed at all from the original sales distribution that you guys envisioned for.
Three phase two.
And.
Second my follow up question is just given the vagaries of numerous psychiatric trials that we have all seen which you kind of consider running a second phase three PTSD study staggered or concurrent.
Or something that could surface I guess, we'd be post marketing study purses. Its a first the successful like just belt and suspenders approach to.
Developing pivotal data is this something you have the resources score is this something that is a priority central priority.
All right.
Thank you, we too I'm going to have Kathleen to answer your first question on geographic distribution of fulfill study sites to help cause all in the U.S., but kits and if you could provide a little bit more color that would be good.
Yes, certainly for chronic pain, we have made some adjustments across our U.S. side.
And.
In some ways trying to diversify so that we can.
Accommodate players with covenant or shutdowns or things like that across the U.S. getting the next carried up enrollment for those trials. So I think we have tried to ship to diversify across the U.S.
With respect to cognition, I think we probably won't change that geographic profile very much because were centered on.
Sites that take care of those patients and I think we'll stick with those sites as we go forward and the new trial, but again, there's enough geographic diversity there across the U.S. that we should be able to keep those trials running even in the challenges that we're facing right now with the pandemic.
And let me turn it to your second question.
It's really important for me.
<unk> point, all said, we are really really satisfied with the results because in this study.
Because it shows very clearly the effect in the life mechanistic way.
Which Kevin a clean is targeted to act primarily on fixed income.
So really the point all its the remarkable.
Well, if you qualify which is now a hallmark of.
Up to nine to five as well as seven eight clean and therefore, I believe subset from its launch.
So, let's let's see where we come out with the discussion that we are seeking with the FDA. The unmet need is of course huge.
There hasn't been a drug approved in more than 20 years.
A lot of talks that are often D indication up always.
As I entered the press and that's I think quite some symptomatic relief what phone cleanly targets the underlying net.
Mechanism that is largely a skier conditioning and therefore, our parts to basically fill extension.
Right and so we will people there and I think what will be required on the path towards commercialization or registration I should say.
And I assume that there will be an open mind to the data and EPS in particular to the fact that the unmet need is increasing and they tested.
It's definitely need for better therapies, and what's out there today, so we will see.
Great. Thanks for taking the questions.
Your next question comes from.
[noise] with Cantor Fitzgerald.
Okay, Yes.
Thanks for take Hey, Thanks for taking my questions. Congrats on a good quarter progress happy you're getting started on sales. These things Ed question on fibromyalgia.
PST and then and then PD technician on.
Fibromyalgia.
Norbert I think I think you mentioned that enrollment was starting to go well. Although it was early days can you provide any additional color on how thats going well not necessarily patient numbers, but how would how would you characterize that.
So it's going well because.
Sites basically up and running.
Hi, Scott actually correct to do this in in the environment in which we operate.
We have really made some adjustments to the protocol that we can speak to which provide further enhancement of.
That safety margin and then we look at.
So.
Cleaning and basically enrollment and while I guess, you're just pointed out don't have an actual number.
I think we on the right path Intel.
We believe at this point that step past guidance as to the first half of Twentytwenty two data readout.
Of course, we will provide as I mentioned for the updates as we come along and.
Advance the program. It is as you mentioned early stage, but so far all the parameters. We have look like they are pointing executives that I correct.
Okay. That's helpful. Norbert on quickly on PTSD.
Think follow up to a previous question I know you are going to be meeting with the agency and so these are yet to be fully defined but if you think about the primary endpoint and the duration of dosing could you provide any additional color on that I guess would you can stick with the caps.
Hi, total score or would you propose using a certain symptom domain as a primary or would that be a secondary endpoint and then in terms of duration of dosing what are you thinking.
Yes, great Chas things, so I pointed out in this clip and its really helpful to reiterate that the study with it was.
Probably a four week study.
And of course, we have and point total cap smell that sub domains of caps.
And I think it's really important to leave the rates that we did.
Separation on total caps Fife.
We did not see statistical separation, but as I mentioned it wasn't.
Pilots to be a statistical study.
So I pointed out to you only the card we are completely open to.
That being an endpoint as a primary endpoint consistent bids for the agency has been looking for up to now.
But we also pointed out that a very relevant highly relevant customer.
Operating profit mechanistic point of view and from a.
All Mark of symptoms point of view, it's clear activity and I've also where we saw highly statistically significant results in four weeks.
I think the other domains will actually show a very clear separation as well in a longer duration study.
And we also pointed out the remarkable responder rate that is a really really important measure of how many patients would actually benefit from a therapy like sales.
If we are talking about 80% of the population having at least one space of better than 30% relative to baseline I think that very very.
Thanks, and hugely helpful, especially been messaging this diverse population that we had in this study.
And so those are the key that I want to just reiterate.
The next time, we will be longer than four weeks.
We will see how much longer than four weeks, but as you know from the studies we.
I mean, we wish to nine to five we had initially a four week study that have the same purpose of highly seasonal and safety in patients.
The next study such Gulf Week studies, and so we will do the same here at PTC, we will make the study comply with what is required to ideally for pivotal design or legislation designed.
And that will all be finalized of course actually come out of the meeting with the FDA.
Okay look forward to that would love to see responder is a secondary endpoint there just quickly on PD cognition.
Interesting to see moving forward there I don't.
See there being a ton of competition, but very clear interesting mechanistic rationale. If your candidate wondering yes in terms of the Nam point would you be looking at you PD. Our asps are number one or total score or some other measure of cognition.
Improvement or stabilization in that setting.
Yes, I'm going to have and Orlando sort of like tag team on and tell you that correct.
Yeah, Charles Thanks for the good question. So we are in the process.
Obviously as we restart that study a finalized.
All of the changes we.
We I think I've talked before about using a range of different domains Pacific neuro cognitive tests to establish effectively what it what is it that the drug really does from a competition perspective and human.
Subjects, and we're going to stick with that approach. We've we've reduced the number of those down to kind of a core.
Set that are easy to administer and informative.
And we'll be able to talk more about that as we move further along with restarting the study, but the two was the most informative endpoints from an efficacy point of view will be those because those are very discriminating quite granular very targeted.
No you're right of course, theres lots of other than.
On broader instruments that can be used to assess cognition assessed function assessed broader Parkinson's symptoms and some of those will be including as additional exploratory endpoints to get a perspective of the correlation that we see with some of the newer specific neurocognitive test improvements in some of those other instruments and I.
And there was a question I think on obviously they are overwatch are possible regulatory endpoints down the line.
Ill, let rolando comment if he wants to add anything.
No I think you covered it it varies so early just to mention.
So do you pdrs.
Of course has a motor component and then the note.
Non motor company doesn't really focus on cognition. So for that there there are a number of other instrument.
Instrument many of them borrowed so frankly from the Alzheimers research areas. So for example, the civic plus.
So.
So, yes, but that should give.
It's a good sense of what the clinical effects will be of the drug.
Got it thanks, Sandy thanks Norbert.
Thank you Chuck.
Your next question comes from the line of Gary Nachman with BMO capital markets.
Good.
Hi, guys good afternoon.
Five to follow up I, just described how some of the protocols have been amended for both the Fibrocell and DPN studies to make it easier to enroll those patients during cove, its and I know you're still targeting 300 for fiber 200 for DPN.
Any possibility the numbers could be smaller if enrollment is ultimately challenging.
So I will answer the second part of your question dynamic on EPS Catherine to give you an answer to the first part.
I think it's really premature to comment on.
What should we think about patient numbers, depending on how things come out, which I think really the main reason why be Paul its wants because this is the gardening.
Bill It's both 50 p. it into a couple of miles of study on very clear and compelling results from our earlier phase two study.
And.
We have designed these trials should take almost always talk salvation.
Interesting design it into consideration and they are designed to be pivotal studies to show definitively that we get a clinically meaningful and statistically with exotic.
In the primary end point and so.
I I would be hard pressed to really compromise that study.
By cutting the number spec or buy or doing other things that were not really serve the purpose well I I hope that we will not be in an environment, where we have to make very very different.
Decisions again.
But if you ask me right off the cuff I would probably have a pause then basically castrate.
The study.
Because I think it is actually designed in the way that we believe is optimized and it would not be serving us well to start passing along with.
Yeah.
And then Catherine can you offer Gary talked of before I hand, it over to go to Catharine does that answer your question.
Yeah no absolutely. That's that's helpful. Okay, and then Kathleen can you add some color to that come off the changes we made to be adapted to weigh cobot environment.
Sure.
Okay. All right. So I think those changes kicking into category I'm personally be descriptions of our population, which in general did not change. These are still patients with TTM for at least four years, who are in pain. So that we can measure change from that baseline likewise.
Likewise in patients with fibromyalgia diagnosed at least one year ago, who has pain.
Thank measure a change from baseline.
We were able to use some of the screening and enrollment information that we gain prior to our called at par.
Just to make some I would say small clarification, so that inclusion and exclusion criteria in order to benefit enrollment going forward, so by and large that changes to the population we were speaking, but we were able.
Got to streamline some things based on what we learn before the studies one on pause.
The other group that will change is that we may have to do with that careful and systematic review of all of the procedures and visits within the trial and what we did is looked at risk to patient risk to study staff.
And were able to remove some procedures that we think post a greater risk again to staff or to subs.
Okay.
We also took an eye toward streamlining those visit so that they can be done efficiently, allowing more patients to come into the site on a day or in a week.
And also allowing room for cleaning and disinfecting things at the sites need to do in their day as well. Thanks.
I think there were specific procedure related changes that we made but then also an overall look.
Okay sure, yes, those visits and our current setting.
And again, we're getting good feedback from our sites about their ability to run. These studies under the current challenges as Robert said things are going well in the early days.
Okay. That's very helpful. And then just one other follow up I mean in your perfect World, assuming everything goes well with enrollment.
Do you think you'll report data on both of those studies at the same time, we're separately in the first half was 22, just thinking of the chronic pain indication more broadly with would you rather have both data sets in hand at the same time just to analyze how you could approach chronic pain.
Pain in general.
Where are you just going to have to release it whenever the data comes through I think you know so very good that's a great question and so yes, you pointed out we tried to couple of motor studies, a 300 patient study thats up and running.
The DPN city is a 200 patient study that is a little bit behind in new equipment.
Thanks.
I think they will read out close to each other.
And it really is.
Close means like within a month or two I don't know at this point our target of coffee is chronic pain much more broadly.
That's why we are in these two indications we have great preclinical data.
That show excellent excellent results between nine to five in chemotherapy induced pain.
We believe that osteoarthritis related joint pain, lower back pain, all qualify as chronic pain conditions for which this mechanism is directly relevant and applicable and so the past.
For what clearly is the goal of a board chronic pain label four to nine to five.
Of which the two ongoing studies are basically examples, but it does not need to be and should not be limited to believing that thats. All we are seeking between nine to five.
And we will.
Make our decisions of course based on the data as they come in and ask the results Guide US just like we have been doing up to now.
Okay, great. Thank you.
Thank you.
Your next question comes from the line of Jim Lee with the truest.
Security.
Hi, Thanks, Hi, Thanks for taking the question so the updates.
I have a question on the the results you saw in the states to part of the PTSD study.
The way I understand the point of doing a sequential parallel compare to design suppressed a placebo response in stage.
Two and to better reflect the drugs efficacy and what I find interesting is that there seems to be an inverse U shaped or actually you seek response curve in the stage two and given how common this type of risk response curve isn't CNN put the result of what you saw in stage to be more reflective of the drugs profile.
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And you know in that regard for DFT actually want you to stick with the sequential parallel concurred design or maybe include also a 10 milligram dose given that it performed better in stage. Two then in 60 milligrams I know you seem to be emphasizing the 50 milligram dose but.
I don't know if it's in lumber.
Robert dosing, but the 10 milligram may start to show yeah. Thanks, great.
Great questions, Jordan and getting people to tech team, Yeah, let me kick it off by saying that the sequential pet other competitive design, it's not designed suitable.
For any kind of registrations.
Registration trial ft.
Hey, it's very clean its guidance on that so the study will be a much more straightforward active against placebo higher just like the problem Algosaibi Trust like the DPN study.
I should also say that and when it does I said, if you look at stage one of our study which.
Yes of course.
The design.
Design of a more traditional study we have all the right guidance. They have with respect to the 10 of the 15 milligram dose separating on total caps separating one sub domain.
Separating on it like PON does rates and so if we use that as a partner.
Oxy for next study then I think that data is fully aligned with the next design of the study.
As it relates to why.
Why did the stage two patients with punk the way. They did I think it's important to we know that those way off course placebo another correspondence.
That way.
Basically randomized to the tenants 50 milligram dose and they of course starts with a much reduced burden of Pts E. Because they have already shown a response and to EPS further onto that by separating between placebo and.
Active of course gives you a sort of like slightly Nevo a delta then in stage one.
We're still trying to dissect the difference between 10 and 15 milligram because as I mentioned in the script it actually is.
Yeah from all the guys that while 50 does that.
10 was also active.
And that gets to your next question of what do we think about that.
Dosing in the next study.
I think the 50 milligram dose is a solid start.
We have it we as a team need to decide if we actually also pick a dose below that.
Abbas that not yet decided but.
But clearly a possibility just like we do the problem as a study with 50 and 100 milligrams.
To actually make sure that we are always operating in a range, where we believe we have the approval.
Dose exposure in the brain to do the fit to do that.
To drop off basically showing the results if you need to see.
I don't know if Tina wants to add anything to that yes. The only thing I would add it's Andy here is if you look at the numbers of patients in each group in stage two and the differences between 10 and 50 milligrams in absolute terms I think you really have to stream quite hard to conclude theres a U shaped so stressed.
Response.
Theres 11 patients in the 10 milligram group and 13 in the 50 milligram group and most of the numerical differences are small so I numerically. There are some instances were 10 milligrams perform better but I think that the numbers are relatively small differences are small and we were not convinced.
Best.
June does that answer your question.
Yes. Thank you. Thank you very much I think you know, but one more question then I'm sure you have any plans to.
Sure sure just additional data at a at a better profit as Matt.
Actually I have one.
One more question actually I'm sorry.
You know one of your findings in the BPM study was that the different patients responded.
Responded better to your drug like people with longer history of peripheral neuropathy responded better because of the way the drug works in the PTSD study are you seeing any differences between the sub groups.
Maybe people who suffered.
Longer may have responded better while you know placebo wasn't placebo response wasn't as high.
For those sub population or maybe a P.S.D. due to different types of drivers of trauma to drivers of respond better to the drug any any sort of and they seem to be you know you're seeing now.
Now or plan to share at a medical conference. Thank you Yeah, I think that's right. So token answer both of those in the same way in which is yeah, we will be going through some of the additional findings from the full data set at a at a medical meeting most likely in the near future and it may include some of those cost.
Since that you asked but I think it'd be premature for us to comment at this point because we're still as you can imagine not completely finished with the analysis.
Thank you.
Thank you.
Your next question comes from the line.
[noise] web security.
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It's Mike Hi, This is kinda fuels on for Laura Chico. Thanks for taking my question and congrats on the progress.
So just on the four or five eight what is your confidence in restarting the study.
I'm 2021 with flare up starting in the U.S. globally for coated are there any specific strategy incurred litigation against delays.
And.
Crewmen in the park the base I mean to be challenging in this environment.
Thanks.
Yeah. So.
Great question, I think Catlin and so it's a good question in a way already even skewing she addressed what we have put in place.
With respect to Hobble my alginate.
Yeah, and namely just making sure that the sites in particular.
A quick payouts and are capable of.
Engaging with patients and is taking vitamins.
Making sure that we minimize exposure all patients through site.
Unnecessarily parts to.
Ponder called the clean quiet interaction with study sites more quickly than necessary.
I would say those are the key the key precautions we are taking.
And.
And we have as I mentioned paused until now to just beach can that be provided as it environments.
We believe a more wind out of the patient population is protected and I think that actually is a belief we know house based on all these data and all the experience in bringing up the problem areas of study and also actually in taking the PTSD study all the way to the finish line dueling Hey, Colby.
It's 19 pandemic so.
So far so good and I hope if the environment stays stable and predictable enough for that to not change.
Yeah, I would just add it's Andy I'd like I do think with respect to the Parkinson's population. Specifically you know we will be obviously vigilant in kind of monitoring.
What changes with respect to co bad in the U.S. and Ah you know that the measures taken to try to control covered in the U.S.
So right now you know our planet's to restart in Q1, but we'll be playing close attention to that obviously and we will be able to update you as we get as we actually restart and then as things unfold.
Okay. Thanks, so much.
Your next question comes from the line of Myles men turn with William Blair.
Hi, Thanks for taking the questions I know of it.
Maybe a question for Kathryn again on just the type.
On same protocol amendments I'm wondering whether you can get specific on differential protocol amendment, you've made to the Fibra my algae, a child versus the painful diabetic peripheral neuropathy population because one that youd see me not come from Austin. The other it's not I'm just trying to get an understanding of additional data point.
So we might see one profit should see on though.
Yeah, I think because we started from a lot of different procedures for Fibra. We did make some different changes then we would have in G.P. on but I think we're still focused on pain scores captured.
The patients on a daily basis in their homes, which is a pretty good endpoint to have in a setting like we're in right now and so I think the streamlining that we did really had more to do with the interactions that patients have with site.
And I think there are slight differences between what they're for Fibrogen DPN, but because our focus in those businesses.
Our safety related they are probably more similar than they are just fine.
Okay codes.
And then just.
Theoretical question, but we talk about fear extinction, which.
Seven I'd three I'm wondering how best to design, a clinical trial to actually prove that out I know right.
At 12 wave randomized trial measuring cats bogs a potential endpoint.
Mike mentioned potential benefit, but it doesn't really capture whether this is actually a functional cure, which when we're talking about here extinction. Maybe it is sorry, obviously he might be the FDIC Mont request a random.
It must be true relapse study or something like that.
I'm just wondering your thoughts on how best to prove out that hypothesis in a potential pivotal trial.
Yeah, So look let's start with.
The mechanism of in BLAIC seven eight we like we have to validate that.
Myles in very I think well his depth isn't validated animal model.
That's the clock is very clearly active in a few extinction paradigm not only does it extinguished FIA.
Also activity allows for the consolidation of six year extension in that.
It's directly related to the mechanism of NYSE seven day, three U.S.A.M.D. it looks at the modulator.
Because he didn't say mess off synaptic plasticity it.
It's a bit off memoli and learning paradigms and as we have discussed before they do.
Off someone separately from PTC is that they can't really and learn.
The fear paradigm.
Initially caused the symptoms of PTSD and they are reminded us that on an ongoing basis, even if the stimulus they encounter.
Not even close to the initial call.
And so.
That is also why we said if mechanistic tilley.
We are right that is happening in the prefrontal cortex, where they say hi, Paul functional.
In India receptor activity there.
That's where we should see that leads us best and we should see them in one of the.
Domains, primarily the activity and have also.
With that I described it in the script as being sort of like the hallmarks associated with PST.
And that is indeed, where we saw that major major change all the way to being statistically significant in a brief four week.
So so far I think we have all the right indicators for that mechanistic rationale to really apply.
And that's that's where we will keep our focus and so.
Okay.
I I don't remember to Chew EPS.
Because also if there's a way of perhaps looking at this more as a disease modifying versus symptomatic treatment for PTC.
Because that that might be the underlying rationale of your question, but I want to clarify that.
Yes, that's what I'm, asking how we potentially prove that out in a clinical trial.
While on our.
Randomized withdrawal rate much monitoring studies are an option, but if youve already to sphere extension in the treatment period, maybe it isn't an option. So I'm just wondering how this hypothesis gets proven in a clinical trial to make those clients.
Yeah Yeah.
So I think we would have to think about that some more and and see what else. We could do that goes beyond the sub domain and trust that we believe is very indicative of FIA in FY extinction.
And there might be a way to also when you get there very subjective lead from patients.
As to how they're sort of like fear paradigm has changed with treatment with seven a clean and I would say great point, let's put us on the investment that we discuss it and then maybe capex to you were in one of our meetings to engage further on that topic.
No I think we're certainly thinking about it Michael.
Sales as you know first things first just trying to get an idea right and what's the best path to do that but but it is a great question to think about.
I see you are starting to allude to in your question, though.
Getting into that in detail may not represent the easiest path forward immediate next step or path forward.
The word for preclinical development. So I think it's an interesting thing to think about something we can discuss with the agency and so on but our our definitely our commitment is trying to find that this is a difficult enough endeavor in the first place trying to find the easiest path to an MD I looked at it has to be clear. Thank you Andy for saying that I do not.
That's all believe Myers that that will be a requirement clause for further development and the legislation into pools I.
Hi, Paul and I believe you asked in the question of can you find ways.
To then further differentiate yourself with the mechanism of action because it is so uniquely different from anything out.
Out there today that is used.
I understood. It more has further augmentation as opposed to a regulatory path to the finish line.
And I think that is how you are meant to be liked yeah. Yeah that was definitely how is framing. The question. Okay. Regularity primary endpoint is one thing the actual exact secondary endpoints you might mention to take.
You talked about the substantially us.
So I feel very clear.
Beautiful thanks for the questions.
Okay nothing else, but.
Okay.
Your next question comes from the line of from so farm reduce with H.C. Wainwright.
Right.
Thanks, So much for taking my questions on 73, I just wanted to know whether and when you think that would be potentially advisable to seek something like a special protocol assessment from the FDA and if you envision that being a possibility at what the the.
Clinical development critical path would it be appropriate.
That's a great question. So look we have pointed to always especially when we discussed the data initially.
That we find it remarkable that we got separation with respect to total cap suffice.
That's very clear and strong efficacy and clinically meaningful and statistically efficacy on Roseland reactivity as well as responder rate.
And when we talk to clinicians clinicians, we really focus on.
What is it.
The ideal profile for me to reach as many patients as they can with its treatment modality.
And clearly responder rate is a huge part of satisfied it doesn't help me much if I ever talk that helps 10% of PTSD patients, but not the other 90% comp.
Compared to us reaching into.
In the study that we just did almost 80% with a 30% improvement at 50% was a 50% improvement, but just remarkable after four weeks of treatment only and so we pointed this out before we'd like to have a dialogue with calix ends with the agency asked to.
What the end point best to service. This patient population. It is fair for one we should consider.
And hopefully we can basically of ice there.
Whether or not that will then be triggered triggering a special protocol assessment.
Or a different path forward, we have to see.
But the primary focus really is initially on dialogue that is relevant to P.S.E. and P.T. so for us and what's in the best interest of patients. Yes, we look at next steps and develop.
That's very helpful. A couple other items on 73, if I may.
Firstly, you know it looks like there.
Here to be enhance the Olympic effect with the drug and I was wondering whether that's specifically is leading you to think about other.
Environment, another context in which the drug could potentially be deployed and demonstrate therapeutic be meaningful effect beyond Pts. So if we think about that.
Specifically as a potential and feel that it is.
Is that likely to provide you with additional directions for new indications to explore with 783 beyond PTSD and if so what might those indications between.
Yeah, No. It's a great question is an interesting finding I can see a b.
The.
Production and anxiety for sure and I think we find just corroborate some of the other findings. It is important to remember that these patients are diagnosed with PGS. The dock with a you know generalized anxiety disorder and their baseline scores on anxiety instruments are relatively low.
Hello, Ah you know compare.
Compared to a generalized anxiety population, so I'd be reluctant to kind of read through that this is the same as showing and a general answer let take effect I think we viewed it as a nice corroborating endpoint and B warrants you know further exploration at some stage, but our primary views.
For follow on indications is still to build off of a platform of some research that we've been doing which as you probably know includes a lot of work on on addiction, which has a similar underlying mechanism to fear extinction, which and we actually have an athree is showing very nice robust effects on pre clinically so I think that.
Thats still our major focus in terms of follow on indications are kind of thinking through what the next steps would be there.
And hopefully we'll have more to say on that in the future.
Great and then just one item regarding.
No discussions that you may have been having I don't know whether this is indeed the case.
Since you reported the Pts the proof of concept results and I was wondering if you had seen any interest in potentially exploring 783 through government sponsored studies from certain government agencies like for example, the Ob or bar.
If if that.
I think thats already under discussion whether you can provide us with any color on what potential government agency sponsored studies might conceivably be undertaken in the foreseeable future, which seveneighty three sort of beyond Europe directly take with your study in PTSD.
Yes.
Great question. So I think now that we have data actually clinical data in patients.
I think we have the right foundation upon which to launch the increasing into.
What it could be actually look floor as it relates to this being.
Almost political and enormous unmet needs.
Where there is many many many sources also off of interest and also funding possible. So we will explore that it's a little bit too soon to comment, but really the unmet need is high, especially all even more so now than they are.
Moving mechanisms that include the department of defense, but also other sources that leave what's absolutely want to explore and now that we have the data that guy does that seven day speak freely is active in P.T. as Steve and.
Really really importantly at its best so without a at the side effects at.
Hi, so many other therapies.
During a very clear and clean Tolerability and safety profile. So I think we are well positioned to.
We'll explore those revenue was four additional funding coming into the organization to support the sudden next study.
Thanks, very much and congrats on all the progress.
Thank you.
Your final question comes from the line of Jeff.
Hi.
Good morning.
Hi, guys. Good evening. Thanks for taking my question I'm, just one from me how long into the restart of the DPN study will it take to confirm that enrollment is tracking in a way that will allow you trip.
For data in the first half of 2022.
So typically yes, yes, you know things take a little time to ramp up.
And so I would say when we get to a state when we believe we are the steady state of enrollment then we have a pretty good measure.
What a likely end state of completion date looks like.
I would say.
It's going to take a few months before we have a steady state since and in particular in this environment.
I think we want to be monitoring that closely so that we don't have.
Ups and downs.
Oh deviate from the norm and you can expect that we would be guiding pretty much on every call as to whether that timeline victory offering today is a term timeline or can be accelerated or needs to be moved out, particularly albeit driven by actual experience in the field.
Which we don't have quite.
That yet.
Okay got it thank you yep.
Yep.
There are no. Other questions you may continue with any closing remarks.
All right. Thank you operator.
Thank you all for your thoughtful questions.
We appreciate your time and.
And your attention please.
Please stay safe irrelevant and enjoy the rest of your day.
Good evening Bye bye.
Ladies and gentlemen, this concludes today's conference call. Thank you for participating you may now disconnect.
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