Aptinyx Inc (APTX) CEO Norbert Riedel on Q3 2020 Results - Earnings Call Transcript
Aptinyx Inc (NASDAQ:APTX) Q3 2020 Results Conference Call November 12, 2020 5:00 PM ET
Company Participants
Nick Smith - Senior Director, Corporate Development & IR
Norbert Riedel - President & Chief Executive Officer
Ashish Khanna - Chief Financial Officer & Chief Business Officer
Andy Kidd - Chief Operating Officer
Kathryn King - Senior Vice President, Clinical Development
Rolando Gutierrez - Senior Vice President, Medical and Pharmacovigilance
Conference Call Participants
Chris Raymond - Piper Sandler
Marc Goodman - SVB Leerink
Ritu Baral - Cowen
Charles Duncan - Cantor Fitzgerald
Gary Nachman - BMO Capital Markets
Joon Lee - Truist Securities
Kenneth Shield - Wedbush Securities
Myles Minter - William Blair
Ram Selvaraju - H.C. Wainwright
Jessica Fye - JPMorgan
Operator
Good afternoon and welcome to the Aptinyx Third Quarter 2020 Financial Results 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 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 and Investor Relations at Aptinyx. Nick, please proceed.
Nick Smith
Thank you, operator. Good afternoon, everyone, and thank you for joining us on today's conference call to discuss Aptinyx' third quarter 2020 financial and operating results. Our press release describing financial results and recent highlights is now available on our website. Before we begin, I'd like to extend a warm welcome to Chris Raymond of Piper Sandler, who recently initiated coverage on Aptinyx. Welcome, Chris. We're glad to have you join us this afternoon.
On today's call, Norbert Riedel, our President and Chief Executive Officer, will discuss our business and clinical progress; then Ashish Khanna, our Chief Financial Officer and Chief Business Officer, will review the financial results. Additionally, for the Q&A portion of the call, we're joined by Andy Kidd, our Chief Operating Officer; Kathryn King, our Senior Vice President of Clinical Development; and Rolando Gutierrez, Senior Vice President of Medical and Pharmacovigilance.
I'd like to remind everyone that statements 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. Please see the forward-looking statements disclaimer in our financial results release issued this afternoon and the risk factors in the Company's current and subsequent filings with the SEC.
It's now my pleasure to turn the call over to Norbert.
Norbert Riedel
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 are safe and in good health. Clearly, the COVID-19 pandemics has been challenging for all of us on both personnel and professional levels throughout 2020. Like many in our industry, our progress at Aptinyx 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 programs.
In response to the pandemic, we have taken measures to support the durable advancement of our pipeline, ensure patient safety and the integrity of the data from our clinical studies and manage our finances in a 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 2 exploratory study of NYX-783 in posttraumatic stress disorder, which demonstrated clinically meaningful improvements in treating patients, living with PTSD. These results surpassed the expectations we had for this signal-seeking study, validating the potential of our mechanistic approach to fear thinking.
The data from this study are highly informative for several reasons. They provide us with the paths for advancing NYX-783 in development for patients with PTSD. And in addition, we have now validated our novel approach of NMDA receptor modulation in three therapeutic indications: fibromyalgia, painful DPN and PTSD.
With respect to NYX-2925, in September, we were pleased to recommend our Phase 2b study of NYX-2925 in patients with fibromyalgia, in which we had previously paused enrollment due to COVID-19-related challenges. Thanks to our team's extensive efforts during the enrollment pause, we are also on track to resume our Phase 2b study of NYX-2925 in painful DPN before the end of the year. And in the coming months, we anticipate the resumption of our clinical investigation of NYX-458 in patients with cognitive impairment.
As we bring these studies back online, we expect to report results from each of them in 2022. Accordingly, last month, we boasted our financial strength with the close of a $48 million common stock offering, to fund our operations through these multiple catalytic data readouts. All of this progress leaves us well positioned and equipped to responsibly advance our novel NMDA receptor modulators, toward patients who may benefit from them.
Let's begin by discussing NYX-2925, our clinical stage development candidate under evaluation in two chronic pain indications; fibromyalgia and painful diabetic peripheral neuropathy, or DPN. Due to the escalation of the COVID-19 pandemic in the U.S., we have to suspend enrollment in both of our Phase 2b studies in chronic pain in late March of this year. Fortunately, in September, we announced that we were able to recommend our Phase 2b fibromyalgia study, and we are presently actively recruiting and enrolling patients.
While it is still early in the recommencement, patient recruitment, screening and enrollment are tracking well. And I'm confident that we have taken necessary precautions to ensure the safety of all those involved in this 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 patients screening and enrollment progresses.
Alongside our fibromyalgia study, we are taking final steps to recommence our Phase 2b study of NYX-2925 in painful DPN. We have taken necessary steps to mitigate the elevated risks posed by COVID-19 to the DPN population, and we have instituted certain protocol changes intended to streamline patient enrollment.
We are confident that the adjustments made to the study's design will enable us to effectively screen and enroll new patients following the study initiation, even in the face of increasing cases of COVID-19, without any compromise to the rigor and quality of the study. We still anticipate that this study will restart before the end of this year. Our current expectation is that both chronic pain studies should read out in the first half of 2022.
Let's move now to NYX-783 and the positive results from our Phase 2 study in patients with posttraumatic stress disorder. The study represents the first time NYX-783 was evaluated in patients with PTSD and tested daily dosing of 10 milligrams and 50 milligrams of NYX-783 against placebo. We enrolled 153 patients in this study, who were evaluated over four weeks of treatment. No patient received more than four weeks of treatment with NYX-783.
Heading into the study, we were seeking to understand whether NYX-783 could demonstrate efficacy signals on the symptoms associated with PTSD. We looked at various efficacy endpoints with the CAPS-5 total score and symptom cluster scores 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 NYX-783 has a therapeutic effect in this patient population and to guide future development. However, we were pleased to find that NYX-783 had clinically meaningful, statistically significant and mechanistically relevant effects 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 effects compared to placebo on certain measures. On the CAPS-5 total score, we saw a clinically meaningful mean reduction with the 50-milligram dose.
In addition, we saw very strong results across responder analysis, showing a significantly greater proportion of patients on NYX-783, achieving clinically meaningful improvements on the CAPS-5 total score. These responder analyses demonstrate that NYX-783 can provide meaningful therapeutic benefit for a substantial proportion of these highly diverse patients, an important factor when physicians are making prescribing decisions.
Potentially, most notably, NYX-783 demonstrated a clinically meaningful and statistically significant improvement on the CAPS-5 arousal and reactivity score in each treatment arm. This symptom cluster score evaluates many of the hallmarks of PTSD including exaggerated startle response, hypervigilance, irritability or aggression, self-destructive behavior and disruptions in concentration in sleep.
The effects observed on this domain of symptoms, aligned with our understanding of the mechanism of NYX-783 and, therefore, enhance our confidence in the reliability and reproducibility of these effects. Importantly, NYX-783 exhibited a favorable safety and tolerability profile with very few treatment-related adverse events.
The most common NYX-783-related AE in the study was headache, with four cases total across both stages, which was comparable to placebo. No dissociative or hallucinogenic adverse events were observed, which further underscores the differentiated safety profile with our novel NMDA receptor modulators.
We believe these are critical results as we consider the tremendous medical burden of 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 effects and tolerability observed in this study as well as the information these results provide to guide the next step in development of NYX-783, 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 as it could be registration supportive if its results are positive. We expect to get regulatory feedback in the first half 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 previous studies in fibromyalgia and DPN. This successful outcome in PTS represents the third Phase 2 study that has demonstrated the therapeutic potential of product candidates from our novel NMDA receptor modulator platform and supports our continued mission of developing numerous novel therapies for CNS disorders.
Finally, let's touch on NYX-458, which is in development for the treatment of cognitive impairment. Following the escalation of the COVID-19 pandemic, we suspended our exploratory study in patients with Parkinson's disease-related cognitive impairment. Based on our compelling preclinical primate data, we are eager to get back into the clinic with NYX-458. We are finalizing a revised study design to evaluate NYX-458 in patients with cognitive impairment and expect to be able to resume this study in the first quarter of next year.
Altogether, these activities of the past several months have 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, PTSD and cognitive impairment with multiple milestones expected over the next 18 months.
With that, I will now hand the call over to Ashish to discuss our third quarter financial results.
Ashish Khanna
Thank you, Norbert. With respect to our third quarter 2020 financials, starting with the balance sheet, we ended the quarter with $103.8 million in cash and 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 our common stock offering in October 2020. And we expect our current cash will fund our anticipated operations into 2023 and enable multiple Phase 2 clinical data readouts.
Revenues for the third quarter of 2020 were $0.3 million compared to $0.9 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 AbbVie. We do not rely upon these revenues to fund our operations. And consistent with the terms of the research collaboration agreement, the joint research activities and associated payments by Allergan to Aptinyx came to their contractual conclusion during the third quarter.
R&D expenses were $6.6 million for the third quarter of 2020 compared to $11.7 million for the same period in 2019. With the recommencement of our fibromyalgia study and the plans to restart our Phase 2 DPN study by the end of this year, we expect to see some ramp-up in our R&D expenses in the coming quarters.
We reported G&A expenses of $5 million for the third quarter of 2020 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 of 2020 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 Norbert.
Norbert Riedel
Thank you, Ashish. As we prepare to turn the page to 2021, we are excited to be moving our clinical pipeline of novel, differentiated NMDA receptor modulators forward across multiple indications of high unmet patient need and substantial commercial opportunity.
We will be happy to begin taking your questions now.
Question-and-Answer Session
Operator
[Operator Instructions] And your first question comes from the line of Chris Raymond with Piper Sandler.
Chris Raymond
So just on the PTSD study, that's commencing next year, I'm 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 it looks like it's now described as registration supportive. I wonder if you could just maybe give any color. Is there additional data required? Are you looking to fill out the package with something else? Or is that just a stylistic change?
Norbert Riedel
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 the design of the next study. But I think we just wanted to clarify that the next study will be one that will be aligned with what typically a registration study should look like, namely with respect to duration of treatment, in particular as well as endpoint. And so that has not changed from when we communicated 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 our meeting with FDA, get the appropriate feedback and guidance, and then we should be off to the races in the second half of next year with the next study.
Operator
Your next question comes from the line of Marc Goodman with SVB Leerink.
Marc Goodman
So two questions. First, 458 cognitive impairment in Parkinson's, just seems to be a lot of companies that are early stage as 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 with respect to what you've seen out there. Anavex has a Sigma 1 that they're trying to move forward there in that area. Just curious what your thoughts were? And then second, you just mentioned on the call a little bit of the R&D thoughts, a little bit of a pickup in the fourth quarter. But trying to give us a sense of really what's going on with next year. And how much we should have R&D really ramping up?
Norbert Riedel
Yes. So Mark, thanks for the questions, as always. Look, I started with 458 and then see if maybe one of my colleagues has further comments on it. I think we, of course, all know that the population of Parkinson's patients is particularly vulnerable to a 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 way of conducting this study that takes into account all the necessary precautions to not expose patients unnecessarily to anyways and to make sure the data intensity is given.
I think we have learned from the PTSD study. We are learning form the fibromyalgia study that -- I have a pretty good handle on what that needs to look like, which is why we have guided in my comments to Q1 as our starting point to kick that study off again. And we are taking the protocol and I'm making necessary adjustments and changes largely in line with providing that safe environment. And I feel pretty confident that once we kick this off, we should be able to conduct it all the way through to completion. And you've mentioned what those changes are. I think if we get to restart and let you know that at the right time.
Andy Kidd
Yes, Andy, here. I think, just to add there, there is a few other companies out there. I think it is all relative when you say a lot it's just a handful and this is an area of huge unmet needs. So, I think it really is more striking that probably. There just hasn't done a lot of activities in the Parkinson's partnership area before and there's a lot of opportunity, and I think, an increasing recognition of how important those symptoms are.
So, you mentioned, the Anavex Sigma-1 agonist, we know that Lilly has a dopamine one agonist, we know that. I think Stages have talked about Parkinson's and also Huntington's and so forth, or fear and MDA. And so, there's a few mechanisms out there, they're all like you said, and relatively early stages. I think that's actually, in some ways refreshing activity in this area, because there's so much unmet need. And I don't think we've used two or three other programs in early stages of anything other than its great for patients and a great recognition of the unmet need.
Ashish Khanna
And then, Mark, it's Ashish. With regard to burn and R&D spend, we talked about our existing cash balance, funding our operations, which are primarily centered on R&D expenditures through 2021, 2022 and into 2023. As we look at that time period and the ramp-up of studies that we've discussed, relatively speaking, 2021, we'll have the higher weight for expenditures, certainly. And I think by a substantial margin as we seek to complete those studies in the 2022 time frame, you'd see that ramp down rationally. And so that's sort of how describe the apportionment of that expenditure over that period of runway.
Operator
Your next question comes from the line of Ritu Baral with Cowen.
Ritu Baral
Glad to hear you guys are still well. Two questions for me. One is a very simple question, but the answer may be extended. Can you describe the geographic distribution of the trial that you will be restarting? And if that's changed at all from the original site distribution that you guys envisioned for the three Phase 2s? And second, my follow-up question is just given the vagaries of neuropsychiatric trials that we have all seen, would you guys consider running a second Phase 3 PTSD study, staggered or concurrent? Or something that could serve as like a 3b post-marketing study, if the first is successful? Like just belt and suspenders approach to developing pivotal data, is this something you have the resources for? Is this something that is a priority, potential priority?
Norbert Riedel
All right. Thank you, Ritu. I'm going to have Kathryn answer your first question on geographic distribution of any sites. They are, of course, all in the U.S., but Kathryn, if you could provide a little bit more color that would be good.
Kathryn King
Yes, certainly. For chronic pain, we have made some adjustments across our U.S. sites, in some ways trying to diversify so that we can accommodate flares with COVID or shutdowns or things like that across the U.S., getting the next period of enrollment for those trials. So I think we have tried to shift to diversify across the U.S. with respect to cognition, I think we probably won't change that geographic profile very much because we're centered on sites that take care of those patients. And I think we will stick with those sites as we go forward in 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.
Norbert Riedel
And Ritu, to your second question, it's really important for me to point out that we are really, really satisfied with the results we got in the study because it shows very clearly the effect in the right mechanistic way in which 783 is targeted to act primarily on fee extension. I also really, really point out the remarkable tolerability profile, which is now a hallmark of 2925 as well as 783. And therefore, I believe the platform at large.
So 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. And those two drugs that are moved in the indication are both SSIs or antidepressants that I think provide some symptomatic relief, but don't really target the underlying mechanism that is largely a fear conditioning, and therefore, our approach to basically fear extinction, right?
And so we will learn, 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 in particular to the fact that the unmet need is increasing and there's a desperate, desperate need for better therapies than what's out there today. So we'll see.
Operator
Your next question comes from the line of with Charles Duncan with Cantor Fitzgerald.
Charles Duncan
Okay. Congrats on a good quarter, progress at the you're getting started on a few of these things, And a question on fibromyalgia, PTSD and then PD cognition. On fibromyalgia, Norbert, I think you mentioned that enrollment was starting to go well, although it was early days. Can you provide any additional color on how it's going well, not necessarily patient numbers, but how would you characterize that?
Norbert Riedel
So it's going well because sites are basically up and running. Sites are actually prepped to do this in the environment in which we operate. We have clearly made some adjustments to the protocol that we can speak to, which provide further enhancement of that safety margin. And then we look at just screening and basically enrollment. And while -- as you just pointed out, don't offer actual numbers. I think we are on the right path. And we believe at this point that, that past guides us to the first half of 2022 data readout. And of course, we'll provide, as I mentioned, further 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 exactly in the right direction.
Charles Duncan
Okay. That's helpful, Norbert. Quickly on PTSD, I think follow-up to a previous question. I know you're 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 you can stick with the CAPS-5 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?
Norbert Riedel
Yes. Great, Charles. Thanks. So I pointed out in the script, and it's really helpful to reiterate that the study we did was only a 4-week study, right? And of course, we had, as endpoints, total caps as well as the sub domains of CAPS-5. And I think it's really important to reiterate that we did see clear separation on total caps. We did not see statistical separation. But as I mentioned, it wasn't powered to be a statistical study. And so I pointed out to you only because we are completely open to that being an endpoint as a primary endpoint, consistent with what the agency has been looking for up to now.
But we also pointed out that a very relevant, highly relevant top domain from a mechanistic point of view and from a hallmark of symptoms point of view is reactivity and arousal, 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 this.
And if we are talking about 80% of the population having at a response rate of better than 30% relative to baseline, I think that's very, very meaningful and hugely helpful, right, especially when measured in this diverse population that we had in this study. And so those are the keys that I want to just reiterate, the next study will be longer than four weeks. We will see how much longer than four weeks.
But as you know from the studies we are doing with 2925, we had initially a 4-week study that had the same purpose of finding signal and safety in patients. The next studies are 12-week studies. And so we will do the same here in PTSD. We will make the study comply with what is required ideally for a pivotal design or registration design and that will all be finalized, of course, as we come out of the meeting with FDA.
Charles Duncan
Okay. Look forward to that and would love to see a responder as 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 with your candidate. Wondering if in terms of an endpoint, would you be looking at UPDRS number one or total score or some other measure of cognition improvement or stabilization in that setting?
Norbert Riedel
Yes. Look, I'm going to have Andy and Rolando sort of like tag team on answering that question.
Andrew Kidd
Yes, Charles, thanks. It's a good question. So we are in the process, obviously, as we restart that study, of finalizing all of the changes. We -- I think, I talked before about using a range of different domain-specific neurocognitive tests to establish effectively what is it the drug really does from a cognition perspective in human subjects. And we're going to stick with that approach.
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 to us, the most informative endpoints from an efficacy point of view will be those because those are very discriminating, quite granular, very targeted.
Now, you're right, of course, there's lots of other than broader instruments that can be used with the assess cognition, assess function, assess 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 specific neuro competitive test improvements and some of those other instruments.
And I still -- and there is a question, I think, then obviously, they're over what our possible regulatory endpoints down the line. But I'll let Rolando comment, if he wants to add anything.
Rolando Gutierrez
Yes. No, I think that you covered it very thoroughly. Just to mention the UPDRS, of course, has a motor component and then the non-motor component. It doesn't really focus on cognition. So for that, there are a number of other instruments. Many of them borrowed, frankly, from the Alzheimer's research areas. So for example, the civic-plus, so yes, that should give us a good sense of what the clinical effects will be of the drug.
Operator
Your next question comes from the line of Gary Nachman with BMO Capital Markets.
Gary Nachman
On 2925, to follow-up, just describe how some of the protocols have been amended for both the fibro and DPN studies to make it easier to enroll those patients during COVID? And I know you're still targeting 300 for Fibro 200 for DPN, any possibility the numbers could be smaller if enrollment is ultimately challenging?
Norbert Riedel
So I'd answer the second part of your question, then I'm going to ask Kathryn 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. I think really the main reason why we paused was because this is a study that builds both the DPN and the fibromyalgia study on very clear and compelling results from our earlier Phase 2 study. And we have designed these trials to take all of those observations into the design and into consideration, and they are designed to be pivotal studies to show definitively that we get a clinically meaningful and statistical result in the primary endpoint.
And so I would be heart-pressed to really compromise that study by cutting the numbers back or by doing other things that would not really serve the purpose well. I hope that we will not be in an environment where we have to make very, very difficult decisions again. But if you ask me right off the cuff, I would probably rather pause than 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 putting around this that.
And then Kathryn, can you offer -- Gary, does that -- before I hand it over to Kathryn, does that answer your question?
Gary Nachman
Yes, yes, absolutely. That's helpful.
Norbert Riedel
Okay. And then, Kathryn, can you add some color to some of the changes we made to be better adapted to a COVID environment?
Kathryn King
Sure. Thanks, Norbert. So I think those changes come in two categories. First would be descriptions of our population, which, in general, did not change. These are still patients with DPN for at least four years who are in pain so that we can measure a change from that baseline. Likewise, these are patients with fibromyalgia diagnosed at least one year ago, who has pain that we can measure a change from baseline.
We were able to use some of the screening and enrollment information that we gained prior to our COVID pause just to make some, I would say, small clarifications to that inclusion and exclusion criteria in order to benefit enrollment going forward. So by and large, there aren't changes to the population we were seeking, but we were able to streamline some things based on what we learned before those studies went on pause.
The other group to changes that we made had 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 we're able to remove some procedures that we think pose a greater risk, again, to staff or to subjects. We also took an eye towards streamlining those visits so that they can be done efficiently, allowing more patients to come into the site down a day or in a week and also allowing room for cleaning and disinfecting things that the sites need to do in their day as well.
So I think there were specific procedure-related changes that we made, but then also an overall look at the efficiency of those visits in 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 Norbert said, things are going well in the early days.
Gary Nachman
Okay. No, that's very helpful. And then just one of the 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 or separately in the first half of '22? Just thinking of the chronic pain indication more broadly, would you rather have both data sets in hand at the same time, just to analyze how you could approach chronic pain in general? Or are you just going to have to release it whenever this data comes through?
Norbert Riedel
No, no. So Gary, that's a great question. And so as you pointed out, right, the fibromyalgia study is a 300-patient study, that's up and running. The DPN study is a 200-patient study that is a little bit behind in its recommencement. I think they will read out close to each other. And even if close means like within a month or two, I don't know at this point, our target, of course, 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 with 2925 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 path forward clearly is the goal of a broad chronic pain label for 2925, of which the two ongoing studies are basically examples, but it does not need to be and should not be limited to believing that, that's all we are seeking with 2925. And we will make our decisions, of course, based on the data as they come in and as the results guide us, just like we have been doing up to now.
Operator
Your next question comes from the line of Joon Lee with Truist Securities.
Joon Lee
I have a question on the results you saw in the Stage 2 part of the PTSD study. The way I understand -- the point of doing a sequential parallel comparator design suppress the placebo response in Stage 2 and to better reflect the drug's efficacy. And what I find interesting is that there seems to be an inverse U-shaped -- or actually U-shaped response curve. In the Stage 2 and given how common this type of response curve is in CNS, put the results of what you saw in Stage 2 be more reflective of the drug's profile? And in that regard, could the FDA actually want you to stick with the sequential parallel comparator design or maybe include also a 10-milligram dose, given that it performed better in Stage 2 than in 50 milligrams? I know you seem to be emphasizing the 50-milligram dose, but I don't know, is a longer dosing, that 10-milligram may start to.
Norbert Riedel
Yes. Great questions, Joon. And again, we're going to take, let me kick it off by saying that the sequential parallel comparison design is not a design suitable for any kind of registration trial. FDA is very clear in its guidance on that. So the study will be a much more straightforward active against the placebo, higher just like the fibromyalgia study, just like the DPN study. I could also say that in either way sketch, which is, of course, the design of a traditional study, we have all right guidance there with 10-milligram to 50-milligram dose separating on total CAP, separating one sub-domain, separating responder rate.
So if we use that as a proxy for next study, then I think that data is fully aligned with the next design of the study. As it relates to why is it the Stage 2 patients with on the way they did. I think it's important to really know those ways of course placebo non-responders that were basically randomized to 10-milligram to 50-milligram dose. And they of course start with a much reduced burden of PTSD because they have already shown a response. And to have further onto that by separating between placebo and active, of course, gives you a sort of like a level delta then in Stage 1.
We've been trying to dissect the difference between10-milligram and 50 milligrams. But as I mentioned, it could, it actually is clear from our results that I think it is better and was also -- and that gets to your next question about what do we think about dosing in the next study? I think the 50 mg dose is a solid dose. We have still need to decide, we actually also picked a dose below that offset what yet decided, but clearly a possibility just like which is a fibromyalgia study with 50 and 100 mg to actually make sure that we are operating in a range that we believe we have the optimal dose exposure in the brain to do the drop off of basically showing the results. Andy Kidd, do you want to add anything to it?
Andy Kidd
Yes. The only thing I would add. This is Andy here. If you look at the numbers of patients in each group in Stage 2 and the differences being 10 and 50 milligrams in absolute terms, I think you really have to restrain quite hard to conclude there's a U-shaped dose-response. There's 11 patients in the 10 mg group and 13 in the 50 mg group. And most of the numerical differences are small. So numerically, there are some instances where 10 mg performed better, but I think that the numbers are relatively small, references are small, and we're not convinced.
Norbert Riedel
Joon, did that answer your question.
Joon Lee
Yes, thank you. Thank you very much. One more question. Do you have any plans to share additional data at a medical conference? Actually, I have one more question. Actually, I'm sorry. One of your findings in the DPN study was that different patients 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 subgroups, maybe people who suffered longer may have responded better, while placebo more than placebo response wasn’t as highs for those subpopulation or maybe PTSD due to different types of drivers, trauma deep drivers like respond better to drug, any sort of additional data you're seeing now or plan to share at a medical conference?
Norbert Riedel
Yes, I think that's right, so I can answer both of those in the same way, which is, yes, we will be going through some of the additional findings from the full dataset at a medical meeting most likely in the near future. And it may include some of those questions that you asked, but I didn't get the parameter for us to comment at this point, because we're still, as you can imagine, not completely finished with the analysis.
Operator
Your next question comes from the line of Laura Chico with Wedbush Securities.
Kenneth Shield
Hi, this is Kenneth Shield on for Laura Chico. Thanks for taking our question and congrats on the progress. So, just on four or five days, what is the confidence in you starting the study on 2021? With flare ups starting in the U.S. globally, for COVID, are there any specific strategies for limitation against delays and recruitment in the Parkinson's disease based in this challenging in this environment?
Norbert Riedel
Yes, so great question. I think Kathryn answered the question in a way already when she was asked what we have put in place with respect to fibromyalgia and DPN. Namely, just making sure that the site in particular, are prepared and capable of engaging with patients in a safe environment, making sure that we minimize exposure of patients to site, unnecessary parts to a protocol that require impacts to the study site most basically they're necessary.
I would say those are the key precaution we are taking, and we have as I mentioned, last until now just reached and that is provided as an environment where we believe a more vulnerable patient population is affected. And I think that actually is the belief we now have based on all the data and all the experience in cleaning up the fibromyalgia study. And it is also actually in taking the PTSD study all the way to the finish during a COVID-19 pandemic.
So far so good, and I hope the environment stay stable and particularly not for them to not change.
Andy Kidd
Yes, I would just add that. I do you think with respect to the Parkinson's population specifically, we will be obviously vigilant in kind of monitoring what changes with respect to COVID in the U.S. and the measures taken to try to control COVID in the U.S. So right now, our plan is to restart in Q1. So, we'll be paying close attention to that obviously, and we will be able to update you as we actually restart and as things unfold.
Operator
And your next question comes from the line of Myles Minter with William Blair.
Myles Minter
Hi. Thanks for a second questions. Hi, Norbert. Maybe a question for Kathryn again on just the COVID-19 protocol amendments, I'm wondering whether you can get specific on differential protocol amendment through the fibromyalgia trial versus the type of diabetic peripheral neuropathy population, because one is immuno compromise and other is not. I'm just trying to get an understanding of additional side effects that we might see in one trial versus the other.
Kathryn King
Yes. I think because we started from different procedures for fibro, we did make some different changes than we would have in DPN. But I think we're still focused on pain scores captured by the patients on a daily basis in their homes, which is a pretty good end point to have in a setting like we're in right now. And so, I think that the streamline 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 fibro and DPN, but because our focus in those businesses, more safety related, they're probably more similar than they are different.
Myles Minter
And then just it's a theoretical question, but we talked about fear extinction with 783. I'm wondering how best to design a clinical trial to actually prove that out. I know at 12-way randomized trial measuring CAPS-5 is a potential end point, you might measure potential benefits. But as the dose really capture whether this is actually a functional cure, which when we talk about fear extinction maybe is. So I was thinking maybe the FDA might request a randomized through relapse study or something like that. I'm just wondering your thoughts on how best to prove out that hypothesis in consensual pivotal trial?
Norbert Riedel
Let's start with the mechanism of NYX-783. As we have validated, Myles, in very I think [indiscernible] delegated animal model that the drug is very clearly active in a fear extinction paradigm, not only does it extinguish fear, it also actually allows for the consolidation effect here in that.
It related to the mechanism NYX-783 as a NMDA receptor modulator. Because it s a matter of synaptic plasticity, It's a matter of memory and learning paradigms. And as we have discussed before, the dilemma of someone suffering from PTSD is that they can't really unlearn the fear paradigm, that initially caused the symptoms of PTSD. And they are reminded of that on an ongoing basis, even if the stimulizing counters are not even close to the initial comma.
And so that is also why we said, if mechanistically, we are right that it is happening in the prefrontal cortex where there's a hypofunction of NMDA receptor activity. That's where we should see the results best, and we should see them in one of the sub-domains, primarily the reactivity in arousal, that I described in the script as being sort of like the hallmarks associated with PTSD. And that is indeed where we saw that major, major change, all the way to being statistically significant in a brief four-week study.
So far, I think we have all the right indicators for that mechanistic rationale to really apply. And that's where we will keep our focus. And so I don't remember. If you ask us also if there is a way of perhaps looking at this more as a disease-modifying versus symptomatic treatment for PTSD because that that might be the underlying rationale of your question, but I want to clarify that.
Myles Minter
Yes, that's what I'm asking, how we potentially prove that out in a clinical trial. I know randomized withdrawal relapse monitoring studies are an option, but if you've already induced fear extinction in the treatment period, maybe it isn't an option. So, I'm just wondering how this hypothesis get proven in a clinical trial to make those claims?
Norbert Riedel
Yes, yes. So I think we would have to think about that some more, and see what else one we could do that goes beyond the sub domain and trust that we believe is very indicative of fear and fear extinction. And there might be a way to also really get a very objective read from patients as to how they are sort of like fear paradigm has changed with treatment with the 783. And I would say, great point. Let's take this under advisement and we discuss it, and then maybe get back to you in one of our meetings to engage further on that topic.
Andy Kidd
I mean I think we're certainly thinking about it, Myles, as first things first is to try to get an NDA, right? And what's the best pass to do that. But it is a great question to think about it. And as you are starting to allude to in your question, though, getting into that in detail may not represent the easiest plot forward, immediate next step or path forward for clinical development. So I think it's an interesting thing to think about something we could discuss with the agency and so on. But our -- definitely, our commitment is to try to find this is a difficult enough endeavor in the first place, trying to find the easiest path to NMDA.
Norbert Riedel
Yes, yes. Look, just to be clear, Thank you, Andy, for saying that. I do not at all believe, Myles, that, that will be a requirement care for further development and registration and approval. I thought and I believe you asked it 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 there today that is used. I understood it more as further augmentation as opposed to a regulatory path to the finish line. And I think that is how you meant it. Correct?
Myles Minter
Yes, yes, that was definitely how it's framing the question. The regulatory endpoint is one thing, the actual secondary endpoints you might measure to keep this hypothesis out.
Norbert Riedel
Yes, very clear.
Operator
Our next question comes from the line of Ram Selvaraju with H.C. Wainwright.
Ram Selvaraju
On 783, I just wanted to know whether at any point, you think it 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 stage in the clinical development critical class would it be appropriate?
Norbert Riedel
That's a great question. So look, we have pointed out, especially when we discussed the data initially, that we find it remarkable that we got separation with respect to total CAPS-5, but very clear and strong efficacy and clinically meaningful and statistical efficacy on rouse and reactivity as well as responder rates.
And when we talk to clinicians, clinicians really focus on what is the ideal profile for me to reach as many patients as they can with the treatment modality and clearly, responder rate is a huge part of that, right? It doesn't help me much if I have a drug that helps 10% of PTSD patients but not the other 90% compared to us reaching in the study that we just did, almost 80% with a 30% improvement and 50% with a 50% improvement, which is remarkable after four weeks of treatment only.
And so we pointed this out before, we'd like to have a dialogue with KOLs and with the agency as to what endpoint best serves this patient population and is, therefore, one we should consider. And hopefully, we can basically arrive there, whether or not that will then be 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 PTSD, and PTSD separable in what's in the best interest of patients as we look at next steps in development.
Ram Selvaraju
That's very helpful. A couple of other items on 783, if I may. Firstly, it looks like there appears to be an anxiolytic effect with this drug. And I was wondering whether that specifically is leading you to think about other environments, other contexts in which the drug could potentially be deployed and demonstrate a therapeutically meaningful effect beyond PTSD. So if we think about this specifically as a potential anxiolytic, 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 decrease?
Andrew Kidd
Yes. No, it's a great question. It's an interesting finding. I think the reduction in anxiety, for sure. And I think we find it that it corroborates some of the other findings. It is important to remember that these patients are diagnosed with PTSD, not with generalized anxiety disorder. And their baseline scores on anxiety instruments are relatively low compared to a generalized anxiety population.
So I'd be reluctant to kind of reach through that this is the same as showing the general anxiolytic effect. I think we viewed it as a nice corroborating end point. And it may warrant further exploration at some stage. But our primary view for follow-on indications is still to build off of a platform of research that we've been doing, which as you probably know, includes a lot of work on addiction, which is a similar underlying mechanism to fear extinction and which NYX-783 has shown very nice robust effects on preclinically.
So I think that's still our major focus in terms of following indications or kind of thinking through what the next steps would be there. And hopefully, we'll have more to say on that in the future.
Ram Selvaraju
Great. And then just one item regarding discussions that you may have been having, I don't know whether this is indeed the case, since you reported the PTSD 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, DoD or BAR. And if that's something that's 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 with 783 sort of beyond of your directly taken with your study in PTSD?
Norbert Riedel
Yes. Great, Ram. So I think now that we have data, actual clinical data in patients, I think we have the right foundation from which to launch the inquiry into what could we actually look for as it relates to this being an enormous person, an enormous unmet need, where there is many, many, many sources 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 or even more so now.
And there are funding mechanisms that include the detergent of defense, but also other sources that we would absolutely want to explore. And now that we have the data that guide us that 783 clearly is active in PTSD and really, really importantly, it does so without the side effects at burden so many other therapies by offering a very clear and clean tolerability and safety profile. So I think we are well positioned to explore those avenues for additional funding coming into the organization to support the sub next study.
Operator
Your final question comes from the line of Jessica Fye with JP Morgan.
Jessica Fye
Just one for 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 to report data in the first half of 2022?
Norbert Riedel
So typically, just as you know, things take a little time to ramp-up. And so I would say when we get to a -- when we believe we are at a steady state of enrollment, then we have a pretty good measure of what a likely end date or completion date looks like. I would say it's going to take a few months before we have a steady state sense.
And in particular, in this environment, I think we want to be monitoring that closely so that we don't have ups and downs that deviate from the norm. And you can expect that we would be guiding pretty much on every call as to whether that's time line that we are offering today is a firm time line or can be accelerated or needs to be moved out. But that will all be driven by actual experience in the field, which we don't have quite yet.
Operator
There are no other questions. You may continue with any closing remarks.
Norbert Riedel
All right. Thank you, operator, and thank you all for your thoughtful questions. We appreciate your time and your attention. Please stay safe, be well, and enjoy the rest of your day.
Good evening. Bye-bye.
Operator
Ladies and gentlemen, this concludes today's conference call. Thank you for participating. You may now disconnect.
