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Edited Transcript of AXSM earnings conference call or presentation 12-Mar-20 12:00pm GMT

Q4 2019 Axsome Therapeutics Inc Earnings Call

New York City Mar 12, 2020 (Thomson StreetEvents) -- Edited Transcript of Axsome Therapeutics Inc earnings conference call or presentation Thursday, March 12, 2020 at 12:00:00pm GMT

TEXT version of Transcript

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Corporate Participants

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* Cedric O'Gorman

Axsome Therapeutics, Inc. - SVP of Clinical Development & Medical Affairs

* David C. Marek

Axsome Therapeutics, Inc. - Chief Commercial Officer

* Herriot Tabuteau

Axsome Therapeutics, Inc. - Founder, Chairman, CEO & President

* Mark L. Jacobson

Axsome Therapeutics, Inc. - COO & Secretary

* Nick Pizzie

Axsome Therapeutics, Inc. - CFO & Principal Accounting Officer

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Conference Call Participants

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* Charles Cliff Duncan

Cantor Fitzgerald & Co., Research Division - Senior Analyst

* Marc Harold Goodman

SVB Leerink LLC, Research Division - MD of Neuroscience & Senior Research Analyst

* Matthew Lee Kaplan

Ladenburg Thalmann & Co. Inc., Research Division - MD & Head of Healthcare Equity Research

* Myles Robert Minter

William Blair & Company L.L.C., Research Division - Analyst

* Raghuram Selvaraju

H.C. Wainwright & Co, LLC, Research Division - MD of Equity Research & Senior Healthcare Analyst

* Robert Cummins Hazlett

BTIG, LLC, Research Division - MD & Biotechnology Equity Research Analyst

* Yatin Suneja

Guggenheim Securities, LLC, Research Division - MD & Senior Biotechnology Analyst

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Presentation

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Operator [1]

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Good morning, ladies and gentlemen, and welcome to the Axsome Therapeutics Conference call. (Operator Instructions)

As a reminder, today's conference call is being recorded. I would now like to turn the conference over to your host, Mark Jacobson, Chief Operating Officer at Axsome Therapeutics. Please go ahead.

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Mark L. Jacobson, Axsome Therapeutics, Inc. - COO & Secretary [2]

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Thank you, operator. Good morning, and thank you all for joining us on today's conference call. Our earnings press release providing a corporate update and details of the company's financial results for the fourth quarter and full year ended December 31, 2019 crossed the wire a short time ago and is available on our website at Axsome.com.

During today's call, we will be making certain forward-looking statements. These statements may include statements regarding, among other things, the efficacy, safety and intended utilization of our investigational agents, our clinical and nonclinical plans; our plans to present or report additional data; the anticipated conduct and the source of future clinical trials; regulatory plans; future research and development; and possible intended use of cash and investments.

These forward-looking statements are based on current information, assumptions and expectations that are subject to change and involve risks and uncertainties that may cause actual results to differ materially from those contained in the forward-looking statements. These and other risks are described in our periodic filings made with the Securities and Exchange Commission, including our quarterly and annual reports. You are cautioned not to place undue weight on these forward-looking statements and the company disclaims any obligation to update such statements.

Joining me on the call today are Dr. Herriot Tabuteau, Chief Executive Officer; Dr. Cedric O'Gorman, Senior Vice President of Clinical Development and Medical Affairs; Dave Marek, Chief Commercial Officer; and Nick Pizzie, Chief Financial Officer. Herriot will first provide a review of significant corporate and clinical developments for Axsome over the course of the past year, and describe upcoming milestones. Following that, Nick will review our financial results. We will then open the line for questions. I shall now turn over the call to Herriot.

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Herriot Tabuteau, Axsome Therapeutics, Inc. - Founder, Chairman, CEO & President [3]

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Thank you, Mark. Good morning, everyone, and thank you all for joining Axsome Therapeutics' Fourth Quarter and Full Year 2019 Results Conference Call. 2019 was a transformative year for Axsome as we generated important clinical data for all of our investigational medicines, which we were developing for serious and difficult-to-treat CNS disorders. But many important achievements of 2019 included: positive NDA enabling clinical trial readouts for AXS-05 in depression; and for AXS-07 in migraine, propelling Axsome potentially towards commercial stage as early as next year. In addition, our AXS-12 product candidate for narcolepsy is progressing to Phase III, based on positive Phase II results and through our collaboration with Pfizer early this year, we expanded our Phase III pipeline with the addition of AXS-14 for the treatment of fibromyalgia.

All in all, we now have 4 differentiated CNS product candidates in clinical development, all of which have demonstrated efficacy in controlled trials, 2 of which are pre-NDA, with the other 2 entering or having completed Phase III trials. These product candidates provide new mechanisms of action and potentially faster, greater and broader efficacy as compared to currently available treatments. Our investigational medicines, therefore, have the potential to change the current standard of care for difficult-to-treat brain disorders and transform the lives of patients living with these conditions.

We expect 2020 to be a year of continued operational, clinical and regulatory progress. Preparations are underway for our planned NDA filing of the AXS-05 in the treatment of major depressive disorder or MDD. AXS-05 has the potential to be the first and only oral NMDA receptor antagonist with multimodal activity for the treatment of depression. In December, we announced positive results from the Phase III GEMINI trial of AXS-05 in patients with confirmed moderate to severe MDD. In the study, the AXS-05 met the primary endpoint by rapidly, substantially and statistically, significantly improving symptoms of depression as compared to placebo. The positive results from the GEMINI trial, along with the previously completed ASCEND trial of AXS-05 in MDD support an NDA filing for AXS-05 in the treatment of MDD. And we are on track to file this NDA in the fourth quarter of 2020.

AXS-05 has been granted breakthrough therapy designation for the treatment of MDD. Preparations are also underway for our planned NDA filing of AXS-07 in the acute treatment of migraine. AXS-07 is a multi-mechanistic approach, is designed to provide enhanced efficacy as compared to currently available treatments.

In December, we announced the positive results from the Phase III MOMENTUM trial of AXS-07 in migraine patients with a history of inadequate response to prior acute treatments. In this study, the AXS-07 met the co-primary and key secondary endpoints by significantly relieving migraine pain as compared to placebo and as compared to the active comparator, rizatriptan. The positive results from the MOMENTUM trial support an NDA filing for AXS-07 in the acute treatment of migraine, and we remain on track to file this NDA in the second half of 2020. With these 2 planned NDA filings, Axsome is on track to transition to commercial stage, potentially as early as next year. And to that end, we have been building out our commercial capabilities. In December, we also announced a positive top line result from a Phase II CONCERT trial of AXS-12 in the treatment of narcolepsy. In this study, AXS-12 significantly reduced cataplexy attacks and excessive daytime sleepiness and improved cognitive function, sleep quality and sleep related symptoms. These results point to a differentiated clinical profile for AXS-12 with the potential to address all the key symptoms with narcolepsy. Based on these positive results, AXS-12 is scheduled to enter Phase III clinical trials in narcolepsy in the second half of this year. We expect the development of AXS-12 to accelerate as a result of our agreement with Pfizer, which we announced in January. This agreement covers an exclusive U.S. license to Pfizer's clinical and nonclinical data and intellectual property for reboxetine, the active pharmaceutical ingredient in AXS-12. This agreement also expands our late-stage CNS pipeline by providing us exclusive rights to develop and commercialize a new product candidate, esreboxetine or AXS-14 in the U.S. for the treatment of fibromyalgia. AXS-14 has previously demonstrated positive and statistically significant results in the Phase III and in the Phase II trial in the treatment of fibromyalgia. In addition to its effects on pain and function, AXS-14 demonstrated in both of these studies, an effect on fatigue, a difficult-to-treat symptom of this condition.

Axsome plans to meet with the FDA this year, to discuss the further clinical development of AXS-14 for fibromyalgia.

Turning now to our ongoing efficacy trials. We recently completed randomization into our STRIDE-1 trial of AXS-05 in treatment-resistant depression and into our INTERCEPT trial of AXS-07 in the early treatment of migraine. We remain on track for top line results from both of these trials before the end of this month. Our ADVANCE-1 Phase II/III trial of AXS-05 in the treatment of agitation associated with Alzheimer's disease is now more than 80% enrolled. And based on this trend, we expect top line results from this trial in the third quarter.

In summary, over the balance of the year, we look forward to several important milestones, including the NDA filings for AXS-05 and MDD, and AXS-07 in migraine, anticipated in the fourth quarter. Top line results from the STRIDE-1 trial of AXS-05 in TRD and the INTERCEPT trial of AXS-07 in migraine this quarter. Top line results from the ADVANCE-1 trial of AXS-05 in Alzheimer's disease agitation in the third quarter, and initiation of Phase III trials of AXS-12 in narcolepsy, in the second half of this year.

I would also like to recognize and congratulate Mark Jacobson on his recent appointment to Chief Operating Officer. Mark has been a valued member of the Axsome team since 2014 and served as Senior Vice President of Operations since 2017. I will now turn the call over to Nick to provide a financial update for the full year of 2019.

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Nick Pizzie, Axsome Therapeutics, Inc. - CFO & Principal Accounting Officer [4]

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Thank you, Herriot, and good morning, everyone. I will focus on key highlights in the quarter and provide some financial guidance. R&D expenses were $19.2 million for the quarter ended December 31, 2019, and versus $7.2 million for the comparable period in 2018. This increase was due to a significant number of new clinical trials that were conducted during the quarter as compared to the prior period, including the CONCERT, GEMINI, MOMENTUM and INTERCEPT trials, along with the AXS-05 and AXS-07 open-label safety studies, in addition to the ongoing progress of the STRIDE-1 and ADVANCE-1 trials.

G&A expenses were $5.2 million for the quarter ended December 31, 2019, and $2.3 million for the comparable period in 2018. The change was primarily due to personnel costs, mainly from higher stock compensation expense, along with the build-out of the commercial functions. In December, we completed an underwritten public offering that yielded aggregate gross proceeds before deducting offering expenses of approximately of $200 million. SVB Leerink and Morgan Stanley acted as joint book running managers for the offering. As a result, we ended the fourth quarter with $220 million in cash compared with $44 million at the end of the third quarter. We believe that our current cash position will be sufficient to fund our anticipated operations based on our current operating plan for at least 2 years.

That concludes our fourth quarter 2019 financial review. I will now turn the call back to Mark to lead the Q&A discussion.

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Mark L. Jacobson, Axsome Therapeutics, Inc. - COO & Secretary [5]

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Thank you, Nick. Operator, may we please have our first question.

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Questions and Answers

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Operator [1]

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(Operator Instructions)

Your first question comes from the line of Charles Duncan with Cantor Fitzgerald.

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Charles Cliff Duncan, Cantor Fitzgerald & Co., Research Division - Senior Analyst [2]

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Herriot and team, congratulations on a very good year, and last year and what could prove to be a very interesting one this year. I wanted to ask you just a couple of quick questions on the pipeline and then maybe one on commercial build. For AXS-05, relative to the upcoming TRD results, I guess, I'm wondering, what you're thinking about the different scenarios that could be, and the impact on the regulatory and/or commercial strategy for AXS-05. Can you lay out a couple of scenarios of either the drug clearly working, the drug may be working, or the drug not working in TRD?

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Herriot Tabuteau, Axsome Therapeutics, Inc. - Founder, Chairman, CEO & President [3]

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Thank you, Charles. With regards to TRD, what's nice about that study is that it is targeting a patient population, which is in great clinical need. What's nice about our regulatory strategy and our commercial strategy is that, we have now demonstrated positive efficacy results in the treatment of MDD with a very differentiated product profile, and we're very much on track to filing our NDA. And our commercial prep is also underway, assuming that the product gets approved for MDD.

If TRD -- if the TRD trial is positive, or if it's negative or if it's mixed, it will not have any impact on our filing strategy. However, should the study be positive, it would be included in our NDA package, and we believe that it would be included in the clinical trial section of the package insert. It would not change the indication for the product. The indications would still be MDD. In order to get a formal label for treatment-resistant depression, we -- it is our understanding that the FDA would require a second trial in TRD. Importantly, as we've said before, that study could be a placebo-controlled trial.

So under the various scenarios, you're assuming that the study is positive, or if it's negative, or if it's somewhere in the middle, I think we're very well positioned. And we'll not speculate on what the results could be, but we certainly have scenarios planned internally. And we're very much looking forward to the readout before the end of the month. Yes, and just from a commercial perspective, I'll turn it over to Dave.

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David C. Marek, Axsome Therapeutics, Inc. - Chief Commercial Officer [4]

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Charles, thank you for the question. As we think about the commercial build out relative to TRD, as we've talked to physicians, the good news is that with MDD, we have the broader indication that already gives us access to a broad range of patients with MDD. And when we talk to physicians, given the clinical data that we've already, produces quite a high degree of enthusiasm towards AXS-05 already. So the question is, well, if we have positive results with TRD, how does that change? And I think the single greatest factor that we've seen in terms of talking with clinicians is that increases their enthusiasm for the patients that they would view for appropriate for AXS-05. So if you look at that, there's already a tremendous degree of enthusiasm for MDD, in general, and this would even further strengthen their conviction around either more patients or perhaps deepening their -- the range of patients that they would consider appropriate for AXS-05?

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Charles Cliff Duncan, Cantor Fitzgerald & Co., Research Division - Senior Analyst [5]

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Yes, and that's helpful. And that's actually consistent with our KOL diligence as well. So it doesn't seem like TRD is needed. It's a nice to have. Moving on just quickly to 07. I think, Herriot, you mentioned, 2H '20 for an NDA filing. And I read in the press release that it was 4Q. So is that just a slip? Or is it possible that the NDA filing for ASX-07 could actually come yet in the third quarter?

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Herriot Tabuteau, Axsome Therapeutics, Inc. - Founder, Chairman, CEO & President [6]

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It is possible that it come in the third quarter, our formal guidance is the fourth quarter, though. And the gating factor to the NDA filing is our safety database, which needs to include 100 patients treated for one year, and that we do have an open-label safety extension trial ongoing. And so we will need to anniversary that 100 patients at the 1-year point in order to file the NDA.

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Charles Cliff Duncan, Cantor Fitzgerald & Co., Research Division - Senior Analyst [7]

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Okay. Final question, commercial capabilities, you say, have been built, I guess, instead of what you've been doing maybe what you will do in terms of sizing and timing? Do you have any thoughts on the -- really, the size of the field force that you'll need to mount to effectively market these 2 drugs and in somewhat different or although somewhat the same markets of -- for prescribers?

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Herriot Tabuteau, Axsome Therapeutics, Inc. - Founder, Chairman, CEO & President [8]

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Yes. Regarding the commercial build-out, you're absolutely correct. When we think about building out our commercial team. We have a solid foundation now, and we want to make sure that we're adding the appropriate resources kind of at the appropriate stage as we march towards -- a potential launch is coming. When we think of field force, the benefit of clarity around both having MDD and migraine allows us to think in a highly efficient way. As you mentioned, around our prescriber base. And we have been looking at a variety of scenarios, in terms of field force sizing, structure, et cetera. I think, one of the kind of tenants that we've been using is how do we make sure that we have a very efficient launch where we're looking at using data and analytics in a very contemporary way to ensure that we are targeting those prescribers who have the highest willingness to prescribe and also ability to prescribe, given what we believe the payer environment could be. And so you could envision a very efficient and targeted launch with the ability to scale very quickly as prescriber adoption and access open up, we would be ready to scale very quickly. So think of an efficient sales force presence at the beginning and one that could scale very quickly.

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Charles Cliff Duncan, Cantor Fitzgerald & Co., Research Division - Senior Analyst [9]

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That sounds like more on that later on this year or into next year as we get closer with the NDA filing?

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Herriot Tabuteau, Axsome Therapeutics, Inc. - Founder, Chairman, CEO & President [10]

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Yes, correct. I mean, if you think about field force being hired about 6 months in advance. We want to keep our options active, as we approach that time period and make sure that we're looking at the latest data, the latest analytics, and that will help us solidify our final sales force sizing structure as we get a little closer to launch.

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Operator [11]

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Your next question comes from the line of Ram Selvaraju with HC.

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Raghuram Selvaraju, H.C. Wainwright & Co, LLC, Research Division - MD of Equity Research & Senior Healthcare Analyst [12]

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I was wondering, if maybe we could start with the narcolepsy program. Can you give us a sense of how large the scope of the clinical trial program is going to be in the pivotal setting, if you have a sense of that at this juncture and whether you expect to seek an SPA from the FDA regarding this? And then also on esreboxetine, I was wondering whether you have, at this point, a sense of whether the discussions with the FDA are going to center around the possibility of being able to file with the data that Pfizer has already generated, or if they are going to center around the need for additional clinical development work in the pivotal setting before you are able to potentially submit an application for that product candidate in fibromyalgia.

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Herriot Tabuteau, Axsome Therapeutics, Inc. - Founder, Chairman, CEO & President [13]

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Thanks, Ram, for the question. With regards to narcolepsy, we have gotten a feedback already in -- from our initial interactions with the FDA with regards to the Phase III plan. And before we actually launch that Phase III trial, actually, we're thinking two Phase III trials for narcolepsy. We really want to refine those plans and make sure that the FDA's thinking has not changed.

So stay tuned with regards to that. What I would say is, if you look at the results of our clinical trial, the results did reflect the pretty large treatment difference, even with approximately -- or the equivalent of 20 patients per arm, assuming that this had been a parallel design trial. With regards to what has been sized in previous trials for registration in cataplexy for example, those studies in the past have been in the range of around 30 to 40 patients per treatment arm. So that gives you a sense. And for, excessive daytime sleepiness, the numbers do go up from there in order to adequately power studies. But we'll be coming back to you, as we finalize the Phase III trial designs before we launch those studies.

With regards to esreboxetine, as I mentioned, we do intend to meet with the FDA to discuss the clinical development plan for esreboxetine going forward. And we think, this is a very differentiated product candidate, especially with regards to the breadth of symptoms of fibromyalgia that it can treat. And as a reminder, there are only 3 products that are currently approved to treat fibromyalgia in the U.S. So we're looking forward to those discussions. Obviously, as part of those discussions, we will like to understand the FDA's view of the data that has been generated to date and the appropriateness of those clinical trials, potentially for registration. And certainly that'll be an important point of discussion, at least as it relates to any further clinical trial that we may have to conduct.

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Raghuram Selvaraju, H.C. Wainwright & Co, LLC, Research Division - MD of Equity Research & Senior Healthcare Analyst [14]

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Okay. And then I wanted to ask a question regarding AXS-05 for the ADVANCE-1 target indication. So I was hoping, you could maybe comment on perspectives regarding what the path forward would be for AXS-05, assuming ADVANCE-1 is positive?

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Herriot Tabuteau, Axsome Therapeutics, Inc. - Founder, Chairman, CEO & President [15]

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Assuming ADVANCE-1 generates positive results. The assumption -- our working assumption is that, we would need one additional trial, one additional efficacy trial in Alzheimer's disease agitation. As a reminder, this is an area of high unmet medical need, there currently is no product that is approved to treat Alzheimer's disease agitation. We're happy with the progress of the clinical trial thus far, and this is a pivotal trial. And so it would make sense, obviously, that we would need an extra trial, in order to get the product approved, that has also been the feedback from the FDA. So that is our working assumption.

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Raghuram Selvaraju, H.C. Wainwright & Co, LLC, Research Division - MD of Equity Research & Senior Healthcare Analyst [16]

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Okay. And then just a couple other very quick ones. I remember that at one point, you were talking about the possibility of monetizing some of the intellectual property that Axsome holds in the area of, I believe, it was complex regional pain syndrome. Is there any update on that, or any way in which you see that situation evolving in the near term?

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Herriot Tabuteau, Axsome Therapeutics, Inc. - Founder, Chairman, CEO & President [17]

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You are correct, that we do have other assets, which are not core assets. So our core focus right now is our CNS pipeline, which consists of four product candidates in active clinical development. And the other assets, we're pretty excited about, and we think that there are potential sources of the non-dilutive funding. And so those have been placed into a separate business unit. We took in order to facilitate business development negotiations. We currently do not have any immediate update on that business unit. However, as things progress, if there is anything that is worth updating you on, we certainly will.

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Raghuram Selvaraju, H.C. Wainwright & Co, LLC, Research Division - MD of Equity Research & Senior Healthcare Analyst [18]

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Okay, perfect. And then this is just a question for David. I was wondering, if at this juncture, since a lot of the clinical efficacy data for AXS-05, and indeed, also 07 is out there. If you are receiving inbound interest from people who might potentially become part of the sales and marketing team at Axsome, and if you could perhaps comment just qualitatively on the caliber of these people, potentially some of their background and promoting drugs in the CNS and neuropsychiatric spaces. And to what extent this could potentially gives you confidence that you can establish a sales and marketing team that would be best-of-breed, as it were in the industry as and when 05 and 07 are approved?

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David C. Marek, Axsome Therapeutics, Inc. - Chief Commercial Officer [19]

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Well, thank you, Ram. I think, you're exactly right that as more and more information comes out and not only regarding those programs, but the company itself, and there are a couple of things that I think gets people really excited. If you have been in the depression space or the migraine space, and you're watching what's happening in terms of the evolution of new potential therapies, there are a lot of commercial people out there who are just as excited as our prescribers are around the potential of what we can do in the marketplace and how we can serve patients.

Also, I think there are commercial individuals who are very excited about the idea of a high analytics function. So we feel very good about the type of interest that we're receiving unsolicited, but also through contacts and relationships that we have in the industry, we feel very, very confident that we're going to be able to field a very strong sales force as well as other commercial talent.

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Operator [20]

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Your next question comes from the line of Yatin Suneja with Guggenheim Partners.

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Yatin Suneja, Guggenheim Securities, LLC, Research Division - MD & Senior Biotechnology Analyst [21]

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Mark, congrats on the new role. Just a couple questions on a few things. Maybe we'll start with TRD, first. Can you talk a little bit about how you are qualifying a nonresponder in the STRIDE study? And then in terms of the dose that you might be using in the open-label phase and we randomized phase, is there a reason to believe that you might be using a different dose in the open-label, what should be randomized phase? And then I have two follow-ups?

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Herriot Tabuteau, Axsome Therapeutics, Inc. - Founder, Chairman, CEO & President [22]

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Thanks, John, for the question, and I'll turn it over to Cedric to answer that.

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Cedric O'Gorman, Axsome Therapeutics, Inc. - SVP of Clinical Development & Medical Affairs [23]

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Yes, thanks. So first of all, with regard to treatment-resistant depression, and the -- I think, it was the definition of treatment-resistant depression that you asked about?

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Herriot Tabuteau, Axsome Therapeutics, Inc. - Founder, Chairman, CEO & President [24]

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Nonresponder.

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Cedric O'Gorman, Axsome Therapeutics, Inc. - SVP of Clinical Development & Medical Affairs [25]

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Nonresponder, right. So there is a standard approach that just as a reminder, within the open-label period, the patient is being treated with the program. And if they fail to respond then they're randomized. Now with regards to both the dosing and the criteria by which we measure responsors on response, the start that we disclosed that, once we have top line data, which will before -- just before the end of the month. So we're on track for that.

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Yatin Suneja, Guggenheim Securities, LLC, Research Division - MD & Senior Biotechnology Analyst [26]

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Okay, I understand. And then in terms of the -- I mean, on the dose, like have you manufactured your own dose to use in the -- in this study for Bupropion? Or are you using it commercially available? Because I remember in the ASCEND study, the dose that you have used is not commercially available. So you have to make your own dose. So help us understand the situation here. And then moving to the education side, can you maybe comment what were the reasons for delay, why it got postpone from first half to Q3?

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Mark L. Jacobson, Axsome Therapeutics, Inc. - COO & Secretary [27]

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So yes, and thanks for all the additional questions. With regards to drug product, which is used in clinical trials. The -- as a reminder, clinical trials do require that products that be blinded. And so therefore, the drug product for the various arms and in the various treatment periods have to be made, that they match in appearance. With regards to Alzheimer's disease agitation, and our updated guidance. So our previous guidance was the first half of this year. And we have talked about roughly midyear. That has not changed significantly. What we wanted to do was to build in some potential buffer. As you know, there are some external factors and not to talk too much about the impact of the coronavirus. But it is something that we do have to be mindful of given that Alzheimer's disease agitation is targeting a patient population, which is elderly. Right now, we've not seen any impact, but we do think that it is prudent to -- in our range of guidance, make sure that we take into account any potential eventualities.

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Operator [28]

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Your next question comes from the line of Marc Goodman with SVB.

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Marc Harold Goodman, SVB Leerink LLC, Research Division - MD of Neuroscience & Senior Research Analyst [29]

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Yes, Dave. I was wondering if you could talk about the migraine space a little bit. Obviously, a crowded market with some new players coming in, one that's already launched and doing pretty well. Talk about how you view the market, how that's changed and how you're thinking about differentiating your product when it comes out?

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David C. Marek, Axsome Therapeutics, Inc. - Chief Commercial Officer [30]

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Yes, well, Mark, thank you. Well, I think when we look at the migraine market, it's -- for those of us who've been associated with the migraine market for many years, it couldn't be a more exciting time and that's really good for clinicians, and it's good for patients. So certainly, we have long wanted to have more options to provide for patients and really help them better manage migraine. When you look at the frustration in the marketplace, the first place we go to when I say this often is to really clarify what are we solving for, in terms of the frustration or the unmet need in the marketplace.

When we talk to clinicians and patients consistently in the acute therapy setting, what physicians and patients are frustrated by is just pure efficacy. While there are a variety of needs out there, the one that rises to the top, and then our research, it was 8 out of 10 physicians prioritize, improved efficacy was their greatest unmet need. And so we built a clinical program around solving that issue of how can we improve efficacy. And I'm really proud of the clinical program we put together because I think with MOMENTUM, it really focused on answering the question that a clinician faces every single day. So you start with who's the right patient population with the greatest unmet need, and those are those who have failed at least one prior therapy. And those are the types of patients that we enrolled in MOMENTUM. Those that were treatment experienced and had a prior suboptimal response to their acute therapy. And again, those are the patients that physicians tell us that, they are dealing with day in and day out. And then we took the next step and said, okay, well, what are the treatment options? And what would they be faced with? And in MOMENTUM, we went head-to-head against rizatriptan, which is largely viewed as one of the most effective treatment options out there, certainly one of the most effective triptans. And by showing superiority, we think that we've really helped clinicians understand for their most difficult patients, where we have the next best step. And those are the data that we think will be highly differentiating in the marketplace. The right patient that they're dealing with, with the right clinical data that allows them to make a treatment decision that we think can deliver better efficacy, which is what the marketplace is looking for.

And I think, that's really the core of our differentiation and why we think there is tremendous enthusiasm for AXS-07 and you may recall in our mindset survey, we ask migraine treating physicians their degree of enthusiasm towards AXS-07, should we be superior to rizatriptan, and I think, we've talked about those results, where the vast majority of physicians to say they would prescribe AXS-07 over currently available treatments, but also over the emerging treatments as well. So we see a very strong commercial opportunity to benefit patients with AXS-07.

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Marc Harold Goodman, SVB Leerink LLC, Research Division - MD of Neuroscience & Senior Research Analyst [31]

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And how does the INTERCEPT study play into it?

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David C. Marek, Axsome Therapeutics, Inc. - Chief Commercial Officer [32]

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Well, I think what INTERCEPT does is, it helps inform and provides a greater foundation for how they treat and direct patients in the real-world setting. We know in the typical clinical trials, we need patients to wait until their pain is moderate to severe, and those data are very helpful for registration processes et cetera. When we looked at how clinicians typically tell their patients to administer their acute therapy, they say, take it as the first sign of migraine pain. And so we think that INTERCEPT will provide kind of the real-world setting information for how we think this product will perform in a manner that they'll likely instruct patients to administer. So we think it's additional information that's not required for the NDA filing, but will be important in the setting, as we educate physicians around the utility of AXS-07.

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Marc Harold Goodman, SVB Leerink LLC, Research Division - MD of Neuroscience & Senior Research Analyst [33]

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And the last question, as you're thinking about the sales force, is it going to be one sales force that sells both products? The commercial product and this one. Will it be two separate ones? How much overlap is there?

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David C. Marek, Axsome Therapeutics, Inc. - Chief Commercial Officer [34]

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Well, as mentioned earlier, yes, we will still look at options around looking at both migraine and depression and how we -- the size and structure of that deal for us. So -- while we have a variety of options, we haven't landed specifically, we'll do more work around that. I will say, though, we will look for efficiencies across promoting both migraine and depression. There is some overlap in -- mainly in the primary care setting, as you can imagine, with some physicians who are high prescribers of both, and we would certainly take advantage of that synergy, the degree to which we're still evaluating as we look at our options. So we haven't landed on one field force or two, but what we will do is, as we make that decision, look for the greatest efficiencies that we can glean.

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Operator [35]

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Your next question comes from the line of Matt Kaplan with Ladenburg.

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Matthew Lee Kaplan, Ladenburg Thalmann & Co. Inc., Research Division - MD & Head of Healthcare Equity Research [36]

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Congrats on the progress. Just wanted to zero in a little bit more on STRIDE-1, given the near-term readout of results expected there. So can you please remind us of the sizing and the powering of the study? I guess, given the fact that you continued enrollment to build your safety database, can you give us a sense of that?

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Herriot Tabuteau, Axsome Therapeutics, Inc. - Founder, Chairman, CEO & President [37]

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Matt, thanks for the question. So in terms of sizing, the last update that we gave was that the study had randomized approximately 300 patients, that was at the end of November. And so the final number will be north of 300. In terms of the powering of the trial, the study was powered at the 90% level to detect an effect size of somewhere between 0.3 and 0.35, which is in line with the effect sizes of anti depressants sort of the jump in work and that have been currently approved.

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Matthew Lee Kaplan, Ladenburg Thalmann & Co. Inc., Research Division - MD & Head of Healthcare Equity Research [38]

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Great. And then in terms of the open-label extension study for 05, that's enrolling both MDD and TRD patients. How is that progressing? And can you give us an update in terms of how many patients you expect in -- ultimately in the open-label extension?

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Herriot Tabuteau, Axsome Therapeutics, Inc. - Founder, Chairman, CEO & President [39]

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That study is progressing well. As you know, that is a gating factor to us filing an NDA. We do need to build the safety database of 300 patients treated for 6 months and 100 patients treated for 1 year, and we're on track to do that. So we're pretty happy with the way that, that is currently enrolling. It is enrolling at a pace that will allow us to meet our guidance of filing NDA before the end of the year.

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Matthew Lee Kaplan, Ladenburg Thalmann & Co. Inc., Research Division - MD & Head of Healthcare Equity Research [40]

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Okay. And I guess, just going back to following up on the questions, that we had on AXS-14 esreboxetine for fibromyalgia. Can you give us a sense, in terms of the potential regulatory paths forward for esreboxetine in fibromyalgia?

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Herriot Tabuteau, Axsome Therapeutics, Inc. - Founder, Chairman, CEO & President [41]

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So we first want to meet with the FDA. As a reminder though, esreboxetine has completed 2 efficacy trials of significant size. One was a Phase III trial, which enrolled and randomized over 1,000 patients. That study was positive, and there was also a Phase II trial, which was also positive. Both of these studies were randomized, double-blind placebo controlled trials. And so we certainly will want to get the FDA's feedback on those clinical studies. Those studies were run by Pfizer. So we want to make sure that we get feedback from the FDA, in terms of what else would be required in order to file an NDA. We will not speculate on what those requirements could be, and until we meet with the agency.

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Operator [42]

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Your next question comes from the line of Bert Hazlett with BTIG.

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Robert Cummins Hazlett, BTIG, LLC, Research Division - MD & Biotechnology Equity Research Analyst [43]

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I just have a couple of quick ones. Just with the -- you have a lot on your plate with a number of different indications. Where does AXS-05 smoking cessation indication sit and in terms of urgency and in terms of corporate priorities?

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Herriot Tabuteau, Axsome Therapeutics, Inc. - Founder, Chairman, CEO & President [44]

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Thanks for the question. So you are correct that AXS-05 does have a number of potential indications, and we do have a lot on our plate. It was -- one of the challenges that we're facing and that we're keeping in mind, is the need to focus, while at the same time, avail ourselves of the various opportunities in the pipeline. So our priorities, clearly, this year are to make sure that we file our NDAs in a timely manner and also that those filings are of significant quality.

So that is our top priority, which is the -- our NDA filing. And then beyond that, this is advancing our -- a product candidate in narcolepsy into Phase III, and are based upon the very positive Phase II data. So that's the -- that's priority #2. And the of course getting a feedback on AXS-014, given the -- a very positive efficacy results there. And then we absolutely will be meeting with the FDA this year, with regards to smoking cessation to delineate a path forward there and understand what the clinical development -- what the further clinical development for that indication will look like.

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Robert Cummins Hazlett, BTIG, LLC, Research Division - MD & Biotechnology Equity Research Analyst [45]

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Okay. Could you just remind us of the CMC progress or characteristics or status of 05 at this point?

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Herriot Tabuteau, Axsome Therapeutics, Inc. - Founder, Chairman, CEO & President [46]

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So the clinical trial material for AXS-05 -- for our AXS-05 trials have been made at commercial scale. So we're in very good shape there and they've also been made at the contractor manufacturer that will be manufacturing the commercial supply. They're all registration batches, which must be made, specifically for the purpose of submitting an NDA. And so that work is underway.

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Robert Cummins Hazlett, BTIG, LLC, Research Division - MD & Biotechnology Equity Research Analyst [47]

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And then just one on migraine. Does it make sense to have a study in combination with the long-acting CGRP and with an acute migraine therapeutics, such as you have with 07? Does that make any sense commercially at all?

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Herriot Tabuteau, Axsome Therapeutics, Inc. - Founder, Chairman, CEO & President [48]

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We certainly don't see any reason why AXS-07 could not be administered with other types of therapies. Right now, we're focusing on getting the product approved, and it will be interesting to see how clinicians eventually choose to use it, as part of their own material.

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Operator [49]

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Your next question comes from the line of Myles Minter with William Blair.

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Myles Robert Minter, William Blair & Company L.L.C., Research Division - Analyst [50]

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Congrats, Mark, on the new position at the company. Just the first question is on ADVANCE, 80% enrolled, pushed the guidance slightly out to third quarter, I'm wondering whether you're proactively cautioning your physicians to enroll new patients into that trial currently, given the unfortunate risk with COVID-19 at the moment in that highly susceptible population? And also, you mentioned 80% enrollment. I'm wondering what proportion of patients were enrolled in that trial at the December 2018 timeline where you discontinued enrollment into the Bupropion alone arm?

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Herriot Tabuteau, Axsome Therapeutics, Inc. - Founder, Chairman, CEO & President [51]

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So thanks for the questions, Myles. The -- with regards to cautioning physicians around enrollment into the trial. I mean, we're not currently doing that. But what we are doing is monitoring very closely from the clinical trial sites and the information that they have, any concerns that they might have with regards to enrollment. Currently, we're not hearing anything that is of concern, but it is a situation that we're monitoring pretty closely. And with regards to the percentage of patients at the time of the -- an interim analysis, at that, the interim analysis, as a reminder, was conducted at the 30% enrollment point. So that should allow you to get a sense of what the subsequent enrollment was after that. So it was enrolled -- so 30% enrollment point. And remember, the size of the trial was 435 subjects total.

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Myles Robert Minter, William Blair & Company L.L.C., Research Division - Analyst [52]

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That's helpful. And then maybe a question for Dave. Just some KOL diligence, we've been doing, suggest that relation specialist centers, they've started using SSRIs, rather than waiting for 6 to 8 weeks for a response there. The China see an early responser 2 weeks with those drugs, maybe a 20% improvement to predict whether those patients are truly going to have a response like they did on the track. Just looking at the data from GEMINI and ASCEND and the early rapidity of response. I'm wondering in your conversations with clinicians, both in specialty centers and in PCPs, whether a focus on their behalf is actually on the rapidity of effects. And maybe not so much focused on MADRS, but maybe on some of those patient, quality of life question is which are more routinely used in the clinic? What are you hearing from the community? What are they most excited about this product, cheers.

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David C. Marek, Axsome Therapeutics, Inc. - Chief Commercial Officer [53]

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Myles, thank you for the question. We are hearing some of the same in our research around what physicians are looking for. And when we put the current data that we have for 05 in front of specialists, one of the things that really -- that they light up about is really around the rapidity of effect. But also what they get excited about is kind of the reason to believe that. And when you look at the NMDA plus multimodal activity, they're really hungry for a different pathway that kind of gets them off the merry go round of the SSRIs, SNRIs, and they're looking for another pathway, and one that would be potentially delivered in a very patient-friendly way.

So that gives them reason to really sit up and look at our data. And then when they see the rapidity of effect and the robust nature of effect, that's where they start to look at this as certainly for patients that they've struggled with, who are treatment experienced. But also, they start to move up very quickly earlier to think about patients where they would want that rapidity of effect and moving it up earlier as kind of foundational therapy. So we're very excited about the response we're getting from the clinical profile. We think, the more there is focus on rapidity of effect is good for clinicians, and it's very good for patients, and we think that we're going to be able to fit nicely in that unmet need.

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Myles Robert Minter, William Blair & Company L.L.C., Research Division - Analyst [54]

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Cool. Just a follow-up, does that logic change between specialist centers that are most likely monitoring their patients consistently over that early treatment period versus PCP that might just prescribe, and then come back 6 weeks later and not actually monitor that patient's response early on?

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David C. Marek, Axsome Therapeutics, Inc. - Chief Commercial Officer [55]

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Yes. I mean, I don't have the data to answer that question. But intuitively, I think it makes sense that those centers who are very actively monitoring rapidity of effect and the types of patients that they may be seeing could be very different in terms of sense of urgency that we might see in the -- with the primary care specialists out in the field. I think the desire is the same that everybody wants their patients to get better sooner. But I think the adoption curve could be different across those 2 areas of treatment.

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Operator [56]

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There are no further questions at this time, I will turn the call back over to the presenters for closing remarks.

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Herriot Tabuteau, Axsome Therapeutics, Inc. - Founder, Chairman, CEO & President [57]

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Well, thank you all for joining us on the call today. Axsome is committed to accelerating the invention, development and adoption of life changing medicines for the many people facing unsatisfactory treatments for CNS diseases. We anticipate a number of important milestones over the balance of the year, and we look forward to updating you on our progress.

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Operator [58]

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This concludes today's conference call. You may now disconnect.