Q4 2019 Earnings Call
Operator: Later, there will be a question and answer session, and instructions will be followed at that time. 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 of Axsome Therapeutics. Please go ahead.
Later, there will be a question and answer session and instructions will be followed at that time.
A reminder, today's conference call is being recorded I would now like to Kinda conference over to your whole Marc Jacobs Chief operating officer at Accenture repeat next please go ahead.
Thank you operator, good morning, and thank you all for joining us on todays conference call.
Mark L. Jacobson: 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
Our earnings press release, providing a corporate updates and details of the Companys financial results for the fourth quarter and full year ended December 30, Onest 2019.
So why are short time ago and is available on our website excellent dot com.
During today's call, we will be making certain forward looking statements.
Mark L. Jacobson: certain forward-looking statements.
Mark L. Jacobson: These statements may include statements regarding, among other things, the efficacy, safety, and intended utilization of our investigational agents, our clinical and non-clinical 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 investment. 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 statement. 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 Merrick, Chief Commercial Officer; and Nick Pizzie, Chief Financial Officer. Ariel will first provide a review of significant corporate and clinical developments for Axsome over the course of the past year and describe upcoming milestones.
These statements May include statements regarding among other things the efficacy safety and intended utilization of our investigational agents are critical and clinical plans.
Our plans to present whole 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 could 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 report Youre cautioned not to place undue weight on these forward looking statements in the company disclaims any obligation to update such statements.
Joining me on the call today are Dr. Aereo to Butoh, Chief Executive Officer, Dr. Cedric O'gorman.
Senior Vice President of clinical development, and medical Affairs things Merrick, Chief Commercial officer, and Nick PZ Chief Financial Officer.
Area will first provide a review of significant corporate and clinical developments for axiom over the course of the past year.
And describe upcoming milestones following that Nick will review our financial result.
Mark L. Jacobson: Following that, Nick will review our financial results, and we will then open the line for questions. I shall now turn over the call to Nick.
We will then open the line for questions I show no turned over the call to area.
Ariel: D'Addario. http://www.youtube.com.au Thank you, Mark.
Thank you Mark good morning, everyone and thank you all for joining excellent therapeutics fourth quarter and full year 2019 results conference call.
Ariel: 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. The many important achievements of 2019 included positive NDA-enabling clinical trial readouts for AXS05 in depression and for AXS07 in migraine, propelling Axsome potentially towards the commercial stage as early as next year, in addition. Our AXS 12 product candidate for narcolepsy is progressing to Phase 3 based on positive Phase 2 results, and through our collaboration with Pfizer early this year, we expanded our Phase 3 pipeline with the addition of AXS 14 for the treatment of fibromyalgia.
We 19, what's a transformative year for axle as we generated an important clinical data.
All of our investigational medicines.
Which were developing for serious and difficult to treat CNS disorders.
The many important achievements of 2019 included positive N D, enabling clinical trial Readouts for access to five in depression and for excess of seven in migraine propelling excellent potentially towards commercial stage as early as next year.
In addition, our.
Our next 12 product candidate from all collect fees progressing the phase three based on positive phase two results.
Throughout collaboration with Pfizer early this year, we expanded our phase three pipeline. The addition of access for team for the treatment of fibromyalgia.
Ariel: All in all, we now have four differentiated CNS product candidates in clinical development, all of which have demonstrated efficacy in control trials, two of which are pre-NDA, with the other two entering or having completed Phase 3 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.
All in all we now have for differentiated CNS product candidates in clinical development.
All of which have demonstrated efficacy in control trials, two of which all pre India.
The other two entering or having completed phase three 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 that change the current standard of care, but difficult to treat rain disorders and transformed the lives of patients living with these conditions.
We expect 2020 to be a year of continued operational clinical and regulatory progress.
Ariel: We expect 2020 to be a year of continued operational, clinical, and regulatory progress. Preparations are underway for our planned NDE filing of XSO5 for the treatment of major depressive disorder, or MDD. AXSO5 has the potential to be the first and only oral and MDA receptor antagonist with multimodal activity for the treatment of depression. In December, we announced positive results from a Phase 3 GemIIni trial of AXSO5 in patients with confirmed moderate to severe MDD. In this study, AXSO5 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 ASCENT trial of AXS05 in MDD, support an NDA filing for AXS05 in the treatment of MDD, and we are on track to file this NDA in the fourth quarter of 2020.
Preparations are underway for our plan to enter you filing the access to five in the treatment of major depressive disorder or MDD.
Excess of fiber has the potential to be the first and only oral and India receptor antagonist with multi modal activity for the treatment of depression.
In December we announced positive results from a phase three Gemini trial on the access to five in patients with confirmed moderate to severe M. D D.
In this study the excess of five met the primary endpoint by rapidly substantially and statistically significantly improving symptoms of depression as compared to placebo.
The positive result from the Gemini trial, along with the previously complete assent trial of excess all five in MPD support and indeed filing for access to five in the treatment of MTD.
And we're on track to file the San Diego and the fourth quarter of 2020.
Ariel: AXS05 has been granted breakthrough therapy designation for the treatment of MDD. Preparations are also underway for our planned NDA filing of AX07 for the acute treatment of migraines. AXS07's multi-mechanistic approach is designed to provide enhanced efficacy as compared to currently available treatments. In December, we announced positive results from the Phase 3 Momentum Trial of XSO7 in migraine patients with a history of inadequate response to prior acute treatments.
Yeah. So five has been granted breakthrough therapy designation for the treatment of MDD.
Preparations also underway for our planned indeed filing or the ACA. So seven in the acute treatment of migraine.
Yes, so seven to 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 three momentum trial of excess seven in migraine patients with a history of inadequate response to prior acute treatment.
Ariel: In this study, AXS07 met the co-primary and key secondary endpoints by significantly relieving migraine pain as compared to placebo and as compared to the active comparator, Ryza Krypton. The positive results from the MOMENTUM trial support an NDA filing for AXS07 in the acute treatment of migraine, and we will remain on track to file this NDA in the second half of 2020. With these two planned NDA filings, Axsome is on track to transition to the commercial stage potentially as early as next year. And to that end, we have been building out our commercial capability.
In this study you access will seven met coal primary and key secondary endpoints I significantly relieving migraine pain as compared to placebo.
And as compared to the active comparator rise a trip that.
The positive result from the momentum trial support in India filing for excess so seven in the acute treatment of migraine and we remain on track to file the San Diego in the second half 2020.
With these two plan and the filings acts on me is on track to transition to commercial stage potentially as early as next year.
To that end, we have been building out our commercial capabilities.
In December we also announced positive top line, resulting from a phase two cancer trial of excess well in the treatment of narcolepsy.
Ariel: In December, we also announced positive top-line results from a Phase 2 concert trial of AXS12 in the treatment of narcolepsy. In this study, AXS12 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 AXS12 with the potential to address all the key symptoms of narcolepsy. Based on these positive results, AXS 12 is scheduled to enter Phase 3 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 non-clinical data and intellectual property for roboxetine, the active pharmaceutical ingredient in AXS12.
In this study excess well significantly reduce the 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 excess 12, what's the potential to address all the key symptoms with narcolepsy.
Based on these positive results. The excess 12 is scheduled to enter phase three clinical trials in norcal EPCI in the second half of this year.
We expect the development of excess well to accelerate as a result of our agreement with Pfizer, which we announced in January.
This agreement covers an exclusive U.S. license, the pfizer's clinical and non clinical data and intellectual property for a box the team the active pharmaceutical ingredient in excess 12.
Disagreement also expands our late stage Sina pipeline by providing us exclusive rights to develop and commercialize a new product candidate Astra box the team or excess 14 in the U.S. for the treatment of fibromyalgia.
Ariel: This agreement also expands our late-stage CNS pipeline by providing us with exclusive rights to develop and commercialize a new product candidate, S-Riboxyteam or AXS-14, in the U.S. for the treatment of fibromyalgia. AXS-14 has previously demonstrated positive and statistically significant results in a phase 3, and in a phase two trial for 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.
Yes, that's 14.
As previously demonstrated positive and statistically significant results in a phase three.
And in a phase two trial and the treatment of fibromyalgia.
In addition to its effects on pain and function excess 14 demonstrated in both of these studies.
Effect on fatigue difficult to treat symptom of this condition.
Axle implants to meet with the FDA this year.
Discuss the further clinical development of the excess 14 for fibromyalgia.
Turning now to our ongoing efficacy trials.
We recently completed randomization into our stride one trial with access to five in treatment resistant depression.
Nick Pizzie: Axsome plans to meet with the FDA this year to discuss the further clinical development of AXS14 for fibromyalgia. But returning now to our ongoing efficacy trial. We recently completed randomization into our Stride 1 trial of XSO5 in treatment-resistant depression and into our Intercept trial of XSO7 in the early treatment of migraines. We remain on track for top-line results from both of these trials before the end of this month. Our Advance 1 Phase 2-3 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 NDF filings for AXS05 in MDD and AXS07 in migraine, anticipated in the fourth quarter.
And into our intercept trial in excess of seven.
In the early treatment of migraine.
We remain on track the topline results from both of these trials before the end of this month.
Our advance one phase two three trial have access to five in the treatment of agitation associated with whole times disease is now more than 80% enrolled.
And based on this trend we expect topline 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 idea of filing sleek special five an MTD and the excess of seven in migraine anticipated in the fourth quarter.
Topline results from stride, one trial in excess of five in PRD and the intercept trial in excess of seven in migraine this quarter.
Topline results from the advanced one trial it makes us feel five an old timers disease agitation in the third quarter.
And initiation of phase three trials of excess 12 in Norcal FC in the second half of this year.
I would also like to recognize and congratulate more jacobson on his recent appointment to Chief operating officer.
Nick Pizzie: Top-line results from the Stride 1 trial of XSO5 in TRD and the Intercept trial of XSO7 in migraine this quarter. Top-line results from the ADVANCE-1 trial of XSO5 and Alzheimer's disease agitation in the third quarter, and initiation of phase 3 trials of AXS12 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 the financial update for the full year of 2019.
Mark has been a valued member of the X. I'm team since 2014.
And served as senior Vice President of operations since 2017.
I will now turn the call over to Nick to provide financial update full year of 29 team.
Thank you area 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 31st 2019 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 excess so five in excess of seven open label Safety Studies. In addition to the ongoing progress of the stride one in advance ones.
Nick Pizzie: Thank you, Ariel, and good morning, everyone. I will focus on key highlights from the quarter and provide some financial guidance. R&D expenses were $19.2 million for the quarter ended December 31, 2019 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 AXS05 and AXS07 Open Label Safety studies, in addition to the ongoing progress of the STR 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, along with the build out of the commercial.
Yes.
DNA expenses were $5.2 million for the quarter ended December 31st 29 team 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 $200 million SCB 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 or anticipated operations based on our current operating plan for at least two years.
That concludes our fourth quarter 2019 financial review I will now turn the call back to Mark to lead the acuity disgusting.
Nick Pizzie: In December, we completed an underwritten public offering that yielded aggregate gross proceeds before deducting offering expenses of approximately 200 million dollars. SVP Larrink 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 two years.
Thank you Nick.
Operator may we please have ever first question.
As a reminder, if you like to ask the question Press Star one on your telephone keypad to withdraw your question I Funky Your first question.
For the nine of Charles Duncan.
I'm sorry, that's Charles Please go ahead.
Good morning, Aereo and team congratulations on a very good year and last year, and what could well 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.
Mark L. Jacobson: That concludes our fourth quarter 2019 financial review. I will now turn the call back to Mark to lead the Q&A. Thank you, Nick.
For access so five [laughter] relative to the upcoming TRD results I guess I'm wondering.
What you're thinking about different scenarios that could be and the impact.
Operator: Operator, may we please have our first question? As a reminder, if you'd like to ask a question, press star 1 on your telephone keypad. To withdraw your question, press the phone key. Come to the line of Charles Duncan with Cantor Fitzgerald, please go ahead. Good morning, Ariel 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 AXS05 relative to the upcoming TRD results, I guess I'm wondering what you think about the different scenarios that could be and the impact on the regulatory and or commercial strategy for AXS05. Can you lay out a couple of scenarios of either the drug clearly working, the drug maybe working, or the drug not working in TRD?
Regulatory and or commercial strategy for access. So five can you can you lay out a couple of scenarios of.
I'll either the drug clearly working the drug may be working or the drug not working integrity.
[noise]. Thank you Charles.
With regards to a tier di na what's what's nice about that's that study is is that it is targeting a patient population, which is in a in great clinical need.
What's nice about our regulatory strategy in our commercial strategy is that now we have now demonstrated positive efficacy results in the treatment of mbd was very differentiated product profile and I'm very much on track to filing on <unk> commercial prep is also a underway.
Assuming that a product gets approved for a for M.D.
If you already or if it's your de trial is positive or if it's a orbitz negative orbitz mixed it will not have any impact on our filing strategy. However should the study be a the positive on it would be included in our Indian package and we believe that it would be.
Ariel: Thank you, Charles. With regard to TRD, what's nice about that study is that it is targeting a patient population that 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 MZD with a very differentiated product profile, and we're very much on track to filing our NDA. Our commercial preparation is also underway, assuming that the product gets approved for MZD.
Included in the clinical trial section of the package insert it would not change the indication.
For the for the product the indication would still be.
The MDB in order to get a woman label for treatment resistant depression. We it is our understanding that DFT. It would require a second trial in T or D. A importantly, as we've said before that's that he could be a placebo controlled trial.
Ariel: 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, if the study is 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 indication would still be MZD.
So you know under the various scenarios I'm, assuming that that that.
This study is a is positive or if it's a negative or if it's somewhere in the middle I think we're very well positioned I'm not speculate on what the results could be but we certainly have a scenario as planned.
Ariel: In order to get a formal label for treatment-resistant depression, 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, you know, 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. We will 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 day. From a commercial perspective, I'll turn it over to Dave.
Internally and I would very much looking forward to the reality and before the end of the month.
Okay, most bench testing, yeah, and it just from a commercial perspective, you know I'll turn it over to today's.
Good morning, Charles Thank you for the question, but it was we think about the commercial build out relative to TRD. There as we've talked to physicians you know the good news or is that with M.D.D.. We have the broader indication that already gives us access to a broad range of.
Of patients with them with M.D. and when we talk to physicians given the the clinical data that we've already producers have quite a high degree of enthusiasm towards access so five already.
So the question as well if we have positive results with TRG, how does that change and and I think the single greatest factor that we've seen in terms of talking with clinicians it increases their enthusiasm.
Dave Merrick: Good morning, Charles. Thank you for the question. As we think about the commercial build-out relative to TRD, as we've talked to physicians, you know, 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 produced, there's quite a high degree of enthusiasm for AXSO5 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 it increases their enthusiasm for the patient that they would view as appropriate for AXS05. 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 the range of patients that they would consider appropriate for AXS05.
For the patients that they would view for appropriate for access. So five so if you look at that there's already a trend tremendous degree of enthusiasm.
Four M.D.D. in general and this would even further strengthen their conviction around either more patients or or perhaps a deepening their the range of patients that they would consider appropriate for a successful five.
Yeah. That's helpful. That's actually consistent with our kalo diligence as well so it doesn't seem like TRD is needed. It's a nice to have moving on just to quickly to O seven.
I think area you mentioned two age 24 in San Diego filing and I read.
The press release said it was for Q. So is is that Chester slip or is it possible that are the indeed filing for access so seven could could actually come yet in in the third quarter.
Charles Cliff Duncan: Yeah, that's helpful. That's actually consistent with our KOL diligence as well. So it doesn't seem like TRD is needed. It's nice to have, though.
<unk>.
It is possible, but did it come in the third quarter. Our formal guidance is the fourth quarter, though and on the gating factor to the India filing is a our safety database, a which that needs to include 100 patients treated for one year and that we do have an open label safety extension trial.
Ariel: Moving on just too quickly to 07. I think, Ariel, you mentioned 2H20 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 AXS 07 could actually come in the third quarter?
Our ongoing and ER and so we will need to anniversary that hundred patients at the one your point a in order to file the India.
Okay Fine I'm question commercial capabilities, he say have been being built I guess.
Ariel: It is possible that it will come in the third quarter. Our formal guidance is for the fourth quarter, though, and the gating factor for the NDA filing is our safety database, which needs to include 100 patients treated for one year. We do have an open-label safety extension trial ongoing, and so we will need to anniversary that 100th patient at the one-year point in order to file the NDA.
Instead of what you've been doing maybe what you will do in terms of sizing and timing do you have any thoughts.
And you know really the size of the field force that you'll need to Mount to effectively market. These these two drugs and in somewhat different or although somewhat the same.
You know markets.
So for prescribers.
Yes regarding the commercial build out you're you're absolutely correct. You know 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 a the appropriate resources kind of at the appropriate stage as we March towards Ah.
Charles Cliff Duncan: 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, you know, really the size of the field force that you'll need to mount to effectively market these two drugs in somewhat different or although somewhat the same markets for prescribers?
Potential launches coming when we think a field force did benefit of clarity around both having M.D.D. and migraine allows us to think in a highly efficient way as you mentioned around our prescriber base and we have been looking at.
Dave Merrick: Yeah, regarding the commercial build out, you're absolutely correct. You know, 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 potential launches. When we think of field forests, 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 fields for sizing, structure, etc., I think one of the kind of tenets that we've been using is, how do we make sure that we have a very efficient launch?
Variety of scenarios.
In terms of fields, where sizing structure et cetera, I think one of the I'm 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 prescribing also ability to prescribe given what we believe the payer or a 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 to open up a we would be ready to scale very quickly so think of it efficient salesforce presence at the beginning.
Dave Merrick: 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 imagine 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 salesforce presence at the beginning and one that could scale very quickly.
And one that can scale very quickly.
That's sounds like more on that later on this year or into next year is as we get closer with us and da filings.
Yeah, correct I mean, if you think about field force being higher you know about six months in advance we want to keep our options active you know as we approach that that time period and make sure that we're looking at the the latest state of the latest analytics and that will help us solidify our.
Charles Cliff Duncan: But sounds like more on that later this year or into next year as we get closer with the NDA filings.
Our final salesforce size and structure as we get a little closer to one.
Sounds good thank you for taking my questions.
Dave Merrick: Yeah, correct. If you think about Field Force being hired, you know, about six months in advance, we want to keep our options open, you know, 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 Salesforce sizing structure as we get a little closer to launch.
Thank you.
Your next question comes from the line of from stuff that Roger with H.C. Please go ahead.
Thanks, very much for taking my questions that 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 a program is going to be in the pivotal setting. If you have a sense if that at this juncture and whether you expect to seek NSP aid from the FDA regarding this.
Charles Cliff Duncan: Sounds good. Thank you for taking my questions.
Mark L. Jacobson: Thank you.
Raghuram Selvaraju: Your next question comes from the line of Raghuram Selvaraju, with HC. Please go ahead. Thanks very much for taking my questions. 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 any sense of that at this juncture, and whether you expect to seek an SBA from the FDA regarding this? And then also on estroboxetine, 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're 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.
Also on a extra box the team 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're going to center around the need for additional clinical development.
The work of the pivotal setting our before you were able to potentially submitted an application for that product candidate in fibromyalgia.
Okay. Thanks, Robert for the question with regards to narcolepsy.
We we have gotten feedback already in from our initial interactions with the FDA with regards to the phase three plan and before we actually launch on that phase three trial actually we're thinking a two phase three trials for narcolepsy, we will want to refine those plans and.
Make sure that that the FDA thinking has not changed so stay tuned with regards to that.
I would say is if you look at the results of our clinical trial results.
Ariel: Thanks, Ron, for the question. With regard to narcolepsy, we have already gotten feedback from our initial interactions with the FDA with regard to the Phase III plan. And before we actually launch that Phase III trial, actually, we're thinking of two Phase III trials for narcolepsy. We will want to refine those plans and make sure that the FDA's thinking has not changed, so stay tuned with regard to that. What I would say is, if you look at the results of our clinical trial, you see, the results did reflect a 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 regard 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, you know, and for excessive daytime sleepiness, the numbers do go up from there in order to adequately power studies.
Uh-huh did reflect a pretty large treatment difference so even with.
Approximately or the equivalent of 20 patients per arm assuming that this this had been he parallel designing trial.
With regards to.
What has been what has been sized a in previous trials.
For registration in Cataplexy for example, no studies in the past had been in the range of around 30 to 40 patients per treatment arm. So that gives you a sense.
You know in for a excessive daytime sleepiness or the numbers do go up from there in order to adequately powered studies.
You know will will be coming back to you as we finalize.
The phase three trial the trial designs on before we launch in those studies.
With regards to asked for box a team.
I mentioned earlier, we do intend to meet with the FDA discuss the clinical development plan for extra box team going forward. We think this is a very differentiated product candidates.
Ariel: But, you know, we'll be coming back to you as we finalize the phase three trial designs before we launch those studies. With regard to S-ruboxetine, as I mentioned, we do intend to meet with the FDA to discuss the clinical development plan for S-ruboxetine going forward. We think this is a very differentiated product candidate, especially with regard to the breadth of symptoms of fibromyalgia that it can treat. As a reminder, there are only three products that are currently approved to treat fibromyalgia in the U.S.
Especially with regards to Ah Ah the breadth of symptoms apart when my Algea that that it can treat it as a reminder, there are only three products that are currently approved to treat fibromyalgia in the U.S. So we're looking forward to those discussions you know obviously as part of those discussions and we will.
HM we will like to understand.
On the FDIC that view of the data that has been generated to date and on the appropriateness of those Oh clinical trials potentially for registration you're certainly on that'll be an important point of discussion at least as it relates to a in further clinical trial that than me.
Ariel: So we're looking forward to those discussions. Obviously, as part of those discussions, we would 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. Certainly, that will be an important point of discussion, at least as it relates to any further clinical trials that we may have to conduct.
Then we may have to conduct.
Okay. Thanks for that clarity and then I wanted to ask a question regarding access so five for the advance one target indication. So I was hoping you could maybe comment on perspectives regarding what the path forward would be for access so five assuming advance one is.
Raghuram Selvaraju: Okay, thanks for that clarification. And then I wanted to ask a question regarding AXS05 for the Advance 1 target indication. So I was hoping you could maybe comment on perspectives regarding what the path forward would be for AXS05, assuming Advance 1 is positive.
Yes.
Assuming assuming advance one generates positive results.
The assumption are working assumption is that we would need a one additional.
Trial, one additional efficacy trial in old timers disease agitation. There's a reminder, this is an area of high unmet medical need. There currently has no product that is approved to treat old-timers disease agitation.
Ariel: Assuming Advance One generates positive results, our working assumption is that we would need one additional FFC trial in Alzheimer's disease agitation. As a reminder, this is an area of high unmet medical need.
You know, where we're happy with the progress of of the clinical trial, thus far and this is a pivotal trial and and so was it.
Ariel: There is currently 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.
It would make sense, obviously that would need an extra trial in order to get the product approved that has also been the feedback from the FDA. So that that is on working assumption.
Okay, and then just a couple other very quick ones I remember that at one point or you were talking about the possibility of monetizing some of the intellectual property that acts home holes in the area of I believe it was complex regional pain syndrome is there any update on that.
Raghuram Selvaraju: 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 Axome 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?
Or any way in which you see that situation evolving in the near term.
Okay.
You are correct that Oh, we do have a of other assets, which are not core assets talk more focus right. Now we saw our CNS pipeline, which consists of for our product candidates an active clinical development and these other assets up we're pretty excited about high and we think that are they all potential.
Ariel: 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. These other assets are pretty exciting, and we think that they are potential sources of non-dilutive funding, and so those have been placed into a separate business unit in order to facilitate business development negotiations. We currently do not have any immediate update on that business unit, but as things progress, if there is anything worth updating you on, we certainly will.
Sources.
Of non dilutive funding and and so you know those have been placed in two separate business unit. We took in order to facilitate a business development negotiations.
We currently do not have any any immediate update I'm on a on on on that business unit. However.
As a as things progress on if there is anything worth updating you on certainly will.
Dave Merrick: Okay, perfect. And then this is just a question for David. I was wondering if at this juncture, since, you know, 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, you know, some of their background in promoting drugs in the CNS and neuropsychiatric spaces, and to what extent this 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?
Okay Perfect and then there's just a question for David I was wondering if.
At this juncture since you know a lot of the clinical efficacy data for excess so five and indeed also seven is out there. If you are receiving inbound interest from people who might potentially become part of the sales and marketing team at XL.
And if you could perhaps comment just qualitatively on the caliber. If these people are potentially you know some of their background and promoting drugs and the CNS indoor psychiatric spaces and to what extent. This 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 O'five Ano seven ARPU.
Well thank you Ron.
Dave Merrick: 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, there are a couple things that I think get 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 about 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 highly efficient and contemporary launch. And so we kind of disproportionately attract those types of talents, whether that's within the marketing function or the data and 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.
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 are there are a couple of things that I think get 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 as our prescribers are around the potential of what we can can due in the marketplace and how we conservations also I think there our commercial individuals who are very excited about the idea of a highly efficient and can temper.
Every launch and so we kind of disproportionately attract those types of talents, whether that's within the marketing function or the data and analytics function. So we feel very good about the type of interest that were 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 are very strong salesforce as well as other commercial talent.
Great. Thank you very much.
Operator: Great, thank you very much. Thank you. Your next question comes from Yatin Suneja with Guggenheim Partners. Please go ahead.
Thank you.
Your next question comes on line of yet tends to nature would cooking Hi partners. Please go ahead.
Yatin Suneja: Hey guys, thank you for taking my question, and Mark, congrats on the new role. Just a couple of questions on a few things. Maybe we'll start with TRD first. Can you talk a little bit about how you are qualifying a non-responder in the STRIPE study? And then, in terms of the dose that you might be using in the open-label phase and the randomized phase, is there a reason to believe that you might be using a different dose in the open-label phase versus the randomized phase? And I have a few follow-up questions.
Hey, guys. Thank you for taking my question in my congrats on the on the New Wells just a couple of question on I'm confident on acute things, maybe we'll start the T.
T D plus can you talk a little bit about how you are qualifying in non responded in the strike study on them in terms of the dose that you might be using in the open label phase under that into my speech is get a reason to believe that you might be using a different dose in the open there, but what should be randomized phase.
We have two follow ups.
Yeah.
Yatin Suneja: Okay.
Cedric O'Gorman: Thanks, Jan, for the question, and I'll turn it over to Cedric to answer that.
Thanks, John for for the question and I'll I'll turn it over to Cedric to answer that.
Cedric O'Gorman: Yeah, thanks. So first of all, with regard to treatment resistant depression and the I think it was the definition of treatment resistant depression, but you asked about the non-responder or non-responder, right? So, you know, there's a standard approach that just as a reminder within the open-label period, the patient is being treated with bupropion, and if they fail to respond, then they are randomized. Now with regard to both the doses and the criteria by which we measure response or non-response. Historically, we disclosed that once we have top-line data, which will be before just before the end of the month So we're on track for that.
Thanks, So first of all with regard to a treatment resistant depression and the I think it was the definition of.
Great.
It's about non responders or non responder right. So you know there's a standard approach that just as a reminder, within the open label period, a piece of patients being treated wood.
And if they fail to respond and in my now with regards to both the dosing and the criteria bike week, we measure sponsors on response to start than we schools that once we have topline data, which will be before just before the end of the most so we're on track a before that.
Yatin Suneja: Okay, I understand. And in terms of the, again, on the dose, like, have you manufactured your own pill to use in this study for bupropion, or are you using one that is commercially available? Because I remember in the ASCEND study, the dose that you used is not commercially available, so you had to make your own pill.
Oh, okay.
I understand.
In terms of the other than on the Bill is like have you manufactured your own killed to use it won't be in this study or bupropion or are you using a commercially available because I remember in D. assent study the dose that you have used is not so much should be available. So you have to make your own though so help us understand.
Yatin Suneja: So help us understand the situation here. And then moving to the agitation side, can you maybe comment on what the reason for the delay was? Why was it pushed from first half to Q3?
The situation here, one moving to the agitation side can you maybe comment what's London reason for would be nave about like approached from far stopped acute three or thank you.
So what you're young but think things little additional questions with regards to a drug product, which is used in clinical trials you know the.
Ariel: Thank you.
Ariel: Thanks for those additional questions. With regard to the drug product used in clinical trials, as a reminder, clinical trials do require that products be blinded, and so therefore, the drug product for the various arms and in the various treatment periods has to be made so that they match in appearance. With regard to Alzheimer's disease agitation and our updated guidance, so our previous guidance was for the first half of this year, and we had talked about roughly mid-year, 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 that is elderly. Right now, we have 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.
It's a as a reminder, clinical trials or do require that products that be blinded and so therefore, the drug product for the there's arms on and into various that treatment periods have to be meets that they match or you know appearance with regards to.
Well times, these agitation and a and our our updated guidance. So our previous guidance was the first half of a of this year and and you know we've talked about your roughly a year that has not changed significantly we wanted to do why was too.
A build in some potential buffer as you know why there are some external factors side.
No.
And not to talk too much about the the impact of the crown of Iris, but it is something that that we do have to be.
Mindful of given that old-timers. The these agitation not is our targeting a patient population, which is elderly or right now we've not seen any impact on but we do thinking that it's a it is prudent aren't too in a range of guidance on make sure that we take into account tiny potential eventuality.
Mark Goodman: Got it. And thank you. I'll get back in the queue. Your next question comes from the line of Mark Goodman with SBB. This morning, Dave, I was wondering if you could talk about the migrant space a little bit. Obviously, it's a crowded market with, you know, 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.
Got it and think I'll get back into queue.
Your next question comes on line, that's Marc Goodman.
The VB please god had.
This morning, Dave I was wondering if you could talk about the migrant space a little bit obviously, a crowded market with.
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.
Dave Merrick: Yeah, well, Mark. Thank you. Well, I think when we look at the migraine market, you know, for those of us who've been associated with the migraine market for many years, it couldn't be a more exciting time. And, and that's really good for clinicians, and it's good for patients.
Yeah, well good morning, Mark Thank you.
Well I think when we look at the migraine market you know, it's it's for those of US who've been associated with might bring market for many years it couldn't be a more exciting time and ER and that's really good for.
Clinicians and it's good for patients so.
Dave Merrick: So, certainly, we have long wanted to have more options to provide for patients and really help them better manage migraines. When you look at the frustration in the marketplace, you know, the first place we go to when I say this often is to really clarify what we are 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 with is just pure efficacy. While there are a variety of needs out there, the one that rises to the top, and in our research, eight out of ten physicians prioritized improved efficacy as 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 it.
Certainly we have long wanted to have more options to provide.
For patients and really help them better managed migrate.
When you look at the frustration in the marketplace you know the first place we go to one nice I'd say this often as to really clarify what do 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.
Well there are a variety of needs out there the one that rises to the top and in our research. It was eight out of 10 physicians prioritize improved efficacy was their greatest unmet need.
And so we built a clinical program around solving that issue or 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 basis every single day.
So you start with.
Dave Merrick: Who's the right patient population with the greatest unmet need? And those are those who have failed, you know, 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, OK, well, what are their treatment options, and what would they be faced with? And in Momentum, we went head to head against Rizotriptan, which is largely viewed as one of the most effective treatment options out there, certainly one of the most effective.
Who is 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.
Are those that were treatment experience and had a prior sub optimal response to to their acute therapy.
Again, those are the patients that physicians tell us that they are dealing with day Annandale.
And then we took the next step and said, okay, well what are their treatment options and what would they be faced with and momentum. We went head to head against rides a trip down which is right largely a viewed as one of the most effective treatment options out there certainly when most effective.
Dave Merrick: 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 differentiated in the market. The right patients 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 ASS 07. And you may recall in our mindset survey, we asked migraine-treating physicians their degree of enthusiasm for AXS07 should it be superior to Rizotriptan, and I think, you know, we've talked about those results where the vast majority of physicians, Unknown Attendee, David Amsellem, Vikram Purohit, Jason Gerberry, Herriot Tabuteau, Mark Jacobson,
Triptan.
And by showing superiority, we think that we've really help clinicians understand for their most difficult patients where we have the next best stuff.
And those are the data that we think will be highly differentiated in the marketplace.
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 marketplaces looking for and I think that's really the core of our differentiation and why we think there is tremendous enthusiasm for access Oh seven.
And you may recall in our mindset survey we out.
Migraine treating physicians.
Their degree of enthusiasm towards access so seven should we be superior to rise a trip Dan and I think we've talked about those those results where the vast majority of physicians.
They would prescribe access so seven over currently available treatments, but also over the emerging treatments as well.
Dave Merrick: [inaudible] So we see a very strong commercial opportunity to benefit patients with AXS 07.
So we see a very strong commercial opportunity to benefit patients with access Sosa.
Dave Merrick: And how does the intercept study play into it?
And how does the intercept study play into it.
Dave Merrick: 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 typical clinical trials, we need patients to wait until their pain is moderate to severe. And those data are very helpful for registration processes, etc. When we look at how clinicians typically tell their patients to administer their acute therapy, they say to take it at the first sign of migraine pain. And so we think that Intercept will provide kind of 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 AXS07.
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 and the typical clinical trials, we need patients to wait until their pain as moderate to severe and those data are very helpful for registration processes and et cetera, when we look at.
How clinicians typically tell their patients to administer their acute therapy. They say take it up 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 ER to administer so we think its is additional information that is not required for the NDA filing, but will be important and the and the setting as we educate physicians.
City of access or so.
Mark Goodman: And last question, as you're thinking about the sales force. Is there going to be one sales force that sells both products, the depression product, and this one? Will it be two separate ones? How much overlap is there?
And last question as you're thinking about the Salesforce.
Is it going to be one salesforce that sells both products. That's a question product in this one.
Two separate ones how much overlap is there.
Well.
Dave Merrick: Well, as mentioned earlier, we will still look at options around looking at both migraine and depression and how we can change the size and structure of that field 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 in promoting both migraine and depression. There is some overlap, 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.
As mentioned earlier, we are we will still look at options around or looking at both migraine and depression and how we the size and structure that field force. So.
Well, 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 Japan, 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 at our option. 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 lean.
Thanks.
Operator: Your next question comes from the line of Matt Kaplan with Ledenberg. Please go ahead. Alright, good morning guys.
Your next question comes online as Matt Kaplan with Ladenburg. Please go ahead.
Hi, good morning, guys.
Matthew Lee Kaplan: Congratulations on the progress. I just wanted to zero in a little bit more on stride one, given the near-term readout of results you expect 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?
Rats on that the progress I'm just wanted to zero in a little bit more on stride, one Ah given the near term read out of results expect.
There so Keith Keith please remind us of the sizing ER and the powering of the study I guess given the fact that you a continued enrollment to build your safety database Oh can you give sense of that.
Ariel: Good morning, Matt, and thanks for the question. So in terms of size, 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 antidepressants that have been shown to work and that are currently approved.
Hi, Good morning, Matt and thanks. Thanks for question so in terms of sizing.
The last update that we gave was that a the study had randomized or approximately 300 patients.
That was a at the end of bomb of November and so why so.
The.
The the final number will be north of a 300.
In terms of the powering of the trial under study was powered at the 90% level to detect and effect size of somewhere between 0.3 0.35, which is in line with side effect sizes I'm antidepressants that don't work in that have been currently approved.
Great. Thank you and then and then in terms of the open label extension study for all size, that's in Raleigh, both MDD and TRG patients hows that progressing and he can give an update in terms how many patients you expect it and ultimately in the open label extension.
Matthew Lee Kaplan: Great, thank you. And then in terms of the Open Label Extension Study for OSA-HIATS 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 do you expect ultimately in the Open Label Extension?
Ariel: That study is progressing well. As you know, that is a gaining factor to us filing it.
That's what he is progressing well I as you know that is a a gating factor to watch the filing an NDA, we do need to build a safety database of 300 patients treated for six months and hundred patients treated for one year and we're on track to do that so we're pretty happy with the way the that is currently.
Ariel: We do need to build a safety database of 300 patients treated for six months and 100 patients treated for one 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 an NDA before the end of the year.
In rolling.
It is enrolling in a piece that will allow us to meet our guidance of a filing the NDA on before the end of year.
Okay. Thank you and.
Matthew Lee Kaplan: Okay, thank you. And, I guess, just going back to following up on the questions that we had about AXS14, S-ruboxetine for fibromyalgia, can you give us a sense in terms of the potential regulatory paths forward for S-ruboxetine in fibromyalgia?
I guess just going back to a following up on on the questions that we had on excess 14 Essar box attain for probably allows you can you give us a sense in terms of the potential directly Pat regulatory paths forward a press reebok's attained in fiber miles you.
Ariel: So we first want to meet with the FDA. As a reminder, though, the S for Boxa team has completed two efficacy trials of significant size. One was a phase 3 trial that enrolled and randomized over 1,000 patients. That study was positive. And there was also a phase 2 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 until we meet with the agency.
So well we.
First want to want to meet with the FDA as a as a reminder, though.
As for box. The team has completed two efficacy trials of significant size. One was the phase three trial, which are enrolled and randomized over a thousand patients and that's that he was positive and there was also what piece to trial, which was also positive over these studies were random.
Mines double blind placebo controlled trials and and so while we certainly will want to I get the Yankees feedback on on those clinical studies those things were run by Pfizer I. So while we want to make sure that that.
Get feedback from the if you in terms of what else would be required in order to file and yet we will not speculate on what those requirements I could be and until we until we meet with the agency.
Matthew Lee Kaplan: Very helpful. Thank you. Your next question comes from the line of Bert Aslett with DTIG. Please go ahead. Thank you. I just have a couple of quick ones.
Great.
Very helpful. Thank you and.
Good luck for.
Your next question comes on line of birds eye slots, but deep <unk>. Please go ahead.
Thank you I just have a couple of quick ones I just would you have a lot on your plate with a number of different indications.
Bert Aslett: Just with you have a lot on your plate with a number of different indications. Where does it go?
Where does the excess o'five smoking cessation indication sit and in in terms of urgency and in terms of corporate priorities. Thanks.
Bert Aslett: 05 the smoking cessation indication sits and in terms of urgency and in terms of corporate priorities. Thanks.
Ariel: Thanks for the question. You are correct that XSO5 does have a number of potential indications and we do have a lot on our plate. So 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 are clearly...
The things work for for the the question.
So you you are correct that to access with five does have a number of potential indications and we do have a lot on our plate. It was one of the challenges that no that we're facing that we're keeping in mind is the need to focus well at the same time avail ourselves of the beer.
There's opportunities in the pipeline.
So I work our priorities on are clearly you know this year or to make sure that you know we file I went to gaze in a timely manner and also why that's that those those those filings or of of significant quality.
Ariel: to make sure that we file our NDAs in a timely manner and also that those filings are of high quality. So that is our top priority, which is our NDA filings. Beyond that, there is...
So that is our top priority, which is the out when do you filings on it and then.
Beyond that is a this is is.
Ariel: Advancing our product candidate in narcolepsy into phase three, you know, based upon the very positive phase two data. So I'd say that that's priority number two. And then, of course, getting FDA feedback on AXS 14, given the very positive efficacy results there. And then we absolutely will be meeting with the FDA this year with regard to smoking cessation to delineate a path forward there and understand what the clinical development, and further clinical development for that indication will look like.
Advancing our product candidate in Norcal EPCI into phase three you know based upon a very positive phase two data. So I'd say I see that that's party not number two and then and of course getting if the feedback on excess 14 given the.
Very positive efficacy results. There and then we are we absolutely will be meeting with the FDA. This year with regards to smoking cessation to a two to delineate a password there and understand what the clinical development what the further clinical development.
For the indication will look like.
Okay. Thank you I'm could you just remind us of the CMC.
Bert Aslett: Okay, thank you. Could you just remind us of the CMC...
Bert Aslett: characteristics or status of O5 at this point.
Progress or or or.
Characteristics or or status of old five at this point.
Ariel: So the clinical trial materials for AXS05, for our AXS05 trials, have been made at commercial scale, so we're in very good shape there, and they've also been made at the contract 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 on the way.
So the or the clinical trial, a material war access so five four hours because we'll five trials.
Have been made at commercial scale. So we're in very good shape, there and have also been made after the.
I've also been made at the a contract manufacturer that will be manufacturing the commercial supply.
They all registration batches would shop.
Must be made in specifically for the purpose of submitting an NDA on and so that work is underway.
Okay. Thank you and then just one on the on migraine doesn't make sense to to have a study in combination with a long acting CRP and with an acute.
Bert Aslett: Okay, thank you. And then just one on migraines.
Bert Aslett: Does it make sense to have a study?
Bert Aslett: in combination with a long-acting CGRP.
Ariel: and with an acute migraine therapeutic such as you have with O7, does that make any sense commercially at all?
Migraine therapeutic such as you have windows, seven but does that make any sense a commercially at all.
Bert Aslett: We certainly don't see any reason why XSO7 could not be administered with other types of therapy. 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 arsenal.
<unk>.
We certainly.
Don't see any reason why it because it was seven could not be administered with other types of therapies.
Right now you know, we're focusing on getting the product approved.
And it will be interesting to see you know how commissions eventually choose to use it as part of the armamentarium.
Okay. Thanks, that's all for me thank you very much.
Operator: Okay, thanks. That's all for me.
Myles Robert Minter: Thank you very much. Your next question comes from the line of Myles Minter with William Blair. Please go ahead.
Your next question comes on line of Smiles Man sounds like volume Blair. Please go ahead.
Myles Robert Minter: Hi guys, thanks for taking the questions and congrats Mark on the new position at the company. Just the first question is on advance, you know, 80% enrolled, so push the guidance slightly out to the third quarter. I'm wondering whether you're proactively, you know, cautioning your physicians to enroll new patients into that trial currently given, you know, 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 when you discontinued enrollment into the bupropionolone arm.
[noise] Hi, guys. Thanks for taking my questions and congrats Mark on the lender position at the company [noise].
Just the first question is on advance.
80% enrolled push the garden slightly out too so I'd call. It off I'm wondering whether youre proactively cautioning you physicians to enroll new patients into that trial kindly given you know the unfortunate risk recall for John staying at the moment and not highly susceptible popular.
And and all sorry, you mentioned, 80% enrollment I'm wondering what proportion of patients were enrolled and not trial at the December 2000, and I chain timeline way you discontinued enrollment into the prepaid ARPU on a line item.
[noise] so was that thank for questions that miles on the.
Ariel: So thanks for the questions, Myles. With regard 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 any information that they have, any concerns that they might have with regard to enrollment. Currently, we're not. I don't hear anything that is of concern, but it is a situation that we're monitoring pretty closely. And with regard to the percentage of patients at the time of the interim analysis, The interim analysis, as a reminder, was conducted at the 30% enrollment point, so
With regards to our cautioning physicians are around enrollment in into the trial I mean.
We're not currently doing that but what youre doing is monitoring very closely from the clinical trial sites any information that they have when you concerns that they might have with regards to enrollment I'm currently Oh, we're not.
Hearing anything that that's that that is of concern, but it is the situation that we're monitoring pretty closely.
And with regards to while the presented a patient at the time of the and interim analysis.
[noise] at that the interim analysis as a reminder was conducted on.
At the 30% enrollment point so won.
Ariel: That should allow you to get a sense of what the subsequent enrollment was after that. So 30% enrollment point, and remember the size of the trial was 435 subjects total.
That should allow you to to get a sense of what the subsequent enrollment was a after that so it was enrolled so 30% enrollment point and remember the size of the trial was 435 subjects total.
Myles Robert Minter: That's helpful. Thanks for that. And then, maybe, a question for Dave.
[laughter] that's a that's helpful. Thanks for that and then maybe a question for Dave.
Dave Merrick: Some COL diligence we've been doing suggests that at least in specialist centers, they're sort of using SSRIs and rather than waiting for, you know, four, six to eight weeks for a response there, they're trying to see an early response at two weeks with those drugs, maybe a 20% improvement to predict whether those patients are truly going to have a response later down the track. You know, 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 effect and maybe not so much focused on Madras but maybe on some of those patient quality of life questionnaires which are more routinely used in the clinic. What are you hearing from the community? What are they most excited about this product? Cheers.
Just some kind of out diligence web pain doing suggests that relation specialist senses. This study using SSR doesn't rather than waiting for you know for six to eight weeks for a response that they're trying to say an early response to two weeks for does drugs might be a 20% improvement.
To predict whether those patients are truly going to have a response light the down the track I'm just looking at the data from Gemini and and a sand and and the early rapidity of response I'm wondering in your conversations with clinicians spicing specialty centers any pay say pays whether a focus on their behalf.
He is actually on the rapidity affects and maybe not so much focused on Madras, but might be on some of those patient quality of life question as which are more routinely use and the plenty what are you hearing from the community what I'm. Most excited about this product she is.
Dave Merrick: Good morning, 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 O5 in front of specialists, one of the things that really 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 NMDA plus multimodal activity, you know, they're really hungry for a different pathway that kind of gets them off the merry-go-round of SSRIs and 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 down and look at our data. And then when they see the rapidity of effect and the robust nature of effect, that's when they start to look at this as
Yeah.
Good morning miles. Thank you for the question.
We are hearing some of the the same and our research around.
You know what physicians are looking for and when we put the the current data that we have for old five in front of specialists one of the things that really are that they light up about is really around the repetitive effect.
But also what they get excited about is kind of the reason to believe that and when you look at the end M.D.A.
Plus multi modal activity you know there, they're really hungry for a different pathway that kind of get them off the Merry go round of the accessorize SNR eyes, and they're looking for another pathway and one that would be potentially deliberate in a very patient friendly.
Way, so that gives us reason to really sit up and look at our data and then when they see the rapidity of effect and the robust nature of of effect, that's where they start to look at this as certainly.
Dave Merrick: Unknown Attendee, David Amsellem, Vikram Purohit, Jason Gerberry, Herriot Tabuteau, Mark Jacobson, Richard Bogan, Darren Opland, Ashwani Verma, Yatin Suneja, Joon Lee, Raghuram Selvaraju, Allan Sheahan, Andrew Cutler, Craig Chepke, Susan McElroy, Ari Maizel, Ari Maizel, Asim Rana, Darren Opland, Allan Sheahan, Andrew Cutler, Craig Chepke, Susan McElroy, Asim Rana, Darren Opland, Ashwani Verma, Ari Maizel, Asim Rana, Darren Opland, Ashwani Verma, Ari Maizel, Asim Rana, Darren Opland, Ashim Verma, Ari Maizel,
For patients that they've struggled with who are treatment experience, but also they start to move up very quickly earlier to to think about patients where they would want that rapidity of effect and 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.
The more there is focused 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 the unmet need.
Myles Robert Minter: Cool, just to follow up, does that logic change between specialist centers that are most likely monitoring their patients consistently over that early treatment period versus a PCP that might just prescribe and then, you know, come back six weeks later and not actually monitor that patient's response early on?
So just a follow up to stop luxury change between specialist sensors that are most likely monitoring that patients consistently over that early treatment period for us as a pay say pay that much ascribe and then you know I come back six like slide off not actually modest about patients response early on.
Dave Merrick: Yeah, 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, you know, the rapidity of effect, and the types of patients that they may be seeing could be very different in terms of the sense of urgency that we might see with 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 two areas of treatment.
Yeah, I mean that I I don't have the data to answer that question, but intuitively I think it it makes sense that those centers, who are very actively monitoring rapidity of effect and the types of patients that they may maybe see could be very different in terms of sense of urgency that we might.
I see in the with 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 the adoption curve could be different across those two areas of treatment.
Myles Robert Minter: Fair enough, thanks for the questions and congrats on the year.
Fair enough. Thanks for the questions and congrats on the you.
Myles Robert Minter: Thank you, Myles. Darren Opland
Thank you Mike.
Operator: There are no further questions at this time. I will turn the call back over to the presenters for closing remarks.
There are no further question at this time I will turn the call back over to the presenters for closing remarks.
Well. Thank you all for joining us on the call today axiom is committed to accelerating the invention development and adoption of life changing medicine for the many people facing on satisfactory treatment for CNS diseases.
Mark L. Jacobson: 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 disease. We anticipate a number of important milestones over the balance of the year, and we look forward to updating you on our progress.
We anticipate a number of important milestones over the balance of the year. When we look forward to updating you on our progress.
Operator: This concludes today's conference call. You may now disconnect.
This concludes today's conference call you may now disconnect.
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