Axsome Therapeutics at Goldman Sachs Conference: Strategic Growth and Challenges

Axsome Therapeutics (NASDAQ:AXSM) presented a comprehensive update at the Goldman Sachs 46th Annual Global Healthcare Conference on Monday, 09 June 2025. The company highlighted its robust pipeline and commercial growth, tempered by regulatory challenges. While products like Avelity show promising expansion, the refusal to file for AXS-14 in fibromyalgia underscores hurdles ahead.

Key Takeaways

• Avelity is experiencing rapid growth, with a $400 million annualized run rate.
• Sunosi continues steady growth, annualizing at $100 million.
• Zimbravo’s launch for acute migraine treatment is imminent.
• NDA submissions for AXS-5 and AXS-12 are planned for this year.
• Financial strength with $300 million in cash resources, aiming for cash flow positivity.

Financial Overview

• Cash Resources:

- $300 million in cash as of Q1, sufficient to achieve cash flow positivity.

• Pipeline Potential:

- Potential peak sales estimated at $16.5 billion for the entire pipeline.

- Avelity, Sunosi, and Zimbravo combined could reach $2 billion to $4.5 billion in peak sales.

Operational Updates

• Avelity:

- Current annualized run rate of $400 million.

- Over 50% of prescriptions are first or second line; 55% of patients use it as monotherapy.

- Growth driven by field force expansion and potential direct-to-consumer (DTC) campaigns.

• Sunosi:

- Approved for excessive daytime sleepiness in narcolepsy and obstructive sleep apnea.

- Potential for label expansion through ongoing R&D efforts.

• Zimbravo:

- Launch set for this month, targeting acute migraine with a novel treatment approach.

Pipeline Development Updates

• AXS-5 (Alzheimer’s Disease Agitation):

- NDA submission on track for Q3, with positive study results supporting the submission.

• Solriamfetol:

- ADHD and major depressive disorder trials planned, with ongoing trials for binge eating and shift work disorders.

• AXS-12 (Narcolepsy):

- NDA submission anticipated in the second half of the year, focusing on cataplexy in NT1 narcolepsy patients.

• AXS-14 (Fibromyalgia):

- Facing a setback with a refusal to file letter, necessitating an additional trial starting in Q4.

Future Outlook

• Axsome emphasizes its diversified portfolio and late-stage pipeline as key growth drivers.
• The company is committed to advancing its novel mechanisms of action and multi-mechanistic approaches.

For further details, please refer to the full transcript provided below.

Full transcript - Goldman Sachs 46th Annual Global Healthcare Conference:

Mark Jacobson, Chief Operating Officer, Axsome Therapeutics: Great. Hi. Thanks for joining us today. My name is Mark Jacobson.

I’m the Chief Operating Officer at Axsome Therapeutics. And thank you to Goldman Sachs for hosting us today. So I may be making forward looking statements, we’d invite you to review our disclosures and summary of risks and uncertainties, which you can find in our filings with the Securities and Exchange Commission. So Axsome Therapeutics, our mission is to develop and deliver transformative, innovative medicines for the hundreds of millions of people living and impacted with or by central nervous system disorders. There are more than one hundred and fifty million people in The United States who are impacted by 10 serious CNS conditions that we’re focused on within neuroscience.

And neuroscience conditions have historically been underserved by the biopharma industry and that is in psychiatry and neurology. Within those categories, underserved unmet needs are twofold. One is areas of unmet need where there are none to only a few treatments approved, think Alzheimer’s disease agitation in this case, to areas where there are multiple products approved but patient outcomes are still lacking in particular with respect to efficacy. So in that case, think migraine, think depression. So we are focused on a number of indications within psychiatry, major depressive disorder, MDD, Alzheimer’s disease agitation, smoking cessation, ADHD, and binge eating disorder.

And we’ll talk about the updates for those programs here. And then within neurology, that’s obstructive sleep apnea, migraine, narcolepsy, fibromyalgia, and shift work disorder. The way we think about delivering innovation to these areas, to patients, to HCPs, there are five pillars. One is novel mechanisms of action. So, within the areas where there are multiple products approved, we think about approaching those areas with novel mechanisms of action that deliver distinct and differentiated treatment outcomes.

We think about multi mechanistic modes of treatment and that would be, for example, Ovelity, Cymbravo, and some of the other programs in the pipeline. Through some of those multi mechanistic approaches, we focus on metabolic pharmacokinetic modulation. So, utilizing central products or product candidates that are active in the central nervous system to also modulate the metabolism of other drug substances. Clinical trial innovation. In CNS, clinical trials have historically had difficulty separating from placebo due to high placebo response rates.

And we work on designing trials that can detect signals if you have an active molecule. And then the final pillar is innovation with respect to molecular drug delivery. So determining ways to deliver molecules that are active in the central nervous system in a way that they can result in distinct pharmacodynamic impacts. Snapshot of the company, we think we have a singular neuroscience pipeline in the industry. And when you think about that, we’re diversified commercially.

We have three commercial products, two of which we developed in house. We have two NDA stage programs, five late stage programs in ongoing Phase III development. And all of those lead to potentially seven additional new products or indications through 2027. And then we’ve covered what we’re focused on, but 10 highly prevalent or difficult to treat areas of unmet need in CNS. Here’s the pipeline broken out in psychiatry and neurology.

And we’ll run through the updates for each program. The pipeline is such that in totality there are $16,500,000,000 of potential peak sales. Just taking Avelity, Synoscience and Bravo, the potential peaks of our approved products, potential peak sales are 2,000,000,000 to $4,500,000,000 Quick snapshot of the year to date and the balance of the year and what’s up for 2026. Cymbravo, that was approved earlier this year. And then we’ve had a number of positive Phase III clinical trial readouts.

I will cover those. And then looking ahead, regulatory and commercial side, launch of CINBRAVO. That is imminent and we’ll look forward to sharing updates there. SNDA submission for Alzheimer’s disease agitation, that is on track for the third quarter. And then we have an NDA submission plan for AXS-twelve and narcolepsy in the second half of the year.

Clinical trial readouts. Right now we have the ENGAGE Phase III trial of soramfetol in binge eating disorder. That top line is on track for 2026, so that’s enrolling through the balance of the year. Same thing with the sustained phase three trial of soramfetol in excessive sleepiness in shift work disorder. Enrollment is ongoing and we expect top line next year.

And then we have a number of trials that we expect to start between now and the end of the year and I’ll cover those. So commercial highlights quickly. Again, approved products. Ovelity in rapid growth phase, annualizing as of the end of the first quarter at a $400,000,000 run rate. Dramatic growth, we expect that to continue.

Sunosi, growing steadily. That is approved for excessive daytime sleepiness in narcolepsy or obstructive sleep apnea. Again, steadily and we’re pleased with how that business is going. And then finally, Simbravo, as I mentioned, that is our new product for the acute treatment of migraine. That was approved in January and that will be launching this month.

Ovality. Oral NMDA receptor antagonist. So this is a dramatically new way of treating depression with oral treatment. The key thing here is it’s fast and it lasts. So a quick snapshot of growth in terms of scripts since launch and a couple callouts as we’re here right now as of today or end of the first quarter.

Just north of 50% of the prescriptions are in first or second line, so first line or first switch. And patients were very pleased with that, especially at this stage of launch. And about fifty five percent of patients start ELETI as monotherapy and we’re very pleased with that as well. Snapshot of quarterly net sales. Again, mentioned the annualized run rate and we expect growth to continue at a nice clip.

That will be in the near term driven by I think three key drivers of growth. One is the field force expansion. We completed an expansion of 40 additional sales reps at the end of the first quarter. They’re in the field. We’re seeing the start of that productivity in terms of pull through new to brand scripts.

We expect continued evolution in terms of covered lives in the commercial channel. That is both improvements in utilization management and increased number of lives covered. And then finally, we will potentially roll out a national DTC campaign in the second half of the year. Turning to Sunosi, as I mentioned, this is our product for excessive sleepiness in OSA and narcolepsy and steady growth here. And just a real quick snapshot, moving nicely.

It’s a very healthy component of the business and we’d expect that growth to continue here. But in particular, we’re very excited about potential efforts on the R and D side for potential label expansion. Annualizing at about $100,000,000 run rate, which is great. And we’re pleased with how that business has been performing since we acquired the product. Zimbravo, this is a novel, again, of our pillars of growth, multi mechanistic treatment options for oral for acute treatment of migraine.

And what’s very interesting about Zimbravo is the clinical data that we generated in a variety of migraine severity. So, these studies position it and allow HCPs to use this in a variety of patient profiles. Early line or mild migraine pain to significant migraine pain, say, in individuals who have had an adequate response to prior oral acute treatments. It uses our MOSAIC technology. This is a technology that we developed in house to deliver meloxicam and rizatriptan.

Offline briefly. Right. Sorry for those joining online. And we should now be back on slide 19. So Sunbrella launch readiness, the field force has been hired.

They’ve gone through training. It’s 100 reps. And they will be in the field imminently and we’ll have an update for you all soon. And the key thing about migraine is there are a number of treatment options, but greater than eighty percent of patients discontinue their acute migraine treatment within the first year. And that’s due if you survey patients and clinicians, that is due to inadequate treatment the efficacy side.

So turning to the development pipeline, we have AXS-five for Alzheimer’s disease agitation. We completed that clinical program at the end of this past year and we are building an NDA submission. As I mentioned, the work is underway now. No patients are being treated and that submission is on track for the third quarter of this year. They have high unmet medical need and currently about seven million adults in The US and about seventy percent of them have agitation.

So about seven million adults with Alzheimer’s or the dementia type. And this is the key symptom that leads to placement in long term care facilities. For the clinical data that we’ve generated, we have three positive studies. So ADVANCE-one here, that is a parallel group study of AXS-five versus placebo and bupropion. And then we show here a COR-two.

This is a randomized withdrawal design study versus placebo. Very consistent results across the studies that we generated. And we met with FDA and announced the we’ve been interacting with FDA throughout the development program and the most recent update was in March, the pre NDA FDA meeting minutes, which are our alignment to proceed with submission of the SNDA. The other program we’re working on for AXS-five is smoking cessation. And we plan to start a study in this indication this year.

So we’ll have more to say soon. Turning to salriamfetol. We are working on a number of additional indications for potential label expansion. This is based on KOL feedback about the activity of the molecule in a number of psychiatric conditions. And we’ve started by prioritizing ADHD, major depressive disorder, binge eating disorder, and excessive sleepiness and shift work disorder.

Starting with ADHD, we have completed one positive trial in adults so far. This is the FOCUS trial. We read out the results earlier this year and those are shown here on the AISRS. The next step for the program here is initiating a pediatric trial. And we plan to do that later this year.

And that is what we’ve discussed with FDA. We would need to complete a package for the indication. Focus on major depressive disorder. We think this is very interesting mechanistically. Saurenipital is a DNRI, so a dopamine norepinephrine reuptake inhibitor.

And mechanistically very interesting with respect to depression. So we wanted to look at this in adults and in particular we were interested if from a precision perspective there are a subset of adults wherein the mechanism could be highly relevant and that we looked at excessive sleepiness. So about half of patients with major depressive disorder have excessive daytime sleepiness. And with the PARADIGN trial that we conducted, we saw a signal in that patient population. So the next steps here are to launch a study, a second study in major depressive disorder with excessive sleepiness, and we’ll be doing that this year, starting that trial this year.

Binge eating disorder, also very interesting with respect to the mechanism of solriamfetol. And we are currently conducting the ENGAGE Phase III trial, as I mentioned. That’s enrolling. So that’s a parallel group trial looking at salriamfetol one hundred and fifty mg and three hundred mg versus placebo. And we expect top line results next year.

Rounding out the development work for salriamfetol, shift to work disorder. There are about fifteen million individuals in The US that may have shift work disorder. And just to give a sense of the impact, about a third of individuals working in The US work on an alternate shift. So highly impactful and prevalent condition. We’re conducting the SUSTAIN trial.

Again, parallel group trial of sarsamfetol one hundred fifty and versus placebo, 300 versus placebo. And we expect results next year. Turning to AXS-twelve, so this is reboxetine in narcolepsy. And Reboxetine is a highly selective norepinephrine reuptake inhibitor. So narcolepsy orphan indication, we are focused on so characterized by cataplexy and excessive daytime sleepiness.

A number of other symptoms including hypnagogic hallucinations. And we are starting to focus on cataplexy, so NT1. That’s in about seventy percent of individuals living with narcolepsy. We have completed the clinical program for AXS-twelve. There are three trials.

The results from two of those are shown here. The CONCERT trial and the SIMPONY trial. We also have results from randomized withdrawal trial, that’s the ENCORE study. And we plan, as a result, are based on this clinical program. We’re planning a new drug application submission in the second half of this year.

Finally, there’s AXS-fourteen, S reboxetine. So this is an S enantiomer Roboxetine and also obviously a norepinephrine reuptake inhibitor. This is a more potent and selective enantiomer of racemic Roboxetine. So we have it’s highly prevalent. We’re very excited about this program.

About seventeen million people in The US have fibromyalgia and it’s highly underserved. So there are only a few products approved and there’s been very little innovation in the space and no new novel therapeutics in over fifteen years. So here are the study results we submitted in NDA based on two studies, two placebo controlled studies. And we heard back from FDA that we received a refusal to file letter from them and we announced that this morning. It is based on the second study, which was an eight week flexible dose design trial.

What they have asked us for is a second trial of twelve weeks in duration and that is fixed dose. So the results here are the phase three trial that was previously conducted. Twelve weeks fixed dose, highly statistically significant. Both studies were positive. No comments on the prior positive results.

So we need to, as a next step, conduct a second twelve week fixed dose trial. We’re on track to do that and start that study by this year in the fourth quarter. So excited about that and the potential for AXS-fourteen to offer a new treatment option to patients. And I’ll summarize quickly and comment on IP. So IP has been a focus of the company.

And each program that we’ve launched and just a very broad patent portfolio covering our pipeline. So we’ve got robust protection depending on the program from 02/1939, ’2 thousand and ’40 to 02/1943. And a key update, I think, for our folks is with respect to OVALITY. We settled earlier this year. We announced the settlement earlier this year with the only the first and only first filer.

And so no see, there are multiple first filers, and we have settled or resolved four of those. And there are two first filers outstanding. So we’re very pleased with the state of the intellectual property portfolio. Financials of the company are strong and the key thing to take away is that we have cash resources on hand that take us to cash flow positivity. So $300,000,000 as of the end of the first quarter.

And I’m very pleased with the financial foundation of the organization. Again, that takes us to cash flow positivity. So the leadership team, we have a strong leadership team across the organization backed by a very strong board of directors. And with that, I want to thank you all for joining us today. And we’ll look forward to providing updates through the balance of the year.

Thank you.

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