Harmony Biosciences at HCW@Home: Strategic Growth and Pipeline Expansion

On Thursday, 17 July 2025, Harmony Biosciences (NASDAQ:HRMY) participated in the H.C. Wainwright "HCW@Home" Series, sharing insights into its strategic direction. The company highlighted its growth, driven by Wakix sales and a promising pipeline, while addressing challenges such as competition in the rare CNS disorder market. Harmony remains optimistic about its future, focusing on innovation and strategic partnerships.

Key Takeaways

• Harmony Biosciences reported a 20% increase in Wakix sales in Q1 2024, with revenue guidance for 2025 set between $820 million and $860 million.
• The company is preparing for the potential launch of ZYN-002 for Fragile X syndrome, with pivotal data expected soon.
• Harmony has over $600 million on its balance sheet, enabling self-funded growth and strategic acquisitions.
• The company is actively expanding its pipeline, with a focus on rare neurological disorders and potential new product launches each year.

Financial Results

• Wakix Performance:

- Q1 2024 net sales reached $184.7 million, marking a 20% increase from the previous year.

- Revenue guidance for 2025 is projected between $820 million and $860 million.

- The average number of patients on therapy in Q1 was 7,200, with projections nearing 8,000 by year-end.

- Wakix aims to achieve $1 billion in narcolepsy revenue before losing exclusivity.

• Balance Sheet:

- Harmony maintains a strong financial position with over $600 million available, supporting its self-funding model.

Operational Updates

• ZYN-002 (Fragile X Syndrome):

- Top-line data is expected this quarter, with potential NDA submission and FDA priority review.

- The Phase 3 RECONNECT study has been extended to 16 weeks, with increased dosing for patients over 50 kg.

• EPX-100 (Epilepsy):

- Ongoing Phase 3 studies for Dravet and Lennox-Gastaut syndromes, with data anticipated in 2026.

- The drug is noted for its safety and tolerability, eliminating the need for certain medical tests.

• Business Development:

- Harmony is actively pursuing acquisitions and collaborations, including a $15 million research collaboration with Cirque Biosciences.

Future Outlook

• Pipeline Expansion:

- Harmony aims for one or more new product or indication launches annually over the next four to five years.

• Wakix Life Cycle Management:

- Gastro-resistant formulation PDUFA is expected in 2026, with a high-dose formulation in 2028.

• Orexin Receptor Agonist:

- First-in-human studies are planned for later this year, with clinical data expected next year.

Q&A Highlights

• ZYN-002:

- The primary endpoint for the study is Social Avoidance.

• Wakix Growth:

- Patient growth is projected to remain consistent with previous years.

• Orexin Receptor Agonist:

- Harmony believes its orexin molecule has the potential to be best in class.

For a more detailed analysis, readers are encouraged to refer to the full transcript.

Full transcript - H.C. Wainwright "HCW@Home" Series:

Patrick Trucchio, Senior Health Care Analyst, HC Wainwright: Good morning, everyone, and welcome to HC Wainwright at Home, our virtual fireside chat series. I’m Patrick Trucchio, a senior health care analyst at HCW. Today, we’re joined by Jeffrey Dano, president and chief executive officer Kumar Boudur, Chief Medical and Scientific Officer Sandeep Tapadia, Chief Financial Officer and Adam Zeski, Chief Commercial Officer of Harmony Biosciences. Harmony’s commercial franchise is anchored by Wakix or Ptolemicent, the first and only FDA approved nonscheduled treatment for narcolepsy, which is on track to generate revenues of $714,000,000 in 2024 and $870,000,000 in 2025 by our estimates. With next generation pitolisin formulations, gastro resistant and high dose advancing and franchise well positioned to surpass $1,000,000,000 in annual revenues by 02/1930, Harmony continues to execute commercially while investing in life cycle innovation.

In parallel, the company is developing ZYN-two, cannabidiol gel and Fragile X syndrome or FXS with pivotal Phase III RECONNECT data expected midyear in EPX-one 100 in Dravet syndrome and Lennox Gastaut syndrome, both in phase three with top line data anticipated in 2026. Harmony also recently presented preclinical data for BP one one five two zero five, an investigational orexin two receptor agonist at the sleep twenty twenty five meeting highlighting dose dependent weight promoting and cataplexy expressing activity in a mouse model of narcolepsy. We look forward to discussing these late stage catalysts and the company’s long term growth strategy in more detail today. So maybe just to start, if you could walk us through the Harmony story, how the commercial and pipeline pieces come together, and where you’re focused in the near term.

Jeffrey Dano, President and Chief Executive Officer, Harmony Biosciences: Yeah. Good morning, Patrick. And, on behalf of the Harmony team, thanks for the invitation this morning. I think I appreciate your your opening remarks. You I think you framed kind of the Harmony story really nicely.

But I I think the top line sort of takeaway that Harmony continues to be a growth story, and I think supported by the continued growth, you know, of Wakix, you know, in the market for, patients with narcolepsy, you know, really based on the strong underlying, you know, business fundamentals. It continues to grow, you know, as you mentioned, on its way to a billion dollar plus, you know, opportunity in in narcolepsy alone. You know, from there, as you’ve highlighted, you know, we have built out a a robust, you know, late stage pipeline with multiple near term catalysts that we’re we’re very excited about. Let me just sort of frame that that pipeline as you were sharing. It it now consists of, you know, three orphan rare CNS franchises with each of those franchises having potential peak sales opportunities of 1,000,000,000 to $2,000,000,000 each.

We now have, as Kumar will expand on, eight assets across 13 development programs and up to six of them in phase three by year end. If we take a step back, we look across the industry, you know, we feel we have one of the strongest pipelines across the industry in, for patients living with rare neurological, disorders. And then with that, you know, we’ve built a unique company profile. You know, we are profitable, self funding kind of, you know, biotech, and really all of this activity, you know, sort of, funded, you know, off of the balance sheet, now over $600,000,000 on the balance sheet. So we are excited about continued growth of our commercial business, next gen formulations of petrolisant that we’ll talk about, and across the pipeline and the the near term catalysts that are coming.

And I’ll highlight, you know, the one that’s right around the corner, z y n zero zero two in fragile x syndrome. So I think that’s a, you know, high level view of, continued growth and opportunities ahead for Harmony and, towards long term value creation.

Patrick Trucchio, Senior Health Care Analyst, HC Wainwright: Right. Right. And so a big catalyst this year is in the this upcoming with this top line data readout for ZYN zero zero two. So maybe you could just sort of frame the timing for that readout and then as well what a successful trial would look like.

Jeffrey Dano, President and Chief Executive Officer, Harmony Biosciences: Sure. Go ahead, Kumar.

Kumar Boudur, Chief Medical and Scientific Officer, Harmony Biosciences: Yeah. Patrick, thanks for the question. Good day, everyone. Yes. Z one zero zero two top line data this quarter is a major catalyst for Harmony Biosciences.

In fact, it’s not a major catalyst. Just for Harmony Biosciences, I would go as far as and say it’s a major catalyst for patients with Frazolek syndrome, the patients, the caregivers, and the providers in general. Because this will be if the data is positive, this is going to be the first and only approved treatment for any symptom domains in patients with Frazolek syndrome. We are very excited about this, Patrick, mainly because we have a high level of confidence and conviction in this program. As we have said before, what we are trying to do in our phase three RECONNECT study is essentially replicating the statistically significant and clinically meaningful data that we saw in patients with complete methylation in the last phase twothree CONNECT study.

In terms of your question regarding what does success look like, obviously, statistically significant data on the primary endpoint, which is social avoidance, and it’s the same endpoint that we used in our phase two, three connect study and clinically meaningful data. In general, a three point within patient change is considered as clinically meaningful. That’s what we saw in our connect study, and we are confident that we’ll be seeing that in our reconnect study as well. The product profile itself is another major differentiation or a very important attribute of this program, z one zero zero two, applied as a topical gel, provides a really nice alternative for the usual treatment options, which is basically off label use of many drugs in these patients to have something especially because these patients have swallowing difficulties and also the topical application also negates a lot of the safety and tolerability issues that are often seen with oral administration of cannabidiol.

Patrick Trucchio, Senior Health Care Analyst, HC Wainwright: Right. And can you walk us through the most important design changes that were made relative to the Connect phase phase two three trial?

Kumar Boudur, Chief Medical and Scientific Officer, Harmony Biosciences: Yeah. Absolutely. The target population is patients with complete methylation. What happened in connect study it was a lot of study, by the way, over 200 patients. And about eighty percent of those patients had complete methylation.

And in those patients with complete methylation, the data was really strong. In fact, those were some of the strongest datasets ever generated in patients with Frazolek syndrome. And it all makes scientific sense because patients with complete methylation have almost complete silencing of the FMR gene, which means that they hardly have any FMR protein, which causes endocannabinoid dysfunction. And z one zero zero two by working with c b one receptors resets the endocannabinoid homeostasis. So that’s one thing.

The target population is patients with complete methylation. The primary endpoint is the same. Couple of changes that we did make based on the learnings from the CONNECT study is we increased the duration of the study from twelve weeks to sixteen weeks. That’s mainly because patients continue to get better towards the last four weeks. And again, given the mechanism of action, it makes sense that the patients do continue to get better for a period of time.

The other thing that we did, Patrick, is we increased the dose in patients who weigh more than fifty kilograms because based on the POP PK, we saw that there was room for us to go higher on the dose in patients who weigh more than fifty kilogram without compromising on safety and tolerability because z one zero zero two safety and tolerability profile is very benign. And lastly, we also made the study much more patient and caregiver friendly mainly because we know how to run orphan disease trials. That’s all we do here at Harmony Biosciences. And we definitely had a lot more resources here at Harmony compared to the previous sponsor of the study.

Patrick Trucchio, Senior Health Care Analyst, HC Wainwright: Right. And just in terms of the timing for the readout, do we have a sense of when we may have the data? And then the path forward once we have the data, you know, what does that look like? Can you you know, would you file the would you file for approval immediately? Or how does what does this look like in terms of the approval and then the eventual launch?

And maybe you could even, talk through that a bit as well.

Kumar Boudur, Chief Medical and Scientific Officer, Harmony Biosciences: Yeah. Sure. The top end data will be available this quarter, Patrick. And if the data is positive, obviously, we will immediately rush to submit an NDA. And then given the nature of the disease, given the unmet need and the fact that there are no other approved treatments in this indication, I think it’s fair to accept expect a priority review from the FDA.

If things go well as planned, we should have a drug approved for these patients by the end of next year, end of twenty twenty six. And in terms of the commercial launch and preparations, I will have Adam, my colleague, answer that question.

Adam Zeski, Chief Commercial Officer, Harmony Biosciences: And apologies. Can you repeat the question?

Patrick Trucchio, Senior Health Care Analyst, HC Wainwright: Just in terms of the the commercial expectations, preparations for commercial launch and how we should think about the, you know, product profile. I mean, clearly, the unmet need is is is very, very high, but just how you’re thinking about that commercial launch.

Adam Zeski, Chief Commercial Officer, Harmony Biosciences: That’s right. I mean, it’s a significant market opportunity as as was mentioned with, 80,000 patients. And, you know, these these patients suffer from a a variety of different symptoms, not only the behavioral, but also cognitive as well as physical. We believe that profile of our compound ZYN is uniquely positioned and has the potential, as was discussed, to be the first and only treatment approved. And we believe that the studies that we’re conducting will demonstrate the value, the risk benefit profile in a manner that will directly address unmet patient needs.

We’re really excited about the opportunity. The commercial organization right now is mobilizing, in preparations for launch with a number of different activities, everything from formal formalizing our marketing strategy, developing our go to market model, building our sales force, planning our payer access and pricing strategy. And we have great confidence that we’ll be able to launch with success.

Patrick Trucchio, Senior Health Care Analyst, HC Wainwright: Right. Terrific. So maybe just, shifting gears here a bit to capital allocation. You know, Harmony has built up this neuro rare disease pipeline over the last several years and have over has over, you know, 600,000,000 on the balance sheet. So I’m wondering what the capital allocation plans are for 2025.

Jeffrey Dano, President and Chief Executive Officer, Harmony Biosciences: Yeah. It’s Sandeep.

Sandeep Tapadia, Chief Financial Officer, Harmony Biosciences: Yeah. I think, look, we continue to be relatively active on the business development front. You know, I think one of the things that we’ve we’ve always talked about is looking to continue to build out our pipeline. As you know, we’re in a very unique position as a company with positive cash flow generating, as you mentioned, over $600,000,000 on the balance sheet. So we really have a lot of flexibility as well as given our profitability.

We have more flexibility to continue to add to our portfolio. And we’re looking at our, you know, assets both early stage as well as, you know, potentially on market, in the the neuro space overall. And and either it’s gonna be to help broaden out our sleep wake franchise or to continue to grow the neurobehavioral or epilepsy franchise as well. So maybe, Jeff, do you wanna talk a bit more about the opportunities?

Jeffrey Dano, President and Chief Executive Officer, Harmony Biosciences: Yeah. No. I I think yeah. Patrick, as Sandeep mentioned, we we feel like from a business development perspective, you know, we’re just getting started with the opportunity we have based on the strong balance sheet. I think, you know, what we’ve demonstrated, you know, is sort of thoughtful, strategic, you know, build out of the pipeline with our foundational sleep wake franchise and our behavioral franchise and and a late stage rare epilepsy assets from the Epigenetics acquisition.

So we look to either go deeper, as Sandeep alluded to, in either of those three franchises, or if there’s something compelling, you know, potentially an adjacent orphan rare sort of neuro opportunity or possibly, you know, go go even larger. I think, you know, we as we grow, we have the capacity to do that. The opportunity, Patrick, is, you know, with our unique commercial model, you know, we’re positioned where we could leverage that such as with Fragile X, and Adam was saying the go to market strategy. We could leverage, you know, the internal, the infrastructure, our single our patient hub, closed specialty pharmacy distribution system. And these indications, you know, are kind of a centers of excellence model.

So it’s not a big incremental spend to sort of go to market with, you know, either of these opportunities. So we continue to be very active. And in fact, you know, we recently we recently entered into a a small research collaboration with another company called Cirque Biosciences, which is actually a very interesting and exciting opportunity. This is a regenerative medicine company that’s developing novel therapies based on reprogramming cells, cellular reprogramming, and has the potential to replace lost cells and restore function. And we are focused on serious neurological disorders.

So the platform is based on chemically reprogramming cells, and it’s using, you know, allogeneic off the shelf, you know, kind of readily sourced GMP cell lines rather than using stem cells with this application. And this could enable a significant competitive manufacturing advantage if this platform proves to be successful. So this research collaboration is strategically aligned with Harmony’s pipeline because we’re focusing on kind of a novel regenerative cellular therapies for either refractory epilepsy. That’s one of the programs in terms of GABAergic cells, or potentially, treatment resistant narcolepsy, and looking to, you know, generate orexigenic cells. The collaboration, you know, with Circ Biosciences would expand our pipeline programs, obviously add to an earlier phase discovery program, focused on either epilepsy or sleep wake.

And this could represent, the next generation of innovative disease modifying therapies to address conditions with high unmet medical needs. So early discovery phase, strategically aligned with our pipeline and another unique opportunity for us. Let me have Sandeep in terms of the the economics. He can share, some of the economics of this research collaboration.

Sandeep Tapadia, Chief Financial Officer, Harmony Biosciences: Yeah. It was a relatively, you know, low upfront overall, as as as Jeff mentioned, fairly early in in in development. So we paid a $15,000,000 upfront for research, to begin the research collaboration, you know, and we’ll recognize a portion of that as an IPR and D charge in in the second quarter, as we as we signed it a couple of weeks back. You know? And then, obviously, as as the programs move forward, we’ll have the option to acquire the programs and take them into the clinic.

If if if successful, that’ll be about $10,000,000 each for each of the programs. And if we decide to do so, we’ll have some customary, you know, downstream milestones and royalties. We’ll share a bit more about it at our next earnings call, but but I think overall, just stepping up in terms of you know, we have a very strong balance sheet over $600,000,000, as I mentioned. So we’re well positioned to continue to look for opportunities both early phase as well as mid stage, late stage as well. So we have, as Jeff mentioned, a dedicated business development team that’s very much focused on looking for some of those opportunities.

And then this one is just, one one step, along the journey.

Patrick Trucchio, Senior Health Care Analyst, HC Wainwright: Right. That that’s really interesting. Is there, an expected, I guess, sort of timing as far as moving programs forward? Or is it, you know, I guess, you’ll you know, would you have more to say on this, you know, I guess, at a a later date?

Jeffrey Dano, President and Chief Executive Officer, Harmony Biosciences: Yeah. Patrick, it’s still early. It’s still early, and and we’ll provide more color upcoming earnings call. And and then, you know, as this platform moves forward, Kumar, obviously, you know, leading on our end with the team at Cirque Biosciences. But it’s novel.

It’s interesting. It’s a it’s a different approach to cellular replacement therapy using chemically, sort of reprogrammed, fibroblasts. And so we will, you know, we’ll share more as this opportunity, moves forward.

Patrick Trucchio, Senior Health Care Analyst, HC Wainwright: Terrific. So maybe just shifting gears on to Wakix. So Wakix grew 20% in the fifth year in the market in 2024. And in 2025, the guidance calls for $820,000,000 to $860,000,000 in revenues. Can you describe the commercial trends and market dynamics that are driving this continued growth in what is now the sixth year of the launch?

Sandeep Tapadia, Chief Financial Officer, Harmony Biosciences: Yes. Go ahead, Adam.

Adam Zeski, Chief Commercial Officer, Harmony Biosciences: Thanks, Jeff. Thanks for the question. You know, we’ve seen Waycase deliver remarkably strong and steady results, actually since launch, and continuing to add patients quarter on quarter in a steady drumbeat. That’s quite impressive. We’re very pleased with the q one results that we announced, achieving a 184,700,000.0 in net sales.

And as you mentioned, that’s a 20% increase from the prior year first quarter, in the sixth year of launch. So so really strong, steady growth. And, that is based on the fact that Wakix is a highly differentiated product as the only, the first and only nonscheduled treatment option for patients with narcolepsy. You know, Wakix having been on the market now several years, we have high awareness with physicians, and that includes all 9,000 physicians that are the prescribing universe for narcolepsy patients. Four thousand of those are oxybate REMS enrolled HCPs, but there’s another five thousand that are not enrolled in an oxybate REMS program, and that’s a unique opportunity for us and for WCAG that we continue to penetrate.

So we’ve seen consistent steady growth. We achieved, seventy two hundred average patients on therapy as of the first quarter. Again, another solid increase from prior quarters. We have broad cover payer coverage with 80% of lives covered. We have a very experienced team, with many of our commercial folks having been part of the Harmony story since its inception.

And we have a very unique commercial model with a single patient hub, three specialty pharmacies, closed distribution, white glove service that we believe provides a lot of value for HCPs and for patients. So, yes, we remain highly confident in our guidance of $8.20 to $860,000,000 for the year, and we’re also confident that we are well on our way to achieving a billion dollars in narcolepsy revenue alone prior to LOE.

Patrick Trucchio, Senior Health Care Analyst, HC Wainwright: Right. And can you tell us what does the guidance embed with respect to patient growth for the full year, and how does that compare to prior years?

Adam Zeski, Chief Commercial Officer, Harmony Biosciences: It’s very consistent with prior years in terms of patient growth. We expect to continue to see the types of additions in patients, average patients that we’ve seen in previous years, actually since inception, and we expect to, approach nearly 8,000 patients by the end of the year. This is a large market with significant unmet need. You know, 80,000 patients diagnosed, potentially another 80,000 undiagnosed. So, with WCAGX at 7,200 average patients in q one, there’s still a tremendous opportunity to continue that growth.

WCAGX is, as I said, physicians are highly aware, highly comfortable with WCAGX. They view it as well tolerated, as low drug drug interactions, and we see continued potential for penetration and the addition of new prescribers, as we continue to grow and move forward.

Patrick Trucchio, Senior Health Care Analyst, HC Wainwright: K. Great. And so when we, think about the life cycle management strategies under development, I’m wondering if, you know, at a at a high level, can you discuss where you see both the gastro resistant and high dose formulations fitting into the Wakix franchise?

Adam Zeski, Chief Commercial Officer, Harmony Biosciences: Jeff, would you like me to take that?

Jeffrey Dano, President and Chief Executive Officer, Harmony Biosciences: Yes. Yeah. Yeah. Go ahead, Adam, please.

Adam Zeski, Chief Commercial Officer, Harmony Biosciences: That’s great. Alright, Patrick. Thank you for the question. So we have two, Wakix or Pitolescent life cycle formulations that we’re really excited about. The first is the GR, the gastro resistant coated formulation of Ptolemicent.

And this is a fast to market strategy, with PDUFA expected in 2026, and also utility patents, filed through 2044. So it really allows us to expand and extend the franchise. The gastro resistant coating, it’s important to note that, you know, eighty percent of narcolepsy patients continue to struggle with GI symptoms. And the GR code coated, Ptolemson formulation, we believe, will help mitigate that need for patients. Not necessarily Wachick’s GI issues, which are relatively low, but actually the comorbid GI issues that, are a function of the disease.

It’s also a formulation that will require no titration. So patients will start immediately, at the seventeen point eight milligram therapeutic dose, which we think is important for patients in securing faster outcomes. The strategy for GR, will be to launch it as an in line, extension, and we’ll be seeking to start new patients, that would have been prescribed Wakix on the TOLIS and GR. We also have the ability because of our unique commercial model to look at patients that may have previously been on WCAG’s therapy but have discontinued, and and to communicate with them that, to ask if PATOLAS and GR might be an appropriate option, for them as well. So it’s really new patient adds as well as potentially previous patients.

Now the HD, that’s the high dose. This is also really exciting. So we have, a formulation that will also have the GR code, will also have, the no titration schedule. It’ll be up to a two times dose, and we’re pursuing a unique indication, in in terms of fatigue. And we know that sixty percent of narcolepsy patients continue to struggle, with fatigue as a symptom.

We also know that seventy five percent of patients, continue to struggle with residual symptoms in general. So we believe the HD can deliver a better therapeutic outcome, and the PDUFA is 2028. And this formulation also has, utility patents filed through 2044. The high level strategy on the HD is all new Wakix patients starting on this formulation as well as previous patients and also transitioning current WCAGX patients, to this new formulation. And we’ve tested this in market research.

We have very favorable response from both HCPs and payers, HCPs indicating they would start all new WCAGX patients on Pitolosin HD, as well as transition the majority of current WCAGX patients to this new formulation. From a payer perspective, also a very positive response. Prior to LOE, no step required through WCAGX. Even post LOE, the only potential step might be patients that have never had any WCAGX experience, in the last six or by the time we launch, eight years. But as we know, even though we’re at seventy two hundred average patients in the in the first quarter, there’s many more patients that have, been on Wakix therapy before.

And so that, that look back for the step, the potential step that it probably is a relatively small population.

Patrick Trucchio, Senior Health Care Analyst, HC Wainwright: Right. And then just a a follow-up on the high dose formulation in particular. What would you see as a potentially clinically meaningful benefit versus Wakix?

Jeffrey Dano, President and Chief Executive Officer, Harmony Biosciences: Ed Kumar.

Kumar Boudur, Chief Medical and Scientific Officer, Harmony Biosciences: Yeah. Patrick, the high dose formulation is designed based on what we know about betalacine. We have talked about this in the past. The do the dose range was never really fully interrogated when it came to betalacine or is an LP water clinical trials in narcolepsy. And we have a body of evidence to show that there is dose response, exposure response evidence for pitola site.

So we here have an opportunity to extract larger efficacy on the core symptoms like excessive daytime sleepiness and also target symptoms like, fatigue that Adam was just mentioning without compromising on the safety and tolerability of pitolacine. In fact, we actually completed a phase one b study where we looked at pitolacent all the way up to one hundred and eighty milligrams in a repeat dose study, and the safety and tolerability profile in general was consistent with the established safety and tolerability profile of pitolacent. So larger efficacy, target unique indications, and no compromise on the safety and tolerability. This is the kind of target product profile we’re looking with our pitolacent HD.

Patrick Trucchio, Senior Health Care Analyst, HC Wainwright: Right. And when you speak with payers in the high dose profile, what are what are they looking for to support moving to the higher dose? Is there a specific magnitude of benefit or endpoint that they’re focused on?

Adam Zeski, Chief Commercial Officer, Harmony Biosciences: Yeah. Thanks for the question. As I mentioned, we did do some extensive, payer market research, and we’re updating that now. And we did test different profiles. Based on the profile that we expect for the HD, that’s where the the response was very clear that there’d be favorable access, for the HD formulation.

As I mentioned, no step edits through, Wakix prior to LOE and even after LOE, a potential step edit, but only for patients that have never had any experience on Wakix, which, as I said, is a a relatively smaller population.

Patrick Trucchio, Senior Health Care Analyst, HC Wainwright: Right. And so last year, you know, acquired an orexin two agonist. I’m curious just, you know, following Takeda’s positive phase three readout, how you see your orexin program position relative to others and what aspects of profile could offer, you know, meaningful differentiation.

Jeffrey Dano, President and Chief Executive Officer, Harmony Biosciences: Kumar, do you wanna comment?

Kumar Boudur, Chief Medical and Scientific Officer, Harmony Biosciences: Yeah. Sure. Patrick, yes, we are aware of Takeda’s top line data readout from both of their phase three studies, which is good. We are happy about it. It’s a new class of innovation for patients with narcolepsy.

This will provide another treatment option if the drug gets approved by the regulatory agencies for patients with the n t one. And we look forward for additional data at the future conferences from Takeda’s compound. But in the meantime, Patrick, we are making all efforts to advance our own orexin receptor agonist. We presented the full comprehensive preclinical safety and efficacy data at the recently concluded sleep meeting last month in Seattle, and we are on track to initiate first in human studies later this year and to share some clinical data next year. What we are excited about our orexin receptor agonist is the product profile based on the data that we have generated thus far.

It continues to be the most potent orexin receptor agonist compared to anything else out there based on the data that is disclosed in the public domain. And we always focused on potency, and we said potency is important because that gives us the dosing flexibility to target all three center disorders of hypersomnolence, n t one, n t two, and IH. The reason for this statement is based on the data that we have seen so far, patients with n t two seems like they, like they need a slightly higher dose than n t one, and patients with IH need a larger dose compared to n t two. Right. So having one of the most potent orexin receptor agonist gives us the ability to tolerate all these three indications without going too high on the dose.

Not only that, good selectivity, a relatively clean preclinical safety profile, and the ability to dose once a day is what we are excited about our orexin receptor agonist. And, of course, when it comes to clinical trials, we know the sites. We know the principal investigators. We have been working with them for almost a decade now. So for us to go ahead and run these clinical trials will be a lot easier, and we are very excited to get this compound to our sites and investigators.

Jeffrey Dano, President and Chief Executive Officer, Harmony Biosciences: Yep. And I I think, Patrick, also in addition, knowing that, you know, the clinical trial sites and investigators, you know, we’re known in the market. And I’d like, you know, Adam, with all with all his commercial experience to kinda comment on, you know, the the prescribing dynamics when a new class of therapy, the the excitement. And it is a very exciting kind of new target for the treatment of narcolepsy, narcolepsy, other essential disorders of hypersomnolence. But, you know, commentary of, when those agents come to market, and kinda how that plays out, our position around that.

Adam?

Adam Zeski, Chief Commercial Officer, Harmony Biosciences: Yeah. So we’ve seen and and I’ve seen personally a lot of examples of new mechanisms of action entering a therapeutic area with significant options already available, with high excitement around efficacy. But I think we still need to wait and understand the full clinical details, see the full details of the of the larger studies, as they come out, and also weigh the the long term, impact and therapeutic impact, the risk benefit profile, and, you know, safety and tolerability in the longer term. And I think that’s, you know, that’s how HCPs will typically think of this, probably see, you know, the the KOLs, and and HCPs with large larger patient populations selectively trying these compounds as they as they are introduced, and then maybe gradual growth over time. We’re excited about the opportunity with orexins as a new treatment option for patients.

We’re confident that our own orexin molecule has potential to be best in class, and we believe the impact on Wakix for the introduction of of of orexins will be limited, at least in the short term after launch. Wakix is highly differentiated. As I mentioned, it’s the only nonscheduled treatment option. HCPs will have eight plus years of clinical experience with WakeEx at the time of of the launch of these new compounds, very familiar, high awareness perceived as well tolerated for a very broad set of patients, low drug drug interactions. And what’s also important to note in narcolepsy, there is definitely a polypharmacy approach to treatment.

So the majority of patients are on two or more therapies, and we expect that approach to continue. We also know and have evidence that orexin and histamine have a synergistic effect, And so we remain confident in the continued growth potential of Wakix even in the light of a new class entering. And that’s actually what we’ve seen some in in the six years since launch with new brands and generics entering the market. Wakix continues its very steady and consistent growth profile.

Patrick Trucchio, Senior Health Care Analyst, HC Wainwright: Right. That that makes sense. And then you’ve also acquired an epilepsy asset, EPX 1 100. I’m wondering if you can just provide some background on this compound, why it was attractive, where you see the unmet need in epilepsy, and then we’ll have a few follow ups.

Jeffrey Dano, President and Chief Executive Officer, Harmony Biosciences: Sure. Yeah. Go ahead, Kumar. We we are very excited about the the late stage, epilepsy, you know, pipeline, and Kumar can, you know, share, you know, what we saw in that and where where the programs are.

Kumar Boudur, Chief Medical and Scientific Officer, Harmony Biosciences: Yeah. Thanks, Jeff. Patrick, if we take a step back, a lot of thought and strategy went into our decision to move into epilepsy space. Because as you know, epilepsy continues to be one of the highest unmet medical needs despite so many medicines abroad Because patients who are currently on various combination of medicines, they either continue to have seizures or they have some safety and tolerability issues that requires regular monitoring of liver function tests regularly, checking the therapeutic levels of the drugs because many of these drugs have not a therapeutic window. What we see in field of epilepsy is even if there is a small incremental improvement in efficacy, safety, tolerability, this is embraced by patients and the providers alike.

So with Epigenetics acquisition, we saw a really nice opportunity to make an impact in developmental and epileptic encephalopathies, which actually has the highest unmet need compared to epilepsy in general. So with Epigenetics acquisition, we got two compounds, epx 100, which is clamsol hydrochloride, the lead compound, and epx 200, which is liquid low acid formulation, which is in the IND enabling stage. With EPX 100, clamsol hydrochloride, established mechanism of action when it comes to treating developmental epileptic encephalopathies. It’s a five s t two serotonin agonist, and the data is very clear that if we increase the serotonergic tone, it helps with seizures in patients with developmental and epileptic encephalopathies. Good preclinical proof of concept data.

Currently, we are running two pivotal phase three registrational studies, one in Dravet syndrome and one in Lennox Gastaut syndrome. We are making good progress, anticipate top line data in 2026. And the differentiation here, Patrick, is really more on the safety and tolerability perspective. Unlike other serotonergic drug that is approved, which requires regular monitoring with echocardiogram, we don’t need that with clamsol hydrochloride. There is a body of safety and tolerability data from clamsol hydrochloride which was originally approved as an antihistamine in nineteen sixties and seventies.

And we did complete preclinical doc studies where we did not see any issues for concern. And the other thing is it also doesn’t require regular monitoring of liver function tests. For example, like Epidiolex, which is also approved for development and epileptic encephalopathies for and ELGS. So we are looking forward to bring a drug to patients with good efficacy, very good safety and tolerability with no need for any special laboratory monitoring or any special medical monitoring.

Patrick Trucchio, Senior Health Care Analyst, HC Wainwright: Great. And so just a a few follow-up questions then. I mean, you know, clearly, differentiation should be on the safety and tolerability side. Epilepsy, though, is also it’s also a a polypharmacy market. So I’m wondering, you know, how we should think about potential for combination of BPX100 with other treatments as well, you know, the indications LGS and Dravet, where the, you know, what you need to demonstrate in terms of of seizure reduction, you know, to have a place in those with those indications.

And then, the potential actually to broaden the label to include, you know, additional additional subtypes of of epilepsy and how you’re viewing that that possibility. So I know there’s a couple of questions in there, but as we get this, you know, the phase three data, you know, would we start to have some answers to this, or how how are you thinking about the program going forward?

Kumar Boudur, Chief Medical and Scientific Officer, Harmony Biosciences: Yeah. I mean, the the trial great questions, Patrick, because this all have implications on how the drug will be described by the prescribers to the patients. Right? So in terms of the polypharmacy, you’re absolutely right. I mean, any patient currently who has DEAs on IVs are on about four different medicines, sometimes up to six different medicines, sometimes on label, and many of many a times off label.

And that would that’s what we are seeing in our clinical trials as well. The clinical trial design itself, Patrick, is very well established. The same design, the same endpoint is accepted not just by FDA and EMA, but other regulatory agencies across the world. In terms of the concomitant medications, we haven’t seen any significant drug drug interactions to the point where we will have to exclude some of the commonly prescribed medicines for DES. In fact, many patients who come into the trials are actually on these medicines and they still have uncontrolled seizures meeting the severity that is required for the inclusion criteria.

So it’s fair to assume that this will be another option for prescribers to prescribe on top of everything else that the patients are already taking. This, again, speaks to the high unmet need in this particular area.

Patrick Trucchio, Senior Health Care Analyst, HC Wainwright: Right. Terrific. So maybe just one one last one just to kind of bring it bring it all together here. You know, it’s, I think, you know, 2025 has been referred to as a transformational year. So I’m wondering what, you know, what would you like investors to understand or revisit about the Harmony story at this point in time?

Jeffrey Dano, President and Chief Executive Officer, Harmony Biosciences: Yeah. Patrick, I think, yeah. You know, as you heard from the the team, you know, this morning, I think Harmony continues to be a growth story, and and not only, you know, wake ups and narcolepsy, but the advancement of the pipeline. And and I think that the main takeaway is kind of the value, you know, the potential value of our pipeline programs that are late stage, you know, with near term catalysts. You know?

In fact, if we look at our existing pipeline, you know, it’s poised to deliver, one or more new product or indication launches each year over the next four to five years, and that’s significant. And these are late stage programs, as Kumar mentioned, often replicating, you know, positive findings from phase two programs designed into into phase three. So I I think that that is the main takeaway. And I think, you know, the time is now. We are focused on advancing these late stage development programs.

We are very excited about the fragile X opportunity with ZYN two. And I think our goal, bringing new products to market, helping these rare disease patient communities with new innovative treatment options while driving long term value creation for our shareholders. And that is the story. And the time is now with these late stage programs moving forward and, you know, on the verge of multiple NDA submissions over the next year or two.

Patrick Trucchio, Senior Health Care Analyst, HC Wainwright: All right. Terrific. Well, we look forward to, you know, continued stellar growth in WCAGX and these late stage readouts as well. It sounds like some very interesting early stage programs as we get this detail going forward. So I’d like to thank the leadership team at Harmony for joining us this morning.

And I’d like to thank everyone for, also joining us, you know, for the for this very interesting discussion. So everyone have a great rest of your day and great rest of your week.

Jeffrey Dano, President and Chief Executive Officer, Harmony Biosciences: Great. Thank you, Patrick. Thanks, everyone.

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