Harmony (HRMY) Q1 2026 Earnings Transcript

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DATE

Thursday, May 7, 2026 at 8:30 a.m. ET

CALL PARTICIPANTS

• Chief Executive Officer — Jeffrey Dayno
• Chief Commercial Officer — Adam Zaeske
• Chief Medical and Scientific Officer — Kumar Budur
• Chief Financial Officer — Glenn Reicin
• Chief Operating Officer — Peter Anastasiou

TAKEAWAYS

• Net Revenue -- $215.4 million, representing 17% growth year over year, with demand attributed to WAKIX performance and partially offset by typical Q1 market access headwinds.
• Patient Metrics -- Average of 8,500 patients on WAKIX in the quarter, exiting with approximately 8,600 patients, in a total diagnosed narcolepsy population of 80,000.
• Full-Year Revenue Guidance -- Management reiterated guidance of $1 billion to $1.04 billion in net revenue for the year.
• Cost of Goods Sold -- 20.7% of net sales, up from 17.3% in the prior year period, driven primarily by royalties under the Novitium license agreement.
• Operating Expenses -- $133.6 million compared to $96.5 million the previous year, including $32 million of upfront licensing fees related to the amorphous pitolisant license; otherwise up approximately 5%.
• GAAP Net Income -- $32.5 million, or $0.55 per share, compared with $45.6 million, or $0.78 per share, in the same quarter last year; the current figure includes $0.45 per share in upfront licensing fees.
• Cash and Debt Position -- $870.5 million in cash and cash equivalents and $160 million in debt at quarter-end, with cash flow generation reduced by licensing fees and working capital impacts.
• WAKIX Franchise Exclusivity -- Patent coverage supports WAKIX exclusivity until March 2030, inclusive of six months pediatric exclusivity, with potential extended protection into the 2040s on the broader pitolisant franchise due to additional patents.
• Pipeline Advancement -- Five ongoing Phase 3 registrational trials across five CNS indications, including EPX-100 for Dravet and Lennox-Gastaut syndromes and continued development of BP-205 (Orexin-2 agonist), with Phase 1 clinical PK data for BP-205 expected mid-year.
• Pitolisant GR NDA Timeline -- NDA submission for Pitolisant GR (gastroretentive) on track this quarter, targeting a PDUFA date in Q1 2027.
• Pitolisant HD Trials -- Two Phase 3 registration trials ongoing, aiming for differentiated labeling in narcolepsy and idiopathic hypersomnia; top-line data expected in 2027 and a target PDUFA in 2028.
• Field Team Expansion -- A 20% increase in field and reimbursement teams completed, representing the largest brand investment to date, effective as of April 1.
• Business Development Focus -- Renewed emphasis on business development, prioritizing assets with revenue potential between 2028 and 2032, focusing on Phase 3, registration-stage, or in-market opportunities in sleep/wake, epilepsy, rare CNS, and adjacent indications.
• Ongoing Litigation -- Six of seven ANDA filers have settled, maintaining loss of exclusivity (LOE) in March 2030; active patent infringement lawsuit against AET Pharma U.S. and Sandoz targets the amorphous pitolisant hydrochloride patent licensed from Novitium.
• EPX-100 Clinical Results -- Data from EPX-100's open-label extension in Dravet Syndrome showed median 50% reduction in seizures after six months, with 50% of patients achieving at least 50% reduction, and a favorable safety profile noted.

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RISKS

• Cost of goods sold increased to 20.7% of net sales from 17.3%, almost entirely driven by new royalties related to the Novitium license agreement.
• Operating expenses rose significantly to $133.6 million versus $96.5 million, including $32 million in upfront licensing fees that impacted profitability.
• cash flow generation in the quarter was muted by the licensing fees and a large reduction in accrued expenses and a modest reduction of debt.
• BP-205 is entering a competitive landscape. So Youn Shim noted, "the flea space may be very crowded with a number of Orexin agonists by the time 205 launches," indicating future commercial challenges.

SUMMARY

Management emphasized execution across four pillars: protecting the pitolisant franchise, accelerating WAKIX commercial growth, advancing a robust CNS pipeline, and deploying significant capital into business development for 2028-2032 revenue opportunities. WAKIX’s average patient count increased, and field expansion initiatives were completed, while full-year revenue guidance was reiterated at $1 billion to $1.04 billion. Novitium-based patents and pending litigation reinforce exclusivity through 2030 with prospects for further extensions. Cash reserves exceeded $870 million after Q1, providing capacity for M&A and licensing. Program milestones include Phase 1 PK data for BP-205 mid-year, pending NDA for Pitolisant GR, and top-line data expectations for both Pitolisant HD and EPX-100 ahead of regulatory submissions in 2027-2028.

• Management stated, "we remain confident in the strength of our IP estate," referencing layered protection through both settled and active legal actions targeting exclusivity.
• Persistency on WAKIX has remained very, very steady for many years, and we have not seen any meaningful change in that dynamic indicating low churn despite access delays.
• Management noted intent to pursue opportunities with revenue potential in the 2028 to 2032 time frame, prioritizing assets that are in Phase 3 development in registration or on market.
• Chief Financial Officer Glenn Reicin said, “We have quite a bit [of deal capacity] given our cash balance and our ability to borrow as well. So we have quite a bit of capacity and size deals, I think, are not a limiting factor.”
• Pitolisant GR is designed to reduce the potential for GI side effects in patients prone to GI symptoms, with NDA expected this quarter and anticipated PDUFA Q1 2027.
• The company confirmed ongoing preclinical work on new CNS indications for BP-205 and additional orexin-2 agonist compounds for both monotherapy and combination therapies.

INDUSTRY GLOSSARY

• Orexin-2 Agonist: A class of compounds designed to activate Orexin-2 receptors, relevant for treating central disorders of hypersomnolence and broader CNS indications.
• PDUFA Date: The target action date by which the FDA is expected to complete review of a New Drug Application (NDA) under the Prescription Drug User Fee Act.
• ANDA Filer: An entity submitting an Abbreviated New Drug Application, often to obtain approval for a generic equivalent of a branded product.
• Amorphous Pitolisant Hydrochloride: A non-crystalline form of pitolisant hydrochloride, now the subject of new patents and ongoing infringement litigation.
• Phase 3 Registrational Trial: Late-stage clinical study intended to provide definitive efficacy and safety data to support regulatory approval in a specific indication.

Full Conference Call Transcript

Jeffrey Dayno: Thank you, Brennan. Good morning, everyone, and thank you for joining our call today. I want to start by highlighting what we have built at Harmony. We are a profitable, self-funding biotech company that continues to operate from a position of strength. We have an exceptionally strong commercial engine, which continues to drive WAKIX growth now in year 7 on the market. We have recently solidified our IP position around the pitolisant franchise through a multilayered approach. We have a robust pipeline centered around BP-205, our potential best-in-class Orexin-2 agonist. And we have a very strong balance sheet with the capacity and conviction to execute on meaningful business development opportunities.

With this solid foundation in place, we are now turning our focus on 4 key pillars of value creation for shareholders. In support of this next phase of growth, we have brought in 2 new members to our management team who further strengthen our ability to execute as we scale. First, I am pleased to welcome our new Chief Financial Officer, Glenn Reicin. Glenn is a seasoned biopharmaceutical executive with extensive experience across publicly traded and privately held companies, guiding them through key growth inflection points. With his experience as a sell-side analyst, investor and biotech CFO, Glenn brings a strong track record of success in strategic planning, capital markets and fundraising.

Next, welcome to Peter Anastasiou, our new Chief Operating Officer, who brings more than 30 years of experience in the industry and comes into this role after having served on Harmony's Board of Directors where he contributed strategic insights across the business during a period of continued growth and operational evolution. Most recently, Peter served as Chief Executive Officer of Capsida Biotherapeutics and previously held multiple leadership roles at Lundbeck, including President of U.S. and Canadian Operations, and U.S. Chief Commercial Officer for its psychiatry and neurology franchises. I am excited to welcome both Glenn and Peter to the Harmony team.

Their combined experience meaningfully strengthens our leadership bench and positions us well to execute on our 4 strategic pillars of value creation, which I will now walk you through. The first pillar is to protect the pitolisant franchise into the 2030s. We are committed to protecting the pitolisant franchise through a multipronged strategy that is based on strong intellectual property protection and exclusivity. Pitolisant's IP estate is multilayered, covering different formulations, methods of use and next-generation applications, which support WAKIX exclusivity into 2030, inclusive of 6 months of pediatric exclusivity with potential IP protection of the franchise into the 2040s via additional issued patents and pending patent applications.

In January, we also acquired an exclusive license from Novitium for certain IP, including their issued patents out to 2042, covering an amorphous form of pitolisant hydrochloride, which provides us with new development opportunities. With regard to the ANDA litigation and the trial that took place in February, we have settled with 6 of the 7 ANDA filers, maintaining LOE until March of 2030, inclusive of 6 months of pediatric exclusivity. During the trial in February, AET, the one remaining ANDA filer who has not settled, stated that their product contains an amorphous form of pitolisant hydrochloride rather than a crystalline form covered by our 197 polymorph patent.

After learning that, Harmony, along with Novitium, recently filed a patent infringement lawsuit against AET Pharma U.S. and Sandoz, alleging infringement of a patent covering an amorphous form of pitolisant hydrochloride. This suit is a new action, different from the legal proceeding of the ANDA litigation trial that took place in February, which is ongoing and is being presided over by the same judge. Based on this legal activity, we remain confident in the strength of our IP estate and continue to vigorously protect it through this multipronged strategy. Second, we are dedicated to the continued growth of the pitolisant franchise in an evolving market.

In Q1, we delivered $215.4 million in net product revenue, up 17% from $184.7 million in the same period last year. This performance reflects continued strong demand, offset by market access headwinds observed every Q1 following 3 of the strongest consecutive quarters in Harmony's history. The average number of patients in Q1 was approximately 8,500, and we exited the quarter with approximately 8,600 average patients on WAKIX. These patient numbers are in a market of 80,000 diagnosed patients with narcolepsy, showing the significant market opportunity that remains to support continued growth of WAKIX and the pitolisant franchise. Based on this, we are reiterating our full year net revenue guidance of $1 billion to $1.04 billion.

In addition to the continued growth of WAKIX in the market, our next-gen pitolisant formulation programs are advancing. Pitolisant GR remains on track for NDA submission this quarter with a target PDUFA date in Q1 2027, and is positioned to extend the WAKIX franchise and our leadership in narcolepsy. Pitolisant HD is enrolling in 2 ongoing Phase 3 registrational trials, one in narcolepsy and in idiopathic hypersomnia, or IH, designed to expand the franchise with differentiated labeling. We have also initiated a new development effort with the amorphous form of pitolisant that we licensed from Novitium. This gives us an opportunity to pursue broader CNS indications, and Kumar will share more color on this new opportunity later in the call.

Our third pillar of value creation is driving value from our robust pipeline, led by BP-205, our potential best-in-class Orexin-2 agonist. We do not believe our current valuation reflects the strength of our pipeline, especially related to our highly potent and selective Orexin-2 receptor agonist, BP-205. This program is on track for top line data readout from our Phase 1 clinical PK single ascending dose study in the middle of this year.

Built on a novel chemical scaffold with strong preclinical PK, safety, efficacy and tox data, BP-205's potential product profile could position it as a highly competitive Orexin-2 agonist, not just in narcolepsy and other central disorders of hypersomnolence, but also for broader indications outside of sleep/wake that are not due to Orexin deficiency for which potency matters. Overall, our robust late-stage pipeline consists of 5 ongoing Phase 3 registrational trials across 5 distinct CNS indications, including the 2 Phase 3 registrational trials with EPX-100 in the rare developmental and epileptic encephalopathies. Kumar will provide more detail on BP-205 and our other pipeline programs during his R&D update. And our fourth value-creating pillar is a renewed emphasis on business development.

We are focused on opportunities with revenue potential in the 2028 to 2032 time frame, prioritizing assets that are in Phase 3 development in registration or on market, and we are maintaining our therapeutic areas of interest that include sleep/wake, epilepsy, rare orphan CNS disorders and CNS adjacencies beyond rare diseases. With about $870 million on the balance sheet, we are moving with urgency to deploy our capital and have clear conviction to execute on strategic business development opportunities. In summary, I've outlined 4 pillars of value creation that we believe matter most to investors and will serve as the framework by which our performance will be measured.

Next, the team will share with you the highlights of how we are executing on each of these strategic pillars. With that, I'll turn the call over to Adam Zeskey, our Chief Commercial Officer, for an update on our commercial performance. Adam?

Adam Zaeske: Thank you, Jeff, and good morning, everyone. We're off to a strong start in the first quarter with WAKIX delivering $215.4 million in net sales, representing 17% year-over-year growth, now in its seventh year on the market, which is in line with the growth required to achieve full year guidance of between $1 billion and $1.04 billion. WAKIX achieved approximately 8,500 average patients in the first quarter. The Q1 typically has lighter patient additions due to seasonal dynamics the entire industry experiences every year. This year, we saw slightly higher market access headwinds than previous years with a higher level of plan changes, planned switching by consumers and higher premium increases, which can delay patient starts.

That said, we're coming off our strongest ever 3 consecutive quarters of patient adds. First quarter demand was actually higher than the first quarter in 2025 and was in line with our recent performance and expectations. In March, we saw a higher level of new prescriptions in all but 2 months in 2025, and we exited the quarter with 8,600 patients on WAKIX. The driving force behind this continued performance is clear. WAKIX owns a unique and highly differentiated position as the only non-scheduled treatment option for narcolepsy patients. After 7-plus years of clinical experience, health care providers are deeply aware of WAKIX.

They believe in its combination of efficacy, safety and tolerability and low drug-drug interactions as well as its broad payer coverage, making it a familiar go-to option for any patient with narcolepsy and in any combination with other therapies in a highly polypharmacy market. And this will continue to remain true as the market continues to evolve. We've now also completed our field team expansion, which has expanded our presence by roughly 20% across field sales, remote sales and field reimbursement. This represents the largest expansion and increase in investment in the history of the brand. All of those positions have been hired and are in place as of April 1.

In Q2, we will launch a new online portal using the process to prescribe WAKIX for healthcare providers and office staff, and we will execute significant changes to our reimbursement support process, helping patients to secure a WAKIX dispense faster and with higher success rate. Looking to the future, we're excited about our 2 life cycle management strategies to extend and expand the pitolisant franchise. Pitolisant GR will build on the strength of WAKIX safety and tolerability profile and allow patients to gain faster results by starting at a therapeutic dose.

Pitolisant HD, or high dose, will bring a totally new optimized formulation to the market with up to 2x the approved dose of WAKIX, all of the benefits of the GR formulation and differentiated labeling regarding fatigue in narcolepsy and sleep inertia in IH. We're also excited about our orexin compound as we see an opportunity remains for Harmony to deliver an orexin that delivers on the promise of efficacy with a better safety and tolerability profile, once daily dosing and indications across NT1, NT2 and IH.

To summarize, we're on track to achieve $1 billion plus in net sales this year, and we're excited about our pipeline with multiple assets that will extend our leadership in sleep/wake for at least the next decade. I'd like to now turn the call over to our Chief Medical and Scientific Officer, Kumar Budur, to discuss the advancements in our clinical development programs. Kumar?

Kumar Budur: Thank you, Adam. Good morning, everyone, and thank you for joining us today. Q1 2026 was a quarter of continued progress across our pipeline. We have 5 ongoing Phase 3 registrational clinical trials across 5 distinct CNS indications, and I'll walk you through the key updates. As just noted, orexin is one of the most exciting areas in sleep/wake and broader neuropsych indications. I'd like to start with the progress we are making with our Orexin-2 receptor agonist, BP-205, an asset we believe has meaningful potential that is not yet reflected in how our pipeline is being viewed today.

BP-206 with its unique chemical scaffold has demonstrated compelling preclinical potency, selectivity, safety and efficacy and the potential for once-a-day dosing all supportive of a potential best-in-class Orexin-2 receptor agonist. It is the most potent Orexin-2 receptor agonist in clinical development and demonstrated weight promoting asset at the lowest dose tested in the transgenic mouse model for narcolepsy. The high potency gives the flexibility to use low doses and treat all 3 central disorders of hypersomnolence that is NT1, NT2 and IH with the same compound. BP-205 also have high selectivity for Orexin-2 receptors over Orexin-1 receptors and 150 other receptors of interest.

We continue to dose healthy volunteers in our Phase 1 SAD/MAD PK study in Europe, and we remain on track for Phase 1 SAD PK data in mid-2026. We are also on track for U.S. IND submission in mid-2026 and commence sleep deprived healthy volunteer study in the second half of this year. As we make progress with our lead asset, DP-205, targeting central disorders of hypersomnolence, we are also working on preclinical experiments in broader neuropsych indications targeting mood, ADHD, cognition and fatigue. Now moving to pitolisant and the life cycle opportunities, beginning with Pitolisant GR. We remain on track for NDA submission in Q2 2026 and a target PDUFA date in Q1 2027.

About 80% to 90% of patients with narcolepsy have GI symptoms related to the pathophysiology of narcolepsy itself. Pitolisant GR is designed with enteric coating meant to reduce the potential for GI side effects in patients prone to GI symptoms. Pitolisant GR demonstrated bioequivalence to WAKIX in our pivotal D study and it also allows patients to start at the therapeutic dose range of 17.8 milligram without titration, an important clinical differentiation. Pitolisant HD continues to advance in 2 Phase 3 registration trial: ONSTRIDE 1 in narcolepsy and ONSTRIDE 2 in idiopathic hypersomnia. We anticipate top line data in 2027 and a target PDUFA date in 2028.

These programs are pursuing differentiated labels, fatigue in narcolepsy and sleep inertia in IH, addressing symptoms for which there are currently no approved treatments. Both Pitolisant GR and Pitolisant HD have utility patents filed until 2044. We are also optimistic about the novel amorphous form of pitolisant we recently licensed from Novitium supported by an issued patent through 2042. We believe this could enable new modes of delivery and broaden pitolisant's potential in larger CNS indications. We are currently optimizing formulation with the goal of commencing a Phase 1 PK study.

On pediatric exclusivity for WAKIX, the top line data from the Phase 3 TEMPO study in Prader-Willi syndrome is anticipated in second half of 2026, a key requirement for achieving pediatric exclusivity, which gives us additional 6-month regulatory exclusivity at the back end of the longest patent for WAKIX. Moving to our epilepsy franchise. EPX-100 continues to advance in 2 global Phase 3 registrational programs, the ARGUS study in Dravet Syndrome and the LIGHTHOUSE study in Lennox-Gastaut Syndrome. We recently presented Encore safety and effectiveness data at the AAN meeting from the open-label extension study in Dravet Syndrome that showed clinically meaningful reduction in seizures and a favorable safety and tolerability profile.

We now have sites actively enrolling patients in India and China in addition to North America and Europe to accelerate recruitment. We anticipate top line data in first half of 2027 and a target PDUFA in 2028. Finally, on behalf of Harmony, I would like to thank all the patients and their families who are participating in our clinical trials as well as the clinical investigators and site personnel for their dedication and commitment in helping us advance our development programs. I now turn the call over to the newest member of our team, CFO, Glenn Reicin, for an update on our financial performance. Glenn?

Glenn Reicin: Thank you, Kumar, and good morning, everyone. I'm delighted to join this organization and help contribute to the growth and significant value creation. And I should mention that I was drawn to Harmony by the strength of its commercial foundation and self-funding financial model that positions us to build and grow from a place of strength. This morning, we issued our first quarter 2026 earnings release and filed a 10-Q where you'll find the details of our financial and operating results. We delivered solid financial results that reflect the continued underlying demand for WAKIX and our disciplined approach to managing expenses across the business.

For the first quarter of 2026, we reported net revenues of $215.4 million compared to $184.7 million in the prior year quarter, representing 17% growth. Performance in the quarter reflects the strong underlying demand for WAKIX, partially offset by the seasonal market access headwinds the industry sees at the start of every year. Cost of goods of products sold was 20.7% of net sales and compares with 17.3% one year ago. This year-over-year increase in cost of products sold as a percentage of net revenue was almost entirely driven by new royalties related to the Novitium license agreement, providing new development opportunities in broader CNS indications.

We reported total operating expenses for the first quarter of $133.6 million compared to $96.5 million in the same quarter in 2025. The growth in expenses reflects continued investment in R&D and ongoing investments in the commercialization of WAKIX in narcolepsy. These operating expenses also include the costs associated with upfront licensing fees of $32 million or $0.45 per share on a fully tax basis related to the amorphous license agreements. Without these costs, operating expenses were up around 5%. GAAP net income for the first quarter of 2026 was $32.5 million or $0.55 per share. This compares with $45.6 million or $0.78 per share.

Of course, that includes $0.45 in costs related to the in-license of the amorphous form of pitolisant and new development opportunities. We ended the first quarter with $870.5 million in cash and cash equivalents cash flow generation in the quarter was muted by the licensing fees and a large reduction in accrued expenses and a modest reduction of debt. We ended the quarter with $160 million in debt. Cash flow generation will reaccelerate in the coming quarters. That said, it's our intent to deploy cash into business development with the objectives of enhancing revenues in the 2028 to 2032 time frame, consistent with our 4 key pillars of value creation for shareholders.

And with that, I'd like to turn the call over to Jeff for his closing remarks. Jeff?

Jeffrey Dayno: Thank you, Glenn. And again, welcome to the team. In closing, I have shared with you our 4 pillars of value creation that we feel matter most to investors and will serve as the framework by which our performance will be measured. First, protect the pitolisant franchise into the 2030s. Second, continued growth of the pitolisant franchise in an evolving market. Third, drive value from our robust pipeline centered around BP-205, our potential best-in-class Orexin-2 agonist. And last, a renewed emphasis on business development with our goal to transact.

We believe when we execute on these 4 strategic pillars, we will be well positioned to bring innovative treatments to patients with unmet medical needs while driving sustained long-term value for our shareholders. Thank you for your attention. I will now turn the call back over to the operator for Q&A. Operator?

Operator: [Operator Instructions] We have the first question comes from the line of David Amsellem of Piper Sandler.

David Amsellem: I have a few questions on BP-205. Just give us a road map on how to think about data milestones as we move through '27, particularly in healthy sleep-deprived volunteers. And then secondly, why not just move right into actual narcolepsy 1 and narcolepsy 2 and IH patients in a Phase 1 B, just given that it's become a pretty crowded landscape just as a way to expedite development. I wanted to get your thought process there.

And then lastly, with other indications, mood, ADHD, cognition, fatigue, as you alluded to, can you talk about early-stage work that you're going to be doing over the next 12 to 18 months that could help you decide what paths to take in terms of the non-sleep break indications?

Jeffrey Dayno: David, thank you for your question. With regards to BP-205, first, let me frame overall our excitement around the potential opportunity we have with our Orexin-2 agonist. And it's really based on, as you've heard us say, the novel chemical scaffold and really the highest potency of any Orexin-2 agonist in the clinic. With that, it gives sort of the differentiated features with a product profile likely to be best-in-class. With regards to timing of the development program and other indications, first, the issue with indications outside sleep/wake and central disorders of hypersomnolence, the opportunity there is that those are not based on a state of orexin deficiency.

And in those conditions, potency matters where we think we would have a significant opportunity, not just with BP-205, but other compounds that we're working on with our partner, Bioprojet. So now I'll ask Kumar to sort of provide what the time line looks like on the current development program. Kumar?

Kumar Budur: Thank you, Jeff. David, you asked several questions, so let me try and answer one by one. In terms of data milestones, we are on track to receive single ascending dose clinical PK data in the middle of this year. We also continue to dose patients in multiple ascending dose study, the food effect study, things like that, that are important before we go to the later clinical stage of development. In terms of your question, why not go straight into clinical proof of concept rather than sleep-deprived healthy volunteer study? Great question, David. One thing to remember is BP-205 is the most potent orexin receptor agonist in clinical development.

So it's good to get a nice bracket around the dose range before we go into clinical proof-of-concept studies. And also another thing to remember is this is not going to add to the time line. This is done in parallel with the multiple ascending dose study and the data we need that before we go into clinical proof-of-concept studies. You also talked about the other indications as we are very excited about the profile of BP-205, most potent orexin receptor agonist, and Jeff alluded to some extent in his response, it's very important that potency translates into efficacy, and we saw that in our preclinical animal models.

And in other indications, it becomes all the more important because we'll be depending on some of the downstream effects of the orexin receptor agonist in conditions like mood, fatigue, cognition, ADHD. We are conducting several preclinical experiments right now. And as the data becomes available, we'll be able to disclose. I hope that answers all of your questions. Thanks, David.

Jeffrey Dayno: Yes. And David, final comment on that. I think as I've discussed with the team, given the potential value of this class of compounds, it's our intent to invest in this program with regards to where we are now on additional compounds and look at the broad opportunity with not just BP-205, but other compounds that we're working on. Thanks, David.

Operator: [Operator Instructions] Next question comes from the line of Ami Fadia from Needham.

Ami Fadia: Maybe one main question and a follow-up. You gave us some sense about the litigation versus AET Sandoz and then there seem to be 2 trials here. Can you talk about kind of what we can expect in terms of next steps with regards to how the litigation might proceed and over the course of the next couple of months? And then a follow-up on the orexin question from earlier. With the sort of higher potency that you've highlighted, how do you see it being differentiated in the sleep/wake area relative to the other orexins that are in development? And then can you elaborate a little bit more on the other indications?

Do you see that other indications such as ADHD, mood, fatigue, et cetera, being ones where you would study it as a combination treatment with other assets? Or do you see it being developed both as a monotherapy and then also explore other combinations?

Jeffrey Dayno: Let me address your first question, and then I'll have Kumar address the follow-up on BP-205. So I want to take a minute or 2 to provide clarity with regards to the IP situation and around the WAKIX and pitolisant IP estate. Before I provide some detail, I first want to be clear about our position. We believe in the strength of our IP, and we continue to vigorously protect it. And it's based on a multipronged strategy with pitolisant IP estate, which is also multilayered. So with regard to some of the detail to provide clarity. So recall, we settled with 6 of the 7 ANDA filers. AET was the one remaining party who went to trial in February.

That legal process continues. It's currently in post-trial briefs, and that part of the process will be completed towards the end of May. So during the trial in February, AET and Sandoz stated that the product will contain an amorphous form of pitolisant hydrochloride rather than a crystalline form, which is covered by our 197 polymorph patent. And they did that in an effort to work around that IP. So we believe that AET and Sandoz infringed our 197 patent and during the trial, they also admitted to the facts that they infringed the 920 amorphous patent that we licensed from Novitium.

Based on that, when we learned about the trial, that is when Harmony Novitium recently filed the patent infringement lawsuit against AET Sandoz, alleging infringement of the 920-patent covering an amorphous form of pitolisant hydrochloride. So that suit is a new and separate action, different from the legal proceedings of the ANDA litigation that took place in February, which is ongoing. And that new suit is also being presided over by the same judge. That is a new legal proceeding, which is just getting started.

So based on this multipronged strategy, the different factors that I just mentioned, we remain confident in the strength of the IP estate, again which is multilayered with multiple components, enabling exclusivity into 2030, inclusive of 6 months of pediatric exclusivity. Kumar, over to you.

Kumar Budur: Thank you, Jeff. Good morning, Ami. So in terms of potency, you may have heard me talk about it many, many times in the past as well why potency matters with Orexin-2 receptor agonist. It matters because it gives us the ability or the flexibility to use low doses to target all 3 central disorders of hypersomnolence. How it could potentially manifest itself in a clinical setting? Well, with low doses, we'll be able to avoid the off-target EAEs. That's number one. The number 2 is having a high potent drug. As you know, the doses required for NT2 and IH are relatively larger based on the data that we have.

So it provides us an opportunity to use the same compound to treat all 3 central disorders of hypersomnolence, NT1, NT2 and IH rather than use different compounds or go to high doses, which may actually result in some EAEs. In terms of the combination, that's a great question, Ami. There is a scientific rationale to potentially realize synergistic effect by combining pitolisant, which is a histaminergic drug with an orexin agonist because the neurocircuitry is such that orexin acts at a higher level and the neuronal connections between the orexinergic neurons and histaminergic neurons in tuberomammillary nucleus is very well established. So this is something that we are very interested in, and we are looking into.

Operator: We have the next question comes from the line of Graig Suvannavejh form Mizuho.

Graig Suvannavejh: I've got 2. My first, just if we could just talk about the WAKIX ramp right now. My apologies, I was a bit late on the call. I know that you're reiterating the guidance for this year and that you added 100 net patients. Should we expect the balance of the year to pretty much reflect what we've seen in the past in terms of second quarter, third quarter and fourth quarter dynamics? Or is there anything this year as the product matures that we need to be aware of? And then just secondly, going back to the ongoing litigations that are ongoing.

I was curious if you could provide a sense of in terms of the timing of when you decided to sue on the second lawsuit on the potential Novitium IP that you licensed in. I was curious as to why you chose to sue when you did versus perhaps waiting for the outcome of the original IND litigation on a view that, at least based on our work, I think there's still a potential chance that you would win.

Jeffrey Dayno: With regards to WAKIX performance, let me just sort of frame and then over to Adam for more commentary. So I think we're off to a strong start with good strong demand that continues for the WAKIX franchise as well as through the quarter, we saw good momentum coming out of the first quarter, exiting with about 8,600 average patients. So with that, we're on track to achieve full year guidance of over $1 billion. And I think Adam can provide more color on the continued growth of the WAKIX franchise.

Adam Zaeske: Thanks, Jeff. Graig, thanks for the question. So in the first quarter, we achieved $215.4 million in net sales. That represents about 17% growth year-over-year. And that is exactly the growth level that we need to achieve in order to hit our full year guidance of $1 billion to $1.04 billion for the full year. Graig, as you know, and we've talked about this before, we typically see seasonal headwinds in the first quarter as we start the year related to market access and payer changes. And we saw that again this year, no surprise. So plan changes, consumer switching plans, increases in premiums. These are factors that can delay some patient starts as we start the new calendar year.

And we saw that again this year. That said, underlying demand remains very strong. We're just coming off 3 consecutive quarters of record growth, and we saw the same level of demand in terms of new prescriptions and new patient starts, consistent with that recent performance. And actually, first quarter demand this year was even higher than it was a year ago. And in March, we saw the highest demand that we've seen in all but 2 months in 2025. So the momentum is there. To answer your question directly, yes, we would expect the remainder of the year to play out similarly as we've seen really in the last 6 years in terms of WAKIX performance.

And that this first quarter is just down to the typical start of the year market access headwinds.

Jeffrey Dayno: Great. With regards to the second question on the timing of our lawsuit with Novitium with regard to the amorphous patent, I want to take the opportunity to welcome Peter and not to see you to the Harmony team, our Chief Operating Officer, who is working very closely with me on all the matters related to IPA and the litigation. Peter?

Peter Anastasiou: Yes. Hello, everyone. It's a pleasure to be on the call with you. I look forward to meeting those of you I don't know in the very near future. To answer your question in terms of timing, first, let me just say, we agree with you that we believe we will prevail in the ANDA case. And so this filing of this lawsuit in no way reflects our -- any other belief than that. It's an effort to make sure that we're asserting our full rights. So we believe that evidence was introduced that there is infringement on our polymorph patent, and we believe that our method of use patent is absolutely valid.

Having said that, during that trial, AET and Sandoz also introduced very clear evidence that they are violating the amorphous patent, the 920 amorphous patent, that we licensed from Novitium. So when you asked about timing, it was after those facts were entered into evidence in the trial that we, along with our partner, Novitium, decided that we were going to fully assert our rights to defend the IP for pitolisant. And as Jeff has said many times, this is a multilayered approach. And so that's why we filed suit in April.

And again, these are 2 different cases with 2 different time courses, and we believe that we will prevail in both cases, and that's why we have strong confidence in our exclusivity going into 2030, inclusive of the 6-month exclusivity.

Operator: Next question, we have the line of Pete Stavropoulos from Cantor Fitzgerald.

Pete Stavropoulos: Congrats to Glenn and Peter on joining the team. Can you touch on EPX-100 for Dravet? The clinical data disclosed at AAN. Can you comment on baseline seizure rates and baseline antiseizure med use? Sort of how do they compare to the real-world patients and patients enrolled in other Dravet clinical trials? And with interim OLE efficacy data and safety data in hand, what's your view on the emerging benefit risk profile? How competitive do you believe the profile is? And where do you see it fitting into the current landscape?

Jeffrey Dayno: Thanks for your question. Kumar?

Kumar Budur: Yes. EPX-100 clinical data that was presented at the recent AAN meeting, that's Encore data that we had already presented at the AAN meeting in December last year. Just to refresh, the data is from the open-label extension part of the Argus study, the Phase 3 pivotal registration study in Dravet syndrome. What the data showed is that in patients who had at least 6 months of exposure to EPX-100, we saw a median reduction of 50% in seizures as measured by CMS 28. And we also saw 50% of patients experiencing 50% reduction in seizures. Both of these measures are considered clinically meaningful.

Now the other side of the coin is safety and tolerability, which is extremely important in this patient population, given many of these patients are on 4 to 6 antiseizure medications and still have residual seizures. And that's exactly how EPX-100 was used in this study as adjunctive therapy. And on average, patients were on about 4 different antiseizure medications. It doesn't require any special monitoring, which is very important, [indiscernible], for example. The lab values were really fine. So there is no need to monitor liver function test as is often the case with some of the antiseizure medications.

And more importantly, the tolerability because a lot of these medicines do cause nausea, vomiting, abdominal graft, diarrhea in patients up to 20% to 30% in some instances. What we saw with EPX-100, the only AE of note was actually diarrhea in about 2% of the patients. So the combination of clinically meaningful efficacy, favorable safety and tolerability profile as an adjunctive therapy in a patient population known for refractory seizures positions EPX-100 very well from a clinician, from a prescribing algorithm perspective. I hope I answered all of your questions.

Operator: Next question comes from the line of Patrick Tutcher from H.C. Wainwright.

Patrick Trucchio: I was hoping to get a little bit more detail on the BD strategy just in terms of the sort of the size and the disease areas that you would be most interested in, particularly as we think about CNS adjacencies beyond rare disease. And then separately, as we approach the BP-205 data in the middle of this year, I'm wondering if you can tell us what specific PK parameters and tolerability bar would validate the best-in-class claim.

Jeffrey Dayno: Patrick, thank you for your questions. Let me first address the question on our BD strategy and then turn it over to Kumar for your second question. So I first want to with regard to the renewed emphasis on business development, just take a minute on the strategy and our thinking there. So it's important to note, I think we have a renewed organizational commitment to transact with regards to business development. And this is from the management team to our Board and then obviously, the 2 members of the management team, Peter and Glenn, that bring very relevant experience doing deals and really strengthens the overall ability for us to move this forward and execute on this key priority.

Obviously, it's one of BD and the renewed emphasis is one of our 4 key pillars of value creation as we see the urgency and conviction to deploy our capital, put it to work to generate sustained value. So I want to ask Glenn to comment on thoughts on capacity, where we are there. And Peter, with regard to what's the strategic focus of what we are considering. Glenn?

Glenn Reicin: Yes. I mean capacity, we can -- we actually have quite a bit given our cash balance and our ability to borrow as well. And obviously, not preferred, but we can always use stock. So we have quite a bit of capacity and size deals, I think, are not a limiting factor.

Jeffrey Dayno: And with regard to strategic focus, it's really -- it's an evolution. It's an evolution of our strategy. It's a refinement. It's not a major pivot sort of in the road. And Peter, if you want to sort of highlight what we're thinking in that area.

Peter Anastasiou: Yes, absolutely. So there's a number of criteria that we're looking at. But I also want to say we're also willing to be opportunistic and keep an open mind to other opportunities as well. But in terms of the areas of focus, it is, of course, revenue potential, especially near-term revenue potential, especially in the '20 to '32 time frame. So that implies something that's in the Phase 3 or registrational phase or even on market, quite frankly. And then we're big believers that the types of things we should look at are things that are related to our core competencies, and we feel we have many core competencies.

But clearly, in the sleep/wake space, we've demonstrated a lot of success and we'll continue to demonstrate success in that space. Also because narcolepsy is in the rare orphan CNS space, we believe that capability can be applied there. We've also been building an epilepsy presence. And so that's an area of focus for us in a variety of different epilepsies. And then certainly other things that are adjacencies to all of those within the CNS space that we believe we could leverage our core competencies. And we are agnostic to deal type. We're looking at M&A. We're looking at licensing. We're looking at collaborations.

So we have kind of a broad aperture in the way we're thinking about accessing and sourcing some of these deals. And then I will also say, while our focus is very heavily on near-term revenue, we are also, in parallel, looking at bolt-on opportunities that fit nicely within our current construct. And so that hopefully gives you some color. And I'll also just end by saying we have a really strong BD team and really strong capabilities that we plan to mobilize and put into action with this renewed focus and commitment from not just this management team, but also the Board.

Jeffrey Dayno: Yes. Thanks, Peter. Thanks, Glenn. In addition to the strong business development team, we also have a very strong commercial engine. So any of these potential bolt-ons or on-market opportunities, we've always wanted to utilize that commercial engine and put it to work in terms of generating more revenue. Kumar to BP-205?

Kumar Budur: Yes. Patrick, great question on the broader CNS indications. Although central disorders of hypersomnolence are the logical natural choice for Orexin-2 receptor agonist. We are also excited at the potential for Orexin-2 receptor agonist in broader CNS indications. And to a large extent, [indiscernible] acquisition of centers have validated the potential for this class of compounds. We are looking at mood disorders, cognition, fatigue, and ADHD in a preclinical setting. In terms of your question regarding what kind of clinical PK data we would like to see, as we have mentioned earlier, BP-205 has demonstrated a potential best-in-class in the preclinical setting, high potency, great selectivity, favorable preclinical safety pharmacology and toxicology and potential for once-a-day dosing.

So in the clinical PK setting, we'll be looking at Tmax, Cmax, AUC, half-life, safety and tolerability to the extent we can infer based on a single dose study.

Operator: Next question comes from the line of Corinne Johnson of Goldman Sachs.

Corinne Jenkins: Maybe as you look at business development, you've been highlighting the importance of relatively near-term revenues. But I'm curious where the path to earnings accretion factors in for deals that you might consider? And then separately, could you just expand a little bit on the newest pitolisant formulation and the types of delivery models that, that formulation could enable?

Jeffrey Dayno: Glenn?

Glenn Reicin: Yes. In terms of earnings accretion, I think what you should -- it's hard to answer that one. What I will represent is, obviously, you have to be looking at returns on invested capital that are quite substantial, which would obviously have low to mid-teens. So that would be the main criteria over time. So we wouldn't be just doing a deal for the purpose of growth. We obviously have to have financial returns. I hope that answers your question.

Jeffrey Dayno: And Corinne, with regards to the new amorphous form of pitolisant and so that was part of the value of the Novitium license. And we saw really an opportunity. We always talked about adjacencies beyond orphan rare with obviously this very strong product, differentiated molecule, WAKIX being very successful in the market in narcolepsy and opportunities to go broad. So with that, we saw new form and potential new formulations and modes of delivery to develop with the Novitium license, the amorphous form. And Kumar, additional thoughts on directionally where we're headed there.

Kumar Budur: Yes. Thanks, Jeff. Corinne, to answer your question, by the way, the physiochemical properties of our amorphous formulation could potentially lend itself to a different mode of oral delivery, resulting in a PK profile that may be more favorable to some of the indications that we plan to target within the broader CNS space. That's all I can say at this stage of development. Right now, we are optimizing the formulation towards a Phase 1 PK study.

Operator: Next question comes from the line of David Hoang from Deutsche Bank.

Samuel Beck: This is Sam on for David. Just a quick one on the OpEx front. Can you help us to think about the trajectory for OpEx for the rest of the year given the slight uptick in cost of products sold from the individual license and the step-up in R&D from the amorphous formulation as well?

Glenn Reicin: Yes. So in terms of gross margins, obviously, they're going to be impacted by the Novitium contract. And then we obviously also have step-up royalties related to Bioprojet. But nothing abnormal beyond that year-over-year gross to net anticipate anything changing there. With respect to R&D, we would, in fact, expect a ramp-up in R&D expense as we move forward on these trials and accelerate these trials. And then with respect to the rest of the P&L, I would not see substantial increase in run rates in terms of -- I hope that helps.

Operator: We have the next question comes from the line of So Youn Shim of UBS.

So Youn Shim: In 1Q, it seems like you saw more friction than usual in terms of access, which may have led to the softness in the patient numbers. Curious if you saw any changes in the discontinuation rate. And as a follow-up on the Orexin agonist program. So now there are several Orexin agonists that are either approaching approval or in late-stage trials in narcolepsy and IH. So for BP-205, although it has the most potency among the competitor Orexin compounds based on preclinical data, it seems like the flea space may be very crowded with a number of Orexin agonists by the time 205 launches.

So just curious here, instead of going head-to-head in those core indications, could it make more sense to just prioritize other indication first where you might have a clear differentiation or even a first-in-class opportunity?

Jeffrey Dayno: Yes. Thanks for your question. With regards to the Q1 dynamics, I'll let Adam share thoughts on that as well as in terms of the commercial perspective on the continued opportunity with BP-205.

Adam Zaeske: Okay. Sure. Thanks, Jeff. Yes. So in 1Q, we typically see these market access-related headwinds. But to be clear, our coverage has not changed. We enjoy very broad coverage over 80% of lives in the U.S. And actually, that coverage was strengthened in 2025 with some improvements in some of our key payer accounts. So that remains the same. What happens is as consumers enter the new calendar year, sometimes they've got to go through co-pay resets. They've got to reestablish coverage or prior authorizations. Sometimes they switch plans. And that dynamic can simply just delay some of the patient starts, which is going to affect your average patients for the quarter. That's something that we see every year.

It's really no surprise. And the -- I think you asked specifically around D.C. rate and persistency. Persistency on WAKIX has remained very, very steady for many years, and we have not seen any meaningful change in that dynamic for a very long time. So it's really just these delays in patient starts as we start the new calendar year.

And then from an Orexin standpoint, kind of a commercial perspective around BP-205, I think we're excited that there still is an opportunity to deliver an Orexin that can deliver on the promise of Orexin level efficacy, but potentially with a better safety and tolerability profile and once-a-day dosing across all 3 domains, NT1, NT2 and IH with a single compound. That's really where we're excited and where we're focused. Thanks, Jeff.

Jeffrey Dayno: Sure. Thanks, Adam. And a final comment on BP-205 and the question. It's not sort of either/or. We see this opportunity in terms of the development program both looking at central disorders of hypersomnolence with BP-205 as well as the broader program that we are working on in addition to some of the broader CNS indications, as Kumar alluded to earlier. So not either/or, we will look to invest in this significant opportunity across the Orexin-2 agonist program.

Operator: We have the next question comes from the line of Jason Gerberry of Bank of America.

Jason Gerberry: Just a question and a follow-up just on the WAKIX IP situation. So if AET does get a non-infringement, the decision in the second half, just wondering about thinking about scenarios and what actually are barriers to an at-risk launch in second half '27? And then as a follow-up, just can you talk a little bit about the diligence that you did just to ensure that the -- I guess, this arrangement survives any FTC scrutiny. If you can just describe a little bit of the history of the license with Novitium versus the settlement agreement that you reached with them during ANDA litigation.

Peter Anastasiou: Yes. So first, on your question with regard to AET and various scenarios. Just as a reminder, the 30-month stay ends in February. And so we would expect February of '27, to be clear. So we would expect that the judge will issue her ruling on the ANDA case somewhere around there. And then, of course, there's appeal opportunities that both parties have for adverse outcomes for either party. So fully expect that there would be appeals. Certainly, we would appeal if there was an issue there. But then also, please keep in mind that this second trial just started, and it could take 24 to 30 months in and of itself plus an appeals process.

And so that kind of gives you a sense of the timelines. And in our view, in almost every scenario, we end up with exclusivity that gets us into 2030, again, inclusive of the 6-month exclusivity. And so hopefully, that gives you some sense of the timelines and the impact. And again, we're going to vigorously defend and use every right that we have to defend our multilayer IP strategy, and that's what we're putting in place.

Jeffrey Dayno: Thanks, Peter. And I would just add that in terms of AET and an at-risk launch, we really can't comment on other parties and what their decisions would be in that regard. And with regards to your question about the antitrust aspect, we obviously always consider that very closely. But most important to remember, when we saw the Novitium license and the amorphous form, the original opportunity we saw with a bona fide development program, which we've already initiated. And that was the opportunity when we did that license in January before what we learned at trial in February. And we continue to pursue that business and development opportunity with the Novitium Amorphous license.

Peter Anastasiou: If I may just add to Jeff's point, just on the at-risk launch. Of course, there are legal remedies that we have access to that we would fully implement in that see how vigorously we are defending our intellectual property, and we would use every tool available.

Operator: That will conclude our question-and-answer session. I will now turn the call back over to Dr. Jeff Dayno, CEO, for closing remarks.

Jeffrey Dayno: Thank you, operator, and thanks, everyone, for your interest in Harmony Biosciences, and have a great rest of your day.

Operator: Thank you. Ladies and gentlemen, that concludes today's call. Thank you all for joining. You may now disconnect.

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