Q1 2025 Harmony Biosciences Holdings Inc Earnings Call
The End
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Angela: Good morning. My name is Angela and I will be your conference operator today. At this time I would like to welcome everyone to Harmony Bioscience's first quarter 2025 Financial Results Conference call.
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Speaker Change: I will now turn the call over to Brennan Doyle, Head of Investor Relations.
Please go ahead.
Speaker Change: Thank you, Operator. Good morning, everyone, and thank you for joining us today as we review Harmony Biosciences 1st quarter 2025 financial results and provide a business update.
Speaker Change: Before we start, I encourage everyone to go to the Investor section of our website to find the materials that accompany our discussion today, including a reconciliation of our gap to non-GAAP financial measures.
Speaker Change: At this stage of our life cycle, we believe non-GAAP financial results better represent the underlying business performance.
Speaker Change: Our speakers today on the call are Dr. Jeffrey Dayno, President and CEO , Adam Zeskey, Chief Commercial Officer, Dr. Kumar Budur, Chief Medical and Scientific Officer, and Sandip Kapadia, Chief Financial Officer.
Speaker Change: As a reminder, we will be making forward-looking statements today, which are based on our current expectations and beliefs.
Speaker Change: These statements are subject to certain risks and uncertainties, our actual results may differ materially and we undertake no obligation to update these statements even if circumstances change. We encourage you to consult the risk factors referenced in our SEC filings for additional details. [inaudible]
Speaker Change: I would now like to turn the call over to our CEO , Dr. Jeffrey Dayno. Jeff?
Speaker Change: Thank you, Brennan. Good morning, everyone, and thanks for joining our call today. Before we get started, I am very excited to introduce and welcome Adam Zewski, our new Chief Commercial Officer to the Harmony team.
Speaker Change: Adam will be sharing a bit about his background in a moment, but let me tell you, in just six weeks at Harmony he has already made a big impact.
Speaker Change: He has been fully embraced by the team, shown himself to be a quick study and strategic thinker, and has brought fresh energy and focus as we continue to grow the patolescent franchise from the strong foundation we built over the past five years.
Speaker Change: Building off of their four years of profitability, Q1 with another strong quarter for Harmony.
Speaker Change: We continue to leverage our commercial success to drive both top and bottom line growth and our well-positioned for significant momentum throughout 2025, fueled by key catalysts from our robust, late stage pipeline.
Speaker Change: Our next major clinical milestone is on track for the third quarter. The top line data read out from the Phase 3 Registrational Trial of ZYN-002, in patients with fragile X syndrome, the ReConnect Study.
Speaker Change: A positive outcome could position us to deliver the first ever FDA approved treatment for fragile X syndrome, a significant milestone for patients and their families.
Speaker Change: I am very proud of the unique profile we have built at Harmony, a profitable self-funding biotech company with a robust pipeline that has the potential to help hundreds of thousands of patients.
Living with rare neurological diseases.
while creating durable long-term value for our shareholders.
First, a few comments in our core commercial business.
Speaker Change: Wakes and narcolepsy continues to deliver strong, consistent growth. Net product revenue for the quarter was $184.7 million, a 20% increase year over year, now in its six year on the market.
Speaker Change: This growth reflects the highly differentiated product profile of WAKIX, as the first and only FDA approved non-schedule treatment for patients living with narcolepsy.
Speaker Change: Our unique commercial model and strong execution continue to deliver results, and we remain confident that WAKIX is on track to reach a billion dollar plus opportunity in narcolepsy alone, well ahead of its loss of exclusivity in 2030.
Speaker Change: Turning to our pipeline, where Harmony's story gets even more exciting.
Speaker Change: Over the last two years, we have been strategic in leveraging our strong balance sheet to build one of the most robust, late-stage pipelines in the industry for people living with rare neurological diseases.
Speaker Change: Today, we have three distinct franchises in sleep wake, nor behavioral disorders, and rare pediatric
Speaker Change: Each with late-stage programs, each with plans to pursue multiple indications, and each one of these franchises with peak sales potential of $1 to $2 billion.
Speaker Change: Our pipeline now includes eight assets across 13 development programs and we expect to have up to six programs in phase three trials by the end of this year.
Speaker Change: Some highlights from our pipeline. First, ZYN002 in patients living with fragile X syndrome. The Phase 3 Reconnect study is on track for top-line data in the third quarter.
Speaker Change: This trial is designed to replicate the positive findings from the pre-specified analysis on the primary outcome from the Phase 2-3 Connect Study in the subgroup of patients with complete methylation of the FMR1 gene.
Speaker Change: Next, the Tulsen HD. We are on track to initiate Phase III Registration trials in narcolepsy and idiopathic hypersomnia in the fourth quarter.
Speaker Change: Patolousin HD is our next-generation high-dose patolousin formulation with an enhanced PK profile.
Speaker Change: Designed to deliver greater efficacy with no change in the well-established Safety Tolerability Profile of the Tollison.
Speaker Change: with utility patents filed out to 2044 and a target padufidate in 2028. Patelison HD provides us with a unique opportunity to extend and expand the Patelison franchise and pursue additional indications for which there are no approved treatments.
Speaker Change: Kumar will provide more colour on our pipeline later in the call.
Speaker Change: On the business development front, with over $600 million in cash and cash equivalents on the balance sheet, we are actively looking to further strengthen the pipeline.
Speaker Change: Our experience business development team has a track record of disciplined smart execution, and we are focused on opportunities that can expand our leadership in sleep wake.
Speaker Change: Billed out our neurobehavioral and epilepsy franchises, and or bring in adjacent rare CNF assets.
Speaker Change: Our financial strength gives us flexibility, and we are well-positioned to deploy our resources to further grow our pipeline and generate significant value for both patients and our shareholders.
Against the current market backdrop.
Speaker Change: Harmony's profile is not only unique because of its profitability and strong cash generation, but Harmony is also well-positioned to navigate the shifting geopolitical landscape, including the potential impact from proposed new tariffs.
Speaker Change: On the supply chain side, while our current manufacturing site for WAKIX is in France,
Speaker Change: We have been working on a secondary manufacturing site for Waykicks in the US and have made good progress toward bringing this site online.
Speaker Change: Looking ahead, Patolison HD is the future of the Patolison franchise, with utility patents for HD extending out to 2044.
Speaker Change: The Manufacturing of Patolson HD has been based in the US from the start of the program.
Speaker Change: Importantly, our exclusive licensing rights for WAKIX and the intellectual property associated with WAKIX along with all the IP related to our pipeline products are domiciled here in the United States.
Speaker Change: I want to emphasize how unique Harmony structure is compared to many others in the biotech industry because of its US-based operational foundation.
Speaker Change: While the outcome of many of these policy proposals is uncertain, we have taken proactive steps to ensure operational independence and minimize exposure to these potential risks.
In closing, I want to leave you with this.
Speaker Change: Harmony is a profitable self-funding biotech company with a strong and growing commercial business.
Speaker Change: Next Generation Formulations of Petolicent on track for PDUFA dates in 2026 and 2028 with utility patents filed out to 2044.
Speaker Change: and a catalyst-rich, late-stage pipeline that is poised to deliver one or more new product or indication launches each year over the next several years, with peak sales potential over $3 billion.
Speaker Change: If you look across our industry today, Harmony's profile stands out and offers a unique and compelling investment opportunity.
Speaker Change: Thank you again for joining us today. I'll now turn the call over to Adam Zewski, our Chief Commercial Officer, to share more about his background and provide an update on our commercial business. Welcome Adam, we are thrilled to have you on the team.
Adam Zesky: Thanks, Jeff. I really appreciate the warm welcome from the entire Harmony bioscience team, and I'm thrilled to be joining Harmony at such an exciting time. I've been very impressed with the clear focus on patients, and what the team has achieved over the past several years with waitings.
I'm excited to continue that success.
Adam Zesky: for many years to come and to expand on that with the next generation, the tallest informulations, as well as the late stage products in the pipeline.
Adam Zesky: I hope to leverage my experience having led marketing sales and market access teams for 10 years in the US.
Adam Zesky: as well as leading large and small country organizations in Europe for 10 years.
Adam Zesky: My most recent role was leading a region of 25 countries, nearly 900 employees and over 2 billion in revenue with double digit growth for the past 3 consecutive years as the largest contributor to revenue and growth for Europe .
Adam Zesky: Prior to that, I led the franchise teams for Europe across rare disease, neuroscience, immunology, hematology, oncology, gastroenterology, and plasma derived therapy.
Adam Zesky: I've been a part of building several multi-billion dollar brands and I've worked on 10 red disease launches in the past 10 years.
Adam Zesky: Turning to Harmony's performance, Q1 2025 demonstrated the ongoing strength of our core business and our ability to deliver durable growth.
Adam Zesky: Notably, we achieved 184.7 million in that sale, and the fundamentals of the business remain strong and stable.
Adam Zesky: In the first quarter, we saw the familiar seasonal rhythm to our performance consistent with prior years. This include the traditional Q1 challenges faced across the industry related to payer dynamics.
Adam Zesky: followed by positive momentum and increased prescription demand coming out of Q1.
Adam Zesky: The continued strong 20% year-over-year first-quarter growth in the Wakix Net Revenant.
Now in its sixth year post-launch.
Adam Zesky: Underscores the sustained high demand for wakix within the narcolepsy market driven by the broad clinical utility of wakix and the fact that it is the first and only nonscheduled treatment for patients with narcolepsy.
Adam Zesky: This differentiated profile allows us to tap into the full potential of the approximately 80,000 diagnosed narcolepsy patients.
Adam Zesky: Even with the introduction of new and generic oxidative alternatives, we continue to see consistent utilization of wakix among the approximately 4,000 HCTs enrolled in oxidative rems.
Adam Zesky: Despite our high penetration within this group, Wake Ex prescriptions for additional narcolepsy patients within this segment continues to grow each quarter.
Adam Zesky: Repleased with the robust growth in WAKIX prescribers beyond those enrolled in Oxidate rents, demonstrating WAKIX's ongoing success in capturing a broader segment of branded prescribers.
Adam Zesky: Additionally, we have now reached over 50% penetration within this approximately 5,000 HCP segment as of the end of Q1.
Adam Zesky: As we look ahead to full year 2025, we're confident in the strength of the underlying business fundamental and optimistic that they will fuel continued growth of Wagex.
Adam Zesky: Based on this, we confirm our net revenue guidance in the range of 820 million to 860 million and we remain on track to achieve $1 billion plus in narcolepsy alone.
Adam Zesky: Leveraging our robust, scalable, commercial infrastructure, we are well positioned to unlock significant longer-term growth and value with our next-generation populism programs.
Adam Zesky: Our development of both patollicent gastroresistant, the GR, and high dose HD formulations is centered on unmet patient needs.
Adam Zesky: Aining to deliver meaningful improvements in care and extend the growth and revenue of the Patollis and Franchise into the mid-2040s.
Adam Zesky: Kumar will elaborate on the specifics of the HD and GR development programs.
Adam Zesky: Initial Market Research among HCTs and payers, regarding the HD target product profile, has demonstrated strong interest from both positions and payers and clear unmet need for patients.
Adam Zesky: In summary, we saw continued strong growth of Wacix and Q1. Our patient-centric drug development approach to the Pisholescent Lifecycle Management Program combined with our unique commercial model will ensure durable growth through the mid-2040s.
Speaker Change: and in closing, my experiences in both rare disease and neurology were tremendously rewarding parts of my career. I'm excited to return to these therapeutic areas and contribute to Harmony's potential to help many more patients living with unmet medical needs.
Speaker Change: I would now like to turn the call over to our Chief Medical and Scientific Officer, Kumar Budur, to discuss the advancements in our clinical development programs.
Two more?
Speaker Change: Thank you, Adam. Good morning, everyone, and thank you for joining us today.
Speaker Change: In R&D, we continue to make progress in advancing our pipeline across 13 development programs, 8 different assets and 3 distinct franchises focused on rare neurological diseases with high
Speaker Change: We have four phase three registrational trials ongoing in four distinct indications and we will have up to six phase three
Speaker Change: This makes our portfolio one of the most robust latest pipelines in the industry with the potential to deliver one or more new products or indication launches every year in the coming years.
Speaker Change: Our Food Clinical Development Pipeline is shown on slide number 5 and the Clinical Development Highlights or on slide 6 through slide 11.
Let's begin with our sleep wig franchise. Good night.
Speaker Change: Our next-gen pitola scent programs, which are patient-centric development programs, are aimed to build upon the product profile of pitola scent.
Speaker Change: We are on track to initiate a Phase 3 Registration Tile of Pidolus and HD in North Colored Sea in Q4 2025 with a target to produce a date in 2028.
Speaker Change: Fierdo's formulation holds the promise of greater efficacy in addressing excessive data and sleepiness.
Speaker Change: The most important unmechanate in patients with narcolepsy, and also target fatigue, a symptom experienced by about 60-70% of patients with narcolepsy, for which there are no currently
Speaker Change: We are also on track to initiate our phase 3 registration trial in Idiabatic Hypersomnia in Q4 2025.
Speaker Change: Pitolocent HD is expected to deliver efficacy in excessive data and sleepiness and also address critical symptoms such as sleep inertia, another core symptom in patients with idiopathic hypersomnia for which there are no approved treatments.
Speaker Change: This double blind, randomized, placebo controlled parallel arm study is designed with FDA input and we anticipate a Poodoo for date in 2028.
Speaker Change: This formulation is designed to address the GI comorbidity, prevalent in almost 80% of patients with narcolepsy, and also designed to give patients an ability to start at the therapeutic dose range with no titration.
Speaker Change: We initiated the Kyoto BE study in Q1 2025 and the top-line data are anticipated in Q3 2025 with the projected producer date in 2026.
Speaker Change: Utility Patents have been submitted for both Pitola Cent GR and Pitola Cent HD with the potential for patent protection until 2044.
Speaker Change: Next in our sleepfake franchise is our Oryxen Deceptor Agonist Program, BP1.5205, Suppotential best in class candidate, currently in pre-clinical development.
Speaker Change: In vitro form of quality data, demonstrate greater potency compared to all other publicly disclosed oraxing to our kinesh.
Speaker Change: The combination of high potency, excellent selectivity, potential for once daily dosing and robust preclinical safety data, underscores its best in class potential.
Speaker Change: We will be presenting a comprehensive pre-clinical safety and efficacy data at the upcoming annual
Speaker Change: and the data will demonstrate efficacy in sleepiness consistent with its high potency.
Speaker Change: We remain on track to file an IMPD by mid-2025 with just in few months studies in the second half of this year and anticipate the shares clinical data in 2026.
Speaker Change: Moving on, I'm very excited about our neurobehavioral franchise which is the next major catalyst in our portfolio.
Speaker Change: The toppling data from the C1-002 phase III resistance tile, the reconnect study in Frasal X syndrome, is in the third quarter of this year.
Speaker Change: Frazel X syndrome, arrays genetic disorders caused by mutation of the SMR-1 gene on the X chromosome results in decreased or no SMR protein production, especially in patients with complete methylation that results in silencing of the gene.
Speaker Change: Lack of FMR protein causes endocannabinoid dysregulation leading to significant neurobehold symptoms, intellectual impairment, developmental delays, and other symptoms.
Speaker Change: In fact, Franois Syndrome is the most common known inherited cause of intellectual impairment and autism spectrum disorders with an estimated prevalence of approximately 80,000 patients each in the US and EU.
V1-002 represents a novel therapeutic approach.
Speaker Change: It's a 100% synthetic canopy, Dior, Deoid of THC in a patent protected, permission enhanced constant mulchow, delivering the drug directly into the systemic circulation.
Speaker Change: ZY-002 is designed to modulate the end-of-canabinoid system by interacting with CD-1 receptors, aiming to restore end-of-canabinoid homeostasis and thereby alleviating the neurobehavioral symptoms.
Notably,
Speaker Change: Pastoral Admi Station of Z1-002 offers a significant advantage in tolerability and safety compared to oral canopy Dior, which is associated with substantial gastrointestinal disturbances such as nausea, vomiting, abdominal cramps, and diarrhea.
Speaker Change: By avoiding the first pass hepatic metabolism, 0.002 is not associated with liver function of normalities that are often observed with oral cannabidiol.
Speaker Change: The ongoing Phase 3 Registration trial, the Reconnect Study, builds upon the data and insights from the large Phase 2-3 Connect Study.
Speaker Change: The Reconnect Study essentially seeks to replicate the clinically meaningful and statistically significant efficacy signals observed in patients with complete matallation in the Connect Study.
We have completed the recruitment and closed new patient screenings.
We are on track for top-line data in Q3 2025.
Speaker Change: If positive, the ReConnect study is expected to support regulatory approvals in both US and EU and Harmony holds global rights for 3.002.
Speaker Change: Z1002's Mechanism of Action, the robust clinical data in patients with complete methylation from the Connect Study, as well as the enhancements in the design of the ReConnect Study based on the learning from the Connect Study, we have a strong conviction in the program success.
Speaker Change: Charles. If approved, ZY-002 will be the first and only treatment approved for any symptoms in patients with phrasalic syndrome.
Speaker Change: For the most, we remain on schedule to initiate a Phase 3 Resistration of Charles in 22 Cube Deletion Syndrome in 2025.
Speaker Change: This rate dissolves with significant neurobehavioral symptoms and no approved therapies affects approximately 80,000 individuals each in the US and Europe .
Speaker Change: Finally, a few words on our epilipsy franchise, where we have the most advanced five-day-two serotonin development programs, developmental and epileptic ends of Lopathy's.
Speaker Change: Our other investigational product in developmental and epileptic and subflopathy, EPX-200, a liquid formulation of locustrine is in the PIND phase.
Speaker Change: In conclusion, we are making strong progress across our latest rare neuro portfolio, with the potential to launch one or more new products or indications each year in the coming years.
Speaker Change: More importantly, this progress holds the promise of providing meaningful new treatment options to hundreds of thousands of individuals affected by rare neurological disorders.
Speaker Change: For many of these patients, there are currently no approved treatments or the existing therapies offer limited efficacy and considerable safety and tolerability issues.
Speaker Change: As always, on behalf of Harmony, I would like to thank all the patients and their families who are participating in our clinical trials.
Speaker Change: As well as our clinical investigators and site personnel for their efforts and commitment in helping us to advance our development programs.
Speaker Change: I'll now turn the call over to our CFO , Sandip Kapadia, for an update on our financial performance, Sandip.
Sandip Kapadia: Thank you, Kumar, and good morning, everyone. This morning we issued our first quarter of 2025 earnings release and filed our 10Q, where you'll find the details of our financial and operating results.
Sandip Kapadia: Our financial performance is also shown on slide 12-14.
Sandip Kapadia: Ferroff to a great start to the year in 2025.
Sandip Kapadia: We reported another quarter for strong growth in revenues of 20% and net income growth of 19%.
Sandip Kapadia: along with closing the quarter with over 600 million of cash, cash, co-reblends and investments on the balance sheet.
Sandip Kapadia: We continue to have a very unique profile in biotech with growing top line profitability and generating positive cash flow.
Sandip Kapadia: As a result, we are well positioned to advance our growth strategy and look for opportunities to increase the drive incremental value for shareholders.
Sandip Kapadia: For the first quarter of 2025, we've reported net revenues of 184.7 million, compared to 154.6 million in the prior year quarter, representing a growth of 20%.
Sandip Kapadia: Performance in the quarter reflects the strong underlying demand for weight-ex, coupled with the typical seasonal dynamics in Q1 that the industry has a whole experiences, including higher growth than method actions, while not seeing its significant change in trade
Sandip Kapadia: Wrote in our expenses were driven by our expanding late-stage pipeline, along with investments for the commercialization of wakix and narcolepsy.
We also reported solid net income and margin.
Sandip Kapadia: Non-gap-adjusted net income for the first quarter of 2025 was $60.4 million or $1.03 per diluted chair compared to $50.7 million or $80.10 per diluted chair in the prior year quarter.
Sandip Kapadia: We believe non-gap-adjusted net income better reflect the underlying business performance.
Sandip Kapadia: Pleased to you, unfortunately, for a reconciliation of Gap to non-GAAP results.
Sandip Kapadia: We also ended the first quarter with 610.2 million in cash, cash quibblance and investments on the balance sheet.
Sandip Kapadia: And looking ahead to the balance of 2025, we are reiterating our net revenue guidance for 2025 of 820 to 860 million, highlighting our progress towards a 1 billion plus opportunity in narcolepsy alone.
Sandip Kapadia: We expect continued strong quarter over quarter growth while noting the potential of trade inventory drawdown of a few days in Q2 as we head into the summer month.
Sandip Kapadia: With respect to expenses, we expect increased R&D investments as we advance our late stage pipeline with multiple programs in Phase 3 Registrational Products.
Sandip Kapadia: That's previously noted, we expect to potentially incur $29 million in R&D-related milestone payments in 2025.
Sandip Kapadia: including milestones for the completion of the Phase 3 trial for ZYN-002 and fragile X syndrome, along with a milestone for positive top-line data from this trial.
Sandip Kapadia: In addition, there is a milestone related to the initiation of the Phase One trial in our REX-2 Agonist Program which we anticipate in the second half of this year.
Sandip Kapadia: In summary, I'm pleased with the great start we're off to in 2025.
Sandip Kapadia: We've once again delivered a quarter with double digit top line growth and healthy operating margins.
Sandip Kapadia: This, along with our US operational footprint, positions as well as we move through the year with the potential for significant value creation through our catalyst-rich pipeline.
Jeff: And with that, I'd like to turn the call back over to Jeff for his closing remarks. Jeff?
Thank you, Sandip.
Jeff: To wrap up, Harmony Biosciences offers a very compelling profile. A strong, commercial, foundational business generating significant cash and profitability.
Jeff: A robust late-stage pipeline with meaningful near-term catalysts poised to deliver one or more new product or indication launches each year over the next several years and peak sales potential over $3 billion.
Jeff: Over $600 million on the balance sheet, funding the entire enterprise, giving us the capacity to build out our pipeline further and create even greater value potential.
Jeff: I am proud of the entire team and what we have accomplished together, transforming Harmony into a high growth biotech company, delivering on our promise to patients and creating durable long-term value for our shareholders.
Jeff: Thank you, and I will now turn the call back over to the operator. Operator?
Jeff: Thank you. At this time, if you would like to ask a question, please press star one on your telephone keypad.
Speaker Change: If you wish to remove yourself from the queue, you may do so by pressing start two.
Jeff: We remind you to please pick up your handset and please limit yourself to one question and one follow-up question.
Thank you. Bye.
Speaker Change: We'll take our first question from Craig Suvannavejh with Mizzouho Securities.
Speaker Change: Hey, thank you very much for taking my question. Congrats on the quarter. Two questions, please. First, just on the pipeline and for ZYN002. Could you please remind us
Speaker Change: What the Powering Assumptions are for the study and also what perhaps risk mitigation strategies and what strategies for optimizing clinical trial success you put in place.
Sandip Kapadia: That's my first question, and then my second question is just for Adam, congrats on the new role as Chief Commercial Officer, but could you please share with us, maybe what you've seen thus far with the commercial team and strategies that are in place supporting WAKIX.
Speaker Change: and are there any changes or tweaks that you think, given your experience, can be put in place? Thanks so much.
Speaker Change: Good morning, Greg. Thank you for your questions. First to Kumar on on ZYN-002, very excited for that opportunity. And Kumar can sort of share with you the powering and how that program is designed and our conviction in the top line data that's on track for Q3, Kumar.
Speaker Change: Hey, good morning, Graig. Thanks for the question. Regarding the powering of the study, the study is powered more than 90% for the primary end point.
Speaker Change: which is changed in the social avoidance severity from baseline to the end of the study, as measured by aberrant behavior checklist, community addition specific for far the less drawn.
Speaker Change: and the study powering and the sample calculation and all the statistical analysis.
Speaker Change: was planned based on the learning from the connect study, whereas we have disclosed in the past, we not just saw a clinically meaningful but also a normally statistically significant difference.
Speaker Change: in patients with complete methylation on the same primary endpoint. Regarding the question on the checks and balances between the clinical trial, Greg Assy, you know, many efforts have done so many neuropsychiatric clinical trials and these clinical trials are very unique. It's very important to make sure.
Speaker Change: that the right patients get into the study. And we have ensured it in a way with Frasal X syndrome, it makes it somewhat easier because all of these patients need to have full methylation, sorry full mutation, that is confirmed while looking at the CGG repeats.
and also full complete ventilation via PCR test.
frequent users.
Speaker Change: As we mentioned in the past, this study is designed based on the learnings from the Connect Study. Not only that, we made several enhancements in the ReConnect Study to increase the probability of success.
Speaker Change: ORA, very excited, high level of conviction and confidence in potentially bringing the first treatment for any symptoms in patients with phrasalic syndrome.
Speaker Change: Thank you, Kumar. And over to Adam for his initial impressions and let me reiterate our welcome to Adam to the Harmony team, Adam.
Adam Zesky: Thank you, Jeff. And thank you, Greg, for the question. Look, it's fantastic to be part of the team here at Harmony now. I'm super excited and I've been thrilled with what I've seen so far. I was originally attracted to Harmony by the sciences for a couple of reasons. I saw an organization that had a very clear purpose.
Adam Zesky: and a shared purpose around really trying to benefit patients and meet on that knee for those patients in neurology. I saw an extremely strong team, a lot of talented individuals.
Adam Zesky: and I think as you've seen here today, the outstanding performance and it just continues. Quarter over quarter, year over year, in addition to having a really exciting pipeline. Pipeline that is developed with a very clear patient orientation. Thank you.
Adam Zesky: and I think smartly designed to really meet the idea of remaining on that needs of patients in each of these areas.
Speaker Change: So I'm just about one month in. I've had a wonderful opportunity to connect with the team and really I'm focused on learning and listening at this stage. My platform is pretty simple.
Speaker Change: I'm focused on how do we continue the strong performance that the organization has delivered for the past five plus years? Consistently?
and how do we prepare for the future? [inaudible]
I think the team has a lot of great ideas!
Speaker Change: and I'm really enjoying the discussion and we're going to be evaluating tweaks as you say, tweaks and opportunities moving forward and prioritize those and I'm confident we will continue the strong performance with WAKIX and we will be prepared for the future with the exciting fun that exists here.
Speaker Change: Yeah, big shoes to fill.
We'll go next to David Amsellem with Piper Sandler. We'll go next to David Amsellem.
David Amsalem: Thanks. So I wanted to ask a high level question on the Aurexan. So you have...
David Amsalem: Three Aurexan Agnes in mid to late-stage development, and it's a space that...
Edmittedly getting crowded. So I guess the question is. [inaudible]
You know, where do you see? [inaudible]
David Amsalem: Your erection to receptor agonist fitting in within this emerging armamentarium in sleep wake, are you looking at the asset?
Insettings, Beyond. [inaudible]
David Amsalem: Slip Wake as other companies are starting to do. So that's number one. And then number two, can you just talk at a high level about? Yeah.
The Payer Landscape for Waykicks and what I guess I'm...
David Amsalem: You know, particularly interested in is as we get more and more generic competition for the oxidative learning. [inaudible]
Next year, how should we think about...
The Pear Landscape, and the Gross-to-Net for WAKIX. Thanks.
Speaker Change: David, thanks for your questions. So, erection to agonist, obviously a lot of attention, a lot of focus, a lot of interest in the next novel target for
Speaker Change: The programs are emerging, still early, still a lot to learn. I think alternative to Kumar in terms of our thoughts on where they could fit in.
C, Nork left, C, in terms of polyformacy market. David.
Speaker Change: Chronic neurologic disorder, multiple mechanisms, you know, always can be helpful. Kumar further thoughts on, you know, the other programs and where we fit in.
Speaker Change: We see all of these programs as still early and the data are emerging. We just need to see how the data will plan out from an efficacy and safety perspective and also from a colorability perspective with a possible exception of the KEDAS 861.
Speaker Change: The other two aspects are in Phase 1B, 2A studies, and we are monitoring these programs very carefully. What we are excited about is really BP1.15.205. As we have disclosed in the past,
Speaker Change: and continues to be the highest potent compound compared to any other products in the subterragnest.
for which the data has been disclosed publicly. Thank you.
And you'll see.
This High Potency,
Speaker Change: and this data will be presented at the upcoming annual sleep meeting in the month of June and will be providing additional color and contact around the pre-clinical safety and efficacy. In terms of your question, David, beyond central disorders of high performance, there is something we are also carefully monitoring and also doing some of our own experiments as well.
Speaker Change: Helpful in certain other symptom domains, for example like cognition and potentially more disorders. These are some of the things that we are working on and also following the field very closely, but it's too early to comment on that.
Speaker Change: George Adam. Thank you, Jeff, and thank you, David, for the question. You know, one of the things I've been really impressed with is how the strength and stability of the fundamentals underpinning the business.
Speaker Change: for WAKIX, and I would include payer coverage in that. I think we experience very broad coverage for WAKIX with over 80% of lives covered, US lives covered for WAKIX.
Speaker Change: And that has really remained consistent, I think, for the past several years, despite entries of additional brand and generic oxipates.
As Jeff mentioned, plans typically don't step.
Thank you.
Speaker Change: Stepped through an oxidative to secure access for for wakix and that is I think largely because it is a highly differentiated product as as the first and only non-schedule treatment option.
Speaker Change: for these patients. And that performance and coverage has really, really been stable and consistent, I would say, to the past several years. We saw no changes in, no significant changes in pair coverage in one queue.
Speaker Change: and that is also a reason why I think we remain confident in confirming our full-year guidance for net revenue of between 820 and 860 million for the year.
Thank you
Thanks.
Thank you, David.
We'll go next to Ami Fadia with Needham.
Amy Faddea: Hi, good morning. Thanks for taking my question and Adam, welcome to the team. And perhaps my post-question is just sort of around market dynamics. And how do you see that evolves with the erections entering the market? I understand that some of the assets are...
Amy Faddea: Silences to, however, in the data that they've generated so far, particularly to Kata, as well as Alkum East.
Speaker Change: With that, is there still likely to be polypharmacy in this market?
Speaker Change: and maybe you could sort of comment on that and comment on whether you think the dynamics could be different across NT-192 and NIH. And then just separately regarding EPX 100.
Oh.
Speaker Change: What are your thoughts around potentially exploring it in a broader DE population as opposed to the SGS? Thank you.
Good morning, Ami. Thank you for your questions.
Speaker Change: Yeah, with regards to the erection to Agnes and...
Speaker Change: sort of the totality of the evidence, if you will, what the ultimate product profile you know will look like. And I'll turn it over to more, you know, any other thoughts, you know, with regard to that piece and then on EPX 100. Thank you, Jeff. Hey, good morning, Tommy. Thanks for the question. Ami, you did mention about the 30 minutes on MWT, that's true. That's the kind of efficacy that we have seen. Thank you.
Speaker Change: Davis, based on the preliminary data from a couple of other centers of data agonization, which is good, but still there are many unanswered questions in terms of the response rate.
Speaker Change: The Durability of Response, and the overall safety and tolerability profile, and these things need to be figured out.
Speaker Change: The way we see it is as we progress our own oraccentral sector agonist, which we believe has the properties to be potentially the best in class oraccentral sector agonist.
Speaker Change: As we make progress, we continue to believe that narcolepsy will be a polyformacy market. Patients with narcolepsy will require more than one medication.
Speaker Change: As you know, it's not just excessive day time sleepiness, they also have disrupted night time sleepiness, they have catapulted me, they have hypnosis, hypnosis, hypnosis, hallucinations, fatigue and a host of other symptoms, that's exactly one of the reasons.
Why we are talking in? [inaudible]
Speaker Change: George Fatigue, which is a symptom that is experienced by about 60% to 70% of patients with narcolepsy for which there are no uproot treatment and histamination mechanism of action specifically is suited to treat fatigue symptoms and we demonstrated that in our DM1 study.
Speaker Change: So, that's where we are with our Alexander Sittar Agonist and how we are thinking about the landscape. Two, your question about EPX-1-handed.
Speaker Change: Yes, definitely the option of going or pursuing a broader DEE indication is available. Right now we are focused on our two phase three studies.
Argus Study, Interface Syndrome,
Speaker Change: and Lighthouse Study in Lanoast, Christophe Sintra, both of these studies are up and running, actively recruiting patients, and we are evaluating, potentially adding other DE indications, but we just need to look at the benefit risk balance.
Speaker Change: By making some of these studies more heterogeneous by adding other patients, so that's an option that is definitely available and we're evaluating.
Thank you.
and Sami.
We'll go next to Patrick Trucchio with HC Wade Wright.
Patrick Truccio: Thanks. Good morning. Just a couple of questions from me. The first is I'm wondering if you can discuss the drivers of your revenue guidance and specifically what we should be looking for to suggest the year's revenue should trend to the higher end of the range.
Patrick Truccio: Rather than the lower end. And then secondly, I'm wondering if you can preview any of the design elements from the upcoming phase three trials for Patolousin HD and Arclapsin IH, including whether fatigue and sleep inertia endpoints may be formally powered in the HD trial for differentiating labeling.
Speaker Change: Morning Patrick, thanks for your questions. So Adam in terms of drivers of the business on Sandip if you want to come in as well.
Speaker Change: and this is something we've seen historically, very consistently, again back to the fundamentals of the business here. A very strong and stable, stable, continued progression of average patients on therapy. It's not going to be this, it's not going to be this.
Porter, Q1, we achieved 7200 patients.
Speaker Change: on WAKIX. We exited the quarter, actually, at 7,300, so we continue to see that strong momentum moving into the rest of the year, and we would expect to see that growth and performance continue. As we think about that, patient growth.
Speaker Change: We typically have been discussing, how do you grow the top line referrals?
Speaker Change: How do you make sure that we're converting patients to ensure that referrals are actually dispensed and what is the adherence and compliance with that therapy over time? I think in each of those buckets you see very strong stable fundamentals and we would continue to expect that through the remainder of this year. Yeah, and I would just add Patrick, I think it speaks to the...
Speaker Change: I think the underlying opportunity in the narcolepsy market. So, again, large market, 80s health and diagnosed patients, year six in the market, continues to grow, continued sort of momentum, and then the differentiated product profile.
Again, we tap into the 9,000 HEPs.
Speaker Change: Prescribing, you know, 5,000 of whom are not, you know, don't participate in and oxidate REMs. Continue to penetrate across that segment, about 50% or so penetrated, as well as the depth of the 4,000 HEPs with the larger clinics more patients.
Speaker Change: Strong underlying business fundamentals that are consistent and we're confident. We are confident in weight kicks being on its way to a billion dollar plus opportunity in narcolepsy alone.
Speaker Change: Thank you, Jeff. Hey, good morning, Patrick. Thank you for your questions on Pitholas and HD Phase 3 programs. We have already disclosed the study design for Idiopartic hyposomnia, which is aligned with the regulatory agency. It's going to be a double-blind, randomized, placebo-controlled parallel-arm study. Phase 3 knock-alepsic study, we haven't disclosed the design yet, and we will provide that information as we get ready to initiate the study. Thank you. Thank you.
Speaker Change: Daniel, other question was around fatigue. We have done some leading edge work in fatigue already. We have been working on this for more than two years now. We completed a qualitative research study.
to look at the instruments.
Speaker Change: To measure specifically fatigue, specifically impatience with narcolepsy and we are about to complete a really large longitudinal study looking at the prevalence and impact and the stability of symptoms of fatigue in patients with narcolepsy.
Speaker Change: The goal is certainly to get fatigued as an indication in narcolepsy, sooner or later, and we will be providing more information on that as we get closer to initiating the narcolepsy study. Thank you.
Thanks Patrick.
We'll go next to David Hoang with Deutsche Bank. Thank you.
David Huang: Hi, thanks so much for taking my questions. I'm Adam Welcome to the team.
David Huang: I want to ask a little bit about ZYNO2 in fragile X. If you do get positive data in hand, how quickly do you think you could file the NDA for that molecule? And could you just give us a sense of what commercialization of O2 in the fragile X market might look like? How do you think about the value proposition in this product and are there other analogs in rare neurological disease that we could look at?
Speaker Change: Yeah, good morning, David. Thanks for your question. Really excited about, you know, these EYN-002 fragile ex-opportunity. Kumar, you want to, you know, talk about.
Speaker Change: The development part, and then we can talk about market opportunity. Sure. Thank you, sir. Hey, good morning, David. Yeah, Frasal Ekcentrom. Look very excited for the top-line data as we disclose to this morning. We completed recruitment. Thank you, sir. Thank you.
and Closed for New Screenings.
Speaker Change: We are on track for top line data in 3Q of this year.
and if the results are positive. Thank you.
Speaker Change: We have the ability, the opportunity to bring the very first approved treatment to patients with Franois X syndrome. Once we get the top-line data, David, the goal is to file NDF as soon as possible. And given the unmet need, significant unmet need in this patient population, I think it's reasonable to expect.
Priority Review in an Indication Like There
Speaker Change: So, there will be an expedited timelines to submission of NDA, and I'm sure the FDI will also show the urgency to quickly review the data package and give their decision on Padufa. And of course, the other thing is in the background, we are working expeditiously on 22Q.
Speaker Change: We have aligned with the regulatory agencies on the primary end point and the phase three study, as you speak, we are getting ready to initiate phase three study in 22Q as well.
Speaker Change: Approximately 80,000 patients each in the US and Europe . And we have to mention in the past we have global rights for the 1002.
Speaker Change: and not how to go back on further like central, one thing is study design.
Speaker Change: Meet the requirement of not just the FDA, but also EMA for an indication for Franois Lexin from. Yeah, yeah. So David, suffice it to say with positive data, we will move swiftly in terms of, you know, preparing an NDA and submitting that. In terms of the, you know, the market opportunity and go to market so. [inaudible]
Speaker Change: So, significant opportunity for rare disease. We see this as a…
Speaker Change: Service Centers of Excellence Model, if you will, you know, with regards to neuro-behavioral, you know, syndromes and those types of disorders. We have a lot of institutional knowledge in the space that came over from our acquisitions, like NERBA.
Speaker Change: Noah Gwell, and then our sort of world-class patient advocacy team is fully engaged, you know, with the fragile ex-patient community, listening to them kind of understanding sort of the unmet needs.
Adam Zesky: You'll hear more from Adam going forward in terms of, you know, launch preparations as we, you know, plan for success with high conviction in the top line data.
We'll go next to Ashwani Verma with UBS.
Adam Zesky: Zatoel Milast, which is reading out in the second quarter. And they've shown privacy to data with cognitive assessment.
Speaker Change: Just what are your thoughts on PDE for the anaesthetic inhibited approach? And then I found like that endpoint is different versus what you're studying this one to make sure that these are both FDA approval or not. And then secondly on weight take so. So, um...
Speaker Change: I want to understand like these new patients that you're getting in, can you provide us a split of how much of that is coming from oxidative experience versus naive patients.
Thanks.
Speaker Change: Okay, good morning, Ash, thanks for your question. In terms of the Shinogi program, just very briefly, obviously following that closely, you know, very different program, as you mentioned, you know, the primary outcome around, you know, cognition.
Speaker Change: and Cognitive Function, only in male patients. So, if Kumar you want to comment further, but our program very different, as well as the importance of any new treatments for patient populations with one that medical need.
Adam Zesky: of Symptoms, when it comes to patients and their caregivers. Thanks, Kumar. Adam, I'm sure you don't like it. Thank you, Jeff. Thanks for the question. Yeah, so I guess in terms of sourcing of patients.
and many others. Thank you.
Adam Zesky: I think Jeff mentioned before the polypharmacy nature of the therapeutic approach here. So the reality is, the majority of patients that we're seeing referred for wakix have been on one or multiple other therapies prior to that referral. That's common, and that's going to be true for any product for sleepwake.
Adam Zesky: and then even when patients are on wake therapy, we continue to see that polypharmacy approach and so the majority of patients on wake therapy are also on one or additional other therapeutic options and certainly oxidates being a prominent
Option that ACTs have access to, that's going to be... [inaudible]
Adam Zesky: A proportional portion of those patients that would also have concomitant oxidative use. Yes, and I think the experience that concomitant use without 10 to 15% of patients on Wagex also in an oxidative, it's been fairly consistent, you know, since launch.
Thank you.
Thanks, Ash.
We'll go next to Jason Gerberry with Bank of America.
Jason Gerberi: Hey guys, thanks for taking my questions. Just on the erection data at sleep, I'm wondering if you can preview a little bit like what we'll get. I think when I looked at some of the other players who were in the clinic when they were
Jason Gerberi: Valuating their drugs preclinically, looking at the DTA mouse models and what endpoints will you give, and I look at like Sintessa's versus
Speaker Change: You know, we give latency to sleep or latency to cataplexy so we can benchmark and get a sense if the potency selectivity argument is starting to translate.
Speaker Change: on the efficacy side, and then just asking the polypharmacy question a little bit differently. What proportion of payers are amenable to two proprietary brand medicines?
Speaker Change: In the narcolepsy setting, just trying to get a sense because a new proprietary presumably expensive medicines come on board just wondering how payers will be amenable to covering multiple brands. Thanks.
Thank you.
Morgan, Jason, thank you for the question. And on...
Speaker Change: Kumar, on upcoming data at sleep? Sure, hey, good morning Jason, thanks for the question. Yes, we will be presenting a comprehensive pre-clinical safety and efficacy data, Jason at the upcoming sleep meeting in June in Seattle. And as I mentioned earlier during the call, I mean you'll see.
Speaker Change: Model, and the parameters that we will be discussing, you can expect to see the standard parameters like sleep latency.
Speaker Change: Wakefulness Time. They are in a mice model that is considered a standard for pre-clinical studies.
Speaker Change: That the efficacy is consistent with the very high potency that we have been talking about and also the preclinical safety data in GNP studies that will look very good.
Uh-huh.
Yeah, wait for the sleep mating. [inaudible]
Speaker Change: And Jason, I think your next question was about, can common use of wakix and oxybates in the payer landscape and um...
Speaker Change: You know, I think that the experience has been and we said this before, you know, given all the plans and. [inaudible]
Speaker Change: You know, each plan with not a lot of patience in the plans, that it's almost more in terms of administratively, you know, with reports regards to managing.
Unknown Executive, Sandip Kapadia
Speaker Change: So, you know, patients on both weight kicks and a branded oxidative has not been, you know, a challenge, you know, in the market for us because, you know, each of the plans don't have a lot of patients given that it's a rare disease.
Got it. Thank you.
Thank you Jason.
We'll go next to Charles Duncan with Cantor Fitzgerald.
Charles Dunkett: Hi, morning, Jeff and team, and Greg's on a nice quarter. Thanks for taking our question. I had a commercial question, and then a pipeline question, may I also have us?
Charles Dunkett: Strategy question at the end, but with regard to the commercial question, I get...
Charles Dunkett: and with the guidance to pen more on, you know, call it external, or external factors such as demand or more on internal efforts such as tweaks to the marketing program that you plan for, that you're contemplating for Wagex.
All right. Thank you.
Charles Dunkett: Thanks for the question, so what I can speak to is really the strong continued performance in Q1. And in Q1 we typically do see the traditional seasonality,
Charles Dunkett: Sinc Launch, and that has to do with the traditional payer resets that happen at the beginning of the year, so as patients move coverage, they have to reestablish prior authorizations and steps.
Charles Dunkett: and that takes a little bit of time. So that does have an impact on Q1 patient numbers but that's something that's seen actually not just for weight kicks and seen across sleep weight. It's also seen actually across.
Charles Dunkett: The Industry. So that's the typical seasonality that we would expect to see, and we weathered that successfully this quarter. As I mentioned, the momentum continues very strong as we're exiting the quarter, and we continue to have confidence for that full-year revenue guidance between 820 and 860 million for the full year.
So it sounds like it's the main driven, correct.
Charles Dunkett: So, I mean, yeah, so certainly the growth is, as Jeff said before, it's going to be primarily driven by demand as a result of the highly differentiated profile of WAKIX, but I will mention as well one of the things I've been really impressed with.
is the commercial model that has been put in place.
to support patients, to support HCPs, as well as caregivers. [inaudible]
Charles Dunkett: I think it's a key driver of performance, it's not just the model itself, but also how it's used with...
All referrals going into a single central hub. [inaudible]
Three specially pharmacies,
Charles Dunkett: and the broad set of data that we're able to capture.
Charles Dunkett: on Patients individually and longitudinally. It allows us to deploy intelligent investment decisions, resource planning, and make real-time decisions as we're seeing that data updated on a daily basis. So I think that's also an element.
Speaker Change: Okay, that's helpful. Sounds like no major changes in terms of sales strategy.
Speaker Change: No, no, no major changes, I think it's really what I said before. You're right, underlying demand remains strong.
Market Opportunity Remains Large, Strong Underlined Fundamentals.
and I think no major...
and changes to the strategy. We will see opportunities, obviously, as a brand mature, to make tweaks along the way. I think Adam is bringing fresh perspective and thinking with regards to that. And we see...
Speaker Change: continued opportunity ahead in our core franchise towards a billion dollar plus in northwest seat, as we prepare...
Speaker Change: to initiate the Phase Three Trials for Patolousin HD in both Norfolk and IH in the fourth quarter. So we are poised with a target to do from 2028 to transition the Patolousin franchise.
Speaker Change: with Utility Patents to 2044, well into the future to extend that franchise and generate significant value.
Speaker Change: That's a great segue, Jeff, to my last question, which is Petola Senn HD, in terms of the timing of that phase three start.
Speaker Change: Are there are there any experimental results you're waiting for to be able to operationalize at phase three with pitolis and HD in narcolepsy as well as perhaps IH by the end of the year?
Speaker Change: Yes, Charles, I think it's mainly just finalizing the formulation work, I'll turn over to Kumar, but all of the preparation is in motion. For those in Kumar, do you want to provide more color? Yeah, sure. Thanks, Jeff. Hey, good morning, Charles. Yeah, I mean, we always said we will initiate...
Loss of Pat and Ton Vegas, which is Q1 2030. [inaudible]
Speaker Change: Thank you. I'm showing no further questions. I would now like to turn the call back over for any closing remarks.
Speaker Change: Thanks Angela, I want to thank everyone for joining our call today. Harmony is well positioned to continue to grow our enterprise and generate durable value going forward. Thank you for your interest in Harmony and have a great day.
Speaker Change: This does conclude today's Harmony Bio-Science's first quarter 2025 Financial Results Conference call.
Speaker Change: You may now disconnect your line and have a wonderful day.