Harmony Biosciences at Goldman Sachs Conference: Strategic Growth Insights

On Monday, 09 June 2025, Harmony Biosciences (NASDAQ:HRMY) presented at the Goldman Sachs 46th Annual Global Healthcare Conference 2025, offering a strategic overview of its growth trajectory. The company highlighted its robust financial performance and ambitious pipeline developments, while also addressing challenges in expanding its prescriber base and managing market exclusivity.

Key Takeaways

• Harmony Biosciences projects Wakix sales of $820 million to $860 million for the year, aiming for $1 billion in narcolepsy alone.
• The company is developing next-generation formulations of pitolisant and expanding into rare neurological disorders.
• With over $600 million in cash, Harmony is poised to acquire additional programs to bolster its portfolio.
• Phase III results for ZYN-002 in Fragile X syndrome are expected in Q3 2025.
• The company plans to launch 1 to 2 new products or indications annually over the next 3 to 4 years.

Financial Results

• Wakix franchise sales for Q1 reached $185 million, marking a 20% growth.
• The company generated over $200 million in cash last year.
• Harmony maintains a strong balance sheet with over $600 million in cash reserves.

Operational Updates

• Wakix remains a strong revenue driver, with efforts to expand prescriber depth and breadth.
• New formulations of Tolucent are in development to address side effects and improve efficacy.
• Adam Zetsky, the newly appointed Chief Commercial Officer, brings 25 years of experience to drive commercial execution.

Future Outlook

• Harmony plans to introduce 1 to 2 new products or indications each year over the next 3 to 4 years.
• The company is focused on expanding its presence in CNS orphan rare diseases and exploring adjacent opportunities.
• Positive data from Phase III trials could lead to FDA and EMA submissions, enhancing the company’s growth prospects.

For more details, readers are encouraged to refer to the full transcript below.

Full transcript - Goldman Sachs 46th Annual Global Healthcare Conference 2025:

Karim, Host, Goldman Sachs: Good morning, and welcome to the Goldman Sachs Healthcare Conference. Thanks so much to the team from Harmony Biosciences for just joining us here today. And yes, I guess, we’ll kick it off. I would love to get started with just maybe an overview of the company. It’s changed a lot in the past, let’s call it year and a half.

So could you give us kind of an overview of the pipeline?

Unidentified speaker, Harmony Biosciences: Yes, sure, Karim. Thank you for the invitation. Great to be here. Yes. So I think at Harmony, we are well positioned.

We’re happy in terms of our position as a profitable self funding biotech company. Building off of four years of profitability in terms of our core commercial franchise in Wakix and narcolepsy that continues to grow. From there preparing for the next gen, the Tolucent formulations that we can talk about in the pipeline. With regards to what we’ve been building over the past two years, with our pipeline now that includes three orphan rare CNS franchises and we’ve got eight assets across 13 development programs and currently four in Phase three and by the end of the year up to six Phase three development programs. So with regards to the sleepwake franchise, neurobehavioral franchise and then also our rare epilepsy franchise, this pipeline is poised to deliver up to one to two new products or indication launches each year over the next three to four years.

So we think a very robust late stage catalyst rich pipeline in the orphan rare CNS space.

Karim, Host, Goldman Sachs: Great. Maybe we’ll start on the WCAGX franchise. This year you’ve put out full year guidance of $820,000,000 to $860,000,000 I guess talk to us about what that embeds with respect to patient growth for the full year? And how does it compare to kind of like the trajectory of patient growth you’ve been on to date?

Sandeep, Harmony Biosciences: Sure. No, we’ve got great momentum coming in with the first quarter. As you know, we announced about $185,000,000 of sales, which is growing about 20%. So really headed into our year at 20% sales growth. We exited the quarter with about 7,300 patients on therapy.

So we expect continued strong patient growth for the balance of the year. So a lot of opportunity as you know over the last five years, we’ve shown very consistent overall growth quarter over quarter. We expect the same for the balance of the year and we’d be approaching about 8,000 patients by the end of the year. So and again reinforce our guidance in terms of eight twenty million to $860,000,000 overall. And I would say well on our way to $1,000,000,000 plus in narcolepsy alone.

So pretty excited about this year. Again, it’s another milestone for the company.

Karim, Host, Goldman Sachs: You talked about nine thousand patients being kind of the level that gets you to $1,000,000,000 in sales at the rate you’re going. That means by the end of next year you’d be analyzing there? I guess what are the puts and takes that would take to get there by the end of twenty twenty six?

Sandeep, Harmony Biosciences: I think it’s really just around continued strong commercial execution as we go through this year and next year. There’s still a large opportunity. As you know, there’s still about 80,000 patients opportunity. We’re about 7,300 as I mentioned at the end of this quarter. So a lot of opportunity for continued growth in the marketplace.

And so we see continued growth like it’s the balance of the year as we go into next year as well. And like I said getting close to where we talked about reaching about 9,000 patients, which will get to $1,000,000,000 plus opportunity. And I think a lot of it is really driven by growing depth of prescribers in the oxybate physicians that the oxybate REMS and then really driving breadth for the physicians outside of the REMS program. And that’s really a unique feature about Wagex for us. So I don’t know if you have anything for us.

Unidentified speaker, Harmony Biosciences: No. Just think the underlying business fundamentals continue to be strong. And I think a lot of it as Sandeep said continued execution and tapping into a sizable market opportunity of about 80,000 patients diagnosed and really growing the business across the 9,000 HCPs that we call on both those in the oxybate REMS and the 5,000 that don’t participate. And a lot of this is based on the broad clinical utility, the differentiated product profile, the and only non scheduled product approved for narcolepsy. And year six in the market, I think it’s been consistent sort of performance and it really continues tapping into that market opportunity.

Karim, Host, Goldman Sachs: And you did recently hire a new Chief Commercial Officer. Can you just talk to me about the perspective that he brings to the role and anything kind of like new that we should expect? Obviously, it’s been successful to date, so you wouldn’t want to it’s not Yes.

Unidentified speaker, Harmony Biosciences: So it’s not broke. And I think that so that’s sort of the takeaway. Yes. So our new Chief Commercial Officer, Adam Zetsky, who’s been on board now about six to eight weeks or so. So Adam, he when he was doing his diligence on Harmony, he saw obviously a successful commercial business continuing to grow.

And I think now that he’s been in, his observations are that really validating what he saw. So he brings twenty five years of experience in the industry, about twenty years at Takeda. His last role is heading up commercial operations for a big part of Europe. He’s been in every commercial role, so we understand from sales and marketing, market access. He’s done a lot of analytics on life cycle management.

So I think his initial observations are strong business continue to build off of that. No major changes with regards to how the marketing strategy and how things are being executed. He also brings a fresh pair of eyes with regards to year six in the market where are some of the levers that potentially that we could pull to kind of accelerate some of the growth and really building off of a successful franchise. And he doesn’t plan any major changes to this

Karim, Host, Goldman Sachs: Of course. You mentioned life cycle management a little bit in that answer and I’d love to talk a little more about that. At a high level, you have this like gastric resistant and high dose formulations. Where do you see this fitting into the Wagix franchise? And maybe you could walk through the development time line for each of those programs?

Unidentified speaker, Harmony Biosciences: Sure. Yes. So I think the opportunity with the Tulsun franchise, now with latex and LOE out to 02/1930, we have the runway in terms of the next generation formulation. So the one is really an opportunistic play pitolacin GR or gastro resistant formulation. So it’s a quick to market strategy based on the demonstration of bioequivalence to WCAG.

It’s got a gastro resistant coating, which can help address. It’s not that there’s a lot of GI side effects in patients on WCAG. But patients with narcolepsy are prone to sort of GI symptoms nausea, dyspepsia. A lot of that is mechanism based because when you have orexin deficiency, orexin drives the vagus nerve in which controls gut motility. So patients with narcolepsy, it’s almost like a comorbidity or a coexisting condition.

So GR can kind of help minimize the potential for GI side effects. In addition, we’re also doing a study to remove the titration dose with a gastro resistant formulation. So we’ll be able to patients will be able to start on the therapeutic dosing range and kind of achieve efficacy quicker. So GR, we are actually doing the pivotal bioequivalent study now and on track for the data readout from the pivotal BE study in the third quarter. Target PDUFA date in 2026.

So that is meant to the design there is meant to expand the base of patients on both Wakix and Fetolacin GR. And also the expanded base is the next formulation Fetolacin high dose. So that is a that’s the more sort of novel in terms of the next generation product. So it’s a new formulation designed with an optimum PK profile for greater bioavailability. And then we have also with a gastro resistant coating.

So driving the major unmet need in the market of greater efficacy in terms of about seventy five percent of patients have residual symptoms both EDS and cataplexy. So Fertullisin HD with regards to the opportunity there, we’re on track to initiate the Phase III pivotal trials in both narcolepsy and idiopathic hypersomnia in the fourth quarter this year, In addition to the usual endpoint, the other opportunity with high dose is to target unique symptoms in both of those patient populations. So in narcolepsy, we’ll be evaluating the potential to treat narcolepsy related fatigue. So fatigue is a different construct. It’s a different symptom than EDS and it’s mediated through histaminergic circuits.

So I think there is a rationale why pitolacin working through histamine could be effective. Fatigue is present in about sixty percent to seventy percent of patients with narcolepsy and that would help in terms of driving a differentiated label for Fertullis in HD. And then in the IH program in addition to EDF, there’s an important symptom of sleep inertia. So that will also be a key endpoint in the Phase III pivotal trial.

Karim, Host, Goldman Sachs: You spoke qualitatively about the benefits of a high dose. Maybe you could help quantify what that means and some of these endpoints to prove kind of the clinical differentiation or meaningful differentiation

Unidentified speaker, Harmony Biosciences: on high

Karim, Host, Goldman Sachs: So I

Unidentified speaker, Harmony Biosciences: think the meaningful differentiation is so in terms of improvement on the FR sleepiness scale score, it’s about a two point improvement within patient improvement. Is felt to be clinically meaningful. So I think that’s sort of the threshold that the Phase three program is designed to demonstrate. So meaningful improvement on EDS compared to WCAG and then obviously gaining an indication in narcolepsy induced fatigue which will help further differentiate the product profile.

Karim, Host, Goldman Sachs: Okay. And how do you think about the right patient population for whom there is this kind of residual unmet need? What portion of patients on wake up would be great candidates for this?

Unidentified speaker, Harmony Biosciences: Yes. I mean, think it’s not just on wake up. I think that there’s a statistic about seventy five percent of patients treated for narcolepsy have residual symptoms with regards to and especially EDS being the primary symptom. There’s a sizable opportunity in those patients that are on therapy with residual symptoms as well as newly diagnosed patients coming in where you’ve got an enhanced PK profile, greater bioavailability, higher dose, gastric resistant coating. So the overall, I think product profile would be I think valuable for a lot of these patients in the market when we come with a target PDUFA in 2028.

Karim, Host, Goldman Sachs: Okay. In terms of dose dependency, what do we know about ptolosin that then kind of supports the thesis that a higher dose is going to deliver these kind of benefits as you’re speaking about?

Unidentified speaker, Harmony Biosciences: Yes. So I think the clinical development program kind of over the years both starting with the pivotal programs in narcolepsy. So for instance in the label the seventeen point eight milligram dose has about a five point improvement in the Epworth. The thirty five point six has about a point five point improvement. Then we have other dose response and exposure response data in our other clinical trials.

I think that supports the benefit in terms of going up on dosage range, which was never fully interrogated. Okay. Yes. And we’ve also shared we have the safety margins. We did a repeat dose safety study up to one hundred and eighty milligrams, which shows adequate safety margins to go up on the dose for pitulsin HD.

Karim, Host, Goldman Sachs: Okay. Great. And as you speak to payers about this profile, the target profile that you just kind of outlined, what are they looking forward to kind of justify paying for this even in the context of a generic?

Unidentified speaker, Harmony Biosciences: So I think that we’ve done some market research with payers that maybe if you want to kind of share the findings.

Sandeep, Harmony Biosciences: Yes. No, I think what we’ve done is the research with payers. And essentially prior to WAKX LOE, I think payers said as long as it’s priced similarly parity, I think we would have the opportunity for patients that are even on WAKICS to transition to that. And I think post LOE, they would have to have some exposure to potentially on ptolacide potentially in fatigue and other symptoms as well. So I think that’s really it’s really the efficacy differentials that would help make the case much stronger for FITELISON HD along with greater bioavailability.

Unidentified speaker, Harmony Biosciences: Yes. Yes. I mean, I think the initial market research both payers as well as HCPs, they see the target product profile as being a superior product with higher efficacy in the core symptoms and then fatigue is an added benefit. Fatigue is in about sixty percent to seventy percent of patients a different sort of construct than sleepiness. So the feedback was that this would be incremental for the current Wagex profile.

Sure.

Karim, Host, Goldman Sachs: And you’ve mentioned LOE a couple of times thus far. You recently settled a couple of the generic patent suits. Maybe just give us the update in terms of the latest on those patent suits? And should we expect everything to now kind of triangulate around January 2030 or July depending on Yes.

Unidentified speaker, Harmony Biosciences: So I think that yes, so we feel good about the progress we’ve made in terms of the ANDA settlements. Yes. So last week we announced that we settled with Lupin, which is sort of the biggest of the ANDA filers. And we settled similar to the two. It’s the settlement and with an entry date of January 2030.

The base case was February with regards to LOE. So I think good settlement terms. And then we’re also on track to gain pediatric exclusivity, which would be an additional six months taking it to February. And I think that with that momentum and as more of the parties settle then there’s less sort of remaining in the It’s more expensive. And I think that gaining good momentum as we continue to engage with the remaining parties.

Karim, Host, Goldman Sachs: How many more are there?

Unidentified speaker, Harmony Biosciences: So there are four remaining. Okay. Are seven.

Karim, Host, Goldman Sachs: Three settled, four remaining. Perfect. Okay. Maybe we can switch gears to the pipeline for a bit. You mentioned I think you mentioned this earlier, Fragile X syndrome is the next clinical readout that’s coming.

You got Phase III results in the third quarter. Maybe let’s just take a step back and revisit the acquisition that brought you this asset. In 2023, you acquired it. What was the impetus behind that acquisition? And what did you kind of like about the product and market?

Unidentified speaker, Harmony Biosciences: Yes. So I think we saw strong clinical data from the Phase II study in another orphan rare sort of neurologic or behavioral disorder. And we had been following Zynerva. It’s actually a company down the road from us and knew some members of the team. And they were doing good work in generating strong data in an area where there are no approved therapies and major unmet medical need.

I mean, maybe Sandeep, two comments on the deal terms in terms

Sandeep, Harmony Biosciences: of what we’re able to achieve? Yes. No, I think we had very attractive deals. Acquired the company for about $60,000,000 We had about $26,000,000 in cash and we were able because we’re a profitable company leverage a lot of the NOLs. So we took a P and L head of probably around like single digit millions in terms of to acquire very late stage Phase III programs.

And we spent probably the last year or so about $30,000,000 to get to an answer for an opportunity that is pretty sizable. We’ll speak a little bit more about it, but a real we have high conviction in the program and we think that it’s successful. Really it offers the treatment potentially for patients with fragile X.

Unidentified speaker, Harmony Biosciences: Yes. I think it’s a really exciting opportunity. I know we don’t have a lot of time. But so the product. So I think it’s an innovative product.

So it’s a purely CYM-two is a purely synthetic cannabidiol, pharmaceutically manufactured, devoid of THC. And it’s delivered through it’s a transdermal application. It’s a permeation enhanced patent protected gel. The benefit of that is it bypasses pass metabolism. So the oral cannabidiol products and I think the one that’s best known is Epidiolex, which has a lot of rate limiting sort of GI tolerability issues about 20% to 30% sort of nausea, vomiting, diarrhea, appetite suppression.

And then you also have to monitor LFTs. So with ZYN-two and transdermal delivery, it bypasses pass metabolism and we don’t see any of the GI tolerability issues from the clinical trials and no elevation in LFTs. So I think that’s a very innovative product. And then we saw the Phase two CONNECT study, which over 200 patients, a sizable trial in patients with fragile X and strong data in the subgroup of patients with complete methylation of the FMR1 gene, I’ll come back to that. It showed a statistically significant and clinically relevant outcome on the primary endpoint in terms of the social avoidance subscale of the ABC Fragile X checklist.

So with that, we learned a lot about in terms of the data and lessons learned from the Phase II study and then design them into the Phase three trial, which is really designed to replicate the positive findings of the Phase two trial. And with that, the primary endpoint is on patients with complete methylation of the FMR1 gene. And the importance of that, those patients have more severe symptoms and greater ability to show benefit with treatment and it’s sort of more sort of homogeneous group. Patients with partial methylation still maintain some FMR protein. So their symptoms are not as severe.

Karim, Host, Goldman Sachs: Okay.

Unidentified speaker, Harmony Biosciences: And we also extended the duration of the trial, because we saw in the Phase two trial patients continue to improve from the twelve week endpoint out to sixteen weeks. So we increased the length of the Phase three RECONNECT trial to basically achieve the continued benefit in those patients. And then lastly, we saw a dose response and added a higher dose patients over fifty kilos in the Phase three RECONNECT trial. So in essence, we’re replicating the findings of the positive Phase two study with those enhancements and have a high degree of conviction in the probability of success for ZYN-two. And we’re on track for top line data readout in the third quarter.

Karim, Host, Goldman Sachs: Okay. You mentioned a number of my questions. I’m going to skip ahead. What is the primary endpoint that you’re looking at in the Phase III? And remind us how it’s kind of measured and evaluated?

Unidentified speaker, Harmony Biosciences: Yes. So the primary outcome is so Fragile X disorder or behavioral symptoms. And so the main ones are social avoidance, which is the primary outcome and then irritability and anxiety. So those are all part of the ABC checklist for fragile X. There’s sort of six domains.

So these are three of the key behavioral symptoms with social avoidance being the primary outcome and then irritability and anxiety key secondary outcomes. So it’s basically it’s a 12 scale on the social avoidance subscale of the ABC checklist. And then a three point improvement is felt to be clinically meaningful. And the study is powered at 90% to demonstrate that clinically meaningful improvement.

Karim, Host, Goldman Sachs: Okay. Understood. In terms of the Phase III, I guess, how did you think about what the placebo arm would do given this is a patient population that I’m sure there’s a good degree of variability? And then can you talk about any sort of execution features that help to manage the placebo cohort?

Unidentified speaker, Harmony Biosciences: Yes. No, I think that question often comes up these scales and how do you manage placebo response. And so I think once we brought this study in, basically through the clinical operations team and a lot of boots on the ground. So we did a lot of rater training. So a lot of it is training.

It’s an observer scale, either the parents or the caregivers. And then training with regards to trying to minimize placebo response and minimize kind of variability in the response rates on that scale.

Karim, Host, Goldman Sachs: Okay. Understood. In terms of next step, let’s say, we see positive data in the third quarter, what comes after that?

Unidentified speaker, Harmony Biosciences: So with positive data, I think we’re looking to engage with the agency and then look to submit an NDA and seek approval. Again, no approved treatments. I think that we the Phase II study will be supported in the positive data there. So be able to with positive data in the Phase III RECONNECT trial build a strong NDA submission and then work with the agency during the review process. The other opportunity is with positive data, there’s another related disorder as a follow on indication in what’s called 22q deletion syndrome, also known as the Georgios syndrome.

So similar neurobehavioral pattern. Zynerva generated positive open label Phase II data. We’ve engaged with FDA on a Phase three trial design on a primary endpoint and has gained acclaim there. So actually Trimur and the team are preparing to initiate the pivotal Phase three in 22q late this year if we read out with positive data in Fragile X and then we’ll follow that program on for that opportunity. In terms of the market opportunity, both of them are about eighty thousand patients in The U.

S. With Fragile X as well as 22q about sixty thousand patients by claims data. And in addition for ZYN-thirty two, we also have global rights to that asset.

Karim, Host, Goldman Sachs: Okay.

Unidentified speaker, Harmony Biosciences: So with positive data, the studies are also designed to satisfy not just submission for FDA, but also EMA as well. Okay. So we would have an opportunity to partner that out ex in other markets.

Karim, Host, Goldman Sachs: Okay. Great. Maybe just remind us on

Unidentified speaker, Harmony Biosciences: the patent side, what’s the patent around this particular asset? Yes. The patent, at this point, are method of use patents in these conditions to the late 2030s.

Karim, Host, Goldman Sachs: Okay. Great. Maybe continuing on with the pipeline. Last year you did acquire anorexin-two agonist. Maybe let’s start with how you think about that class of therapy.

There’s a lot of noise there and the potential role given your experience in narcolepsy that those drugs could play?

Unidentified speaker, Harmony Biosciences: Yes. So obviously, erection to Agnes, a lot of excitement there, a lot of interesting work there. And I think the main update is for this week, the sleep the annual sleep conference is taking place in Seattle. So we will be presenting on Wednesday, a full panel of our preclinical safety and efficacy data out of sleep. And what it will show, we’ve been talking about the importance of potency in the OREXAN2 agonist compound.

And I think what it will demonstrate is what we’ve seen that our compound has the highest potency of what’s kind of in the public And that will translate and show the high degree in the preclinical narcolepsy models of efficacy and the benefit of in terms of the properties of our REXN2 agonist. Okay.

Karim, Host, Goldman Sachs: How do you think this class is going to be used? Do you expect it will be used beyond the NT1 patient population? And why or why not?

Unidentified speaker, Harmony Biosciences: Yes. So I think that, obviously, we’re still learning a lot about the orexin-two agonist in the I think the importance of potency is that the lower the experience to date, the lower you can dose and generate good efficacy and minimize some of the safety and tolerability features then that will help possibly be beneficial beyond NT1.

Sandeep, Harmony Biosciences: Okay.

Unidentified speaker, Harmony Biosciences: So I think Takeda obviously the most advanced program and their initial target is on NT1. And then you have Alkermes in the clinic looking at both NT1 and NT2 and now looking at IH. But it’s still I think the ultimate product profiles of what these products will look like from a safety and efficacy perspective, I think it’s still playing out in

Karim, Host, Goldman Sachs: we think about the bar for moving forward or the threshold you’d like to hit to move forward with this program, anything we should be mindful of with the Phase one data?

Unidentified speaker, Harmony Biosciences: Yes. So I think at this point, we are on track to submit an IMPD middle of this year and then go into the clinic half of this year in human studies. And then we’ll have our initial clinical data next year. And we would look to based on the preclinical data that we’ve seen generate strong efficacy signals. And at that point based on the work to date and what’s known about this class then look to accelerate our clinical development program.

Karim, Host, Goldman Sachs: Okay. Maybe we can talk a little bit about epilepsy. You also acquired a couple of assets last year. The DPX-one hundred in development for Lennox Gastaut and Dravet syndrome. The preclinical data is pretty supportive of the efficacy of the agent.

But let’s talk about how you designed the Phase three study to capture that activity.

Unidentified speaker, Harmony Biosciences: Yes. So I think that the so for EPX-one hundred, so clamazol hydrochloride, a five HT2 agonist. So I think importantly, right now we have the most advanced five HT2 agonist programs in the clinic for the rare epilepsies. So as you mentioned, DPX-one hundred in Phase three for Dravet syndrome and then we initiated late last year and now in Phase three for Lennox. So I think they are the trial designs are pretty standard in terms of demonstrating reduction in countable motor seizures in those patient populations.

I think similar to some of the other products on the market that are approved for those indications.

Karim, Host, Goldman Sachs: You went straight into Phase III given this is a drug with tons of safety data. So talk to us how you thought about dose selection and powering around demonstrating the appropriate clinical endpoints?

Unidentified speaker, Harmony Biosciences: Yes. I think dose selection was based on some of the preclinical models and then the studies were adequately powered in terms of four registrational Phase three trial. And that’s kind of what guided in terms of the UPX100 into the clinic.

Karim, Host, Goldman Sachs: You’ve talked about one of the areas of differentiation for this versus some of the other programs in development is reduced monitoring requirements. So can you talk about what that means? Explain a little bit more and how you expect that to play out in the clinic?

Unidentified speaker, Harmony Biosciences: Yes. So the benefit, yes, so Climazole hydrochloride is was a generation antihistamine. So it’s actually on the market for about twenty years. So a lot of post marketing safety data, which didn’t show any major safety signals. It was sunsetted when the generation antihistamine product came in.

So I think compared to what you have currently on the market that are effective agents for the rare epilepsy, so Epidiolex and then FINTEPLA. And I think with Epidiolex, as I mentioned, the need to monitor LSTs and some of the rate limiting GI side effects. FINTEPLA, tenfluramine, I actually know well from my days at Viropharma, when we actually looked at that many moons ago. But there it’s really the cardiac risk in terms of the valvulopathy and pulmonary hypertension. Hypertension.

So it’s actually a REMS program for you need to do echocardiogram prior to initiating therapy and then you have to sort of follow with echocardiogram. So in EPX-one 100, the idea is comparable efficacy or possibly better with a improved safety tolerability profile could deliver an overall benefit risk profile favorable for these kids that still have severe refractory seizures and a new therapeutic options would be very valuable.

Karim, Host, Goldman Sachs: And how do you see this market shaping up in terms of polypharmacy or competitive dynamics?

Unidentified speaker, Harmony Biosciences: Where does this fit? Probably similar to the narcolepsy market. I think polypharmacy is common. Really refractory seizure is difficult to treat. Multiple mechanisms are often kind of included.

So I think it’s polypharmacy market, new product offerings will be important. I think patients likely will cycle on and off therapy based on what combinations may or may not work for an

Karim, Host, Goldman Sachs: So you mentioned this drug has existed for a long time. So talk to us about the patent.

Unidentified speaker, Harmony Biosciences: So patents similar to ZYN-two into the late 30s based on methods of use for Dravet, LGS as well as broader PEEs.

Karim, Host, Goldman Sachs: Okay. And where are you in terms of determining a registrational path

Unidentified speaker, Harmony Biosciences: for other DEEs? I think that we said that we’ve taken the approach sort of a more focused approach in Dravet, which is more a homogeneous condition and now into Lennox Gastaut. From there we have optionality either with EPX-one hundred potentially going broader after it’s successful gaining those indications. But we also have EPX-two hundred, which is a liquid lorcasiran, which is a more five HT2C specific agent. So that we are working on final formulation and looking to go into the clinic.

That could be another option for us to go right into kind of a broader VEE type of basket trial.

Karim, Host, Goldman Sachs: Perfect. Maybe in our remaining time, you can just speak briefly to the capital allocation priorities for the company.

Unidentified speaker, Harmony Biosciences: So priorities are to I’ll start and Sandeep chime in, continue to grow. So, obviously, strong balance sheet over $600,000,000 We feel that we’re just getting started. We’ve built sort of the enterprise. We’ve built a team, a lot of experience, a lot of expertise, unique commercial model. Obviously, Kumar has built a strong R and D bench.

And I think with the balance sheet, we’re looking and the current market backdrop to continue to add to either our current CNS orphan rare franchises or possibly an adjacent opportunity if we see something that’s compelling.

Sandeep, Harmony Biosciences: No, think you covered. I mean, we have a very unique profile of the company, right? We’re positive cash flow generating. Last year, generated over $200,000,000 of cash. We have really high EBITDA.

So really ability to leverage not only our company financial profile, but also the strength of the balance sheet with over $600,000,000 to be able to acquire additional programs. Again, as we’ve done already, we’ve demonstrated over the years to get many late stage programs. So we continue to look for opportunities to do that and continue to drive value for shareholders.

Karim, Host, Goldman Sachs: Great. Well, with that, I think that takes us to the end of time and all my questions. Appreciate it today guys. Thanks, Thanks, Thanks,

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