Ladies and gentlemen, thank you for standing by. And welcome to the Harmony Biosciences First Quarter 2021 Financial Update Call. At this time, all participant lines are in a listen-only mode. After the speakers' presentation, there will be a question-and-answer session. [Operator instructions] Please be advised that today's conference is being recorded. [Operator Instructions]. I would now like to hand the conference over to your host today, Lisa Caperelli, Investor Relations. Please go ahead.

Thank you, operator. Good morning, everyone and thank you for joining us today as we review Harmony Biosciences' first quarter 2021 financial performance and provide business updates. Before we begin, I encourage everyone to go to the Investors section of the Harmony Biosciences website to find the press release and slides that accompany our discussion today, including a reconciliation of the GAAP to non-GAAP financial measures. At this stage of our company life cycle, we believe non-GAAP financial results better represent the economics of our business. Our presenters on today's call are John Jacobs, President and CEO; Dr. Jeffrey Dayno, Chief Medical Officer; Jeffrey Dierks, Chief Commercial Officer; and Sandip Kapadia, Chief Financial Officer. Moving to Slide 2. As a reminder, we will be making forward-looking statements today, which are based on our current expectations and beliefs. These statements are subject to certain risks and uncertainties, and our actual results may differ materially. I encourage you to consult the risk factors discussed in our SEC filings for additional details. At this time, I'd like to turn the call over to our CEO, John Jacobs. John?

Thank you for the introduction, Lisa. And I would also like to thank all of the participants for joining our first quarter 2021 conference call this morning. I am pleased to share with you that once again, we reported strong sales for WAKIX, with Q1 2021 revenues of approximately $60 million and achieved profitability for the first time in our company history. We delivered these results despite the lingering challenges of a pandemic, and the anticipated seasonal payer dynamics that impact all branded and specialty products in the first quarter, largely due to insurance resets at the beginning of each year. We believe this performance driven by continued strong demand for WAKIX underscores that WAKIX is addressing significant unmet medical needs in the narcolepsy market, as our first-in-class meaningfully differentiated products for people living with this rare neurological condition. Since our inception in 2017, we have focused on developing and marketing novel therapeutic options for people living with rare neurological diseases. Our commitment to these patients has never faltered. And we continue to help an expanding number of narcolepsy patients with WAKIX, while making significant progress on our new clinical programs, which are designed to expand the utility of WAKIX to new patient populations beyond narcolepsy, including Prader-Willi Syndrome and myotonic dystrophy. Harmony's success is driven by our hard-working employees, the patients and healthcare professionals that have embraced WAKIX and the investigators and patients involved in our clinical trials. The momentum we saw in 2020 has continued into 2021. With our robust sales trajectory and strong cash position, Harmony is well positioned to continue to execute on our three pillars of growth this year, which are seen on Slide 3 and include: pillar one, optimize the commercial performance of WAKIX. We reported Q1 net revenues of $59.7 million driven by growth in the average number of patients and the number of healthcare professionals prescribing WAKIX since launch. Pillar two, expand the clinical utility of WAKIX. By mid-year we expect to have to Phase 2 programs in the clinic investigating pitolisant for potential new indications. And pillar three, acquire new assets to expand our portfolio. We have a dedicated team which is focused on identifying new assets for potential acquisition within our area of expertise in order to expand our pipeline. We are starting this process early in our company lifecycle, so we have the time to be thoughtful in our approach and to execute well without unnecessary distraction to our core business with pitolisant. With more than $140 million in cash and cash equivalents on the balance sheet, continued positive sales growth and now having reached profitability, we are well positioned to act. In summary, I am very pleased with our first quarter performance and look forward to keeping you updated on our progress as we continue to advance our three-pillar strategy to grow the company. I would now like to turn over the call to Jeff Dierks, our Chief Commercial Officer. Jeff?

Thanks, John. I continue to be impressed with the momentum and progress we've achieved through our launch, as indicated by another strong quarter of performance as noted on Slide 4. Net revenues for the first quarter were $59.7 million, demonstrating strong performance and representing our fifth consecutive quarter of net revenue growth. Moving on to Slide 5. For our q1 commercial results, I'd like to highlight a few key performance metrics. We continue to increase the average number of WAKIX patients. In the first quarter, the average number of patients on WAKIX increased to approximately 2800, demonstrating continued strong demand for WAKIX and the meaningfully differentiated product profile. The WAKIX prescriber base continued to increase with more than 2700 unique healthcare professionals have prescribed WAKIX since launch through the end of the first quarter. This represents approximately 33% of the approximate 8000 healthcare professionals who treat the diagnosed narcolepsy patient population. Market access for WAKIX continues to be strong. We've maintained strong formulary access across commercial Medicare and Medicaid insurance plans. And continue to see additional favorable access for WAKIX subsequent to the approval of the cataplexy indication and October of 2020. And with respect to the cataplexy indication, the volume of prescription requests for narcolepsy patients with cataplexy, or Type 1 narcolepsy continued to increase in the first quarter. This growth demonstrates the added value of the cataplexy indication to the overall benefit risk profile of WAKIX. Additional managed care plans made positive formulary changes for Type 1 patients in Q1, building upon the decisions we saw in q4 2020, further helping to accelerate patient's ability to get access to WAKIX. Now, prior to the approval of WAKIX, we heard through market research from the Narcolepsy Community, that there were a number of unmet needs in the market. And…

Thank you, Jeff. And good morning everyone. At Harmony, the second pillar of our growth strategy is to expand the clinical utility of pitolisant by pursuing new indications in additional orphan rare neurological disorders, with unmet medical needs, and by expanding the label in narcolepsy to include pediatric patients. Our current development programs are shown on our pipeline chart as seen on Slide number 9. With regard to pursuing new indications in additional orphan rare neurological disorders, all sites have been activated and are currently enrolling patients in our Phase 2 trial in patients with Prader-Willi Syndrome or PWS. This trial is evaluating pitolisant for excessive daytime sleepiness or EDS, behavioral symptoms and impaired cognitive function. PWS effects approximately 15,000 to 20,000 patients in the United States and represents an unmet medical need for which Harmony is committed to pursuing a new treatment option for this patient community. A potential larger market opportunity is highlighted by another one of our development programs. That being in patients with myotonic dystrophy or DM, which is the most common form of adult onset muscular dystrophy. And one that is a genetic disorder inherited in an autosomal dominant pattern. Latest estimate suggests a prevalence of about five per 10,000 people with a genetic defect for myotonic dystrophy Type 1, or DM1, which is the most common form of this disorder. This equates to about 160,000 people in the U.S. with a genetic defect for DM1. Based on the clinical presentation, with 50% of this population likely being symptomatic, and of those about 50% being diagnosed. The current number of people diagnosed with DM1 in the U.S. is approximately 40,000. Of these, 80% to 90% experience excessive daytime sleepiness and over 90% also report fatigue. So EDS and fatigue are the two most common non-muscular…

Thank you, Jeff. And good morning, everyone. I'm excited to join Harmony Biosciences and look forward to meeting many of you. Earlier today, we posted our first quarter 2021 press release and filed our 10-Q, where you can find details on our financial and operating results for the quarter. I'd like to take the opportunity to share with you some of the highlights from the quarter which can be seen on Slide 12 and 13. I'm pleased to see that the momentum from 2020 has continued into the first quarter of 2021. We grew WAKIX sales to our highest quarter-to-date, managed our operating expenses, and posted our first quarter positive net income and earnings per share. For the first quarter of 2021, Harmony posted $59.7 million in WAKIX net product revenues, as compared to $19.8 million in the prior year quarter. We're pleased with the continued sequential growth of revenues, especially in the first quarter, as there are typical headwinds from insurance reauthorizations and copays resetting. We continue to see an increase in average number of patients on therapy during the first quarter, as well as the number of healthcare professionals prescribing WAKIX. For the first quarter 2021 cost of product sold were $10.4 million, versus 3.5 million in the prior year quarter. For the first quarter of 2021, GAAP operating expenses were $34.7 million, up significantly versus the prior year quarter of $26 million. The increase was anticipated as we in 2020 advanced our pipeline programs in PWS DM, increased sales and marketing activities associated with the WAKIX launch and expanded our operations as we became a public company. For the first quarter of 2021, Harmony posted GAAP net income of $7.4 million or $0.13 per share. This compares favorably to last year's first quarter net loss of $38.6…

Thank you, Sandip, and the entire harmony team for your continued dedication. In summary, I am very pleased with our continued progress on our three pillars of growth. Q1 was an outstanding quarter, representing our fifth consecutive quarter of sequential revenue growth. We continue to advance our development programs in Prader-Willi Syndrome and myotonic dystrophy. Financially we are in a strong cash position, and for the first time in our company history, have reached profitability. This success positions as well to pursue our third pillar of growth, acquiring additional assets to expand our pipeline. Operator, we will now take questions from the audience?

Thank you. [Operator Instructions] Our first question comes on the line of David Amsellem with Piper Sandler. Your line is now open.

Hey, thanks. So just a couple. So first, wanted to get your high-level thoughts on more aggressive payer contracting down the road to the extent you think you need to do it. And the reason I ask is that, Jazz has contracted fairly aggressively on Xywav and with some success. Obviously not an apples-to-apples comparison here, but with contracting in the space, how do you think about your contracting position for WAKIX? So that's number one. And then number two is, in terms of the cadence versus for sales this year. And you talked about some headwinds for 1Q seasonality, things that are customary across the space. With that in mind, can you just talk about what that means for say 2Q in terms of moderation of gross to net? Thanks.

Hey, Jeff, why don't you take David's first question?

Good morning, David. Thank you for the question. So with respect to managed care, we're extremely pleased with the favorable market access coverage that we've seen with WAKIX since launch. And as we shared in previous calls, we've secured approximately 80% coverage of all U.S. insured lives across commercial Medicare, Part D and Medicaid. And since the recent approval of the cataplexy indication, we've seen even additional favorable access for WAKIX, building on those that we've seen in Q4. And really what that doing is either eliminating or reducing some of the step edits for Type 1 lives. And so we believe we're very well positioned within the market access landscape to support our patients. We have selectively contracted with a handful of plans that we do not believe that there will be a need for aggressive contracting, given the overall benefit risk profile of WAKIX and what we've been seeing in the market. So with that, if that answers your question, I can turn it over to Sandip and answer the second part of your question with respect to cadence on sales.

Yeah. Go ahead, Sandip.

Yeah, hi. We had a really strong quarter of dry [ph] placement $59.7 million in top-line revenue, really solid growth versus fourth quarter and certainly versus prior year. As you mentioned, typically, we have first quarter headwinds, which results in slightly higher growth in that in the first quarter. We obviously see that reversing as we go through the rest of the year. And we expect, strong quarter-over-quarter growth for the balance of the year. So very, very pleased with how we came out of Q1. And I think, well-positioned for the balance of the year as well. Hopefully that gives you some color there.

Yeah. No, that's really helpful. If I may just ask one more - sneak in one more just on business development and M&A. Obviously, you've been talking about that for a while. Can you give us a sense of how aggressive you're willing to be in terms of potential deal size? I know that's a tough one to answer, you're probably looking at a lot of different assets. But, how big can you go for an asset, whether it's commercial stage or pipeline?

David, what I will say is we're determined to broaden our pipeline with additional assets. As you've stated, we're starting early in our company history. So we can take our time to do this thoughtfully. And we want to make sure that whatever we acquire, that it's complimentary to what we do now at Harmony, what we know how to do well. And that it allows us internal synergy, leverage our current infrastructure and expertise. And it should be in the rare orphan neurological and CNS arena where we have that infrastructure deployed right now. And certainly, we want to make sure that we have the right - the capability to acquire that. And that's one of the reasons we chose to go public last year. We're in a very strong cash position right now. We have optionality on how to finance a potential deal when we do find the correct deal for Harmony.

Okay. That's great. Thanks.

Thank you. Our next question comes from the line of Chris Howerton with Jefferies. Your line is now open.

Hi. Good morning, everybody. Thanks so much for taking the questions. And congratulations on the progress this quarter. So I think maybe just a couple questions from me, likely mostly related to Jeff Dayno. I guess first of all, with respect to myotonic dystrophy, I guess, if you can just give us a sense in terms of what does it take to obtain that diagnosis? And maybe, if you have some thoughts as to what the shape or trajectory of that diagnosis rate might be, when and if therapies become available for those patients such as WAKIX? So that would be question one. And then question two is just with respect to the pediatric narcolepsy study that was described by Bioprojet. Just curious if we could get a little more color, I might have missed it in terms of what exactly the headwinds were to that study? And maybe just a reiteration of what we can know about the timelines at this point? Thank you.

Yeah. Sure, Chris. Good morning. Thanks for the question. So with regards to myotonic dystrophy, so the diagnosis, it's a genetic disorder. And it's inherited in an autosomal dominant pattern. So it's basically a genetic diagnosis, that's how the diagnosis is confirmed. Symptoms include what's called myotonia, so the problems - difficulty muscle relaxing which results in muscle weakness. But also what we've learned and as the disorder is more recognized over the years that EDS, excessive daytime sleepiness and fatigue are the two most common non-muscular symptoms in 80% to 90% of those patients. So, they come to medical attention, and eventually getting to a neurologist. And then the diagnosis is confirmed, as I said, through genetic testing. Defect basically on chromosome 19, of a certain protein. Then with regards to over time - with regards to any disease state as it becomes more recognized, onset is usually symptoms late adolescence into early-20s. But then, it is a bit of a long journey to diagnosis because there are no approved treatment options for myotonic dystrophy. There are other sponsors working on new treatments. And as we step into this arena, and bring more attention and awareness, I think over time, diagnosis rates will increase. So does that answer your question, Chris for myotonic dystrophy?

Yeah, it does. If I might just maybe ask a quick follow up to that is that, I noticed that at least for many genetic abnormalities, there's newborn screening. So is there any discussion about the potential for that genetic defect to be detected in such a manner?

Chris, it's a great question. It is included on some of the newborn screening panels. The latest epidemiologic data that we have actually comes from a large study from the New York State Department of Health and their newborn screening, which is - what we cited in terms of about 150,000 patients in the U.S. potentially with a genetic defect from myotonic dystrophy Type 1 or DM1, which is the most common form of myotonic dystrophy. So there is similar with other rare orphan disorders in terms of genetic screening. There is the opportunity to pick up the genetic defects much earlier in life. And then with greater awareness in the medical community, hopefully leading to earlier diagnosis.

Okay, all right. Very good.

So, turning to your question about pediatric narcolepsy and Bioprojet study with regards to a little more color. So first, let me say, we remain committed to patients with pediatric narcolepsy. And it was always our plan to wait for Bioprojet's pediatric narcolepsy trial to read out before beginning our own trial. So Bioprojet's trial is delayed. Basically they amended the protocol, as I mentioned in my earlier comments. They amended the protocol and increased the sample size and sort of to make the study, the more robust. And that led to a delay in in the readout of the trial. So, along with our partner Bioprojet, we made a strategic decision to wait for the Phase 3 trial to readout before initiating our own, which was our original plan. At this point, I think it makes the most sense to wait for the data before starting another trial, and let the data inform us going forward. And to provide us some optionality. We feel that's a prudent approach and thoughtful. And we're working with our partner Bioprojet to that end. So we remain committed to the pediatric population obtaining on that indication. And we'll provide an update in the coming months on that program.

Okay. And so at this point, it's difficult to say when those data may become available. And that'll be the expected update maybe?

That'll be the expected update in the coming months. Yes.

Got it. Okay. All right. Well, thank you very much, Jeff. Appreciate it.

Thanks, Chris.

Thank you. Our last question comes from the line of Graig Suvannavejh with Goldman Sachs. Your line is now open.

Hey, good morning, everyone. And thanks for taking my questions and nice progress with the commercial launch. I had a couple of questions, and thanks for the granularity on some of the details in your slide deck. But I could just ask on the metrics you provide on the average number of WAKIX patients of 2800. I think that compares to about 2500 from the last quarter. And then your unique prescribers of 2700 compares with about 2400. So just trying to see if I get your perspective on what you think, should be a good rate going forward on each of those metrics. And then if I could ask a follow up question with regards to the Patient Assistance Program, it's been a focus for some over the past several quarters. And given where we are with the recovery or reopening or unemployment. I was just hoping if you could provide some color on the latest update on how you think that dynamic is evolving as a relates to WAKIX sales? And perhaps getting some sales back that you had perhaps previously lost? Thank you.

Thank you for those questions, Greg. Jeff Dierks, why don't you handle those two for Greg?

Sure. Good morning, Greg. Great to hear from you. So with respect to your first question on average number of patients and unique number of healthcare professionals prescribing since launch. So clearly, we're extremely pleased with our continued strong performance in Q1. As we've stated, it was a fifth consecutive quarter of growth in average number of patients in net revenue and number of unique prescribers. And in Q1, we saw a really strong underlying demand, resulting in about 9% increase in the average number of patients quarter-over-quarter. You've noted about an 11% increase in the average number of prescribers quarter-over-quarter. And that growth is seeing even with the key one seasonal pair dynamics and the lingering pandemic. And we do expect to continue to grow the average number of patients on WAKIX and the number of unique healthcare professionals each quarter during 2021. And the research that we recently feel that really shows strong outlook for future growth for WAKIX. So the research demonstrated that physicians believe that WAKIX is an appropriate treatment option for the vast majority of patients. Physician shared that WAKIX was extremely strong for EDS. 95% of physicians stated it was effective for EDS. 90% stated WAKIX was effective for cataplexy. And 90% of the healthcare professionals expect to prescribe the same or increase their use of WAKIX in more than narcolepsy patients in the future. So I know we're not providing guidance, but we believe that we are going to expect to grow average number of patients and unique healthcare professionals each quarter in '21. So hopefully that helps answer your first question just with respect to growth moving forward. And then second question you asked about Patient Assistance Program. And sort of what have we seen related to the reopening and a little bit broader COVID vaccination. So, as we shared previously, throughout the pandemic we have seen an increased demand for our Patient Assistance Program. And it's been relatively consistent. And that's even until into the first quarter of 2021. It's difficult to speculate when there will be less reliance on patient assistance as the country continues to open up. We certainly do have a strong conviction to support eligible patients through this program, knowing the difference that WAKIX can make in these patients lives. And importantly, as you've seen we've been able to demonstrate strong growth in net revenues for five consecutive quarters, even with this elevated demand. And we expect to continue to grow our net revenue and average number of patients throughout '21.

Okay, thanks. If I could just follow up on an answer you gave previously. When we think about the prescribers that you've been able to target. As it relates to 2021, in terms of your ability to do face-to-face interactions with those prescribers from the 2700 that you have now. Is there a number we should be thinking about for as a goal for calendar 2021? I know you really don't want to give guidance. But I'm just trying to figure out in terms of what that penetration might look like, of the 8000 patients who treat narcolepsy?

No. I appreciate the question, Greg. And our expectation is that we will continue to grow the number of unique healthcare professionals. Our 2700 unique healthcare professionals represent about 33% of the overall target universe. And so we believe that there's significant opportunity to continue to penetrate that group. We've seen the receptivity to the broad clinical utility and the overall benefit risk profile of WAKIX. We have seen with COVID, there are more healthcare professionals' offices that are opening up and seeing patients. We are starting to see more of our Field Sales Team, make more in-person sales calls. And you certainly know the benefit of in person education, especially in a launch. So while we're not providing guidance. We do expect to grow the average number and the number of unique healthcare professionals quarter-over-quarter throughout 2021.

Yeah, Greg, it's John. We're optimistic about the opportunity as COVID lifts as vaccinations continue throughout the United States. And we have yet to really - I think get a feel for the true run-rate. As COVID starts to lift throughout the rest of this year, we'll get a much better feeling for the true-run rate. But I think what you've seen here hopefully today and heard from us today is very strong underlying demand. We're getting excellent feedback from physicians and patients on their experience with our products. And there remains tremendous unmet need in the space. Whether there is a chronic disease, these patients are living with this disease before during and they will be after COVID. And we're here to help them. And that demand isn't going anywhere. So as we get into the next couple of quarters, I think we'll be excited to share with you the way we're able to penetrate the marketplace as COVID starts to lift.

Okay, thank you very much.

Thank you. There are no further questions. I will now turn the call back to John Jacobs for closing remarks.

Thanks again for joining us today. I hope that you can appreciate that Harmony has continued our strong launch momentum for WAKIX. And that we are poised well to execute on our three pillars of growth strategy, which should enable us to grow our business and shareholder value as we continue on our mission to help patients who are living with orphan rare neurological disorders. Thank you.

Ladies and gentlemen, this concludes today's conference call. Thank you for your participation. You may now disconnect.