Cuộc họp Báo cáo Kết quả Kinh doanh Quý 3/2026 của Harmony Biosciences (HRMY): Doanh thu WAKIX Đạt 269 Triệu USD

Điểm tin chính

• Doanh thu thuần của WAKIX tăng 12,6% so với cùng kỳ năm ngoái lên 269 triệu USD, nhờ vào số lượng bệnh nhân trung bình khoảng 9.350 người và có thêm 400 bệnh nhân mới ròng trong quý.
• Harmony Biosciences tái khẳng định dự báo doanh thu thuần của WAKIX cho năm 2026 là từ 1,00 tỷ USD đến 1,04 tỷ USD, giúp sản phẩm đi đúng hướng để vượt mốc 1 tỷ USD lần đầu tiên.
• Lợi nhuận thuần giảm xuống còn 48 triệu USD, tương đương 0,81 USD trên mỗi cổ phiếu pha loãng, do chi phí bản quyền mới từ Novitium và khoản phí cột mốc 25 triệu USD từ MSN Labs làm tăng chi phí.
• Dữ liệu sơ bộ về liều tăng dần nhiều lần trong Giai đoạn I cho thấy BP-205 nhìn chung được dung nạp tốt sau 14 ngày dùng liều một lần mỗi ngày. Theo ban quản lý, thời gian bán thải hiệu dụng khoảng 29 giờ của thuốc hỗ trợ tiềm năng sử dụng một lần mỗi ngày.
• Harmony kết thúc quý 3 năm 2026 với hơn 1 tỷ USD tiền mặt, các khoản tương đương tiền và khoản đầu tư, cùng với 150 triệu USD nợ. Ban quản lý dự định tài trợ cho hoạt động R&D và theo đuổi nhiều giao dịch phát triển kinh doanh.
• Jeffrey Dayno sẽ từ chức CEO vào cuối năm. Peter Anastasiou sẽ trở thành Chủ tịch kiêm CEO vào ngày 1 tháng 1 năm 2027 và công ty không có kế hoạch thay đổi các ưu tiên chiến lược.

Kết quả tài chính cốt lõi

Kết quả hoạt động và kinh doanh

WAKIX đã có thêm 400 bệnh nhân ròng trong quý 3, nâng số lượng bệnh nhân trung bình hàng quý lên khoảng 9.350 người. Harmony cho biết sản phẩm này đã ghi nhận ít nhất 400 bệnh nhân mới ròng trong 5 trên 6 quý gần nhất, cho thấy sự tăng trưởng mạnh mẽ hơn trong năm thứ bảy có mặt trên thị trường.

Ban quản lý cho biết nhu cầu tăng là nhờ vị thế của WAKIX như một liệu pháp điều trị không thuộc danh mục kiểm soát đặc biệt đối với tình trạng buồn ngủ quá mức vào ban ngày và đột ngụy chứng (cataplexy) ở bệnh nhân ngủ róc người lớn và trẻ em, cùng với tỷ lệ chi trả từ bên thứ ba đạt hơn 80%. Việc mở rộng các đội ngũ bán hàng, hoàn chi phí và tiếp cận bệnh nhân cũng làm tăng sự gắn kết với các nhà cung cấp dịch vụ y tế và cải thiện việc thực hiện đơn thuốc.

Công ty dự kiến ORZEYFUL sẽ sớm được ra mắt và lưu ý rằng sản phẩm này sẽ chỉ được chỉ định cho bệnh chứng ngủ róc loại 1. Ban quản lý tin rằng việc WAKIX được áp dụng rộng rãi hơn đối với chứng ngủ róc loại 1 và loại 2, ở cả bệnh nhân người lớn lẫn trẻ em, cũng như trong liệu pháp phối hợp, sẽ hỗ trợ tăng trưởng liên tục cho đến hết năm 2027.

Pitolisant GR có ngày PDUFA của FDA là ngày 1 tháng 4 năm 2027 và tiếp tục đi đúng hướng để ra mắt trong nửa đầu năm 2027. Công thức này được thiết kế để cho phép khởi đầu điều trị ở liều điều trị 17,8 miligam mà không cần chỉnh liều.

Đối với pitolisant HD, các nghiên cứu Giai đoạn III ONSTRIDE 1 về chứng ngủ róc và ONSTRIDE 2 về chứng ngủ nhiều vô căn vẫn đang được tiến hành, với dữ liệu sơ bộ dự kiến vào năm 2027 và ngày PDUFA dự kiến vào năm 2028. Nghiên cứu Giai đoạn III TEMPO của WAKIX đối với hội chứng Prader-Willi dự kiến sẽ báo cáo dữ liệu sơ bộ vào giữa năm 2027.

BP-205 và chuỗi sản phẩm lâm sàng

Kết quả sơ bộ về liều tăng dần nhiều lần của BP-205 được thực hiện trên 94 tình nguyện viên khỏe mạnh dùng liều một lần mỗi ngày ở mức 0,1; 0,6; 1 hoặc 3 miligam trong 14 ngày. Thời gian trung bình đạt nồng độ tối đa là khoảng 60 phút, trong khi thời gian bán thải hiệu dụng trung bình là khoảng 29 giờ.

BP-205 nhìn chung được dung nạp tốt, không có biến cố bất lợi nghiêm trọng hoặc nặng phát sinh trong quá trình điều trị. Không có báo cáo nào về biến cố bất lợi về gan hoặc thị giác, các biến cố tim mạch có ý nghĩa lâm sàng hoặc bất thường về điện tâm đồ.

Đau đầu xảy ra ở khoảng 25% số người tham gia. Mất ngủ được báo cáo ở dưới 20% đối với tất cả các liều được đánh giá, trong khi đi tiểu nhiều lần và tiểu gấp xảy ra ở dưới 20% và chỉ xuất hiện ở các liều 1 và 3 miligam. Các biến cố này ở mức độ nhẹ và không làm cho ai ngừng tham gia thử nghiệm.

Ban quản lý cho biết các phát hiện này hỗ trợ tiềm năng dùng liều một lần mỗi ngày của BP-205 nhưng thừa nhận rằng hiệu quả có ý nghĩa lâm sàng vẫn chưa được xác lập. Một nghiên cứu trên các tình nguyện viên khỏe mạnh bị thiếu ngủ dự kiến sẽ cung cấp tín hiệu hiệu quả đầu tiên vào quý 1 năm 2027 và giúp xác định khoảng liều lượng cho Giai đoạn II. Nhiều nghiên cứu Giai đoạn II trên các chỉ định về thần kinh trung ương (CNS) đang được lên kế hoạch vào giữa năm 2027.

Harmony cũng đang tiến hành các nghiên cứu Giai đoạn III cho EPX100 trong hội chứng Lennox-Gastaut và hội chứng Dravet. Dữ liệu sơ bộ từ cả hai chương trình dự kiến sẽ có vào giữa năm 2027, với các ngày PDUFA dự kiến vào năm 2028.

Dự báo của Ban quản lý

Harmony tái khẳng định dự báo doanh thu thuần cả năm 2026 của WAKIX là 1,00 tỷ USD đến 1,04 tỷ USD. Ban quản lý cho biết đà tăng trưởng số lượng bệnh nhân tiếp tục kéo dài sang quý 4 và dự kiến sản phẩm này sẽ vượt mốc 1 tỷ USD doanh thu thuần hàng năm lần đầu tiên.

Công ty dự kiến các hoạt động vận hành sẽ tiếp tục tạo ra dòng tiền. Các ưu tiên phân bổ vốn bao gồm phát triển BP-205, các cột mốc trong hợp đồng phát triển sản phẩm và phát triển kinh doanh.

Ban quản lý đang hướng tới hai nhóm giao dịch: các tài sản thương mại hoặc giai đoạn muộn có khả năng đóng góp doanh thu trong khoảng thời gian từ năm 2028 đến năm 2032, và các tài sản đổi mới sáng tạo ở giai đoạn sớm hơn mang lại sự tăng trưởng dài hạn hơn. Các lĩnh vực được quan tâm bao gồm rối loạn giấc ngủ, động kinh, các bệnh hiếm về thần kinh trung ương và các chỉ định rộng hơn về thần kinh trung ương. Công ty dự kiến sẽ theo đuổi nhiều giao dịch thay vì triển khai toàn bộ nguồn vốn hiện có vào một thương vụ duy nhất.

Rủi ro và các điểm cần theo dõi

Giá vốn hàng bán của WAKIX đã tăng lên 30% doanh thu do phí bản quyền mới từ Novitium. Ban quản lý cho biết quý 3 đã phản ánh đầy đủ tác động của phí bản quyền này và chỉ ra rằng cấu trúc chi phí tương tự dự kiến sẽ tiếp diễn trong thời gian tới.

Chiến lược độc quyền WAKIX của Harmony vẫn đang trong quá trình tranh tụng bằng sáng chế tích cực. Công ty đã đạt được thỏa thuận giải quyết với 6 trong số 7 bên nộp đơn đăng ký thuốc mới rút gọn (ANDA) và tiếp tục tự tin giữ vững quyền độc quyền cho đến tháng 3 năm 2030, bao gồm cả 6 tháng độc quyền dành cho chỉ định nhi khoa.

Quyết định trong vụ tranh tụng AET dự kiến sẽ có vào khoảng thời gian hết hạn tạm hoãn 30 tháng vào tháng 2 năm 2027. Phiên phân xử lệnh cấm sơ bộ liên quan đến bằng sáng chế pitolisant vô định hình được ấn định vào ngày 26 tháng 1 năm 2027, với quyết định dự kiến đưa ra trước ngày 14 tháng 2. Ban quản lý cho biết phiên tòa riêng về bằng sáng chế dạng vô định hình có thể được lên lịch vào năm 2029.

Kết quả của BP-205 vẫn chỉ là sơ bộ và thu được từ các tình nguyện viên khỏe mạnh. Ban quản lý cho biết khả năng dung nạp ở bệnh nhân ngủ róc loại 1 bị thiếu hụt orexin vẫn chưa chắc chắn và phải được đánh giá trên lâm sàng. Mối quan hệ giữa mức độ phơi nhiễm và biến cố bất lợi cũng chưa được công bố đầy đủ.

Những điểm nổi bật trong phần Q&A với chuyên gia phân tích

• Liều lượng BP-205: Ban quản lý cho biết liều lặp lại 3 miligam đã tạo ra mức phơi nhiễm đủ dựa trên mô hình của họ. Các biến cố bất lợi do gắn kết mục tiêu được ghi nhận ở liều thấp tới 0,1 miligam, hỗ trợ bằng chứng cho thấy thuốc vượt qua hàng rào máu não và gắn kết với thụ thể orexin-2.
• Tiềm năng dùng 1 lần/ngày: Theo ban quản lý, thời gian bán thải hiệu dụng khoảng 29 giờ, hiệu lực và sự gắn kết mục tiêu hỗ trợ chế độ liều dùng một lần mỗi ngày. Nghiên cứu về tình trạng thiếu ngủ sẽ đánh giá xem liệu sự tỉnh táo có thể duy trì đến cuối chiều hoặc đầu buổi tối hay không.
• Cân bằng độ an toàn: Harmony không kỳ vọng triệt tiêu toàn bộ các biến cố bất lợi liên quan đến orexin. Mục tiêu của công ty là xác định các mức liều mang lại hiệu quả có ý nghĩa lâm sàng trong khi giữ cho tác dụng phụ gây mất ngủ và tiểu tiện ở mức nhẹ và kiểm soát được.
• Sự cạnh tranh đối với WAKIX: Ban quản lý kỳ vọng nhãn chỉ định rộng rãi, trạng thái không thuộc danh mục kiểm soát đặc biệt và phạm vi chi trả bảo hiểm đã được xác lập của WAKIX sẽ hỗ trợ đà tăng trưởng tiếp tục sau khi ORZEYFUL ra mắt.
• Bảo vệ bằng sáng chế: Harmony cho biết việc yêu cầu lệnh cấm sơ bộ không nên được hiểu là sự giảm sút niềm tin vào vụ án ANDA đang chờ xử lý. Công ty tin tưởng có thể giành chiến thắng dựa trên các yêu cầu bảo hộ bằng sáng chế dạng đa hình, phương pháp sử dụng hoặc dạng vô định hình.
• Pitolisant vô định hình: Công ty có kế hoạch nghiên cứu các chỉ định tâm thần-thần kinh rộng hơn, bao gồm các triệu chứng liên quan đến mệt mỏi, cũng như các công thức và phương pháp đưa thuốc vào cơ thể thay thế.

Toàn bộ biên bản cuộc họp kết quả kinh doanh

Toàn văn cuộc gọi công bố kết quả kinh doanh

Phần trình bày của ban lãnh đạo

Operator

Good morning. My name is Nikki, and I will be your conference operator today. At this time, I would like to welcome everyone to Harmony Biosciences Third Quarter Financial Results Conference Call. [Operator Instructions] Please be advised that today's conference is being recorded. [Operator Instructions]

I will now turn the call over to Brennan Doyle, Head of Investor Relations. Please go ahead.

Brennan Doyle

Thank you, operator. Good morning, everyone, and thank you for joining us today as we review Harmony Biosciences third quarter 2026 financial results and provide a business update.

Before we start, I encourage everyone to go to the Investors section of our website to find the materials that accompany today's discussion. Our speakers on today's call are Dr. Jeffrey Dayno, President and CEO; Peter Anastasiou, Senior Executive Vice President, Chief Operating Officer and Incoming President and CEO; Adam Zaeske, Chief Commercial Officer; Dr. Kumar Budur, Chief Medical and Scientific Officer; Steve Mollichella, Interim Principal Financial Officer.

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 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.

I would now like to turn the call over to our CEO, Dr. Jeffrey Dayno. Jeff?

Jeffrey Dayno

Thank you, Brennan. Good morning, everyone, and thank you for joining our call today. Before I get into the quarter, I first want to say a few words about the CEO transition we announced last month.

I will be stepping down at the end of the year, and Peter Anastasiou, our Senior Executive Vice President and Chief Operating Officer, will succeed me as President and Chief Executive Officer, effective January 1, 2027. I am confident that Harmony is in very good hands with Peter, and you will hear from him next.

The main themes regarding the CEO transition are continuity, strength and growth. This leadership transition does not signal any change in our overall business or our strategic priorities.

We are doing this from a position of strength and executing a well-coordinated plan. Harmony continues to be a growth story. And during Q3, we moved that story forward in each of our 4 priorities of value creation: continue to grow WAKIX in an evolving market; advancing a novel and robust clinical stage pipeline; transact on strategic business development deals; and protect the pitolisant franchise.

I will provide some more color on our first 2 strategic priorities of value creation and then turn it over to Peter to expand on our other 2 strategic priorities.

The key highlights that I want you to take away from today's call are sustained patient demand-driven growth for WAKIX in its seventh year on the market, keeping us firmly on track to exceed $1 billion in net revenue this year and preliminary multiple ascending dose data for our orexin-2 agonist, BP-205, that further strengthened its emerging best-in-class profile.

First, growing the WAKIX franchise. In the third quarter, WAKIX delivered $269 million in net revenue with 400 net patient adds, resulting in approximately 9,350 average patients on WAKIX in the quarter. For the first time in company history, we expect to exceed $1 billion in revenue for WAKIX this year.

We want to recognize our commercial team for their executional excellence, and Adam will take you through our commercial performance in more detail later in the call.

Our second strategic priority is advancing our novel clinical stage pipeline. The strength and durability of the pitolisant franchise gives us the flexibility to fund our pipeline, creating new growth drivers for the company. The centerpiece of our pipeline is BP-205, and we continue to have firm conviction in its best-in-class potential.

Today, we announced preliminary results from the multiple ascending dose portion of our Phase I study for BP-205. After 14 days of once-daily dosing, the safety, tolerability and PK profile was consistent with what we saw in the single ascending dose study, giving us confidence in the emerging best-in-class profile of BP-205.

We initiated a study in sleep-deprived healthy volunteers in the third quarter with data expected in the first quarter of 2027. And Phase II studies across multiple CNS indications are planned to initiate in mid-2027.

There is a clear line of sight to the multiple data catalysts for BP-205, and they are right around the corner. Kumar will provide more color on BP-205 and the rest of our pipeline programs during his R&D update.

Peter will now walk you through our 2 other value creation priorities related to business development and protecting the pitolisant franchise. Peter?

Peter Anastasiou

Thank you, Jeff, and good morning, everyone. First, I want to thank Jeff for his leadership and for everything he has done for Harmony over the last 9 years. WAKIX is on track to exceed $1 billion in net revenues this year.

We have a novel pipeline led by BP-205, and we have a strong balance sheet to fund our business development efforts. That is the foundation I get to build on, and I am honored to become Harmony's next CEO.

Our ambition is to build Harmony into an enduring leading CNS company with multiple products across multiple franchises, and our strategic priorities will enable us to achieve this ambition.

You heard Jeff speak to our first 2 strategic priorities regarding our strength in the WAKIX business and the advancement of our pipeline. Our third strategic priority is transacting on business development.

With over $1 billion on our balance sheet, we have both the capacity and the conviction to execute on meaningful transactions. We are pursuing assets that can add near-term revenues to support our self-funded model and assets that can continue to drive long-term value creation.

Our fourth strategic priority is protecting the pitolisant franchise. We have settled with 6 of the 7 ANDA filers, and our multilayered IP strategy across active legal proceedings reinforces our confidence in WAKIX exclusivity to March 2030, inclusive of 6 months of pediatric exclusivity.

In summary, Harmony is a growth story, and we are executing on all 4 strategic priorities. To tell you more about our first strategic priority of growing the WAKIX franchise, I'll turn it over to our Chief Commercial Officer, Adam Zaeske. Adam?

Adam Zaeske

Thank you, Peter, and good morning, everyone. We continue to see extremely strong demand-driven growth for WAKIX this quarter. WAKIX achieved estimated average patients of 9,350, an increase of 400 patients in the quarter.

WAKIX has now demonstrated a meaningful acceleration in growth in year 7 on the market with 400 net patient additions in 5 of the last 6 quarters. Prior to 2025, WAKIX had achieved 400 net patient additions only twice in the history of the brand.

As a result, WAKIX delivered $269 million in net product revenue, which was right in line with our internal expectations. And we remain on track to achieve our full year guidance of $1 billion to $1.04 billion.

The drivers of this growth and performance are clear. WAKIX holds a unique position as the only non-scheduled treatment option for excessive daytime sleepiness and cataplexy in adults and pediatric patients with narcolepsy. With more than 7 years of clinical experience, health care providers are highly aware of WAKIX and believe in its unique combination of efficacy, safety and tolerability with low drug-drug interactions as well as its broad payer coverage with more than 80% of lives covered.

This makes WAKIX a familiar go-to option for any adult and pediatric patients with narcolepsy type 1 and type 2 and in any combination with other therapies in a highly polypharmacy market. And this will continue to remain true as the market continues to evolve, including with the introduction of orexin.

We executed the expansion of our field sales, remote sales, field reimbursement and patient outreach teams in the second quarter and are seeing higher levels of HCP engagement, fueling a strengthening of top line demand. We also implemented changes to our reimbursement and patient support programs, which are decreasing the time required and increasing the success rate of patients filling prescriptions.

In addition, significant growth opportunity remains. Approximately 170,000 people in the U.S. are living with narcolepsy, fewer than 50% are diagnosed and brand penetration is approximately 20%.

Looking ahead, we're excited about our 2 life cycle programs to extend and expand the franchise. Pitolisant GR is on track to launch in the first half of 2027 with a PDUFA date of April 1, 2027, and build on the WAKIX safety and tolerability profile while enabling patients to start at a therapeutic dose.

Pitolisant HD offers an opportunity to extend the pitolisant franchise with differentiated labeling for fatigue in narcolepsy and sleep inertia in IH with up to 2x the highest label dose of WAKIX and all the benefits of the GR formulation.

Finally, our orexin program gives us the opportunity to deliver on the promise of efficacy with a favorable safety and tolerability profile, once-a-day dosing and potential use across NT1, NT2 and IH in addition to a broad range of CNS indications.

To summarize, WAKIX performance has never been stronger. We are on track to achieve more than $1 billion in net sales this year, and our pipeline is positioned to extend our leadership in sleep-wake for at least the next decade.

I'll now turn the call over to our Chief Medical and Scientific Officer, Dr. Kumar Budur, to discuss the advancements in our clinical development programs. Kumar?

Kumar Budur

Thank you, Adam. Good morning, everyone, and thank you for joining us today. We continue to make good progress across our robust clinical stage pipeline, including 5 Phase III registrational studies in 5 distinct rare neurological disorders and BP-205, our potential best-in-class orexin-2 receptor agonist.

The headline for R&D this quarter is again BP-205. So let me start there. BP-205 is discovered by Teijin Pharma and developed in partnership with Bioprojet. It is designed to deliver a differentiated profile with high potency, excellent selectivity, emerging strong PK, and favorable safety tolerability, all supportive of a best-in-class profile.

BP-205 is built on a unique chemical scaffold and has the highest potency among known orexin agonists in clinical development and has demonstrated excellent selectivity for orexin-2 receptors over orexin-1 receptor and over 150 other receptors of interest.

As a reminder, we reported top line data from the single ascending dose or SAD portion of the study in August. Today, we announced preliminary results from the multiple ascending dose or MAD portion of the Phase I study. The multiple ascending dose study was a 14-day randomized double-blind placebo-controlled study evaluating once daily doses of 0.1, 0.6, 1 and 3 milligrams across 6 cohorts, 4 male and 2 female of healthy volunteers.

A total of 94 healthy volunteers participated. The preliminary data shows that the PK profile is consistent with what was expected based on the preclinical and the SAD data. The mean Tmax was approximately 60 minutes with a range of 32 to 90 minutes pointing to the potential for rapid onset of efficacy.

The mean effective half-life was approximately 29 hours, not meaningfully different from the SAD study and it supports once daily dosing and the potential for durable efficacy throughout the day and potentially with no troublesome insomnia as evidenced by the tolerability profile of BP-205 from the SAD and the MAD data.

Exposure was dose-related and we saw target engagement adverse events, which further highlights its potential to deliver meaningful efficacy at low clinical doses. The most common adverse event was headaches, observing approximately 25% of participants.

On-target adverse events of insomnia were reported in less than 20% of participants at all doses evaluated. These events were mild in severity and did not lead in any discontinuations.

On target adverse events of urinary frequency and urgency were reported in less than 20% of participants and only at the 1 milligram and 3 milligram doses. These events were also mild in severity and did not lead to any discontinuations.

BP-205 was generally well tolerated with no serious or severe treatment emergent adverse events. In addition, no liver or visual adverse events were reported and no clinically significant cardiovascular events or EKG abnormalities were recorded.

This data strengthens our conviction of BP-205's emerging best-in-class profile and will inform our dosing strategy as we accelerate the development program. The complete MAD data sets will be presented at an upcoming medical meeting.

We initiated the Phase volunteer in the U.S. in the third quarter with top line data expected in the first quarter of 2027. That study is the one to watch because it will provide the first signals of efficacy for BP-205.

Reflecting our commitment to investing in a robust development program for BP-205, we expect to initiate Phase II studies across multiple CNS indications by mid-2027.

Of the portfolio, we have highlighted the potential synergistic effects of orexin and [indiscernible] mechanisms of action based on the neurocircuitry and orexin neurons resulting in downstream effects on various neurotransmitters, including histamine.

At the recently concluded Sleep EU 2026 meeting, Bioprojet presented a poster showing an in vivo synergistic effect of orexin and histamine on wakefulness in a narcoleptic mouse model.

Specifically, pitolisant added to BP-205 drove maximum histamine release for the BP-205 of pitolisant alone and this translated into increased duration of wakefulness in this model. The relevance of this data is that combining an orexin-2 agonist with a treatment that works through histamine could be a new therapeutic approach for patients with narcolepsy and other central disorders of hypersomnolence.

Of note WAKIX along with pitolisant GR and HD are the only agents that work through histamine that is either approved or in late-stage clinical development. Speaking of pitolisant, starting with pitolisant GR, we are on track to launch in the first half of 2027.

The NDA was accepted by the FDA with a PDUFA date of April 1, 2027. Pitolisant GR has enteric coating, enables patients to initiate treatment at a therapeutic dose of 17.8 milligrams without titration, an important clinical differentiation.

Pitolisant HD is our key value driver to extend the pitolisant franchise with differentiated labeling. It's an enhanced formulation of pitolisant with an optimized PK profile, enteric coating and a higher dose to drive greater efficacy.

Our Phase III registrational clinical trials ONSTRIDE 1 in narcolepsy and ONSTRIDE 2 in idiopathic hypersomnia are ongoing with top line data expected in 2027 and anticipated PDUFA date in 2028. Utility patents are filed for both pitolisant GR and pitolisant HD to extend the pitolisant franchise into the 2040s.

For WAKIX, the Phase III TEMPO study in Prader-Willi syndrome is actively recruiting with top line data expected in mid-2027 and an anticipated PDUFA date in 2028. We remain on track for pediatric exclusivity.

Completion of the TEMPO study fulfills the second and last requirement agreed with the FDA for WAKIX pediatric exclusivity, which will provide an additional 6 months of regulatory exclusivity for WAKIX. We are also exploring the amorphous form of pitolisant to pursue broader CNS indication.

This opportunity is based on our exclusive license to Novitium's issued amorphous form of pitolisant patent with protection until 2042. Current efforts are focused on formulation optimization and new modes of delivery.

Finally, our Epilepsy franchise. EPX100 is actively enrolling patients in 2 global Phase III registrational trials in rare epilepsies, the Lighthouse study in Lennox-Gastaut syndrome and the ARGUS study in Dravet syndrome. The top line data are expected in mid-2027 and anticipated PDUFA date in 2028.

In December, we will present updated 18-month data from the open-label extension of the ARGUS study at the American Epilepsy Society Annual Meeting.

In summary, the MAD data further strengthened the emerging profile of BP-205 as a potential best-in-class orexin-2 agonist and our next data readout from the sleep-deprived healthy volunteer study comes in the first quarter of 2027. We also continue to make progress on the 5 Phase III registrational clinical trials across our other pipeline programs, which will contribute to additional data catalysts in 2027 and target PDUFA date in 2028.

On behalf of Harmony, I want to thank the patients and their families for participating in our clinical trials, along with the investigators and the site personnel for their dedication in advancing our clinical trials. I'll now turn the call back over to Peter. Peter?

Peter Anastasiou

Thank you, Kumar. I'll now provide further color on our final 2 strategic priorities of value creation, transacting on business development and protecting the pitolisant franchise. Let me start with business development.

We are pursuing 2 types of opportunities. The first is assets that can add near-term revenue in the 2028 to 2032 time frame to support our self-funded model. For this category, we are prioritizing assets in Phase III in registration or on the market.

The second category is innovative pipeline assets that drive long-term value creation, the way we believe BP-205 can. Our therapeutic areas of interest include sleep disorders, epilepsy, rare orphan CNS and broader CNS indications.

We see the opportunity to broaden our reach beyond orphan rare into larger CNS patient populations through both our orexin strategy and business development efforts. We are directing our search and evaluation efforts accordingly, and we are considering a variety of deal types, including M&A and licensing.

We have a strong balance sheet with over $1 billion. We will be good stewards of capital, seeking value-creating opportunities. You should not expect that we will allocate all of that capital on one transaction.

Instead, we will likely engage in multiple transactions. Together, they can be transformative for Harmony and drive long-term value creation. With regard to our fourth strategic priority of protecting the pitolisant franchise, our IP estate is multilayered across formulations, methods of use and next-generation applications. It supports WAKIX exclusivity to March 2030, inclusive of 6 months of pediatric exclusivity.

There is potential protection of the franchise through other formulations into the 2040s via additional patents and applications. We have settled with 6 of the 7 ANDA filers. Harmony is asserting its pitolisant IP in active legal proceedings.

First, the Polymorph 197 and method of use 947 patents. The bench trial in the AET ANDA litigation has concluded and closing arguments were held on October 22. Harmony believes AET's proposed product infringes the 197-polymorph patent and that the 947 patent is valid. We expect a ruling from the judge around the time of the 30-month stay expiring in February 2027.

Second, the amorphous Form 920 patent. Harmony and Novitium filed a lawsuit alleging patent infringement in April against AET Pharma US and its marketing partner, Sandoz. The licensed Novitium patent provides protection to 2042.

A scheduling conference was held on October 21. The amorphous 920 patent trial has not yet been scheduled, but would be expected to be scheduled for some time in 2029. Yesterday, November 3, the judge scheduled a preliminary injunction hearing on January 26, 2027, with an expected decision by February 14, 2027, when the 30-month stay expires. This schedule is identical to the preliminary injunction schedule Harmony proposed to the judge on October 26.

In Q1 this year, we obtained the exclusive rights to a patent covering methods of making amorphous pitolisant hydrochloride from our licensee MSN Labs. That patent was issued by the PTO in Q3.

The patent strengthens our patent portfolio and protects our development efforts for a new amorphous pitolisant hydrochloride product. Together with our settlements with the 6 other ANDA filers, these actions reinforce our confidence in WAKIX exclusivity to March 2030.

I will now turn the call over to our Interim Principal Financial Officer, Steve Mollichella, to walk through our financial results for the quarter. Steve?

Stephen Mollichella

Thank you, Peter, and good morning, everyone. This morning, we issued our third quarter 2026 earnings release and filed our 10-Q, where you can find details of our financial and operating results. We continue to deliver financial results that reflect strong underlying demand for WAKIX as well as our disciplined approach to managing expenses across the business.

For the third quarter of 2026, we reported net revenue of $269 million compared to $239 million in the prior year quarter. Our performance this quarter reflects continued strong demand for WAKIX within the large narcolepsy market, coupled with typical quarter-to-quarter buying patterns that the industry as a whole experiences.

Cost of products sold was 30% of net revenue compared to 25% one year ago. This year-over-year increase was primarily driven by a new royalty related to the Novitium license agreement, which we signed in Q1 of this year.

We reported total operating expenses of $134 million for the third quarter compared to $114 million in the prior year quarter. The increase in expenses was primarily driven by a $25 million charge related to a milestone achieved by MSN Labs, continued investments in R&D and the expansion of our field-based team, which we completed earlier this year. These increases were partially offset by a $15 million charge related to a clinical milestone for ZYN002 in the prior year quarter.

Net income for the third quarter was $48 million or $0.81 per diluted share compared to $51 million or $0.87 per diluted share for the prior year period. The year-over-year decline in net income was primarily due to the Novitium royalty and the MSN milestone charge I mentioned earlier.

We ended the third quarter with over $1 billion of cash, cash equivalents and investments and $150 million in debt. Looking forward, we continue to expect our operations to generate cash, maintaining our strong balance sheet.

Our capital allocation priorities include investment in R&D, driven by our BP-205 development plans, along with potential business development opportunities and contractual milestones, which may be triggered by continued success in our pipeline. Finally, we are reiterating our full year guidance of $1 billion to $1.04 billion for WAKIX net revenue.

And with that, I will turn the call back to Jeff for his closing remarks. Jeff?

Jeffrey Dayno

Thanks, Steve. In closing, this was another productive quarter for Harmony centered around 2 key accomplishments: sustained patient demand-driven growth for WAKIX that keeps us firmly on track to exceed $1 billion in net revenue this year and preliminary MAD data that further strengthened the emerging best-in-class profile of our orexin-2 agonist BP-205.

We have been leaders in the sleep disorder space for almost a decade now and are leveraging our expertise to accelerate our BP-205 development program for multiple CNS indications to become a leader in the orexin space. We believe that when we execute on our 4 strategic priorities of value creation, Harmony is well positioned to become an enduring leading CNS company with multiple products across multiple franchises, bringing innovative treatments to patients living with CNS diseases while driving sustained long-term value for our shareholders.

I would like to end this call today on a personal note before I step down as CEO at the end of the year. It has been my honor and privilege to have been a part of building Harmony Biosciences from the beginning over the past 9 years and leading the organization as the CEO these past 4 years.

I am grateful to our Board of Directors for the confidence they had in me and the opportunity to have served in the CEO role as well as staying on as an adviser to the company throughout 2027. I want to thank the analysts I have worked with and members of the investment community for your interest in what we have been building at Harmony and recognizing the value of the enterprise that we are advancing.

Lastly, I want to thank the entire Harmony organization for their dedication, commitment and their passion for why we do what we do. That is what I am most proud of because for an empathy and innovation need, a better life can begin for patients with unmet medical needs. I wish Peter all the best of success in the role of CEO, and I am confident in his leadership and the entire organization in driving Harmony's next chapter of growth.

Thanks, everyone. And I will now turn the call back over to the operator for Q&A. Operator?

Operator

[Operator Instructions] We'll take our first question from Ami Fadia with Needham.

Phần hỏi đáp

Ami Fadia

Firstly, Jeff, congrats on a fantastic career and it's been great working with you. So my first question is, with regards to the MAD data, while this was a safety study, can you give your preliminary thoughts on the therapeutic index across the doses studied based on target engagement?

And what are your thoughts around being able to explore a dose even higher than the range that you studied in the MAD study, because you went up to 6 milligrams in the SAD study? And then I have one more question.

Jeffrey Dayno

Okay, Kumar.

Kumar Budur

Thanks for the question. In terms of the therapeutic index, we do have a wide range of therapeutic index with BP-205. Regarding your question about limiting the dose to 3 milligram in the multiple ascending dose study, and in the SAD study we went all the way up to 6 milligram.

The repeat dose of 3 milligram at steady state equals something like around 6 milligram single ascending dose anyway. So in terms of could we have gone higher on the dose? Absolutely, yes, we could have. We limited the MAD at 3 milligram, starting at 0.1 milligram, because based on our preclinical PK/PD modeling and the modeling that we have from the single ascending dose study, at 3 milligram multiple ascending dose we will get more than enough exposure that we need based on what we find in the preclinical PK/PD relationship.

So we are actually quite happy with the range that we have studied. And more importantly, as we disclosed this morning, in terms of target engagement, we saw target engagement at a very low dose of 0.1 milligram where we saw insomnia, which is a class-related AE, and we saw other target engagement AEs, like urinary frequency and urgency at 1 and 3 milligram.

So what it says is in the dose range that we have evaluated, the drug crosses the blood-brain barrier, interacts with orexin-2 receptor agonist, and is doing what it is expected to do. So with that, these data support the potency of BP-205, which is highest compared to other orexin-2 receptor agonists in clinical development, and gives us an opportunity to explore low doses to treat all 3 central disorders of hypersomnolence.

Ami Fadia

One other quick question. The half-life obviously supports once-daily dosing. However, based on what we've seen from the other orexin programs, what are your thoughts around exploring a split dosing when you initiate the Phase II planned middle of next year?

Kumar Budur

Yes. A great question, Ami. In terms of what we have seen with the half-life and the target engagement AEs that we are seeing, we remain confident that once-a-day dosing is what's going to work with BP-205.

Jeffrey Dayno

Yes, I would add to that, I think that the data from the sleep-deprived healthy volunteer study, Ami, will inform that in terms of the ability to demonstrate sustained wakefulness throughout the day and until late day, early evening. So that data set will also inform the potential, what we believe could be once-daily dosing.

Kumar Budur

Yes. And there are also other factors other than half-life, like the ability to cross the blood-brain barrier, the potency, this activity, the features that we have talked about, especially that are very unique for BP-205.

Operator

We will move next with Graig Suvannavejh with Mizuho.

Graig Suvannavejh

Jeff, congrats on a great career. Look forward to connecting with you on whatever might be next for you. But that being said, I have 2 questions, if I could. One just on the existing business.

WAKIX seems to be in a good place, but I think we're within the month that Takeda's ORZEYFUL may be launching, and maybe this is a question for Adam, but how should we think about that launch and what the impact might be to WAKIX both in the very near term or perhaps in the midterm?

Just thinking about you're getting good traction on new patient adds, but just if you could give some other higher-level comments because we are pretty much here -- we've been talking about it for a while now.

And then second, if I could just revisit the patent litigation. It is very complex. I know you had mentioned that the amorphous patent litigation, the timing there would be perhaps a trial start in 2029, but if we get to know a final decision in the original ANDA-based litigation, which could come, I guess, in February, if you end up losing on that, could you just remind us around the risks around really a potential launch at risk?

I know you've got the PI hearing, but just trying to think about how we all should be thinking, given the many moving parts of this multilayered IP strategy that you have.

Jeffrey Dayno

Yes, sure, Graig. Over to Adam, first for thoughts on potential impact of the ORZEYFUL launch. Adam?

Adam Zaeske

Yes. We're thrilled with the results that we saw this quarter. Another now 5 out of the last 6 quarters of 400-plus patient adds. Really, really excited and enthusiastic about that continued growth.

We expect ORZEYFUL launch probably this month. It's looking like DEA scheduling will be wrapped up potentially this week. We expect ORZEYFUL to be scheduled for. So what that means for WAKIX, and ORZEYFUL will be indicated only in NT1.

So for WAKIX, we expect everything that's driven the performance to date will continue to drive our performance in the future, and we're confident in continued growth through the end of this year and into next year.

WAKIX will remain the only non-scheduled treatment option, broad clinical utility across NT1 and NT2 pediatric and adult patients with EDS and cataplexy. So broad indications and broad utilization, with strong efficacy, very clean safety and tolerability profile, which really differentiates it and makes it a great addition for any combination of treatments in a highly polypharmacy market.

We continue to enjoy broad coverage with more than 80% of lives covered, and we believe all of that will remain unchanged with the launch of ORZEYFUL and will continue to propel our growth through 2027.

Jeffrey Dayno

Peter?

Peter Anastasiou

Yes. And on your second question about the patent litigation, you're right, it is complicated, Graig, and I'll try to make it as simple as possible. The headline basically is that the schedule is proceeding exactly as we expected and actually even proposed.

So there is a schedule. Yesterday the judge issued a scheduling order that there is a scheduled preliminary injunction hearing. To be clear, we're the ones asking for the preliminary injunction, and she granted that.

We proposed it in 2 forms. We proposed it on October 26th in a letter that she asked us to provide on our proposed schedule, and we actually proposed it yesterday again in the morning in a letter we sent to the judge. And then in the afternoon she issued the schedule.

So this is progressing exactly the way we expected and wanted. So this is a positive for us. And so the preliminary injunction hearing will take place on the 26th. That then gives her a couple of weeks prior to the stay, the 30-month stay ending in mid-February, and then we expect a decision both on the injunction and the end of case.

Importantly, the amorphous case still has not been scheduled, and as you pointed out, just based on past history, those things typically get scheduled a couple of years out. So we expect it to be scheduled in 2029 and then a ruling from the judge sometime after that.

So this is progressing exactly as we had proposed, and we are looking forward to the upcoming PI hearing and stating our case. And we continue to reiterate the confidence that we have in March of 2030, when you look at both the ANDA case, the still outstanding amorphous case, and our requested preliminary injunction hearing.

Graig Suvannavejh

Just a quick follow-up. I mean, just the request for the preliminary injunction, which sounds like it's going the way that you had proposed, I guess from a more bearish perspective it gives a sense that you're already anticipating that you could lose in the final ruling.

So could you just address, like, how those who are trying to think about all the implications, the fact that you did request a hearing for the preliminary injunction, like how that may -- how people may interpret that, whether rightfully or wrongfully?

Peter Anastasiou

I mean, no one should interpret that as our belief that we won't prevail in the ANDA trial. We still have very strong confidence that we will prevail in the ANDA trial. But as you've seen, we've vigorously defended our IP and we're going to take advantage of every opportunity we have to continue to defend our IP.

So it's well within our rights to ask for a preliminary injunction, and we asserted that right. And so we -- and you also have seen that we asserted our rights by suing AET and Sandoz with the amorphous trial. So it just is consistent with the very assertive approach that we have taken to defend WAKIX IP. And we believe we will prevail ultimately, but we also believe we'll prevail specifically on the ANDA trial.

Operator

We will move next with Jay Olson with Oppenheimer.

Jay Olson

Thank you, Jeff, for your leadership over the years, and congrats on all the impressive progress you've made on behalf of patients.

Jeffrey Dayno

Thank you. Thank you, Jay. Thank you.

Jay Olson

Look forward to staying in touch with you. We have a question about BP-205, which is already demonstrating clear points of differentiation based on the clean safety and favorable PK profile. Can you talk about what to expect from the sleep-deprived volunteer study?

Beyond demonstrating wake-promoting activity, will you assess late-day efficacy, attention, next night's sleep, or other points of differentiation? And then as a follow-up, how will those findings factor into your clinical development plan, especially in terms of potential indications you would pursue?

Jeffrey Dayno

Yes, Jay. Thank you for your question. I'll turn it over to Kumar, but I think you're highlighting the multiple catalysts for BP-205 that are coming right around the corner, and the important one, the sleep-deprived healthy volunteer study data. And Kumar can provide more color on what we anticipate, how that will inform the multiple Phase II trials. Kumar?

Kumar Budur

Right. Thanks for the question and thank you for highlighting some of the features that we discussed today in terms of the PK differentiation, in terms of safety and tolerability differentiation for BP-205.

Regarding sleep-deprived healthy volunteer study, the goal is we anticipate wakefulness that is expected from this class of drugs that's orexin [indiscernible], and a favorable safety and tolerability profile. This is going to be a single dose in sleep-deprived healthy volunteers.

As long as we hit the benchmark, or the ballpark, what we anticipate in terms of efficacy for this class of compounds, that's good for us to move to the next stage of development.

What the sleep-deprived healthy volunteer study also does is it will help us to bracket the dose range that we'll be taking into Phase II studies. We already have an idea based on what we saw with our single ascending dose study, especially with the multiple ascending dose study, based on the target engagement AEs that we have seen with small doses, doses ranging from 0.1 to 3 milligrams in multiple ascending doses.

So the data from the sleep-deprived healthy volunteers will add to the data that we are generating, and that will help us to determine the dose ranges and the design of our multiple Phase II clinical trials that we plan to initiate in mid-2027 in multiple CNS indications.

Operator

We will move next with Rudy Lee with Wolfe Research.

Rudy Lee

Congrats on the tenure, Jeff. Hope you enjoy your journey out there. So follow-up question. For BP-205, can you provide more color on the headache AEs? Are they dose-dependent? Do you have any patients discontinued because of headache AEs? And what doses will you be testing in the sleep-deprived subjects? Are you dosing just QD or include split dosing? So what do you think would be the right therapeutic dose?

Kumar Budur

Thank you for the question. In terms of headaches, about 25% of the participants experienced headache. They were mild in severity and did not lead to any discontinuation. So headache did not lead to any discontinuation.

In terms of headache itself, it's actually very common in any Phase I studies to see headache raise to the top 1, 2, or 3 most commonly reported adverse events. Because all these studies are done in an inpatient unit, the healthy volunteers are in the unit sometimes for up to 10 days, and it's actually very common for these healthy volunteer participants to report headache.

On the second question, in terms of QD, BID, split dosing: the data that we have generated, Rudy, thus far in terms of terminal half-life from our single ascending dose study, in terms of effective half-life in our multiple ascending dose study, and the target engagement AEs that we saw with the QD dosing, mind you, the multiple ascending dose study was dose every day in the morning for 14 consecutive days, based on everything that we are seeing, we are confident that BP-205 will be QD dosing at this point in development.

Operator

We will move next with Patrick Trucchio with H.C. Wainwright.

Patrick Trucchio

I have a question on WAKIX. Just wondering, Steve, where should we be modeling the cost of sales going forward? And did this quarter include catch-up charges or royalty tier effects?

Is approximately 30% appropriate going forward? And as far as WAKIX buying patterns, could you quantify the third quarter revenue impact of the buying patterns that you referenced and clarify whether you expect the effect to reverse in the fourth quarter?

Jeffrey Dayno

Yes. Thanks for your question. I think Adam can respond in terms of the buying patterns and Steve can comment on the other impact.

Adam Zaeske

Yes, sure. I mean, the short answer is we really saw no impact from inventory fluctuations for third quarter results. Steve, over to you on cost of sales.

Stephen Mollichella

On cost of sales, third quarter represents the full impact of the new royalty from the Novitium agreement we signed in Q3. So I would expect that you would see that kind of going forward.

Patrick Trucchio

Okay. And then if I could just on BP-205, Kumar, separately from the single-dose sleep-deprived study, were any objective overnight sleep measurements collected during the MAD portion of program, or was the nighttime tolerability assessment based on participant-reported insomnia?

Kumar Budur

Patrick, we did not collect any information like attention, concentration, things like that. It's in healthy volunteers. And in terms of AE collection, obviously we collected the AEs based on the standard format that is used in Phase I studies.

And that's where we noticed insomnia in less than 20% of the participants. We saw urinary urgency and frequency in less than 20% of participants, only at 1 and 3 milligram doses. Both of these AEs were mild in severity. And importantly to note, neither of these target engagement AEs led to any discontinuations.

Operator

We will move next with David Amsellem with Piper Sandler.

David Amsellem

First, Jeff, I just want to wish you all the best. I've really enjoyed working with you over the years, so all my best to you. And now to my questions. Just a couple.

First on 205, as you think about evaluation of the drug in orexin-deficient patients, how do you think it will behave from a tolerability perspective? In other words, insomnia, Polycoria. I know that you have the data in healthies, but do you expect it to behave differently once you're putting it into actual orexin-deficient patients, as you think about tolerability, particularly insomnia and Polycoria? So that's number one.

And then secondly, appreciate, Peter, the transparency on business development, M&A. You did talk about a broader aperture within CNS, and that sort of leads me to wonder, are you thinking about, say, psychiatry?

And part of the reason I'm asking is because we are seeing a number of psychiatry-focused orexin programs that are moving forward, such as Alkermes' program in MDD for one of its orexins. So these are obviously much bigger markets, but how are you thinking about that? That to me is a big change from what Harmony had historically been thinking regarding M&A. So I'd love to hear more thoughts on that.

Jeffrey Dayno

Thanks for your question, and thanks for your kind words. I've enjoyed working with you as well over the years. First, let me frame in terms of BP-205 and disorders beyond orexin deficiency.

I think it, you know, as we said, it starts with regard to the potential, the high potency. So I think framing this opportunity is the most potent orexin-2 agonist in the clinic and the opportunity for lower clinical doses overall achieving driving toward a favorable risk-benefit profile.

And that's, I think, the opportunity that we see in the emerging data. I think Kumar can expand on that, but that is what we believe is the real opportunity in the high potency of BP-205.

Kumar Budur

Thank you, Jeff. Thanks for the question. [Indiscernible] question. Look, from a clinical development perspective, what happens is, in general, I'm not talking specifically for BP-205 or narcolepsy, in general, the tolerability profile is much better in patients compared to healthy volunteers.

That's what we have seen. But specifically in NT1, that's a question that I don't know the answer, to be honest with you, because these are also the patients whose physiological system has never ever been exposed to any meaningful exposure to orexins.

So we just need to see what happens in the clinical setting. But outside of NT1, David, in NT2, IH, in broader CNS indications, these individuals have intact orexin physiology, and the healthy volunteers also have intact orexin physiology. So I'm actually pretty confident that the safety and tolerability profile, which is very favorable that we saw in the MAD dose, extends to patient populations who have intact orexin physiology that is, NT2, IH and broader CNS indications.

Jeffrey Dayno

Thanks, Kumar. Peter, on non-business development.

Peter Anastasiou

Yes. Thanks for the question, David. First of all, just to reiterate, sort of our priority areas are where we have franchises now and emerging franchises. So clearly sleep is a high priority given our leadership in the sleep space.

We have an emerging franchise in epilepsy, so that's a high area of interest. In terms of those broader indications that you asked about, we've been silent to date on what specific indications we're pursuing with orexin, but we have said that the broad categories of interest are things that relate to areas of orexin's role in various disease processes, and those include cognition, attention, mood, fatigue just as broad categories.

And so as we continue to move forward our planning for our orexin strategy, those final indications that we ultimately start studies in next year will also be areas of interest that we would also pursue BD opportunities in. So I can't be really specific yet, but I think that gives you some broad strokes.

And when we become more transparent with you next year, when we kick off those Phase II studies, I think you'll have a better understanding of what those areas are and how that could be pulled through in our BD strategy.

Jeffrey Dayno

Yes. David, just to add, I think that the opportunity with BP-205 and the broader orexin space in broader CNS indications is really what's sort of opening up the aperture from a BD perspective. So given the excitement there, given the opportunity with BP-205, we see the BD aperture opens up with regards to opportunities in those broad CNS indications as well.

Operator

We will move next with David Hoang with Deutsche Bank.

David Hoang

Jeff, let me echo everyone else. It was a pleasure working with you and I wish you only the best in your next chapter.

So in terms of questions: first, noticing a little bit of quarter-over-quarter deceleration for WAKIX in terms of the net patient adds, is there anything to read there? And if you could just remind us and maybe speak to the underlying demand this quarter versus last.

And then in terms of the amorphous formulation of pitolisant, where you licensed that in from, I believe, Novitium, how does that now fit into your lifecycle management plans alongside pitolisant HD and GR?

Adam Zaeske

Thanks for the question. Very pleased with quarter-over-quarter results, achieving 400 patient adds in third quarter. As you know, over the last 6 years WAKIX typically has added between 1 and 400 patients in a quarter.

This quarter marks now 5 out of the last 6 quarters with 400 or more patient adds. So that really emphasizes an acceleration of growth in year 7. And we're very pleased with that momentum and we can see continued momentum as we enter the fourth quarter and continue that growth through the end of the year.

We're fully on track with our estimates internally through 3 quarters. Very confident in our ability to achieve guidance of $1 billion to $1.04 billion for the year and looking forward to hitting that major milestone for the company.

Jeffrey Dayno

Thanks, Adam. Kumar?

Kumar Budur

Yes. Thanks for the question. Regarding amorphous form of pitolisant. This is the form of pitolisant that has been issued patent until 2042. In terms of how it fits within the broader pitolisant franchise, one of the things that we always wanted to explore is certain symptoms like fatigue in broader neuropsychiatric indications.

And amorphous form of pitolisant offers us that opportunity to look into some of those broader indications that we couldn't pursue with, for example, WAKIX or pitolisant HC, where we are specifically looking into rare orphan disorders. Just as an FYI, with the WAKIX formulation we actually did generate positive data both in sleep and fatigue in myotonic dystrophy.

So we have shown there is a proof of concept that histaminergic mechanism of action can be helpful in fatigue. And that's what we want to do. We want to look at certain symptoms in broader neuropsychiatric indications with the amorphous form of pitolisant.

Peter Anastasiou

And it also gives us the opportunity to explore new formulations with that formulation as well. So not just new indication space, but also with a new route of delivery.

Kumar Budur

New mode of delivery, right. Thank you, Peter.

Operator

We will move next with Danielle Brill with Raymond James.

Danielle Brill

Also want to wish you the best, Jeff. It's been great getting to know you over the years and likewise hope to connect again in the future.

I just wanted to ask on -- sorry, BP-205. So you saw target engagement at 0.1 milligrams and urinary AEs at 1 and 3. So how confident are you that you can get clinically meaningful efficacy below the exposure where these on-target AEs emerge? And do you think this separation is being driven more by 205 potency, Tmax, or overall exposure?

Kumar Budur

All right. So Danielle, when you say the separation of AEs, I assume the separation of insomnia that was observed at all 4 doses, whereas urinary frequency and urgency were observed only at 1 and 3 milligram doses. Good question.

What we can say, as I mentioned earlier, is the observation of target engagement. AEs at 0.1 milligram was not a surprise for us given the potency of BP-205 and what we had seen in the preclinical model.

In terms of how we can extrapolate these to efficacy, we will be testing these doses. We haven't disclosed exactly the doses that we'll be testing in sleep-deprived healthy volunteers, and we will see. Ultimately the goal is to derive efficacy, good efficacy, clinically meaningful efficacy without causing troublesome AEs. That includes target engagement AEs which are insomnia, urinary frequency, and urgency.

Peter Anastasiou

And if I could just add a perspective, I think people should not expect that we will completely avoid those target engagement side effects. They are part and parcel of raising orexin levels, and anything that raises orexin levels is likely to have those.

What we aim to do is have them be low enough where they're not problematic. And that's what we saw in the MAD study. After 14 days of repeated dosing, they were mild in severity, they didn't lead to discontinuation, and they were by definition not problematic.

And so that's what we're aiming for in terms of striking the right balance in the doses we select to give us that efficacy that we think that will generate from the strong potency that we have, but also minimize, but probably not avoid, the on-target AEs.

Jeffrey Dayno

Yes, yes. I would just add that I was going to mention the same thing, that we're not going to avoid the on-target AEs, but it's the overall again, back to the overall benefit-risk profile what we're driving towards. Obviously, Danielle, some of the other orexin development programs, you're seeing higher rates, you're seeing some of that in the [indiscernible] label.

That is what we are trying to prove out here and achieve. There's an interesting analog in the market and it's called WAKIX. WAKIX has a very favorable overall safety, tolerability and efficacy profile, risk-benefit profile, which leads to its broad clinical utility. So that is how the development program is designed to sort of demonstrate that with BP-205.

Operator

We will move next with Jason Gerberry with Bank of America.

Unknown Analyst

Hi, this is [ Melanie ] on for Jason. Just one on the litigation. So if the generic gets a non-infringement ruling on polymorph, can you talk about potential at-risk launch scenarios that investors should understand heading into 2027?

Peter Anastasiou

Yes. A couple of thoughts. First of all, there's 2 patents that are the subject of the ANDA. One is the polymorph patent that you mentioned. The other one is a method-of-use patent. And so they are claiming that they do not infringe on the polymorph patent and introduced lots of evidence in the trial that they have an amorphous formulation. And so we'll come back to that in a second. And then the method-of-use, they're challenging the validity of that patent.

And this is an issued patent, and issued patents that get screened and approved by the Patent and Trademark Office are presumed valid patents. And so all we have to do is prevail on one of those 2.

And so then the amorphous trial after they introduced all this evidence to try to avoid the claim that they're infringing on our polymorph patent, they introduced lots of evidence about amorphous. And so we have an exclusively licensed amorphous patent.

Actually now we have a second one as well that was issued this quarter that was mentioned in the press release also protecting amorphous form of pitolisant. And so we talked about how we're developing that due to an earlier question, but we're also asserting those patent rights against AET and Sandoz because it's very clear that they do have an amorphous formulation.

So ultimately there's 3 patents at play here. All we have to do is prevail on one of them, and we believe very strongly that we can prevail on all 3. But all we need to do is prevail on one of them.

But as mentioned before, we have every right to ask for a preliminary injunction. And so we did that and that hearing will be scheduled because we think it's important for the judge to hear the amorphous case prior to any generic entering, given the obvious connectivity of the amorphous lawsuit to the formulation that AET and Sandoz plan to launch.

Operator

We will move next with So Youn Shim with UBS.

So Youn Shim

Jeff, congrats on your retirement, and thank you for all the helpful discussion over the years. On the PI proceeding, given AET's argument that Harmony does not practice the 920 patent with WAKIX and that monetary damages could therefore be adequate, can you discuss how you plan to demonstrate the irreparable harm from a potential generic launch to support the PI?

And if I can squeeze in on BP-205, so you have noted that urinary frequency, urgency were only shown at 1 and 3 mg across the cohorts, the 0.1, 0.6, 1, 3 mg cohorts. Did you see any dose-dependent trends in insomnia rates as well?

Peter Anastasiou

I'll just quickly mention on the first one, we're not going to be able to reveal to you the strategy and the arguments that we will put forth in the upcoming PI hearing. But all of those documents will be public along the way once we file them, and certainly they will be public during the PI hearing.

So I won't disclose any of that now, other than to say we feel we have a very strong case to not only demonstrate irreparable harm, but also the importance of the amorphous trial. And it's important to note that AET and Sandoz have never claimed that our lawsuit is a sham lawsuit, that they argue against the merits.

And so we believe that along with a strong irreparable harm argument will be laid out in the documents and in front of the court, and we're looking forward to that opportunity.

Kumar Budur

Thank you, Peter. Thanks for asking the question. In terms of the relationship between dose and AE, we haven't disclosed that information. These are preliminary data from the multiple ascending dose study. We are still looking into exposure-AE relationship, which is much more meaningful than the dose-AE relationship. Once we have all of those data, we'll present the full data set at an upcoming medical conference.

Operator

Thank you. This concludes our Q&A session. I would now like to turn the call back to Jeff Dayno for any closing remarks.

Jeffrey Dayno

Thank you, Nikki, and I just want to thank everyone for joining our call today and for your continued interest in Harmony Biosciences. Thanks, and have a great day.

Operator

Thank you. This does conclude today's Harmony Biosciences' third quarter 2026 financial results conference call. You may now disconnect your lines and have a wonderful day.