Harmony Biosciences Reports Strong 2024 Revenues, Provides 2025 Revenue Guidance and Highlights Key Pipeline Catalysts

WAKIX$(R)$ (pitolisant) Preliminary Net Revenue of $201 Million for Fourth Quarter and $714 Million for Full Year 2024; Representing Growth of 23% in Year-Five of Launch

2025 WAKIX Net Revenue Guidance Between $820 - $860 Million; On Track to a Potential $1 Billion+ Opportunity

Value-Creating Catalysts Anticipated Every Quarter in 2025:

Q1 -- FDA Decision on File Acceptance of Pitolisant sNDA for Idiopathic Hypersomnia; Potential Approval in 2025

Q2 -- Orexin/BP1.15205 Data Presentation at SLEEP 2025 Conference

Q3 -- Pivotal Phase 3 Topline Data for ZYN002 in Fragile X Syndrome

Q4 -- Initiation of Pitolisant-HD Pivotal Phase 3 Trial in Narcolepsy

Robust, Catalyst-Rich Pipeline Includes Up to Six Phase 3 Clinical Programs by Year End 2025

PLYMOUTH MEETING, Pa.--(BUSINESS WIRE)--January 13, 2025-- 

Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY) today announced strong preliminary, unaudited net product revenues for Q4 and full year 2024 of $201 million and $714 million, respectively. Building on this performance, the company is well positioned for a catalyst-rich 2025, with value-creating milestones anticipated every quarter. Harmony will be highlighting its catalysts for 2025 and other key milestones during its presentation at the 43rd Annual J.P. Morgan Healthcare Conference on Wednesday, January 15, 2025, at 11:15 a.m. PT / 2:15 p.m. ET.

"2025 is a pivotal year for Harmony Biosciences, as we focus on unlocking the full potential of our catalyst-rich neuroscience pipeline, " said Jeffrey M. Dayno, M.D., President and Chief Executive Officer of Harmony Biosciences. "Building upon our foundation of strong and continued revenue growth for WAKIX, our multi-franchise pipeline has come into focus with value-creating catalysts expected each and every quarter this year. We are entering 2025 with strong momentum and a clear path toward long-term growth, building a company with the potential to deliver over $3 billion in net revenue going forward."

Fourth Quarter and Full Year 2024 Net Product Revenue for WAKIX (Preliminary and Unaudited)

   -- 
 Preliminary, unaudited net product revenue for the quarter ended 
      December 31, 2024, was approximately $201 million, compared to $168.4 
      million for the same period in 2023 
 
 
   -- 
 Preliminary, unaudited net product revenue for the full year ended 
      December 31, 2024, was approximately $714 million, compared to $582 
      million for the same period in 2023, representing 23% growth in its 
      fifth year on the market 
 
 
   -- 
 The average number of patients on WAKIX increased by approximately 300 
      sequentially to approximately 7,100 for the quarter ended December 31, 
      2024 
 

2025 Net Revenue Guidance

   -- 
 Net revenue projected between $820 to $860 million 
 

Key Catalysts for 2025

   -- 
 Q1 -- FDA decision on file acceptance of pitolisant sNDA for Idiopathic 
      Hypersomnia $(IH)$ 
 
          -- 
 IH sNDA for pitolisant submitted in Q4 2024 
 
 
          -- 
 Potential FDA approval in 2025 
 
 
 
 
   -- 
 Q2 -- Preclinical data for BP1.15205 (OX2R agonist) to be presented at 
      SLEEP 2025 
 
          -- 
 Data to include preclinical safety and efficacy 
 
 
          -- 
 BP1.15205 on track for first-in-human study in Q3 2025 
 
 
 
 
   -- 
 Q3 -- Topline data readout for ZYN002 from pivotal Phase 3 trial in 
      Fragile X syndrome $(FXS)$ 
 
          -- 
 Potential for first and only approved treatment for patients 
             with FXS 
 
 
          -- 
 Estimated 80,000 people living with FXS in the US; worldwide 
             rights 
 
 
 
 
   -- 
 Q4 -- Initiation of Pitolisant-HD Pivotal Phase 3 trial in Narcolepsy 
 
          -- 
 Designed to address the largest unmet need in patients with 
             narcolepsy by providing greater efficacy for both excessive 
             daytime sleepiness and cataplexy 
 
 
          -- 
 Program to include novel endpoint to assess narcolepsy-related 
             fatigue 
 
 
          -- 
 Preliminary IP filed out to 2044 to extend the pitolisant 
             franchise 
 
 
 

Robust, Multi-Franchise Pipeline

   -- 
 Anticipates up to six phase 3 clinical development programs by year end 
      2025 
 
          -- 
 Initiated pivotal phase 3 study in Lennox-Gastaut syndrome (LGS) 
             for EPX-100 in Q4 2024 
 
                 -- 
 EPX-100 is the most advanced 5HT2 (serotonin) agonist in 
                    clinical development 
 
 
 
 
          -- 
 Initiation of Pitolisant-GR pivotal bioequivalence study in Q1 
             2025 with readout anticipated Q3 2025 
 
 
 
 
   -- 
 Pipeline poised to deliver at least one new product or indication 
      approval every year for the next four years (2028) 
 
 
   -- 
 Current pipeline has the potential to generate over $3B in net revenue 
 
 
   -- 
 Additional program updates to be provided during the Management 
      presentation at the upcoming J.P. Morgan Healthcare Conference on January 
      15, 2025, at 11:15 a.m. PT / 2:15 p.m. ET. Presentation slides are 
      available on the investor page of the Harmony Biosciences website: 
      https://ir.harmonybiosciences.com/ 
 

The financial information included in this press release is preliminary, unaudited, and subject to change. It does not present all the information necessary for an understanding of the company's financial results for the fourth quarter or full year 2024.

About WAKIX(R) (pitolisant) Tablets

WAKIX, a first-in-class medication, is approved by the U.S. Food and Drug Administration for the treatment of excessive daytime sleepiness (EDS) or cataplexy in adult patients with narcolepsy and for the treatment of EDS in pediatric patients 6 years of age and older with narcolepsy. It was granted orphan drug designation for the treatment of narcolepsy in 2010, and breakthrough therapy designation for the treatment of cataplexy in 2018. WAKIX is a selective histamine 3 (H ) receptor antagonist/inverse agonist. The mechanism of action of WAKIX is unclear; however, its efficacy could be mediated through its activity at H receptors, thereby increasing the synthesis and release of histamine, a wake promoting neurotransmitter. WAKIX was designed and developed by Bioprojet (France). Harmony has an exclusive license from Bioprojet to develop, manufacture and commercialize pitolisant in the United States.

Indications and Usage

WAKIX is indicated for the treatment of excessive daytime sleepiness (EDS) or cataplexy in adult patients with narcolepsy and for the treatment of excessive daytime sleepiness (EDS) in pediatric patients 6 years of age and older with narcolepsy.

Important Safety Information

Contraindications

WAKIX is contraindicated in patients with known hypersensitivity to pitolisant or any component of the formulation. Anaphylaxis has been reported. WAKIX is also contraindicated in patients with severe hepatic impairment.

Warnings and Precautions

WAKIX prolongs the QT interval; avoid use of WAKIX in patients with known QT prolongation or in combination with other drugs known to prolong the QT interval. Avoid use in patients with a history of cardiac arrhythmias, as well as other circumstances that may increase the risk of the occurrence of torsade de pointes or sudden death, including symptomatic bradycardia, hypokalemia or hypomagnesemia, and the presence of congenital prolongation of the QT interval.

The risk of QT prolongation may be greater in patients with hepatic or renal impairment due to higher concentrations of pitolisant; monitor these patients for increased QTc. Dosage modification is recommended in patients with moderate hepatic impairment and moderate or severe renal impairment. WAKIX is contraindicated in patients with severe hepatic impairment and not recommended in patients with end-stage renal disease (ESRD).

Adverse Reactions

In the placebo-controlled clinical trials conducted in patients with narcolepsy with or without cataplexy, the most common adverse reactions (>=5% and at least twice placebo) for WAKIX were insomnia (6%), nausea (6%), and anxiety (5%). Other adverse reactions that occurred at >=2% and more frequently than in patients treated with placebo included headache, upper respiratory tract infection, musculoskeletal pain, heart rate increased, hallucinations, irritability, abdominal pain, sleep disturbance, decreased appetite, cataplexy, dry mouth, and rash.

In the placebo-controlled phase of the clinical trial conducted in pediatric patients 6 years and older with narcolepsy with or without cataplexy, the most common adverse reactions (>=5% and greater than placebo) for WAKIX were headache (19%) and insomnia (7%). The overall adverse reaction profile of WAKIX in the pediatric clinical trial was similar to that seen in the adult clinical trial program.

Drug Interactions

Concomitant administration of WAKIX with strong CYP2D6 inhibitors increases pitolisant exposure by 2.2-fold. Reduce the dose of WAKIX by half.

Concomitant use of WAKIX with strong CYP3A4 inducers decreases exposure of pitolisant by 50%. Dosage adjustments may be required.

H1 receptor antagonists that cross the blood-brain barrier may reduce the effectiveness of WAKIX. Patients should avoid centrally acting H1 receptor antagonists.

WAKIX is a borderline/weak inducer of CYP3A4. WAKIX may reduce the effectiveness of sensitive CYP3A4 substrates, including hormonal contraceptives. Patients using hormonal contraception should be advised to use an alternative non-hormonal contraceptive method during treatment with WAKIX and for at least 21 days after discontinuing treatment.

Use in Specific Populations

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