First-quarter highlights include increased patient uptake of Wakix and steps toward advancing pitolisant for idiopathic hypersomnia.

Summary: Harmony Biosciences Holdings Inc reported first-quarter results, showcasing progress in its sleep/wake portfolio. Key highlights include a rise in patients on Wakix, advancements in the idiopathic hypersomnia program with plans for pitolisant, and positive data on next-gen pitolisant-based formulations. The company is on track to extend Wakix exclusivity and has entered an exclusive licensing agreement for TPM-1116. Financially, net product revenue for Q1 2024 reached $154.6 million, a 30% growth attributed to robust commercial sales of Wakix.

Key Takeaways:

Harmony Biosciences saw a rise in the number of patients using Wakix in Q1, totaling approximately 6,100.
The company is progressing with plans to submit a supplemental new drug application for pitolisant in idiopathic hypersomnia.
Harmony Biosciences’ first-quarter financial report reveals 30% growth in net product revenue compared to the same period last year, driven primarily by strong sales of Wakix.

Harmony Biosciences Holdings Inc announced first-quarter results, highlighting progress in its sleep/wake portfolio, including growth in the number of patients on Wakix, advancements in the idiopathic hypersomnia program, and positive data on next-gen pitolisant-based formulations.

“We believe Harmony is well-positioned to become the leading patient-focused [central nervous system] biotechnology company delivering innovative treatments to patients living with unmet medical needs. We have transformed our business by executing a best-in-class launch of Wakix in narcolepsy, advancing our pipeline through life cycle management and new indications, and diversifying our portfolio through strategic business development,” says Jeffrey M. Dayno, MD, president and chief executive officer of Harmony, in a release.

Key Q1 Sleep/Wake Highlights:

The average number of patients on Wakix increased by approximately 150 patients sequentially to approximately 6,300 for the quarter ended March 31.
Following a March meeting with US Food and Drug Administration, Harmony is moving forward with the idiopathic hypersomnia program and plans to submit a supplemental new drug application for pitolisant in idiopathic hypersomnia in the second half of 2024.
Reported positive pharmacokinetic data on next-gen pitolisant-based formulation 1. Pivotal bioequivalence and dosing optimization studies will be initiated in the fourth quarter of 2024. Prescription Drug User Fee Act date expected in 2026. Provisional patent filed with the potential for patent protection out to 2044.
On track to receive pharmacokinetic data on next-gen pitolisant-based formulation 2 in the first half of 2024.
Pediatric narcolepsy supplemental new drug application on track for Prescription Drug User Fee Act date of June 21.
Initiated the phase 3 TEMPO study in patients with Prader-Willi syndrome in March.
On track toward gaining pediatric exclusivity to extend Wakix exclusivity to September 2030 based on progress in the pediatric narcolepsy submission and advancement of the Prader-Willi Syndrome phase 3 TEMPO study.
Strengthened its leadership position and created opportunity for long-term revenue generation in sleep medicine with an exclusive licensing agreement with Bioprojet to develop, manufacture, and commercialize TPM-1116, a highly potent and selective oral orexin-2 receptor agonist that will be evaluated for the treatment of narcolepsy and other sleep-wake disorders. Expect to file IND by mid-2025 and initiate first-in-human studies in the second half of 2025.

Q1 2024 Financial Results

Net product revenue for the quarter ended March 31 was $154.6 million, compared to $119.1 million for the same period in 2023.

The 30% growth versus the same period in 2023 is primarily attributed to strong commercial sales of Wakix driven by continued organic demand tapping into a large market opportunity (approximately 80,000 patients diagnosed with narcolepsy in the US), according to a release from the company.