PLYMOUTH MEETING, Pa. - Harmony Biosciences (NASDAQ: HRMY) has revealed findings from a Phase 2 study indicating that its drug pitolisant reduced symptoms of excessive daytime sleepiness (EDS) and fatigue in adults with Myotonic dystrophy Type 1 (DM1).
The study, which was not designed to demonstrate statistical significance but rather to detect potential signals of efficacy, showed that patients receiving higher doses of pitolisant experienced greater improvements compared to those on lower doses or a placebo.
The research, presented today, assessed the drug's performance over an 11-week period involving a 3-week titration and an 8-week stable dose phase. The primary efficacy endpoint focused on changes in the Daytime Sleepiness Scale score from baseline to Week 11, with secondary endpoints including other measures of sleepiness and fatigue, as well as overall disease burden.
Pitolisant, which is believed to promote wakefulness by targeting the histamine H₃ receptor, is currently FDA-approved under the brand name WAKIX® for the treatment of EDS or cataplexy in adult patients with narcolepsy. It is not yet approved for use in DM1 but is under investigation for this indication.
With over 40,000 individuals diagnosed with DM1 in the U.S., and up to 90% of them suffering from EDS and fatigue, the potential market for pitolisant in this new indication is significant. Harmony Biosciences plans to advance the drug through a pivotal Phase 3 study using a Next-Generation 2 (NG2) formulation, which aims to offer an optimized pharmacokinetic profile and higher dosage strength.
The safety and tolerability profile of pitolisant in the study appeared consistent with its known safety profile, and the rate of adverse events was similar between the drug and placebo groups. The company's broader lifecycle management programs are targeting over 100,000 patients living with sleep/wake disorders who currently have unmet medical needs.
This information is based on a press release statement from Harmony Biosciences.
In other recent news, Harmony Biosciences has seen a flurry of developments. The company's Q1 net revenues have surged by 30%, hitting $154.6 million, primarily driven by the performance of its narcolepsy treatment, WAKIX. Harmony Biosciences maintains its 2024 net revenue guidance of $700 million to $720 million.
Needham, a prominent investment firm, has raised its stock price target for Harmony Biosciences to $52, up from the previous $50, in light of the company's strategic efforts to expand and diversify its central nervous system franchises. The acquisition of Epygenix Therapeutics, a firm focusing on epilepsy treatments, and TMP1116 for narcolepsy, are seen as significant steps in bolstering the growth and longevity of its existing WAKIX franchise.
These recent developments also indicate Harmony Biosciences' commitment to diversifying away from its dependency on Zygel, a treatment that previously dominated its portfolio. Harmony Biosciences now has 13 programs at various stages of development across three CNS franchises, a move seen as a diversification of the company's offerings and a mitigation of risks associated with its development pipeline.
Investors and analysts are eagerly anticipating key milestones for Harmony Biosciences, including the pharmacokinetic data for the NG2 formulation expected in the first half of 2024, and the submission of a supplemental New Drug Application for Idiopathic Hypersomnia in the second half of the year. These upcoming events are critical for the company's continued growth and market positioning.
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