PLYMOUTH, Pa. – Harmony Biosciences (NASDAQ: HRMY) announced results from a Phase 2 study showing its drug pitolisant reduces excessive daytime sleepiness (EDS) in adults with myotonic dystrophy type 1 (DM1) and symptoms of fatigue.
The study, which was not designed to prove statistical significance but to detect a potential signal of efficacy, showed that patients who received higher doses of pitolisant experienced greater improvements than those who received lower doses or placebo.
The study published today evaluated the drug’s performance over 11 weeks, including a 3-week titration period and an 8-week stable-dose phase. The primary efficacy endpoint focused on change from baseline to week 11 in Daytime Sleepiness Scale scores, with secondary endpoints including other measures of sleepiness and fatigue and overall disease burden.
Pitolisant is believed to promote wakefulness by targeting histamine H₃ receptors and has been approved by the FDA under the trade name WAKIX® for the treatment of EDS or cataplexy in adults with narcolepsy. It is not yet approved for use in DM1, but is being studied for this indication.
With more than 40,000 people in the United States diagnosed with DM1, up to 90% of whom suffer from EDS and fatigue, the potential market for Pitolisant in this new indication is huge. Harmony Biosciences plans to advance the drug’s development through a pivotal Phase 3 study using its Next Generation 2 (NG2) formulation, which is designed to provide optimized pharmacokinetic profiles and higher dose strength.
The safety and tolerability of pitolisant in the study appeared to be consistent with its known safety profile, with adverse event rates similar between the drug and placebo groups. The company’s broader lifecycle management program targets the more than 100,000 patients with sleep/wake disorders who currently have unmet medical needs.
This information is based on a press release from Harmony Biosciences.
In other recent news, Harmony Biosciences has also made a series of advances. The company’s first-quarter net income soared 30% to $154.6 million, driven by the performance of its narcolepsy treatment drug WAKIX. Harmony Biosciences maintained its 2024 net revenue guidance of $700 million to $720 million.
Prominent investment firm Needham raised its price target on Harmony Biosciences to $52 from the previous target of $50 as the company works to expand and diversify its central nervous system franchise. The acquisition of epilepsy-focused company Epygenix Therapeutics and narcolepsy treatment TMP1116 is seen as an important step in boosting the growth and longevity of its existing WAKIX franchise.
These recent developments also demonstrate Harmony Biosciences’ efforts to diversify away from its reliance on Zygel, a treatment that previously dominated its portfolio. Harmony Biosciences currently has 13 projects in various stages of development across three CNS franchises, a move seen as diversifying the company’s products and mitigating risks associated with its development pipeline.
Investors and analysts are eagerly anticipating Harmony Biosciences’ key milestones, including pharmacokinetic data for the NG2 formulation expected in the first half of 2024 and the submission of a supplemental new drug application for the treatment of idiopathic narcolepsy in the second half of this year. These upcoming events are critical to the company’s continued growth and market positioning.
This article was generated with the support of artificial intelligence and reviewed by an editor. For more information, please see our terms and conditions.
