Harmony Biosciences - Beyond Wakix And Narcolepsy

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Shares of Harmony Biosciences Holdings Inc. (HRMY) have gained 42% over the last three months to trade around $39.

Harmony Biosciences is a revenue-generating pharmaceutical company developing innovative therapies for patients with rare neurological diseases. The company markets Wakix for the treatment of excessive daytime sleepiness in adults with narcolepsy. Known generically as Pitolisant, Wakix is the first and only FDA approved non-scheduled treatment option for narcolepsy.

There has been continued sequential quarter over quarter growth in the sales of Wakix since its launch in November 2019.

The annual net product revenue of the drug, which was $5.99 million in 2019, climbed to $159.7 million in 2020. In the six months ended June 30, 2021, Wakix generated sales of $133.5 million compared to $57.8 million in the year-earlier period.

Harmony Biosciences is also exploring the clinical utility of Wakix beyond narcolepsy.

-- A phase II clinical trial of Wakix for excessive daytime sleepiness (EDS) and other non-muscular symptoms in adult patients with type 1 myotonic dystrophy was initiated by the company in June of this year.

Myotonic dystrophy is a rare, neurological and multi-system disease and is the most common form of adult-onset muscular dystrophy. Type 1 myotonic dystrophy (DMI) is the most common form of the disorder. It is estimated that there are 40,000 people currently diagnosed with DM1 in the U.S., with up to 90% of them reporting EDS and fatigue and over 60% of them experiencing cognitive dysfunction.

Topline results from the phase II trial of Wakix in myotonic dystrophy are anticipated in the second half of 2022.

-- A phase II trial evaluating Wakix for the treatment of excessive daytime sleepiness (EDS) and other key symptoms in patients with Prader-Willi Syndrome (PWS) was initiated last December.

Prader-Willi Syndrome is a rare, genetic multi-system disease characterized by hypothalamic dysfunction. About 15,000-20,000 patients in U.S. have PWS and more than 50% of them have Excessive Daytime Sleepiness (EDS) due to sleep-wake state instability of central origin and other factors. There are no approved treatments for EDS in patients with PWS.

Topline results from the phase II trial of Wakix in patients with Prader-Willi Syndrome are expected in the first half of next year.

As the first addition to the pipeline beyond Wakix, the company acquired CSTI-100, now known as HBS-102, a potential first-in-class molecule with a novel mechanism of action, from ConSynance Therapeutics Inc in August of this year.

HBS-102, a phase II-ready compound, is a Melanin Concentrating Hormone Receptor 1 (MCHR1) antagonist that has the potential to offer a novel approach to the treatment of narcolepsy including the symptoms of Rapid Eye Movement (REM) sleep dysregulation, such as cataplexy, hallucinations and sleep paralysis.

Cash position:

The company ended June 30, 2021, with cash and cash equivalents of $159.7 million.

Harmony Biosciences made its debut on the Nasdaq Global Select Market on August 19, 2020, offering its shares at a price of $24 per share.

HRMY has traded in a range of $25.09 to $52.74 in the last 1 year.

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