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Harmony Biosciences announced it will acquire Zynerba Pharmaceuticals, a pioneer in transdermal cannabinoid therapies for orphan neuropsychiatric disorders, including Fragile X syndrome (FXS). Harmony will pay up to $200M.

“This acquisition affords us the opportunity to advance the development and delivery of a potentially transformative treatment for the symptoms of Fragile X syndrome and other rare neuropsychiatric disorders,” said Jeffrey M. Dayno, MD, president and CEO at Harmony Biosciences.

Harmony is the developer of Wakix (pitolisant) tablets, a potential blockbuster treatment for narcolepsy. The company describes itself as “dedicated to developing and commercializing innovative therapies for patients with rare neurological diseases.”

FXS is a rare genetic disorder that affects approximately 80,000 people in the United States, causing intellectual disabilities and behavioral challenges, among other medical issues.

Zynerba’s lead asset, Zygel, is the first and only pharmaceutically manufactured, synthetic cannabidiol, a non-euphoric cannabinoid, formulated as a patent-protected permeation-enhanced gel for transdermal delivery through the skin. Zygel is manufactured through a synthetic process in a cGMP facility, is not extracted from the cannabis plant, and does not contain THC.

The treatment is currently in a pivotal Phase III clinical trial (RECONNECT) for patients living with FXS. It is also being studied for 22q11.2 deletion syndrome (22q). There are currently no FDA-approved therapies for this condition. The company has completed a Phase II trial of the drug in 22q (INSPIRE). In this trial, the total score and all five subscales of the Anxiety, Depression, and Mood Scale (ADAMS) showed statistically significant improvements at 14 weeks of treatment compared to baseline. There are plans for Phase III trials in this indication.

So far, the only other FDA-approved cannabidiol-based treatment is Epidiolex, an oral solution used to treat seizures associated with Lennox-Gastaut syndrome (LGS), Dravet syndrome, or tuberous sclerosis complex (TSC).

According to a release, Harmony will commence a tender offer to acquire all outstanding shares of Zynerba for a purchase price of $1.1059 per share in cash, or $60 million in the aggregate, plus the right to receive potential additional payments of up to $140M.

“In addition to the strength of our core business in narcolepsy and our current life cycle management programs, led by idiopathic hypersomnia, we are excited to continue to diversify our portfolio beyond sleep/wake by adding Zynerba’s clinical development programs to our pipeline,” said Dayno.

“Harmony’s development and commercial expertise, technologies, people, and focus on rare neurological diseases are an excellent strategic fit with Zynerba,” said Armando Anido, chairman and CEO of Zynerba. “With Harmony’s scale, resources, and proven commercial excellence, they are well positioned to potentially bring to market the first pharmaceutical product indicated for the treatment of behavioral symptoms of Fragile X syndrome and to maximize the value of Zygel.”

Sales of Wakix have been encouraging. “This quarter represented the highest top-line prescription demand since our first full quarter of launch in 2020 and the strongest quarter of new patient starts in our history,” said Dayno.

“This sustained momentum in our core business, driven by robust underlying demand, reinforces our confidence in Wakix being a $1-billion-plus opportunity in narcolepsy alone.”

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