Harmony Biosciences Gains U.S. Rights to Pitolisant, Raises $270 Million

Harmony Biosciences Gains U.S. Rights to Pitolisant, Raises $270 Million October 6, 2017
By Alex Keown, BioSpace.com Breaking News Staff

PLYMOUTH MEETING, Penn. – Startup Harmony Biosciences has spun out of Chicago-based Paragon Biosciences with $270 million in financing and the exclusive U.S. rights to narcolepsy drug pitolisant.

Harmony will enter the U.S. with rights to pitolisant, which it acquired from the French company Bioprojet SCR. Pitolisant is a selective histamine H3-receptor antagonist/inverse agonist that enhances the activity of histaminergic neurons. It is marketed under the brand name Wakix in Europe, but has not been approved in the United States. In Europe, it has been approved for the treatment of narcolepsy in adult patients with and without cataplexy, a sudden and uncontrollable muscle weakness associated with narcolepsy. Pitolisant increases the release of the brain chemical histamine to increase a patient's wakefulness and alertness, according to Harmony.

The new Harmony is helmed by life sciences veteran Bob Repella, the former head of global commercial operations at CSL Behring. Parent Paragon is banking on his more than 30 years of experience in bringing drugs to market to treat orphan and rare disorders. As chief executive officer, Repella’s primary course of action will be securing U.S. Food and Drug Administration approval for pitolisant.

"We're excited to research, develop and seek US approval for pitolisant, a potential first-in-class therapy to treat patients with narcolepsy," Repella said in a statement. "In addition to working with the FDA, Harmony will be working closely with healthcare providers, advocacy organizations and other stakeholders to help address the unmet needs of the patient community."

It’s estimated that one in every 2,000 people in the United States is affected by the sleep disorder Narcolepsy. The disorder involved irregular patterns in Rapid Eye Movement (REM) sleep and significant disruptions of the normal sleep/wake cycle, according to the Narcolepsy Network.

Harmony said it intends to establish an expanded access program (EAP) for pitolisant in the United States in 2018. Additionally, the company is expected to submit a New Drug Application (NDA) for pitolisant for the treatment of narcolepsy in adult patients during the first half of 2018.

Harmony’s narcolepsy drug will have to compete with Jazz Pharmaceuticals ’ Xyrem, which is indicated for the treatment of cataplexy in narcolepsy and for the treatment of excessive daytime sleepiness (EDS) in narcolepsy. In 2016, Xyrem was responsible for the lion’s share of Jazz revenue stream, bringing in about $1.1 billion.

Harmony’s first years will be supported by the $270 million in financing. Investors include Valor Equity Partners, Fidelity Management & Research Company, HBM Healthcare Investments, Vivo Capital, venBio Partners, Novo Holdings and Nan Fung Life Sciences. In addition to seeking a revenue stream from pitolisant, Harmony said it will use the funding to provide novel treatments for patients with orphan and rare conditions, with a focus on CNS disorders.

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