Translating Innovative Science
into Therapeutic Possibilities

Harmony Biosciences pipeline

CLINICAL TRIALS

Prader-Willi syndrome

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Efficacy and Safety Study of Pitolisant Followed by an Open-Label Extension in Patients With Prader-Willi Syndrome

Fragile X syndrome

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Efficacy and Safety Study of ZYN002 in Children, Adolescents, and Young Adults With Fragile X Syndrome

DEVELOPMENT PORTFOLIO

PITOLISANT

Pitolisant is a histamine-3 receptor (H3R) antagonist/inverse agonist, which was designed and developed by Bioprojet Societe Civile de Recherche (Bioprojet), and was approved in Europe by the European Medicines Agency in 2016. Harmony has an exclusive license from Bioprojet to develop, manufacture, and commercialize pitolisant in the United States.

Pitolisant, initially developed for treatment of excessive daytime sleepiness and cataplexy in patients with narcolepsy, is also being evaluated in patients with idiopathic hypersomnia, Prader-Willi syndrome, and myotonic dystrophy type 1.

NEXT-GENERATION PITOLISANT FORMULATIONS

Two next-generation formulations of pitolisant tablets (NG1 and NG2) represent modifications to the current pitolisant tablet formulation.

NG1 is being developed as a delayed-release formulation. NG2 is a reformulation with excipient modification in which the pharmacokinetic profile and different and higher dosage strengths will be evaluated.

ZYN002

ZYN002 is an investigational drug product in development for the potential treatment of behavioral symptoms associated with Fragile X syndrome (FXS) and 22q11.2 deletion syndrome (22q). ZYN002 is a pharmaceutically manufactured synthetic cannabidiol formulated as a patent-protected permeation-enhanced gel for transdermal delivery through the skin and into the circulatory system. ZYN002 does not contain tetrahydrocannabinol (THC), the compound that causes the euphoric effect of cannabis.

HBS-102

HBS-102, an investigational compound, is a melanin-concentrating hormone receptor 1 (MCHR1) antagonist that has the potential to offer a novel approach to the treatment of a variety of rare neurological diseases. Melanin-concentrating hormone neurons are located in the hypothalamus and are involved in several different functions, including feeding behaviors, energy balance and control of metabolic functions, rapid eye movement (REM) sleep, and mood and behavioral processing, among others.