Pipeline

A Growing Pipeline of Innovative Solutions

Harmony Biosciences’ patient-focused drug development model allows us to build a pipeline focused on potential, next-generation, innovative treatments to help people living with rare neurological disease and unmet medical needs.

Sleep/Wake

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.

Two next-generation formulations of pitolisant tablets, Pitolisant GR and Pitolisant HD, represent modifications to the current pitolisant tablet formulation.

Pitolisant GR is being developed to have the ability to initiate treatment at a therapeutic dose range. Pitolisant HD is a reformulation with excipient modification in which we are looking to optimize the pharmacokinetic profile and target new symptoms, such as fatigue, in patients with narcolepsy.

PRE-IND
PHASE 1
PHASE 2
PHASE 3
REGULATORY REVIEW
Idiopathic Hypersomnia (IH)
PHASE 3
Prader-Willi Syndrome (PWS)
PHASE 2
Myotonic Dystrophy Type 1 (DM1)
PHASE 2
Pitolisant GR
PHASE 1
Pitolisant HD
PHASE 1

BP1.15205 (orexin-2 receptor agonist)

BP1.15205, an investigational compound, is a highly potent and selective oral orexin-2 receptor (OX2R) agonist that will be evaluated for treatment of symptoms of narcolepsy and other sleep/wake disorders. BP1.15205 represents a new chemical series of OX2R agonists.

PRE-IND
PHASE 1
PHASE 2
PHASE 3
REGULATORY REVIEW
Sleep/Wake Disorders
PRE-IND

HBS-102 (MCHR1 antagonist)

HBS-102, an investigational compound, is a melanin-concentrating hormone receptor 1 (MCHR1) antagonist. 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.

PRE-IND
PHASE 1
PHASE 2
PHASE 3
REGULATORY REVIEW
Prader-Willi Syndrome (PWS)
PRE-IND

Neurobehavioral

ZYN002 (cannabidiol gel)

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 produced (not plant-derived) cannabidiol that has been uniquely formulated as a gel for transdermal delivery. ZYN002 does not contain tetrahydrocannabinol (THC), the compound that causes the euphoric effect of cannabis.

PRE-IND
PHASE 1
PHASE 2
PHASE 3
REGULATORY REVIEW
Fragile X Syndrome (FXS)
PHASE 3
22q11.2 Deletion Syndrome (22q)
PHASE 2

Rare Epilepsy

EPX-100 (clemizole hydrochloride)

EPX-100, an investigational compound, modulates serotonin signaling by targeting central 5-hydroxytryptamine (5-HT) receptor subtypes. EPX-100 has received Orphan Drug Designation (ODD) from the FDA for treatment of Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS).

PRE-IND
PHASE 1
PHASE 2
PHASE 3
REGULATORY REVIEW
Dravet Syndrome (DS)
PHASE 3
Lennox-Gastaut Syndrome (LGS)
PHASE 2

EPX-200 (lorcaserin)

EPX-200 is a potent, oral, centrally active and selective 5HT2C agonist and is currently in IND-enabling studies. EPX-200 is a liquid formulation of lorcaserin.

PRE-IND
PHASE 1
PHASE 2
PHASE 3
REGULATORY REVIEW
Developmental and Epileptic Encephalopathy (DEE)
PRE-IND
Safety and efficacy have not been established for the investigational uses of pitolisant or any of the investigational agents shown above.

Clinical Trials

Tempo: a clinical trial in patients with 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

Trial Website
Reconnect: a clinical trial in children, adolescents, and young adults with 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

Trial Website
Argus: a clinical trial in children and adults with Dravet Syndrome

A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of EPX-100 as Adjunctive Therapy in Children and Adults with Dravet Syndrome

Trial Website

Resources for the Rare Neurological Disease Community

Patients and their caregivers can explore some of the advocacy organizations we work with and the resources they offer to help support the patient community.

Get free resources

Investigator Sponsored Studies (ISS)

Harmony Biosciences supports Investigator Sponsored Studies (ISS) aimed at furthering the understanding of our products and that align with our areas of scientific interest. Such studies may advance disease-related knowledge, improve patient care, or explore unmet clinical needs.

Apply for funding