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 diseases 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 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.

DISCOVERY
PRE-CLINICAL
PHASE 1
PHASE 2
PHASE 3
REGULATORY REVIEW
Prader-Willi Syndrome (PWS)
PHASE 3
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.

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

CBS-105*

CBS-105 is an investigational novel regenerative cellular therapy being developed with the aim to replace lost neurons and restore their function in patients with treatment-resistant narcolepsy.

DISCOVERY
PRE-CLINICAL
PHASE 1
PHASE 2
PHASE 3
REGULATORY REVIEW
Treatment-Resistant Narcolepsy
DISCOVERY

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.

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

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 the first-and-only pharmaceutically manufactured synthetic cannabidiol devoid of THC and formulated as a patent-protected permeation-enhanced gel for transdermal delivery through the skin and into the circulatory system. The product is manufactured through a synthetic process in a cGMP facility and is not extracted from the cannabis plant. ZYN002 does not contain THC, the compound that causes the euphoric effect of cannabis.

Cannabidiol, the active ingredient in ZYN002, has been granted orphan drug designation by the United States Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of FXS and for the treatment of 22q. Additionally, ZYN002 has received FDA fast track designation for the treatment of behavioral symptoms in patients with FXS.

DISCOVERY
PRE-CLINICAL
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 been granted orphan drug designation by the United States Food and Drug Administration (FDA) for the treatment of Dravet syndrome (DS) and for the treatment of Lennox-Gastaut syndrome (LGS). Additionally, EPX-100 has received FDA rare pediatric disease designation for the treatment of DS and for the treatment of LGS.

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

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.

DISCOVERY
PRE-CLINICAL
PHASE 1
PHASE 2
PHASE 3
REGULATORY REVIEW
Developmental and Epileptic Encephalopathy (DEE)
PRE-CLINICAL

CBS-104*

CBS-104 is an investigational novel regenerative cellular therapy being developed with the aim to replace lost neurons and restore their function in patients with refractory epilepsy.

DISCOVERY
PRE-CLINICAL
PHASE 1
PHASE 2
PHASE 3
REGULATORY REVIEW
Refractory Epilepsies
DISCOVERY

Safety and efficacy have not been established for the investigational uses of pitolisant or any investigational agents shown above.

*Research collaboration with CiRC Biosciences.

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.

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.