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.

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.

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
Pitolisant GR in Narcolepsy
PHASE 3
Pitolisant HD in Narcolepsy
PHASE 3
Pitolisant HD in Idiopathic Hypersomnia (IH)
PHASE 3
Pitolisant HD in Myotonic Dystrophy Type 1 (DM1)
PHASE 2

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

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 Encephalopathies
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 Epilepsy
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.

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
Lighthouse: a clinical trial in children and adults with Lennox-Gastaut 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 Lennox-Gastaut 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