Translating Innovative Science
into Therapeutic Possibilities

We see pitolisant as a portfolio in a product opportunity and are taking a mechanism-based approach to our clinical development programs, driven by our conviction that innovative science translates into therapeutic possibilities. It is clear to us that the burden of living with rare neurological diseases is significant and affects daily functioning. This has led us to using the model of patient-focused drug development, in which patient insights inform our clinical research programs, as we build a pipeline focused on potential, next-generation treatments to help people living with rare neurological diseases, where the needs are many and options are few.

Jeffrey M. Dayno, M.D., President and Chief Executive Officer


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.


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.


The company is currently evaluating the novel mechanism of action of pitolisant for application in other neurological diseases that may be mediated by histamine and H3 receptors. We are currently in the clinic with 3 development programs evaluating pitolisant in patients with idiopathic hypersomnia, Prader-Willi syndrome, and myotonic dystrophy.

We are evaluating the potential of HBS-102, an early-stage asset with a novel mechanism of action, for a variety of indications in patient populations living with rare neurological diseases.

For more information about Harmony’s clinical trials, please visit NCT05156047 (idiopathic hypersomnia), NCT04257929 (Prader-Willi syndrome), or NCT04886518 (myotonic dystrophy).