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Agios Pharmaceuticals Inc is a biopharmaceutical company with a focus on developing treatments geared towards cancer and rare genetic disorders of metabolism. The company's primary focus is to develop potentially transformative small-molecule medicines. The clinical development plan for Agios' product candidates includes a precision approach with initial study designs that allow for genetically or biomarker-defined patient populations. The company seeks the potential for proof of concept early in clinical development, along with any potential for accelerated approval.
Harmony Biosciences Holdings Inc is a commercial-stage pharmaceutical company focused on developing and commercializing therapies for patients living with rare neurological diseases who have unmet medical needs. The company's product WAKIX (pitolisant), is a molecule with a novel mechanism of action specifically designed to increase histamine signaling in the brain by binding to H3 receptors and used for the treatment of cataplexy in adult patients with narcolepsy.