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Agios Pharmaceuticals Inc is a biopharmaceutical company focused on the field of cellular metabolism to create differentiated medicines for rare diseases, with a focus on classical hematology. The company's primary focus is to develop potentially transformative small-molecule medicines. Its product candidate, Pyrukynd (mitapivat), is an activator of both wild-type and mutant pyruvate kinase enzymes, developed for the treatment of hemolytic anemias. The other drug candidates in its pipeline include Tebapivat (PK activator), being developed as a potential treatment for MDS-associated anemia and sickle cell disease, AG-181 (PAH stabilizer), AG-236, and others.
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 is used for the treatment of cataplexy in adult patients with narcolepsy. The other drug candidates in its pipeline are Pitolisant, HBS-102 (MCHR1 antagonist), BP1.15205 (orexin-2 receptor agonist), ZYN002 (cannabidiol gel), EPX-100 (clemizole hydrochloride), and EPX-200 (lorcaserin), among others.