At Achillion, we are driven to transform the lives of patients and families affected by rare diseases of the complement system, an integral part of the innate immune system. Scientific and clinical evidence has implicated the complement system, and specifically the complement Alternative Pathway(AP), in numerous devastating diseases and conditions. Our principal focus at Achillion is to advance our clinical-stage portfolio of orally administered factor D inhibitors into late-stage development and potential commercialization for patients with devastating rare disorders of the AP.
Factor D is an essential enzyme upstream in the alternative pathway of the complement system and a target for selective suppression of AP activity. With drug candidates targeting a critical control point for the generation of AP activity, Achillion has prioritized the development of oral factor D inhibitors as potential treatments for patients with C3 glomerulopathy (C3G), a disease affecting the kidneys, and paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disease. Both conditions are devastating rare disorders in which overactivity of the AP is recognized as the underlying cause and where there are no approved therapies or existing therapies are inadequate for patients.
Our first-generation, oral factor D inhibitor, ACH-4471, has demonstrated initial proof-of-mechanism in both C3G and PNH with clinical development expanded into global, Phase 2 proof-of-concept clinical programs. In addition to ACH-4471, our next-generation oral factor D inhibitors, ACH-5228 and ACH-5548, have advanced into Phase 1 studies. To advance factor D inhibition into late-stage development and potential commercialization, our team plans to partner with key stakeholders including patients and their families, payors, regulators and healthcare professionals.
By combining great science, a growing commercial capability, and a keen understanding of patient needs, Achillion is building an integrated commercial pharmaceutical company capable of bringing oral, factor D inhibition to patients with AP-mediated diseases. Complement focused. Patient driven.