About Achillion

Complement Focused. Patient Driven.

At Achillion, we are driven to transform the lives of patients and families affected by 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 disorders of the complement system. We are actively assessing two clinical stage, oral factor D inhibitors, danicopan (ACH-4471) and, second-generation, ACH-5228 in Phase 2 and 1 studies, respectively.

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 paroxysmal nocturnal hemoglobinuria (PNH), a blood disease, and C3 glomerulopathy (C3G), a disease affecting the kidneys. Both conditions are devastating 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, danicopan (ACH-4471), has demonstrated proof-of-concept in PNH and initial proof-of- mechanism in C3G with clinical development expanded into global, Phase 2 clinical programs. In addition to danicopan, a second-generation, oral factor D inhibitor, ACH-5228, has completed a Phase 1 multiple ascending dose (MAD) study. We are preparing to submit an investigational new drug (IND) application to the U.S. FDA for ACH-5228 in the fourth quarter of 2019. Achillion has also identified a series of third-generation factor D inhibitors, which we believe will provide additional optionality and durability for our factor D development program. We plan to nominate a lead candidate from the third-generation factor D inhibitors for clinical development in 2020. To achieve our goal of late-stage development and potential commercialization, Achillion is partnering with key stakeholders including patients and their families, regulators, healthcare professionals and payors.

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.