Achillion Pharmaceuticals

About Achillion

Inspiring science. Innovative medicine.

Achillion Pharmaceuticals, Inc. (Nasdaq: ACHN) is a science-driven, patient-focused company leveraging its strengths across the continuum from drug discovery to commercialization to provide better treatments for people with serious diseases.

Achillion’s rational, iterative approach to discovery and development has created a powerful engine for advancing novel small-molecule drugs. Achillion pairs this rigorous science with insights about patients, caregivers, and the healthcare market to choose product candidates with disruptive potential – or the potential to change treatment paradigms. This has already enabled Achillion to generate a proprietary library of over 2,000 potent and specific complement inhibitors.

The company’s commitments to scientific excellence and market impact are reinforced by a culture that encourages cross-functional collaboration, organizational nimbleness and resilience, professional courage, and informed risk-taking.

By combining great science, a growing commercial capability, and a keen understanding of patient needs, Achillion is rapidly becoming a fully-integrated commercial pharmaceutical company capable of bringing its factor D inhibitors to patients with rare diseases on its own.

Achillion is headquartered in New Haven, Connecticut. The company’s common stock trades on the NASDAQ Global Select Market under the symbol: ACHN.

Achillion is motivated to be as heroic in all of its efforts as the patients and families we are working to help, as they cope with serious diseases.


Delivering on the Potential of Alternative Complement Pathway Inhibitors to Provide New Treatments for Rare Diseases and Other Serious Conditions

Novel therapeutic approaches that provide potent and targeted suppression of complement, overcome the limitations of current therapy and provide for ease of administration are needed by patients who suffer from complement-mediated diseases. These patients also need improved access to additional treatment options. Achillion is currently clinical trials with ACH-4471, a first-in-class, oral complement factor D inhibitor, and anticipates advancing a next-generation factor D inhibitor, ACH-5228, into clinical development by the end of 2017.

Potential indications being evaluated for Achillion’s compounds include paroxysmal nocturnal hemoglobinuria (PNH), C3 Glomerulopathy (C3G), dry age-related macular degeneration (dry AMD), and other therapeutic indications where dysregulation of the complement alternative pathway is implicated. Achillion anticipates that its platform could play a role in addressing the needs of all PNH patients, including patients who have suboptimal response to, or fail to respond to, the currently available treatment, as well as for patients suffering from other complement-mediated diseases.