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 three clinical-stage oral chronic hepatitis C (HCV) candidates and a proprietary library of over 1,300 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.

Achillion’s Portfolio will Help Redefine Best-in-Class Treatment for HCV

Achillion discovered and developed a comprehensive portfolio of antivirals for the treatment of HCV, including a unique second-generation NS5A inhibitor, odalasvir (also known as ACH-3102); a novel nucleotide NS5B polymerase inhibitor, ACH-3422; and a NS3 protease inhibitor, sovaprevir.

The potential of Achillion’s three lead HCV candidates has been validated by a worldwide license and collaboration agreement with Janssen Pharmaceuticals worth up to $905 million in milestones plus mid-teen to low-twenties percentage royalties on worldwide sales.

Janssen is aggressively pursuing clinical trials with Achillion candidates in combination regimens that may redefine the standard of care for the more than 150 million patients with HCV worldwide.

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.

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.