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 enabled Achillion to generate a proprietary library of over 2,700 potent and specific complement inhibitors, with a pipeline including a first-in-class, and two next-generation, oral factor D 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 conducting clinical trials with ACH-4471, a first-in-class, oral complement factor D inhibitor, as a potential treatment for C3G and for PNH, both rare, and potentially life-threatening diseases. Furthermore, Achillion has advanced two next-generation factor D inhibitors, ACH-5228 and ACH-5548, into Phase 1 clinical development and IND-enabling studies, respectively, during 2018.


For additional information on Achillion’s C3G clinical trials, please visit:

  • Proof of Concept Study for a 12 Month Treatment in Patients With C3 Glomerulopathy (C3G) or Immune-Complex Membranoproliferative Glomerulonephritis (IC-MPGN):NCT03459443
  • Proof of Concept Study for 6 Month Treatment in Patients With C3 Glomerulopathy (C3G):NCT03369236
  • Proof-of-Mechanism Study to Determine the Effect of ACH-0144471 on C3 Levels in Patients With C3G or IC-MPGN:NCT03124368


For additional information on Achillion’s PNH clinical trials, please visit:

  • A Treatment Study of ACH-0144471 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) With Inadequate Response to Eculizumab (PNH):NCT03472885
  • A Treatment Study of ACH-0144471 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)):NCT03053102
  • A Long-term Treatment Study of ACH-0144471 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH):NCT03181633