Company Update (NASDAQ:ACHN): Achillion Pharmaceuticals, Inc. Initiates Phase 1 Study of ACH-4471, First Orally-Administered Small Molecule Complement Factor D Inhibitor

Achillion Pharmaceuticals, Inc. (NASDAQ:ACHN) announced the initiation of a phase 1 study with ACH-4471, the Company’s first orally-administered, highly potent and specific small molecule that has been shown to inhibit complement factor D. The phase 1 study is being conducted in healthy volunteers and is evaluating single ascending doses of ACH-4471 to assess safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD). The Company expects to report interim clinical data from this study during the second quarter of 2016.

“We believe our molecular-level understanding of factor D, based upon proprietary high-resolution x-ray structures, has enabled us to design ACH-4471 with the potential to be the first orally-bioavailable, highly differentiated treatment for PNH and other ultra-rare diseases. We look forward to advancing ACH-4471, with the goal of demonstrating its safety, tolerability, proof-of-mechanism and working to establish its potential benefit for patients,” commented Milind Deshpande, Ph.D., President and Chief Executive Officer.

Dr. Deshpande further stated, “In addition to ACH-4471, we have developed a substantial library of complement factor D inhibitors and are now working to advance distinct small molecule candidates with traits appropriate for ophthalmic administration, to potentially treat dry AMD, and for inhalation, in order to address respiratory indications such as COPD.” (Original Source)

Shares of Achillion Pharmaceuticals closed yesterday at $5.92, down $-0.10 or -1.66%. ACHN has a 1-year high of $12.94 and a 1-year low of $5.67. The stock’s 50-day moving average is $8.09 and its 200-day moving average is $8.37.

On the ratings front, Leerink Swann analyst Howard Liang downgraded ACHN to Hold, in a report issued on January 29.

According to, Liang has a total average return of 19.0%, a 54.0% success rate, and is ranked #105 out of 3569 analysts.

Achillion Pharmaceuticals Inc is a biopharmaceutical company which focuses on the discovery, development and commercialization of treatments for infectious diseases like hepatitis C virus, or HCV, infection that are once-daily and ribavirin-free.

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