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Achillion Begins Study on First Complement Factor D Inhibitor

Achillion Pharmaceuticals, Inc.ACHN announced that it has started a phase I study on its first orally administered small molecule complement factor D inhibitor, ACH-4471. The randomized, placebo-controlled, single-ascending dose study is being conducted in healthy volunteers to evaluate single ascending doses of ACH-4471 in order to assess its safety, tolerability, pharmacokinetics and pharmacodynamics.

The primary endpoint of the study, which will enroll about 28 patients, is the evaluation of safety and tolerability. The company expects interim results from the study in the second quarter of 2016.

Additionally, a second phase I study evaluating multiple-ascending doses of ACH-4471 in healthy volunteers is expected to commence in the second quarter of 2016. Interim data should be out in the third quarter of 2016.

Achillion said in its press release that ACH-4471 has the potential to be developed as the first orally-bio-available, highly differentiated treatment of paroxysmal nocturnal hemoglobinuria (PNH) and other ultra-rare diseases.

We note that Achillion's complement inhibitor platform is focused on advancing compounds that inhibit factor D and have the potential to be used for the treatment of immune-related diseases. Some of the indications that are being evaluated for these compounds include PNH, atypical hemolytic uremic syndrome, dry age-related macular degeneration and chronic obstructive pulmonary disease.

We remind investors that in May 2015, Achillion announced an agreement with Johnson & Johnson's JNJ Janssen Pharmaceuticals, Inc. The agreement gives Janssen the global rights to develop and commercialize one or more of Achillion's lead hepatitis C virus (HCV) assets including ACH-3102, ACH-3422, and sovaprevir.

With Achillion giving the rights to its HCV portfolio to Janssen, the company intends to completely focus its research and development efforts on the complement inhibitor platform.

Achillion is a Zacks Rank #3 (Hold) stock. A couple of better-ranked stocks in the health care sector are Anika Therapeutics Inc. ANIK and Gilead Sciences Inc. GILD , each carrying a Zacks Rank #1 (Strong Buy).

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The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.


The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.

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