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ACHN > up 16% on Phase I results for factor D inhibitor ACH-5228...achieves 95% mean inhibition of alternative pathway.

http://ir.achillion.com/news-releases/news-release-details/achillions-ach-5228-achieves-positive-results-phase-1-multiple

In the randomized Phase 1 MAD study, ACH-5228 was administered to 43 healthy volunteers. Study subjects received oral doses of ACH-5228 ranging from 40 mg to 200 mg twice a day (BID) for fourteen days. The study also included a single dose cohort of 240 mg. The results demonstrated that ACH-5228, when dosed 120 mg BID or higher, achieved near complete and sustained Alternative Pathway (AP) inhibition with a mean value of >95% at steady state concentrations as measured by AP Hemolysis and AP Wieslab assays.

The study demonstrated that ACH-5228 was generally well tolerated over the dose ranges tested, which include the doses expected to be evaluated in Phase 2 trials. The Company expects to submit an IND application to the U.S. Food and Drug Administration supporting clinical development for ACH-5228 in the fourth quarter of 2019.

Our plan is to advance ACH-5228 into Phase 2 clinical trials in multiple diseases. Danicopan, our first-generation alternative pathway inhibitor, validated factor D as a target and we plan to begin a Phase 3 registrational study in early 2020,” said Joe Truitt, Chief Executive Officer of Achillion Pharmaceuticals.