Achillion Meets Enrollment Target In Kidney Disease Studies
Zacks Investment Research | Apr 03, 2019 10:23PM ET
Achillion Pharmaceuticals, Inc. (NASDAQ:ACHN) announced completion of enrollment target in two phase II studies evaluating its lead pipeline candidate, ACH-4471. Both the studies, a six-month study and a 12-month study, are evaluating ACH-4471 in C3 glomerulopathy (C3G), a devastating disease affecting the kidney.
The company will submit data from these studies to the FDA in an end-of-phase II meeting planned for the fourth quarter of 2019.
Achillion’s shares gained 4.8% on Apr 3 following the update. Shares of the company rallied 107% so far this year compared with the achieved enrollment target in this indication last week. Preliminary data from PNH and C3G studies have shown proof-of concept for the candidate.
Achillion is focussed on developing oral inhibitors of complement Factor D. Targeted therapeutic areas include PNH, C3G, IC-MPGN, atypical hemolytic uremic syndrome and dry age-related macular degeneration.
Factor D, an essential protein of the complement pathway, is integral to the human innate immune system. Though this area has commercial opportunity, the complement-mediated space is extremely crowded with many biotech companies working to introduce these treatments to the market.
The company is developing its next-generation factor D inhibitors, ACH-5228 and ACH-5548, in two separate phase I studies for PNH and other complement-mediated diseases.
Please note that there is growing interest in developing treatments for PNH and C3G. Apart from Alexion’s Soliris and its recently approved Ultomiris, complement inhibitor therapies for these indications are being developed by companies such as Ionis Pharmaceuticals (NASDAQ:IONS) , Novartis (NYSE:NVS) and Regeneron Pharmaceuticals among others.
Achillion Pharmaceuticals, Inc. Price
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