Achillion Pharmaceuticals, Inc. (ACHN - Free Report) 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 industry’s rise of 13.8%.
The phase II studies are evaluating the safety and efficacy of the factor D inhibitor, ACH-4471 in C3G patients. The endpoints of these studies include proteinuria and estimated glomerular filtration rate after six and 12 months of treatment as well as changes in kidney biopsy compared to baseline.
A successful completion of these mid-stage studies will likely advance the candidate in phase III development for C3G indication and will bring the drug closer to its commercialization potential. The candidate enjoys orphan drug status for this indication. We remind investors that there is no approved therapy for treating C3G, suggesting a good opportunity for Achillion. There are 4000 C3G patients in the United States and an equal number in Europe.
Please note that the company is also developing ACH-4471 in combination with Alexion’s (ALXN - Free Report) Soliris as a treatment for paroxysmal nocturnal hemoglobinuria (“PNH”) in a mid-stage study. The company 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 (IONS - Free Report) , Novartis (NVS - Free Report) and Regeneron Pharmaceuticals among others.
Achillion currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
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