Achillion Pharmaceuticals, Inc. (ACHN - Free Report) announced completion of enrollment in a phase II study evaluating its lead pipeline candidate, ACH-4471, as a treatment for paroxysmal nocturnal hemoglobinuria (“PNH”), a rare life-threatening disease of the blood.
The company is developing the factor D inhibitor candidate in combination with Alexion’s (ALXN - Free Report) C5 Inhibitor, Soliris (eculizumab) for the indication.
Updated interim data from the study is expected to be presented at a meeting organized by the Italian PNH association in May 2019. The company is targeting an end-of-phase II meeting with the FDA in the second half of 2019.
Shares of Achillion have rallied 83% so far this year compared with the industry’s rise of 11.2%.
The phase II study is evaluating the combination of ACH-4471 and Soliris in PNH patients who have inadequate response to Soliris monotherapy. The primary endpoint of the study is increase in hemoglobin levels. The study will also observe reduction in blood transfusions over the treatment period of 24 weeks.
In December 2018, the company announced interim data from the study, which showed that treatment with the combination reduced blood transfusion to zero from 14 over a period of one year. The combination therapy also had a positive impact on hemoglobin levels in patients.
We remind investors that Achillion has also completed a phase II study evaluating ACH-4471 as monotherapy for PNH. The candidate has shown proof of concept in the study.
Apart from PNH, the company is also developing ACH-4471 for treating C3 glomerulopathy (C3G) and immune complex membranoproliferative glomerulonephritis (IC-MPGN) in mid-stage studies. The company is planning an end of phase II meeting in the fourth quarter of 2019 for these indications as well.
Achillion is focused on its factor D portfolio. The company is working 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 has 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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