Achillion Pharmaceuticals’ (NASDAQ:AHCN) 14-day Phase II ACH-4471 trial for complement (C3) glomerulopathy (C3G) is likely to face patient enrollment hurdles, affecting an on-time readout, experts said. Nonetheless, they earmarked the study for success, considering ACH-4471’s more targeted and upstream approach to modulating the complement system.
Even the interim Phase II data boasts early hints that it may be efficacious in reducing proteinuria, which also signifies that the Phase II (NCT03124368) will meet the primary endpoint of increasing blood C3 levels, experts added. Yet, because C3G is an ultrarare disease and the small Phase II has tight enrollment criteria, recruitment could be a limiting factor.
Interim data is expected in 3Q18, according to a company report. Achillion also has results from two other trials expected in 4Q18 and 1Q19 based on the same report. It is testing the drug in six-month (NCT03369236) and 12-month open label studies (NCT03459443).
According to one analyst report, peak sales for ACH4471 are estimated at USD 1bn. Achillion has a market cap of USD 392m. Analysts are also encouraged about ACH-4471’s prospects, attributing their sentiments to encouraging data presented in May, though they noted the small, four-patient sample size.