Achillion initiated a program to discover and develop drug candidates effective against the hepatitis C virus in 2002. Since that time, Achillion has successfully leveraged its expertise in medicinal chemistry and virology to develop its NS4A antagonist program, on which it is now collaborating with Gilead Sciences. Achillion has also used this expertise in a proprietary program against a more traditional and proven target, NS3 protease.
Achillion believes its lead drug candidate from this program, ACH-1625, has the following benefits:
Pharmacokinetics. With its rapid and extensive partitioning to the liver, as well as high liver/plasma ratios demonstrated in vivo, ACH-1625 has good potential for once daily dosing.
Virology. ACH-1625 has shown low single-digit nanomolar potency that is specific to HCV. It is equipotent against HCV genotypes 1a and 1b at IC50 ~1nM.
Safety. High safety margins have been established in both single ascending dose and repeat dose studies in humans. Overall, the compound is well tolerated with minimal side effects.
