CLL & Follicular Lymphoma

Single Agent
Phase I
Phase II
Phase III

The PI3K/AKT/mTOR pathway is an important signaling pathway for many cellular functions such as cell survival, cell cycle progression and cellular growth. PI3Ks are a family of enzymes within this pathway that have been shown to play a critical role in the proliferation and survival of certain cancer cells. There are several isoforms of PI3K that are expressed in different types of cells. The delta isoform is believed to be important for survival of certain B-cell leukemias and lymphomas.

We acquired exclusive worldwide rights to ME-401 (formerly PWT143) in September 2013. Data from pre-clinical studies show ME-401 to be a potent and selective oral inhibitor of PI3K delta, a molecular target that plays a critical role in the proliferation and survival of certain hematologic cancer cells. PI3K delta is a class of drugs that has shown promise in the treatment of B-cell malignancies, but with particular toxicities. We believe this provides an opportunity for development of a next-generation oral drug that can produce therapeutic responses at a safe, effective dose. ME-401 has a distinct chemical structure from certain other PI3K delta inhibitors, including idelalisib (marketed as Zydelig®). Data presented at the American Society of Hematology (ASH) Annual Meeting in December 2012 demonstrated that ME-401 has superior pre-clinical activity compared to idelalisib.

Data from a first-in-human, single ascending dose clinical study of ME-401 in healthy volunteers demonstrated on-target activity at very low plasma concentrations. In addition, the results suggest that ME-401 has the potential for a superior pharmacokinetic and pharmacodynamic profile and an improved therapeutic window compared to idelalisib, with a half-life that supports once-daily dosing. These results were presented at the American Association for Cancer Research (AACR) Annual Meeting in April 2016.

In March 2016, the U.S. Food & Drug Administration (FDA) approved our Investigational New Drug (IND) application for ME-401 in B-cell malignancies. A Phase Ib dose-escalation study of ME-401 in patients with recurrent chronic lymphocytic leukemia (CLL) or follicular lymphoma opened for enrollment in September 2016. The goal of the study is to demonstrate an improved therapeutic window with repeated dosing in cancer patients. The study is now actively dosing patients and interim data are expected in June 2017.

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