Santaris Pharma has reported that the FDA has approved an investigational new drug application for the company's international Phase I/II study of SPC2996 in chronic lymphocytic leukemia (CLL).
SPC2996 is the first of a new class of LNA-based investigational drugs known as RNA antagonists and is designed to reduce the level of Bcl-2 protein within tumor cells by binding and inactivating Bcl-2 messenger RNA, thereby inducing programmed cell death (apoptosis). Bcl-2 is highly over-expressed in CLL cells and appears to play a role in pathogenesis of the disease, preventing lymphocyte apoptosis and being strongly correlated with poor clinical outcome.
The clinical trial, the European arm of which is already ongoing in Denmark and France, is an open-label, escalating, repeated-dose, multicenter study of SPC2996 in patients with relapsed or refractory CLL requiring therapy. The primary objective is to investigate the safety and potential efficacy of SPC2996 in CLL patients and the company plans to enroll an overall total of 42 patients. The participation of U.S. centers is intended to facilitate the further development of this novel investigational agent.