In the trial, the company will broaden the clinical scope and usefulness of sevuparin to treat sickle cell disease by testing its safety when administrated subcutaneously (under the skin).
This follows the acceptance by the U.S Food and Drug Administration (FDA) of an investigational new drug (IND) application for sevuparin.
Modus Therapeutics is currently evaluating the therapeutic activity of sevuparin to manage painful crisis, also known as acute vaso-occlusive crisis (VOC), in patients with sickle cell disease in an ongoing Phase 2 study (NCT02515838).
The study is still recruiting adults and adolescents older than 12 with sickle cell disease. Study locations include hospitals in The Netherlands, Turkey, Lebanon, Bahrain, Oman, Saudi Arabia, and Jamaica. Participants will be randomized to receive sevuparin or placebo through intravenous infusion for two to seven continuous days.
“The FDA’s decision to accept the IND application for the development of sevuparin in sickle cell disease will allow us to start our clinical development program in the U.S. and constitutes an important milestone in our efforts to provide patients with effective and convenient therapies to treat their disease,” Ellen K. Donnelly, PhD, CEO of Modus Therapeutics, said in a press release.
Sevuparin is based on the commonly used blood thinner heparin. It was designed in order to retain the anti-adhesive and anti-inflammatory potential, but with overall reduced anti-clotting activity. The new compound has the ability to restore blood flow and prevent obstruction of small blood vessels caused by abnormal blood cells, such as those affecting SCD patients.
In November 2016, Modus received two U.S. patents covering sevuparin chemical composition and its manufacturing method (U.S. Patent 9,480,701), and for its use for treating sickle cell disease (U.S. Patent 9,480,702).
Preclinical data supporting the patent application showed that sevuparin could reduce adhesion of both sickle red cells and a specific type of white blood cells to the interior of blood vessels, as well as to inhibit vessels’ obstruction.
This investigational compound also has been granted orphan drug designation for treating sickle cell disease by the FDA and the European Medicines Agency (EMA). Orphan drug status is expected to support and expedite the clinical development and approval of sevuparin in both Europe and the U.S.