Raptor Pharma Gets IP License For Huntington Disease Treatment

The Weizmann and Niigata patents cover the use of transglutaminase inhibitors, a class of molecules chemically similar to Cysteamine, in the potential treatment of Huntington’s Disease and other neurological disorders.

Raptor Pharma said that these patents add to its portfolio of intellectual property related to its programs utilising DR Cysteamine, Raptor’s proprietary formulation of delayed-release cysteamine bitartrate, licensed exclusively, with worldwide rights, from the University of California, San Diego (UCSD).

Further, Raptor plans to initiate a Phase II clinical trial this summer of DR Cysteamine in patients with Huntington’s Disease under a previously signed collaboration agreement with CHU d’Angers of France. The company is also developing DR Cysteamine as a treatment for nephropathic cystinosis (cystinosis) and non-alcoholic steatohepatitis (NASH).

Ted Daley, president of clinical division at Raptor Pharma, said: “This exclusive license covering the Weizmann and Niigata patents significantly strengthens and expands our proprietary position as the compounds claimed in these patents are closely related to our lead clinical compound DR Cysteamine.

“Additionally, this strategic move to enhance our intellectual property position coincides with our planned clinical trial of DR Cysteamine in collaboration with The Centre Hospitalier Universitaire d’Angers (CHU d’Angers) for the potential treatment of Huntington’s Disease, as well as our future plans to explore potential treatments for multiple indications utilising cysteamine and DR Cysteamine.”

Patrice Rioux, chief medical officer of Raptor Pharma, said: “This clinical trial is intended to build on preclinical work published by Sandrine Humbert and Frederic Saudou from the Curie Institute in France. Their work demonstrated a potential mechanism for cysteamine in an in-vivo preclinical Huntington’s Disease model that showed increased brain and blood levels of brain-derived neurotrophic factor (BDNF), through its inhibition of transglutaminase, a key regulating enzyme in BDNF production.”

Dr Steinman said: “In previous animal studies published in the journal, Nature Medicine, the team that I worked with found that in a Huntington’s Disease preclinical model those given cysteamine had fewer tremors and other abnormal movements and reduced weight loss, as compared to the untreated subjects.

“Additionally, cystamine appeared to work differently than other Huntington’s Disease drugs, and it may be capable of adding to the benefits of existing therapies. This could be a powerful option in the treatment of Huntington’s Disease.”

In addition, Raptor Pharma has added Lawrence Steinman, an inventor on the Weizmann patent, to its advisory board.