Medtronic, Inc. and Alnylam Pharmaceuticals, Inc. announced that the companies are advancing their collaboration initiated in February, 2005, following positive pre-clinical data generated under the initial joint technology development phase of the program.
Under the terms of the agreement, Alnylam and Medtronic will focus on developing a drug-device combination for the treatment of Huntington's disease. The product is expected to consist of an RNAi therapeutic targeting the Huntington's disease gene that will be delivered by Medtronic's implantable infusion pump.
In addition, the companies may jointly decide to collaborate on the development of similar drug-device combinations using Alnylam's RNAi therapeutics platform and Medtronic's implantable infusion pump for the treatment of other neurodegenerative diseases, such as Parkinson's disease.
The agreement has also been revised as a 50/50 relationship in the United States. Medtronic will commercialize the therapy consisting of the identified RNAi compound and advanced delivery devices. In the United States, Alnylam has the opportunity to invest in clinical development of this therapy through product launch. In Europe, Medtronic is solely responsible for development and commercialization.
"We have been very pleased with the progress made to date by the scientific teams at Alnylam and Medtronic. Pre-clinical data from our Huntington's disease program provide a strong rationale to advance an RNAi therapeutic program forward for this important disease where there are simply no effective therapies for patients today," said John Maraganore, President and Chief Executive Officer of Alnylam.
"We have proven expertise in delivering targeted therapies throughout the body, while Alnylam brings leadership in the evolving science of RNAi technology," said Stephen Oesterle, M.D., Medtronic senior vice president, medicine and technology. "Together, we are well positioned to explore drug-device combinations with a goal of discovering better ways to treat serious neurodegenerative diseases."
Alnylam has reported on data in its Huntington's disease program in a number of scientific meetings. Most recently, at the Keystone Symposium "RNAi for Target Validation and as a Therapeutic" in January, 2007, Alnylam collaborators presented in vivo data demonstrating that an siRNA targeting the huntingtin gene inhibited the progression of Huntington's disease in a mouse model.
These results showed both a reduction of neuronal pathology and an improvement in abnormal behavior, where pathological protein aggregates were decreased by about 70 percent and two types of abnormal behavior - clasping and footslips - were ameliorated by approximately 50 percent and 70 percent, respectively. In addition, levels of the huntingtin protein that mediates Huntington's disease were reduced by about 70 percent.