Isis Pharmaceuticals received a $5 million milestone payment from partner GlaxoSmithKline following news of its investigational drug ISIS-GSK5Rx being granted the development candidate designation. ISIS-GSK5Rx is the second medication created as a result of the collaboration between the two companies and is an antisense drug, indicated for the treatment of an undisclosed ocular disease. Among other investigational drugs, Isis has been dedicated to develop a therapy to pediatric spinal muscular atrophy.
“ISIS-GSK5Rx is the second drug in our collaboration with GSK to enter development to treat an ocular disease,” said the chief operating officer at Isis, B. Lynne Parshall. “This drug utilizes the unique properties of antisense to selectively reduce the production of a target known to be involved in various ocular diseases. We look forward to advancing this program and sharing more information on the target and disease.”
“Our collaboration with GSK has been very productive and has resulted in a number of novel antisense drugs in our pipeline to treat a diverse range of diseases,” added Parshall, at a point when the company’s broad pipeline includes 33 drug treatments for different diseases, with an emphasis on treating cardiovascular, metabolic, severe and rare diseases, such as neurological disorders and cancer.
Specifically, Isis Pharmaceuticals has been working on establishing itself as a leader in developing antisense technology, and in addition to GlaxoSmithKline, has also established a partnership with Genzyme for the commercialization in the United States and other countries of Kynamro, a pharmaceutical product designed to treat patients suffering from homozygous FH.
The company also has several other drugs in phase 3 clinical development, including a novel triglyceride lowering drug for the treatment of familial chylomicronemia syndrome, ISIS-APOCIIIRx, a treatment for the polyneuropathy form of TTR amyloidosis being developed with GSK, ISIS-TTRRx, as well as a therapy for infants and children with spinal muscular atrophy, being developed with Biogen Idec, ISIS-SMNRx.
Isis Pharmaceuticals has recently presented encouraging results from its ISIS-SMNRx, at the 19th International World Muscle Society Congress in Berlin, which revealed its efficacy in the treatment of spinal muscular atrophy in infants and children, according to the ongoing open-label phase 2 clinical trial. The company is also enrolling patients for the phase 3 trial ENDEAR, and is planning on initiate another phase 3 study called CHERISH.
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