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Feb 19, 2013

Mystery Rare Disease Drug Earns Isis $5M from GSK

  • Sometimes, it pays to be secretive: Isis Pharmaceuticals received a $5 million milestone payment from GlaxoSmithKline (GSK) toward the development of a drug to treat an undisclosed rare and serious disease. Isis will develop the drug to Phase II proof-of-concept, at which time GSK has the option to license it.

    "We are pleased to have achieved this milestone so early in our collaboration with GSK. This alliance is an important step in enhancing our focus on rare disease, and we are excited to move our third drug in this area into our pipeline," said Stanley T. Crooke, M.D., Ph.D., chairman and CEO of Isis. "Under this new model of partnership we are able to retain control of this asset to facilitate its rapid development, while benefiting from GSK's expertise and new focus in rare diseases, and financially from milestone payments even before GSK exercises its license option at Phase II proof-of-concept."

    In March 2010, Isis entered into a strategic alliance with GSK to develop RNA therapeutics for rare and infectious diseases, with the specific aim of applying Isis' antisense drug discovery platform toward the discovery and development of new therapeutics against targets for rare and serious diseases, including infectious diseases and some conditions causing blindness. The agreement covers up to six programs. Isis received an up-front $35 million payment from GSK and is eligible to receive license fees and milestone payments, totaling nearly $1.5 billion, in the event all six programs are successfully developed for one or more indications and commercialized through to pre-agreed sales targets.

    The first $5 million milestone payment was made five months into the partnership for an equally mysterious development candidate. In May 2011, the launch of a Phase I study of ISIS-TTRRx in transthyretin amyloidosis—the first antisense drug to enter development under the alliance—triggered another $5 million payment.

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