GlaxoSmithKline (GSK) and Isis Pharmaceuticals Inc announced a new strategic alliance that will apply the Isis antisense drug discovery platform to seek out and develop new therapeutics against targets for rare and serious disease, including infectious diseases and some conditions causing blindness.

Under the terms of the agreement, which covers up to six programmes, Isis will receive an upfront US$ 35 million payment from GSK and is eligible to receive on average up to US$ 20 million in milestones per programme up to phase-2 proof of concept (PoC). GSK will have the option to license compounds at PoC, and will be responsible for all further development and commercialisation. Isis will be eligible to receive license fees and milestone payments, totaling nearly US$ 1.5 billion, in the event all six programmes are successfully developed for one or more indications and commercialised through to pre-agreed sales targets. In addition Isis will receive up to double-digit royalties on sales, from any product that is successfully commercialised.

“As a platform, the Isis antisense approach offers us an exciting opportunity to target certain severe diseases in a way that has not previously been possible,” said Dr Patrick Vallance, senior vice-president and Head of Drug Discovery at GSK. “Isis Pharmaceuticals is a leader in antisense technology, and this new alliance will enhance our discovery platform in this promising research area.”

Antisense therapies target the proteins involved in disease processes through the RNA that is involved in building these proteins. The Isis discovery platform develops specific therapies that bind to messenger RNA (mRNA) and inhibit the production of disease-causing proteins. Isis recently announced data from a phase-3 trial in heterozygous familial hypercholesterolemia patients that demonstrated the therapeutic effect of this approach.

This alliance provides GSK with access to Isis’ expertise in drug discovery and development of RNA-targeted therapeutics, with Isis retaining responsibility for the discovery and development of compounds to the alliance targets from inception to PoC.

“We are excited to be working with GSK to apply antisense technology to these new therapeutic areas. We are particularly excited to work on the novel targets GSK brought to the alliance,” said Dr Stanley T Crooke, chairman and chief executive officer for Isis Pharmaceuticals. “This alliance is exactly the type of deal we want to do. We retain control of the discovery and early development of our drugs while working together with a very high-quality partner to maximise the value of the drugs in late-stage development and commercialisation.”

RNA-targeted therapeutics, or antisense therapies such as oligonucleotides, represent an opportunity for a new drug class.

Isis is exploiting its expertise in RNA to discover and develop novel drugs for its product pipeline and for its partners.