GSK signs RNA therapeutics alliance

pharmafile | March 31, 2010 | News story | Research and Development GlaxoSmithKline, Isis, RNA 

GlaxoSmithKline has forged a new strategic alliance with Isis Pharmaceuticals to exploit the specialist company’s expertise in anti-RNA therapeutics.

California-based Isis has a drug discovery platform based on ‘antisense therapies’, focusing on the promising area of research which targets a disease one stage earlier than other drugs.

Where most other medicines are small molecules or biologics that target a specific protein in a disease process, antisense therapies prevent protein synthesis by eliminating the mRNA – the template or pattern that guides the production of the protein.

GSK will work with the company to develop new therapies against targets for rare and serious disease, including infectious diseases and some conditions causing blindness.

“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 VP 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.”

The agreement covers up to six programmes, and Isis will receive an upfront $35 million payment from GSK, and is eligible to receive on average up to $20 million in milestones per programme up to phase II proof of concept (PoC).

GSK will have the option to license compounds at PoC, and will oversee all further development and commercialisation.

Isis will receive license fees and milestone payments totalling nearly $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. The specialist company is also in line for up to double-digit royalties on sales from any product successfully brought to market.

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 III 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 control of 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 Crooke, chairman and chief executive of 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.”

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