There was big news from drug giant GlaxoSmithKline yesterday, which won European approval for Duodart and signed a deal with Isis to develop RNA therapeutics for rare diseases.

The UK drugmaker said it has entered into a strategic alliance with US firm Isis Pharmaceuticals, under which the latter’s antisense drug discovery platform will be used to identify and develop new therapeutics against targets for rare and serious diseases, including infectious diseases and some causing blindness.

Opposed to targeting a specific protein in a disease process, antisense therapies actually prevent protein synthesis by elimating the messenger RNA guiding its production. Isis’ discovery platform has been constructed to develop specific therapies that bind to mRNA and thereby inhibit the production of disease-causing proteins.

“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 Patrick Vallance, Senior Vice-President and Head of Drug Discovery at GSK, explaining the group’s interest in the deal. “This new alliance will enhance our discovery platform in this promising research area,” he added.

Under the terms of the deal, which covers up to six programs, Isis stands to receive an upfront cash pile of $35 million, and is also eligible an average of up to $20 million in milestones per program up to the Phase II proof-of-concept stage, at which point GSK can license the compounds if it chooses to and take over all further development and commercialisation.

The deal is certainly huge for Isis, as the group could gain total payments of nearly $1.5 billion if all six programmes are successfully developed in one or more indication, as well as double-digit royalties on sales any product that makes it to market.

Duodart green light
Meanwhile, GSK also announced yesterday that European regulators have waved through Duodart - a fixed dose combination of dutasteride and tamsulosin - for the treatment of moderate-to-severe symptoms of Benign Prostatic Hyperplasia (BPH) and reduction in the risk of acute urinary retention (AUR) and surgery in patients with moderate-to-severe symptoms.

The drug’s approval came on the back of results from the CombAT, which study showed that the combination of dutasteride (marketed as Avodart) and tamsulosin offers patients with moderate-to-severe symptoms of BPH a “significantly superior and sustained symptom improvement” compared with tamsulosin, the most frequently prescribed medication, as well less risk of complications associated with BPH, including a 66% reduction in the risk of AUR and surgery.

According to Eddie Gray, President, Pharmaceuticals Europe at GSK, the firm developed the new fixed-dose combination medicine “to provide patients and physicians a convenient, once-daily treatment, which reduces the impact of the bothersome symptoms of this common condition, and the risk of potential complications and related surgery,” factors which can “create uncertainty and anxiety for many men and may also lead to additional unplanned costs for healthcare providers”.