GlaxoSmithKline is paying out $7.5 million to partner Isis Pharmaceuticals as an antisense drug being developed for transthyretin amyloidosis, "a severe and rare genetic disease," goes into a Phase II/III study.

TTR amyloidosis is characterised by progressive dysfunction of peripheral nerve and/or heart tissues and affects 50,000 patients worldwide and current treatments are limited. The 15-month study of the drug, known as ISIS-TTRRx, will involve some 200 patients with familial amyloid polyneuropathy who experience TTR build-up in their peripheral nerves and experience the loss of motor functions, such as walking.

Lynne Parshall, chief operating officer at Isis, said that "the rapid development of ISIS-TTRRx from a research-stage programme to a drug in late-stage clinical development in just over two years represents the strong commitment of both teams". She added that the encouraging data from a Phase I study, "in which our drug was well tolerated and produced significant reductions in TTR protein, supported the advancement of ISIS-TTRRx directly into this registration-directed Phase II/III study".

The drug is part of an Isis-GSK strategic alliance to develop RNA therapeutics for rare and infectious diseases. That pact was recently amended and apart from the $7.5 million fee, Isis is eligible to earn an additional $50 million to support the study, plus regulatory and sales milestone payments, plus double-digit royalties.

COPD combo accepted for US review

Meantime, GSK and partner Theravance have confirmed that the New Drug Application for their new once-daily chronic obstructive pulmonary disease combination therapy Anoro has been accepted for review by the US Food and Drug Administration.

The drug is a combination of two investigational molecules - GSK573719 or umeclidinium bromide, a long-acting muscarinic antagonist (LAMA) and vilanterol, a long-acting beta2 agonist (LABA), administered using the Ellipta inhaler. The Prescription Drug User Fee Act goal date has also been confirmed as December 18, 2013.