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news.Isis Pharmaceuticals has earned an $18m milestone payment from GlaxoSmithKline (GSK) for the advancement of Phase II/III study of ISIS-TTR in patients with familial amyloid polyneuropathy (FAP).
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ISIS-TTR is an antisense drug intends for the treatment of transthyretin amyloidosis, a genetic disease characterized by progressive dysfunction of peripheral nerve and/or heart tissues.
For the ISIS-TTR development, Isis has secured $45m of the total $70m so far, including the latest payment.
The company will also receive a license fee, regulatory and sales milestone payments and double-digit royalties on sales of ISIS-TTR, if GSK decides to license the ISIS-TTR program.
Isis chief operating officer Lynne Parshall said: "ISIS-TTRRx is the most advanced drug in our collaboration with GSK, and aside from KYNAMRO, it is also the furthest advanced in our late-stage clinical pipeline.
"The rapid development of ISIS-TTRRx from a research-stage program to a drug in a late-stage clinical trial is a testament to the efficiency and productivity of our drug discovery technology platform."
The 15-month long Phase II/III study is a randomized, double-blind, placebo-controlled, international study aims to support an application for marketing approval of ISIS-TTRRx in patients with FAP.
This will assess the effects of ISIS-TTRRx both on neurological dysfunction and quality-of-life.
It is also an investigational drug developed to reduce the production of all forms of TTR as well as to treat all types of transthyretin-related amyloidosis.