First GSK milestone for Isis as antisense amyloidosis drug progresses
This article was originally published in Scrip
With the initiation of a Phase II/III clinical trial for the antisense drug ISIS-TTRRx as a treatment for transthyretin (TTR) amyloidosis Isis Pharmaceuticals earned a $7.5 million milestone payment from GlaxoSmithKline under the companies' 2010 alliance to develop RNA therapeutics for rare and infectious diseases.
GSK and Isis expect the late-stage trial for ISIS-TTRRx to support regulatory applications for the drug, which has been granted both fast-track and orphan drug designations by the US FDA for the treatment of familial amyloid polyneuropathy (FAP). ISIS-TTRRx has moved from a research-stage asset to a late-stage program in just over two years, according to Isis chief operating officer Lynne Parshalle.
TTR amyloidosis is a rare genetic disease characterized by progressive dysfunction of peripheral nerve and/or heart tissues. Patients with FAP have TTR build-up in their peripheral nerves and experience loss of motor skills, including walking, as a result, Indiana University medical genetics professor Merrill Benson explained in a statement from Isis.
Dr Benson said that if a drug, such as ISIS-TTRRx, were to reduce TTR levels it "could potentially slow or arrest progression" of FAP.
Isis said the company and GSK recently amended their clinical development plan and financial terms related to ISIS-TTRRx to support the Phase II/III study for the antisense drug.
The allies signed an agreement during the first quarter of 2010 that gave Isis $35 million upfront in return for GSK's option to license up to six compounds after Phase II proof-of-concept studies (scripintelligence.com, 31 March 2010). Isis stands to earn up to $1.5 billion in license fees and milestone payments plus double-digit royalties under the alliance.
For ISIS-TTRRx, Isis is eligible to earn another $50 million in pre-licensing milestone payments in addition to the $7.5 million fee tied to initiation of the Phase II/III clinical trial. Isis also is entitled to certain other regulatory and sales milestone payments plus royalties related to development of the amyloidosis therapy.
Isis will continue to manage clinical development of ISIS-TTRRx with input from GSK.