(By Balachander) ISIS Pharmaceuticals Inc. (NASDAQ: ISIS) said it has earned a milestone payment of $7.5 million from GlaxoSmithKline Plc (NYSE: GSK) related to the initiation of an antisense drug study.
Carlsbad, California-based ISIS said the Phase 2/3 study of ISIS-TTRRx - an antisense drug in development with GlaxoSmithKline to treat transthyretin (TTR) amyloidosis - is designed to support an application for marketing approval of ISIS-TTRRx in patients with familial amyloid polyneuropathy (FAP).
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ISIS said it is eligible to earn an additional $50 million in pre-licensing milestone payments to support the Phase 2/3 study of ISIS-TTRRx. The company is also eligible to receive regulatory and sales milestone payments and double-digit royalties on sales of ISIS-TTRRx.
The companies recently amended the clinical development plan and financial terms relating to ISIS-TTRRx to support this registration-directed Phase 2/3 clinical study on ISIS-TTRRx.
ISIS noted that TTR amyloidosis is a severe and rare genetic disease that leads to death. TTR amyloidosis affects roughly 50,000 patients worldwide and current treatments are limited.
"Patients with familial amyloid polyneuropathy, or FAP, experience TTR build up in their peripheral nerves and experience the loss of motor functions, such as walking," said Merrill Benson, Professor of Medical Genetics at Indiana University.
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By inhibiting the production of TTR protein, ISIS-TTRRx could offer an alternative approach to treating patients with FAP, Benson said. A drug that can reduce TTR levels could potentially slow or arrest progression of this terrible disease.
The U.S. Food and Drug Administration (FDA) granted ISIS-TTRRx fast track designation and orphan drug status for the treatment of FAP.
ISIS shares, which have been trading in the 52-week range of $7.02 to $15.61, ended at $14.43 on Friday.