GSK opts out of rare disease drugs under Ionis deal

14 August 2017

Shares of Ionis Pharmaceuticals (Nasdaq: IONS) fell 6.7% in pre-market trading on Friday, but recovered for a 2.6% rise to $46.76 by close of business, after it announced that UK pharma major GlaxoSmithKline (LSE: GSK) was pulling out of a development deal on two drug candidates.

As part of a reprioritization of its pipeline and strategic review of its rare diseases business, GSK declined its options on inotersen and IONIS-FB-LRx, meaning that Ionis, a California-based biotech, now retains rights to these.

Ionis plans to file for marketing authorization for inotersen this year to support a commercial launch of the product in 2018. Inotersen is a drug designed to treat patients with TTR amyloidosis (ATTR). The first indication Ionis is pursuing for inotersen is to treat patients with polyneuropathy due to hereditary TTR amyloidosis (hATTR-PN).

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