The payment primarily relates to initiation of a phase II/III clinical study for ISIS-TTRRx. The study will span 15 months enrolling 200 patients. Isis is developing ISIS-TTRRx in collaboration with GlaxoSmithKline for the treatment of transthyretin (TTR) amyloidosis.
Amyloidosis is a genetic disease, which is typically characterized by progressive dysfunction of peripheral nerve and/or heart tissues.
We remind investors that Isis entered into a strategic alliance with GlaxoSmithKline in Mar 2010. The alliance, covering up to six programs, will primarily develop new drugs for rare and serious diseases, including infectious diseases and some conditions causing blindness.
In 2011, Iris initiated a phase 1 clinical study on ISIS-TTRRx as part of its collaboration with GlaxoSmithKline. In Oct 2012, Isis and GlaxoSmithKline amended the original agreement to accelerate the clinical development for ISIS-TTRRx.
Under the amended agreement, Isis has received an upfront payment of $2.5 million and $7.5 million as milestone payment following the initiation of the phase II/III study on ISIS-TTR Rx. Isis will also be entitled to receive an additional $50 million in pre-licensing milestone payments associated with the phase II/III clinical study.
We note that the candidate received fast track designation from the US Food and Drug Administration (FDA) and orphan drug status for the treatment of FAP, a type of TTR amyloidosis.
Isis already received approval for Kynamro (mipomersen sodium) injection in Jan 2013. Kynamro, Isis’ lead pipeline candidate, is a second generation compound being developed for the treatment of familial hypercholesterolemia (FH) with Sanofi (SNY).
Isis Pharma currently carries a Zacks Rank #3 (Hold). Medivation Inc. (MDVN) looks more attractive in the pharma sector with a Zacks Rank #2 (Buy).