News Feature | June 26, 2014

Isis Receives $1M Milestone From GSK For FAP Drug

By Estel Grace Masangkay

Isis Pharmaceuticals announced this week that it has earned a $1 million milestone payment from GlaxoSmithKline after advancing its investigational drug ISIS-TTR Rx in a Phase II/III study in patients with familial amyloid polyneuropathy (FAP).

Antisense drug ISIS-TTR Rx is currently under development with Isis collaborator GSK to treat the rare genetic disease transthyretin amyloidosis. People suffering from the disease experience progressive dysfunction of the peripheral nerve and/or the heart tissue. The company has completed a Phase I study establishing the investigational drug’s safety and clinical activity in healthy volunteers. In February 2013, the Phase II/III study of the drug was launched to assess efficacy of the drug in FAP patients, for which the company received a milestone payment of $7.5 million from GSK.

The milestone payment is the fourth of the $50 million in milestone payments GSK agreed to pay Isis as the Phase II/III progresses. Isis initially received $26 million in upfront and milestone payments for advancing the investigational drug, which has also received a positive opinion from the Committee for Orphan Medicinal Products (COMP) to designate ISIS-TTR Rx as an orphan drug.

B. Lynne Parshall, COO at Isis, said, “We are very pleased with the progress we are making with ISIS-TTR Rx. In addition to being on schedule for enrollment, we have initiated an open-label extension study of ISIS-TTR Rx, which is being offered to those patients with FAP who have completed dosing in the Phase 2/3 study of ISIS-TTR Rx.”

The double-blind, randomized, international, placebo-controlled Phase II/III study will help form the basis for the application of a marketing approval of ISIS-TTR Rx in patients with FAP. The trial will be conducted for fifteen months and will measure the effect of the drug on neurological dysfunction and on quality-of-life of patients.

If GlaxoSmithKline chooses to use its option to gain exclusive license of the ISIS-TTR Rx program, Isis will receive additional license fee, regulatory, and sales milestone payments, and double-digit royalties on sales of the drug.