Isis Pharmaceuticals, Inc. (NASDAQ:ISIS) announced that it has earned a $15 million milestone payment from GSK related to advancing the Phase 2/3 study of ISIS-TTRRx in patients with familial amyloid polyneuropathy (FAP). “We are very pleased with the progress we are making with ISIS-TTRRx, and plan to report data from the Phase 2/3 study in 2017. We have many patients who have completed the fifteen months of dosing and are now receiving ISIS-TTRRx in an open-label extension study,” said B. Lynne Parshall, chief operating officer at Isis Pharmaceuticals.
ISIS-TTRRx is an antisense drug Isis is developing with GSK for the treatment of transthyretin amyloidosis, a severe and rare genetic disease characterized by progressive dysfunction of peripheral nerve and/or heart tissues. Including this milestone payment, Isis has generated $60 million in upfront and milestone payments for advancing ISIS-TTRRx. In addition, if GSK elects to exercise its option to exclusively license ISIS-TTRRx, Isis is eligible to receive a license fee, regulatory and sales milestone payments and double-digit royalties on sales of ISIS-TTRRx.
The Phase 2/3 study of ISIS-TTRRx is a randomized, double-blind, placebo-controlled, international study designed to support an application for marketing approval of ISIS-TTRRx in patients with FAP. The fifteen month study will measure the effects of ISIS-TTRRx on neurological dysfunction and on quality-of-life. ISIS-TTRRx is a drug that is designed to inhibit the production of all forms of TTR, and to treat all types of transthyretin-related amyloidosis. For further study information, please visit www.clinicaltrials.gov and search for the identifier number NCT01737398.
Shares of Isis Pharmaceuticals closed last Friday at $68.56 . ISIS has a 1-year high of $75.24 and a 1-year low of $22.25. The stock’s 50-day moving average is $67.09 and it’s 200-day moving average is $51.96.
On the ratings front, Isis Pharmaceuticals has been the subject of a number of recent research reports. In a report issued on February 17, Deutsche Bank analyst Alethia Young reiterated a Buy rating on ISIS, with a price target of $80, which represents a potential upside of 16.7% from where the stock is currently trading. Separately, on February 4, Piper Jaffray’s Joshua Schimmer maintained a Hold rating on the stock and has a price target of $68.
In total, 2 research analysts have assigned a Hold rating and 4 research analysts have given a Buy rating to the stock. When considering if perhaps the stock is under or overvalued, the average price target is $68.56 which is 2.8% above where the stock closed last Friday.
Isis Pharmaceuticals is engaged in antisense drug discovery and development, exploiting a novel drug discovery platform it created to generate a broad pipeline of first-in-class drugs.