What: Shares of Isis Pharmaceuticals (NASDAQ:IONS), a biopharmaceutical company focused on developing therapies using its proprietary antisense technology, surged 19% in December, according to data from S&P Capital IQ, after releasing a trio of positive clinical data.
So what: Perhaps no tidbit of clinical data stood as more important than its phase 2 data on ISIS-FXIrx for the prevention of venous thrombosis in patients that underwent total knee replacement surgery. ISIS-FXIrx is an experimental drug designed to reduce the production of Factor XI, which plays a role in coagulation. Ultimately the results showed a seven-fold lower incidence in venous thrombosis in ISIS-FXIrx-treated patients than those who took enoxaparin, a commonly prescribed anticoagulant. Isis noted in its press release that it planned to bring in a partner to help with the costs of later-stage development.
In early December, Isis published the results from its midstage study involving ISIS-APOCIIIrx in patients with Familial Chylomicronemia in The New England Journal of Medicine. The three-patient study highlighted how ISIS-APOCIIIrx-treated patients saw their baseline triglyceride levels fall from a range of 1,406 mg/dL - 2,083 mg/dL down to less than 500 mg/dL during the study.
Lastly, Isis excited shareholders by announcing that it was initiating a phase 1/2 study involving ISIS-DMPKrx for patients with myotonic dystrophy, the most common form of muscular dystrophy. The drug is being developed in cooperation with Biogen Idec, and is designed to target the genetic defect that leads to myotonic dystrophy type 1.
Now what: The story with Isis is pretty much the same month in and month out: data, data, and more data! Isis is working with a pipeline that's nearly three dozen compounds deep and its partner list has grown to around a dozen. By not being shy about seeking funding and expertise, Isis has amassed ample ways to monetize its technology to fund additional research while leaving the eventual logistics of marketing a drug to its partners.
As I've often said, Isis' pipeline is like stepping up to home plate during a home run derby. You're going to get about 35 pitches to hit two or three home runs -- and that's all it would really take to justify Isis' current valuation. With nearly unmatched diversity for a biotech stock of its size, I continue to believe its shares have upside over the long run.