ISIS Pharmaceuticals, Inc. (NASDAQ:ISIS), a developer and marketer of RNA-based novel drugs to treat a variety of diseases, has been one of the best performing stocks in the biotechnology sector this year. Year-to-date, the stock has gained almost 250%. Despite the strong gains, there are reasons to believe that Isis Pharmaceuticals still has growth potential. There are four reasons that make Isis Pharmaceuticals an attractive proposition.
Isis is a position in antisense technology. The technology has been designed to alter a gene’s function, silence a mutation, or activate a gene to compensate for an underlying genetic defect. Antisense technology makes it possible to synthesize a strand of nucleic acid (DNA, RNA or a chemical analog) that binds to the messenger RNA (mRNA) produced by the gene and effectively turn that gene on or off. With antisense technology, Isis has been able to generate a broad pipeline of first-in-class drugs. One of the biggest advantages of the antisense platform is that it reduces costs and time to discover and develop drugs when compared to traditional small molecule drugs.
Collaboration with Genzyme for Kynamro
Isis’ lead product candidate is Kynamro for the treatment of patients with homozygous familial hypercholesterolemia, or HoHF. The company has partnered with Genzyme, a subsidiary of French drugmaker Sanofi SA (ADR) (NYSE:SNY), to commercialize Kynamro in the U.S. Genzyme is also pursuing marketing approval for the drug in other markets. An extremely rare disease, HoHF occurs in one out of every 1 million people. However, the actual prevalence of the disease might be underestimated. Currently, some 6,000 people worldwide suffer from HoHF.
Currently, there is just one other approved drug for the treatment of HoHF: Juxtapid from Aegerion Pharmaceuticals, Inc. (NASDAQ:AEGR). Juxtapid was launched in January this year after the Food and Drug Administration approved the drug in December 2012. The once-daily pill costs about $295,000 a year. This is where Isis’ Kynamro has an edge. Kynamro, which is an injectable treatment, will cost $176,000 a year.
In addition to costs, Isis’ patents offer strong and extensive protection for the drug and the technology. The FDA has already approved the new drug application for Kynamro in January.
Strategic collaboration in neurology
This month, Biogen Idec Inc. (NASDAQ:BIIB) and Isis entered into a strategic collaboration to advance treatment of neurological disorders. Under the terms of the six-year research collaboration agreement, Biogen will have the exclusive rights to use Isis’ antisense technology to develop therapies for neurological disorders.
