Some people are completely fascinated with fashion trends and the latest jeans highlighted in GQ. For those of you who are fascinated by the other type of genes, this one's for you!
RNA aye aye aye!
Specifically, I want to take a closer look at biotechnology companies that are focused on RNA interference therapies, which I feel could become an area of large financial interest and research in the coming years -- especially with big pharmaceutical company pipelines aging and many willing to reach out to new fields for growth.
RNA interference is a biological process that can modulate the expression of proteins in the body. The process that's most often targeted involves something as simple as whether a protein is produced by interfering with the messenger RNA that would translate that specific sequence. The possible treatment areas for RNAi therapies range from treating viruses to helping treat cancer.
One downside of RNAi therapies is that they're still in the early stages of their existence, so their usefulness, side effects, and the ability to target specific sequences (since some sequences are very similar) still pose a challenge. Merck (NYSE:MRK), for instance, spent $1.1 billion in 2006 to acquire Sirna Therapeutics, but last year decided to shut down its RNA interference research facility. Merck will continue to utilize Sirna's RNAi technology throughout its R&D processes, however. The failure of Marina Biotech (formerly MDRNA) to deliver a successful RNAi-based drug is another example of the noted struggles within the sector to bring a viable RNAi drug to market.
The one name to know in RNAi
One company, though, could have the fast-track to success in RNAi therapeutics, if not for its diverse pipeline but because of the lack of competition.
Alnylam Pharmaceuticals (NASDAQ:ALNY) is the leader in developing RNAi therapies with its 5x15 strategy, which it unveiled last year. Alnylam is focusing on areas of treatment with largely unmet needs, and plans to have five RNAi-based drug hopefuls in late-stage trials by 2015 (ergo "5x15"). But, even Alnylam hasn't had a perfect go of things, with its phase 2b clinical trial for ALN-RSV01 -- developed in collaboration with Cubist Pharmaceuticals (NASDAQ:CBST) and targeted at treating progressive bronchiolitis obliterans syndrome -- failing to meet its endpoint. Yet the remaining pipeline, and even ALN-RSV01 for that matter, still shows promise.
In 2008, Japan's Takeda Pharmaceuticals made what could amount to a $1 billion bet on Alnylam's success, which included $100 million in upfront cash, as well as multiple future milestone payments assuming drug approval within its RNAi therapy line of treatments. Alnylam has other collaborations in place with Roche, Novartis, and Biogen Idec. These speak volumes to the funding that companies are willing to throw at RNAi research.
Alnylam's pipeline (unsurprisingly) has five areas of focus, including transthyretin-mediated amyloidosis, hemophilia, hypercholesterolemia, refractory anemia, and hemoglobinopathies. Three of these five focus areas are still in development, with only its TTR-amyloidosis treatment and hypercholesterolemia treatment advancing to clinical trials (currently phase 2 and end of phase 1, respectively). These studies are still early; however, they offer incredible promise and a lot of financial backing.
But don't forget these two names, either...
Although Alnylam is the most direct play on RNAi therapeutics, both ISIS Pharmaceuticals (NASDAQ:IONS) and Sangamo BioSciences (NASDAQ:SGMO) offer similar treatment pathways as RNAi and shouldn't be discounted, either.
ISIS Pharmaceuticals might ring a bell as its lead drug candidate -- Kynamro, for the treatment of homozygous familial hypercholesterolemia, or HoFH -- went before the Food and Drug Administration advisory panel last week and received a tentative nod of approval by a vote of 9 to 6, despite concerns with regard to fat buildup in the liver of treated patients, and the occurrence of cancerous growths in 3.1% of all tested patients.
What's really unique about ISIS' method of treatment is that it utilizes antisense technology in many cases to bind to messenger RNAs and inhibit the production of disease-causing proteins. Kynamro is just one example of this -- ISIS actually has a ridiculously impressive pipeline featuring 26 (yes, 26!) drug hopefuls, of which 21 are in some stage of clinical trials. That's strength in numbers!
Sangamo BioSciences doesn't have anywhere near the pipeline that ISIS has; its lead drug candidate -- SB-509, targeted at diabetic neuropathy -- failed to reach its endpoint in mid-stage trialslast year. It currently has just one hopeful in clinical trials and a handful of others in pre-clinical trials, but the potential for its ZFP technologies is, for lack of a better word, "cool!" According to Sangamo's website, the company uses "engineered zinc finger DNA-binding transcription factors to 'up-regulate' or 'down-regulate' gene expression to achieve a desired therapeutic effect." Similar to RNAi, but working at a different point in the protein production pathway, Sangamo uses zinc finger nucleases to disrupt protein synthesis. The company has a collaborative partnership with Shire in the focus fields of hemophilia and Huntington's disease, and could draw more interest assuming it can bring more than just one drug to clinical trials.
Advancing toward a cure
Now it's a matter of seeing whether Alnylam, and/or the RNA-similar twins, ISIS and Sangamo, can turn their revolutionary pipeline products into FDA-approved treatments.
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Fool contributor Sean Williams has no material interest in any companies mentioned in this article. You can follow him on CAPS under the screen name TMFUltraLong, track every pick he makes under the screen name TrackUltraLong, and check him out on Twitter, where he goes by the handle @TMFUltraLong.
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