Small interfering RNA (siRNA) is a relatively new kind of technology that is being developed into a new generation of therapeutics that targets diseases in a very novel way. While traditional therapies often target proteins through direct inhibition, manipulation of RNA through small interfering molecules can inhibit the production of these proteins, which removes some of the barriers that stood in the way of traditional inhibitors.
siRNA has received a fair amount of attention from biotech investors in recent years, although I think that many companies in the space remain undiscovered. One notable example is Tekmira Pharmaceuticals (NASDAQ: TKMR), which will be one of the major players in the iRNA space as the technology is developing into more therapeutic products targeting more indications.
One fairly recent and particularly notable display of the power of RNA inhibition was seen when Sarepta Therapeutics (NASDAQ: SRPT) published the results of a phase II trial for the drug eteplirsen (AVI-4658) in the treatment of Duchenne Muscular Dystrophy (NYSE:DMD).
Although patient enrollment into the trial was miniscule (12) due to the small population of the target indication, we saw very significant slowdowns in the deterioration of dystrophin fibers in these patients on top of significant improvements in secondary measures like 6-minute walk test (6-MWT) distances and lymphocyte counts that also show that the drug had a noticeably beneficial effect. While it's still being debated whether or not eteplirsen will become an FDA-approved drug on these results alone, I also see it as a very encouraging sign for all RNA-based therapeutics going forward.
If Sarepta was able to see such stellar results in a previously untreatable and degenerative disease, what else can RNA therapies do for other indications that traditional drugs struggle with? Although their RNA technology is a bit different, Tekmira is one of a handful of companies that is focusing entirely on this with its developmental pipeline.
They currently have five programs in preclinical or phase I development, and have partnerships for another five drugs. The three partnered programs that catch my attention are ALN-VSP, ALN-TTR, and ALN-PCS which are three products being developed with the larger iRNA company Alnylam Pharmaceuticals (NASDAQ: ALNY) for the treatment of amyloidosis, solid tumors, and hypercholesterolemia respectively.
It's also encouraging to see that Tekmira also holds the rights to five pipeline compounds that target very diverse indications, with my personal favorite being TKM-PLK1 which has demonstrated promising anti-tumor activity in animal models.
While we don't understand the full implications (and limitations) of this new RNA interference technology, the signs that I've seen thus far are very encouraging for both biotech investors and millions of patients that will need next-generation therapeutics that tackle diseases from a different angle.
The main problem for iRNA developers now is the overall lack of clinical trial data supporting the technology, although progress is being made at a slow (but steady) pace. I think that biotech & small pharma investors that are interested in holding stocks with a long-term outlook should definitely investigate iRNA companies like Tekmira, who are at the very front of the RNA revolution and have intellectual property rights to iRNA development platforms that could be worth significantly more if the industry really does take off.
It's because of this in particular that these companies may have a better-than-average chance of being acquired by larger pharmaceutical companies in coming years, as cash-rich big pharma sees dwindling potential in more traditional drugs and development platforms and look to newer sectors for growth potential.
Tekmira may garner a lot more interest from Wall Street next week as it presents the results of its phase 1 dose-escalation trial for its oncology therapy TKM-PLK1 at AACR 2013 (American Association for Cancer Research), which is scheduled to take place on April 9th, 2013. The abstract; however, should be available as early as April 5th, 2013.
I think this may provide some liquidity and possible upside potential if this event garners some much-needed attention for Tekmira throughout the month.