Shoppers- Attention Please! Marina Bio High-Affinity Nucleotides On Sale

Why pay tens of millions to ISIS Pharmaceuticals or Santaris for modified nucleic acid chemistry when you can get the same, if not better from Marina Bio for just a million?

This is the question that Novartis probably was answering when they non-exclusively licensed CRN technology (for single and double-strand oligo Rx) from Marina Bio for just $1 million in upfront considerations.  CRN stands for conformationally restricted nucleotides, similar in shape and thermodynamic behavior to Santaris’ LNA technology, a technology that is also being researched by ISIS Pharmaceuticals.

To me, CRN has been the best bet for Marina Bio's corporate survival.  The reason is that selling a nucleotide modification, especially for antisense therapeutics should be fairly straight-forward: there is no formulation involved requiring support by a functioning laboratory, just include it in standard phosphorothioate oligos like everybody else.  This comes in handy when you have become a largely virtual, IP-based biotech company like $3M market cap Marina Bio.   

I had wondered whether the reason why Marina Bio had not been able to capitalize on this opportunity so far was either due to IP uncertainties or manufacturing issues with this novel nucleotide.  The latter issue seemed to have been resolved when Marina in May of this year struck a cGMPsupply agreement with Girindus. The IP issue I have to admit I have not researched in too much detail, but suffice it to say that my understanding of Santaris’ and ISIS’ CRN patents is that they are quite structure-specific which would move the IP issue more to questions surrounding their use in general (e.g. in gapmers and the like).  

Today’s announcement also confirms that Novartis is still in the game of oligonucleotide therapeutics after picking their 30+ targets from Alnylam and a product-specific deal on an RNAi Therapeutics from Quark.  Although $1M is not much to a Big Pharma, nobody wants to part just like that with a million, even if it means being able to rescue old friend Michael French (now for the second time after the 2009 mdRNA cash crunch, Marina Bio’s predecessor).

Today’s deal further fuels the rekindled fire of commercial small RNAs:

1)      impressive clinical results by Alnylam facilitated by Tekmira’s technology;

2)      penny stock investors multiplying their money following the Medicare coverage announcement for Rosetta Genomics' Cancer of Unknown Primary miRNA diagnostics;

3)      Rosetta Genomics gearing up for the commercialization of the test in what is a whopping $>25M offering for the company;

4)      new investors in Silence Therapeutics doubling and tripling their money overnight in a new share offering;

5)      and the Tekmira-Alnylam trade secret theft show-down coming to a widely followed climax.

Big investors may be jeering at such nanocap biotech companies, but it is a fact of life that most small silencing RNA companies have become pennystocks, which also means that their resuscitation could make for some nice investment returns.