The COO of RNAi specialist Alnylam ($ALNY) was critical of Big Pharma in the wake of Novartis' ($NVS) decision to slam the breaks on its RNAi research. The news made investors nervous about the future of the promising but nascent technology and Alnylam's stock price is down more than 20% in the last week.
"I think the best way to think about the news around RNAi versus the [scientific] progress is that big pharma--as evidenced by the story of recombinant DNA and monoclonal antibodies--has been a miserable barometer of high impact technologies," said Alnylam COO Barry Greene.
"Novartis pulling out is an exemplar of Big Pharma not being able to innovate, and historically they have never been able to innovate," he continued.
He maintained that the company is making progress in the field, saying, "We've made unbelievable progress with the translation of RNAi into what I believe is going to be an entirely new class of innovative medicines."
"Probably the best exemplar of that progress and the pipeline we're building is the deal we did with Genzyme," he said, referring to the Sanofi subsidiaries' move to purchase a 12% stake in his company for $700 million in January.
"I don't think anybody who understands the data and understands the state of the technology is nervous," he said.
But Novartis isn't the first Big Pharma company to pull out of RNAi research, which some say has the potential to create an entire new class of drugs, akin to monoclonal antibodies. Merck ($MRK) recently sold off its RNAi work to Alnylam for a fraction of what it had paid to get into the field, and Roche ($RHHBY) has exited the space as well.
The biggest technical challenge of the technology is drug delivery, Greene said.
"When we say delivery, we're talking about getting the siRNA [small interfering RNA], which is the drug that intermediates RNAi into the cytoplasm of the cell, which is where the RNAi machinery works." Alnylam's GalNAc-siRNA platform uses the sugar molecule GalNAc conjugated to the RNA molecule to enable subcutaneous delivery.
"We've created a conjugate platform where we're attaching a sugar moiety to specifically target the asialoglycoprotein receptor of hepatocyte," he said. "The delivery is now very elegant, and very targeted, and specific for hepatocytes [liver cells]."
Greene said Alnylam took a "pragmatic" approach and targeted liver cells because that is where the delivery challenges are most easily overcome. Novartis focused oncogenes, according to an Alnylam spokeswoman.
"Our approach was to figure the best place to develop RNAi. Big companies have different discovery research approaches. Sometimes they took more of a pathway approach or a franchise disease approach. So when you take those franchises, like oncology, you'll come up with oncogenes that are ubiquitously expressed, or expressed in tumor cells, and that's not where delivery is optimized. Delivery is optimized in the liver. So we've gone where delivery is optimized, and we think we're going after really important targets," Greene said.
Don't expect any Alnylam drugs to hit the market before 2017. Greene said the development program for Patisiran for the treatment of the orphan indication transthyretin-mediated amyloidosis is farthest along and should complete Phase III trials in 2017.
He blamed the decline in the stock price on the generally bearish sentiment on Wall Street regarding biotech stocks in particular.
Greene said Alnylam plans on updating investors and the scientific community about the results of its preclinical and clinical programs at a variety of scientific symposia in May.