Alnylam Racing to Prove RNAi Works, For a Disease You’ve Never Heard Of

Xconomy Boston — 

Alnylam Pharmaceuticals has been in the doghouse for a while now, as its technology of choice has fallen out of favor in the industry. But while many people have written it off, the Cambridge, MA-based company has started to show hints that it might—just might—be onto something that works against a rare and deadly disease.

The company (NASDAQ: ALNY) reported last month at a scientific meeting that one of its experimental RNA-interference drugs was able to shut down production of a protein that causes a disease called TTR amyloidosis. The findings, from an early-stage clinical trial of 31 patients, marked the first time that Alnylam has been able to show that an RNAi treatment can specifically block a disease-causing protein in human beings.

The potential of RNA interference has electrified scientists for years, because it offers the potential of blocking disease targets inside cells that can’t be effectively halted by conventional small-molecule or larger biotech drugs. But the excitement has faded the past couple years, as researchers have struggled to deliver these RNA-silencing molecules into cells, and major companies like Roche, Merck, and Novartis have cut back on their investments in the field. Through it all, Alnylam CEO John Maraganore has insisted that the only way to overcome the doubters is to deliver convincing proof from clinical trials, not just mouse or monkey experiments.

Alnylam has made some headway in the clinic the past couple years, and while it’s too early to say it has proved the skeptics wrong, it’s moving full steam ahead on this new flagship program, betting that it will prove that RNAi will someday live up to its promise.

“The level of confidence for us at Alnylam, and among our collaborators, has gone up enormously as a result of this data,” Maraganore says.

Based on the clinical findings (which I’ll get to in a minute), the company has raced ahead with a more potent-second generation delivery technology of its TTR drug, which it plans to move into its first clinical trial in early 2012. The plan is then to move ahead at breakneck speed by wrapping up that study before year’s end, starting a mid-stage study later in 2012, and then leaping all the way into the third and final phase of clinical trials normally required for FDA approval in 2013, Maraganore says.

There are scientific and business reasons why Alnylam has honed in on TTR to be its flagship program. The disease, transthyretin (TTR) amyloidosis, allows excessive amounts of amyloid proteins to build up in tissues throughout the body. Alnylam likes going after this disease partly because … Next Page »

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