Alnylam Pharmaceuticals has a technology, RNA interference, that scientists say has potential to break new ground against hundreds of diseases for decades to come. But when you have a proverbial “platform” technology that can do so much, how exactly should you set priorities, and decide which drugs to develop first?
I got a sense for how Alnylam CEO John Maraganore thinks about this a few weeks ago when I visited his office in Cambridge, MA, and asked about a treatment his company is developing for something called TTR amyloidosis. I’m sure he’ll have more to say about this when he appears today at an Xconomy event on life sciences innovation in San Diego.
Alnylam (NASDAQ: [[ticker:ALNY]]) has made waves the past few years as the leading company developing RNA interference drugs, which have the potential to specifically silence disease-related genes that have been inaccessible to drugs of the past. Alnylam has chosen to remain small, with just 180 employees, while pursuing many opportunities through its network of partnerships with big drugmakers like Roche, Novartis, GlaxoSmithKline, and others. The partnerships are critical, because they provided early validation for Alnylam’s technology, along with more than $600 million in cash, and helped it build a stock market valuation of more than $680 million. That’s all helped Alnylam advance two RNAi treatments into clinical trials, one for respiratory syncytial virus and another for liver cancer.
But the third drug in Alnylam’s pipeline, ALN-TTR, reveals another kind of strategy at work at Alnylam, one that looks a lot like the trail blazed twenty years ago by Cambridge, MA-based Genzyme. This is the model that says it’s better to retain 100 percent of the commercial rights to drugs that can make a big difference for a small number of patients with a severe genetic disease, and command premium prices for those drugs. ALN-TTR is one drug Alnylam wants to keep to itself, at least in the world’s top two pharmaceutical markets, the U.S. and Europe.
“This is an area we can build our company around,” Maraganore says.
The disease, transthyretin (TTR) amyloidosis, appears in patients with a mutation of a gene, concentrated in the liver, that allows excessive amounts of amyloid proteins to build up in the body. People are born with the condition, but usually don’t see symptoms until their 30s or 40s. Once symptoms appear, people suffer progressive damage to the nerves and the heart. The only treatment is a liver transplant. People usually die about nine to 11 years after symptoms appear, Alnylam has said.
About 10,000 people worldwide have the nerve-damaging form of the disease (familial amyloidotic polyneuropathy) and an estimated 40,000 worldwide have the heart-damaging effect (familial amyloidotic cardiomyopathy). That’s a small population in the global scheme of things, but Maraganore notes that only about 10,000 people worldwide have Gaucher’s disease, and that still became the basis for a billion-dollar annual market for Genzyme’s imiglucerase (Cerezyme).
But why does TTR amyloidosis make an attractive target from a science standpoint? Maraganore zipped through the logic. The disease symptoms are clearly linked to the gene mutation, without
