Alnylam Seeks to Prove RNAi Skeptics Wrong After Layoffs, End of Novartis Deal

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liver cancer. This trial is primarily to show whether the drug is safe for people. But the drug is also the first RNAi treatment that the company is using to circulate throughout the body to treat cancer. The gene-silencing drugs are notoriously difficult to deliver deep inside the body because they are made of short pieces of RNA, which can be chewed up in the blood stream before they reach their intended destination inside cells to block the activity of disease-related genes. [RNAi drugs were mistakenly described as short pieces of DNA. They should be described as short pieces of RNA.]

To deliver its liver cancer drug, Alnylam is using engineered particles licensed from Tekmira Pharmaceuticals in Vancouver, BC. Alnylam has already shown that its drugs can be delivered safely to the livers of non-human primates and other lab animals. Time will tell how well this success translates in humans. Alnylam is also using Tekmira’s technology for a separate drug in a Phase I study for treating TTR amyloidosis, and the drug targets the liver to essentially turn off the gene responsible for a mutant protein that causes the disease.

Alnylam CEO John Maraganore

Alnylam CEO John Maraganore

Leerink Swann, the Boston-based banking and analyst firm, said in a September 27 note to investors that they lowering their valuation of Alnylam from $20 to $14 per share “given the slow progress in solving RNAi delivery plus our remaining uncertainty as to whether liver-targeting RNAi therapies can be delivered safely.”

Partly because of the challenge of delivering RNAi therapies throughout the body, Alnylam has picked a localized delivery vehicle for its lead drug program. Alnylam’s most advanced candidate, ALN-RSV01, is inhaled into the lungs to treat respiratory syncytial virus infections.

Maraganore contends that concern over RNAi delivery might be blown out of proportion. “We’re beyond the point of any reasonable line of questioning as it relates to our ability to deliver these molecules,” the CEO says. “We are now at a stage of execution around those discoveries and technologies. So it’s no longer, in my mind, conceivable that this basic approach will never create medicines.”

Alnylam has had no shortage of partners that are willing to bet that RNAi can live up to its promise, which includes the ability to target genes that traditional small molecule drugs haven’t been able to hit. There’s no question Alnylam has a lot of support. The company hasn’t needed to raise money from the public markets since 2006 because it’s generated enough cash to keep growing from its partnerships with large drug makers, Maraganore says.

But while $325 million in the bank sounds like a lot, Alnylam will likely need even more financial help from its pharma friends to complete the long and expensive journey toward FDA approval.

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