Alnylam Looks to ASCO as First “Bell-Ringing” for RNAi Resurgence

5/26/11Follow @arleneweintraub

When Cambridge, MA-based Alnylam (NASDAQ: ALNY) made its debut last year at the annual American Society of Clinical Oncology (ASCO) conference, it only had a pittance of safety data on its drug, which is designed to treat advanced solid tumors that have spread to the liver. At this year’s conference, which starts June 3 in Chicago, Alnylam CEO John Maraganore hopes the company will make more of a splash, especially since one of the patients in the trial being presented has been on its cancer drug for more than a year.

Maraganore can’t reveal much about that patient until the ASCO presentation on June 4, but it’s clear he’s excited she’s done well on the experimental drug, called ALN-VSP02. “This patient failed all other therapies,” he says. “As long as she’s responding, she’ll continue to receive our drug.”

ASCO could end up being a much-needed bright spot in what has been a tough year for Alnylam. The company has three drugs in clinical trials based on the technology RNA interference (RNAi), a once-hot technique that involves silencing key genes involved in a range of diseases. But last September, Novartis ended a partnership with Alnylam. Two months later, Roche pulled out of RNAi all together, which also involved axing a program with Alnylam.

Then in mid-March, seemingly out of the blue, Alnylam partner Tekmira (NASDAQ: TKMR)—which which provides a key nanoparticle technology used in ALN-VSP and other RNAi drugs—sued Alnylam for trade-secret violations. All told, Alnylam’s stock has lost 34 percent of its value during this string of events, falling from nearly $15 last September to a recent $9.68 at yesterday’s close.

But Maraganore hasn’t lost faith in Alnylam—or in RNAi as a potential game-changer. “I’ve seen this play out before,” says Maraganore, who met with Xconomy New York when he was in town on May 25 for the ThinkEquity Healthcare Conference. He points out that the pharmaceutical industry once abandoned monoclonal antibodies. Then the biotech industry picked up the ball and monoclonals became the fastest-growing category in all of pharma. “The driver for RNAi is going to be clinical data,” Maraganore says. “When it happens, you’ll see pharma come back.”

Maraganore says that the Phase 1 data being released at ASCO will be the first of three key datasets Alnylam plans to release this year. In the third quarter, the company will present data from its trials of ALN-TTR, which treats a rare hereditary disease called TTR-mediated amyloidosis. And in the fourth quarter, it will release clinical data from … Next Page »

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  • Homer Yeh

    I believe it is worth to give RNAi a trial from molecular point of view since cancer is a molecular disease. From theorectical point of view, using RNAi for gene silencing is nothing wrong but a perfect approach. But unlike monoclonal antibodies that tackles antigenic epitodes that are located on the surfaces of the cells. Those mutated genes are usually located deep in the nuclear of malignant cells, having problems of accessibility. But that is not a problem that can’t be overcome. It may take a little while to find ways of problem solving. In math, key to the problem solving usually is located in the problem itself. Good luck! Ms. Maraganore!

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