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 a trial of ALN-PCS, which is designed for patients with severely high cholesterol who don’t respond to traditional statins. “We think these are going to be pretty important for the whole RNAi field,” Maraganore says.
The first hurdle, though, is the ASCO conference. ALN-VSP02 targets two genes that promote the growth and development of cancer cells: vascular endothelial growth factor (VEGF) and kinesin spindle protein (KSP). This drug is important for Alnylam not just because of its potential value against liver cancer, but also because it is the company’s first RNAi treatment designed to circulate throughout the bloodstream—the way it hopes to deliver other experimental treatments in the future. The abstract for the trial of ALN-VSP02, which was published May 18, says the drug is well tolerated and is showing activity “consistent with an anti-VEGF effect” that it is shooting for. Maraganore says further details on the trial—and the patient who has been on the drug for 12 months—will be provided at the meeting.
He hopes pharmaceutical executives will be paying attention, because Alnylam is looking to ally with another company to take the drug into Phase 2 clinical trials. “ASCO will be important for that program’s partnership potential,” he says. “For Alnylam it’s also important, because in a sense, it’s a bell-ringing for this drug class.”
Maraganore points out that there are still plenty of drugmakers interested in RNAi. Alnylam has ongoing partnerships with such pharma giants as Takeda Pharmaceuticals, GlaxoSmithKline (NYSE: GSK), and Cubist Pharmaceuticals (NASDAQ: CBST). And Merck (NYSE: MRK), which was an early investor in the technology, is still researching RNAi therapeutics. “Other companies remain committed to this field,” he says.
As for the drop of Alnylam’s stock, Maraganore isn’t worried. “Obviously it caused anxiety, but for Alnylam it hasn’t changed a thing,” he says. The company has $343 million in cash on its books, placing it in the top 12 in the biotech industry in net cash positions, according to Alnylam’s internal analysis. It helped that when Roche came aboard in 2007, Alnylam got $331 million right off the bat. “That was an enormous amount of money up front,” Maraganore says.
Maraganore can’t comment on the Tekmira suit because of the ongoing dispute. At the time, he told Xconomy that the suit was a “big surprise” and the allegations were “completely untrue.” The dispute hasn’t disrupted the manufacturing partnership the two companies have on ALN-VSP02, he says.
The ASCO conference will also feature a presentation by Silence Therapeutics, a UK-based company that’s developing an RNAi drug to treat solid tumors. Maraganore hopes that data will help build positive attention for the beaten-down technology. “These are evolutionary trials,” he says. “But we’re pretty excited. As these bells get jingled, increasingly it will be a resounding sound.”
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