Alnylam Maps Out First Steps in ‘RNA Decade’
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metabolizing so-called “bad” LDL cholesterol. The company’s drug candidate has been shown to reduce LDL cholesterol by more than 50 percent in primate studies. That candidate is being primed to enter its first clinical trial before the end of 2011, Maraganore says.
—Alnylam expects to do “a couple” new partnerships this year, although it’s not promising specific number to investors like it did in 2009, Maraganore says. While Wall Street likes to count on a specific number of deals in a given time frame, that also can take away a bit of leverage when a prospective partner can sense that the other side is under pressure to take whatever’s on the table by a certain deadline. Alnylam doesn’t want to give away any of that leverage this year by promising a fixed number of deals, Maraganore says.
Even though Alnylam didn’t deliver any major new partnerships in 2009 as it forecasted, the Big Pharma companies still have a strong appetite for Alnylam’s RNA interference technology, Maraganore says. “It’s [pharma interest] never been stronger,” Maraganore says. “We are having conversations at the CEO level all the time. They are increasingly convinced that RNAi is a disruptive technology, and that Alnylam is a leading, if not the leading, company.”
—Like last year, Alnylam has set a goal of getting at least 15 scientific papers published in top quality, peer-reviewed journals. That’s important for the company to keep its research sharp, even though it may not be enough to move the needle anymore among shareholders. “Investors want to see proof of concept in the field,” Maraganore says. “The goalposts are moving, and that’s OK. First it was animal data. Now they want to see human data. Then it will be about the marketed products and how they do. The goalposts are always moving, and we’re ready for it.”
—The intellectual property battles certainly aren’t over either. Alnylam expects to receive at least 30 new patents this year to strengthen the position it wants to hold, as a gatekeeper that RNAi drug developers need to pay in order to get in the game or stay in it.
—Last but not least, this will all take a lot of cash. Alnylam forecast a year ago that it would ring in 2010 with about $435 million of cash and investments in the bank, and now it says it ended 2009 just about right on target, with a little more than $430 million. It expects to tap a big chunk of that stockpile, and finish this year with at least $325 million, Maraganore says. But that number is clearly a conservative estimate, because Alnylam isn’t counting on a $100 million fee that Novartis may elect to pay to get a non-exclusive license to continue developing the Alnylam RNAi technology on its own, Maraganore says. The $325 million cash guidance figure also doesn’t assume that Alnylam will secure any major partnerships, he says.
Before we hung up, I asked Maraganore about his predictions on what will happen in the external world, beyond his control, that will have a big effect on Alnylam’s year. He conceded that he was worried a year ago when industry estimates said one-fourth to one-third of all biotech companies were operating on less than a year’s worth of cash. Most of the companies in that precarious position found some way to survive, either by cutting costs, or by producing enough clinical trial results that they could raise more money. “In general, the industry has been resilient, and made it through a tough period,” Maraganore says.
That led him to his one big prediction for the year. If Congress passes the health care reform bill and it wins President Obama’s signature, then big institutional investors will breathe a sigh of relief that health reform won’t harm biotech, Maraganore says. Essentially, the pricing power of biotech and pharma companies who develop innovative medicines will remain intact, and investors will see the benefits for drugmakers, who will essentially be able to sell their high-priced products to an even larger base of customers with insurance coverage.
“If you cure cancer, or at least make a big difference in people’s lives, you can still create a lot of value,” Maraganore says.