Alnylam, Tekmira Look Ahead After RNAi Divorce

Xconomy Boston — 

Alnylam Pharmaceuticals and Tekmira Pharmaceuticals were married, more or less, for eight years. But now that their divorce is final, each side sounds like it is relieved to be going off in separate directions. And shareholders of the companies who watched this dispute from afar basically agreed yesterday.

After news of a legal settlement broke late Monday, shares of Cambridge, MA-based Alnylam Pharmaceuticals (NASDAQ: ALNY) climbed 5 percent to $16.94 yesterday. Tekmira (NASDAQ: TKMR) got a bigger lift, up 9 percent to $5.60.

The two companies, which began working together in 2004 when Tekmira went by the name Protiva, had been embroiled in a tense legal dispute since March 2011. That was when Tekmira accused Alnylam of what it called “relentless and egregious” misappropriation of its trade secrets. Tekmira sought $1 billion in damages related to the intellectual property, which covers lipid nanoparticles that are supposed to make it possible for RNA interference drugs to be effectively delivered inside cells. On the eve of taking this case to a jury trial, Alnylam agreed to pay Tekmira $65 million upfront to terminate a manufacturing agreement and amend a license to use the technology. Both sides agreed to drop all litigation, and amend their relationship so it’s unlikely that they’ll ever end up fighting each other in court again.

Mark Murray, CEO of Tekmira Pharmaceuticals

After the settlement was announced yesterday, each side was able to show something of value to its shareholders. Tekmira, in particular, is getting two years’ worth of extra operating cash which it can pour into its R&D programs.

“This leaves us in a great position,” says Tekmira CEO Mark Murray. “We’ve been able to recover our IP. There will be an assignment of patents back to us, control will go to us, we can license and use them as we see fit. We’ve re-established and shored up our platform, which is the gold standard in the RNAi delivery business. We now have some additional cash reserves that will allow us to push forward our product pipeline. Now we can get back to work of building our business.

John Maraganore, CEO of Alnylam Pharmaceuticals

Maraganore, the Alnylam CEO, said his company believed to the end that it did nothing wrong and that Tekmira’s accusations were “meritless.” But he said Alnylam chose not to take the risk of putting the case before a jury. “We viewed our case extremely strongly, but nobody knows when you go to a jury trial what the jury will decide. Reaching a settlement is a way to achieve certainty. It’s a smart business decision,” Maraganore says.

Each side had a lot to lose. For Murray, the decision to bring the lawsuit was a “bet-the-company decision,” he says. Tekmira had just $6.9 million in cash left at the end of June, and it only had a little more than a year’s worth of cash left in the bank. If it had walked away empty-handed, it would have been in trouble. “I suppose there are people in the industry who thought [the lawsuit] wasn’t a good idea. I felt, and the people around me felt, it was essential for the survival of the company.” He added: “A lot of the value in our business is based on our intellectual property. We need to remember that.”

Even though Alnylam is writing a $65 million check to settle a case it considers “meritless,” Maraganore insists that his company is getting some of what it wanted too. That’s because the new agreement calls for Alnylam to pay Tekmira some extra upfront cash, while sacrificing some bigger money it could have collected in the long term. Alnylam had previously agreed to pay Tekmira a “low single digit” percentage royalty on sales of products that use the Tekmira delivery technology, but the new agreement ratchets that royalty down to a “very low single digit” percentage, Maraganore says. And while the old agreement called for Alnylam to pay as much as $16 million, per product, in milestone payments to Tekmira for hitting certain development goals, the new agreement lowers the milestones to $5 million per product for drugs that treat transthyretin-mediated amyloidosis and liver cancer, and “basically zero” for other products in Alnylam’s pipeline, Maraganore says.

The reduced royalties and milestones make this “an attractive economic proposition for us,” Maraganore says. And even after writing checks for $65 million, Alnylam still has plenty of cash to run its business. It expects to end the year with more than $215 million in cash.

But now each company is on its own, and can’t count on the other side for help. Instead of relying on Tekmira as a contract manufacturer, Alnylam plans to focus on doing in-house manufacturing of the lipid-nanoparticles that it needs to deliver RNAi drugs into cells. The company has been building up that capability for a year, he says, and it has enough capacity to make batches of its lead drug candidate ALN-TTR02 for clinical trials, and into “early commercialization” of the product, Maraganore says. Alnylam also has been focusing on building up internal R&D strength for RNAi delivery. Alnylam’s in-house delivery group developed some new technology that is enabling it to start the first clinical trials later this year with an RNAi drug can be given through a subcutaneous injection—which goes just beneath the skin and is generally more convenient than intravenous delivery.

Tekmira, for its part, has been seeking to recast itself as a drug developer in its own right, not just a technology company that out-licenses RNAi delivery systems. Tekmira plans to use a new lipid nanoparticle technology in its TKM-PLK1 cancer drug candidate, which Murray says is 10 times more potent than earlier delivery technologies it developed and licensed to Alnylam. That drug is supposed to enter a mid-stage clinical trial in 2013.

Plus, Tekmira caught a fortunate break last month when the U.S. Department of Defense decided to keep funding an antiviral drug that Tekmira is developing to counteract the deadly Ebola virus. The U.S. military threatened to drop the program in August because of budget cuts, but decided in early October to keep supporting Tekmira’s work, while dropping another program it had supported at Cambridge, MA-based Sarepta Therapeutics (NASDAQ: SRPT).

Murray also says that Tekmira’s RNAi delivery technology is on the leading edge in the field, and that by settling and clarifying its dispute with Alnylam, it will now be in a better position to license it to other pharmaceutical companies. Any new licensing deals presumably won’t include Alnylam. As Maraganore put it: “This is, in many ways, a divorce. We don’t intend to work with them [Tekmira] in the future.”

Both Murray and Maraganore agreed on one thing—they are both glad to see the litigation come to an end, so both companies can concentrate more of their time and attention on drug development. Maraganore said that the case probably consumed one-fourth of his time in the past couple months. Plus, both executives said they believe the settlement will clarify ownership of key RNA intellectual property, which will remove some uncertainty that had been hanging over the field.

“It’s certainly good for the whole RNAi field that this lawsuit is over. It would have been better for the RNAi field if this lawsuit had never been started,” Maraganore says.