to allow the RNAi molecules to be released into the cytoplasm of cells so they can go to work, Murray says.
Last month, Alnylam started the first significant clinical trial in 55 patients, for an RNAi drug that can circulate throughout the body, built on Tekmira’s delivery technology. The drug, ALN-VSP, is designed to treat liver tumors by blocking two critical genes for that type of malignancy. Alnylam has run tests in mice that show its drug can suppress the intended genes, shrink tumors, and help the cancerous animals to live longer. ALN-VSP also passed safety tests in rats and monkeys, Alnylam says.
Tekmira stands to gain milestone payments for every drug candidate Alnylam or its partners advances using its lipid nanoparticle delivery technology, plus royalties on sales if they ever become marketed products.
That could generate some income to mitigate the company’s need to burn through its cash, but Murray says it’s only part of Tekmira’s business strategy. It hopes to keep some of the delivery technology in-house for its own drugs, including one RNAi drug to block a protein called ApoB that carries the so-called “bad” LDL cholesterol in the blood.
This drug—which hasn’t yet entered clinical trials—aims to be a competitor to Genzyme and Isis Pharmaceuticals’ mipomersen, which uses a different technology to block ApoB. That drug is years ahead in development, as those companies have said they aim to seek FDA approval in the second half of 2010.
There are plenty of competitors looking to solve the RNAi delivery problem. London-based Silence Therapeutics, Seattle-based PhaseRx, and Palo Alto, CA-based Intradigm are a few companies with different technology approaches to the problem, Murray says.
Like I do with every company without a product on market, I had to ask how Tekmira’s cash is holding out. The company had about C$30 million in the bank at its last financial update, which is enough to run into mid-2011, Murray says. The market gave the company a lift when it announced the Roche deal, but now investors want to see promising Phase I data. The company deserves a valuation of more than $75 million, Murray said, but he didn’t sound whiny about it.
“If we continue to put products into the clinic and build value there, we’ll be sustainable,” he says.
