Innovation Northwest Wrapup: Alder, Tekmira, Acucela, & Other Emerging Little Biotechs

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that the world’s biggest drug company, Pfizer, has formed a partnership to use the technology. That means big players like Alnylam Pharmaceuticals, Roche, and now Pfizer, are beating a path to Vancouver for help in solving this vexing delivery challenge. RNAi will need to solve this problem in order to fulfill its potential to block all sorts of disease targets at the RNA level that have been inaccessible to other drugs. By the end of this year, five products using the Tekmira technology will be in clinical trials, CEO Mark Murray said.

“We’re fortunate at Tekmira to have a technology that a lot of people are interested in, and we’re the only ones who can provide it,” Murray said during a panel discussion.

Acucela CEO Ryo Kubota reminded the audience in his talk that his company has become “cash-flow positive” on the basis of a partnership with Japan-based Otsuka Pharmaceutical. That’s almost unheard of for a development-stage biotech company with no marketed products. While that makes life more comfortable in a tough fundraising climate—and is enabling Acucela to grow from a staff of 40 to 100 this year—what matters a lot more will be further clinical trial data to suggest its lead candidate, ACU-4429, has potential to become a real drug for the “dry” form of age-related macular degeneration.

—One of the things I always try to do at an investor meeting is to learn about new companies. Jennifer Hamilton of Nomura Phase4 Ventures introduced me to Vancouver, BC-based Celator Pharmaceuticals. It has some well-known VCs backing it (Domain Associates, Ventures West, and Quaker Bioventures), and some support from Frazer, PA-based Cephalon and the Leukemia & Lymphoma Society.

The idea is to develop “cocktails” of chemotherapy drugs in precise ratios, so they can minimize any overlapping side effects, while getting maximum tumor-killing kick. The lead drug candidate, CPX-351, has shown an ability to completely wipe out tumors for nine out of 45 patients with acute myeloid leukemia in an early-phase trial, which was better than what researchers expected based on historical comparisons to older trials, said David Wood, Celator’s head of finance and corporate development. Those kinds of comparisons are apples-to-oranges, but a more rigorous trial of 126 patients is expected to produce interim results before the end of June, followed by full results before the end of the year, Wood said.

—Seattle-based ZymoGenetics (NASDAQ: ZGEN) is sticking to its guns with its lone marketed product, recombinant thrombin (Recothrom) for surgical bleeding. This drug, a genetically engineered human protein that assists in clotting, was supposed to almost completely supplant a rival drug derived from cow blood. It hasn’t yet, although spokeswoman Susan Specht said it has captured 17 percent of the market as of the most recent quarter, and is on track to start generating positive cash flow for the Zymo pipeline by the end of 2010. (Specht stood in for president Stephen Zaruby, who was injured in a skiing accident.)

Most of the value at Zymo these days is derived from pegylated interferon lambda, a drug being developed with Bristol-Myers Squibb for hepatitis C. It’s supposed to eliminate the nasty flu-like side effects that plague patients taking a similar interferon drug that is part of the standard regimen for Hepatitis C. Results on the Zymo drug’s antiviral activity after four weeks, and 12 weeks of observation, are expected this year, Specht said.

HemaQuest Pharmaceuticals CEO Ron Berenson said that the Seattle company is close to nailing down the remaining $6 million out of a $12 million financing round I reported on last month. HemaQuest, a developer of treatments for blood diseases, is working on an oral pill for sickle-cell disease, and an intravenous product for lymphomas related to Epstein-Barr virus.

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