Cequent Pharmaceuticals, the Cambridge, MA-based developer of RNA interference drug technology, said today it has filed an application with the FDA to begin the first clinical trial of an orally-delivered RNAi treatment. Cequent is seeking to test its drug, CEQ508, at a variety of doses in 18 patients with polyps that lead to colon cancer (familial adenomatous polyposis). Cequent hopes to get the trial underway before the end of March, at the Fred Hutchinson Cancer Research Center in Seattle.

Comments | Reprints | Share:        

Cambridge, MA-based Aileron Therapeutics has bet the company on the idea that it has discovered a whole new class of drugs that, like RNA interference, can hit targets in the body that are beyond the reach of conventional chemical compounds and biotech therapies. Today, scientists are reporting the drugs can achieve this goal and block one of the prized targets that has eluded cancer researchers for years.

Scientists at Harvard University, the Dana-Farber Cancer Institute, and the Broad Institute of Harvard and MIT say they have used a synthetic “stapled peptide” from Aileron to get inside the nucleus of cells and stop the production of a protein called Notch that’s implicated in uncontrolled growth of cancer cells, according to research being published this week in Nature. The work was repeated in multiple disease models and in animal tests, which showed blocking this target led to cancer cell death, without the side effects of previous drugs, the researchers said.

This finding is bound to stir curiosity in the cancer research world for Aileron’s stapled peptide drugs. Buzz for the new drug technique picked up in June when Aileron raised $40 million in venture capital from a syndicate that included four major drugmakers—Roche, Novartis, Eli Lilly, and GlaxoSmithKline. While a few other peptide treatments are on the market for diabetes and osteoporosis, most of these drugs don’t work because they get chewed up by enzymes in the body before they can hit their target. Aileron’s key insight is to chemically “staple” these peptides in a way that holds them together in a properly folded shape, protecting them and preserving the unique structure that gives them the ability to hit very specific protein targets inside cells, like Notch.

“There have been many valiant efforts that have gone after this target, and they’ve all failed,” says Aileron CEO Joe Yanchik. “This is the first potentially viable therapy.”

As Yanchik explained to me in a profile of Aileron a year ago, traditional small-molecule chemical drugs, like Pfizer’s atorvastatin (Lipitor), usually need “a nice deep pocket” on the targeted protein for the compound to settle into. The problem is that only about one-tenth of proteins have this kind of pocket, while many more have long, flatter pockets inside that are “like a hot dog bun, for lack of a better term,” he said. Engineered peptides, which are protein fragments, are thought to have improved properties because they are larger than traditional small molecules and able to nestle into some of those bigger pockets, but they aren’t so big they can’t get inside cells, like traditional antibody drugs that operate on the cell surface, Yanchik says. Done right, a stapled peptide ought to be efficient at penetrating cells, and bind tightly enough and long enough to its target to have the intended effect.

Researchers led by James Bradner at Dana-Farber and the Broad Institute as well as Gregory Verdine at Harvard, said they found that the Aileron drug was able to bind directly and tightly to Notch in the nucleus of cells. That target is known as a transcription factor—a protein that binds to DNA in the nucleus of cells and regulate important biological processes. By blocking Notch, the scientists found they could prevent a cancer-causing gene from assembling the necessary proteins to grow, and suppress the production of other growth proteins that cancer cells need to live.

This idea of blocking transcription factors is important because they have been traditionally inaccessible, and there are an estimated 1,500 of these proteins involved in regulating key biological processes involved in diseases such as arthritis, asthma, diabetes, infectious diseases, and cancer, Aileron says.

“These results are tantamount to a declaration of open season on transcription factors,” said Verdine, a professor of chemistry at Harvard University and co-chair of Aileron’s scientific advisory board, in a statement.

Getting inside cells to specifically target previously “undruggable” targets sounds a lot like what gets so many scientists are excited about RNA interference …Next Page »

Comments | Reprints | Share:        

RXi Pharmaceuticals (NASDAQ: RXII), the Worcester, MA-based developer of RNA interference drugs, said today that Noah Beerman has replaced Tod Woolf as president and CEO. Beerman, 47, was most recently chief business officer of Indevus Pharmaceuticals. The company said the leadership switch was part of a succession plan as it looks to advance into product development. Woolf will remain on the company’s scientific advisory board.

Comments | Reprints | Share:        

[Update: 7:15 pm Eastern, 10/30/09] Cambridge, MA-based Cequent Pharmaceuticals, one of the many aspiring firms in the field of RNA interference drug technology, has raised $3.35 million in equity in the first tranche of a venture round that could be worth as much as $15 million, according to CEO Peter Parker. A regulatory filing released earlier today said the initial financing was worth $2.7 million.

Cequent has not formally announced the financing, although this is part of a second round of financing since Ampersand Ventures, Pappas Ventures, Yasuda, and Novartis Option Fund pumped in $15 million for a Series A round in 2007.  The first tranche of equity, from inside investors, is worth $3.35 million, while a second tranche from that group is expected to be worth $3.15 million, Parker says. The new financing will also include a $3.5 million venture loan from Gold Hill Capital and Silicon Valley Bank, which will be used to help get Cequent’s lead drug candidate into clinical trials, Parker says. Cequent is also seeking capital from outside investors, which it expects to receive after the clinical trial starts, he says. [Updated comments from Parker at 7:15 pm Eastern, 10/30/09].

RNA interference, which is supposed to allow scientists to silence disease-causing genes in ways that traditional drugs can’t, has been one of the big ideas to emerge in the biotech and pharmaceutical fields this decade. Boston has more than its share of companies vying in the space, including the leader in the field, Alnylam Pharmaceuticals (NASDAQ: ALNY), as well as smaller upstarts like Worcester, MA-based RXi Pharmaceuticals (NASDAQ: RXII), Watertown, MA-based Dicerna Pharmaceuticals, and Norwood, MA-based AiRNA Pharmaceuticals.

The biggest challenge with RNAi is with delivering these drugs effectively, because in their pure form, they get broken down in a matter of minutes in the body before they can block the RNA target of interest. Cequent is attempting to tackle this problem, and differentiate itself from competitors, by pushing the first orally delivered RNAi drug into clinical trials for a rare condition called FAP, a type of gastrointestinal polyp that leads to colon cancer. Other drugs are given via injection with lipid nanoparticles to make them more stable in the blood, or through localized delivery such as aerosols.

The idea at Cequent is to deliver the RNAi with an engineered form of E. coli bacteria, which would be at home in the bacteria-rich environment of the colon and wouldn’t need to overcome the challenges of being absorbed into the bloodstream that injectable drugs have. In this case, the target it would need to hit is right there in the gut anyway. Cequent has said it expects to file an application to start its first clinical trial with this drug before the end of the year.

Cequent lists a seven-member board of directors, which includes two insiders—CEO Peter Parker and founder Chiang Li, who’s also the CEO of Boston Biomedical and an adjunct faculty member at Beth Israel Deaconess Medical Center in Boston. The outside directors include Herbert Hooper of Ampersand; Eric Linsley of Pappas Ventures; Lauren Silverman of Novartis Option Fund; Michael Taylor of Ensemble Discovery; and Thomas Hancock of New England Partners.

Comments | Reprints | Share:        

Why would a Big Pharma company want to remake itself as a biotech? In a recent Xconomy piece, I discussed the changes in Roche’s corporate culture brought on by their recent acquisition of Genentech, along with recommendations for creating an innovative biotech research culture. What I didn’t have the space to dive into was a discussion of why Roche is attempting to transform itself from Big Pharma to biotech (OK, Big Biotech). The answer can be summed up in one word. Biologics. Here’s how I see it all coming together:

Biologics are Hot, Hot, Hot

Plant-based drugs have been used by cultures around the world for thousands of years. Drugs based on chemical synthesis have been with us since the latter half of the 1800s. Now, the era of biologics—genetically engineered protein drugs made in living cells—is upon us. Biologics now account for 20 percent of the global drug market, according to market research firm IMS Health. In 2000, only one biologic made the top ten list of worldwide drug sales (Amgen’s recombinant erythropoietin in 4th place). By 2008, five of the top 10 drugs in sales were biologics, and by 2014 biologics are expected to occupy six of the top ten positions, according to EP Vantage.

The trend is clear: worldwide sales of biologics are on track to hit the $100 billion level by 2011. As recently as 35 years ago there were no recombinant protein drugs in the marketplace. Today the marketplace contains many types of recombinant proteins, including growth factors, monoclonal antibodies, soluble receptors, clotting factors, replacement enzymes, vaccine components, and immune system stimulators. Biologics comprise about 25 percent of new drugs, according to the Washington Post, and accounted for about 16 percent of total prescription drug spending in 2008, according to IMS Health.

Small molecules, however are not likely to go away anytime soon, because they retain …Next Page »

Comments (2) | Reprints | Share:        

Alnylam Pharmaceuticals is looking to the frontiers of science for ideas on how to best deliver RNA interference drugs throughout the body, and the Cambridge, MA-based company sounds most jazzed about what it sees emerging from laboratories in Vancouver, BC.

Back in May, I wrote about how Alnylam—one of the richest companies in biotech—was leaning on partners at Vancouver, BC-based Tekmira to deliver an RNAi drug that can circulate through the body. Last week, Alnylam said it is doubling down with its Canadian friends. The company has picked a second drug candidate that uses the same technology, and is sponsoring research at a new company made up of four former Tekmira employees who are pushing further on the leading edge of drug delivery, at Vancouver-based AlCana Technologies.

These are important developments to watch, because while RNAi has been one of biotech’s hot concepts for years, scientists still have plenty of work left to prove these drugs can be efficiently delivered. RNAi treatments are thought to be superior to conventional pills because they specifically hit targets on cells that other drugs can’t, and can silence malfunctioning proteins that cause disease. It sounds great, but when small interfering RNA drugs are injected into the body on their own, they get chewed up by enzymes, or flushed through the kidneys long before they ever get to the desired target on diseased cells. Alnylam has worked around this with locally-delivered drugs that don’t have to circulate through the body, but only a limited number of diseases can be treated that way (and that approach poses challenges too, as I described earlier this week.)

Alnylam, and Swiss pharma giant Roche, see big potential to deliver RNAi drugs through the body using Tekmira’s approach, with lipid nanoparticle capsules (sort of like little grease balls, as Alnylam CEO John Maraganore told me last December) designed to protect the drug in the body until it gets to the diseased cells. This is the approach that Alnylam has implemented with ALN-VSP, an experimental drug for liver cancer that is in clinical trials, and it’s the same technology it plans to use for a second candidate called ALN-TTR, for a rare genetic disease called amyloidosis.

“It’s great technology. It works well, you can manufacture it,” says Maraganore. “It’s ready for prime time.”

But this being the leading edge of biology, what’s prime time today can turn obsolete really quickly. I wanted to know from Maraganore what advantages he sees …Next Page »

Comments | Reprints | Share:        

Navigating the world of cutting-edge drug development can be thrilling, but you can sometimes find yourself in a gray zone. That’s the impression I got from a conversation yesterday with Alnylam Pharmaceuticals CEO John Maraganore about the many options he’s carefully mulling over before deciding whether to bet bigger company resources on his lead drug candidate.

Cambridge, MA-based Alnylam (NASDAQ: ALNY) announced last month at a medical meeting in Beijing that its lead drug, ALN-RSV01, reached its goal of showing safety in adult patients with respiratory syncytial virus infections of the lungs. The study, a Phase II trial with 24 patients, wasn’t big enough to provide statistical proof that the drug was working, although researchers saw signs that it was helping patients breathe better than a placebo for as long as 90 days. Alnylam’s partner, Lexington, MA-based Cubist Pharmaceuticals, said in a statement that it was “encouraged” by the results.

This is important stuff to scientists, since Alnylam is a leader in the field of RNAi interference, which seeks to specifically silence disease-causing genes in ways that traditional treatments don’t. Since this inhalable form of RNAi appeared safe, and it offered signs of effectiveness in patients with the lung disease, why not charge right ahead into the third and final phase of testing required for FDA approval? Respiratory syncytial virus (RSV) is a serious infection that is the leading reason why children in the U.S. get hospitalized, Alnylam says. It also common among adults, especially elderly people with weakened immune systems.

“We and Cubist and [[our partner]] Kyowa Hakko remain very excited about RSV,” Maraganore says.

Alnylam CEO John Maraganore

Yet there are a lot of factors now to carefully consider before Alnylam and its partners make their next significant move. First, there are two different patient populations to consider—adults and children. As many pediatricians will tell you, children aren’t simply littler adults that can take a proportionally lower dose, so these patient populations need to be treated differently, and the product for each group may need to be differentiated, Maraganore says.

Then—and this is a familiar dilemma to many biotechs—RSV research didn’t just stop when Alnylam settled on its lead candidate and pushed it into clinical trials in 2005. The company’s researchers continued working on a molecule with “improved attributes” for treating this condition, by chemically modifying a new compound so that it would be more stable in the body—potentially enabling longer-lasting effects and less-frequent dosing, Maraganore says. This second-generation RNAi drug for RSV also can avoid activating a component of the immune system, which means it can potentially be given at a higher dose, Maraganore says.

“RSV01 is a great compound, but we may be able to improve on it quite a bit in the second generation,” Maraganore says.

This has essentially left Alnylam with three alternatives. It can advance …Next Page »

Comments | Reprints | Share:        

The big news of the week was Microsoft’s sale of Razorfish to Publicis (see directly below), but there were a few other important deals in software, biotech, and energy.

—Microsoft’s online advertising subsidiary, Seattle-based Razorfish, was bought by French marketing firm Publicis for approximately $530 million, as Bob reported. The payment is expected to include cash and Publicis Groupe treasury shares. In addition, Microsoft and Publicis have entered into a five-year strategic alliance whereby Publicis clients can purchase display and search advertising from Microsoft on favorable terms. Microsoft (NASDAQ: MSFT) originally acquired Razorfish in its 2007 purchase of aQuantive.

—Bellevue, WA-based Confirma, a medical imaging software firm, has been acquired by Merge Healthcare, a Milwaukee, WI-based health IT provider, for about $22 million, as Eric reported. Merge will incorporate Confirma’s MRI software into its IT offerings for doctors.

—Vancouver, BC-based Tekmira (TSX: TKM) formed a two-year partnership with Cambridge, MA-based Alnylam Pharmaceuticals to develop new particles to deliver RNA-interference drugs to diseased cells deep in the body, as Ryan reported. Financial terms of the deal weren’t given. Alnylam is funding the research effort and has exclusive rights to new discoveries, while Tekmira can use the discoveries for some of its own RNAi treatment programs.

—Seattle-based EnerG2 won $21.3 million in federal stimulus funding from the U.S. Department of Energy to build a new manufacturing plant in Albany, OR. The University of Washington energy-storage spinout is developing nano-scale materials to make better ultracapacitors for electric and hybrid vehicles and other applications.

—A few more terms of the Microsoft-Yahoo search deal, in which Yahoo will use Bing as its search engine and will control ad sales for five years, were spelled out in a filing with the SEC. Microsoft (NASDAQ: MSFT) will pay Yahoo $50 million a year for three years to cover transition and implementation costs. It will also hire 400 Yahoo employees, plus another 150 to assist with the transition. Yahoo (NASDAQ: YHOO) can opt out of the deal if it isn’t approved within a year, or if Microsoft and Yahoo’s combined share of the search market dips below an undisclosed percentage.

—Seattle-based Oncothyreon (NASDAQ: ONTY), a developer of cancer drugs, raised $15 million by securing commitments from investors to buy new shares and warrants, as Luke reported. Last week, the company said a small group of lung cancer patients showed long-lasting responses after taking Stimuvax, the immune-boosting vaccine therapy Oncothyreon is co-developing with Germany-based Merck KGaA.

—Seattle-based WatchGuard Technologies, a network security company, acquired Toronto-based BorderWare Technologies, an e-mail and Web security firm, as Eric reported. Financial terms were not given. WatchGuard plans to use BorderWare’s technology to make its security software more comprehensive and competitive.

Comments | Reprints | Share:        

Worcester, MA-based RXi Pharmaceuticals (NASDAQ: RXII) said today it is raising $8.3 million in working capital through a stock offering that’s expected to close on August 4. The company, which is developing drugs based on RNA interference for the treatment of conditions such as rheumatoid arthritis, is selling about 2.4 million shares of common stock at $3.50 per share, as well as warrants for almost a million shares at $4.50 per share. It expects to realize about $7.8 million in net proceeds from the sale.

Comments | Reprints | Share:        

RXi Pharmaceuticals, the Worcester, MA-based developer of RNA interference drugs, said today it has obtained an exclusive worldwide license to RNAi delivery technologies from Advirna. The technology is supposed to make it easier to deliver RNAi without liposomes, nanoparticles, or other methods that add complexity, RXi said (NASDAQ: RXII). Terms of the deal weren’t disclosed.

Comments | Reprints | Share:        

Dicerna Pharmaceuticals has set its sights on silencing problematic genes using what it calls the “second doorway” of RNA interference—an approach to the burgeoning field that other RNAi companies aren’t pursuing. But to get its drugs over the transom and where they need to go in the body, the Watertown, MA-based company will need a little help, and for that it has turned to Cambridge, MA-based Archemix.

The two firms have partnered up to develop combination treatments that will merge Archemix’s proprietary “aptamer” treatments with Dicerna’s gene-silencing drugs. Both companies hope the alliance will yield drugs that can precisely—and potently–home in on the molecular roots of disease. Specific financial terms of the deal aren’t being disclosed, although Dicerna and Archemix will split the early development costs, and Dicerna has an option to get exclusive rights to take the new drugs through development, leaving the usual milestone payments and product royalties to Archemix if the collaboration bears fruit.

The idea behind RNAi-based drug development is to create specially engineered RNA molecules that selectively turn off disease-causing genes. Entrants to the field typically have to pay some sort of toll to Cambridge, MA-based Alnylam Pharmaceuticals (NASDAQ: ALNY), which has an extensive RNAi IP estate, but Dicerna insists it has found a “second doorway.” Simply put, it’s designing drugs that are a little longer than so-called small interfering RNA molecules being developed by Alnylam and others—and which are therefore not covered by Alnylam’s patents. Dicerna says its drugs may have the added advantage of being more potent that other RNAi-based treatments.

Importantly, these slightly longer RNAi drugs can be made with a handle on them that allows them to be welded together with other drug compounds that could give them extra kick. …Next Page »

Comments | Reprints | Share:        

We had a smorgasbord of life sciences news this week, featuring updates on Northwest companies developing drugs, devices, vaccines, and scientific instruments.

—Cell Therapeutics has bet the company on pixantrone for non-Hodgkin’s lymphoma, and this week it hit one of its key goals for the year, by turning in its new drug application to the FDA before the end of June. The Seattle company (NASDAQ: CTIC) fell short in its bid to unload all of its $118.9 million in debt, instead getting rid of about $52.9 million of its liabilities.

—Seattle-based Targeted Genetics (NASDAQ: TGEN) has been in survival mode for some time now, and this week it said it will cut its staff down to as little as 10 to 15 employees. The company hopes the cuts will stretch its cash reserves into August, giving it enough time to close a deal to keep the company from closing its doors.

—Sanofi-Aventis CEO Chris Viehbacher traveled to Seattle last week for a global health meeting, and took time to offer a glimpse into his strategy to make the Paris-based drugmaker a bigger player in global health. He also shared his perspective on partnerships with biotech companies.

—Seattle-based Amnis shared its story of how it has rolled out a second-generation version of its sophisticated lab instrument, which combines properties of a microscope with a high-speed cell counter called a flow cytometer. Orders are building up, and this could be the company’s first full year of profitability after a decade in business, says CEO David Basiji.

—Oncothyreon (NASDAQ: ONTY), the Seattle-based developer of cancer drugs, said that its German partner, Merck KGaA, started up a pivotal trial of its immune-boosting therapy, Stimuvax. It’s the second big trial of this drug, which is already being tested against lung cancer. If it’s successful, Oncothyreon will stand to get royalties on product sales.

—AVI Biopharma (NASDAQ: AVII), the Portland, OR-based developer of RNA-based therapies, said it is working on a contract to develop swine flu drugs.

—Bothell, WA-based Mirabilis Medica told its story about how it is developing the first use of high-intensity therapeutic ultrasound waves to treat uterine fibroids, as a non-invasive alternative to surgery.

—MDRNA (NASDAQ: MRNA), the Bothell, WA-based developer of RNA interference drugs, gained full rights to a technology license from Denmark-based RiboTask to make its treatments more stable, and less likely to spark inflammation.

—Global health funding has quadrupled over the past two decades, and while a lot of this new money comes from foundations like the Bill & Melinda Gates Foundation, the biggest financier of this work is now the U.S. taxpayer, according to this intriguing new study from the University of Washington.

—Anthony Fauci, the head of the division of the National Institute of Allergy and Infectious Diseases, called for a transformative research agenda to fight tuberculosis, like the country did for HIV in the 1980s, during a stop last week at the Pacific Health Summit in Seattle.

Comments (2) | Reprints | Share:        

It has been a fairly busy start to the summer in the Northwest. In the past week, we’ve seen a number of deals in software, Internet, displays, biotech, and energy.

—Portland, OR-based Reductive Labs raised a $2 million Series A round led by True Ventures in Palo Alto, CA. Reductive, whose founders are in Salt Lake City and Nashville, TN, but are relocating most of the company to Portland, has developed open source software to help companies automate the management of their IT systems. True Ventures is also an investor in Seattle-based 1000 Markets and RescueTime.

—Redmond, WA-based Microvision (NASDAQ: MVIS), a maker of display technologies, received a $15 million investment from Taipei-based Walsin Lihwa, in the form of a common stock sale to Walsin’s subsidiary, Max Display Enterprises Limited, and a warrant to buy more shares.

—Bellevue, WA-based LiquidPlanner, a collaborative software startup focused on online project management, raised $1 million from angel investors, including $235,000 from the Seattle-based Alliance of Angels’ new seed fund. Angel investor Geoff Entress has joined LiquidPlanner’s board of directors.

—Luke reported that Bothell, WA-based MDRNA (NASDAQ: MRNA), a developer of RNA interference drugs, renegotiated a key technology license with Denmark-based RiboTask. MDRNA now has full control and no longer needs to make payments for the technology, which could make RNA interference drugs more stable and less likely to cause inflammation. Terms of the deal were not disclosed.

—Seattle-based Madrona Venture Group led a $4.4 million funding round for Animoto, a New York-based video and slideshow software startup. Amazon is also an investor in the company, which …Next Page »

Comments | Reprints | Share:        

AVI Biopharma, a Portland, OR-based developer of RNA-based drugs, said today it has secured a contract with the U.S. Defense Threat Reduction Agency to develop drugs for H1N1, or swine flu. Under the contract, worth as much as $5.1 million, AVI will analyze the H1N1 sequence, determine appropriate targets and identify lead and back-up candidate drugs. The company (NASDAQ: AVII) also plans to do animal tests.

Comments (5) | Reprints | Share:        

MDRNA, the Bothell, WA-based developer of RNA interference drug technology, said today it has renegotiated a key technology license so that it has full control and no longer needs to make future payments for it. MDRNA (NASDAQ: MRNA) bought the rights in October to unlocked nucleobase analog technology from Denmark-based RiboTask, which has been studying its ability to make RNA interference drugs more stable, and less likely to spark inflammation. Terms of the deal with RiboTask weren’t disclosed, although MDRNA raised $10.5 million from investors this month.

Comments (2) | Reprints | Share:        

Center stage in Seattle life sciences moved to the waterfront this week, as 250 global movers and shakers in science, global health, and the pharmaceutical business gathered for the Pacific Health Summit.

—The Pacific Health Summit, an invitation-only event of global health stars, in its fifth year, focused this year on multidrug resistant tuberculosis, as I wrote in this preview story. Big names from global health came to brainstorm, including Margaret Chan of the World Health Organization, Anthony Fauci of the National Institutes of Health, and Chris Viehbacher, the CEO of Sanofi-Aventis, the world’s largest vaccine maker. Seattle’s Infectious Disease Research Institute is one of the players in this field, too, through work supported by Eli Lilly.

—Every good conference like the Pacific Health Summit needs power players competing for media attention. This year, we had two pieces of international news. Johnson & Johnson announced a deal to co-develop a new drug with the nonprofit TB Alliance; the deal could lead to the first new drug against tuberculosis in more than 40 years. Sanofi-Aventis also broke some news by announcing it will donate 100 million doses of flu vaccine to the WHO, to help poor countries cope with the swine flu pandemic.

—I profiled Beat Biotherapeutics, a Bellevue, WA-based company that envisions a way of generating stem cells that could perform the function of a cardiac pacemaker, or maybe someday even regenerate heart muscle that’s been damaged by heart attack. This company is built on years of research by UW stem cell scientists Chuck Murry and Michael Laflamme, and is married to bioengineering techniques from Buddy Ratner’s lab at the UW.

—Mukilteo, WA-based CombiMatrix (NASDAQ: CBMX) never captured much of the market for sophisticated gene chips used in modern biotech labs, which is now dominated by Santa Clara, CA-based Affymetrix and San Diego-based Illumina. But now CombiMatrix hopes to carve out an emerging niche by marketing its DNA microarray instruments as a diagnostics service to physicians, who are looking for accurate ways to diagnose the aggressiveness of an individual patient’s form of cancer, and to use genetic screening to catch malignancies earlier.

—MDRNA (NASDAQ: MRNA), the Bothell, WA-based developer of RNA interference drugs, has been working hard to clean up its balance sheet the last few months. It faced a cash crisis earlier in the year, and then raised $7.25 million from Novartis, another $10.5 million from investors, and used some of the proceeds this week to pay off its $5.5 million debt to GE Capital.

—Seattle’s Infectious Disease Research Institute said it is offering up its immune-stimulating compounds known as adjuvants to the world’s major vaccine makers. With the right partnerships in place, these adjuvants have potential to greatly amplify the world’s supply of flu vaccine, says IDRI founder Steve Reed. This may come in especially handy if the swine flu pandemic takes a severe turn for the worse.

Comments | Reprints | Share:        

MDRNA, the Bothell, WA-based developer of RNA interference drug technology, said today it has paid back all of its debt to GE Capital. The company paid back a $5.5 million loan, removing all liens on its intellectual property, equipment or other assets, MDRNA said. The company (NASDAQ: MRNA) ran low on cash earlier this year, although it struck a deal with Novartis in March that brought in $7.25 million and raised $10.5 million from investors earlier this month to replenish its balance sheet.

Comments | Reprints | Share:        

This is definitely not in sync with the economic theme of 2009, but surprisingly, there was a lot of good news in Seattle biotech the past week.

—Dendreon CEO Mitchell Gold fielded a lot of questions at the company’s annual shareholder meeting yesterday, and the biggest one of all is whether he’s looking to sell the company. He said all the right things a CEO is supposed to say. But afterwards, when I asked him to elaborate, he went further—saying that not only does he want Dendreon (NASDAQ: DNDN) to remain independent, he wants it to become the next big anchor for Seattle biotech, like Immunex in the late 1990s and early 2000s.

—Seattle-based NanoString Technologies pulled in a whopping $30 million round of venture capital to continue building momentum for its marketed genetic analysis tool, nCounter. The company had the unfortunate timing to introduce this product last July, right before the economic crisis, but it raised the new capital anyway, partly because rave product reviews trickled in to Clarus Ventures, one of the largest biotech venture investors in the U.S.

—Bothell, WA-based OncoGenex Pharmaceuticals (NASDAQ: OGXI), the developer of a new prostate cancer drug, doesn’t seem to be very well known in its backyard, but this company has been on an amazing bull run on Wall Street, and it has made some serious money for Vancouver, BC-based Ventures West. I dug through the regulatory filings to show that Ventures West, which had almost 17 percent ownership in the company last fall when it traded down in the $2 range, has cashed out all its shares. The stock closed yesterday at $25.90.

—Seattle-based Cell Therapeutics (NASDAQ: CTIC) has run dangerously low on cash this year, and is performing a “tight-wire” act to hold everything together. Yesterday I discovered in the regulatory filings that the company’s compensation committee decided to reward the company’s senior executives with $250,000 of bonuses that had been deferred until they considered the company “sufficiently liquid” to make the payments.

—MDRNA, the Bothell, WA-based developer of RNA interference drug technology, had two bits of good news this week. The company (NASDAQ: MRNA), ran extremely low on cash earlier this year, but has recovered enough to raise $10.5 million in a stock sale to existing investors. The company also said that a generic calcitonin nasal spray it developed for osteoporosis was approved by the FDA and introduced to the market by Par Pharmaceutical. MDRNA sold the product to Par back in March, but it can still receive an undisclosed profit share from the product over the next five years.

—Bothell, WA-based Alder Biopharmaceuticals, the developer of targeted antibody drugs, expanded its partnership with pharmaceutical giant Schering-Plough, in which it will develop a number of antibody drug candidates for a major neurological disease, says Alder CEO Randy Schatzman. Yes, that’s cryptic, but the understated Schatzman said this deal provides “significant” upfront cash to his company, milestone payments for success in development, and royalties on future sales. Since Alder raised $40 million in venture capital in January 2008, I’m going to surmise this new deal pulls in at least $5 million upfront.

—Seattle-based Calistoga Pharmaceuticals is starting to build a preliminary, but impressive, body of evidence for its experimental drug candidate for blood cancers. Two weeks ago, the company showed an earlier slice of data that showed its CAL-101 drug was at least partially shrinking tumors for 6 of the first 12 patients who enrolled in a clinical trial, even when given at lower-than-optimal doses. As more patients enroll in the clinical trial, the data is holding up, with 12 of the first 24 patients now reporting partial tumor shrinkage.

—Portland, OR-based AVI Biopharma (NASDAQ: AVII), the developer of RNA-based therapies, said last week that it will receive $3 million from Charley’s Fund to continue testing a treatment for Duchenne Muscular Dystrophy.

Comments | Reprints | Share:        

MDRNA, the Bothell, WA-based developer of RNA interference drug technology, said today it has raised $10.5 million in a stock sale to existing investors. The company (NASDAQ: MRNA) agreed to sell 5.25 million shares at $2 apiece, plus warrants to buy the same number of shares at $2.38 over the next 5.5 years. Canaccord Adams served as the sole placement agent on the deal. MDRNA, which ran dangerously low on cash earlier this year, didn’t say how long the new infusion is expected to last.

Comments | Reprints | Share:        

Bothell, WA-based MDRNA (NASDAQ: MRNA) said today that a generic calcitonin nasal spray for osteoporosis has received FDA approval, and has been introduced to the market. The Bothell, WA-based company sold the product in March to Woodcliff Lake, NJ-based Par Pharmaceutical (NYSE: PRX), although MDRNA still stands to receive an undisclosed profit share from the product over the next five years. MDRNA has switched its strategy from developing nasal spray products to RNA interference technology.

Comments | Reprints | Share: