Author: Luke Timmerman

Luke Timmerman wrote:

The Bill & Melinda Gates Foundation said today that it is pledging $10 billion to foster more work on research, development, and delivery of vaccines over the coming decade. The goal is to save the lives of more than 8 million children by 2020. Bill and Melinda Gates made the announcement at the World Economic Forum in Davos, Switzerland. “We must make this the decade of vaccines,” said Bill Gates, in a statement. “Vaccines already save and improve millions of lives in developing countries. Innovation will make it possible to save more children than ever before.”

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Luke Timmerman wrote:

Steve Burrill, the San Francisco-based merchant banker with one of the best Rolodexes in the life sciences industry, is getting serious about Seattle. And his name and connections might be just the thing to help lift the Northwest’s annual biotech investing conference out of obscurity.

Burrill & Co. made it official yesterday that it has agreed to sign on as a sponsor and partner on Life Sciences Innovation Northwest, the regional biotech showcase that the Washington Biotechnology & Biomedical Association organizes every year in March.

This means Burrill, an in-demand public speaker, will deliver one of his trademark keynote talks on the state of the biotech industry. More importantly, his organization will provide money and organizational horsepower to help draw a crowd of national biotech players—something that’s never easy in this soggy corner of the country in March.

“There’s a lot of strength in the Pacific Northwest,” Burrill said yesterday, phoning from Paris. “This is a way for us to be connected into an enormous band of technology that stretches from north of the San Francisco Bay to Vancouver, BC.”

Burrill added that while his organization is “geographically agnostic,” he’s drawn to the Northwest partly because of its computing expertise, as IT becomes more vital to life sciences in the genomics age. While Burrill has organized events in other parts of the Northwest in the past—including Portland, OR, last year—this is the first time he’s thrown his energy into a conference in the Seattle biotech cluster.

Steve Burrill

For those who are unfamiliar, Burrill has been analyzing, prognosticating, investing, dealmaking, speaking, and writing about biotech for more than two decades. His organization has more than $950 million under management to invest in drugs, devices, diagnostics, agricultural biotech, and biomaterials. Every year, Burrill & Co. hosts an event for top industry CEOs in Laguna Beach, CA. Just two weeks ago at the JP Morgan Healthcare Conference in San Francisco—the biggest public biotech investing event of the year—Burrill hosted a reception that drew more than 1,000 people.

That sort of high-powered networking has been only a dream for the folks at the WBBA, who have been trying for years to raise the profile of the local biotech scene, and have struggled to put the local investing event on the national map.

“This helps validate life sciences here,” says Chris Rivera, head of the WBBA.

Last year, Rivera’s first as WBBA president, the conference drew a little more than 700 attendees to the Seattle waterfront. Even though the economy looked grim, the conference had …Next Page »

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Luke Timmerman wrote:

Flexion Therapeutics is a little company built on the idea it can steer through early clinical trials in a faster and cheaper way than a lumbering Big Pharma giant. Now the Woburn, MA-based startup, founded by a pair of Eli Lilly veterans, has secured backing from four major drugmakers to put the idea to the test.

The world’s largest pharma company, Pfizer, has agreed to pump in $9 million in venture financing to Flexion, bringing the startup’s total Series A financing to $42 million. Flexion is also announcing today that it has secured partnerships with U.K.-based AstraZeneca, Germany-based Merck KGaA, and one other unnamed drugmaker. The mission will be to run early-stage clinical trials on four drug candidates that have been sitting on the shelf at the bigger companies, to see whether they have potential to treat inflammatory diseases.

We first broke news on Flexion back in October when the company raised money from Versant Ventures, 5AM Ventures, and Sofinnova Partners. The biotech, which pronounces its name FLEK-shun, is, like the name suggests, focused on bringing drug candidates to a value-creating “inflection point.” Flexion’s founders, Mike Clayman and Neil Bodick, built their reputations doing that at an incubator in Lilly called Chorus. The model there, as with Flexion, was to take raw drug candidates and design fast, cheap, elegant experiments that would demonstrate they have what it takes to go to the later, more expensive stages of development.

Drug development, of course, is a horrifically inefficient, time-consuming, risky, and expensive business. An estimated one out of every 10 drugs entering clinical trials ever makes it all the way to FDA approval, and the development process takes a decade or more and costs more than a billion dollars on average—depending on whose figures you want to believe. Regardless of the actual math, pharma is crying out for ways to increase its batting average in clinical trials, and lower its costs. So Clayman and Bodick found plenty of interest in Flexion, and ultimately had “in-depth” talks with 10 major drugmakers from the U.S. and Europe, Clayman says.

Mike Clayman

“What we offered them was a track record that says we’ve done this before, and if they outlicense their products to us, they will be in good development hands,” Clayman says.

The plan is to take “de-prioritized” early phase assets of a pharmaceutical company and seek to turn them into diamonds in the rough. Sometimes these drugs have been put on the back burner because the pharma company dropped out of an entire disease category, or went through budget-cutting that forced it to place its bets on drugs in the more expensive, final phase of development.

Flexion isn’t afraid to make some pretty specific, and bold, claims about what it can do. Instead of investing three to four years and $15 million to $40 million in generating proof of concept for a new molecule, Flexion aims to use “compact experimental designs” to cut that time frame in half, while slashing the costs of proof of concept to as little as $3 million to $5 million.

The company only has six employees, and says it intends …Next Page »

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Luke Timmerman wrote:

Amira Pharmaceuticals looks and feels like a typical small biotech company, with the sort of irreverent sense of humor that you don’t often find at a Big Pharma shop. While CEO Bob Baltera showed me around the Amira offices on my last visit to San Diego, he opened up a storage room where employees had stashed away some belongings.

There was an old turkey baster, next to a bench press.

“That’s how we burn off a few calories around here before Thanksgiving,” Baltera cracked.

Amira may be able to joke around and have fun, but it’s actually borrowed more than a few pages from the old Big Pharma drug development playbook. The company was founded in 2005 by three scientists—Peppi Prasit, Jilly Evans, and John Hutchinson—who worked together for years at Merck before the giant pharma company closed its San Diego operation. As I described in a feature last March, they played important roles in developing montelukast sodium (Singulair), the asthma drug that generates $4 billion a year in revenue, and they resolved to try to do it again at their own company in San Diego. They’ve had some success already, raising a little more than $28 million in venture capital, building a staff of 50 employees, and scoring a partnership with GlaxoSmithKline.

But what I found most interesting when I met with Baltera was how Amira has combined some of the best cultural features of biotech and pharma in one place. That means while softball trophies are one prominent display of how the team literally plays together, Amira also insists on being a little extra careful and cautious, like a Big Pharma company, when it comes to building a body of evidence to support new treatments.

Bob Baltera

What does that mean? For one thing, Amira scientists identify not just one lead drug candidate to block a particular target, but they identify a lead and backup contender that generally follows six months behind in development. That way if a safety issue emerges with the lead candidate, the entire program won’t have to go in the trash. And by running experiments on both molecules, Amira hopes to answer whether the problem was in picking a dangerous target, or whether it was just something with the molecule that needs a minor tweak. From a business perspective, this means Baltera isn’t betting the company on a single molecule, which biotech startups often do.

“We try to sell a drug development program, not a molecule,” Baltera says.

This definitely costs more money, significantly more, to run simultaneous clinical trials of different molecules, Baltera says. He concedes that investors have occasionally asked why the company really needs to spend money on two candidates instead of one. Amira hasn’t really had to aggressively defend the strategy during the downturn, because the Glaxo partnership has provided enough support that it hasn’t had to raise money since Baltera arrived in July 2007. But he insists that going the extra mile mitigates …Next Page »

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Luke Timmerman wrote:

Biotech companies took a vow of silence during this week of breathless national celebration over the latest creation from Apple. The sarcasm you detect is intended. A few other things happened this week.

—The University of Washington has hired two heavy hitters from the world of high tech and biotech to help it get better at spinning out its inventions into the business world. Rick LeFaivre of OVP Venture Partners, and Pathway Medical founder Tom Clement talked about what they hope to accomplish on campus.

—Accelerator, the biotech startup machine in Seattle, actually slowed down a fair bit in 2009, according to CEO Carl Weissman. The place endured a six-month dry spell last year when it wasn’t finding promising new company ideas, but Weissman hints that could soon change in the first part of 2010.

—Ekos, the Bothell, WA-based developer of ultrasound catheters that help dissolve blood clots, told me that it is starting an ambitious new clinical trial to take its device from its original application in the legs, to a new use in the lungs.

—I stopped by Merck’s Sirna Therapeutics operation during my last trip to San Francisco, and came away with this exclusive interview with the company’s RNA interference leader, Alan Sachs. He should be familiar to Seattle biotechies, since he formerly ran Merck’s Rosetta Inpharmatics operation.

—Xconomist Ken Stuart, the founder and president of Seattle Biomedical Research Institute, wrote a guest editorial on how genomics have laid a foundation for discoveries in global health that will pay off over the next decade.

—My colleague in Boston, Ryan McBride, gathered some insight from Cambridge, MA-based Millennium: The Takeda Oncology Company and its new partnership with Seattle Genetics (NASDAQ: SGEN). Millennium has high hopes that the brentuximab vedotin antibody will be useful for autoimmune disorders, in addition to its original application for Hodgkin’s disease and related lymphomas.

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Luke Timmerman wrote:

One of the more intriguing experimental drugs for multiple sclerosis has just entered its first clinical trial. Cambridge, MA-based Biogen Idec (NASDAQ: BIIB) has now started what it believes to be the first-ever clinical trial of a drug with the potential to regenerate the fatty protective coating around nerves that gets damaged in people with multiple sclerosis.

Biogen plans to enroll 64 healthy volunteers in the Netherlands, who will get a single intravenous infusion of what’s being called BIIB-033, says company spokeswoman Tracy Vineis. The study, listed in detail here on clinicaltrials.gov, will look at whether the targeted antibody drug is safe and well-tolerated, along with measurements on how long it stays in the body. Results from this initial test should be available in 2011, Vineis says.

This trial is more interesting than the usual run-of-the-mill study. That’s because the current standard treatments for MS, a neurodegenerative disease, are only thought to slow down the progressive damage of the disease. Biogen scientists hope to change the paradigm of treatment with this newer drug, by developing an antibody that blocks a new target called the Lingo-1 protein. By hitting this marker on cells, the company hopes that it will restore the body’s natural process of building a fatty protective layer around nerve fibers, known as myelin. As I described in an in-depth feature on the program back in August 2008, this is sort of like putting rubber coating back around electrical wires so they don’t short-circuit.

“As far as we know, this is the first drug in clinical trials with the potential to repair MS damage,” Vineis says. “We’re very excited about it.”

It’s too early to say what Biogen’s ultimate strategy will be for the best use of the anti-Lingo1 antibody. But it’s possible that patients could start on existing drugs that stop the progressive nerve damage, and then switch to the new drug that regenerates the protective myelin coating. If Biogen can prove the drug is safe, and does what it is supposed to do scientifically, this could be a very big deal in a few years for the 400,000 or so people in the U.S. with multiple sclerosis.

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Luke Timmerman wrote:

The man hired to turn Cambridge, MA-based Vertex Pharmaceuticals into biotechnology’s next big success story might never have gotten into the industry if he hadn’t been a security guard.

It was the early 1970s, and Matt Emmens was a middle-class New Jersey kid with a vague idea of studying business at nearby Fairleigh Dickinson University. He worked as an auto mechanic for a while to help pay the steep private school tuition. He even dropped out for a while to save cash before he got a lucky break: During his senior year, he scored free tuition and room and board as head of security on campus.

Being the man with a lot of keys meant Emmens got to know a lot of people on campus who sometimes needed help. That included people in the career office, who suggested he interview with a drug company: Merck. “It was a godsend,” Emmens says.

Thirty-five years later, this 58-year-old executive with humble beginnings, finds himself as a leader in an industry full of hard-charging brainiacs. He took over last May as the chairman and CEO of Vertex (NASDAQ: VRTX).

Today, Vertex is a company that aspires to do no less than shake up medicine like only a handful of biotechs have ever done, namely Genentech and Gilead Sciences. Vertex plans to finish up the final phase of clinical trials of a drug that it hopes will transform the treatment of hepatitis C liver infections, and which analysts predict will exceed more than $2 billion in U.S. sales after just a couple years on the market. Vertex plans to follow that up with a first-of-its-kind oral pill for cystic fibrosis that experts say has the potential to be the first to correct the underlying protein abnormality at the roots of the fatal lung disease.

Matt Emmens

The pressure to perform is on, and plenty of people will be watching Emmens closely this year. Vertex’s market capitalization has swollen to $7 billion, and it spent a staggering $516 million on R&D in 2008. Vertex added 200 employees last year, and now has about 1,100 on staff in Cambridge, MA, and another 175 in San Diego.

“This is about getting a group of people together, and developing a vision for something that seems impossible,” Emmens says. “This is about involving people in an undertaking they’ll remember their whole life.”

I talked to Emmens in depth about his life journey a couple weeks ago in San Francisco while we were both attending the JP Morgan Healthcare Conference. I’ve been itching to interview him one-on-one since May. He officially took over at that time when founder and CEO Josh Boger stepped down after a 20-year run at the helm. The rationale was pretty straightforward. Boger was the scientist with the adventurous entrepreneurial spirit who built the company’s R&D engine. But as Vertex inched toward the anticipated commercial introduction of telaprevir in 2011, it needed an experienced commercial guy to take over.

The official corporate biography of Emmens doesn’t say much about who he is. He had previous stints as CEO of U.K-based Shire Pharmaceuticals, president of Germany-based Merck KGaA’s pharmaceuticals division, CEO at Astra Merck, and a series of managerial jobs at Merck.

Meeting Emmens in person, he doesn’t strike an imposing figure, at about 5-foot-10 with a medium build. He speaks in a soft monotone, and has the standard-issue conservatively cut gray hair …Next Page »

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Luke Timmerman wrote:

Any fair assessment of Seattle biotech over the past five years would have to count Accelerator as one of the bright spots. But the past year or so has been unusually quiet at the biotech startup incubator. Accelerator recently endured a six-month dry spell when it didn’t see any exciting new investment ideas enter its pipeline, according to CEO Carl Weissman.

“We can survive these kind of times easily, because what the board really expects is that the quality [of investments] never goes down,” Weissman said when I stopped by his office on Seattle’s Eastlake Avenue last week. “We don’t have to lower our standards on quality in order to satisfy a quota.”

For those who aren’t familiar, Accelerator is one of the central players in the local life sciences innovation scene. Biotech pioneer Leroy Hood, along with a number of prominent venture firms, founded Accelerator in 2003 to provide some lab space and operational support for scientific entrepreneurs with potentially groundbreaking ideas that need a little more proof before they can secure serious venture dollars. Accelerator has raised a total of $43.8 million, some of which it has put to work in 10 startups so far. The roster includes four Seattle companies that have emerged and raised a combined $144 million since graduating—VLST, Theraclone Sciences, Allozyne, and Integrated Diagnostics.

That last company, known as InDi for short, is the most recent startup with roots at Accelerator to score venture bucks. It was known as Homestead Clinical in a previous incarnation, and it didn’t win follow-on financing from Accelerator’s primary VC backers, but it emerged in October anyway with a $30 million commitment from outside VCs largely because of Hood’s “force of will,” Weissman says.

Carl Weissman

OK, so InDi may be another notch in the Accelerator belt. But I wondered about the other three startups that were founded at Accelerator in the past two years—PharmSelex (formerly known as GPC-Rx) in June 2008, Mirina in August 2008, and Xori in May of 2009. All three are still operating, and Weissman wouldn’t say much about their future prospects other than, “We’re really pleased with the progress from two out of the three.”

So we’ll have to sit tight a while longer to see whether any of those three emerge, or fade away. What was more surprising to me is what he said about the slowdown in the flow of new ideas for companies. From about June through December, “there wasn’t much,” in the way of exciting new scientific ideas coming in to Accelerator, Weissman says, even though it has historically been inundated with pitches. That improved a bit last month, when Accelerator found three new ideas that it considered “very exciting,” Weissman says.

The feast-and-famine cycle at Accelerator “is to be expected,” he says, and that is why the organization doesn’t have a hard quota …Next Page »

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Luke Timmerman wrote:

Big news is out this morning from the Burnham Institute for Medical Research. The San Diego-based nonprofit says it’s getting a $50 million donation from T. Denny Sanford, enough for the whole institution to be re-named in honor of the philanthropist.

The center will now be called the Sanford-Burnham Medical Research Institute. That decision was made now that Mr. Sanford, who made his fortune in the private equity business, has committed to donate $70 million to the research center over the past three years. Besides his direct support of the Burnham, the financier gave another $30 million in 2008 to create the Sanford Consortium for Regenerative Medicine to support stem cell research in San Diego.

This is really just the latest coup for the Sanford-Burnham Institute, which has been on a roll over the past couple of years. The institute scored a six-year $98 million grant from the National Institutes of Health in September 2008 to establish one of four new drug discovery centers at academic centers around the U.S. Its annual budget has seen double-digit annual increases in recent years, climbing to $154 million. The staff has grown to 1,000 people at campuses in San Diego, Santa Barbara, CA and Orlando, FL. The institute loves to talk about how its scientists were ranked No. 1 worldwide in terms of impact over the past decade, as measured by citations in peer-reviewed scientific journals.

That’s all impressive stuff when scientific publications are the coin of the realm, but the Burnham is also well aware its research has yet to deliver a major breakthrough for human health like, say, Novartis’ imatinib (Gleevec) for leukemia. So over the past year, the center has intensified its push to pursue that lofty goal by hiring two key people: One is Michael R. Jackson, a former Johnson & Johnson drug discovery expert; the other is Paul Laikind, a former biotech CEO, to negotiate more deals with drug companies with the money and expertise to create the next generation of Gleevecs. So the Sanford donation is really about fueling a transformation engine that can help the Burnham cross the gap between basic research and real-world applications.

Paul Laikind

“Denny Sanford’s continuing generosity will help us make a greater impact on human health,” said John Reed, the Institute’s president and CEO, in a statement.

As it so happens, I heard about this big donation just as I was sitting down to write a feature about how Burnham intends to do a better job of applying its ideas in the wider world, by hiring Laikind as its new chief business officer. Laikind is a biochemist by training, with a 25-year track record as a biotech startup executive, most recently as the CEO of San Diego-based Metabasis Therapeutics (acquired by Ligand for $3.2 million in October). I’ve written a lot in the past couple years about academic centers around the country trying to do a better job of this notoriously tricky business of relating to the business world, including the University of Washington and The Scripps Research Institute, so I was curious to hear Laikind’s perspective.

Donations like the one from Sanford—spread out over five years in $10 million annual chunks—provide a certain amount of budget stability, and could enable researchers there to get tantalizing early experimental data that can be used to help win further federal grants, and maybe entice …Next Page »

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Luke Timmerman wrote:

The folks at Life Technologies, the giant Carlsbad, CA-based maker of supplies and instruments for biologists, like to say they seek to “democratize” molecular biology. That means simplifying sophisticated tests so they don’t have to be done in a few hard-core, central labs, but can be done at an everyday lab bench. This is the same philosophy that allowed early PCs to crush the old-school, hard-to-operate mainframe computers.

But Life Tech isn’t the only company in San Diego seeking to apply that idea to biology. Just a few miles down the road in Carlsbad, I met an intriguing startup called Helixis that could shake up the business of real-time PCR technology. I heard about it a few weeks ago from CEO Alex Dickinson and the company’s vice president of marketing, Judy Macemon.

The basic idea goes something like this: Advances in molecular biology depend on scientists being able to precisely measure how DNA is switched on or off in a given sample of, say, cancer cells, compared with healthy cells. Sophisticated real-time PCR machines are one of the workhorse technologies used for those experiments. Three major players—Life Tech, Roche, and Bio-Rad Laboratories—dominate the market. They sell machines that can cost as much as $50,000, need to be shared typically by an average of 10 researchers, and are operated in central labs, Dickinson says.

The idea at Helixis, hatched at the Caltech labotatories of Nobel laureate David Baltimore and Axel Scherer, is to create a real-time PCR technology that’s small enough to sit on the average lab bench, costs just $10,000, and performs with a higher degree of accuracy and consistency, Dickinson tells me.

Alex Dickinson

“This will allow researchers much greater access to the [real-time PCR] machines, and enable them to be much more productive,” says Dickinson.

And this is not just a research project. The company has more than 150 orders for the product, to be marketed as Pixo. Helixis plans to start shipping its first versions to commercial customers in April, Macemon says.

If Helixis has sized up the market correctly, this tool should eliminate some bottlenecks that exist in the research world for RT-PCR. The company estimates that 10 researchers share the average existing machine that sits in a central lab.

The key enabling technology from Caltech, which makes this more broadly accessible, is in faster, simpler thermocycling. That really means that Helixis can heat up and cool down biological samples faster than the older machines, and make more copies of DNA for analysis. The Helixis approach also involves more precision, offering temperature variation from sample to sample that only sways 0.1 degrees Centigrade during an experiment, instead of 0.5 degrees on existing machines. That means there is one less variable researchers have to worry about that might mess up their experiments, Dickinson says.

That difference might not sound like much, but Dickinson insists it is “a big advantage.”

So the science is cool. But part of the innovation story here also is in the marketing. Helixis isn’t following the standard playbook …Next Page »

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Luke Timmerman wrote:

Two big names from the Seattle high tech and biotech scene—Rick LeFaivre of OVP Venture Partners and Pathway Medical’s Tom Clement—are taking new jobs at the University of Washington to help turn some of its most promising research ideas into new startup companies.

LeFaivre and Clement are joining the rebranded UW Center for Commercialization as key deputies to vice provost Linden Rhoads, the high-tech entrepreneur who was hired 18 months ago to light a fire for entrepreneurship on campus. LeFaive will split his time evenly between the university and his other job as a managing director at OVP in Kirkland, WA, while Clement is expected to work full-time for about 18 months, Rhoads says.

Janis Machala, the super-connected startup adviser who Rhoads hired as her deputy in November 2008, is leaving the UW at the beginning of February to go back to her consulting business. Machala says she feels “great” about the progress made in stirring more commercial activity at UW in 2009. “New VCs are also engaging from the Bay Area and other locales now that they are seeing commercial progress” coming from UW research, she says.

The hiring of two people with deep expertise in high tech and cleantech (LeFaivre) as well as life sciences (Clement) is a recognition of how the UW needs specialized talent on staff to help nurture more startups, Rhoads says. The UW conducted more than $1 billion worth of research in 2008, paid for mostly by the feds, charitable foundations, and corporations. It ranks second in federal research funding nationally behind Johns Hopkins University. Yet before Rhoads and Machala came aboard, business leaders long complained that the university was mediocre at best when it came to transforming all that research into new startups or useful products. Getting more experienced industry talent in house to mentor scientists and engineers about the ways of business is a key part of bridging that traditional gap, Rhoads says.

Rick LeFaivre

“This takes a lot of expertise. I know a lot about tech startups, but I think it sums it up to point out that two 23-year-olds can’t start a biotech company,” Rhoads says. “There’s a lot of domain expertise required in knowing the safety and efficacy requirements, regulatory issues, product reimbursement.” A similar thing is true for cleantech, she adds.

That rationale was what led her to LeFaivre and Clement. “I wanted to make sure we had the right expertise on both sides of the house,” she says.

Both of these guys are already familiar faces in the local innovation community. LeFaivre, 62, has a doctorate in computer science and formerly worked as a professor at Rutgers and UC San Diego. He has a long track record in the tech world, including stints in management roles at Apple Computer, Silicon Graphics, Sun Microsystems, and Tektronix, before he joined OVP. LeFaivre lately has been carving out greater expertise in the cleantech scene, and he was OVP’s point man on its 2008 investment in EnerG2, an intriguing UW spinoff that’s seeking to develop more efficient ways to store energy.

Tom Clement

Clement, 54, is known as one of the most successful medical device entrepreneurs in the Northwest, after a two-decade run as a co-founder of Heart Technology and Pathway Medical Technologies. He stepped down as CEO of Pathway a year ago as the company graduated from pure R&D mode into commercialization mode with its novel tool that drills through, and vacuums out, all sorts of fatty blockages and clots in leg arteries.

While he has backed off from day-to-day demands of running a company, Clement has stayed busy. He’s the chairman of the Washington Biotechnology & Biomedical Association, and has spent the last eight months or so as an entrepreneur-in-residence at UW for scouting and advising budding medical device entrepreneurs.

“A lot of people might say, ‘Why take this job?’” Clement says. “I haven’t had a boss for a while, and there’s bureaucracy. But I’m really very excited, and Rick is too, that I can help form connections and do something positive for the community. It’s also not a commitment for the rest of my life. I’ll get an opportunity to run another company, which I do want to do.”

Part of Clement’s responsibility will be …Next Page »

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Luke Timmerman wrote:

Amylin Pharmaceuticals CEO Dan Bradbury likes to list his company’s priorities in a well-organized series of little bullet points. But this is the year the San Diego-based company is counting on one event to dwarf all others, as it seeks FDA approval for an injectable diabetes treatment that only needs to taken once a week.

I met Bradbury at his hotel last week during the JP Morgan Healthcare Conference in San Francisco, just a few hours before he gave a speech about the year ahead.. We discussed all the usual highlights about how Amylin (NASDAQ: AMLN) is (still) striving to turn consistently cash-flow positive by the end of this year, boost sales of its existing products, and advance some of the interesting products in the pipeline—including the novel obesity drug that few people are paying much attention to at all.

But that’s really taking a backseat to what is expected to happen by March 5. That’s the FDA’s deadline to complete its review of Amylin and Eli Lilly’s application to start selling exenatide once-weekly in the U.S. This is a first-of-its kind treatment that takes the active ingredient in Amylin’s top-selling drug, marketed as Byetta, and combines it with a polymer from Cambridge, MA-based Alkermes to make it last longer in the bloodstream. This is potentially a huge advance, in that the existing drug must be taken through twice-daily injections, while the newer one can control blood sugar levels more effectively with just one shot a week. Patients won’t have to worry as much about the peaks and valleys of drug concentration in their bloodstream that leads to a lot monitoring through pinpricks of blood.

An estimated 25 million people in the U.S. have diabetes, and the incidence is expected to double over the next 25 years as more young people get diagnosed, Bradbury says. Based on the average of nine Wall Street analysts, who are predicting revenues of $1.45 billion for Amylin in 2013, the bet appears to be that the company will double its sales from the current form of exenatide. The new drug has been tested in head-to-head studies against big-sellers like Merck’s sitagliptin (Januvia), Takeda Pharmaceuticals’ pioglitazone (Actos), and Sanofi-Aventis’ insulin glargine (Lantus) that are taken once a day.

Dan Bradbury

“We have a tremendous opportunity with exenatide once-weekly,” Bradbury says “This product has demonstrated efficacy that’s greater, in terms of glucose lowering, than all commonly used branded diabetes medicines. It has the potential for weight loss. It’s dosed just once a week to provide continuous control. That means you have control 24 hours a day, seven days a week, 365 days a year. That whole concept of just providing control all the time is really important.”

Many stock analysts are predicting that Amylin and Lilly will be delayed by the FDA for one reason or another. This will obviously be one of the key points of speculation over the next six weeks, so I pressed Bradbury for a little more information that might help people handicap this long-anticipated event.

Right before Christmas, the FDA completed its inspection of Amylin’s $500 million factory in Ohio, which is the only place in the world set up to manufacture exenatide once-weekly, Bradbury says. The agency’s inspectors made some “observations” which he wouldn’t characterize, although he tried to downplay their significance. The agency’s remarks are “addressable,” probably without …Next Page »

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Luke Timmerman wrote:

Ekos has been hearing scuttlebutt from doctors over the past few months about a new way of using its ultrasound technology to treat dangerous blood clots in a new organ—the lungs. The company hadn’t seriously considered investing in it, but over the past few months, anecdotes from doctors kept pouring in. So now the Bothell, WA-based company is spending some serious bucks to test this idea, and see if it can generate real medical evidence that it hopes will crack open a lucrative new market.

Regular readers of this space know that Ekos has been around a long time, and it has had its ups and downs with its novel technique for ultrasound-guided therapy. The technology uses a miniature ultrasound probe that slithers inside blood vessels, and gently amplifies the effect of clot-dissolving drugs. The intent is to get rid of clots faster, which ought to help people live healthier lives after they get out of the hospital.

Ekos raised $12.5 million a year ago to commercialize this system for its first big application—blood clots in the legs, known medically as deep vein thrombosis. This is a chronic condition that affects 250,000 people in the U.S. each year, and puts them at risk for clots that break off and create blockages in the lungs. These are serious blockages, known as pulmonary embolisms, that are thought to kill 100,000 people in the U.S. each year, making it the third-leading cause of death in hospitals, according to the National Institutes of Health.

Ekos has had some modest success by trying to convince doctors they need to aggressively treat deep vein thrombosis patients—the company is on track to reach cash-flow break even later this year. But a number of doctors, including Peter Lin of Baylor Medical College in Houston, reported anecdotal success stories that convinced the company it ought to go after sicker patients who have what are known as “sub-massive” pulmonary embolisms that have already gotten to the lungs.

So after going over the anecdotes and doing the market research, Ekos has decided the doctors might be onto something. About 40 percent of all patients with pulmonary embolisms have the “sub-massive” forms, which means they have a one-in-five chance of dying within 90 days, yet they are thought to remain healthy enough for an interventional treatment like catheter-guided ultrasound, says Ekos CEO Bob Hubert.

“Deep-vein thrombosis is not life-threatening, but pulmonary embolisms are life-threatening,” Hubert says. “Deep vein thrombosis reduces quality of life, but we think we’ll get a lot more attention with a [pulmonary embolism] solution. If you have a patient with this, you need to intervene.”

While medical device companies often get flak for running clinical trials that are sketchy at best, Ekos is staking out a plan which could provide some solid medical evidence that says its treatment works.

The plan is to enroll 50 patients with the pulmonary condition who are randomly assigned to get either a standard blood-thinner, or a low-dose of a clot-busting drug that’s being gently …Next Page »

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Luke Timmerman wrote:

Merck hasn’t said much in public about what it’s doing in the field of RNA-based therapies, since it paid the jaw-dropping sum of $1.1 billion to acquire Sirna Therapeutics back in October 2006. So when I had the chance last week to sit down for an exclusive interview in San Francisco with Merck’s RNA therapeutics leader, Alan Sachs, I jumped at it.

What’s the big idea? RNA-based therapies hold the promise of silencing specific disease-related genes in ways conventional drugs don’t. They can potentially reach targets inside cells that have previously been inaccessible for a whole array of diseases.

This promise of a new wave of pharmaceuticals has enticed a generation of RNA-based drug companies to exploit this emerging science, including Cambridge, MA-based Alnylam Pharmaceuticals (NASDAQ: ALNY), Vancouver, BC-based Tekmira Pharmaceuticals, and microRNA drug startups like Carlsbad, CA-based Regulus Therapeutics. So whatever Merck does has a big impact not just on its shareholders, but ripples through an emerging technology sector.

“This is a big story as it relates to RNA therapeutics in general,” says Regulus CEO Kleanthis Xanthopoulos.

There was a lot of ground to cover during my 45-minute interview with Sachs. Before diving in, I should provide a little background on him. He’s a former professor of molecular and cell biology at the University of California Berkeley. He joined Merck in July 2001, and has been given a lot of managerial experience running a couple of leading edge operations inside Merck, including Sirna and Rosetta Inpharmatics.

Here are the highlights of the conversation, edited for length and clarity as always. Merck spokesman Ian McConnell was also there for the meeting.

Xconomy: How did you first get exposed to RNAi?

Alan Sachs: At Berkeley, I worked on RNA post-transcriptional control. I joined Merck in 2001 to help start a clinical genomics group. At that time, we had just acquired [Seattle-based] Rosetta Inpharmatics, and so that first year I worked with [Stephen Friend, Rosetta’s founder] to build up a molecular profiling unit which included gene expression genetics, proteomics, [and] informatics of course.

Alan Sachs

Molecular profiling at Merck was broad. It extended beyond Seattle to research sites in Boston, West Point, PA, just outside of Philadelphia, and Rahway, NJ. Then as part of the work in Seattle, we were developing siRNA as a tool for [drug] target discovery using cell-based screens. Thanks to Stephen, we really had more of a therapeutic push. We initiated a partnership with Alnylam, it must have been around 2005.

Somewhere around the middle of 2006, we realized that Alnylam did a big deal with Roche, and we realized the area was heating up. We needed to do more than a collaboration per se. It ultimately led to the decision to acquire Sirna Therapeutics, which closed at the end of 2006.

So I was asked at that time to lead a new department at Merck called RNA Therapeutics. The Sirna site which you’re sitting in now is a geographic identifier. The department at Merck is called RNA Therapeutics. Sirna San Francisco is responsible for lead discovery and optimization. On the East Coast, we have a very large effort in RNAi delivery in West Point, PA. In Rahway, NJ, all of our manufacturing of oligonucleotides and delivery vehicles occurs. So RNA Therapeutics is split over three research sites within the company.

What you often read about, but many people don’t understand, is how hard …Next Page »

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Luke Timmerman wrote:

News flow was light on the biotech beat this week, partly because of the MLK Day holiday, and everybody needed a little rest after the networking frenzy at the JP Morgan Healthcare Conference.

—One of the more interesting interviews I had last week at the JP Morgan conference was with Hans Bishop, the new chief operating officer for Seattle-based Dendreon (NASDAQ: DNDN). He talked about manufacturing capacity, the need to hire a marketing ace, and even how to correctly pronounce his name.

—Bothell, WA-based Acucela has quietly embarked on an ambitious growth plan this year. CEO Ryo Kubota told me in an exclusive interview that the company is planning to expand its staff from 40 to about 100 this year as it advances its novel drug for age-related macular degeneration further along in clinical trials.

—Instead of doing the usual recap on the JP Morgan Healthcare Conference like every other news outlet which said the mood was upbeat, I ruminated on the value of all the impromptu meetings that happen when so many people gather and pool their energy in one place.

—We are getting close to the end of the amazing series of editorials we’ve been been running daily from Xconomists and other technology leaders who are offering their views on the big innovations of the coming decade. This past week, we ran a piece on innovations in genomics from Eric Schadt, the chief scientific officer of Pacific Biosciences and a co-founder of Sage Bionetworks, as well as a great editorial about the future of molecular medicine from Matt O’Donnell, the dean of the University of Washington College of Engineering.

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Luke Timmerman wrote:

Brain teaser time: Name the Bothell, WA-based biotech company that has never raised a nickel from local VCs, has scored a partnership with a Big Pharma company, operates on a cash-flow positive basis, and plans to more than double in size from 40 employees to about 100 this year.

Think it’s Alder Biopharmaceuticals? Guess again. It’s Acucela.

Acucela has the ambitious goal of fundamentally changing the way doctors treat the so-called “dry” form of age-related macular degeneration, which damages the eyesight of an estimated 29 million elderly people worldwide. The company was spun out of the University of Washington in 2002 by Ryo Kubota, an ophthalmologist on the faculty at the time. His dream is to create a once-daily oral pill that can slow down the progressive vision loss that comes with the dry form of macular degeneration, and ultimately to keep these aging patients from going blind.

The company got a big lift in September 2008 when it struck a partnership with Japan-based Otsuka Pharmaceutical to co-develop its lead drug candidate, and this month it took another big step by entering the first clinical trial that will assess what kind of effect its drug will have on the eyesight of between 50 to 100 elderly patients.

“We want to build a great company, and we want to build it to last. Our investors and our partner believes in it,” Kubota says.

Since Acucela doesn’t usually make a lot of news, some background is certainly in order. Acucela is vying to develop the first effective therapy for the dry form of age-related macular degeneration. It’s caused by yellowish buildups and a loss of pigment in the retina as people age. There is no approved treatment for this disease, unlike the “wet” form of age-related macular degeneration, which can be treated with Roche’s bevacizumab (Avastin) and ranibizumab (Lucentis), which cut off blood flow to leaky vessels behind the eye. The dry form of the disease is generally milder, but the market opportunity is thought to be huge, because as populations around the world age, it is expected to double in incidence over the next two decades.

Acucela’s idea comes in the form of a once-daily pill it calls ACU-4429. The drug is designed to block an enzyme called isomerase. By blocking that enzyme, Acucela hopes to slow down the visual cycle in which Vitamin A is constantly replenished. While people need a constant source of Vitamin A to see, a fast visual cycle can also contribute to the buildup of toxic Vitamin A byproducts in the eye, particularly one called A2E.

Back in May, Acucela presented the first evidence from a clinical trial that said its drug was on track. The initial trial enrolled 36 healthy volunteers at an escalating series of doses, and basically concluded that the drug was safe and well-tolerated.

This month, Acucela is attempting to pass a tougher test. The company started enrolling elderly people with severe cases of the dry form of macular degeneration. The trial, called Envision, will randomly assign patients to get the drug, or a placebo, at a variety of once-daily doses. Patients will be followed up for three months, and researchers will look at how well they can read letters on the eye chart, and a secondary goal that will assess whether the drug is working as intended, by reducing the amount of toxic byproduct buildup in the eye. It’s possible that the trial, as long as it appears safe at escalating doses, could enroll as many as 100 patients, Kubota says. It’s expected to take about one year to complete enrollment, and results should be available by mid-2011, he says.

Although Acucela is entering uncharted territory with a new way of treating this disease, it has competition from Tampa, FL-based Sirion Therapeutics. The competing drug is an oral pill like Acucela’s, made to circulate through the bloodstream, although it is designed to hit a different target than Acucela’s, Kubota says.

While Acucela awaits results from its mid-stage clinical trial, it is working to build up a lot of organizational strength this year, Kubota says. People with expertise in clinical development, preclinical development, and regulatory affairs are being recruited this year. By the end of 2010, Acucela expects to have hired about 60 new people, bringing its staff to about 100, he says. The company hopes to have that organizational horsepower in place by the time it expects to enter the final phase of clinical trials with ACU-4429, for an even more rigorous study that will assess patients’ vision for 18 to 24 months, Kubota says.

Although it’s rare among biotech companies without marketed products, Acucela has been able to operate on a cash-flow positive basis of late, Kubota says. That’s because Otsuka is paying for the company’s R&D expenses, while Acucela has received undisclosed milestone payments for getting this far in development, Kubota says.

“We’re going to be running big clinical trials,” Kubota says. “We think we can change the disease paradigm. Our goal is to cure blindness. It’s a huge ambition.”

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Luke Timmerman wrote:

The new CEO of Quanterix isn’t afraid to dream big, and say it out loud.

“We participate in an $8 billion market,” says CEO Dave Okrongly, referring to the business of antibody-based diagnostics. “Quanterix can be a platform for that whole $8 billion market.”

Okrongly was named CEO of the Cambridge, MA-based company in September, and now that he’s been settling in for a while, I got a fresh update just yesterday. The company is the latest brainchild of Tufts University chemistry professor David Walt, who previously hit gold with Illumina (NASDAQ: ILMN), the San Diego-based genetic analysis powerhouse. Quanterix hasn’t said much publicly since it raised $15 million in venture capital back in August 2008, so I’ve been curious for some time about the company’s game plan for approaching the first segments of that $8 billion antibody-based diagnostics market.

Before diving into that, I first wanted to know a little about Okrongly. It turns out he’s got a doctorate in chemistry, and a resume with a lot of commercial experience, most recently as the senior vice president of the molecular diagnostics unit at Siemens Healthcare Diagnostics. Earlier in his career, he caught the startup bug at Applied Immune Sciences, an early gene therapy company that was acquired in 1995 by what’s now Sanofi-Aventis.

So while Okrongly, 51, rose in the ranks at big companies, and introduced a number of clinical diagnostic tests around the world, he felt the urge to come back to a startup to create something new. When he first heard about Quanterix, he didn’t think it was for him. “It seemed early and raw,” he says.

Then he met personally with Walt, who changed his mind. “I thought, holy cow, this could change the whole way we do immunodiagnostics. I gotta give this a run. This is a transformative technology with top-notch people around it.”

Dave Okrongly

Walt is equally enthused about the new leader at Quanterix. “Dave has exactly the right blend of diagnostics experience and strong technical background. He has helped push the company’s market strategy forward while helping it achieve its technical milestones,” Walt says.

The initial idea at Quanterix is to find a way to detect trace quantities of proteins in the blood, which could be an early warning sign for cancer or a neurodegenerative disease like Alzheimer’s or Parkinson’s. Quanterix says its test is about 1,000 times more sensitive than the gold standard ELISA assays. The older tests employ specific antibodies that are made to bind with certain biomarkers, known as antigens.

Some of these older tests can be quite cheap and reliable, but they also have their limits. One of the emerging ideas in biology is that tumors and other diseased tissues spill off specific proteins in the bloodstream, but these proteins generally come in trace amounts too small to be detected by the standard ELISA tests. The Quanterix system is designed to detect thousands of single molecules simultaneously, with proprietary chemistry and what the company calls “a relatively simple” instrument with a light source, optics, a digital camera, and an automated handling system. Software is then used to analyze the images.

Quanterix is betting that its ultra-sensitive tests can fill some of the gaps that ELISA tests struggle with. One well-known example is the prostate-specific antigen (PSA) screening test …Next Page »

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Luke Timmerman wrote:

Dendreon has a new man on the spot, and his name is Hans Bishop. The Seattle biotech company (NASDAQ: DNDN) settled the raging debate last year about whether its immune booster can help men with prostate cancer live longer without serious side effects. Now the company has graduated to a less glamorous, but equally important phase in which it must show that it can effectively manufacture and market its first-of-a-kind drug, sipuleucel-T (Provenge).

That responsibility falls to Bishop, its new chief operating officer. Bishop, 45, is a native of the U.K. who most recently worked as president of the specialty medicine business at Bayer Healthcare. He officially started at Dendreon on Jan. 4. He and his wife—who oversees communications at the Bill & Melinda Gates Foundation—have already moved into a new place in downtown Seattle which is about a 15 to 20-minute walk from Dendreon’s Belltown office.

I met Bishop and Dendreon’s vice president of communications, Katherine Stueland, while they were attending the JP Morgan Healthcare Conference last week at San Francisco’s Union Square. Before diving too deep, it’s worth noting that Bishop’s first name is correctly pronounced as “Hahnce,” not the Germanic-sounding “Hahnz.” He’s heard it both ways, and told me he doesn’t care when people get it wrong, but there you have it.

Like most of our interviews, I sought to get to know the person as well as the situation he is stepping into at an important local biotech company. Here are the highlights of the conversation.

Xconomy: Where are you from, and how did you get started in the pharmaceutical business?

Hans Bishop: I’m from New York, in the sense that’s where I was living last. My last job was as president of specialty medicine at Bayer. That was a 3 billion Euro business comprised of oncology, hemostasis, urology, and ophthalmology. It’s all of the specialty medicine parts of the Bayer business.

Hans Bishop

But I was born and raised in the U.K., trained as an organic chemist. I’ve spent pretty much all of my career, not all, but pretty much all, in healthcare and pharmaceuticals.

X: How did you find out about the Dendreon position?

HB: I met [CEO] Mitch [Gold] and the team during a business development discussion. I’m embarrassed to tell you that I wasn’t that aware of Dendreon in my prior job. I met them through business development talks, and I was really amazed. I learned about the product for the first time when I met with them, and the clinical data they had generated. I was really impressed with the product, all the technology they had built around the product, and the management team. So I came away really impressed. I was really delighted two weeks later when the phone rang and I was asked if I was interested in meeting with them about this position.

X: So Mitch Gold recruited you, after he met you when you were on the other side of the bargaining table?

HB: You should get the quote from Mitch, but Mitch clearly came away impressed with me. While it was unclear if he wanted Bayer as a partner, it was clear that he wanted …Next Page »

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Luke Timmerman wrote:

Sequenom (NASDAQ: SQNM) has reached a legal settlement in a class action suit filed by a group of  shareholders. The San Diego-based company said today that it has agreed to pay shareholders $14 million from its insurance proceeds, and issue them new shares worth a 9.95 percent stake in the company.

The settlement still needs approval from the U.S. District Court in for the Southern District of California. The settlement agreement doesn’t include any admission of liability, but the company is settling the lawsuit “to avoid potentially lengthy, costly, distracting and time-consuming litigation,” according to a statement.

Sequenom’s troubles surfaced last April 29, when it said it “mishandled” clinical trial to support the commercialization of its non-invasive prenatal test for Down syndrome. The company hasn’t fully explained what happened, but after an internal investigation, the company ousted CEO Harry Stylli, former R&D chief Elizabeth Dragon, and three other employees in September. The SEC has opened an investigation to the company’s mishandling of its data and the Justice Department also is asking questions. There was no update today on the status of those inquiries.

Sequenom’s chairman and interim CEO, Harry Hixson, said in a statement that settling the shareholder lawsuit, “is in the best interest of the company and its shareholders. I look forward to closing this chapter for the company and focusing on meeting our 2010 milestones, which include the launch of a number of molecular diagnostic tests.”

Shares of Sequenom climbed about 5 percent to $4.35 at 10:05 am Eastern time after the announcement.

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