Bor-Wen Wu says he had the North Star in mind in 2006 when he founded San Diego’s Polaris Group, a small holding company with a promising lead drug candidate for treating liver cancer, malignant melanoma, and other related cancers. As an explorer in science, Wu says, “I need a North Star to tell me where to go.”

Yet the path Wu has followed has been anything but a sure and constant course. In his quest to develop the drug ADI-PEG 20, Wu has formed eight companies since 2002 that are affiliated with Polaris; raised more than $60 million from individual investors in Taiwan; and battled to retain control of ADI-PEG after paying millions to acquire a predecessor company, Phoenix Pharmacologics of Lexington, KY.

Despite a sometimes-circuitous path, though, Wu has kept the Polaris Group focused on a distant goal. The FDA recently approved the company’s plans for a late-stage clinical trial of ADI-PEG 20, an enzyme also known as pegylated arginine deiminase. ADI-PEG 20 is incredibly effective in breaking down arginine, an amino acid that is critical to the growth of hepatocellular carcinoma—the primary type of liver cancer.

Among cancer drugs in Phase 3 trials, Wu boasts, “We’re not the first in class. We’re the only one in the class. There’s nothing in the rear-view mirror.”

A study published last year in the British Journal of Cancer estimates there are 500,000 new cases of hepatocellular cancer diagnosed worldwide annually, with a five-year survival rate of less than 10 percent in the United States and Europe. Polaris, which contends the liver cancer is far more prevalent in Asia, estimates that worldwide deaths from hepatocellular carcinoma is closer to 700,000 people a year, with more than 330,000, or nearly half, in China.

ADI-PEG 20 represents an especially hot area of cancer research, which has focused on finding ways to starve tumors by depriving them of key nutrients. In the case of liver cancer, Wu says a key genetic mutation that triggers hepatocellular carcinoma coincides with the specific gene that makes arginine in normal cells. The company says the correlation is more than 70 percent in the patients studied so far. As a result, most liver tumor cells are unable to manufacture their own arginine and depend on some other source of … Next Page »

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French drug giant Sanofi-Aventis’s long-sought takeover of Cambridge, MA-based biotech company Genzyme (NASDAQ:GENZ) might happen within the next week or so, sources told The New York Times yesterday. The report came the same day that Genzyme announced that it had opened its books to Sanofi to allow its potential buyer to conduct due diligence.

Sanofi—which made its desire to buy Genzyme for $18.5 billion or $69 per share public back in August—has agreed to raise its acquisition offer by an unspecified amount, The Boston Globe reported today. Now the two sides are said to be at the table working out the details of the deal, which would put the Bay State’s largest biotechnology outfit in the hands of the Paris-based drugmaker.

Genzyme’s stock closed at $73.35 on Monday, giving the company a market cap of $19 billion, according to Google Finance. The closing price was just shy of its 52-week high of $73.46 per share.

A key aspect of closing the deal appears to hinge on the two companies coming to terms on the value of Genzyme’s experimental multiple sclerosis treatment, alemtuzumab. While Genzyme sees the drug, which is already approved as a treatment for leukemia, as a potential $3.5 billion seller, Sanofi expects the drug to reach only about $700 million in annual sales, according to The Times report.

The Globe reports that the buyout deal will likely include terms that would provide additional payments to Genzyme shareholders if alemtuzumab hits certain financial targets. This term would obviously be a big compromise for Genzyme, and the company’s long-time chairman and CEO, Henri Termeer, is known for getting his way in negotiations.

Termeer has insisted for months that Sanofi’s offer undervalued Genzyme, a global leader in treatments for rare genetic diseases, and its potential multiple sclerosis drug. We’ll see whether the companies can strike a deal in the next week or so.

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It was a busy New England area life sciences news week, with headlines of drug collaborations, failed applications with the FDA, clinical trial results, plus some lengthier profile stories.

—Providence, RI-based Shape Up The Nation is using social networking to invite people’s friends to encourage them to eat better and make healthier choices, Ryan wrote. The four-year-old startup raised $5 million earlier this year from Excel Venture Management and Cue Ball Group, landed CVS Caremark (NYSE: CVS) as its first big customer, and is adding other Fortune 500 companies to the list.

—The diabetes drug developed in a collaboration by San Diego-based Amylin Pharmaceuticals (NASDAQ: AMLN), Waltham, MA-based Alkermes (NASDAQ: ALKS), and drug giant Eli Lilly (NYSE: LLY) failed to garner FDA approval, the companies announced on Tuesday. The regulatory agency is requiring a new study looking at whether the drug—the once-weekly injectable version of exenatide (Bydureon)—is connected to irregular heartbeat in trial patients. The application for FDA approval of the drug was delayed earlier this year, due to manufacturing questions and the agency’s request for a risk mitigation strategy of the drug. By Wednesday morning Amylin’s and Alkermes’ share prices had fallen 50 percent and 29 percent, respectively. Lilly, with a more diversified drug pipeline, saw its shares drop by about 5 percent.

—Cubist Pharmaceuticals (NASDAQ: CBST), a Lexington, MA-based maker of acute care drugs, announced it had priced a $400 million offering of convertible notes, which have an annual interest rate of 2.5 percent and mature on November 1, 2017. The deal underwriters have the option to purchase $50 million in additional notes. Cubist will put the proceeds toward repurchasing about $191 million in notes that carry a 2.25 annual interest rate and are due June 15, 2013, as well as its investing in its pipeline products, corporate expenses, and acquisitions.

—Cambridge-based Genzyme (NASDAQ: GENZ) reported data from a late-stage clinical trial comparing its drug clofarabine (Clolar) in combination with chemotherpy to treatment with chemotherapy and a placebo. Those treated with the drug did not live longer than those only given the placebo, but the clofarabine group saw its remission rate double, to 47 percent, Genzyme said. The firm will … Next Page »

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[Editor's note, Aug. 23, 2010: Updated and corrected, see below.] David Schenkein, the CEO of the biotech startup Agios Pharmaceuticals, walked into the firm’s Cambridge, MA, headquarters last week with building plans rolled up in his hand. It was evident that his three-year-old company is growing quickly to pursue new drugs to starve cancer cells, and that it has the funds to do it because of the $130 million the firm received this spring through a deal with the cancer drugmaker Celgene (NASDAQ:CELG).

Summit, NJ-based Celgene’s large payment to Agios is uncommon. Not only is it a huge sum for a young biotech to grab, the deal gives Celgene the exclusive option to license any of the potentially cancer-starving drugs that Agios discovers for a certain period of time (neither company will say how long). The unique quality of this deal prompted me to head down to Agios’s labs and also talk to Celgene last week to learn why exactly this deal came together.

For starters, Schenkein has always been clear about his desire to keep Agios on the cutting edge of the emerging cancer metabolism field. The firm wants to develop drugs that target mutated enzymes that are believed to be culprits in feeding certain cancer cells’ addiction to specific nutrients that enable them to grow out of control. The idea sounds simple: wipe out the mutated enzymes, starve the cancer cells to death. But key discoveries that have exposed these cancer-enabling enzymes have only just surfaced in academic journals over the past several years, Schenkein says, even though it’s been known since the 1920s that cancer cells metabolize nutrients differently than healthy cells do.

This emerging field is drawing interest from drugmakers around the world. To find its own drugs to target cancer metabolism, British drugmaker AstraZeneca says it formed a three-year research deal with Cancer Research UK in February. London-based drug giant GlaxoSmithKline has been assembling its own internal drug discovery group to find molecules to home in on cancer metabolism targets. Those are just two of several groups in the hunt. And all alone, it’d be impossible for Agios to match the level of resources that larger outfits like AstraZeneca and Glaxo can deploy.

“We view this space as really beginning to explode,” Schenkein said. “Over the next three to five years, as more players move into this space, it’s going to be a bit of a … Next Page »

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This week we were jamming on our big event on the 20-year outlook for Seattle’s life sciences hub, but we still found a way to squeeze in a lot of news and features.

—Biotech pioneer Chris Henney offered an insightful and entertaining talk about how to invest in biotech at a recent speech before the CFA Society of Seattle. I included his six tips on how to spot a winning biotech in last week’s roundup, but that piece didn’t include the sequel on the five red flags investors should watch out for. The In Vivo Blog did a fun follow up on this story, too.

—If Seattle wants to grow as a biotech hub over the next 20 years, it could use a few more bars, and less university red tape. That’s according to Stephen Friend, the Rosetta Inpharmatics founder who’s now trying to ignite an open source movement for biology, in comments he made at the Xconomy event Monday evening on the 20-year outlook for Seattle life sciences. I summed up some of the other great insights I heard from his fellow panelists Ben Shapiro and Steve Gillis, and also posted a big photo gallery here that captured the energy in the room.

—Kirkland, WA-based Pathway Medical Technologies is one of the big success stories of the past year in the local medical device community, but even it has had to endure a few lessons from the school of hard knocks. CEO Paul Buckman offered a candid look at how the company is adjusting to the new reality of the medical device business.

—Amgen (NASDAQ: AMGN), the giant biotech company with 900 employees in Seattle, said its experimental drug for osteoporosis, denosumab (Prolia), was delayed by FDA requests for more information. That didn’t seem like much of a big deal at first glance because the FDA didn’t ask for any new clinical trials, and it has been missing a lot of deadlines lately. But then Amgen said yesterday in its earnings release that the FDA is asking for more clinical trials to demonstrate safety of denosumab for cancer patients. Ouch.

—Sonosite, the Bothell, WA-based maker of portable ultrasound machines, got a lift this week when it settled all of its patent litigation with General Electric over ultrasound devices that weigh less than 10 pounds. Sonosite (NASDAQ: SONO) will get $21 million upfront and an undisclosed royalty on sales of GE’s portable ultrasound machines until 2016.

—Seattle-based Calistoga Pharmaceuticals released some preliminary, but encouraging, results over the weekend from a small leukemia trial. The company’s experimental drug was able to shrink tumors for 29 percent of patients in the trial, but based on some secondary findings, the company thinks it might be able to do much better than that in combination with other treatments, or with longer follow-up.

—San Diego-based Fate Therapeutics, the company co-founded by a group of top stem cell scientists that includes Randall Moon of the University of Washington, announced over the weekend that it has taken a big step toward “industrialized” production of stem cells for drug discovery. The advance for inducing adult cells into a pluripotent, stem-cell like state, came from the lab of Fate co-founder Sheng Ding, a professor at The Scripps Research Institute in San Diego.

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Fate Therapeutics plans to announce this morning that it has begun its first clinical trial of a drug built on its knowledge of stem cell biology. Rather than injecting certain kinds of adult stem cells to regenerate tissues, this treatment involves a conventional small-molecule drug that’s designed to spur growth of blood-forming stem cells that patients need to recover from certain kinds of leukemia and lymphomas.

Xconomy reported in March that La Jolla, CA-based Fate—which was founded by top stem cell scientists at Harvard University, The Scripps Research Institute, the University of Washington, Stanford University, and the Whitehead Institute for Biomedical Research—was destined to test its first drug in humans. But the company is now disclosing that the study will be done at the Dana-Farber Cancer Institute in Boston, and provided more details on how its treatment could better the way hematopoietic stem cells from umbilical cord blood are transplanted into some cancer patients to restore their bone marrow function.

Fate’s drug, dubbed FT-1050, is a small molecule that the company plans to use to treat blood-forming stem cells from cord blood before the cells are transplanted into humans. By activating certain genes in the stem cells, the drug is intended to boost the ability of the cells to multiply and take up residence in bone marrow, Pratik Multani, vice president of clinical development at Fate, explains. The clinical trial is testing this use of the drug in patients with blood or immune system cancers whose bone marrow function—which is vital to blood production and the immune system—has been destroyed by chemotherapy and other cancer treatments.

Fate’s treatment was initially discovered in the lab of Leonard Zon, the director of stem cell research at Children’s Hospital Boston. Zon is one of several stem cell experts who was recruited by Polaris Venture Partners and … Next Page »

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There are about 11,000 physicians in the U.S. whose job is to diagnose and treat people thought to have cancers of the blood. These specialty physicians need to sort through a dizzying number of lab tests to identify subtle differences in types and stages of leukemias, lymphomas, and myelomas. Get it exactly right, and it might cure the patient. Get it wrong, and it could be a disaster.

Most lab service companies specialize in running specific tests accurately and fast. They often get their orders one by one, and send back results that way, not in a comprehensive package for the hematologist/oncologists (known as heme-onks for short) who have to make the bottom line diagnosis. San Diego-based Genoptix (NASDAQ: GXDX) has seized upon this gap in the marketplace by bundling the tests a doctor needs to make such conclusions. Founder and CEO Tina Nova, a veteran biotech entrepreneur with experience at Hybritech, Ligand Pharmaceuticals, and Nanogen, filled me in on why her newest company has been so successful.

Something like 850,000 patients are living with blood malignancies of some kind in the U.S., and about 150,000 new cases are diagnosed each year. This typically involves a battery of expensive tests that are run by giant contract labs like Laboratory Corp of America, or Quest Diagnostics. Such tests require a small sample of a patient’s bone marrow to run analyses such as flow cytometry, which helps sort out malignant cells from healthy ones, and pathology tests, which examine cells under a microscope. More sophisticated analyses put cells in a lab dish and examine genes associated with malignancies.

Usually a combination of these tests will give the doctor an answer, like whether a patient has chronic myelogenous leukemia and is therefore a candidate for Novartis’ wonder drug imatinib (Gleevec) or whether he or she needs some other treatment altogether. Nova says Genoptix is the only company that runs all the tests in-house and puts together a report that offers a diagnosis under the direction of employees trained in hematology/oncology. As Nova put it, they speak the same language as the physician.

“No one else uses a model like ours,” Nova says. “We came to this because we learned that the diagnosis of leukemia and lymphoma was not well-served. The physicians told us that diagnosis is the biggest problem they are faced with.”

Diagnostics companies traditionally take a back seat to the more glamorous work of drug development, but Genoptix is clearly much better positioned heading into in the downturn than most traditional drug developers. Revenues were $59.3 million in 2007, which doubled to $116.2 million last year. Genoptix is profitable and expects to continue growing despite this year’s recession, with forecasted revenue this year of $170 million. Nova insists the company is just warming up, having captured just 6 percent … Next Page »

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Seattle-based Trubion Pharmaceuticals said today it is cutting one-fourth of its workforce, about 25 jobs, leaving it with a staff of about 75. The company says it expects the cuts will help it conserve enough cash to operate into the second half of 2010.

The Trubion (NASDAQ: TRBN) cuts will affect “most areas of the company,” a spokeswoman said. The restructuring is designed to invest in the company’s “strongest near-term opportunities,” including TRU-016 for chronic lymphocytic leukemia, and select drug candidates in animal testing that it says could become valuable over time. Trubion says it will “continue to support” the programs it has in a collaboration with Madison, NJ-based Wyeth, TRU-015 and SBI-087, a pair of experimental drugs for rheumatoid arthritis.

Trubion was dealt a big dose of uncertainty last month when the world’s largest pharmaceutical company, New York-based Pfizer, reached an agreement to acquire Wyeth for $68 billion. Trubion has no marketed products of its own, so it depends heavily on the partnership with Wyeth to support its research and development. A clinical trial of 270 patients who took Trubion’s lead drug, TRU-015, or a placebo, disappointed investors in the fall of 2007 because it didn’t show conclusive superiority over Genentech’s rituximab (Rituxan) for rheumatoid arthritis.

Wyeth has shown willingness to stick with Trubion’s plan, pushing forward another mid-stage trial that’s expected to produce results by the end of 2009, but investors have run low on patience. Trubion had about $61.6 million in cash and investments left at the end of September, the last time it reported financial results, yet investors are saying the company is worth a whole lot less than that, giving it a market value of $23 million. Trubion stock fell 87 percent during 2008.

For an updated look at job cuts at Seattle high-tech and life sciences companies, check out our updated layoff tracker.

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Merck is consolidating its cancer research in Boston, and now it has landed an academic research star from Harvard to run the place. The pharmaceutical giant said it has hired D. Gary Gilliland, a renowned Harvard cancer researcher, to be its new senior vice president of Merck Research Labs in charge of its oncology franchise.

Gilliland, 54, has spent 20 years on the Harvard faculty, where his work has won a number of awards, including the William Dameshek Prize from the American Society of Hematology, and support from the Howard Hughes Medical Institute. Gilliland’s research has explored the genetic basis of leukemia and other malignancies of the blood and bone marrow. His hiring “may raise the competitive juices of other big drugmakers that, like Merck, have stepped up their own pushes into cancer treatments,” says the Wall Street Journal’s Health Blog.

Gilliland will replace Stephen Friend as Merck’s cancer research boss. Friend joined the company in 2001 after he sold the startup he led, Rosetta Inpharmatics, to Merck for more than $620 million. Merck said in October, as part of sweeping cutbacks that include 7,200 companywide layoffs, that it plans to close down the 300-person Rosetta operation in Seattle. The Rosetta division uses computing techniques to try to predict how patients with certain genetic profiles will respond to drugs in development. The company plans to keep doing the work in a more comprehensive cancer research center in Boston, the company has said.

Friend is leaving later this year to help build an open-access database system that will allow scientists to better collaborate, Merck said in a statement. “Merck has been and will remain a key supporter of Dr. Friend on his ambitious and promising endeavor,” the company said in a statement.

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Seattle Genetics said today its experimental drug for acute myeloid leukemia passed an interim safety review. The Bothell, WA-based biotech company (NASDAQ: SGEN) said an independent monitoring committee recommended the study continue of lintuzumab (SGN-33). Full results from the trial, which is two-thirds of the way to its enrollment goal of 210 patients, should be available in the first half of 2010, the company said.

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The American Society of Hematology (ASH) is one of those scientific meetings that usually flies below the radar of the wire services and big newspapers. Still, this year’s event in San Francisco has drawn more than 21,000 physicians, scientists, and intensely interested folks from biotech companies and Wall Street. They are all scouting new treatments for diseases of the blood, like leukemias, lymphomas, myelomas, and every clotting disorder you can shake a stick at.

If you don’t think this sounds like a big deal, check out the sales of anti-clotting drug clopidrogel (Plavix): $4.7 billion worldwide for Bristol-Myers Squibb and Sanofi-Aventis in 2007, even while they faced competition from cheaper generics. Then there’s old ASH standbys like Genentech and Biogen Idec’s rituximab (Rituxan) for non-Hodgkin’s lymphoma, which eclipsed $2 billion in U.S. sales last year and keeps growing.

So, I’ve been keeping an eye on the next big thing for the blood. Here are the newsiest stories we saw cross our biotechnology desk in our three-city network of Boston, San Diego, and Seattle:

Seattle Genetics

The Bothell, WA-based biotech company (NASDAQ: SGEN) caused a stir with its “empowered” antibody drug, which has the tumor-targeting ability of an antibody loaded with a potent toxin to give it extra tumor-killing kick. The drug, SGN-35, was able to completely wipe out aggressive forms of Hodgkin’s disease and related lymphomas with minimal side effects. In a study of 44 patients who were seriously ill and relapsed after a median of three prior rounds of chemotherapy, the Seattle Genetics drug caused tumors to completely disappear or mostly go away in more than one-third of patients (38 percent).

This data is promising enough that Seattle Genetics is preparing to go straight to pivotal studies for Hodgkin’s disease and anaplastic large cell lymphoma in early 2009, which could lead this drug to be FDA approved. Seattle Genetics CEO Clay Siegall said investigators at the ASH meeting are itching to participate in the pivotal studies. “There’s a lot of buzz here,” Siegall said during a break, when he called me yesterday. “The investigators are excited. We’ve never had this much excitement in the 10.5 year history of Seattle Genetics. The doctors really want to get involved in the trial.” He adds that there’s a backlog of patients who could enroll in the next studies, and he’s hopeful that they can be recruited to sign up quickly.

Millennium: The Takeda Oncology Company

Millennium Pharmaceuticals had a breakout performance at last year’s ASH. The Cambridge, MA-based biotech company, now the cancer division of Japan-based Takeda Pharmaceuticals, showed last year that bortezomib (Velcade) could cause complete remissions for 35 percent of patients with multiple myeloma on a combination treatment with its drug, compared with 5 percent who did that well on the combination alone. This year, it showed follow-up data from the trial, called Vista, that showed that early glimpse of promise is translating into an ability to help patients live longer.

Ligand Pharmaceuticals

This San Diego-based biotech company (NASDAQ: LGND) saw some positive data reported by its partner, GlaxoSmithKline. The drug giant reported … Next Page »

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Genzyme said today it has applied for FDA approval of clofarabine (Clolar) for patients over age 60 with acute myeloid leukemia, in addition to its existing use for acute lymphoblastic leukemia. The Cambridge,MA-based biotech company (NASDAQ: GENZ) has asked for an expedited six-month review of the new use, which means the treatment could be approved for sale in the U.S. in the first half of 2009. The product, which Genzyme obtained through the $345 million acquisition of Bioenvision last year, has potential to generate peak annual sales of $600 million if it is cleared for multiple forms of leukemia, Genzyme said.

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