Life Science

Bruce V. Bigelow

Waiting for the biotech industry to advance new treatments for male pattern baldness (as well as female hair loss) might seem about as exciting as watching hair grow. But some people have a lot of skin in the game, if you know what I mean.

More than just a few people, actually. San Diego’s Histogen estimates that hair loss affects some 40 million men and 21 million women in the United States alone. Histogen, which has been developing a bio-engineered treatment for stimulating hair growth, says less than 7 percent seek treatment for their hair loss “due to the limitations of available options.”

For these people, any incremental improvement in hair growth can be thrilling—even life-changing—which helps explain a burst of enthusiasm we’ve noticed in recent days in messages penned below a 2011 article about Boston-based Follica on the Xconomy Boston website. With close to 1,500 comments posted over the past 14 months our readers have shown no loss for words, making this one article a defacto online message board and a virtual support group for the follically challenged.

As I reported in 2010, few life sciences companies have gotten as much mileage from a pilot trial that enrolled two dozen patients as Histogen has for its much-anticipated treatment. The company says its formulation consists of proteins, including growth factor molecules, secreted by human fibroblast cells grown in a laboratory culture. The concoction is injected just below the scalp.

Over the past week, gentle and not so-gentle visitors to the Follica comment page have seized on the early results of Histogen’s latest experimental trial, which Histogen CEO Gail Naughton presented a week ago in Raleigh, NC, during the annual meeting of the Society for Investigative Dermatology. Naughton gave an oral summary of the data on May 11 and presented a poster the following day on “Stimulation of hair growth in humans by cell-secreted proteins.”

The data has not yet been published in a peer-reviewed scientific journal, according to a Histogen spokeswoman. “We are not releasing an announcement at this time,” she adds.

Yet Histogen has summarized its early findings on its website, where a PDF copy of the May 12 poster also can be downloaded. The company says 56 patients were enrolled in … Next Page »

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Luke Timmerman

CardioKinetix has spent 10 years and $80 million in venture capital working on an implantable device for people with heart failure. Now it’s got some evidence that suggests it could be on track with a real product.

The Menlo Park, CA-based company is announcing today it has passed a study of 31 patients who got an implantable device designed to help the heart beat more efficiently after the muscle is damaged by a heart attack. About 16 percent of the patients died or had to be hospitalized within one year of getting the heart failure implant, and 32.3 percent suffered that fate within two years.

The trial lacked a control group, so it’s impossible to directly compare how those patients would have done otherwise. But historic studies have shown that about 40 percent of patients can expect to die or be hospitalized after one year, and about 60-65 percent would normally end up that way on standard treatments, according to CardioKinetix’s executive director of therapy development, Barry Templin.

The findings, being presented today at the EuroPCR meeting in Paris, mean that CardioKinetix is now in position to see if its product can really pass muster in a more rigorous study of 500 heart failure patients in the U.S.—the kind of evidence it will need to win FDA approval. The CardioKinetix device isn’t intended to help all of the 2.5 million people worldwide diagnosed annually with different types of heart failure, but it could end up being a new option for about 20-30 percent of patients with a form of this chronic disease, Templin says.

“Our device isn’t just a quick fix, we’re seeing long term benefit,” Templin says. One of the study’s investigators, Marco Costa of the Interventional Cardiovascular Center at Case Western Reserve University, gushed in a company statement saying, “it is difficult to contain our enthusiasm because the procedure is relatively simple and the outcomes are surprisingly remarkable for such a high-risk population. The sustained and concordant improvements in symptoms, heart function, and clinical outcomes are compelling, with a very acceptable safety profile.”

Before diving in too deep into the details of this study, a little background is required on what CardioKinetix is attempting to do. The private company got its start in 2002, and has raised its cash from a well-known syndicate that includes SV Life Sciences, New Leaf Venture Partners, U.S. Venture Partners, Panorama Capital, and H&Q Healthcare Investors. CardioKinetix has about 30 employees today, Templin says.

The big idea at CardioKinetix is to develop what it calls its Parachute Ventricular Partitioning Device. The way this works, a technician slides a narrow catheter into a patient’s femoral artery in the leg, threads it into the heart with the Parachute device tightly bound to the catheter. Once inside the heart’s left ventricle, the device gets popped open like a parachute, and it has tiny hooks on the edge that enable to to anchor itself in position in the heart muscle. The procedure is supposed to take 40-60 minutes.

Once the implant is in place, physicians hope to essentially wall off part of the heart muscle that gets loose and damaged after a heart attack, which makes it harder for the heart to pump blood in and out efficiently. Walling off part of the heart … Next Page »

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Erin Kutz

Drugs, devices, and nonprofits showed up in this week’s New England life sciences news.

—Promedior, a maker of treatments for tissue damage known as fibrosis, is moving its headquarters from Pennsylvania to Boston and has hired Suzanne Bruhn, a veteran of the Irish drug giant Shire’s Human Genetic Therapies division, as its new CEO.

—Cambridge, MA-based Aveo Pharmaceuticals (NASDAQ: AVEO) and its Japan-based partner Astellas Pharma announced their experimental kidney cancer drug tivozanib reached the main goal in a pivotal of 517 patients. Aveo said tivozanib prevented tumors from spreading for a longer period than sorafenib (Nexavar), a rival drug from Bayer and Onyx Pharmaceuticals, and also showed an edge in safety and ease of use.

—Cambridge-based Ra Pharmaceuticals emerged from stealth mode to reveal it would be focusing on the rara immune system disorder hereditary angioedema as its lead drug program.

—Mitralign, a Tewksbury, MA-based maker of cardiac devices, announced it had closed on $35 million in Series D financing, led by Forbion Capital Partners. The deal also included previous venture investors, such as Oxford Bioscience Partners, Triathlon Medical Ventures, Medtronic Corporation, and Johnson & Johnson Development Corporation.

—My colleague Arlene Weintraub wrote about Xconomist Tom Maniatis’ involvement in a non-profit known as Prize4Life. The Cambridge-based organization offers a $1 million prize to scientists making strides in research for amyotrophic lateral sclerosis, also known as Lou Gehrig’s disease.

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Bruce V. Bigelow

We’re anticipating a lot of news out of a big cancer conference that begins next week in Chicago. Here is your head start.

—Cancer researchers have talked for decades about finding the silver bullet that could kill cancer cells without harming the healthy cells nearby. These days, the industry is focusing on the development of “empowered” or “armed” antibodies, or antibody-drug conjugates (ADCs). Luke put together a list of venture-backed life sciences companies that are focusing on ADCs. His list includes San Diego’s AnaptysBio, Fabrus, and Ambrx.

—San Diego’s Elevation Pharmaceuticals said a mid-stage dosing trial of its lead drug candidate, an aerosol dubbed EP-101, generated positive results among patients with moderate to severe chronic obstructive pulmonary disease (COPD). The company said it tested four doses of EP-101, with all four doses showing EP-101 was safe and effective in helping COPD patients breathe easier. Elevation raised $30 million in a Series B round five months ago that should enable the company to complete a follow-up round of testing this year.

—With the American Society of Clinical Oncology (ASCO) meeting set to begin next week in Chicago, Luke used his BioBeat column to provide a rundown on eight cancer drugs to watch from biotech companies throughout the U.S. He picked them because they are either on the cusp of reaching the market, or just beginning to show their potential.

—BioSurplus, a San Diego company that provides pre-owned lab instruments to the life sciences industry, said it raised $1.5M to fund its expansion into Boston. The company recently opened similar equipment showrooms in the San Francisco Bay Area and Korea. BioSurplus said most of the funding for the move came from San Diego-based KI Investment Holdings.

—Good News for Xconomy: Our national biotech editor, Luke Timmerman, is one of four finalists in the blogging category of this year’s Gerald Loeb Awards for Distinguished Business and Financial Journalism. Whatever happens, he’s put Xconomy in good company. The other blogging finalists work for The New York Times, The Washington Post, and Reuters. Winners will be announced June 26.

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Arlene Weintraub

A few years back, molecular geneticist Tom Maniatis was approached by a Harvard Business School student with a heart-wrenching story. The student, Avichai Kremer, then 29, had been diagnosed with amyotrophic lateral sclerosis (ALS), otherwise known as Lou Gehrig’s disease. Kremer had an unusual idea: He wanted to advance ALS research by offering million-dollar prizes to scientists who made meaningful contributions to research into the disease.

Maniatis was both skeptical and intrigued. He had lost his sister to ALS, and was the longtime chair of the research and drug-development committees for the Amyotrophic Lateral Sclerosis Association. “The prize model had never been tried in life sciences,” says Maniatis, who is now a professor and chair of the department of biochemistry and molecular biophysics at the Columbia University College of Physicians and Surgeons (and one of our Xconomists). “At the time Avi entered the scene, the ALS community was pretty small. And the drug companies did not see it as a profitable pursuit.

As it turns out, Maniatis says, ALS was a perfect model for a prize-based research approach. In 2006, Kremer’s idea became Prize4Life, a Cambridge, MA-based nonprofit that is now one of the most influential forces in ALS research. In addition to offering a $1 million research prize each year—-the latest of which will be announced at a fundraising gala in New York on June 6—Prize4Life has spearheaded several programs designed to mobilize and energize scientists working in ALS. Kremer and a handful of his HBS classmates are running the entire endeavor, says Maniatis, who is a member of Prize4Life’s scientific advisory board. “They had such an affection and respect for Avi that many of them put off jobs to stay in Cambridge and work for this nonprofit,” he says.

Kremer and his classmates spent more than 1,000 hours talking to experts from the drug industry, academia, and nonprofits, before officially launching Prize4Life’s model. “What inspired me was the success of the Ansari X-Prize,” Kremer says in an e-mail, referring to the California organization that provides incentives to researchers in education, energy, the environment, life sciences, and exploration. “It proved to me that a prize can correct a market failure.” In Prize4Life’s case, he explains, that market failure is the lack of capital “to focus innovation coming from academia and small biotechs on ALS,” he says.

This year’s award will mark the third effort by Prize4Life to support ALS scientists with $1 million in research funding. In 2010, 33 teams of scientists—about half of whom came from academia and the other half from pharma companies—competed for the organization’s first $1 million award by proposing treatment protocols meant to extend the lives of mice with ALS by 25 percent. Ultimately, none of the teams met the goals set out by Prize4Life, and the $1 million prize went unclaimed, though several of the applicants are continuing their research with the organization’s support. (Prize4Life also sponsors a number of smaller research grants.)

Last year, Prize4Life awarded $1 million to Seward Rutkove, co-founder of Woburn, MA-based Convergence Medical Devices, for his work developing a biomarker that can measure the progression of ALS in patients. The tool is designed to make clinical trials of potential new drugs more efficient.

This year’s major prize will be a revival of the inaugural award’s goal—the promise of $1 million for research that demonstrates 25 percent survival in ALS mice. “In the previous round, no one was able to achieve that, but there are so many … Next Page »

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Luke Timmerman

From the moment Dendreon started in business 20 years ago, most scientists have said that if its immune-booster for prostate cancer was going to work, it would probably work best at an early stage of disease, before tumors had gotten too powerful for the immune system to contain. Today, Dendreon is offering the latest slice of data that suggests that may be the case.

The Seattle-based biotech company (NASDAQ: DNDN) is announcing today a new analysis of its pivotal 512-patient study of sipuleucel-T (Provenge), which separated patients into four quartiles based on how high or low their prostate-specific antigen (PSA) scores were. The study wasn’t designed to get a statistically significant answer to this question, meaning the finding could be a fluke. Still, the results do align with what many scientists believe based on the history of clinical trials with the product—that it should work better among patients with less-aggressive forms of disease. The analysis, by lead author Gerald Chodak at Weiss Memorial Hospital in Chicago and colleagues, was posted online today on the American Society of Clinical Oncology’s website, in advance of its annual meeting.

“All of these analyses support the robustness of clinical benefit for patients, and this PSA quartile data is helpful for patients when you think about sequencing of therapy,” says Mark Frohlich, Dendreon’s chief medical officer. “The data strongly argues that using it as early as possible is the best for the patient and still allows you to go on and get other therapies.”

Dendreon won FDA approval back in April 2010 for the first-of-its-kind immune-boosting therapy, after the 512-patient study showed patients lived a median time of about four months longer on the drug than on a placebo, with minimal side effects. Since then, the company has faced a series of new competitive threats, particularly from Johnson & Johnson’s abiraterone (Zytiga) and Medivation’s enzalutamide. Those drugs are starting out by aiming to treat the sickest of prostate cancer patients, whose disease has worsened after getting chemotherapy. Dendreon’s drug is approved for patients with a less-severe form of disease, which hasn’t yet prompted them to go all the way to chemotherapy. Since prostate cancer is a slow-growing malignancy, part of Dendreon’s challenge is to persuade doctors to get more aggressive in treating patients early.

PSA, which isn’t a perfectly reliable marker, is still used almost universally by doctors and patients to track a patient’s progress. The lower the score, generally speaking, the better. In this analysis of the Dendreon study, researchers crunched the data to look at survival times of patients who entered the trial with PSA scores of less than or equal to 22.1; between 22.1 and 50.1; between 50.1 and 134; and over 134. As you can see from the chart below, Provenge patients lived longer than placebo patients in all four quartiles, but the difference in median survival times was the largest among those with lower PSA scores.

PSA of 22.1 or less PSA of 22.1-50.1 PSA of 50.1-134 PSA of 134 and up Provenge 41.3 months 27.1 months 20.4 months 18.4 months Placebo 28.3 months 20.1 months 15 months 15.5 months Difference 13 months 7.1 months 5.4 months 2.8 months –Source, Gerald Chodak et al

 

This isn’t really a surprising finding, given that Dendreon has previously said that patients with lower PSA scores had better outcomes on Provenge, about to a summary of the trial published in the New England Journal of Medicine. But it’s the first time Dendreon looked more closely at the PSA scores, by dividing patients into quartiles, Frohlich says.

Given how much debate there has always been about the data to support Dendreon’s prostate cancer drug, I’d love to hear readers thoughts on how meaningful the latest PSA analysis is. Do you think the consistency of the survival advantage in all PSA groups means something, or do you chalk this up to mere data dredging that does little more than stir up a new hypothesis? Let me know your thoughts in the comment section below.

 

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Luke Timmerman

Aveo Oncology is entering a competitive world for the treatment of kidney cancer, and it made a bold bet that its drug would prevail in the first head-to-head comparison of its kind against an active drug. Now the company is preparing to enter that competition with data that says its drug has a slight advantage over a rival on effectiveness, and a more meaningful edge on safety and ease of use.

Cambridge, MA-based Aveo (NASDAQ: AVEO) and its partner, Japan-based Astellas Pharma, are announcing today that their experimental drug tivozanib reached the main goal in a pivotal study of 517 patients with renal cell carcinoma. The new drug showed it was able to keep tumors from spreading a median time of 11.9 months, compared with 9.1 months for those who were randomly assigned to get Bayer and Onyx Pharmaceuticals’ sorafenib (Nexavar). The most common side effects for the new drug were high blood pressure, diarrhea, a skin rash called hand-foot syndrome, and fatigue.

Those effects are common for this class of therapy that cuts off blood flow to tumors, but what’s noteworthy here is that fewer patients on the new drug had to go on unplanned drug “holidays,” or have their doses reduced, because of severe side effects. A detailed summary of the findings is being posted on the American Society of Clinical Oncology’s website today, as a preview of an oral presentation at the ASCO conference on June 2.

Aveo CEO Tuan Ha-Ngoc

Aveo, which is seeking to introduce its first marketed product, has said it plans to seek FDA approval later this year based on the results described online today. Its drug, a once-daily pill, is designed to cut off blood flow to tumors by interfering with three different forms of the VEGF receptor, a marker on cells. If the drug can win FDA clearance, it will be in position to compete not just with Bayer/Onyx’s sorafenib (Nexavar), but also with Pfizer’s sunitinib (Sutent) and axitinib (Inlyta), Roche/Genentech’s bevacizumab (Avastin), and GlaxoSmithKline’s pazopanib (Votrient). There’s certainly a large pool of people who are candidates for the new drugs. About 61,000 new cases of kidney cancer were diagnosed in the U.S. last year, and 13,000 people died from the disease, according to the American Cancer Society.

“There are a lot of options but there are also problems with all the existing options,” says Michael Atkins, the deputy director of the Georgetown Lombardi Comprehensive Cancer Center in Washington D.C., and an investigator on the study. “This drug addresses one of those problems, and is therefore an advance.”

Aveo reported the basic headline that the study was a success back in January, but today’s abstract goes into more detail about what researchers found in the study called TIVO-1. The key new information is in the product’s safety profile.

According to research being published today, about 44 percent of patients on the Aveo drug experienced high blood pressure, and 25 percent of those cases were graded moderate to severe, researchers said. That was a slightly higher rate than for patients who got the Bayer/Onyx drug. But that’s not much of a concern because that effect can be managed with anti-hypertensives, Atkins says.

More importantly, fewer patients on the Aveo drug reported hand-foot syndrome (skin rash) or diarrhea. About 13 percent on the Aveo drug reported hand-foot syndrome, compared with 54 percent who got the Bayer/Onyx drug, researchers said. About 22 percent on the Aveo drug reported diarrhea, compared with 32 percent in the control group.

While that might not sound like much of a difference, it translated into a significantly easier drug to administer to patients, Atkins says. Because the Aveo drug caused fewer … Next Page »

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Sarah Schmid

If the turnout at yesterday’s Michigan Growth Capital Symposium is any indication, our fair state is on the upswing. The conference, in its 31st year, had roughly 450 venture capitalists, entrepreneurs, academics, and government officials in attendance. Jason Mendelson, called “the Elvis of innovation” by this very publication, gave the keynote speech titled, “The Velocity of the Midwest Venture Capital Ecosystem: Sleeping Giants or Momentary Blips?”

Mendelson is the managing director and co-founder of the Foundry Group, a Boulder, CO fund that makes investments in early-stage information technology, Internet, and software startups. He grew up in metro Detroit and graduated from the University of Michigan. Upon finishing law school at U-M, he realized he was frustrated with what he saw as Michigan’s prevailing attitude—fostered by the auto industry—that seniority was all that mattered. Nobody seemed to care what the new guy had to say, even if the new guy had a potentially million-dollar idea. So he decamped to Silicon Valley and went on to serve as managing director and general counsel for Mobius Venture Capital.

Mendelson was in Silicon Valley for the first tech bubble, and he was still there, he says, after “the keg ran dry.” He describes Silicon Valley as an ice-hearted entrepreneurial machine, and not one other cities should necessarily seek to replicate. In his description, Silicon Valley is a place so consumed with competition that virtually the only pleasure in life comes from relishing in the setbacks and failures of others. It’s a toxic place that nobody much likes living in, he claims. Not surprisingly, he fled to Boulder in 2006.

Once he and his partners established the Foundry Group, they decided they would need to “level up” Boulder’s entrepreneurial scene by leveraging the University of Colorado, working to attract capital from outside the region, establishing rigorous entrepreneurial mentorship and development programs through the TechStars accelerator, and cultivating a community where collaboration is key. Now, Mendelson says, Boulder, a city of about 100,000 people, is ranked fourth in the nation in terms of new company formations and companies getting funded.

Before setting up shop in Boulder, Mendelson came to Ann Arbor in 2005 as “a VC with a checkbook.” He came ready to invest in the software/IT space, but he found … nothing. Not one company piqued his interest enough to cut a check. He came back … Next Page »

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Luke Timmerman

Doctors often ask themselves, after a leukemia or lymphoma patient gets high-dose chemotherapy, whether they really wiped out every last rugged cancer cell in the patient. That’s always been a difficult question, but now a team of Seattle researchers is showing they might have found a powerful new technology to consistently deliver that answer.

Scientists at the Fred Hutchinson Cancer Research Center, the University of Washington and Seattle-based Adaptive Biotechnologies are reporting they’ve found what could become a more clear and consistent way to spot “minimal residual disease” that doesn’t show up on imaging scans, but can be deadly. Researchers looked at blood samples from 43 leukemia patients who got high-dose chemo, and found that a new DNA sequencing-based technology was able to spot trace amounts of cancer in 22 patients. For comparison, the gold standard flow cytometry method was only sensitive enough to spot residual cancer cells in 12 patients. The detailed findings are being reported in Science Translational Medicine.

More accurate detection of “minimal residual disease” is important to cancer treatment, because it can influence a physician’s decision on whether or not another round of chemotherapy is really necessary for an individual patient. So if the Seattle scientists can go on to reproduce these findings in other cancers, it could open the door to a potentially lucrative new diagnostic line of business for Adaptive Biotechnologies, a company that has already made some headway by marketing its test to scientists.

“We’re excited, we think this is an important step in the right direction,” says Adaptive co-founder Harlan Robins, a computational biologist at the Fred Hutchinson Cancer Research Center and lead author of the study.

Harlan Robins

Researchers sought to get this answer through a new technology based on advances in DNA sequencing. Adaptive Biotechnologies, a Fred Hutch spinoff, was founded on the idea that while the 6 billion letters of DNA that make up a human genome are consistent in almost every cell of the body, the immune system’s B cells and T cells are an exception. In these cells, DNA gets shuffled around in wildly complex combinations, allowing T cells to recognize specific invaders, such as viruses and bacteria that people get exposed to over time, and enabling B cells to produce specific antibodies against those intruders.

Most of the time, those adaptations are what keep us healthy and able to fend off the wily pathogens in the environment. But sometimes those mutations lead to T cells or B cells that divide out of control, leading to leukemia or lymphoma.

To look for those nasty T and B cells, Adaptive has built a new kind of technology platform based on DNA sequencing, which it calls ImmunoSEQ. The company uses San Diego-based Illumina’s high-speed HiSeq DNA sequencing instruments, and it has custom chemical reagents and software that enable researchers to look specifically at DNA sequences of these variable T and B cells, Robins says. Adaptive found demand for this new technology from scientists looking to answer basic immunology questions. But there’s long been interest in extending this technology into a bigger commercial opportunity with diagnostics.

Adaptive is hopeful that today’s findings will help open that door to the diagnostic world. Flow cytometry, a technique that scientists have used for years, looks for certain biomarkers that can be found on the surface of cancer cells. While it’s not as sensitive at detecting rare cancer cells, that’s not its only weakness, Robins says. The technology isn’t easy to standardize from lab to lab, depends a lot on the skill of the technician running the test, and therefore is subject to human error. Adaptive’s bet is that its technology will be more automated, and easier to standardize, largely because it’s one of only a handful of organizations developing this sort of T and B cell genomic technology (San Francisco-based Sequenta is another).

Still, Adaptive has a ways to go before … Next Page »

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Luke Timmerman

[Updated: 1:30 pm PT, 5/17/12] One of the big dreams in biotech over the past 35 years has been to make drugs that work like “smart bombs” by destroying tumors while minimizing collateral damage. Scientists have learned this is no easy thing, but now that a couple of these types of drugs have been shown to work, a new wave of companies is emerging to see if they can finally turn this vision into reality.

Targeted antibody drugs have been around for a long time, and have been shown to do a lot of good for patients. Some of the world’s best-selling medicines are designed to specifically hone in on cancer cells while mostly sparing healthy tissues. Yet it’s only been in the last several years that a couple of companies—Seattle Genetics and Genentech—have shown proof in clinical trials that they can go a step further than what’s been done with so-called “naked” antibodies. The concept is simple: Take a regular antibody, link it to a toxin, and design the combination so that it unleashes a killer payload on tumors. Done right, you ought to have a drug with more punch than traditional antibodies or chemotherapy.

The idea of making “empowered” or “armed” antibodies is known more formally in industry circles as the antibody-drug conjugate (ADC) business. Most previous attempts to amplify antibodies in the past failed because the toxins broke off and started floating around the bloodstream. That meant the drug never got to the right place, and the treatment caused similar side effects to standard chemotherapy. Seattle Genetics overcame that hurdle with the FDA approval last year of its lymphoma drug brentuximab vedotin (Adcetris). And Genentech is in late-stage trials of its souped-up version of Herceptin called trastuzumab emtansine (T-DM1). Both of these drugs have shown in clinical trials that they can be powerful anti-tumor weapons in very sick cancer patients. And not surprisingly, these successes have inspired a new group of genetic engineers to see what they can do to turn antibody-drug conjugates into mainstream cancer medicines.

“We as an industry now have a lot of experience with naked antibodies for cancer, and some of them are very good, but we know they aren’t perfect,” says Bill Newell, the CEO of South San Francisco-based Sutro Biopharma, a venture-backed startup. “They aren’t magic bullets. But I think as people recognize the valuable contribution antibodies have made to cancer, they naturally ask themselves, ‘how can we make them better?’ Essentially, antibodies are good, but antibodies with a payload may be even better.”

There is so much enthusiasm for the emerging antibody-drug conjugate movement that there’s even a World ADC Summit, now in its third year, scheduled for this October in San Francisco. Given the increasing interest among venture-backed companies that are seeking to come up with new antibody-drug conjugates, or provide new enabling technologies, I thought it would useful to put together a list of companies seeking to play a role. If I’ve overlooked a company you know of, please send me a note at ltimmerman@xconomy.com so I can update the list.

T-DM1. Image courtesy of Genentech

Genentech (South San Francisco). The biotech giant, part of Switzerland-based Roche, has the broadest and deepest experience with antibody-drug conjugates in the world. The company uses technology from Seattle Genetics and ImmunoGen in some cases to make ADCs, but it also has its own proprietary techniques which it doesn’t license outside the company. Genentech has had its most high-profile success with T-DM1, but that’s just one of 25 different antibody-drug conjugates in various stages of development, from discovery through late clinical trials. Nine of Genentech’s 38 cancer drugs in clinical trials—roughly one-fourth of the portfolio—belong to this new class of empowered antibodies. “We’ve really invested heavily in this technology and have the breadth and depth of our pipeline to show for it,” says Genentech spokeswoman Krysta Pellegrino. For a detailed rundown of Genentech’s ADC programs, click here.

Seattle Genetics (Bothell, WA). Seattle Genetics (NASDAQ: SGEN) is one of the two mainstays of the armed antibody field, along with Waltham, MA-based ImmunoGen (NASDAQ: IMGN). The company was founded in 1998 after Bristol-Myers Squibb closed down a Seattle research center that had been dedicated to developing antibody-drug conjugate technology. Besides its work on the new lymphoma drug Adcetris, the company lists six other empowered antibodies in clinical and preclinical development on its website. The company also licenses out its antibody-drug linking technology to other drug developers working on specific projects. The list of collaborators includes Genentech, Bayer, Celldex Therapeutics, Progenics Pharmaceuticals, Astellas Pharma, Daiichi Sankyo, Millennium:Takeda, GlaxoSmithKline, Genmab, Pfizer, and Abbott Laboratories.

ImmunoGen (Waltham, MA). ImmunoGen (NASDAQ: [[ticker:IMGN]) is the other stalwart of the empowered antibody world, having been founded way back in 1981. The company has never made a profit in all those years, and has had some very lean years, but it has been resurgent of late. That’s because it licensed its antibody-drug linking technology more than a decade ago to Genentech, which has used it to make T-DM1. That drug isn’t yet FDA approved, but it passed a pivotal clinical trial back in March, which was the last major milestone it needed to clear before seeking the regulatory green-light. ImmunoGen stands to collect a modest royalty on that product, and like Seattle Genetics, it seeks to use its antibody-drug conjugate technology for its own internal drug candidates, while also making some money by licensing it to other companies working on specific projects. Eli Lilly, Novartis, Amgen, Genentech, Biotest, Bayer, and Sanofi are among its collaborators.

Bristol-Myers Squibb (New York). The pharmaceutical company (NYSE: BMY), which once invested so heavily … Next Page »

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Arlene Weintraub

Biotech firm Promedior announced today that it will be moving its headquarters from Malvern, PA, to Boston, and that it has hired a new CEO, Suzanne Bruhn,who was previously senior vice president for planning and program management for Shire’s Human Genetic Therapies division.

Promedior is working on several drugs to treat fibrosis—a type of tissue scarring that contributes to a range of diseases affecting major organs such as heart, lung, and kidney, as well as the eyes. In March, the company raised $21.5 million in a Series D funding round from its previous investors, Morgenthaler Ventures, HealthCare Ventures, Polaris Venture Partners, Forbion Capital Partners, and Easton Capital Investment Group. New investor Fibrotec Ventures also participated in the round, which brought the total amount raised by the company to $62 million.

Promedior’s drug-development platform is centered around an engineered version of a naturally occurring protein called pentraxin-2. The company’s therapeutics are designed to exploit the mechanisms by which the protein modulates fibrosis. “Pentraxin-2 directs cells to a pathway that promotes healing instead of fibrosis,” says Bruhn (pictured above). “We think this could have a big impact in many different indications.”

The company is testing its lead drug, PRM-151, in the lung disease idiopathic pulmonary fibrosis (IPF) and in myelofibrosis, which is a disorder of the bone marrow. Next in line is PRM-167, which it is plans to develop for eye diseases such as age-related macular degeneration. Bruhn says Promedior’s evolution from a research-based company to a full-fledged drug developer precipitated the move to Boston. “The focus of the company is moving into later-stage clinical development,” she says. “The Boston/Cambridge area is a great source of talent.” Promedior is planning new hires in research and drug development, she says.

Bruhn spent most of her career at Transkaryotic Therapies, which was acquired by Shire (NASDAQ: SHPGY) in 2005. She had a hand in developing four major products—agalsidase alfa (Replagal) for Fabre disease, idursulfase (Elaprase) for Hunter syndrome, velaglucerase alfa (Vpriv) for Gaucher disease, and icatibant (Firazyr) for hereditary angioedema. The products now bring in combined annual sales of more than $1 billion. In today’s announcement, Promedior’s board chairman, Morgenthaler partner Jim Broderick, calls Bruhn a “proven biotech leader with strong experience in commercializing novel therapeutics.”

Promedior expects to complete a mid-stage trial of PRM-151 in IPF this year. The company plans to initiate a trial early next year in myelofibrosis. Bruhn says she hopes to move PRM-167 into early-stage clinical trials next year for retinal diseases.

As for developing the pipeline, Bruhn says Promedior intends to maintain its focus on fibrosis. “It’s such a huge opportunity,” she says. “The challenge is to make sure we’re lean and focused and that we’re choosing indications that make sense, both from a pharmaceutical perspective and a marketing perspective. That’s the strategy we’re working on going forward.”

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Luke Timmerman

Theraclone Sciences has taken its first step ahead toward showing it might have an antibody drug that could work in humans.

The Seattle-based biotech company is announcing today that it passed its first clinical trial with an antibody drug designed to fight a wide variety of flu strains. The study randomly assigned 40 healthy volunteers to get a dose of Theraclone’s TCN-032 or a placebo. Researchers found that the treatment was safe and well-tolerated at all five doses studied, didn’t appear to cause any serious side effects, and didn’t provoke an immune reaction against the drug.

“This study exceeded our expectations,” says Leni Ramos, Theraclone’s chief medical officer.

Leni Ramos, Theraclone Sciences' chief medical officer

Theraclone plans to release more detailed results at a future scientific meeting, which will include strategically important information on how long the drug lasts in the bloodstream. And today’s study results don’t say anything about whether the drug is effective at killing flu viruses in human beings. But the results are promising enough that the company plans to take the next step in clinical trials, advancing to a mid-stage clinical trial later this year in which the drug will be tested among people who get exposed to flu virus in a carefully controlled environment. That study will be placebo-controlled, and is being designed as the first to establish biological proof in humans that TCN-032 can effectively kill the virus, Ramos says.

While there are already a couple of antiviral pills on the market—Roche’s oseltamivir phosphate (Tamiflu) or GlaxoSmithKline’s zanamivir (Relenza)—scientists worry about mutant strains like H5N1 “bird flu” that could emerge and resist today’s antivirals. Even without some mutant supervirus in the air, an estimated 200,000 people are hospitalized in the U.S. each year with flu, and 36,000 people in the U.S. still die from annually. Most are elderly, young children, or those with weakened immune systems.

Theraclone, founded in 2005, has spent years working to get to this point by developing its technology for discovering “broad-neutralizing” antibodies. Theraclone has shown in the lab that it can find antibodies that can fight a wide variety of infectious invaders, from HIV to flu to cytomegalovirus (CMV). Japan-based Zenyaku Kogyo helped finance the anti-flu antibody work, and it retains commercial rights to the Theraclone product candidate in Japan. Zenyaku remains committed to support further development, Theraclone CEO Cliff Stocks says.

Exactly how such a flu antibody might be used, in practical terms, is still an open question. Since Theraclone’s antibody is given intravenously, it’s not the kind of flu-fighting product people will ever get at their neighborhood Safeway. But it could be valuable to hospitals looking to have a potent anti-flu weapon on reserve for tough cases, and for public health officials, who may want to stockpile it in case a particularly nasty pandemic flu strain emerges.

Theraclone isn’t the only company out there with visions of creating a potent antibody drug against flu. Netherlands-based Crucell, acquired last year for $2.3 billion by Johnson & Johnson, has said it is working on a “universal antibody” for flu. It has said that its antibody candidate is designed to hit the hemagglutinin protein that appears on various flu viruses.

Theraclone’s program is different in a few key respects, Stocks says. For starters, Theraclone believes it is ahead in development, since the Crucell program is thought to still be in preclinical development. Second, the Theraclone antibody is made to hit a different target on flu viruses, known as matrix 2 protein (M2e). That distinction is important because an estimated 98 percent of flu strains contain the M2e region in their DNA. Viruses can easily mutate when confronted with a powerful new drug that threatens their survival, but Ramos says if flu viruses mutate away from the assault of Theraclone’s TCN-032, then they would lose their ability to be infectious.

The next study will say a lot about whether Theraclone has a real drug it will be able to sell, and it won’t have to wait eons for the answer. The next study will be based on volunteers getting a single dose, and since the antibody is designed to last several weeks in the bloodstream, it won’t need to be given in repeat doses over time to prove it’s effective against the virus, Ramos says.

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Erin Kutz

Startup accelerators like Y Combinator and TechStars are known for producing the latest and greatest in Web startups. But there’s a set of established and accomplished companies—like ZipCar (NASDAQ: ZIP), Xenogen, and Constant Contact (NASDAQ: CTCT)—that also received startup coaching, mentoring, and connections to investors back in their day.

They went through a different program, run by Washington, DC-based Springboard Enterprises. Unlike the aforementioned accelerators (and the dozens upon dozens of imitators they’ve spawned), Springboard doesn’t directly offer funding or take equity. Instead, through its six-month-long venture forums, it focuses on connecting women entrepreneurs with mentors for intense coaching on their presentation and business strategy, and for connections to investors.

“It’s trying to build a network of individuals in entrepreneurial and venture communities who are supportive of these emerging growth ventures,” said Springboard director of programming Joshua Henderson in an interview about the organization.

Springboard, founded in 2000, is in the midst of its 23rd venture forum, which is focused on life sciences companies. It took its current crop of entrepreneurs to New York for a day of interviews with mentors last month, and brought the group to Boston last week for a full-day boot camp.

This past Thursday’s event included panels on topics like term sheets, getting press, and crafting the perfect pitch to investors. But much of the day was structured around active feedback sessions for the participating startups.

It featured an impressive roster of entrepreneurs, including doctors, lawyers, PhDs, and several who had built and exited multiple companies. But being a serial entrepreneur or accomplished professional doesn’t automatically make you a strong public speaker, so Springboard dedicated the first part of its boot camp to a session where the women presented for a few minutes on themselves and their accomplishments, while the audience of investors and mentors offered feedback. Below are some takeaways I caught from that part of the day.

—“I think any of these degrees shows stick-to-it-iveness. I value that tremendously,” said one audience participant after an entrepreneur started her presentation describing her failed attempt as an attorney (she passed the bar but decided she didn’t like the field). This sparked an interesting discussion on many of the candidate’s tendencies to downplay their achievements and degrees.

—This also brought up the suggestion by one audience member to “never disparage any profession.” Smart advice, given you never know the full backgrounds of the investors you’re pitching. Presenters were encouraged to say why they jumped into starting their company without negatively describing their previous job experiences.

—“You’re not learning the ropes, you’re running a company.” This came in response to one entrepreneur who described getting her footing at her new CEO gig. Audience members said she was clearly well qualified and understood the space, but spending time talking about how she was still learning “negated the positive.”

—Audience members advised entrepreneurs to go beyond rehashing their resume to telling their unique story—like one entrepreneur whose grandfather’s heart attack inspired her to start her medical monitoring company.

—The audience feedback showed that subtle changes in wording and phrasing can impact the overall feel of a presentation. One entrepreneur was cautioned for overusing the word “passion” and advised instead to be specific about what made her qualified to run that company. Another was told that the phrase “I believe” is stronger than “I think” when describing her company’s potential impact.

In its 10-year history, Springboard has worked with 481 companies, which have collectively raised … Next Page »

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Arlene Weintraub

In March, global consulting firm Ernst & Young released an annual report for its life sciences clients called “The Third Place: Healthcare Everywhere.” It discusses a range of topics, from apps designed to improve patient adherence to medical treatments, to games that encourage healthy habits.

Today Ernst & Young is holding a summit in Princeton, NJ, which will bring together players across the healthcare system to discuss opportunities for using technology to get closer to patients—and to change their behavior for the better. In advance of the event, Xconomy spoke with David DeMarco, Ernst & Young’s northeast pharmaceutical and medical device leader, on how the changing technology landscape is affecting the life sciences industry.

Xconomy: Ernst & Young’s report refers to “the third place” in healthcare. Explain.
Dave DeMarco: The idea came from Starbucks, which talked about wanting to be the third place where you enjoyed a cup of coffee—after your home or your work. And they didn’t just want you to enjoy a cup of coffee, they wanted you to enjoy a social experience. Traditionally healthcare is provided in two places—the hospital and the doctor’s office. But in the future world, the third place is wherever the patient is, i.e. everywhere. In the third place, you have the potential for continuous monitoring. You have the potential for real-time data that helps drive behavior. The system is more transparent, and patients have a lot more control.

X: Have you seen any good examples of healthcare providers going into the third place and influencing patient behavior effectively?
DD: There was a study done at the University of Pennsylvania and Carnegie Mellon University involving [the blood thinner] warfarin, which is given to patients who are at risk of suffering pulmonary embolisms or other serious events. You would think the compliance to that drug would be pretty darn high, but it’s only 67 percent. So in this study, they gave patients electronic pill boxes that were hooked up through the Internet to their providers. They knew if you took your pill or not. Then, through either a text or e-mail, they gave each patient a two-digit number every day and held a lottery. If a patient matched both digits, which only happened with a 1 percent probability, he or she won $100. What I loved was the catch: You could only win the money if you took your pill.

The last thing they did, which was clever, was if you had a day where you weren’t compliant, they sent you a text or an e-mail saying, “You know, if you had taken your pill today you could have won $100.” Compliance went from 67 percent to 97 percent.

Is this going to save the industry? No. But we’ve been working on disease management for a long time, with mixed results. We really believe that behavioral change is the next frontier. Understanding the biases around behavioral economics is key—for pharma and biotech companies, too.

X: You also write about making medical compliance fun for consumers. How can life sciences companies—and even consumer companies—do that effectively?
DD: One of the things that we believe is going to be part of this healthcare-everywhere future world is the medicalization of consumer devices and the consumerization of medical devices.

An interesting example of the medicalization of a consumer device is Nike’s development of embedded sensors in their products to give real-time feedback on exercise. It’s called Nike+. This is just an early example of sensor technology blending with devices to be able to give some credence to the fact that yes, healthcare in the future can be delivered in other settings than the doctor’s office and the hospital.

An example of the consumerization of a medical device is a pilot that Bayer Healthcare and Nintendo did where they partnered to create a game for children with diabetes that centered around proper glucose control.

Life science companies have to figure out how to collaborate with organizations that they’re really not used to dealing with, so they can bring a net outcome advantage to the market.

X: How is the use of social media evolving, and how important will it be for life science companies to understand it and participate in it?
DD: I think that represents a real challenge. Most of our clients know that it’s going to be important. Yet there are regulatory issues that are unclear. So they’re all taking a cautious approach to do what they can. One of our large pharma clients has people online every day. They’re not pushing their products, but rather trying to advance awareness and education of disease states.

X: And what about apps? How can health-related companies best use them?
DD: The whole idea of apps is something that really harkens back to the theme of the consumer being the center. In a patient-centric world, where healthcare is everywhere, apps should enable and empower consumers.

Some of our clients believe that creating clinical decision tools for healthcare professionals is a very important strategy to wrap value around their molecules. Their thinking is now evolving, and they’re beginning to wonder about the power of consumer-based clinical decision tools. They’re recognizing that the consumer is really going to shape things in the future. I think the more life sciences companies can get information into the hands of consumers, the more they’re going to be able to extract value.

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Sarah Schmid

JumpStart Inc., a Cleveland-based nonprofit focused on entrepreneurship and economic development, will team with the New Economy Initiative (NEI) to open a new high-tech accelerator in Detroit, says NEI executive director David Egner. Egner (an Xconomist) says a variety of state and regional investors will fund the accelerator and he expects it to have an annual operating budget of between $1.5 million and $3 million.

Egner says the accelerator, which will likely open this fall inside TechTown, will join a field of local accelerators and incubators that some feel is already too crowded given the relatively small geographic area they cover in Southeast Michigan. But Egner says that although accelerators like TechTown and Ann Arbor SPARK nurture high-tech startups, they aren’t solely devoted to them. “We don’t have a designated organization doing marketing to increase deal flow,” he says. “There currently isn’t capacity to go out and find new deals. We’ll have a $5 million fund with the ability to move $250,000 per deal.”

What Detroit has that still hasn’t been fully capitalized upon, Egner argues, is an innovation corridor that stretches from Dan Gilbert’s buildings downtown to Henry Ford Hospital in New Center. “That three and a half miles could be one of the most innovative districts in the country, and we’ll help to accelerate that corridor,” he says, adding that innovation is already flourishing in places like Wayne State, the Detroit Medical Center, Next Energy, Bizdom, D:Hive, Sustainable Water Works, the Detroit Creative Corridor Center, Inforum, and all the startups housed in the Madison Building. The NEI is itself a $100 million initiative formed in 2008 to increase economic activity in … Next Page »

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Luke Timmerman

The pistons of the biggest publicity engine in cancer R&D will start firing this week. It’s time to behold the annual rite of the American Society of Clinical Oncology (ASCO) meeting, the biggest event for showing off what’s new and interesting in the treatment of cancer.

This conference, officially held June 1-5 at McCormick Place in Chicago, draws more than 25,000 physicians, pharmaceutical companies, investors, and journalists every year. Organizers have spent years carefully orchestrating this show to make sure it’s the place for all kinds of market-moving, medical practice-changing, and front-page leading news about cancer. Partly to help drum up suspense, things really get started this week, as thousands of (often outdated and incomplete) abstracts of clinical trial results are posted on the ASCO website, as a preview of coming attractions in Chicago. The abstracts are due out at 6 pm ET on Wednesday.

Despite the excessive hype in this business, where a few extra months of survival counts as a breakthrough, there are really encouraging things happening in cancer treatment. There’s undoubtedly tons of money to be made, which explains a lot about the spectacle that is ASCO. Health insurers now spend an estimated $80 billion a year on cancer care worldwide, and spending in the U.S. is expected to climb an eye-popping 42 percent by the end of 2013, according to a report last year by Medco Health Solutions. There are now about 900 cancer drugs in development.

Most of those drugs will suffer quiet deaths, and never get close to the marketplace, because they aren’t safe enough or don’t work well enough. But here’s a rundown on eight drugs from biotech companies around the U.S. that are sure to make news at this year’s ASCO because they are either on the cusp of reaching the market, or just beginning to scratch the surface of their potential:

—Genentech’s trastuzumab-DM1 (T-DM1) for breast cancer. Evidence has been mounting for years that this “souped-up” antibody drug could be much more potent than Genentech’s original trastuzumab (Herceptin), the pioneering breast cancer medicine that’s almost 15 years old. Now the South San Francisco-based company, a unit of Roche, is getting ready to present data from a pivotal study of 991 women with breast cancer. These women had previously gotten Herceptin, and were randomly assigned to get either T-DM1 or a combo of GlaxoSmithKline’s lapatinib (Tykerb) and capecitabine (Xeloda) chemotherapy.

Genentech said in March that T-DM1 met its main goal of slowing the spread of tumors, and that it was good enough for the company to seek FDA approval of the drug later this year. This study, known as Emilia, also measured survival times. Data on the tumor progression times, and an interim look at overall survival time, will be made public the first full day of ASCO presentations on Saturday, June 2, according to Genentech spokeswoman Emmy Wang. Besides Genentech, this disclosure also means quite a bit to Waltham, MA-based ImmunoGen (NASDAQ: IMGN), which developed antibody-drug linking technology that it licensed to Genentech, in exchange for a “mid-single digit” percentage royalty on worldwide sales of T-DM1.

Partly because this drug is working against a high-profile disease like breast cancer, and partly because of its groundbreaking science as a sort of anti-cancer smart bomb, I’d bet that this drug will be the star of the show at this year’s ASCO. The presentation also happens to be timed for the deadlines of the nation’s major Sunday newspapers. Watch for the headlines on June 3.

Aveo Pharmaceuticals CEO Tuan Ha-Ngoc

—Aveo Pharmaceuticals’ tivozanib for renal cell carcinoma (kidney cancer). The Cambridge, MA-based biotech company (NASDAQ: AVEO) has its biggest-ever presentation coming up this year at ASCO. The company released basic results in January, from a study of 517 patients with renal cell carcinoma, which showed its tivozanib compound was able to keep tumors from spreading for a median of 11.9 months, compared with 9.1 months for sorafenib (Nexavar), an FDA-approved drug sold by Bayer and Onyx Pharmaceuticals. The study was designed to show the Aveo drug could keep tumors from spreading for about an extra three months, so it barely passed. Aveo’s stock fell after the results, but the company has said it is hiring aggressively and getting ready to seek FDA approval. Physicians, investors, and competitors will get their first detailed glimpse at ASCO into what has made Aveo so bullish about tivozanib’s prospects. New details “could provide additional evidence to support a differentiated product profile based on safety,” said Jason Kantor, an analyst with RBC Capital Markets, in a note to clients May 3.

—Onyx Pharmaceuticals/Bayer’s regorafenib for colorectal cancer and GIST. South San Francisco-based Onyx (NASDAQ: ONXX) has been on a roll of late, and it will give physicians and investors a lot of information to chew over at ASCO. Onyx is planning to present data from a pivotal study of regorafenib, a follow-on cancer compound that builds on its success with sorafenib (Nexavar). The company said in January that this new compound offered a slim advantage of about six weeks extra survival time for some very sick patients with colorectal cancer. More data on that trial is expected at ASCO, and so is some new information about regorafenib as a treatment for gastrointestinal stromal tumors (GIST).

The company also will have more to say about … Next Page »

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Erin Kutz

News of financings, clinical developments, hires, and IPO moves came out of New England life sciences companies this week.

—Cambridge, MA-based Vertex Pharmaceuticals revealed promising data showing it could potentially reach more cystic fibrosis patients by combining its treatment ivacaftor (Kalydeco) with an experimental drug called VX-809. The company plans to advance the combination regimen into a third and final stage of clinical trials.

—JAFCO, Third Rock Ventures, and Flagship Ventures pumped $20 million in Series A funding into Eleven Biotherapeutics. The Cambridge-based startup will use the money to fund the first human trial of its lead drug EBI-005, a treatment for dry eye syndrome.

—RXi Pharmaceuticals, a Worcester, MA-based biotech working in the area of RNA interference, said Thursday that it had named Geert Cauwenbergh as its new president and CEO. The company also announced its stock had begun trading on the OTC Bulletin Board under the symbol RXII.

—BioSurplus, a San Diego-based company that has a platform for buying and selling pre-owned laboratory equipment, announced it had raised $1.5 million from KI Investment Holdings to fund an expansion to the Boston area.

—New Haven, CT-based antibiotics developer Rib-X Pharmaceuticals has postponed its initial public offering, a day after slicing its projected share price by 50 percent to $6 to $7 per share, Renaissance Capital reports. The company filed paperwork late last month indicating it had hoped to go public at $14 per share.

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Bruce V. Bigelow

Verdezyne, the Carlsbad, CA-based company developing industrial biotechnology processes for making adipic acid and other sustainable chemicals and fuels, has raised more than $10.6 million, according to a recent regulatory filing.

A spokesman for the company confirmed the financing, but would not comment beyond the filing, which shows the financing consisted of debt and rights to acquire securities.

Last year at this time, Verdezyne raised an undisclosed amount of funding from BP Alternative Energy Ventures, DSM Venturing B.V., OVP Venture Partners, and Monitor Ventures. Previous investors include Southern California’s Tech Coast Angels, the Life Science Angels, and other individual investors.

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Bruce V. Bigelow

Massachusetts’ Vertex and San Diego’s Celladon are advancing some promising gene therapy treatments. Here’s our roundup of life sciences news over the past week.

—San Diego’s Celladon, which is developing a gene therapy to treat patients with a certain type of heart failure, extended a recent investment round by $10 million. Two new investors, MPM Capital and LSP Life Sciences Partners, increased Celladon’s current round to $53 million. Celladon’s gene therapy is intended to restore a key enzyme that helps a healthy heart maintain a normal heartbeat.

—Cambridge, MA-based Vertex (NASDAQ: VRTX), which has operations in San Diego, reported that a combination of its ivacaftor (Kalydeco) therapy and an experimental drug called VX-809 significantly improved lung function for adult patients with the most common genetic mutation (F508del) in cystic fibrosis. The FDA approved ivacaftor in January for treating CF patients with a different mutation (G551D).

—San Diego’s MediciNova (NASDAQ: MDNV) says it is nearing completion of a mid-stage trial of its lead drug candidate, bedoradrine sulfate. MediciNova licensed the compound from Japan’s Kissei Pharmaceutical, and has been advancing the drug as an intravenous treatment for treating acute asthma attacks. MediciNova expects to report results of its latest trial in the next six weeks or so.

—Luke voiced support in his BioBeat column for legislation known as the Generating Antibiotic Incentives Now (GAIN) Act. The bill would provide antibiotic developers an additional five years of market exclusivity for their products.

—Just in time for the Hologic acquisition, San Diego’s Gen-Probe (NASDAQ: GPRO) said the FDA cleared its new molecular diagnostic platform, the Panther system. Gen-Probe says its technology can initially be used to test for the common sexually transmitted infections Chlamydia trachomatis and Neisseria gonorrhoeae. Hologic disclosed its plans to acquire Gen-Probe in a $3.7 billion deal earlier this month.

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Luke Timmerman

A lot of people think tuberculosis is a thing of the past, but it remains one of the most deadly diseases in the world. Now the Seattle-based Infectious Disease Research Institute is getting ready to take it on with a new vaccine being prepped for clinical trials.

IDRI said today it has formed a partnership with another nonprofit, Rockville, MD-based Aeras, to test a new vaccine candidate for tuberculosis. IDRI and Aeras said they expect to start an initial study to assess the vaccine’s safety in healthy volunteers later this year.

There already is an available TB vaccine that was developed 90 years ago, but it didn’t stop an estimated 1.4 million people from dying of the infectious disease in 2010. As many people infected with HIV in the developing world end up getting TB, and as multi-drug resistant forms of bacterial infection have continued to evolve, it has created new urgency for scientists to come up with an effective TB vaccine. Both IDRI and Aeras are funded by the Bill & Melinda Gates Foundation, and have a lot of experience in the fight against global infectious disease. Aeras has invented or is supporting the development of six TB vaccine candidates, five of which are in clinical trials today, while IDRI has invented the key intellectual property in this newest candidate.

“Given Aeras’ focus and breadth of experience in supporting the clinical development of tuberculosis vaccines, we believe this collaboration will speed the development of this promising new vaccine,” said Steve Reed, IDRI’s founder and chief scientific officer, in a statement.

The new vaccine candidate, dubbed ID93/GLA-SE, uses a genetically engineered fusion protein designed by IDRI to alert the immune system to a marker on TB bacteria, combined with an immune-boosting compound known as an adjuvant. This adjuvant, GLA, is a synthetic compound designed to be more potent than past adjuvants made from natural products. Scientists are hopeful it can be manufactured consistently and cheaply at the kind of large scales that would be necessary for an effective TB vaccination campaign. The adjuvant has already been used in a wide array of experiments, including some for Seattle-based Immune Design, which is seeking to develop commercial immune-boosting therapies for Herpes Simplex Virus 2 (genital herpes) and cancer.

IDRI said in today’s statement that the vaccine candidate has shown “an acceptable safety profile” in animal tests and substantial protection from the TB bug. The vaccine appeared effective in animals that had previously gotten the existing vaccine, BCG, to prime their immune systems, and among animals that hadn’t gotten the old vaccine.

Today’s statement didn’t name a commercial partner, or discuss the financial resources needed to push the vaccine through this phase of clinical trials. But IDRI has conducted all of the preclinical development of the vaccine so far, with about $35 million of support from the National Instiutes of Health, according to Erik Iverson, IDRI’s executive vice president of business development. Most of the funding for the clinical trials is coming from the Gates Foundation and the Paul G. Allen Family Foundation, Iverson said.

Aeras and IDRI said in today’s statement they are committed to make sure that new vaccines they develop “will be accessible and affordable to those who need them most in developing countries.”

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