News of FDA drug approvals shone the spotlight on some New England biotechs this week.

—Waltham, MA-based Alkermes (NASDAQ: ALKS) and San Diego-based Amylin Pharmaceuticals(NASDAQ: AMLN) won FDA approval for their injectable diabetes drug exenatide once-weekly (Bydureon). This was their third time seeking clearance for the drug, which uses technology from Alkermes to last long enough in the bloodstream to turn it into a once-weekly injection

—Additionally, the FDA cleared a drug developed by Lexington, MA-based Curis and its partner Genentech as treatment for a common form of skin cancer called basal cell carcinoma. The drug, vismodegib (Erivedge), is the first approved product for Curis.

—They say good news comes in threes. Cambridge-based Vertex Pharmaceuticals also got a faster-than-expected FDA OK to start selling its drug ivacaftor (Kalydeco) as a treatment for a rare form of cystic fibrosis. That’s the second drug approval for Vertex (NASDAQ: VRTX), which also sells a treatment for hepatitis C. Bonnie Ramsey, one of the key people involved in developing the drug, talked to my colleague Luke about the implications of the approval.

—But there was some bad news, too: A week after reporting data that its pancreatic cancer treatment saridegib (IPI-926) showed some success in a small study, Infinity Pharmaceuticals (NASDAQ: INFI) halted a bigger, mid-stage clinical trial of the drug when it showed patients were living longer in the placebo group. The Cambridge, MA-based company’s shares fell more than 30 percent after the news last Friday.

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Bad news out today from Infinity Pharmaceuticals. The Cambridge, MA-based biotech company said it is halting a mid-stage clinical trial of its drug for pancreatic cancer early after learning that patients were living longer in the placebo comparison group. Infinity (NASDAQ: INFI) shares fell more than 30 percent after the news.

The trial of 122 patients showed that when patients got Infinity’s saridegib (IPI-926) in addition to gemcitabine chemotherapy, they were living less than the six months they were expected to based on historical studies with the chemo drug alone. No unexpected side effects were seen among patients on the Infinity drug or in the control group, the company said.

This is a painful setback for Infinity. The company just last week released some more encouraging data from an early-phase study of the drug in 16 patients, which suggested it offered a benefit by shrinking tumors and helping them live a median time of about 10 months. The plan for this year was to wait for the results from the more rigorous study of 122 patients, to get a firm answer on whether it could help pancreatic cancer patients live more than the expected six months. Even though the drug failed in that study, Infinity said it still believes in the drug’s potential because it inhibits a pathway known as hedgehog that plays a role in multiple cancers. Infinity currently is testing the new compound in mid-stage trials against myelofibrosis and chondrosarcoma. Those studies are continuing, the company said.

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Steve Holtzman got his first taste of corporate venture capital back in 1987, when he raised money from SR One, back when it was part of an old company known as Smith, Kline & French.

The concept was unorthodox 25 years ago, yet over time, most every Big Pharma company has become an active equity investor in biotech startups. But Holtzman, early in his tenure as lead dealmaker at Weston, MA-based Biogen Idec (NASDAQ: BIIB), chose to buck the trend, helping put the kibosh on the company’s VC investment group in the past year. This was no small decision, given that Biogen started its venture investing group in 2004, committed $200 million to it, and made investments in companies like San Diego-based Intellikine, South San Francisco-based iPierian, San Diego-based Calcimedica, and Cambridge, MA-based Aveo Pharmaceuticals (NASDAQ: AVEO), among others.

Holtzman outlined four main reasons why Biogen has gotten out of the VC game during an interview earlier this month at the JP Morgan Healthcare Conference. But before diving into those reasons, they should be placed in the context of what’s been a busy first year of dealmaking he has overseen as part of CEO George Scangos‘s new management team at Biogen.

Besides shutting down the venture investment effort, Biogen also closed down its startup incubator, so that more resources could go to internal R&D projects, Holtzman says. Given that Biogen has plenty of drug candidates (six) in the third and final phase of clinical trials usually required for FDA approval, Holtzman’s mandate has been to in-license drugs that can fill gaps in the early-stage part of the pipeline. Two recent partnerships, with South San Francisco-based Portola Pharmaceuticals and Carlsbad, CA-based Isis Pharmaceuticals (NASDAQ: ISIS), reflect that desire. Expect more in-licensing of early-stage drug candidates for autoimmune and neurological disorders in the year to come, Holtzman says.

Given that backdrop, here are the four arguments that Holtzman says are commonly made in favor of corporate VC activity, which he says don’t make sense for Biogen:

1. “Corporate VC investing provides a window on novel technologies.” That might sound reasonable on the surface, but Holtzman says it’s not necessary, and not the best way to stay plugged in. “I’d submit to you that the best window on novel technologies comes from your scientists, who are identifying new things all the time,” Holtzman says.

2. “It’s a good way to network with VCs who have an inside look at what’s hot. If you don’t invest with them, you aren’t connected to them.” This argument doesn’t apply to Biogen, because its senior executives—particularly Scangos, R&D head Doug Williams, and Holtzman—have all been in the biotech business for 25 years and have extensive Rolodexes in the venture capital world.

“We are intimately familiar with all the VC players on a first-name, friendly basis,” Holtzman says. “We have multiple meetings here [at the JP Morgan conference] with the leading VCs in the industry, where they sit down and go through every company in their portfolio that might be of interest to us. We are very happy to do that. We get calls regularly from VCs who say, ‘We’re thinking of starting a company in the following area. Are you interested in that area?’ And beyond that, they ask us, ‘What do you think are the interesting areas where we ought to start companies?’

Personal relationships with the VCs can be forged … Next Page »

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It was a busy news week in the New England life sciences scene, with acquisitions, clinical data, and partnership deals.

—Thanks to a new co-promotion deal, Genzyme will begin marketing a diagnostic test for thyroid cancer developed by South San Francisco-based Veracyte. Genzyme, the Cambridge, MA-based unit of Sanofi (NYSE: SNY) makes a common drug for treating the disease.

—Cambridge-based Infinity Pharmaceuticals (NASDAQ: INFI) presented data from a 16-patient study indicating saridegib, its experimental once-daily pill, could potentially shrink tumors and help people live longer when combined with chemotherapy.

—Millennium, also of Cambridge, nabbed FDA clearance to begin selling a version of the multiple myeloma drug bortezomib (Velcade) that can be injected just under the skin, as well as intravenously. It’s significant because the under-the-skin drug seems to be more tolerable, reducing the drug’s side effect of nerve damage in the fingers and toes.

—Xconomy national biotech editor Luke Timmerman wrote about how Cambridge-based Vertex Pharmaceuticals (NASDAQ: VRTX) and its South San Francisco-based partner Alios Biopharma are positioning themselves to defend their share of the hot growing market for hepatitis C treatments.

—And my colleague Arlene wrote about the family-run business that is Woburn, MA-based Courtagen Life Sciences. Three brothers fill the roles of CEO, president, and chief scientific officer, while their dad chairs the board. Read about more about how it all came together.

—Bedford, MA-based Avila Therapeutics, a maker of so-called covalent drugs for treating cancer, was bought by New Jersey-based Celgene (NASDAQ: CELG) for $350 million upfront. Potentially $575 million more could come to Avila through milestones. The startup’s venture investors included Polaris Venture Partners, Atlas Venture, Abingworth Management, and Advent Venture Partners. Check out investors’ and friends’ reactions to the deal across the Web here.

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Infinity Pharmaceuticals released some preliminary data last June that suggested it may be onto something for pancreatic cancer, and now it has gathered a bit more evidence to support its case.

The Cambridge, MA-based biotech company (NASDAQ: INFI) is announcing results today from a small study of 16 patients that suggest its experimental once-a-day pill, saridegib (IPI-926), may be able to shrink tumors and help people live longer when given in combination with chemotherapy.

The data is still from a small sample, so it will need to be confirmed in more patients before it be considered convincing. As reported back in June, five of the 16 patients (31 percent) had significant tumor shrinkage. Now with more follow-up time, researchers are reporting that the tumors were kept from spreading for a median of 7.6 months, and the patients lived a median time of 10.2 months, according to data being presented at the American Society of Clinical Oncology’s Gastrointestinal Cancers Symposium in San Francisco. There was no way to compare how good this performance was since there was no control group in the trial, but historical studies have shown patients with this diagnosis can expect to live about six months.

Infinity is betting big that it can confirm the results from this small study, in an ongoing trial of 120 patients, which will randomly assign patients to the new drug or a control group, and measure how long each group of patients lives. Once enough patients have died to get a good statistical comparison between the two groups, Infinity will unblind the data and release it. That eagerly awaited result is expected in the second half of 2012.

Infinity Pharmaceuticals CEO Adelene Perkins and R&D head Julian Adams

“We’re definitely in the game,” said Julian Adams, Infinity’s president of R&D, in an interview last week at the JP Morgan Healthcare Conference in San Francisco. “If the next trial performs like this one, we will have very significantly changed the landscape for pancreatic cancer.”

Many biotech companies have tried, and failed, to make much difference for pancreatic cancer, which often isn’t diagnosed until it has already spread through the body. About 36,800 people in the U.S. die each year from pancreatic cancer, according to the American Cancer Society.

The trial of this drug (pronounced “suh-RID-uh-gib”) will carry a lot of meaning for both physicians and cancer biologists. That’s because the compound is designed to inhibit a biological target known as Smoothened, a component of the Hedgehog pathway that is thought to help tumors grow and thrive when it’s turned into a mutated form. Infinity scientists showed, in a paper published in Science in 2009, that the drug disrupted the dense tissue matrix that encases tumors, Adams says. By altering the tumor’s microenvironment, the Infinity drug is thought to break down the tumor’s “cement-like fibrotic shell” and allow chemotherapy to get inside where it can kill cancer cells.

The drug’s mechanism means that it wouldn’t be used as single treatment on its own, but rather in combination with chemotherapy, Adams says.

Side effects of the drug appeared to be pretty typical for cancer trials. Of the 16 patients in this study, seven got the highest dose, 160 milligrams, which was selected for the ongoing trial of 120 patients. At that dose, two of the seven patients had moderate to severe depletion of their infection-fighting white blood cells, one had moderate to severe anemia, and another case was reported of elevated liver enzymes, which can be a sign of liver damage. No patient deaths were attributed to the Infinity drug or the chemo agent, researchers said.

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Japan-based Takeda Pharmaceuticals, which has its cancer drug development operations in Cambridge, MA, said today it is acquiring San Diego-based Intellikine to get ahold of the startup’s portfolio of cancer drugs.

Takeda said it has agreed to pay $190 million upfront, plus another $120 million in future milestone payments, to acquire privately held Intellikine. Through this deal, Takeda is getting its hands on a portfolio of small-molecule drugs that are made to block many of the different variations of a hot target in cancer biology-the PI3 kinase pathway.

This move inserts Millennium:The Takeda Oncology Company into one of the most competitive fields in the cancer drug business, pitting it against GlaxoSmithKline, Roche, Novartis, and Gilead Sciences, among others. Intellikine has long said it differentiates itself through its in-depth understanding of the four common variations of the PI3k pathway, and marrying that biology with a prolific chemistry team that created more than 1,500 different drug candidates to block those various targets. Two drug candidates in particular, dubbed INK218 and INK1117, were singled out in today’s statement by Takeda as having potential to be best-in-class compounds.

“As single agents or in different combinations with novel molecules within our robust pipeline, we anticipate that these assets will be able to deliver transforming therapies to cancer patients,” Millennium: Takeda’s CEO, Deborah Dunsire, said in a statement.

Intellikine, founded in September 2007 with a $12.5 million venture financing, grew out of science from the lab of Kevan Shokat at the University of California San Francisco. The company raised a $28 million Series B venture round in July 2009, from a syndicate that included Novartis Bioventures, Biogen Idec, FinTech Global Capital, and US Venture Partners, as well as previous investors Sofinnova Ventures, Abingworth Management, and CMEA Ventures. In July 2010, Intellikine pulled in another $13.5 million upfront through a collaboration with Cambridge, MA-based Infinity Pharmaceuticals (NASDAQ: INFI).

The Intellikine takeover marks the second time this year that a maker of PI3kinase inhibitors has been acquired. Back in February, Gilead Sciences acquired Seattle-based Calistoga Pharmaceuticals for $375 million upfront, plus another $225 million in potential milestone payments.

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Genetics Institute, one person told me last night, “was like college, only more fun.”

When’s the last time you heard anybody describe a corporate workplace that way?

There was something special about the culture and people who made up Genetics Institute, one of Boston’s original biotech highfliers from the ’80s and ’90s. We felt the energy in the air last night at an Xconomy event, “The Genetics Institute Impact.” This gathering, marking the 15th anniversary of American Home Products’ $1.25 billion acquisition of GI, brought together more than 250 people to the Koch Institute at MIT to reminisce and re-connect with old friends.

A great cast of characters from GI shared some revealing and funny stories. Here are some of my favorites:

—Adelene Perkins, now the CEO of Cambridge, MA-based Infinity Pharmaceuticals (NASDAQ: INFI), started smoothly, by observing that “we all look the same,” even though 15 years had gone by since the company was acquired. (Made me wonder, was there a secret anti-aging project going on at GI?)

As she went on, Perkins recalled how as a young dealmaker she was terrified about messing up a negotiation, and asked CEO Gabe Schmergel for advice about whether to ask for $10 million or $20 million upfront. Her boss replied, “your reputation stays with you,” which at the time, she says, “was not even remotely helpful.” Only after time went on, did she appreciate that Schmergel trusted she would figure it out and do the right thing.

—John Knopf, one of the early GI scientists, showed he clearly has potential as a stand-up comic if this biotech thing doesn’t quite work out.

But one of the serious points Knopf wanted to make was that GI was made up of more than a bunch of clever businesspeople and scientists. He told the story of how Bill Lonergan, a one-time Marine and the company’s former head of security, did his job in the least obtrusive way possible, and with good humor. Years later, Knopf recalled inviting Lonergan on a tour of a hot new biotech startup, Acceleron Pharma, which had raised more than $100 million. Lonergan teased Knopf and his colleague, Jas Seehra, about how he couldn’t believe those two young guys he knew from GI had done it.

“If two dumb shits like you can have success, I wonder what I could have done if I’d been a scientist,” Knopf quoted Lonergan as saying.

—Gabe Schmergel, in the closing keynote chat, shared some of his insight into what made GI special. Essentially, lots of CEOs pay lip service to the idea of building an organization by surrounding themselves with people who are more talented than they are. But, truth is, most of them are a bit too insecure to actually do it. “But I did hire people who were better than me,” he said.

For some of these memories, you just had to be there. Special thanks go out to the Koch Institute for Integrative Cancer Research at MIT for hosting this event, and to our event sponsors Alexandria Real Estate Equities, Cubist Pharmaceuticals, Infinity Pharmaceuticals, Aveo Pharmaceuticals, and Medidata Solutions Worldwide. And thanks to Keith Spiro for capturing so many of these great moments on camera. If you have some other photos of your own you’d like to share with the GI network, send me an e-mail at ltimmerman@xconomy.com, with a link to a photo-sharing website.

Enjoy the photos!

	NEXT IMAGE >>
	The Genetics Institute Impact — GI’s former chief operating officer, Pat Gage (left), catches up with co-founder Tom Maniatis as the event was getting started.
	photo by Keith Spiro

… Next Page »

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Medical device makers, drug developers, charity foundations, and health IT startups rounded out the New England life sciences news pot this week.

—Cambridge, MA-based MedicalRecords.com is raising $500,000 to put toward the development of its website, which connects sellers of electronic medical records software with doctors looking to adopt the technology.

—Rib-X Pharmaceuticals, a New Haven, CT-based antibiotics developer with two drugs in clinical trials, filed documents with the SEC indicating its intent to go public. The company, founded in 2000, has developed a drug-discovery platform based around an atomic, three-dimensional picture of the interactions between the antibiotics and the bacteria they target.

—The Michael J. Fox Foundation for Parkinson’s Research awarded grant money to Chelsea, MA-based Civitas Therapeutics, a startup working to improve the standard treatment for Parkinson’s disease. Patients usually take the drug, levodopa (L-dopa), in pill form, but Civitas is developing a version that they could inhale to get quicker, more precise doses of the medication. The company, spun out by the Waltham, MA, biotech Alkermes (NASDAQ: ALKS) will test the inhalable drug in a pair of clinical trials over the next year.

—Mundipharma will provide $50 million to Cambridge-based Infinity Pharmaceuticals in 2013 to support the development of Infinity (NASDAQ: INFI) drug candidates, like IPI-145, a molecule that’s being designed to treat blood cancers and inflammatory conditions. Infinity and Mundipharma first inked a collaboration deal in 2008, in which Mundipharma and its Stamford, CT-based affiliate Purdue Pharma, the maker of the drug oxycodone (Oxycontin), said they would pay $75 million to Infinity to access the company’s research and development capabilities.

—Cambridge-based Idera Pharmaceuticals regained the rights to a cancer drug it was previously co-developing with Merck KGaA, until the German drugmaker cancelled the collaboration this past July. Merck will continue an ongoing clinical trial of the drug, IMO-2055, in combination with Eli Lilly’s cetuximab (Erbitux) in 104 patients with a form of head and neck cancer, and Idera will reimburse the company for about 1.8 million Euros in expenses associated with the trial across 12 months. Idera (NASDAQ: IDRA) will own the data from that trial and other clinical studies Merck helped conduct and finance.

—A bioreactor made by Holliston, MA-based Harvard Bioscience was recently used in the world’s first two synthetic trachea implantations. Harvard’s InBreath Bioreactor helped create the tracheas by using the patients’ own stem cells. The company (NASDAQ: HBIO) has been around for 110 years as a maker of scientific instruments such as pumps and glassware for drug research, but it’s expanding its presence in the regenerative medicine field.

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The holiday weekend didn’t seem to slow New England deals making down.

—MedicalRecords, a Cambridge, MA-based startup connecting doctors with sellers of health record software, is working on raising its first outside funding round. It’s got about 60 percent of a targeted $500,000 round committed, from investors like Ty Danco and Peter Bordes.

—New Haven, CT-based antibiotics developer Rib-X Pharmaceuticals filed federal documents that reveal its plans to go public. The company has raised $208.4 million in venture funding and recently inked a partnership with Sanofi that could be worth as much as $772 million.

—Mundipharma will provide more than $50 million in funding in 2013 to Cambridge-based Infinity Pharmaceuticals as an expansion of a drug development partnership first inked in 2008. The money will go to developing drug candidates from Infinity (NASDAQ: INFI) such as IPI-145, which is being designed to target blood cancers and inflammatory conditions.

—VMTurbo, a Waltham, MA-based virtualization management software developer, announced that it had wrapped up a $10 million Series B investment led by its return backers Bain Capital Ventures and Highland Capital Partners. The new funding will go to product development, customer support, sales, and marketing.

—Skyhook Wireless has inked a deal to get its location-finding technology into the new Norton Anti-Theft Web service, from Mountain View, CA-based Symantec (NASDAQ: SYMC). The Norton service enables people to lock, locate, and, ideally, recover a lost or stolen Windows-based laptop, and Android smartphone or tablet, with Skyhook’s technology that use GPS, WiFi and cellular signals to track a device.

—ChoiceStream, a Cambridge-based developer of software for personalizing e-commerce shopping experiences for consumers, raised $8.7 million of a targeted $12.3 million equity and options offering, an SEC filing showed.

—Boston-based Summit Partners led a $10 million investment LogiXML, a maker of Web-based business intelligence software. Other LogiXML investors include Grotech Ventures and Updata Partners.

—Civitas, a Chelsea, MA-based spinout of the biotech Alkermes (NASDAQ: ALKS), will get an undisclosed grant award from the Michael J. Fox Foundation for Parkinson’s Reseach to put towards development of an inhaled form of the Parkinson’s treatment levodopa (L-dopa).

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Cambridge, MA-based Infinity Pharmaceuticals has found a way to secure more than $50 million for its R&D programs, through an extension of its partnership with Mundipharma.

Infinity (NASDAQ: INFI) said today that Mundipharma has committed more than $50 million in 2013 to the development of drug candidates from Infinity’s pipeline, including IPI-145, a molecule designed to block two specific forms of the PI3 kinase pathway. That drug is being developed to go after a variety of blood cancers and inflammatory conditions.

Infinity first struck the global alliance with Mundipharma back in November 2008. That’s when Stamford, CT-based Purdue Pharma, the maker of oxycodone (Oxycontin) and Mundipharma, an affiliated company with operations outside the U.S., agreed to pay $75 million upfront to tap into Infinity’s R&D capabilities. The original deal also provided Infinity with access to a $50 million line of credit from Purdue, and today Infinity said it has chosen to borrow all of that money. The debt will have to paid back by 2019, Infinity said in a statement.

“Since our alliance began in 2008, Infinity has advanced and expanded its portfolio of innovative product candidates,” said Antony Mattessich, regional director of Europe at Mundipharma, in an Infinity statement.

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Cambridge, MA-based Verastem has big names and big science on its side, which a lot of startups have, but now it’s doing something quite unconventional for a company that’s just 15 months old—it’s trying to go public.

Verastem, the company led by former Sirtris Pharmaceuticals CEO Christoph Westphal and founded on science from MIT luminaries Bob Weinberg and Eric Lander, most recently made news in July when it raised $32 million in venture capital. But today it looked to raise even more more, filing a prospectus for an initial public offering to raise as much as $50 million. The proposed deal is being underwritten by UBS, Leerink Swann, Lazard Capital Markets, Oppenheimer & Co., and Rodman & Renshaw.

Unlike most biotech companies seeking to go public, Verastem doesn’t have any drugs yet in clinical trials, which are easier for public investors to evaluate. Instead, the company’s IPO prospectus says it has drug discovery technology that seeks “to create a stable population of cancer stem cells to screen for and identify small molecule compounds that target cancer stem cells.” These cancer stem cells, sometimes called tumor-initiating cells, are thought to be one of the reasons why cancers often develop resistance to existing therapies and enable cancer cells to spread.

Scientists have long struggled to isolate enough of these elusive cells in the lab, and have found it difficult to keep them stable for experiments, and there has been great debate in academia about their ultimate value to cancer treatment. Verastem says it will seek to prove its concept with a lead drug known as VS-507, which targets breast cancer stem cells. The drug is still in preclinical studies, Verastem says.

What Verastem lacks in clinical proof, it makes up for with scientific and drug development experience. The company’s scientific advisory board includes George Daley of Children’s Hospital Boston; Nobel laureate Phil Sharp of MIT; Jose Baselga of Massachusetts General Hospital; Roger Tung, the CEO of Concert Pharmaceuticals; and Julian Adams, the president of Infinity Pharmaceuticals. Former Genzyme CEO Henri Termeer is on the board of directors.

Christoph Westphal is chairman and CEO of Verastem

Verastem has raised about $48 million in a pair of venture financings since it was founded in August 2010, and had burned through about $8.5 million of its cash through the end of September, according to the prospectus.

Several venture investors stand to quickly get some liquidity in their ownership stakes if Verastem can entice public investors to bite on an IPO. The biggest holders in Verastem, according to the filing, are Longwood Founders Fund, with a 15.5 percent stake; CHP of Princeton, NJ with 13.5 percent; MPM Bioventures with 13.2 percent; Bessemer Venture Partners with 12.9 percent; Eastern Capital Limited with 7.8 percent; and Advanced Technology Ventures with 5.1 percent. Westphal, 43, personally has 1.76 million shares of common stock in his name, or about a 3.4 percent ownership stake, according to the filing.

Verastem isn’t the only company going after cancer stem cells. Redwood City, CA-based OncoMed Pharmaceuticals has partnerships to develop drugs that go after cancer stem cells with Bayer and GlaxoSmithKline. The Verastem IPO filing also lists divisions of Astellas Pharma, Sanofi, and Glaxo as competitors.

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One of the scientists at Biogen Idec pushing for a new kind of regenerative medicine got some of her most valuable experience, and inspiration, back in the day at Genetics Institute. So we’re thrilled to have Sha Mi join what is shaping up to be a truly special gathering of alumni from GI, one of the pioneering companies in the Boston biotech scene.

Sha Mi, the distinguished investigator in Biogen’s discovery neurobiology group, has agreed to join the lineup of confirmed speakers at the next big Xconomy Boston life sciences event—”The Genetics Institute Impact” on Dec. 14. Sha Mi, who worked at GI from 1995 to 2000, will join the ranks of speakers who will offer a short story about one of their favorite memories from their GI days.

Besides Sha Mi (who sometimes goes by “Misha”) you can expect to hear these short stories from Adelene Perkins of Infinity Pharmaceuticals, Tuan Ha-Ngoc of Aveo Pharmaceuticals, Abbie Celniker of Eleven Biotherapeutics, Tony Evnin of Venrock Associates, and John Knopf of Acceleron Pharma. Plus, we’ll have a keynote chat with the two scientific co-founders of GI—Tom Maniatis and Mark Ptashne—along with longtime CEO Gabe Schmergel.

Sha Mi, distinguished investigator, Biogen Idec

Sha Mi is best known these days for her discovery work on a drug that seeks to be the world’s first regenerative medicine for multiple sclerosis. This experimental drug, which Biogen Idec has advanced into clinical trials, is designed to block a protein called Lingo-1 that interferes with the body’s production of myelin, the fatty insulating coating around nerve fibers. People with multiple sclerosis have an overactive immune system that eats away at the myelin layer, and they have no ability to regenerate myelin to protect their nerves. That means nerve impulses that control speech, vision, and movement get short-circuited, sort of like when an electrical wire is stripped of its insulation.

According to Sha Mi’s hypothesis, the new drug should be able to help regenerate the protective myelin coating around nerves. And if you can regenerate myelin, it’s conceivable you can restore lost neurological function, like the ability to see, talk, and walk. It will take a long time to find out if this works in humans, but the experimental drug has already cleared some significant hurdles by making it into the clinic.

I’m personally excited to hear Sha Mi tell her story about a memorable time at GI that help propel her along her current path. And I’ve heard great feedback from many GI alumni, and friends of GI alumni, who are looking forward to this special event. Tickets are going fast, so if you haven’t gotten one yet, be sure to sign up here at the registration page. See you there Dec. 14.

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Quite a few people study genetics in Boston. But there was—and can only be—one true “Genetics Institute.” Even though 15 years have gone by since the company was swallowed up in a corporate merger, the people who made “GI” one of the biotech industry’s pioneers in the ’80s are still making waves throughout the Boston biotech scene.

So I’m super excited to announce today we are bringing together a dream team of GI alumni for Xconomy’s next big public life sciences event in Boston, “The Genetics Institute Impact,” which will be held on Dec. 14.

Here are the confirmed speakers who will be there at the Koch Institute at MIT on Dec. 14:

—Mark Ptashne, Genetics Institute co-founder; Ludwig Chair of Molecular Biology, Memorial Sloan-Kettering Cancer Center

—Tom Maniatis, Genetics Institute co-founder; Professor and Chair, Biochemistry and Molecular Biophysics, Columbia University

—Gabriel Schmergel, former Genetics Institute CEO

—Tony Evnin, early venture investor in Genetics Institute; partner, Venrock Associates

—Tuan Ha-Ngoc, former executive vice president of Genetics Institute; CEO, Aveo Pharmaceuticals

—Adelene Perkins, former president of GI business unit DiscoverEase; CEO, Infinity Pharmaceuticals

—John Knopf, former scientist at Genetics Institute; co-founder and CEO, Acceleron Pharma

—Abbie Celniker, former director of antibody technologies at Genetics Institute; CEO, Eleven Biotherapeutics

I plan to make sure the speeches are short and sweet, so that everybody here has time to mingle and catch up with old friends. I’ll moderate a keynote chat with the two scientific founders, Ptashne and Maniatis, along with the former CEO, Gabe Schmergel. The other speakers—Tony Evnin, Tuan Ha-Ngoc, Adelene Perkins, John Knopf, and Abbie Celniker—will offer up one of their favorite memories from their time at GI in a couple of minutes.

Now, for the key details. This special event will go from 4:30 pm to 7:30 pm on Dec. 14 at the shiny new Koch Institute for Integrative Cancer Research at MIT. You can get your tickets by clicking here. I’m really looking forward to seeing a great crowd of GI alumni, friends of GI alumni, and stakeholders in the Boston biotech community. See you there Dec. 14.

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San Diego’s Intellikine will be four years old in another month, and CEO Troy Wilson tells me he’s feeling very excited about the venture-backed biotech’s progress so far in developing its pipeline of anti-cancer drugs, including its lead candidate designated INK128.

As Luke has reported previously, Intellikine’s research is focused on PI3K, shorthand for a group of kinase enzymes that help control an important cellular signaling pathway. The PI3K group is made up of four variations, or isoforms, that play slightly different roles in sending signals that help control protein synthesis, blood vessel growth, proliferation, and other cell processes.

Because PI3K enzymes are implicated in a wide variety of solid tumors and blood cancers, the PI3K pathway has become a hot target for cancer research. More than a dozen companies, including Big Pharmas like Novartis, Pfizer, and Bayer, have drug development programs targeting PI3K, as do rival biotechs like Exelixis of South San Francisco and Seattle’s Calistoga Pharmaceuticals, which was recently acquired by Gilead.

But Wilson says he likes the work Intellikine has done so far with INK128, a lead molecule designed to inhibit a key target, mTOR kinase, in a specific part of the PI3K signal pathway known as the TORC1/2 complex. The 30-employee company is planning to begin mid-stage trials of INK128 early next year, says Wilson, who calls INK128 “an exceptional drug candidate.”

Intellikine has raised a total of $62.5 million from investors, and Wilson says the company still has about $22.5 million available to move ahead to the next stage of drug development. Nevertheless, he says Intellikine also is generating strong interest among new investors. “Those discussions are ongoing,” he says, “and it’s likely the company will raise another venture round before the end of the year”.

Last year, Intellikine signed a global drug development and commercialization deal with Cambridge, MA-based Infinity Pharmaceuticals to develop small … Next Page »

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Whoever came up with the phrase “drinking from the fire hose,” could have easily been thinking of the American Society of Clinical Oncology annual meeting.

The information on cancer R&D has been flowing furiously the past few days at ASCO, which draws about 30,000 cancer doctors, drug company executives, investors, and journalists. I didn’t attend the meeting this year in person, but I’ve still had plenty to write about. Here are some quick thumbnail summaries and links that caught my eye from companies in our national network.

BOSTON

Ariad Pharmaceuticals (NASDAQ: ARIA). The Cambridge, MA-based company built up a lot of suspense around its presentation of a pivotal study for its lead drug candidate for sarcoma, a deadly bone cancer. We already knew heading into the conference that this drug, ridaforolimus, hit its primary goal in a study of 711 patients, showing it kept tumors in check for a median of 17.7 weeks, compared with 14.6 weeks for a placebo. Yesterday at ASCO, researchers said an interim look at survival times suggests an advance for the Ariad drug over placebo—a median of 21.4 months vs. 19.2 months—but more patients need to be followed over time before statisticians can say with any confidence that the drug helps people live longer. Ariad’s partner, Merck, repeated that it plans to seek approval in the U.S. and European Union based on the success of the trial.

Infinity Pharmaceuticals (NASDAQ: INFI). Infinity is the Cambridge, MA-based company that has dared to take on pancreatic cancer, a nasty malignancy that has pretty much whipped every drugmaker that has tried to take it on. When taken in combination with chemotherapy, Infinity’s drug, IPI-926, was able to produce meaningful tumor shrinkage in 31 percent of pancreatic cancer patients (5 of 16). There was no control group in this early-phase safety study, so this finding needs to be taken with a grain of salt, but historically less than 10 percent of patients have had that kind of response. Infinity is now in the process of attempting to prove this drug is a winner—through a randomized study of 120 patients that will measure whether IPI-926 in tandem with chemotherapy can help patients live longer than the chemo alone.

Synta Pharmaceuticals. Lexington, MA-based Synta (NASDAQ: SNTA) offered up some preliminary data that showed it has been thinking hard about how to pick patients with the right genetic profiles who are most likely to respond to its experimental drug, ganetespib. This drug, which works against a target called Hsp90, appeared most effective in lung cancer patients whose tumors expressed mutant forms of the genes ALK and KRAS. Patients with those mutations are especially difficult to treat, and the numbers of patients studied in that group is small, so it would be premature to raise any hopes about what this drug can do. Synta said six of the eight patients with ALK mutations (75 percent) had some tumor shrinkage in target lesions.

Alnylam Pharmaceuticals. The Cambridge, MA-based developer of RNA interference drugs (NASDAQ: ALNY) offered an update on an early-stage safety study of its experimental treatment for liver cancer, ALN-VSP. The company said most of the side effects experienced by the first 41 patients were mild in (fatigue, nausea, and fever) although there was one patient who suffered liver failure and died, in a case that Alnylam said “was deemed possibly related to study drug.” About two-thirds (7 of 11 patients) had their tumors stabilize at a dose of 1 milligram per kilogram of body weight—the dose Alnylam has selected as ideal for further development. The company didn’t talk specifically about next steps in clinical trials for ALN-VSP, but CEO John Maraganore did say “Clearly, these data are not only important for the continued advancement of our ALN-VSP program, but they also significantly increase our confidence in our entire pipeline of systemically delivered RNAi therapeutics.”

Constellation Pharmaceuticals. The venture-backed company in Cambridge, MA, developing cancer drugs based on advances in epigenetic science gave everybody a break on crunching hard data on drug response rates and side effects. It made some financing news, by raising $15 million in an extension of its Series B venture round, pulling in cash from Third Rock Ventures, The Column Group, Venrock Associates, SR One and Altitude Life Science Ventures.

San Francisco

Genentech/Plexxikon. Many of the big headlines at this year’s ASCO went to … Next Page »

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You wouldn’t know we had a holiday this week, with the rate Boston life sciences companies are pumping out news. They’ve inked partnerships, announced fundings, made acquisitions, and are gearing up for a big medical convention this weekend.

—Cambridge, MA-based Ariad Pharmaceuticals (NASDAQ: ARIA) will reveal data from a clinical trial of its drug ridaforolimus to an eager audience at next week’s American Society of Clinical Oncology’s (ASCO) annual meeting in Chicago, Luke wrote. The drug was developed in a collaboration with Merck and is designed to block a protein that causes tumor growth.

—Woburn, MA-based drug developer Excelimmune said it nabbed $10.5 million in Series B financing, from new and previous individual backers. The money will help advance Staphguard, the company’s treatment for Staph infections resistant to methicillin.

—Aveo Pharmaceuticals of Cambridge said it entered into a partnership with Johnson & Johnson’s (NYSE: JNJ) Centocor Ortho Biotech unit to work together on Aveo’s antibodies targeting a receptor involved in tumor growth. Aveo (NASDAQ: AVEO) received $15 million upfront for this, and could get $540 million more down the road in milestones.

—Cambridge-based Dossia, a nonprofit consortium offering online medical records to employees of its member companies, inked a partnership this spring with Denver-based Health Language. Dossia CEO Michael Critelli says the collaboration will help translate the medical jargon in the clinical records into plain English for patients to better understand and manage their care.

—Luke took a look at Cambridge-based Infinity Pharmaceuticals (NASDAQ: INFI) as it readies itself for a presentation at ASCO. The drug developer will reveal data from the first 16 months of a trial of its new drug candidate for pancreatic cancer, IPI-926.

—My colleague Arlene wrote about Newton, MA-based NeuroHealing, a virtual biotech company that looks for already approved drugs and reformulates them to treat central nervous system disorders. The company has been getting grants and other investments to fund the development of its drugs for treating patients with traumatic brain injuries, Parkinson’s disease, and premature ejaculation.

—Lumicell Diagnostics, a Waltham, MA-based developer of a cancer diagnostic device, took in $2.7 million in equity-based funding.

—Bedford, MA-based diagnostic and medical imaging firm Hologic (NASDAQ: HOLX) announced it had acquired TCT International, a China-based distributor of medical products, for $135 million in cash.

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Infinity Pharmaceuticals’ top execs sometimes get strange looks when they say they are developing a new drug for pancreatic cancer. This, after all, is a wicked malignancy that usually kills patients in just a few months, and has dashed the hopes of many cancer drug developers over the years.

Yet Cambridge, MA-based Infinity (NASDAQ: INFI) is moving full steam ahead with a new drug candidate for pancreatic cancer, IPI-926, in a mid-stage trial of 120 patients this year. Infinity will offer a clearer picture of why it has moved ahead with this program during a presentation this weekend at the American Society of Clinical Oncology in Chicago. That’s where researchers will present data on the first 16 patients with pancreatic cancer who got the Infinity drug in combination with a standard chemo drug.

An interim peek at the data from the first-phase trial showed that three of the first nine patients (33 percent) had their tumors shrink by half or more when they got IPI-926 in combination with standard gemcitabine (Gemzar) chemotherapy. The study was designed to look at safety of a variety of doses, and there was no control group, so it’s impossible to say for sure how good that really is compared to anything else. But it was an eye-opening result nonetheless, given that only about 5 percent of patients will typically see that kind of tumor shrinkage when getting the gemcitabine alone, says Julian Adams, president of R&D at Infinity. Common side effects were fatigue, nausea, and an elevation in liver enzymes which can be a sign of liver damage, but which weren’t considered serious in this trial, and which were reversible.

“We are circumspect. We don’t want to create unbridled enthusiasm. We are humbled by the challenge of this disease,” Adams says. That said, he notes that the ongoing mid-stage study of 120 patients is recruiting patients fast, and should be completely enrolled by the end of this year. “The investigator community is jazzed,” Adams says.

Julian Adams

Pancreatic cancer, once it has spread through the body, usually gives people a life expectancy of just under six months, Adams says. About 36,800 people in the U.S. die each year from pancreatic cancer, according to the American Cancer Society. The really big test for Infinity will be to see if its drug can help those pancreatic cancer patients live longer, which is the main goal of the ongoing trial of 120 patients. Results from that study are expected by late in 2012, Adams says.

Part of what draws attention to the Infinity program is the science. The drug is designed to inhibit a biological pathway known as Hedgehog, which is thought to help tumors grow and thrive when flipped into a mutated form. Infinity scientists showed, in a paper published in Science in 2009, that the drug by itself had no effect on pancreatic cancer in mice, but that it disrupted the dense tissue matrix surrounding tumors, Adams says. By disrupting the tumor’s microenvironment, it is thought to help enable cell-killing chemotherapy like gemcitabine to do a better job of penetrating the tumor, Adams says.

The drug helped mice live longer with cancer, but curing cancer in mice isn’t the same … Next Page »

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Calistoga Pharmaceuticals CEO Carol Gallagher was playing with a strong hand of cards, holding onto an emerging cancer drug she could have sold for a mint to any number of Big Pharma companies hungry for innovative new products.

But she and Calistoga’s board took an unusual tack, selling the Seattle-based company for as much as $600 million to an HIV drug powerhouse that’s still a newbie to the cancer drug business—Gilead Sciences (NASDAQ: GILD).

While Gilead may not have the experience of a Pfizer, Roche, or Novartis in cancer, it sees the cancer drug landscape evolving into something right up Gilead’s alley, Gallagher says. The vision is that a cancer diagnosis will become less like a short-term death sentence, and more of a chronic disease that must be managed regularly, like HIV. As DNA sequencing gets fast and cheap enough for many more doctors to see what’s going wrong at a molecular level, patients will be treated with cocktails of drugs aimed at specific molecular targets, through convenient oral pills, that offer mild side effects compared to chemotherapy, says Gilead’s chief scientific officer, Norbert Bischofberger. Calistoga’s drug candidates fit right into this vision, he says.

If Gilead and Calistoga are right about this broad shift, then they could capture a toehold in a cancer drug market estimated to be worth $84 billion in 2012, according to Cowen & Co.

“We think a lot about where the field is going, and like us, Gilead believes people can live with the disease without a lot of side effects,” Gallagher says. “Patients will live longer lives, and have better quality of life. That’s what Gilead has done in the HIV space. They have this shared belief with us.”

Calistoga, of course, had lots of other reasons to sell the company now. Its venture capitalists, like all VCs, wanted to see a return on the more than $90 million they’ve invested since 2006. The company, as you’d expect, looked at all the usual strategic options it had to maximize the opportunity with its lead drug—an IPO, a partnership, an acquisition, Gallagher says.

Carol Gallagher

The time was right to get acquired, Gallagher says, because of the resources Gilead can throw behind its cancer drug vision. Calistoga’s drugs also “won’t get lost in the shuffle,” like they might in a massive portfolio of cancer drugs at another Big Pharma company, Gallagher says. Calistoga’s lead drug, CAL-101, will instantly become the most valuable cancer drug asset at a company with a market valuation of $31 billion, and which generated $7.95 billion in sales last year. And cancer isn’t the end of the story—Bischofberger says there is reason to believe that drugs like Calistoga’s could be effective against inflammatory diseases.

“We really have a shared vision of how we can use targeted therapies to change patients’ lives in cancer and inflammatory disease,” Gallagher says.

Of course, Calistoga wouldn’t be in a position to sell at all without some promising results for its drug from clinical trials.

A little bit of science is required to understand why Calistoga became a hot property the past couple years. It is one of the companies with a specific drug aimed at a molecular target known as the PI3 kinase pathway—which is involved in multiple cell processes like proliferation, growth, migration, and cell survival. Research has shown over several years that when this pathway gets switched into an overactive mode, it can lead to cancer and inflammatory diseases. Multiple big pharma companies, including GlaxoSmithKline, Novartis, Roche, and Sanofi-Aventis, as well as small companies like San Diego-based Intellikine and Cambridge, MA-based Infinity Pharmaceuticals (NASDAQ: INFI), have wagered on this field over the past few years.

Calistoga has sought to separate itself from the pack by specifically designing its lead drug, and others in its pipeline, to hit a certain variation of the PI3 kinase known as the delta isoform, which is believed to only appear on certain blood cells. The idea is that you can have … Next Page »

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Infinity Pharmaceuticals (NASDAQ: INFI) said today in its financial forecast that the company has enough cash on hand, and committed research funding, to run its R&D operation into 2014. The Cambridge, MA-based developer of cancer drugs said it enters this year with $345 million in committed capital, which includes slightly more than $100 million of cash and investments, another $195 million in committed R&D funding from its partner Mundipharma, and a $50 million line of credit from Purdue Pharma. Infinity also described a number of clinical development goals in a statement.

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New England’s life sciences companies and investors are wasting no time this new year. We saw headlines of financings, clinical trial results, acquisitions, and executive moves at biotech and pharma companies this week.

—Genocea Biosciences of Cambridge, MA, wrapped up $35 million in Series B financing from new investors Johnson & Johnson Development Corporation, MP Healthcare Venture Management, and Skyline Ventures. Previous Genocea backers Auriga Partners, Cycad Group, Lux Capital Management, Morningside Ventures, Polaris Venture Partners, and SR One also participated in the deal for the vaccine discovery and development firm.

—Year-old biotech venture firm Longwood Founders Management announced it had raised its first fund, with help from Cambridge-based Genzyme (NASDAQ: GENZ) and GlaxoSmithKline (NYSE: GSK), which employs two of the fund’s founders, Christoph Westphal and Michelle Dipp. The fund raise totaled to $86.9 million, according to an SEC filing. Ryan caught up with the other founder, biotech investor Rich Aldrich, for a look at Longwood’s strategy.

—Boston-based Relay Technology Management is ready to launch its search and analytics engine for biotech business development groups early this year, Ryan wrote. The startup was formed by a duo of Tufts University researchers in 2008.

—George Cotsarelis, co-founder of baldness treatment developer Follica, was part of a team that released research this week showing that men with pattern baldness have plenty of hair follicle stem cells in their scalps. Follica CEO William Ju said the findings fit with the rationale behind the experimental drug and device under development at the mostly virtual biotech firm, which was founded and incubated at PureTech Ventures in Boston.

—Cambridge weight loss drug developer Zafgen announced that its experimental treatment for severe obesity was well-tolerated and helped patients lose about 1 kilogram (2.2 pounds) of body weight per week in its first human study, which lasted one month. The biotech still has more to prove—obesity drugs must show sustained benefits for at least a year—but the first 24 people tested saw better weight loss effects than those with placebo.

—Ryan previewed our upcoming Xconomy Xchange: HHS CTO Todd Park and Friends on the Future of Health IT. Park, who co-founded Watertown, MA-based Athenahealth (NASDAQ: ATHN) before working in Washington, will dish on how the government can help innovate in the health IT space.

—Medical device maker Boston Scientific (NYSE: BSX) of Natick, MA, announced it had bought Intelect Medical, a maker of neuromodulation technologies for deep brain stimulation, for about $60 million in cash. Boston Scientific, which already had an equity stake in the company, said it will complement its existing technology to help physicians better visualize brain stimulation fields.

—Weston, MA-based Biogen Idec (NASDAQ: BIIB) enlisted former ZymoGenetics CEO Doug Williams to lead its R&D group. It also brought on a new vice president of corporate development: Steve Holtzman, the former chief executive of Cambridge-based Infinity Pharmaceuticals (NASDAQ: INFI). Both new hires will report to George Scangos, who joined Biogen as CEO in summer of 2010.

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