VC, funding, Life Sciences Wade Roush wrote: Link Medicine, a secretive Cambridge, MA, startup that’s spent the last three years investigating new therapies for neurodegenerative diseases like Alzheimer’s, Parkinson’s, Huntington’s, and Amyotrophic Lateral Sclerosis (ALS), said today that it has completed a $40 million Series C venture round. The investment comes from Clarus Ventures, a Cambridge- and San Francisco-based fund focused on life science companies, and SV Life Sciences, another biotech-oriented fund based in Boston, San Francisco, and London. It’s a significant jump over Link’s first two venture rounds, which together brought in $16.5 million. The company’s drug discovery approach is focused around the work of its chief scientific officer, Harvard Medical School neurologist Peter Lansbury, who is developing ways to counter the buildup of misfolded proteins in neural tissue that seems to be a common factor in several neurodegenerative conditions. But as the Boston Globe reports today, the company’s first drug is actually a compound it’s in-licensing from an unnamed pharmaceutical company that unsuccessfully tested it against a different illness. The company says the funding round will help it move this compound from preclinical programs into human testing, which it hopes to begin next year. Link’s CEO, Adam Rosenberg, told the Globe that the company’s ability to raise such a significant round of working capital is a sign that venture capital firms are still interested in life science startups with “novel science in areas of high unmet need.” Nick Galakatos, managing director of Clarus Ventures, said in the company’s funding announcement that its approach is “a novel way to tackle Alzheimer’s disease and related disorders. It is particularly attractive because it can be potentially used as a monotherapy, or as a complementary combination with other medicines.” But the company has said little about the compound it’s testing or its mechanism of action. Comments | Permalink | Share |  E-mail UNDERWRITERS AND PARTNERS Less

Biotech, Alzheimer's, Proteostasis Luke Timmerman wrote: One of those foggy concepts from eighth-grade biology came back to me the other day when I spoke with David Pendergast. It’s homeostasis, the idea that the human body naturally makes constant adjustments on the fly to maintain a proper balance of things like body temperature, water, or salt in your system. Pendergast, the CEO of Cambridge, MA-based Proteostasis Therapeutics, is attempting to take advantage of new insights in biology that may enable scientists to promote or restore the homeostasis of proteins—molecules that are key players in every tissue and organ in the body and which can cause a host of problems when they’re out of whack. Restoring protein homeostasis might mean revving up the protein machinery that normally does the job of sweeping up degraded proteins; this machinery can break down as we age, causing diseases like Alzheimer’s or Parkinson’s. Or it could help patients with rare genetic disorders, such as Huntington’s disease, who might just need a nudge to their protein networks to keep their symptoms at bay, Pendergast says. Proteostasis recently raised a whopping $45 million in its initial round of financing, from HealthCare Ventures, Fidelity Biosciences, New Enterprise Associates, Novartis Option Fund, and Genzyme Ventures. Pendergast was formerly the president of human genetics therapies at Shire Pharmaceuticals, and before that was CEO of Cambridge, MA-based Transkaryotic Therapies. He’s now placing a big bet on a field of biology that isn’t being tapped by traditional pharmaceutical or biotech companies, and that’s still three to five years away from entering clinical trials, Pendergast says. “We’re talking about restoring a natural function that works for people,” Pendergast says. “It has a lot of appeal, as opposed to a lot of other drugs that try to block something, and you may not always understand the unintended consequences of that.” Some of the pioneering work in this field of biology comes from the lab of Proteostasis co-founder Andrew Dillin at the Salk Institute in San Diego. A series of experiments there and other labs have given the venture capitalists confidence that it’s time to invest, Pendergast says. The researchers were able to show they could stimulate the pathways that control protein homeostasis using conventional small-molecule drugs that are cheaper and easier to make than drugs produced through genetic engineering, he says. The effect is reversible, and reproducible in rodents, worms, and tissue cultures growing in lab dishes, he says. It sounded to me like a technology that could potentially be applied as a preventive medicine. Who wouldn’t want a pill, for example, that might be able to ensure 70-year-olds will maintain order in the protein network responsible for clearing up amyloid plaques in the brain, which are thought to play a key role in Alzheimer’s disease? It’s theoretically possible to do that, Pendergast says, but that’s not what the company has in mind. To get a drug approved by the FDA, the company would aim to treat patients who already have mild-to-moderate Alzheimer’s disease, then give them the drug for a six-month or one-year period, and use standard measures tracking their cognition. It would take many more years, and loads of more capital, to show, say, that healthy 70-year-olds who take a Proteostasis drug have a lower risk of getting Alzheimer’s by the time they turn 80. That’s a pretty far-out, futuristic vision, and not really on the company’s agenda, Pendergast says. For now, Pendergast is focused on hiring a chief scientific officer and scientific team of 20 people over the next 12 to 18 months, to better understand the protein networks it is thinking about targeting. It’s a new challenge for Pendergast, whose experience is with leading bigger companies. “Here, you’re starting with a blank sheet of paper,” he says. “It’s a building project. We’re excited, and we think this is an exciting area of biology, and we hope we can show significant benefit for patients.” Comments | Permalink | Share |  E-mail Less

Biotech, Diagnostics, Quanterix Luke Timmerman wrote: Quanterix made the news this week when it raised the second half of a $15 million financing round from Arch Venture Partners, Bain Capital Ventures, and Flagship Ventures. The Cambridge, MA-based company has no trouble commanding attention, since its technology comes from the lab of Tufts University researcher David Walt, a co-founder of Illumina (NASDAQ: ILMN), the high-flying San Diego-based maker of biological research tools. Despite the company’s pedigree, though, Quanterix’s latest round was covered as a run-of-the-mill VC financing blurb. So, I contacted CEO Nick Naclerio to learn more. The company, which started in June 2007, is working on what it calls a Single Molecule Array, or SiMoA for short. It’s supposed to be 1,000 times more sensitive than the standard Elisa tests, made by big players like Abbott Laboratories (NYSE: ABT) and Becton Dickinson (NYSE: BDX), that are used to detect antibodies in a blood sample and which can diagnose whether a patient has been exposed to HIV, West Nile Virus, or other pathogens. Quanterix has a prototype device, Naclerio says, and the new capital will be used both to further develop the technology platform and for preliminary clinical trials. The company’s aim is to use SiMoA to find the tiny concentrations of proteins that can’t be seen by Elisa. One example might be a millimeter-wide tumor, at the very early stages of growth, that sheds minute amounts of signature proteins into the blood. It’s theoretically possible the test could spot tiny amounts of proteins that are linked to Alzheimer’s, Naclerio says. “We are able to count individual molecules,” he says. “Generally, the more sensitive you are with a diagnostic, the better.” Lots of work still needs to be done to establish how useful such a test might really be, Naclerio says. For instance, clinical studies will have to be done to show whether tiny concentrations of a protein in the blood are really an early sign of bad things to come with cancer. Until a trial says otherwise, it’s possible that small tumors might simply be mopped up by the immune system, and might cause people to be worried for no good reason, I suggested. Naclerio didn’t disagree, which is why he added that the company is working with physician collaborators on which meaningful proteins they really would like to look for in the blood if they had a powerful device that could find them. The company plans to concentrate initially on known proteins, and isn’t going to spend its research budget looking for new proteins that could help with early detection of cancer. However, one well-known project of that kind is the International Cancer Biomarker Consortium, led by Nobel Laureate Lee Hartwell, president of the Fred Hutchinson Cancer Research Center in Seattle. Given how much promise Hartwell and his collaborators see with proteins serving as early warning signs of cancer, it seems likely Quanterix won’t have much trouble finding researchers who want to use its precise new tool to learn about those diagnostic clues lurking in the blood. “This technology has the potential to open up a lot of new applications in diagnostics,” Naclerio says. Comments | Permalink | Share |  E-mail Less

Biotech, Alzheimer's, Epix Luke Timmerman wrote: Epix Pharmaceuticals, the Lexington, MA-based developer of an experimental drug for Alzheimer’s disease, said today that Kingsbridge Capital has committed to provide as much as $50 million of capital over the next three years through buying new shares. The actual amount raised by the company (NASDAQ: EPIX) will depend on the number of shares it sells and the market value of Epix stock at the time of the purchases. Epix can sell a maximum number of 8.3 million shares under the agreement. Permalink | Share |  E-mail Less

Biotech, Resignation, Alzheimer's Luke Timmerman wrote: Here’s a CEO resignation that caught me by surprise this morning. Epix Pharmaceuticals CEO Michael Kauffman stepped down as CEO and as a member of the company’s board of directors, as of last Friday, according to a statement from the Lexington, MA-based company. He left for the proverbial chance to “pursue other opportunities.” Kauffman, 44, became Epix’s CEO in August 2006, through the company’s acquisition of Predix Pharmaceuticals, the company said. He is being replaced by Elkan Gamzu, an Epix director, who will be interim CEO. Epix (NASDAQ: EPIX) has had a bumpy ride under Kauffman. Its Vasovist product, designed to help doctors get a clearer picture from magnetic resonance angiography tests that look at blood vessels, has been dealt a series of delays from the FDA since Epix first submitted it for approval in December 2003. An experimental depression drug failed in a mid-stage clinical trial in March. And Epix’s Alzheimer’s drug candidate showed promising results in December in a small clinical trial, yet the company later discovered its contract research organization made errors in data-crunching that ended up making the drug’s cognitive benefit appear more modest. Not surprisingly, the stock has dropped 49 percent so far this year, trading at $2 a share at 12:27 p.m. Eastern today. Still, Kauffman hasn’t been in hiding. I spoke to him less than two weeks ago, for a feature story about how he was preparing for a big Alzheimer’s conference in Chicago that runs through Thursday. He touted a couple of anecdotal cases of patients who stayed on the Alzheimer’s drug for six months, and showed “profound” improvement. “You don’t have to be a physician to see progress when a patient can suddenly play cards when they couldn’t before,” Kauffman said at the time. The drug, being developed in partnership with pharmaceutical giant GlaxoSmithKline, is currently being tested in larger mid-stage clinical trials. We put a call in to a PR agency representing Epix, with the two big questions: Why’s Kauffman leaving, and what’s he going to do next. I also wonder what he gets in terms of a golden parachute. The company’s most recent proxy filing with the SEC says he got $1.3 million in total compensation last year, made up of salary, bonus, the value of stock option awards and perks. If we hear back anything, or we see some answers in any new regulatory filings, we’ll let you know. Comments | Permalink | Share |  E-mail Less

Biotech, Alzheimer's, clinical trials Luke Timmerman wrote: Epix Pharmaceuticals is having a wild year with its Alzheimer’s drug development program. Shares in the Lexington, MA-based company (NASDAQ: EPIX) shot up 35 percent in one day in December after it said its drug produced one of the best improvements ever in memory and thinking skills for Alzheimer’s patients in a clinical trial. One month later, the gains were wiped out when a major goof-up was revealed. Epix said its contract research organization made mistakes crunching the numbers, so the improvement in patients’ cognition score was 3.6 points on a standard scale, not the 5.7 point gain previously reported. “Obviously, we are not using those folks anymore,” says Epix CEO Michael Kauffman. Now Epix is trying to set the record straight for good. The company plans to present full results from the mid-stage trial at the International Conference on Alzheimer’s Disease in Chicago that runs July 26-31. About 5,000 researchers will gather there to learn about the latest research against this incurable disease, which robs people of their memory and thinking skills and affects more than 5 million Americans, according to the Alzheimer’s Association. The key finding for Epix is still the same as what the company reported January—that 10 patients who took a daily, 150 milligram dose of the drug by itself for two weeks had a 3.6 point improvement in their ADAS-cog score, a standard measurement of memory and thinking skills, compared with a 0.9 point worsening for 10 patients on placebo. Some more data is available from a computer test that patients took, which verifies the benefit, and suggests Epix’s drug may also work in tandem with Aricept, the Alzheimer’s treatment from New York-based Pfizer (NYSE: PFE) and Tokyo-based Eisai, Kauffman says. No serious side effects of Epix’s drug were seen in the study. One of the strengths of the study is that it showed a benefit so quickly, although critics will say Epix still needs to demonstrate that the drug has a sustained effect over six months, Kauffman says. The company doesn’t have that proof yet, Kauffman says, but it plans to present some interesting results from a couple of trial volunteers who asked to continue taking the drug after the two-week study ended. In response to the patients’ request for more drug, the company made a special petition to the FDA for a six-month extension of the study for those two individuals. The request was granted, Kauffman says, because Alzheimer’s patients have so few treatment options. The two patients in the extended trial had periods where they went off treatment, and showed “profound” improvement when they went back on, Kauffman says. “You don’t have to be a physician to see progress when a patient can suddenly play cards when they couldn’t before,” Kauffman says. Epix’s drug, called PRX-03140, is designed to work unlike any marketed Alzheimer’s drug, by stimulating production of a neurotransmitter in the brain called acetylcholine. In the study, it didn’t produce the key side effect of Aricept, nausea. The market is obviously huge for Alzheimer’s—Aricept, the most-prescribed Alzheimer’s medicine worldwide, generated $2.16 billion in worldwide sales in the fiscal year ended in March 2007. That’s for a drug that doesn’t really improve cognition, but can slow down the patients’ decline. Epix and partner GlaxoSmithKline (NYSE: GSK) are recruiting patients into larger trials that could go a long way toward demonstrating if the experimental drug really has legs. One trial of 420 patients will compare two different doses of the Epix drug in combination with Aricept against placebo, measuring the ADAS-cog score after six months. A second study of 240 patients will compare the Epix drug to Aricept and to placebo. That trial will run for three months, with another three-month extension. The latter study should produce its first results in mid-2009, Kauffman says, while the larger trial in combination with Aricept will take longer, delivering findings in the first half of 2010, he says. By that time, more data will be available on competitors like drug giant Wyeth (NYSE: WYE) and Elan’s bapineuzumab, and Medivation’s (NASDAQ: MDVN) Dimebon. Some of the buzz at the meeting will also be about the failure last month of a clinical trial of Flurizan, an experimental drug from Salt Lake City-based Myriad Genetics. There may be something to learn from it, Kauffman says. The failure of Flurizan has gotten some researchers casting around again for promising new leads. It certainly hasn’t hurt a company like Epix, with a new method of treatment, at an earlier stage of development. “People in the field are starting to feel like we’re asking the right questions,” Kauffman says. “We’re getting some more calls and more interest about our program.” Comments | Permalink | Share |  E-mail Less

Dr. Sue Halpern is author of the book, Can’t Remember What I Forgot: The Good News from The Front Lines of Memory Research. She is also scholar in residence at Middlebury College in Vermont. Here’s what she says about herself: “I was educated at Yale and at Oxford, where I was a Rhodes Scholar, and [...] Less