[Updated, 3/30/15, 8:20 am ET, see below] For those looking to get away from the broader biotech indices’ plummet this week (the Nasdaq Biotechnology Index (NASDAQ: [[ticker:IBB]]) fell 7 percent), we’ve got your comfort food here: a feature about gene therapy’s long quest to cure hemophilia, a scientific journey that began in the 1980s. We’ve also got news of a high-flying biotech executive’s new venture, a $537 million fund for one of Boston’s life sciences startup creators, and plenty more in this week’s roundup. Details below.
—Gene therapy’s most competitive race will unfold over the next few years, with seven groups trying to prove they can provide a long-lasting treatment, if not a cure, for the blood disease known as hemophilia. This race has been in the making for decades, and runs through the highs and lows of the gene therapy field—from its enormous promise, to the safety issues that eviscerated investment in the field many years ago. I explored the story of gene therapy in hemophilia, and some of the key figures who kept research alive in its darkest days.
—Jeremy Levin, the former Bristol-Myers Squibb dealmaker and Teva Pharmaceutical CEO, is back; he’s become the chairman of a New York startup called Ovid Therapeutics, which is developing treatments for orphan diseases of the brain. Additional details about the startup are scarce, but I spoke with Levin about Ovid and found a few other tidbits that give some insight into its plans.
—Just weeks after it inked its first partnership, a $265 million deal with Alexion Pharmaceuticals (NASDAQ: [[ticker:ALXN]]) Cambridge, MA-based Blueprint Medicines has outlined plans to go public.
—Shares of Waltham, MA-based ImmunoGen (NASDAQ: [[ticker:IMGN]]) surged over 20 percent on Monday after the firm cut a $440 million deal with Takeda to develop cancer drugs using its antibody-drug conjugate technology. ImmunoGen has since lost some of those gains amidst a broader biotech selloff.
—Boston-based Gelesis nabbed $22 million in equity financing to fund more trials for its anti-obesity pill, which swells up in the stomach to make people feel full. The pill, Gelesis100, isn’t absorbed by the body, so Gelesis is trying to win regulatory approval as a medical device, not a drug.
—Cambridge-based Flagship Ventures closed a whopping $537 million fund to back more biotech startups. The fund is almost double the previous $269 million one Flagship closed in 2012.
—Cambridge-based Semma Therapeutics emerged from stealth with a bang: a $44 million Series A round from MPM Capital, Fidelity Biosciences, Arch Venture Partners, and Medtronic, and an undisclosed deal with Novartis. Semma is advancing a cell therapy for Type 1 diabetes created by Harvard University professor Doug Melton.
—In case you missed it, we posted the agenda for “What’s Hot in Boston Biotech,” our latest East Coast biotech event. It’ll take place on April 8 at the Broad Institute of MIT and Harvard.
—Good news for New York-based Intercept Pharmaceuticals (NASDAQ: [[ticker:ICPT]]) this week, as rival GenFit’s competing drug for nonalcoholic steatohepatitis (NASH) failed a mid-stage trial (though GenFit, for its part, claimed that the study would have met its goal “with correction for baseline severity and site heterogeneity.”). Shareholders weren’t buying GenFit’s explanation, sending shares down more than 30 percent. Shares of Intercept, now the clear frontrunner to bring the first NASH drug to market, meanwhile, climbed about 8.5 percent.
—Less than two years after paying MorphoSys $92 million up front (and investing in $50 million of Morphosys stock) to co-develop a multiple myeloma drug called MOR202, Summit, NJ-based Celgene (NASDAQ: [[ticker:CELG]]) has scrapped the deal. The drug, which targets the protein CD38, is well behind competing drugs from Johnson & Johnson and Sanofi.
—[An earlier version of this story had Vertex based in Cambridge] Shares of Boston-based Vertex Pharmaceuticals (NASDAQ: [[ticker:VRTX]]) fell about 4 percent after investors digested the data released from a mid-stage study of VX-661, another one of its experimental cystic fibrosis drugs. VX-661 is meant to be an improved version of the experimental CF drug lumacaftor, used in combination with Vertex’s marketed CF drug ivacaftor (Kalydeco); the idea is a VX-661/ivacaftor combination could open up Vertex’s drugs to thousands more CF patients with different genetic traits. Vertex had said previously that the data from the study were good enough to move VX-661 into Phase 3 testing, but as TheStreet.com and Bloomberg wrote this week, the numbers fell short of expectations.
—Waltham, MA-based Proteon Therapeutics (NASDAQ: [[ticker:PRTO]]) released results from a long-term follow-up analysis of patients in a Phase 2 study of vonapanitase (previously known as PR-201), a drug meant to boost the success rates of a surgical procedure used to prepare kidney failure patients for dialysis. The drug helped reduce the number of “corrective procedures”—needed when that surgical procedure procedure, known as an arteriovenous fistula, fails—compared to a placebo.
—Shares of Cambridge-based OvaScience (NASDAQ: [[ticker:OVAS]]) climbed this week on the results of a study showing its fertility treatment, Augment, helped some women who had failed multiple cycles of in vitro fertilization get pregnant.
—New Brunswick, NJ-based Johnson & Johnson (NYSE: [[ticker:JNJ]]) has teamed with Google (NASDAQ: [[ticker:GOOG]]) on an effort to develop robotic tools that would help physicians perform surgical procedures. Financial terms of the deal weren’t disclosed.
Photo courtesy of flickr user Bill Damon via Creative Commons.
