East Coast Biotech Roundup: Vertex, Cubist, Editas, Polaris, & More
There was a lot of activity back East the past seven days, most of it in Boston and Cambridge, biotech’s ground zero. Vertex released excellent Phase 3 data, and Cubist got a green light from the FDA. Gene editing startup Editas Medicine found a new CEO, while the more advanced gene therapy firm Dimension Therapeutics found a new corporate partner and a new investor. Merrimack Pharmaceuticals took a step toward figuring out a treatment for pancreatic cancer, while Dana-Farber and J&J teamed up to unravel the complexities of lung cancer. And cornerstone life science investors Polaris Partners let the feds know they’re raising a new fund. Let’s round ‘em up.
—Vertex Pharmaceuticals (NASDAQ: VRTX) of Cambridge released Phase 3 data Tuesday for its combination of ivacaftor (Kalydeco) and lumacaftor that showed improved lung function in a genetic subset of cystic fibrosis patients. Its stock rose 40 percent Tuesday, and three days later it remains in the low $90 range.
—As Xconomy reported Tuesday, Editas Medicine of Cambridge, seven months after announcing a $43 million Series A round of funding, unveiled its choice for CEO. It’s Katrine Bosley, a veteran of the local biotech scene whose last company, Avila Therapeutics, had only the bare minimum of clinical data when it was snapped up by Celgene for $350 million upfront. Bosley, an Xconomist, will now try to steer a new technology—gene editing based on the recently discovered bacterial defense system called, in shorthand, CRISPR/Cas9—from a highly popular research tool to a breakthrough way of delivering cures.
—Cubist Pharmaceuticals (NASDAQ: CBST) won FDA marketing approval on June 20 for its antibiotic to treat skin infections, tedizolid phosphate (Sivextro). Cubist acquired the drug when it bough Trius one of several antibiotic programs to get a boost from the federal government’s so-called GAIN Act, a set of incentives to develop much-needed drugs to combat drug-resistant infections, which kill more than 23,000 people a year in the U.S. Sivextro is aimed at a group of bacteria that includes methicillin-resistant Staphylococcus aureus, more commonly known as MRSA.
—Diversified venture firm Polaris Partners filed SEC paperwork to indicate it is raising a new fund, its seventh. The target is $400 million, which is a few ticks higher than the $375 million fund Polaris closed in 2011.
—Mersana Therapeutics of Cambridge said Tuesday that EMD Serono, the US arm of German pharma Merck KGaA, will pay as much as $792 million to share technology and build antibody-drug conjugates (ADCs), although it was not disclosed how much Mersana is receiving upfront. ADCs attach cytotoxic, or tumor cell-killing, drugs to antibodies that act as homing devices and ferry the drugs directly to tumors. Drug companies have worked on the problem for decades, but only two ADCs to date are marketed in the US, one from Seattle Genetics, another from Roche’s Genentech group. (A third gained approval in 2000 but was yanked off the market in 2010.) Mersana specializes in toxic agents and the linkers—the chemical bonds—that attach the agents to antibodies.
—Dimension Therapeutics of Cambridge nailed down a $30 million round of venture funding from new investor OrbiMed Advisors and existing investor Fidelity Biosciences. The gene therapy developer also licensed its hemophilia A program to Bayer HealthCare. For $20 million immediately, Bayer gains worldwide rights to the program, taking over development if Dimension can advance it through Phase 1/2a trials. It is currently in preclinical development. If Bayer brings the therapy to market, Dimension could earn up to $232 million in payments, plus sales royalties.
—Boston’s Dana-Farber Cancer Institute will work with various groups within Johnson & Johnson (NYSE: JNJ) to assess immunotherapies as treatments for lung cancer. Dana-Farber’s Belfer Institute will contribute disease models, and the two groups will look for biomarkers and try to tease out the mechanisms behind drug resistance, a big problem in lung cancer. The collaboration is slated to last three years.
—Ohr Pharmaceutical (NASDAQ: OHRP) of New York released Tuesday interim Phase 2 data for its squalamine eye drops, a treatment for wet age-related macular degeneration (AMD). Both the patients receiving squalamine and those receiving placebo also received injections of the Genentech drug ranibizumab (Lucentis). Ohr is aiming to give patients with wet AMD a topical treatment that would boost eyesight and lower the needed frequency of Lucentis injections. The interim results helped with the former, but not the latter, and investors sold upon the news, cutting the price nearly in half the day after the data release to a low of $6.93 a share. The price rebounded to some extent Thursday, closing at $9.90 a share.
—Cambridge’s GNS Healthcare said Friday it has co-authored a study with insurance firm Aetna that shows deep computational analysis of patient records can predict future risk of metabolic syndrome, which is a catch-all phrase for the five risk factors that increase a person’s chances of heart disease, diabetes, and other serious problems. For the study, health-care analytics specialist GNS combed through the records of 37,000 Aetna patients. The study was published in the American Journal of Managed Care.
—Merrimack Pharmaceuticals (NASDAQ: MACK) of Cambridge announced Wednesday detailed Phase 3 results for its pancreatic cancer treatment MM-398. They were mixed, as we reported when the top-line results came out in May, but were good enough for Merrimack to put the drug, a reformulated version of the chemotherapy irinotecan (dubbed MM-398), on course for a marketing application this year as a combination therapy with 5-FU and leucovorin. Patients taking the combination had an overall survival rate of 6.1 months versus a 4.2 overall survival rate for patients on 5-FU and leucovorin alone.