East Coast Biotech Roundup: Regeneron, Sarepta, Jounce, & More
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establish it as a treatment for other inflammatory disorders like psoriasis. Celgene, for its part, said that it still hasn’t given up in ankylosing spondylitis, pointing to a subgroup of patients who responded better than others. The company may run another Phase 3 trial after it collects more data.
—Newton, MA-based AesRx became a notable win for the National Institutes of Health’s National Center for Advancing Translational Sciences this week when it was acquired, for an undisclosed sum, by Baxter International (NYSE: BAX). AesRx was formed in 2008 by Stephen Seiler, who scooped up a sickle cell disease prospect from a now-defunct New Jersey company called Xechem and kept it on life support until he formed a key partnership with the NIH’s NCATS group in 2010. That deal enabled AesRx to nurture its drug prospect, now called AES-103, through the “Valley of Death”—a translational gap where projects can’t get the funding they need to become opportunities that venture firms or drugmakers want to advance—and ultimately into the hands of Baxter. NCATS director Christopher Austin noted in a statement that the deal “validates the NCATS model.” AES-103 is now in Phase 2 testing.
—Newly-named, Cambridge-based Akashi Therapeutics (formerly Dart Therapeutics), a startup founded by a group of disease foundations, presented the first clinical data on its prospective Duchenne Muscular Dystrophy treatment, HT-100. The drug candidate is meant to help remedy the fibrosis and inflammation associated with Duchenne. Akashi has received an orphan drug and fast-track designation from the FDA for HT-100.
—Capitalizing on the recent clinical success of one of its gene therapies, Cambridge-based Bluebird Bio (NASDAQ: BLUE) cashed in this week, raising about $95.6 million by pricing 3 million shares at $34.00 apiece. It’ll primarily use the cash for clinical studies of its gene therapy programs, like Lenti-Globin for beta-thalassemia and sickle cell disease, and Lenti-D for childhood cerebral adrenoleukodystrophy.
—Merck will likely be the first company to bring a PD-1 checkpoint inhibitor cancer treatment to market in the U.S., but New York-based Bristol-Myers Squibb may not be that far behind. Bristol announced this week that it plans to file an application with the FDA in the third quarter to approve nivolumab, its big cancer immunotherapy prospect, as a melanoma treatment. Nivolumab was approved in Japan earlier this week as well. Bristol also aims to file an application in lung cancer by the end of the year, and is testing nivolumab in a variety of other cancers as well.
—Long Island’s Cold Spring Harbor Laboratory got a big $50 million gift from Jim and Marilyn Simons to establish the Simons Center for Quantitative Biology, which will bring together experts in applied mathematics, computer science, physics, and engineering to advance research into diseases like autism, cancer, bipolar disorder, and depression. Cornell University professor Adam Siepel will leave his post to chair the Simons Center starting in September. Jim Simons is the billionaire mathematician and hedge fund manager who founded Renaissance Technologies. Marilyn Simons heads the Simons Foundation, a financier of basic scientific research.
—Fresh off its $96 million IPO, Cambridge-based Zafgen (NASDAQ: ZFGN) was awarded an orphan drug designation by the European Commission for its prospective fat-burning drug, beloranib, as a treatment for Prader-Willi Syndrome. Zafgen has already received that designation from the FDA. Zafgen has said it plans to use its IPO cash partly to fund a late-stage trial in Prader-Willi, a rare genetic disorder that causes severe overeating.