East Coast Biotech Roundup: NJ Blues, Genzyme, Amicus, Biogen & More

Xconomy Boston — 

The late summer doldrums are here, and unsurprisingly, biotech news slowed to a crawl this week. Before you know it, Labor Day will be here, the pigskins will be flying, and the newswire will be humming again. But until then, I’m headed for a short break. Enjoy the last days of summer, everyone. Here’s your roundup:

—Large pharmaceutical companies have been beating a path to Massachusetts over the past few years to join its bubbling life sciences cluster. And this week, GE Healthcare Life Sciences, a unit of the industry conglomerate General Electric (NYSE: GE), became the latest to make the move—dealing a blow to New Jersey’s life sciences scene in the process. GE Healthcare Life Sciences is shuttering its Piscataway, NJ, headquarters and a second facility in Princeton, and opening up a new 160,000-square-foot home in Marlborough, MA. The move will bring GE Healthcare Life Sciences, which makes tools used in diagnostics, imaging, and drug discovery, closer to a lot of its customers. But it will also lead to a number of job cuts in New Jersey. I spoke with GE Healthcare Life Sciences president and CEO Kieran Murphy about both sides of the story.

—What does it take to build an innovative biotech from scratch, persevere, and then convince public investors to buy into it? That’s the major theme of our upcoming Boston biotech event, “Boston’s Life Science Disruptors,” which will take place on Oct. 8 at the Novartis Institutes for Biomedical Research. We’ll hear from founding investors and leaders of three startups that have been able to navigate their way to some of the most successful recent biotech IPOs—Sage Therapeutics (NASDAQ: SAGE), Zafgen (NASDAQ: ZFGN), and Epizyme (NASDAQ: EPZM). You can grab discounted tickets to the event here if you register by Sep. 3.

—Biotech’s big winner this week was Cranbury, NJ-based Amicus Therapeutics (NASDAQ: FOLD), which revealed that its second Phase 3 study of its oral Fabry Disease drug migalastat met its goals and proved comparable to enzyme-replacement therapies in patients with certain specific genetic mutations. Migalastat succeeded in its first Phase 3 trial a few months ago, and with the new data in hand, will now make its case for the approval of its first drug. I spoke with CEO John Crowley and investigator Raphael Schiffmann about the results. As I wrote last week, it’s been a long time coming for Amicus, which has suffered through a number of setbacks before getting to this point. Shares jumped more than 20 percent on the news.

—Sanofi’s Cambridge, MA-based unit Genzyme won FDA approval of an oral pill for Gaucher Disease this week, called eliglustat (Cerdelga). Gaucher, like Fabry, is a disorder caused by mutations in the genetic instructions for important enzymes that help our cells clear out waste, such as extra fats and sugars. And as with Fabry, patients are typically treated with enzyme-replacement therapy, which requires IV infusions every few weeks and as its name suggests, replaces the faulty or missing enzymes with synthetic ones. Genzyme, in fact, sells this therapy, imiglucerase, under the brand name Cerezyme. Eliglustat, on the other hand, suppresses the production of the excess fats that build up in the cells of Gaucher patients. Genzyme said the treatment, which is approved for the most common form of Gaucher, should be available in about a month. The drug will likely be priced on par with enzyme-replacement therapy, which costs about $300,000 annually, according to a report from Bloomberg.

—The FDA this week also approved Biogen Idec’s (NASDAQ: BIIB) latest multiple sclerosis drug, peginterferon beta-1a (Plegridy). The therapy is an improved form of Biogen’s flagship multiple sclerosis treatment, Avonex, in that patients can self-administer subcutaneous injections of it every two weeks, instead of weekly. The drug is already approved in Europe.

—A number of companies have been teaming up to play the immuno-oncology combination trial game this year, aiming to find the best possible pairing of cancer drugs to take forward. New York-based Bristol-Myers Squibb (NYSE: BMY), for instance, has already cut a few deals to pair its cancer immunotherapy drug nivolumab with other drug prospects in clinical trials, like Celldex Therapeutics’ (NASDAQ: CLDX) varlilumab. This week, Bristol joined up with Summit, NJ-based Celgene (NASDAQ: CELG) to test nivolumab in tandem with the New Jersey company’s approved cancer drug, protein-bound paclitaxel (Abraxane); the companies are running a Phase 1 trial in multiple tumor types like pancreatic cancer, HER-2 negative metastatic breast cancer, and non-small cell lung cancer. The study will begin in the fourth quarter of this year.

—Pfizer (NYSE: PFE) this week filed a new drug application with the FDA for its closely-watched developmental breast cancer drug, palbociclib, earlier than expected. The drug is an inhibitor of the enzymes cyclin-dependent kinases 4 and 6 (CDK4/6), and Pfizer has taken data to the FDA after a successful Phase 2 study. Palbociclib is one of several CDK4/6 inhibitors in development, such as Eli Lilly’s abemaciclib and Novartis’s LEE011.