East Coast Biotech Roundup: Emulate, Accelerator, Retrophin, & More

Xconomy Boston — 

A new high-profile startup, an evolving biotech soap opera, and a long-awaited new entrant to New York City’s life sciences scene dot the roundup this week—all as we say goodbye to a volatile July and Thursday’s market carnage, which saw many biotech indexes plummet. Those stories and more below:

—After about five years of incubation at Harvard University’s Wyss Institute and some $40 million in grant dollars, Cambridge, MA-based Emulate ramped up this week with a $12 million Series A round from venture firm NanoDimension, Cedars-Sinai Medical Center, and Swiss billionaire Hansjorg Wyss (the founder of the Wyss Institute). Emulate, which plans to move into Kendall Square, is developing what it claims is the most comprehensive “organ-on-a-chip” system created to date, aiming to prove that its technology can be used to supplant current preclinical testing methods—such as in vitro or animal tests. Still, Emulate is by no means the only company with such technology, and there’ll be a long road ahead to create a successful business out of it. I spoke with CEO James Coon about the effort.

—Seattle’s Accelerator has been talking about expanding and opening up an office in New York City for some time, and it finally did so this week, announcing that it’s raised $51.1 million from a group of venture and industry players and christened a new outlet in the Alexandria Center for Life Science on Manhattan’s East Side. Returning investors Alexandria Venture Investments, Arch Venture Partners, and WRF Capital contributed to the round, as did the VC arms of three pharma companies—Pfizer, Johnson & Johnson, and Eli Lilly—as well as the Partnership Fund for New York City and Harris & Harris Group. Accelerator, started up in 2003 by Leroy Hood and others, helps build early-stage biotechs—and will try to do so in a city where it’s notoriously difficult due to the cost.

—New York-based Retrophin (NASDAQ: RTRX) was one of the investors in the recent $2 million seed round for the Perlstein Lab, Ethan Perlstein’s previously boostrapped effort to unearth treatments for ultra-rare diseases. Perlstein, who got his doctorate from Stuart Schreiber’s Harvard University lab and did some post-doctoral work at Princeton University, is taking an unorthodox route to biotech entrepreneurship—he moved to San Francisco and with the help of social networking tools, built his own lab, unaffiliated with a school, company, or other institution. Retrophin’s participation, for instance, dates back to a twitter exchange between CEO Martin Shkreli and Perlstein in January. Alex Lash has the details here.

—Cambridge-based Sarepta Therapeutics (NASDAQ: SRPT) was already a controversial stock when the big story surrounding the company was whether the FDA would approve its Duchenne Muscular Dystrophy drug, eteplirsen, off a small trial. But over the past few weeks, drama spilled into Sarepta’s boardroom. First, Sarepta abruptly fired chief scientific officer Art Krieg, a veteran of RNA drug development who’d only been hired in January—something sources at the time claimed had to do with a lack of progress in drug discovery activities. Then, the Wall Street Journal published a few articles painting a picture of boardroom dissension within Sarepta. Krieg, for one, was quoted as having “serious disagreements” with CEO Chris Garabedian in one report. In another, the Journal, citing unnamed sources, reported that Sarepta chairman William Goolsbee recently said that Garabedian wouldn’t attend any more of the company’s FDA meetings. Sarepta didn’t refute the report, but on Thursday Goolsbee resigned—replaced by interim chairman John Hodgman—and Sarepta said that the board “fully supports” Garabedian. (Goolsbee will remain an independent director). The whole soap opera comes at a critical time for Sarepta. It’s preparing to file a new drug application for eteplirsen with the FDA.

—New York-based Regeneron Pharmaceuticals (NASDAQ: REGN) had an active week. First, it won FDA approval to begin selling aflibercept (Eylea) as a treatment for diabetic macular edema. Next, Regeneron and partner Sanofi posted the results from their final Phase 3 study of cardiovascular drug alirocumab, and said they’d file an application with the FDA by the end of the year. Then, the companies said they’d bought a “priority review” voucher from BioMarin Pharmaceutical for $67.5 million, enabling the two companies to cut down the FDA’s review of alirocumab as they attempt to catch up with a rival drug Amgen is developing. Separately, Regeneron is also one of the big investors in Menlo Park, CA-based Avalanche Biotechnologies (NASDAQ: AAVL), which raised $102 million in an IPO this week and then rocketed more than 60 percent in its first day of trading.

—Former Pfizer general counsel Amy Schulman this week was named a venture partner at Boston’s Polaris Partners. She’s also become the CEO of Arsia Therapeutics, a Polaris seedling startup co-founded by Robert Langer that’s developing a technology to deliver antibody drugs via subcutaneous injections, rather than through intravenous infusions. The startup is currently housed in LabCentral, the Kendall Square biotech incubator that launched in November. Schulman, meanwhile, will serve on the boards of a number of other Polaris companies. She’s already on the board of Alnylam Pharmaceuticals (NASDAQ: ALNY).

—Waltham, MA-based Kala Pharmaceuticals started a Phase 2 trial for KP-121, its amped up formulation of the corticosteroid drug loteprednol etabonate (Lotemax), in patients with diabetic macular edema, and a few other ophthalmic diseases. Kala is trying to prove that its drug delivery technology enables it to make eye drops that are either better than existing therapies, or for middle- or back-of-the-eye disorders, which typically require injectable drugs to treat. Kala recently began testing KP-121 in a Phase 3 study for the inflammation people get after cataract surgery, and a mid-stage trial for dry eye disease.