East Coast Biotech Roundup: Cellectis, Bristol, What’s Hot & More

We’re going on a biotech road trip this week, from the Garden State to Beantown. Just after finding the venture backers for a $150 million life sciences fund, New York biotech got another jolt with the arrival of a high-profile French company. A startup in New Jersey grabbed over $70 million and looks poised for an IPO. A Connecticut biotech inked a $434 million deal with Merck. And then there’s our Boston biotech bash. There are plenty of pit stops in between, so let’s get right to it.

—On Wednesday we held our latest life sciences event, “What’s Hot in Boston Biotech,” at the Broad Institute of MIT and Harvard. Our speakers touched on the ups, downs, and looming issues facing a host of emerging (and re-emerging) areas of biotech innovation, from synthetic biology to gene therapy, cancer immunotherapy, and microbiome research. If you happened to miss it, you can catch my recap of the festivities here.

—In the latest step in gene therapy’s resurgence, New York-based Bristol-Myers Squibb (NYSE: [[ticker:BMY]]) formed a broad collaboration with Amsterdam’s UniQure (NASDAQ: [[ticker:QURE]]) to develop gene therapies for a variety of cardiovascular diseases, among them congestive heart failure. Bristol took a 4.9 percent stake in UniQure as part of the deal and could own as much as 19.9 percent of the Dutch company within the next few years as the alliance progresses.

—New York biotech got a boost this week as one of cellular immunotherapy’s major players, France’s Cellectis (NASDAQ: [[ticker:CLLS]]), established a U.S. headquarters in Manhattan at the Alexandria Center for Life Science. Cellectis will be cutting the ribbon on that new facility later this week.

—Berkeley Heights, NJ-based Edge Therapeutics said it closed two separate private financing rounds worth about $72.5 million. For the first time, Edge has identified its venture backers, among them Venrock and Sofinnova Ventures; it was previously only financed by an unspecified group of “high net worth individuals.”

—Alzheon raised a $10 million Series A led by Ally Bridge Group, a private equity investor based in Hong Kong. Alzheon, which also moved its headquarters to Framingham, MA, from Lexington, will use the cash to bring a revamped version of tramiprosate—an Alzheimer’s drug that failed a large study several years ago—to the brink of a Phase 3 trial for the memory-robbing disorder.

—Another gene editing startup has joined the fray in Boston, as London, U.K.-based CRISPR Therapeutics announced plans to establish an R&D hub in Cambridge. Newly named CRISPR chief scientific officer Bill Lundberg, a former Alexion Pharmaceuticals (NASDAQ: [[ticker:ALXN]]) executive, will lead the effort.

—Francois Nader, just a few months after steering NPS Pharmaceuticals to a $5.2 billion buyout from Shire, has become the chairman of Acceleron Pharma (NASDAQ: [[ticker:XLRN]]).

—Alkermes (NASDAQ: [[ticker:ALKS]]), based in Dublin and Waltham, MA, released more positive data from a Phase 2 test of schizophrenia drug prospect ALKS 3831. Alkermes aims to move the drug into Phase 3 testing later this year.

—The FDA granted an orphan drug designation to Lexington-based Curis’s (NASDAQ: [[ticker:CRIS]]) prospective lymphoma drug, CUDC-907, which is currently in Phase 1 trials.

—Cambridge-based Ariad Pharmaceuticals (NASDAQ: [[ticker:ARIA]]) said its leukemia drug ponatinib (Iclusig) was approved in Canada for certain types of patients with chronic myeloid leukemia or acute lymphoblastic leukemia who don’t respond to, or can’t tolerate, other therapies.

—-Shares of Bedford, MA-based Ocular Therapeutix (NASDAQ: [[ticker:OCUL]]) plummeted more than 20 percent after OTX-DP—an implantable device that delivers the steroid dexamethasone into the eye over a period of four weeks—failed a Phase 3 trial for pain after cataract surgery. This was the second of two Phase 3 trials for OTX-DP in post-surgical pain; Ocular said the first one hit its main goal in March. It blamed the poor results in the latest study on a “significantly higher than expected” placebo response.

—Biotech investor Brad Loncar has started the “Loncar Cancer Immunotherapy Index,” an index fund that tracks 25 biotech and pharmaceutical companies developing therapies that harness the immune system to fight cancer. You can read more about the fund in Forbes and TheStreet.com.

—Cambridge-based Raze Therapeutics named former Takeda oncology executive Mark Manfredi its chief scientific officer. Raze launched with a $24 million Series A from a group of venture firms and pharma companies in October.

—Two headlines from Kenilworth, NJ-based Merck (NYSE: [[ticker:MRK]]). First, it formed a potentially $434 million alliance with a New Haven, CT, startup called Arvynas, which is developing small molecule drugs that aim to disrupt proteins that have a role in promoting a variety of diseases.

—Merck also got two breakthrough therapy designations for a combination pill of two hepatitis C drugs—grazoprevir and elbasvir—for patients with chronic genotype 4 infection of hep C, and those with genotype 1 with kidney failure and on dialysis. The FDA rescinded breakthrough status for grazoprevir/elbasvir in genotype 1 patients in February.

Author: Ben Fidler

Ben is former Xconomy Deputy Editor, Biotechnology. He is a seasoned business journalist that comes to Xconomy after a nine-year stint at The Deal, where he covered corporate transactions in industries ranging from biotech to auto parts and gaming. Most recently, Ben was The Deal’s senior healthcare writer, focusing on acquisitions, venture financings, IPOs, partnerships and industry trends in the pharmaceutical, biotech, diagnostics and med tech spaces. Ben wrote features on creative biotech financing models, analyses of middle market and large cap buyouts, spin-offs and restructurings, and enterprise pieces on legal issues such as pay-for-delay agreements and the Affordable Care Act. Before switching to the healthcare beat, Ben was The Deal's senior bankruptcy reporter, covering the restructurings of the Texas Rangers, Phoenix Coyotes, GM, Delphi, Trump Entertainment Resorts and Blockbuster, among others. Ben has a bachelor’s degree in English from Binghamton University.