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East Coast Biotech Roundup: CAR-T, Celgene, Aerie, ITCI, IPOs, & More

Xconomy Boston — 

A little tip for you non-New Yorkers: September is the city’s best month of the year. The summer humidity starts giving way to fall crispness, tourist levels dissipate, the beaches clear out, and baseball and football seasons are in full swing (parking is still impossible but, shame on you for trying to drive here). Just something to think about the next time you’re planning an East Coast trip. On to the biotech news!

—There is a lot of excitement around an emerging method of cellular immunotherapy known as “CAR-T.” But as with any new area of science, a lot of hurdles must be cleared before CAR-T can really make an impact on cancer care. Those challenges were the subject of a panel of experts at the inaugural International Cancer Immunotherapy Conference in New York in this past week. I recounted some of the problems brought up by the panel, and polled some CAR-T experts for their thoughts as well.

—Shares of New York-based Intra-Cellular Therapies (NASDAQ: ITCI) more than doubled after the company said that its experimental schizophrenia drug, ITI-007, succeeded in the first of two Phase 3 trials. ITI-007 bested a placebo in the study, and now its day of reckoning lies ahead—a second trial pitting the drug against mainstay schizophrenia drug risperidone (Risperdal) will produce data next year.

—Earlier this year, the FDA let Bedminster, NJ-based Aerie Pharmaceuticals (NASDAQ: AERI) change the goal of one of the Phase 3 trials for its experimental glaucoma drug, Rhopressa. That paid dividends this week after Aerie’s drug hit its revamped mark in the trial, sending shares soaring more than 60 percent. Aerie believes it’s on track to file an application for FDA approval next year, but there are still looming questions for it to answer regarding the drug’s side effects and durability.

—Summit, NJ-based Celgene (NASDAQ: CELG) inked two more deals this week. First, it paid $25 million to a Bay Area academic consortium working on antibody drugs for cancer. Then Celgene paid $150 million to enter into a deal with San Francisco startup Nurix, which is developing drugs that impact protein homeostasis. Celgene also took an equity stake in Nurix as part of the partnership and agreed to share rights to some of the drugs that come out of the collaboration. It’s the type of arrangement Celgene has done several times over with other biotechs like Forma Therapeutics and Agios Pharmaceuticals, among others.

—A few IPO developments on the gene therapy front this week. First, Cambridge, MA-based Dimension Therapeutics filed papers to go public and fund its work in gene therapy for hemophilia and rare metabolic diseases—an expected move following its $65 million Series B round. Then, RegenXBio, of Rockville, MD, raised $139 million in an upsized IPO when it sold 6.3 million shares at $22 apiece, beating its projected range. The two companies have one important tie in common: RegenX is based on the work of gene therapy pioneer Jim Wilson—who is also a Dimension founder and chairs its scientific advisory board.

—There’s just a week left until our latest biotech event, “Boston’s Life Science Disruptors,” which will take place on Sept. 30 at the Novartis Institutes for Biomedical Research in Cambridge, MA. This past week, I previewed the festivities, which will include an inside look at the ups and downs from three Boston-area biotechs: Seres Therapeutics (NASDAQ: MCRB), Foundation Medicine (NASDAQ: FMI), and Synlogic.

—A few weeks back, Turing Pharmaceuticals bought U.S. rights to a drug called pyrimethamine (Daraprim) that targets a parasitic infection called toxoplasmosis. According to reports from USA Today, Healio, and the New York Times this past weekend, Turing has since jacked up the price from $13.50 to $750 per tablet—the type of thing its CEO, Martin Shkreli, has come under fire for in the past with a drug known as tiopronin (Thiola)—and is now facing protests from non profits, patient groups, and experts in the field.

—An FDA advisory panel voted unanimously in favor of an extended-release oxycodone drug being developed by Canton, MA-based Collegium Pharmaceutical (NASDAQ: COLL). The FDA will decide whether to approve the drug, which would be sold as Xtampza ER, by Oct. 12.

Photo of the Queensboro Bridge courtesy of flickr user Matt Kane via Creative Commons.