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East Coast Biotech Roundup: Celldex, Ensemble, CRISPR Battle & More

Xconomy Boston — 

Biotech indices had rallied over the past week, but that’s before the drug pricing issue returned to the spotlight in Washington. The Centers for Medicare & Medicaid Services, as the New York Times wrote here, proposed new ideas to change the reimbursement for prescription drugs under Part B of Medicare—infused and injectable drugs, like chemotherapies and many treatments for rheumatoid arthritis that are administered in hospitals and doctors’ offices. The idea is to find ways to reduce the spending on Medicare Part B, which totaled $20 billion last year.

Even as sell-side analysts tried to allay investors’ concerns—RBC Capital Markets’ Michael Yee wrote that the proposed changes don’t “really impact the utilization of most biotech drugs”—it put another scare into biotech. The Nasdaq Biotechnology Index (NASDAQ: IBB) promptly fell almost 5 percent in the two days following the CMS announcement, squelching the recent rally.

Meanwhile on the East Coast, a closely watched cancer vaccine received a disappointing result, and biotech’s most high-profile patent fight continued apace. Those stories and more below.

—Shares of Hampton, NJ, and Needham, MA-based Celldex Therapeutics (NASDAQ: CLDX) fell more than 50 percent this week after its cancer vaccine for glioblastoma, rindopepimut (Rintega), failed a Phase 3 trial. Celldex stopped the study early after an independent monitoring board deemed that rindopepimut wouldn’t succeed—patients on the drug lived for a median time of 20.4 months, less than the 21.1 month median of those on a placebo. Rindopepimut’s future is now very much in doubt, leaving Celldex to rally around a group of other cancer drugs in earlier-stage testing.

—The patent fight between two groups in Boston and San Francisco over the gene editing system CRISPR-Cas9 heated up as lawyers for both sides filed key documents that could help shape the proceedings. In this piece, Science explains the revelations to come forth and the next steps in the case. Check out Alex Lash’s extensive coverage of the CRISPR patent dispute and the important role the overhaul of American patent law in 2011 will play in the proceedings.

—Cambridge, MA-based Ensemble Therapeutics is one of the rare biotech startups that’s been able to survive more than eight years without a big funding round, thanks to a string of drug discovery deals. But Ensemble’s bills are about to escalate, because it has quietly been building an in-house pipeline of cancer drugs, the first of which could begin human clinical testing next year. I spoke with CEO John Ripple, a longtime biotech veteran, about the plan and challenges ahead for Ensemble.

—Cambridge-based Sarepta Therapeutics (NASDAQ: SRPT) announced plans to move all of its operations to Massachusetts and close its facility in Corvallis, OR, in the process. FierceBiotech reported that 30 employees will lose their jobs amidst the reshuffling, which Sarepta expects to complete by Dec. 30. The FDA on Thursday also penciled in a new date for the advisory panel on Sarepta’s Duchenne muscular dystrophy drug, eteplirsen—it’ll take place on April 25.

—Cambridge-based Akebia Therapeutics (NASDAQ: AKBA) won a patent suit against rival Fibrogen (NASDAQ: FGEN) in Europe related to drugs the two companies are developing for anemia. CEO John Butler said in a statement that the company will now look for a partner in Europe for its prospective anemia drug, vadadustat, to complement the $350 million deal it has already signed with Mitsubishi Tanabe in various Asian countries.

—Cambridge-based Eleven Biotherapeutics (NASDAQ: EBIO) received a notice that its shares could be delisted from the Nasdaq if they don’t close above $1 apiece for at least ten days by Aug. 30. Eleven’s shares currently trade at just $0.33 apiece; the company shelved its lead drug, isunakinra, after it failed the second of two late-stage trials in January.

—Be sure to check out the agenda for our upcoming March 29 New York biotech event, “New York’s Life Science Disruptors,” which we posted last week.

—Boston-based Vertex Pharmaceuticals (NASDAQ: VRTX) continued its rollout of the combination cystic fibrosis treatment ivacaftor/lumacaftor (Orkambi), winning approval to sell the drug in Australia. Ivacaftor/lumacaftor is already approved in the U.S. and Europe.

—Tewksbury, MA-based medtech startup Corvia Medical, which has a transcatheter device to treat heart failure, has found a potential acquirer. The company said it has signed an exclusive “option-to-purchase” deal with an unnamed strategic partner. Corvia didn’t disclose the terms or much else about the agreement—like what would trigger the buyout—but it was just cleared by the FDA to run a randomized, controlled study testing its product. Corvia is backed by Third Rock Ventures, General Catalyst Partners, AccelMed, and Lumira Capital.

—Waltham, MA-based EyeGate Pharma (NASDAQ: EYEG) acquired Jade Therapeutics, a Salt Lake City company with hydrogel-based technology that can be used to help deliver drugs to the eye. Eyegate will repay $300,000 in Jade debt and issue its backers 765,728 shares of stock. If a Jade product wins FDA approval, EyeGate would also shell out about $2.2 million in cash.

—Tom Daniel, Celgene’s head of research, joined Zafgen’s (NASDAQ: ZFGN) board of directors.

Photo of Spring in Central Park courtesy of flickr user Shinya Suzuki via Creative Commons.