Bio Roundup: CAR-T & Migraine Races, Intra-Cellular, Rockefeller & More

Xconomy National — 

The presidential race captivated millions of TV viewers this week as the first debate took place in Hofstra. But biotech watchers had other races on their mind. Who will be first to market with a new type of cancer treatment known as CAR-T? Who will break free of the pack of companies developing drugs meant to prevent migraines? As with the election, both races are heading down the homestretch. The latest updates, and the rest of this week’s biotech headlines, are below.


—Kite Pharma (NASDAQ: KITE) released data from an ongoing non-hodgkin lymphoma trial that, while early, bolster the view that the Santa Monica, CA, company could be the first to market with a cell-based immunotherapy product known as CAR-T.

—Amgen (NASDAQ: AMGN), of Thousand Oaks, CA, and partner Novartis released positive results from the first Phase 3 trial of erenumab, one of a group of drugs being developed to prevent migraines, not just lessen their impact when they happen. That keeps Amgen ahead of two other groups—Teva Pharmaceutical (NYSE: TEVA) and a collaboration between Alder Biopharmaceuticals (NASDAQ: ALDR) and Eli Lilly (NYSE: LLY)—developing similar migraine drugs, known as CGRP antibodies.

—Amgen also disclosed that carfilzomib (Kyprolis), the drug it acquired in a $10 billion buyout of Onyx Pharmaceuticals a few years ago, failed in a head-to-head study against mainstay multiple myeloma drug bortezomib (Velcade). has more here.


—Xconomy’s Alex Lash sat down with Cell Design Labs co-founder Wendell Lim to talk about and his work with colleagues at University of California, San Francisco, building “cellbots,” programmable T cells that can sense when they’re near a tumor, and produce drugs on the spot to kill only tumor cells.

—A leadership void was created for New York biotech when Marc Tessier-Lavigne left Rockefeller University to run Stanford. I spoke with new Rockefeller president Richard Lifton, the former genetics chief at Yale University, about his plans to engage the local life sciences scene.

—Xconomy’s Jeff Buchanan reported from a panel at BioForward in Wisconsin, where biotech executives discussed the implications of last week’s controversial approval of Sarepta Therapeutics’ (NASDAQ: SRPT) eteplirsen (Exondys 51) for Duchenne muscular dystrophy.

—Caribou Biosciences president, CEO and co-founder Rachel Hurwitz spoke at the University of Texas Health Science Center at San Antonio about the many challenges she faced trying to form Caribou, one of a group of companies using CRISPR-Cas9 gene editing technology.


—Shares of Gilead Sciences (NASDAQ: GILD) have dropped to lows the Foster City, CA, company hasn’t seen in two years as the market for its blockbuster hepatitis C drugs sofosbuvir (Sovaldi) and sofosbuvir/ledipasvir (Harvoni) continues to wane. In a research note this week, Leerink Partners analyst Geoffrey Porges wrote that hep C is “turning out to be a ‘flash in the pan’ market” and questioned Gilead’s lack of speed in addressing its revenue decline.

—Shares of Boulder, CO-based Array BioPharma (NASDAQ: ARRY) surged more than 80 percent after experimental cancer drug encorafenib succeeded in an Phase 3 study in skin cancer.

—Shares of New York-based Intra-Cellular Therapies (NASDAQ: ITCI) plummeted almost 70 percent after its experimental schizophrenia drug came up short in the second of two Phase 3 trials. Intra-Cellular still aims to talk to regulators about potential FDA approval despite the setback.

—Sarepta inked a research pact with Catabasis Pharmaceuticals (NASDAQ: CATB) to develop a combination therapy for Duchenne. Shares of Catabasis surged 43 percent.


—Amgen teamed with RNA interference drugmaker Arrowhead Pharmaceuticals (NASDAQ: ARWR) to develop two treatments for cardiovascular disease.

—By exercising a $20 million option, Summit, NJ-based Celgene (NASDAQ: CELG) got rights to ABX-1431, a potential treatment for multiple sclerosis developed by Abide Therapeutics.

—Cambridge-based Bluebird Bio (NASDAQ: BLUE) will work with Germany’s Medigene to develop T cell-based immunotherapy products for cancer. Bluebird paid Medigene $15 million up front, and could shell out over $1 billion total if a variety of milestones are hit.

—Boston-based SQZ Biotech raised a $16 million Series B round to develop a broad range of cell therapies for cancer, and possibly infectious and autoimmune diseases as well.

—Avalon Ventures led an $18 million Series A round for new cancer immunotherapy startup Fortis Therapeutics.


—Cambridge-based Genocea Biosciences (NASDAQ: GNCA) said an early look at the latest study of its experimental genital herpes vaccine yielded positive results, but questions still remain for the company to answer going forward. Phase 3 trials should start next year.

—Waltham, MA-based ImmunoGen (NASDAQ: IMGN) will ax 65 employees (17 percent of its workforce) and seek to partner off some of its drug candidates as part of a restructuring.