Bio Roundup: Big Apple Mo’, Checkpoint Yes and No, CAR-T Death & More

Xconomy National — 

“New York, New York, it’s a hell of a town. The Bronx is up, but we’re Brooklyn down.”

For all you old-school Brooklyn swingers out there, we’ll take a short break from the madness in the nation’s capital and start our roundup in the greatest city in the world.

You might disagree if you’ve got biotech dreams. To little avail, New York has long tried to turn its financial prowess, population density, and deep biomedical bench into a hub to rival Boston and San Francisco. Is a ton of new government money — $1.15 billion promised in recent years —changing the momentum? Xconomy deputy biotech editor Ben Fidler is based in New York and keeps his finger on the city’s biotech pulse. At this week’s annual NewYorkBio conference, he quizzed attendees about the local sector’s progress in 2017. If you’ve got a New York minute to spare, read his report. Then let’s get to the roundup.


—Investors who bet that a once-stalled Roche cancer drug could become a treatment for eye disease were rewarded when Horizon Pharma (NASDAQ: HZNP) paid $145 million up front for New York startup River Vision Development.

—According to a letter obtained by Endpoints, Turing Pharmaceuticals is weighing a $100 million cash offer for daraprim, the generic drug that made headlines after the New York company raised its price by more than 5,000 percent.

—When it was run by “pharmabro” Martin Shkreli, Turing stoked the U.S. political firestorm around drug prices. Companies have since tried to prove their good citizenship. The latest is Sanofi (NYSE: SNY), which pledged this week to keep its price increases below the annual inflation rate of healthcare costs.

—Then again, if this makes it to Broadway, Shkreli–one of New York hip-hop’s oddest fanboys—might have the last laugh.


—A Genentech drug that was granted accelerated FDA approval last year to treat the most common form of bladder cancer failed a Phase 3 clinical trial in patients with a more advanced form of the disease.

—CAR-T cell therapy developer Kite Pharma (NASDAQ: KITE) of Santa Monica, CA, revealed that a patient died last month from cerebral edema, or severe brain swelling, the first reported case among hundreds of patients treated with Kite’s most advanced product, KTE-C19. The FDA is currently reviewing KTE-C19 for approval in non-Hodgkin lymphoma. Officials said the single case would not delay other studies. A cluster of similar deaths last year spelled doom for the top CAR-T product of Kite’s rival Juno Therapeutics (NASDAQ: JUNO).

—Immunotherapy leader Merck (NYSE: MRK) notched FDA approval of its pembrolizumab (Keytruda) combined with chemotherapy to treat patients with previously untreated metastatic non-small cell lung cancer whether or not their tumors have the telltale protein PD-L1. It’s an expansion of pembrolizumab’s use in lung cancer. Last year, the FDA said it could be used on its own but only for cancers that use PD-L1 to evade the immune system.


—National Institutes of Health director Francis Collins said the Trump administration’s proposed $5.8 billion NIH budget cut was “not the main focus” of a White House meeting that gathered Collins, academia and industry life sciences leaders, and administration officials.

—The law that lets the FDA fund its drug-review operations through fees charged to industry is a step closer to reauthorization. A key Senate committee voted 21-2 to re-up the current “PDUFA,” which expires in September. As happens every five years, members of Congress have attached various FDA reforms to the reauthorization bill.

—Scott Gottlieb, who worked as a deputy FDA commissioner during President George W. Bush’s presidency, is headed back to the agency as commissioner after the U.S. Senate confirmed his nomination by a 57 to 42 vote.

—A former Beltway scientist was in the news. Former National Institute of Mental Health director Thomas Insel has left Verily Life Sciences after less than two years. Insel jumped in 2015 to South San Francisco-based Verily, formerly known as Google Life Sciences. CNBC first reported the news of Insel’s departure.

—More people on the move: Owen Wallace is leaving Novartis (NYSE: NVS), where he was head of the global discovery chemistry division, to become chief scientific officer of Cambridge, MA-based Fulcrum Therapeutics… Verge Genomics of San Francisco, led by 28-year-old Alice Zhang, has hired neuroscience drug veteran Tony Altar as chief scientific officer.


—PTC Therapeutics’s (NASDAQ: PTCT) announcement of a $35,000 net price for its Duchenne muscular dystrophy drug delfazacort led some analysts to speculate that the South Plainfield, NJ-based biotech would face insurance company pushback on the cost.

—A settlement between Morris Plains, NJ, biotech Immunomedics (NASDAQ: IMMU) and activist investment firm venBio, its largest shareholder, unwound a deal to license the company’s lead drug to Bothell, WA-based Seattle Genetics (NASDAQ: SGEN). Immunomedics’ new management team will now try to win accelerated FDA approval for the drug in triple negative breast cancer.

—Pfizer (NYSE: PFE) is paying Sangamo Biosciences (NASDAQ: [[SGMO]]) $70 million upfront for rights to a gene therapy for hemophilia A.

—Dell Technologies Capital led a $22 million investment in Edico Genome, a San Diego maker of hardware and software that speeds up the process of genome mapping.

—An OncoMed Pharmaceuticals (NASDAQ: OMED) drug aimed at non-small cell lung cancer failed a Phase 2 study, marking the third clinical failure in the last month for the Redwood City, CA-based biotech. The failure comes two weeks after OncoMed announced it would lay off half of its staff.

Ben Fidler and Frank Vinluan contributed to this report.

Photo of the Queensboro Bridge by Matt Kane via Creative Commons.