Bio Roundup: Fast “Cures,” Trump In Time, CRISPR In Court & More

Xconomy National — 

What a week. The 21st Century Cures Act is now waiting President Obama’s promised signature after easy passage through Congress. President-elect Donald Trump re-stirred the drug-pricing pot with comments in Time Magazine and a reported FDA commissioner candidate who doesn’t seem to think much of the regulatory process. And a trio of judges met to hear brief—can we say edited?—arguments from both sides of the epic CRISPR patent fight.

We’ll start with the national news, check in with news from a big Alzheimer’s meeting, then dig into plenty more about the annual American Society of Hematology conference, surprising M&A, fundings, and famous genome guy Craig Venter, never far from the headlines. It’s roundup time….


—The 21st Century Cures Act, a vast biomedical bill, breezed through Congress and is poised to become law. It includes new NIH and FDA funding with uncertain guarantees, controversial regulatory reforms that critics say would unsafely lower the bar for approval, and more.

—The good cheer among biopharma backers for the Cures Act was complicated by two bits of news from Trumpland. The President-elect told Time magazine that he would bring down prescription drug costs, without offering any concrete plans. And Bloomberg reported that hedge-fund libertarian Jim O’Neill, an associate of Trump backer Peter Thiel, is under consideration for FDA commissioner. O’Neill has no medical background and has endorsed changing FDA rules to require only safety testing for approval. “Let’s prove efficacy after they’ve been legalized,” he told a 2014 conference. He is also a proponent of establishing new societies on floating ocean platforms out of the reach of national governments.

—Three federal patent judges gave lawyers for The Broad Institute of MIT and Harvard and the University of California, Berkeley—the two sides of the CRISPR gene editing patent fight—20 minutes each to present their cases in a long-awaited public hearing Tuesday. The Scientist has a summary of the hearing here. It’s not clear when the judges will decide the case.

—Late Thursday, data trickled in on experimental Alzheimer’s drugs solanezumab (from Eli Lilly) and aducanumab (from Biogen) at a medical meeting in San Diego. In solanezumab’s case, more details from Lilly’s failed Phase 3 trial affirmed the small therapeutic signal Lilly reported previously. As for aducanumab, more data from Biogen’s Phase 1b study continued to support the early promise the drug has shown so far.


—The American Society of Hematology’s annual meeting provided updates on a group of experimental treatments for hemophilia, the progress of immunotherapy “checkpoint” inhibitors in blood cancers, and much more. Xconomy rounded up many of the stories and put the meeting in context.

—Closely watched Juno Therapeutics (NASDAQ: JUNO), knocked back by 5 patient deaths and a second suspension of its lead product candidate JCAR015, reported results at ASH from other parts of its CAR-T therapy pipeline.

—Gene therapies for hemophilia continue to show promise, but they might not work for everybody. At ASH, Spark Therapeutics (NASDAQ: ONCE) reported that two patients taking its experimental therapy developed an immune reaction that stifled the treatment. Spark also announced a license of technology from Selecta Biosciences (NASDAQ: SELB) that could help with the problem.

—Xconomy’s running resource of cell therapy trials that have reported patient outcomes got several ASH updates.


The Wall Street Journal detailed ups and downs in the long-running alliance between French drug maker Sanofi and Boston startup Warp Drive Bio. Xconomy explained in this article why Warp Drive scientists think they’ve found the answer to making drugs to fight cancers driven by mutations in the RAS gene family.

—Months after passing on an option to buy all of Acetylon Pharmaceuticals, Celgene (NASDAQ: CELG) struck a new deal to acquire only part of the Boston startup. The rest will be spun into a new company called Regenacy Pharmaceuticals. Xconomy spoke to CEO Walter Ogier about Regenacy’s plans and the rationale behind the deal.


—Early clinical data from Cambridge, MA-based Voyager Therapeutics (NASDAQ: VYGR) hinted that its experimental gene therapy for Parkinson’s disease, VY-AADC01, might help patients no longer responding to levodopa—a problem many Parkinson’s patients deal with over time. Shares of Voyager surged 35 percent.

—Seattle-based Cascadian Therapeutics (NASDAQ: CASC), a biotech formerly known as Oncothyreon, reported early clinical data on its drug ONT-380 for breast cancer patients whose tumors have spread to the brain. Forbes and have more here.

—Cambridge-based Sage Therapeutics (NASDAQ: SAGE) said the FDA has signed off on a speedy path towards potential approval for SAGE-547, an experimental treatment for postpartum depression. Sage released Phase 2 data for SAGE-547 in PPD over the summer. Data from the Phase 3 trial are expected next year.


—Geneticist J. Craig Venter, famous for leading one of the two teams to decode the human genome and for having his own genome published, told a conference crowd that he had undergone prostate cancer surgery three weeks before. The elite health clinic he runs, Health Nucleus, had detected the cancer.

—The former head of Novartis’ now-disbanded gene and cell therapy unit, Uzman Ozam, was named the CEO of Philadelphia startup, Tmunity Therapeutics, which was founded by cell therapy pioneer Carl June.


—Days after releasing promising early data for a cell therapy for multiple myeloma and seeing its shares rise more than 16 percent, Bluebird Bio (NASDAQ: BLUE) sold nearly $250 million in stock.

—Tenaya Therapeutics spun out of the San Francisco-based Gladstone Institutes with a $50 million Series A round. It plans to develop regenerative medicine for heart failure.

—Gurnet Point Capital, the healthcare fund managed by ex-Sanofi CEO Chris Viehbacher, put $92 million into Aliso Viejo, CA-based Crossover Health. The startup works with self-insured employers to provide healthcare and related services.

—New York-based Elysium Health raised a $20 million Series B to roll out its first product, a supplement that Elysium bills as a way to “support the long term health of your cells,” and to fund research into the science of aging.

Ben Fidler contributed to this report