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Bio Roundup: Trump Budget, FDA Chief, CAR-T Qs, CRISPR Cash, & More

Xconomy National — 

If it wasn’t clear that the Trump administration disdained most types of scientific inquiry and practice, the White House’s proposed budget drove the point home. The top-line figures: a nearly 20 percent cut in the National Institutes of Health, and a 30 percent cut in the Environmental Protection Agency. These and other cuts to programs for foreign aid and the poor prompted this tweet from Sue Desmond-Hellman, the head of the Bill and Melinda Gates Foundation.

And these thoughts, via e-mail, from Robert Darnell, the founding director of the New York Genome Center: “The proposed Trump NIH budget cuts expose, at their root, a fundamental lack of appreciation of the value of investing in science and discovery.”

Details of the cuts at NIH, which funds biomedical research across the country as well as within its own walls, were scant. The White House hinted of a major reorganization, which would be nothing new for the sprawling agency. But the two changes mentioned in the budget—eliminating the global-health focused Fogarty Center and moving the FDA’s health-care quality group into NIH—do “not bode well for the Administration’s reorganization plan,” said Ellie Dehoney, vice president of policy and advocacy at Research!America, a nonprofit advocate for science research.

“Global health research tends to get short shrift, despite the fact that not only do some of the most profound threats to American lives emerge in other countries, but working collaboratively with the global community is a cost-efficient approach to medical progress,” Dehoney wrote in an e-mail.

One big question now: Which Republicans, who control Congress, will stand up for science funding? So far, there has been no Arlen Specter for our era—a moderate Republican senator who made sure the NIH got a $10 billion bump from President Obama’s 2009 recession stimulus package.

There were plenty of other health and life science related stories this week from Washington and around the country, so let’s get to the roundup.

—Late last Friday, the Trump administration nominated Scott Gottlieb to be FDA commissioner. Biopharma breathed a sigh of relief; Gottlieb, a physician, venture capitalist, and former FDA deputy commissioner, was considered least likely among the floated candidates to cause drastic upheaval.

—Seema Verma, a longtime healthcare consultant in Indiana, was sworn in to run the Centers for Medicare and Medicaid Services. She and Health and Human Services secretary Tom Price immediately issued a letter to all 50 state governors that called Medicaid’s expansion under the Affordable Care Act a “clear departure from the core, historic mission of the program.”

—Price also questioned the idea behind a House bill that would let employers require genetic testing of their employees as part of “workplace wellness” programs.

CANCER NEWS AND VIEWS

—Xconomy examined the current state of CAR-T therapies, which use live modified T cells to fight cancer. The first-ever CAR-T approvals could come this year, but the field faces safety and affordability obstacles that could prevent use beyond small patient populations.

—The FDA approved Merck’s pembrolizumab (Keytruda) for classical Hodgkin’s lymphoma patients who have failed at least three treatments. Bristol-Myers Squibb’s rival nivolumab (Opdivo) is also approved for cHL patients who have failed multiple prior therapies.

—Shares of Waltham, MA-based Tesaro (NASDAQ: TSRO) slid 11 percent after AstraZeneca released Phase 3 data for a “PARP” blocking cancer drug, olaparib (Lynparza), that rivaled Tesaro’s own niraparib in ovarian cancer. TheStreet.com has more here.

—The FDA approved ribociclib (Kisqali) for previously untreated HR-positive/HER2-negative advanced breast cancer, and owner Novartis said it would put in place a flexible pricing scheme.

TRIAL TRIBULATIONS

—Following the failure of its experimental microbiome drug last summer, Seres Therapeutics (NASDAQ: MCRB) revealed its plan to try again—this time using a different diagnostic, a higher dose, and a larger sample of patients.

—Waltham, MA-based Proteon Therapeutics (NASDAQ: PRTO) is trying to overcome the failure of one study of its blood vessel drug vonapanitase with a larger trial that has a modified goal.

—Cempra (NASDAQ: CEMP) retained Morgan Stanley to explore “strategic options.” The Chapel Hill, NC-based antibiotics developer recently cut 67 percent of its staff after its lead drug failed to win FDA approval in pneumonia, then failed in a separate clinical trial for gonorrhea.

—AstraZeneca’s hyperkalemia drug, ZS-9—acquired in its $2.7 billion buyout of ZS Pharma in 2014—was rejected by the FDA for a second time due to manufacturing problems.

CRISPR AND OTHER DEALS

—Allergan (NYSE: AGN) is the latest large biopharma company to place a bet—albeit a small one—on emerging CRISPR-Cas9 gene editing technology. It paid $90 million to Cambridge, MA-based Editas Medicine (NASDAQ: EDIT) for an option to grab rights to up to five experimental eye disease therapies.

—Harvard University spinout eGenesis raised $38 million in its pursuit of the once-abandoned field of xenotransplantation. The firm is using CRISPR-Cas9 to engineer pigs that grow up with human-compatible organs.

—One of eGenesis’s lead investors, Biomatics Capital Partners, announced the close of a $200 million fund, its first. The two Biomatics principals have ties to Bill Gates.

—San Francisco-based Pivotal bioVenture Partners closed a $300 million fund, its first, to invest in early stage biotechs. The fund is led by Tracy Saxton, who previously had stints at Roche Venture Fund and SV Life Sciences Advisors.

—South Plainfield, NJ-based PTC Therapeutics (NASDAQ: PTCT) acquired Duchenne muscular dystrophy drug deflazacort (Emflaza) from Marathon Pharmaceuticals, and in the process inherited the pricing controversy surrounding the drug.

Ben Fidler and Frank Vinluan contributed to this report.

White House photo by jason goulding via Creative Commons license