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Bio Roundup: Trump’s Rx Silence, PCSK9 News, Amgen’s Migraines & More

Xconomy National — 

Speculation swirled this week around the incoming U.S. president’s intent to make good on his angry campaign rhetoric on drug prices, while the Associated Press reported that naming-and-shaming—that is, Congress hauling executives into hearings and penning angry letters—certainly hasn’t done the trick.

For one new kind of cholesterol drug, insurers and their agents have tried the “just say no” strategy: refusing to pay what they deem high prices until the drug makers produce broad (and expensive) evidence that the drugs save lives. We’ll start the roundup with a slate of news about these cholesterol drugs, called PCSK9 inhibitors, then carry on to CRISPR, Theranos, and a lot more. Let’s get to the news.

I (HEART) CLINICAL DATA

—At the American Heart Association’s annual meeting in New Orleans, companies provided updates on cholesterol-fighting drugs known as PCSK9 inhibitors, whose high price tags have met with significant resistance from insurers. Amgen (NASDAQ: AMGN) reported that its PCSK9 blocker, evolocumab (Repatha), helps reduce artery-clogging plaques. But the study wasn’t designed to show whether evolocumab leads to fewer heart attacks and strokes, which are the data payers are waiting for. Disappointing sales are unlikely to change for now. Those data are expected next year from Amgen…

—…and from Amgen’s rival Regeneron Pharmaceuticals (NASDAQ: REGN), as well. Regeneron said Thursday that an independent committee reviewed the interim data and told Regeneron and partner Sanofi (NYSE: [[Ticker:SNY]]) that its Odyssey Outcomes study must run the full course. A third PCSK9 competitor, Pfizer (NYSE: PFE), abandoned the field recently without presenting Phase 3 data.

—Also at AHA, The Medicines Co. (NASDAQ: MDCO) and Alnylam Pharmaceuticals (NASDAQ: ALNY) released new Phase 2 data supporting a potentially longer lasting alternative to Amgen and Regeneron’s approved PCSK9 blockers. Years more clinical testing are likely needed to prove the drug’s potential and commercial viability.

THE CUTTING EDGE

—Startup Magenta Therapeutics spun out of Harvard University with $48 million in venture cash and several experimental methods to improve hematopoietic stem cell transplants, which can save lives but come with big risks.

Nature reported that a Chinese medical team has performed the first test of the CRISPR-Cas9 gene editing system in a human. Lu You at Sichuan University has injected CRISPR-modified T cells into a lung cancer patient in a trial slated to treat 10 people. The first U.S. trial using CRISPR could come next year.

NEWS AROUND OUR NETWORK

—Amgen released highlights of Phase 3 data that suggest its migraine drug erenumab could come up for FDA approval next year. Amgen is competing with Alder Pharmaceuticals (NASDAQ: ALDR) and Teva Pharmaceutical (NYSE: TEVA) to develop a new class of drug meant to prevent migraines instead of just treating symptoms.

—Corbus Pharmaceuticals (NASDAQ: CRBP) reported positive results in the first of several Phase 2 trials for anti-inflammatory drug resunab. Shares climbed more than 50 percent.

—Indianapolis startup Apexian Pharmaceuticals is reviving an old hepatitis C drug, shelved by Japanese firm Eisai, to go after pancreatic cancer. The firm’s chief scientific officer is the chair of pediatric oncology research at Indiana University.

—In a major restructuring, Dutch gene therapy pioneer UniQure (NASDAQ: QURE) will cut up to 60 jobs, partner off a few experimental programs, and center all manufacturing in Lexington, MA.

—The state of Texas’s cancer fund granted $32 million to two companies, Bellicum Pharmaceuticals (NASDAQ: BLCM) and Molecular Templates, for their blood cancer work.

BIG NAMES

—Celgene (NASDAQ: CELG) executive Rich Bagger was removed from the Trump transition team as part of a big shakeup that saw Vice President-elect Mike Pence take over the transition from New Jersey governor Chris Christie. Bagger was a top Christie aide.

—Former Secretary of State George Schulz’s grandson Tyler Schulz, a Theranos employee, was the first whistle blower to attract scrutiny to dubious practices at the blood diagnostic firm. The act has caused a rift in his family, the Wall Street Journal reported. George Schulz was a Theranos board member from 2011 to 2015.

—In other Theranos news, former partner Walgreens alleged the firm voided more than 11 percent of its blood test results. The claim was in filings related to Walgreens’ breach-of-contract lawsuit against Theranos.

HANDSHAKES AND SETBACKS

—The FDA rejected hepatitis B vaccine Heplisav-B from Dynavax Technologies (NASDAQ: DVAX) for the second time, knocking the Bay Area firm’s share price from nearly $12 to $4.50 at Thursday’s close.

—Sanofi exercised an option to share rights to an experimental Alnylam hemophilia drug, but passed on a second Alnylam drug prospect for a rare enzyme deficiency, hepatic porphyria. The firms entered an alliance in 2014.

—Gilead Sciences (NASDAQ: GILD) reported a Phase 3 stumble for its myelofibrosis drug momelotinib, layering more doubt upon the company’s pipeline and adding pressure to use its cash pile for M&A.

—Gilead also terminated a drug development partnership with Louisville, CO-based Globeimmune, which reported that its president and CEO, Timothy Rodell, and various boardmembers have resigned. Globeimmune was delisted from the Nasdaq in July.

—Bristol-Myers Squibb (NYSE: BMY) and Paris-based Enterome have teamed up to explore the relationship between the human microbiome and cancer, hoping to unearth new cancer immunotherapy drugs and companion diagnostics. Enterome received $15 million up front.

—The Broad Institute and microchip behemoth Intel expanded a long-running collaboration with a $25 million project to combine and analyze disparate genomic data sets.

Ben Fidler contributed to this report.

Photo of America at night courtesy of NASA.