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Bio Roundup: Crazy ’16, Trials of ’17, Senate on Drugs & More

Xconomy National — 

You’ve probably seen the Internet memes. “When 2016 started, I looked like this. [Insert picture of young Leo DiCaprio.] Now I look like this. [Leo, with mountain beard, after mud-wrestling with a grizzly in The Revenant.]” In life science terms, let’s just say all our telomeres feel a lot shorter than 12 months ago.

The biggest issues of 2016 are also the biggest questions of 2017: What do the Trump administration and Republican-led Congress have in store for federal health initiatives, laws, and agency budgets? Will government and public pressure force change on drug prices? Or will drug makers and insurers proactively adopt new schemes that preserve profits and the public good?

Early this year, the circus hit full tilt when Martin Shkreli smirked his way through a Congressional hearing on price gouging. More executive grilling followed. (The Associated Press reported last month that “naming and shaming” hasn’t had much impact.)

So it seems appropriate that we’re ending the year with a Senate price-gouging report slamming Shkreli and his former companies, Retrophin and Turing Pharmaceuticals.

Cutting-edge biomedical research, once out of the lab, hit hurdles in 2016. A leading microbiome program to combat deadly C. difficile infections failed a major trial that could have led to a landmark approval.

In cancer immunotherapy, one leading developer of CAR-T cell therapies, Novartis (NYSE: NVS), dismantled its research group, even while pressing toward FDA approval of its lead product for kids with leukemia. The approval could come next year, as could approval for a lymphoma CAR-T therapy from Kite Pharma (NASDAQ: KITE).

Another top CAR-T developer, Juno Therapeutics (NASDAQ: JUNO), revealed in 2016 that five people died while taking its most advanced experimental therapy. Officials earlier this month could not explain why the therapy was causing the deadly brain swelling.

The new year will also bring gene editing news. Patent judges could issue a decision about the ownership of CRISPR-Cas9, while a few trials using the breakthrough technology could begin. But new versions of CRISPR are in the works and might eventually make the original Cas9 patents a footnote.

For pre-holiday developments on all these fronts, and more, let’s get to the roundup. Happy holidays, everyone.

TOP STORIES

—In a two-part series, Xconomy previewed several key clinical studies expected to produce data in 2017 and possibly shape healthcare in the years to come. Part one digs into trials in hemophilia, cystic fibrosis and heart disease, among others. Part two looks at studies in Zika infection, migraines, multiple sclerosis, and more.

—Along with the Senate’s drug-pricing report, a group of Democrats and independents in the chamber urged Trump to help reform drug prices. Another Democratic senator, Joe Manchin of West Virginia, called for a new “war on drugs”—namely, prescription painkillers that have ravaged communities across the country. The Los Angeles Times reported that the maker of Oxycontin turned to overseas markets when U.S. prescriptions began to fall.

—Cambridge, MA-based Biogen (NASDAQ: BIIB) stayed in-house to replace departing CEO George Scangos. Michel Vounatsos, a Merck veteran who joined Biogen as chief commercial officer earlier this year, will take over Jan. 6. He said he would make Biogen more “efficient” and pump up its core multiple sclerosis franchise.

—Looking beyond the CRISPR-Cas9 patent fight, the Broad Institute sold an exclusive license for a new type of CRISPR to Editas Medicine (NASDAQ: EDIT), which has close ties to the Broad’s top gene edit researcher Feng Zhang. Meanwhile, the Broad’s CRISPR patent rival, UC Berkeley, said that its scientists have found new CRISPR elements in microbes dug out of soil, mines, and geysers.

—In a week in which New York City and state governments pledged $1.15 billion to boost the local life sciences industry, Xconomy held New York Life Sciences 2021 to hear from local industry experts about shaping New York’s biotech future. Here are a few takeaways and photos.

PARP-MANIA

—Clovis Oncology (NASDAQ: CLVS) won FDA approval of rucaparib (Rubraca) for advanced ovarian cancer patients with a specific genetic signature.  Clovis announced a price of $165,000 per patient per year. The approval is the latest of a so-called “PARP” inhibitor, which are being tested in a variety of cancers.

—Meanwhile, the FDA began a priority review—faster than usual—of Tesaro’s (NASDAQ: TSRO) rival PARP blocker niraparib and said it wouldn’t convene a panel of outside experts to debate the drug’s merits—a positive sign for niraparib’s approval chances. The agency will make a decision by June 30.

ROCHE DATA AND MORE

—While experimental gene therapies for hemophilia have attracted attention this year, other drugs continue through the clinic. The Roche empire (including Genentech and Chugai) this week reported positive data in the first of three Phase 3 trials for emicizumab. The drug is meant for the subset of hemophilia A patients who develop immune responses, or “inhibitors,” to current treatments.

—The FDA will delay three months to make its approval decision on ocrelizumab (Ocrevus), a multiple sclerosis treatment from Roche’s Genentech group. Data from recent trials were published in the New England Journal of Medicine.

—San Diego’s Acadia Pharmaceuticals reported positive Phase 2 data for its drug pimavanserin in a study of psychosis related to Alzheimer’s disease.

—Shares of Cambridge-based Merrimack Pharmaceuticals (NASDAQ: MACK) fell 18 percent after the company stopped a Phase 2 breast cancer trial early. The drug MM-302 was unlikely to benefit patients.

PRE-CHRISTMAS DEALS

—San Antonio-based Acelity sold its LifeCell regenerative medicines unit to Allergan (NYSE: AGN) for $2.9 billion.

—Otsuka Pharmaceutical committed $265 million to Cambridge, MA-based Akebia Therapeutics (NASDAQ: AKBA) to help fund Phase 3 trials of anemia pill vadadustat.

—Novartis will pay $50 million upfront to Conatus Pharmaceuticals (NASDAQ: CNAT) for rights to develop the fatty liver disease drug emricasan if Conatus successfully brings it through Phase 2 studies.

—New York-based Bristol-Myers Squibb (NYSE: BMY) paid U.K.-based PsiOxus Therapeutics $50 million up front for rights to a type of cancer therapy called an oncolytic virus—engineered to invade and kill tumor cells—called NG-348.

—Five months after Tokai Pharmaceuticals’s (NASDAQ: TKAI) prostate cancer drug failed a Phase 3 trial, the company agreed to sell most of its shares to privately held Otic Pharma, which will rebrand Tokai as a developer of drugs for ear, nose, and throat disorders.

—Summit, NJ-based Celgene (NASDAQ: CELG) picked up an option to acquire an experimental cancer drug, MSC-1, from Toronto startup Northern Biologics. Terms weren’t disclosed.

Ben Fidler contributed to this report.

Photo courtesy of flickr user MK Feeney via a Creative Commons license.