Bio Roundup: CRISPR Drama, Marathon and PhRMA, the Price of Vision & More

Xconomy National — 

News may be coming out of Washington, DC, at a breakneck pace, but concrete plans regarding the future of the nation’s healthcare system, the FDA, and the pharmaceutical industry have been much slower to materialize. The consternation about President Donald Trump’s coming pick for FDA commissioner was palpable this week at the BIO CEO & Investor Conference in New York. Speakers universally panned the idea of dramatically shaking up the FDA or lowering the drug approval bar; one biotech investor said significant deregulation, like not testing drugs for efficacy, could turn the industry into the “wild west.”

Meanwhile, plans to replace the Affordable Care Act crept forward, the potential new Medicare/Medicaid chief testified before a congressional committee, and the legal feud over ownership of the gene editing technology CRISPR-Cas9 continued. Let’s get it all rolling below.


—Seema Verma, President Trump’s nominee to run the agency that oversees Medicare and Medicaid, testified before the Senate Finance Committee on Thursday about her plans for programs that cover a third of all Americans. Here’s more in the Washington Post, which says a vote on her appointment isn’t expected for at least a week.

—On Valentine’s Day, a trio of U.S. Senators sent new Health and Human Services secretary Tom Price a letter urging new rules to allow importation of drugs from Canada to combat price gouging.

—On Thursday, GOP lawmakers outlined their plan to replace Obamacare, which, according to the New York Times, rolls back Medicaid expansion and leans “heavily on tax credits” to fund individual insurance purchases.


—The Patent and Trademark Office added its own splash to the firehose of news this week from the nation’s capital. A panel of PTO judges said the Broad Institute of MIT and Harvard’s work on the groundbreaking gene editing technology CRISPR-Cas9 was not an obvious extension of work by a team led by UC Berkeley researcher Jennifer Doudna. The ruling validates the patents Broad received in 2014, for now, but it also leaves open a door for the UC group to get their own patents.

—Merck (NYSE: MRK) joined the long queue of drug developers with failed Alzheimer’s disease treatments. The firm halted a Phase 3 study of verubecestat in mild-to-moderate Alzheimer’s patients after an oversight board said the drug would not meet the goal of improving their cognition. There were no safety concerns, however, and Merck for now will continue a Phase 3 study of verubecestat in Alzheimer’s patients showing little or no symptoms.

—Marathon Pharmaceuticals received no valentines this week. Amid public outrage and threat of a congressional probe, the Northbrook, IL-based firm paused the launch of deflazacort (Emflaza), a Duchenne muscular dystrophy-treating steroid. Marathon priced the drug at $89,000 a year, even though it is available in other countries at a fraction of the cost. (Endpoints asked drug development veterans to scrutinize Marathon’s justifications for the price.) According to Bloomberg, Marathon, whose CEO is on the board of industry lobbying group PhRMA, could be ousted from the group.


—Drug pricing was (of course) on plenty of biopharma minds this week, as well. Xconomy examined the case of Philadelphia’s Spark Therapeutics (NASDAQ: ONCE), which this year could win the first ever U.S. approval of a gene therapy … Next Page »

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