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 with its treatment for a form of genetic blindness. At the BIO CEO conference in New York, the company’s CEO, Jeff Marrazzo, acknowledged that a creative pricing solution will be necessary—and not easy to hammer out with insurers.

—Investment in private companies developing microbiome-related medicines has jumped more than 20-fold since 2013. But a big clinical setback, a lack of biological understanding, and now, uncertainty about the near-term future of the FDA, have added challenges to an already difficult prospect, a panel of experts explained at the BIO CEO conference.


—Lots of news in HIV from the CROI gathering in Seattle: Merck had positive Phase 3 news for doravirine in previously untreated patients; GlaxoSmithKline (NYSE: GSK) touted a two-drug cocktail featuring its dolutegravir; and Gilead Sciences (NASDAQ: GILD) said its new bictegravir posted similarly positive results to dolutegravir in a head-to-head Phase 2 study of combination therapies.

—Celgene (NASDAQ: CELG) announced positive results in the first of two Phase 3 trials of ozanimod, the multiple sclerosis drug it acquired in its $7.2 billion buyout of Receptos in 2015.

—Ardelyx (NASDAQ: ARDX) of Fremont, CA, announced positive Phase 3 results for tenapanor, a treatment for hyperphosphatemia.

—Axovant Sciences (NASDAQ: AXON) of New York will move its nelotanserin into Phase 3 after positive Phase 2 results treating Lewy body dementia.

—Sage Therapeutics (NASDAQ: SAGE) touted early-stage data from a tiny trial of an experimental depression drug. CEO Jeff Jonas then told Bloomberg that the company is “on top of everyone’s M&A list,” igniting a stock surge that Sage had to douse with a follow-up statement saying Jonas “did not imply the company was in discussion with potential buyers.”


—Botox-maker Allergan (NYSE: AGN) is adding another medical aesthetics product to its portfolio with a $2.4 billion deal to acquire Zeltiq Aesthetics (NASDAQ: ZLTQ, a Pleasanton, CA-based company that commercialized a medical device that uses cold temperatures to eliminate fat.

—In another sign of growing interest in medical devices that address body appearance, Marlborough, MA-based women’s health company Hologic (NASDAQ: HOLX) agreed to pay $1.44 billion to acquire Cynosure (NASDAQ: CYNO), a company in Westford, MA, whose portfolio includes the fat-zapping laser device SculpSure.

—Boston’s Rhythm raised $41 million from private investors to push its lead drug, meant to treat metabolic disorders, into Phase 3 studies.

—South San Francisco-based Surrozen raised a $33 million Series A round to develop drugs that can regulate tissue repair in the body.

—University of California San Diego spinout Jecure Therapeutics launched with $20 million in funding from Versant Ventures to develop drugs to treat the liver disease nonalcoholic steatohepatitis.


—After a solid start to 2017, biotech IPOs stumbled. The two most recent companies to try for the Nasdaq—Visterra and Braeburn Pharmaceuticals—both pulled their offerings, citing market conditions.

—Biotech investment fund venBio, which owns nearly 10 percent of Immunomedics (NASDAQ: IMMU), slammed the deal that the Morris Plains, NJ-based biotech reached to license its lead cancer drug to Seattle Genetics (NASDAQ: SGEN) for $250 million up front, plus up to $1.7 billion in milestones. VenBio says Immunomedics rushed the deal to avoid losing a proxy fight aiming to oust executives.

—Executives at Intercept Pharmaceuticals (NASDAQ: ICPT) said their drug obeticholic acid (Ocaliva) now has an easier path to FDA approval to treat the liver disease nonalcoholic steatohepatitis, thanks to a new trial design. Here’s more from Endpoints.

Alex Lash and Frank Vinluan contributed to this report.