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Bio Roundup: #JPM18, Axovant’s Fall, CRISPR Caveat, Impact M&A & More

Xconomy National — 

Have you gotten your flu shot yet this year? This winter’s flu season is shaping up to be a particularly nasty one and the Centers for Disease Control and Prevention this week upped its tally of states reporting widespread flu activity to 46.

Flu awareness was top of mind at the J.P. Morgan Healthcare Conference held this week in rainy San Francisco. While the annual gathering provides a way for life science companies, investors, and others to make connections and strike deals, some attendees chose fist bumps instead of handshakes to stem the spread of germs. Xconomy emerged from the conference disease-free, but we couldn’t shake the malaise of seeing men wearing fleece vests under their suit jackets. We’ll have more news to report from our meetings there soon.

Also this week, a leukemia drug company was the target of a billion-dollar buyout, a dementia drug failed again in clinical trials, a CRISPR-Cas9 research paper made deep cuts, and several pharma companies announced restructuring plans. Let’s get to those stories and more in this week’s roundup.

BIOTECH MOVERS & SHAKERS

—The J.P. Morgan Healthcare Conference in San Francisco, biopharma’s biggest yearly gathering, came and went this week, along with its wall-to-wall meetings and schmooze-fests in Union Square’s hotels, bars, and cramped restaurants. As in years past, Xconomy provided a glimpse into the meeting via tidbits from our notebooks that include gene-therapy observations, drug-price notes, a close eye on Alnylam Pharmaceuticals (NASDAQ: ALNY), and the above-mentioned sartorial face-palm.

—Several companies also chose to reveal corporate restructuring plans at the conference. Shire (NASDAQ: SHPG) is splitting off its brain drug R&D into a separate unit, leaving the company with two divisions, neuroscience and rare diseases. Medtronic’s (NYSE: MDT) corporate shakeup is expected to save as much as $700 million over the next five years, though it didn’t say how many jobs would be affected.

—Pfizer (NYSE: PFE) snuck in its restructuring news before J.P. Morgan started, quietly disclosing it will stop its Alzheimer’s and Parkinson’s disease drug research. The shakeup will cut 300 jobs in Massachusetts and Connecticut, the Wall Street Journal reported.

—Some small biotech companies are also starting the new year with downsizing plans. Madison, WI-based drug developer Cellectar Biosciences (NASDAQ: CLRB) said it would shutter in-house manufacturing and transfer that work to partners in order to save money.

OUT OF WASHINGTON

–A report from the non-partisan Congressional Budget Office says extending the Children’s Health Insurance Program (CHIP) for 10 years would actually yield net savings for the federal government because the alternatives to the program, which benefits an estimated 9 million children whose families don’t qualify for Medicaid, would cost more. Nevertheless, Congress has yet to agree on an extension for CHIP funding, which by some estimates, could dry up in some states as soon as Jan. 19.

—Though FDA Commissioner Scott Gottlieb has been on the job for less than a year, he didn’t hesitate to tout the agency’s 2017 accomplishments: a record-breaking number of approvals for new drugs and biologics (56), generic drugs (1,027) and medical devices (95). As the FDA pursues several initiatives to speed up the review of novel drugs, generics, and digital therapeutics, he hopes the approval trends continue.

DATA REVELATIONS

—Last Friday after market close, a non-peer-reviewed paper described a human immune response to the CRISPR-Cas9 gene editing system. Come Monday, the stocks of the main three companies racing to use CRISPR-Cas9 as medicine—Intellia Therapeutics (NASDAQ: NTLA), Editas Medicine (NASDAQ: EDIT), and CRISPR Therapeutics (NASDAQ: CSRP)—took a big hit, even though the paper was not peer reviewed and the authors were careful to note many caveats, including workarounds that companies have already begun to explore. By Thursday close, CRISPR shares had rebounded, closing above last Friday’s peak. Intellia and Editas had not quite climbed back.

—Shares of Axovant (NASDAQ: AXON) lost more than half of their value after the company reported its drug intepirdine failed two mid-stage studies in patients who have dementia with Lewy bodies. It’s the drug’s second failure in a little more than three months. The Switzerland-based company tried for a positive spin by pairing the news with encouraging early results testing a different drug as a treatment for hallucinations associated with Parkinson’s disease. But the company later had to correct its report of those data.

—After reporting positive results in a late-stage study testing migraine-prevention drug eptinezumab in patients with chronic migraine, Bothell, WA-based Alder Therapeutics (NASDAQ: ALDR) said it is on track to file for FDA approval in the second half of the year.

–A Revance Therapeutics (NASDAQ: RVNC) drug developed to treat plantar fasciitis failed to beat a placebo in a mid-stage study, but the Newark, CA, company plans to try again with a different trial design.

—Merck (NYSE: MRK) gave a look at early results from a Phase 3 study of its anti-PD-1 checkpoint inhibitor, pembrolizumab (Keytruda), in patients who have had their melanoma surgically removed and are at risk of seeing the cancer return. The company reported that the trial hit its main goal of improving patients’ recurrence-free survival compared to those on placebo. The Merck cancer drug already has FDA approval to treat metastatic melanoma and several other cancer types.

DEALS & DOLLARS

—Celgene (NASDAQ: CELG) is paying $1.1 billion upfront to acquire San Diego-based Impact Biomedicines, including its drug fedratinib, a potential treatment for a slow-growing form of leukemia.

—Regeneron Pharmaceuticals (NASDAQ: REGN) is partnering with five other biopharma companies to sequence all the exomes (the protein-coding part of the genome) from the UK Biobank, one of the world’s largest repositories of human genetic data and health records, containing samples and medical information from 500,000 British volunteers. Regeneron will do the sequencing and AbbVie (NYSE: ABBV), Alnylam Pharmaceuticals (NASDAQ: ALNY), AstraZeneca (NYSE: AZN), Biogen (NASDAQ: BIIB), and Pfizer will each pitch in $10 million to fund the effort, which will be completed by the end of 2019. Then they’ll have one year to work with the data before their release to other researchers at the end of 2020.

—Eyenovia turned an inkjet printing technique into a way to administer tiny amounts of a drug to the eye, and the New York startup this week updated its plans for an IPO to finance late-stage studies testing its technology.

Image of H1N1 influenza by Flickr user NIAID via a Creative Commons license.