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Bio Roundup: Skinny Cuts, Genomic Approvals, Unfrozen FDA & More

Xconomy National — 

Team Trump unveiled its 2018 federal spending proposal—the so-called “skinny budget”—with health and science in the crosshairs. The NIH is looking at a 22 percent cut and the FDA 31 percent, although the administration wants to backfill the FDA loss with a huge boost in user fees, which drug and device companies pay to have their products reviewed for market.

The Centers for Disease Control and Prevention are line for an 18 percent cut, and Medicaid would also take a hit on top of what the GOP healthcare bill, in its current form, would cut.

About that bill… Already expected to be overhauled in the Senate, the American Healthcare Act fared badly on the Congressional Budget Office’s highly anticipated scorecard. For example, the nonpartisan CBO estimated that 23 million Americans would lose health insurance by 2026, and that people with pre-existing conditions would see a big spike in premiums or a loss of coverage despite the bill’s promises to shore up so-called “high risk pools” in states that opt out of mandatory coverage for pre-existing conditions.

According to a roundup in Science, high-profile health projects funded by last year’s 21st Century Cures Act would stay well-funded under the Trump plan, although the proposal might be moot. Congress has already showed this year its willingness to shun the administration’s attempts to cut biomedical funding.

Let’s stay on that story as we segue from this Beltway-heavy introduction into the heartland of our weekly roundup.

TOP STORIES

—One of those Cures-funded projects was on NIH director Francis Collins’s mind this week. Speaking at the Precision Medicine World Conference in Durham, NC, Collins said that the Precision Medicine Initiative, a longitudinal health study aiming to enroll 1 million volunteers by 2022, will kick off its “All of Us” recruitment next week in Pittsburgh.

—Picking up where his predecessor Robert Califf left off, new FDA commissioner Scott Gottlieb pledged to find ways to use the agency’s regulatory authority to curb the nation’s opioid epidemic.

—For the first time, the FDA has approved a cancer drug based on the genetic abnormalities present in a tumor, rather than where the tumor originated. The nod was for Merck’s pembrolizumab (Keytruda), an immunotherapy drug already marketed for skin, lung, and other cancers.

— Alexion Pharmaceuticals (NASDAQ: AXLN) continued its executive overhaul in the wake of sales fraud charges—practices Bloomberg detailed this week in a long expose.

MORE FDA

—The agency’s hiring freeze was unfrozen.

—Regeneron Pharmaceuticals (NASDAQ: REGN) and partner Sanofi won FDA approval of a new drug for rheumatoid arthritis, sarilumab (Kevzara), and priced it at a discount to rival therapies from AbbVie (NYSE: ABBV) and others already entrenched on the market. The FDA’s decision had been delayed several months due to manufacturing problems at a Sanofi site.

—The FDA granted marketing approval to a medical device that Bloomington, IN-based Cook Medical developed to treat a rare birth defect affecting the esophagus.

—Celgene (NASDAQ: CELG) is preparing to file for FDA approval of its experimental multiple sclerosis drug ozanimod after the Summit, NJ, company reported that the pill met the goals of a two-year study.

—Amgen (NASDAQ: AMGN) disclosed new safety concerns for its closely watched osteoporosis drug, romosozumab (Evenity), that will at minimum delay an FDA approval decision for the drug. The news was a boost for Radius Health (NASDAQ: RDUS), whose rival osteoporosis treatment, abaloparatide (Tymlos), was approved in April.

DEALS AND DOLLARS

—Biogen (NASDAQ:BIIB) spinoff Bioverativ (NASDAQ: BIVV) paid $400 million for South San Francico, CA-based True North Therapeutics as part of a plan to focus on rare blood diseases. True North itself was once a spinoff. Bristol-Myers Squibb acquired its predecessor iPerian three years ago and later flipped those assets to Biogen.

ViaCyte raised $10 million that the San Diego biotech plans to use to finance a clinical trial testing its experimental cell replacement therapy for Type 1 diabetes.

—Bain Capital raised $720 million for its first life sciences fund, which is being run by former Biogen executive Adam Koppel. Here’s more from Reuters.

—Symic Bio closed a $30 million Series B round of funding that the San Francisco company plans to apply to its drug pipeline, including SB-030, a compound developed to reduce the scarring that leads to vein graft failure.

—Another Bay Area biotech, OrphoMed, raised $39 million in a Series A funding round to support clinical trials testing a drug developed to treat irritable bowel syndrome with diarrhea.

—And South San Francisco, CA-based Harpoon Therapeutics raised $45 million in a Series B round of funding to bring two cancer immunotherapies into clinical trials.

—Swiss firm Debiopharm International paid $25 million up front to buy an experimental cancer drug from Waltham, MA-based ImmunoGen (NASDAQ: IMGN), IMGN529, currently in Phase 2 testing.

—Shares of Bedminster, NJ-based Aerie Pharmaceuticals (NASDAQ: AERI) jumped more than 32 percent after the company’s combination glaucoma treatment, netarsudil-latanoprost (Roclatan), succeeded in its second Phase 3 trial. Aerie could file for FDA approval of the drug by early next year.

—Cambridge, MA-based Constellation Pharmaceuticals made sweeping executive changes and outlined plans to change its strategy. The company now aims to develop therapies used in combination with immuno-oncology drugs.

—Ben Fidler and Frank Vinluan contributed to this report.

Photo by Flickr user Jake Rustenhoven via a Creative Commons license.