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Bio Roundup: Light For Spark, California Drug Law, IPO Queue & More

Xconomy National — 

In a year of tragedies across the U.S., natural or otherwise, it seemed this week was California’s turn. Fueled by high winds, low humidity, and ample dry vegetation, fires swept through several towns across the state and, as of this writing, have barely been contained. With our minds on friends and loved ones affected by the fires, we turn to this week’s roundup. It includes the ongoing national health reform fight and plenty of deals and data. And even as California’s skies filled with hazardous smoke, state lawmakers passed a bill to shine more daylight on murky drug prices. Let’s get to it.

HEALTH, LAW, AND MARKETS

—With Congressional Republicans unable to replace the Affordable Care Act with their own healthcare overhaul, President Trump took executive action on two fronts. First, he signed an executive order to let trade and business groups form insurance pools across state lines with fewer restrictions than the Affordable Care Act allows. How soon and how broadly the new rules take effect remains to be seen. Critics said the new pools could favor predominantly healthy consumers and undermine the concept of shared risk that is key to the ACA’s structure. Then Politico broke the news that the administration would end key federal subsidies to insurers who sell coverage under the ACA.

—California Gov. Jerry Brown signed a bill that requires drug makers to notify private and government insurers if they plan to raise prices more than 16 percent over a two-year period. Companies would also have to explain publicly the reasons for the hikes. Opposed by industry, the law takes effect in 2019.

—CNBC’s Christina Farr reported last Friday that Amazon will decide before Thanksgiving whether to sell pharmaceuticals online, sending pharmacy stocks like Walgreens (NASDAQ: WBA) down several percentage points.

THE REGULATORY RUMPUS

—A panel of FDA advisors voted 16-0 to recommend that the agency approve a one-dose gene therapy for a rare form of blindness. If approved, voretigene neparvovec (Luxturna) would be the first therapy in the U.S. that replaces a faulty gene with a working one. The drug’s owner, Spark Therapeutics (NASDAQ: ONCE) of Philadelphia, is pitching it as a one-shot cure. “The data suggest that it could be very long-lasting, if not lifelong,” CEO Jeff Marrazzo told Xconomy.

—Despite hitting its goal in a large trial, Merck (NYSE: MRK) won’t seek FDA approval of cholesterol-lowering drug anacetrapib, concluding that the data aren’t strong enough.

—The FDA approved Flexion Therapeutics’ (NASDAQ: FLXN) extended release formulation of triamcinolone acetonide (Zilretta), a non-opioid treatment for osteoarthritis knee pain. Flexion then raised $122 million that the Burlington, MA, company will use for manufacturing and commercialization.

—The FDA told AcelRx Pharmaceuticals (NASDAQ: ACRX) it would not approve its opioid painkiller Dsuvia. AcelRx, of Redwood City, CA, said it would address the FDA’s concerns and try again.

DATA TIME

—Eli Lilly’s (NYSE: LLY) drug abemaciclib failed a key lung cancer trial, which stalls the Indianapolis-based company’s strategy to compete with similar treatments from Pfizer and Novartis. Abemaciclib has FDA approval in advanced breast cancer.

—Ardelyx (NASDAQ: ARDX) reported positive Phase 3 data for tenapanor, a treatment for irritable bowel syndrome with constipation. Shares were up 44 percent Thursday.

—AnaptysBio (NASDAQ: ANAB) of San Diego said its experimental treatment for atopic dermatitis met the main goal of a Phase 2 study. The stock price jumped more than 67 percent to $58.83 per share.

—Researchers at the Fred Hutchinson Cancer Research Center in Seattle have reported in depth on the side effects from an ongoing, 133-patient CAR-T cell therapy study. The two papers focus on signals, or biomarkers, that could warn doctors who is at risk for life-threatening reactions to CAR-T treatment.

MONEY IN

—The IPO window remains open to life science companies, evidenced by the $52 million debut of Warsaw, IN-based medical device maker OrthoPediatrics (NASDAQ: KIDS).

—More companies joined the IPO queue: Cambridge, MA-based Spero Therapeutics and Allena Pharmaceuticals, based in Newton, MA.

Merck paid KalVista Pharmaceuticals $37 million upfront for the option to license the Cambridge, MA, biotech’s Phase 2-ready drug that could treat diabetic macular edema, a diabetes complication that can progress to blindness. KalVista shares soared nearly 50 percent.

—AbbVie (NYSE: ABBV) paid Turnstone Biologics of New York and Ottawa an undisclosed sum to license up to three immunotherapies that use tumor-killing viruses to fight cancer.

—Investment firm Deerfield Management is committing $50 million toward a new five-year research partnership with the Broad Institute of MIT and Harvard University.

—U.K. infectious disease startup VirionHealth raised a Series A round of £13 million from investment firm Abingworth.

URBAN PLANNING

The San Francisco Business Times reported that San Francisco’s Mission Bay neighborhood, once seen as a biotech enclave to rival Cambridge’s Kendall Square, has favored tech over biotech for new projects. “We’ve lost a chance to diversify the city’s economy,” one broker told the paper.

—Speakers and guests will convene Dec. 11 at Xconomy’s “The Future Is Now” event to discuss whether New York’s biotech scene is ready for prime time.

Image by Dave Winer via Creative Commons 2.0.