Bio Roundup: Cholesterol Check, J&J’s Opioid Hit, AbbVie’s Flop & More

Xconomy National — 

You might be camping, barbecuing, or sleeping on Monday. It’s Labor Day, after all. But in Paris, this cardiologist will be in a conference center, walking an audience through a slide deck packed with Phase 3 data for a new cholesterol-lowering drug, inclisiran.

Inclisiran’s owner, the Medicines Co., jumped the presentation by a week, promising in a statement Monday that the data were very good, indeed. Xconomy and many others will pore over the results to make sure. (Stay tuned for our coverage.)

Even if, as MedCo says, inclisiran is safe, effective, and more convenient, needing only two doses a year, there’s no guarantee insurers will pay for it. As Xconomy reported this week, two approved drugs which, like inclisiran, work by blocking the protein PCSK9, continue to meet resistance despite doctor support, price cuts, and evidence of health benefits.

Speculation grew this week that one of those drugs, Praluent, might not be worth the effort for its owners. Well-known analyst Geoffrey Porges published a note with the headline “Will REGN Bail?” (That’s the stock ticker for Regeneron Pharmaceuticals (NASDAQ: REGN).)

But Regeneron and Praluent co-owner Sanofi (NYSE: SNY) had some good PCSK9 news this week, as well. A federal judge ruled for them and against rival Amgen (NASDAQ: AMGN) in the latest twist of a long-running patent fight. Amgen, which has been more enthusiastic lately about prospects for its drug Repatha, will appeal. Short term, it’s more likely that insurers, plus competition from inclisiran and other drugs, will have more sway on the PCSK9 market than patent scuffles.

PCSK9 drugs were at the heart of this week’s life-sciences headlines, but there was a larger body of news to digest. Let’s get to the roundup.


—AbbVie (NYSE: ABBV) said experimental cancer treatment rovalpituzumab tesirine, or Rova-T, failed a Phase 3 study in small-cell lung cancer. The company will pull the plug on Rova-T three years after paying $5.8 billion to acquire its owner, Stemcentrx.

—GlaxoSmithKline (NYSE: GSK) reported that its experimental multiple myeloma drug belantamab mafodotin succeeded in a pivotal study. GSK plans to file for regulatory approval by year’s end.

—A year after failing a late-stage study, the AstraZeneca (NYSE: AZN) lupus drug anifrolumab succeeded in a second Phase 3 trial.

—The Boston Globe published a pro/con debate over the merits of releasing genetically engineered mice into the wild to combat Lyme disease.

—Duchenne muscular dystrophy mostly affects boys. STAT profiled the rare case of two young girls with the disease and their struggles to receive experimental therapies.

—The FDA’s adverse events database reported that 30 patients taking the Vertex Pharmaceuticals (NASDAQ: VRTX) cystic fibrosis medicine Symdeko have died. But Vertex responded that it hasn’t been contacted by the FDA and has no evidence tying the deaths to its drug. Shares rebounded after a 4 percent dip.


— Celgene (NASDAQ: CELG) reached a deal to sell anti-inflammatory drug apremilast (Otezla) to Amgen/ (NASDAQ: AMGN) for $13.4 billion cash. The deal was needed to win the blessing of antitrust regulators for Celgene’s pending $74 billion acquisition by Bristol-Myers Squibb (NYSE: BMY).

Celgene also cut a deal with German biotech Immatics for three cell therapies being developed for solid tumors. Celgene paid $75 million upfront.

—GSK paid Ionis Pharmaceuticals (NASDAQ: IONS) $25 million for rights to two experimental hepatitis B drugs following positive Phase 2 results.

—Rare disease drug developer Zogenix (NASDAQ: ZGNX) agreed to pay $250 million upfront for privately held Modis Therapeutics. Modis’s top drug candidate is in late-stage testing for thymidine kinase 2 deficiency, an often-fatal inherited disorder.

—HiFiBiO Therapeutics closed a $67 million Series C round to continue development of its antibody drugs for cancer and autoimmune diseases.


—An Oklahoma judge ruled that Johnson & Johnson (NYSE: JNJ) contributed to the state’s opioid crisis and ordered the company to pay $575 million—far less than the $17.5 billion prosecutors sought. Meanwhile, opioid maker Purdue Pharma and its owners, the Sackler family, reportedly offered to pay $10 billion to $12 billion to settle more than 2,000 lawsuits against the company.

—After a long legal battle, the Kentucky Supreme Court ordered the release of secret documents related to Purdue Pharma’s marketing of Oxycontin.

—In a Wall Street Journal op-ed, ex-FDA commissioner Scott Gottlieb called on Congress to change laws to boost biosimilars and drive down drug costs. One idea: force branded drug makers to sell samples to biosimilar developers at a discount to reduce the cost of clinical studies.

—Yearly out-of-pocket costs for Medicare Part D beneficiaries with multiple sclerosis spiked from $372 to $2673 between 2006 and 2016. The annual costs of their medicines climbed from an average of $18,660 to $75,847 per year, according to a study in JAMA Neurology.


—FibroGen (NASDAQ: FGEN) chairman and CEO Thomas Neff, who founded the San Francisco-based firm in 1993, passed away unexpectedly. FibroGen didn’t provide more details. Board member James Schoeneck was named interim CEO.

—Biotech analyst Mark Schoenebaum passed away over the weekend at the age of 46. He was held in high regard as an analyst, a mentor, and a friend. His death was first announced by a former colleague at Evercore ISI.


—This week we posted the agendas for Boston’s Life Science Disruptors, on Oct. 17 at the Broad Institute in Cambridge, MA; and The New York Breed of Startups, on Oct. 15 at the SUNY Global Center in Manhattan. You can grab discounts for each if you register by Sept. 6.

—The biopharma industry in Massachusetts added more than 4,300 new jobs last year. It’s the highest annual increase in the past decade, according to the Massachusetts Biotechnology Council.

—Richard Fitzgerald is stepping down as chief financial officer of Sesen Bio (NASDAQ: SESN) and will be replaced by Monica Forbes … Tom Neary joined SEngine Precision Medicine as CFO … Maninder Hora was named chief technical operations officer at Amunix Pharmaceuticals … Mark Sampson was appointed chief scientific officer of Azitra … Scholar Rock (NASDAQ: SRRK) CFO Rhonda Chicko is departing at the end of September … and Rodger Currie left the Pharmaceutical Research and Manufacturers of America to become head of government affairs at Grail.

Ben Fidler and Frank Vinluan contributed to this report.