Bio Roundup: CBO’s Praise, Celgene’s Shock, Trump’s Opioid Plan & More

Xconomy National — 

The White House and the FDA turned the spotlight on the nation’s opioid crisis. Blockbuster drugs from Alexion and Tesaro gained expanded approvals, but bad earnings news from Celgene and Biogen brought stocks down. Academics in the U.S. and China made progress with the gene editing technology CRISPR. And as the week’s biggest biotech deal centered on Alzheimer’s, researchers in the field argued for a new approach in the fight against the disease, and the NIH poured more money into brain research. For a broader swath of the week’s news, read on.


—Unlike its assessments of GOP efforts to repeal Obamacare earlier this year, the Congressional Budget Office gave a positive score to the proposed Obamacare fix, negotiated by Sens. Lamar Alexander (R-TN) and Patty Murray (D-WA) and endorsed by 24 senators on both sides of the aisle.

—President Trump declared the opioid crisis a national health emergency and promised several initiatives, including “really great advertising” to keep people away from drugs, but the declaration did not include a request for new funding. The previous day, FDA commissioner Scott Gottlieb told a Congressional committee that his agency would push for more medication-based treatment for substance abuse.

—California’s new drug price transparency law won’t make much difference, opined STAT’s pharma columnist Ed Silverman.

—The FDA expanded approval of Alexion Pharmaceuticals’ (NASDAQ: ALXN) drug eculizumab (Soliris) to include generalized myasthenia gravis, an autoimmune disorder that causes muscle weakness. The FDA first approved the New Haven, CT, biotech’s drug in 2011 for atypical hemolytic syndrome, a rare inherited disorder that leads to progressive damage of organs.

—The intravenous form of the Tesaro (NASDAQ: TSRO) drug rolapitant (Varubi) received FDA approval to treat nausea and vomiting in cancer patients receiving chemotherapy. The regulator approved the pill version of the Waltham, MA, company’s drug in 2015.

—PTC Therapeutics (NASDAQ: PTCT) failed in its third attempt to win FDA approval for its Duchenne muscular dystrophy drug atalursen (Translarna). The South Plainfield, NJ, company plans to appeal the decision.

—Eli Lilly (NYSE: LLY) recently filed for FDA approval of its migraine drug galcanezumab, but the Indianapolis drug maker already faces a competitive challenge. Reuters reported that Israel’s Teva Pharmaceutical (NYSE: TEVA) filed a lawsuit claiming Lilly’s drug infringes on five patents covering Teva’s migraine drug, which also has yet to be approved.


—Xconomy reported that a group of Alzheimer’s disease experts are urging their peers to spend more time and resources on the brain’s cholinergic system. It has been overlooked, they say, because of the fruitless obsession with beta-amyloid as the point of attack against Alzheimer’s.

—Broad Institute researchers published work detailing a new form of the CRISPR-Cas gene editing system that can replace single letters within a strand of RNA.

—In the journal PNAS, Chinese scientists showed that they had created leaner pigs with CRISPR-Cas9. Headlines about low-fat bacon sizzled across the Internet.

—The National Institutes of Health announced $169 million in grants for brain research under its BRAIN Initiative. A major chunk of the cash is going to the Allen Institute for Brain Science in Seattle, which is working to create an atlas of every cell type in the brain. Xconomy wrote about the launch of the atlas project in 2015.

Gizmodo wrote about desperate cancer patients home-brewing their own immunotherapy.


—Celgene (NASDAQ: CELG) shook biotech markets Thursday with a gloomier sales outlook than expected. As Matthew Herper of Forbes noted, the company has lost a third of its value in three weeks.

—On its quarterly earnings call, Biogen (NASDAQ: BIIB) announced lower-than-expected sales of its high-priced spinal muscular atrophy drug nusinersen (Spinraza).

—While its lead drug candidate GBT440 heads toward a major data readout in sickle cell disease, Global Blood Therapeutics (NASDAQ: GBT) pulled the plug on an effort to develop the compound for idiopathic pulmonary fibrosis.

—Gilead Sciences (NASDAQ: GILD) reported positive Phase 2 data for its nonalcoholic steatohepatitis drug GS-0976.


—Abbvie (NYSE: ABBV) paid $205 million upfront to South San Francisco, CA-based Alector and will share development costs and profits on any Alzheimer’s drug that comes to market from the tie-up.

—Alzheon of Framingham, MA, needs to raise another $50 million to push its Alzheimer’s disease treatment ALZ-801 into … Next Page »

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