Bio Roundup: Spark’s Road Ahead, Juno Rumors, Merck’s Big Day & More

Xconomy National — 

The most overblown health story of the week was President Trump’s health exam. America learned Trump is in perfect health and has “great genes.” He takes Propecia. He doesn’t have heart problems, had a perfect cognitive test score, and is one pound shy of obese.

This scintillating information led to a new social media phenomenon, the “girther” movement, and a number of articles questioning the findings of longtime White House physician Ronny Jackson.

Meanwhile, there was life-science and biomedical news actually pertinent to the public health of the U.S. Spark Therapeutics is trying to influence how our healthcare system pays for its expensive gene therapy, which could have ripple effects on the drug industry for years to come. The treatment of lung cancer, a leading cause of cancer death, continues to evolve rapidly. And the first U.S. human trial of a medicine using the gene editing technology CRISPR-Cas9 could start soon.

What were you paying attention to this week? Your exam is below.


—The first U.S. gene therapy, voretigene neparvovec (Luxturna), approved in December for an inherited form of vision loss, faces big commercial challenges. Xconomy spoke with the CEO of Luxturna’s developer, Spark Therapeutics (NASDAQ: ONCE), about the complexities of pricing, forging new types of payment models, and paving the way for future gene therapies.

—In an effort to help lower the prices of generic drugs and prevent shortages at hospitals, a group of some 300 hospitals are banding together to create a nonprofit company that will provide generic drugs to hospitals, the New York Times reports.


MIT Technology Review reported that the University of Pennsylvania could soon start the first U.S. study of a medicine that deploys CRISPR-Cas9. The study, funded by the San Francisco-based Parker Institute for Cancer Immunotherapy, got a thumbs-up in 2016 from National Institutes of Health reviewers.

The Wall Street Journal reported late Tuesday that Celgene (NASDAQ: CELG) was mulling a buyout of T cell therapy developer Juno Therapeutics (NASDAQ: JUNO), bumping Juno’s share price more than 50 percent. Celgene already owns 10 percent of Juno and rights to some of its products, thanks to this 2015 deal.


—In another positive development for the rapidly changing treatment landscape for lung cancer, Merck (NYSE: MRK) reported that a combination regimen utilizing its immunotherapy drug, pembrolizumab (Keytruda), and two chemotherapies has succeeded in a confirmational Phase 3 study in patients with newly diagnosed non-small cell lung cancer.

–Shares of Eiger BioPharmaceuticals (NASDAQ: EIGR) lost nearly half their value after the Palo Alto, CA, company announced that its pulmonary arterial hypertension drug ubenimex failed a Phase 2 study.


—The Senate HELP committee approved the nomination of former Eli Lilly (NYSE: LLY) executive Alex Azar to run the Health and Human Services Department, which oversees the FDA, NIH, Centers for Disease Control and Prevention, and the vast Medicare and Medicaid insurance programs. Azar is expected to win full Senate approval and succeed Tom Price, who resigned in September.

—The FDA wants to allow more public access to the clinical data that drives drug approvals. Commissioner Scott Gottlieb outlined the plans in a speech Tuesday, but it remains to be seen how much information currently kept private will become public.

—With an FDA nod to treat breast cancer, AstraZeneca’s (NYSE: AZN) olaparib (Lynparza) became the first member of an emerging class of therapies called PARP inhibitors cleared for a disease other than ovarian cancer. Rival drugs from Tesaro (NASDAQ: TSRO) and Clovis Oncology (NASDAQ: CLVS) are both being tested in breast cancer as well.

—As of this writing, health insurance for 9 million low-income children remained uncertain as lawmakers wrangled over a federal spending plan to avoid a government shutdown. Though Republicans put reauthorization of the Children Health Insurance Plan (CHIP) in their budget proposal, President Trump complicated the matter with a Tweet Thursday saying CHIP shouldn’t be attached to that temporary funding measure.

—The Patent Trial and Appeal Board struck down a patent protecting Johnson & Johnson’s (NYSE: JNJ) prostate cancer drug abiraterone (Zytiga), clearing the way for generic versions of the treatment from Argentum Pharmaceuticals and others. Here’s more from Reuters.


–Cambridge, MA-based Pandion Therapeutics emerged from stealth with $58 million in Series A financing, which it will use to develop a pipeline of bispecific antibody drugs for autoimmune and inflammatory disorders.

—China and Cambridge-based cancer drugmaker BeiGene (NASDAQ: BGNE) bagged $750 million in a stock offering.

—Agios Pharmaceuticals (NASDAQ: AGIO), also of Cambridge, snagged $475 million in a stock sale.

—Struggling Cambridge-based Genocea Biosciences (NASDAQ: GNCA), which still hasn’t find a partner to move its experimental genital herpes vaccine into phase 3 testing and whose shares now trade at just $0.82 apiece, raised $55 million in stock offerings to help fund its other work in cancer.

Alex Lash and Frank Vinluan contributed to this report.