Bio Roundup: Trials to Watch, Mega Deals, Video Game Therapy & More

Xconomy Boston — 

With 2018 around the corner, this was a week to look ahead. At Xconomy, we zeroed in on several clinical trials that could become major stories in the life sciences. Other healthcare milestones are on the way, too.

The FDA will consider for the first time whether a video game should be approved as medicine. The agency could also give its first nod for a gene therapy. We could see the first clinical trial by a U.S. company for a CRISPR-Cas9 gene editing treatment. And, of course, there are several healthcare implications for the Republican tax bill, the fate of which could be decided by the time the ball drops in Times Square at the end of the month.

Let’s look ahead by looking back at this week’s headlines. It’s roundup time.


—Xconomy published a two-part preview of 15 key clinical trials expected to produce results next year. Part one includes studies in Alzheimer’s disease, lung cancer, anemia, and spinal muscular atrophy. Part two includes trials for migraine drugs, cell therapies, and schizophrenia treatments.

—Akili Interactive Labs plans to ask the FDA to approve its therapeutic video game, a treatment for attention deficit/hyperactive disorder, or ADHD. Until Akili shares more details from the key study, however, several questions remain about the product, including the magnitude of the benefit it provided to patients.

—CRISPR Therapeutics (NASDASQ: CRSP) submitted the first clinical trial application by a company to begin a human study of a CRISPR-Cas9 gene editing therapeutic. The trial, testing a treatment for the blood disease beta-thalassemia, could begin in Europe next year.


—Drug store chain and pharmacy benefits company CVS (NYSE: CVS) aims to enter the health insurance business via a $69 billion bid to acquire Aetna (NYSE: AET).

—UnitedHealth Group unveiled a deal of its own, a $4.9 billion cash acquisition of health clinic and outpatient surgical center operator DaVita Medical Group.

—Stepping up its efforts to develop a pipeline of cell therapies for cancer, Gilead Sciences (NASDAQ: GILD) paid $567 million to acquire University of California, San Francisco spinout Cell Design Labs. Xconomy profiled some of the immunotherapy work being done by Cell Design Labs founder Wendell Lim in 2016.

—San Francisco Bay Area neurodegenerative disease drug developer Denali Therapeutics priced the biggest biotech IPO of 2017, raising nearly $250 million by selling 13.9 million shares at $18 apiece.

—Alexion Pharmaceuticals (NASDAQ: ALXN) paid Halozyme Therapeutics (NASDAQ: HALO) $40 million upfront for use of its technology that allows drug injection just under the skin.


—Despite the drug industry’s dismal public reputation, some companies are doing well making their clinical data available. A report from Bioethics International, headed by NYU professor Jennifer Miller, covered the world’s largest companies and gave Johnson & Johnson and Sanofi top marks for their clinical disclosures. Allergan and Valeant trailed the field.

—The New England Journal of Medicine published a promising update from an ongoing early-stage study of an experimental hemophilia gene therapy from Spark Therapeutics (NASDAQ: ONCE). Spark’s treatment is one of several hemophilia gene therapies for both hemophilia A and B in clinical development.

—Shares of Sage Therapeutics (NASDAQ: SAGE) soared to record highs after its experimental SAGE-217 succeeded in a Phase 2 study for major depressive disorder.

—A Revance Therapeutics (NASDAQ: RVNC) injectable treatment for facial frown lines hit its main goals in a Phase 3 study, results that the Newark, CA, company says put it on track to file for FDA approval in 2019.

—Roche unveiled two sets of positive study results this week. Its hemophilia A treatment emicizumab (Hemlibra) showed it could control bleeding when administered once every four weeks. And it made its case to enter the immunotherapy lung cancer race with data from a combination of its own bevacizumab (Avastin) and atezolizumab (Tecentriq) plus chemotherapy.

—Shares of Ra Pharmaceuticals (NASDAQ: RARX) fell nearly 50 percent after Ra released interim Phase 2 data for a drug it has considered a threat to Alexion’s blockbuster treatment eculizumab (Soliris), a treatment for paroxysmal nocturnal hemoglobinuria. While Ra’s RA101495 may end up a potential alternative to eculizumab, Leerink Partners analyst Geoffrey Porges wrote that it looks “much worse… and less convenient” than Alexion’s own in-house alternative, ALXN1210, currently in Phase 3 testing.


—Investment firm OrbiMed, and its managing partner and co-founder Sam Isaly, were accused of creating a toxic work culture of sexual harassment, according to a STAT investigation. Isaly resigned from OrbiMed Thursday evening, though the firm didn’t mention the charges in a prepared statement announcing the move.

—Obsidian Therapeutics, one of two startups currently run by serial biotech entrepreneur Michael Gilman, debuted with $49.5 million in funding. It wants to exert more therapeutic control over CAR-T cells, a cutting edge type of cancer immunotherapy that just hit the market this year.

—Mike Pellini, chairman and former CEO of Cambridge, MA-based Foundation Medicine (NASDAQ: FMI), is joining San Francisco venture capital firm Section 32 as a managing partner, according to Pitchbook.

—Cambridge, MA-based Neon Therapeutics added $36 million more to a Series B round the company first announced in January and closed it out at $106 million.


—Flagship Pioneering CEO Noubar Afeyan talked to Xconomy about immigration’s importance for innovation, and how his views were shaped by his family’s own refugee experiences.

—Dynavax Technologies (NASDAQ: DVAX) CEO Dennis Carson talked to Xconomy about his company’s 21-year odyssey to win FDA approval for hepatitis B vaccine Heplisav-B.

—Acorda Therapeutics (NASDAQ: ACOR) resubmitted an FDA approval application for inhalable Parkinson’s disease drug Inbrija, and expects to hear within 74 days whether the agency will review the document. The FDA refused to look over Acorda’s previous application.


—Life Science Cares, a nonprofit organization run by former Cubist executive Robert Perez to help fight poverty in Boston, handed out over $300,000 in grants to 19 social service organizations, among them Food for Free and More than Words.

Frank Vinluan and Alex Lash contributed to this report.