Bio Roundup: CRISPR Drama, Marathon and PhRMA, the Price of Vision & More

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with its treatment for a form of genetic blindness. At the BIO CEO conference in New York, the company’s CEO, Jeff Marrazzo, acknowledged that a creative pricing solution will be necessary—and not easy to hammer out with insurers.

—Investment in private companies developing microbiome-related medicines has jumped more than 20-fold since 2013. But a big clinical setback, a lack of biological understanding, and now, uncertainty about the near-term future of the FDA, have added challenges to an already difficult prospect, a panel of experts explained at the BIO CEO conference.


—Lots of news in HIV from the CROI gathering in Seattle: Merck had positive Phase 3 news for doravirine in previously untreated patients; GlaxoSmithKline (NYSE: GSK) touted a two-drug cocktail featuring its dolutegravir; and Gilead Sciences (NASDAQ: GILD) said its new bictegravir posted similarly positive results to dolutegravir in a head-to-head Phase 2 study of combination therapies.

—Celgene (NASDAQ: CELG) announced positive results in the first of two Phase 3 trials of ozanimod, the multiple sclerosis drug it acquired in its $7.2 billion buyout of Receptos in 2015.

—Ardelyx (NASDAQ: ARDX) of Fremont, CA, announced positive Phase 3 results for tenapanor, a treatment for hyperphosphatemia.

—Axovant Sciences (NASDAQ: AXON) of New York will move its nelotanserin into Phase 3 after positive Phase 2 results treating Lewy body dementia.

—Sage Therapeutics (NASDAQ: SAGE) touted early-stage data from a tiny trial of an experimental depression drug. CEO Jeff Jonas then told Bloomberg that the company is “on top of everyone’s M&A list,” igniting a stock surge that Sage had to douse with a follow-up statement saying Jonas “did not imply the company was in discussion with potential buyers.”


—Botox-maker Allergan (NYSE: AGN) is adding another medical aesthetics product to its portfolio with a $2.4 billion deal to acquire Zeltiq Aesthetics (NASDAQ: ZLTQ, a Pleasanton, CA-based company that commercialized a medical device that uses cold temperatures to eliminate fat.

—In another sign of growing interest in medical devices that address body appearance, Marlborough, MA-based women’s health company Hologic (NASDAQ: HOLX) agreed to pay $1.44 billion to acquire Cynosure (NASDAQ: CYNO), a company in Westford, MA, whose portfolio includes the fat-zapping laser device SculpSure.

—Boston’s Rhythm raised $41 million from private investors to push its lead drug, meant to treat metabolic disorders, into Phase 3 studies.

—South San Francisco-based Surrozen raised a $33 million Series A round to develop drugs that can regulate tissue repair in the body.

—University of California San Diego spinout Jecure Therapeutics launched with $20 million in funding from Versant Ventures to develop drugs to treat the liver disease nonalcoholic steatohepatitis.


—After a solid start to 2017, biotech IPOs stumbled. The two most recent companies to try for the Nasdaq—Visterra and Braeburn Pharmaceuticals—both pulled their offerings, citing market conditions.

—Biotech investment fund venBio, which owns nearly 10 percent of Immunomedics (NASDAQ: IMMU), slammed the deal that the Morris Plains, NJ-based biotech reached to license its lead cancer drug to Seattle Genetics (NASDAQ: SGEN) for $250 million up front, plus up to $1.7 billion in milestones. VenBio says Immunomedics rushed the deal to avoid losing a proxy fight aiming to oust executives.

—Executives at Intercept Pharmaceuticals (NASDAQ: ICPT) said their drug obeticholic acid (Ocaliva) now has an easier path to FDA approval to treat the liver disease nonalcoholic steatohepatitis, thanks to a new trial design. Here’s more from Endpoints.

Alex Lash and Frank Vinluan contributed to this report.

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