This week’s headlines are all about second chances and new beginnings. A gene therapy company that survived on bridge loans many years ago is now one of biotech’s biggest stars. A company developing a therapy for Duchenne Muscular Dystrophy is starting to win back investor confidence after a tumultuous year. And a former Pfizer executive has turned in his Big Pharma badge to lead a startup. Those stories and much more to get you through your long Memorial Day weekend below:
—Cambridge, MA-based Bluebird Bio (NASDAQ: [[ticker:BLUE]]) was in the headlines a few times this week. First, it reached a deal with regulators in the U.S. and Europe outlining just what it’ll take to win approval for its gene therapy for beta-thalassemia, LentiGlobin. Then, it gave people an early look at some data from the first sickle cell disease patient to have been treated with a gene therapy. Shares of the company climbed about 10 percent this week; at over $170 apiece, they’re worth ten times the price Bluebird went public at in 2013.
—Sarepta Therapeutics (NASDAQ: [[ticker:SRPT]]) has seen more than its share of ups and downs over the past few years, but this past week saw a big rally for the Cambridge company. Sarepta said following an FDA meeting, it plans to begin what’s known as a “rolling submission” of an application to approve its Duchenne drug, eteplirsen—basically, a tool that allows companies to submit completed portions of their FDA applications to the agency, rather than wait until they finish the whole document. The move sent Sarepta shares soaring some 60 percent, as it implied the FDA is willing to review the eteplirsen data Sarepta has on hand, rather than require the company to gather more first. There’s also at least a chance for a potentially dramatic biotech story later this year—a dual FDA advisory panel reviewing both eteplirsen, and a competing Duchenne drug owned by BioMarin Pharmaceutical (NASDAQ: [[ticker:BMRN]]), drisapersen, on the same day.
—Jose-Carlos Gutierrez-Ramos stepped down from a high-ranking post at Pfizer a few weeks ago to join a biotech, and this week he revealed which one—Cambridge-based Synlogic, a startup with $35 million in backing from Atlas Venture, New Enterprise Associates, and The Bill & Melinda Gates Foundation. I spoke with Gutierrez-Ramos about the move, and the startup’s plans for its programmable, custom-built bacteria.
—Shares of Cambridge-based Eleven Biotherapeutics (NASDAQ: [[ticker:EBIO]]) plummeted more than 70 percent on Monday after its lead drug candidate, EBI-005, failed a late-stage trial for dry eye disease. Eleven will now turn to its other Phase 3 program for EBI-005, for pink eye, which is expected to begin later this year.
—New York-based Intercept Pharmaceuticals (NASDAQ: [[ticker:ICPT]]) reached a deal with the FDA on the design of a late-stage study for its closely watched drug for nonalcoholic steatohepatitis, obeticholic acid. The trial will be a randomized, placebo-controlled, 2,500-patient trial; Intercept will conduct an interim analysis of about 1,400 patients after 72 weeks of treatment. The trial design disappointed investors, however, who sent shares down about 16 percent.
—Cambridge-based Genocea Biosciences (NASDAQ: [[ticker:GNCA]]) said the latest results from a mid-stage study of its experimental vaccine for genital herpes, GEN-003, came back positive, enabling it to pick a dose of the vaccine to move into further testing. For more on Genocea and GEN-003, check out this story.
—New Brunswick, NJ-based Johnson & Johnson (NYSE: [[ticker:JNJ]]) cut a wide-ranging deal with Achillion Pharmaceuticals (NASDAQ: [[ticker:ACHN]]) for full rights to “one or more” of the New Haven, CT, company’s experimental hepatitis C drugs. Achillion stands to get as much as $1.1 billion in various payments in the deal. J&J also paid $225 million for 18.4 million Achillion shares at $12.25 apiece in a separate transaction.
—Two Boston-area companies got some FDA support this week, as the agency granted fast track designations to Agios Pharmaceuticals’ (NASDAQ: [[ticker:AGIO]]) AG-120, for patients with acute myelogenous leukemia and a specific genetic mutation; and to Acceleron Pharma’s (NASDAQ: [[ticker:XLRN]]) luspatercept, for people with certain types of beta-thalassemia.
—Cambridge startup Aura Biosciences received an orphan drug designation for AU-011, its experimental virus nanoparticle treatment for a rare eye cancer called uveal melanoma. Here’s more on Aura’s approach from a piece I wrote back in March.
—Alnylam Pharmaceuticals (NASDAQ: [[ticker:ALNY]]) also got an orphan drug tag for revusiran, a subcutaneous RNA interference drug in late-stage testing for familial amyloidotic cardiomyopathy.
—Exosome Diagnostics said that its urine test for prostate cancer met its main goal in a large clinical trial—when added to the current standard of care (prostate-specific antigen, or PSA testing), the test did a better job of predicting high-grade prostate cancer than PSA tests alone, reducing the need for unnecessary biopsies. Exosome is developing a type of “liquid biopsy,” a catch-all term for fluid-based, rather than surgical cancer diagnostics. You can read more about Exosome’s method here. And for more detail on the trial results, check out this report from Targeted Oncology.
—Tarrytown, NY-based Regeneron Pharmaceuticals (NASDAQ: [[ticker:REGN]]) rolled out more data from two antibody drugs it’s developing with Sanofi: dupilumab, for allergic asthma, and sarilumab, for rheumatoid arthritis. Regeneron aims to file an application for sarilumab later this year. A phase 3 study of dupilumab is underway.
Photo of Boston skyline from the Prudential skywalk courtesy of flickr user Bill Damon via Creative Commons.
