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Bio Roundup: Wrapping ASCO, Dreaming IPO, Rebooting Axovant & More

Xconomy National — 

Most of the cancer news this week happened at the massive ASCO meeting in Chicago. But not all, as we’ll see in a moment.

Also this week, Biogen and Eisai reported good news about an Alzheimer’s drug, but with plenty of caveats. Axovant Sciences, which hit the skids last year because of a massive Alzheimer’s failure, announced comeback plans in Parkinson’s disease. Axovant’s IPO three years ago was one biotech’s largest ever, and it certainly won’t be matched by the IPO aspirants lining up this week. But plenty of money continues to roll into biotech companies. We’ll catch you up on all the latest. Let’s roll into the roundup.

ONCOLOGY NEWS

—The American Society of Clinical Oncology’s yearly meeting is in the books. Xconomy wrapped up some of the major themes, including the race to develop cancer immunotherapy combinations, the progress of “tissue-agnostic” cancer drugs, the state of lung cancer care, and more.

—Later this year, Loxo Oncology (NASDAQ: LOXO) could become just the second company with a tissue-agnostic cancer drug on the market. Leading up to ASCO, Xconomy spoke with Loxo executives about early data on Loxo’s newest experimental therapy LOXO-292 and the challenges of this new frontier of treating cancer based on its genetic fingerprint, not on the part of the body where it began growing.

—The immunotherapy nivolumab (Opdivo) seemed to make patients worse, not better, in a study of the uncommon blood cancer adult T-cell leukemia–lymphoma. The doctors running the trial penned a warning that was published in the New England Journal of Medicine last month and reported this week by the New York Times.

VENTURE & IPO

—Boston-area biotechs Neon Therapeutics and Translate Bio joined the growing line of life-science companies filing for IPOs to finance clinical development of their drug pipelines. Forty Seven, a Menlo Park, CA-based company developing immunotherapies targeting a cancer protein called CD47, also joined the queue.

—MeiraGTx (NASDAQ: MGTX), a Kadmon (NASDAQ: KDMN) spinout developing gene therapies for rare diseases of the eye and other tissues, raised $75 million in an IPO by selling 5 million shares at $15 apiece.

—Pfizer (NYSE: PFE) is plowing $600 million into its venture arm, with roughly a quarter of that cash ticketed for neuroscience startups.

—The Bill & Melinda Gates Foundation has launched its own nonprofit biotech, the Gates Foundation Medical Research Institute. The Cambridge, MA-based institute’s director talked to reporters at the BIO conference in Boston this week.

—Nimbus Therapeutics of Cambridge, raised $65 million to press ahead with a pipeline split between drugs partnered with larger pharmaceutical companies and compounds it plans to develop itself.

—Seven months after closing $22 million in financing, San Diego’s Metacrine raised another $65 million in a Series C round to bring its lead drug into clinical trials for nonalcoholic steatohepatitis, a liver disorder.

APPROVALS, PRICES, AND STRATEGY

—The Institute for Clinical and Economic Review (ICER), a drug price watchdog, concluded that the new migraine-prevention drug from Amgen (NASDAQ: AMGN) is cost-effective as long as patients have first tried and failed to respond to older, less expensive treatments. After the FDA approved erenumab (Aimovig) last month, Amgen set a $6,900 annual price.

—ICER also published a report reiterating its earlier conclusion that the price of the cystic fibrosis drugs sold by Vertex Pharmaceuticals (NASDAQ: VRTX) should be cut by up to 77 percent. ICER said Vertex “should abandon vague claims that prices are justified by the need to invest in future research” and instead provide “a transparent, explicit justification” for its prices. Vertex has previously called ICER’s assessment a “sham.”

—Eli Lilly (NYSE: LLY) won FDA approval for rheumatoid arthritis drug baricitinib (Olumiant) at a lower dose than it hoped for. The FDA also slapped a warning on its label about potential health risks. Those limitations will make it difficult to take market share from top-selling adalimumab (Humira).

—Fresh off an Alzheimer’s failure that cost top executives their jobs, Axovant Sciences (NASDAQ: AXON) rebooted as a gene therapy company, starting with a Parkinson’s disease treatment it licensed from Oxford Biomedica for $30 million. Axovant tapped Spark Therapeutics (NASDAQ: ONCE) co-founder Fraser Wright to run its gene therapy work. Shares nearly tripled to close Thursday at $4.80 apiece.

—Activist shareholders at Allergan (NYSE: AGN) who own nearly 2 percent of the company are advocating that it split the CEO and chairman roles, both held by Brent Saunders, and rethink its aggressive acquisition strategy.

—Ex-FDA top drug official John Jenkins, who left the agency after its controversial decision to approve Sarepta Therapeutics’s (NASDAQ: SRPT) Duchenne muscular dystrophy drug, joined the board of Corbus Pharmaceuticals (NASDAQ: CBRP).

DATA & DEALS

—Biogen (NASDAQ: BIIB) and partner Eisai reported that in a small, randomized Phase 2 test, their experimental Alzheimer’s treatment elenbecestat lowered levels of amyloid beta plaques in the brain. But a “numerical slowing of decline” in patients’ cognition, as the companies noted in a statement, was not statistically significant. The drug is already in Phase 3, with results expected in 2020.

—The experimental cystic fibrosis drug PTI-801 from Proteostasis Therapeutics (NASDAQ: PTI) failed to improve patients’ lung function when combined with ivacaftor/lumacaftor (Orkambi), from Vertex, in an early stage study (compared to Orkambi and a placebo). Proteostasis shares fell 38 percent. The company is still using PTI-801 in experimental two- and three-drug regimens for cystic fibrosis, which represent a possible future threat to Vertex’s dominant CF franchise if effective.

—Roche will scrap experimental cancer drug taselisib, a PI3 kinase inhibitor, after disappointing results in a breast cancer study. Here’s more from Fierce Biotech.

—To add to its neurology pipeline, Biogen agreed to pay $4 million ufront for the option to acquire a drug that Japan-based TMS is testing in mid-stage studies as a treatment for stroke.

Ben Fidler and Frank Vinluan contributed to this report.

Map by Dave Winer.