What You Need To Know In Biotech: An ASCO Disappointment, Big Money In Brains And A New CF Drug

A flood of cancer drug data poured into the public domain, a huge neuroscience startup got financed, and an FDA advisory panel gave a green light to an important combo drug for cystic fibrosis.

Catch up on your biotech and pharmaceutical industry news highlights below:

ASCO DATA DUMP

The American Society of Clinical Oncology, the big annual gathering of cancer physicians to talk about advances in treatment, is coming up May 29-June 2 in Chicago. But all eyes in the pharmaceutical industry, and Wall Street, were on the ASCO website this week as conference organizers released short summaries of the clinical trial presentations to come. While the abstracts are often incomplete and out of date by the time the actual conference rolls around, they are often newsy and can move cancer drug stocks.

Many ASCO abstract watchers were disappointed with data from Puma Biotechnology. The company’s drug, neratinib, appeared to offer only the slimmest of benefits, if any, when compared with a placebo in a study of more than 2,800 patients with breast cancer that overexpresses the HER2 protein. See Matthew Herper’s story for more of the details.

Elsewhere on the ASCO agenda, the FT wrote up a quick summary with the drug names every self-respecting cancer R&D follower needs to know. There’s Bristol-Myers Squibb ’s ipilimumab (Yervoy) and nivolumab (Opdivo), as well as Merck ’s pembrolizumab (Keytruda), as well as a few other drugs from the class of “checkpoint inhibitors” that enable the immune system to do a better job of fighting tumors. Adam Feuerstein at TheStreet.com compiled a helpful rundown of 10 abstracts to watch in this emerging class of cancer therapy.

One experimental drug for multiple myeloma from Bristol-Myers and AbbVie , called elotuzumab, delivered a 30% reduction in cancer worsening or death when compared with a standard combo regimen that includes a big-selling drug from Celgene , as Feuerstein reported.

Any real improvement over standard treatment is news at ASCO, but the stock market didn’t move much on the data. None of this week’s top five stock movers in biotech tracked by FactSet (Revance Therapeutics, Ultragenyx Pharmaceutical, Radius Health, Blueprint Medicines, or Galapagos) was an ASCO newsmaker. “The stock moves for larger cap names may be more modest than we have seen in the past given the limited surprises in the abstracts and also a number of key abstracts still being under embargo until the meeting starts at the end of the month,” said Vamil Divan, an analyst with Credit Suisse, in a note to clients.

DENALI DEBUTS

A South San Francisco startup called Denali Therapeutics, inspired by the highest peak in North America and led by some prominent neuroscience veterans from Genentech , secured a truly gargantuan initial financing of $217 million. The company is backed by some of the same people who invested in last year’s hit cancer immunotherapy IPO—Juno Therapeutics. The idea is to use the better/faster/cheaper tools of DNA sequencing to help identify promising new avenues for treating common neurological diseases like Alzheimer’s and Parkinson’s. See Matthew Herper’s story on Forbes for more.

VERTEX TAKES ANOTHER BIG STEP IN CF

Despite some doubts in the stock market heading into this week, Boston-based Vertex Pharmaceuticals won a convincing 12-1 endorsement for its new cystic fibrosis combo drug ivacaftor and lumacaftor (Orkambi) from an FDA advisory panel. The new combo is designed to help about half of the 30,000 cystic fibrosis patients in the U.S. who have a genetic mutation called F508del. Vertex already has a hit on its hands in the original ivacaftor (Kalydeco), which was designed to treat for about 4% of cystic fibrosis patients with a certain genetic abnormality called G551D. The FDA usually follows the advice of its advisory panels, so most observers expect the combo will be cleared for sale sometime before the agency’s July 5 deadline to complete its review. See Andrew Pollack’s coverage in the New York Times for more.

STRING OF INSIDER SALES AT BLUEBIRD BIO

Bluebird Bio, an exciting new player in gene therapy based in Cambridge, Mass., has been an investor darling for a couple years, especially after it presented some promising clinical trial data for patients with beta-thalassemia last December at the American Society of Hematology. But this week, some investors were less enthused. Filings with the Securities and Exchange Commission showed that a number of insiders were selling quite a few of their shares through the usual 10b5-1 rules. Several executives cashed out at prices over $130 a share and in some cases over $160, according to SEC records. Bluebird, in its quarterly filing with the SEC on May 6, acknowledged the sales and said they were done within usual 10b5-1 rules, and company policy. Bluebird stock declined 5.7% this week. Hat tip to Red Acre Investments (@redacre on Twitter) for flagging the sales. Go here for a full list the Form 4 disclosures.

SAGE THERAPEUTICS DELIVERS FOR RARE SEIZURE DISORDER

Cambridge, Mass.-based Sage Therapeutics is developing a drug for a super-refractory status epilepticus (SRSE), a disease in which the brain is in a persistent state of seizure, and where there are no FDA-approved therapies. Sage had some encouraging news this week from a small, preliminary clinical trial. Sage reported that 77% of 22 evaluable patients hit the main goal of an early-to-mid-stage clinical trial, as they were successfully weaned off their anesthetic agents while the experimental drug was being given. That same number of patients remained stable, without having a recurrence, for 24 hours after being weaned off the experimental drug.

OREXIGEN BATTLES WITH INVESTIGATOR

San Diego-based Orexigen Therapeutics got into a hissing match with Cleveland Clinic researcher Steve Nissen, and its partner Takeda Pharmaceuticals, over a cardiovascular outcomes study for its obesity drug that was recently shut down. Once again, Matthew Herper of Forbes was all over this story, quoting Nissen as saying the company misled patients and investors. Orexigen stock dropped 27%, making it the biggest biotech decliner of the week, according to data compiled by FactSet.