Bio Roundup: Not-BIO Party Foul, CRISPR Drama, Sickle Cell Updates

Xconomy National — 

This week brought a sobering reminder of how far away we are from true gender equality in the life sciences. An industry party held during BIO’s annual meeting in Boston last week featured scantily clad women with company logos painted on their bodies—two years after the infamous party at the J.P. Morgan party in San Francisco that similarly sparked outrage, mea culpas, and initiatives to help advance womens’ careers in the life sciences.

Bio-Twitter erupted as details from the party spread, and BIO president John Maraganore reportedly vowed to kick any company that sponsors the event next year out of the group. That’s all well and good, but hopefully this time around, there are some teeth to those threats and the industry actually learns its lesson. More on that story and the rest of the week’s biotech headlines below.


—No, PABNAB is not a new oncology formulation. It stands for Party at BIO Not Associated with BIO, and BioCentury first reported its organizers decided to hire female dancers, some reportedly topless, effectively thumbing their noses at the outrage that stemmed from the infamous cocktail party at the 2016 J.P. Morgan healthcare conference that featured hired models in short dresses. It didn’t stop there. Some dancers had the party sponsors’ logos painted on their bodies, which sponsors said they were unaware would happen. (One of the sponsors, EBD Group, is owned by the same parent company as Xconomy.) Here’s more on the fallout from PABNAB from STAT and Bloomberg.


—June 19 is World Sickle Cell Day, and the next two weeks will bring updates from experimental treatments that patients hope eventually will add to the scant options available to them. Xconomy took a look at some of the treatments on the horizon and the questions they’ll face as they advance through clinical testing.

—The first of those updates comes from Bluebird Bio (NASDAQ: BLUE), which presented results Friday morning from a Phase 1 test of its experimental gene therapy LentiGlobin. While early and in a small number of patients, data show that recent manufacturing and other changes to the gene therapy, an effort to boost its effectiveness, seem to be working so far.


—At a hearing before the Senate Health Committee, Health and Human Services (HHS) Secretary Alex Azar encouraged drug makers to voluntarily drop prices and took aim at secretive rebate deals between biopharma companies and drug pricing middlemen.

—Congress awarded the National Institutes of Health $500 million to fight the nationwide opioid addiction epidemic. The agency’s leaders outlined how they’ll spend the money.

—After meeting with the FDA, Sage Therapeutics (NASDAQ: SAGE) outlined what could be a fast approval path for SAGE-217, an experimental treatment for both major depressive disorder and postpartum depression. In Phase 3 trials, Sage plans to test the drug as a two-week treatment, not a chronic therapy like other depression medicines. Separately, the company struck a licensing deal with Shionogi for rights to SAGE-217 in Japan, Taiwan, and South Korea.

—For the second time, Mylan (NASDAQ: MYL) failed in its bid to win FDA approval for its generic version of the GlaxoSmithKline (NYSE: GSK) lung drug Advair due to “minor deficiencies.” Mylan said more details will be explained when it receives the full correspondence from the FDA later this month.

—Merck’s (NYSE: MRK) cancer immunotherapy pembrolizumab (Keytruda) picked up two more FDA approvals, for advanced cervical cancer and primary mediastinal large B-cell lymphoma patients who have failed other therapies.

—With drug-resistant microbes on the rise, the FDA is open to new ideas to regulate and pay for new antibiotics. One proposal the agency says it will discuss with the Centers for Medicare and Medicaid Services is to let health providers pay for antibiotic doses the way large companies license and use software.

—The FDA approved venetoclax (Venclexta) to treat chronic and small lymphocytic lymphoma (CLL and SLL). The drug was developed by AbbVie (NYSE: ABBV) and Genentech.


—Two papers published in Nature Medicine revealed potential problems for CRISPR-Cas9 gene editing. First, edited cells might end up with a mutation to a gene, p53, that protects the body from cancer. Second, the gene editing method might not work well unless that protective gene is disabled. It’s unclear whether the findings apply to CRISPR therapies advancing toward human testing, investors sent shares of developers Editas Medicine (NASDAQ: EDIT), CRISPR Therapeutics (NASDAQ: CRSP), and Intellia Therapeutics (NASDAQ: NTLA) downward. Here’s more from The New York Times.


—Shares of Boston-based Flex Pharma (NASDAQ: FLKS) plummeted 70 percent after “oral tolerability concerns” caused the company to abruptly end two clinical trials of its experimental muscle cramp treatment FLX-787. The company, co-founded by serial biotech executive Christoph Westphal—who left the board in March—will cut much of its workforce and pursue strategic alternatives, including a sale.

—Allergan (NYSE: AGN) released encouraging data for its migraine pill atogepant, part of a class of drugs meant to head off migraine pain before it starts. The drug will now advance into a larger Phase 3 study.

—An Alzheimer’s drug in Phase 3 testing has joined the growing list of clinical trial failures for the disease. Partners Eli Lilly (NYSE: LLY) and AstraZeneca (NYSE: AZN) are stopping work on the drug, lanabecestat, after an independent committee concluded it wouldn’t succeed.


—Rumors that medical device giant Stryker (NYSE: SYK) plan to acquire Boston Scientific (NYSE: BSX) are apparently untrue. In a tersely worded regulatory filing, the Kalamazoo, MI, company denied acquisition talks with its rival.

–According to the Financial Times, Celgene (NASDAQ: CELG) president of hematology and oncology Nasim Ahmed blamed the FDA’s refusal to review the approval application for multiple sclerosis pill ozanimod on Receptos, which Celgene bought for $7.2 billion. Ex-Receptos CEO Faheem Hasnain fired back at Celgene in this Endpoints News story.

—Sanofi paid Cambridge, MA-based Translate Bio (NASDAQ: TBIO), which could soon go public, $45 million to develop messenger RNA vaccines for up to five unspecified infectious diseases. Sanofi is also aligned with BioNTech on mRNA cancer vaccines.

—Palo Alto, CA, startup creator BridgeBio Pharma is adding two more to its lineup. The first is Origin Biosciences, which will develop a compound from Alexion Pharmaceuticals (NASDAQ: ALXN) for olybdenum cofactor deficiency Type A, a rare metabolic disease. The second is CoA Therapeutics, which will test a treatment for kinase-associated neurodegeneration (PKAN), a rare nervous system disorder. BridgeBio didn’t disclose financial details.

—Johnson & Johnson (NYSE: JNJ) accepted a $2.1 billion bid for its blood glucose monitoring business, LifeScan. The sale to private equity firm Platinum Equity is expected to close by the end of the year.


—Don’t miss our latest New York biotech event next Thursday, “Bringing Back the Expats,” which will feature some New Yorkers who have gone on to biopharma prominence in other hubs. Here’s your last chance to grab a ticket.

Frank Vinluan and Alex Lash contributed to this report.