Bio Roundup: FDA Says No, PCSK9 Value Fight, CRISPR Intrigue & More

Xconomy National — 

When the Olympics roll around, many watchers get into a sport they previously hadn’t thought much about. This time for me, it’s volleyball.

The men’s and women’s games, both indoor and beach versions, are fast, aggressive, with lightning-quick adjustments and subtle teamwork required every time a ball is dug out and popped in the air. The best part, though, are the rejections. Just like in basketball, there’s nothing more dramatic than emphatically denying an opponent a chance to score. And once in a while, a defender uses his or her face, inadvertently, to block a ball spiked over the net. Talk about sacrifice.

If drug approval were an Olympic sport, rejections would also be high drama. All those years of hard work to reach the promised land, then just as a drug owner is ready to spike it home and take a victory lap, the FDA’s hands rise over the net… OK, it’s not a perfect analogy, but it’s better than comparing the patent fight over the gene editing system CRISPR-Cas9 to table tennis. (Be our guest and try it.)

The CRISPR news this week doesn’t need a metaphor; the drama that MIT Technology Review reported can stand alone. We’ll get to that, and to a busy week of drug rejections, trial failures, lawsuits, and more. Time for the gold-medal roundup.

—Rejections: The FDA denied approval of Chiasma’s lead drug, for acromegaly, in April and asked for more data. Waltham, MA-based Chiasma (NASDAQ: CHMA) announced plans to slash 44 percent of its workforce… Portola Pharmaceuticals (NASDAQ: PTLA) of South San Francisco, CA, said the FDA needs more information to make an approval decision about andexanet alfa (AndexXa), an emergency drug that reverses the effect of blood thinners. Shares were down 14 percent mid-day Thursday.

—The FDA last summer approved two new cholesterol fighting drugs known as PCSK9 inhibitors, but so far the market has rejected them, resulting in paltry sales. Everyone is waiting until 2017, when huge studies of the drugs’ ability to prevent heart attacks and strokes start to provide data. Meanwhile, the latest study reporting that the drugs’ $14,000-a-year list price isn’t cost effective, published this week in the Journal of the American Medical Association, has the drugs’ owners, Amgen (NASDAQ: AMGN) and Regeneron Pharmaceuticals (NASDAQ: REGN), up in arms.

MIT Technology Review reported this week that a graduate student in the lab of Feng Zhang, Broad Institute gene-editing star, had accused the Broad of misleading the U.S. Patent Office in its successful push to get pioneering patents for the CRISPR-Cas9 gene-editing technology. The student, Shuailiang Lin, was in Zhang’s lab at the time but has since moved on to the University of California, San Francisco. Lin made his claims in an email to University of California, Berkeley professor Jennifer Doudna, Zhang’s rival in the dispute over who invented key aspects of CRISPR-Cas9. The email has surfaced in Berkeley’s filings as part of its patent fight with the Broad. The fight is more expensive than expected: Editas Medicine (NASDAQ: EDIT), which has licensed the Broad patents, has spent nearly $11 million this year so far on legal fees helping the Broad and Harvard University. STAT found the figures in Editas’s quarterly earnings report.

—In other CRISPR news, Bayer and CRISPR Therapeutics have christened Casebia Therapeutics, the name for the joint venture the two established through their $335 million partnership last year. Casebia is developing gene editing drugs for blood disorders, blindness, and congenital heart disease, and has leased 33,000 square feet of lab space in Cambridge to set up shop.

— Radius Health (NASDAQ: RDUS) published Phase 3 trial data for its experimental osteoporosis drug abaloparatide, which is currently under FDA review. Amgen is expected to ask FDA for approval of a rival osteoporosis drug, romosuzumab, later this year. Here’s more on Radius’s drug and osteoporosis care from the New York Times.

—More Phase 3 news: Boston-based Vertex Pharmaceuticals (NASDAQ: VRTX) said one of the four Phase 3 trials testing the experimental cystic fibrosis drug VX-661 with ivacaftor (Kalydeco) failed and is being shut down. The combination is meant to improve upon Vertex’s approved pairing of CF drugs ivacaftor-lumacaftor (Orkambi), and Vertex is testing it in a variety of genetic subsets of CF patients. Data from the other three trials should come in late 2016 or early 2017.

—Terrible (Phase) Twos: Shares of Waltham-based Cerulean Pharma (NASDAQ: CERU) fell more than 60 percent after its nanoparticle cancer drug, CRLX101, failed its second Phase 2 trial, this time in kidney cancer. Cerulean later announced plans to slash 48 percent of its workforce by the end 2016… A cancer drug developed by OncoGenex Pharmaceuticals (NASDAQ: OGXI) also flubbed a second Phase 2 trial, leading the Bothell, WA, company to explore … Next Page »

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