Bio Roundup: FDA Approvals, Intrexon’s Shift, Melinta Bankruptcy & More

Xconomy National — 

In the week leading up to Christmas, six drugs won FDA approvals. The decisions covered insomnia, migraine, and several different forms of cancer.

The FDA’s final tally for the year totaled 48 new drug approvals. If you’re keeping score, that compares with 59 approvals in 2018 and 46 in 2017. Most of the nods went to small molecule drugs and cancer drugs continued to represent a significant chunk of the new approved medicines. For what it’s worth, the FDA didn’t say how many drugs it rejected in 2019, but the final week of the year included one such denial.

In other news to close out 2019 and kick off the new year, a billion-dollar merger was abandoned, a synthetic biology company announced a strategy shift, and a slew of biotechs reported data that they hope will earn them a spot on the 2020 list of approved therapies. Let’s get to those stories and more in the first biotech news roundup of the year.


—Illumina (NASDAQ: ILMN) and Pacific Biosciences (NASDAQ: PACB) abandoned their proposed $1.2 billion merger due to opposition by US and UK antitrust regulators.

—Synthetic biology company Intrexon (NASDAQ: XON) is selling most of its non-healthcare businesses and turning its focus to human health. The Maryland company is adopting the name of Precigen, its cell and gene therapy subsidiary that has cancer therapeutic candidates in early-stage clinical testing.

—Acceleron Pharma (NASDAQ: XLRN) is paying Fulcrum Therapeutics (NASDAQ: FULC) $10 million up front to kick off a partnership aiming to identify small molecule drugs to treat pulmonary disease.

—Pfizer (NYSE: PFE) is taking the reins on experimental hemophilia A treatment SB-525 after partner Sangamo Therapeutics (NASDAQ: SGMO) completed Phase 1/2 tests of the gene therapy. The pharmaceutical giant has already started enrolling patients in a Phase 3 study and will be responsible for further development.

—Antibiotics company Melinta Therapeutics (NASDAQ: MLNT) is looking for new owners after filing for Chapter 11 bankruptcy as part of a voluntary “restructuring support agreement” with its supporting lenders.

—Johnson & Johnson (NYSE: JNJ) subsidiary Janssen completed its $750 million acquisition of bermekimab, an XBiotech (NASDAQ: XBIT) drug candidate with potential applications in multiple skin disorders. The antibody drug is currently in mid-stage testing for atopic dermatitis and hidradenitis suppurativa.

—Tokyo-based Astellas Pharma acquired San Francisco immuno-oncology company Xyphos Biosciences in a deal worth $665 million.


—Intra-Cellular Therapeutics won FDA approval for lumateperone (Caplyta), a treatment for schizophrenia in adults. But the drug’s label carries a boxed warning stating that elderly patients treated with antipsychotic drugs have an increased risk of death.

—Weeks after an FDA advisory committee voted against recommending approval of Correvio Pharma (NASDAQ: CORV) atrial fibrillation drug vernakalant (Brinavess), the agency formally rejected the therapy because of safety risks. Vancouver, BC-based Correvio says it will ask to meet with the FDA to discuss a potential study to address the concerns.

—The FDA approved Eisai insomnia therapy lemborexant (Dayvigo), but with the recommendation that the US Drug Enforcement Administration classify the pill as a controlled substance. The drug’s label cautions that patients with a history of substance abuse may face greater risk of addiction to the Eisai drug.

—Allergan (NASDAQ: AGN) received FDA approval for acute migraine drug ubrogepant (Ubrelvy). The drug blocks a protein called calcitonin gene-related peptide, but unlike the injectable therapies previously approved in this class of new treatments, Allergan’s drug is a pill.

—Lynparza, a drug marketed by AstraZeneca (NYSE: AZN) and Merck (NYSE: MRK) for ovarian and breast cancers, won an additional FDA nod for pancreatic cancer. Like the earlier approvals, the decision covers cancer that has a particular genetic mutation.

—Health Canada approved Amarin Pharma (NASDAQ: AMRN) drug icosapent ethyl (Vascepa) for reducing the risk of cardiovascular events in certain high-risk patients who are already taking statins. The decision comes two weeks after the fish oil-derived pill won a similar approval from the FDA.

—Shares of Wave Life Sciences (NASDAQ: WVE) took a dip after the company reported disappointing early clinical trial results of its experimental Huntington’s disease drug. The Cambridge, MA, biotech is hoping for better data from tests of a higher dose of the drug.


—Seattle Genetics (NASDAQ: SGEN) filed for regulatory review of tucatinib, an investigational cancer drug for HER2-positive breast cancer.

—Roxadustat, a FibroGen (NASDAQ: FGEN) drug developed to treat anemia in patients with chronic kidney disease, joined the FDA’s review queue. The San Francisco company is collaborating on the drug with AstraZeneca (NASDAQ: AZN) in the US, China, and other markets, and with Astellas Pharma in other areas including Japan and Europe.

—BioMarin Pharmaceutical (NASDAQ: BMRN) submitted an application for FDA review of valoctocogene roxaparvovec, its experimental gene therapy for hemophilia A. The San Rafael, CA, company, says it is the first US regulatory submission for a gene therapy for any type of hemophilia.

—Chinese company Luye Pharma Group submitted an application for LY3005, an experimental treatment for major depressive disorder. The drug is part of a new class of antidepressants called triple reuptake inhibitors because they target three brain chemicals: serotonin, norepinephrine, and dopamine.

—Irvine-CA-based Urovant Sciences (NASDAQ: UROV), one of the former Roivant Sciences subsidiaries recently sold to Sumitomo Dainippon Pharma, asked the FDA to review its experimental drug vibegron, a once-daily treatment for patients with overactive bladder.

—Zosano Pharma (NASDAQ: ZSAN) of Fremont, CA, filed for FDA approval of Qtrypta, its proprietary form of migraine drug zolmitriptan. Instead of a pill, Zosano’s formulation is delivered via titanium microneedles coated with the drug.


—Rubius Therapeutics (NASDAQ: RUBY) appointed Christina Coughlin to serve as chief medical officer, taking over from Chris Carpenter, who will take on an advisory role.

Aridis Pharmaceuticals promoted Michael Nazak to chief financial officer, succeeding Fred Kurland, who has retired.

Melissa Fassbender and Sarah de Crescenzo contributed to this report.

Photo by Flickr user @yb_woodstock via a Creative Commons license