Biotech Roundup: Gottlieb v. Juul, Solid Slumps, Avidity Gets $100M & More

Xconomy National — 

Lung injuries connected to the use of electronic cigarettes have topped 2,000 and killed 40 people, according to the Centers for Disease Control and Prevention.

It’s still unclear whether the cases are tied to legally sold products or devices modified by e-cigarette users, but the CDC this week identified a “potential toxin of concern”—Vitamin E acetate, which health officials detected in all 29 lung tissue samples from patients that they tested.  The compound is used as an additive in THC-containing vaping products, and THC has been found in most of the samples the FDA has tested in connection to the illnesses.

There may be more than one cause of these illnesses, and the CDC continues its investigation. But this week, former FDA commissioner Scott Gottlieb said products made by market leader Juul should be removed from the market entirely. “It’s very clear that Juul can’t keep their products out of the hands of kids,” he told CNBC. “It could be that this product can’t exist on the market anymore.”

In other news this week, the FDA halted a gene therapy clinical trial, an Ebola vaccine won regulatory approval, and a biotech startup raised $100 million. Those stories and more in this week’s roundup.


—For the second time in less than two years, the FDA stopped a Solid Bio (NASDAQ: SLDB) clinical trial testing a gene therapy for Duchenne muscular dystrophy due to safety concerns. Solid’s stock plummeted 69 percent on the news.

Roche drug risdiplam achieved the goals of a pivotal study in spinal muscular atrophy, better positioning the experimental medicine as a potential competitor to a Biogen (NASDAQ: BIIB) product.

—Celgene (NASDAQ: CELG) and Acceleron Pharma (NASDAQ: XLRN) won FDA approval for luspatercept (Rebloyzl), a treatment for anemia in patients who have the blood disease beta thalassemia.

—A Reata Pharmaceuticals (NASDAQ: RETA) succeeded in a Phase 3 study testing it Alport syndrome, a rare, inherited kidney disorder. The Plano, TX, company said it would file for FDA review of its drug.

—San Francisco, CA-based FibroGen (NASDAQ: FGEN) and partners AstraZeneca (NYSE: AZN) and Astellas Pharma reported safety data for roxadustat, an experimental treatment for anemia associated with chronic kidney disease. FibroGen said the data showed that the cardiovascular safety was comparable to a placebo and to an anemia injection already in use.

—Regenxbio (NASDAQ: RGNX) sued the FDA over the partial clinical hold the agency placed on tests of an experimental gene therapy, and the full clinical hold placed on another. FDA Law Blog was the first to report the news.

—A Merck (NYSE: MRK) Ebola vaccine received conditional approval from the European Commission for use in the EU and some additional countries. The FDA is also reviewing the vaccine.

—The merger of Mylan (NASDAQ: MYL) and Pfizer (NYSE: PFE) unit Upjohn now has a name. When the deal closes sometime next year, the combined business will be called Viatris.

—An FDA advisory panel voted unanimously to recommend that approval of Amarin (NASDAQ: AMRN) pill Vascepa, which is derived from fish, be expanded to include the reduction of cardiovascular problems in high-risk patients. An FDA decision is expected by Dec. 28.


—Novo Nordisk (NYSE: NVO) added a potential NASH treatment to its pipeline through a licensing deal it inked with Ube Industries in Japan.

—Roche struck a deal to acquire Lexington, MA-based Promedior and its clinical-stage fibrosis drug, for $390 million up front.

—Sarepta Therapeutics (NASDAQ: SRPT) started a gene therapy R&D partnership with StrideBio aiming to develop treatments for central nervous system disorders and muscular conditions. Sarepta is paying Research Triangle Park, NC-based StrideBio $48 million in cash and stock up front.

—Foamix Pharmaceuticals (NASDAQ: FOMX) and Menlo Therapeutics (NASDAQ: MNLO) agreed to an all-stock merger, forming a company focused on skin drugs. The combined business will be majority-owned by Foamix shareholders.

—Merck acquired Calporta, a biopharma startup created as part of a collaboration between San Diego investment firm Avalon Ventures and GlaxoSmithKline (NYSE: GSK), in a deal worth up to $576 million.

—Medical genetics company Invitae (NYSE: NVTA) agreed to acquire San Francisco software startup Clear Genetics for $50 million in cash and stock. Clear Genetics has developed a chatbot for patients undergoing genetic testing and other software tools.

—Sandoz, the generics and biosimilar division of Novartis (NYSE: NVS), struck a deal with Aspen Global Incorporated (AGI) to acquire its Japanese business for about $330 million.


Avidity Biosciences closed a $100 million Series C round to continue development of its pipeline of antibody-oligonucleotide conjugate drugs for rare muscular disorders.

—Artificial intelligence-powered startup X-37 closed a $14.5 million Series A round to fund its drug discovery research.


Join us in San Francisco on Nov. 19 for the latest in our Xchange series of events. What’s Next in Neuroscience Therapies will feature speakers from the Christopher & Dana Reeve Foundation, the California Institute for Regenerative Medicine, BlackThron Therapeutics, and Alector (NASDAQ: ALEC).

—Tune in on Nov. 20 at 2 p.m. ET for the latest in the Xconomy Insight digital health webinar series, which will focus on the impact of AI on drug discovery and development. Hear from Echo Health Ventures partner Jessica Zeaske and Celsius Therapeutics CEO Tariq Kassum. Register here.


—Neurana Pharmaceuticals named Randall Kaye its chief medical officer… TransEnterix (NYSE American: TRXC) CEO Todd Pope is stepping down, succeeded by Anthony Fernando… Prakash Raman left the Novartis Institutes for BioMedical Research to become senior partner and chief business development officer of Flagship Pioneering… and Equillium (NASDAQ: EQ) announced Bruce Steel, its president and chief business officer, would succeed CEO Dan Bradbury come Jan. 1.

Frank Vinluan contributed to this report.

Photo by Flickr user Ecig Click via a Creative Commons license