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Bio Roundup: Zolgensma Fallout, Duchenne Redux, Ebola Boost & More

Xconomy National — 

We learned last week that the FDA was investigating Novartis for manipulating animal data related to its $2 million-a-dose gene therapy Zolgensma. The activity took place at AveXis, the firm Novartis bought for $8.7 billion in 2018, and Novartis reportedly knew but failed to report it to the FDA before the agency approved Zolgensma in May. There are still many unanswered questions, such as when the data-cooking took place. Before Novartis bought AveXis? After?

AveXis spun out of Nationwide Children’s Hospital in Columbus, OH, where the Zolgensma program originated. The manipulation didn’t take place there, according to hospital spokesperson Gina Berrichia.

“To our understanding, the reported data manipulation did not occur in preclinical studies or clinical trials conducted at Nationwide Children’s,” Berrichia wrote in an e-mail to Xconomy late Thursday. “We have not been contacted by the FDA, Novartis, or Avexis regarding this matter.”

The fallout first intensified late last week, when a group of Democratic senators wrote to FDA commissioner Ned Sharpless that “anything short of a forceful response would signal a green light to future pharmaceutical misbehavior.” This week, Novartis said it had taken action against brothers Brian and Allan Kaspar, the scientific founder and research chief of AveXis, respectively, while the FDA was still reviewing Zolgensma. The Kaspars “haven’t been involved in any operations at AveXis since early May,” were replaced by Novartis veteran Page Bouchard on Aug. 5, and are no longer with the company, Novartis said in a statement. Former FDA commissioner Robert Califf penned an op-ed in Stat about the FDA’s unusually harsh response.

Before he cofounded AveXis, Brian Kaspar led the development of Zolgensma at Nationwide, which—thanks in large part to Zolgensma’s success—has rapidly become a gene therapy research powerhouse, as Xconomy detailed back in April.

Oh, and Novartis also said US pharma chief Fabrice Chouraqui will step down at the end of the month, but it doesn’t seem Zolgensma was within Chouraqui’s purview.

There’s plenty more news to round up this week, so let’s get to it.


—Angry about a Trump administration proposal to import cheaper drugs from Canada, Canadian officials are considering ways to thwart the plan. Stat has more.

—With the import issue in the spotlight, Sen. Chuck Grassley (R-IA), chairman of the Senate finance committee, called on the FDA to conduct more snap inspections of foreign drug-making facilities. The safety and quality of drug manufacturing in India and China has also recently been under scrutiny.

—A US judge last Friday rejected a challenge to Amgen (NASDAQ: AMGN) patents that protect its blockbuster autoimmune disease drug etanercept (Enbrel) from generic competition. Challenger Novartis, which wants to market a biosimilar in the US, will appeal the ruling.

—In the wake of last year’s revelation of the first babies to come from gene-edited embryos, scientists want to produce clearer rules about human embryo editing, Stat reported.


—Two experimental drugs produced better survival rates among people infected with Ebola virus in the Democratic Republic of the Congo than previously available treatments, prompting study leaders to put everyone on the new drugs. People with low levels of the virus had survival rates of 94 percent and 89 percent with the two experimental drugs, REGN-EB3 from Regeneron Pharmaceuticals (NASDAQ: REGN) of Tarrytown, NY, and mAb114 from Ridgeback Biotherapeutics of Miami.

—The Global Alliance for TB Drug Development, a New York nonprofit, won FDA approval for Pretomanid, a tablet to treat drug-resistant forms of tuberculosis. It’s the second approval under a new regulatory pathway to encourage more antibacterial and antifungal products.

—By next week, the FDA could also give the nod to up to four new drugs for Duchenne muscular dystrophy, bacterial pneumonia, cancer, and rheumatoid arthritis. Xconomy previewed the approval decisions and the significance of each drug, including Roche’s entrectinib (Rozlytrek), which got the agency’s green light late Thursday.

—Shares of Waltham, MA-based Deciphera Pharmaceuticals (NASDAQ: DCPH) doubled after the company reported success in a Phase 3 study of ripetinib, a drug for gastrointestinal stromal tumors. Deciphera will file for FDA approval next year.

— Sarepta Therapeutics (NASDAQ: SRPT) of Cambridge, MA, has yet to begin a study meant to confirm the benefits of Exondys 51, a Duchenne muscular dystrophy drug the FDA controversially approved in 2016 on a slim data set. Stat reports that Sarepta submitted plans for the trial in June, but the study wouldn’t wrap up until 2024, three years later than the FDA had required.

—The Merck (NYSE: MRK) and AstraZeneca (NYSE: AZN) PARP inhibitor olaparib (Lynparza) passed another Phase 3 trial, this time when added to standard-of-care bevacizumab (Avastin) as a maintenance treatment for ovarian cancer patients following chemotherapy.

—Researchers at the UK’s Wellcome Sanger Institute described how the bacterium C. difficile, which can infect the gut and cause deadly diarrhea, has evolved into two species, one of which is highly adapted to spread in hospital settings, thriving on humans’ sugar-rich diets and is also resistant to clean-up.


—In the months leading up to the June AbbVie (NYSE: ABBV) announcement of its $63 billion deal to acquire Allergan (NYSE: AGN), no other companies offered a competing bid, according to Allergan securities filings. Meanwhile, filings also show Allergan CEO Brent Saunders is in line for a $14.9 million payment when the deal closes.

—Blacksburg, VA-based Landos Biopharma, a portfolio company of biotech startup accelerator Xontogeny, closed $60 million in Series B financing to advance its lead drug into Phase 2 for ulcerative colitis and Crohn’s disease.

—Ultragenyx Pharmaceutical (NASDAQ: RARE) of Novato, CA, paid $20 million up front for the option to acquire GeneTx Biotherapeutics of Downers Grove, IL, and its preclinical treatment for Angelman syndrome, a rare neurological disorder.


—On Aug. 27, Xconomy will host an evening of discussions about the progress of healthcare in the genomics era at our “Turning Genomics into Better Health” forum in South San Francisco. Speakers include insitro CEO Daphne Koller, UCSF chief genomics officer Aleks Rajkovic, Sangamo Therapeutics CEO Sandy Macrae, and more. Check out the agenda and full speaker list here.

—Xconomy announced two new events in New York and Boston in October: The New York Breed of Startups, on Oct. 15, and Boston’s Life Science Disruptors on Oct. 17.


—Jack Bailey, president of US pharmaceuticals for GlaxoSmithKline (NYSE: GSK), will step down at the end of the year, succeeded by EMD Serono executive Maya Martinez-Davis … Christopher Barys left Philips Healthcare to become CEO of On Target Laboratories… former Acceleron Pharma (NASDAQ: XLRN) executive Robert Zeldin joined Immunovant as chief medical officer… and Francis Perier was named chief financial officer of SpringWorks Therapeutics.

Image courtesy of Joe Flintham via Creative Commons.

Ben Fidler and Frank Vinluan contributed to this report.