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Bio Roundup: Surprise News, a Gene Therapy Mess & a CAR-T Step

Xconomy National — 

Gene therapy has become one of the hottest fields in biomedicine, with two treatments approved in the US already and several more on the way. But surprise news this week regarding Zolgensma, the recently approved spinal muscular atrophy treatment, cast a cloud over its progress.

The FDA revealed that Novartis subsidiary AveXis had “manipulated” data underlying the review of Zolgensma, and only told the agency after it had approved the treatment in May. The FDA won’t take Zolgensma off the market—it remains convinced of its safety and effectiveness—but it could sue those involved, or alter its opinion of the data pending an investigation.

Novartis promptly and fiercely defended Zolgensma’s clinical track record, and its own actions over the past months, among them an internal investigation and plans to fire people involved in the alleged malfeasance. But the mess leaves several unanswered questions as the FDA conducts its query. Among them: How deep did the data manipulation go? What will the fallout be for patients and families clamoring to get Zolgensma? How does it affect the decisions of payers who are forming policies to cover the drug’s $2 million price tag? And will there be any ripple effects for other gene therapy developers?

As the first gene therapy to ever go head to head with an established product—Spinraza, from Biogen (NASDAQ: BIIB)—Zolgensma is already making history. But what happens next could be every bit as important.

Biotech had a few more twists and turns this week. We’ve got those details and much more below.


—The FDA said that data underlying the approval of the spinal muscular atrophy gene therapy Zolgensma had been “manipulated” by its developer, Novartis (NYSE: NVS) subsidiary AveXis. The issue concerned mouse testing that supports the process Novartis uses to make Zolgensma, not patient data, and the FDA said it “remains confident” that the treatment should stay on the market. But an investigation is underway and the agency may “amend [its review] as appropriate.”

—A day after the announcement, Novartis held a conference call with analysts defending its actions. CEO Vas Narasimhan said that despite the fact that the company learned of the falsified data before Zolgensma was approved and didn’t tell the FDA until afterwards, the company “tried to do the right thing” and is getting rid of the AveXis scientists to blame for the fiasco. Here’s more from STAT.

—The nonprofit spinal muscular atrophy patient advocacy group Cure SMA has also begun its own investigation. It has asked Novartis and AveXis for a report on the nature of the manipulated data, and whether they were presented to patients and families.

—Meanwhile, it was a tumultuous Thursday for another gene therapy developer, Sarepta Therapeutics (NASDAQ: SRPT). The FDA’s adverse event database showed that a patient in a trial of its Duchenne muscular dystrophy gene therapy was hospitalized in February with rhabdomyolysis, a breakdown in muscle tissue that can lead to serious health problems. Shares quickly fell 13 percent, but then began to bounce back after Sarepta called the disclosure “erroneous.” The patient didn’t end up with any serious health problems, and may not have even gotten the gene therapy, Sarepta said.


—Less than two weeks ago, Amarin (NASDAQ: AMRN) said it was “unlikely” that the FDA would schedule an advisory panel meeting to debate whether it could expand the label of its prescription grade fish oil pill Vascepa to include claims that it can cut the risk of heart disease. On Thursday, the FDA did just that, setting a Nov. 14 meeting and delaying a decision on Amarin’s drug until “late December.” Shares plummeted about 20 percent.

—On a conference call, Nektar Therapeutics (NASDAQ: NKTR) executives disclosed that some patients taking its cancer drug bempegaldesleukin in clinical trials got “suboptimal” product and that may have affected its results. The disclosure “reduces our confidence in the clinical signal and will likely diminish investor confidence in Nektar management,” wrote SVB Leerink analyst Daina Graybosch, and shares fell 31 percent.


—German drug maker Bayer agreed to acquire BlueRock Therapeutics, a company developing cell therapies for Parkinson’s and a variety of other diseases. BlueRock is one of multiple companies Bayer has started and funded with Versant Ventures; it’s paying $240 million up front, and potentially $360 million more, to buy out BlueRock’s remaining shares.

—The Centers for Medicare and Medicaid Services finalized a policy to cover pricey CAR-T cancer immunotherapies nationwide. But CMS administrator Seema Verma told the Washington Post that the agency is “extremely concerned” about how the healthcare system will pay for the pricey therapies over the long term.


—A GlycoMimetics (NASDAQ: GLYC) drug for sickle cell disease failed a Phase 3 trial, sending shares down more than 50 percent. Pfizer (NYSE: PFE) owns rights to the drug through a 2011 partnership.

—An experimental drug from Allakos (NASDAQ: ALLK) met all of its goals in a Phase 2 trial in patients with either eosinophilic gastritis or eosinophilic gastroenteritis, two rare gastrointestinal disorders. Shares nearly tripled, from $31.05 to $83.81 apiece.

—Guardant Health (NASDAQ: GH) published a study in the journal Clinical Cancer Research showing that its blood-based cancer diagnostic can detect tumors with microsatellite instability-high (MSI-H) or mismatch repair deficiency (dMMR), defects that make them more vulnerable to immunotherapies like pembrolizumab (Keytruda).

—Australian biotech Opthea reported that its wet age-related macular degeneration drug OPT-302, when combined with ranibizumab (Lucentis), was better than Lucentis alone in a Phase 2 study.

—The Merck (NYSE: MRK) and AstraZeneca (NYSE: AZN) drug olaparib (Lynparza) succeeded in a Phase 3 trial in a subset of patients with metastatic castration resistant prostate cancer—a first for a relatively new class of drugs known as PARP inhibitors.

—-Rhythm Pharmaceuticals (NASDAQ: RYTM) reported that setmelanotide, an experimental drug for rare genetic obesity disorders, aced two Phase 3 studies. The Boston company plans to file for FDA approval in two diseases, pre-opiomelancortin deficiency and Lepin receptor deficiency.


—The FDA extended its review of experimental Intra-Cellular Therapies (NASDAQ: ITCI) schizophrenia drug lumateperone by three months, giving it more time to review safety data from animal tests.

—The agency rejected a dry eye disease medication from Kala Pharmaceuticals (NASDAQ: KALA), pending data from an additional trial. Kala has another Phase 3 study underway with results expected next year.

—An FDA advisory panel voted 16 to 2 to recommend approval of Gilead Sciences (NASDAQ: GILD) drug Discovy for HIV prevention in men and transgender women.

—European regulators approved the Regeneron Pharmaceuticals (NASDAQ: REGN) and Sanofi (NYSE: SNY) drug dupilumab (Dupixent) for adolescents with moderate to severe eczema. Dupixent also succeeded in a Phase 3 trial in six to 11 year old eczema patients.

—Running out of cash to advance an experimental liver patch product to human testing, San Diego-based Organovo (NASDAQ: ONVO) announced a corporate restructuring that could lead to a sale of the business or a merger with another company.

—Last month, Mallinckrodt (NYSE: MNK) announced it would spin off its generics subsidiary, which markets opioids. This week, the drug maker suspended those plans, citing market conditions that include “increasing uncertainties created by the opioid litigation.”


—Boston biotech Luca Biologics launched with a pipeline of preclinical microbiome treatments for women’s health.

—Neoleukin (NASDAQ: NLTX), a University of Washington spinout making protein drugs, went public via a reverse merger with Aquinox Pharmaceuticals of Vancouver, BC.

—A new report from CB Insights showed that healthcare startups around the world raised a record $26.9 billion through 2,258 deals in the first half of 2019.

—Bridge Bio (NASDAQ: BBIO) offered to buy out the shares of Eidos Therapeutics (NASDAQ: EIDX) it doesn’t already own in a stock deal that values Eidos shares at $38.31 apiece.

—Nonalcoholic steatohepatitis drug developer 3-V Biosciences of San Mateo, CA, changed its name to Sagimet Biosciences and announced the second tranche of its Series E funding, which now totals $25 million.

—Exo Imaging raised a $35 million Series B to develop smaller, more affordable ultrasound diagnostic tools.


—Former Gilead executive John Hutchison was named CEO of Assembly Biosciences (NASDAQ: ASMB)… Ribon Therapeutics hired Sudha Parasuraman as chief medical officer… Teva Pharmaceutical (NYSE: TEVA) chief financial officer Mike McClellan is stepping down… Rafael Amado left Adaptimmune for the chief medical officer job at Allogene Therapeutics (NASDAQ: ALLO)… Genentech’s Priti Hegde was appointed chief scientific officer of Foundation Medicine… former Achaogen executive Janet Dorling joined CymaBay Therapeutics (NASDAQ: CBAY) as chief commercial officer… Editas Medicine (NASDAQ: EDIT) named interim CEO Cynthia Collins its full-time president and CEO.

Frank Vinluan contributed to this report.