With two mRNA COVID-19 vaccines already approved for use in the U.S. and Johnson & Johnson’s (J&J) vaccine available through an emergency use authorization (EUA), a few members of the FDA’s Vaccines and Related Biologic Products Advisory Committee today questioned the grounds for granting an EUA for Novavax Inc.’s vaccine. Under the Federal Food, Drug and Cosmetics Act, EUAs are reserved for certain emergencies when there are no adequate, approved and available alternatives. Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, explained that the statute allows some leeway to meet an unmet need. In this case, since the J&J vaccine isn’t currently used as frontline, there are no options for people who may not want an mRNA vaccine. “We do have a problem with vaccine uptake that is very serious in the United States,” Marks said, adding that health officials are compelled to do anything they can to make people more comfortable with getting a COVID-19 vaccine. Having a protein-based alternative such as the Novavax product may help with that effort, he said.

Code raises a series A to develop Duchenne and diabetes therapies

Code Biotherapeutics Inc. has raised an upsized and oversubscribed series A financing to develop programs for treating rare and genetic diseases that include Duchenne muscular dystrophy and type 1 diabetes. The Philadelphia-based company’s platform is designed to deliver RNAi and gene therapies. The platform leverages targeting molecules such as peptides, antibodies and small molecules that bind to cell surface proteins expressed on target cells. Northpond Ventures led the round along with Amgen Ventures, Hatteras Venture Partners and UCB Ventures. Existing investors include New Enterprise Associates, 4BIO Capital, Cureduchenne Ventures, the JDRF T1D Fund, UPMC Enterprises and Takeda Ventures.

Immunos adds $74M in series B round to progress myeloid checkpoint inhibitors

Immunos Therapeutics AG closed a $74 million series B round to progress its first-in-class, multifunctional immuno-oncology drug candidate, IOS-1002, and to expand further its pipeline of biologic drugs based on immuno-stimulatory human leukocyte antigen (HLA) molecules. The Schlieren, Switzerland-based firm is also setting up a U.S. subsidiary in Gaithersburg, Md., in order to conduct future U.S. trials and to expand its international reach. IOS-1002 has completed IND-enabling studies, and the company is filing for approval in Australia to begin a first-in-human phase Ia trial in patients with solid tumors later this year.

Wall Street upbeat about Bluebird adcom briefing docs for two gene therapy prospects 

The market was responding favorably to mixed briefing documents posted by the U.S. FDA with regard to the upcoming review of Bluebird Bio Inc.’s two gene therapy prospects, with shares of the Somerville, Mass.-based company (NASDAQ:BLUE) trading midday at $3.30, up 32 cents, or 10%. The Cellular, Tissue and Gene Therapies Advisory Committee June 9-10 will sort data yielded by Bluebird’s elivaldogene autotemcel (eli-cel) for early active cerebral adrenoleukodystrophy in patients without a matched sibling donor, and betibeglogene autotemcel (beti-cel) for people with beta-thalassemia who require regular red blood cell transfusions. Beti-cell seemed to pass muster but reviewers sounded less than certain about eli-cel.

US FDA’s accelerated approvals: Time to reform?

The U.S. FDA’s accelerated approval path is front burner these days, what with Congress looking to modernize the path through provisions added to the must-pass user fee legislation, the controversy still boiling over the FDA’s accelerated approval last year of Biogen Inc.’s Alzheimer’s drug, Aduhelm (aducanumab), and a number of recent withdrawals of drugs granted accelerated approval years ago.

Henlius’ PD-1 drug shows positive data in phase III trial to treat ES-SCLC

Shanghai Henlius Biotech Inc.’s anti-PD-1 monoclonal antibody serplulimab has generated positive data in a phase III trial as a first-line treatment for extensive-stage small-cell lung cancer (ES-SCLC). “The study results demonstrated that serplulimab, in combination with chemotherapy, can significantly extend the median overall survival (OS) to 15.4 months when compared to the control group in first-line SCLC,” said Ying Cheng, the trial’s lead investigator. It was the first anti-PD-1 antibody to show OS benefit in untreated ES-SCLC patients, according to Henlius.

Three NMEs added, but U.S. approvals are at five-year low

Global regulatory activity has fallen slightly, partially due to a decline in pandemic activity, and U.S. FDA approvals of drugs and biologics are at their lowest levels in five years. Regulatory news tracked by BioWorld has reached 1,127 entries so far this year, reflecting a drop of about 2% from last year, while the FDA has approved 64 drugs and biologics, down from 76 by this point in 2021.

The BioWorld Insider Podcast: New therapies vie to change fatal course of amyotrophic lateral sclerosis

As multidisciplinary care extends survival and improves the quality of life for people with amyotrophic lateral sclerosis (ALS), can a host of new ALS drugs vying to slow its progression make a difference? A surprise extension of the U.S. FDA review for Amylyx Pharmaceuticals Inc.’s AMX-0035 to review new data, plus near-term readouts from a major ALS platform trial may soon provide answers. Join the BioWorld Insider podcast as we talk with executives from Amylyx and Clene Inc., and a key Penn State ALS expert on what the future of medicine may hold for ALS patients.

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