Zolgensma Gene Therapy Data Manipulation, GRF6019 Phase 2 Results in Mild to Moderate Alzheimer, Mind Moments Podcast

This week, Neurology News Network covered the statement that the FDA released announcing that early testing of Zolgensma was manipulated, positive top-line data from a phase 2 studying GRF6019 for mild-to-moderate Alzheimer disease, and the first episodie of NeurologyLive's Mind Moments podcast (transcript below).

Welcome to Neurology News Network. I’m Jenna Payesko. Let’s get into the news from this week.

The FDA has released a statement announcing that data from early testing of ON-A-SEMONO-GENE AB-PARVO-VEC, or Zolgensma, was manipulated. The agency said that the therapy, which is the first and only gene therapy approved for SMA in patients aged 2 years of age and younger with SMN1 mutations, should remain on the market while it assesses the situation.

AveXis Inc., a subsidiary of Novartis, informed the agency of the data manipulation on June 28, just over a month following the therapy's momentous approval. Of the entirety of the data submitted and reviewed by the agency, at this time, the manipulation concerns are confined to only a small portion which was performed in animals and submitted in the BLA. Thus far, the manipulated information does not change the FDA’s positive assessment of data from the human clinical trials.

Alkahest reported positive top-line data from its phase 2 clinical trial studying GRF6019, which is under investigation for mild-to-moderate Alzheimer disease. Investigators reported that dosing with the agent was safe and well-tolerated, and those receiving GRF6019 experienced no decline in cognition measured by the ADAS-Cog11 and the MMSE. A minor decline in function measured by the ADCS-ADL₂₃, and the CDR-SB was reported. These data, the group noted, demonstrate that subjects maintained cognitive and functional status throughout the 6-month period.

NeurologyLive is proud to introduce the first episode of the Mind Moments podcast, your resource for expert insights on the diagnosis, treatment, and management of neurologic disorders. It will feature exclusive interviews with leaders in the field discussing the latest research and disease management strategies across the breadth of neurology, including epilepsy, multiple sclerosis, Parkinson disease, dementia, sleep disorders, and more.

Episode 1, "State of CBD: Not All Cannabidiol Is Created Equal" features an interview with Dr. Anup Patel section chief of Pediatric Neurology at Nationwide Children’s Hospital in Columbus, Ohio, and associate professor of Clinical Pediatrics and Neurology at The Ohio State University College of Medicine. He discusses the current state of CBD in the US, and why increasing public access to the molecule via legalized marijuana is becoming a big problem for clinicians treating patients with serious neurologic disorders.

For more direct access to expert insight, head to neurologylive.com. This has been Neurology News Network. Thanks for watching.