• Rafael Holdings subsidiary Cyclo Therapeutics submitted an FDA application for remibetadex to treat Niemann-Pick disease type C in adults and children from birth.
• The Phase 3 trial missed its primary endpoint, showing a 64% relative slowing of disease progression versus placebo (p=0.19).
• A prespecified subgroup receiving background therapy, approximately 83% of participants, showed a 71% relative slowing of progression (p=0.046).
• A survival analysis linked remibetadex to an 85% lower mortality risk in infantile-onset patients versus matched registry controls (p=0.044).
• Serious adverse events occurred in 35.9% of remibetadex recipients versus 16.7% with placebo; no new safety signals emerged.

Disclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. Rafael Holdings Inc. published the original content used to generate this news brief via GlobeNewswire (Ref. ID: 202610290700PRIMZONEFULLFEED9855853) on October 29, 2026, and is solely responsible for the information contained therein.