Rafael Holdings says Phase 3 TransportNPC trial of Trappsol Cyclo misses primary endpoint in Niemann-Pick type C

Bitget2026/09/30 11:02

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Rafael Holdings reported topline Phase 3 results for Trappsol Cyclo in Niemann-Pick disease type C; full results will be presented later.
The study missed its primary endpoint, although results favored Trappsol Cyclo over placebo on disease progression.
A prespecified subgroup on background miglustat and/or leucine showed a statistically significant benefit, supporting a differentiated regulatory narrative.
Safety was broadly consistent with prior studies, with no new safety signals reported.
The company kept its plan to file a U.S. NDA in the fourth quarter of 2026.

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: 202609300700PRIMZONEFULLFEED9836379) on September 30, 2026, and is solely responsible for the information contained therein.

Disclaimer: The content of this article solely reflects the author's opinion and does not represent the platform in any capacity. This article is not intended to serve as a reference for making investment decisions.

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Rafael Holdings says Phase 3 TransportNPC trial of Trappsol Cyclo misses primary endpoint in Niemann-Pick type C

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