Rafael announces continuation of Phase 3 TransportNPC study following DMC review
Clinical Trial Progress: Rafael Holdings' subsidiary Cyclo Therapeutics will continue its 96-week phase 3 TransportNPC study for Niemann-Pick Disease Type C1 after a positive review from the Data Monitoring Committee at the 48-week interim analysis.
Regulatory Developments: The FDA has accepted the statistical analysis plan for the TransportNPC study, highlighting the company's commitment to advancing its investigational candidate towards regulatory approval and commercial readiness.
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Rafael Holdings Reports $9.8M Net Loss in Q1, R&D Expenses Rise to $7.5M
- Financial Performance: As of October 31, 2025, Rafael Holdings reported a net loss of $9.8 million for Q1, translating to a loss of $0.19 per share, which is an increase from last year's $9.0 million and $0.37 per share, reflecting the impact of consolidating Cyclo Therapeutics' expenses post-acquisition.
- Surge in R&D Expenses: Research and development expenses soared to $7.5 million this quarter, compared to $1.3 million in the same period last year, primarily due to the inclusion of Cyclo's costs, indicating the company's ongoing commitment to drug development.
- Cash Flow Status: As of October 31, 2025, the company had cash and cash equivalents of $45.5 million, down from $52.8 million on July 31, 2025, highlighting liquidity pressures amid ongoing losses.
- Clinical Trial Progress: Rafael Holdings' Trappsol®Cyclo™ received a recommendation to continue from the Data Monitoring Committee in its pivotal Phase 3 trial for Niemann-Pick Disease Type C1, suggesting the drug could become a significant new treatment option for this rare and fatal genetic disorder.
Rafael Holdings Announces Continuation of its Phase 3 Study for the Treatment of Niemann-Pick Disease Type C1 (NPC1) Following Independent Data Monitoring Committee (DMC) Review of Prespecified 48-Week Interim Data
Continuation of Phase 3 Study: Rafael Holdings' subsidiary, Cyclo Therapeutics, will continue its pivotal phase 3 TransportNPC study for Trappsol® Cyclo™ in treating Niemann-Pick Disease Type C1 (NPC1) after a positive interim analysis by an independent Data Monitoring Committee (DMC).
Trappsol® Cyclo™ Overview: Trappsol® Cyclo™, administered intravenously, aims to address the root cause of NPC1 by mobilizing cholesterol from cells, and has shown a favorable safety profile consistent with earlier studies.
