Rafael Holdings Advances Phase 3 Study for NPC1 Treatment

Rafael Holdings ( (RFL) ) just unveiled an announcement.

On June 18, 2025, Rafael Holdings announced the continuation of its 96-week pivotal phase 3 TransportNPC study, evaluating Trappsol® Cyclo™ for Niemann-Pick Disease Type C1 (NPC1), following a positive review of safety and efficacy data at the 48-week interim analysis by an independent Data Monitoring Committee. This decision, along with the FDA’s acceptance of the statistical analysis plan, highlights the company’s commitment to advancing its investigational candidate through late-stage clinical development, potentially impacting global regulatory and commercial readiness. The study is significant as it is the most comprehensive trial for NPC1, involving 94 patients across 13 countries, and aims to address the need for effective treatments for this rare and fatal genetic disease.

Spark’s Take on RFL Stock

According to Spark, TipRanks’ AI Analyst, RFL is a Neutral.

Rafael Holdings’ overall score reflects significant financial challenges, including declining revenues and negative profitability, which weigh heavily on its financial performance. While the technical analysis also indicates bearish signals, the recent merger with Cyclo Therapeutics provides a potential positive outlook by strengthening strategic assets and leadership. However, the valuation remains unattractive with a negative P/E ratio. Overall, the company’s current financial struggles and weak technical indicators are only partially offset by recent strategic moves, resulting in a moderate stock score.

To see Spark’s full report on RFL stock, click here.

More about Rafael Holdings

Rafael Holdings, Inc. is a biotechnology company with interests in clinical and early-stage pharmaceutical companies. It has a 100% interest in Cyclo Therapeutics, LLC, which is focused on developing Trappsol® Cyclo™, a lead clinical candidate for treating Niemann-Pick Disease Type C1 (NPC1). Rafael Holdings also holds interests in other pharmaceutical and medical device companies.

Average Trading Volume: 199,133

Technical Sentiment Signal: Buy

Current Market Cap: $101.8M

For an in-depth examination of RFL stock, go to TipRanks’ Overview page.