Innovative Advances in Niemann-Pick Disease Treatment Showcase

Exciting Opportunities in Niemann-Pick Disease Research

Rafael Holdings, Inc. (NYSE: RFL; NYSE American: RFL-WT) is making waves with its subsidiary Cyclo Therapeutics, which has had the honor of having two insightful abstracts selected for presentation at a prestigious international event focused on inborn errors of metabolism. This year, the International Congress of Inborn Errors of Metabolism (ICIEM) is taking place in Kyoto, Japan, and it runs from September 2 to 6. Rafael Holdings is particularly excited about the potential discussions surrounding innovative treatments for Niemann-Pick Disease Type C1 (NPC1), a rare but severe genetic disorder.

Presentation Details to Watch For

Rafael Holdings is set to present cutting-edge research that highlights the advancements made with Trappsol Cyclo™. The presentations include:

Oral Presentation

Title: Hydroxypropyl-beta-cyclodextrin for the Long-Term Treatment of Niemann-Pick Type C1: Efficacy and Safety Data from 4 Clinical Studies and the Ongoing Expanded Access Program.
Presenter: Dr. Caroline Hastings, a distinguished pediatric hematologist and professor, will share valuable insights on Wednesday, September 3, 2025, from 14:00 - 16:00 (JST) in Room 3 at the Kyoto International Conference Center.

Poster Presentation

Title: Trappsol Cyclo Open Label Treatment in the TransportNPC Sub-Study in Patients Under the Age of 3 Diagnosed with Niemann-Pick Disease Type C1.
Poster No: P-75
Presenter: Dr. Orna Staretz Chacham, an expert in neonatal care and rare diseases. This presentation is scheduled for Wednesday, September 3, 2025, from 18:00 - 19:00 (JST) at Poster 1, Annex Hall.

Impact of Trappsol Cyclo™ on NPC1

Trappsol Cyclo™ represents a significant breakthrough in treatment methods for NPC1, functioning by mobilizing lysosomal cholesterol. This intravenously administered medication could alter the trajectory of this disorder by directly addressing the cholesterol accumulation that leads to various organ damages, particularly affecting the brain, liver, and spleen.

Understanding Niemann-Pick Disease Type C1

Niemann-Pick Disease Type C1 is characterized by genetic mutations impacting cholesterol transport, resulting in severe health complications and often premature mortality. Symptoms include cognitive decline, impaired motor functions, and other debilitating issues. Awareness and education about this disease are essential as around 1 in 100,000 live births are affected globally.

The Pioneering Phase 3 TransportNPC Study

The ongoing Phase 3 TransportNPC study is crucial as it evaluates the safety and efficacy of Trappsol Cyclo™. Currently, 94 patients aged 3 years and older are participating, and the study is structured as a randomized, double-blind, placebo-controlled trial. This design aims to ensure that the results can be widely accepted and trusted.

Moreover, an intriguing aspect of this study is an open-label phase tailored for babies and toddlers aged from birth to 3 years to better understand treatment implications in early childhood. This sub-study has enlisted ten participants so far.

About Rafael Holdings, Inc.

Operating at the forefront of biotechnology, Rafael Holdings, Inc. has invested in several innovative pharmaceutical ventures, including Cyclo Therapeutics. They are committed to developing effective treatments for serious medical conditions. Their portfolio includes other partnerships, such as with LipoMedix Pharmaceuticals and Rafael Medical Devices, each focused on different aspects of healthcare innovation.

Conclusion: Future of Niemann-Pick Disease Treatment

As the presentations at ICIEM draw near, the anticipation surrounding Trappsol Cyclo™ only continues to grow, representing hope for families affected by Niemann-Pick Disease Type C1. With ongoing research and clinical trials, Rafael Holdings is pioneering pathways that could result in significant improvements in patient outcomes and lives.

Frequently Asked Questions

What is the significance of the presentations at ICIEM?

The presentations will showcase important research on Niemann-Pick Disease Type C1 treatments, highlighting advancements in the understanding and management of the condition.

Who is presenting at the ICIEM?

Dr. Caroline Hastings and Dr. Orna Staretz Chacham are the key presenters, each bringing extensive expertise in their respective fields.

What is Trappsol Cyclo™?

Trappsol Cyclo™ is a proprietary formulation aimed at treating Niemann-Pick Disease Type C1 by mobilizing lysosomal cholesterol.

How is the TransportNPC study structured?

The TransportNPC study is a randomized, double-blind, placebo-controlled trial designed to evaluate the efficacy and safety of Trappsol Cyclo™.

What are the goals of the ongoing research?

The primary goal is to assess the effect of Trappsol Cyclo™ on clinical severity and disease progression in NPC1 patients.

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