Full Press Release Details
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
Phase 3 TransportNPC study to continue based
on the independent DMC review of safety and efficacy data at prespecified 48-week interim analysis
Data on the investigational candidate Trappsol
Cyclo (hydroxypropyl-beta-cyclodextrin), indicates that it is well-tolerated and has a safety profile consistent with the previously
completed phase 1 and 2 studies and ongoing phase 1 open-label extension study
NJ - June 18, 2025 (GLOBE NEWSWIRE) - Rafael
Holdings, Inc. (NYSE: RFL; NYSE American: RFL-WT) announced today that its subsidiary Cyclo Therapeutics' 96-week pivotal phase
3 TransportNPC study evaluating intravenous (IV) Trappsol Cyclo for the potential treatment of Niemann-Pick Disease Type C1
(NPC1) will continue based on the independent Data Monitoring Committee (DMC) review of safety and efficacy data at the prespecified 48-week
interim analysis. Additionally, the Food and Drug Administration (FDA) has accepted the statistical analysis plan for the TransportNPC
study. These developments underscore the company's continuing commitment to advancing its lead investigational candidate through
late-stage clinical development to support global regulatory and commercial readiness.
"NPC is a rare, fatal, and progressive genetic disease, and there
is a need for safe and effective treatment that addresses its root cause," said Howard S. Jonas, CEO of Rafael Holdings. "The
recommendation made by the independent DMC to continue the study to 96 weeks, boosts our determination to the continued clinical evaluation
of the potential of TrappsolCyclo as a systemic and neurological treatment option for people living with NPC1. We
recently enhanced our financial position with the closing of a $25 million rights offering earlier this month which will support our strategic
"It is a privilege to lead and continue the TransportNPC study,
the most comprehensive, controlled pivotal study of an investigational therapy for NPC ever conducted in terms of patient size, global
footprint, duration, and clinical outcomes," commented N. Scott Fine, Chief Executive Officer of Cyclo Therapeutics. "We are
grateful to the study participants, their families, investigators, and clinical trial sites who are dedicated to this important research."
About Trappsol Cyclo (hydroxypropyl-beta-cyclodextrin)
Trappsol Cyclo (hydroxypropyl-beta-cyclodextrin) is a first-in-class
propriety cyclodextrin formulation administered intravenously (IV) that mobilizes lysosomal cholesterol. TrappsolCyclo is designed to
directly impact the root cause of Niemann-Pick Disease Type C1 (NPC1) by mobilizing cholesterol from late-stage endosomes and lysosomes.
TrappsolCyclo has also been shown to cross the blood-brain barrier after IV administration, suggesting that therapeutic concentrations
are reached in the central nervous system over the infusion time window. The potential clinical significance of those concentrations will
be evaluated based upon the results of the phase 3 TransportNPC study.
About the TrappsolCylco Study Program
The phase 3 TransportNPC study is a prospective, randomized, double-blind,
placebo-controlled study evaluating the safety and efficacy of TrappsolCyclo (hydroxypropyl-beta-cyclodextrin) administered intravenously
(2000 mg/kg dose every 2 weeks) in patients aged 3 years and older with confirmed diagnosis of Niemann-Pick Disease Type C1 (NPC1) (CTD-TCNPC-301;
NCT04860960). The TransportNPC study enrolled 94 patients in over 25 sites across 13 countries. The study duration is 96 weeks, an unblinded
interim analysis was reviewed by an independent DMC when all patients reached 48 weeks and recommended to continue the study for the full
96 weeks. A phase 3 open-label extension study of up to 96 weeks follows the interventional study.
The primary endpoints of the phase 3 TransportNPC study are the mean
change in the 4-domain NPC Clinical Severity Scale (4D-NPC-CSS) score in the United States and the 5-domain NPC Clinical Severity Scale
(5D-NPC-CSS) score in Europe. The 4D-NPC-CSS score (inclusive of ambulation, fine motor, speech, and swallow) and 5D-NPC-CSS score (inclusive
of ambulation, fine motor, speech, swallow, and cognition) are measures of NPC disease progression that look at items that patients with
NPC and their caregivers and physicians have identified as most relevant. Important secondary and exploratory endpoints will also be assessed
across measures of disease activity.
As part of the phase 3 study, a phase 3 open-label sub-study is being
conducted in NPC1 patients from birth to 3 years of age outside of the United States. Ten patients have been recruited and are continuing
in the study. Outcomes for the sub-study include safety, clinical, and caregiver impression of the disease.
Cyclo Therapeutics has completed 2 studies, including a phase 1 study
(CTD-TCNPC-101; NCT02912793) and a phase 2 study (CTD-TCNPC-201; NCT02912793). Patients who completed the phase 1 study continue to receive
TrappsolCyclo treatment in the ongoing phase 1 open-label extension study (CTD-TCNPC-102; NCT03893071).
About Niemann-Pick Disease Type C1 (NPC1)
Niemann-Pick Disease Type C1 (NPC1) is a rare genetic disease that
affects approximately 1 in 100,000 live births globally and often leads to premature death. NPC1 is characterized by an inability for
cells to transport and process cholesterol, resulting in excessive amounts of cholesterol accumulating and damaging affected organs, including
the liver, spleen, and brain. The disease can be life-limiting, with symptoms including progressive intellectual decline, loss of motor
skills, seizures, and dementia. Approximately 95% of individuals with NPC have mutations in the NPC1 gene, and 5% have mutations in the
About Rafael Holdings, Inc.
Rafael Holdings, Inc. is a biotechnology company with interests in
clinical and early-stage pharmaceutical companies including a 100% interest in Cyclo Therapeutics, LLC, a biotechnology company dedicated
to developing Rafael's lead clinical candidate, Trappsol Cyclo (hydroxypropyl-beta-cyclodextrin), which is being evaluated
in clinical trials, including an ongoing phase 3 trial for the potential treatment of Niemann-Pick Disease Type C1 (NPC1), a rare, fatal,
and progressive genetic disorder. Rafael also holds a majority interest in LipoMedix Pharmaceuticals Ltd., a clinical stage pharmaceutical
company, Barer Institute Inc., a wholly owned preclinical cancer metabolism research operation, a majority interest in Cornerstone Pharmaceuticals,
Inc., formerly known as Rafael Pharmaceuticals Inc., a cancer metabolism-based therapeutics company, a majority interest in Rafael Medical
Devices, LLC, an orthopedic-focused medical device company developing instruments to advance minimally invasive surgeries, and a majority
interest in Day Three Labs, Inc., a company which empowers third-party manufacturers to reimagine their existing cannabis offerings, enabling
them to bring to market better, cleaner, more precise and predictable versions by utilizing Day Three's technology and innovation
About Cyclo Therapeutics, LLC
Cyclo Therapeutics, LLC ("Cyclo") is a wholly owned subsidiary
of Rafael Holdings, Inc. (NYSE: RFL). Cyclo is a clinical-stage biotechnology company dedicated to developing life-changing medicines
through science and innovation for patients and families living with rare and neurodegenerative diseases. The company's investigational
drug Trappsol Cyclo (hydroxypropyl-beta-cyclodextrin), an orphan drug designated product in the United States and Europe,
is the subject of 4 formal clinical trials for Niemann-Pick Disease Type C1, a rare and fatal genetic disease, (www.ClinicalTrials.gov
NCT02939547, NCT02912793, NCT03893071 and NCT04860960). Cyclo is also conducting a phase 2b clinical trial using TrappsolCyclo intravenously
in early Alzheimer's disease (NCT05607615) based on encouraging data from an Expanded Access program for Alzheimer's disease
Forward-Looking Statements
This press release contains forward-looking statements within the meaning
of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters
of historical fact should be considered forward-looking statements, including without limitation statements regarding our expectations
surrounding the potential safety, efficacy, and regulatory and clinical progress of our product candidates; plans regarding the further
evaluation of clinical data; and the potential of our pipeline, including our internal cancer metabolism research programs. These statements
are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our
actual results, performance or achievements to be materially different from any future results, performance or achievements expressed
or implied by the forward-looking statements, including, but not limited to, those disclosed under the caption "Risk Factors"
in our Annual Report on Form 10-K for the year ended July 31, 2024, and our other filings with the SEC. These factors could cause actual
results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking
statements represent management's estimates as of the date of this press release. While we may elect to update such forward-looking
statements at some point in the future, we disclaim any obligation to do so, even if subsequent events cause our views to change.