Cyclo Therapeutics Achieves Landmark Milestone with Completion of Enrollment of Last Patient in Phase 3 Pivotal TransportNPC™ Trial of Niemann-Pick Type C1

Rhea-AI Summary

Cyclo Therapeutics has completed enrollment for its Phase 3 TransportNPC™ trial, the largest study of its kind for Niemann-Pick Disease Type C1 (NPC1). The study includes 104 patients and aims to evaluate the efficacy of Trappsol® Cyclo™ in treating systemic and neurological symptoms of NPC1.

Topline data from the 48-week interim analysis is expected in the first half of 2025. Positive results could lead to the submission of a New Drug Application (NDA) to the FDA and a Marketing Authorization Application (MAA) to the EMA in the second half of 2025.

The trial also features a substudy for newborns to 3-year-olds, targeting early-stage interventions. The company has received Orphan Drug Designation in both the U.S. and EU, Fast Track and Rare Pediatric Disease designations in the U.S., which could qualify them for a Priority Review Voucher.

Positive

• Completion of enrollment for Phase 3 TransportNPC™ trial with 104 patients.
• 48-week interim data expected in H1 2025.
• Potential for NDA submission to FDA and MAA to EMA in 2H 2025.
• Enrolled 10 patients in a substudy targeting early-stage interventions.
• Received Orphan Drug Designation in the U.S. and EU.
• Gained Fast Track and Rare Pediatric Disease designations in the U.S.
• Largest ever controlled pivotal study for NPC1.
• Substantial support from FDA and EMA noted.

Negative

• Topline data not expected until H1 2025, implying a long wait for investors.
• No guarantee that 48-week interim data will meet the required significance.
• Potential risks associated with the clinical trial outcomes not being favorable.
• Financial instability could arise if the trial results are not as expected.
• Possible delay in revenue realization even if the drug gets approved.
• High costs and financial strain associated with conducting large-scale trials.

Insights

Medical Research Analyst

The completion of patient enrollment for Cyclo Therapeutics' pivotal Phase 3 trial of Trappsol® Cyclo™ represents a significant milestone in the treatment of Niemann-Pick Disease Type C1 (NPC1), a severe genetic condition with no currently approved therapies. The scale and rigor of this trial, involving 104 patients globally, ensure robust data which will be critical in assessing the efficacy and safety of Trappsol® Cyclo™.

From a medical research perspective, the fact that approximately half of the patients will have completed the 96-week timepoint by the 48-week interim analysis provides additional long-term data that could bolster the potential for a successful New Drug Application (NDA) submission. This is especially important for conditions like NPC1, where long-term efficacy and safety are paramount.

The inclusion of a pediatric substudy targeting the youngest age group underscores the comprehensive approach Cyclo Therapeutics is taking. Early intervention in NPC1 could have a preventative effect, potentially reducing overall symptom development, which, if proven effective, could transform patient care standards.

Rating: 1

Financial Analyst

For retail investors, the completion of patient enrollment in Cyclo Therapeutics' pivotal Phase 3 trial signifies a significant step towards regulatory approval and potential commercialization. The expected milestone of 48-week interim data by H1 2025 is crucial. If positive, it will likely lead to NDA and MAA submissions in H2 2025, with the possibility of receiving a Priority Review Voucher (PRV). PRVs are valuable assets, as they can be sold or used to expedite the review process for future drugs.

Financially, successful interim results could catalyze stock price movement and attract more investments. However, investors should be cautious about the inherent risks in clinical trials, such as potential delays or unfavorable outcomes. It is also worth noting that Cyclo Therapeutics received Orphan Drug Designation, which provides certain incentives including market exclusivity upon approval.

While the prospective impact on Cyclo Therapeutics' financial health appears positive, the volatility associated with clinical stage biotech firms should not be underestimated. This development is promising but still carries risks commonly associated with drug development.

Rating: 1

Market Research Analyst

The significance of Cyclo Therapeutics' Phase 3 trial completion for Trappsol® Cyclo™ extends beyond the immediate clinical implications. The rarity of NPC1, affecting approximately 1 in 100,000 live births globally, means that any advancement in treatment will likely garner significant attention from the medical community and patient advocacy groups.

From a market perspective, the unmet need for NPC1 treatments creates a niche but potentially lucrative market. The Orphan Drug Designation in the U.S. and Europe, along with Fast Track and Rare Pediatric Disease Designations, provide regulatory and marketing advantages that can expedite the drug's path to market and protect its exclusivity.

Given the global footprint of the study, successful results could position Cyclo Therapeutics as a leader in the rare disease sector. However, the market potential is somewhat limited by the rarity of NPC1, so investors should balance the high impact of a potential breakthrough treatment against the relatively small patient population.

Rating: 1

TransportNPC™ is the most comprehensive ongoing controlled pivotal study regarding patient size, global footprint, duration and clinical outcomes for the treatment of Niemann-Pick Disease Type C1 (NPC1)

Topline data from the 48-week interim analysis of 104 enrolled patients is anticipated for H1 2025

If 48-week data demonstrate significance, submission of New Drug Application (NDA) to the Food and Drug Administration (FDA) and Marketing Authorization Application (MAA) to European Medicines Agency (EMA) is targeted for 2H 2025; Qualification for Priority Review Voucher (PRV) upon NDA submission

GAINESVILLE, Fla.--(BUSINESS WIRE)-- Cyclo Therapeutics, Inc. (Nasdaq: CYTH) (“Cyclo Therapeutics” or the “Company”), a clinical stage biotechnology company dedicated to developing life-changing medicines through science and innovation for patients and families living with diseases, today announced the last patient has been enrolled in the Company’s pivotal Phase 3 study (“TransportNPC™”) evaluating Trappsol^® Cyclo™ for the treatment of systemic and neurological symptoms of Niemann-Pick Disease Type C1 (NPC1).

The TransportNPC™ study is the most comprehensive controlled pivotal study regarding patient size, global footprint, duration and clinical outcomes of an investigational therapy for NPC1. The study has dosed its 93^rd (final) and 94^th (over enrolled) patients. Additionally, the Company has enrolled ten (10) patients in its substudy per their adopted Paediatric Investigational Plan (PIP) treating newborn to 3 years of age. The substudy is evaluating Trappsol^® Cyclo™ in the youngest age subsets as it targets also the visceral aspects of the disease and may achieve its most optimal results when administered early in the disease course, thus having the potential of a preventative effect in overall symptom development.

“The completion of enrollment represents by far the most significant milestone for Cyclo Therapeutics to date. Not only is this study the largest controlled pivotal study for NPC1 ever to be conducted with 104 patients enrolled, but we will also have approximately half of those 104 patients who completed the 96-week (two-year) timepoint at the time of our 48-week interim analysis, thus providing important additional long-term data supporting our potential submissions to the Health Authorities,” commented N. Scott Fine, Chief Executive Officer of Cyclo Therapeutics. “With the positive support, feedback and alignment from our recent health authority interactions with both the FDA and EMA, we are excited to take this critical step toward the interim data readout, which is expected in H1 2025 and most importantly, the potential to provide a much-needed treatment option for the NPC community.”

Professor Caroline Hastings, MD, Global Principal Investigator for the Company’s ongoing TransportNPC™ study, added, “We are extremely pleased with the progress of this trial and to be a step closer to potentially providing a much-needed treatment option for this devastating disease that is in desperate need of a safe and effective approved treatment for the systemic and neurological symptoms of NPC. Together with the global NPC community, we have steadily increased our understanding of NPC and Trappsol^® Cyclo™ over the course of nearly two decades and look forward to the 48-week interim data readout in the first half of 2025. We believe this has the potential to provide a transformational impact for all people living with NPC and what this represents for this area of significant unmet need.”

“We are grateful for the continued support of our investors, including Rafael Holdings, Inc. (RFL) and other longtime shareholders, who stand shoulder to shoulder with us to see the clinical development of Trappsol^® Cyclo™ successfully cross the finish line,” added Joshua Fine, Chief Financial Officer of the Company.

The Company’s ongoing TransportNPC™ study is a randomized, double-blind, placebo-controlled, parallel group, multicenter study designed to evaluate the safety, tolerability, and efficacy of 2,000 mg/kg doses of Trappsol^® Cyclo™ administered intravenously and standard of care (SOC), compared to placebo administered intravenously and SOC alone, in patients with NPC1, a rare, genetic disease causing cholesterol accumulation in cells, leading to dysfunction of the liver, lung, spleen and brain and premature death. The study duration is a 96-week study, with a 48-week comparative interim analysis. Should the 48-week interim data meet statistical significance, the Company, in alignment with the FDA and EMA, intends to submit marketing applications for approval based on the 48-week interim data.

The Company’s ongoing single-arm sub-study is evaluating patients from birth to less than 3 years of age with NPC1 irrespective of symptoms to evaluate safety and to obtain descriptive data on global disease severity and the response to Trappsol^® Cyclo™. The substudy is being conducted in countries outside of the United States per their adopted PIP.

For more information about the Company’s TransportNPC™ pivotal Phase 3 study, visit www.ClinicalTrials.gov and reference identifier NCT04860960.

Cyclo Therapeutics received Orphan Drug Designation for Trappsol^® Cyclo™ to treat NPC1 in both the U.S. and EU and Fast Track and Rare Pediatric Disease Designations in the U.S. The Rare Pediatric Disease Designation is one of the chief requirements for sponsors to receive a Priority Review Voucher in the U.S. upon marketing authorization.

About Niemann-Pick Disease Type C1 (NPC)

NPC is a rare genetic disease affecting 1 in 100,000 live births globally. Approximately 95% of individuals with NPC have mutations in the NPC1 gene and 5% have mutations in the NPC2 gene. NPC affects nearly every cell in the body due to a deficiency in either the NPC1 or NPC2 protein, which are required for the transport and processing of cholesterol within the cell. As cholesterol accumulates within cells, NPC causes symptoms that affect the brain, liver, spleen, lung, and other organs and often leads to premature death.

About Cyclo Therapeutics

Cyclo Therapeutics, Inc. is a clinical-stage biotechnology company dedicated to developing life-changing medicines through science and innovation for patients and families living with disease. The Company’s Trappsol^® Cyclo™, an orphan drug designated product in the United States and Europe, is the subject of four formal clinical trials for Niemann-Pick Disease Type C1, a rare and fatal genetic disease, (www.ClinicalTrials.gov NCT02939547, NCT02912793, NCT03893071 and NCT04860960). The Company is conducting a Phase 2b clinical trial using Trappsol^® Cyclo™ intravenously in early Alzheimer’s disease (NCT05607615) based on encouraging data from an Expanded Access program for Alzheimer’s disease (NCT03624842). Additional indications for the active ingredient in Trappsol^® Cyclo™ are in development. For additional information, visit the Company’s website: www.cyclotherapeutics.com.

Safe Harbor Statement

This press release contains “forward-looking statements” about the company’s current expectations about future results, performance, prospects and opportunities, including, without limitation, statements regarding the satisfaction of closing conditions relating to the offering and the anticipated use of proceeds from the offering. Statements that are not historical facts, such as “anticipates,” “believes” and “expects” or similar expressions, are forward-looking statements. These statements are subject to a number of risks, uncertainties and other factors that could cause actual results in future periods to differ materially from what is expressed in, or implied by, these statements. The factors which may influence the company’s future performance include the company’s ability to obtain additional capital to expand operations as planned, success in achieving regulatory approval for clinical protocols, enrollment of adequate numbers of patients in clinical trials, unforeseen difficulties in showing efficacy of the company’s biopharmaceutical products, success in attracting additional customers and profitable contracts, and regulatory risks associated with producing pharmaceutical grade and food products. These and other risk factors are described from time to time in the company’s filings with the Securities and Exchange Commission, including, but not limited to, the company’s reports on Forms 10-K and 10-Q. Unless required by law, the company assumes no obligation to update or revise any forward-looking statements as a result of new information or future events.

View source version on businesswire.com: https://www.businesswire.com/news/home/20240530051022/en/

Investor Contact:

JTC Team, LLC

Jenene Thomas

(833) 475-8247

CYTH@jtcir.com

Source: Cyclo Therapeutics, Inc.

FAQ

What is the significance of Cyclo Therapeutics completing enrollment for the TransportNPC™ Phase 3 trial?

Completing enrollment signifies Cyclo Therapeutics' progress in its Phase 3 trial for Niemann-Pick Disease Type C1 (NPC1), which is the largest study of its kind and could lead to significant regulatory submissions if successful.

When are the topline data from Cyclo Therapeutics' TransportNPC™ trial expected?

Topline data from the 48-week interim analysis are expected in the first half of 2025.

How might the TransportNPC™ trial results impact Cyclo Therapeutics?

If the interim results are positive, Cyclo Therapeutics plans to submit a New Drug Application (NDA) to the FDA and a Marketing Authorization Application (MAA) to the EMA in the second half of 2025.

What designations has Trappsol® Cyclo™ received?

Trappsol® Cyclo™ has received Orphan Drug Designation in the U.S. and EU, as well as Fast Track and Rare Pediatric Disease designations in the U.S.

What is the purpose of the substudy in Cyclo Therapeutics' TransportNPC™ trial?

The substudy aims to evaluate Trappsol® Cyclo™ in newborns to 3-year-olds, focusing on early-stage intervention and the potential preventative effects on systemic and neurological symptoms.

Why is the TransportNPC™ study significant for the treatment of NPC1?

The study is the most comprehensive controlled pivotal study for NPC1 to date, involving a large number of patients and a global footprint, which could potentially lead to a new treatment for this rare and fatal disease.