Cyclo Therapeutics Announces Positive Outcome from Type C Meeting with the U.S. FDA Discussing Trappsol® Cyclo™ Clinical Program for the Treatment of Niemann-Pick Disease Type C1
- Positive FDA feedback from the Type C meeting
- Direction for comparative interim analysis of the ongoing pivotal Phase 3 study
- Alignment with the company's proposal to continue with the Phase 3 96-week study
- Suggestive evidence of potential therapeutic benefit from clinical data
- None.
Ongoing pivotal Phase 3 study (“TransportNPC™”) evaluating Trappsol® Cyclo™ for the treatment of Niemann-Pick Disease Type C1 (NPC1)
FDA feedback provides direction for the comparative interim analysis
With aligned strategy, interim analysis data readout is anticipated for Q1 2025
FDA and Cyclo Therapeutics to meet in the first half of 2024 for pre-NDA meeting
N. Scott Fine, Chief Executive Officer of Cyclo Therapeutics, added, “We view our Type C meeting with FDA as a catalytic and transformational milestone for our ongoing pivotal Phase 3 NPC program. By working closely and in alignment with FDA, I am confident our leadership team has positioned Cyclo Therapeutics for rapid continued advancement and has established the appropriate path to generate the data required to successfully drive our TransportNPC study across the finish line.”
In summary, the Company’s clinical data (NCT02939547, NCT03893071 and NCT02912793) provided suggestive evidence, based on individual patient data, of potential therapeutic benefit which may be observed at or before 48 weeks of treatment. Given the significant unmet need within this Orphan Disease and the lack of available effective pharmacotherapy, and that aggregate clinical and nonclinical data argue for a potentially clinically relevant therapeutic effect, the FDA aligned with Cyclo Therapeutics’ proposal to continue with the agreed upon Phase 3 96-week study, but change the timing of the planned comparative interim analysis to execute data readout in Q1 2025.
“The FDA feedback we received allows us to proceed as planned to meet our full enrollment goals as well as our regulatory and corporate objectives to bring a potential treatment option to this underserved patient community as quickly as possible. We look forward to implementing these changes and to our future pre-NDA meeting with the FDA as we continue to execute across all aspects of our drug development program,” stated Michael Lisjak, Chief Regulatory Officer and Senior Vice President of Business Development for Cyclo Therapeutics.
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 Phase 3 study intends to enroll at least 93 pediatric (age 3 years and older) and adult patients with NPC1 and is now active in 12 countries.
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
About Cyclo Therapeutics
Cyclo Therapeutics, Inc. is a clinical stage biotechnology company focused on developing transformative therapies for rare and neurological diseases with limited treatment options. The Company’s Trappsol® Cyclo™, an orphan drug designated product in
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Investor Contact:
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Source: Cyclo Therapeutics, Inc.
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