Cyclo Therapeutics Presents Encouraging Preliminary Safety Data from Ongoing Pivotal Phase 3 Study and Substudy for the Treatment of Niemann-Pick Disease Type C1 (NPC1)
Two posters presented at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium 2024
Pivotal TransportNPC™ study is the most comprehensive ongoing controlled pivotal study regarding patient size, global footprint, duration and clinical outcomes for NPC1
Topline data from the 48-week interim analysis of 104 enrolled patients in TransportNPC™ on track for H1 2025
Trappsol® Cyclo™ demonstrated to be well tolerated in ongoing substudy with a safety profile consistent with that of completed phases
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 presentation of positive preliminary data from its ongoing pivotal Phase 3 study ("TransportNPC™") and substudy evaluating Trappsol® Cyclo™ for the treatment of Niemann-Pick Disease Type C1 (NPC1). The data were presented in two poster presentations at the SSIEM Annual Symposium 2024 being held September 3-6, 2024 in Porto, Portugal.
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 completed enrollment in May 2024. Additionally, the Company completed enrollment with ten (10) patients in a substudy per the adopted Paediatric Investigational Plan (PIP) treating newborn to 3 years of age. The substudy is evaluating Trappsol® Cyclo™ in the youngest age groups, focusing on its ability to target the visceral aspects of the disease. Administering treatment early in the disease course may lead to the most optimal results and could potentially have a preventative effect on overall symptom development.
"We are incredibly pleased with the progress made in our pivotal TransportNPC™ trial. These findings continue to underscore the potential of Trappsol® Cyclo™ to address a devastating disease with significant unmet need. We are committed to getting this important, global program across the finish line and are extremely hopeful for the interim data readout expected in H1 2025," commented N. Scott Fine, CEO of Cyclo Therapeutics.
Professor Caroline Hastings, MD, Global Principal Investigator for the Company's ongoing TransportNPC™ study, added, "We continue to believe Trappsol® Cyclo™ has the potential to provide a transformational impact for all people living with NPC. We are highly encouraged by these preliminary safety data and believe Trappsol® Cyclo could address the systemic and neurological symptoms of NPC which is considered the holy grail for the NPC community."
A summary of key highlights from the poster presentations are as follows:
The Company's ongoing Phase 3 study (96-week, double-blind, placebo-controlled) (TransportNPC™) will evaluate change in 5D-NPC-CSS (ambulation, fine motor, speech, swallow, cognition) between Trappsol® Cyclo™ and placebo as primary endpoint for the EU. A total of 104 patients have been enrolled into the study and substudy, the majority of patients enrolled fall within the ASIS score limits targeted (0.5 to 2.0) with the average ASIS at recruitment was 1.04; average age at inclusion was 20.2 years (3 – 65 years). Average participation time in the blinded phase of the study is 44 weeks as of June 30, 2024. As of June 30, 2024, six patients terminated study participation before week 96; following protocol, six patients switched to open-label Trappsol® Cyclo™ before week 96 due to disease worsening.
Safety and Tolerability Highlights:
- A total of 625 Adverse Events (AEs) were reported, 80% were Grade 1 (mild) in severity most related to underlying NPC disease; Grade 2 were 16.7%, and Grade 3 (Severe) were 5.3%
- 9 Serious Adverse Events (SAEs) reported, possibly related or related to treatment
- One patient death unrelated to study-drug
- No patients have withdrawn due to safety concerns
- Safety profile found consistent with that of completed studies
Topline data from the 48-week interim analysis is anticipated for H1 2025. 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 goal of the substudy is to evaluate the safety of biweekly IV Trappsol® Cyclo™ (HPβCD) (2000 mg/kg) for 192 weeks in very young patients (birth to ≤ 3 years), in order to obtain descriptive data regarding safety, global severity, and improvement in response to HPβCD from investigators and patients/caregivers. Ten patients were recruited, of which two terminated the study after 48 weeks.
Safety and Tolerability Highlights:
- AE profile is in line with prior findings from compassionate use, from earlier studies, and from a double-blind Phase 3 study running in parallel
- Overall, AEs are limited (87), majority are mild (73%) or moderate (17%) and 1 AE severe; most considered unrelated to study drug
- No SAEs were considered as related to or possibly related to study drug
- At baseline patients had a mixture of very mild to severe disease based on CGI
- Progress in patients with neurological symptoms from the early pediatric age is understood to be more rapid than in patients with late pediatric, juvenile, or even adult neurological onset of NPC (Yanjanin, 2010; Vanier, 2010). Disease progression is carefully monitored throughout duration of the study.
The substudy has completed enrollment and the treatment has been well tolerated with a safety profile consistent with prior studies, including the larger TransportNPC™ study. In addition to the clinical endpoints, this study will provide additional critical information to address the concept of early intervention and prevention of disease progression. By the first half of 2025, all currently enrolled patients will have completed at least 48 weeks of treatment; Three patients will likely have completed 96 weeks of treatment. This will be the first data in NPC1 on treatment in this age group over a period of 48 weeks.
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, (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.
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