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)
Cyclo Therapeutics (Nasdaq: CYTH) presented encouraging preliminary safety data from its ongoing pivotal Phase 3 study (TransportNPC™) and substudy evaluating Trappsol® Cyclo™ for Niemann-Pick Disease Type C1 (NPC1) at the SSIEM Annual Symposium 2024. The TransportNPC™ study, with 104 enrolled patients, is the most comprehensive controlled pivotal study for NPC1. Key highlights include:
1. Topline data from the 48-week interim analysis expected in H1 2025
2. Trappsol® Cyclo™ demonstrated to be well-tolerated with a consistent safety profile
3. 625 Adverse Events reported, 80% Grade 1 (mild)
4. No patients withdrew due to safety concerns
5. Substudy completed enrollment with ten patients under 3 years old
Cyclo Therapeutics (Nasdaq: CYTH) ha presentato dati preliminari di sicurezza incoraggianti dallo studio pivotale di Fase 3 in corso (TransportNPC™) e dalla sottostudio che valuta Trappsol® Cyclo™ per la malattia di Niemann-Pick di tipo C1 (NPC1) al Simposio Annuale SSIEM 2024. Lo studio TransportNPC™, con 104 pazienti arruolati, è lo studio controllato pivotale più completo per NPC1. I punti salienti includono:
1. I dati principali dell'analisi intermedia di 48 settimane sono attesi nella prima metà del 2025
2. Trappsol® Cyclo™ ha dimostrato di essere ben tollerato con un profilo di sicurezza costante
3. 625 eventi avversi segnalati, 80% di Grado 1 (lievi)
4. Nessun paziente ha ritirato dal trattamento per preoccupazioni di sicurezza
5. La sottostudio ha completato l'arruolamento con dieci pazienti di età inferiore ai 3 anni
Cyclo Therapeutics (Nasdaq: CYTH) presentó datos preliminares de seguridad alentadores de su estudio pivotante de Fase 3 en curso (TransportNPC™) y un subestudio que evalúa Trappsol® Cyclo™ para la enfermedad de Niemann-Pick tipo C1 (NPC1) en el Simposio Anual SSIEM 2024. El estudio TransportNPC™, con 104 pacientes inscritos, es el estudio crucial controlado más completo para NPC1. Los puntos destacados incluyen:
1. Se esperan datos principales del análisis intermedio de 48 semanas en la primera mitad de 2025
2. Trappsol® Cyclo™ demostró ser bien tolerado con un perfil de seguridad consistente
3. Se reportaron 625 Eventos Adversos, 80% Grado 1 (leves)
4. Ningún paciente se retiró debido a preocupaciones de seguridad
5. El subestudio completó la inscripción con diez pacientes menores de 3 años
사이클로 테라퓨틱스(Cyclo Therapeutics, Nasdaq: CYTH)는 2024 SSIEM 연례 심포지엄에서 니만-픽병 유형 C1(Niemann-Pick Disease Type C1, NPC1)에 대한 트랩솔® 사이클로™(Trappsol® Cyclo™)를 평가하는 진행 중인 주요 3상 연구(TransportNPC™)와 하위 연구의 초기 안전성 데이터에 대한 고무적인 결과를 발표했습니다. 104명의 환자가 등록된 TransportNPC™ 연구는 NPC1에 대한 가장 포괄적인 통제된 기준 연구입니다. 주요 하이라이트는 다음과 같습니다:
1. 48주 중간 분석의 주요 데이터는 2025년 상반기에 예상됨
2. 트랩솔® 사이클로™는 일관된 안전성 프로필을 가진 잘 견디는 것으로 나타남
3. 625건의 부작용이 보고되었으며, 80%가 1등급(경미)임
4. 안전성 문제로 치료를 중단한 환자는 없음
5. 하위 연구는 3세 이하 환자 10명의 등록을 완료함
Cyclo Therapeutics (Nasdaq: CYTH) a présenté des données préliminaires de sécurité encourageantes lors de son étude pivotante de phase 3 en cours (TransportNPC™) et de la sous-étude évaluant Trappsol® Cyclo™ pour la maladie de Niemann-Pick de type C1 (NPC1) lors du Symposium annuel SSIEM 2024. L'étude TransportNPC™, avec 104 patients inscrits, est l'étude pivotale contrôlée la plus complète pour le NPC1. Les points forts incluent :
1. Les données principales de l'analyse intermédiaire de 48 semaines sont attendues dans la première moitié de 2025
2. Trappsol® Cyclo™ a démontré qu'il était bien toléré avec un profil de sécurité constant
3. 625 événements indésirables signalés, 80 % de Grade 1 (léger)
4. Aucun patient n'a interrompu son traitement en raison de préoccupations de sécurité
5. La sous-étude a terminé le recrutement avec dix patients de moins de 3 ans
Cyclo Therapeutics (Nasdaq: CYTH) hat ermutigende vorläufige Sicherheitsdaten aus seiner laufenden pivotalen Phase-3-Studie (TransportNPC™) und einer Unterstudie zur Bewertung von Trappsol® Cyclo™ für die Niemann-Pick-Krankheit Typ C1 (NPC1) auf dem jährlichen SSIEM-Symposium 2024 präsentiert. Die TransportNPC™-Studie mit 104 eingeschriebenen Patienten ist die umfassendste kontrollierte Hauptstudie für NPC1. Die wichtigsten Highlights sind:
1. Topline-Daten aus der interimistischen Analyse über 48 Wochen werden für das erste Halbjahr 2025 erwartet
2. Trappsol® Cyclo™ hat sich als gut verträglich mit einem stabilen Sicherheitsprofil erwiesen
3. 625 unerwünschte Ereignisse wurden gemeldet, 80% Grad 1 (mild)
4. Keine Patienten haben die Studie aufgrund von Sicherheitsbedenken abgebrochen
5. Die Unterstudie hat die Rekrutierung mit zehn Patienten unter 3 Jahren abgeschlossen
- Completed enrollment of 104 patients in the TransportNPC™ study
- Trappsol® Cyclo™ demonstrated to be well-tolerated in ongoing substudy
- 80% of reported Adverse Events were Grade 1 (mild) in severity
- No patients withdrew due to safety concerns
- Topline data from 48-week interim analysis expected in H1 2025
- Six patients terminated study participation before week 96
- Six patients switched to open-label Trappsol® Cyclo™ before week 96 due to disease worsening
- One patient death reported (unrelated to study-drug)
Insights
The preliminary safety data from Cyclo Therapeutics' Phase 3 TransportNPC™ study for Trappsol® Cyclo™ in NPC1 patients is encouraging. Key points:
- Of 625 Adverse Events (AEs),
80% were mild, with only5.3% severe. - Only 9 Serious Adverse Events (SAEs) reported, with one possibly related to treatment.
- No withdrawals due to safety concerns.
The substudy in patients under 3 years old also shows promise, with mostly mild to moderate AEs. This early intervention approach could be groundbreaking for NPC1 treatment. However, efficacy data is still pending and the H1 2025 interim analysis will be important for assessing the drug's true potential.
Cyclo Therapeutics' progress with Trappsol® Cyclo™ is noteworthy for investors. The company has achieved:
- Full enrollment (104 patients) in the largest NPC1 pivotal study to date.
- Completion of a 10-patient substudy in very young patients.
- A favorable preliminary safety profile.
The H1 2025 interim data readout is a critical catalyst. If positive, it could lead to marketing applications, potentially accelerating the path to commercialization. However, investors should note that efficacy data is still pending and NPC1's rarity may limit market size. The Alzheimer's trial (NCT05607615) could offer significant upside if successful, given the larger patient population.
Cyclo Therapeutics' approach to NPC1 treatment is innovative and comprehensive. The TransportNPC™ study's global scope and inclusion of a wide age range (3-65 years) are commendable. The substudy focusing on patients under 3 years old is particularly exciting, as early intervention could potentially prevent symptom development.
The safety profile thus far is reassuring, but the true test will be efficacy. The 5D-NPC-CSS endpoint is appropriate, covering key disease aspects. If Trappsol® Cyclo™ can demonstrate significant improvements across these domains, it could become a game-changer for NPC1 patients. The H1 2025 interim analysis will be a pivotal moment for both patients and the company.
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
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 were16.7% , and Grade 3 (Severe) were5.3% - 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
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Investor:
JTC Team, LLC
Jenene Thomas
(833) 475-8247
CYTH@jtcir.com
Source: Cyclo Therapeutics
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