Home-based intravenous infusions of Trappsol® Cyclo™ for up to one year show improvement in disease features or disease stabilization
Cyclo Therapeutics, Inc., a clinical stage biotechnology company developing a cyclodextrin platform for the treatment of neurodegenerative diseases, including its lead candidate Trappsol® Cyclo™ for the treatment of Niemann-Pick Disease Type C (NPC), announced initial data from its “Open-Label Study of Long-Term Safety and Efficacy of Intravenous Trappsol® Cyclo™ (HPBCD) in Niemann-Pick Disease Type C,” NCT03893071. Eight patients living in the US who completed the Phase I trial (NCT02939547) were eligible for the extension program. All eight enrolled.
The first patient was dosed in the Open-Label extension study in May 2019 and the last patient enrolled was in February 2020. The initial efficacy results presented below have a data cut-off of September 2020.
All patients included in this analysis demonstrated disease stability or improvement as measured by the 17-domain NPC Severity Score scale (NPC-SS, Yanjinin et al, 2010). The NPC-SS measures primarily neurologic features of the disease, including ambulation, fine motor skills, ability to speak and swallow, and cognition. Without intervention, over the course of one year, NPC patients would be expected to worsen by one to two points using this scale.
During this Open-Label Study, patients receive intravenous infusions of Trappsol® Cyclo™ in their homes under the care of healthcare professionals every two weeks, with regular assessments for NPC-SS at UCSF Benioff Children’s Hospital Oakland under the care of Dr. Caroline Hastings and Dr. Benny Liu, Co-Principal Investigators.
Of the patients who received Trappsol® Cyclo ™ at the 1500 mg/kg body weight dose level, four patients remained stable in terms of their NPC-SS and two patients improved:
Patient 1 had a baseline NPC-SS of 24; and the same score 10 months later.
Patient 2 had a baseline NPC-SS of 18, with the same score 4 months later and again 6 months after the second assessment.
Patient 3 had a baseline NPC-SS of 11 and the same score 7 months later.
Patient 4 had a baseline NPC-SS of 12 and the same score 6 months later.
Patient 5 had a baseline NPC-SS of 16, a score of 15 five months later (improvement of 1 point), and a score of 13 eight months later (representing improvement by total 3 points).
Patient 6 had a baseline NPC-SS of 12 and a score of 10 eight months later (an improvement of 2 points).
Of the patients in the 2500 mg/kg dose group, both improved overall:
Patient 7 had a baseline NPC-SS of 18, followed by a score of 20 four months later (a worsening of 2 points) and a score of 15 six months later (a total improvement of 3 points compared to baseline during the 10-month assessment period).
And, Patient 8 had a baseline NPC-SS of 12 and a score of 8 five months later (an improvement of 4 points).
The safety profile was favorable with 16 Adverse Events (AEs) in total between May 2019 and September 2020, all mild (Grade 1 and 2) and none related to the study drug, per the treating physician.
Cyclo Therapeutics has authorization from FDA to begin enrollment in its phase 3 trial and plans to enroll the first patient early in 2021.