Cyclone Therapeutics

Trappsol Cyclo, rare neurological disorders

clearingsmall molecule

San Diego, California, United States·Acquired·$13M total funding

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Plate C · C-T581

Projects

Trappsol Cyclo for early Alzheimer's Disease

undisclosed

drug program · high · Fri Sep 23 2022 00:00:00 GMT+0000 (Coordinated Universal Time)

Evaluate the safety and potential efficacy of Trappsol Cyclo in patients aged 50 to 80 with early Alzheimer's Disease.

Six-month randomized, double-blind clinical study with approximately 90 patients in the United States, using two active dose groups and one placebo group, with screening, treatment, and follow-up periods.

Clinical study registered to evaluate Trappsol Cyclo in early Alzheimer's Disease.

Trappsol Cyclo for Niemann-Pick Disease Type C1

phase 3

drug program · high · Tue Jul 20 2021 00:00:00 GMT+0000 (Coordinated Universal Time)

Evaluate Trappsol Cyclo as a disease-modifying treatment for pediatric and adult patients with Niemann-Pick Disease Type C1 by improving or stabilizing clinical signs, neurologic symptoms, quality-of-life-relevant domains, and biomarkers related to cholesterol metabolism, neurodegeneration, and inflammation.

Intravenous hydroxypropyl-beta-cyclodextrin (HP-beta-CD; Trappsol Cyclo) administered every two weeks; studied in Phase I/II dose-ranging trials, open-label long-term access, and a randomized double-blind placebo-controlled Phase 3 study using 2000 mg/kg compared with standard of care.

Phase 3 TransportNPC study evaluating intravenous Trappsol Cyclo in pediatric and adult NPC1 patients; prior 48-week Phase I/II trial reported clinical benefits and supported the global pivotal trial.

In the 48-week Phase I/II trial, 9 patients completed treatment; 8 of 9 improved in at least two domains of the 17-domain NPC Clinical Severity Scale, 7 were judged by treating physicians to have improved and 2 remained stable, and all three tested doses were generally well tolerated.

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0-100 chain-logic scale · 15 dimensions · scored on public evidence