Cyclo Therapeutics (NASDAQ: CYTH) presented positive preliminary data from its ongoing Phase 3 TransportNPC™ open-label sub-study in patients under 3 years old with Niemann-Pick Disease Type C1 (NPC1). The data shows promising results, with 87% of patients (7 of 8) at 24 weeks and 86% (6 of 7) at 48 weeks demonstrating stabilization or improvement in Clinical Global Impression – Change Scale.
The sub-study is part of the company's comprehensive TransportNPC™ pivotal Phase 3 global study evaluating Trappsol® Cyclo™. The main study completed enrollment in May 2024 with 104 patients, and topline data is expected in H1 2025. The safety profile remains consistent with previous findings, with mostly mild (77%) or moderate (22%) adverse effects, and no serious adverse events related to the study drug.
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