Neurology Daily Report: 01/25/2024
Duchenne Muscular Dystrophy (DMD)
A Phase 2 open-label study investigated the effect of ATL1102 in 9 patients with non-ambulatory DMD aged 10-18 years and found that ATL1102 was generally safe and well tolerated with no serious adverse events reported . The most commonly reported adverse events were injection site erythema and skin discoloration. There was no statistically significant change in lymphocyte count from baseline to week 8, 12, or 24 of dosing. However, CD3+CD49d+ T lymphocytes were statistically significantly higher at week 28 compared to week 24. Functional muscle strength and MRI fat fraction of the forearm muscles were stable throughout the trial period. This study supports the continued development of ATL1102 for the treatment of DMD. The trial was registered at the Australian New Zealand Clinical Trials Registry Number: ACTRN12618000970246.
Reference
Woodcock IR, Tachas G, Desem N, Houweling PJ, Kean M, Emmanuel J, Kennedy R, Carroll K, de Valle K, Adams J, Lamandé SR, Coles C, Tiong C, Burton M, Villano D, Button P, Hogrel JY, Catling-Seyffer S, Ryan MM, Delatycki MB, Yiu EM. A phase 2 open-label study of the safety and efficacy of weekly dosing of ATL1102 in patients with non-ambulatory Duchenne muscular dystrophy and pharmacology in mdx mice. PLoS One. 2024 Jan 25;19(1):e0294847. doi: 10.1371/journal.pone.0294847. PMID: 38271438; PMCID: PMC10810432.
Alzheimer’s Disease
A Phase 1/2 randomized, double-blind, placebo-controlled study investigated the effect of CT1812 in 23 participants with mild to moderate dementia due to Alzheimer's disease and found no treatment differences relative to placebo in the change from baseline at 24 weeks in synaptic vesicle glycoprotein 2A (SV2A) or fluorodeoxyglucose (FDG) PET signal, cognitive clinical rating scales, or in cerebrospinal fluid biomarkers (1). However, a trend towards tissue preservation was observed in volumetric MRI in participants treated with CT1812, with nominally significant differences in the pericentral, prefrontal, and hippocampal cortices compared to placebo. CT1812 was safe and well tolerated, supporting its further clinical development. The trial was registered at clinicaltrials.gov (NCT03493282).
Reference
van Dyck CH, Mecca AP, O'Dell RS, Bartlett HH, Diepenbrock NG, Huang Y, Hamby ME, Grundman M, Catalano SM, Caggiano AO, Carson RE. A pilot study to evaluate the effect of CT1812 treatment on synaptic density and other biomarkers in Alzheimer's disease. Alzheimers Res Ther. 2024 Jan 25;16(1):20. doi: 10.1186/s13195-024-01382-2. PMID: 38273408; PMCID: PMC10809445.