Welcome to this special edition of Neurology News Network. I'm Marco Meglio.
Positive topline results from the pivotal phase 3 IB1001-303 trial (NCT06673056) showed that levacetylleucine (Aqneursa; IntraBio), which is a therapy approved for Niemann-Pick disease type C, met its primary end point and secondary end points by demonstrating statistically significant improvement in ataxia symptoms compared with placebo in patients with ataxia-telangiectasia (A-T). Based on these data, the company stated that it plans to advance regulatory submissions to the FDA and the European Medicines Agency, as well as other global regulatory authorities. IB1001-303 is a phase 3, randomized, placebo-controlled, double-blind crossover phase study, followed by a long-term open-label extension assessing the efficacy, safety, and tolerability of levacetylleucine in pediatric and adult patients with A-T. At 12 weeks, findings showed that treatment with the agent was associated with statistically significant improvement on the primary end point, change in Scale for the Assessment and Rating of Ataxia (SARA) score, compared with placebo (−1.92 vs −0.14; between-group difference, −1.88; P<.001).
In a new company update, Capricor Therapeutics announced that the FDA has formally requested the complete clinical study report and supporting data from the phase 3 HOPE-3 trial (NCT05126758) of deramiocel, as the agency continues to review of the company’s biologics license application (BLA). The investigational cell therapy, which remains under review, is aiming to become the first cell-based product for patients with Duchenne muscular dystrophy (DMD) cardiomyopathy. Capricor stated in its company update that preparation of the HOPE-3 clinical study report is already underway and that the requested materials are expected to be submitted to the FDA in February 2026. The company noted that it anticipates that this submission will support continued review of the BLA and may lead to the assignment of a new PDUFA action date.
Recently announced data from a small-scale, investigator-initiated trial showed that treatment with COYA 302 (Coya Therapeutics), a combination of low-dose interleukin-2 (IL2) plus CTLA-4 Ig, met its target engagement and led to stable cognitive outcomes in patients with frontotemporal dementia (FTD) over a 6-month period. Coya expects to move the investigational agent into more controlled phase 2 studies to further understand its therapeutic effects. The trial featured 9 patients with FTD who received subcutaneous CTLA4-IgG, along with a 5-day course of low-dose IL-2 every 4 weeks for a treatment period lasting 22 weeks in total. Although not powered for significance, results revealed relatively unchanged scores on Montreal Cognitive Assessment (MoCA), a cognitive outcome, over the 22-week period (baseline: 13.5; week 22: 14). While there were slightly higher Clinical Dementia Rating-Frontotemporal Lobe Dementia (CDR-FTLD) scores at week 22, these were still considered unchanged relative to baseline (baseline: 4.8; week 22: 5.5).
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