TRAILBLAZER-ALZ-2 Data of Donanemab Published, Risdiplam Effective in SMA up to 4 Years, Preclinical Alzheimer Study of Lecanemab Announced

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Welcome to this special edition of Neurology News Network. I’m Marco Meglio.

At the 2023 Alzheimer’s Association International Conference (AAIC), held June 16-20, in Netherlands, Amsterdam, full data from the pivotal, phase 3 TRAILBLAZER-ALZ 2 trial highlighted donanemab’s (Eli Lilly) impact on disease progression in individuals with early symptomatic Alzheimer disease (AD). The results, which were simultaneously published in JAMA, showed that donanemab, an antiamyloid therapy, was effective across all cognitive and functional secondary end points, and across multiple analysis methods. When the initial topline results of the study were announced in May 2023, Eli Lilly noted it will proceed with global regulatory submissions as soon as possible, with a potential FDA submission occurring this quarter. In January, when the FDA issued its CRL for donanemab, the agency felt as though there was not enough sufficient, longer-term evidence to approve the agent, considering its application was supported by the phase 2, 12-month TRAILBLAZER-ALZ study.

Recently announced long-term data from the pivotal FIREFISH study showed continued improvements in infants with spinal muscular atrophy (SMA) treated with risdiplam (Evrysdi; Genentech) for up to 4 years. All treated children who were alive at the time of the primary analysis were still alive after 48 months. Results of the OLE, presented at the Cure SMA Research & Clinical Care Meeting, held June 28-30, showed that after 4 years of treatment, many of the babies, now young children, continued to improve their ability to sit, stand, and walk without support. A major of the infants included in the OLE (n = 50) maintained their ability to feed by mouth and swallow up to month 48. At the same time point, treated individuals showed either maintained or improved motor function, as demonstrated through the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development Third Edition and the Hammersmith Infant Neurological Examination 2 assessments.

Less than a week after the FDA granted traditional approval to Eisai’s antiamyloid agent lecanemab (Leqembi), the Alzheimer’s Clinical Trial Consortium announced a new study, AHEAD, that will assess the agent in individuals with preclinical Alzheimer disease (AD) who have no cognitive symptoms present. Funded by the National Institutes of Health, the trial aims to enroll 1165 participants from North America to see whether lecanemab can help stave off the memory problems caused by AD. With more than 100 study locations worldwide, the trial will use change from baseline in Preclinical Alzheimer Cognitive Composite 5 (PACC5) score and change in amyloid PET standard uptake value ratio (SUVr) over a 216-week period as the primary end points.

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