Solanezumab Fails A4 Alzheimer Study, FDA Lifts Hold on FREEDOM-DM1 Trial, CMS Announces Coverages of PET Amyloid-ß Imaging

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

After topline data were announced earlier this year, investigators have published full findings from the phase 3 Anti-Amyloid Treatment in Asymptomatic Alzheimer’s Disease (A4) study (NCT02008357) in the New England Journal of Medicine. All told, treatment with solanezumab (Eli Lilly), an investigational antiamyloid agent, failed to slow cognitive decline as compared with placebo over a period of 240 weeks in patients with preclinical Alzheimer disease (AD). The trial featured 1169 patients aged 65 to 85 years of age with a global Clinical Dementia Rating score of 0, indicating no cognitive impairment, and elevated brain amyloid levels on 18F-florbetapir PET. At the conclusion of a 240-week treatment period, findings showed a mean change in Preclinical Alzheimer Cognitive Composite (PACC) score of –1.43 in the solanezumab group and –1.13 in the placebo group.

According to an announcement from PepGen, the FDA has lifted a clinical hold on its investigational new drug application (IND) for its phase 1 study assessing PGN-EDODM1, an investigational antisense oligonucleotide (ASO), in patients with myotonic dystrophy type 1 (DM1). The company expects to obtain proof-of-concept data, including transcript splicing and clinical outcome measures, as well as safety data, for treated patients in the study in 2024. Originally started in Canada, FREEDOM-DM1 is a randomized, double-blind, placebo-controlled, single-ascending dose study assessing the safety and tolerability of the ASO in 3 cohorts of 5 mg/kg, 10 mg/kg, and 20 mg/kg dose levels. The study also will look at correction of mis-splicing of transcripts and other clinical functional outcome measures throughout.

After proposing coverage determination earlier in the summer, the Centers for Medicare and Medicaid Services (CMS) announced they will cover PET imaging for the diagnosis of Alzheimer disease (AD), a major step toward ensuring access to novel treatments for patients who may be eligible. The CMS policy to pay for amyloid PET scans beyond clinical trials also removes the current limitation of 1 scan per lifetime. Typically, out-of-pocket amyloid PET scans average $3000 per test or greater. In its coverage memo, CMS also noted that the harms of antiamyloid treatments would be avoided because the drug would not be given to patients that do not have brain amyloid and potentially stopped when brain amyloid was completely removed.

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