CNM-Au8 Improves Survival in ALS, New Parkinson Risk Factor Identified, Pamrevlumab Falls Short in Phase 3 Study

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

New data from the ongoing phase 2 RESCUE-ALS open-label extension (OLE) trial investigating CNM-Au8 (Clene Nanomedicine) demonstrated survival improvement and improved time to clinical worsening among patients with ALS treated with the agent. These findings produced by using the rank-preserving structural failure time model, a well-recognized method to estimate crossover treatment effects in ALS trials, are cut from the 24-month long-term data of RESCUE-ALS as of July 2023. At the conclusion of the data cutoff point, findings showed a median survival benefit of 19.3 additional months among 45 patients with ALS in comparison with placebo. Notably, the results displayed a 75% decreased risk of long-term all-cause mortality in patients who originally started treatment with CNM-Au8 compared with placebo after adjusting for benefit received by placebo following the switch to CNM-Au8.

A recently published genome-wide association study of nearly 200,000 individuals found a novel genetic risk factor affecting GBA1 in people of African ancestry, which had not been seen in European populations, and could be a major mechanistic basis of Parkinson disease in African populations. The variant on the GBA1 gene was identified by researchers from the Global Genetics Program (GP2), led by those at the National Institutes of Health, the University of Lagos in Nigeria, and University College London (UCL). From the 3 cohorts, 1488 cases of PD and 196,430 controls of African and African admixed ancestry were included in the GWAS meta-analyses. In the overall meta-analysis, the odds ratio (OR) for risk of PD was 1.58.

In recent news, topline data from the phase 3 LELANTOS-2 trial showed that pamrevlumab (FibroGen) in combination with systemic corticosteroids failed to improve ambulatory function in ambulatory patients with Duchenne muscular dystrophy (DMD). FibroGen announced that they are in the process of assessing the totality of the data in order to decide the next steps and plan to communicate the full results of the study at an upcoming medical forum. From baseline to week 52, the treatment did not meet the primary end point of change in North Star Ambulatory Assessment (NSAA) total score. The secondary end points, change from baseline at week 52 in 4-stair climb velocity, 10-meter walk/run test, time to stand, time to loss of ambulation, and proportion of patients with greater than 10 seconds in the 10-meter walk/run test, were also not met.

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