ALS Agent PrimeC Moves to Phase 3, FDA Clears IND for AB126, APOE Carrier Status Impacts ARIA

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

Shortly after positive topline data from the phase 2 PARADIGM study of PrimeC was released, NeuroSense Therapeutics has announced that the FDA has confirmed its development plans for a phase 3 pivotal trial with the intention of subsequent marketing approval. In collaboration with Biogen, the company also plans on releasing data from PARADIGM assessing PrimeC in biomarkers relevant to the pathology of ALS. The agent met its primary end point in PARADIGM, demonstrating a 37.4% (P = .03) difference on ALS Functional Rating Scale Revised at 6 months vs placebo, while maintaining a strong safety profile. Additionally, investigators recorded a 13% treatment difference in slow vital capacity favoring PrimeC.

The FDA has cleared Aruna Bio’s investigational new drug (IND) application for the company’s lead candidate AB126, an unmodified neural-derived exosome, according to a new announcement. This clearance by the agency paves the way for investigating AB126 in a phase 1b/2a clinical trial among patients with acute ischemic stroke, which the company noted is expected to initiate in the first half of 2024. AB126 has intuitive ability to cross the blood-brain barrier and modulate inflammasome in the central nervous system by action of anti-inflammatory and neuroprotective mechanisms, giving it the potential to treat a range of neurodegenerative disorders, according to Aruna Bio. In addition, the company noted that its neural exosome platform can be combined with therapeutics, such as small molecules, small interfering RNAs, and proteins.

Newly published in Neurology, a genome-wide association study (GWAS) of the phase 3 ENGAGE and EMERGE trials of aducanumab (Aduhelm; Biogen) revealed a strong, significant link between apolipoprotein (APOE) carrier status and the risk of amyloid-related imaging abnormalities. Overall, the research highlights the value genetics add to guiding clinical treatment decisions and the importance of reporting heterozygosity by genotype, given the substantially larger effects observed among ε4 homozygotes vs heterozygotes. All told, participants homozygous for APOE ε4 exhibited 4.28 greater odds of experiencing AIRA-H, 4.58 greater odds of experiencing ARIA-H microhemorrhage, and 7.84 greater odds of experiencing ARIA-H superficial siderosis.

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