FDA doubles down on neurodegeneration

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Regulator bestows Fast Track and Expanded Access designations on two biotech companies targeting Alzheimer’s and beyond.

Announcements made today by two US biotechs could lead to the accelerated development of novel therapies for neurodegenerative diseases. ProMIS Neurosciences and NKGen Biotech separately revealed progress with the FDA to speed up or expand the potential use of therapeutics targeting conditions marked by progressive neuronal damage, including Alzheimer’s.

ProMIS Neurosciences announced that it has received Fast Track designation from the FDA for its lead therapeutic candidate, PMN310, a monoclonal antibody in development for Alzheimer’s disease. PMN310 is designed to selectively target soluble amyloid-beta oligomers, which are thought to be primary neurotoxins contributing to Alzheimer’s pathology. Unlike treatments that focus on removing amyloid plaques, PMN310 avoids plaque altogether, potentially minimizing the risk of amyloid-related imaging abnormalities (ARIA), a known complication associated with some anti-amyloid therapies.

The Fast Track designation provides ProMIS with opportunities for closer regulatory interaction and potentially quicker review timelines. It follows the launch of the company’s PRECISE-AD Phase 1b clinical trial, which is assessing safety, tolerability, pharmacokinetics and biomarker response in patients with early-stage Alzheimer’s, after last year’s successful Phase 1a trial in healthy volunteers. With plans to report interim biomarker and safety data in mid-2026, and final results later that year, ProMIS aims to clarify whether PMN310’s precision-targeting mechanism can translate into clinical benefit without the common drawbacks of plaque-binding agents.

“We designed PMN310 with a goal of providing Alzheimer’s patients with a safer and more efficacious treatment option, which we believe represents the next generation of Alzheimer’s therapeutics,” said ProMIS CEO Neil Warma. “By selectively targeting only the most harmful, toxic forms of amyloid-beta, we believe PMN310 has the potential to reduce the serious side effects seen with current Alzheimer’s treatments, namely brain swelling and bleeding known as ARIA, while also delivering improved therapeutic benefit to patients.”

Separately, NKGen Biotech disclosed that it has received FDA authorization for an Expanded Access Program (EAP) that allows the use of its investigational therapy, troculeucel, in patients with a range of neurodegenerative diseases. Troculeucel is an enhanced natural killer (NK) cell therapy developed from the patient’s own cells and designed to restore immune function impaired by aging and disease. The therapy has previously shown clinical activity in early-stage trials in Alzheimer’s disease without associated drug-related adverse events, and received its own Fast Track designation earlier this year.

The new EAP authorization enables NKGen to offer troculeucel under an open-label, multi-center protocol to patients diagnosed with various neurodegenerative conditions beyond Alzheimer’s. These include Parkinson’s disease, amyotrophic lateral sclerosis, frontotemporal dementia, progressive supranuclear palsy, corticobasal degeneration, multiple system atrophy, Lewy body dementia and multiple sclerosis. The company can now enroll up to 20 patients through the EAP as it continues its ongoing randomized Phase 2a trial for moderate Alzheimer’s disease.

“There is a common element of autoimmune neuroinflammation in all of these neurodegenerative diseases due to autoreactive T cells, many in response to either an amyloid, alpha-synuclein, or tau protein deposition,” said NKGen CEO Paul Y Song. “We believe there is ample scientific and clinical rationale to offer this to patients with other neurodegenerative diseases for which there is no effective therapy and little hope.”