Massachusetts-based biotech NeuroSense Therapeutics’ combination amyotrophic lateral sclerosis (ALS) therapy, PrimeC, has been shown to reduce a key biomarker of progression in the neurodegenerative disease – a development that an ALS specialist calls “compelling”.
During the Phase IIb PARADIGM study (NCT05357950), PrimeC triggered a statistically significant reduction in the levels of a protein called TDP-43 – meeting the trial’s primary endpoint.
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TDP-43, a key protein associated with RNA and DNA processing in healthy cells, is commonly considered a key driver of ALS pathology, as its misfolding and aggregation are often linked to neurodegeneration within patients. NeuroSense estimates that TDP-43 pathology is associated with 97% of all ALS cases and is widely linked to disease progression.
Alongside its TDP-43-reducing activity, PrimeC prompted a sustained and durable reduction in the protein’s levels over an 18-month treatment period, highlighting the drug’s potential to offer long-lasting benefits for patients.
NeuroSense’s mid-stage win comes shortly after the drug significantly slowed ALS decline by 32.8% at the 18-month mark, while offering a 15-month median survival benefit over placebo, as per an earlier readout from the PARADIGM study. The biotech recently secured clearance from the US Food and Drug Administration (FDA) to initiate the global Phase III PARAGON study of PrimeC.
NeuroSense’s PrimeC combines the common antibiotic ciprofloxacin and anti-inflammatory therapy, celecoxib, by addressing three key underlying disease mechanisms associated with ALS. Through the drug’s dual-pronged approach, NeuroSense proposes that PrimeC can address brain inflammation, while stemming the abnormal activity of microRNAs (miRNAs) linked to gene control and preventing the buildup of iron in the neurones – factors that have all previously been linked to disease progression in ALS.
Tackling unmet needs in ALS
According to NeuroSense, the updated results from the PARADIGM study make PrimeC the first ALS therapy to facilitate a significant drop of TDP-43 protein, which CEO Alon Ben-Noon says provides a “compelling and highly differentiated body of evidence” that touts PrimeC’s potential as a disease-modifying therapy (DMT) in ALS.
Merit Cudkowicz, director of the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital, echoed Ben-Noon’s sentiments, noting that PrimeC’s impact on TDP-43 “suggests target engagement of a pathological process present in the majority of people with ALS”.
“When viewed together with the previously reported safety, biomarker and clinical outcome data, and the high unmet need, these results provide compelling data supporting advancement into a confirmatory Phase III clinical trial,” added Cudkowicz.
As NeuroSense develops PrimeC further, several other players are also looking to take their drugs to the market, with Spinogenix advancing its DMT, SPG302, to a registrational trial following the positive outcome of a mid-stage study in ALS.
AL-S Pharma is also taking its drug, AP-101, to Phase III after the drug met its efficacy and safety endpoints in a Phase II trial.
According to a report from GlobalData, parent company of Clinical Trials Arena, there are 12 novel ALS agents projected to launch between 2024 and 2029, which will become “key drivers” of future market growth.
