NeuroSense Reports Positive Phase 2b Results for PrimeC in ALS, Meets Primary Study Endpoint

PrimeC Achieves Primary Goal in Phase 2b ALS Trial

NeuroSense Therapeutics has announced positive results from its Phase 2b PARADIGM clinical trial evaluating PrimeC for the treatment of amyotrophic lateral sclerosis (ALS).
The study successfully met its primary efficacy endpoint by demonstrating a statistically significant reduction in TDP-43 levels compared with placebo after 180 days of treatment. According to the company, this is the first randomized, double-blind, placebo-controlled clinical trial to show that a treatment can reduce TDP-43 levels in people living with ALS.
The analysis was performed using NeuroDex's ExoSORT technology, which isolates neuron-derived extracellular vesicles from blood samples. This method allows researchers to measure TDP-43 originating specifically from nerve cells rather than from other tissues in the body.

Why TDP-43 Matters in ALS

TDP-43 is considered one of the main biological markers of ALS and is found in more than 97% of patients with the disease.
Researchers believe abnormal accumulation of this protein plays a major role in disease progression. By reducing TDP-43 levels, PrimeC may be targeting one of the underlying causes of ALS rather than only managing symptoms.
The company said the reduction in TDP-43 provides biological evidence that the treatment is engaging a core disease mechanism.

Benefits Continued Throughout the Study

The positive effect on TDP-43 was not limited to the primary analysis at Day 180.
Patients who continued receiving PrimeC maintained lower TDP-43 levels throughout the full 18-month study. At Day 540, the difference between the PrimeC and placebo groups became even more pronounced, showing a highly significant result.
These findings suggest the treatment effect remained consistent over long-term use.

Clinical Results Build on Earlier Findings

The latest biomarker data support previously reported clinical results from the PARADIGM study.
Earlier analyses showed that PrimeC significantly slowed the decline in ALS Functional Rating Scale-Revised (ALSFRS-R) scores at both 12 and 18 months. The treatment also demonstrated an estimated median survival benefit of approximately 15 months compared with placebo.
Researchers also observed favorable changes in several biomarkers linked to ALS, including TDP-43, iron regulation, and disease-associated microRNAs. Throughout the study, PrimeC maintained a favorable safety and tolerability profile, with no new safety concerns reported during up to 18 months of treatment.

Experts See Strong Evidence for Disease Modification

NeuroSense believes the combination of clinical outcomes and biomarker findings strengthens the case for PrimeC as a disease-modifying therapy for ALS.
According to the company, demonstrating a reduction in TDP-43 alongside slower disease progression and improved survival provides one of the most comprehensive clinical datasets currently available for an investigational ALS treatment.
Independent experts involved in the study also noted that showing an effect on TDP-43 is an important step because it suggests the therapy is directly influencing the underlying biology of the disease rather than only treating symptoms.

Company Prepares for Global Phase 3 Trial

Following these positive Phase 2b results, NeuroSense is moving forward with preparations for its global Phase 3 PARAGON study.
The company has already received clearance from the U.S. Food and Drug Administration (FDA) to begin the pivotal trial and is continuing discussions with regulatory agencies in multiple countries, including Canada.
The Phase 3 study is expected to enroll approximately 300 participants, with most patients being recruited in the United States.

About PrimeC

PrimeC is NeuroSense's lead investigational therapy for ALS. It is an extended-release oral formulation that combines two FDA-approved medicines, ciprofloxacin and celecoxib, into a fixed-dose treatment.
The therapy is designed to target several biological pathways involved in ALS progression, including neuroinflammation, oxidative stress, abnormal iron metabolism, and RNA regulation. By addressing multiple disease mechanisms at the same time, PrimeC aims to slow the progression of ALS.

Understanding ALS

Amyotrophic lateral sclerosis, commonly known as ALS, is a progressive neurodegenerative disease that damages nerve cells responsible for controlling voluntary muscle movement.
The disease gradually leads to muscle weakness, paralysis, and eventually respiratory failure. Most patients survive between two and five years after diagnosis.
More than 5,000 people are diagnosed with ALS every year in the United States, and the number of patients is expected to continue increasing over the coming decades, highlighting the need for new treatment options that can modify disease progression.

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