Biotechnology Company Announces Positive Biomarker Data From Major Clinical Trial
NeuroSense Therapeutics Announces Positive Biomarker Data from ALS Phase 2b Clinical Trial.
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NeuroSense Therapeutics Ltd. (NASDAQ: NRSN) ("NeuroSense"), a late-clinical stage biotechnology company known for developing novel treatments for severe neurodegenerative diseases, has recently announced positive 12-month iron biomarker data from its Phase IIb study (PARADIGM). This study evaluated both the safety and efficacy of PrimeC in individuals living with Amyotrophic Lateral Sclerosis (ALS). The newly released data aligns with the company's recent announcements highlighting improved survival rates by 43% and a slowed disease progression by 36%.
Understanding ALS and Its Pathophysiology
Amyotrophic lateral sclerosis (ALS) is a multifactorial and incurable neurodegenerative disease that leads to complete paralysis and eventual death, typically within 2-5 years post-diagnosis. Every year, over 5,000 people in the United States are diagnosed with ALS, posing an annual economic burden estimated at $1 billion. The number of individuals affected by ALS is projected to grow by 24% by 2040 in both the U.S. and the EU.
Iron metabolism plays a critical role in the pathophysiology of ALS. Elevated iron levels, particularly involving proteins such as transferrin and ferritin, contribute to the disease's progression. Accumulation of iron in different brain regions of ALS patients has been associated with neuronal damage. Higher ferritin levels, an indicator of iron storage, are frequently observed in ALS patients and are linked to reduced survival and potential oxidative stress. Conversely, lower levels of transferrin, the primary iron transport protein, contribute to iron dysregulation and disease progression. In the PARADIGM Phase IIb study, NeuroSense Therapeutics investigated the impact of PrimeC on iron biomarkers over a 12-month period. The results demonstrated meaningful changes in iron metabolism, which in turn correlated with improved clinical outcomes. Specifically, the study revealed a decrease in ferritin levels and an increase in transferrin levels among patients treated with PrimeC.
The stability of iron levels over the 12-month dosing period was notable, with a mean difference of 4.536 µmol/L (95% CI [1.143, 7.929], p=0.01) when comparing patients who started on placebo and later transitioned to PrimeC after the initial 6-month double-blind phase. These alterations in iron metabolism suggested that PrimeC effectively targets iron regulation, which could mitigate ALS pathology and enhance survival rates.
Clinical Outcomes and Expert Insights
The improvement in iron biomarkers coincided with better functionality and survival rates among patients on PrimeC compared to those on placebo. Merit Cudkowicz, M.D., M.Sc., Chair of Neurology and Director of the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital, remarked on the significance of these findings. Dr. Cudkowicz highlighted that the 12-month results from the PARADIGM Phase IIb study are promising, showing a slowing of disease progression and improved survival outcomes.
With these promising results, NeuroSense Therapeutics is preparing to share additional data with the FDA to determine the clinical and regulatory path forward for PrimeC. The company is also engaged in advanced discussions with several potential development partners to explore marketing opportunities contingent on the successful completion and approval of PrimeC for ALS. Disease progression in ALS is commonly measured using the ALS Functional Rating Scale-Revised (ALSFRS-R). This scale is the most widely used tool for tracking ALS progression, accepted by the FDA, and utilized by neurologists in both clinical practice and trials. The ALSFRS-R assesses patient capabilities across various functional domains, providing a comprehensive measure of disease status and progress.
The announcement of positive biomarker data from NeuroSense Therapeutics’ Phase IIb PARADIGM study represents a significant step forward in the fight against ALS. By demonstrating meaningful changes in iron metabolism, PrimeC has shown promise in mitigating disease pathology and improving survival outcomes for ALS patients. As NeuroSense moves forward with its clinical and regulatory plans, the potential for PrimeC to offer a new therapeutic option for ALS brings hope to many affected by this devastating disease.
NeuroSense Therapeutics Announces Positive Biomarker Data from ALS Phase 2b Clinical Trial
NeuroSense Therapeutics Ltd. (NASDAQ: NRSN) ("NeuroSense"), a late-clinical stage biotechnology company known for developing novel treatments for severe neurodegenerative diseases, has recently announced positive 12-month iron biomarker data from its Phase IIb study (PARADIGM). This study evaluated both the safety and efficacy of PrimeC in individuals living with Amyotrophic Lateral Sclerosis (ALS). The newly released data aligns with the company's recent announcements highlighting improved survival rates by 43% and a slowed disease progression by 36%.
Amyotrophic lateral sclerosis (ALS) is a multifactorial and incurable neurodegenerative disease that leads to complete paralysis and eventual death, typically within 2-5 years post-diagnosis. Every year, over 5,000 people in the United States are diagnosed with ALS, posing an annual economic burden estimated at $1 billion. The number of individuals affected by ALS is projected to grow by 24% by 2040 in both the U.S. and the EU.
Iron metabolism plays a critical role in the pathophysiology of ALS. Elevated iron levels, particularly involving proteins such as transferrin and ferritin, contribute to the disease's progression. Accumulation of iron in different brain regions of ALS patients has been associated with neuronal damage. Higher ferritin levels, an indicator of iron storage, are frequently observed in ALS patients and are linked to reduced survival and potential oxidative stress. Conversely, lower levels of transferrin, the primary iron transport protein, contribute to iron dysregulation and disease progression.
The PARADIGM Phase IIb Study
In the PARADIGM Phase IIb study, NeuroSense Therapeutics investigated the impact of PrimeC on iron biomarkers over a 12-month period. The trial was prospective, multinational, randomized, double-blind, and placebo-controlled. It involved 68 participants living with ALS from Canada, Italy, and Israel. Notably, 96% of the participants who completed the initial 6-month double-blind portion opted to continue receiving treatment with PrimeC through a 12-month open-label extension.
As previously reported, the 6-month double-blind segment showed clinically meaningful signs of efficacy. Specifically, there was a 29% difference in favor of PrimeC versus placebo in the intent-to-treat (ITT) population analysis. Additionally, the per-protocol (PP) top-line analysis from PARADIGM observed a statistically significant slowing of disease progression, with a 37.4% (p=0.03) difference in ALS Functional Rating Scale-Revised (ALSFRS-R) scores favoring PrimeC over placebo.
Most patients enrolled in both the active and placebo arms of the trial were concurrently treated with Riluzole, the standard of care medication for ALS. This indicates that PrimeC's efficacy in slowing disease progression extends beyond the benefits offered by existing FDA-approved ALS medications. The results demonstrated meaningful changes in iron metabolism, which correlated with improved clinical outcomes. Specifically, the study revealed a decrease in ferritin levels and an increase in transferrin levels among patients treated with PrimeC.
The stability of iron levels over the 12-month dosing period was notable. There was a mean difference of 4.536 µmol/L (95% CI [1.143, 7.929], p=0.01) when comparing patients who started on placebo and later transitioned to PrimeC after the initial 6-month double-blind phase. These alterations in iron metabolism suggested that PrimeC effectively targets iron regulation, which could mitigate ALS pathology and enhance survival rates.
Clinical Outcomes and Expert Insights
The improvement in iron biomarkers coincided with better functionality and survival rates among patients on PrimeC compared to those on placebo. Merit Cudkowicz, M.D., M.Sc., Chair of Neurology and Director of the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital, remarked on the significance of these findings. Dr. Cudkowicz highlighted that the 12-month results from the PARADIGM Phase IIb study are promising, showing a slowing of disease progression and improved survival outcomes. She emphasized that this analysis shows PrimeC's ability to regulate the iron panel in people living with ALS, demonstrating the drug’s target engagement.
With these promising results, NeuroSense Therapeutics is preparing to share additional data with the FDA to determine the clinical and regulatory path forward for PrimeC. The company is also engaged in advanced discussions with several potential development partners to explore marketing opportunities contingent on the successful completion and approval of PrimeC for ALS.
PrimeC is NeuroSense's lead drug candidate. It is a novel extended-release oral formulation composed of a unique fixed-dose combination of two FDA-approved drugs: ciprofloxacin and celecoxib. PrimeC is designed to synergistically target several key mechanisms of ALS, including motor neuron degeneration, inflammation, iron accumulation, and impaired RNA regulation, potentially inhibiting ALS progression. NeuroSense completed a Phase 2a clinical trial that met its safety and efficacy endpoints. These included reducing functional and respiratory deterioration and generating statistically significant changes in ALS-related biological markers, indicating PrimeC's biological activity. PrimeC has been granted Orphan Drug Designation by both the U.S. Food and Drug Administration and the European Medicines Agency.
NeuroSense Therapeutics, Ltd. is a clinical-stage biotechnology company focused on discovering and developing treatments for patients suffering from debilitating neurodegenerative diseases. NeuroSense believes that these diseases, which include ALS, Alzheimer's disease, and Parkinson's disease, represent some of the most unmet medical needs of our time, with limited effective therapeutic options available for patients to date. Due to the complexity of neurodegenerative diseases and based on strong scientific research on a large panel of related biomarkers, NeuroSense's strategy is to develop combined therapies targeting multiple pathways associated with these diseases.
The announcement of positive biomarker data from NeuroSense Therapeutics’ Phase IIb PARADIGM study represents a step forward in the fight against ALS. By demonstrating meaningful changes in iron metabolism, PrimeC has shown promise in mitigating disease pathology and improving survival outcomes for ALS patients. As NeuroSense moves forward with its clinical and regulatory plans, the potential for PrimeC to offer a new therapeutic option for ALS brings hope to many affected by this devastating disease.
Disclaimer: This article is intended for informational purposes only and should not be considered a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of your physician or other qualified health provider with any questions you may have regarding a medical condition.
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