NeuroSense Therapeutics Announces Transformative Phase 2b MicroRNA Data, Highlighting PrimeC's Promise as a Disease-Modifying ALS Treatment
NeuroSense Therapeutics (NASDAQ: NRSN) has unveiled promising new findings from its Phase 2b PARADIGM clinical trial for PrimeC, its investigational ALS treatment. The data demonstrates PrimeC's significant impact on microRNA (miRNA) modulation, showing a 33% reduction in disease progression and 58% improvement in survival rates.
Key findings include the consistent downregulation of 161 miRNAs in PrimeC-treated patients, while no significant changes were detected in the placebo arm. The treatment specifically downregulated ALS-related miRNAs miR-199 and miR-181, which are associated with disease progression and survival.
The research, presented at the 77th Annual American Academy of Neurology Meeting, was conducted in collaboration with Prof. Noam Shomron of Tel Aviv University and presented by Dr. Jeffrey Rosenfeld of Loma Linda University School of Medicine.
NeuroSense Therapeutics (NASDAQ: NRSN) ha svelato risultati promettenti dal suo studio clinico di fase 2b PARADIGM per PrimeC, il suo trattamento sperimentale per la SLA. I dati dimostrano l'impatto significativo di PrimeC sulla modulazione dei microRNA (miRNA), mostrando una riduzione del 33% nella progressione della malattia e un miglioramento del 58% nei tassi di sopravvivenza.
I risultati chiave includono la downregulation costante di 161 miRNA nei pazienti trattati con PrimeC, mentre non sono stati rilevati cambiamenti significativi nel gruppo placebo. Il trattamento ha specificamente downregolato i miRNA correlati alla SLA, miR-199 e miR-181, associati alla progressione della malattia e alla sopravvivenza.
La ricerca, presentata al 77° Congresso Annuale dell'Accademia Americana di Neurologia, è stata condotta in collaborazione con il Prof. Noam Shomron dell'Università di Tel Aviv ed è stata presentata dal Dr. Jeffrey Rosenfeld della Loma Linda University School of Medicine.
NeuroSense Therapeutics (NASDAQ: NRSN) ha revelado hallazgos prometedores de su ensayo clínico de fase 2b PARADIGM para PrimeC, su tratamiento experimental para la ELA. Los datos demuestran el impacto significativo de PrimeC en la modulación de microARN (miARN), mostrando una reducción del 33% en la progresión de la enfermedad y una mejora del 58% en las tasas de supervivencia.
Los hallazgos clave incluyen la regulación a la baja constante de 161 miARN en pacientes tratados con PrimeC, mientras que no se detectaron cambios significativos en el grupo placebo. El tratamiento específicamente reguló a la baja los miARN relacionados con la ELA, miR-199 y miR-181, que están asociados con la progresión de la enfermedad y la supervivencia.
La investigación, presentada en el 77º Congreso Anual de la Academia Americana de Neurología, fue realizada en colaboración con el Prof. Noam Shomron de la Universidad de Tel Aviv y presentada por el Dr. Jeffrey Rosenfeld de la Escuela de Medicina de la Universidad de Loma Linda.
NeuroSense Therapeutics (NASDAQ: NRSN)는 ALS 치료제 PrimeC에 대한 2b 단계 PARADIGM 임상 시험에서 유망한 새로운 발견을 공개했습니다. 데이터는 PrimeC가 미세 RNA (miRNA) 조절에 미치는 중요한 영향을 보여주며, 질병 진행률을 33% 감소시키고 생존율을 58% 개선했습니다.
주요 발견으로는 PrimeC 치료를 받은 환자에서 161개의 miRNA가 일관되게 하향 조절된 반면, 위약 그룹에서는 유의미한 변화가 감지되지 않았습니다. 이 치료는 질병 진행 및 생존과 관련된 ALS 관련 miRNA인 miR-199 및 miR-181을 특히 하향 조절했습니다.
이 연구는 제77회 미국 신경학회 연례 회의에서 발표되었으며, 텔아비브 대학교의 Noam Shomron 교수와 협력하여 진행되었고, 로마 린다 대학교 의과대학의 Jeffrey Rosenfeld 박사가 발표했습니다.
NeuroSense Therapeutics (NASDAQ: NRSN) a dévoilé des résultats prometteurs de son essai clinique de phase 2b PARADIGM pour PrimeC, son traitement expérimental contre la SLA. Les données montrent l'impact significatif de PrimeC sur la modulation des microARN (miARN), affichant une réduction de 33 % de la progression de la maladie et une amélioration de 58 % des taux de survie.
Les résultats clés incluent la régulation à la baisse constante de 161 miARN chez les patients traités avec PrimeC, tandis qu'aucun changement significatif n'a été détecté dans le groupe placebo. Le traitement a spécifiquement régulé à la baisse les miARN associés à la SLA, miR-199 et miR-181, qui sont liés à la progression de la maladie et à la survie.
La recherche, présentée lors de la 77e réunion annuelle de l'American Academy of Neurology, a été réalisée en collaboration avec le Prof. Noam Shomron de l'Université de Tel Aviv et présentée par le Dr. Jeffrey Rosenfeld de la Loma Linda University School of Medicine.
NeuroSense Therapeutics (NASDAQ: NRSN) hat vielversprechende neue Ergebnisse aus seiner Phase 2b PARADIGM-Studie für PrimeC, seine experimentelle ALS-Behandlung, vorgestellt. Die Daten zeigen die signifikante Wirkung von PrimeC auf die Modulation von Mikro-RNA (miRNA) und belegen eine Reduktion der Krankheitsprogression um 33% sowie eine Verbesserung der Überlebensraten um 58%.
Wichtige Ergebnisse umfassen die konstante Herabregulierung von 161 miRNAs bei mit PrimeC behandelten Patienten, während im Placebo-Arm keine signifikanten Veränderungen festgestellt wurden. Die Behandlung hat speziell die mit ALS assoziierten miRNAs miR-199 und miR-181 herabreguliert, die mit Krankheitsprogression und Überleben in Verbindung stehen.
Die Forschung, die auf dem 77. jährlichen Treffen der American Academy of Neurology präsentiert wurde, wurde in Zusammenarbeit mit Prof. Noam Shomron von der Universität Tel Aviv durchgeführt und von Dr. Jeffrey Rosenfeld der Loma Linda University School of Medicine vorgestellt.
- 33% reduction in disease progression demonstrated in Phase 2b trial
- 58% improvement in survival rates in treated patients
- Consistent downregulation of 161 miRNAs showing clear therapeutic effect
- Successful targeting of specific ALS-related miRNAs (miR-199 and miR-181)
- None.
Insights
NeuroSense's Phase 2b PARADIGM trial data reveals significant microRNA modulation that substantiates PrimeC's potential as a disease-modifying ALS treatment. The consistent downregulation of 161 miRNAs in treated patients - particularly ALS-associated miR-199 and miR-181 - provides a molecular explanation for the previously reported
This microRNA data is particularly valuable as it offers mechanistic validation beyond symptomatic improvement. ALS therapeutics historically struggle to demonstrate disease-modifying effects, making these findings exceptionally meaningful. The downregulation of miR-199 (linked to neuroinflammation) and miR-181 (associated with mortality risk) directly addresses underlying ALS pathophysiology rather than merely masking symptoms.
The consistency of miRNA modulation across multiple timepoints strengthens confidence in these findings, while the absence of significant changes in the placebo arm further validates these effects as treatment-specific. This robust biomarker data provides crucial support for PrimeC's continued development and positions it favorably for potential Phase 3 investigation.
NeuroSense's latest PARADIGM trial data represents a critical inflection point for this
The presentation at AAN's Late Breaker session – a highly selective forum typically reserved for groundbreaking research – provides external validation of these findings' significance. Collaboration with established researchers from Tel Aviv University and Loma Linda University further strengthens credibility.
The consistent molecular signature across all timepoints demonstrates reliable biological activity, addressing a key investor concern for early-stage therapeutics. Most importantly, this data bridges the gap between clinical outcomes and underlying biology, providing a compelling scientific narrative that explains how PrimeC delivers its clinical benefits.
For a small neurodegenerative disease-focused biotech, establishing this level of mechanistic understanding significantly enhances PrimeC's development prospects and partnership potential. These findings position NeuroSense favorably for advancing toward pivotal trials with a differentiated, potentially disease-modifying ALS treatment.
New miRNA findings unveiled at the AAN, reveal key biological mechanisms behind PrimeC's clinical benefit in ALS.
These data are particularly significant because they:
- Represent an important development in understanding how the therapy may slow disease progression in people living with ALS.
- Reveal the consistent effect of PrimeC on altering the expression of miRNAs associated with ALS, reinforcing its potential role in addressing key pathological processes in ALS and underscoring its potential as a transformative, multi-targeted therapeutic.
- Align with the previously reported clinical benefits, including the
33% reduction in disease progression and58% improvement in survival rates observed with PrimeC treatment.
"We are excited by these results from the PARADIGM trial, which provide further compelling support of PrimeC's potential to modify disease progression in people living with ALS," said Alon Ben-Noon, CEO of NeuroSense Therapeutics. "The ability of PrimeC to affect miRNA expression and the underlying disease pathways not only validates our approach but positions PrimeC as a promising therapeutic candidate for ALS."
Key Findings:
- Consistent downregulation of 161 miRNAs in PrimeC-treated patients
In ALS patients treated with PrimeC, the study observed a profound and consistent downregulation of 161 mature miRNAs across all time points in the double-blind period. In contrast, no significant changes were detected in the placebo arm, emphasizing PrimeC's targeted biological activity and potential for disease modification. - Clinical relevance: miRNA modulation and disease progression
PrimeC treatment led to the significant downregulation of ALS-related miRNAs, including miR-199 and miR-181, both of which are associated with ALS disease progression and survival. Notably, both miR-199 and miR-181 are upregulated in ALS. miR-199 is associated with neuroinflammation and reduced neuronal survival, while miR-181 correlates with a higher risk of mortality. This downregulation of both miRNAs in patients corresponds with the already reported improvement in clinical function.
This research, conducted in collaboration with Prof. Noam Shomron of
"What stood out to us was the consistent modulation of key miRNAs closely associated with ALS biology," said Prof. Shomron. "Observing these changes as part of a rigorous and well-controlled analysis was particularly compelling. It speaks to the potential of PrimeC to directly influence key molecular mechanisms in ALS, in a manner that is both data-driven and unbiased."
As NeuroSense advances PrimeC's clinical development, the company remains focused on deepening mechanistic insights and identifying miRNA-based biomarkers for predicting and monitoring treatment response. The strength of the Phase 2b PARADIGM miRNA data provides a solid foundation for the next phase of development, including further investigations into PrimeC's impact on miRNA maturation and its relevance in other neurodegenerative indications.
About MicroRNAs
MicroRNAs (miRNAs) are crucial regulators of gene expression, controlling cellular processes such as differentiation, proliferation, and apoptosis. Dysregulation of miRNA are known to contribute to neurodegenerative diseases such as ALS, exacerbating inflammation, impairing neuronal survival, and disrupting critical cellular pathways.
About ALS
Amyotrophic lateral sclerosis ("ALS") is an incurable neurodegenerative disease that causes complete paralysis and death within 2-5 years from diagnosis. Every year, more than 5,000 people are diagnosed with ALS in the
About PrimeC
PrimeC, NeuroSense's lead drug candidate, 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 that contribute to motor neuron degeneration, inflammation, iron accumulation and impaired ribonucleic acid ("RNA") regulation to potentially inhibit the progression of ALS. NeuroSense completed a Phase 2a clinical trial which met its safety and efficacy endpoints including reducing functional and respiratory deterioration and statistically significant changes in ALS-related biological markers indicating PrimeC's biological activity. PrimeC was granted Orphan Drug Designation by the
About NeuroSense
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 amyotrophic lateral sclerosis (ALS), Alzheimer's disease and Parkinson's disease, among others, represent one of the most significant 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.
For additional information, we invite you to visit our website and follow us on LinkedIn, YouTube and X. Information that may be important to investors may be routinely posted on our website and these social media channels.
Forward-Looking Statements
This press release contains "forward-looking statements" that are subject to substantial risks and uncertainties. All statements, other than statements of historical fact, contained in this press release are forward-looking statements. Forward-looking statements contained in this press release may be identified by the use of words such as "anticipate," "believe," "contemplate," "could," "estimate," "expect," "intend," "seek," "may," "might," "plan," "potential," "predict," "project," "target," "aim," "should," "will" "would," or the negative of these words or other similar expressions, although not all forward-looking statements contain these words. Forward-looking statements are based on NeuroSense Therapeutics' current expectations and are subject to inherent uncertainties, risks and assumptions that are difficult to predict and include statements regarding the potential of PrimeC. Further, certain forward-looking statements are based on assumptions as to future events that may not prove to be accurate. The future events and trends may not occur and actual results could differ materially and adversely from those anticipated or implied in the forward looking statements. These risks include the risk that regulatory approvals for PrimeC will be delayed or not obtained in
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FAQ
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