NeuroSense Therapeutics Announces Positive Biomarker Data from ALS Phase 2b Clinical Trial
NeuroSense Therapeutics (NASDAQ: NRSN) has announced positive 12-month iron biomarker data from its Phase IIb PARADIGM study of PrimeC in ALS patients. The results show significant improvements in iron regulation, aligning with previously reported 36% slower disease progression and 43% improved survival rates. Key findings include a decrease in ferritin levels and an increase in transferrin levels, indicating alleviation of ALS pathology. Iron levels remained stable over the 12-month dosing period, with a mean difference of 4.536 µmol/L compared to the placebo group. These positive changes in iron metabolism correlate with better functionality and survival rates in patients treated with PrimeC. The company is preparing to share additional data with the FDA and is in discussions with potential development partners for marketing opportunities.
NeuroSense Therapeutics (NASDAQ: NRSN) ha annunciato dati positivi sui biomarcatori del ferro a 12 mesi dallo studio Phase IIb PARADIGM di PrimeC in pazienti affetti da SLA. I risultati mostrano significativi miglioramenti nella regolazione del ferro, in linea con una progressione della malattia rallentata del 36% e tassi di sopravvivenza migliorati del 43%. Tra i risultati chiave si evidenziano una diminuzione dei livelli di ferritina e un aumento dei livelli di transferrina, che indicano un miglioramento della patologia della SLA. I livelli di ferro sono rimasti stabili durante il periodo di somministrazione di 12 mesi, con una differenza media di 4,536 µmol/L rispetto al gruppo placebo. Questi cambiamenti positivi nel metabolismo del ferro sono correlati a una migliore funzionalità e a tassi di sopravvivenza nei pazienti trattati con PrimeC. L'azienda si sta preparando a condividere ulteriori dati con la FDA ed è in discussione con potenziali partner di sviluppo per opportunità di marketing.
NeuroSense Therapeutics (NASDAQ: NRSN) ha anunciado datos positivos de biomarcadores de hierro a 12 meses del estudio Phase IIb PARADIGM de PrimeC en pacientes con ELA. Los resultados muestran mejoras significativas en la regulación del hierro, alineándose con un 36% de progresión de la enfermedad más lenta y un 43% de aumento en las tasas de supervivencia. Los hallazgos clave incluyen una disminución en los niveles de ferritina y un aumento en los niveles de transferrina, indicando una mejora de la patología de la ELA. Los niveles de hierro se mantuvieron estables durante el período de dosificación de 12 meses, con una diferencia media de 4,536 µmol/L en comparación con el grupo placebo. Estos cambios positivos en el metabolismo del hierro se correlacionan con una mejor funcionalidad y tasas de supervivencia en pacientes tratados con PrimeC. La empresa se está preparando para compartir datos adicionales con la FDA y está en conversaciones con posibles socios de desarrollo para oportunidades de comercialización.
NeuroSense Therapeutics (NASDAQ: NRSN)는 ALS 환자를 대상으로 한 Phase IIb PARADIGM 연구에서 PrimeC에 대한 12개월 철 바이오마커 데이터 긍정적인 결과를 발표했습니다. 결과는 철 조절에서의 유의미한 개선을 보여주며, 이는 병이 36% 느리게 진행되고 생존율이 43% 향상된 이전 보고와 일치합니다. 주요 발견으로는 페리틴 수치의 감소와 트랜스페린 수치의 증가가 있으며, 이는 ALS 병리의 완화를 나타냅니다. 철 수치는 12개월 투여 기간 동안 안정적으로 유지되었으며, 위약 그룹에 비해 평균 4.536 µmol/L의 차이를 보였습니다. 철 대사의 이러한 긍정적인 변화는 PrimeC로 치료받은 환자들의 기능과 생존율의 개선과 관련이 있습니다. 회사는 FDA와 추가 데이터를 공유할 준비를 하고 있으며, 마케팅 기회를 위한 잠재적 개발 파트너와 논의 중에 있습니다.
NeuroSense Therapeutics (NASDAQ: NRSN) a annoncé des données positives sur les biomarqueurs de fer à 12 mois issues de son étude Phase IIb PARADIGM sur PrimeC chez des patients atteints de SLA. Les résultats montrent des améliorations significatives dans la régulation du fer, en accord avec un ralentissement de la progression de la maladie de 36 % et une augmentation de 43 % des taux de survie précédemment rapportés. Les résultats clés incluent une diminution des niveaux de ferritine et une augmentation des niveaux de transferrine, indiquant un soulagement de la pathologie SLA. Les niveaux de fer sont restés stables pendant la période de traitement de 12 mois, avec une différence moyenne de 4,536 µmol/L par rapport au groupe placebo. Ces changements positifs dans le métabolisme du fer sont corrélés à une meilleure fonctionnalité et à des taux de survie chez les patients traités avec PrimeC. L'entreprise se prépare à partager des données supplémentaires avec la FDA et est en discussion avec des partenaires potentiels pour des opportunités commerciales.
NeuroSense Therapeutics (NASDAQ: NRSN) hat positive 12-Monats-Eisen-Biomarker-Daten aus der Phase-IIb PARADIGM-Studie zu PrimeC bei ALS-Patienten bekannt gegeben. Die Ergebnisse zeigen signifikante Verbesserungen in der Eisenregulation, im Einklang mit zuvor berichteten 36% langsamerem Krankheitsverlauf und 43% verbesserten Überlebensraten. Zu den wichtigsten Ergebnissen gehören ein Rückgang der Ferritinkonzentration und ein Anstieg der Transferrinwerte, was auf eine Linderung der ALS-Pathologie hinweist. Die Eisenwerte blieben während der 12-monatigen Behandlungsdauer stabil, mit einem mittleren Unterschied von 4,536 µmol/L im Vergleich zur Placebo-Gruppe. Diese positiven Veränderungen im Eisenmetabolismus korrelieren mit einer besseren Funktionalität und Überlebensrate bei Patienten, die mit PrimeC behandelt wurden. Das Unternehmen bereitet sich darauf vor, zusätzliche Daten bei der FDA vorzulegen und führt Gespräche mit potenziellen Entwicklungspartnern über Vermarktungsmöglichkeiten.
- PrimeC demonstrated 36% slower disease progression and 43% improved survival rates in ALS patients
- Significant improvements in iron regulation biomarkers, aligning with better clinical outcomes
- Decrease in ferritin levels and increase in transferrin levels, indicating alleviation of ALS pathology
- Stable iron levels over 12-month dosing period, with a mean difference of 4.536 µmol/L compared to placebo
- Positive results support proceeding to Phase 3 testing of PrimeC in ALS
- None.
The 12-month biomarker data from NeuroSense's Phase IIb PARADIGM study for PrimeC in ALS patients is highly significant. The results demonstrate PrimeC's ability to regulate iron metabolism, a important factor in ALS pathology. Specifically:
- Ferritin levels decreased significantly, indicating reduced iron storage and potentially less oxidative stress.
- Transferrin levels increased, suggesting improved iron transport and regulation.
- Iron levels remained stable over the 12-month period, with a mean difference of
4.536 µmol/L (p=0.01) compared to the placebo-to-PrimeC transition group.
These biomarker changes align with the previously reported 36% slowing of disease progression and 43% improvement in survival rates. The data strongly supports PrimeC's mechanism of action and its potential as a transformative ALS therapy. However, it's important to note that while these results are promising, they still need to be confirmed in a larger Phase III trial to establish definitive efficacy and safety.
The endorsement from Dr. Merit Cudkowicz, a leading ALS researcher, adds credibility to these findings. Her support for proceeding to Phase III testing is a positive signal for the drug's potential. As NeuroSense prepares for FDA discussions and explores partnerships, investors should watch for announcements regarding the Phase III trial design and any potential collaborations, as these will be critical next steps in PrimeC's development journey.
This announcement presents a significant positive development for NeuroSense Therapeutics (NASDAQ: NRSN). The biomarker data from the Phase IIb trial provides strong support for PrimeC's efficacy in treating ALS, potentially positioning it as a breakthrough therapy in a market with treatment options. Key financial implications include:
- Market Potential: Given the improved survival rates and slowed disease progression, PrimeC could command premium pricing if approved, potentially generating substantial revenue for NeuroSense.
- Partnership Opportunities: The company's ongoing discussions with potential development partners could lead to lucrative collaboration agreements, providing additional funding and expertise for Phase III trials and commercialization.
- Investor Confidence: Positive clinical data typically boosts investor confidence, which could lead to increased stock valuation and easier access to capital if needed for future development stages.
However, investors should be cautious of potential risks:
- The need for a successful Phase III trial before potential FDA approval.
- Possible dilution if additional funding is required for the Phase III trial.
- Competition from other ALS therapies in development.
Overall, this news significantly de-risks NeuroSense's lead asset and enhances the company's value proposition. Investors should closely monitor upcoming FDA interactions and potential partnership announcements as these could be significant catalysts for the stock price.
12-month data demonstrated regulation of iron levels, which aligns with improved ALS survival and disease mitigation
Previous results showed PrimeC slowed disease progression by
ALS is a multifactorial disease in which iron and its related proteins play a critical role in its pathophysiology. These new results demonstrate the target engagement of PrimeC in iron regulation, which is linked to disease mitigation and improved survival. Iron metabolism is crucial in ALS pathology, with transferrin and ferritin being significant contributors to the progress of the disease. Iron accumulation in various brain regions of ALS patients has been linked to neuronal damage. Elevated ferritin levels, an indicator of iron storage, are consistently observed in ALS patients and are associated with reduced survival and potential oxidative stress. Conversely, lower levels of transferrin, the primary iron transport protein, contribute to iron dysregulation and disease progression.
The 12-month study results show a significant decrease in ferritin levels and a corresponding increase in transferrin levels, both indicating alleviation of the pathology. Iron levels remained stable over the 12-month dosing period, with a mean difference of 4.536 µmol/L (
These positive changes in iron metabolism align with improved clinical outcomes. Patients on PrimeC maintained better functionality and survival rates compared to those on placebo.
"The 12-month results from the PARADIGM Phase IIb study are encouraging, showing slowing of disease progression and improved survival outcomes," said Merit Cudkowicz, M.D., M.Sc., Chair of Neurology and Director of the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital. "This new analysis highlights PrimeC's ability to regulate the iron panel in people living with ALS, underscoring the drug's target engagement. These findings strongly support the proceeding to phase 3 testing of PrimeC in ALS."
The Company is currently compiling additional data to share with the FDA for discussion to determine the clinical and regulatory path forward. Additionally, the Company is continuing advanced discussions with several potential development partners to explore marketing opportunities following the potential completion and approval of PrimeC for ALS.
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 ALSFRS-R
Disease progression is measured by the ALS Functional Rating Scale-Revised (ALSFRS-R), which is the most widely used ALS tracking tool accepted by the FDA, utilized by neurologists treating ALS patients, in clinical trials, and by other regulators to determine disease progression. It tracks 12 changes in a person's physical abilities over time including functions such as: speech, walking, climbing stairs, dressing/hygiene, handwriting, turning in bed, cutting food, salivation, swallowing, and breathing. A single point change on the ALSFRS-R has a significant impact on ALS patients, such as the transition from independent feeding to requiring assistance or independent breathing to needing to use a machine ventilator.
About PARADIGM
PARADIGM is a prospective, multinational, randomized, double-blind, placebo-controlled Phase 2b (NCT05357950) clinical trial of PrimeC in ALS. The trial included 68 participants living with ALS in Canada, Italy, and Israel.
As previously reported, in the 6-month double-blind segment of the trial, the data showed clinically meaningful signs of efficacy with a
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 PrimeC as a potential treatment for people with ALS. 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 the Company will not enter into an agreement with a development partner at all or on favorable terms; unexpected R&D costs or operating expenses, a delay in the reporting of additional results from PARADIGM clinical trial, a delay in a Phase 3 trial for PrimeC in ALS, the timing of expected regulatory and business milestones, risks associated with meeting with the FDA to determine the best path forward following the results from PARADIGM clinical trial, including a delay in any such meeting; the potential for PrimeC to safely and effectively target ALS; preclinical and clinical data for PrimeC; the uncertainty regarding outcomes and the timing of current and future clinical trials; timing for reporting data; the development and commercial potential of any product candidates of NeuroSense; the ability of NeuroSense to remain listed on Nasdaq; and other risks and uncertainties set forth in NeuroSense's filings with the Securities and Exchange Commission (SEC). You should not rely on these statements as representing our views in the future. More information about the risks and uncertainties affecting NeuroSense is contained under the heading "Risk Factors" in the Annual Report on Form 20-F filed with the Securities and Exchange Commission on April 4, 2024 and NeuroSense's subsequent filings with the SEC. Forward-looking statements contained in this announcement are made as of this date, and NeuroSense undertakes no duty to update such information except as required under applicable law.
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FAQ
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