DelveInsight’s, “Amyotrophic Lateral Sclerosis Pipeline Insight 2024” report provides comprehensive insights about 75+ companies and 80+ pipeline drugs in Amyotrophic Lateral Sclerosis pipeline landscape. It covers the Amyotrophic Lateral Sclerosis pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Amyotrophic Lateral Sclerosis therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
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Key Takeaways from the Amyotrophic Lateral Sclerosis Pipeline Report
- In September 2024:- Ionis Pharmaceuticals Inc.- A Phase 1-3 Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION363 in Amyotrophic Lateral Sclerosis Patients With Fused in Sarcoma Mutations (FUS-ALS). The primary purpose of this study is to evaluate the efficacy of ION363 on clinical function and survival in carriers of fused in sarcoma mutations with amyotrophic lateral sclerosis (FUS-ALS).
- In September 2024:- UniQure Biopharma B.V.- A Phase 1/2, Multicenter, Single Ascending Dose Study to Evaluate the Safety, Tolerability, and Exploratory Efficacy of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants with SOD1 Amyotrophic Lateral Sclerosis (SOD1-ALS). This is the study of AMT-162 in Participants with SOD1-ALS and is designed to evaluate the safety, tolerability, and exploratory efficacy of intrathecally administered gene therapy AMT-162. AMT-162-001 is a Phase 1/2, multi-center, single ascending dose study.
- In September 2024:- Rapa Therapeutics LLC- RAPA-501-ALS is an Intermediate-Size Expanded Access Trial of RAPA-501 autologous hybrid TREG/Th2 cells in patients living with amyotrophic lateral sclerosis (pwALS). This is an open-label, non-randomized, multi-center intermediate size expanded access clinical trial of single-agent RAPA-501 cells in patients with high-risk ALS who are not eligible for other ALS clinical trials.
- In September 2024:- Revalesio Corporation- Nebulized RNS60 for the Treatment of Amyotrophic Lateral Sclerosis. The purpose of this study is to determine whether nebulized RNS60 is effective in the treatment of amyotrophic lateral sclerosis (ALS).
- In September 2024:- Everfront Biotech Co., Ltd.- The study drugs (including placebo) will be administered at the study site by following the investigator’s instructions to either perform blood sampling for pharmacokinetic evaluation or maximize the treatment compliance.
- DelveInsight’s Amyotrophic Lateral Sclerosis pipeline report depicts a robust space with 75+ active players working to develop 80+ pipeline therapies for Amyotrophic Lateral Sclerosis treatment.
- The leading Amyotrophic Lateral Sclerosis Companies such as Ionis Pharmaceuticals, 1ST Biotherapeutics, Scholar Rock, Revalesio, QurAlis Corporation, Sanofi, MediciNova, Helixmith, Verge Genomics, UCB, and others.
- Promising Amyotrophic Lateral Sclerosis Therapies such as Dazucorilant 300 mg, AP-101, Fasudil (WP-0512), ION363, and others.
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Amyotrophic Lateral Sclerosis Emerging Drugs Profile
- MN-166: MediciNova
MN-166 is a first-in-class, orally bioavailable, small molecule glial attenuator that suppresses pro-inflammatory cytokines IL-1ß, TNF-a, and IL-6, and may upregulate the anti-inflammatory cytokine IL-10. It has additionally been shown to be a toll-like receptor 4 (TLR4) functional antagonist that may contribute to its attenuation of neuroinflammation. The drug is currently being evaluated under Phase II/III clinical trial for the treatment of ALS.
- RNS60: Revalesio
RNS60 is being developed to provide disease modifying and potentially restorative treatments for neurological diseases. The drug activates intracellular signaling pathways to increase mitochondrial biogenesis and function and reduce inflammation. RNS60 safely protects neurons and oligodendrocytes and modulates the activity of immune cells to restore homeostasis. RNS60 has been granted Orphan Drug and Fast Track designations for ALS from the US Food and Drug Administration. The drug is currently being evaluated under Phase II clinical trial for the treatment of ALS.
- VM202: Helixmith
VM202 is an innovative gene therapy drug that provides fundamental treatment through tissue regeneration. The drug is injected along the peripheral nerves and their branches, the internal production of HGF may protect nerves and promote the growth of neurons while also ameliorating atrophic conditions. The US FDA granted orphan drug and fast track designation to VM202. The drug is currently being evaluated under Phase II clinical trial for the treatment of ALS.
- QRL-201: QurAlis Corporation
QRL-201 is a first-in-class therapeutic product candidate aiming to restore STMN2 expression in ALS patients. STMN2 is a well-validated protein important for neural repair and axonal stability, the expression of which is significantly decreased in nearly all ALS patients. QRL-201 rescues STMN2 loss of function in QurAlis ALS patient-derived motor neuron disease models in the presence of TDP-43 pathology. The drug is currently being evaluated under Phase I clinical trial for the Amyotrophic Lateral Sclerosis treatment.
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Amyotrophic Lateral Sclerosis pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration
- Intravenous
- Subcutaneous
- Oral
- Intramuscular
Amyotrophic Lateral Sclerosis Products have been categorized under various Molecule types such as
- Monoclonal antibody
- Small molecule
- Peptide
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Scope of the Amyotrophic Lateral Sclerosis Pipeline Report
- Coverage- Global
- Amyotrophic Lateral Sclerosis Companies- Ionis Pharmaceuticals, 1ST Biotherapeutics, Scholar Rock, Revalesio, QurAlis Corporation, Sanofi, MediciNova, Helixmith, Verge Genomics, UCB, and others.
- Amyotrophic Lateral Sclerosis Therapies- Dazucorilant 300 mg, AP-101, Fasudil (WP-0512), ION363, and others.
- Amyotrophic Lateral Sclerosis Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
- Amyotrophic Lateral Sclerosis Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III
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Table of Content
- Introduction
- Executive Summary
- Amyotrophic Lateral Sclerosis: Overview
- Pipeline Therapeutics
- Therapeutic Assessment
- Amyotrophic Lateral Sclerosis– DelveInsight’s Analytical Perspective
- Late Stage Products (Phase III)
- Drug name: Company name
- Drug profiles in the detailed report…..
- Mid Stage Products (Phase II/III)
- MN-166: MediciNova
- Drug profiles in the detailed report…..
- Early Stage Products (Phase I)
- QRL-201: QurAlis Corporation
- Drug profiles in the detailed report…..
- Preclinical and Discovery Stage Products
- Drug name: Company name
- Drug profiles in the detailed report…..
- Inactive Products
- Amyotrophic Lateral Sclerosis Key Companies
- Amyotrophic Lateral Sclerosis Key Products
- Amyotrophic Lateral Sclerosis- Unmet Needs
- Amyotrophic Lateral Sclerosis- Market Drivers and Barriers
- Amyotrophic Lateral Sclerosis- Future Perspectives and Conclusion
- Amyotrophic Lateral Sclerosis Analyst Views
- Amyotrophic Lateral Sclerosis Key Companies
- Appendix
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