Amyotrophic Lateral Sclerosis Pipeline Drugs and Companies Insight Report: Analysis of Clinical Trials, Therapies, Mechanism of Action, Route of Administration, and Developments

DelveInsight's, "Amyotrophic Lateral Sclerosis (ALS) - Pipeline Insight, 2024," report provides comprehensive insights about 75+ companies and 80+ pipeline drugs in Amyotrophic Lateral Sclerosis (ALS) pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

DelveInsight's "Amyotrophic Lateral Sclerosis Pipeline Insight" report provides comprehensive insights about key companies and pipeline drugs in the Amyotrophic Lateral Sclerosis pipeline landscapes.
The report comprises Amyotrophic Lateral Sclerosis pipeline drug profiles, including clinical and non-clinical stage products. It also includes the Amyotrophic Lateral Sclerosis therapeutics assessment by product type, stage, route of administration, and molecule type and further highlights the inactive Amyotrophic Lateral Sclerosis pipeline products.
Download sample report @ https://www.delveinsight.com/report-store/amyotrophic-lateral-sclerosis-als-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr

Amyotrophic Lateral Sclerosis Overview
Amyotrophic Lateral Sclerosis (ALS), commonly known as Lou Gehrig's disease, is a neurodegenerative disorder that affects motor neurons. The exact cause of ALS remains uncertain, with multiple pathways-both genetic and sporadic-leading to the development of the disease. ALS involves the progressive degeneration of both upper and lower motor neurons, often starting with symptoms of lower motor neuron damage in the proximal limbs. As the disease progresses, it leads to paralysis and, ultimately, death.

While no single cause has been identified for sporadic ALS, various mechanisms have been proposed, including abnormal RNA processing leading to prion-like aggregation, mutations in the superoxide dismutase type 1 (SOD1) gene causing free radical toxicity, inflammatory responses, and excessive glutamate concentrations. Familial ALS, though rarer, is often associated with specific genetic mutations, most notably repeat expansions in the C9ORF72 gene and various SOD1 gene mutations. Mutant SOD1 proteins misfold and aggregate, causing cellular damage and apoptosis. These genetic mutations are typically inherited in an autosomal dominant manner. Overall, ALS results from a complex interplay of diverse etiological factors rather than a single underlying cause.

Effective respiratory management is critical for ALS patients, who commonly experience chronic respiratory failure due to weakening of the diaphragm and intercostal muscles. Noninvasive ventilation is advised when symptoms such as orthopnea, nocturnal hypoxia, or a forced vital capacity (FVC) below 50% are present. In cases where noninvasive ventilation is insufficient or not tolerated, invasive ventilation may be considered. Additionally, managing swallowing difficulties (dysphagia) often begins with dietary adjustments, and enteral nutrition via percutaneous gastrostomy (G-tube) should be considered if oral intake becomes inadequate, as weight loss can worsen prognosis.

ALS is associated with numerous complications, including respiratory decline necessitating ventilatory support, dysphagia, dysarthria, malnutrition, muscle spasms, spasticity, fatigue, sialorrhea, thick mucus secretions, and pseudobulbar affect. Some complications may also result from medications used to manage ALS symptoms. For example, mexiletine can cause gastrointestinal upset and arrhythmias, riluzole may lead to transaminitis and asthenia, and edaravone can result in gait disturbances and headaches.
To know more about amyotrophic lateral sclerosis, causes, signs and symptoms, visit: https://www.delveinsight.com/report-store/amyotrophic-lateral-sclerosis-als-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr

Some of the key takeaways from the Amyotrophic Lateral Sclerosis Pipeline Report:
• Companies across the globe are diligently working toward the development of novel treatment therapies with a considerable amount of success over the years. Key players, such as Orion Pharma, Brainstorm-Cell Therapeutics, Orphazyme, MediciNova, Biogen, Biohaven Pharmaceutical, Prilenia Therapeutics, Apellis Pharmaceuticals, etc., are developing therapies for the treatment of Amyotrophic Lateral Sclerosis.
• Emerging therapies such as Levosimendan, NurOwn, Arimoclomol, Ibudilast, Tofersen, Verdiperstat, Pridopidine, AMX0035, Pegcetacoplan, are expected to have a significant impact on the Amyotrophic Lateral Sclerosis market in the coming years.
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Emerging Drugs for Amyotrophic Lateral Sclerosis

MN-166: MediciNova

MN-166 is a pioneering, orally administered small molecule designed to attenuate neuroinflammation. It works by suppressing pro-inflammatory cytokines such as IL-1ß, TNF-a, and IL-6, while potentially increasing the levels of the anti-inflammatory cytokine IL-10. Additionally, MN-166 acts as a functional antagonist of toll-like receptor 4 (TLR4), which may further contribute to its anti-inflammatory effects. This drug is currently undergoing evaluation in Phase II/III clinical trials for ALS.

RNS60: Revalesio

RNS60 is being developed as a disease-modifying and potentially restorative treatment for neurological disorders. It activates intracellular signaling pathways to enhance mitochondrial biogenesis and function while reducing inflammation. RNS60 offers neuroprotection for neurons and oligodendrocytes and modulates immune cell activity to restore homeostasis. The US FDA has granted RNS60 Orphan Drug and Fast Track designations for ALS. The drug is currently in Phase II clinical trials for ALS.

VM202: Helixmith

VM202 is an advanced gene therapy aimed at tissue regeneration for ALS treatment. It is injected along peripheral nerves and their branches to stimulate the internal production of hepatocyte growth factor (HGF), which may protect nerves, promote neuronal growth, and improve atrophic conditions. VM202 has received Orphan Drug and Fast Track designations from the US FDA and is under investigation in Phase II clinical trials for ALS.

QRL-201: QurAlis Corporation

QRL-201 is a novel therapeutic candidate designed to restore the expression of STMN2, a crucial protein for neural repair and axonal stability, which is notably reduced in ALS patients. By rescuing the loss of STMN2 function in motor neuron disease models with TDP-43 pathology, QRL-201 aims to counteract this protein deficiency. The drug is currently being tested in Phase I clinical trials for ALS.
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Amyotrophic Lateral Sclerosis Pipeline Therapies along with Key Players:
• Levosimendan: Orion Pharma
• NurOwn: Brainstorm-Cell Therapeutics
• Arimoclomol: Orphazyme
• Ibudilast: MediciNova
• Tofersen: Biogen
• Verdiperstat: Biohaven Pharmaceutical
• Pridopidine: Prilenia Therapeutics
• AMX0035: Amylyx
• Pegcetacoplan: Apellis Pharmaceuticals
Scope of Amyotrophic Lateral Sclerosis Pipeline Drug Insight
• Coverage: Global
• Major Players: Orion Pharma, Brainstorm-Cell Therapeutics, Orphazyme, MediciNova, Biogen, Biohaven Pharmaceutical, Prilenia Therapeutics, Apellis Pharmaceuticals, and others.
• Pipeline Therapies: Levosimendan, NurOwn, Arimoclomol, Ibudilast, Tofersen, Verdiperstat, Pridopidine, AMX0035, Pegcetacoplan, and others.
Table of Contents
1 Amyotrophic Lateral Sclerosis Report Introduction
2 Amyotrophic Lateral Sclerosis Executive Summary
3 Amyotrophic Lateral Sclerosis Overview
4 Amyotrophic Lateral Sclerosis- Analytical Perspective In-depth Commercial Assessment
5 Amyotrophic Lateral Sclerosis Pipeline Therapeutics
6 Amyotrophic Lateral Sclerosis Late Stage Products (Phase II/III)
7 Amyotrophic Lateral Sclerosis Mid Stage Products (Phase II)
8 Amyotrophic Lateral Sclerosis Early Stage Products (Phase I)
9 Amyotrophic Lateral Sclerosis Preclinical Stage Products
10 Amyotrophic Lateral Sclerosis Therapeutics Assessment
11 Amyotrophic Lateral Sclerosis Inactive Products
12 Company-University Collaborations (Licensing/Partnering) Analysis
13 Amyotrophic Lateral Sclerosis Key Companies
14 Amyotrophic Lateral Sclerosis Key Products
15 Amyotrophic Lateral Sclerosis Unmet Needs
16 Amyotrophic Lateral Sclerosis Market Drivers and Barriers
17 Amyotrophic Lateral Sclerosis Future Perspectives and Conclusion
18 Amyotrophic Lateral Sclerosis Analyst Views
19 Appendix
20 About DelveInsight
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