Amyotrophic Lateral Sclerosis - Pipeline Insight, 2024

This “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 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.

Geography Covered

• Global coverage

Amyotrophic Lateral Sclerosis: Understanding

Amyotrophic Lateral Sclerosis: Overview

Amyotrophic Lateral Sclerosis, also known as “Lou Gehrig disease,” is a neurodegenerative disease of the motor neurons. No single etiology has been proven; rather, multiple pathways (both heritable and sporadic) have been shown to result in unmistakably similar disease entities. ALS necessarily affects both upper and lower motor neurons with variable patterns of onset, most commonly beginning with signs of lower motor neuron degeneration within proximal limbs. As it is a progressive disease, it will eventually lead to paralysis and, inevitably, death.

Although many potential mechanisms have been proposed, a precise, single etiology of sporadic amyotrophic lateral sclerosis is yet unproven. These mechanisms include altered RNA processing leading to prion-like self-aggregation, superoxide dismutase type 1 SOD1 mutations leading to free radical toxicity, cascading inflammatory responses, and excessive concentrations of glutamate, among others. The rarer entity of familial ALS has numerous genetic mechanisms, most frequently repeat expansion of the C9ORF72 gene and various mutations of the SOD1 gene. Mutated SOD1 protein misfold and forms aggregates, leading to cellular injury and eventually apoptosis. Both genetic aberrations are inherited in a mainly autosomal dominant pattern. Ultimately, rather than a single unifying cause, ALS is an etiologically diverse clinical entity, which is the result of a multitude of separate potential preceding aberrations.

Respiratory management is crucial, as ALS patients often develop chronic respiratory failure due to weakening of the diaphragmatic and intercostal muscles. Noninvasive ventilation is recommended when the patient experiences orthopnea, nocturnal hypoxia, or a forced vital capacity (FVC) below 50%. Invasive ventilation may be considered if noninvasive ventilation is not tolerated or if the patient remains hypoxic or hypercarbic. Swallowing difficulties (dysphagia) are also common in ALS patients and should be managed initially with diet modifications. Enteral nutrition via percutaneous gastrostomy (G-tube) should be considered if the patient's oral intake is impaired, as weight loss is associated with a worse prognosis.

There are numerous complications associated with amyotrophic lateral sclerosis, including respiratory decline with eventual need for ventilatory support, dysphagia, dysarthria, malnutrition, spasms, spasticity, and fatigue, functional decline due to muscular weakness, sialorrhea, thick mucus secretions and pseudobulbar affect. In addition, some complications may arise due to the less-common medications meant to manage symptoms of the disease itself. These side effects include gastrointestinal upset and arrhythmias in mexiletine, transaminitis and asthenia in Riluzole, and gait disturbance and headache in edaravone.

"Amyotrophic Lateral Sclerosis- Pipeline Insight, 2024" report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Amyotrophic Lateral Sclerosis pipeline landscape is provided which includes the disease overview and Amyotrophic Lateral Sclerosis treatment guidelines. The assessment part of the report embraces, in depth Amyotrophic Lateral Sclerosis commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Amyotrophic Lateral Sclerosis collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

The companies and academics are working to assess challenges and seek opportunities that could influence Amyotrophic Lateral Sclerosis R&D. The therapies under development are focused on novel approaches to treat/improve Amyotrophic Lateral Sclerosis.

Amyotrophic Lateral Sclerosis Emerging Drugs Chapters

This segment of the Amyotrophic Lateral Sclerosis report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, II/III, II, I, preclinical and discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Amyotrophic Lateral Sclerosis Emerging Drugs

• 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 treatment of ALS.

Amyotrophic Lateral Sclerosis: Therapeutic Assessment

This segment of the report provides insights about the different Amyotrophic Lateral Sclerosis drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Amyotrophic Lateral Sclerosis

There are approx. 75+ key companies which are developing the therapies for Amyotrophic Lateral Sclerosis. The companies which have their Amyotrophic Lateral Sclerosis drug candidates in the most advanced stage, i.e. Phase II/III include, MediciNova.

Phases

This report covers around 80+ products under different phases of clinical development like

• Late stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

Route of Administration

Amyotrophic Lateral Sclerosis pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Intravenous
• Subcutaneous
• Oral
• Intramuscular
• Molecule Type

Products have been categorized under various Molecule types such as

• Monoclonal antibody
• Small molecule
• Peptide
• Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Amyotrophic Lateral Sclerosis: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Amyotrophic Lateral Sclerosis therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Amyotrophic Lateral Sclerosis drugs.

Amyotrophic Lateral Sclerosis Report Insights

• Amyotrophic Lateral Sclerosis Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Amyotrophic Lateral Sclerosis Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Amyotrophic Lateral Sclerosis drugs?
• How many Amyotrophic Lateral Sclerosis drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Amyotrophic Lateral Sclerosis?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Amyotrophic Lateral Sclerosis therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Amyotrophic Lateral Sclerosis and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Ionis Pharmaceuticals
• 1ST Biotherapeutics
• Scholar Rock
• Revalesio
• QurAlis Corporation
• Sanofi
• MediciNova
• Helixmith
• Verge Genomics
• UCB

Key Products

• ION363
• FB418
• SRK-015
• RNS60
• QRL-201
• SAR443820
• MN-166
• VM202
• VRG-50635
• Zilucoplan

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