DelveInsight’s, “Amyotrophic Lateral Sclerosis Pipeline Insight, 2023,” report provides comprehensive insights about 100+ companies and 100+ pipeline drugs in the Amyotrophic Lateral Sclerosis pipeline landscape. It covers the Amyotrophic Lateral Sclerosis pipeline drug profiles, including Amyotrophic Lateral Sclerosis clinical trials 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.
In the Amyotrophic Lateral Sclerosis pipeline report, detailed description of the drug is given which includes mechanism of action of the drug, Amyotrophic Lateral Sclerosis clinical trials studies, Amyotrophic Lateral Sclerosis NDA approvals (if any), and product development activities comprising the technology, Amyotrophic Lateral Sclerosis (ALS) collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
Key takeaways from the Amyotrophic Lateral Sclerosis Pipeline Insight Report
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Amyotrophic Lateral Sclerosis Overview
Amyotrophic Lateral Sclerosis (ALS) (ALS) is a rare neurological disease that primarily affects the nerve cells (neurons) responsible for controlling voluntary muscle movement (those muscles we choose to move). Voluntary muscles produce movements like chewing, walking, and talking. The disease is progressive, meaning the symptoms get worse over time. Currently, there is no cure for ALS and no effective treatment to halt or reverse the progression of the disease. ALS belongs to a wider group of disorders known as motor neuron diseases, which are caused by gradual deterioration (degeneration) and death of motor neurons. Motor neurons are nerve cells that extend from the brain to the spinal cord and to muscles throughout the body.
Recent Developmental Activities in the Amyotrophic Lateral Sclerosis Treatment Landscape
For further information, refer to the detailed Amyotrophic Lateral Sclerosis Unmet Needs, Amyotrophic Lateral Sclerosis Market Drivers, and Amyotrophic Lateral Sclerosis Market Barriers, click here for Amyotrophic Lateral Sclerosis Ongoing Clinical Trial Analysis
Amyotrophic Lateral Sclerosis Emerging Drugs Profile
AMX0035: Amylyx Pharmaceuticals
AMX0035 is an investigational product comprised of two complementary active agents, sodium phenylbutyrate (PB) and taurursodiol (TURSO), which were combined in a co-formulation to reduce neuronal death and dysfunction. AMX0035 was specifically co-formulated and manufactured by Amylyx to ensure proper absorption, exposure, and quality. AMX0035 targets endoplasmic reticulum and mitochondrial dependent neuronal degeneration pathways in ALS and other neurodegenerative diseases. The company has submitted a New Drug Submission (NDS) to Health Canada for AMX0035 (sodium phenylbutyrate (PB)-taurursodiol (TURSO)) for the treatment of ALS.
BIIB067: Biogen
BIIB067 (tofersen) is thought to reduce the production of SOD1 protein and is being evaluated in Phase III clinical trial to see if it can potentially slow the fatal progression of SOD1-ALS. Tofersen (BIIB067) previously called IONIS-SOD1Rx is an investigational therapy to slow the progression of familial Amyotrophic Lateral Sclerosis (ALS) (ALS). The therapy was developed in a collaboration between Ionis Pharmaceuticals and Biogen but is now being developed solely by Biogen.
Masitinib: AB Sciences
Masitinib is currently the only tyrosine kinase inhibitor in late-stage development for ALS. Masitinib distinguishes itself from other ALS developmental drugs by exerting neuroprotection in both central and peripheral nervous systems. Masitinib appears exceptional among other ALS-developmental drugs, exerting neuroprotection in both central nervous system and peripheral nervous system via selective kinase inhibition that modulates the functionality of different cells implicated in ALS pathogenesis. In recognition of the critical need for new treatments, masitinib received orphan drug designation for ALS from both the European Medicine Agency (EMA) and the U.S. Food and Drug Administration (FDA).
Ravulizumab: Alexion Pharmaceuticals
Ravulizumab is the first and only approved long-acting C5 complement inhibitor. It is administered intravenously every eight weeks or every four weeks for pediatric patients less than 20 kg, following a loading dose. ULTOMIRIS works by inhibiting the C5 protein in the terminal complement cascade, a part of the body’s immune system. The terminal complement cascade, when activated in an uncontrolled manner, plays a role in severe ultra-rare disorders. ULTOMIRIS is approved in the U.S., Japan, and the EU as a treatment for adults with PNH and in the U.S. for aHUS to inhibit complement-mediated thrombotic microangiopathy (TMA) in adult and pediatric (one month of age and older) patients. The drug is being evaluated in Phase III clinical trial to treat patients with ALS.
Autologous MSC-NTF cells: BrainStorm Cell Therapeutics
BrainStorm has entered into agreements with Dana-Farber Cancer Institute (Dana-Farber) in Boston, Massachusetts and the City of Hope National Medical Center in Duarte, California to provide clean room facilities for production of autologous MSC-NTF cells. The company has completed a phase III clinical trial of autologous MSC-NTF cellular therapy in Amyotrophic Lateral Sclerosis.
Amyotrophic Lateral Sclerosis Pipeline Therapeutics Assessment
There are approx. 100+ key companies which are developing the therapies for Amyotrophic Lateral Sclerosis (ALS). The companies which have their Amyotrophic Lateral Sclerosis (ALS) drug candidates in the most advanced stage, i.e. Preregistration include, Amylyx Pharmaceuticals.
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Scope of the Amyotrophic Lateral Sclerosis Pipeline Report
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Table of Content
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