DelveInsight’s, “Amyotrophic Lateral Sclerosis Pipeline Insight, 2023,” report provides comprehensive insights about 90+ 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 Amyotrophic Lateral Sclerosis pipeline therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
For Amyotrophic Lateral Sclerosis Emerging drugs, the Amyotrophic Lateral Sclerosis pipeline analysis report provides a 360° view of the therapeutics landscape by development point, product type, route of administration, molecule type, and MOA. The Amyotrophic Lateral Sclerosis pipeline research covers business opportunities, challenges, future partnerships, strong competitors, and growth strategies.
In the Amyotrophic Lateral Sclerosis Pipeline Report, a detailed description of the drug is given which includes the 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 collaborations, licensing, mergers and acquisition, funding, designations, and other product-related details.
Key takeaways from the Amyotrophic Lateral Sclerosis Pipeline Report
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Amyotrophic Lateral Sclerosis Overview
Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s Disease, is a rare neurological disease that affects motor neurons—those nerve cells in the brain and spinal cord that control voluntary muscle movement. Voluntary muscles are those we choose to move to produce movements like chewing, walking, and talking. The disease is progressive, meaning the symptoms get worse over time. ALS has no cure and there is no effective treatment to reverse its progression.
Recent Developmental Activities in the Amyotrophic Lateral Sclerosis Treatment Landscape
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Amyotrophic Lateral Sclerosis Emerging Drugs Profile
Tofersen is an antisense drug being evaluated for the potential treatment of SOD1-ALS. Tofersen binds to SOD1 mRNA, allowing for its degradation by RNase-H in an effort to reduce synthesis of SOD1 protein production. Tofersen is also being studied in the Phase 3 ATLAS study, which is designed to evaluate the ability of tofersen to delay clinical onset when initiated in presymptomatic individuals with a SOD1 genetic mutation and biomarker evidence of disease activity. Biogen licensed tofersen from Ionis Pharmaceuticals, Inc. under a collaborative development and license agreement. Biogen Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted a New Drug Application (NDA) for tofersen, an investigational drug for superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS). The application has been granted priority review and given a Prescription Drug User Fee Act action date of January 25, 2023.
RNS60 is being developed to provide disease modifying and potentially restorative treatments for neurological diseases. It 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 U.S. Food and Drug Administration.
ANX005 is an antibody-based medication designed to bind and block the activity of C1q, which is expected to keep synapses healthy and slow or halt neurodegeneration. The antibody is being developed for a number neurodegenerative diseases, such as ALS, Guillain-Barré syndrome (GBS), Huntington’s disease (HD), and cold agglutinin disease (CAD). Clinical trials are ongoing in all these indications. The ongoing ALS trial is investigating ANX005 in patients whose first symptoms of weakness began in the past three years. Participants will receive two induction doses about 5–6 days apart, followed by every-two-week injections up to week 22. All then will be followed for another 14 weeks. Preliminary data showed that ANX005 treatment resulted in a reduction in neurofilament light chain (NfL) levels, a marker of nerve cell damage, during the initial 12 weeks of treatment. But levels rose to their initial value when patients stopped receiving treatment.
Amyotrophic Lateral Sclerosis Pipeline Therapeutics Assessment
There are approx. 90+ 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. Preregistration include, Biogen.
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Scope of the Amyotrophic Lateral Sclerosis Pipeline
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Table of Content
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