Los Angeles, June 15, 2021 (GLOBE NEWSWIRE) -- Amyotrophic Lateral Sclerosis Pipeline: Current Treatments, Drug Pipeline and Clinical Trial Outlook | Insights by DelveInsight
The pipeline of Amyotrophic Lateral Sclerosis is very vast and in the coming years, it is expected that the market will grow with the approval of late-stage products in the pipeline.
DelveInsight’s ‘Amyotrophic Lateral Sclerosis (ALS) Pipeline Insights’ report offers exhaustive coverage of the emerging therapies and landscape in different stages of development from pre-clinical till late-stage, along with dormant, inactive, and abandoned therapeutic agents.
Some of the key highlights from the Amyotrophic Lateral Sclerosis Pipeline report:
- Amyotrophic Lateral Sclerosis Pipeline report offers a rich analysis of 100+ key players and 100+ key therapies.
- Amyotrophic Lateral Sclerosis pipeline comprises therapies in different stages of the clinical phase such as BIIB067, ANX005, AMX0035, MN-166, BLZ945, AP-101, AS-202, Arimoclomol, ALZT-OP1a, AT1501, HK001, Q-Cells, and several others expected to get launched in the next decade.
- Some of the key companies engaged in expanding the Amyotrophic Lateral Sclerosis Pipeline are Biogen, Annexon Biosciences, Amylyx Pharmaceuticals, MediciNova, Novartis, AL-S Pharma, Acurastem, Orphazyme, AZTherapies, ALS TDI, Everfront Biotech, Q Therapeutics, among others.
- DelveInsight estimates that the AMX0035 is expected to emerge as the trailblazer as the 24-week CENTAUR study comprising 137 participants with ALS met its primary endpoint of slowing ALS progression, as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) and Amylyx Pharmaceuticals has announced its intention to submit a marketing authorization application for its investigational amyotrophic lateral sclerosis (ALS) therapy AMX0035 to the European Medicines Agency (EMA) by the end of 2021.
- In May 2021, Seelos Therapeutics announced it has received European Orphan Drug Designation for SLS-005 in amyotrophic lateral sclerosis (ALS) from the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP).
- In May 2021, Aeterna Zentaris announced the commencement of the previously announced preclinical program to qualify macimorelin for clinical development as a potential treatment option for amyotrophic lateral sclerosis (ALS; Lou Gehrig's disease).
- In May 2021, Orphazyme announced that the ORARIALS-01 pivotal trial of arimoclomol in amyotrophic lateral sclerosis (ALS) did not meet its primary and secondary endpoints to show benefit in people living with ALS. No important safety signals were reported in the trial.
- In March 2021, Retrotope announced that the first patient has been dosed in a multicenter Phase 2 clinical trial evaluating RT001, the company’s lead development candidate, in patients with amyotrophic lateral sclerosis (ALS or Lou Gehrig’s disease).
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The Amyotrophic Lateral Sclerosis Pipeline reports lay down a complete picture of the ongoing clinical trials, partnerships taking place in the domain, recent happenings in space and growth prospects across the Amyotrophic Lateral Sclerosis domain.
Amyotrophic Lateral Sclerosis: Overview
Amyotrophic Lateral Sclerosis is one of the groups of disorders known as motor neuron diseases, characterized by the progressive degeneration of nerve cells (motor neurons) in the brain and spinal cord eventually leading to their death. The condition hampers the communication between the nervous system and voluntary muscles of the body thus, affecting both the upper and lower motor neurons.
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Amyotrophic Lateral Sclerosis Pipeline Drugs
| Drug | Company | Phase | MoA | RoA |
| BIIB067 | Biogen | Phase III | Superoxide dismutase inhibitors | Intrathecal |
| ANX005 | Annexon Biosciences | Phase II | Complement C1 inhibitors | Intravenous |
| AMX0035 | Amylyx Pharmaceuticals | Phase II/III | Ammonia scavengers; Histone deacetylase inhibitors; Phosphotransferase inhibitors | Oral |
| MN-166 | MediciNova | Phase II/III | Astrocyte inhibitors; Cytokine modulators; Glial cell modulators; Leukotriene receptor antagonists | Oral |
| BLZ945 | Novartis | Phase II | Macrophage colony-stimulating factor receptor antagonists | Oral |
| AP-101 | AL-S Pharma | Phase I | Superoxide dismutase inhibitors | Intravenous |
| AS-202 | Acurastem | Preclinical | PIKFYVE protein inhibitors | NA |
| Arimoclomol | Orphazyme | Phase III | Heat shock transcription factor stimulants | Oral |
| ALZT-OP1a | AZTherapies | Phase II | Glial cell modulators; Mast cell stabilizers | Inhalation |
| AT1501 | ALS TDI | Phase II | CD40 ligand inhibitors | Intravenous |
| HK001 | Everfront Biotech | Phase I | Everfront Biotech | Oral |
| Q-Cells | Q Therapeutics | Preclinical | Cell replacements | NA |
| Verdiperstat | Biohaven Pharmaceuticals | Phase II/III | Peroxidase inhibitors | Oral |
| NPT 520-34 | Neuropore Therapies | Phase I | 1 Phosphatidylinositol 3 kinase modulators; Autophagy stimulants; Organic anion transport protein 3 inhibitors | Oral |
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The Amyotrophic Lateral Sclerosis Pipeline report proffers detailed insights into active pipeline assets segmented by Stage, Product Type, Route of Administration, Molecule Type, Target, and Mechanism of Action.
By Product Type
- Mono
- Combination
By Stage
- Discovery
- Pre-clinical
- IND
- Phase I
- Phase II
- Phase III
- Pre-registration
By Molecule Type
- Gene therapies
- Small molecule
- Vaccines
- Polymers
- Peptides
- Monoclonal antibodies
By Route of Administration
- Infusion
- Intradermal
- Intramuscular
- Intranasal
- Oral
- Parenteral
- Subcutaneous
- Inhalation
- Intrathecal
By Mechanism of Action
- Glial cell modulators
- Histone deacetylase inhibitors
- Heat shock transcription factor stimulants
- C1q Inhibitors
- Superoxide dismutase inhibitors
- Immunomodulators
By Targets
- Superoxide dismutase
- Complement C1
- Histone deacetylase
- Macrophage colony-stimulating factor receptor
- CD40 ligand
- PIKFYVE protein
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Scope of the ALS Pipeline Report
Coverage: Global
Key Players: Biogen, Annexon Biosciences, Amylyx Pharmaceuticals, MediciNova, Novartis, AL-S Pharma, Acurastem, Orphazyme, AZTherapies, ALS TDI, Everfront Biotech, Q Therapeutics, Biohaven Pharmaceuticals, Neuropore Therapies, Retrotope, Seelos Therapeutics, among others.
Key Amyotrophic Lateral Sclerosis Pipeline Therapies: BIIB067, ANX005, AMX0035, MN-166, BLZ945, AP-101, AS-202, Arimoclomol, ALZT-OP1a, AT1501, HK001, Q-Cells, Verdiperstat, NPT 520-34, RT001, SLS-005, among others.
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Table of Contents
| 1 | Report Introduction |
| 2 | Executive Summary |
| 3 | Amyotrophic Lateral Sclerosis Disease Overview |
| 4 | ALS Pipeline Therapeutics Comparative Analysis |
| 5 | Amyotrophic Lateral Sclerosis Therapeutic Assessment |
| 6 | Amyotrophic lateral sclerosis – DelveInsight’s Analytical Perspective |
| 7 | In-depth Commercial ALS Assessment |
| 8 | Amyotrophic Lateral Sclerosis Collaboration Deals |
| 9 | Late Stage ALS Products (Phase III) |
| 10 | Mid Stage ALS Products (Phase II) |
| 11 | Early Stage ALS Products (Phase I) |
| 12 | Pre-clinical and Discovery Stage ALS Products |
| 13 | Inactive Amyotrophic Lateral Sclerosis Pipeline Products |
| 14 | Key Amyotrophic Lateral Sclerosis Products |
| 15 | Unmet Needs |
| 16 | Amyotrophic Lateral Sclerosis Market Drivers and Barriers |
| 17 | Future Perspectives and Conclusion |
| 18 | Analyst Views |
| 19 | Key Amyotrophic Lateral Sclerosis Companies |
| 20 | Appendix |
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