Motor Neuron Disease Clinical and Non-Clinical Studies, Key Companies, Therapeutic Assessment, Emerging Therapies, Treatment Algorithm, and Pipeline Analysis | Biohaven Pharmaceuticals, Inc., Prilenia Therapeutics, Helixmith Co., Ltd., Transposon Therapeu

Motor Neuron Disease Companies are Biohaven Pharmaceuticals, Inc., Prilenia Therapeutics, Helixmith Co., Ltd., Transposon Therapeutics, Inc., GeneCradle Therapeutics, Verge Genomics, QurAlis Corporation, Zydus Lifesciences Limited, Ra Pharmaceuticals, Guangzhou Magpie Pharmaceuticals Co., Ltd., Scholar Rock, Inc., Spinogenix, Seelos Therapeutics, Inc., Sanofi, Hoffmann-La Roche, Revalesio Corporation, Cytokinetics, Rapa Therapeutics LLC, Q Therapeutics, Inc., PTC Therapeutics, ProJenX, NeuroSense Therapeutics Ltd., Knopp Biosciences, MediciNova, Amylyx Pharmaceuticals Inc., Neuropore Therapies Inc, NMD Pharma A/S, Supernus Pharmaceuticals, Inc., Mitsubishi Tanabe Pharma America Inc., MaaT Pharma

Motor Neuron Disease Pipeline constitutes 180+ key companies continuously working towards developing 200+ Motor Neuron Disease treatment therapies, analyzes DelveInsight.

Motor Neuron Disease Overview:

Motor neuron diseases (MNDs) are a group of progressive neurological disorders that damage motor neurons, which control essential muscle functions like movement, breathing, speech, and swallowing. This category includes conditions such as amyotrophic lateral sclerosis (ALS), primary lateral sclerosis, spinal muscular atrophy, and others. MND is a rare but serious disease that weakens muscles over time, significantly impacting daily life. While there is no cure, treatments can help manage symptoms, and some individuals live with the condition for years. The disease is often caused by a combination of genetic, environmental, and lifestyle factors, with about 10% of cases being inherited due to genetic mutations.

Symptoms develop gradually and may initially go unnoticed. Early signs include muscle weakness in the legs or ankles, difficulty speaking or swallowing, weak grip, muscle cramps, weight loss, and uncontrollable emotional responses like sudden laughter or crying. MND progresses through three stages-early, middle, and advanced-affecting individuals at different rates and severity levels. Ultimately, the disease shortens life expectancy and leads to a decline in physical function.

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"Motor Neuron Disease Pipeline Insight 2025" report by DelveInsight provides a comprehensive analysis of the ongoing clinical development activities and growth prospects across the Motor Neuron Disease Therapeutics Market.

Key Takeaways from the Motor Neuron Disease Pipeline Report

DelveInsight's Motor Neuron Disease pipeline report depicts a robust space with 180+ active players working to develop 200+ pipeline therapies for Motor Neuron Disease treatment.
Key Motor Neuron Disease companies such as Biohaven Pharmaceuticals, Inc., Prilenia Therapeutics, Helixmith Co., Ltd., Transposon Therapeutics, Inc., GeneCradle Therapeutics, Verge Genomics, QurAlis Corporation, Zydus Lifesciences Limited, Ra Pharmaceuticals, Guangzhou Magpie Pharmaceuticals Co., Ltd., Scholar Rock, Inc., Spinogenix, Seelos Therapeutics, Inc., Sanofi, Hoffmann-La Roche, Revalesio Corporation, Cytokinetics, Rapa Therapeutics LLC, Q Therapeutics, Inc., PTC Therapeutics, ProJenX, NeuroSense Therapeutics Ltd., Knopp Biosciences, MediciNova, Amylyx Pharmaceuticals Inc., Neuropore Therapies Inc, NMD Pharma A/S, Supernus Pharmaceuticals, Inc., Mitsubishi Tanabe Pharma America Inc., MaaT Pharma, and others are evaluating new drugs for Motor Neuron Disease to improve the treatment landscape.
Promising Motor Neuron Disease pipeline therapies in various stages of development include Talditercept alfa, Pridopidine, VM202, TPN-101, GC 101, VRG 50635, QRL 201, and others.

Recent breakthroughs in the Motor Neuron Disease Pipeline Segment:

In December 2024, Celosia Therapeutics, a biotech spin-out from Macquarie University, secured $16.8 million in funding to advance its ALS treatment, CTX-1000, into clinical trials. CTX-1000 is a disease-modifying therapy targeting the harmful protein TDP-43, which affects motor neurons in ALS patients. The therapy aims to halt disease progression with a single administration, showing promise even in severe animal models. Human trials are expected to commence by late 2025 at Macquarie University Hospital.
In October 2024, Scholar Rock announced positive results from a Phase 3 trial of Apitegromab, a treatment for spinal muscular atrophy (SMA). The therapy demonstrated significant improvements in motor function, with 30.4% of patients showing over a 3-point improvement on the Hammersmith Functional Motor Scale Expanded (HFMSE), compared to 12.5% on placebo. Scholar Rock plans to submit applications for U.S. Biologics License and European Union marketing authorization in early 2025.
In May 2024, the FDA cleared Spinogenix's Investigational New Drug (IND) application for SPG302, a novel therapy aimed at treating Amyotrophic Lateral Sclerosis (ALS). SPG302 is designed as a once-daily oral treatment with the potential to regenerate synapses, thereby addressing declines in cognitive and motor functions associated with ALS.

Motor Neuron Disease Pipeline Analysis
The report provides insights into:
The report provides detailed insights into the key companies that are developing therapies in the Motor Neuron Disease Market.
The report also evaluates different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Motor Neuron Disease treatment.
It analyzes the key companies involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
It navigates the emerging drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement, and financing details for future advancement of the Motor Neuron Disease market.

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Motor Neuron Disease Emerging Drugs

Talditercept alfa: Biohaven Pharmaceuticals, Inc.
Pridopidine: Prilenia Therapeutics
VM202: Helixmith Co., Ltd.
TPN-101: Transposon Therapeutics, Inc.
GC 101: GeneCradle Therapeutics
VRG 50635: Verge Genomics
QRL 201: QurAlis Corporation

Motor Neuron Disease Companies

More than 180 key companies are actively developing therapies for Motor Neuron Disease. Among them, Biohaven Pharmaceuticals, Inc. has a drug candidate in the most advanced stage of development, specifically Phase III clinical trials.

DelveInsight's report covers around 200+ 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

Motor Neuron Disease 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

Motor Neuron Disease Products have been categorized under various Molecule types such as

Monoclonal antibody
Small molecule
Peptide

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Motor Neuron Disease Pipeline Therapeutic Assessment
• Motor Neuron Disease Assessment by Product Type
• Motor Neuron Disease By Stage
• Motor Neuron Disease Assessment by Route of Administration
• Motor Neuron Disease Assessment by Molecule Type

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Table of Content

1. Report Introduction
2. Executive Summary
3. Motor Neuron Disease Current Treatment Patterns
4. Motor Neuron Disease - DelveInsight's Analytical Perspective
5. Therapeutic Assessment
6. Motor Neuron Disease Late-Stage Products (Phase-III)
7. Motor Neuron Disease Mid-Stage Products (Phase-II)
8. Early Stage Products (Phase-I)
9. Pre-clinical Products and Discovery Stage Products
10. Inactive Products
11. Dormant Products
12. Motor Neuron Disease Discontinued Products
13. Motor Neuron Disease Product Profiles
14. Motor Neuron Disease Key Companies
15. Motor Neuron Disease Key Products
16. Dormant and Discontinued Products
17. Motor Neuron Disease Unmet Needs
18. Motor Neuron Disease Future Perspectives
19. Motor Neuron Disease Analyst Review
20. Appendix
21. Report Methodology

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Contact Info:
Kritika Rehani
krehani@delveinsight.com
info@delveinsight.com
+14699457679

About DelveInsight

DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance.

This release was published on openPR.