Motor Neuron Disease Clinical Trials

DelveInsight reports that the Motor Neuron Disease pipeline involves over 180 key companies actively developing more than 200 investigational therapies.

Motor Neuron Disease Overview:

Motor neuron diseases (MNDs) are a group of progressive neurological disorders that damage motor neurons, the cells responsible for controlling vital muscle functions such as movement, breathing, speech, and swallowing. This group includes conditions like amyotrophic lateral sclerosis (ALS), primary lateral sclerosis, spinal muscular atrophy, and others. Although rare, MND is a serious condition that gradually weakens muscles, profoundly affecting daily activities. While no cure currently exists, treatments can help manage symptoms, and some patients live with the disease for many years. The causes of MND are multifactorial, involving genetic, environmental, and lifestyle factors, with approximately 10% of cases inherited due to genetic mutations.

Symptoms often develop slowly and may be subtle at first. Early indicators include weakness in the legs or ankles, difficulty speaking or swallowing, reduced grip strength, muscle cramps, weight loss, and involuntary emotional reactions such as sudden laughter or crying. The disease typically progresses through three stages—early, middle, and advanced—affecting individuals differently in terms of speed and severity. Ultimately, MND leads to a decline in physical function and reduces life expectancy.

Request for a detailed insights report on Motor Neuron Disease pipeline insights

“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 (MND) pipeline report highlights a vibrant landscape, with over 180 companies actively developing more than 200 investigational therapies for MND.

• Key players in this field include 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, all working to advance treatments and improve the therapeutic landscape for MND.

• Promising pipeline candidates in various stages of development include Talditercept alfa, Pridopidine, VM202, TPN-101, GC 101, VRG 50635, QRL 201, and more.

• 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.

Download our free sample page report on Motor Neuron Disease pipeline insights

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

Over 180 leading companies are actively pursuing therapies for Motor Neuron Disease, with Biohaven Pharmaceuticals, Inc. having a candidate in the most advanced stage, currently undergoing 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

Download Sample Pages to Get an in-depth Assessment of the Emerging Motor Neuron Disease Therapies and Key Companies: Motor Neuron Disease Clinical Trials and advancements

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

Download Motor Neuron Disease Sample report to know in detail about the Motor Neuron Disease treatment market @ Motor Neuron Disease Therapeutic Assessment

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

Request the Sample PDF to Get Detailed Insights About the Motor Neuron Disease Pipeline Reports Offerings

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.

Media Contact
Company Name: DelveInsight Business Research LLP
Contact Person: Kritika Rehani
Email: Send Email
Phone: +14699457679
Address:304 S. Jones Blvd #2432
City: Las Vegas
State: Nevada
Country: United States
Website: https://www.delveinsight.com/