Motor neuron disease is a progressive neurodegenerative disorder causing muscle weakness, paralysis, and respiratory failure. According to Weifang Tong et al., 2025, the global MND incidence rose 74.54%, from approximately 36,769 cases in 1990 to 64,177 in 2021 over the past three decades. It encompasses emerging therapies such as gene therapies, antisense oligonucleotides, neuroprotectants, and cell‑based approaches, with growing emphasis on biomarkers, delivery systems, and patient stratification. According to the motor neuron disease pipeline analysis by Expert Market Research, these factors are driving increased clinical activity and investment, supporting market growth in the coming years.
Major companies involved in the motor neuron disease pipeline analysis include MediciNova, Ionis Pharmaceuticals, Inc., and others.
Leading drugs currently in the pipeline include MN-166, PLL001, QRL-101, and others.
Pipeline growth in motor neuron disease is driven by advancing gene therapies, expanding antisense oligonucleotide research, and increasing clinical trials targeting neuroprotection and disease-modifying treatments.
The Motor Neuron Disease Pipeline Analysis Report by Expert Market Research gives comprehensive insights into motor neuron disease therapeutics currently undergoing clinical trials. It covers various aspects related to the details of each of these drugs under development for motor neuron disease. The motor neuron disease report assessment includes the analysis of over 100 pipeline drugs and 50+ companies. The motor neuron disease pipeline landscape will include an analysis based on efficacy and safety measure outcomes published for the trials, including their adverse effects on patients suffering from the condition, and alignment with motor neuron disease treatment guidelines to ensure optimal care practices.
The assessment part will include a detailed analysis of each drug, drug class, clinical studies, phase type, drug type, route of administration, and ongoing product development activities related to motor neuron disease.
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Motor neuron disease (MND) is a progressive neurological disorder that damages motor neurons in the brain and spinal cord, leading to muscle weakness, wasting, and impaired movement. It occurs when these nerve cells gradually degenerate and die, disrupting communication between the nervous system and muscles.
Motor neuron disease treatments aim to slow progression, manage symptoms, and improve quality of life through medications, physiotherapy, occupational therapy, and supportive care, although no definitive cure currently exists. In June 2025, the MND-SMART trial introduced tacrolimus as its fourth investigational drug arm, testing its potential to slow disease progression by targeting abnormal protein clumping and neuron inflammation, highlighting advancements in the motor neuron disease drug pipeline.
According to the World Health Organization (WHO), neurological conditions affect over 1 in 3 people worldwide, making them a leading cause of illness and disability. Research by Weifang Tong et al. (2025) reported that the global incidence of motor neuron disease (MND) increased by 74.54%, rising from 36,769 cases in 1990 to 64,177 cases in 2021. In the United States, Delaram Imantalab et al. (2024) estimated an annual incidence of 2 cases per 100,000 population.
This section of the report covers the analysis of motor neuron disease drug candidates based on several segmentations, including:
By Phase
The pipeline assessment report covers 50+ drug analyses based on phase:
By Drug Class
The motor neuron disease pipeline analysis report covers 50+ drug analyses based on drug classes:
By Route of Administration
The pipeline assessment report covers 50+ drug analyses based on the route of administration.
The report covers phase I, phase II, phase III, phase IV, and early-phase drugs. The coverage includes an in-depth analysis of each drug across these phases. According to EMR analysis, phase II covers a major share of the total motor neuron disease clinical trials, with 39%. It is followed by phase I at 30%. Phase III contributes around 15%, and early phase I accounts for 7%. This balanced pipeline across multiple phases reflects strong research efforts, ensuring continued advancements that can significantly enhance treatment options in the motor neuron disease market.
The drug molecule categories covered under the motor neuron disease pipeline analysis include small molecules, monoclonal antibodies, gene therapies, peptides, and polymers. The motor neuron disease report provides a comparative analysis of the drug classes for each drug in various phases of clinical trials for motor neuron disease. Gene-targeted drug therapies are gaining attention in the motor neuron disease pipeline. For instance, Tofersen, developed by Biogen, is designed to treat patients with superoxide dismutase 1 (SOD1) mutation-associated motor neuron disease. Clinical trials have demonstrated its potential to slow disease progression, making it a significant advancement in gene-specific treatment approaches for this rare neurological disorder.
The EMR report for the motor neuron disease pipeline covers the profile of key companies involved in clinical trials and their drugs under development. It provides a detailed motor neuron disease therapeutic assessment, analyzing the competitive dynamics of the clinical trial landscape. Below is the list of a few players involved in motor neuron disease clinical trials:
This section covers the detailed analysis of each drug under multiple phases, including phase I, phase II, phase III, phase IV, and emerging drugs for motor neuron disease. It includes product description, trial ID, study type, drug class, mode of administration, and recruitment status of motor neuron disease drug candidates.
MN-166 (Ibudilast), sponsored by MediciNova, is being evaluated in the Phase 2b/3 COMBAT-ALS trial. It is a 12‑month randomized, double‑blind study with a 6‑month open‑label extension. The study is examining efficacy, safety, and tolerability in ALS, assessing function and survival. MN‑166 is an oral allosteric MIF inhibitor and selective PDE‑3/4/10 inhibitor that crosses the blood–brain barrier, attenuates glial activation, and shows neuroprotective, autophagy‑enhancing effects.
PLL001, is being evaluated in a Phase 1/2, randomized, double-blind, placebo-controlled ALS study with an open-label extension. The trial is examining safety, tolerability, efficacy, and pharmacodynamics of daily subcutaneous dosing for up to 48 weeks. PLL001 consists of multiple APIs using patented Poly‑L‑Lysine to deliver four short‑chain fatty acids to gut epithelium and across the BBB, restoring microbiome integrity and reducing gut leak.
QRL-101, by QurAlis Corporation, is a first‑in‑class selective Kv7.2/7.3 ion‑channel opener targeting motor‑neuron hyperexcitability caused by mis‑spliced Kv7.2 in ALS. This randomized, double‑blind, placebo‑controlled Phase 1 study is examining single oral doses in approximately 12 adults, evaluating safety, tolerability, and PK/PD relationships across escalating cohorts. The trial is assessing how QRL‑101 is modulating excitability biomarkers while characterizing exposure–response to inform dose selection for subsequent studies.
*Please note that this is only a partial list; the complete list of drugs will be available in the full report.*
The Motor Neuron Disease Pipeline Analysis Report provides a strategic overview of the latest and future landscape of treatments for motor neuron disease. It provides necessary information for making informed investment decisions along with research, development, and strategic planning efforts. The stakeholders will benefit from the essential insights into motor neuron disease collaborations, regulatory environments, and potential growth opportunities.
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*While we strive to always give you current and accurate information, the numbers depicted on the website are indicative and may differ from the actual numbers in the main report. At Expert Market Research, we aim to bring you the latest insights and trends in the market. Using our analyses and forecasts, stakeholders can understand the market dynamics, navigate challenges, and capitalize on opportunities to make data-driven strategic decisions.*
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