Motor Neuron Disease - Pipeline Insight, 2024

This “Motor Neuron Disease- Pipeline Insight, 2024” report provides comprehensive insights about 180+ companies and 200+ pipeline drugs in Motor Neuron Disease pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Geography Covered

• Global coverage

Motor Neuron Disease: Understanding

Motor Neuron Disease: Overview

Motor neuron diseases (MNDs) are a group of progressive neurological disorders that destroy motor neurons, the cells that control skeletal muscle activity such as walking, breathing, speaking, and swallowing. This group includes diseases such as amyotrophic lateral sclerosis, progressive bulbar palsy, primary lateral sclerosis, progressive muscular atrophy, spinal muscular atrophy, Kennedy's disease, and post-polio syndrome. Motor neuron disease (MND) is an uncommon condition that affects the brain and nerves. It causes weakness that gets worse over time. There's no cure for MND, but there are treatments to help reduce the impact it has on a person's daily life. Some people live with the condition for many years. MND can significantly shorten life expectancy and, unfortunately, eventually leads to death. Generally, MND is believed to be caused because of a combination of environmental, lifestyle and genetic factors. Most cases of MND develop without an obvious cause. Around 1 in 10 cases are 'familial', meaning the condition is inherited. This is due to a genetic mutation, or an error in the gene.

Symptoms of motor neuron disease happen gradually and may not be obvious at first. Early symptoms can include: weakness in ankle or leg that might trip, or find it harder to climb stairs, slurred speech, which may develop into difficulty swallowing some foods a weak grip might drop things, or find it hard to open jars or do up buttons, muscle cramps and twitches, weight loss the arms or leg muscles may have become thinner over time, difficulty stopping from crying or laughing in inappropriate situations. The different types of MND cause similar symptoms and have three stages: early, middle, and advanced. The diseases progress at different speeds and vary in severity.

In many cases, there are no specific tests to diagnose MNDs. Symptoms may vary among individuals and, in the early stages, may be similar to those of other diseases, making diagnosis difficult. However, there are gene tests for SMA, Kennedy's disease, and some causes of familial ALS. A physical exam should be followed by an extensive neurological exam. These tests, usually done together, can identify the differences between muscles diseases and MNDs that are Electromyography (EMG) is used to diagnose disorders of lower motor neurons, and a nerve conduction study. Additional tests may include, laboratory tests of blood, urine, or other substances, Magnetic resonance imaging (MRI), muscle or nerve biopsy can help confirm nerve disease and nerve regeneration. There is no cure or standard treatment for MNDs. Symptomatic and supportive treatment can help individuals be more comfortable while maintaining their quality of life. Muscle relaxers such as baclofen, tizanidine, and the benzodiazepines may reduce muscle stiffness and help muscle spasms.

Botulinum toxin injections may be used to treat muscle stiffness by weakening overactive muscles. They may be injected into the salivary glands to stop drooling. Excessive saliva also can be treated with medications such as amitriptyline, glycopyrrolate, and atropine. Physical therapy and rehabilitation may help improve posture, prevent joint immobility, and slow muscle weakness and atrophy. Stretching and strengthening exercises may help reduce stiffness, as well as increase range of motion and circulation.

Motor Neuron Disease- Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Motor Neuron Disease pipeline landscape is provided which includes the disease overview and Motor Neuron Disease treatment guidelines. The assessment part of the report embraces, in depth Motor Neuron Disease commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Motor Neuron Disease collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

The companies and academics are working to assess challenges and seek opportunities that could influence Motor Neuron Disease R&D. The therapies under development are focused on novel approaches to treat/improve Motor Neuron Disease.

Motor Neuron Disease Emerging Drugs Chapters

This segment of the Motor Neuron Disease report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Motor Neuron Disease Emerging Drugs

Talditercept alfa: Biohaven Pharmaceuticals, Inc.Taldefgrobep alfa (also known as BMS-986089) is a modified adnectin designed to specifically bind to myostatin (GDF-8). Adnectins are an established proprietary protein therapeutic class based on human fibronectin, an extracellular protein that is naturally abundant in human serum. The intrinsic properties of an adnectin align with the properties needed to make a successful drug, including high potency, specificity, stability, and favorable half-life. Currently, the drug is in Phase III stage of its development for the treatment of Spinal Muscular Atrophy.

Pridopidine: Prilenia Therapeutics Pridopidine is an oral investigational drug. It is administered in a small, easy-to-swallow capsule twice daily. Multiple clinical studies have been conducted providing important understanding about pridopidine’s safety, mechanism of action, and efficacy. Imaging studies in humans show that pridopidine enters the brain and spinal cord, where it activates a protein called the sigma-1 receptor (S1R). Currently, the drug is in Phase II/III stage of its development for the treatment of Amyotrophic lateral sclerosis.

VM202: Helixmith Co., Ltd.Engensis (VM202) is an innovative gene therapy drug that provides fundamental treatment through tissue regeneration. It is non-viral plasmid DNA product, Engensis, is designed to express recombinant HGF protein in nerve and Schwann cells to promote nerve system regeneration and induce the formation of microvascular blood vessels. Data from previous clinical studies suggest that Engensis is well tolerated and has the potential to provide durable analgesic and/or symptomatic relief in a variety of disease settings. Currently, the drug is in Phase II stage of its development for the treatment of Amyotrophic lateral sclerosis.

TPN-101: Transposon Therapeutics, Inc.TPN-101, is the most potent known small molecule that is inhibitor of LINE-1 reverse transcriptase and has excellent systemic and brain bioavailability with once daily oral dosing. TPN-101, also known as censavudine. Currently, the drug is in Phase II stage of its development for the treatment of Amyotrophic lateral sclerosis.

GC 101: Gene Cradle Therapeutics GC101 injection is a gene replacement therapy drug based on AAV viral vector. Its structure and administration method are optimized according to the disease characteristics and biodistribution characteristics to achieve safer and more effective purposes. The drug candidate is been developed by GeneCradle Therapeutics. Currently, the drug is in Phase I/II stage of its development for the treatment of Spinal muscularatrophy.

VRG 50635: Verge Genomics VRG50635 is a potent, orally bioavailable PIKfyve inhibitor that improves survival in ALS patient neurons and has shown efficacy in multiple preclinical studies in ALS-relevant models of motor neuron degeneration. VRG50635 is the only PIKfyve inhibitor in clinical development that has been specifically optimized for treatment of central nervous system disorders like ALS, and has the potential to become a best-in-class therapy. Currently, the drug is in Phase I stage of its development for the treatment of Spinal muscularatrophy.

QRL 201: Qur Alis Corporation QRL-201 is a first-in-class precision therapeutic product candidate aiming to restore STATHMIN-2 (STMN2) expression in ALS patients. STMN2 is a well-validated protein important for neural repair and axonal stability and is the most significantly regulated gene by TDP-43 exclusively in humans. Its expression is significantly decreased in nearly all ALS patients and it is the most consistently decreased gene over all sporadic ALS patient data sets. QRL-201 rescues STMN2 loss of function in QurAlis ALS patient-derived motor neuron disease models in the presence of TDP-43 pathology. Currently, the drug is in Phase I stage of its development for the treatment of Amyotrophiclateralsclerosis.

Motor Neuron Disease: Therapeutic Assessment

This segment of the report provides insights about the different Motor Neuron Disease drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Motor Neuron Disease

• There are approx. 180+ key companies which are developing the therapies for Motor Neuron Disease. The companies which have their Motor Neuron Disease drug candidates in the most advanced stage, i.e. phase III include, Biohaven Pharmaceuticals, Inc.

Phases

This 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

Route of Administration

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

• Oral
• Intravenous
• Subcutaneous
• Parenteral
• Topical

Molecule Type

Products have been categorized under various Molecule types such as

• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy

Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Motor Neuron Disease: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Motor Neuron Disease therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Motor Neuron Disease drugs.

Motor Neuron Disease Report Insights

• Motor Neuron Disease Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Motor Neuron Disease Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Motor Neuron Disease drugs?
• How many Motor Neuron Disease drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Motor Neuron Disease?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Motor Neuron Disease therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Motor Neuron Disease and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Biohaven Pharmaceuticals, Inc.
• Prilenia Therapeutics
• Helixmith Co., Ltd.
• Transposon Therapeutics, Inc.
• Gene CradleTherapeutics
• Verge Genomics
• Qur AlisCorporation
• 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
• Neuro Sense TherapeuticsLtd.
• Knopp Biosciences
• MediciNova
• Amylyx Pharmaceuticals Inc.
• Neuropore Therapies Inc
• NMD Pharma A/S
• Supernus Pharmaceuticals, Inc.
• Mitsubishi Tanabe Pharma America Inc.
• MaaT Pharma

Key Products

• Talditercept alfa
• Pridopidine
• VM202
• Censavudine
• GC 101
• VRG 50635
• QRL 201
• ZYIL1
• Zilucoplan
• Tetramethylpyrazine nitrone
• SRK-015
• SPG302
• SLS-005
• SAR443820
• RO7204239
• RNS60
• Reldesemtiv
• RAPA-501
• Q-Cells
• PTC857
• Prosetin
• PrimeC
• Dexpramipexole
• MN-166
• AMX0035
• NPT520-34
• NMD670
• MYOBLOC
• MT-1186
• MaaT033

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