Dublin, April 16, 2024 (GLOBE NEWSWIRE) -- The "Motor Neuron Disease - Pipeline Insight, 2024" drug pipelines has been added to ResearchAndMarkets.com's offering.
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.
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: GeneCradle TherapeuticsGC101 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 muscular atrophy.
VRG 50635: Verge GenomicsVRG50635 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 muscular atrophy.
QRL 201: QurAlis CorporationQRL-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 Amyotrophic lateral sclerosis.
Key Questions
- 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.
- 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
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
Motor Neuron Disease: Therapeutic Assessment
Phases
- 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
- Oral
- Intravenous
- Subcutaneous
- Parenteral
- Topical
Molecule Type
- Recombinant fusion proteins
- Small molecule
- Monoclonal antibody
- Peptide
- Polymer
- Gene therapy
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
For more information about this drug pipelines report visit https://www.researchandmarkets.com/r/pk4jo3
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