Amyotrophic Lateral Sclerosis Als Pipeline Insight
DelveInsight’s, “Amyotrophic lateral sclerosis – Pipeline Insight, 2020,” report provides comprehensive insights about 250+ companies and 250+ pipeline drugs in Amyotrophic lateral sclerosis 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.
Amyotrophic lateral sclerosis Understanding
Amyotrophic lateral sclerosis: Overview
Amyotrophic lateral sclerosis (ALS) is a group of rare neurological diseases that mainly involve the nerve cells (neurons) responsible for controlling voluntary muscle movement. Voluntary muscles produce movements like chewing, walking, and talking. The disease is progressive, meaning the symptoms get worse over time. Currently, there is no cure for ALS and no effective treatment to halt, or reverse, the progression of the disease.
The onset of ALS can be so subtle that the symptoms are overlooked but gradually these symptoms develop into more obvious weakness or atrophy that may cause a physician to suspect ALS. Some of the early symptoms include:
- Fasciculations (muscle twitches) in the arm, leg, shoulder, or tongue
- Muscle cramps
- Tight and stiff muscles (spasticity)
- Muscle weakness affecting an arm, a leg, neck or diaphragm.
- Slurred and nasal speech
- Difficulty chewing or swallowing.
No one test can provide a definitive diagnosis of ALS. ALS is primarily diagnosed based on detailed history of the symptoms and signs observed by a physician during physical examination along with a series of tests to rule out other mimicking diseases. However, the presence of upper and lower motor neuron symptoms strongly suggests the presence of the disease. Physicians will review an individual’s full medical history and conduct a neurologic examination at regular
No cure has yet been found for ALS. However, there are treatments available that can help control symptoms, prevent unnecessary complications, and make living with the disease easier.
The U.S. Food and Drug Administration (FDA) has approved the drugs riluzole (Rilutek) and edaravone (Radicava) to treat ALS. Riluzole is believed to reduce damage to motor neurons by decreasing levels of glutamate, which transports messages between nerve cells and motor neurons. Physicians can also prescribe medications to help manage symptoms of ALS, including muscle cramps, stiffness, excess saliva and phlegm, and the pseudobulbar affect (involuntary or uncontrollable episodes of crying and/or laughing, or other emotional displays).
Amyotrophic lateral sclerosis Emerging Drugs Chapters
This segment of the Amyotrophic lateral sclerosis report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, 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.
Amyotrophic lateral sclerosis Emerging Drugs
- Autologous MSC-NTF cells: BrainStorm Cell Therapeutics
Autologous MSC-NTF cells is being developed by BrainStorm Cell Therapeutics for the treatment of of ALS. BrainStorm is currently enrolling a phase III pivotal trial using repeat-administration of autologous MSC-NTF cells in ALS at six leading clinical sites in the United States, supported by a grant from the California Institute for Regenerative Medicine.
- Pegcetacoplan: Apellis Pharmaceuticals
Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate excessive activation of the complement cascade. Apellis plans to initiate a clinical trial in ALS to understand if targeting C3 with pegcetacoplan may have the potential to slow the progression of ALS by reducing the inflammation that causes motor neuron death. It is currently in phase II stage of development.
BIIB078 targets specific messenger RNA emerging from the C9orf72 gene and degrades it, removing the RNA and preventing production of the abnormal protein it encodes. Importantly, BIIB078 largely preserves the normal C9orf72 protein coming from the gene. Preclinical researchers have found this approach to be useful, decreasing ALS-related pathology in preclinical models and improving behavioral function. It is currently in phase I stage of development.
- ALS-205: Alsonex Pharmaceuticals
ALS-205 is a potent antagonist of the human C5a1 receptor with no activity at the C5a2 receptor. It binds to the human C5a1 receptor on white blood cells and is a functional insurmountable antagonist. ALS-205 has shown similar effects on other cell types including lymphocytes, monocytes and monocyte-derived macrophages. It is also highly receptor selective, failing even at 100 μM concentrations to antagonize myeloperoxidase release from leukocytes stimulated with fMet-Leu-Phe, leukotriene B4, platelet-activating factor, C3a, or IL-8. ALS-205 is chemically stable to peptidase degradation in blood and gastric fluids and is both small enough and sufficiently lipophilic to be orally active at ≤1 mg/kg/day in vivo and readily accesses the brain. It is currently in preclinical stage of development.
Further product details are provided in the report……..
Amyotrophic lateral sclerosis: Therapeutic Assessment
This segment of the report provides insights about the different Amyotrophic lateral sclerosis drugs segregated based on following parameters that define the scope of the report, such as:
- Major Players in Amyotrophic lateral sclerosis
There are approx. 250+ key companies which are developing the therapies for Amyotrophic lateral sclerosis. The companies which have their Amyotrophic lateral sclerosis drug candidates in the most advanced stage, i.e. phase III include BrainStorm Cell Therapeutics and others
DelveInsight’s report covers around 250+ products under different phases of clinical development like
- Late-stage products (Phase III)
- Mid-stage products (Phase II)
- Early-stage products (Phase I) along with the details of
- Pre-clinical and Discovery stage candidates
- Discontinued & Inactive candidates
- Route of Administration
Amyotrophic lateral sclerosis pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as
- Molecule Type
Products have been categorized under various Molecule types such as
- Gene therapies
- Small molecule
- Monoclonal antibodies
- Product Type
Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.
Amyotrophic lateral sclerosis: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase III, II, I, preclinical and discovery stage. It also analyses Amyotrophic lateral sclerosis 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 Amyotrophic lateral sclerosis drugs.
- The companies and academics are working to assess challenges and seek opportunities that could influence Amyotrophic lateral sclerosis R&D. The therapies under development are focused on novel approaches to treat/improve Amyotrophic lateral sclerosis.
- ALS-205 has been granted orphan drug status by the US FDA for ALS
- In October 2020, Catalent and BrainStorm Cell Therapeutics announced an agreement for the manufacture of NurOwn, BrainStorm’s autologous cellular therapy being investigated for the treatment of amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease or motor neuron disease.
Amyotrophic lateral sclerosis Report Insights
- Amyotrophic lateral sclerosis Pipeline Analysis
- Therapeutic Assessment
- Unmet Needs
- Impact of Drugs
Amyotrophic lateral sclerosis Report Assessment
- Pipeline Product Profiles
- Therapeutic Assessment
- Pipeline Assessment
- Inactive drugs assessment
- Unmet Needs
Current Treatment Scenario and Emerging Therapies:
- How many companies are developing Amyotrophic lateral sclerosis drugs?
- How many Amyotrophic lateral sclerosis drugs are developed by each company?
- How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Amyotrophic lateral sclerosis?
- What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Amyotrophic lateral sclerosis 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 Amyotrophic lateral sclerosis and their status?
- What are the key designations that have been granted to the emerging drugs?
- BrainStorm Cell Therapeutics
- Apellis Pharmaceuticals
- Alsonex Pharmaceuticals
- Astellas Pharma
- Grifols Therapeutics
- AL-S Pharma
- Mitsubishi Tanabe Pharma Corporation
- Autologous MSC-NTF cells