Amyotrophic Lateral Sclerosis - Pipeline Insight, 2024

This “Amyotrophic Lateral Sclerosis- Pipeline Insight, 2024” report provides comprehensive insights about 90+ companies and 100+ 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.

Geography Covered

• Global coverage

Amyotrophic Lateral Sclerosis: Understanding

Amyotrophic Lateral Sclerosis: Overview

Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's Disease, is a rare neurological disease that affects motor neurons - those nerve cells in the brain and spinal cord that control voluntary muscle movement. Voluntary muscles are those we choose to move to produce movements like chewing, walking, and talking. The disease is progressive, meaning the symptoms get worse over time. ALS has no cure and there is no effective treatment to reverse its progression. ALS is a type of motor neuron disease. As motor neurons degenerate and die, they stop sending messages to the muscles, which causes the muscles to weaken, start to twitch (fasciculations), and waste away (atrophy). Eventually, the brain loses its ability to initiate and control voluntary movements. Early symptoms include: Muscle twitches in the arm, leg, shoulder, or tongue, Muscle cramps, Tight and stiff muscles (spasticity), Muscle weakness affecting an arm, a leg, the neck, or diaphragm, Slurred and nasal speech and Difficulty chewing or swallowing. Some studies suggest that military veterans are about one and half to two times more likely to develop ALS, although the reason for this is unclear. Possible risk factors for veterans include exposure to lead, pesticides, and other environmental toxins. Nearly all cases of ALS are considered sporadic. This means the disease seems to occur at random with no clearly associated risk factors and no family history of the disease. Although family members of people with sporadic ALS are at an increased risk for the disease, the overall risk is very low and most will not develop ALS. There is no single test that can definitely diagnose ALS. Your healthcare provider will conduct a physical exam and review your full medical history. A neurologic examination will test your reflexes, muscle strength, and other responses and will be held at regular intervals to assess whether symptoms such as muscle weakness, muscle wasting, and spasticity are progressively getting worse. There is no treatment to reverse damage to motor neurons or cure ALS. However, treatments can make living with the disease easier.

Amyotrophic Lateral Sclerosis- Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Amyotrophic Lateral Sclerosis pipeline landscape is provided which includes the disease overview and Amyotrophic Lateral Sclerosis treatment guidelines. The assessment part of the report embraces, in depth Amyotrophic Lateral Sclerosis 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, Amyotrophic Lateral Sclerosis 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 Amyotrophic Lateral Sclerosis R&D. The therapies under development are focused on novel approaches to treat/improve Amyotrophic Lateral Sclerosis.

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 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

Tofersen: BiogenTofersen is an antisense drug being evaluated for the potential treatment of SOD1-ALS. Tofersen binds to SOD1 mRNA, allowing for its degradation by RNase-H in an effort to reduce synthesis of SOD1 protein production. Tofersen is also being studied in the Phase 3 ATLAS study, which is designed to evaluate the ability of tofersen to delay clinical onset when initiated in presymptomatic individuals with a SOD1 genetic mutation and biomarker evidence of disease activity. Biogen licensed tofersen from Ionis Pharmaceuticals, Inc. under a collaborative development and license agreement. Biogen Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted a New Drug Application (NDA) for tofersen, an investigational drug for superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS). The application has been granted priority review and given a Prescription Drug User Fee Act action date of January 25, 2023.

RNS60: Revalesio CorporationRNS60 is being developed to provide disease modifying and potentially restorative treatments for neurological diseases. It activates intracellular signaling pathways to increase mitochondrial biogenesis and function and reduce inflammation. RNS60 safely protects neurons and oligodendrocytes and modulates the activity of immune cells to restore homeostasis. RNS60 has been granted Orphan Drug and Fast Track designations for ALS from the U.S. Food and Drug Administration.

ANX005: AnnexonANX005 is an antibody-based medication designed to bind and block the activity of C1q, which is expected to keep synapses healthy and slow or halt neurodegeneration. The antibody is being developed for a number neurodegenerative diseases, such as ALS, Guillain-Barré syndrome (GBS), Huntington’s disease (HD), and cold agglutinin disease (CAD). Clinical trials are ongoing in all these indications. The ongoing ALS trial is investigating ANX005 in patients whose first symptoms of weakness began in the past three years. Participants will receive two induction doses about 5-6 days apart, followed by every-two-week injections up to week 22. All then will be followed for another 14 weeks. Preliminary data showed that ANX005 treatment resulted in a reduction in neurofilament light chain (NfL) levels, a marker of nerve cell damage, during the initial 12 weeks of treatment. But levels rose to their initial value when patients stopped receiving treatment.

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. 90+ 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. Preregistration include, Biogen.

Phases

This report covers around 100+ 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

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

• Intra-articular
• Intraocular
• Intrathecal
• Intravenous
• Ophthalmic
• Oral
• Parenteral
• Subcutaneous
• Topical
• Transdermal

Molecule Type

Products have been categorized under various Molecule types such as

• Oligonucleotide
• Peptide
• Small molecule

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 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.

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

Key Questions

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?

Key Players

• Molecular Partners
• Biogen
• Ionis Pharmaceuticals
• Ferrer Internacional S.A.
• AbbVie
• Calico Life Sciences LLC
• Genuv Inc.
• Kadimastem
• Corcept Therapeutics
• AL-S Pharma
• Rapa Therapeutics LLC
• Cytokinetics
• MediciNova
• Retrotope, Inc.
• Woolsey Pharmaceuticals,
• Sanofi, PTC Therapeutics
• Helixmith Co., Ltd.
• Annexon, Inc.
• Denali Therapeutics Inc.
• Revalesio Corporation
• Clene Nanomedicine
• Ashvattha Therapeutics, Inc.
• Apellis Pharmaceuticals, Inc.
• Procypra Therapeutics
• Knopp Biosciences
• InFlectis BioScience
• AI Therapeutics, Inc.
• Cellenkos
• ZZ Biotech, LLC
• QurAlis Corporation
• Alector Inc.
• NeuroSense Therapeutics Ltd.
• Novartis Pharmaceuticals
• Eledon Pharmaceuticals

Key Products

• BIIB067
• FAB122
• ABBV-CLS-7262
• Trametinib
• BIIB105
• AstroRx
• Dazucorilant
• AP-101
• RAPA-501
• ION-363
• Reldesemtiv
• MN-166
• RT001
• Fasudil
• SAR443820
• PTC857
• ANX005
• DNL343
• RNS60
• BIIB100
• CNMAu8
• 18F-OP-801
• Pegcetacoplan (APL-2)
• Cu(II)ATSM
• KNS-760704
• IFB-088
• LAM-002A
• CK 0803
• 3K3A-APC
• QRL-201
• Dexpramipexole
• AL001
• PrimeC
• BLZ945
• AT-1501

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