Lou Gehrig's disease - Pipeline Insight, 2024

This “Lou Gehrig’s disease - Pipeline Insight, 2024” report provides comprehensive insights about 75+ companies and 80+ pipeline drugs in Lou Gehrig’s 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

Lou Gehrig’s disease: Understanding

Lou Gehrig’s disease: Overview

Lou Gehrig’s Disease, formally known as Amyotrophic Lateral Sclerosis (ALS), is a progressive neurodegenerative disorder that affects the nerve cells responsible for controlling voluntary muscle movements. These nerve cells, known as motor neurons, are located in the brain and spinal cord. Over time, ALS leads to the degeneration and death of motor neurons, which prevents them from transmitting signals to muscles, resulting in loss of muscle function. As muscles weaken and waste away, individuals experience increasing difficulty with basic motor tasks such as walking, speaking, swallowing, and breathing. ALS is relatively rare, but it is one of the most serious forms of motor neuron disease.

There are two main forms of ALS: sporadic and familial. Sporadic ALS is the most common form, accounting for the vast majority of cases, and it typically occurs with no clear family history of the disease. Familial ALS, which is hereditary, constitutes a smaller percentage of cases and is linked to genetic mutations. Regardless of the form, ALS symptoms usually begin with subtle muscle weakness, often in the hands, legs, or throat. As the disease progresses, this weakness spreads to other parts of the body, leading to widespread paralysis. However, the sensory functions, cognitive abilities, and autonomic functions (such as bladder and bowel control) usually remain intact for most of the disease course.

The exact causes of ALS are not well understood, but research suggests a combination of genetic, molecular, and environmental factors may play a role. Studies indicate that disruptions in cellular processes such as protein folding, oxidative stress, and inflammation could contribute to motor neuron death in ALS. Environmental factors, including exposure to toxins, head trauma, and certain occupational risks, have been investigated as potential contributors, though none have been definitively proven. The complexity of ALS pathology makes it challenging to pinpoint a singular cause, but scientists continue to study how these mechanisms work together in the progression of the disease.

There is currently no cure for ALS, and treatment focuses on symptom management and slowing the progression of the disease. Multidisciplinary care, including physical therapy, occupational therapy, and speech therapy, is often employed to help patients maintain their quality of life as much as possible. Supportive equipment, such as ventilators or communication devices, may also be necessary as the disease advances. Ongoing research is exploring ways to understand the biological processes behind ALS, with the aim of developing new treatments that could potentially modify the disease or target its underlying causes.

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

Lou Gehrig’s disease Emerging Drugs Chapters

This segment of the Lou Gehrig’s disease 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.

Lou Gehrig’s disease Emerging Drugs

Ulefnersen: Ionis Pharmaceuticals

Ulefnersen, formerly known as ION363, is an investigational antisense medicine designed to reduce the production of the fused in sarcoma (FUS) protein to treat people with amyotrophic lateral sclerosis (ALS) caused by mutations in the FUS gene. Ulefnersen is also known as Jacifusen (not an official USAN name) in honor of Jaci Hermstad, the first patient treated with the drug under an expanded access program. Because antisense-mediated reduction of mutant FUS protein in a FUS-ALS mouse model demonstrated the ability to prevent motor neuron loss, it is hypothesized that reduction of FUS protein will reverse or prevent disease progression in FUS-ALS patients. The drug is currently being evaluated under Phase III clinical trial for the treatment of ALS.

MN-166: MediciNova

MN-166 is a first-in-class, orally bioavailable, small molecule glial attenuator that suppresses pro-inflammatory cytokines IL-1ß, TNF-a, and IL-6, and may upregulate the anti-inflammatory cytokine IL-10. It has additionally been shown to be a toll-like receptor 4 (TLR4) functional antagonist that may contribute to its attenuation of neuroinflammation. The drug is currently being evaluated under Phase II/III clinical trial for the treatment of ALS.

RNS60: Revalesio

RNS60 is being developed to provide disease modifying and potentially restorative treatments for neurological diseases. The drug 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 US Food and Drug Administration. The drug is currently being evaluated under Phase II clinical trial for the treatment of ALS.

QRL-201: QurAlis Corporation

QRL-201 is a first-in-class therapeutic product candidate aiming to restore STMN2 expression in ALS patients. STMN2 is a well-validated protein important for neural repair and axonal stability, the expression of which is significantly decreased in nearly all ALS patients. QRL-201 rescues STMN2 loss of function in QurAlis ALS patient-derived motor neuron disease models in the presence of TDP-43 pathology. The drug is currently being evaluated under Phase I clinical trial for the treatment of ALS.

Lou Gehrig’s disease: Therapeutic Assessment

This segment of the report provides insights about the different Lou Gehrig’s disease drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Lou Gehrig’s disease

• There are approx. 75+ key companies which are developing the therapies for Lou Gehrig’s disease. The companies which have their Lou Gehrig’s disease drug candidates in the most advanced stage, i.e. Phase III include, Ionis Pharmaceuticals.

Phases

The report covers around 80+ 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

Lou Gehrig’s 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.

Lou Gehrig’s disease: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Lou Gehrig’s 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 Lou Gehrig’s disease drugs.

Lou Gehrig’s disease Report Insights

• Lou Gehrig’s disease Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Lou Gehrig’s 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 Lou Gehrig’s disease drugs?
• How many Lou Gehrig’s disease drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Lou Gehrig’s disease?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Lou Gehrig’s 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 Lou Gehrig’s disease and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Ionis Pharmaceuticals
• 1ST Biotherapeutics
• Scholar Rock
• Revalesio
• QurAlis Corporation
• Sanofi
• MediciNova
• Helixmith
• Verge Genomics
• UCB

Key Products

• Ulefnersen
• FB418
• SRK-015
• RNS60
• QRL-201
• SAR443820
• MN-166
• VM202
• VRG-50635
• Zilucoplan

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