Spinal muscular atrophy - Pipeline Insight, 2021

DelveInsight’s, “Spinal muscular atrophy - Pipeline Insight, 2021,” report provides comprehensive insights about 10+ companies and 10+ pipeline drugs in the Spinal muscular atrophy 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

Spinal muscular atrophy Understanding
Spinal muscular atrophy: Overview
Spinal muscular atrophy (SMA) is a group of inherited disorders characterized by a loss of certain nerve cells in the spinal cord called motor neurons or anterior horn cells. Motor neurons receive the nerve impulses transmitted from the brain to the spinal cord (brainstem) and, in turn, transmit the impulses to the muscle via the peripheral nerves. The loss of motor neurons leads to progressive muscle weakness and muscle wasting (atrophy) in muscles closest to the trunk of the body (proximal muscles) such as the shoulders, hips and back. These muscles are necessary for crawling, walking, sitting up and head control. The more severe types of SMA can affect muscles involved in feeding, swallowing and breathing.

""Spinal muscular atrophy - Pipeline Insight, 2021"" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Spinal muscular atrophy pipeline landscape is provided which includes the disease overview and Spinal muscular atrophy treatment guidelines. The assessment part of the report embraces, in depth Spinal muscular atrophy 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, Spinal muscular atrophy 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 Spinal muscular atrophy R&D. The therapies under development are focused on novel approaches to treat/improve Spinal muscular atrophy.

Spinal muscular atrophy Emerging Drugs Chapters
This segment of the Spinal muscular atrophy 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.

Spinal muscular atrophy Emerging Drugs
● Apitegromab: Scholar Rock
Apitegromab is a selective inhibitor of the activation of myostatin and is an investigational product candidate for the treatment of patients with spinal muscular atrophy (SMA). Myostatin, a member of the TGFβ superfamily of growth factors, is expressed primarily by skeletal muscle cells, and the absence of its gene is associated with an increase in muscle mass and strength in multiple animal species, including humans. Scholar Rock believes that inhibiting myostatin activation with apitegromab may promote a clinically meaningful improvement in motor function in patients with SMA. The U.S. Food and Drug Administration (FDA) has granted Fast Track (FTD), Orphan Drug (ODD) and Rare Pediatric Disease (RPD) designations, and the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) and Orphan Medicinal Product designations, to apitegromab for the treatment of SMA. Currently, it is in phase II stage of development.

● ACTX-401: Novartis
ACTX-401 is a gene therapy being developed for spinal muscular atrophy with respiratory distress type 1 (SMARD1) and is currently in IND stage of development.
Further product details are provided in the report……..

Spinal muscular atrophy: Therapeutic Assessment
This segment of the report provides insights about the different Spinal muscular atrophy drugs segregated based on following parameters that define the scope of the report, such as:
● Major Players in Spinal muscular atrophy
There are approx. 10+ key companies which are developing the therapies for Spinal muscular atrophy. The companies which have their Spinal muscular atrophy drug candidates in the most advanced stage, i.e. phase II include, Scholar Rock.

● Phases
DelveInsight’s report covers around 10+ 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
Spinal muscular atrophy 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
● Parenteral
● intravenous
● Subcutaneous
● Topical.

● Molecule Type
Products have been categorized under various Molecule types such as
● Monoclonal Antibody
● Peptides
● Polymer
● Small molecule
● Gene therapy
● Product Type
Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Spinal muscular atrophy: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Spinal muscular atrophy 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 Spinal muscular atrophy drugs.

Spinal muscular atrophy Report Insights
● Spinal muscular atrophy Pipeline Analysis
● Therapeutic Assessment
● Unmet Needs
● Impact of Drugs

Spinal muscular atrophy 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 Spinal muscular atrophy drugs?
● How many Spinal muscular atrophy drugs are developed by each company?
● How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Spinal muscular atrophy?
● What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Spinal muscular atrophy 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 Spinal muscular atrophy and their status?
● What are the key designations that have been granted to the emerging drugs?

Key Players
• Scholar Rock
• Biogen
• Astellas Pharma
• Alcyone Therapeutics
• AndroScience Corporation
• Hanugen Theraputics
• Voyager Therapeutics
• Genethon
• Astellas Pharma
• Hoffmann-La Roche

Key Products
• Apitegromab
• BIIB 110
• Reldesemtiv
• ACTX 401
• ASCJ 9
• Reldesemtiv
• RO7204239