DelveInsight’s, “Spinal Muscular Atrophy – Pipeline Insight, 2020,” report provides comprehensive insights about 40+ companies and 40+ pipeline drugs in 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. SMA is divided into subtypes based on age of onset and maximum function achieved. SMA types 0, 1, 2, 3 and 4 are inherited as autosomal recessive genetic disorders and are associated with abnormalities (mutations) in the SMN1 and SMA2 genes which are located on chromosome 5.
Symptoms
The primary symptom of chromosome 5-related (SMN-related) SMA is weakness of the voluntary muscles. The muscles most affected are those closest to the center of the body, such as those of the shoulders, hips, thighs, and upper back. The lower limbs seem to be affected more than the upper limbs, and deep tendon reflexes are decreased.
Special complications occur if the muscles used for breathing and swallowing are affected, resulting in abnormalities in these functions. If the muscles of the back weaken, spinal curvatures can develop.
Diagnosis
Spinal muscular atrophy (SMA) is sometimes difficult to diagnose, as symptoms can resemble other conditions or medical problems. Doctors usually diagnose SMA after a child has muscle weakness and decreased muscle tone.
If the clinician suspects SMA, they may use the following tests to diagnose the condition:
• Genetic blood tests, which can confirm the diagnosis of SMA
• An electromyography (EMG) test that measures the electrical activity of a muscle or a group of muscles (in some cases)
• A creatine kinase (CPK) test (to distinguish from other types of neuromuscular diseases, if necessary)
Treatment
The management of children with spinal muscular atrophy starts with the diagnosis and classification into 1 of the 5 categories. Health issues specific to spinal muscular atrophy are as follows:
• Pulmonary management: Children with SMA1 can survive beyond 2 years of age when offered tracheostomy or noninvasive respiratory support.
• An intermittent positive-pressure breathing device (mechanical in-exsufflator) has proven effective.
• Nutrition: Bulbar dysfunction is universal in SMA1 patients. Early gastrostomy should be considered as part of the management of such patients. The bulbar dysfunction eventually becomes a serious problem for spinal muscular atrophy II patients and only very late in the course of disease for spinal muscular atrophy III patients.
• In 2017, Spinraza (nusinersen) was FDA-approved as the first drug to treat children and adults with SMA.
• In 2019, the FDA approved Zolgensma (onasemnogene abeparvovec-xioi) for the treatment of children less than two years of age with SMA.
• In 2020, the FDA approved Evrysdi (risdiplam) to treat patients two months of age and older with SMA. Evrysdi is the first orally administered drug approved for the treatment of SMA.
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
• Sodium valproate: Kowa
Sodium valproate is being developed by Kowa for the treatment of Spinal muscular atrophy. It is currently in phase III stage of development.
• SRK-015: Scholar Rock
SRK-015, our most advanced product candidate, is a selective and local inhibitor of the activation of myostatin. Based on existing research on the mechanism of myostatin in muscle growth and strength, Scholar Rock believes the inhibition of the activation of myostatin with SRK-015 may promote a clinically beneficial increase in motor function. Scholar Rock is developing and investigating SRK-015 as a potential treatment to improve muscle strength and motor function in patients with Spinal Muscular Atrophy (SMA).it is currently in phase II stage of development.
• BIIB110: Biogen
BIIB110 is a hybrid activin II receptor (ACTIIR) ligand trap that sequesters both myostatin and activins while sparing the related ligand bone morphogen protein 9 (BMP9). This targeted mechanism of action may result in greater muscle mass, function and improved safety compared to other myostatin inhibition approaches. It is currently in phase I stage of development.
• Research programme: SNA based therapeutics: Exicure
SNA based therapeutics is being developed by Exicure for the treatment of Spinal muscular atrophy. It is currently in preclinical 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. 40+ 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 III include Kowa and others
• Phases
DelveInsight’s report covers around 40+ products under different phases of clinical development like
• Late-stage products (Phase II and Phase II/III)
• Mid-stage products (Phase II and Phase II/III)
• Early-stage products (Phase I/II and 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
• Infusion
• Intradermal
• Intramuscular
• Intranasal
• Intravenous
• Oral
• Parenteral
• Subcutaneous
• Topical.
• Molecule Type
Products have been categorized under various Molecule types such as
• Gene therapies
• Small molecule
• Vaccines
• Polymers
• Peptides
• Monoclonal antibodies

• 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.

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.
• In August 2020, Scholar Rock announced that the US Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation for SRK-015 for the treatment of Spinal Muscular Atrophy (SMA).
• In March 2018, Scholar Rock announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its lead antibody product candidate, SRK-015, for the treatment of spinal muscular atrophy (SMA).
• In July 2018, Biogen announced the acquisition of the muscle enhancement programme from AliveGen, which includes candidate, BIIB 110 (previously known as ALG 801).
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
• Kowa
• Scholar Rock
• Biogen
• Exicure
• AveXis
• Novartis Pharmaceuticals
• Catalyst Pharmaceuticals
• ReveraGen Biopharma
• Translate Bio

Key Products
• Sodium valproate
• SRK-015
• BIIB110
• Leadiant Biosciences
• Vamorolone
• Research programme: SNA based therapeutics
• Amifampridine Phosphate
• Nusinersen
• Onasemnogene
• Branaplam
• Valproic Acid
• Research programme: mRNA therapeutics