Multiple System Atrophy - Pipeline Insight, 2024

This “Multiple System Atrophy- Pipeline Insight, 2024” report provides comprehensive insights about 20+ companies and 22+ pipeline drugs in Multiple System 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

Multiple System Atrophy: Understanding

Multiple System Atrophy: Overview

Multiple system atrophy (MSA) is a rare disease, affecting potentially 15,000 to 50,000 Americans, including people of all racial groups. The cause of MSA is unknown. The vast majority of cases are sporadic, meaning they occur at random. Multiple system atrophy (MSA) is a progressive neurodegenerative disorder characterized by a combination of symptoms that affect both the central nervous system (which controls how a person moves), and the autonomic nervous system, which controls involuntary functions such as blood pressure or digestion.

The symptoms of MSA reflect the progressive loss of function and death of different types of nerve cells in the brain and spinal cord. MSA is one of a family of neurological disorders known as an atypical parkinsonian disorder. The initial symptoms can be difficult to distinguish from those of Parkinson's disease, and can include slowness of movement, tremor, or stiffness, clumsiness or lack of coordination, croaky, quivering voice, fainting or lightheadedness, bladder control problems, and many others.

There are two different types of MSA, which are categorized by the person’s most prominent symptoms when they’re evaluated by a doctor: a) Parkinsonian type MSA (MSA-P) which consists of primary symptoms similar to Parkinson's disease (such as slowness of movement, stiffness, and tremor) along with problems with balance, coordination, and autonomic nervous system dysfunction (such as urinary problems, sweating abnormalities, and digestion difficulties). b) Cerebellar type MSA (MSA-C) is associated with balance and coordination problems (ataxia), difficulty swallowing, speech problems or a quivering voice, and abnormal eye movements.

Diagnosing MSA can be difficult, particularly in the early stages because many of the features are similar to those observed in Parkinson's disease. In addition to taking a person’s medical and family history and performing a neurological examination, a doctor may order tests to support the diagnosis. These tests might include- Autonomic testing (such as blood pressure and heart rate control), Assessment of bladder function, Magnetic resonance imaging (MRI) may identify changes that suggest MSA or rule out other causes of the symptoms, Positron emission tomography (PET) scans to monitor metabolic function in specific parts of the brain, and Dopamine transporter (DaT) scans can assess the distribution and activity of dopamine in the brain.

Currently, there are no treatments to stop or slow the progression of MSA, and there is no cure. However, there are treatments to help people cope with the symptoms. Medications like levodopa may help improve motor function, Botulinum toxin injections to ease abnormal muscle postures, medicines to help with the bladder control and certain sleep problems are used. While therapies include Speech therapy that may help identify strategies to address swallowing difficulties and Physical therapy helps maintain mobility, reduce contractures, and decrease muscle spasms and abnormal posture.

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

Multiple System Atrophy Emerging Drugs Chapters

This segment of the Multiple System 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.

Multiple System Atrophy Emerging Drugs

Ampreloxetine: Theravance Biopharma

Ampreloxetine, an investigational, novel, selective, long-acting, once-daily norepinephrine reuptake inhibitor in development for the treatment of symptomatic neurogenic orthostatic hypotension (nOH) in patients with multiple system atrophy (MSA). The unique benefits of ampreloxetine treatment reported in MSA patients from Study 0170 included an increase in norepinephrine levels, a favorable impact on blood pressure, clinically meaningful and durable symptom improvements, and no signal for supine hypertension. The company has been granted an orphan drug designation in the US and, if results support it, plans to file an NDA for full approval based on the Phase III CYPRESS study. The drug is currently investigated in Phase III clinical trial for MSA patients.

ATH434: Alterity Therapeutics

ATH434, is an oral agent designed to inhibit the aggregation of pathological proteins implicated in neurodegeneration. ATH434 has been shown preclinically to reduce α-synuclein pathology and preserve neuronal function by restoring normal iron balance in the brain. As an iron chaperone, it has excellent potential to treat Parkinson’s disease as well as various Parkinsonian disorders such as Multiple System Atrophy (MSA). ATH434 successfully completed Phase I studies demonstrating the agent is well tolerated and achieved brain levels comparable to efficacious levels in animal models of MSA. ATH434 is currently being studied in two clinical trials: Study ATH434-201 is a randomized, double-blind, placebo-controlled Phase II clinical trial in patients with early-stage MSA and Study ATH434-202 is an open-label Phase II Biomarker trial in patients with more advanced MSA. ATH434 has been granted Orphan drug designation for the treatment of MSA by the US FDA and the European Commission.

AB-1005: AskBio

AB-1005 is an investigational gene therapy based on adeno-associated viral vector serotype 2 (AAV2) containing the human glial cell line-derived neurotrophic factor (GDNF) transgene, which allows for stable and continuous expression of GDNF in localized regions of the brain after direct neurosurgical injection with magnetic resonance imaging (MRI)-monitored convection enhanced delivery. GDNF is a homodimer that is a distantly related member of the transforming growth factor-β superfamily. In midbrain neuronal cell cultures, recombinant human GDNF promoted the survival and morphological differentiation of dopaminergic neurons and increased their high-affinity dopamine uptake.

Multiple System Atrophy: Therapeutic Assessment

This segment of the report provides insights about the different Multiple System Atrophy drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Multiple System Atrophy

• There are approx. 20+ key companies which are developing the therapies for Multiple System Atrophy. The companies which have their Multiple System Atrophy drug candidates in the most advanced stage, i.e. Phase III include, Theravance Biopharma.

Phases

This report covers around 22+ 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

Multiple System 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
• 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.

Multiple System Atrophy: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Multiple System 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 Multiple System Atrophy drugs.

Multiple System Atrophy Report Insights

• Multiple System Atrophy Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• Theravance Biopharma
• Alterity Therapeutics
• AskBio
• Kainos Medicines
• CORESTEM, Inc.
• ProMIS Neuroscience
• H. Lundbeck A/S

Key Products

• Ampreloxetine
• ATH434
• AB-1005
• KM-819
• Lu AF82422
• CE111BR16
• PMN442

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