DelveInsight’s, “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 Multiple System Atrophy pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Multiple System Atrophy therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
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Key Takeaways from the Multiple System Atrophy Pipeline Report
- June 2024:- H. Lundbeck A/S– This study will consist of a double-blind period (DBP) and will include an optional open-label treatment extension (OLE) period. Participants in the DBP will be randomized to Lu AF82422 or placebo (2:1). All participants entering the OLE will receive Lu AF82422 during the OLE.
- June 2024:- Theravance Biopharma– A Phase 3, Multi-center, Randomized Withdrawal and Long Term Extension Study of Ampreloxetine for the Treatment of Symptomatic Neurogenic Orthostatic Hypotension in Participants With Multiple System Atrophy. This is a Phase 3, multi-center, randomized withdrawal study to evaluate the efficacy and durability of ampreloxetine in participants with MSA and symptomatic nOH after 20 weeks of treatment. This study includes 4 periods: Screening, open label, randomized withdrawal, and long-term treatment extension (LTE).
- June 2024:- Vertex Pharmaceuticals Incorporated- A Phase 1/2, Randomized, Double-blind, Placebo-controlled Single- and Multiple-dose Escalation Study Evaluating the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of VX-670 in Adult Subjects With Myotonic Dystrophy Type 1. The purpose of the study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VX-670 at different single and multiple doses in participants with DM1.
- June 2024:- BioMarin Pharmaceutical- This is Phase 1/2, open-label, multi-center study consisting of 2 parts to evaluate the safety and tolerability of BMN 351 at escalating doses in participants with Duchenne Muscular Dystrophy (DMD) with genetic mutations amenable to exon 51 skipping. The purpose of this study is to test the safety and tolerability of BMN 351 in participants with Duchenne Muscular Dystrophy (DMD) with a genetic mutation amenable to exon 51 skipping.
- June 2024:- Dyne Therapeutics– The primary purpose of this study is to evaluate the safety, tolerability, and dystrophin protein levels in muscle tissue following multiple intravenous (IV) doses of DYNE-251 in participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.
- June 2024:- ARTHEx Biotech S.L.- A Phase 1/2a Double-Blind, Placebo-controlled, Single- and Multiple Ascending Dose Study to Assess the Safety, Tolerability, PK, PD and Efficacy of IV Administration of ATX-01 In Male and Female Participants Aged 18 to 64 With Classic DM1. The goal of this clinical trial is to test ATX-01 in participants with myotonic dystrophy type 1 (DM1). The main question it aims to answer is if ATX-01 is safe and well tolerated. The trial will compare the safety and tolerability of ATX-01 and a matching placebo.
- DelveInsight’s Multiple System Atrophy pipeline report depicts a robust space with 20+ active players working to develop 22+ pipeline therapies for Multiple System Atrophy treatment.
- The leading Multiple System Atrophy Companies such as Theravance Biopharma, Alterity Therapeutics, AskBio, Kainos Medicines, CORESTEM Inc., ProMIS Neuroscience, H. Lundbeck A/S, and others.
- Promising Multiple System Atrophy Therapies such as Lu AF82422, hOMSC300, TAK-341, KM-819, ONO-2808, Ampreloxetine, Nusinersen, OAV101, and others.
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Multiple System Atrophy Emerging Drugs Profile
- 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.
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Multiple System Atrophy pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration.
- Oral
- Intravenous
- Subcutaneous
- Parenteral
- Topical
Multiple System Atrophy Products have been categorized under various Molecule types such as
- Recombinant fusion proteins
- Small molecule
- Monoclonal antibody
- Peptide
- Polymer
- Gene therapy
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Multiple System Atrophy Companies and Therapies
- Theravance Biopharma: Ampreloxetine
- Biogen: Nusinersen
- Novartis Pharmaceuticals: OAV101
- GeneCradle Inc.: GC101
- Hoffmann- La Roche: RO7204239
Scope of the Multiple System Atrophy Pipeline Report
- Coverage- Global
- Multiple System Atrophy Companies- Theravance Biopharma, Alterity Therapeutics, AskBio, Kainos Medicines, CORESTEM Inc., ProMIS Neuroscience, H. Lundbeck A/S, and others.
- Multiple System Atrophy Therapies- Lu AF82422, hOMSC300, TAK-341, KM-819, ONO-2808, Ampreloxetine, Nusinersen, OAV101, and others.
- Multiple System Atrophy Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
- Multiple System Atrophy Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III
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Table of Content
- Introduction
- Executive Summary
- Multiple System Atrophy: Overview
- Pipeline Therapeutics
- Therapeutic Assessment
- Multiple System Atrophy – DelveInsight’s Analytical Perspective
- Late Stage Products (Phase III)
- Ampreloxetine: Theravance Biopharma
- Drug profiles in the detailed report…..
- Mid Stage Products (Phase II)
- ATH434: Alterity Therapeutics
- Drug profiles in the detailed report…..
- Early Stage Products (Phase I)
- AB-1005: AskBio
- Drug profiles in the detailed report…..
- Preclinical and Discovery Stage Products
- Drug name: Company name
- Drug profiles in the detailed report…..
- Inactive Products
- Multiple System Atrophy Key Companies
- Multiple System Atrophy Key Products
- Multiple System Atrophy- Unmet Needs
- Multiple System Atrophy- Market Drivers and Barriers
- Multiple System Atrophy- Future Perspectives and Conclusion
- Multiple System Atrophy Analyst Views
- Multiple System Atrophy Key Companies
- Appendix
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