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DelveInsight’s “Multiple System Atrophy Pipeline Insight 2026” report provides comprehensive insights about 20+ companies and 22+ pipeline drugs in the 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 Pipeline Therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Curious about the latest updates in the Multiple System Atrophy Pipeline? @ Multiple System Atrophy Pipeline Outlook Report

Key Takeaways from the Multiple System Atrophy Pipeline Report

• On May 01, 2026- AstraZeneca initiated a phase I/II study will evaluate the safety, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary efficacy of AZD9750 as monotherapy and in combination with saruparib in participants with metastatic prostate cancer. Additional combinations with other anticancer agents may be added via protocol amendment as separate modules. The study follows a modular design, allowing initial assessment of safety, tolerability, and preliminary efficacy across multiple treatment arms. Each Module has 2 parts: Part A (monotherapy dose escalation or combination dose finding) and Part B (monotherapy dose optimization and expansion or combination dose expansion). Treatment continues until disease progression, unacceptable toxicity, withdrawal of consent, or other reasons to discontinue study intervention occur.
• On April 30, 2026- Precision BioSciences Inc. conducted a Phase 1/2a trial is to evaluate the safety, tolerability, and preliminary efficacy of PBGENE-DMD in patients with DMD harboring mutations amenable to excision of exons 45-55. Given the limitations of existing therapeutic strategies, PBGENE-DMD represents a novel, innovative approach with the potential for a one-time, durable correction of the underlying genetic defect in the largest molecular subset of patients with DMD.
• On April 29, 2026- Dasher Neuroscience Inc. initiated a Phase 2, double-blind, placebo-controlled, multi-center, Phase II, dose escalation study to evaluate the safety, tolerability, pharmacokinetics and efficacy of Ya-101 in subjects with multiple system atrophy.
• On April 28, 2026- PepGen Inc. announced a phase 2 study is to learn about the effects of an investigational medicine, PGN-EDODM1, to see how safe and tolerable multiple administrations of PGN-EDODM1 are for people with myotonic dystrophy type 1 (DM1) compared to 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 Cytora Ltd., Alterity Therapeutics, H. Lundbeck A/S, AskBio, Corestemchemon, Inc., Tiziana Life Sciences Ltd., Kainos Medicine Inc., Ono Pharmaceutical Co. Ltd., Teva Pharmaceutical Industries Ltd., ProMIS Neurosciences, Inc., Ionis Pharmaceuticals, Inc., and Yoda Therapeutics Inc. andothers.
• Promising Multiple System Atrophy Therapies such as TEV-56286, YA-101, Foralumab Nasal, Verdiperstat, TAK-341, ATH434, Safinamide Methanesulfonate, Rasagiline Mesylate, ONO-2808, AZD3241, and others.

Want to know which companies are leading innovation in Multiple System Atrophy? @ Multiple System Atrophy Clinical Trials Assessment

The Multiple System Atrophy Pipeline Report provides disease overview, pipeline scenario and therapeutic assessment of the key pipeline therapies in this domain. The Multiple System Atrophy Pipeline Report also highlights the unmet needs with respect to the Multiple System Atrophy.

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.

Multiple System Atrophy Emerging Drugs Profile

• Amlenetug: H. Lundbeck A/S

Amlenetug (also known as Lu AF82422), developed by H. Lundbeck A/S, is an investigational human monoclonal antibody designed to target neurodegenerative processes in multiple system atrophy. The drug specifically binds to extracellular α-synuclein, a pathological protein implicated in MSA, with the aim of preventing its uptake into cells and inhibiting the seeding and spread of toxic protein aggregates across the brain, additionally, through its active Fc region, it may enhance immune-mediated clearance of α-synuclein via microglial activity, thereby potentially slowing disease progression. The drug has received several important designations, including FDA Fast Track designation (2025), Orphan Drug Designation in the US, EU, and Japan, and SAKIGAKE designation in Japan, highlighting its potential to address a significant unmet medical need. Currently, amlenetug is in Phase III clinical development, being evaluated in the global MASCOT trial (NCT06706622), a randomized, double-blind, placebo-controlled study assessing its efficacy, safety, and tolerability in patients with MSA, with dosing administered via intravenous infusion approximately every four weeks.

• ATH434: Alterity Therapeutics

ATH434, developed by Alterity Therapeutics, is an investigational oral small-molecule therapeutic designed to treat neurodegenerative diseases such as multiple system atrophy (MSA). The drug acts as an iron chaperone, targeting pathological processes by redistributing excess iron in the brain and inhibiting α-synuclein aggregation, which is a key driver of neurodegeneration in MSA, ATH434 aims to protect neurons and slow disease progression. ATH434 is being developed primarily for MSA, a rare and rapidly progressive neurodegenerative disorder with significant unmet medical need. The drug has received Orphan Drug Designation from the U.S. FDA and the European Commission, as well as FDA Fast Track designation, underscoring its potential clinical importance. Clinically, ATH434 has completed Phase II trials (ATH434-201/202), and based on positive data, the program is advancing toward Phase III development, positioning it as a late-stage asset in Alterity’s pipeline.

• hOMSC300: Cytora Ltd.

hOMSC300, developed by Cytora Ltd., is an investigational allogeneic, off-the-shelf cell therapy, a unique stem cell population derived from the neural crest with properties of neural, mesenchymal, and regenerative cells. These cells exhibit a dual mechanism of action involving cell replacement and paracrine trophic effects, enabling neuroprotection, regeneration, and modulation of neuroinflammation, which are key pathological processes in neurodegenerative diseases. The therapy is being developed for multiple system atrophy (MSA), a rare and progressive neurodegenerative disorder affecting autonomic and motor functions, and aims to act as a disease-modifying treatment by slowing disease progression and preserving neuronal function. From a clinical development perspective, Cytora’s pipeline clearly indicates that hOMSC300 is currently in Phase I clinical studies for MSA, with ongoing trials evaluating safety and preliminary efficacy following intrathecal administration.

• AB-1005: AskBio

AB-1005 is an investigational gene therapy developed by AskBio, a wholly owned subsidiary of Bayer AG. The therapy is based on an adeno-associated viral vector serotype 2 (AAV2) designed to deliver the gene encoding glial cell line-derived neurotrophic factor (GDNF) directly into the brain. Mechanistically, AB-1005 enables sustained local production of GDNF within targeted brain regions, which is intended to support the survival and function of dopaminergic neurons and enhance dopamine signaling, thereby addressing the underlying neurodegeneration. The therapy is currently being investigated in the Phase I stage of its development for the treatment of Multiple System Atrophy – parkinsonian type (MSA-P).

If you’re tracking ongoing Multiple System Atrophy Clinical trials, this press release is a must-read @ Multiple System Atrophy Treatment Drugs

The Multiple System Atrophy Pipeline report provides insights into:-

• The report provides detailed insights about companies that are developing therapies for the treatment of Multiple System Atrophy with aggregate therapies developed by each company for the same.
• It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Multiple System Atrophy Treatment.
• Multiple System Atrophy Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
• Multiple System Atrophy Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
• Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Multiple System Atrophy market.

Multiple System Atrophy Companies

Cytora Ltd., Alterity Therapeutics, H. Lundbeck A/S, AskBio, Corestemchemon, Inc., Tiziana Life Sciences Ltd., Kainos Medicine Inc., Ono Pharmaceutical Co. Ltd., Teva Pharmaceutical Industries Ltd., ProMIS Neurosciences, Inc., Ionis Pharmaceuticals, Inc., and Yoda Therapeutics Inc. andothers.

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,

• Intra-articular
• Intraocular
• Intrathecal
• Intravenous
• Ophthalmic
• Oral
• Parenteral
• Subcutaneous
• Topical
• Transdermal

Multiple System Atrophy Products have been categorized under various Molecule types such as,

• Oligonucleotide
• Peptide
• Small molecule

From emerging drug candidates to competitive intelligence, the Multiple System Atrophy Pipeline Report @ Multiple System Atrophy Market Drivers and Barriers, and Future Perspectives

Scope of the Multiple System Atrophy Pipeline Report

• Coverage- Global
• Multiple System Atrophy Companies- Cytora Ltd., Alterity Therapeutics, H. Lundbeck A/S, AskBio, Corestemchemon, Inc., Tiziana Life Sciences Ltd., Kainos Medicine Inc., Ono Pharmaceutical Co. Ltd., Teva Pharmaceutical Industries Ltd., ProMIS Neurosciences, Inc., Ionis Pharmaceuticals, Inc., and Yoda Therapeutics Inc. and others.
• Multiple System Atrophy Therapies- TEV-56286, YA-101, Foralumab Nasal, Verdiperstat, TAK-341, ATH434, Safinamide Methanesulfonate, Rasagiline Mesylate, ONO-2808, AZD3241, 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

Stay ahead in Healthcare Research – discover what’s next for the Multiple System Atrophy Treatment landscape in this detailed analysis @ Multiple System Atrophy Emerging Drugs and Major Players

Table of Contents

1. Introduction
2. Executive Summary
3. Multiple System Atrophy: Overview
4. Pipeline Therapeutics
5. Therapeutic Assessment
6. Multiple System Atrophy– DelveInsight’s Analytical Perspective
7. Late Stage Products (Preregistration)
8. NASP: Swedish Orphan Biovitrum A
9. Drug profiles in the detailed report…..
10. Mid Stage Products (Phase II/III)
11. ABP-671: Jiangsu Atom Bioscience and Pharmaceutical Co., Ltd.
12. Drug profiles in the detailed report…..
13. Early Stage Products (Phase I)
14. PRX-115: Protalix Biotherapeutics Inc.
15. Drug profiles in the detailed report…..
16. Preclinical and Discovery Stage Products
17. Drug Name: Company Name
18. Drug profiles in the detailed report…..
19. Inactive Products
20. Multiple System Atrophy Key Companies
21. Multiple System Atrophy Key Products
22. Multiple System Atrophy- Unmet Needs
23. Multiple System Atrophy- Market Drivers and Barriers
24. Multiple System Atrophy- Future Perspectives and Conclusion
25. Multiple System Atrophy Analyst Views
26. Multiple System Atrophy Key Companies
27. Appendix

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