Below is MSA pharmaceutical clinical testing trial info, sent to a friend dealing with MSA. Hope it helps all.
Current pharmaceutical clinical testing for Multiple System Atrophy (MSA) is heavily focused on disease-modifying therapies, specifically targeting the reduction of
-synuclein protein aggregation, which is believed to drive disease progression. As of early 2026, the field has seen several key developments, including Phase 2/3 trials for investigational drugs and FDA Fast Track designations.
National Institutes of Health (.gov)National Institutes of Health (.gov) +4
Here are the key ongoing and recent clinical trials and therapeutic candidates:
Top Clinical Trial Candidates (2025–2026)
Amlenetug (Lu AF82422 / Lundbeck): This is a human monoclonal antibody designed to bind to
-synuclein and inhibit its aggregation.
Status: A Phase 3 trial, MASCOT (NCT06706622), was initiated in late 2024/early 2025 following positive results from the Phase 2 AMULET trial. It was granted FDA Fast Track designation in February 2025.
Emrusolmin (TEV-56286 / MODAG / Teva): A small molecule designed to target
-synuclein oligomers.
Status: In Phase 2 development (TOPAS-MSA study). It was granted FDA Fast Track and Orphan Drug designation in September 2025.
ATH434 (Alterity Therapeutics): An oral iron-targeting agent designed to reduce iron-induced
-synuclein aggregation in the brain.
Status: Reported positive Phase 2 results in early 2025, demonstrating reduced brain atrophy.
AB-1005 (AskBio / Bayer): An investigational gene therapy (AAV2-GDNF) delivered to the brain to promote the survival of dopaminergic neurons.
Status: Completed enrollment for the Phase 1 REGENERATE MSA-101 trial in September 2025.
Ampreloxetine (Theravance Biopharma): A norepinephrine reuptake inhibitor focusing on symptomatic treatment of neurogenic orthostatic hypotension (nOH) in MSA.
Status: Active, but reported in March 2026 that the Phase 3 CYPRESS study did not meet its primary endpoint.
ION464 (Ionis Pharmaceuticals): An antisense oligonucleotide (ASO) designed to prevent the production of
-synuclein protein.
AskBioAskBio +10
Key Areas of Investigation
-Synuclein Reduction: Antibodies (amlenetug) or small molecules (emrusolmin) to clear toxic protein buildup.
Neuroprotection/Iron Regulation: Agents like ATH434 to prevent brain atrophy.
Gene Therapy: Using GDNF to protect cells.
Symptomatic Management: Treating debilitating orthostatic hypotension (e.g., ampreloxetine).
Natural History Studies: Studies such as TRACK-MSA (NCT04450992) are ongoing to identify better outcome measures for future trials.
NYU Grossman School of MedicineNYU Grossman School of Medicine +3
Recent Trial Outcomes
Sirolimus: A study supported by the NIH was ended early after interim analysis indicated it was not effective in slowing MSA progression.
Trial Challenges: The rapid progression of MSA makes trial recruitment and retention challenging, with a high failure rate for neuroprotective compounds.
National Institutes of Health (.gov)National Institutes of Health (.gov) +3
Regards,
Sagan
@shelleystolfi
I'm so sorry to hear of Joe's death. I know from your previous posts, that you both worked really hard to find help.
If you are so inclined, I would like to invite you to post about your grief on Connect's Loss and Grief support group. Here is a link to those discussions: https://connect.mayoclinic.org/group/loss-grief/
Would you be willing to share a few ideas for those who are facing life with MSA?
Of course I would invite anyone with this disease to share or ask questions about MSA. I learned a lot about it as we didn't have much help here in Maine. I found my Joe had adverse reactions to medications, as do I. As an example, for swelling, I would feed him pineapple.
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Below is MSA pharmaceutical clinical testing trial info, sent to a friend dealing with MSA. Hope it helps all.
Current pharmaceutical clinical testing for Multiple System Atrophy (MSA) is heavily focused on disease-modifying therapies, specifically targeting the reduction of
-synuclein protein aggregation, which is believed to drive disease progression. As of early 2026, the field has seen several key developments, including Phase 2/3 trials for investigational drugs and FDA Fast Track designations.
National Institutes of Health (.gov)National Institutes of Health (.gov) +4
Here are the key ongoing and recent clinical trials and therapeutic candidates:
Top Clinical Trial Candidates (2025–2026)
Amlenetug (Lu AF82422 / Lundbeck): This is a human monoclonal antibody designed to bind to
-synuclein and inhibit its aggregation.
Status: A Phase 3 trial, MASCOT (NCT06706622), was initiated in late 2024/early 2025 following positive results from the Phase 2 AMULET trial. It was granted FDA Fast Track designation in February 2025.
Emrusolmin (TEV-56286 / MODAG / Teva): A small molecule designed to target
-synuclein oligomers.
Status: In Phase 2 development (TOPAS-MSA study). It was granted FDA Fast Track and Orphan Drug designation in September 2025.
ATH434 (Alterity Therapeutics): An oral iron-targeting agent designed to reduce iron-induced
-synuclein aggregation in the brain.
Status: Reported positive Phase 2 results in early 2025, demonstrating reduced brain atrophy.
AB-1005 (AskBio / Bayer): An investigational gene therapy (AAV2-GDNF) delivered to the brain to promote the survival of dopaminergic neurons.
Status: Completed enrollment for the Phase 1 REGENERATE MSA-101 trial in September 2025.
Ampreloxetine (Theravance Biopharma): A norepinephrine reuptake inhibitor focusing on symptomatic treatment of neurogenic orthostatic hypotension (nOH) in MSA.
Status: Active, but reported in March 2026 that the Phase 3 CYPRESS study did not meet its primary endpoint.
ION464 (Ionis Pharmaceuticals): An antisense oligonucleotide (ASO) designed to prevent the production of
-synuclein protein.
AskBioAskBio +10
Key Areas of Investigation
-Synuclein Reduction: Antibodies (amlenetug) or small molecules (emrusolmin) to clear toxic protein buildup.
Neuroprotection/Iron Regulation: Agents like ATH434 to prevent brain atrophy.
Gene Therapy: Using GDNF to protect cells.
Symptomatic Management: Treating debilitating orthostatic hypotension (e.g., ampreloxetine).
Natural History Studies: Studies such as TRACK-MSA (NCT04450992) are ongoing to identify better outcome measures for future trials.
NYU Grossman School of MedicineNYU Grossman School of Medicine +3
Recent Trial Outcomes
Sirolimus: A study supported by the NIH was ended early after interim analysis indicated it was not effective in slowing MSA progression.
Trial Challenges: The rapid progression of MSA makes trial recruitment and retention challenging, with a high failure rate for neuroprotective compounds.
National Institutes of Health (.gov)National Institutes of Health (.gov) +3
Regards,
Sagan
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Of course I would invite anyone with this disease to share or ask questions about MSA. I learned a lot about it as we didn't have much help here in Maine. I found my Joe had adverse reactions to medications, as do I. As an example, for swelling, I would feed him pineapple.
-
Like -
Helpful -
Hug
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