PRECLINICAL STUDIES OF POTENTIAL DISEASE-MODIFYING TREATMENTS FOR MSA

A Phase 1 clinical trial in MSA patients was previously completed.

For historical Phase 1 clinical trial details See: https://www.clinicaltrials.gov/ct2/show/NCT02270489

Two vaccines aimed at preventing the buildup of the alpha-synuclein protein are under development.

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September 2020: Targeting α-synuclein by PD03 AFFITOPE® and Anle138b rescues neurodegenerative pathology in a model of multiple system atrophy: clinical relevance

September 2020: A Phase 1 Randomized Trial of Specific Active α‐Synuclein Immunotherapies PD01A and PD03A in Multiple System Atrophy

October 2018: Specific active immunotherapy (SAIT) against alpha-synuclein with AFFITOPE® PD01A and PD03A: Results from the AFF009 phase I trial

March 2018: AFFiRiS Announces Results of a Phase I Clinical Study Using AFFITOPEs® PD01A and PD03A, Confirming Safety and Tolerability for Both Compounds as well as Immunogenicity for PD01A in Early MSA patients

September 2016: Boost Vaccination Data Encourage Continued Development of AFFiRiS Therapeutic Parkinson’s Disease Vaccine Against Alpha-Synuclein

NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.

A Phase 1 clinical trial in healthy volunteers was previously completed. See: https://www.clinicaltrials.gov/ct2/show/NCT03022799

KM-819 is a new drug candidate targeting FAS-associated factor 1, which inhibits the death of dopamine-secreting cells, thereby stopping the progression or slowing the progress of the disease. KM-819 has been shown to inhibit the accumulation of alpha-synuclein.

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NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.

The drug company has filed a request with the FDA requesting orphan drug designation for Alpha-SYN SM in the treatment of MSA.

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NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.

MPLA is a clinically approved vaccine adjuvant. It is being tested in MSA experimental animal models in Austria.

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NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.

Belnacasan is currently in Phase 2 clinical trials for Epilepsy and is also being tested in MSA experimental models in the lab by a collaborative research consortium in Austria, France and Germany.

See: ARTEMIS: Targeting Alpha-Synuclein for Treating Multiple System Atrophy

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August 2016: Reducing C-terminal truncation mitigates synucleinopathy and neurodegeneration in a transgenic model of multiple system atrophy

NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.

YTX-7739 has been shown in experimental lab studies to inhibit many of the key aspects of alpha-synuclein toxicity.

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NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.

NaPB provided GCI reduction, neuroprotection and motor improvement in experimental MSA mouse models when therapy started at the time of motor symptom onset.

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October 2016: Neuroprotection by Epigenetic Modulation in a Transgenic Model of Multiple System Atrophy

NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.

Benztropine provided remyelination, neuroprotection and motor improvement in experimental mouse models of MSA.

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July 2016: α-Synuclein-induced myelination deficit defines a novel interventional target for multiple system atrophy.

NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.

A-443654 is an inhibitor of the multiple functional kinase AKT. A-443654 and AKT inhibition may be a potential strategy for reducing SNCA expression and treating Parkinson’s disease pathology.

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September 2021: The AKT modulator A-443654 reduces α-synuclein expression and normalizes ER stress and autophagy

NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.

In January 2020, the Company initiated a first-in-human Phase 1 trial evaluating its second-generation pipeline compound, SBT-272, in healthy subjects. The Company anticipates that preclinical data expected later this year in models of amyotrophic lateral sclerosis (ALS) and multiple system atrophy (MSA) will help inform SBT-272’s clinical development pathway for rare neurodegenerative diseases.

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NOTE: All of the above information is intended to assist MSA patients and their families in having a conversation with doctors, none of it should be considered medical advice or endorsements of any particular drugs or therapies. Always consult a licensed medical practitioner for expert care.