PRECLINICAL STUDIES OF POTENTIAL DISEASE-MODIFYING TREATMENTS FOR MSA

Click on each compound to learn more details about these experimental laboratory studies and what has been discovered so far.

PD01A & PD03A (alpha-synuclein vaccines) - Phase 1 Completed 2017

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.

Related Videos:

October 2019: A vaccine for Parkinson’s disease shows promising results

Related Articles:

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.

KM-819 - Phase 1 Completed 2017

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.

Related Articles:

February 2019: Kainos Medicine starts developing treatment for multiple system atrophy

January 2019: Kainos Medicine Reaches Milestone in Bringing “KM-819” Treatment for Parkinson’s Disease to Market

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.

α-SYN SM (alpha-synuclein-SMART Molecule)

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

Related Articles:

December 2017: ICBII Announces the Filing of Orphan Drug Designation Application for the Use of α-Synuclein-SMART Molecule in the Treatment of Multiple System Atrophy

May 2014: Third Party Validation of BBB Permeability and Target Specificity of SMs by Dr. Eliezer Masliah, UCSD

April 2014: Brain Imaging Tracer for Parkinson’s Disease

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 (monophosphoryl lipid A)

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

Related Articles:

July 2017: Toll-like receptor 4 stimulation with monophosphoryl lipid A ameliorates motor deficits and nigral neurodegeneration triggered by extraneuronal α-synucleinopathy.

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 (VX-765)

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

Related Articles:

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

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

Related articles:

September 2018: Yumanity Therapeutics Announces First Clinical Candidate for the Potential Treatment of Parkinson’s Disease.

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.

SYNUCLEAN-D

SynuClean-D, a small compound that inhibits alpha-synuclein aggregation, disrupts mature amyloid fibrils, prevents fibril propagation, and abolishes the degeneration of dopaminergic neurons in an animal model of Parkinson’s disease.

Related articles:

October 2018: New Compound Can Prevent Formation of Toxic Alpha-Synuclein Aggregates, Animal Study Finds

October 2018: Small molecule inhibits α-synuclein aggregation, disrupts amyloid fibrils, and prevents degeneration of dopaminergic neurons

September 2018: Molecule capable of halting and reverting neurodegeneration caused by Parkinson’s disease identified

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

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

Related Articles:

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

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

Related Articles:

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.

SBT-272

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.

Related Articles:

November 2020: Stealth BioTherapeutics Reports Third Quarter 2020 Financial Results And Recent Business Highlights

May 2020: Stealth BioTherapeutics Reports First Quarter 2020 Financial Results and Recent Business Highlights

January 2020: Stealth BioTherapeutics Initiates First-in-Human Study of SBT-272

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.

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