About Our Science.
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We are developing a transformative therapy for Parkinson’s disease (PD). Our lead candidate, buntanetap, simultaneously inhibits the production of multiple neurotoxic brain proteins that have been shown to impair axonal transport and lead to nerve cell death. It is distinct from symptomatic dopaminergic drug, because it improves function on top of dopamine. Further it is distinct from disease-modifying drugs in development, because,rather than removing one toxic protein, buntanetap inhibits all the major toxic proteins before they can form.
IRB approved Alzheimer Study Flyer: Buntanetap in Treating Alzheimer’s and Parkinson’s Disease
The data from our phase 2 study in Alzheimer’s and Parkinson’s patients was published in JPAD (Journal of Prevention of Alzheimer’s Disease) and we have attached the paper. Here is a summary of what we found and why we have progressed our drug into phase 3.
Buntanetap is an orally available small molecule that inhibits the translation of multiple neurotoxic aggregating proteins only under conditions where their translation is elevated – in sick nerve cells. Because it only affects these neurotoxic proteins when they are overexpressed, it restores their normal levels without affecting other proteins and restores homeostasis in the brain.
Both Alzheimer’s disease and Parkinson’s disease have been shown to have mixed pathology and mixed proteinopathy. All four neurotoxic aggregating proteins are present in the brain of Alzheimer and Parkinson patients – Aβ forms plaques, tau forms tangles, alpha-synuclein forms Lewy bodies, and TDP43 forms its aggregates.
Amyloid β
Alzheimer’s - Parkinson’s
Tau
Tauopathies - AD, PD, FTD, CTE
αSynuclein
Parkinson’s - Alzheimer’s
TDP43
ALS, AD, PD, FTD, CTE
Buntanetap inhibits translation of all the above four neurotoxic proteins, thus is well positioned to treat both diseases, which is an advantage compared to removing a single neurotoxic protein alone. In a double-blind, placebo-controlled, multi-center phase 2, study we enrolled 14 early AD and 54 early PD participants who were treated for 25 days with buntanetap or placebo. Buntanetap was safe and well tolerated. Biomarker data indicated a trend in lowering levels of neurotoxic proteins and inflammatory factors and improving axonal integrity and synaptic function in both AD and PD cohorts, confirming target and pathway engagement of buntanetap. Psychometric tests showed statistically significant improvements in ADAS-Cog11 and WAIS coding in AD patients and MDS-UPDRS and WAIS coding in PD patients. These findings support the hypothesis that lowering levels of neurotoxic aggregating proteins leads to stabilization and improvement in cognition and motor function.
