Update on Selection of Potential Drug Candidates May, 27 2026

(AI-Unbiased drug screening & known neurotransmitter modulators)

MAODF is pursuing multiple approaches to identify potential therapies for MAOD, including AI-driven unbiased drug screening and evaluation of known neurotransmitter-regulating compounds.

In collaboration with Unravel Biosciences, approximately 40,000 compounds were screened using their proprietary BioNAV platform, which analyzes RNA sequence (RNAseq) profiles to identify drugs capable of reversing disease-associated molecular signatures. The analysis compared RNAseq profiles from normal parents with MAOD-A/B patients. They also analyzed RNAseq profiles of brain tissues from WT and MAOD knockout (KO) mice in addition to testing RNAseq from human brain organoids treated with MAOD gene therapy and Fenclonine (pCPA).

Based on BioNAV scoring, 14 lead compounds were selected for further preclinical testing, including Withaferin-A, Albendazole, Disulfiram, and Allopurinol. Interestingly, none of these lead candidates are traditional neurotransmitter modulating drugs, but instead represent diverse classes of drugs, suggesting broader metabolic involvement in MAOD disease pathology beyond neurology. MAOD patients have demonstrated abnormal levels of certain proteins, carbohydrates
and fatty acids indicating that these compounds may improve both neurological and other symptoms.

In parallel, MAODF is evaluating several serotonin- and dopamine-regulating compounds that have shown potential symptomatic benefit in neurological disorders or within the MAOD community. These include Pimavanserin (Nuplazid), Sertraline (Zoloft), Tetrabenazine (Xenazine), para-Chlorophenylalanine (Fenclonine), Risperidone (Risperdal) and Ketanserin (Sufrexal, Serefrex).

These ongoing studies aim to identify the most promising candidates for future clinical development in MAOD patients.