lovastatin (AMO Pharma Limited)

AMO-01 is an investigational intravenous formulation of lovastatin, a small molecule HMG-CoA reductase inhibitor, being developed by AMO Pharma in collaboration with Alexander Kolevzon at the Seaver Autism Center for Research and Treatment at Mount Sinai. It is specifically intended for the treatment of Phelan-McDermid syndrome (PMS), a rare genetic disorder caused by SHANK3 gene mutations that lead to intellectual disability, autism, and epilepsy. The therapeutic mechanism involves the modulation of the Ras-MAPK signaling pathway, which is overactive in PMS. By inhibiting HMG-CoA reductase, lovastatin reduces the production of farnesyl pyrophosphate, thereby preventing the farnesylation and membrane activation of Ras proteins. A Phase 2 clinical trial (NCT04009603) was conducted to evaluate the safety and pharmacokinetics of a single-dose AMO-01 infusion in adolescents and adults with PMS and epilepsy.

Other names

lovastatin