reMYND identifies novel target and small molecules with potential to halt and reverse disease progression in Huntington’s disease through oral administration
reMYND NV, a clinical stage company tackling neurodegeneration, today announces positive preclinical results from its drug discovery program ReS18-H to treat Huntington’s Disease (HD), a rare monogenic disease that causes the progressive breakdown of nerve cells in the brain by neurotoxic mutant huntingtin, leading to movement, cognitive and psychiatric dysfunction.
reMYND has discovered a novel drug target and small molecule modulators counteracting the formation of HD pathophysiology and subsequent neurodegeneration. Oral administration of ReS18-H compounds in the gold-standard mutant huntingtin mouse model has shown to regenerate brain mass and nerve outgrowth, re-functionalize communication between brain areas (cortex and striatum) and reverse symptoms such as hypokinesia and anxiety. Similar effectiveness was also demonstrated in patient-derived iPSC neurons to ensure clinical translatability. This concept represents a novel way of treating Huntington’s with the potential to halt disease progression and reverse disease effects through a straightforward oral application.
The drug candidates selected, which have a potency of less than 1 nM, have shown to be well-tolerated in non-clinical models and have excellent brain exposure. reMYND is gearing up the program for pre-clinical GLP toxicology studies. This discovery program has been made possible through grants from Flanders Innovation & Entrepreneurship (VLAIO).
Gerard Griffioen, Chief Scientific Officer of reMYND commented: “These early data suggest that our Huntington’s program has the potential to not only halt disease progression, but also reverse disease effects. Our next step will be to leverage the expertise and network acquired through our Alzheimer’s program to assess the translatability of the animal data from our Huntington’s program to patients in clinical studies in the years to come.”
This Huntington’s program adds to reMYND’s existing neurodegeneration pipeline. The company’s ReS19-T Alzheimer’s program will shortly be entering Phase 2a. This Alzheimer’s program was discovered through the same discovery platform as the Huntington’s program. Research so far has demonstrated that this compound also combines fast symptomatic relief with disease modification albeit through a different target and with a different mechanism.