Leuven Belgium, 31 August 2022: 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.
About Huntington’s Disease
Huntington's Disease (HD) is a rare monogenic disease that causes the progressive breakdown of nerve cells in the brain by mutant huntingtin. HD is typically inherited from an affected parent, who carries a mutation in the huntingtin gene (HTT). However, up to 10% of cases are due to a new mutation. This genetic basis was discovered in 1993. The disease affects about 4 to 15 in 100,000 people. The earliest symptoms are often subtle problems with mood or mental abilities. A general lack of coordination and an unsteady gait often follow. As the disease advances, uncoordinated, involuntary body movements of chorea become more apparent. Physical abilities gradually worsen until coordinated movement becomes difficult and the person is unable to talk. Mental abilities generally decline into dementia. Symptoms usually begin between 30 and 50 years of age, with a life expectancy of 15–20 years from when the disease is first detected.
No cure or effective treatments for HD are available, and full-time care is required in the later stages. Current treatments can relieve at best some symptoms.
reMYND is a clinical stage company developing novel treatments for Alzheimer’s, Huntington’s, epilepsy, ALS and other neurodegenerative diseases caused by neuronal dysfunction. It is backed by a proprietary drug discovery platform, which enables the identification of novel mechanisms-of-action, targets and first-in-class small molecules.
reMYND’s most advanced program is ReS19-T, an investigational compound for the treatment of Alzheimer’s and is entering Phase 2a. Similarly, reMYND’s Huntington program fully restores cortico-striatal transmission and regenerates the mouse brain in the standard Huntington knock-in mice. This program is 12-18 months pre-IND.
In addition, reMYND has a dedicated Contract Research Organization (CRO), which focuses on CNS disorders. The team helps clients to assess the pharmacokinetics, pharmacodynamics and efficacy of their experimental treatments in reMYND’s proprietary animal models. The CRO has a global client base, including the US, Europe and Japan.
reMYND was founded in 2002 as a spin-off from the University of Leuven, and has been substantially supported by grants from VLAIO/IWT (Flanders, Belgium). Find out more at https://www.remynd.com.