Scilex Bio, a Controlling Interest of Joint Venture by Scilex Holding Company, Reports KDS2010 a Novel Oral Tablet Phase 2 Trial for Alzheimer’s Disease (AD) Currently Enrolling with U.S. Patient Cohort to be Added in 2025
SCLX 12.11.2024

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- Currently enrolling randomized, double-blind, placebo-controlled, dose-finding, Phase 2 clinical trial to evaluate the safety and efficacy of KDS2010 in patients with Alzheimer’s disease with mild cognitive impairment and mild dementia is currently recruiting in
South Korea in 114 patients andU.S. cohort will be added in 2025. - KDS2010 (Tisolagiline) is a new reversible selective monoamine oxidase B (MAO-B) inhibitor being studied for its potential in treating neurodegenerative diseases like Alzheimer's disease.
- Apart from its role as a MAO-B inhibitor, it also influences astrocytic GABA inhibition.
- In neurodegenerative diseases like Alzheimer’s, altered GABAergic activity and dysfunction in astrocytic regulation of neurotransmitter systems contribute to cognitive decline and neuroinflammation.
- A highly selective and reversible MAO-B inhibitor, KDS2010 overcomes the disadvantages of the irreversible MAO-B inhibition.
- Long-term treatment with KDS2010 significantly attenuates increased astrocytic GABA levels and astrogliosis, enhances synaptic transmission, and improves learning and memory in APP/PS1 mice.1
- KDS2010 pharmacokinetics, lack of food effect, safety and dose selection have been characterized in Single Ascending Dose and Multiple Ascending Dose Phase 1 clinical trials with 88 healthy young adults and elderly subjects and between Korean and Western population, demonstrating favorable safety and tolerability and adequate pharmacokinetics for once-daily dosing.
- Per Health Care analyst reports 2023, Alzheimer’s global drug market size is expected to rise above
$15 billion by 2030 in the eight major markets, as new drugs show promise and are being launched with FDA approval to slow cognitive decline.2
“Current treatments in development for Alzheimer’s Disease are mostly injectable antibodies or peptides targeting amyloid, tau or neuroinflammation. Advancement of a small molecule with great potential to improve cognitive function that can be delivered with once-daily oral dosing holds promise for people living with Alzheimer's disease,” said
GABA (gamma-aminobutyric acid) is the primary inhibitory neurotransmitter in the brain, playing a key role in regulating neuronal activity and maintaining balance between excitation and inhibition. Astrocytes, a type of glial cell in the brain, have been shown to be involved in the regulation of GABA activity. In neurodegenerative diseases like Alzheimer’s, altered GABAergic activity and dysfunction in astrocytic regulation of neurotransmitter systems contribute to cognitive decline and neuroinflammation. By inhibiting astrocytic GABA signaling, KDS2010 helps to reduce neuroinflammation and normalize the balance between excitatory and inhibitory signals in the brain, potentially leading to enhancing cognitive function in Alzheimer's disease.
Mechanistically, reactive astrocytes precipitate pathological hallmarks of Alzheimer’s disease via hydrogen peroxide (H2O2) production. KDS2010 blocks MAO-B-dependent aberrant GABA/ H2O2production in reactive astrocytes and eliminates neuronal inhibition, neuroinflammation, and neurodegeneration, while enhancing neuroregeneration.3
In the dentate gyrus of mouse models of AD, the released GABA reduces spike probability of granule cells by acting on presynaptic GABA receptors. Suppressing GABA production in reactive astrocytes restores the impaired spike probability, synaptic plasticity, and learning and memory in mice. In the postmortem brain of individuals with AD, astrocytic GABA and MAO-B are significantly upregulated. Selective inhibition of astrocytic GABA synthesis and release is a new therapeutic strategy for treating memory impairment in AD.4
Clinically, short-term treatment with known irreversible MAO-B inhibitors, such as selegiline, improves cognitive deficits in AD patients, long-term treatments have shown disappointing results. Interestingly, prolonged treatment with selegiline fails to reduce aberrant astrocytic GABA levels and rescue memory impairment in APP/PS1 mice, an animal model of AD, because of increased activity in compensatory genes for a GABA-synthesizing enzyme, diamine oxidase (DAO). A highly selective and reversible MAO-B inhibitor, KDS2010 overcomes the disadvantages of the irreversible MAO-B inhibition. Long-term treatment with KDS2010 significantly attenuates increased astrocytic GABA levels and astrogliosis, enhances synaptic transmission, and improves learning and memory in APP/PS1 mice.1
KDS2010 pharmacokinetics, lack of food effect, safety and dose selection have been characterized in Single Ascending Dose and Multiple Ascending Dose Phase 1 clinical trials with 88 healthy young adults and elderly subjects and between Korean and Western populations, demonstrating favorable safety and tolerability and adequate pharmacokinetics for once-daily dosing. A Randomized, Double-Blind, Placebo-Controlled, Dose-Finding, Phase 2 Clinical Trial to Evaluate the Safety and Efficacy of KDS2010 in Patients with Alzheimer’s Disease with Mild Cognitive Impairment and Mild Dementia is currently recruiting in
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IPMC is a private biopharmaceutical company focused on the development of new medicines for the treatment of cardiometabolic and neurodegenerative diseases.
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References
Jong-Hyun Park , et al. Science Advances. 2019 Mar 20;5(3):eaav0316- www.ihealthcareanalyst.com/global-alzheimers-disease-market/
Heejung Chun , et al. Nature Neuroscience 2020 Dec;23(12):1555-1566Seonmi Jo , et al. Nature Medicine. 2014 August ; 20(8): 886–896. doi:10.1038/nm.3639
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