Galimedix Therapeutics initiates Phase 1 trial of oral GAL-101

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Galimedix Therapeutics Inc. announced the initiation of dosing in its phase 1 clinical trial evaluating oral GAL-101, an amyloid beta (Aβ) aggregation modulator. This trial is designed to assess the safety, tolerability, and pharmacokinetics of single and multiple ascending doses of the orally administered therapy.

The phase 1 study aims to enroll up to 40 healthy volunteers in the single ascending dose (SAD) cohort and 32 participants in the multiple ascending dose (MAD) cohort. The trial will also investigate GAL-101’s ability to cross the blood-brain barrier, among other parameters. Overall, up to 120 participants are expected to be enrolled.¹

Alexander Gebauer, MD, PhD, co-founder and executive chairman of Galimedix Therapeutics, highlighted the compound's promising safety profile and preclinical efficacy.

“GAL-101 eyedrops have already demonstrated excellent safety and tolerability in early clinical testing, as well as compelling efficacy in relevant ophthalmic and Alzheimer’s preclinical models,” he said. “We look forward to the initial results of this Phase 1 trial, which will guide the development of our oral formulation for Alzheimer’s disease and inform future studies in dry age-related macular degeneration (AMD) and glaucoma.”

About GAL-101

GAL-101 is a small molecule designed to target misfolded Aβ monomers, thereby preventing the formation of toxic Aβ oligomers and protofibrils. The molecule is being developed in both oral and topical (eyedrop) formulations for the treatment of dry AMD, glaucoma, and Alzheimer’s disease.

Studies have implicated toxic Aβ aggregates as a major underlying cause of neurodegenerative diseases of the eye, and recent approvals of anti-Aβ drugs have validated Aβ as a key target in Alzheimer’s disease.

Preclinical and early clinical results

In prior Phase 1 studies, GAL-101 eyedrops exhibited an excellent safety and tolerability profile. Preclinical research has demonstrated GAL-101’s ability to prevent and eliminate toxic Aβ species while preserving healthy forms of Aβ. The compound has shown potential for neuroprotection and symptomatic alleviation in preclinical Alzheimer’s disease models.¹

Additionally, oral GAL-101 has demonstrated favorable attributes, including:

• Absence of antibody-specific immunological side effects, such as amyloid-related imaging abnormalities (ARIA).
• Minimal systemic toxicity.
• Robust storage stability.
• Ease of manufacturing at a low cost.

Preclinical ophthalmic studies have further demonstrated GAL-101’s efficacy in protecting neuronal retinal cells from toxic damage.

Recruitment for a pivotal Phase 2 study (NCT06659549) evaluating GAL-101 eyedrops in dry AMD is anticipated to begin soon.

Reference:

1. Galimedix Therapeutics initiates Phase 1 study with oral small molecule, GAL-101, an amyloid beta (Aβ) aggregation modulator – Galimedix Therapeutics. Galimedix.com. Published 2024. Accessed December 5, 2024. https://galimedix.com/article/galimedix-therapeutics-initiates-phase-1-study-with-oral-small-molecule-gal-101-an-amyloid-beta-a