Galimedix finishes Phase 1 single dose trial of GAL-101, an amyloid beta modulator

Galimedix Therapeutics, Inc., a biotech firm in the Phase 2 clinical stage, has reported successful results from the single ascending dose (SAD) portion of its Phase 1 clinical trial for GAL-101. This novel small molecule is designed to address misfolded amyloid beta (Aβ) monomers, which are implicated in neurodegenerative diseases. GAL-101, developed for oral administration, was tested on 40 healthy volunteers who were randomized in a 3:1 ratio to receive either the GAL-101 capsules or a placebo. The results demonstrated that GAL-101 is well tolerated, showcasing a favorable safety and pharmacokinetic profile that supports its oral formulation.

Dr. Hermann Russ, Co-founder and Chief Scientific Officer of Galimedix, emphasized the significance of these findings as a critical advancement in their clinical development. The data corroborate GAL-101's safety and efficacy, reinforcing its potential as an oral treatment for central nervous system (CNS) and eye diseases related to amyloid beta, including Alzheimer's disease. The company is progressing to subsequent stages of the study, with completion anticipated later in the year.

The ongoing Phase 1 trial is set to involve up to 120 participants. Its design covers essential elements needed to commence Phase 2 trials for Galimedix’s primary targets: Alzheimer's disease, dry age-related macular degeneration (AMD), and glaucoma. Concurrently, a Phase 2 clinical trial named eDREAM is underway to evaluate GAL-101 eyedrops for dry AMD, with recruitment expected to conclude within a year.

GAL-101 functions by targeting misfolded Aβ monomers, thus preventing the development of toxic Aβ oligomers and protofibrils. The molecule is available in oral and topical forms, and its focus on Aβ monomers is supported by recent approvals of anti-Aβ drugs in Alzheimer’s treatment. Previous Phase 1 studies with GAL-101 eyedrops have shown strong safety and tolerability. Additionally, preclinical tests indicate that GAL-101 can neutralize all toxic Aβ variants while preserving healthy Aβ forms, suggesting potential neuroprotective benefits and symptom relief in Alzheimer's models.

The oral form of GAL-101 has demonstrated minimal immunological side effects, such as ARIA, low systemic toxicity, stability in storage, and cost-effective manufacturing. Preclinical ophthalmic models have shown significant efficacy in safeguarding retinal neurons from toxic damage. An ongoing Phase 2 study (NCT06659549) is further investigating the impacts of GAL-101 eyedrops in dry AMD.

Galimedix, a private company at the forefront of Phase 2 clinical research, is committed to pioneering oral and topical neuroprotective treatments for severe eye and brain disorders. It was established by a team of seasoned bio-entrepreneurs, pharmaceutical leaders, and scientists. The company’s innovative molecules target diseases influenced by amyloid beta, with an initial focus on dry AMD, glaucoma, and Alzheimer’s disease. Galimedix aims to transform disease management in these areas, offering hope for substantial therapeutic advancements.