Galimedix Therapeutics, Inc. has dosed the first patients in its phase 1 study evaluating the safety, tolerability, and pharmacokinetics of GAL-101, a neuroprotective therapy, for the potential treatment of three neurodegenerative diseases.
First, this company.
Based in Kensington, Maryland, the phase 2 clinical-stage biotechnology company is developing novel first-in-class drugs intended to stop or slow the progression of neurodegenerative disease as well as improve function in:
- Glaucoma
- Dry age-related macular degeneration (AMD)
- Alzheimer’s disease
Explain the basis for its scientific approach.
First and foremost: Clinical research has identified accumulations of toxic amyloid beta (Aβ) oligomers and protofibril as an underlying cause for retina-based neurodegenerative diseases in the eye (including those three mentioned above).
In the case of normal Aβ monomers: These are key to neuronal function; however, when misfolded (in other words, nonfunctional and resistant to breakdown), these monomers can result in the development of toxic forms of Aβ that damage the retina and brain cells.
- See here for a visual of how this works.
Which brings us to Galimedix’s technology?
Indeed. The company is developing a product candidate category of “orally and topically-administered Aβ aggregation modulators” that target the beginning of this Aβ accumulation to prevent damage to the retina and brain cells.
Its lead asset: GAL-101.
Tell me more.
This is a novel small molecule designed to provide neuroprotection and improved visual function for retinal diseases by targeting Aβ aggregates to slow the progression of blindness associated with dry AMD and glaucoma.
- To note: GAL-101 is under clinical development in both oral and topical (eye drop) formulations.
Plus, while we’re on the subject: Back in March 2023, Galimedix and Théa Open Innovation agreed to a licensing agreement for the development and commercialization of GAL-101.
Any clinical data on it so far?
Yes—preclinical and phase 1 included.
On the preclinical front: Galimedix reported that the GAL-101 was found to prevent and eliminate all forms of toxic Aβ species while leaving healthy Aβ forms intact.
Plus: “GAL-101 has also demonstrated the potential for neuroprotection and for symptomatic alleviation in pre-clinical models of Alzheimer’s disease,” the company stated.
- And when administered orally, the asset previously demonstrated “no antibody-specific immunological side effects.”
And beyond the pre-clinical side?
In a previous randomized, double-masked, placebo-controlled, single center phase 1 study, GAL-101 demonstrated a favorable safety profile and was well tolerated among 70 patients (40 healthy; 30 with glaucoma).
- Its administration form for this trial: topical (eye drop)
Regulatory note: That study’s protocol was accepted by the FDA to support the launch of larger clinical studies for glaucoma and dry AMD—including this new phase 1 study.
Tell me about this trial.
The phase 1 study is designed to evaluate the safety, tolerability, and pharmacokinetics of GAL-101, administered orally as a single-ascending dose (SAD) and multiple-ascending doses (MAD) of GAL-101.
About the participants: Up to 120 (in total):
- (Up to) 40 healthy volunteers in the SAD part
- 32 patients in the MAD part
Study details: The study will also evaluate GAL-101’s ability to cross the blood-brain barrier as well as other parameters (which will include an unspecified number of participants).
Alrighty, so when can we expect data from this?
With initiation only just started, it’s difficult to give an exact timeframe—patient enrollment will likely continue into the new year.
Mmm. Any other clinical updates to know about, then?
Yes! Galimedix also announced recruitment is expected to start soon for a double-masked, randomized, multicenter, parallel group, placebo-controlled, pivotal phase 2 eDREAM study (NCT06659549) of GAL-101 for dry AMD.
In this trial: GAL-101 will be administered topically (as an eye drop) among an estimated 110 participants (aged 55+) diagnosed with non-foveal geographic atrophy (GA) secondary to dry AMD.
Its estimated conclusion: May 2027.