Maryland-based biotech Galimedix Therapeutics is ready to take a big swing on its oral Alzheimer’s disease candidate after no serious adverse events were reported and the small molecule successfully crossed the blood-brain barrier in a phase 1 trial.
The first-ever clinical trial for GAL-101 tested single and multiple ascending doses of the asset in more than 100 healthy volunteers, Galimedix reported in a Sept. 12 release. The company is now initiating fundraising for a phase 2 trial.
“Oral GAL-101 was well tolerated with a highly favorable safety profile. Additionally, the pharmacokinetic profile strongly supports the planned administration route,” Galimedix co-founder and executive chair Alexander Gebauer, M.D., Ph.D., said in the release. “GAL-101 is expected to be first-in-class and has the potential to become the future standard of care for all stages of Alzheimer’s, including mild cognitive impairment.”
Like approved Alzheimer’s meds Kisunla (donanemab) and Leqembi (lecanemab), as well as the discontinued Aduhelm (aducanemab), GAL-101 is designed to target misfolded amyloid beta protein in the brain. By securing itself to the protein, the small molecule is meant to prevent amyloid from bunching together into harmful aggregations.
However, GAL-101 differentiates itself in delivery, with the three approved amyloid beta blockers administered via IV infusions.
The prevalence of amyloid beta clumps in Alzheimer’s disease has made the protein an attractive target for new drugs for decades. But a 2021 analysis found that many trials targeting amyloid plaques failed to improve patient cognition.
“There isn't a tight correlation between amyloid levels and cognition,” Matthew Schrag, M.D., Ph.D., a neuroscientist at Vanderbilt University Medical Center, told Fierce Biotech in August when discussing the success of Denali Therapeutics’ own amyloid antibody at reducing plaques in mice.
“We put heavily mutated, huge amounts of beta amyloid into mice, and they don't get neurodegeneration,” Schrag added. “They get some symptoms, but we don't see massive amounts of neuronal loss.”
That said, back in 2009, scientists showed that the compound now known as GAL-101 was able to reduce amyloid beta and improve learning ability in a small sample of mice with Alzheimer’s disease.
Galimedix is not limiting GAL-101’s potential to neurodegenerative diseases. The company is also testing the molecule’s ability to break up amyloid plaques in the retina, as a potential topical treatment for age-related macular degeneration (AMD). The biotech launched a phase 2 study of GAL-101 in dry AMD in December 2024.