Galmed Develops Brain-Penetrating Aramchol Formulation for Parkinson's Disease and Synucleinopathies
核心洞察
Galmed Pharmaceuticals and Barcode Nanotech (搜索) have developed a breakthrough brain-penetrating formulation of Aramchol using lipid nanoparticles to cross the blood-brain barrier.
The new formulation targets stearoyl-CoA desaturase 1 (搜索) (SCD1 (搜索)) and demonstrated dose-dependent reduction of α-synuclein (搜索) aggregation in vitro studies without toxicity.
This represents the first potential disease-modifying therapy for Parkinson's disease (搜索) and related synucleinopathies (搜索), with Phase 1b/2 studies planned for H2 2026.
Galmed Pharmaceuticals Ltd. (NASDAQ: GLMD) announced a breakthrough development in central nervous system therapeutics with the creation of a brain-penetrating formulation of its SCD1 (搜索) inhibitor Aramchol. The new formulation, developed in collaboration with Barcode Nanotech (搜索), represents a potential first-in-class disease-modifying therapy for Parkinson's disease (搜索) and related synucleinopathies (搜索).
Overcoming the Blood-Brain Barrier Challenge
The development addresses a fundamental challenge in CNS drug development: 98% of drugs do not reach the brain due to the blood-brain barrier (BBB) that separates peripheral blood circulation from the central nervous system. The new Aramchol formulation is characterized by sequestration of the drug in lipid nanoparticles, which will be administered by subcutaneous injection for delivery across the BBB.
Barcode Nanotech (搜索)'s proprietary platform enabled simultaneous screening of hundreds of different nanoparticle formulations in vivo, coupled with AI analysis tools to select the optimal delivery vehicle to the brain. "Based on our proprietary platform and lipids library we have identified a unique formulation of Aramchol to cross the BBB and deliver the molecule to the brain," said Ronen Eavri, Co-founder & CEO of Barcode Nanotech.
Targeting α-Synuclein Aggregation
The therapeutic approach targets stearoyl-CoA desaturase 1 (搜索) (SCD1 (搜索)), which has been identified as an important therapeutic target for CNS diseases characterized by the aggregation of the protein α-synuclein (搜索) (αSyn). These diseases, collectively known as synucleinopathies (搜索), include Parkinson's disease (搜索), multiple systems atrophy (搜索) (MSA), and dementia with Lewy bodies (搜索) (DLB).
In-vitro studies demonstrated that Aramchol effectively down-regulated αSyn-aggregation in a dose-dependent manner. According to Allen Baharaff, Galmed's Co-founder and CEO, "Based on our preliminary in-vitro data which demonstrated that Aramchol dose-dependently down-regulated αSyn-aggregation as well as indicating that the treatment was not associated with toxicity, we believe that treatment with Aramchol could prevent the pathophysiological cascade that leads to αSyn-aggregation which induces a chronic inflammatory state that is associated with PD disease progression and severity."
Addressing Unmet Medical Need
Currently, no disease-modifying therapies are available for treatment of Parkinson's disease (搜索) or related synucleinopathies (搜索). These diseases are each characterized by presence of Lewy bodies enriched in αSyn protein. Recent evidence from cell-based screens of αSyn toxicity has identified SCD1 (搜索) as a potential target for treatment of synucleinopathies.
The breakthrough medicinal chemistry work converting Aramchol into a brain-penetrant SCD1 (搜索) inhibitor positions the compound as an attractive therapeutic asset for synucleinopathies (搜索) such as Parkinson's disease (搜索), multiple systems atrophy (搜索), dementia and other CNS indications of unmet need.
Clinical Development Timeline
Subject to regulatory advice, Galmed is planning to advance Aramchol to proof-of-concept Phase 1b/2 studies in Parkinson's disease (搜索) patients in the second half of 2026. "I am excited to report today the fruits of our collaboration with Barcode Nanotech (搜索), advancing our lead compound Aramchol as a first in class brain penetrating SCD1 (搜索) inhibitor," Baharaff commented.
Technology Platform
Barcode Nanotech (搜索)'s NeoRNA™ Targeting Technology enables efficient discovery and optimization of lipid-based nanoparticles (LNPs) through in silico and in vivo mass-screening. The solution combines the design of thousands of new lipids and LNPs, advanced AI tools and in vivo barcoding-based biodistribution analysis at a single cell resolution. Using this platform, Barcode has developed unique LNPs that can direct RNA and other therapies to cells in the brain, kidney, lungs and additional target organs.