Galmed Pharmaceuticals Ltd. has announced the breakthrough development of a brain penetrating new formulation of Aramchol. Crossing of the blood-brain barrier (BBB) is an essential step to achieve effective treatment effects in Parkinson’s disease (PD) and other CNS diseases.
The new Aramchol formulation is characterised by sequestration of Aramchol in lipid nanoparticles which will be administered by subcutaneous injection for delivery across the BBB.
This new Aramchol formulation was co-developed by Galmed and Barcode Nanotech, based on Barcode Nanotech’s platform which enables simultaneous screening of hundreds of different nanoparticle formulations in vivo, coupled with AI analysis tools to select the optimal delivery vehicle to the brain.
There are currently no disease-modifying therapies available for treatment of PD or related synucleinopathies such as multiple systems atrophy (MSA) and dementia with Lewy bodies (DLB). These diseases are each characterised by presence of Lewy bodies enriched in αSyn protein and are thus collectively known as synucleinopathies. Recent evidence from cell-based screens of αSyn toxicity has identified stearoyl-CoA desaturase 1 (SCD1) as a potential target for treatment of synucleinopathies.
Based on the evidence for the role of SCD1 inhibition in mitigating synucleinopathies, Galmed’s breakthrough medicinal chemistry work converting Aramchol into a brain-penetrant SCD1 inhibitor position Aramchol as an attractive therapeutic asset for synucleinopathies such as PD, multiple systems atrophy (MSA), dementia and other CNS indications of unmet need.
Allen Baharaff, Galmed’s co-founder and CEO, saidd: “The largest challenge in developing innovative CNS therapeutics is delivering these molecules to the brain. I am excited to report today the fruits of our collaboration with Barcode Nanotech, advancing our lead compound Aramchol as a first in class brain penetrating SCD1 inhibitor. 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. Subject to the regulatory advice we are now seeking, we are planning to advance Aramchol to a PoC Ph1b/2 studies in PD patients in H2 2026.”
Ronen Eavri, co-founder and CEO of Barcode Nanotech, said: “Barcode Nanotech has developed a library of novel lipids for RNA and DNA, LNP-based delivery, through a unique in vivo & AI-based screening platform which allow simultaneously screening of hundreds of different nanoparticle formulations. 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. We look forward to continuing our collaboration with Galmed to develop target-specific selective delivery vehicles of Aramchol to enable precise target-site activity and release of this promising drug.”