Matinas BioPharma's LNC Platform Delivers Small Oligonucleotides: New In Vitro Data at TIDES USA 2024

Matinas BioPharma Holdings, Inc., a clinical-stage biopharmaceutical company that focuses on developing innovative therapies through its lipid nanocrystal (LNC) platform, has disclosed new in vitro data demonstrating the effective delivery of small oligonucleotides using LNCs. This data was presented during a poster session at TIDES USA 2024: Oligonucleotide & Peptide Therapeutics, a hybrid conference held in Boston.

The poster, entitled "Intracellular Delivery of Small Oligonucleotides with Lipid Nanocrystals (LNCs): in vitro studies," was presented by Hui Liu, PhD, Chief Technology Officer of Matinas. The research showcased how LNC formulations containing various cytokine-targeted small oligonucleotides were efficiently taken up by murine macrophages, achieving up to 70% gene knockdown for each targeted cytokine.

Dr. Liu emphasized that small oligonucleotides hold immense potential across multiple therapeutic fields. However, their systemic delivery has posed significant challenges, often leading to toxicity and adverse immunogenicity. The in vitro studies revealed that Matinas' LNCs could encapsulate and protect antisense oligonucleotides (ASOs), small interfering RNA (siRNA), and other small oligonucleotides even when exposed to gastric fluid. Moreover, these LNCs can deliver these molecules to various cells with observable biological activity. These findings suggest that LNCs could be a highly promising platform for the oral delivery of small oligonucleotides in future therapeutic applications.

Matinas BioPharma is dedicated to leveraging its LNC platform to introduce breakthrough therapies. One of its leading LNC-based therapies is MAT2203, an oral formulation of the broad-spectrum antifungal drug amphotericin B. Although amphotericin B is highly effective, it is often associated with significant toxicity. Matinas' LNC technology allows for the oral administration of amphotericin B, potentially reducing the nephrotoxicity typically linked with intravenous formulations. Combining this targeted delivery with comparable fungicidal activity may lead to a safer and more effective treatment for invasive fungal infections.

MAT2203 has shown promising results in the completed Phase 2 EnACT study conducted on HIV patients with cryptococcal meningitis, achieving its primary endpoint and demonstrating robust survival rates. The next step involves evaluating MAT2203 in a single Phase 3 registration trial, known as the ORALTO trial. This trial aims to compare MAT2203 as an oral step-down monotherapy following treatment with AmBisome® (liposomal amphotericin B) against the standard care for patients with invasive aspergillosis who have limited treatment options.

Beyond MAT2203, both preclinical and clinical data have indicated that Matinas' LNC technology could address many challenges associated with the safe and effective intracellular delivery of small molecules and larger, complex molecular cargos, including small oligonucleotides like ASOs and siRNA. The unique mechanism of action and flexibility in routes of administration, including oral, position Matinas' LNC technology as a potential next-generation, orally available intracellular drug delivery platform.

Matinas BioPharma continues to explore and develop its LNC technology, aiming for broad applications across various therapeutic areas. The company remains committed to advancing its innovative platform to meet the evolving needs of modern medicine.