MAT2501: Encochleated Formulation of Amikacin

Candidate & Indication Development Stage
IND Preparation Phase 1 Development Phase 2 Development Phase 3 Development Market
MAT2501 Gram-Negative Bacterial Infections
IND Preparation Phase in progress
Phase 1 Development Phase not started
Phase 2 Development Phase not started
Phase 3 Development Phase not started
Market Phase not started

MAT2501 is a lipid-crystal nano-particle formulation of Amikacin. Amikacin is an aminoglycoside antibiotic specific for Gram-negative bacterial infections.

Amikacin is most often used for treating severe, hospital-acquired infections with multidrug-resistant Gram-negative bacteria such as Pseudomonas Aeruginosa, Acinetobacter, and Enterobacter. Serratia marcescens, Mycobacterium and Providencia stuartii are also included in the spectrum. Amikacin may be combined with a beta-lactam antibiotic for empiric therapy for people with neutropenia and fever. The emergence of treatment-resistant bacterial infections has elevated the clinical importance of Amikacin.

Unfortunately, in the currently available formulations Amikacin is intravenously (IV) administered and has significant side-effects. The potential damage to the kidneys (nephrotoxicity) and hearing loss is of most concern. These side effects are dose dependent and the nephrotoxicity is the consequence of sensitive kidney tissue being exposed directly to Amikacin. The data for our cochleate lipid-crystal nano-particle formulation of Amikacin [see Our Science Page] indicates two advantages over previously approved Amikacin formulations:

  • The lipid-crystal nano-particle is a solid particle, and unlike liposomes, does not “leak” its nephrotoxic content while circulating. The particle only releases its medication pay-load when inside the target cells, and thus protects the kidney and other sensitive tissues from many of the Amikacin side effects.
  • Because of the targeted approach, the required dose level is typically lower than other formulations. So besides the protective effects of the technology itself, the lower dose further contributes to a more beneficial side-effect profile.

In addition, in the vast majority of animal studies the MAT2501 product was orally administered. If confirmed in human efficacy studies, the oral administration is a second major differentiator of our technology which offers significant health-economic benefit since patients do not need to stay in the hospital to receive the therapy. Because of these strong differentiators brought by the cochleate lipid-crystal nano-particle technology, we believe that MAT2501 will be able to satisfy a significant need in the treatment of life-threatening Gram-negative bacterial infections.

MAT2501 is undergoing formal GLP animal toxicology studies in preparation for an IND filing with the FDA.

About Gram-Negative Bacterial Infections

Serious or life-threatening Gram-negative bacterial infections include infections by Pseudomonas Aeruginosa in Cystic Fibrosis patients or in a hospital acquired setting, infections by Acinetobacter and Enterobacter in immunocompromised patients and ventilator-bound patients, and infections by various forms of Mycobacterium, including M.Avium and M.Tuberculosis.

Approximately 30,000 patients in the US (about 70,000 worldwide) have cystic fibrosis, while approximately 140,000 patients with haematological malignancies or transplants are significantly immunocompromised each year. In addition, elderly and patients with HIV and other chronic viral infections are at risk for opportunistic Gram-negative bacterial infections. Approximately 800,000 hospitalizations in the US involved mechanical ventilation each year as estimated by AAST. According to the CDC, approximately 10,000 cases of Tuberculosis occur in the US every year, and the emergence of drug-resistant TB is of significant concern.