Matinas gets FDA nod to start phase 1 study of MAT2501 to treat NTM infections

Matinas BioPharma president and CEO Roelof Rongen said: "The encouraging preclinical data demonstrating MAT2501’s oral bioavailability and its targeted delivery of amikacin directly to the site of infection in NTM infections, gives us confidence as we prepare for our upcoming Phase 1 study.

"Importantly, we believe our oral encochleated formulation of MAT2501 has the potential to address the many shortcomings that currently exist in the treatment of both chronic and acute bacterial infections with IV administration of amikacin, including major side effects associated with toxicity."

MAT2501, an orally-administered, encochleated formulation of the broad spectrum IV-only aminoglycoside antibiotic agent amikacin, utilizes the Company’s proprietary, lipid-crystal, nanoparticle delivery technology.

Amikacin is currently used to treat different types of chronic and acute bacterial infections, including NTM infections and various multidrug-resistant gram negative bacterial infections. IV-administered amikacin is associated with major side effects including nephrotoxicity and ototoxicity (permanent loss of hearing) with long-term use.

Matinas BioPharma’s MAT2501 encochleated formulation of amikacin is specifically designed to provide for the targeted delivery of this potent antibiotic while providing a significantly improved safety and tolerability profile. In preclinical studies MAT2501 demonstrated oral bioavailability and targeted delivery of amikacin directly to the site of infection in both pulmonary (lung) and disseminated NTM infections.

The FDA designated MAT2501 as a Qualified Infectious Disease Product (QIDP) for the treatment of NTM infections in December of 2015.

Matinas BioPharma co-founder and chief business officer Jerome Jabbour said: "The achievement of this regulatory milestone underscores the significance of MAT2501’s robust preclinical data package. We look forward to promptly advancing this program into clinical development.

"We believe MAT2501 has the potential to be the first orally-available aminoglycoside, representing a disruptive, game-changing shift in the treatment paradigm for both chronic and acute bacterial infections, including both NTM and gram negative bacterial infections, and an important solution for physicians and patients."

Matinas BioPharma expects to commence the Phase 1 program of MAT2501 during the first half of 2016. The first planned Phase 1 trial is a placebo-controlled, single ascending dose study designed to evaluate the safety, tolerability, and pharmacokinetics (PK) of MAT2501 following oral administration in healthy adult subjects.

After completion of this first study, the second study will be a placebo-controlled, multiple ascending dose study designed to evaluate the safety, tolerability and PK of MAT2501 at up to three different doses in healthy volunteers and is designed to establish a complete PK profile of MAT2501 including peak and trough levels in a multiple-dose regimen.

About Nontuberculous Mycobacteria

Nontuberculous mycobacteria (NTM) are naturally occurring organisms found in water, soil, plants and animals. NTM causes many serious and life-threatening diseases, including pulmonary disease, skin and soft tissue disease, joint infections and, in immunocompromised individuals, disseminated infection.

The most common clinical manifestation of NTM disease is pulmonary, or lung, disease. NTM lung infection occurs when a person inhales the organism from their environment. While most people do not become ill, some individuals develop a slow, progressive and destructive disease when NTM infects the airways and lung tissue leading to inflammation in the respiratory system. Individuals susceptible to the infection often have an unknown defect in their lung structure or immune system, lung damage from a pre-existing chronic obstructive pulmonary disease (COPD), such as emphysema and bronchiectasis, cystic fibrosis, or an immune deficiency disorder, such as HIV or AIDS.

There are about 50,000 to 90,000 people with NTM pulmonary disease in the United States, with a much higher frequency in older adults, and these numbers appear to be increasing. However, NTM can affect any age group. Without treatment, the progressive lung infection caused by NTM results in severe cough, fatigue, and often weight loss. In some people NTM infections can become chronic and require ongoing treatment. Treatment may be difficult because NTM bacteria may be resistant to many common types of antibiotics. Severe NTM lung disease can have a significant impact on quality of life and can be life-threatening.

About MAT2501

MAT2501 is an orally administered, encochleated formulation of the broad spectrum aminoglycoside antibiotic amikacin which may be used to treat different types of multidrug-resistant bacteria, including non-tubercular mycobacterial infections (NTM), as well as various multidrug-resistant gram negative and intracellular bacterial infections.

Currently, amikacin cannot be absorbed enterally and must be given by intravenous, intramuscular or nebulization routes with the significant risk of nephrotoxicity and ototoxicity, which makes it an impractical choice when treating serious infections which often require long courses of therapy, often 12 to 18 months or longer. MAT2501, taking advantage of its disruptive, nano-encapsulation delivery technology, is being developed to provide an orally administered, safer and targeted therapy for improved treatment of these serious and life-threatening bacterial infections in patients, including those who are severely immunocompromised.