Matinas BioPharma Holdings, Inc. (NYSE AMER: MTNB), a clinical-stage biopharmaceutical company focused on developing next generation therapeutics to advance standards of care in areas of significant unmet medical need, today announced that it has been awarded up to $3.75 million from the Cystic Fibrosis Foundation (CFF). The award will support preclinical development of MAT2501, Matinas’ lipid nano-crystal (LNC) oral formulation of the broad-spectrum aminoglycoside amikacin, toward an indication to treat nontuberculous mycobacterial (NTM) lung disease, including infections in patients with cystic fibrosis (CF).
“We are grateful to the Cystic Fibrosis Foundation for their support in accelerating the development of MAT2501 as a potential best in class treatment for NTM lung disease. These are debilitating, potentially life-threatening, and increasingly prevalent pulmonary infections, especially in patients with cystic fibrosis,” commented Jerome D. Jabbour, Chief Executive Officer of Matinas. “We believe that an orally bioavailable amikacin, which takes advantage of our LNC delivery platform, would be the first oral aminoglycoside and would represent a significant improvement over currently available therapy. Furthermore, an oral, well tolerated, and targeted aminoglycoside would also potentially be of considerable value in treating other acute bacterial infections, especially gram-negative infections, where oral options are very limited and drug resistance is an increasing challenge. We look forward to continuing to work with the CF Foundation on realizing the potential of our LNC delivery platform.”
The CFF award will allow Matinas to rapidly advance the development of MAT2501 and will support preclinical in vitro and in vivo studies, along with several of the toxicology studies necessary to progress MAT2501 into Phase 2. Pending a successful preclinical program, the CFF has indicated to Matinas a willingness to consider a request for further monetary support for the continuation of clinical studies, including dose determination and Phase 2 efficacy studies in CF patients suffering from NTM lung disease.
MAT2501 has been designated as a Qualified Infectious Disease Product (QIDP) and as an Orphan Drug for the treatment of NTM by the U.S. Food and Drug Administration (FDA). Orphan Drug designation of MAT2501 provides for a seven-year marketing exclusivity period against competition in the United States upon FDA approval, as well as other incentives and exemptions, including waiver of Prescription Drug User Fee Act (PDUFA) filing fees and tax credits for the cost of the clinical research. If MAT2501 is ultimately approved by the FDA, the seven-year period of marketing exclusivity from orphan designation combined with the additional five years of marketing exclusivity provided by the QIDP designation, provides for a potential total of 12 years of marketing exclusivity.
About NTM Lung Disease
NTM lung disease is a chronic, debilitating condition arising from an NTM infection in the lungs and is associated with significant patient morbidity and mortality. The signs and symptoms of NTM lung disease often overlap with the underlying lung conditions that increase risk for NTM, like cystic fibrosis, bronchiectasis, COPD, and asthma. The most common pathogens for NTM infections in the United States are Mycobacterium avium complex (MAC), which accounts for more than 80% of all NTM infections in the U.S. Patients with NTM lung infections frequently require lengthy hospital stays and prolonged courses of antibiotics to manage their disease.
The prevalence of human disease attributable to NTM has increased over the past two decades and is now growing at more than 8% per year and is even more prevalent than tuberculosis in the U.S. In 2018, it was estimated that between 75,000 and 100,000 patients were diagnosed with NTM lung disease in the U.S. alone.
MAT2501 is an oral, encochleated formulation of the broad-spectrum aminoglycoside antibiotic agent amikacin, which utilizes the Company’s proprietary LNC platform to achieve oral bioavailability, limit toxicity and enable targeted delivery to sites of infection. Currently, amikacin can only be delivered parenterally or through inhalation and is used to treat a variety of chronic and acute bacterial infections, including both NTM infections and various multidrug-resistant gram-negative bacterial infections. IV and inhaled amikacin, however, are associated with major side effects including nephrotoxicity and ototoxicity (permanent loss of hearing) with long-term use. Matinas believes that MAT2501’s ability to orally deliver high levels of amikacin directly to the lung and without use-limiting toxicity, distinguishes it from all available therapies and could provide an important solution for patients and physicians.