CFF Grant Will Support Early Studies of Oral Antibiotic, MAT2501, to Treat NTM Infections in Cystic Fibrosis

Daniela Semedo, PhD avatar

by Daniela Semedo, PhD |

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Under a research contract award granted by the Cystic Fibrosis Foundation Therapeutics (CFFT), Matinas BioPharma and the Colorado State University (CSU) will jointly conduct preclinical studies assessing the efficacy of MAT2501, a potential oral antibiotic, in treating nontuberculous mycobacterium infection (NTM) in a cystic fibrosis (CF) lung model.

NTM are ubiquitous organisms responsible for opportunistic infections with a broad spectrum of virulence. The incidence and prevalence of NTM infections in people with chronic lung diseases, such as cystic fibrosis, or with compromised immune systems are increasing worldwide and becoming a major public health problem. These infections are difficult to treat, and progressively more resistant to most available antibiotics. Treating NTM infections is also complicated because of the potential toxicity of existing therapies, in addition to their increasingly limited efficacy.

MAT2501 is an encochleated formulation of the broad spectrum aminoglycoside antibiotic agent amikacin, that is currently used to treat bacterial infections, including NTM and various drug-resistant gram-negative bacterial infections. But amikacin is now given by intravenous injection, and its use is associated with major side effects, including toxicity to the kidneys and ototoxicity (permanent loss of hearing) with long-term use.

“We are honored to receive the support of Cystic Fibrosis Foundation Therapeutics for this important research program, and our hope is that this is the beginning of what promises to be a long-term relationship as we advance the clinical development of MAT2501. Mycobacterium infections in people with cystic fibrosis are very difficult to treat, to a significant degree, because of the unique complications associated with CF,” Raphael Mannino, PhD, chief scientific officer of Matinas BioPharma and principal investigator of the CF research program, said in a press release.

“In earlier pre-clinical work, we demonstrated the efficacy of MAT2501 against several mycobacterium species. Anti-infectives formulated in our cochleate technology are uniquely targeted toward the site of infection while significantly reducing toxicities. We are looking forward to working with CFFT and CSU with the goal to expand the treatment options for CF patients battling these multi-drug resistant mycobacterium infections,” Mannino added.

MAT2501, Matinas’ lead antibiotic product candidate, is an orally administered version of amikacin (encochleated amikacin) based on a lipid-crystal, nanoparticle delivery technology for more targeted treatment, improving its safety and tolerability to allow for multiple dosing. Preclinical studies performed on both pulmonary and disseminated NTM infections demonstrated anti-bacterial activity with no noted toxicity, the company reported in September 2015.

The U.S. Food and Drug Administration (FDA) classified MAT2501 as a Qualified Infectious Disease Product (QIDP) for the treatment of NTM infections in December 2015. The potential therapy was also designated an Orphan Drug for the treatment of NTM by the FDA.

CFFT is the non-profit drug discovery and development arm of the Cystic Fibrosis Foundation.