CFF Awards Up to $3.75M to Develop Oral Antibiotic for NTM Infections

The Cystic Fibrosis Foundation (CFF) awarded up to $3.75 million to Matinas BioPharma to support preclinical studies of MAT2501, a potential oral antibiotic to treat nontuberculous mycobacterium (NTM) infections in people with cystic fibrosis (CF) and other lung diseases.

A goal of this early research work is to prepare the potential antibiotic for use in clinical trials in patients.

NTM is an umbrella term for opportunistic lung infections caused by mycobacteria. This type of infection most commonly affects people with an underlying lung disease, like CF, or with a compromised immune system. The disease is termed “nontuberculous” to distinguish it from the mycobacterium that causes tuberculosis.

NTM infections are difficult to treat, requiring prolonged course of antibiotics and lengthy hospital stays.

“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,” Jerome D. Jabbour, CEO of Matinas, said in a press release.

MAT2501 is an oral version of the broad spectrum aminoglycoside antibiotic amikacin (encochleated amikacin) used to treat bacterial infections, including NTM and various drug-resistant, gram-negative bacteria.

Amikacin, currently administered by injection or inhalation, has been linked with serious side effects over long-term use that include toxicity to the kidneys and ototoxicity or permanent hearing loss.

MAT2501 is designed to deliver high levels amikacin directly to the lungs, based on a lipid nano-crystal delivery technology that allows for a more targeted treatment, while improving the antibiotics’ safety and tolerability, the company reported.

The CFF award will allow Matinas to conduct further preclinical research, including toxicity studies that are required to bring the candidate therapy into Phase 2 clinical trials.

A previous Phase 1 study in healthy adults tested single ascending doses of oral MAT2501 — 200 mg, 400 mg, or 800 mg — regarding treatment safety, tolerability and pharmacokinetics, which refers to the movement of a medicine into, through, and out of the body.

MAT2501 blood levels at all three doses were below the safety levels recommended for injected amikacin, Matinas reported on its website. The antibiotic was also well-tolerated with no serious adverse events.

“We believe that an orally bioavailable amikacin … would be the first oral aminoglycoside and would represent a significant improvement over currently available therapy,” Jabbour said.

“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,” he added.

Upon successful completion of the preclinical studies, the CFF is willing to further support financially clinical trials of MAT2501, including doses optimizing studies and Phase 2 studies in CF patients with NTM lung infections.

“We look forward to continuing to work with the CF Foundation on realizing the potential of our LNC delivery platform,” Jabbour said.

MAT2501 was designated a qualified infectious disease product (QIDP) and an orphan drug as a possible treatment of NTM infections by the U.S. Food and Drug Administration.

Both FDA designations provide benefits to a therapy’s developer, including market exclusivity for up to 12 years (seven years through the orphan drug designation plus five years through the QIDP) should the treatment gain agency approval.