FDA Gives Nod to Start of Phase 1 Drug Trial for NTM Infections

FDA Gives Nod to Start of Phase 1 Drug Trial for NTM Infections

Matinas BioPharma Holdings of Bedminster, N.J., recently announced that its investigational new drug (IND) application to the U.S. Food and Drug Administration is open and the company has been authorized to begin a Phase 1 clinical trial for its novel drug MAT2501 (encochleated amikacin), as a therapy for infections with non-tuberculous mycobacterium (NTM).

Patients with bronchiectasis often suffer from cough, chest pain, shortness of breath, wheezing and fatigue, and are known to be more susceptible to NTM infections. NTM are natural organisms found in many elements, such as plants, animals, soil, and water. They can provoke serious and life-threatening reactions, including pulmonary disease and even disseminated infection in immunocompromised individuals.

While most people will not become ill when exposed to NTM, some might develop a slow and progressive condition in which NTM infects the airways and lung tissue, eventually leading to inflammation in the respiratory system. There are currently between 50,000 and 90,000 people with NTM-associated pulmonary disease in the U.S. alone.

MAT2501 is an orally administered encochleated formulation of the broad spectrum aminoglycoside antibiotic agent amikacin. Amikacin is typically used in the treatment of different types of chronic and acute bacterial infections, like NTM infections. However, intravenously (IV) administered amikacin has been linked to significant side effects, such as ototoxicity (loss of hearing) and nephrotoxicity with long-term use.

“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,” Roelof Rongen, president and CEO of Matinas BioPharma, said in a press release. “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.”

The company’s MAT2501 formulation of amikacin is specifically developed to provide a targeted delivery of the antibiotic at the same time that it offers a considerably better safety and tolerability profile, potentially resulting in fewer side effects associated with drug intake.

The FDA had previously designated MAT2501 as a Qualified Infectious Disease Product (QIDP) for the treatment of NTM infections in December 2015, based on a preclinical study that demonstrated the successful oral bioavailability and targeted delivery of amikacin administered directly to the site of infection.

The study, “Oral Encochleated Amikacin Demonstrates Activity In Vivo and In Vitro in Biofilm Models of Mycobacterium avium,” was presented at the American Society for Microbiology’s Interscience Conference of Antimicrobial Agents and Chemotherapy and International Society of Chemotherapy’s International Congress of Chemotherapy and Infection (ICAAC/ICC 2015) scientific meeting in San Diego, California.

“The achievement of this regulatory milestone underscores the significance of MAT2501’s robust preclinical data package,” said Jerome D. Jabbour, Matinas BioPharma’s co-founder and CBO. “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.”

The company will initiate its Phase 1 clinical trial on MAT2501 before the second half of 2016. This first trial will be a placebo-controlled, single ascending dose study, planned to assess the drug’s safety and tolerability, together with the pharmacokinetics (PK) profile, after oral administration in healthy participants. After the completion of this initial stage, the second study will evaluate MAT2501 in up to three different doses in order to establish a complete PK profile, also in healthy volunteers.