MAT2501: LNC Formulation of Amikacin

Candidate & Indication Development Stage
IND Preparation Phase 1 Development Phase 2 Development Phase 3 Development Market
MAT2501 Non-Tuberculous Mycobacterium (NTM) Infections
IND Preparation Phase complete
Phase 1 Development Phase in progress
Phase 2 Development Phase not started
Phase 3 Development Phase not started
Market Phase not started

MAT2501, an orally-administered, LNC formulation of the broad spectrum aminoglycoside antibiotic agent amikacin, which is currently available to patients only in IV formulations. Amikacin is currently used to treat different types of chronic and acute bacterial infections, including non-tuberculous mycobacterium (NTM) infections and various multi-drug resistant gram negative bacterial infections. IV-administered amikacin is associated with major side effects including nephrotoxicity and ototoxicity (permanent loss of hearing).

MAT2501 is designed to provide targeted delivery of the potent antibiotic amikacin while providing a significantly improved safety and tolerability profile. In preclinical studies MAT2501 demonstrated efficacy after oral bioavailability in murine models of both pulmonary (lung) and disseminated NTM infections. The FDA designated MAT2501 as a QIDP and an Orphan Drug for the treatment of NTM infections.

In May 2017, we completed and announced topline results from a Phase 1 single escalating dose clinical trial of MAT2501 in healthy volunteers in which no serious adverse events were reported and where oral administration of MAT2501 at all tested doses yielded blood levels that were well below the safety levels recommended for injected amikacin, supporting further development of MAT2501 for the treatment of NTM infections.

We intend to initially develop MAT2501 for the treatment of NTM infections and are also exploring the development of MAT2501 for the treatment of a multi-drug resistant, gram negative bacterial infections. If approved, we believe MAT2501 would become the first orally bioavailable aminoglycoside and represent a significant improvement over existing therapies from a treatment and health economic perspective.