The Food and Drug Administration (FDA) has granted Orphan Drug designation to MAT2203 (Matinas BioPharma) for the treatment of cryptococcosis.

Cryptococcosis is a rare, fatal infection caused by the fungus Cryptococcus neoformans. The infection typically manifests in the lungs or the central nervous system, but may also spread to other parts of the body. 

MAT2203 is an orally-administered formulation of amphotericin B that uses the Company’s proprietary lipid nano-crystal (LNC) drug delivery technology to target infections in a manner with minimal toxicity. Currently, amphotericin B is only available for intravenous infusion and has significant treatment-limiting side effects, including nephrotoxicity.

The designation is supported by data from a phase 1 trial that demonstrated a strong safety and tolerability profile with no serious adverse events or observed nephrotoxicity in 48 healthy patients. Additionally, MAT2203 met the primary efficacy end point in a phase 2 trial conducted by the National Institutes of Health (NIH) in 3 out of 3 enrolled patients; 2 out of 3 patients did not show signs of kidney or other toxicity over 1 year.

The FDA previously granted Fast Track and Qualified Infectious Disease Product designations to MAT2203 for the treatment of candidiasis and aspergillosis and for the prevention of invasive fungal infections in patients on immunosuppressive therapy.

The Company is currently enrolling patients in a phase 2a trial of MAT2203 for the treatment of refractory mucocutaneous candidiasis.

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“Orphan drug designation is yet another major step forward for MAT2203 in the treatment of life-threatening fungal infections, and adds to the prior Qualified Infectious Disease Product (QIDP) and Fast Track designations this product has already received,” commented Theresa Matkovits, PhD, Chief Development Officer of Matinas. “We believe MAT2203 represents a promising new approach for the treatment of severe fungal infections and addresses one of the most important limitations of current antifungal treatment options.”

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