Matinas BioPharma Holdings said Monday that the National Institutes of Health (NIH) has revealed positive data from a third patient enrolled in the collaborative Phase 2a clinical study of Matinas’ lead anti-infective product candidate MAT2203 for the treatment of chronic refractory mucocutaneous candidiasis (CMC) infection.
According to Matinas, this third patient, with long-standing azole resistant mucocutaneous candidiasis, met the primary endpoint of the Phase 2a study in achieving ≥ 50% clinical response with treatment of MAT2203. MAT2203 was well tolerated with any adverse events observed being mild in severity and unrelated to study drug. With this third positive response, the study has met its statistical hurdle for success.
MAT2203 is Matinas’s orally-administered, encochleated formulation of the broad spectrum fungicidal medication amphotericin B. Matinas BioPharma’s proprietary lipid-crystal nano-particle formulation of amphotericin B has a novel mechanism of absorption and distribution to infected tissues and has the potential to transform the way this potent fungicidal agent is administered and used in clinical practice.
“With the statistical success hurdle that was prospectively set at a 20% patient-response probability and now seeing a clinical response in three out of three patients, we have successfully achieved the 3 out of 16 possible clinical responders required for the study to meet its primary endpoint. This is a major milestone for this study and our platform technology more broadly,” said Roelof Rongen, Chief Executive Officer. “We continue to develop invaluable data in patients receiving MAT2203 and are exploring options with our valued collaborators at the NIH to determine next best steps for this study. In addition to impressive efficacy results in this difficult-to-treat patient population, we remain very excited about the long-term safety data of MAT2203. Whereas today amphotericin B can only be used in limited, acute settings due to its significant toxicity, we now have multiple patients who have been taking MAT2203 for almost a full year with no signs of kidney or liver toxicity.”
About the patient
The third patient in this study was diagnosed with a dual Candida albicans and C. glabrata infection with azole resistance. The predominant manifestation was esophageal candidiasis, which had been refractory to treatment for a prolonged period. Patient 03 achieved a reduction in clinical symptoms at an efficacious orally administered dosage of 800 mg MAT2203 per day, meeting the response criterium of ≥ 50% reduction in clinical symptoms. MAT2203 was generally well tolerated by Patient 03 and there were no signs of nephrotoxicity, hypokalemia or hepatoxicity (measured by ALT and AST). Indicators of kidney and liver toxicity remained within normal limits throughout the treatment period. For this patient, no underlying immunocompromising condition was diagnosed. Patients 01 and 02, both with an underlying hereditary immunodeficiency called Job’s Syndrome, also known as Autosomal Dominant Hyper IgE Syndrome (AD-HIES), enrolled earlier in this trial and achieved reduction in clinical symptoms of 57% (at 800mg/day) and 85% (at 400mg/day). The first two patients have enrolled in a long-term study extension and have shown no signs of kidney or liver toxicity over the approximately twelve months of being administered MAT2203. Furthermore, the clinical response to MAT2203 seen in these patients has been maintained and/or improved during the extension period in addition to patients reporting meaningful quality-of-life improvements.
“We remain extremely grateful to the patients for their ongoing participation and to the NIH for conducting this study,” said Raphael J. Mannino, Matinas’s Chief Scientific Officer. “As we look forward to our upcoming meeting with the FDA, we are positioning MAT2203 to be used by and for patients who today have very few treatment or prevention options, either due to increasing drug resistance, toxicity or use-limiting drug to drug interaction with other therapies.”