NovelMed Therapeutics announced positive efficacy results from the administration of NM3086 to a rabbit model of paroxysmal nocturnal hemoglobinuria (PNH). The results, announced via news release, highlight the ability of NL3086 to regulate the extravascular and intravascular hemolysis mechanisms that occur in PNH.

“PNH continues to be an unmet clinical need as none of the FDA-approved therapies have provided complete remission from existing complications,” said Rekha Bansal, PhD, chief executive officer of NovelMed Therapeutics. “NM3086’s novel and well-differentiated mechanism of action is expected to result in a superior safety and efficacy profile compared to those FDA-approved for the treatment of PNH.”

Read more about PNH therapies

PNH is a rare disease characterized by red blood cell hemolysis and increased free hemoglobin and lactate dehydrogenase (LDH) levels in the blood. The condition is due to alternative pathway dysregulation and is currently treated in part with C3 and C5 blockers, which block the entire complement system.

Current therapies have undesirable side effects including anemia, persistent infections, and only limited reduction of LDH levels. Furthermore, approximately 36% of patients with PNH on treatment with C5 blockers require more than one transfusion per year and 70% continued to have low hemoglobin levels.

NM3086 is an anti-properdin humanized monoclonal antibody that blocks only the alternative pathway and leaves the classical pathway intact. Therefore it is able to block the hemolysis found in PNH and reduce LDH levels while also reducing free hemoglobin. The researchers speculate that the treatment has the potential to provide complete remission in PNH.

“The animal data clearly highlight that NM3086 can control the mechanisms of both extravascular and intravascular hemolysis in PNH and may potentially change the way PNH is being treated,” said Robert Bard, chief regulatory officer of NovelMed Therapeutics.

Given that alternative pathway disruption is a key player in several other complement-mediated diseases, NM3086 is being developed to potentially treat multiple rare and non-rare conditions associated with anemia and alternative pathway dysregulation.

Reference

Anti-properdin antibody (NM3086) reduced hemolysis, LDH, and free hemoglobin in an animal model of paroxysmal nocturnal hemoglobinuria (PNH) — a rare disease. News release. Novelmed Therapeutics; December 12, 2022.