Phase 2 Data Support a Safer Immunotherapy Option for PNH

In a phase 2 clinical trial, ruxoprubart showed impressive efficacy, along with good tolerance and safety, as monotherapy for untreated paroxysmal nocturnal hemoglobinuria (PNH). NovelMed Therapeutics Inc., the biopharmaceutical company developing this agent, announced the results in a press release.

Ruxoprubart is a novel immunotherapy antibody that targets the complement system, specifically blocking the disease-afflicted alternative pathway while sparing the classical pathway. According to NovelMed, this will distinguish it as the first PNH immunotherapy not to have a Black Box warning for immunosuppression of the classical pathway.

“We are excited to announce that our Alternative Pathway strategy for treating PNH is delivering exceptional results,” commented NovelMed chief strategy advisor Alex Kumar.

In the ongoing, open-label, multi-dose phase II trial, ruxoprubart monotherapy was administered to 12 adults with PNH. This trial was launched following encouraging results from a phase 1 study in which 40 healthy volunteer enrollees received ruxoprubart at dose levels ranging from 0.3 to 20 mg/kg, with good tolerability and no safety issues.

At 12 weeks, the trial had met all primary endpoints regarding protection of PNH Type III red blood cells against lysis, achievement of complete transfusion avoidance, increase of hemoglobin levels above baseline, and reduction of lactate dehydrogenase levels to baseline. The treated patients also had increased PNH Type III clone size and normal clearance of infection and reported no side effects.

“The promising interim results from our phase 2 PNH study solidify Ruxoprubart as a novel treatment for patients with advanced hematological conditions, offering hope where existing therapies fall short,” remarked NovelMed chief executive officer Rekha Bansal, PhD.

Ruxoprubart currently has Orphan Drug designation from the FDA for treating PNH and NovelMed intends to apply for a Breakthrough Therapy designation for this agent. The company continues to develop ruxoprubart for use in other complement-mediated hematological diseases, as well as complement-mediated dermatological, ocular, and renal disorders.

Reference

NovelMed Therapeutics Inc. Accessed May 19, 2025. https://www.globenewswire.com/news-release/2025/05/19/3083995/0/en/Phase-II-Data-in-Treatment-Na%C3%AFve-Paroxysmal-Nocturnal-Hemoglobinuria-PNH-Patients-Ruxoprubart-Demonstrates-Best-in-Class-Efficacy-as-Monotherapy.html