NovelMed’s PNH antibody therapy NM8074 now called ruxoprubart

Phase 2 trial testing safety, efficacy of treatment candidate in patients

Andrea Lobo, PhD avatar

by Andrea Lobo, PhD |

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NM8074, an investigational antibody therapy for paroxysmal nocturnal hemoglobinuria (PNH), will now be called ruxoprubart, its developer NovelMed has announced.

The name change came after the USAN, or United States Adopted Names Council, selected ruxoprubart as the unique generic, or nonproprietary, name for the therapy candidate. According to the company, the new name reflects a novel nomenclature system for antibodies, based on antibody type and its mechanism of action.

“The assignment of the nonproprietary name to NM8074 represents a significant milestone as we advance this potential PNH treatment towards study completion and regulatory filings,” Rekha Bansal, PhD, NovelMed’s CEO, said in a company press release.

“This allows us to establish a well-recognized name for future publications, labeling, and marketing materials,” Bansal added.

NovelMed is now evaluating the therapy’s safety and efficacy in people with PNH in a Phase clinical 2 trial (NCT05646524) launched last fall.

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In PNH, proteins of the complement cascade — a part of the immune system that normally helps fight off infections — attack healthy blood cells, particularly red blood cells that are responsible for transporting oxygen through the bloodstream. This leads to disease symptoms that include fatigue, shortness of breath, and blood in the urine.

The complement cascade can become activated through varying pathways: the classical and the alternative. Several approved medications to treat PNH work to block both pathways, thereby preventing complement-mediated blood cell destruction. However, such treatment also can increase the risk of infections.

Ruxoprubart is an antibody-based therapy that’s designed to target a protein called Bb, which is part of the alternative complement pathway. It mostly drives blood cell destruction in PNH, while leaving the classic pathway untouched. As such, its use is expected to help control the disease while reducing the risk of infections, as compared with other therapies now available.

NovelMed completed a Phase 1 study (NCT05642546) in 2022 that tested ruxoprubart in 40 healthy volunteers. The study demonstrated that the treatment was safe and tolerated well when given at doses ranging from 0.3 to 20 mg/kg. Moreover, it completely blocked the alternative pathway in a dose-dependent manner.

The ongoing Phase 2 trial expects to enroll 12 adults with PNH who have not received previous treatment with complement inhibitors. All participants will receive ruxoprubart via an intravenous or into-the-vein infusion for 13 weeks, or about three months.

The treatment will be administered according to one of two dosing schedules: Half of the patients will receive the therapy at a dose of 20 mg/kg every two weeks, while the other half will receive it at a dose of 10 mg/kg weekly for the first four weeks followed by 20 mg/kg every two weeks thereafter.

Early data from the study demonstrated that when given biweekly, ruxoprubart was safe and well tolerated. Moreover, the therapy was found to be able to prevent blood cell destruction and reduce the need for blood transfusions. Such transfusion typically are used to help ease PNH symptoms, such as fatigue, that are caused by low blood cell counts.

NovelMed also is conducting two Phase 2 trials — NCT05646563 and NCT05731050 — that are testing ruxoprubart in PNH patients treated with Soliris (eculizumab). The FDA also has approved trials evaluating ruxoprubart in other complement-mediated diseases, including atypical hemolytic uremic syndrome (aHUS).