Phase 2 trial of NM8074 in untreated PNH patients underway

Eight patients have been enrolled so far, two have started treatment

Marisa Wexler MS avatar

by Marisa Wexler MS |

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NovelMed Therapeutics has launched a Phase 2 clinical trial to test its experimental therapy, NM8074, in people with paroxysmal nocturnal hemoglobinuria (PNH) who haven’t received treatment for the disease.

Eight patients have been enrolled so far and two have started treatment, according to the company.

The Phase 2 trial (NCT05646524) seeks to enroll 12 adults and all will be treated with NM8074, given by infusion into the bloodstream, for 13 weeks (about three months), according to one of two dosing schedules. Half will receive 20 mg/kg every two weeks, and the other half will receive 10 mg/kg weekly for the first four weeks, followed by 20 mg/kg every other week.

The study’s main goal is to evaluate the treatment’s safety. The researchers also will monitor how it affects patients’ need for blood transfusions and biomarkers of disease activity, such as lactate dehydrogenase (LDH), a marker of blood cell destruction in PNH.

In PNH, a group of immune proteins that make up the complement cascade, which normally help fight off bacteria and other infectious agents, become activated and attack and destroy healthy blood cells. There are several approved PNH treatments, all of which work by blocking the complement cascade’s activation to prevent blood cell destruction.

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How is NM8074 different from available treatments?

Two major molecular pathways can be used to activate the complement cascade: the so-called classical and the alternative pathway. Available treatments block both pathways, which can control the disease, but also increase the risk of infections.

NM8074 is designed to block the alternative complement pathway — the one that mostly drives blood cell destruction in PNH — by targeting an alternative complement protein known as Bb. Since NM8074 leaves the classical pathway untouched, NovelMed expects it may help keep PNH under control and reduce the risk of infection compared with other available treatments.

Early data from the ongoing Phase 2 study haven’t revealed any noteworthy safety concerns and early biomarker data indicate the therapy can prevent blood cell destruction, as expected, according to the company.

“Preliminary data from the Phase II clinical trial in treatment-naïve PNH patients confirms NM8074’s potential to comprehensively address intravascular and extravascular hemolysis [red blood cell destruction]-mediated anemia and bolster patient host defense systems, thus reducing the risk of infections,” Rekha Bansal, PhD, NovelMed’s CEO, said in a company press release.

NovelMed completed a Phase 1 study (NCT05642546) wherein NM8074 was given to 40 healthy volunteers. No safety issues were reported when the therapy was administered at doses ranging from 0.3 to 20 mg/kg.

The company also is sponsoring two other Phase 2 trials (NCT05731050 and NCT05646563) to test NM8074 in people with PNH who’ve been treated with the approved therapy Soliris (eculizumab).

NovelMed is also developing NM3086, another experimental therapy that’s designed to block the alternative complement pathway through a different mechanism. The company announced positive data from a study of NM3086 in healthy volunteers earlier this year.

NovelMed expects these therapies may offer a meaningful alternative in PNH and other diseases driven by complement activation.

“This wide application to multiple diseases underscores our commitment to serving diverse patient populations,” Bansal said.