Hematology Month in Review: May 2025

May was a slow month for hematology at HCPLive, with relative silence from the FDA and an even spread of trial successes and failures. NovelMed’s Ruxoprubart, a novel complement-targeting immunotherapy, showed efficacy for treating paroxysmal nocturnal hemoglobinuria (PNH) in interim results from its phase 2 trial. On the other hand, Prothena Corporation’s birtamimab failed to achieve its primary phase 3 endpoint for treating AL amyloidosis.

Meanwhile, new research has indicated a connection between iron deficiency anemia (IDA) and ischemic stroke in young adults, as well as between dietary diversity and anemia risk. Another study determined the superiority of thalidomide and hydroxyurea combination therapy over hydroxyurea (HU) alone for treating sickle cell anemia.

Trial News in Hematology

Ruxoprubart Shows Efficacy for PNH in Interim Phase 2 Trial Results

On 19 May, NovelMed announced positive interim results from the phase 2 trial of Ruxoprubart, which demonstrated efficacy in treating PNH. The medication binds to Bb and selectively inhibits the protease activity of the Alternative Pathway C3 convertase, while maintaining the Classical Pathway. The phase 2 trial was ongoing at the time of the report, with 2 of 12 participants still to complete; however, Ruxoprubart had already cleared all efficacy endpoints by avoiding transfusion and increasing hemoglobin above baseline by 1.4 to 2 g/dL.

Birtamimab Misses Primary Phase 3 Endpoint for AL Amyloidosis

May 27 saw the unexpected failure of Prothena Corporation’s AL amyloidosis treatment birtamimab. In the phase 3 AFFIRM-AL trial, the medication missed the primary endpoint of time to all-cause mortality, which led to discontinuation of development and halting the open-label extension. Despite the failed trial, Prothena noted that birtamimab was generally safe and well-tolerated, consistent with its existing profile.

Recent Studies in Hematology

Poor Dietary Diversity Potentially Linked to Risk of Anemia in Adolescents

A systematic review and meta-analysis from Henan University drew a correlation between poor dietary diversity and anemia in adolescents and adults. Micronutrient deficiencies are the cause of a variety of diseases worldwide; folic acid deficiency leads to excess birth defects, and vitamin D deficiency leads to bone fractures and osteoporosis. Likewise, iron deficiency is one of the leading causes of anemia; therefore, proper observation of recommended dietary nutrients can prevent this and similar diseases.

Iron Deficiency Anemia Significantly Increases Ischemic Stroke Risk in Young Adults

According to research from the University of Cincinnati, iron deficiency anemia independently and significantly increases the odds of ischemic stroke in young adults. Roughly 10-15% of all strokes occur in adults aged 18-50, a group which also exhibits an increased prevalence of IDA. The sheer size of the study – roughly 21 million individual health records – also allowed researchers to roughly estimate the prevalence of IDA more generally.

Thalidomide, Hydroxyurea Combination Bests Hydroxyurea Alone for Sickle Cell Anemia

An open-label quasi-experimental clinical trial from India conclusively determined the safety and efficacy of thalidomide and HU versus HU alone for treating sickle cell anemia. This combination therapy has been suggested for patients who have received subpar results from HU alone. Side effects did arise during testing, but investigators noted that they were consistent with previous knowledge of the effects of thalidomide.

Why Screen for Iron Deficiency? It's Common, Consequential & Curable

Given the broad prevalence of iron deficiency, potentially affecting as much as a quarter of the global population, a recent study has indicated the critical need for more accurate and widespread ferritin screening. By comparing the efficacy of various methods and treatments, this study posited that regular screening has clear and consistent value in mitigating and potentially even forestalling symptoms of iron deficiency anemia.