Q2 2025 Recap: Hematology News and Updates

Hematology has been relatively silent compared to other fields in recent months, with few new treatments developed and fewer approvals from the US Food and Drug Administration (FDA). The majority of conditions, despite still being prevalent, are relatively well-handled.

Q2 2025 largely followed this pattern, with the FDA handing out a few designations and no significant approvals. Trial results varied as well, with a handful of advances tempered by the destructive failure of birtamimab to reach its primary endpoint. Now that the end of Q2 has arrived, HCPLive Hematology has put together a recap of the biggest news and headlines from April to June 2025.

In this recap, we highlight 3 regulatory updates from the FDA, our 4 biggest trial announcement articles, and 2 key insights from prominent experts featured within our coverage.

Q2 Regulatory Updates in Hematology

FDA Grants Orphan Drug Designation to DIAG723 for Treatment of HHT

On June 18, DIAG723 received Orphan Drug designation from the FDA and a positive opinion from the European Medicines Agency (EMA), both for the treatment of hereditary hemorrhagic telangiectasia. Parent company Diagonal Therapeutics recently began a natural history study of adults with HHT earlier this year, partnering with the organization Cure HHT to characterize the variability of patient-reported outcomes, such as hematologic support, quality of life, and epistaxis.

FDA Grants Fast Track Designation to Mavorixafor for Chronic Neutropenia

On June 10, X4 Pharmaceuticals announced the receipt of Fast Track designation from the FDA for mavorixafor, a treatment for chronic neutropenia. Mavorixafor is currently being examined in the phase 3 4WARD trial, which aims to evaluate the efficacy, safety, and tolerability of oral, once-daily treatment with or without injectable G-CSF. The trial will last 52 weeks with a primary endpoint based on 2 separate outcome measures of annualized infection rate and positive ANC response.

VAS-101 (Vasceptor) Receives Orphan Drug Designation for Sickle Cell Disease

On June 12, Vascarta announced receipt of an Orphan Drug designation from the FDA for VAS-101, a treatment for sickle cell disease. A phase 1 clinical trial is currently underway to investigate the safety and tolerability of VAS-101, as well as its effects on impaired blood flow dynamics like adhesion molecule expression and erythrocyte fragility parameters. Investigators are also examining the mean change in red blood cell sickling kinetics and oxygen dissociation curves.

Q2 2025 Trial Announcements in Hematology

Ruxoprubart Shows Efficacy for PNH in Interim Phase 2 Trial Results

Ruxoprubart, a novel complement-targeting immunotherapy typically administered as a monotherapy, reached all primary efficacy endpoints in interim data released by parent company NovelMed. These endpoints included transfusion avoidance, increased hemoglobin levels, reduced lactate dehydrogenase, and increased PNH clone size. NovelMed has also indicated an interest in partnerships to further develop the treatment.

Birtamimab Misses Primary Phase 3 Endpoint for AL Amyloidosis

Prothena Corporation announced the failure of birtamimab in its phase 3 AFFIRM-AL clinical trial, accompanied by a plan to cut operating costs and shrink its workforce. An investigational, humanized monoclonal antibody, birtamimab was designed to selectively target and clear the amyloid leading to organ dysfunction and failure. Despite receiving Orphan Drug designation from both the FDA and EMA, as well as an agreement between Prothena and the FDA for a Special Protocol Assessment, the phase 3 trial was an overall failure.

Iptacopan (Fabhalta) Hits Primary Endpoint in Phase 3 APPULSE-PNH Trial

Novartis announced iptacopan’s success in APPULSE-PNH, a phase 3b trial investigating its safety and efficacy. A twice-daily oral monotherapy for the treatment of paroxysmal nocturnal hemoglobinuria, iptacopan exhibited no new safety signals and was well-tolerated by patients. In addition, it resolved extravascular hemolysis control and reduced absolute reticulocyte count in treated patients.

Genetically Modified Cell Therapy BEAM-101 Successful in Sickle Cell Disease Trial

Announced by parent company Beam Therapeutics, BEAM-101, a genetically modified cell therapy for the treatment of sickle cell disease, has demonstrated its strong clinical profile in the BEACON phase 1/2 clinical trial. BEAM-101 is intended to facilitate the production of non- and anti-sickling fetal hemoglobin; among these results, investigators also found normalization of a variety of hemolysis markers, such as indirect bilirubin, haptoglobin, and lactate dehydrogenase.

Q2 2025 Expert Perspectives in Hematology

Pediatric Year in Review: New Therapies in Sickle Cell Disease, with Shaina Willen, MD

Shaina Willen, MD, a board-certified provider in Pediatric Hematology/Oncology and Pediatric Pulmonology from UC Davis Health, speaks on new therapies for sickle cell disease and the recent drastic increase in options for patients.

A Positive Outlook on Gene Therapy for Hemophilia B Post-Discontinuation, with Guy Young, MD

Guy Young, MD, director of the Hemostasis and Thrombosis program at the Children’s Hospital of Los Angeles and professor of pediatrics at the Keck School of Medicine of the University of Southern California, discusses the discontinuation of fidanacogene elaparvovec development and its implications for the future of gene therapy in treating hemophilia B.