ReAlta Life Sciences, Inc. (“ReAlta” or the “Company”), a clinical-stage biopharmaceutical company dedicated to saving lives by rebalancing the inflammatory response to address rare and acute inflammatory diseases, today announced that the European Medicines Agency (EMA) has granted Orphan Drug Designation to RLS-0071 (pegtarazimod) for the treatment of Graft-versus-Host Disease (GvHD).
The Orphan Drug Designation was supported by preliminary data from ReAlta’s ongoing Phase 2 trial, which included data from the initial cohort of steroid-refractory Acute GvHD (aGvHD) patients. ReAlta is currently enrolling the Phase 2, open-label clinical trial of pegtarazimod for hospitalized patients with steroid-refractory aGvHD (NCT06343792) at clinical sites in the United States, Germany, and Spain. The Company expects to report data from additional cohorts of patients enrolled in this trial in 2026. In August 2024, the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation and Fast Track Designation to pegtarazimod for the treatment of aGvHD.
“Receiving EMA Orphan Drug Designation represents a significant new regulatory milestone in our efforts to address the urgent unmet need in aGvHD, and we are particularly encouraged by the EMA’s positive feedback to our initial cohort of Phase 2 data,” said David Marek, Chief Executive Officer of ReAlta. “This designation, alongside our existing FDA Orphan Drug and Fast Track Designations for aGvHD, validates the potential of our novel dual-targeting approach to modulate both neutrophil and complement-mediated inflammation as we advance our Phase 2 trial to bring this potential therapy to aGvHD patients in Europe.”
Kenji Cunnion, MD, MPH, Chief Medical Officer of ReAlta, commented, “Our targeted intervention addresses the specific pathways driving tissue damage, including the inhibition of extracellular myeloperoxidase, NETosis and neutrophil elastase, while preserving beneficial immune function, unlike broadly immunosuppressive approaches to treat aGvHD. The compelling preclinical and clinical data that we have generated shows pegtarazimod’s potential to address the neutrophil-driven disease process in patients with lower gastrointestinal (GI) aGvHD that is the most difficult to treat and has the highest rate of mortality.”
The EMA grants Orphan Drug Designation to medicines intended for the treatment, diagnosis or prevention of rare, life-threatening or chronically debilitating diseases or conditions that affect fewer than five in 10,000 people in the European Union. This designation provides pharmaceutical companies with certain benefits including reduced regulatory fees, clinical protocol assistance, and up to 10 years of market exclusivity in the European Union, if approved.
About the Phase 2 aGvHD Trial and RLS-0071 (pegtarazimod)
The Phase 2 trial (NCT06343792) is an open-label, prospective, dose-escalation trial currently enrolling patients across clinical sites in the United States, Germany, and Spain, evaluating pegtarazimod in hospitalized patients with moderate to very severe steroid-refractory acute graft-versus-host disease. Pegtarazimod is a 15-amino-acid peptide that uniquely targets both humoral and cellular inflammation and is the Company’s lead therapeutic candidate. The peptide works by selectively inhibiting complement activation at C1, as well as myeloperoxidase (MPO) activity and neutrophil extracellular trap (NET) formation – key mechanisms that drive the inflammatory cascade and tissue damage when donor immune cells attack recipient tissues following hematopoietic stem cell transplantation.
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