ReAlta Life Sciences 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 designation was supported by preliminary data from the company's ongoing Phase 2 trial, which included data from the initial cohort of steroid-refractory Acute GvHD (aGvHD) patients.
Regulatory Milestone Builds on FDA Designations
This European designation follows the U.S. Food and Drug Administration's grant of both Orphan Drug Designation and Fast Track Designation to pegtarazimod for the treatment of aGvHD in August 2024. "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.
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.
Novel Dual-Targeting Mechanism
Pegtarazimod represents a 15-amino-acid peptide that uniquely targets both humoral and cellular inflammation. 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.
"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," commented Kenji Cunnion, MD, MPH, Chief Medical Officer of ReAlta. "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."
Ongoing Phase 2 Trial
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 Phase 2 trial is an open-label, prospective, dose-escalation trial evaluating pegtarazimod in hospitalized patients with moderate to very severe steroid-refractory acute graft-versus-host disease. The Company expects to report data from additional cohorts of patients enrolled in this trial in 2026.
EPICC Platform Technology
ReAlta's therapeutic approach is built on the company's EPICC peptide platform, which leverages discoveries from the human astrovirus (HAstV-1) that uniquely inhibits components of the innate immune system. The Company's therapeutic peptides employ the dual-targeting mechanisms of HAstV-1 to modulate both complement and innate inflammatory pathways by inhibiting complement cascade activation and two key neutrophil-driven mechanisms: myeloperoxidase (MPO) and neutrophil extracellular traps (NETs).
Beyond aGvHD, RLS-0071 has received FDA Orphan Drug Designation and Fast Track Designations for hypoxic ischemic encephalopathy (HIE), a devastating disease that causes brain damage and high mortality in oxygen-deprived newborns, as well as Orphan Drug Designation by the European Medicines Agency for both HIE and GvHD. The drug has also received FDA IND clearance for acute exacerbations of chronic obstructive pulmonary disease (COPD).
