ReAlta Life Sciences Announces New Clinical Data for RLS-0071 (pegtarazimod) from Hypoxic Ischemic Encephalopathy Program at Pediatric Academic Societies 2025 Meeting

NORFOLK, Va.--(BUSINESS WIRE)--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 new clinical data from its hypoxic ischemic encephalopathy (HIE) program, to be presented at the Pediatric Academic Societies (PAS) Annual Meeting, taking place April 24-28, 2025 in Honolulu, Hawaii.

First Human Evidence of Predicted Inflammatory Mechanisms in HIE Aligned with RLS-0071 Mechanism of Action

Poster Presentation: “Early Granulocyte Inflammatory Biomarkers in Newborn Babies with Moderate or Severe Hypoxic Ischemic Encephalopathy”

Biomarker data from the ongoing Phase 2 STAR trial provide the first confirmatory evidence in humans of inflammatory mechanisms previously observed only in animal models of HIE. The findings show elevated levels of myeloperoxidase (MPO) in newborns with HIE, as predicted, with generally higher levels observed in those with severe versus moderate cases. These data validate a critical component of the disease process. Extracellular MPO contributes to brain tissue damage in two distinct ways: direct tissue injury through hypochlorous acid generation and activation of neutrophils and microglia to a pro-inflammatory state, leading to the formation of extracellular traps that further contribute to tissue damage. The formation of extracellular traps is supported by finding elevated cell-free DNA in the plasma of many of these babies.

“These biomarker findings represent a significant advancement in our understanding of the inflammatory pathways driving HIE in humans,” said Kenji M. Cunnion, MD, MPH, Chief Medical Officer at ReAlta. “This is the first clinical evidence linking MPO-driven mechanisms in HIE, validating a key component of the disease process. These insights strongly support the rationale for targeting MPO and neutrophil activation with RLS-0071 (pegtarazimod). Pegtarazimod is designed to inhibit these processes, including neutrophil and microglia activation and extracellular trap formation, and these data suggest that our mechanism is well positioned to address these two drivers of inflammation and tissue damage in HIE.”

Predictable Pharmacokinetic Profile in Vulnerable Newborn Population

Poster Presentation: “Pharmacokinetics of RLS-0071, a Novel Anti-Inflammatory Peptide, in Newborns With Moderate or Severe Hypoxic Ischemic Encephalopathy”

In pharmacokinetic (PK) data from the first two dosing cohorts of the STAR trial, RLS-0071 (pegtarazimod) at both 3 mg/kg and 10 mg/kg dose levels demonstrates highly consistent results with pre-clinical PK models and adult humans. This is a particularly notable achievement in a newborn population, where PK profiles often vary due to rapid physiological changes. ReAlta's PK modeling and planned dose levels are effectively translating into the targeted patient population, with plasma concentrations remaining within previously established safety parameters.

“The data being presented at PAS advance both our understanding of the disease and the strong potential of pegtarazimod in treating newborns with HIE,” said Paolo Martini, PhD, Chief Research & Development Officer of ReAlta. “We are encouraged by the consistency of the pharmacokinetics and the emerging biomarker data. Together, these results reinforce our confidence in pegtarazimod’s innovative dual-targeting mechanism and its potential to address significant unmet needs in HIE, where no approved pharmacological treatments currently exist beyond therapeutic hypothermia.”

About the STAR Trial and RLS-0071 (pegtarazimod)

The STAR trial (NCT05778188) is a two-stage, randomized, double-blind, placebo-controlled study currently enrolling patients across 13 NICUs in the United States, evaluating RLS-0071 (pegtarazimod) in newborns with moderate or severe HIE undergoing therapeutic hypothermia. Pegtarazimod is a 15-amino-acid peptide that uniquely targets both humoral and cellular inflammation and is the Company's lead therapeutic candidate for HIE treatment. The peptide works by inhibiting complement activation at C1, as well as myeloperoxidase activity and extracellular trap formation – key mechanisms implicated in the inflammatory cascade of reperfusion injury that contributes to brain damage in HIE.

About ReAlta Life Sciences

ReAlta Life Sciences, Inc. is a clinical mid-stage biopharmaceutical company dedicated to addressing life threatening rare and acute inflammatory diseases by rebalancing the inflammatory response. The Company’s EPICC peptide platform leverages discoveries from the human astrovirus (HAstV-1), which uniquely inhibits components of the innate immune system. ReAlta’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). The Company’s lead candidate, RLS-0071 (pegtarazimod), 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; Orphan Drug Designation by the European Medicines Agency for HIE; FDA Orphan Drug and Fast Track Designations for acute graft-versus-host disease (aGvHD) associated with bone marrow and stem cell transplants; and FDA IND clearance for acute exacerbations of chronic obstructive pulmonary disease (COPD). Founded in 2018, ReAlta operates in Norfolk, Virginia and Aguadilla, Puerto Rico. For more information, please visit www.realtalifesciences.com.