Pegtarazimod demonstrates significant clinical improvement and reduction in inflammatory markers in patients with lower gastrointestinal acute GvHD
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 a presentation of interim Phase 2 clinical data on RLS-0071 (pegtarazimod) for the treatment of acute graft-versus-host disease (aGvHD) at the 67th American Society of Hematology (ASH) Annual Meeting.
The new findings will be presented in a poster titled “Anti-inflammatory drug pegtarazimod (RLS-0071) demonstrates clinical improvement in lower gastrointestinal acute graft-versus-host disease (aGvHD) and target engagement of key inflammatory marker myeloperoxidase (MPO).” The data highlight interim results from ReAlta’s Phase 2 clinical trial, an open-label, prospective, dose-escalation study evaluating pegtarazimod in hospitalized patients with steroid-refractory aGvHD, underscoring the program’s continued clinical momentum.
The study design features multiple cohorts exploring different dosing strategies, including a treatment optimization cohort of 6 patients receiving 10mg/kg of pegtarazimod with ruxolitinib for 7 days. Preliminary efficacy of pegtarazimod was evaluated by clinical response in the lower GI, skin and liver, and characterized as Grade II, III or IV, per Mount Sinai Acute GVHD International Consortium (MAGIC) guidelines. Plasma levels of MPO were evaluated as a pharmacodynamic (PD) measurement of drug activity in a qualified assay.
Key Highlights from ASH 2025 Poster Presentation:
- Clinical MAGIC Stage improvement observed for 6 of 6 patients with steroid-refractory aGvHD, with 4 of 5 participants demonstrating lower GI MAGIC Stage improvement of 1, 2, or 3 stages over 7 days of treatment
- Median plasma MPO levels decreased by 68% (p = 0.02), suggesting modulation of neutrophil-driven inflammatory mechanisms
- Safety profile characterized by no dose-limiting toxicities or serious adverse events attributed to the study drug
“Pegtarazimod treatment shows compelling clinical improvements for patients with steroid-refractory aGvHD, particularly in lower gastrointestinal involvement,” said Robert Zeiser, M.D., from the Medical Center, University of Freiburg and lead author of the study. “The statistically significant decrease in plasma myeloperoxidase levels suggests a direct pathway to reducing tissue damage and potentially improving outcomes for patients with this life-threatening condition.”
Kenji Cunnion, M.D., Chief Medical Officer of ReAlta, said, “This data demonstrates pegtarazimod’s unique ability to target multiple inflammatory pathways simultaneously, particularly in lower gastrointestinal aGvHD, a condition with historically poor patient outcomes. By selectively inhibiting complement activation and neutrophil-driven mechanisms, we are exploring a novel therapeutic approach that could transform the clinical management of this complex and life-threatening disease.”
About Pegtarazimod (RLS-0071)
Pegtarazimod (RLS-0071) is a pipeline-in-a-product that represents a paradigm shift in treating immune-mediated inflammatory disease. Pegtarazimod inhibits C1 and MBL activation within the classical and lectin pathways to block the initiation of the inflammatory cascade at its earliest stages, while simultaneously blocking myeloperoxidase (MPO) in order to prevent the activation of toxic reactive oxidative species (ROS) and formation of neutrophil extracellular traps (NETs) that are known to accelerate tissue damage.
With demonstrated biomarker validation across neurology, immunology and pulmonology, pegtarazimod is currently advancing through multiple Phase 2 clinical trials, including ongoing studies in newborns with moderate to severe hypoxic ischemic encephalopathy (HIE) and patients with steroid-refractory acute graft-versus-host disease (aGvHD), along with a recently completed Phase 2 proof-of-concept study in hospitalized patients with acute exacerbations of chronic obstructive pulmonary disease (COPD).
ReAlta has secured multiple FDA Orphan Drug and Fast Track Designations for pegtarazimod in HIE and aGvHD, as well as European Medicines Agency Orphan Drug Designations for both indications, positioning ReAlta to deliver transformative outcomes for patients facing life-threatening inflammatory conditions with no adequate treatment options.
About ReAlta Life Sciences
ReAlta Life Sciences is clinical-stage biopharmaceutical company redefining the treatment possibilities for patients with devastating and historically untreatable inflammation-driven diseases by developing first-in-class tailored peptides designed to achieve life-changing outcomes. The company’s lead candidate, pegtarazimod (RLS-0071) is a next-generation dual-targeting intervention that represents a potential solution that rebalances the body’s inflammatory response and prevents tissue damage by selectively blocking both complement- and neutrophil-mediated pathways at the outset, disrupting inflammation at the core. To learn more about ReAlta, visit https://realtalifesciences.com and follow us on LinkedIn.
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