Pegtarazimod

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 that the first patient has been dosed in its Phase 2 study of RLS-0071, the Company’s lead therapeutic candidate, for the treatment of hospitalized patients with moderate to very severe steroid-refractory acute graft-versus-host disease (aGvHD), a serious and often fatal complication following hematopoietic stem cell transplantation. “Hematopoietic stem cell transplantation is one of the most impactful treatments available for people with cancer, autoimmune disease, sickle cell disease and other life-threatening disorders. However, the procedure can quickly turn into a severe and life-threatening condition for those who are affected by acute graft-versus-host disease in which the transplanted immune cells attack the patient, leading to severe inflammation and tissue damage,” said ReAlta Chief Medical Officer Kenji Cunnion, MD, MPH. “We believe that RLS-0071 has the potential to be a powerful new tool to improve outcomes for individuals with this serious complication of transplantation. We aim to leverage RLS-0071's unique dual mechanism-of-action (MOA) to inhibit unwanted inflammatory side-effects from the donor cells. We are committed to advancing this clinical program on behalf of patients facing steroid-refractory aGvHD.” Patients with aGvHD who do not respond to standard steroid treatments have limited therapeutic options and face severe inflammation, tissue damage and a high risk of mortality. Each year, approximately 4,000 patients develop steroid-refractory aGvHD within the U.S., EU and Japan. RLS-0071, an investigational medicine based on the Company’s novel EPICC peptide platform, seeks to address this unmet medical need by capitalizing on its unique dual-targeting MOA to modulate both the complement and innate inflammatory pathways, offering a new hope for patients with this devastating condition. RLS-0071 was recently granted Orphan Drug Designation and Fast Track Designation for the treatment of steroid-refractory aGvHD by the U.S. Food and Drug Administration (FDA). The Company is also currently conducting Phase 2 clinical trials of RLS-0071 in newborns with hypoxic ischemic encephalopathy (HIE) ( NCT05778188 ) and hospitalized patients with acute exacerbations of chronic obstructive pulmonary disease (AE-COPD) ( NCT06175065 ). About the Study The Phase 2 study ( NCT06343792 ) is an open-label, prospective, dose-escalation trial to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, dosing and efficacy of RLS-0071 for the secondary treatment of acute graft-versus-host disease (aGvHD) in hospitalized patients who are steroid-refractory. Participants will receive RLS-0071 at their assigned dose level(s) for 7 or 14 days as either a monotherapy or concurrent with the standard-of-care treatment, ruxolitinib. Primary outcomes measures include safety and overall response rate. About ReAlta Life Sciences ReAlta Life Sciences, Inc. is a clinical mid-stage biopharmaceutical company dedicated to saving lives by rebalancing the inflammatory response to address life threatening rare and acute inflammatory diseases. The Company’s EPICC peptide platform is based on discoveries related to the human astrovirus, HAstV-1, which causes a non-inflammatory, self-limiting gastroenteritis by inhibiting components of the innate immune system – a trait unique among viruses. By inhibiting activation of the complement cascade and two key neutrophil-driven mechanisms of inflammation and host tissue destruction - myeloperoxidase (MPO) and neutrophil extracellular traps (NETs), ReAlta’s therapeutic peptides leverage the dual-targeting mechanisms of this human enteric virus – and its millions of years of evolutionary adaptations – to modulate both complement and innate inflammatory pathways. The Company’s pipeline is led by RLS-0071, which has received both Orphan Drug Designation and Fast Track Designation by the U.S. Food and Drug Administration (FDA) and Orphan Drug Designation by the European Medicines Agency, for the treatment of hypoxic ischemic encephalopathy (HIE), a devastating disease that causes brain damage in oxygen-deprived newborns and is a leading cause of cerebral palsy; Orphan Drug Designation and Fast Track Designation by the FDA for the treatment of acute graft-versus-host disease (aGvHD), a disease associated with bone marrow and stem cell transplants whereby transplanted white blood cells attack the new host in an inflammatory response; and IND clearance by the FDA for the treatment of acute exacerbations of chronic obstructive pulmonary disease (COPD), an inflammatory lung disease characterized by progressive airflow limitations and tissue destruction that makes it challenging for patients to breathe. The company launched in 2018 and is located in Norfolk, Virginia and Aguadilla, Puerto Rico. For more information, please visit www.realtalifesciences.com .

Pegtarazimod is being studied as a potential treatment to aGvHD patients after haematopoietic stem cell transplant where steroid therapy is ineffective. Image: Shutterstock / Gorodenkoff. The US Food and Drug Administration (FDA) has granted an orphan drug designation and fast track designation to ReAlta Life Sciences’ lead candidate pegtarazimod (RLS-0071) to treat steroid-refractory acute graft-versus-host disease (aGvHD). These designations come as the company continues its open label Phase II AURORA trial (NCT06343792) with pegtarazimod in this indication. The FDA designations will grant ReAlta seven years of market exclusivity for pegtarazimod upon approval and expedite the review process. aGvHD is an often fatal complication following haematopoietic stem cell transplantation in which donor immune cells attack the recipient’s tissue. Standard treatment involves steroid regimens, but patients who are unresponsive to steroid therapy face limited further options and a high risk of mortality. There are an estimated 4,000 such patients in the US, EU, and Japan. Pegtarazimod is based on ReAlta’s EPICC peptide platform. The technology is based on the human astrovirus HAstV-1, which causes non-inflammatory, self-limiting gastroenteritis by inhibiting components of the immune system. Dr. Kenji Cunnion, chief medical officer at ReAlta, said the FDA decision underscored, “the significant potential of RLS-0071 and its novel dual mechanism-of-action to address critical unmet needs for patients with this life-threatening condition”. See Also: Licensing agreements for innovator GLP1R drugs weigh in with over $6.2bn in 2024 Top 20 biopharmas’ market cap up 4.3% in Q2 2024 as GLP-1 drug innovators hold top spots If successfully developed and approved, pegtarazimod is set to compete in a market inhabited by Johnson & Johnson ’s Imbruvica (ibrutinib), Novartis ’s Jakavi or Jakafi (ruxolitinib phosphate), and Bristol Myers Squibb ’s Orencia (abatacept) among others. These therapies are projected by GlobalData to generate $2.56bn, $2.25bn, and $3.44bn in annual sales by 2030, respectively. GlobalData is the parent company of Pharmaceutical Technology . The Virginia-based ReAlta is currently investigating their lead therapeutic candidate’s application in a variety of indications, fuelled in part by $20m raised in Series A financing in 2021 . Alongside the AURORA trial, the company is conducting Phase II trials to determine the efficacy of pegtarazimod in newborns with hypoxic-ischemic encephalopathy (NCT05778188) and in patients with acute exacerbations of chronic obstructive pulmonary disease (NCT06175065). ReAlta also completed a Phase I trial to investigate the therapeutic application of RLS-0071 against asthma (NCT05351671) and was conducting a Phase I trial exploring the drug’s use in Covid-19-associated acute lung injury (NCT04574869) before the trial was withdrawn due to the “low probability of enrolling patients prior to expiry of study drug”, as per ClinicalTrials.gov . Earlier this year, the company collaborated with US National Institute of Allergy and Infectious Diseases (NIAID) to evaluate the drug’s use in acute radiation syndrome.