Rallybio Corporation (Nasdaq: RLYB) has announced the approval of its clinical trial applications (CTAs) by the European Medicines Agency (EMA) and the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA) for a Phase 2 clinical trial of RLYB212. This trial aims to assess the drug's efficacy in preventing fetal and neonatal alloimmune thrombocytopenia (FNAIT) in pregnant women at higher risk for HPA-1a alloimmunization.
The Phase 2 trial, a single-arm, dose confirmation study, is set to commence screening participants in the fourth quarter of 2024. It will be conducted across five European countries: Belgium, the Netherlands, Norway, Sweden, and the UK. The trial will involve eight pregnant women who are at higher risk for HPA-1a alloimmunization and FNAIT.
Trial Design and Objectives
The primary objective of the trial is to evaluate the pharmacokinetics (PK) and safety of RLYB212, a monoclonal anti-HPA-1a antibody. The drug will be administered subcutaneously, starting at Gestational Week 16, with doses given every 4 weeks until parturition. Secondary objectives include assessing pregnancy and neonatal/infant outcomes, as well as monitoring the occurrence of emergent HPA-1a alloimmunization.
Addressing an Unmet Medical Need
FNAIT is a rare, potentially life-threatening condition that can cause uncontrolled bleeding in fetuses and newborns. It arises from an immune incompatibility between a mother and her fetus, specifically involving the human platelet antigen 1 (HPA-1). Alloimmunization occurs when an HPA-1a-negative mother carries an HPA-1a-positive fetus, leading to the development of antibodies that cross the placenta and destroy fetal platelets. This can result in severe thrombocytopenia and potentially devastating consequences, including miscarriage, stillbirth, or severe neurological disabilities in surviving infants. Currently, there is no approved therapy for the prevention or prenatal treatment of FNAIT.
Leadership Perspective
Stephen Uden, M.D., Chief Executive Officer of Rallybio, stated, “Securing these CTA approvals from European health authorities to advance RLYB212 into a Phase 2 trial is a significant achievement... We are activating clinical sites and expect to initiate screening this quarter, which will mark another important step toward achieving our mission to prevent FNAIT and its potentially devastating consequences.”
Steven Ryder, M.D., Chief Medical Officer at Rallybio, added, “These approvals recognize Rallybio’s commitment to safely and effectively progress a novel prophylactic approach for preventing alloimmunization in pregnant women at higher FNAIT risk... We are proud to be leaders in bringing much needed innovation to this area of women’s health, which has gone unaddressed for far too long.”
