A New Haven biotech started by three former Alexion executives has begun human testing on its first experimental drug — a potential treatment for a rare condition that causes uncontrolled bleeding in newborns.
Rallybio announced the launch of the Phase 1-2 study on Tuesday, saying the first dose of the drug had been administered.
The drug, called RLYB211, aims to prevent the rare condition FNAIT (fetal and neonatal alloimmune thrombocytopenia), which occurs during pregnancy.
The condition is the result of a mismatch between mother and fetus of a specific human platelet antigen, according to Rallybio.
The mismatch causes the mother to develop antibodies that attack her fetus’ platelets, which can cause uncontrolled bleeding either in the womb or shortly after birth.
Rallybio says its drug, a human hyperimmune globulin derived from human plasma, aims to prevent the condition in mothers who are identified as being at risk by giving them a low dose of the problematic antibodies.
“Our hearts break for the mothers, babies and families who have been impacted by this terrible disorder, and with RLYB211, we hope to prevent this disease from ever occurring,” CEO Martin Mackay said in a statement.
Rallybio acquired the drug from a small startup in Norway.
The proof-of-concept study, being conducted in Frankfurt, Germany, will consist of three cohorts of healthy male volunteers, each to be given a different dose of the drug.
The trial will test the safety of the drug before it can be administered to pregnant women in later-stage studies, according to Rallybio.
Alexion veterans Mackay, Jeffrey Fryer and Dr. Stephen Uden launched Rallybio in 2018 and have raised $182 million in venture capital to date, including a $145 million Series B round last spring.
The company, which targets rare diseases in the areas of hematology, immuno-inflammation and metabolism, has 20 employees at its 234 Church St. headquarters and an office at UConn’s Technology Incubation Program (TIP) in Farmington.
Contact Natalie Missakian at news@newhavenbiz.com
