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January 7, 2022

Rallybio launches trial for promising rare disease drug

PHOTO | Steve Laschever Rallybio’s co-founders (from left to right) are: Dr. Stephen Uden, Jeffrey Fryer and Martin Mackay.

New Haven-based Rallybio announced on Thursday the launch of a Phase 1 clinical trial for its latest drug candidate to fight a rare disease in newborns and outlined its goals for the coming year. 

The company reported that the first subjects have been dosed in its study of RLYB212, a novel human monoclonal antibody drug candidate designed to prevent fetal and neonatal alloimmune thrombocytopenia (FNAIT). 

FNAIT is a life-threatening rare disease that can cause uncontrolled bleeding in fetuses and newborns. 

In December Rallybio announced positive data from a Phase 1/2 study of its RLYB211 for the same ailment, with results showing significant benefit over a placebo. 

Rallybio’s plans for 2022 include launching a study of its RLYB116, a potential treatment for  patients with paroxysmal nocturnal hemoglobinuria and generalized Myasthenia Gravis.

Also on tap are studies later of a slate of drug candidates for the treatment of hypophosphatasia, a metabolic disorder. That work is in partnership with U.K.-based biotech Exscientia, which utilizes artificial intelligence in drug discovery.

Rallybio CEO Martin Mackay said, “Our mission is to build Rallybio into a leading biotechnology company, with a broad and sustainable pipeline of product candidates that can transform the lives of people with severe and rare disorders and shatter expectations of what’s possible.”

Rallybio, a startup founded by three former Alexion executives, launched in January 2018 and has built a portfolio of candidates to treat rare diseases in the areas of hematology, immuno-inflammation, maternal fetal health and metabolic disorders.

Rallybio’s IPO in August brought in nearly $93 million, with shares debuting at $13 each. Shares were trading around $9 this week. 

Contact Liese Klein at

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