LogicBio Therapeutics announced that the U.S. FDA has placed a clinical hold on the Investigational New Drug (IND) submission for LB-001 for the treatment of methylmalonic acidemia (MMA) pending the resolution of certain clinical and nonclinical questions. LogicBio expects that the FDA questions will be provided in writing within 30 days. LogicBio plans to work closely with the FDA to resolve these questions as quickly as possible.
The Company submitted the IND in January 2020 to support the initiation of a Phase 1/2 clinical trial in patients with MMA. The Phase 1/2 trial is designed for pediatric MMA patients and was anticipated to begin in the first half of 2020, with preliminary data expected in the second half of 2020.
LB-001 has received both orphan drug and rare pediatric disease designations from the U.S. FDA.
LB-001 is an investigational pediatric genome editing therapy based on LogicBio’s GeneRide™ technology. GeneRide enables site-specific integration and lifelong expression of therapeutic transgenes, without the use of exogenous promoters or nucleases. LB-001 is designed to incorporate a functioning version of the faulty human methylmalonyl-COA mutase (MMUT) gene into the genome of MMA patients. LogicBio has demonstrated preclinical proof-of-concept of GeneRide in multiple animal models of the disease, improving survival and reversing disease pathology. In preclinical MMA models, LogicBio has shown that cells into which GeneRide has inserted a transgene demonstrate a selective survival advantage over cells not expressing the transgene.
Methylmalonic acidemia, or MMA, is a rare and life-threatening genetic disorder, affecting 1 in 25,000 to 50,000 newborns. A mutation in the MUT gene prevents the body from properly processing certain fats and proteins. As a result, toxic metabolites accumulate in the liver, in muscle tissue and in the brain. Symptoms include vomiting, lethargy, seizures, developmental delays and organ damage.