Exhibit 99.1
LogicBio Therapeutics Announces Submission of Investigational New Drug Application (IND) forLB-001 for Methylmalonic Acidemia (MMA) and Highlights Key Corporate Milestones
– Filed IND for lead GeneRide™ candidateLB-001 in pediatric MMA patients –
– Phase 1/2 trial initiation planned for H1 2020, with preliminary data in H2 2020 –
– Established research collaboration with Takeda for GeneRide in Crigler-Najjar Syndrome (CN) –
CAMBRIDGE, Mass., January 10, 2020 – LogicBio Therapeutics, Inc. (Nasdaq:LOGC), a genome editing company focused on developing medicines to durably treat rare diseases in pediatric patients, today announced it has submitted an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) to initiate a Phase 1/2 trial ofLB-001, a recombinant adeno-associated viral vector with human methylmalonyl-COA mutase (MMUT) gene for the treatment of methylmalonic acidemia (MMA).LB-001 leverages LogicBio’s proprietary, promoterless, nuclease-free genome editing technology, GeneRide™, and has previously received both orphan drug and rare pediatric disease designations from the FDA.
LogicBio intends to disclose additional details regarding the planned Phase 1/2 trial, including trial size, endpoints, and timelines, once the FDA accepts the IND. LogicBio plans to initiate a Phase 1/2 trial in pediatric MMA patients in the first half of 2020, with preliminary data expected in the second half of 2020.
“We founded LogicBio with the mission of bringing genetic medicines to children with rare diseases. Both the IND submission and the nomination of our second indication represent significant steps in advancing our goal,” said Fred Chereau, CEO of LogicBio. “MMA and CN are devastating early onset diseases with no approved pharmacological therapies, and we are committed to developing novel medicines based on our GeneRide platform for pediatric patients. We look forward to a transformational year for LogicBio as we work to advance our programs, validate our platform, and expand our pipeline.”
Today, LogicBio also highlighted key recent and upcoming milestones.
| • | | Established collaboration with Takeda to leverage theGeneRide platform in a second indication,Crigler-Najjar Syndrome.LogicBio and Takeda will further research and developLB-301, an investigational pediatric genome editing therapy based on LogicBio’s GeneRide technology for the treatment of CN. TheLB-301 construct, utilizing the modularity of GeneRide, is expected to share several components withLB-001 to facilitate development. Those components include:LK-03 as the capsid; the albumin gene as the target genetic locus for integration; and a 2A peptide sequence to facilitate polycistronic expression and serving as a circulating biomarker. A separate press release with more details can be found on LogicBio’s investor relations website atinvestor.logicbio.com. |
| • | | Initiated Retrospective Natural History Study in MMA. This study is designed to evaluate disease progression in pediatric patients (born since 2010) with severe MMA, with the aim of informing LogicBio’s future development in MMA and its discussions with regulatory agencies. |
| • | | Reported positive data on Next Generation Capsid Development program at European Society of Gene and Cell Therapy Annual Meeting.Data were presented from a set of novel, synthetic adeno-associated virus (AAV) capsid candidates tested against references AAV2, AAV8, andLK-03. All capsids showed selective tropism and more potent transduction and gene expression than the reference capsids in human hepatocytes of a chimeric FRG mouse model. LogicBio, working in partnership with the Children’s Medical Research Institute, intends to advance this research and present additional findings at a scientific conference in 2020. |