LogicBio Therapeutics Announces SUNRISE Phase 1/2 Clinical Design for LB-001 for the Treatment of Methylmalonic Acidemia in Pediatric Patients

– LogicBio’s First IND Clearance Leveraging GeneRide, an In Vivo Homologous Recombination-based Genome Editing Platform –

– Enrollment to Start with Patients as Young as 3 Years Old, De-escalating Down to 6 Months old –

– Webcast and Conference Call Today at 8 a.m. ET to Discuss LB-001 Update –

LEXINGTON, Mass., Aug. 10, 2020 (GLOBE NEWSWIRE) -- LogicBio Therapeutics, Inc. (Nasdaq:LOGC) (LogicBio or the Company), a company dedicated to extending the reach of genetic medicine with pioneering targeted delivery platforms, today announced the clinical trial design for the planned Phase 1/2 clinical trial for LB-001 in pediatric patients with methylmalonic acidemia (MMA) .

“We’re pleased to announce our plans for developing LB-001 in pediatric patients with MMA. This is an important milestone for patients, their families, our company and for the genetic medicines space more broadly as we believe this is the first Investigational New Drug application (IND) clearance for an in vivo gene editing program harnessing homologous recombination,” said Fred Chereau, CEO of LogicBio. “We have maintained a steadfast commitment to execution and the IND clearance is the first of several near-term milestones we expect to reach in our determined efforts to leverage cutting-edge medicines to address high unmet medical needs. I look forward to sharing those updates as we advance our LB-001 program.”

Daniel Gruskin, M.D., SVP, head of clinical development, commented, “The effects of MMA usually appear shortly after birth and can quickly become severe and life-threatening. Early intervention in this vulnerable population is critical to combat the manifestation of irreversible clinical disease pathologies including neurological damage. That’s the reason MMA is on the newborn screening panel in the United States. Our protocol allows for the first cohort to enroll patients as young as three years old and, once certain safety parameters are met, we can age de-escalate to as young as six months old. Our goal is to provide a safe and durable therapeutic with a single administration at an age when we can make a difference for these patients.”

SUNRISE - Phase 1/2 Clinical Trial of LB-001 in Pediatric Methylmalonic Acidemia (MMA)

The SUNRISE trial is a multi-center, open-label, Phase 1/2 clinical trial designed to assess the safety and tolerability of a single intravenous infusion of LB-001 in pediatric patients with MMA characterized by methylmalonyl-CoA mutase gene (MMUT) mutations. Six leading centers in the United States are expected to participate in the SUNRISE Phase 1/2 trial. The trial is expected to enroll eight pediatric patients with ages ranging from 6 months to 12 years, initially starting with 3 to 12 year-old patients and then adding patients aged 6 months to 2 years. The SUNRISE Phase 1/2 trial will evaluate two doses of LB-001. Patients will participate in a pre-dosing observational period and will be administered a prophylactic steroid regimen. The primary endpoint of the SUNRISE trial is to assess the safety and tolerability of LB-001 at 52 weeks after a single infusion. Additional endpoints include changes in disease-related biomarkers, including serum methylmalonic acid, clinical outcomes such as growth and healthcare utilization, and the pharmacodynamic marker albumin-2A. The Company expects to enroll the first patient in early 2021.