FDA Lifts Hold on LogicBio’s Phase 1/2 SUNRISE Trial of LB-001 for MMA

May 9, 2022

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The hold was originally placed after reports of 2 serious adverse events in pediatric patients.

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The US Food and Drug Administration (FDA) has lifted its clinical hold on LogicBio Therapeutics’ phase 1/2 SUNRISE trial (NCT04581785), which is evaluating LB-001 for the potential treatment of methylmalonic acidemia (MMA) in pediatric patients.¹

The hold on the company’s Investigational New Drug (IND) application was issued in February² after reports surfaced that 2 patients dosed with 5 x 1013 vg/kg LB-001 developed thrombotic microangiopathy (TMA), considered a serious adverse event (AE). The cases resolved within weeks, according to LogicBio.

"We are pleased that the FDA has completed its review of the information we provided and that the hold on our LB-001 IND has been lifted," Frederic Chereau, president and chief executive officer of LogicBio, said in a statement.¹ "We look forward to dosing the next patient in our SUNRISE trial, which we expect will occur in the third quarter of 2022."

After talks with the FDA, LogicBio amended its SUNRISE protocol to include enhanced monitoring measures and frequent testing for complement activation, which is associated with TMA.

LB-001 is developed using LogicBio's proprietary GeneRide platform, which uses homologous recombination to edit the genome without exogenous nucleases and promoters. The therapy delivers a corrected MMUT gene intravenously to the liver. The FDA previously granted fast track, rare pediatric disease, and orphan drug designations to the therapy for the treatment of MMA. The European Medicines Agency, meanwhile, has granted orphan drug designation.

In the SUNRISE trial, LB-001 is administered intravenously to participants aged 6 months to 12 years old at a dose concentration of 5e13 vg/kg. The primary end points of the study include the incidence of adverse events and incidence of infusional toxicities over the course of 52 weeks.

The younger age group was previously added to the study after a positive review from the independent data safety monitoring board in October 2021.³ Data announced at that time from the SUNRISE trial demonstrated site-specific gene insertion and protein expression via albumin-2A biomarker expression.

FDA Lifts Hold on LogicBio’s Phase 1/2 SUNRISE Trial of LB-001 for MMA

References:

LogicBio Therapeutics announces FDA lifts clinical hold on SUNRISE trial in pediatric patients with methylmalonic acidemia. News release. May 9, 2022. https://investor.logicbio.com/news-releases/news-release-details/logicbio-therapeutics-announces-fda-lifts-clinical-hold-sunrise

Johnson, V. LogicBio’s methylmalonic acidemia gene-editing therapy program placed on clinical hold. CGTL website. Published February 2, 2022. Accessed May 9, 2022. https://www.cgtlive.com/view/logicbio-methylmalonic-acidemia-gene-editing-therapy-clinical-hold