The US Food and Drug Administration (FDA) has lifted the clinical hold placed on LogicBio Therapeutics’ Phase I/II SUNRISE clinical trial of LB-001 for methylmalonic acidemia (MMA) in paediatric patients.
An investigational, single-dose genome editing therapy, LB-001 is intended for use as an early intervention in MMA leveraging the company’s GeneRide drug development platform.
The regulatory agency noted in a letter that LogicBio had adequately addressed all the issues linked to the clinical hold.
In February this year, the regulatory agency had placed on hold the Investigational New Drug application (IND) for LB-001 after two serious adverse events were reported in the trial.
These events were classified as cases of thrombotic microangiopathy (TMA) and both cases were claimed to be resolved within weeks.
With the latest development, the company modified the trial protocol as per the discussions with the FDA.
The protocol changes comprise additional assessment measures such as regular testing for complement activation, a TMA characteristic, and using a complement inhibitor in the event where lab results show a potential TMA.
In line with the lifting of the clinical hold, LogicBio will begin enrolling paediatric subjects in the SUNRISE trial and has commenced activities to restart subject dosing soon.
The open-label, multi-centre trial will evaluate the safety, tolerability and initial efficacy of a single intravenous dose of LB-001 in paediatric subjects with MMA that are characterised by methylmalonyl-CoA mutase gene (MMUT) mutations.
Subjects aged six months to 12 years will be part of the trial.
The company anticipates reporting interim findings from the trial by the end of the second quarter of this year.
A rare and life-threatening genetic disorder, MMA affects newborns.
LogicBio president and CEO Frederic Chereau said: “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.
“We look forward to dosing the next patient in our SUNRISE trial, which we expect will occur in the third quarter of 2022.”