LogicBio Therapeutics, Inc., a clinical-stage company advancing a diversified pipeline of genetic medicines addressing rare disorders from infancy through adulthood, May 9th announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold on the company’s LB-001 Investigational New Drug Application (IND), allowing patient enrollment to resume in the Phase 1/2 SUNRISE trial for pediatric patients with methylmalonic acidemia.
In its letter, the FDA acknowledged that the company satisfactorily addressed all clinical hold issues. The company has initiated activities to resume dosing as soon as possible.
Methylmalonic acidemia refers to a group of inherited conditions in which the body can’t breakdown certain parts of proteins and fats. This leads to a build-up of toxic substances and bouts of serious illness called decompensation events or metabolic crises.
Frederic Chereau, president and chief executive officer of LogicBio, 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.
As previously disclosed, the FDA placed the IND for LB-001 on clinical hold following the occurrence of two serious adverse events, categorized as cases of thrombotic microangiopathy (TMA), in the company’s SUNRISE trial. Both cases of TMA resolved within weeks.
In connection with the lifting of the clinical hold, LogicBio amended the SUNRISE protocol in a manner that reflected its dialogue with the FDA. LogicBio expects to proceed with dosing after it implements the changes to the SUNRISE protocol, which include enhanced monitoring measures, such as frequent testing for complement activation, a characteristic of TMA, as well as the use of a complement inhibitor in the event there are laboratory findings indicating a potential TMA. LogicBio plans to treat the next patients, who may be as young as six months old, at the 5e13 vg/kg dose and continually assess safety outcomes.
Following the lifting of the clinical hold, the company announced that it is reinstating its previous guidance and expects to present interim clinical data from the SUNRISE trial by the end of the second quarter of 2022.