FDA slams clinical hold on LogicBio's gene editing drug after clotting cases in child patients
Another day, another AAV-delivered gene therapy running into roadblocks with the FDA.
The agency has placed a clinical hold on LogicBio’s AAV-delivered gene editing therapy LB-001 after two adolescent patients in an early trial for the drug were diagnosed with thrombotic microangiopathy (TMA), a clinical syndrome defined by the formation of tiny clots in blood vessels, the company said Wednesday.
The biotech is trying LB-001 in a Phase I/II trial dubbed SUNRISE to treat children with the rare metabolic disease methylmalonic acidemia, which results in patients not being able to break down certain fats and proteins.
In that study, two of four patients reported cases of TMA, neither of which proved fatal. In the first case, LogicBio instituted new safety protocols in the SUNRISE trial following the patient’s diagnosis. That case was resolved as of December 2021.
In the second case, the patient was diagnosed with TMA in January of this year and is currently being monitored.
With now two cases reported, the FDA opted to halt development until more info on what exactly caused those side effects is picked out. In the meantime, LogicBio has said it’s putting trial data updates on pause and working with the agency to get the study back up and running.
TMA has been a common stumbling block before in gene therapy studies, a fact which LogicBio admitted in its release. But it’s still not a welcome result for the company or its lead drug. LB-001 purports to work by inserting a corrective copy of the methylmalonyl-CoA mutase gene into human cells, driving therapeutic production of that enzyme in the liver.
Shares in $LOGC were trading down 34% before the bell Wednesday.