The sun and shares are rising again at LogicBio after the FDA lifted a clinical hold on a genome editing therapy for children with a rare disorder.
Shares of the Lexington, Massachusetts-based biotech were sent skyward by 60% as the markets opened Monday morning on the news that the phase 1/2 SUNRISE trial will get underway again. Even with that rapid climb, the shares are only worth 79 cents apiece, but the rally was much needed after the hold put a cash-strapped LogicBio in a tough position.
In February, the phase 1/2 methylmalonic acidemia study was placed on hold after the second patient dosed followed the same path as the first and developed thrombotic microangiopathy, a condition defined by the destruction of red blood cells, low platelets and organ damage. The report from the first patient came in during 2020. Both patients have since recovered.
According to LogicBio, the FDA said the biotech “satisfactorily addressed all clinical hold issues.” The next patients will receive the same dose as before but will be continually assessed.
With the hold now lifted, the biotech reinstated its guidance of releasing interim data by the end of this year's second quarter. The company aims to recruit eight patients. Methylmalonic acidemia is a life-threatening inherited disorder that prevents the body from breaking down certain proteins and fats.
LogicBio has now agreed to additional screening and monitoring requirements with the FDA so that enrollment can resume. The trial will now include additional checks for complement activation and the use of a complement inhibitor if lab tests reveal a potential case of thrombotic microangiopathy. The complement system is a part of the immune system that helps defend against pathogens and keeps the body in a normal state. Activation of the complement system is a hallmark of thrombotic microangiopathy, which leads to the formation of microscopic blood clots.
The FDA’s hold is not the only time the regulator has stepped in to halt work on LB-001. The therapy was also briefly placed on hold in 2020 as the FDA considered an application for human testing. The problems with the application were resolved by August 2020; however, details were scant on the FDA’s original concerns.