Shares of LogicBio Therapeutics have fallen more than 57% this morning after the company announced that its gene therapy for pediatric patients with methylmalonic acidemia has been placed on clinical hold by the U.S. Food and Drug Administration.
LogicBio CEO Fred Chereau/Courtesy of LogicBio
Shares of LogicBio Therapeutics have fallen more than 57% this morning after the company announced that its gene therapy for pediatric patients with methylmalonic acidemia had been placed on clinical hold by the U.S. Food and Drug Administration.
Lexington, Massachusetts-based LogicBio conducted a Phase I/II study on LB-001, an investigational, first-in-class, single-administration, adeno-associated virus (AAV) genome editing therapy that uses LogicBio’s proprietary GeneRide drug development platform. This morning, the company said the hold was placed following a severe adverse event involving two of the four patients dosed in the SUNRISE clinical trial.
In December, LogicBio disclosed that the third patient dosed in the SUNRISE study, a pediatric patient between 6 months and 2 years, experienced a severe adverse event following treatment with LB-001. The patient was treated with a 5 x 1013 vg/kg dose of LB-001. The SAE was categorized as a case of thrombotic microangiopathy (TMA), which was previously been reported as a potential negative reaction with other AAV genetic therapies. The patient was hospitalized and treated for the TMA. In a call with reporters this morning, Fred Chereau, president and chief executive officer of LogicBio, said the patient had been discharged from the hospital’s critical care unit after improved conditions.
The company disclosed that the fourth patient, also in the 6 months and 2 years age group, treated with the same dose level of LB-001, has also experienced a SAE that has been classified as TMA. The patient is currently undergoing treatment to address the issue according to the company. After reporting the SAE to the FDA, the trial was placed on hold.
LogicBio said it will work closely with the FDA to determine the next steps going forward. They anticipate a written communication from the FDA regarding the hold. Chereau noted that patient safety is of primary importance to the company and thanked the care team treating the pediatric patients.
The company’s announcement stated that the first two patients dosed with LB-001 have done well after receiving a 5 x 1013 vg/kg of LB-001. These two patients were older, between 3 to 12 years old. Per the protocol of the trial, they are being monitored.
“We look forward to working closely with the FDA and the DSMB to determine the next steps for the trial and the program,” Chereau said in a statement.
Methylmalonic acidemia is a disorder in which the body cannot break down specific proteins and fats. The result is a buildup of methylmalonic acid in the blood.
