FDA Clears Phase I PKD Study

The US Food and Drug Administration (FDA) is clearing a new multi-platform development approach dubbed RP-L301 to treat Pyruvate Kinase Deficiency (PKD), a rare disorder that results in red cell destruction with mild to life-threatening anemia.

The Investigational New Drug (IND) clearance allows Rocket Pharmaceuticals to move forward with the well-established lentiviral vector (LVV) and adeno associated viral vector (AAV) gene therapy platforms to treat the rare disease.

“RP-L301 represents the first gene therapy candidate in development for PKD, a hematologic disorder in which the current treatment options, chronic blood transfusions and splenectomy, are associated with burdensome side effects and end-organ damage,” Gaurav Shah, MD, Chief Executive Officer and President of Rocket, said in a statement. “Promising preclinical RP-L301 data suggest correction of multiple key disease elements, including hemoglobin increases, reticulocyte reduction, and correction of splenomegaly.”

The RP-L301 clearance, which marks the fourth Rocket-sponsored IND in the last year, allows the company to move forward with a global phase I study to assess the safety, tolerability, and preliminary efficacy of the treatment.

The open-label, single-arm, clinical trial will involve 6 adult and pediatric transfusion-dependent PKD patients in the US and Europe. The trial will be comprised of 3 cohorts assessing the new approach in young pediatric, older pediatric, and adult populations.

Once the adult cohort is complete, Rocket plans to move forward with the younger patient population studies.

Lucile Packard Children’s Hospital Stanford will serve as the lead site in the US for adult and pediatric patients, while Hospital Infantil Universitario Niño Jesús will serve as the lead site in Europe for pediatrics and Hospital Universitario Fundación Jiménez Díaz will serve as the lead site in Europe for adult patients.

PKD is a rare, monogenic red blood cell disorder resulting from a mutation in the PKLR gene encoding for the pyruvate kinase enzyme, a key component of the red blood cell glycolytic pathway.

The disorder, which commonly affects children, impacts between 3000-8000 patients in the US and European Union.