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Orphan Drug Designation for AUTO3, a New Therapy for ALL

May Offer Improved Safety Profile for Pediatric Patients

The FDA has given an orphan drug designation to AUTO3 (Autolus Therapeutics plc), autologous enriched T-cells genetically modified with a retroviral vector to express two chimeric antigen receptors that target CD19 and CD22, for the treatment of acute lymphoblastic leukemia (ALL).

ALL is the most common pediatric cancer and represents approximately 25% of cancer diagnoses in children younger than 15 years of age. Annually, approximately 3,100 children and adolescents below age 20 are diagnosed with ALL in the U.S., and rates have been steadily increasing over the last few decades. Although pediatric patients usually respond well to first-line treatment, a substantial number relapse with chemotherapy-resistant disease.

AUTO3 is a programmed T cell therapy containing two independent chimeric antigen receptors targeting CD19 and CD22 that have been optimized for single target activity. The treatment is designed to minimize relapse caused by single antigen loss in patients with B-cell malignancies. AUTO3 is currently being tested in pediatric ALL in the AMELIA clinical trial and in diffuse large B-cell lymphoma in the ALEXANDER clinical trial.

AUTO3 may also provide an improved safety profile over current CAR-T therapies as low levels of severe cytokine release syndrome and neurotoxicity have been observed in clinical studies.

Source: Seeking Alpha, April 23, 2019

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