Autolus Publishes Data on Novel CAR-T AUTO1 Leukemia Treatment

 Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies, on Tuesday announced that the journal Nature Medicine has published both pre-clinical results and clinical data from the ongoing Phase I CARPALL trial of AUTO1, demonstrating the potential of the company’s novel CAR T therapy targeting CD19 in development for the treatment of pediatric acute lymphoblastic leukemia (ALL).  The paper reports that AUTO1, or CAT Chimeric Antigen Receptor T cells (CAT CAR T), utilizes a binder with a fast off rate and showed both increased proliferation/cytotoxicity in vitro and enhanced proliferative capacity and anti-tumor activity when compared to FMC63 CAR T therapies in vivo.  In the Phase 1 clinical trial, 86% (n=14) of recurrent/refractory pediatric ALL patients achieved molecular complete remission after a single dose, with a median duration of remission of 7.4 months and no severe cytokine release syndrome (CRS; ≥ grade 3 or 4), in this relapsed and/or refractory patient population.

“The safety profile emerging from this pediatric study is encouraging.  AUTO1 was well-tolerated and we did not see severe cytokine release syndrome or neurotoxicity seen in other ALL programs,” said Sara Ghorashian, PhD, Molecular and Cellular Immunology Section, UCL Great Ormond Street Institute of Child Health and a co-author of the paper.  “It is very promising to see these strong remission rates and excellent CAR T cell expansion and persistence, which give us hope that AUTO1 could improve outcomes for these patients.”

“The publication in Nature Medicine is a nice validation of our AUTO1 pre-clinical and Phase 1 clinical data,” said Dr. Christian Itin, chairman and chief executive officer of Autolus.  “AUTO1 CAR- T cells are designed to effectively engage leukemic cells while avoiding excessive immune stimulation. This profile results in an improved safety profile compared to current treatments, while achieving a high level of clinical activity.  We are currently testing the activity of AUTO1 in adult patients who typically are even more susceptible to severe immunological adverse events than pediatric patients.”

https://www.globenewswire.com/news-release/2019/09/03/1909704/0/en/Autolus-Announces-Publication-in-Nature-Medicine-of-Data-Supporting-the-Development-of-AUTO1-for-Treatment-of-Patients-with-ALL.html