Autolus presents initial AUTO1 data at AACR

1st April 2019

Initial Results from Autolus Therapeutics’ ALLCAR19 Phase 1/2 Trial in Adult Acute Lymphoblastic Leukemia Presented at the AACR Annual Meeting

Initial results from the trial show 88% molecular complete response at one month with

well-tolerated safety profile

Management to Hold Conference Call on April 2, 2019 at 8:00am ET / 1:00pm BST

LONDON, UK, April 1, 2019 -- Autolus Therapeutics plc (Nasdaq: AUTL), a clinical-stage biopharmaceutical company developing next-generation programmed T cell therapies for the treatment of cancer, announced that Claire Roddie MB, PhD, FRCPath, honorary senior lecturer, Cancer Institute, University College London (UCL), presented today initial data from the ongoing Phase 1/2 ALLCAR19 trial of AUTO1 in adult acute lymphoblastic B cell leukemia (ALL) as a late-breaking poster presentation at the American Association of Cancer Research (AACR) Annual Meeting 2019 in Atlanta, Georgia.

The outlook for adult patients with ALL is poor, and no CD19 CAR-T therapy has been approved in this indication. Key challenges seen in clinical studies with other CD19 CAR T therapies in adult ALL are considerable toxicity associated with severe cytokine release syndrome (CRS) and high-grade neurological toxicity.

AUTO1 uses a novel CD19 binder that allows the CAR T cells to disengage rapidly after target cell encounter and kill. Data from the ongoing Phase 1/2 CARPALL trial of AUTO1 in pediatric ALL presented at the EHA CAR-T Meeting in February 2019 has shown that AUTO1 has a well-tolerated safety profile without inducing high grade CRS in pediatric patients.

As of the data cutoff date of March 18, 2019 in the ongoing Phase 1/2 ALLCAR19 trial of AUTO1 in adult ALL patients, 13 patients were leukapheresed, and products for 12 patients were manufactured, including 7 with Autolus’ semi-automated, fully enclosed manufacturing process. Two patients are pending infusion. Among the 10 infused patients to date, the median age is 41 and 70% were male, with median lines of treatment of 4 (the range is 2-7). Five of the ten treated patients had ≥ 50% BM blasts and were considered to be high-risk for severe CRS. Patients received a split dose based on disease burden for a total dose of up to 410 million cells.

Safety results

Using the Lee criteria, there were no patients with severe CRS (≥ Grade 3), and 2 of 10 patients (20%) with Grade 2 CRS. Tocilizumab was used in 2 of 10 patients (20%). None of the patients were admitted to intensive care due to CRS. One patient developed delayed G3 neurotoxicity following high levels of CAR T expansion, which was quickly reversed with steroids. Four patients died on study, two due to progression of leukemia and two due to sepsis, a common complication of advanced ALL.

Efficacy results

Nine patients were evaluable for response at 1 month and 8 (88%) had a molecular complete response. One patient died of sepsis before the one-month evaluation point. At a median follow up of 5 months (range 0.62-10.6 months), 6/10 patients are alive and continue to be in molecular remission. There continues to be evidence of ongoing B cell aplasia and CAR T persistence.

“AUTO1 delivered promising early remission rates, CAR T cell expansion and persistence in this adult ALL trial cohort. Despite enrolling patients with high tumor burden, the safety profile in the trial appears to compare very favorably to other CD19 CARs and is consistent with the safety profile of AUTO1 observed in pediatric patients in the CARPALL trial.”

Dr Claire Roddie MB, PhD, FRCPath, honorary senior lecturer, Cancer Institute, University College London (UCL)

“These data from the ALLCAR19 study of AUTO1 in relapsed refractory ALL, while early, are extremely encouraging, with a high response rate we now associate with CAR T cell therapies, but with a potentially improved safety profile. If AUTO1 continues to be associated with a lower incidence of adverse events with additional patients treated, this could represent an important advance for more vulnerable adult patients, as side effects of these therapies, including serious cytokine release syndrome and neurotoxicity, limit our ability to treat these individuals."

Krishna Komanduri, M.D., Kalish Family Chair in Stem Cell Transplantation and Director, Adult Stem Cell Transplant Program at the Sylvester Comprehensive Cancer Center at the University of Miami Miller School of Medicine.

“The strong persistence of the CAR T cells over time, coupled with the low frequency of severe CRS events seen in these patients, represent encouraging initial data for AUTO1 in relapsed/refractory adult ALL. We expect AUTO1 in adult ALL to move into a registration trial towards the end of this year.”

Dr. Christian Itin, Chairman and CEO OF Autolus

For information about the ALLCAR19 trial, visit

https://clinicaltrials.gov/ct2/show/NCT02935257?term=ALLCAR19&rank=1