dm+d

Unassigned

New Medicines

Acute lymphoblastic leukaemia (ALL) in adults

Information

New molecular entity
Autolus
Autolus

Development and Regulatory status

Phase II Clinical Trials
None
Phase II Clinical Trials
Yes
Aug 21Obecabtagene autoleucel receives Promising Innovative Medicine (PIM) designation from the MHRA for the treatment of relapsed / refractory (r/r) B-cell ALL in patients 18 years and older [7].
Jun 21Autolus Therapeutics receives innovative licensing and access pathway (ILAP) designation from the MHRA for obecabtagene autoleucel for treatment of relapsed / refractory (r/r) B-cell ALL in patients 18 years and older [7].
Apr 21EMA grant AUTO1 PRIME designation for treatment of adult ALL [6].
Apr 20FDA accepts the Investigational New Drug (IND) application for AUTO 1 for the treatment of adults with ALL. The IND allows initiation of the first pivotal study, AUTO1-AL1 in the US [3].
Nov 19Granted orphan drug status in US for treatment of ALL (precursor cell lymphoblastic leukaemia lymphoma) [3].

Category

CD19-targeting CAR T cell therapy with fast target binding off-rate designed to minimize excessive activation and associated cytokine release, which may reduce toxicity. In addition, the fast off-rate may reduce T cell exhaustion and enhance persistence.
Global incidence is about 3 per 100,000 population, with about 3 out of 4 cases occurring in children aged under 6 years. ALL represents 12% of all leukaemia (but 80% in children) [1].
Acute lymphoblastic leukaemia (ALL) in adults
Intravenous

Trial or other data

Nov 21Company announces that one-month complete response rate and safety data from initial 16 patients in the PIb cohort of the PI/II FELIX trial (AUTO1-AL1; NCT04404660) is consistent with data reported from the academic ALLCAR19 study (in which data from Cohort A involving patients with acute lymphoblastic B cell leukaemia showed that of 20 patients evaluable for efficacy, 17 (85%) of patients achieved minimum residual disease-negative complete remission at one month) [7].
Nov 20PI/II AUTO1-AL1 (NCT04404660) is recruiting [2].
Nov 20Autolus expects to announce long-term data from the PI ALLCAR19 trial at ESMO [5].
Mar 20Autolous announces data from the PI ALLCAR19 trial (NCT02935257). As of the most recent cut-off date of November 25, 2019, of the 15 patients evaluable for efficacy, 13 patients (87%) achieved minimal residual disease, or MRD, negative complete responses at one month and all patients had ongoing CAR T cell persistence at last follow-up. 10 of the 15 evaluable patients (67%) remain disease-free at a median follow up of 11 months (range of 0.5 month - 21 months). Of the 16 patients dosed, ten patients were dosed with AUTO1 manufactured using our semi-automated, fully enclosed system for manufacturing. In this cohort, nine patients were evaluable and achieved MRD negative complete responses of 100%. The median follow-up in this cohort was 6.7 months (range of 1.1 month-14.5 months). The event free survival or EFS and overall survival or OS data are preliminary considering the small number of patients [4].
Mar 20Pivotal PI/II trial to evaluate AUTO 1 in adults with ALL who have previously been treated and subsequently progressed starts (AUTO1-AL1; NCT04404660). 145 patients will be enrolled in the US and UK (University College London Hospitals, Kings College Hospital in London, Manchester Royal Infirmary, Queen Elizabeth University Hospital in Glasgow, Freeman Hospital in Newcastle upon Tyne, University Hospitals Bristol and Weston NHS Foundation Trust). Primary outcome is overall response rate and safety; collection of these data is due to complete Mar 23 [2].