dm+d

Unassigned

New Medicines

Persistent or recurrent acute myeloid leukaemia (AML) in adults - second-line

Information

New molecular entity
Intellia Therapeutics
Intellia Therapeutics

Development and Regulatory status

Phase II Clinical Trials
None
Phase II Clinical Trials
Yes
Mar 22FDA grants orphan drug designation to NTLA-5001 for the treatment of AML [3].

Category

An autologous CRISPR/Cas9-engineered T cell receptor (TCR)-T cell therapy designed for AML patients with the HLA-A*02:01 allele and whose tumors carry the Wilms’ Tumor 1 (WT1) antigen, which is highly expressed in AML and many other haematologic and solid tumours.
AML is the most common acute leukaemia in adults. The incidence of AML in the UK is 4.05 per 100,000. This is the highest in Europe. AML can occur at any age but the incidence increases with age and the median age of onset is 67 [1].
Persistent or recurrent acute myeloid leukaemia (AML) in adults - second-line
Intravenous infusion

Trial or other data

Nov 21PI/IIa study to evaluate the safety, tolerability, cell kinetics and anti-tumor activity of a single dose of NTLA-5001 in adults who have detectable AML after having received standard first-line therapy starts (NCT05066165). The study includes a dose escalation and expansion phase, with up to 54 adults recruited in the US and UK (sites in London, Leeds and Manchester - no further details provided). The dose-escalation phase of the study includes two independent arms of up to three cohorts each: Arm 1 consists of adults with AML with lower disease burden, defined as those with less than 5% blasts in bone marrow, while Arm 2 consists of adults with AML with higher disease burden, defined as those with greater than or equal to 5% blasts in bone marrow. Once a dose is identified in each arm, two expansion cohorts will be opened for further safety assessment. Cyclophosphamide and fludarabine will be administered on Day -5, -4, and -3 as intravenous infusion, prior to a single dose of NTLA-5001 given as an IV infusion. Primary outcome is safety and tolerability; collection of these data is due to complete Nov 23 [2].