AstraZeneca, along with its global biologics research and development arm MedImmune, has reported positive results from the Phase III 1053 clinical trial that investigated moxetumomab pasudotox to treat patients with relapsed or refractory hairy cell leukaemia (HCL).
The single-arm, multi-centre trial met its primary endpoint of durable complete response (CR).
It has been designed to evaluate the efficacy, safety, immunogenicity and pharmacokinetics of moxetumomab pasudotox monotherapy in HCL patients who have received at least two prior therapies.
A total of 80 patients were enrolled for the trial across 34 sites in 14 countries worldwide.
The trial’s secondary outcome included overall response rate, relapse-free survival, progression-free survival, time to response, safety, pharmacokinetic and immunogenic potential.
Findings of the trial showed that moxetumomab pasudotox demonstrated a 75% objective response (OR) rate, a 41% complete response (CR) rate, and a 30% durable CR rate.
In addition, 73% of the patients with a complete response had a durable response and achieved a negative minimal residual disease (MRD) status.
AstraZeneca Global Medicines Development executive vice-president and chief medical officer Sean Bohen said: “Moxetumomab pasudotox is an investigational, first-in-class immunotoxin which we believe has the potential to advance outcomes for patients with relapsed or refractory hairy cell leukaemia, a condition with a high unmet need.
“It is also the first agent to be submitted for regulatory review from our Antibody Drug Conjugates platform, and as such demonstrates our commitment to developing novel treatments for blood cancer.”
HCL is a rare, incurable slow-growing leukaemia that leads the bone marrow to overproduce abnormal B-cells or lymphocytes.
It can cause serious and life-threatening conditions, such as infections, bleeding and anaemia.