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).

Go deeper with GlobalData

Go deeper with GlobalData

The gold standard of business intelligence.

Find out more

Discover B2B Marketing That Performs

Combine business intelligence and editorial excellence to reach engaged professionals across 36 leading media platforms.

Find out more

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.

GlobalData Strategic Intelligence

US Tariffs are shifting - will you react or anticipate?

Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis.

By GlobalData
“A total of 80 patients were enrolled for the trial across 34 sites in 14 countries worldwide.”

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.

Clinical Trials Arena Excellence Awards - Nominations Closed

Nominations are now closed for the Clinical Trials Arena Excellence Awards. A big thanks to all the organisations that entered – your response has been outstanding, showcasing exceptional innovation, leadership, and impact.

Excellence in Action
YPrime won the Innovation award for AI in Clinical Trials and the Environmental award for Sustainable Trials, thanks to its eCOA, IRT and eConsent platforms. Explore how purpose-built AI, paperless workflows and circular hardware practices are reshaping timelines, data quality and ESG performance in clinical research.

Discover the Impact