AstraZeneca's (NASDAQ: AZN) Alexion announces positive high-level results from the FoCus Phase III trial in Wilson disease.

ALXN1840 met the primary endpoint with a statistically significant improvement in daily mean copper mobilisation from tissues, demonstrating superiority compared with standard-of-care (SoC) treatments.

The primary endpoint gauged the daily mean Area Under the Effect Curve (AUEC) for directly measured non-ceruloplasmin-bound copper (dNCC) over 48 weeks. This novel measure assesses the daily mean copper mobilised from tissues, reflecting the underlying burden of the copper accumulation.

Wilson disease is a rare and progressive genetic condition in which the body’s pathway for removing excess copper is compromised.1 Damage from toxic copper build-up in tissues and organs leads to liver disease, psychiatric and/or neurological symptoms.1

ALXN1840, a potential new once-daily, oral medicine, demonstrated approximately three times greater copper mobilisation than SoC. The trial enrolled 214 patients, including treatment-naïve participants and those who have been on SoC therapy for an average of ten or more years. Additional analyses, including individual patient-reported outcomes and clinician-reported functional assessments, are ongoing and will be presented at an upcoming medical meeting.

Marc Dunoyer, Chief Executive Officer, Alexion, said: “Where existing treatments remove copper from the blood, these 48-week Phase III results demonstrate ALXN1840’s significant impact in mobilising copper from tissues. As we advance this first innovation in Wilson disease treatment in more than 30 years, we will continue to follow these patients long term to further assess clinical impact on disease symptoms. We look forward to learning more about how we can evolve the treatment of this progressive and devastating disease.”

Dr Michael Schilsky, Medical Director of Adult Liver Transplant at Yale-New Haven Transplantation Center, Yale University, New Haven, Connecticut, US and principal investigator of the FoCus Phase III trial, said: “It is encouraging to see the effect of ALXN1840 on both treatment-naïve patients and those who have been on SoC for an average of ten or more years. The Phase III results provide evidence that tissue-bound copper remains built-up in the organs even in patients who have been on SoC therapy for many years, and the potential for ALXN1840 to provide a new approach to mobilise and safely sequester copper from tissues.”

ALXN1840 was generally well-tolerated with most reported adverse events considered mild to moderate, and no neurological worsening upon initiation of treatment was observed. In the ALXN1840 treatment group, the most frequently reported adverse event was a reversible increase in transaminase levels.

Alexion is working closely with health authorities worldwide and intends to submit these data for review in the coming months.