AstraZeneca upbeat on Wilson disease trial, Forxiga approval

At the same time, the FTSE 100 drugmaker said ‘Forxiga’, or dapagliflozin, has been approved in Japan for the treatment of chronic kidney disease in adults with and without type-2 diabetes.

On the ALXN1840 trial, the primary endpoint gauged the daily mean area under the effect curve for directly-measured non-ceruloplasmin-bound copper (dNCC) over 48 weeks.

That “novel measure” assessed the daily mean copper mobilised from tissues, reflecting the underlying burden of the copper accumulation, the company explained.

It described Wilson disease as a rare and progressive genetic condition, in which the body's pathway for removing excess copper is compromised.

Damage from toxic copper build-up in tissues and organs leads to liver disease, psychiatric, and neurological symptoms.

ALXN1840, a potential new once-daily, oral medicine, demonstrated about three times greater copper mobilisation than standard-of-care.

The trial enrolled 214 patients, including treatment-naive participants and those who had been on standard-of-care therapy for an average of 10 or more years.

Additional analyses, including individual patient-reported outcomes and clinician-reported functional assessments, were ongoing, and would be presented at an upcoming medical meeting.

“Where existing treatments remove copper from the blood, these 48-week phase 3 results demonstrate ALXN1840's significant impact in mobilising copper from tissues,” said Marc Dunoyer, chief executive officer of AstraZeneca’s rare diseases division Alexion.

“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.”

On the approval of Forxiga by Japan's Ministry of Health, Labour and Welfare (MHLW), AstraZeneca said it was based on positive results from the ‘DAPA-CKD’ phase 3 trial.

The decision followed the priority review designation granted by the MHLW earlier in the year.

AstraZeneca described chronic kidney disease as a “serious, progressive condition” defined by decreased kidney function, often associated with an increased risk of heart disease or stroke.

The condition affects 840 million people worldwide, but diagnosis rates remained low, and up to 90% of patients were unaware they had the disease.

Forxiga is the first ever approved medicine for the treatment of the disease in Japan.

“This approval is an important step towards realising our ambition of improving outcomes for patients with chronic kidney disease,” said executive vice-president of biopharmaceuticals research and development Mene Pangalos.

“While new medicines like Forxiga advance the standard of care, we are also committed to the prevention and early detection of this often debilitating and life-threatening disease.”

At 0949 BST, shares in AstraZeneca were up 0.37% at 8,584.59.