Amgen has announced positive topline results from a phase 3 study evaluating the safety and efficacy of ABP 959 – a biosimilar candidate to Alexion’s Soliris (eculizumab) – in adult patients with paroxysmal nocturnal haemoglobinuria (PNH).
PNH is a rare, acquired, life-threatening disease of the blood characterised by intravascular haemolytic anaemia, bone marrow failure and thromboembolic episodes, and is associated with a significant increase in mortality, development of arterial and venous thromboembolic episodes, visceral organ damage and a rapid deterioration in quality of life.
The most widely used drug to treat PNH is Soliris, the first therapy approved for the treatment of patients with PNH to reduce haemolysis, and approved for the treatment of patients with PNH in nearly 50 countries worldwide, including the US, EU and Japan.
The phase 3 DAHLIA study was a randomised, double-blind, active-controlled, two-period crossover study in adult patients with PNH, who have previously been treated with Soliris for at least six months. The study met its primary endpoints, showing ABP 959 to be as effective as Soliris on key measures of disease activity.
ABP 959 demonstrated no ‘clinically meaningful’ difference to Soliris at controlling intravascular haemolysis, based on its effects on lactate dehydrogenase (LDH) levels. Critically, the safety and immunogenicity profile of ABP 959 was comparable to Soliris.
Commenting on the positive results, David Reese, executive vice president of research and development at Amgen, said: “Today’s positive results with ABP 959 demonstrate similar efficacy, safety and immunogenicity as the reference product, further highlighting Amgen’s commitment to providing patients with access to high-quality, biologic therapies. We look forward to working with regulators to make this potential biosimilar option available to patients.”
Biosimilars, according to the US Food and Drug Administration (FDA), are biological products that are ‘highly similar to and have no clinically meaningful differences’ from an existing FDA-approved reference product. The drugs have no clinically significant differences in terms of safety or effectiveness from the reference product, but they have the potential to lower health care costs.
In line with this, ABP 959 has the same amino acid sequence as Soliris and has an equivalent non-clinical pharmacologic function, based on comprehensive bioanalytical assays.
The company outlined its plans to present detailed results from the study at a future medical congress, and to submit the results for publication.