AstraZeneca has announced successful results from its set of Phase III trials, evaluating the efficacy and cardiovascular (CV) safety of roxadustat in anaemia-related chronic kidney disease (CKD).

The drug, an oral first-in-class treatment, did not increase the risk of MACE, MACE+ and all-cause mortality in non dialysis-dependent (NDD) patients compared to placebo and dialysis-dependent (DD) patients compared to epoetin alfa.

For patients on dialysis for four months or less, roxadustat also reduced the risk of MACE and MACE+ by 30% and 34% respectively, as well as showing a trend towards lower risk of all-cause mortality relative to epoetin alfa.

Joris Silon, senior vice president of the company’s BioPharmaceuticals Business Unit, told PharmaTimes that it was an “overall exciting” data set, and confirmed that AZ plans to “submit to the FDA in 2019, which the team are currently working hard on.”

The data marks an important milestone for CKD-related anaemia patients, as it is the "first innovation of its kind for this market in many years.”

Joris continued, “Maybe what’s most exciting is the DD segment of the trial, as more or less 20% of patients who are taking dialysis have only recently started on dialysis, so they’re a very vulnerable group at high risk of cardiovascular event. It’s very exciting that in this population we see such a significant impact on MACE and MACE+ with roxadustat.”

The drug in question is a hypoxia-inducible factor prolyl hydroxylase inhibitor, or HIF-PHI, which is a relatively novel kind of treatment. It works by “mimicking the body’s natural response to hypoxia,” explained AZ’ global medicine leader, John Houghton.

He also explained that the results show a "good efficacy in the inflamed patients - a difficult-to-treat group - without having to increase the dose. So, when you think about efficacy; yes, we do what we say we do in terms of increasing haemoglobin, we do it very well. But, I think you also have to bare in mind those other benefits as well.

“Patients who are still suffering from their anaemia, and as you can imagine because their renal function is declining, they’re very tired, they’re fatigued. It’s a huge quality of life aspect when you can’t get off the sofa or you can’t go downstairs.

“So, in that respect they tend to be an area of unmet need because they don’t actually have that therapy made available for them. It’s not going to be an easy thing to get the backing, but we do hope that with the profile that we’re presenting today, we stand a good chance of offering these patients hope going forward.”

Earlier results showed that the drug increased haemoglobin levels, demonstrating a mean increase from baseline in haemoglobin levels averaged over weeks 28 to 52 of 1.85 g/dL in the NDD population.

Meanwhile in the DD population, roxadustat demonstrated a statistically significant mean increase from baseline in Hb levels averaged over weeks 28 to 52 with 1.22 g/dL compared to epoetin alfa.

The results were presented in an oral late-breaking abstract session at the American Society of Nephrology (ASN) Kidney Week 2019 in Washington DC.

Anaemia from CKD is associated with increased risk of hospitalisation, CV complications and death, also frequently causing significant fatigue, cognitive dysfunction and decreased quality of life.