Shield Therapeutics PLC (AIM:STX, OTCQX:SHIEF), a UK-based pharmaceutical company specialising in treatments for iron deficiency, announced on Wednesday successful results from a pivotal phase III clinical trial that could herald a broader indication for its oral iron deficiency anaemia treatment.
The drug, known commercially as Accrufer or Feraccru (scientifically referred to as ferric maltol), demonstrated significant efficacy, safety, and tolerability in paediatric patients.
The study marks the completion of Shield's comprehensive development programme, which is aligned with regulatory requirements set by both the European Medicines Agency (EMA) and the United States Food and Drug Administration (FDA).
The company plans to submit the full dataset for peer review and publication. Shield aims to file for approval to use the medicine in children older than one month in both Europe and the US by the first half of 2025.
A 'delighted' Anders Lundstrom, interim CEO, said: "We will now initiate the work with the regulatory applications to be able to expand the patient population who can benefit from a safe and effective oral iron treatment."
Financially, the company stands to receive €1 million from its European partner, Norgine, contingent upon the EMA's Paediatric Committee granting the indication. This payment reflects development milestones achieved under their partnership agreement.
In the trial, children aged between 2 and 17 who were treated with ferric maltol showed an average increase of 1.25 grams per decilitre (g/dL) in haemoglobin levels over 12 weeks. Haemoglobin is the protein in red blood cells responsible for carrying oxygen throughout the body.
Infants treated with the medicine exhibited an even higher average increase of 1.77 g/dL. For comparison, patients receiving ferrous sulphate, a standard iron supplement, had an average haemoglobin increase of 1.15 g/dL.
Importantly, none of the patients in the ferric maltol group discontinued treatment due to adverse events, which are unwanted side effects that emerge during clinical trials. In contrast, one patient in the ferrous sulphate group did discontinue for this reason.
Moreover, no serious adverse events related to the treatment were reported in the ferric maltol group. Only two patients aged between 2 and 17 reported treatment-related side effects, compared to four patients in the same age group receiving ferrous sulphate.
The absence of serious adverse events and the low incidence of side effects suggest that ferric maltol is well-tolerated among paediatric patients.
Iron deficiency anaemia occurs when the body lacks sufficient iron to produce healthy red blood cells, leading to fatigue, weakness, and other health issues. It is particularly concerning in children, as it can affect growth and cognitive development.