Shield Therapeutics has announced positive top-line results from its Phase III clinical trial evaluating the efficacy and safety of Accrufer (ferric maltol) in pediatric patients with iron deficiency. The study achieved its primary endpoint, demonstrating a statistically significant increase in hemoglobin levels from baseline to week 12.
The Phase III trial was a multi-center, open-label study that enrolled pediatric patients aged 1 to 17 years with confirmed iron deficiency. Participants received Accrufer orally twice daily for 12 weeks. The primary endpoint was the change in hemoglobin levels from baseline to week 12. Secondary endpoints included changes in other iron stores, such as ferritin and transferrin saturation, as well as safety and tolerability.
Key Findings
The study demonstrated a statistically significant increase in hemoglobin levels from baseline to week 12 (p<0.001). Secondary endpoints also showed improvements in iron stores. Accrufer was well-tolerated, with a safety profile consistent with previous studies in adults. The most common adverse events were gastrointestinal-related and mild to moderate in severity.
Clinical Significance
Iron deficiency is a common nutritional disorder in children, affecting growth, development, and overall health. Current treatment options, such as ferrous sulfate, are often associated with gastrointestinal side effects, leading to poor adherence and suboptimal outcomes. Accrufer offers a novel mechanism of action with improved tolerability, potentially addressing the unmet medical need in pediatric iron deficiency.
Accrufer: A Novel Oral Iron Therapy
Accrufer is a non-salt based oral iron product. It has demonstrated efficacy and safety in adults with iron deficiency anemia and is approved in the United States and Europe for this indication. The positive results from the pediatric Phase III trial support the potential of Accrufer as a valuable treatment option for children with iron deficiency.
