Shield Therapeutics has announced positive results from its Phase 3 FORTIS/ST10-01-305 trial evaluating the efficacy and safety of its oral liquid suspension of ferric maltol in pediatric patients with iron deficiency. The trial met all primary endpoints, demonstrating a clinically relevant increase in hemoglobin (Hb) levels over 12 weeks. These findings pave the way for regulatory submissions to the US FDA and the EMA in Europe for a pediatric indication in children older than one month, expected in the first half of 2025.
Efficacy and Safety Data
The study revealed significant improvements in Hb levels across different age groups. In children aged 2 to 17, the mean Hb increase was 1.25 grams per deciliter (g/dL). Infants showed an even more pronounced increase of 1.77 g/dL. Comparatively, the ferrous sulphate group, a common treatment for iron deficiency, saw a mean increase of 1.15 g/dL. Importantly, the ferric maltol group demonstrated excellent tolerability, with no patients discontinuing the study due to treatment-related adverse events.
Regulatory Pathway and Commercial Implications
Shield Therapeutics intends to use the data from the FORTIS/ST10-01-305 trial to support its regulatory filings for pediatric use in both the US and Europe. The company anticipates receiving a €1 million development milestone payment from its European partner, Norgine, following compliance notifications and approval of the pediatric indication by the EMA. Anders Lundstrom, interim chief executive officer, stated, "We are delighted that the results in this important Accrufer/Ferracru paediatric phase three study show similar levels of efficacy and safety as in prior trials with adults. 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."
Current Iron Deficiency Treatment Landscape
Iron deficiency is a prevalent condition, particularly in young children, and can lead to anemia and impaired development. Current treatments often involve ferrous sulphate, which can be associated with gastrointestinal side effects, leading to poor adherence. The availability of a safe and effective oral iron treatment with good tolerability, such as Shield Therapeutics' ferric maltol suspension, could significantly improve outcomes for pediatric patients with iron deficiency.
