Results from Phase 3 paediatric study

Data will be used to support filing obligations with the US FDA and the European EMA for a paediatric indication in children older than 1 month for ACCRUFeR®/FeRACCRU® in H1 2025

London, UK, 25 September 2024: Shield Therapeutics plc (LSE: STX), the commercial stage pharmaceutical company specialising in iron deficiency, announces results from the Phase 3 paediatric clinical trial (FORTIS/ST10-01-305), confirming the efficacy, safety, and tolerability of the new oral liquid paediatric suspension in children with iron deficiency anaemia (IDA).

This trial is the final study in the comprehensive development program supporting the paediatric investigation plan (PIP/PSP) agreed with the European EMA and the US FDA. The full data set, including pharmacokinetic (PK) sub-study parameters, will be submitted for peer-review and subsequent presentation/publication. The data will be used to support a filing with the US FDA and European EMA for a paediatric indication in children older than 1 month for ACCRUFeR®/FeRACCRU® in H1 2025.

Additionally, Shield is due a total of €1 million as development milestones from its European partner, Norgine BV, upon the compliance notification of the PIP by the Pediatric Committee (PDCO) and the granting of the paediatric indication by EMA.

All primary endpoints were met, and the outcomes were:

• Change in Hb concentration from baseline to week 12: Patients in the ferric maltol group demonstrated a highly clinically relevant mean change in Hb concentration at week 12 compared to baseline.

o The mean change was 1.25 g/dl from baseline to week 12 in the 2 - 17-year-old cohort and was 1.77 g/dl from baseline to week 12 in the infants

o The mean change was 1.15 g/dl from baseline to week 12 in patients in the ferrous sulphate group.

• Frequency of discontinuations from the study because of Treatment Emergent Adverse Events (AEs): No patients in the ferric maltol group discontinued from the study as a result of AEs.

o In the 2 - 17-year-old cohort, no patient (0/31; 0%) discontinued the ferric maltol treatment due to an AE compared to one patient (1/30; 3.3%) who discontinued from the ferrous sulphate arm.

o None of the infants discontinued ferric maltol treatment due to an AE.

• Frequency of AEs/serious adverse events (SAEs): No patient in the ferric maltol group reported a treatment related SAE and two (2) patients reported treatment related AEs.

o No treatment related SAEs were reported in either group.

o In the 2 - 17-year-old cohort, treatment related AEs were reported in 2 out of 31 patients treated with ferric maltol compared to 4 out of 30 patients treated with ferrous sulphate.

o None of the ferric maltol-treated infants reported treatment related AEs .

Anders Lundstrom, interim CEO commented: "We are delighted that the results in this important ACCRUFeR®/FeRACCRU® Paediatric Phase 3 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."

Professor Richard Russell, a FORTIS Principal Investigator, commented: "These results indicate that the newly developed paediatric liquid formulation will provide a welcome additional well-tolerated and effective therapeutic option for the treatment of IDA in young children and adolescents."

Clinically low iron levels (aka iron deficiency, ID) can cause serious health problems for adults of all ages, across multiple therapeutic areas. Together, ID and ID with anemia (IDA) affect about 20 million people in the US and represent a $2.3B market opportunity. As the first and only FDA approved oral iron to treat ID/IDA, ACCRUFeR® has the potential to meet an important unmet medical need for both physicians and patients.

Details of the FORTIS/ST10-01-305 Phase 3 study

The open label randomized Phase 3 study included children aged 1 month to 17 years with mild to moderate iron-deficiency anaemia (IDA), who also had serum ferritin levels below 30 μg/L or ferritin levels below 50 μg/L and transferrin saturation below 20%. Children aged 2 to 17 years were randomized 1:1 to receive either ferric maltol (N=31) or ferrous sulphate (N = 30). Children 1 month to under 2 years (N=4, and 3 were treated) were all assigned to receive ferric maltol treatment. The study was not powered to detect a statistical difference in primary endpoints between ferric maltol and the ferrous sulphate comparator group.