Shield Therapeutics (Shield), an independent specialty pharmaceutical company focused on the development of mineral-derived hospital pharmaceuticals, has announced strongly positive top-line data from the pivotal Phase 3 programme of ST10 for the treatment of iron deficiency anaemia (IDA) in inflammatory bowel disease (IBD).
ST10, a novel orally-dosed form of ferric iron, delivered a mean improvement in haemoglobin levels of 2.3g/dL (p <0.0001), clearly meeting the primary endpoint of haemoglobin change after 12 weeks' treatment compared to placebo. More than 65% of treated subjects experienced normalised haemoglobin levels by week 12 and ST10 also rapidly delivered significant improvements in haemoglobin at 4 weeks (1.1g/dL, p <0.0001) and 8 weeks (1.8g/dL, p <0.0001) of therapy. From a safety perspective, ST10 did not adversely affect IBD symptoms in treated subjects and was well tolerated for the duration of exposure.
The two AEGIS protocols recruited 128 patients with anaemia secondary to either Crohn's disease or ulcerative colitis who had previously failed therapy with oral ferrous products due to intolerance and/or inadequate therapeutic benefit. Patients were recruited from expert centres in Austria, Germany, Hungary and the UK and were randomised (1:1) to receive ST10 or a matched placebo capsule. Other iron therapies were not permitted during the study. Adverse events recorded during the study were mainly gastrointestinal in nature and occurred in the ST10-treated arm with placebo-like frequency (38% of ST10-treated subjects and 40% of placebo-treated subjects). Withdrawal from the study before 12 weeks (due to adverse events or subject decision) occurred in seven ST10-treated and nine placebo-treated subjects. Patients who completed the 12-week double-blind phase of the study continued treatment in a 12-month open-label extension phase and results of this will be available later this year.
These results provide confirmatory pivotal clinical data which will form the basis of a Marketing Authorisation Application submission to the European regulatory authorities during 2014. In conjunction with data that will be generated from the study of ST10 in the treatment of IDA in pre-dialysis chronic kidney disease patients that is currently progressing, these results will also form part of a subsequent New Drug Application submission to the FDA in the USA.
Full findings from the AEGIS studies will be presented at scientific congresses in due course.
Commenting on the results, Professor Christoph Gasche, Professor of Medicine, Medical University of Vienna, Austria, said:
"I have studied the problem of anaemia in inflammatory bowel disease (IBD) patients for many years, and the data from the AEGIS studies are very encouraging. Until now there has been no acceptable oral iron treatment available for the majority of my IBD patients with iron deficiency anaemia. These results are statistically very convincing and the 2.3 g/dL rise in haemoglobin at 12 weeks together with the more than 1.1g/dL rise in haemoglobin at 4 weeks compared to placebo are clinically meaningful for anaemic patients. The safety results from this study also show that ST10 is well tolerated with just a very small number of subjects withdrawing because of adverse effects and the frequency of GI side effects was similar in the placebo and ST10 groups. My personal experience of the patients that I treated in this study fits with these results."
Also commenting on the results, Dr Tariq Ahmad, Consultant Gastroenterologist at the Royal Devon and Exeter Foundation Trust, Exeter, UK, said:
"Iron deficiency anaemia is a real problem for many of my patients. Unfortunately patients often struggle with GI side effects caused by conventional ferrous iron salts. Currently we offer such patients intravenous iron in hospital. This is costly to administer, inconvenient for patients and associated with a small but significant risk of anaphylaxis. The exciting AEGIS Phase 3 data suggests that ST10 will provide an effective, safe and more convenient alternative to intravenous iron for this group of patients as demonstrated by ST10 keeping our patients out of the infusion room by correcting and maintaining their haemoglobin in the normal range."
Shield's Founder and Chief Executive Officer, Carl Sterritt, commented:
"Delivering such resoundingly positive findings from these pivotal trials of ST10 in IBD is a tremendous achievement for Shield; and the whole team is indebted to the patients and investigators who participated in the protocols. Our next aim is to make this therapy available as quickly as possible on a commercial basis to as wide an audience of prescribers and patients as we can, so we will be working diligently with regulatory authorities to achieve this goal.
"The results reflect an important milestone in the development of ST10 as the only effective, low-dose oral iron-replacement therapy without the significant GI side-effects of ferrous iron or the high risks associated with intravenous administration of iron. ST10 presents a significant opportunity for Shield to develop a paradigm changing treatment that will address a broad range of indications with a strong commercial potential and we look forward to rapidly advancing the programme."
Shield Therapeutics is soon to commence a Phase 3 study of ST10 for the treatment of iron deficiency anaemia in patients with chronic kidney disease.