Shield Therapeutics’ success in showing that its oral iron supplement can treat anaemia in inflammatory bowel disease will not necessarily trigger the active licensing talks pursued by most small venture-backed biotechs following a trial win. A solo launch or acquisition are the outcomes sought by the UK-based group as it prepares to file for approval in Europe and launch a second clinical study to support a US submission, says its chief executive, Carl Sterritt.
While not ruling out a licensing deal, Mr Sterritt tells EP Vantage that the group is considering raising more cash to build a commercial team for ST10 if a suitable buyout offer does not appear. “My primary goal is to return value to shareholders as quickly as possible,” he says. “The first two options (solo launch or acquisition) would provide that.”
If pivotal trial results are anything to go by, ST10 has a good shot at making it to the market. UK-based Shield hopes to offer it as an alternative to oral and intravenous iron supplementation in several settings, but the first target has been inflammatory bowel disease patients who are intolerant of oral iron supplements because they represent the most difficult anaemia population – anaemia is a frequent complication of these diseases. Chronic kidney disease and dialysis patients are in the group’s longer-term plans.
ST10 is formulated to allow absorption at a significantly lower dose than existing medications and thus could avoid many of the gastrointestinal side effects.
The Aegis trials, the results of which were announced today, pitted ST10 against placebo in 128 patients with Crohn’s disease or ulcerative colitis – at 12 weeks it resulted in a statistically significant 2.3g/dl improvement in haemoglobin levels in patients when compared with placebo. Statistically significant improvements were measured at four and eight weeks, and more than 65% of patients taking ST10 achieved normalised haemoglobin levels at week 12.
The data should be sufficient to submit a marketing authorisation application to the European Medicines Agency, Mr Sterritt says. The group will submit to US authorities once it has also completed a trial in chronic kidney disease, a study scheduled to begin recruiting patients in the second half of 2014, the same timeframe in which the company should be able to submit the European application.
While the trial was not intended to demonstrate that ST10 was better than or at least non-inferior to the IV iron treatments, the hope is that the magnitude of improvement will encourage physicians to look to it as an alternative.
“IV iron works; there’s no doubt about that. That’s demonstrated by the way the market’s expanding,” Mr Sterritt says. This claim is backed up by the growth of such supplements as Galenica’s Ferinject, forecast to grow at 23% a year between 2012 and 2018, according to EvaluatePharma’s consensus.
However, while they require less frequent dosing than the twice daily of ST10, IV drugs like Venofer carry with them the risk of hypersensitivity reactions: “It’s a low risk but, if you’re the unfortunate patient who experiences it, it’s very real.”
It is on this anaphylaxis issue that Rockwell Medical Technologies is hoping to build the case for Triferic, as an example (Rockwell steels itself for review of iron supplement, September 9, 2013).
Furthermore, Mr Sterritt touts an economic argument – a self-administered oral drug represents a cost savings over an IV one that requires a hospital or clinic visit, something that should appeal to healthcare payers.
More shots on goal
Moving into the chronic kidney disease population is the next step, and the trial is required before the FDA will look at a new drug application. For now, the pre-dialysis population will be the Shield’s focus in part because so many dialysis patients make frequent clinic visits and where they receive IV iron supplementation, Mr Sterritt says.
Home dialysis patients, though a small part of the overall kidney failure population, is a potential market, and is growing quickly, he adds. However, any clinical work in those patients will wait until the pre-dialysis study has been completed.
The potential to widen the market, and quickly, could serve as an enticement for any acquisitive company, he notes: “If this asset was held by a large pharma, it could do these things in parallel.”
|Aegis-1||Anaemia in ulcerative colitis||NCT01340872|
|Aegis-2||Anaemia in Crohn's disease||NCT01352221|