While all eyes have been on the commercial progress of Efmody in the rare congenital adrenal hyperplasia (CAH) disorder, post-approval in Europe, its ability to mimic the human physiological circadian release of cortisol makes it relevant also to those with adrenal insufficiency (AI).
The release this morning, reporting that the first patient has been dosed in the European CHAMPAIN study, is a reminder that this is a significant opportunity for Efmody. While patients may not suffer from overnight androgen build up, they still suffer from the risk of hyper and hypocortisolism despite available hydrocortisone replacement therapy. Hypocortisolism runs the risk of adrenal crises, while hypercortisolism can result in fatigue, low libido, and cognitive issues. Moreover, there remains significant inter and intrapatient variability with no easy diagnostic means of ensuring that patients are adequately controlled.
CHAMPAIN will compare Efmody versus Plenadren, an existing modified-release hydrocortisone preparation that was also designed to provide a better solution for AI patients. However, the available evidence suggests that Plenadren does not replicate the early morning cortisol peak - the primary endpoint of CHAMPAIN. Unlike Plenadren, Efmody successfully provides physiological peak hydrocortisone levels on awakening. Consequently, we believe there is a reasonable prospect of Efmody delivering a favourable comparison versus Plenadren with a primary endpoint measuring cortisol levels in the morning. A positive result from CHAMPAIN should position Efmody appropriately to best capitalise on its differentiated profile.
The release suggests that CHAMPAIN will read out in H2 2022, with potential approval in 2023. Although this is also an orphan disorder affecting 298,000 patients in the UK and Europe, it is a significantly larger market than CAH representing a market opportunity of approximately $1.9bn. Approval for the AI indication is also important to the extent that it should also help deliver significant operating leverage being sold through the existing European commercial infrastructure.
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Diurnal Group is a client of Calvine Partners, and as such, this publication is not independent and should be considered a marketing communication under FCA Rules. None of the information contained in this publication should be considered as any form of advice.