Diurnal has provided a business update this morning that reflects the broad range of clinical studies ongoing in support of its emerging endocrinology franchise.
While all eyes are on the European Efmody roll out in congenital adrenal hyperplasia (CAH) for now, the important US market is also in sight. The US (and Japan) Phase 3 trial (CONnECT) design is secured under the auspices of a Special Protocol Assessment (SPA), and this should reduce risk and increase the probability of success for Efmody in the US. Today's release confirms that plans are on track, with the first clinical study site recently opened to recruitment in the US and enrolment anticipated shortly. The story is much the same in Japan, with ethical and regulatory approvals gained and enrolment expected in early 2022. As we have noted previously, the design of CONnECT has benefited from the previous European Phase 3 study. As a result, CONnECT is a double-blind, 52-week study with a biochemical responder analysis versus immediate-release (IR) hydrocortisone in a non-inferiority design as the primary endpoint. Secondary endpoints should also provide important information comparing Efmody with IR hydrocortisone on key measures, including steroid-sparing, fertility, body weight and quality of life. Results from CONnect are anticipated in early 2024.
Sticking with the adrenal disorder franchise, the larger adrenal insufficiency (AI) opportunity beckons, with no overnight androgen complications suggesting a much more straightforward replacement therapy proposition for Efmody compared to CAH. As Diurnal seeks to position Efmody in the AI indication optimally, the Phase 2 CHAMPAIN trial will study Efmody's ability to deliver higher concentrations of cortisol versus Plenadren (modified-release hydrocortisone) in the morning (primary endpoint). Secondary endpoints will include fatigue and quality of life measures, as well as looking at the response to therapy with respect to cortisol levels in the morning reaching a predefined level. This morning's update reports that the first clinical study site for CHAMPAIN was opened for recruitment in the UK in December 2021, with Germany awaiting final ethics committee approval. Reassuringly, the CHAMPAIN study remains on track to provide results in H2 2022, suggesting the potential for significant operating leverage in Europe from 2023 onwards. The addressable market for Efmody in AI in Europe is estimated to be over $1.9bn.
Outside of the adrenal franchise, Diurnal is progressing DITEST - the only orally available testosterone replacement therapy (TRT) for the treatment of classical hypogonadism. DITEST does not require patients to eat a high-fat meal and promises greater convenience in the treatment of hypogonadism. Diurnal has already agreed a streamlined (505(b)(2)) US development pathway with the FDA for DITEST, suggesting lower costs and a lower risk clinical opportunity. Today's release noted that application for an IND will be filed with the FDA during January, which would lead to clinical evaluation through a Phase 1 multiple ascending dose study. If its promising clinical profile is confirmed, we believe that DITEST should overcome some of the limitations associated with the profile of currently available oral TRT preparations. The addressable market for TRT is over $5bn in the US and Europe.
Clearly, there is much to look forward to as Diurnal continues to progress its burgeoning pipeline and seeks to establish its endocrinology (ex diabetes) franchise. In this regard, we look forward to more detail and discussion at the Company's forthcoming R&D Day scheduled for February 2nd 2022.
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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.