COSCIENS Biopharma Reports Phase 3 Results for Childhood Growth Hormone Deficiency Trial

COSCIENS Biopharma Inc., a specialized biopharmaceutical company, revealed the top-line results from its Phase 3 safety and efficacy study AEZS-130-P02, referred to as the DETECT-trial. This study assessed macimorelin for diagnosing Childhood Onset Growth Hormone Deficiency (CGHD). The results pointed out that although macimorelin successfully stimulated growth hormone release, it did not meet the primary endpoint as per the study protocol. The comparison tests, arginine and clonidine, appeared to contribute to a high false positive rate, impacting macimorelin’s efficacy outcome. 

The DETECT-trial was an international study conducted in multiple countries, including the USA, Germany, and Italy, and enrolled 102 subjects aged 3 to 17 years. The protocol included a macimorelin test at the initial visit and subsequent visits, with standard growth hormone stimulation tests (GHSTs) using arginine and clonidine conducted in between. An independent expert panel adjudicated the final growth hormone deficiency status of each child based on the collected medical data and GHST outcomes.

Nicola Ammer, Chief Medical Officer of COSCIENS, noted that while macimorelin's safety profile was confirmed, the high growth hormone stimulation seen in many subjects diagnosed as deficient by arginine and clonidine tests was unexpected. The team plans to delve deeper into the detailed data to understand these anomalies.

The study's results indicated an optimal growth hormone cut-off point of 25.59 ng/mL, much higher than the standard 7-10 ng/mL. This high cut-off point led to failures in sensitivity and specificity assessments, which are crucial for determining a diagnostic test's accuracy in identifying ill and healthy subjects.

CEO Gilles Gagnon remarked that while further analysis and discussions with health authorities are anticipated, macimorelin remains approved in the USA and marketed in Europe for diagnosing adult growth hormone deficiency (AGHD). The company will consider its future strategy for macimorelin following additional analyses of the DETECT-trial and will continue to prioritize its pipeline to drive growth.

The DETECT-trial was the second required study under agreements with the U.S. FDA and the European Medicines Agency (EMA) as part of COSCIENS' Pediatric Investigation Plan. Complete trial results are expected to be published later in the year.

Macimorelin, marketed as GHRYVELIN® in Europe and Macrilen® in the USA, South Korea, and Israel, is an orally administered ghrelin receptor agonist. It stimulates growth hormone secretion from the pituitary gland, measured through blood samples. Macimorelin was approved for diagnosing AGHD by the FDA in 2017 and by the EMA in 2019, based on Phase III data showing its accuracy and favorable safety profile compared to insulin tolerance testing (ITT).

COSCIENS Biopharma Inc. focuses on developing and commercializing a diverse range of pharmaceutical and diagnostic products. One of its leading products is macimorelin, the first and only oral test approved by both the U.S. FDA and the EMA for diagnosing AGHD. The company is also developing therapeutic assets and proprietary extraction technology for producing active ingredients from renewable plant resources, used in cosmeceuticals and potentially in nutraceuticals and pharmaceuticals. COSCIENS is listed on the NASDAQ Capital Market and the Toronto Stock Exchange under the ticker symbol “CSCI”.

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