Aeterna Zentaris Announces Last Patient Last Visit in Pivotal DETECT-Trial for the Diagnosis of Childhood-Onset Growth Hormone Deficiency

Aeterna Zentaris Inc. recently announced the clinical completion of its Phase 3 safety and efficacy study AEZS-130-P02 (the “DETECT-trial”) evaluating macimorelin for the diagnosis of Childhood Onset Growth Hormone Deficiency (CGHD).

The company confirms that the last patient conducted the last study visit successfully in Europe on June 13, 2024. The DETECT-trial is a multicenter, open-label trial to investigate the efficacy and safety of a single oral dose of 1.0 mg/kg macimorelin acetate as growth hormone stimulation test (GHST) in pediatric patients with suspected growth hormone deficiency (GHD). The study enrolled 100 subjects in Europe and North America. The company remains on track to report top-line data for the DETECT-trial in Q3 2024.

Gilles Gagnon, Chief Executive Officer of the company, said “The completion of the DETECT-trial represents a major milestone for the company. In addition to the already approved indication for adults, the successful development of macimorelin as a unique diagnostic tool for the detection of growth hormone deficiency in children would represent a major benefit for patients while significantly expanding this market in endocrinology. On behalf of the company, I would like to thank the patients and clinical staff for their continued efforts to bring this study across the finish line as well as our shareholders for their continued support. Looking ahead, we are eagerly anticipating the presentation of the top-line data as well as the full study results in the third quarter of this year.”

The DETECT-trial is the second and final study needed evaluating the potential use of macimorelin as diagnostic test in children as agreed upon with the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) in the company’s Pediatric Investigation Plan.

For more information about the DETECT-trial, please visit EU Clinical Trials Register and reference EudraCT #2018-001988-23 and clinicaltrials.gov identifier NCT04786873.

Macimorelin, an oral drug used for the diagnosis of adult growth hormone deficiency (AGHD) is approved for marketing under the brand name GHRYVELIN in the European Economic Area and Macrilen in the US, South Korea, and Israel.

Macimorelin (Macrilen; GHRYVELIN), a ghrelin receptor agonist, is an orally administered peptidomimetic molecule that stimulates the secretion of growth hormone from the pituitary gland. Stimulated growth hormone levels are measured in blood samples taken after oral administration of macimorelin for the assessment of AGHD. Approval of macimorelin for use in adult was granted by the FDA in 2017 and by the EMEA in 2019, based on Phase III data showing that oral macimorelin provides accuracy comparable to that of standard insulin tolerance testing (ITT), but has a more favorable safety profile compared to ITT. Oral macimorelin also reduces false positive test results, helping to avoid unnecessary treatment of patients.

Aeterna is a specialty biopharmaceutical company engaged in the development and commercialization of a diverse portfolio of pharmaceutical and diagnostic products, including those focused on areas of significant unmet medical need. One of Aeterna’s lead products is macimorelin (Macrilen; Ghryvelin), the first and only US FDA and European Commission approved oral test indicated for the diagnosis of adult growth hormone deficiency (AGHD). Aeterna is also engaged in the development of therapeutic assets and proprietary extraction technology, which is applied to the production of active ingredients from renewable plant resources currently used in cosmeceutical products (ie, oat beta glucan and avenanthramides which are found in leading skincare product brands like Aveeno and Burt’s Bees formulations) and being developed as potential nutraceuticals and/or pharmaceuticals.

The company is listed on the NASDAQ Capital Market and the Toronto Stock Exchange, and trades on both exchanges under the ticker symbol AEZS. For more information, visit www.zentaris.com.