TORONTO - COSCIENS Biopharma Inc. (NASDAQ: CSCI) (TSX: CSCI), a biopharmaceutical company, announced unexpected results from its Phase 3 DETECT-trial of macimorelin, a diagnostic agent for Childhood Onset Growth Hormone Deficiency (CGHD). The trial did not meet its primary efficacy endpoint, according to the study protocol.
The international multicenter trial aimed to evaluate the safety and efficacy of macimorelin, which stimulates growth hormone release for diagnostic purposes. Despite the drug's proven ability to stimulate hormone release, the primary endpoint was not reached due to a high false positive rate in the comparator tests, arginine and clonidine, which affected the trial's outcome.
The DETECT-trial, involving 102 subjects aged 3 to 17 across several countries, established an 'optimal' growth hormone cut-off point of 25.59 ng/mL, significantly higher than the current 7-10 ng/mL standard, leading to a failure in sensitivity and specificity assessments.
Chief Medical Officer Nicola Ammer stated that further analysis is needed to understand the unexpected results. The company confirmed macimorelin's safety profile, consistent with previous clinical and commercial experience in diagnosing adult growth hormone deficiency (AGHD).
CEO Gilles Gagnon expressed gratitude towards the participants and clinical teams and indicated that the company would consider its action plan for macimorelin following further analysis. He also mentioned COSCIENS's commitment to focusing resources on programs and products that will drive the company's growth.
Macimorelin is currently approved in the USA and marketed in Europe for AGHD under the brand names Macrilen® and GHRYVELIN®. The DETECT-trial was the second study required for evaluating macimorelin's use in children as agreed with the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) in the company's Pediatric Investigation Plan.
Full trial result reporting is expected later this year. Information on the trial can be found on the EU Clinical Trials Register and clinicaltrials.gov. This news article is based on a press release statement.
In other recent news, COSCIENS Biopharma, formerly known as Aeterna Zentaris (NASDAQ:CSCI) Inc., has made significant strides in its operations. The company recently disclosed top-line results from its Phase 3 DETECT-Trial, a critical step in developing a diagnostic test for childhood-onset growth hormone deficiency (GHD). Additionally, COSCIENS Biopharma reported its Q2 2024 financial results and emphasized its reliance on the success of its product Macrilen™.
In the same vein, Aeterna Zentaris and Ceapro have finalized their all-stock merger, creating a combined entity with a diversified portfolio. The merger is expected to enhance the company's revenue base and development programs. The company also implemented a reverse stock split, reducing the number of issued and outstanding common shares.
These are recent developments that could have implications for the company's position in the market for pediatric endocrine diagnostics. As always, investors and interested parties are advised to consider the risks detailed in the company's filings with the SEC, which provide insight into the company's financial position and strategic direction.
InvestingPro Insights
Following the recent news on COSCIENS Biopharma Inc.'s (NASDAQ: CSCI) unexpected trial results, investors may be keen to understand the company's financial health and market performance. According to InvestingPro data, CSCI has a market capitalization of approximately $18.26 million. Despite a challenging revenue growth rate of -43.63% over the last twelve months as of Q2 2024, the company has demonstrated a gross profit margin of 81.67%, indicating a strong capability to generate profit from its sales.
InvestingPro Tips suggest that CSCI holds more cash than debt on its balance sheet, which could provide some financial flexibility in the face of the recent trial setback. Moreover, analysts predict the company will be profitable this year, which may offer a glimmer of hope to investors concerned about the company's future prospects. It's worth noting that the company does not pay a dividend, which might be a factor for income-focused investors to consider.
InvestingPro offers several additional tips for those interested in a deeper dive into COSCIENS Biopharma's financial metrics and market predictions. As of now, there are 9 more tips available, which can be found on the InvestingPro platform: https://www.investing.com/pro/CSCI. These tips could provide valuable insights for investors looking to make informed decisions about their investments in the biopharmaceutical sector.
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