Macrilen (macimorelin) / COSCIENS Biopharma, Xeris Biopharma, Megapharm - LARVOL DELTA

Coxboost Six-Gene Proliferation Signature Improves Multiple Myeloma Risk Stratification and Identifies Therapeutic Vulnerabilities: A Tp53-Anchored ML Approach (IMS 2026) - "Drug sensitivity analysis identified three compounds — MACIMORELIN, MW-150, and BROFAROMINE — with selective activity against high-risk MM cells across all 16 lines (p< 0.001). A TP53-anchored machine learning approach identified a 6-gene proliferation signature that substantially improves MM risk stratification beyond clinical variables, is robust to overfitting, generalises externally, and exposes actionable therapeutic vulnerabilities. Prospective validation in treatment-stratification trials is warranted."

Hematological Malignancies • Multiple Myeloma • PTTG1 • STMN1 • TP53 • UBE2C

Real-World Diagnostic Performance of the Macimorelin Stimulation Test in the Diagnosis of Adult Growth Hormone Deficiency (ENDO 2026) - "Although the 5.1 ng/mL cut-off diagnoses more AGHD cases, this occurs mainly among patients with higher BMI and low pre-test AGHD probability, suggesting potential obesity-related over classification. Therefore, a tailored approach incorporating BMI and clinical likelihood may be more appropriate than a uniform threshold. In addition, the test may be shortened to 60 minutes without affecting accuracy."

Clinical • Real-world • Real-world evidence • Endocrine Disorders • Genetic Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Adult) • Obesity

A machine learning (ML)–based six-gene signature for risk stratification and therapeutic target identification in multiple myeloma. (ASCO 2026) - "In multivariable Cox analysis, the CoxBoost risk score remained highly significant (HR 1.95, 95%CI:1.51-2.52,p<0.001) independent of ISS stage (HR 2.89, p<0.001) and age (HR 1.39 per decade, p<0.001).External validation on GSE24080 showed improved performance (C-index 0.683,+6.8% over clinical variables).The signature showed 50% gene overlap with LASSO (3/6 genes) and outperformed UAMS-70 (C-index 0.742 vs 0.631).Drug sensitivity analysis identified three compounds (MACIMORELIN, MW-150, BROFAROMINE) with selective activity against high-risk MM cells (p<0.001),validated across 16 cell lines... TP53 and its downstream pathways are central to MM progression. CoxBoost modeling produced a 6-gene signature that improved MM risk stratification over clinical variables, identifies therapeutic vulnerabilities, and is computationally efficient model suitable for clinical deployment, warranting prospective validation in MM treatment stratification. Performance of..."

Gene Signature • Machine learning • Hematological Malignancies • Multiple Myeloma • PTTG1 • STMN1 • UBE2C

Neurophysin-I dynamics upon different pituitary provocation tests in healthy participants. (PubMed, Endocr Connect) - "Sixty-six participants underwent a pituitary provocation test, 11 the hypertonic saline test, 20 the arginine infusion test, 25 the oral macimorelin test, and 10 the glucagon injection test...NP-I showed minimal responses to pituitary provocation tests, paralleling OXT dynamics. These findings highlight NP-I's potential as a reliable and technically robust surrogate biomarker for OXT activity, warranting further evaluation in clinical and experimental settings."

Journal • Metabolic Disorders • OXT

Facts and news about growth hormone replacement therapy in adults. (PubMed, Ann Endocrinol (Paris)) - "Traditional stimulation tests, such as the insulin tolerance and glucagon tests, are gold-standards for diagnosis, but the oral macimorelin test offers a safe, reliable and well-tolerated alternative...Long-acting growth hormone (LAGH) formulations, such as somapacitan, lonapegsomatropin and somatrogon, now enable weekly administration, improving adherence while maintaining efficacy and safety comparable to daily therapy. Early evidence suggests enhanced patient satisfaction and similar metabolic outcomes. Ongoing longitudinal studies are essential to clarify long-term safety."

Journal • Review • Cardiovascular • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Adult) • Musculoskeletal Diseases • Obesity • IGF1

Real-world diagnostic performance of the macimorelin stimulation test in the diagnosis of adult growth hormone deficiency. (PubMed, Pituitary) - No abstract available

Journal • Real-world evidence • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Adult)

Computational repurposing of approved drugs targeting KRAS G12D and EGFR for colorectal cancer therapy. (PubMed, PLoS One) - "This in silico study predicts Carteolol as a potential dual-targeting therapeutic agent, requiring biochemical and cellular validation before clinical relevance can be established."

Journal • Colorectal Cancer • Oncology • Solid Tumor • EGFR • KRAS

Molecular recognition of two approved drugs Macimorelin and Anamorelin by the growth hormone secretagogue receptor. (PubMed, Acta Pharmacol Sin) - "In addition, structural comparison of GHSR in complex with different G protein subtypes elucidated the mechanisms driving G protein selectivity. Our results provide crucial insights into GHSR-drug interactions and offer valuable guidance for designing more selective and potent GHSR agonists."

Journal • Cachexia • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Adult) • Oncology

Consensus and controversies about diagnosing GH deficiency: a Delphi survey by the GH research society. (PubMed, Pituitary) - "Consensus on the GHD diagnosis was lower in pediatric practice, mainly with respect to choice and interpretation of GH stimulation tests."

Journal • Review • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Adult) • Growth Hormone Deficiency (Pediatric) • Pediatrics • IGF1

Effect of urea ingestion on growth hormone levels in healthy adults – An analysis of a double-blind, randomized, placebo-controlled cross-over trial (ESPE-ESE 2025) - "Macimorelin, an oral GH stimulation test, offers an alternative but is unavailable in many countries, highlighting the need for accessible, non-invasive oral options...Conclusion Our study demonstrated no relevant changes in serum GH after the ingestion of urea in comparison with placebo. These findings suggest that urea does not stimulate GH secretion and cannot be used as a stimulation test for GH deficiency."

Clinical • Cardiovascular • Growth Hormone Deficiency • Growth Hormone Deficiency (Adult) • Heart Failure • Hypoglycemia

Macimorelin Test: A Safer and Reliable Alternative to the Gold Standard Insulin Tolerance Test (ESPE-ESE 2025) - "Conclusion The availability of a safer, quicker and more-tolerable GH stimulation test like MT, should no longer prevent the provision of appropriate and timely diagnosis and treatment of patients with GHD. Considering both the financial and personal burden of performing an ITT, MT should be considered as a reliable alternative as it is easy and quick to perform, associated with minimal adverse effects and has a diagnostic accuracy comparable with ITT."

Gold standard • Hypoglycemia

ECE2025 Best Nursing Poster Presentation | Macimorelin Test: A Safer and Reliable Alternative to the Gold Standard Insulin Tolerance Test (ESPE-ESE 2025) - No abstract available

Gold standard

DETECT: A Research Study of How Well Macimorelin Works to Find Out if Children Have a Lack of Growth Hormone and How Safe it is (clinicaltrials.gov) - P3 | N=101 | Completed | Sponsor: AEterna Zentaris | Active, not recruiting ➔ Completed

Trial completion • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Pediatric) • Pediatrics

Insights into the Effects of Ligand Binding on Leucyl-tRNA Synthetase Inhibitors for Tuberculosis: In Silico Analysis and Isothermal Titration Calorimetry Validation. (PubMed, Biomolecules) - "Compound 1054 (Macimorelin) was found to be the most potent molecule, with better antimycobacterial activity, enzyme binding affinity, and significant inhibition of biofilm formation, as well as inhibition of the LeuRS gene expression. Compound 1054, the top hit compound, has the potential to be used as a lead to develop successful leucyl t-RNA synthetase inhibitors."

Journal • Infectious Disease • Pulmonary Disease • Respiratory Diseases • Tuberculosis

DETECT: A Research Study of How Well Macimorelin Works to Find Out if Children Have a Lack of Growth Hormone and How Safe it is (clinicaltrials.gov) - P3 | N=100 | Active, not recruiting | Sponsor: AEterna Zentaris | Recruiting ➔ Active, not recruiting | Trial completion date: Sep 2023 ➔ Jun 2024 | Trial primary completion date: Sep 2023 ➔ Jun 2024

Enrollment closed • Trial completion date • Trial primary completion date • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Pediatric) • Pediatrics

Once upon a time: the glucagon stimulation test in diagnosing adult GH deficiency. (PubMed, J Endocrinol Invest) - "This document provides an update on the utility of GST, summarizes how to perform the test, shows which cut-points should be used in interpreting the results, and discusses its drawbacks and caveats referring to the most recent studies."

Journal • Review • Growth Hormone Deficiency • Growth Hormone Deficiency (Adult) • Obesity

New insights on diagnosis and treatment of AVP deficiency. (PubMed, Rev Endocr Metab Disord) - "Glucagon-stimulated copeptin also showed promising results, while the oral growth hormone secretagogue Macimorelin failed to provide a sufficient stimulus. In general, treatment of AVP-D focuses on desmopressin substitution, with oral formulations currently showing the best tolerance and safety profile. However, in addition to desmopressin substitution, recent data also showed that psychopathological factors play an important role in managing AVP-D patients."

Journal • Review • Metabolic Disorders

Pilot study of macimorelin for cancer cachexia (SCWD 2023) - No abstract available.

Clinical

"$AEZS U.S. sales of Macrilenfor adult use to be temporarily discontinued as of May 23rd" (,@BioStocks)

Pilot clinical trial of macimorelin to assess safety and efficacy in patients with cancer cachexia. (PubMed, J Cachexia Sarcopenia Muscle) - "Daily oral macimorelin for 1 week was safe and numerically improved body weight and QOL in patients with cancer cachexia compared with placebo. Longer term administration should be evaluated for mitigation of cancer-induced reductions in body weight, appetite and QOL in larger studies."

Journal • Cachexia • Fatigue • Oncology • IGF1

Macimorelin Acetate for the Diagnosis of Childhood-onset Growth Hormone Deficiency. (PubMed, touchREV Endocrinol) - "Despite the widespread use of these tests, various criticisms have been levelled against them, such as the labour-intensive nature of the tests, their potential for serious adverse effects and their questionable reproducibility. Macimorelin acetate, a ghrelin mimetic approved for the diagnosis of adult growth hormone deficiency, could serve an unmet need in the diagnosis of childhood-onset growth hormone deficiency based on its good tolerability and benign side effect profile."

Journal • Endocrine Disorders • Growth Hormone Deficiency • Growth Hormone Deficiency (Adult)

Aeterna Zentaris Provides Business Update and Outlines Key Upcoming Milestones (Market Screener) - "Aeterna Zentaris Inc...provided a business update and outlined upcoming key milestones....Substantial progress in the development of a suitable, alternative formulation for use in ALS. Ongoing evaluation of AEZS-130 in transgenic mouse ALS models to demonstrate the therapeutic potential of macimorelin in this indication....Proof-of-concept results from ongoing pre-clinical studies expected in the first quarter of 2023. Following potential achievement of proof-of-concept, the Company will seek to have a scientific advice meeting with regulatory authorities to discuss program development next steps..."

Preclinical • Amyotrophic Lateral Sclerosis • CNS Disorders

The effect of glucose dynamics on plasma copeptin levels upon glucagon, arginine, and macimorelin stimulation in healthy adults (ENEA 2022) - "These findings suggest that a drop in glucose levels might be a possible cause for the increase in copeptin levels upon glucagon and arginine stimulation. Whether the drop in glucose levels itself leads to copeptin secretion, or whether it mirrors other stimuli such as hypoglycemia-induced stress cannot be answered with our analysis and should be further investigated."

Clinical • Hypoglycemia

"$AEZS Aeterna Zentaris Set to Regain Full Rights to Macrilen™ (Macimorelin) in U.S. and Canada from Novo Nordisk https://t.co/QG2vbW55YB" (,@stock_titan)

Pilot Clinical Trial of Repeated Doses of Macimorelin to Assess Safety and Efficacy in Patients With Cancer Cachexia (clinicaltrials.gov) - P2 | N=8 | Completed | Sponsor: Garcia, Jose M., MD, PhD | Active, not recruiting ➔ Completed

Trial completion • Cachexia • Oncology • Solid Tumor • IGF1 • IGFBP3 • IL6