THE EARLY-PHASE INNOVATIVE OBESITY PIPELINE: A SYSTEMATIC SCREENING OF NOVEL WEIGHT-LOSS ASSETS IN CLINICAL DEVELOPMENT
Author(s)
Thi Phuong Thao Nguyen, MD, MSc1, Attila Imre, PharmD1, Tamas Agh, MSc, PhD, MD2.
1Center for Health Technology Assessment, Semmelweis University & Syreon Research Institute, Budapest, Hungary, 2Center for HTA and Pharmacoeconomic Research, University of Pecs & Syreon Research Institute, Budapest, Hungary.
1Center for Health Technology Assessment, Semmelweis University & Syreon Research Institute, Budapest, Hungary, 2Center for HTA and Pharmacoeconomic Research, University of Pecs & Syreon Research Institute, Budapest, Hungary.
OBJECTIVES: To characterize the early-phase clinical pipeline of novel, investigational pharmaceutical assets under development for weight loss in individuals with obesity or overweight.
METHODS: We searched Clinical Trial Nexus (NCT, CTIS, and EudraCT) and removed duplicates before screening. Two reviewers independently assessed each record against pre-defined criteria. To be eligible, a trial had to test a new molecular or biological entity (but not an advanced-therapy product) that was still unapproved for any indication and was administered for weight loss to people with obesity or overweight, regardless of comorbidity. We excluded approved or repurposed agents, lifestyle-only studies, supplements, microbiome studies, observational studies, and mechanistic studies. When an approved drug appeared only as the comparator, we judged the trial on its new arm.
RESULTS: Of 355 records, 90 trials were included. Phase 2 accounted for most (72.2%); the rest sat at Phase 1 or 1/2, and nearly all were recruiting or close to it. This is a young field with almost 88.9% opened in 2024 or later, and the industry ran 85.6%. About a third enrolled with obesity and a comorbidity; the rest studied uncomplicated obesity. China led on sponsorship (45.6%), ahead of the United States (31.1%) and Europe (17.8%). By molecule type, peptides dominate (58.9%), with small molecules following (26.7%) and antibodies or fusion proteins (10.0%); no advanced-therapy products were observed. Regarding the mechanism, more than half of the assets act via incretin biology. Amylin and amylin-calcitonin agonists comprised the largest non-incretin group (14.4%), muscle-preserving agents targeting the myostatin-activin pathway accounted for 5.6%, and 8.9% targeted other mechanisms (NLRP3, cannabinoid, leptin, MAO-B, PDE4, or RNA).
CONCLUSIONS: The early pipeline is large, recent, and peptide- and incretin-heavy, with China now its biggest sponsor. The differentiation and open evidence questions are amylin biology, lean-mass preservation, oral delivery, and a few non-incretin targets.
METHODS: We searched Clinical Trial Nexus (NCT, CTIS, and EudraCT) and removed duplicates before screening. Two reviewers independently assessed each record against pre-defined criteria. To be eligible, a trial had to test a new molecular or biological entity (but not an advanced-therapy product) that was still unapproved for any indication and was administered for weight loss to people with obesity or overweight, regardless of comorbidity. We excluded approved or repurposed agents, lifestyle-only studies, supplements, microbiome studies, observational studies, and mechanistic studies. When an approved drug appeared only as the comparator, we judged the trial on its new arm.
RESULTS: Of 355 records, 90 trials were included. Phase 2 accounted for most (72.2%); the rest sat at Phase 1 or 1/2, and nearly all were recruiting or close to it. This is a young field with almost 88.9% opened in 2024 or later, and the industry ran 85.6%. About a third enrolled with obesity and a comorbidity; the rest studied uncomplicated obesity. China led on sponsorship (45.6%), ahead of the United States (31.1%) and Europe (17.8%). By molecule type, peptides dominate (58.9%), with small molecules following (26.7%) and antibodies or fusion proteins (10.0%); no advanced-therapy products were observed. Regarding the mechanism, more than half of the assets act via incretin biology. Amylin and amylin-calcitonin agonists comprised the largest non-incretin group (14.4%), muscle-preserving agents targeting the myostatin-activin pathway accounted for 5.6%, and 8.9% targeted other mechanisms (NLRP3, cannabinoid, leptin, MAO-B, PDE4, or RNA).
CONCLUSIONS: The early pipeline is large, recent, and peptide- and incretin-heavy, with China now its biggest sponsor. The differentiation and open evidence questions are amylin biology, lean-mass preservation, oral delivery, and a few non-incretin targets.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
SA66
Topic
Clinical Outcomes, Study Approaches
Topic Subcategory
Literature Review & Synthesis
Disease
Diabetes/Endocrine/Metabolic Disorders (including obesity), No Additional Disease & Conditions/Specialized Treatment Areas