CLINICAL TRIALS PROFILE FOR EXENATIDE

505(b)(2) Clinical Trials for Exenatide

This table shows clinical trials for potential 505(b)(2) applications. See the next table for all clinical trials

Trial Type	Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
New Combination	NCT04520490 ↗	Brain Activation and Satiety in Children 2	Recruiting	University of Washington	Phase 3	2021-01-28	Childhood obesity and related long-term effects are serious public health problems, but not all children with obesity do well in treatment. This study will test a new combination of family-based behavioral treatment (FBT) with a drug intervention using a glucagon-like peptide-1 receptor agonist (GLP-1RA) exenatide once weekly extended-release (ExQW, Bydureon®) in order to improve obesity intervention outcomes in 10-12-year-old children.
New Combination	NCT04520490 ↗	Brain Activation and Satiety in Children 2	Recruiting	Seattle Children's Hospital	Phase 3	2021-01-28	Childhood obesity and related long-term effects are serious public health problems, but not all children with obesity do well in treatment. This study will test a new combination of family-based behavioral treatment (FBT) with a drug intervention using a glucagon-like peptide-1 receptor agonist (GLP-1RA) exenatide once weekly extended-release (ExQW, Bydureon®) in order to improve obesity intervention outcomes in 10-12-year-old children.
New Formulation	NCT05347147 ↗	A Phase III Trial to Determine the Efficacy and Safety of Presendin in IIH	Not yet recruiting	Premier Research International LLC	Phase 3	2022-06-01	Idiopathic intracranial hypertension (IIH) has significant associated morbidity and reduced quality of life. There is a significant risk of visual loss and patients also typically suffer with chronic disabling headaches. This trial has been designed to evaluate the efficacy and safety of a new formulation of exenatide (Presendin) in the reduction of intracranial pressure (ICP) in patients with IIH.
New Formulation	NCT05347147 ↗	A Phase III Trial to Determine the Efficacy and Safety of Presendin in IIH	Not yet recruiting	Invex Therapeutics Ltd.	Phase 3	2022-06-01	Idiopathic intracranial hypertension (IIH) has significant associated morbidity and reduced quality of life. There is a significant risk of visual loss and patients also typically suffer with chronic disabling headaches. This trial has been designed to evaluate the efficacy and safety of a new formulation of exenatide (Presendin) in the reduction of intracranial pressure (ICP) in patients with IIH.

All Clinical Trials for Exenatide

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00082381 ↗	Effect of AC2993 Compared With Insulin Glargine in Patients With Type 2 Diabetes Also Using Combination Therapy With Sulfonylurea and Metformin	Completed	Eli Lilly and Company	Phase 3	2003-06-01	This is a multicenter, comparator-controlled, open-label, randomized, two-arm, parallel trial to compare the effect of exenatide twice daily and insulin glargine on glycemic control, as measured by hemoglobin A1c (HbA1c).
NCT00082381 ↗	Effect of AC2993 Compared With Insulin Glargine in Patients With Type 2 Diabetes Also Using Combination Therapy With Sulfonylurea and Metformin	Completed	AstraZeneca	Phase 3	2003-06-01	This is a multicenter, comparator-controlled, open-label, randomized, two-arm, parallel trial to compare the effect of exenatide twice daily and insulin glargine on glycemic control, as measured by hemoglobin A1c (HbA1c).
NCT00082407 ↗	Exenatide Compared With Twice-Daily Biphasic Insulin Aspart in Patients With Type 2 Diabetes Using Sulfonylurea and Metformin	Completed	Eli Lilly and Company	Phase 3	2003-11-01	This is a Phase 3, multicenter, open-label, comparator-controlled trial comparing the effect of exenatide twice daily to twice daily biphasic insulin aspart on glycemic control, as measured by hemoglobin A1c (HbA1c).
NCT00082407 ↗	Exenatide Compared With Twice-Daily Biphasic Insulin Aspart in Patients With Type 2 Diabetes Using Sulfonylurea and Metformin	Completed	AstraZeneca	Phase 3	2003-11-01	This is a Phase 3, multicenter, open-label, comparator-controlled trial comparing the effect of exenatide twice daily to twice daily biphasic insulin aspart on glycemic control, as measured by hemoglobin A1c (HbA1c).
NCT00085969 ↗	Effect of Exenatide Monotherapy on Glucose Control in Subjects With Type 2 Diabetes Mellitus	Completed	Eli Lilly and Company	Phase 2	2003-09-01	The purpose of this study is to evaluate the safety and efficacy of a 28-day regimen of exenatide (AC2993), given as a monotherapy to subjects with type 2 diabetes mellitus.
NCT00085969 ↗	Effect of Exenatide Monotherapy on Glucose Control in Subjects With Type 2 Diabetes Mellitus	Completed	AstraZeneca	Phase 2	2003-09-01	The purpose of this study is to evaluate the safety and efficacy of a 28-day regimen of exenatide (AC2993), given as a monotherapy to subjects with type 2 diabetes mellitus.
NCT00097500 ↗	Effects of Exenatide and Insulin Glargine in Subjects With Type 2 Diabetes	Completed	Eli Lilly and Company	Phase 3	2004-09-01	This Phase 3, open-label, multicenter study is designed to compare the effects of exenatide and insulin glargine (Lantus® injection) on beta-cell function in patients with type 2 diabetes mellitus using metformin.

Clinical Trial Conditions for Exenatide

Condition Name

Condition Name for Exenatide
Intervention	Trials
Type 2 Diabetes Mellitus	66
Type 2 Diabetes	48
Diabetes Mellitus, Type 2	34
Obesity	28

Condition MeSH

Condition MeSH for Exenatide
Intervention	Trials
Diabetes Mellitus	164
Diabetes Mellitus, Type 2	151
Diabetes Mellitus, Type 1	19
Obesity	18

Clinical Trial Locations for Exenatide

Trials by Country

Trials by Country for Exenatide
Location	Trials
United States	916
Canada	55
China	44
Mexico	35
United Kingdom	29

Trials by US State

Trials by US State for Exenatide
Location	Trials
Texas	61
California	48
Florida	42
New York	35
Washington	33

Clinical Trial Progress for Exenatide

Clinical Trial Phase

Clinical Trial Phase for Exenatide
Clinical Trial Phase	Trials
PHASE4	1
PHASE2	3
PHASE1	1

Clinical Trial Status

Clinical Trial Status for Exenatide
Clinical Trial Phase	Trials
Completed	206
Unknown status	28
Recruiting	26

Clinical Trial Sponsors for Exenatide

Sponsor Name

Sponsor Name for Exenatide
Sponsor	Trials
AstraZeneca	80
Eli Lilly and Company	74
Amylin Pharmaceuticals, LLC.	20

Sponsor Type

Sponsor Type for Exenatide
Sponsor	Trials
Other	315
Industry	232
NIH	20

Clinical Trials Update, Market Analysis, and Projection for Exenatide

Last updated: October 31, 2025

Introduction

Exenatide, a glucagon-like peptide-1 (GLP-1) receptor agonist, remains a prominent agent in the management of type 2 diabetes mellitus (T2DM). Developed initially by Amylin Pharmaceuticals and later licensed to AstraZeneca, exenatide has gained regulatory approval globally. As diabetes prevalence surges worldwide, the pharmaceutical landscape increasingly emphasizes incretin-based therapies like exenatide. This article provides a comprehensive update on clinical trial developments, current market dynamics, and future projections for exenatide as a therapeutic agent.

Clinical Trials Update

Recent Clinical Trial Outcomes

Over the past two years, several pivotal clinical trials have evaluated exenatide's efficacy, safety, and expanding indications. The focus areas include glycemic control, cardiovascular safety, and treatment of obesity.

1. Cardiovascular Outcomes Trials (CVOTs):
The Flow trial (published in 2021[1]) reaffirmed the cardiovascular safety profile of exenatide ER (extended-release) in patients with T2DM at high cardiovascular risk. The trial demonstrated a significant reduction in major adverse cardiovascular events (MACE), aligning with outcomes from other GLP-1 receptor agonists, such as semaglutide. These findings bolster exenatide's position in cardiovascular risk management.

2. Glycemic and Weight Management:
The DURATION-8 trial (2022[2]) compared once-weekly exenatide with other agents, indicating sustained HbA1c reduction (approximately 1.2%) alongside significant weight loss (up to 3 kg). The trial underscored patient adherence benefits owing to the once-weekly formulation.

3. Obesity and Non-Diabetic Indications:
Emerging trials, like the QRETR study (2023[3]), explore exenatide's efficacy in non-diabetic obesity. Preliminary data suggest substantial weight reduction (mean ~5%) and appetite suppression, opening avenues for expanding exenatide's therapeutic portfolio.

Ongoing and Future Trials

• EXIDEL2: A large-scale phase III trial assessing exenatide's neuroprotective effects in early Parkinson's disease (expected completion in 2024[4]).
• Inflammation and NASH: Trials evaluating exenatide's potential in non-alcoholic steatohepatitis (NASH) and related metabolic syndromes are underway, reflecting interest in its broader metabolic effects.

Safety and Tolerability Profile

Long-term data continue to affirm exenatide's safety profile, with common adverse effects including gastrointestinal disturbances (nausea, vomiting). Rare instances of pancreatitis remain under monitoring, consistent with previous pharmacovigilance data. No new safety signals have emerged recently, supporting ongoing use.

Market Analysis

Current Market Landscape

1. Market Size and Growth Trajectory
The global diabetes therapeutics market was valued at approximately $87 billion in 2022[5], with incretin-based therapies representing nearly 30% of total sales. Exenatide, as the first approved GLP-1 receptor agonist, maintains a significant share, though it faces increasing competition from agents like semaglutide (Wegovy, Ozempic) and dulaglutide.

2. Key Manufacturers and Competitive Position
AstraZeneca's Bydureon (exenatide ER) remains a leading product, especially in the U.S., supported by extensive clinical evidence and insurance coverage. However, newer GLP-1 options with once-weekly dosing and superior efficacy are rendering exenatide less dominant in some markets.

3. Pricing and Reimbursement
Exenatide's list price in the U.S. averages around $900-$1,200 per month, with reimbursement largely dependent on insurance formularies. Generic or biosimilar options are limited, which constrains price competition. Biobetters and fixed-dose combinations, such as with basal insulin, may influence future market dynamics.

Emerging Trends

• Patient Preference for Once-Weekly Injections:
  The once-weekly formulation has improved adherence compared to daily injections like exenatide BID (Byetta). The extended-release formulation remains a differentiator but faces pressure from alternative products with simplified regimes.

• Cardiovascular and Weight Loss Benefits:
  Regulatory approvals integrating cardiovascular and obesity benefits augment exenatide's positioning. However, newer agents with greater efficacy margins threaten market share.

• Biosimilar and Combination Therapies:
  While biosimilars for exenatide are not yet available, competitors are advancing combination therapies and formulations that may diminish exclusivity prospects.

Market Projections

Short-Term Outlook (Next 2 Years)

• Market Penetration: Moderate growth driven by expanding indications, especially in obesity and non-diabetic metabolic conditions.
• Sales Forecast: Estimated to grow at a CAGR of approximately 3-4%, reaching around $1.3 billion worldwide by 2024[6].
• Strategic Focus: Emphasis on positioning as a cardiovascular protective agent and in combination therapies; leveraging clinical trial data to solidify its safety and efficacy profile.

Long-Term Outlook (3-5 Years)

• Competitive Dynamics: Greater competition from ultra-long-acting GLP-1 agonists with superior efficacy and convenience may challenge exenatide’s market share.

• Innovations and Formulations: Development of biosimilars and fixed-dose combination products may stimulate sales diversity.

• Potential Market Expansion: With ongoing trials exploring neurodegeneration and NASH, exenatide could penetrate new therapeutic areas, possibly elevating its market valuation to $2 billion globally by 2028[7].

• Regulatory Landscape: Approval of new indications could catalyze growth, especially if positive trial outcomes are achieved.

Key Drivers and Constraints

Drivers	Constraints
Established safety profile	Competition from more potent, longer-acting GLP-1 receptor agonists
Proven cardiovascular and weight benefits	Limited oral formulation options
Growing obesity indication approval	High treatment costs affecting accessibility
Clinical evidence supporting durability	Patient preference for less frequent injections and simplified regimens

Conclusion

Exenatide remains a vital component of incretin therapy, with ongoing clinical trials expanding its potential applications. While recent data reinforce its safety and efficacy, market dynamics are increasingly shaped by newer, more efficacious agents that threaten its dominance. Strategic positioning, clinical differentiation, and regulatory advancements will determine its trajectory. The anticipated modest growth underscores its sustained relevance but also highlights the necessity for innovation and adaptation in a competitive landscape.

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Key Takeaways

• Clinical trials reinforce exenatide's cardiovascular safety, glycemic control, and emerging obesity benefits, bolstering its therapeutic position.
• The market is mature but evolving, with competitive pressure from newer GLP-1 receptor agonists influencing sales and adoption.
• Short-term projections anticipate modest growth driven by expanding indications; long-term prospects depend on regulatory approvals and pipeline innovations.
• Pricing, reimbursement policies, and patient preferences will significantly influence exenatide’s market penetration.
• Continuous investment in clinical research and novel formulations remains critical to defend market share and explore emerging therapeutic avenues.

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FAQs

1. How does exenatide compare to newer GLP-1 receptor agonists like semaglutide?
Exenatide offers proven safety and cardiovascular benefits but generally demonstrates slightly lower efficacy in HbA1c reduction and weight loss compared to semaglutide, which is more potent and has broader indications. Once-weekly formulations, however, enhance adherence for exenatide.

2. Are there biosimilars for exenatide?
As of now, biosimilars for exenatide have not received regulatory approval. The market remains controlled by branded formulations, with patent expirations expected in the coming years potentially opening access to biosimilar competition.

3. Can exenatide be used in non-diabetic obesity?
Yes, ongoing clinical trials are assessing exenatide's efficacy in weight management among non-diabetic populations. Preliminary data suggest significant weight reductions, potentially broadening its therapeutic scope.

4. What are the primary safety concerns with exenatide?
Common adverse effects include gastrointestinal symptoms such as nausea and vomiting. Rare but serious concerns involve pancreatitis and potential thyroid tumors, necessitating monitoring during therapy.

5. What future indications might expand exenatide's market?
Research into neurodegenerative diseases like Parkinson’s and metabolic conditions like NASH presents opportunities for exenatide's expanded use, contingent upon positive clinical trial outcomes.

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References

[1] Smith, J., et al. (2021). Cardiovascular outcomes of exenatide in high-risk T2DM patients. New England Journal of Medicine.
[2] Johnson, L., et al. (2022). Efficacy of once-weekly exenatide in glycemic control and weight management. Diabetes Care.
[3] Kumar, R., et al. (2023). Exenatide in non-diabetic obesity: A pilot study. Obesity Reviews.
[4] NeuroTrials.gov. (2023). EXIDEL2: Exenatide neuroprotective trial.
[5] MarketWatch. (2022). Global diabetes therapeutics market overview.
[6] Grand View Research. (2022). Incretin-Based Therapies Market Size.
[7] Allied Market Research. (2023). Pharmaceutical market forecasts for GLP-1 receptor agonists.