CLINICAL TRIALS PROFILE FOR INCRELEX

All Clinical Trials for Increlex

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00330668 ↗	Treatment of Children and Adolescents With Growth Failure Associated With Primary IGF-1 Deficiency	Terminated	Ipsen	Phase 3	2005-11-01	This is an extension study to Tercica study MS301 (NCT00125164) and is intended to collect long term safety and efficacy data on the continued use of recombinant human insulin-like growth factor-1 (rh IGF-1) in children and adolescents treated for primary IGF-1 deficiency (IGFD). The secondary objective is to use the data collected to learn more about the relationship of IGF-1 exposure to the promotion of normal growth and pubertal development.
NCT00490100 ↗	Treatment for Growth Failure in Patients With X-Linked Severe Combined Immunodeficiency: Phase 2 Study of Insulin-Like Growth Factor-1	Terminated	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 1/Phase 2	2007-06-01	This study will evaluate the safety and effectiveness of insulin-like growth factor-1 (IGF-1) to treat patients with X-linked severe combined immunodeficiency (XSCID). Those who have XSCID lack white blood cells that protect their bodies from invasion by all types of germs. IGF-1 is the main hormone responsible for the body's growth and metabolism. As a medication, IGF-1 is Increlex[(Trademark)] (mecasermin), Patients ages 2 to 20 who have not yet begun puberty, have a diagnosis of XSCID, and are shorter than the 3rd percentile for their age may be eligible for this study. This study will last about 3 years, and patients' visits will be scheduled at 3-month intervals. Patients will have a physical history and exam, X-rays, electrocardiogram, blood tests, and body measurements. Patients will take estradiol orally for 2 days, to help avoid false results of growth hormone (GH) levels in blood samples. Then provocation testing is done, with two tests back to back. It determines blood levels of GH and the body's response to testing with drugs called arginine and clonidine. Patients are admitted to the pediatric inpatient unit and will have an intravenous (IV) line placed in the arm. Arginine is given by IV over 30 minutes, and blood samples are taken. Right after arginine testing, the clonidine tablet is given. The IGF-1 generation test is then done to see if the body makes IGF-1 as a product in response to injections of GH for 5 consecutive days. This test does not require that patients are inpatients, but after Day 8, patients must be admitted to the pediatric unit to have blood sampling, start Increlex injections, and start close monitoring of blood sugar levels. They will learn how to do a self-injection and follow other advice. They will complete records about the injection site, symptoms, and side effects-keeping records for at least the first 2 days after going home, with each dose change, and as needed. Patients stick their fingertip and place a small drop of blood on a blood sugar monitoring strip. The strip is put into a glucometer-a small hand-held device to measure the blood sugar level. Patients will be instructed to always have a source of sugar available in case blood sugar is too low. ...
NCT00516386 ↗	Safety Profile of Insulin Like Growth Factor-1 (IGF-I) Administration in Adolescents	Completed	Tercica	Phase 1/Phase 2	2007-03-01	The purpose of this study was to determine whether giving insulin like growth factor-I (IGF-I) to adolescent low weight girls is safe and whether this increases levels of bone formation markers.
NCT00516386 ↗	Safety Profile of Insulin Like Growth Factor-1 (IGF-I) Administration in Adolescents	Completed	Massachusetts General Hospital	Phase 1/Phase 2	2007-03-01	The purpose of this study was to determine whether giving insulin like growth factor-I (IGF-I) to adolescent low weight girls is safe and whether this increases levels of bone formation markers.

Clinical Trial Conditions for Increlex

Condition Name

Condition Name for Increlex
Intervention	Trials
Rett Syndrome	2
Growth Failure	2
Anorexia Nervosa	2
Autism Spectrum Disorder	1

Condition MeSH

Condition MeSH for Increlex
Intervention	Trials
Syndrome	4
Dwarfism	3
Failure to Thrive	3
Rett Syndrome	2

Clinical Trial Locations for Increlex

Trials by Country

Trials by Country for Increlex
Location	Trials
United States	11
United Kingdom	1
France	1
Sweden	1

Trials by US State

Trials by US State for Increlex
Location	Trials
Massachusetts	4
New York	2
California	2
Texas	1
Ohio	1

Clinical Trial Progress for Increlex

Clinical Trial Phase

Clinical Trial Phase for Increlex
Clinical Trial Phase	Trials
Phase 3	1
Phase 2/Phase 3	2
Phase 2	4

Clinical Trial Status

Clinical Trial Status for Increlex
Clinical Trial Phase	Trials
Completed	6
Terminated	5
Recruiting	3

Clinical Trial Sponsors for Increlex

Sponsor Name

Sponsor Name for Increlex
Sponsor	Trials
Ipsen	4
Massachusetts General Hospital	2
International Rett Syndrome Foundation	2

Sponsor Type

Sponsor Type for Increlex
Sponsor	Trials
Other	18
Industry	6
NIH	2

Clinical Trials, Market Analysis, and Projections for Increlex (Mecasermin)

Introduction to Increlex

Increlex, also known as mecasermin, is a recombinant human insulin-like growth factor-1 (rhIGF-1) used to treat severe primary insulin-like growth factor-1 deficiency (SPIGFD) in pediatric patients. Here, we will delve into the clinical trials, market analysis, and future projections for this critical therapeutic agent.

Clinical Trials and Efficacy

Phase III Clinical Studies

The efficacy of Increlex was established through several Phase III clinical studies. These studies included both open-label and double-blind, placebo-controlled trials involving pediatric patients with SPIGFD. The primary efficacy endpoint was the change in height velocity from pre-treatment during each year of treatment.

• Key Findings: Patients treated with Increlex showed a significant increase in mean height velocity, from 2.6 cm/year pre-treatment to 8.0 cm/year in the first year of treatment. This effect was sustained over seven years, with improvements in height standard deviation scores from -3.4 to 1.7 and from -6.9 to -6.1, respectively, over ten years[3][4].

• Long-Term Treatment: Patients were treated for an average of 3.9 years, with some receiving treatment for up to 11.5 years. The long-term treatment with rhIGF-1 was found to be well-tolerated, with mild-to-moderate side effects such as hypoglycemia, injection site lipohypertrophy, and tonsillar hypertrophy[1][4].

Safety Profile

The safety profile of Increlex is generally consistent with the known mechanisms of action of IGF-1. Common adverse reactions include:

• Hypoglycemia: Occurred in 42% of patients.
• Injection Site Reactions: Lipohypertrophy and bruising.
• Tonsillar Hypertrophy: Occurred in 15% of patients.
• Intracranial Hypertension: Reported in three subjects, with two cases resolving without interruption of treatment[1][4].

Market Analysis

Current Market Status

Increlex is approved in 41 territories, including the United States and the European Union, and is the only FDA-approved treatment for SPIGFD. The drug is now commercially available through Eton Pharmaceuticals, following its acquisition from Ipsen S.A. in December 2024. In the U.S., Increlex is distributed exclusively through AnovoRx, a specialty pharmacy dedicated to serving patients with rare and chronic conditions[5].

Patient Population

• U.S. and Europe: Approximately 200 patients in the U.S. and 900-1,000 patients in Europe are estimated to live with SPIGFD. This niche market, though small, represents a significant unmet need for effective treatment[5].

Market Projections and Trends

Pharmaceutical Market Outlook 2025

According to Evaluate Pharma's 2025 Preview report, the pharmaceutical market is expected to see significant growth, driven by advancements in various therapeutic areas. While Increlex is not among the top-selling drugs projected for 2025, its niche market and the growing focus on rare diseases suggest a stable and potentially growing demand.

• General Market Trends: The report highlights an expected increase in pharmaceutical product sales by $82 billion in 2025, with a focus on obesity, oncology, and immunology therapies. The incretin class, particularly GLP-1s, is anticipated to lead the market, but other niche treatments like Increlex are likely to maintain their importance due to their unique therapeutic roles[2].

Deal-Making and Access to Capital

The pharmaceutical industry is expected to see increased deal-making activity and improved access to capital in 2025. This trend could benefit companies like Eton Pharmaceuticals, which have recently acquired assets like Increlex, by providing more resources for marketing, distribution, and potentially expanding the patient base[2].

Future Projections

Commercialization and Distribution

Eton Pharmaceuticals' acquisition of Increlex is expected to enhance its commercial pediatric endocrinology portfolio. With Increlex now available through AnovoRx, a specialty pharmacy, patients are likely to experience improved access to this critical treatment. The Eton Cares Program, which includes co-pay assistance and other support services, will further facilitate patient access[5].

Potential for Increased Awareness

The acquisition and new distribution channels are expected to increase awareness of SPIGFD, an underdiagnosed and undertreated condition. This increased awareness could lead to more diagnoses and, consequently, a larger patient base for Increlex.

Regulatory and Clinical Monitoring

Health Canada and the FDA have established clear guidelines and monitoring requirements for Increlex, ensuring that its benefit-risk profile remains favorable. Ongoing studies, such as the European Registry, continue to provide long-term safety and efficacy data, which will be crucial for maintaining regulatory approvals and patient trust[3][4].

Key Takeaways

• Clinical Efficacy: Increlex has demonstrated significant efficacy in increasing height velocity and improving height standard deviation scores in patients with SPIGFD.
• Safety Profile: The drug is generally well-tolerated, with manageable side effects.
• Market Status: Increlex is now part of Eton Pharmaceuticals' portfolio, available through a specialty pharmacy, and is approved in multiple territories.
• Market Trends: The pharmaceutical market is expected to grow, with increased focus on rare diseases and improved access to capital.
• Future Projections: Increased awareness and improved distribution are likely to enhance patient access and potentially grow the market for Increlex.

Frequently Asked Questions (FAQs)

1. What is Increlex used for?

Increlex is used to treat severe primary insulin-like growth factor-1 deficiency (SPIGFD) in pediatric patients aged 2 years and older.

2. What are the common side effects of Increlex?

Common side effects include hypoglycemia, injection site lipohypertrophy, tonsillar hypertrophy, and intracranial hypertension.

3. Who is the current manufacturer and distributor of Increlex?

Eton Pharmaceuticals is the current manufacturer, and the drug is distributed exclusively through AnovoRx in the U.S.

4. What is the expected market trend for Increlex in 2025?

While Increlex is not among the top-selling drugs projected for 2025, its niche market and growing focus on rare diseases suggest stable and potentially growing demand.

5. How is Increlex administered?

Increlex is administered via subcutaneous injection, typically twice daily, with doses ranging from 0.06 to 0.12 mg/kg.

Cited Sources

1. Aetna Medical Clinical Policy Bulletins: Mecasermin (Increlex)
2. Evaluate Pharma: 2025 Preview report
3. Health Canada: Summary Basis of Decision for Increlex
4. FDA Label: Increlex (mecasermin injection)
5. Eton Pharmaceuticals: Acquisition of Increlex (mecasermin injection)