Congenital Hyperinsulinism - Pipeline Insight, 2024

This “Congenital Hyperinsulinism - Pipeline Insight, 2024” report provides comprehensive insights about 4+ companies and 4+ pipeline drugs in Congenital Hyperinsulinism pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Geography Covered

• Global coverage

Congenital Hyperinsulinism: Understanding

Congenital Hyperinsulinism: Overview

Congenital hyperinsulinism (HI) is the most frequent cause of persistent hypoglycemia in infants and children. Delays in diagnosis and initiation of appropriate treatment contribute to a high risk of neurocognitive impairment. HI represents a heterogeneous group of disorders characterized by dysregulated insulin secretion by the pancreatic beta cells, which in utero, may result in somatic overgrowth. There are at least nine known monogenic forms of HI as well as several syndromic forms. Molecular diagnosis allows for prediction of responsiveness to medical treatment and likelihood of surgically-curable focal hyperinsulinism. Timely genetic mutation analysis has thus become standard of care.

The diagnosis of HI is made based upon the critical sample, the blood specimen obtained at the time of spontaneous or provoked hypoglycemia, and the glycemic response to glucagon. The threshold plasma glucose for obtaining the critical sample is < 50mg/dL to permit investigation of the biochemical counter-regulatory response to hypoglycemia and to limit the likelihood of false positive results. Biochemical findings consistent with inappropriate insulin action include inappropriately low plasma beta-hydroxybutyrate and free fatty acid concentrations, and a glycemic response to glucagon of 30 mg/dL or more at the time of hypoglycemia.

The primary goal for hyperinsulinism treatment is to maintain plasma glucose > 70 mg/dL, above the threshold for activation of neuroendocrine responses to hypoglycemia. This target is supported by physiology and reflects current consensus opinion recognizing an absence of outcomes data comparing different therapeutic thresholds. Diazoxide, which acts to open pancreatic β-cell ATP-sensitive potassium (KATP) channels and decrease insulin secretion, is the first-line therapeutic agent. However, diazoxide is ineffective in treating hyperinsulinism caused by inactivating mutations in the genes encoding the KATP channel.

"Congenital Hyperinsulinism- Pipeline Insight, 2024" report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Congenital Hyperinsulinism pipeline landscape is provided which includes the disease overview and Congenital Hyperinsulinism treatment guidelines. The assessment part of the report embraces, in depth Congenital Hyperinsulinism commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Congenital Hyperinsulinism collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

The companies and academics are working to assess challenges and seek opportunities that could influence Congenital Hyperinsulinism R&D. The therapies under development are focused on novel approaches to treat/improve Congenital Hyperinsulinism.

Congenital Hyperinsulinism Emerging Drugs Chapters

This segment of the Congenital Hyperinsulinism report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Congenital Hyperinsulinism Emerging Drugs

RZ358: Rezolute, Inc.

RZ358 is a fully human monoclonal antibody developed by Rezolute to treat congenital hyperinsulinism (HI) and other conditions characterized by excessive insulin activity. It works downstream from the pancreas and binds to a unique allosteric site on insulin receptors in the liver, fat, and muscle, counteracting the effects of excess insulin binding and activity to correct hypoglycemia. RZ358 targets the insulin receptor to reduce insulin's signal to take sugar from the bloodstream, helping to maintain safer blood sugar levels and protect the brain. It is administered intravenously and the dosage depends on the participant's weight and treatment group. The drug is currently in Phase III stage of clinical trial evaluation for the treatment of Congenital Hyperinsulinism.

Congenital Hyperinsulinism: Therapeutic Assessment

This segment of the report provides insights about the different Congenital Hyperinsulinism drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Congenital Hyperinsulinism

• There are approx. 4+ key companies which are developing the therapies for Congenital Hyperinsulinism. The companies which have their Congenital Hyperinsulinism drug candidates in the most advanced stage, i.e. Phase III include, Rezolute, Inc.

Phases

This report covers around 4+ products under different phases of clinical development like

• Late stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

Route of Administration

Congenital Hyperinsulinism pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Oral
• Intravenous
• Subcutaneous
• Parenteral
• Topical
• Molecule Type

Products have been categorized under various Molecule types such as

• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy
• Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Congenital Hyperinsulinism: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Congenital Hyperinsulinism therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Congenital Hyperinsulinism drugs.

Congenital Hyperinsulinism Report Insights

• Congenital Hyperinsulinism Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Congenital Hyperinsulinism Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Congenital Hyperinsulinism drugs?
• How many Congenital Hyperinsulinism drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Congenital Hyperinsulinism?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Congenital Hyperinsulinism therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Congenital Hyperinsulinism and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Rezolute, Inc.
• Hanmi Pharmaceutical

Key Products

• RZ358
• HM 15136

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