Congenital Hyperinsulinism (CHI) - Pipeline Insight, 2024

This “Congenital Hyperinsulinism (CHI)- Pipeline Insight, 2024” report provides comprehensive insights about 4+ companies and 6+ 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 is a genetic disorder in which the insulin cells of the pancreas, called beta cells, secrete too much insulin. Excess insulin causes low plasma sugar (hypoglycemia) or low blood sugar. Ordinarily, beta cells secrete just enough insulin to keep the blood sugar in the normal range. In children with HI, the secretion of insulin is not properly regulated, causing excess insulin secretion and low blood sugar. Low blood sugar can be very dangerous, because the brain needs a constant source of sugar. If the brain doesn't get the sugar it needs, it can lead to seizures, brain damage, and possibly death.

Congenital hyperinsulinism causes low plasma sugar (hypoglycemia).The symptoms of hypoglycemia in infants are often difficult to identify, as they can be similar to normal infant activities. Common symptoms of hypoglycemia include irritability, sleepiness, lethargy, excessive hunger and rapid heart rate. More severe symptoms, such as seizures and coma, can occur with a prolonged low plasma sugar or an extremely low plasma sugar.

The genetic and molecular cause of CHI includes defects in key genes regulating insulin secretion from the pancreatic ß-cell. Molecular defects in previously described genes (ABCC8, KCNJ11, GLUD1, GCK, HADH, SLC16A1, UCP2, HNF4A, HNF1A, HK1, PGM1, and PMM2) have been reported. However, recent studies have linked the role of other genes (CACNA1D, FOXA2) to hyperinsulinaemic hypoglycaemia (HH) but in some of these cases the underlying molecular mechanisms are still not fully elucidated. Understanding the molecular mechanisms of CHI due to these genetic abnormalities has provided unique insight into the normal physiological mechanisms which regulate the insulin release.

Broadly, these genetic defects leading to unregulated insulin secretion can be grouped into four main categories. The first group consists of defects in genes encoding the pancreatic KATP channels (ABCC8 and KCNJ11) and other channel/transporter proteins (KCNQ1, CACNA1D, SLC16A1). Pancreatic KATP channels have a critical role in the regulation of insulin release and defects in their encoding genes cause the most prevalent and severe forms of CHI. The second and third categories of conditions are enzymatic gene defects (GLUD1, GCK, HADH, UCP2, HK1, PMM2, PGM1) and defects in genes encoding the transcription factors (HNF1A, HNF4A, FOXA2) leading to changes in nutrient flux into metabolic pathways which converge on insulin secretion. The ß-cell insulin release is coupled to the metabolic signals and so any perturbation in these pathways will ultimately result in inappropriate insulin secretion. The underlying molecular mechanism leading to unregulated insulin secretion has yet to be clarified.

Repeat episodes and/or dangerously low blood sugars increase the risk of neurological and developmental complications, including persistent feeding problems, learning disabilities, recurrent seizures, and/or brain damage, or even death. Existing medical options were not developed for CHI and are often either ineffective since certain groups of patients do not respond to these therapies, or are associated with substantial side effects that discourage compliance and lead to suboptimal treatment outcomes. Surgical removal of the pancreas is also an option, but this approach is invasive, may require repeat surgery, and ultimately leads to the development of lifelong insulin-dependent diabetes. CHI is a rare pediatric disease that affects mainly newborns, infants and toddlers. Due to a genetic defect in the insulin producing cells, these children have increased insulin levels, resulting in persistent and recurrent hypoglycemia throughout childhood. Current treatment options are limited, complex and may be insufficient to adequately control hypoglycemia.

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 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

Dasiglucagon: Zealand PharmaDasiglucagon is being investigated by Zealand Pharma for subcutaneous continuous infusion using a wearable pump system as a potential treatment of CHI. Dasiglucagon is a glucagon receptor agonist that works by causing the liver to release stored sugar to the blood. Zealand Pharma has a collaborative development and supply agreement with DEKA Research & Development Corporation and affiliates for the wearable subcutaneous infusion pump system. In June 2023, Zealand Pharma announced the submission of a New Drug Application (NDA) to the US Food and Drug Administration (FDA) for dasiglucagon for the prevention and treatment of low blood sugar (hypoglycemia) in pediatric patients 7 days of age or older with congenital hyperinsulinism.

RZ358: RezoluteRZ358 is an intravenously administered human monoclonal antibody that binds to a unique site (allosteric) on the insulin receptor throughout the body, such as in the liver, fat, and muscle. The antibody modifies insulin’s binding and signaling to maintain glucose levels in a normal range which counteracts the effects of elevated insulin in the body. Rezolute believes that RZ358 is ideally suited as a potential therapy for conditions characterized by excessive insulin levels and is developing it to treat the hyperinsulinism and low blood sugar characteristic of diseases such as congenital hyperinsulinism (CHI). As RZ358 acts downstream from the beta cells, it has the potential to be universally effective at treating HI caused by any of the underlying genetic defects. RZ358 received Orphan Drug Designation in the United States and European Union as well as Pediatric Rare Disease Designation in the US. Currently, the drug is in the Phase II stage of its development 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. preregistration include, Zealand Pharma.

Phases

This report covers around 6+ 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

• Crinetics Pharmaceuticals
• Hanmi Pharmaceutical
• Rezolute
• Zealand Pharma

Key Products

• HM 15136
• RZ358
• Dasiglucagon
• CRN-04777

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