Homocystinuria - Pipeline Insight, 2024

This “Homocystinuria - Pipeline Insight, 2024,” report provides comprehensive insights about 3+ companies and 3+ pipeline drugs in Homocystinuria 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

Homocystinuria Understanding

Homocystinuria: Overview

Homocystinuria is a disorder of amino acid metabolism that is caused by a lack of the enzyme cystathionine beta-synthase, which is needed to metabolize the amino acid homocysteine. This disorder can cause a number of symptoms, including decreased vision, intellectual disability, and skeletal abnormalities. Homocystinuria occurs when parents pass the defective genes that cause this disorder on to their children. Infants with this disorder are normal at birth. The first symptoms, including dislocation of the lens of the eye causing severely decreased vision, usually begin after 3 years of age. Most children have skeletal abnormalities, including osteoporosis. Children are usually thin with a curved spine, chest deformities, elongated limbs, and long, spiderlike fingers. Without early diagnosis and treatment, mental (psychiatric) and behavioral disorders and intellectual disability are common. Homocystinuria makes the blood more likely to clot spontaneously, resulting in strokes, high blood pressure, and many other serious problems.

Homocystinuria - Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Homocystinuria pipeline landscape is provided which includes the disease overview and Homocystinuria treatment guidelines. The assessment part of the report embraces, in depth Homocystinuria 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, Homocystinuria collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

• A better understanding of disease pathogenesis contributing to the development of novel therapeutics for Homocystinuria.
• In the coming years, the Homocystinuria market is set to change due to the rising awareness of the disease, and incremental healthcare spending across the world; which would expand the size of the market to enable the drug manufacturers to penetrate more into the market.

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

• A detailed portfolio of major pharma players who are involved in fueling the Homocystinuria treatment market. Several potential therapies for Homocystinuria are under investigation. With the expected launch of these emerging therapies, it is expected that there will be a significant impact on the Homocystinuria market size in the coming years.
• This in-depth analysis of the pipeline assets (in early-stage, mid-stage and late stage of development for the treatment of Homocystinuria) includes therapeutic assessment and comparative analysis. This will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the research and development activities.

Homocystinuria Emerging Drugs Chapters

This segment of the Homocystinuria 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.

Homocystinuria Emerging Drugs

Pegtibatinase: Travere Therapeutics Pegtibatinase is a novel investigational human enzyme replacement candidate being evaluated in Phase 1/2 development for the treatment of classical homocystinuria (HCU). Classical HCU is a rare metabolic disorder characterized by elevated levels of plasma homocysteine that can lead to vision, skeletal, circulatory and central nervous system issues. Pegtibatinase is currently advancing in a Phase 1/2 dose escalation study to assess its safety, tolerability, pharmacokinetics, pharmacodynamics and clinical effects in patients with classical HCU. Pegtibatinase has been granted Rare Pediatric Disease and Fast Track designations by the US Food and Drug Administration (FDA), as well as orphan drug designation in the US and Europe. On December 15, 2021, Travere announced positive topline results from the ongoing Phase 1/2 COMPOSE Study of pegtibatinase. In the highest dose cohort to date evaluating 1.5mg/kg of pegtibatinase twice weekly (BIW), treatment with pegtibatinase resulted in rapid and sustained reductions in total homocysteine (tHcy) through 12 weeks of treatment, including a 55.1% mean relative reduction in tHcy from baseline as well as maintenance of tHcy below a clinically meaningful threshold of 100 µmol. To date in the study, pegtibatinase has been generally well-tolerated.

AGLE-177: Aeglea Biotherapeutics AGLE-177 is a novel engineered human enzyme designed to degrade both homocysteine and homocystine (two homocysteine molecules bound together) to lower abnormally high levels of homocysteine in the blood. We have initiated a first-in-human Phase 1/2 clinical trial to investigate the effectiveness of AGLE-177 in lowering levels of total homocysteine (homocysteine plus homocystine) in people with Homocystinuria. AGLE-177 has been granted Rare Pediatric Disease and Orphan Drug Designations by the U.S. FDA and received Orphan Drug Designation from the European MedicinesAgency.

Homocystinuria: Therapeutic Assessment

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

Major Players in Homocystinuria

There are approx. 3+ key companies which are developing the therapies for Homocystinuria. The companies which have their Homocystinuria drug candidates in the most advanced stage, i.e. phase I/II include, Travere Therapeutics.

Phases

This report covers around 3+ 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

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

• Inhalation
• Inhalation/Intravenous/Oral
• Intranasal
• Intravenous
• Intravenous/ Subcutaneous
• NA
• Oral
• Oral/intranasal/subcutaneous
• Parenteral
• Subcutaneous

Molecule Type

Products have been categorized under various Molecule types such as

• Antibody
• Antisense oligonucleotides
• Immunotherapy
• Monoclonal antibody
• Peptides
• Protein
• Recombinant protein
• Small molecule
• Stem Cell
• Vaccine

Product Type

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

Homocystinuria: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Homocystinuria 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 Homocystinuria drugs.

Homocystinuria Report Insights

• Homocystinuria Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Homocystinuria 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 Homocystinuria drugs?
• How many Homocystinuria drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Homocystinuria?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Homocystinuria 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 Homocystinuria and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Travere Therapeutics
• Aeglea Biotherapeutics
• Synlogic
• Codexis

Key Products

• Pegtibatinase
• ACN00177
• SYNB1353
• CDX 6512