PUBLISHER: DelveInsight | PRODUCT CODE: 1105486
PUBLISHER: DelveInsight | PRODUCT CODE: 1105486
DelveInsight's, "Homocystinuria - Pipeline Insight, 2022," 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.
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, 2022" report by DelveInsight 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.
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 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 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 Medicines Agency.
Further product details are provided in the report……..
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:
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.
DelveInsight's report covers around 3+ products under different phases of clinical development like
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
Products have been categorized under various Molecule types such as
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.
Key Questions
Current Treatment Scenario and Emerging Therapies:
Key Players
Key Products
Introduction
Executive Summary
Homocystinuria: Overview
Pipeline Therapeutics
Therapeutic Assessment
Homocystinuria - DelveInsight's Analytical Perspective
Late Stage Products (Phase III)
Drug name: Company name
Drug profiles in the detailed report…..
Mid Stage Products (Phase II)
Drug name: Company name
Drug profiles in the detailed report…..
Early Stage Products (Phase I/II)
Pegtibatinase: Travere Therapeutics
Drug profiles in the detailed report…..
Preclinical and Discovery Stage Products
SYNB 1353: Synlogic
Drug profiles in the detailed report…..
Inactive Products
Homocystinuria Key Companies
Homocystinuria Key Products
Homocystinuria- Unmet Needs
Homocystinuria- Market Drivers and Barriers
Homocystinuria- Future Perspectives and Conclusion
Homocystinuria Analyst Views
Homocystinuria Key Companies
Appendix