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Methylmalonic academia - Pipeline Insight, 2021

May 2021 | 60 pages | ID: MC0D627C56E1EN
DelveInsight

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DelveInsight’s, “Methylmalonic academia - Pipeline Insight, 2021,” report provides comprehensive insights about 6+ companies and 6+ pipeline drugs in Methylmalonic academia 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
Methylmalonic academia Understanding

Methylmalonic academia: Overview

Methylmalonic acidemia refers to a group of inherited conditions in which the body can’t breakdown certain parts of proteins and fats. This leads to a build-up of toxic substances and bouts of serious illness called decompensation events or metabolic crises. Symptoms of a decompensation event include poor feeding, vomiting, trouble breathing, and lack of energy (lethargy). These can occur at different ages and can range from mild to severe. Methylmalonic acidemia is caused by changes in several different genes and is inherited in an autosomal recessive fashion. Treatment includes aggressive management of decompensation events, a low-protein diet, certain medications, antibiotics and, in some cases, liver and kidney transplantation. Some subtypes of methylmalonic acidemia respond to vitamin B12. Long-term complications can include growth delay, intellectual disability, kidney disease, and pancreatitis. Methylmalonic acidemia can be isolated or may occur along with another condition called homocystinuria.

Report Highlights
  • The companies and academics are working to assess challenges and seek opportunities that could influence Methylmalonic academia R&D. The therapies under development are focused on novel approaches for Methylmalonic acidemia.
Methylmalonic academia Emerging Drugs Chapters

This segment of the Methylmalonic academia 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.

Methylmalonic academia Emerging Drugs
  • HST5040: HemoShear Therapeutics
HST5040 is an investigational oral small molecule therapy developed by HemoShear to address metabolic abnormalities associated with MMA and PA. Because HST5040 is a small molecule, it has the ability to distribute to multiple affected tissues and thus has the potential to be active throughout the body, including the brain, heart, liver, kidneys, and muscles. HST5040 is designed for convenient daily administration at home as a liquid formulation taken either orally or through a gastric feeding tube.

In December, 2020, HemoShear Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designations to HemoShear’s HST5040 oral small molecule for treatment of methylmalonic acidemia (MMA) and propionic acidemia (PA). HST5040 was granted Fast Track and Rare Pediatric Disease designations to treat MMA and PA earlier in 2020.
  • LB-001: LogicBio Therapeutics
LB-001 is being developed by LogicBio Therapeutics for Methylmalonic academia. It is currently in phase I/II stage of development. LB-001 acts as Gene transference; Methylmalonyl-CoA mutase replacements.

In November, 2020, LogicBio Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to its clinical candidate, LB-001 for the treatment methylmalonic acidemia (MMA). According to the FDA, the purpose of Fast Track designation is to get important new drugs to patients earlier by facilitating the development, and expediting the review, of drugs to treat serious conditions and fill an unmet medical need.

Further product details are provided in the report

Methylmalonic academia: Therapeutic Assessment

This segment of the report provides insights about the different Methylmalonic academia drugs segregated based on following parameters that define the scope of the report, such as:
  • Major Players working on Methylmalonic academia
There are approx. 6+ key companies which are developing the Methylmalonic acidemia. The companies which have their Methylmalonic academia drug candidates in the most advanced stage, i.e. Phase II include, HemoShear Therapeutics.
  • Phases
DelveInsight’s report covers around 6+ products under different phases of clinical development like
  • Late-stage products (Phase III and
  • Mid-stage products (Phase II and
  • Early-stage products (Phase I/II and Phase I) along with the details of
  • Pre-clinical and Discovery stage candidates
  • Discontinued & Inactive candidates
  • Route of Administration
Methylmalonic academia pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as
  • Infusion
  • Intradermal
  • Intramuscular
  • Intranasal
  • Intravaginal
  • Oral
  • Parenteral
  • Subcutaneous
  • Topical
  • Molecule Type
Products have been categorized under various Molecule types such as
  • Vaccines
  • Monoclonal Antibody
  • Peptides
  • Polymer
  • Small molecule
  • Product Type
Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Methylmalonic academia: Pipeline Development Activities

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

Methylmalonic academia Report Insights
  • Methylmalonic academia Pipeline Analysis
  • Therapeutic Assessment
  • Unmet Needs
  • Impact of Drugs
Methylmalonic academia Report Assessment
  • Pipeline Product Profiles
  • Therapeutic Assessment
  • Pipeline Assessment
  • Inactive drugs assessment
  • Unmet Needs
Key Questions

Current Scenario and Emerging Therapies:
  • How many companies are developing Methylmalonic academia drugs?
  • How many Methylmalonic academia drugs are developed by each company?
  • How many emerging drugs are in mid-stage, and late-stage of development for Methylmalonic academia?
  • What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Methylmalonic academia 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 Methylmalonic academia and their status?
  • What are the key designations that have been granted to the emerging drugs?
Key Players
  • HemoShear Therapeutics
  • LogicBio Therapeutics
  • Selecta Biosciences
  • Asklepios BioPharmaceutical
  • Moderna Therapeutics
  • Poseida Therapeutics
Key Products
  • HST5040
  • LB-001
  • AAV8
  • MMA-101
  • mRNA-3705
  • P-MMUT-101
Introduction
Executive Summary
Methylmalonic academia: Overview
  Structure
  Mechanism of Action
Pipeline Therapeutics
  Comparative Analysis
Therapeutic Assessment
  Assessment by Product Type
  Assessment by Stage and Product Type
  Assessment by Route of Administration
  Assessment by Stage and Route of Administration
  Assessment by Molecule Type
  Assessment by Stage and Molecule Type
Methylmalonic acidemia– DelveInsight’s Analytical Perspective
In-depth Commercial Assessment
  Methylmalonic academia companies’ collaborations, Licensing, Acquisition -Deal Value Trends
Methylmalonic acidemiaCollaboration Deals
  Company-Company Collaborations (Licensing / Partnering) Analysis
  Company-University Collaborations (Licensing / Partnering) Analysis
Late Stage Products (Phase III)
  Comparative Analysis
Drug Name: Company Name
  Product Description
  Research and Development
  Product Development Activities
Drug profiles in the detailed report
Mid Stage Products (Phase II)
  Comparative Analysis
HST5040: HemoShear Therapeutics
  Product Description
  Research and Development
  Product Development Activities
Drug profiles in the detailed report
Early Stage Products (Phase I/II)
  Comparative Analysis
LB-001: LogicBio Therapeutics
  Product Description
  Research and Development
  Product Development Activities
Drug profiles in the detailed report
Pre-clinical and Discovery Stage Products
  Comparative Analysis
mRNA-3705: Moderna Therapeutics
  Product Description
  Research and Development
  Product Development Activities
Drug profiles in the detailed report

Inactive Products
  Comparative Analysis
Methylmalonic acidemiaKey Companies
Methylmalonic acidemiaKey Products
Methylmalonic academia- Unmet Needs
Methylmalonic academia- Market Drivers and Barriers
Methylmalonic academia- Future Perspectives and Conclusion
Methylmalonic academia Analyst Views
Methylmalonic academia Key Companies
Appendix

LIST OF TABLES

Table 1 Total Products for Methylmalonic academia
Table 2 Late Stage Products
Table 3 Mid Stage Products
Table 4 Early Stage Products
Table 5 Pre-clinical & Discovery Stage Products
Table 6 Assessment by Product Type
Table 7 Assessment by Stage and Product Type
Table 8 Assessment by Route of Administration
Table 9 Assessment by Stage and Route of Administration
Table 10 Assessment by Molecule Type
Table 11 Assessment by Stage and Molecule Type
Table 12 Inactive Products

LIST OF FIGURES

Figure 1 Total Products for Methylmalonic academia
Figure 2 Late Stage Products
Figure 3 Mid Stage Products
Figure 4 Early Stage Products
Figure 5 Preclinical and Discovery Stage Products
Figure 6 Assessment by Product Type
Figure 7 Assessment by Stage and Product Type
Figure 8 Assessment by Route of Administration
Figure 9 Assessment by Stage and Route of Administration
Figure 10 Assessment by Molecule Type
Figure 11 Assessment by Stage and Molecule Type
Figure 12 Inactive Products


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