Propionic Acidemia - Pipeline Insight, 2024

This “Propionic Acidemia - Pipeline Insight, 2024,” report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in Propionic Acidemia 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

Propionic Acidemia Understanding

Propionic Acidemia: Overview

Propionic acidemia is a rare metabolic disorder affecting from 1/20,000 to 1/250,000 individuals in various regions of the world. It is characterized by deficiency of propionyl-CoA carboxylase, an enzyme involved in the breakdown (catabolism) of the chemical “building blocks” (amino acids) of proteins. Symptoms most commonly become apparent during the first weeks of life and may include abnormally diminished muscle tone (hypotonia), poor feeding, vomiting, listlessness (lethargy), dehydration and seizures. Without appropriate treatment, coma and death may result. Rarely, the condition may become apparent later in life and may be associated with less severe symptoms and findings. Propionic acidemia is inherited in an autosomal recessive pattern. Individuals with this condition have to follow a specific diet including a low protein intake and specific food formulas (medical foods). Liver transplant is a surgical option that can help decrease the frequency of acute metabolic episodes (decompensation).

Propionic Acidemia - Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Propionic Acidemia pipeline landscape is provided which includes the disease overview and Propionic Acidemia treatment guidelines. The assessment part of the report embraces, in depth Propionic Acidemia 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, Propionic Acidemia 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 Propionic Acidemia.
• In the coming years, the Propionic Acidemia 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 Propionic Acidemia 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 Propionic Acidemia treatment market. Several potential therapies for Propionic Acidemia are under investigation. With the expected launch of these emerging therapies, it is expected that there will be a significant impact on the Propionic Acidemia 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 Propionic Acidemia) 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.

Propionic Acidemia Emerging Drugs Chapters

This segment of the Propionic Acidemia 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.

Propionic Acidemia Emerging Drugs

m RNA-3927: Modernam RNA-3927 is designed to instruct the body to restore the missing or dysfunctional proteins that cause PA. mRNA-3927 contains two mRNAs that encode for the alpha and beta subunits of the mitochondrial enzyme propionyl-CoA carboxylase (PCC), encapsulated within Moderna’s proprietary lipid nanoparticle (LNP). mRNA-3927 is intended to treat patients with PA regardless of whether they are missing the alpha or beta subunits. mRNA-3927 uses the same proprietary LNP formulation used in the Company’s antibody against chikungunya virus (mRNA-1944) and MMA (mRNA-3704) programs. In addition to Fast Track designation, mRNA-3927 has also been granted Orphan Drug and Rare Pediatric Disease designations from the FDA and Orphan Designation by the European MedicinesAgency(EMA).

HST 5040: Hemo Shear Therapeutics HST5040 is an investigational oral small molecule therapy being developed by HemoShear to reduce the levels of toxins associated with methylmalonic acidemia (MMA) and propionic acidemia (PA), rare genetic disorders caused by the de?ciency of certain enzymes required to metabolize amino acids. HST5040 is formulated for convenient daily administration at home as a liquid taken either orally or through a gastrostomy tube. The FDA has granted HST5040 Orphan Drug, Fast Track and Rare Pediatric Disease designations for the treatment of MMA andPA.

Propionic Acidemia: Therapeutic Assessment

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

Major Players in Propionic Acidemia

There are approx. 5+ key companies which are developing the therapies for Propionic Acidemia. The companies which have their Propionic Acidemia drug candidates in the most advanced stage, i.e. phase I/II include, Moderna.

Phases

This report covers around 5+ 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

Propionic Acidemia 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.

Propionic Acidemia: Pipeline Development Activities

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

Propionic Acidemia Report Insights

• Propionic Acidemia Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• Moderna
• Hemo ShearTherapeutics
• CoA Therapeutics, Inc.
• Agios Pharmaceuticals
• Imbria Pharmaceuticals
• Comet Therapeutics

Key Products

• mRNA-3927
• HST 5040
• BBP-671
• Research programme - BCAT2 inhibitors
• IMB-203
• VB 1197