Vascular Ehlers-Danlos Syndrome - Pipeline Insight, 2025

This “Vascular Ehlers-Danlos Syndrome - Pipeline Insight, 2025,” report provides comprehensive insights about 3+ companies and 3+ pipeline drugs in Vascular Ehlers-Danlos Syndrome 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.

Vascular Ehlers-Danlos Syndrome Understanding

Vascular Ehlers-Danlos Syndrome: Overview

Vascular Ehlers-Danlos syndrome is an inherited connective tissue disorder that is caused by defects in a protein called collagen. It is generally considered the most severe form of Ehlers-Danlos syndrome (EDS). Common symptoms include thin, translucent skin; easy bruising; characteristic facial appearance; and fragile arteries, muscles and internal organs. Vascular EDS is usually caused by a change in the COL3A1 gene. Rarely, it may be caused by a genetic change in the COL1A1 gene. Inheritance is autosomal dominant. Typical physical signs associated with vEDS are predominantly cutaneous, as easy bruising unrelated to trauma, thin translucent skin predominantly on the upper torso and abdomen, with abnormally visible veins. Wound healing may be delayed and may result in widened, papyraceous scars, particularly over prominent bony pressure points (knee). Extremities, particularly hands may appear prematurely aged (acrogeria). Characteristic facial features are prominent eyes, thin lips, sunken cheeks and a pinched nose. Diagnosis is suspected on the base of physical signs and clinical complications in probands, and requires the identification of a pathogenic variant within the COL3A1 gene by molecular genetic testing (single gene testing or multigene panel including COL3A1).

'Vascular Ehlers-Danlos Syndrome - Pipeline Insight, 2025' report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Vascular Ehlers-Danlos Syndrome pipeline landscape is provided which includes the disease overview and Vascular Ehlers-Danlos Syndrome treatment guidelines. The assessment part of the report embraces, in depth Vascular Ehlers-Danlos Syndrome 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, Vascular Ehlers-Danlos Syndrome 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 Vascular Ehlers-Danlos Syndrome.

In the coming years, the Vascular Ehlers-Danlos Syndrome 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 Vascular Ehlers-Danlos Syndrome 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 Vascular Ehlers-Danlos Syndrome treatment market. Several potential therapies for Vascular Ehlers-Danlos Syndrome are under investigation. With the expected launch of these emerging therapies, it is expected that there will be a significant impact on the Vascular Ehlers-Danlos Syndrome market size in the coming years.

Our in-depth analysis of the pipeline assets (in early-stage, mid-stage and late stage of development for the treatment of Vascular Ehlers-Danlos Syndrome) 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.

Vascular Ehlers-Danlos Syndrome Emerging Drugs Chapters

This segment of the Vascular Ehlers-Danlos Syndrome 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.

Vascular Ehlers-Danlos Syndrome Emerging Drugs

Celiprolol: Acer Therapeutics

Acer is developing EDSIVO™, a new chemical entity (NCE), for the treatment of COL3A1-positive vEDS patients. Celiprolol received FDA Orphan Drug Designation for the treatment of vEDS in 2015. The EDSIVO™ NDA was originally submitted based on data obtained from the BBEST trial4 and accepted for filing in October 2018 with priority review. Following FDA review, Acer received a Complete Response Letter (CRL) in June 2019 stating that it will be necessary to conduct an adequate and well-controlled trial to determine whether EDSIVO™ reduces the risk of clinical events in patients with vEDS. Acer subsequently appealed the FDA decision and while FDA denied the appeal, it described possible paths forward for Acer to explore. In a Type B meeting in May 2021, the Company discussed with FDA the conduct of an U.S.-based prospective, randomized, double-blind, placebo-controlled, decentralized clinical trial in patients with COL3A1-positive vEDS, and sought FDA’s opinion on various proposed design features of the study. The official Type B meeting minutes outlined: the acceptability of a decentralized (virtual) clinical trial design and use of an independent centralized adjudication committee; acceptability of a primary endpoint based on clinical events associated with disease outcome; agreement with modest safety data collection (based on the known safety profile of the drug; and a statistical plan that considers the rare disease classification of vEDS.

AR101 (enzastaurin): Aytu BioPharma

Enzastaurin is a well characterized PKCβ inhibitor that has been evaluated in over 50 clinical trials, with more than 3300 patients. This includes a Phase 3 study of nearly 500 patients with 3 years of enzastaurin treatment. Mutations in the COL3A1 gene have been linked to the loss of structural integrity of the extracellular matrix and increased clinical presentation of VEDS related symptoms, including arterial dissection and/or rupture. Recent findings from animal studies, in a VEDS mouse model, with similar Col3A1 mutations have shown that the mutation is a key mediator in increased PKC/ERK pathway signaling. Additionally, in this model, treatment with an inhibitor of PKCβ significantly prevented death due to spontaneous aortic rupture. Further investigation will be necessary to determine the potential of PKC inhibition as a treatment option.

Vascular Ehlers-Danlos Syndrome: Therapeutic Assessment

This segment of the report provides insights about the different Vascular Ehlers-Danlos Syndrome drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Vascular Ehlers-Danlos Syndrome

There are approx. 3+ key companies which are developing the therapies for Vascular Ehlers-Danlos Syndrome. The companies which have their Vascular Ehlers-Danlos Syndrome drug candidates in the most advanced stage, i.e. preregistration include, Acer Therapeutics.

Phases

The 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

Vascular Ehlers-Danlos Syndrome 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.

Vascular Ehlers-Danlos Syndrome: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Vascular Ehlers-Danlos Syndrome 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 Vascular Ehlers-Danlos Syndrome drugs.

Vascular Ehlers-Danlos Syndrome Report Insights

• Vascular Ehlers-Danlos Syndrome Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• Acer Therapeutics
• Aytu BioPharma
• Castle Creek Pharmaceuticals

Key Products

• Celiprolol
• AR101 (enzastaurin)
• CCB 003
• CCB 005