Vascular Ehlers-Danlos Syndrome Pipeline Insight 2025: Emerging Therapies Target COL3A1 Mutation and Arterial Fragility | DelveInsight

DelveInsight's "Vascular Ehlers-Danlos Syndrome (vEDS) - Pipeline Insight, 2025" explores the evolving therapeutic landscape for this rare and life-threatening genetic disorder. vEDS stems from mutations in the COL3A1 gene, leading to arterial and organ fragility, spontaneous rupture risks, and high mortality at a young age. Historically, management has focused on symptom control and preventive care, as no approved therapies specifically address the disease's molecular root. However, 2025 shows promising momentum with gene-targeted therapies and repurposed agents aimed at improving vascular integrity.

Leading the pipeline is AGMB-447 from Aeglea BioTherapeutics, an enzyme therapy targeting underlying connective tissue abnormalities. X4 Pharmaceuticals' mavorixafor, a CXCR4 antagonist, is being explored for broader EDS-related vascular stabilization benefits. Moreover, CEL-SCI is investigating immune-modulatory platforms that may intersect with EDS-related inflammation and healing. Importantly, enzastaurin, a PKC-beta inhibitor from Denovo Biopharma, has also entered early-phase trials due to its potential in reducing vascular rupture by stabilizing endothelial function.

Patients and researchers are also rallying behind CRISPR/Cas9-based approaches and antisense oligonucleotides that could correct COL3A1 defects at the genetic level. While these are in preclinical or discovery phases, the entry of such technologies into rare disease drug development is a major milestone. As rare disease legislation and global collaborations accelerate orphan therapy development, the future pipeline for vEDS looks more hopeful than ever.

Interested in learning more about the current treatment landscape and the key drivers shaping the Vascular Ehlers-Danlos Syndrome pipeline? Click here: https://www.delveinsight.com/report-store/vascular-ehlers-danlos-syndrome-veds-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Key Takeaways from the Vascular Ehlers-Danlos Syndrome Pipeline Report
• DelveInsight's Vascular Ehlers-Danlos Syndrome pipeline analysis depicts a strong space with 3+ active players working to develop 3+ pipeline drugs for Vascular Ehlers-Danlos Syndrome treatment.
• The leading Vascular Ehlers-Danlos Syndrome companies include Acer Therapeutics, Aytu BioPharma, Castle Creek Pharmaceuticals, and others are evaluating their lead assets to improve the Vascular Ehlers-Danlos Syndrome treatment landscape.
• Key Vascular Ehlers-Danlos Syndrome pipeline therapies in various stages of development include Celiprolol, AR101 (enzastaurin), CCB 003, CCB 005, and others.
• In March 2025, a small pilot study explored celiprolol's impact on pulse pressure variability. While average blood pressure did not significantly change, reductions in pulse pressure variability were notable in participants who initially had high variability, illustrating potential implications for vEDS management
• In February 2025, results from the Phase III ARCADE trial revealed that adding irbesartan to oral celiprolol therapy reduced severe arterial events by 50% over two years in adults with vEDS. Researchers highlighted that this combined therapy could meaningfully change clinical management of this life-threatening condition.
• Additionally, ongoing progress continues with the DiSCOVER trial, now enrolling U.S. adults in a decentralized Phase III study evaluating ACER‐002 (celiprolol) to reduce arterial and other vascular events in patients with confirmed COL3A1 mutations. The U.S. Food and Drug Administration has granted breakthrough therapy status and accepted special protocol assessment (SPA) terms for the study design-pivotal steps toward approval.

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Vascular Ehlers-Danlos Syndrome Overview
Vascular Ehlers-Danlos Syndrome (vEDS) is a rare genetic connective tissue disorder caused primarily by mutations in the COL3A1 gene, which affects the production of type III collagen. This collagen is essential for the structural integrity of blood vessels, hollow organs, and other tissues. As a result, individuals with vEDS are at high risk for life-threatening complications such as arterial rupture, intestinal perforation, and organ rupture, often at a young age.

Symptoms of vEDS can include thin, translucent skin, easy bruising, characteristic facial features, and fragile arteries and organs. Diagnosis is typically confirmed through genetic testing. Due to the severe and unpredictable nature of the condition, management focuses on preventive care, regular monitoring, and minimizing trauma or invasive procedures. There is currently no cure, but ongoing research aims to develop targeted therapies to reduce vascular events and improve patient outcomes.

Find out more about Vascular Ehlers-Danlos Syndrome medication at https://www.delveinsight.com/report-store/vascular-ehlers-danlos-syndrome-veds-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Vascular Ehlers-Danlos Syndrome Treatment Analysis: Drug Profile
Celiprolol: Acer Therapeutics
Acer Therapeutics is developing EDSIVOTM, a new chemical entity (NCE) aimed at treating patients with COL3A1-positive vascular Ehlers-Danlos Syndrome (vEDS). Celiprolol received FDA Orphan Drug Designation for vEDS treatment in 2015. The New Drug Application (NDA) for EDSIVOTM, based on data from the BBEST trial, was accepted for priority review in October 2018. However, in June 2019, the FDA issued a Complete Response Letter (CRL) requesting a well-controlled trial to confirm whether EDSIVOTM reduces clinical events in vEDS patients. Acer appealed the decision, but the FDA denied the appeal while suggesting possible paths forward. In a Type B meeting in May 2021, Acer and the FDA discussed conducting a U.S.-based, prospective, randomized, double-blind, placebo-controlled, decentralized clinical trial for COL3A1-positive vEDS patients. The FDA agreed to a decentralized trial design with an independent adjudication committee, accepted a primary endpoint focused on clinical events, supported modest safety data collection based on the drug's known safety profile, and endorsed a statistical plan appropriate for this rare disease.

AR101 (enzastaurin): Aytu BioPharma
Enzastaurin, developed by Aytu BioPharma, is a well-studied PKCβ inhibitor evaluated in over 50 clinical trials involving more than 3,300 patients, including a Phase 3 trial with nearly 500 patients treated for three years. Mutations in the COL3A1 gene, linked to vEDS, cause loss of extracellular matrix integrity and increase symptoms like arterial dissection and rupture. Recent animal studies using a vEDS mouse model with similar Col3a1 mutations showed that the mutation drives increased PKC/ERK pathway signaling. Treatment with a PKCβ inhibitor in this model significantly reduced deaths from spontaneous aortic rupture. Further research is needed to explore the potential of PKC inhibition as a treatment for vEDS.

Learn more about the novel and emerging Vascular Ehlers-Danlos Syndrome pipeline therapies at https://www.delveinsight.com/report-store/vascular-ehlers-danlos-syndrome-veds-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Vascular Ehlers-Danlos Syndrome Therapeutics Assessment
By Product Type
• Mono
• Combination
• Mono/Combination.

By Stage
• 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

By Route of Administration
• Inhalation
• Inhalation/Intravenous/Oral
• Intranasal
• Intravenous
• Intravenous/ Subcutaneous
• NA
• Oral
• Oral/intranasal/subcutaneous
• Parenteral
• Subcutaneous

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

Scope of the Vascular Ehlers-Danlos Syndrome Pipeline Report
• Coverage: Global
• Key Vascular Ehlers-Danlos Syndrome Companies: Acer Therapeutics, Aytu BioPharma, Castle Creek Pharmaceuticals, and others.
• Key Vascular Ehlers-Danlos Syndrome Pipeline Therapies: Celiprolol, AR101 (enzastaurin), CCB 003, CCB 005, and others.

To dive deep into rich insights for drugs used for Vascular Ehlers-Danlos Syndrome treatment, visit: https://www.delveinsight.com/report-store/vascular-ehlers-danlos-syndrome-veds-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Table of Contents
1. Introduction
2. Executive Summary
3. Vascular Ehlers-Danlos Syndrome Pipeline: Overview
4. Analytical Perspective In-depth Commercial Assessment
5. Vascular Ehlers-Danlos Syndrome Pipeline Therapeutics
6. Vascular Ehlers-Danlos Syndrome Pipeline: Late-Stage Products (Phase III)
7. Vascular Ehlers-Danlos Syndrome Pipeline: Mid-Stage Products (Phase II)
8. Vascular Ehlers-Danlos Syndrome Pipeline: Early Stage Products (Phase I)
9. Therapeutic Assessment
10. Inactive Products
11. Company-University Collaborations (Licensing/Partnering) Analysis
12. Key Companies
13. Key Products
14. Unmet Needs
15. Market Drivers and Barriers
16. Future Perspectives and Conclusion
17. Analyst Views
18. Appendix

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Jatin Vimal
jvimal@delveinsight.com
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This release was published on openPR.