Steroid Refractory Acute Graft-versus-host Disease - Pipeline Insight, 2025

This “Steroid refractory acute graft-versus-host disease - Pipeline Insight, 2025” report provides comprehensive insights about 3+ companies and 4+ pipeline drugs in Steroid refractory acute graft-versus-host disease 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.

Steroid refractory acute graft-versus-host disease: Understanding

Steroid refractory acute graft-versus-host disease: Overview

Steroid-refractory acute graft-versus-host disease (SR-aGVHD) is a severe complication following allogeneic hematopoietic stem cell transplantation, where the disease persists or worsens despite high-dose steroid treatment. Affecting 35-50% of patients, SR-aGVHD is associated with a poor prognosis, with a 6-month survival rate of approximately 49%. Although alternative immunosuppressive therapies may provide partial responses in some patients, the overall outcomes remain challenging, and long-term survival is rare. The lack of effective treatment options has made SR-aGVHD a major cause of morbidity and mortality post-transplant. Ongoing research is focused on developing more targeted therapies to enhance survival and quality of life for patients with this complex disease.

The pathophysiology of acute graft-versus-host disease (aGVHD) unfolds in three distinct phases. Initially, conditioning treatments (e.g., chemotherapy or radiation) damage recipient tissues, triggering the release of pro-inflammatory cytokines like TNF-α and IL-6, which activate host antigen-presenting cells (APCs). In phase II, these APCs activate donor-derived T cells through cytokines like IL-12 and IL-23, promoting Th1 cytokine production and T cell migration to target organs. In phase III, T cells and other immune cells, including natural killer cells, induce tissue damage through apoptosis. In contrast, the pathophysiology of steroid-refractory GVHD (SR-aGVHD) remains poorly understood. Although initial treatment targets T cells, refractory cases continue to experience organ damage, as indicated by persistently elevated levels of cytokeratin-18 fragments, a marker of epithelial apoptosis.

Steroid-refractory acute Graft-versus-Host Disease (SR-aGVHD) is diagnosed through a comprehensive clinical evaluation involving laboratory analysis, imaging, and potentially endoscopic examination to exclude alternative diagnoses. The diagnosis typically involves grading the severity from mild (grade I) to very severe (grade IV) using the modified Glucksberg staging system. SR-aGVHD is defined as disease progression after 3-5 days of steroid treatment or no response after 5-7 days of treatment, with the specific criteria potentially varying between medical centers. Histopathological confirmation through skin, liver, or mucous membrane biopsies can help substantiate the diagnosis, although these biopsies may lack complete sensitivity and specificity.

The treatment of steroid-refractory acute graft-versus-host disease (SR-aGVHD) remains a significant clinical challenge, with limited effective therapeutic options. Ruxolitinib, an FDA-approved Janus kinase inhibitor, has emerged as a key treatment, demonstrating substantial improvements in organ function and survival in clinical trials. In addition to ruxolitinib, second-line treatments include various immunosuppressive agents, as well as emerging therapies such as mesenchymal stromal cell (MSC) therapy with remestemcel-L. Despite these advances, the weighted average 6-month survival estimate for patients with SR-aGVHD remains low, around 49%, underscoring the urgent need for more effective therapies. Treatment decisions are typically individualized, taking into account factors such as disease progression, patient characteristics, and the potential for treatment-related toxicity.

'Steroid refractory acute graft-versus-host disease- Pipeline Insight, 2025' report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Steroid refractory acute graft-versus-host disease pipeline landscape is provided which includes the disease overview and Steroid refractory acute graft-versus-host disease treatment guidelines. The assessment part of the report embraces, in depth Steroid refractory acute graft-versus-host disease 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, Steroid refractory acute graft-versus-host disease collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

The companies and academics are working to assess challenges and seek opportunities that could influence Steroid refractory acute graft-versus-host disease R&D. The therapies under development are focused on novel approaches to treat/improve Steroid refractory acute graft-versus-host disease.

Steroid refractory acute graft-versus-host disease Emerging Drugs Chapters

This segment of the Steroid refractory acute graft-versus-host disease 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.

Steroid refractory acute graft-versus-host disease Emerging Drugs

MaaT013: MaaT Pharma

MaaT013 is an innovative, first-in-class, pooled allogeneic microbiome therapeutic developed by MaaT Pharma for treating steroid-refractory acute Graft-versus-Host Disease (SR-aGVHD). This therapeutic approach involves administering a standardized, carefully screened fecal microbiota preparation derived from multiple donors, designed to restore gut microbiome diversity and modulate immune responses in patients who have failed standard steroid treatments. It is a donor-derived, standardized, high-richness, high-diversity microbiome ecosystem therapy, containing Butycore, a group of bacterial species known to produce anti-inflammatory short-chain-fatty acids. MaaT013 has received Orphan Drug Designation from FDA and EMA. Currently, the drug is in Phase III stage of its development for the treatment of Steroid refractory acute graft-versus-host disease.

RLS-0071: ReAlta Life Sciences, Inc.

RLS-0071 (pegtarazimod) is an innovative investigational therapeutic candidate developed by ReAlta Life Sciences, Inc. for treating Steroid-Refractory Acute Graft-Versus-Host Disease (SR-aGvHD). Based on the company's unique EPICC peptide platform, RLS-0071 features a dual-targeting mechanism designed to modulate both complement and innate inflammatory pathways, offering a novel approach to addressing this serious post-transplantation complication. The drug has received Orphan Drug Designation and Fast Track Designation from the FDA. Currently, the drug is in Phase II stage of its clinical trial for the treatment of Steroid refractory acute graft-versus-host disease.

Steroid refractory acute graft-versus-host disease: Therapeutic Assessment

This segment of the report provides insights about the different Steroid refractory acute graft-versus-host disease drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Steroid refractory acute graft-versus-host disease

• There are approx. 3+ key companies which are developing the therapies for Steroid refractory acute graft-versus-host disease. The companies which have their Steroid refractory acute graft-versus-host disease drug candidates in the most advanced stage, i.e. Phase III include, MaaT Pharma

Phases

The report covers around 4+ 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

Steroid refractory acute graft-versus-host disease pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Oral
• Intravenous
• Subcutaneous
• Parenteral
• Topical

Molecule Type

Products have been categorized under various Molecule types such as

• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy

Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Steroid refractory acute graft-versus-host disease: Pipeline Development Activities

The report provides insights into different therapeutic candidates in Phase III, II, I, preclinical and discovery stage. It also analyses Steroid refractory acute graft-versus-host disease 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 Steroid refractory acute graft-versus-host disease drugs.

Steroid refractory acute graft-versus-host disease Report Insights

• Steroid refractory acute graft-versus-host disease Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• MaaT Pharma
• ReAlta Life Sciences, Inc.

Ironwood Pharmaceuticals

• Wuhan Optics Valley Vcanbiopharma Co., Ltd.

Key Products

• MaaT013
• RLS-0071
• Apraglutide
• VUM02

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