Mesoblast and NIH’s BMT CTN Launch Pivotal Trial for Ryoncil in Severe Adult aGvHD

Mesoblast and NIH’s BMT CTN Launch Pivotal Trial for Ryoncil in Severe Adult aGvHD

By, Admin 24 November 2025

Mesoblast has announced a major step toward expanding treatment options for adults with severe acute graft-versus-host disease (aGvHD) who no longer respond to corticosteroids.
The company will partner with the NIH-funded Blood and Marrow Transplant Clinical Trials Network (BMT CTN) on a pivotal trial evaluating Ryoncil (remestemcel-L-rknd) as part of first-line therapy for steroid-refractory aGvHD (SR-aGvHD).

This collaboration is significant. BMT CTN represents U.S. centers performing nearly 80% of all allogeneic bone marrow transplants—making it one of the most influential bodies in the field.

Why This Trial Matters?

Adults with severe SR-aGvHD face poor outcomes:

  • 44–58% of patients treated with ruxolitinib do not respond by Day 28
  • Those who fail ruxolitinib show only 20–30% survival by Day 100
  • Mortality remains high despite available therapies

Early data from Mesoblast’s Expanded Access Program is more encouraging. In patients 12+ years old who failed ruxolitinib or other second-line agents, Ryoncil recorded 76% survival at Day 100.

This disparity underscores the urgent need for better first-line strategies in adults.

What the New Trial Will Explore?

Mesoblast and FDA recently aligned on the appropriate patient population and timing for Ryoncil’s pivotal trial.
The trial will:

  • Randomize patients immediately after they develop steroid refractoriness
  • Compare ruxolitinib alone versus ruxolitinib + Ryoncil
  • Target adults with Grade III/IV SR-aGvHD
  • Aim to start enrollment in Q1 2026

The goal is clear: deliver treatment earlier, when patients have the best chance of survival.

Leadership Insights

Dr. John Levine, Chair of the BMT CTN Steering Committee, said:

“We are delighted to be partnering with Mesoblast. We aim to extend the use of this potentially life-saving treatment, already FDA-approved in children, to adults with severe SR-aGvHD.”

Silviu Itescu, CEO of Mesoblast, added:

“This remains a major unmet need. The adult market is 3–4 times larger than the pediatric market, and our partnership with BMT CTN is critical for expanding access.”

About the Organizations

Mesoblast

A global leader in allogeneic, off-the-shelf cellular medicines for life-threatening inflammatory diseases. The company focuses on developing therapies that address major unmet needs in conditions like aGvHD.

BMT CTN

A premier NIH-funded network responsible for high-quality, multi-institutional clinical trials in cellular therapies and bone marrow transplantation.
To date, BMT CTN has:

  • Completed 52 Phase II and III trials
  • Enrolled 16,600+ patients
  • Operated across 125+ transplant centers

Its collaborative structure includes the CIBMTR, NMDP, and Emmes Company LLC, ensuring scientific rigor and broad clinical reach.

Optimize Your trial insights with Clival Database.

Are you exhausted from the uncertainty of trial insights pricing? Clival Database ensures the clarity in the midst of the global scenario for clinical trials to you.

Clival Database is one of the best databases that offers an outstanding number of clinical trial data in terms of 50,000+ molecules and from primary regulatory markets as well as new entrants like Indian and Chinese markets.

With Clival, you get accurate positioning of historical sales data, patent database, company profiling, safety & efficacy, and prediction of launch of new innovative molecules helping you to align your research and driving down the cost.

To add value, we further break down our analytics for you so that improving your operational effectiveness; optimizing your clinical trials; and offering you accurate and high-quality data at lowest possible prices becomes possible.

Elevate your trial success rate with the cutting-edge insights from Clival database.

Check it out today and make more informed sourcing decisions! Learn More!