Real-World Commercial Experience with Ryoncil® Shows 84% Survival of Children with SR-aGvHD After Completing 28-Days of Treatment

Mesoblast (Nasdaq:MESO) reported real-world pediatric outcomes for Ryoncil (remestemcel-L-rknd) since commercial launch in March 2025.

Rhea-AI Summary

Mesoblast (Nasdaq:MESO) reported real-world pediatric outcomes for Ryoncil (remestemcel-L-rknd) since commercial launch in March 2025. Of the first 25 children treated post-launch, 21 (84%) completed the FDA-label 28-day regimen and were alive; four patients who did not complete treatment died within 28 days. Mesoblast has onboarded 45 transplant centers toward a 64-center target covering 94% of U.S. transplants, and reports payer coverage for over 260 million U.S. lives. A CMS HCPCS J-Code issued Oct 1, 2025 increased CMS usage in the last quarter. A pivotal adult trial with BMT-CTN is expected to begin site enrollment this quarter to seek FDA label expansion.

Positive

• 84% survival (21 of 25) completed 28-day pediatric regimen
• 45 transplant centers onboarded toward 64-center target
• Payer coverage for 260 million U.S. lives including CMS Medicaid
• CMS issued specific HCPCS J-Code (Oct 1, 2025) improving reimbursement

Negative

• Small early cohort: real-world results based on only 25 patients
• Four treated children died within 28 days and did not complete therapy
• Recent uptake skewed toward CMS coverage versus commercial payers

Argus Jan 27 session

+2.62% close to close 0.8x rel. volume Open Argus

Details

News Market Reaction – MESO

On Jan 27, the first trading day after this news, MESO closed 2.62% above the previous close.

Data tracked by StockTitan Argus for the Jan 27 session.

Key Figures

Patients treated: 25 children 28-day survival: 84% Completed regimen: 21 patients +5 more

Patients treated

25 children

First real-world Ryoncil-treated SR-aGvHD patients post-March 2025 launch

28-day survival

84%

21 of 25 children alive after completing 28-day Ryoncil regimen

Completed regimen

21 patients

Children who completed initial 28-day Ryoncil treatment and were alive

Deaths within 28 days

4 patients

Did not complete 28-day course; died of severe SR-aGvHD

Initial treatment course

28 days

Standard Ryoncil regimen per FDA-approved label

Transplant centers onboarded

45 centers

Ryoncil commercial onboarding in U.S. transplant centers

Target centers coverage

64 centers / 94%

Target transplant centers representing 94% of U.S. transplants

Lives covered

260 million

U.S. lives covered by government and commercial payers for Ryoncil

Historical Context

5 past events · Latest: Jan 18

5 events

1. Jan 18

   FDA feedback CLBP

   24h Move

   -3.5%

   FDA Type B feedback that Phase 3 pain data support rexlemestrocel-L efficacy.

2. Jan 08

   Sales and financing

   24h Move

   +3.2%

   Ryoncil quarterly sales rose to US$35.1M with a 60% sequential increase.

3. Jan 01

   Board changes

   24h Move

   +0.7%

   Board leadership reshaped to support shift toward revenue-generating commercialization.

4. Dec 29

   Credit facility

   24h Move

   -2.0%

   New five-year, interest-only facility used to retire senior debt and refinance.

5. Dec 11

   ASH meta-analysis

   24h Move

   -1.8%

   Meta-analysis showed remestemcel-L superior to ruxolitinib in SR-aGvHD outcomes.

24h Move is the share-price change in the day after each event; other market factors may also have contributed.

Key Terms

steroid-refractory acute graft-versus-host disease, sr-agvhd, mesenchymal stromal cell, u.s. food & drug administration, +3 more

7 terms

steroid-refractory acute graft-versus-host disease medical

"with steroid-refractory acute graft-versus-host disease (SR-aGvHD)."

A severe condition that can follow a stem cell or bone marrow transplant in which the donor immune cells attack the patient’s organs and tissues; “steroid-refractory” means it does not improve after standard steroid treatment. Investors pay attention because it represents an urgent unmet medical need that drives demand for new drugs, influences clinical trial success and regulatory approvals, and can materially affect the commercial prospects and valuation of companies developing effective therapies — like a fire that the usual extinguisher cannot put out.

sr-agvhd medical

"in children 2 months and older with steroid-refractory acute graft-versus-host disease (SR-aGvHD)."

Steroid-refractory acute graft-versus-host disease (sr‑aGVHD) is a severe immune reaction that can occur after a patient receives a donor stem cell or bone marrow transplant; it means the inflammation did not improve after standard steroid therapy. For investors, it signals a high medical need and potential market opportunity for new therapies because affected patients face worse outcomes, longer hospital stays and higher costs, similar to a fire that keeps flaring up despite the usual extinguisher.

mesenchymal stromal cell medical

"Ryoncil® is the first mesenchymal stromal cell (MSC) product approved by the U.S."

Mesenchymal stromal cells are a type of adult cell found in bone marrow, fat and other tissues that can act like a repair crew, supporting tissue healing, reducing inflammation and helping other cells grow. Investors care because these cells are the basis for many experimental therapies and diagnostics; clinical success, manufacturing scale-up, safety and regulatory approval determine commercial potential and can strongly affect a company’s valuation.

u.s. food & drug administration regulatory

"product approved by the U.S. Food & Drug Administration (FDA) for any indication."

The U.S. Food & Drug Administration (FDA) is the federal agency that reviews and approves medicines, medical devices, vaccines, food safety measures and related health products for use in the United States. Investors watch FDA decisions because approvals, rejections or safety warnings can quickly change a company’s sales prospects and stock value—similar to a referee’s call that can determine whether a product can legally enter the market and compete.

hcpcs regulatory

"Issuance on October 1, 2025, of a specific Healthcare Common Procedure Coding System (HCPCS) J-Code"

HCPCS (Healthcare Common Procedure Coding System) is a standardized set of alphanumeric codes used by providers and insurers to describe medical services, supplies and durable equipment for billing and reimbursement. Think of it as the industry’s SKU list that determines how treatments and devices are recorded and paid for; changes to HCPCS codes or payment rules can directly affect revenue, pricing and market access for healthcare companies.

j-code regulatory

"Healthcare Common Procedure Coding System (HCPCS) J-Code by CMS for billing and reimbursement"

A J-code is a standardized billing code used by U.S. healthcare payers to identify specific injectable or infused drugs and biologic therapies given in clinics or hospitals. For investors, J-codes matter because they determine how treatments are billed and reimbursed by insurers and government programs; having a clear code can make it easier for a medicine to be paid for and for a company to forecast sales, much like a product SKU that allows a store to track and sell an item.

pivotal trial medical

"A pivotal trial of Ryoncil® in adults with severe SR-aGvHD will be conducted"

A pivotal trial is a key test of a new medicine or treatment to see if it works and is safe enough to be approved by health authorities. It's like a final exam for a new product, and passing it is essential for bringing the treatment to the public.

AI-generated analysis. How Rhea-AI works. Not financial advice.

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NEW YORK, Jan. 26, 2026 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today provided an update on use of Ryoncil^® (remestemcel-L-rknd) since commercially available in March 2025 for the approved label in children 2 months and older with steroid-refractory acute graft-versus-host disease (SR-aGvHD). Ryoncil^® is the first mesenchymal stromal cell (MSC) product approved by the U.S. Food & Drug Administration (FDA) for any indication.¹

Of the first 25 patients treated with Ryoncil^® in a ‘real-world’ clinical setting post launch, 21 were alive (84%) and completed the initial 28-day treatment regimen as per the FDA approval label. The four patients who did not complete the 28-day treatment course had been offered and failed other therapies prior to use of Ryoncil^® and died of severe SR-aGvHD within 28 days. These early data are consistent with the prior clinical experience with Ryoncil^®. The outcomes highlight our focus on getting patients on Ryoncil^® therapy as early as possible following steroid resistance to enable completion of an initial 28-day treatment course and maximize survival.

To ensure that no patient is left behind in receiving this potentially life-saving therapy, Mesoblast has established a patient access hub termed MyMesoblast™, where Ryoncil^® is available for ordering. Additional information is available on ryoncil.com, where valuable resources for healthcare providers, patients and caregivers can be found.

To date 45 transplant centers have been onboarded, with a target of 64 centers which account for 94% of transplants performed in the U.S. Ryoncil^® coverage by government and commercial payers already extends to over 260 million U.S. lives with Federal Medicaid coverage by U.S. Centers for Medicare & Medicaid Services (CMS) and mandatory fee-for-service Medicaid coverage in all U.S. states. Issuance on October 1, 2025, of a specific Healthcare Common Procedure Coding System (HCPCS) J-Code by CMS for billing and reimbursement resulted in greater usage of Ryoncil^® under CMS coverage versus commercial coverage in the last quarter.²

These commercial activities will continue to serve the company well as it seeks to expand the FDA label for Ryoncil^® to adults with severe SR-aGvHD, a market size approximately three times that of the pediatric SR-aGvHD population. A pivotal trial of Ryoncil^® in adults with severe SR-aGvHD will be conducted with the NIH-funded Bone Marrow Transplant Clinical Trials Network (BMT-CTN) and is expected to commence site enrollment this quarter.

“We are delighted to see the excellent early survival rates in the real-world experience with Ryoncil^® in children with this devastating disease,” said Mesoblast Chief Executive Dr. Silviu Itescu. “Our strong early results and the streamlined process that is in place to provide access to the product underscores the importance of early physician referral and treatment initiation in order to give Ryoncil^® the best chance to save as many precious lives as possible."

About Mesoblast
Mesoblast (the Company) is a world leader in developing allogeneic (off-the-shelf) cellular medicines for the treatment of severe and life-threatening inflammatory conditions. The therapies from the Company’s proprietary mesenchymal lineage cell therapy technology platform respond to severe inflammation by releasing anti-inflammatory factors that counter and modulate multiple effector arms of the immune system, resulting in significant reduction of the damaging inflammatory process.

Mesoblast’s Ryoncil^® (remestemcel-L-rknd) for the treatment of steroid-refractory acute graft versus host disease (SR-aGvHD) in pediatric patients 2 months and older is the first FDA-approved mesenchymal stromal cell (MSC) therapy. Please see the full Prescribing Information at www.ryoncil.com.

Mesoblast is committed to developing additional cell therapies for distinct indications based on its remestemcel-L and rexlemestrocel-L allogeneic stromal cell technology platforms. Ryoncil^® is being developed for additional inflammatory diseases including SR-aGvHD in adults and biologic-resistant inflammatory bowel disease. Rexlemestrocel-L is being developed for heart failure and chronic low back pain. The Company has established commercial partnerships in Japan, Europe and China.

About Mesoblast intellectual property: Mesoblast has a strong and extensive global intellectual property portfolio, with over 1,000 granted patents or patent applications covering mesenchymal stromal cell compositions of matter, methods of manufacturing and indications. These granted patents and patent applications provide commercial protection extending through to at least 2044 in all major markets.

About Mesoblast manufacturing: The Company’s proprietary manufacturing processes yield industrial-scale, cryopreserved, off-the-shelf, cellular medicines. These cell therapies, with defined pharmaceutical release criteria, are planned to be readily available to patients worldwide.

Mesoblast has locations in Australia, the United States and Singapore and is listed on the Australian Securities Exchange (MSB) and on the Nasdaq (MESO). For more information, please see www.mesoblast.com, LinkedIn: Mesoblast Limited and Twitter: @Mesoblast

References / Footnotes

1. Please see the full Prescribing Information at www.ryoncil.com
2. Coding and coverage decisions are made by payers, and coverage cannot be guaranteed

Forward-Looking Statements
This press release includes forward-looking statements that relate to future events or our future financial performance and involve known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, performance or achievements to differ materially from any future results, levels of activity, performance or achievements expressed or implied by these forward-looking statements. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. Forward-looking statements should not be read as a guarantee of future performance or results, and actual results may differ from the results anticipated in these forward-looking statements, and the differences may be material and adverse. Forward-looking statements include, but are not limited to, statements about: the initiation, timing, progress and results of Mesoblast’s preclinical and clinical studies, and Mesoblast’s research and development programs; Mesoblast’s ability to advance product candidates into, enroll and successfully complete, clinical studies, including multi-national clinical trials; Mesoblast’s ability to advance its manufacturing capabilities; the timing or likelihood of regulatory filings and approvals, manufacturing activities and product marketing activities, if any; the commercialization of Mesoblast’s RYONCIL for pediatric SR-aGvHD and any other product candidates, if approved; regulatory or public perceptions and market acceptance surrounding the use of stem-cell based therapies; the potential for Mesoblast’s product candidates, if any are approved, to be withdrawn from the market due to patient adverse events or deaths; the potential benefits of strategic collaboration agreements and Mesoblast’s ability to enter into and maintain established strategic collaborations; Mesoblast’s ability to establish and maintain intellectual property on its product candidates and Mesoblast’s ability to successfully defend these in cases of alleged infringement; the scope of protection Mesoblast is able to establish and maintain for intellectual property rights covering its product candidates and technology; estimates of Mesoblast’s expenses, future revenues, capital requirements and its needs for additional financing; Mesoblast’s financial performance; developments relating to Mesoblast’s competitors and industry; and the pricing and reimbursement of Mesoblast’s product candidates, if approved. You should read this press release together with our risk factors, in our most recently filed reports with the SEC or on our website. Uncertainties and risks that may cause Mesoblast’s actual results, performance or achievements to be materially different from those which may be expressed or implied by such statements, and accordingly, you should not place undue reliance on these forward-looking statements. We do not undertake any obligations to publicly update or revise any forward-looking statements, whether as a result of new information, future developments or otherwise.

Release authorized by the Chief Executive.

For more information, please contact:

Corporate Communications / Investors
Paul Hughes
T: +61 3 9639 6036

Media – Global
Allison Worldwide
Emma Neal
T: +1 603 545 4843
E: emma.neal@allisonworldwide.com

Media – Australia
BlueDot Media
Steve Dabkowski
T: +61 419 880 486
E: steve@bluedot.net.au

FAQ

AI-generated questions and answers. How Rhea-AI works. Not financial advice.

What were the real-world pediatric survival results for Ryoncil (MESO) reported Jan 26, 2026?

Of the first 25 children treated post-launch, 21 (84%) completed the 28-day regimen and were alive.

When did Ryoncil become commercially available and for which patients is it approved?

Ryoncil launched in March 2025 and is FDA-approved for children aged 2 months and older with steroid-refractory acute GvHD.

How broad is payer coverage for Ryoncil (MESO) in the U.S. as of Jan 2026?

Coverage extends to over 260 million U.S. lives, including CMS Medicaid and mandatory fee-for-service Medicaid in all states.

What reimbursement change occurred Oct 1, 2025 related to Ryoncil (MESO)?

CMS issued a specific HCPCS J-Code on Oct 1, 2025, which increased Ryoncil usage under CMS coverage in the last quarter.

How many transplant centers are using Ryoncil and what is the target network size?

To date 45 transplant centers have been onboarded, with a target of 64 centers representing 94% of U.S. transplants.

Is Mesoblast planning an adult trial of Ryoncil (MESO) for severe SR-aGvHD?

Yes; a pivotal adult trial with the NIH-funded BMT-CTN is expected to begin site enrollment this quarter to pursue FDA label expansion.