Mesoblast Achieves Target of 300 Treated Patients in Pivotal Phase 3 Trial for Chronic Low Back Pain
Rhea-AI Summary
Mesoblast (Nasdaq:MESO; ASX:MSB) announced it has reached its target of at least 300 treated patients in the MSB-DR004 pivotal Phase 3 randomized, placebo-controlled trial of rexlemestrocel-L for chronic low back pain (CLBP) associated with degenerative disc disease.
The trial aims to confirm durable pain reduction from a single intra-discal injection seen in the earlier MSB-DR003 study. The primary endpoint is reduction in low back pain at 12 months versus sham control, with secondary endpoints including function, quality of life, and cessation of pain medication, including opioids. Rexlemestrocel-L has RMAT designation from the FDA, enabling eligibility for priority review once a BLA is filed. Mesoblast is progressing commercial manufacturing in parallel, with top-line Phase 3 results expected in mid‑CY2027. The CLBP indication is described by Mesoblast as a potential blockbuster with possible peak year revenue >US$10 billion at single-digit market penetration.
Positive
- 300 patients treated in pivotal Phase 3 CLBP trial, meeting target
- Primary endpoint powered for 12‑month pain reduction vs sham controls
- RMAT designation from FDA, enabling eligibility for priority review of BLA
- Commercial manufacturing proceeding in parallel with Phase 3 trial
- Mesoblast cites potential peak year revenue of >US$10 billion at single-digit penetration
Negative
- None.
News Market Reaction – MESO
In the Jul 14 session, MESO gained 3.21%, reflecting a moderate positive market reaction. Argus tracked a peak move of +2.0% during that session. Our momentum scanner triggered 7 alerts that day, indicating moderate trading interest and price volatility.
Data tracked by StockTitan Argus on the day of publication.
Key Figures
Previous Clinical trial Reports
| Date | Event | Sentiment | 24h Move | Catalyst |
|---|---|---|---|---|
| Apr 28 | Phase 3 CLBP enrollment | Positive | +2.2% | Pivotal Phase 3 CLBP trial reached recruitment target of at least 300 patients. |
| Apr 10 | DMD IND clearance | Positive | -2.0% | FDA IND clearance to proceed directly to registrational Ryoncil trial in DMD. |
| Apr 07 | DMD IND clearance | Positive | +1.7% | Announcement of FDA IND clearance for registrational DMD trial using Ryoncil. |
| Mar 11 | Leadership appointment | Neutral | -3.6% | Appointment of Dr. Teresa Montagut as head of Clinical Development and Medical Affairs. |
| Jul 21 | Phase 3 CLBP enrollment start | Positive | +4.4% | Commencement of enrollment in pivotal Phase 3 trial of rexlemestrocel-L for CLBP. |
24h Move is the share-price change in the day after each event; other market factors may also have contributed.
Clinical trial announcements have typically produced small positive price moves, with one notable negative reaction among recent events.
Key Terms
pivotal phase 3 clinical
randomized controlled trial clinical
intra-discal injection medical
regenerative medicine advanced therapy regulatory
biologics license application regulatory
AI-generated analysis. How Rhea-AI works. Not financial advice.
NEW YORK, July 13, 2026 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today announced that it has achieved its target of at least 300 patients treated in the MSB-DR004 pivotal Phase 3 randomized controlled trial of rexlemestrocel-L for chronic low back pain (CLBP) associated with degenerative disc disease. The trial aims to confirm the durable pain reduction from a single intra-discal injection of rexlemestrocel-L seen in the earlier MSB-DR003 trial.
Silviu Itescu, Chief Executive of Mesoblast, said: “Completing our target of treating at least 300 patients in the placebo-controlled pivotal back pain trial ensures the trial is well powered for success. Commercial manufacturing is proceeding in parallel so that we can file for approval as soon as possible after trial results readout.”
CLBP caused by inflammation and degenerative disc disease is a serious condition with a prevalence of over 7 million people in the U.S. alone. The indication is a potential blockbuster for Mesoblast with potential peak year revenue of >US
The trial's primary endpoint is powered to show a significant difference in reduction of low back pain at 12 months between rexlemestrocel-L and sham controls. Secondary endpoints include improvements in function, quality of life, and cessation of pain medication, including opioids.
Rexlemestrocel-L has Regenerative Medicine Advanced Therapy (RMAT) designation from the United States Food and Drug Administration (FDA) for treatment of CLBP due to degenerative disc disease providing eligibility for priority review once the Biologics License Application (BLA) has been filed. Top-line results are expected in mid-CY2027 after the last treated patient has completed 12 months follow-up.
About Rexlemestrocel-L for Chronic Low Back Pain associated with Degenerative Disc Disease
The 300-patient randomized controlled confirmatory Phase 3 trial of Mesoblast’s second generation allogeneic, STRO3-immunoselected, and industrially manufactured stromal cell product candidate rexlemestrocel-L in combination with hyaluronic acid (HA) as delivery agent for injection into the lumbar disc is actively enrolling in patients with chronic low back pain (CLBP) due to inflammatory degenerative disc disease (DDD) of less than five years duration at multiple sites across the U.S.
FDA has previously agreed on the design of this 300-patient randomized, placebo-controlled confirmatory Phase 3 trial, and the 12-month primary endpoint of pain reduction as an approvable indication. This endpoint was successfully met in Mesoblast’s first Phase 3 trial. Key secondary measures include improvement in quality of life and function.
A particular focus is on treatment of patients on opioids, since discogenic back pain accounts for approximately
FDA has designated rexlemestrocel-L a Regenerative Medicine Advanced Therapy (RMAT) for the treatment of chronic low back pain. RMAT designation provides all the benefits of Breakthrough and Fast Track designations, including rolling review and eligibility for priority review on filing of a Biologics License Application (BLA).
About Chronic Low Back Pain
Back pain is the leading cause of disability in Americans under 45 years,1 with an annual prevalence in the general U.S. adult population of 10
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
- American Academy of Pain Medicine - Get the Facts on Pain. The American Academy of Pain Medicine. http://www.painmed.org/patientcenter/facts-on-pain/ Accessed on June 28, 2017.
- Urits I, Burshtein A, Sharma M, et al. Low Back Pain, a Comprehensive Review: Pathophysiology, Diagnosis, and Treatment. Current Pain and Headache Reports. 2019;23(3):1-10. doi:10.1007/s11916-019-0757-1.
- Navigant: Commercial Assessment for a Proprietary Cell-Based Therapy for DDD in the U.S. and the EU3 – August 2014.
- Decision Resources: Chronic Pain December 2015.
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 | Media – Australia |
| Rubenstein | BlueDot Media |
| Caroline Nelson | Steve Dabkowski |
| T: +1 703 489 3037 | T: +61 419 880 486 |
| E: cnelson@rubenstein.com | E: steve@bluedot.net.au |
