Consistent Outcomes Using Ryoncil as First-Line Treatment or Salvage Therapy in 309 Children With Steroid-Refractory Acute GVHD – BioSpace

NEW YORK, Feb. 24, 2020 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB) today announced that aggregated results from 309 children treated with Ryoncil (remestemcel-L) were presented atthe American Society for Transplantation Cellular Therapy and the Center for International Blood & Bone Marrow Transplant Research (TCT) meeting in Orlando, Florida on February 22. The data showed that treatment with RYONCIL across three separate trials resulted inconsistent treatment responses and survival outcomesinchildren with steroid-refractory acute graft versus host disease (SR-aGVHD).

Key findings and conclusions were:

Mesoblast Chief Medical Officer Dr Fred Grossman said: These aggregated data from three studies demonstrate consistent efficacy and safety of RYONCIL in children suffering from steroid refractory acute graft versus host disease. If approved, RYONCIL has the potential to be an effective and safe therapy to improve survival outcomes in the most vulnerable population of children with severe forms of this disease who can have mortality rates as high as 90 percent.

In January, Mesoblast filed a Biologics License Application (BLA) to the United States Food and Drug Administration (FDA) for RYONCIL for the treatment of children with steroid-refractory aGVHD. The Company has requested Priority Review of the BLA by the FDA under the product candidates existing Fast Track designation. If approved, RYONCIL is expected to be launched in the US in 2020.

About Acute GVHDAcute GVHD occurs in approximately 50% of patients who receive an allogeneic bone marrow transplant (BMT). Over 30,000 patients worldwide undergo an allogeneic BMT annually, primarily during treatment for blood cancers, and these numbers are increasing.1 In patients with the most severe form of acute GVHD (Grade C/D or III/IV) mortality is as high as 90% despite optimal institutional standard of care.2,3. There are currently no FDA-approved treatments in the US for children under 12 with SR-aGVHD.

About Ryoncil Mesoblasts lead product candidate, RYONCIL, is an investigational therapy comprising culture- expanded mesenchymal stem cells derived from the bone marrow of an unrelated donor. It is administered to patients in a series of intravenous infusions. RYONCIL is believed to have immunomodulatory properties to counteract the inflammatory processes that are implicated in SR- aGVHD by down-regulating the production of pro-inflammatory cytokines, increasing production of anti-inflammatory cytokines, and enabling recruitment of naturally occurring anti-inflammatory cells to involved tissues.

References1. Niederwieser D, Baldomero H, Szer J. (2016) Hematopoietic stem cell transplantation activity worldwide in 2012 and a SWOT analysis of the Worldwide Network for Blood and Marrow Transplantation Group including the global survey.2. Westin, J., Saliba, RM., Lima, M. (2011) Steroid-refractory acute GVHD: predictors and outcomes. Advances in Hematology.3. Axt L, Naumann A, Toennies J (2019) Retrospective single center analysis of outcome, risk factors and therapy in steroid refractory graft-versus-host disease after allogeneic hematopoietic cell transplantation. Bone Marrow Transplantation.

About MesoblastMesoblast Limited (Nasdaq: MESO; ASX: MSB) is a world leader in developing allogeneic (off-the-shelf) cellular medicines. The Company has leveraged its proprietary mesenchymal lineage cell therapy technology platforms to establish a broad portfolio of commercial products and late-stage product candidates. Mesoblasts proprietary manufacturing process yields 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 filed a Biologics License Application to the United States Food and Drug Administration (FDA) to seek approval of its product candidate Ryoncil (remestemcel-L) for steroid-refractory acute graft versus host disease (acute GvHD). Remestemcel-L is also being developed for other rare diseases. Mesoblast is completing Phase 3 trials for its rexlemestrocel product candidates for advanced heart failure and chronic low back pain. If approved, RYONCIL is expected to be launched in the United States in 2020 for pediatric steroid-refractory acute GVHD. Two products have been commercialized in Japan and Europe by Mesoblasts licensees, and the Company has established commercial partnerships in Europe and China for certain Phase 3 assets.

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 http://www.mesoblast.com, LinkedIn: Mesoblast Limited and Twitter: @Mesoblast

Mesoblasts Forward-Looking StatementsThis announcement 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 timing, progress and results of Mesoblasts preclinical and clinical studies; Mesoblasts ability to advance product candidates into, enroll and successfully complete, clinical studies; the timing or likelihood of regulatory filings and approvals; and the pricing and reimbursement of Mesoblasts 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 Mesoblasts 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.

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Consistent Outcomes Using Ryoncil as First-Line Treatment or Salvage Therapy in 309 Children With Steroid-Refractory Acute GVHD - BioSpace