Mesoblast submits new information to the FDA’s IND dossier in
NEW YORK, Oct. 02, 2022 (GLOBE NEWSWIRE) — Mesoblast Limited (Nasdaq: MESO; ASX: MSB), the world leader in allogeneic cell-based drugs for inflammatory diseases, today announced that it has submitted to the Food and Drug Administration United States (FDA) substantial new information on the clinical test and potency elements identified in the Complete Response Letter (CRL) received from the FDA in September 2020 to the Biologics License Application (BLA) for remestemcel -L in the treatment of children with acute steroid-refractory graft versus host disease (SR-aGVHD).
Mesoblast has maintained an active dialogue with the FDA since receiving the CRL, and the substantial new information submitted to the Investigational New Drug (IND) dossier for remestemcel-L in the treatment of children with SR-aGVHD, such as guided by the FDA, represent a major step in the Company’s comprehensive response to the FDA. Remestemcel-L has been granted Fast Track designation and BLA priority review by the FDA.
Survival outcomes have not improved over the past two decades for children or adults with the most severe forms of SR-aGVHD.1-3 The lack of approved treatments for children under 12 means there is an urgent need for a therapy that improves dismal survival outcomes in children.
“The submission summarizes controlled data providing further evidence of remestemcel-L’s ability to save lives,” said Dr. Silviu Itescu, Chief Executive Officer of Mesoblast. “Furthermore, the enhanced process controls we have in place to ensure a robust and consistent commercial product, along with a potency test that predicts consistent survival outcomes, make remestemcel-L a compelling treatment for these children. .”
About Steroid-Refractory Acute Graft-versus-Host Disease
Acute GVHD occurs in approximately 50% of patients who receive allogeneic bone marrow transplantation (BMT). More than 30,000 patients worldwide undergo allogeneic GMO each year, mainly during the treatment of blood cancers, of which approximately 20% are pediatric patients.4.5 SR-aGVHD is associated with up to 90% mortality and significant prolonged hospitalization costs.6.7 There are currently no FDA-approved treatments in the United States for children younger than 12 with SR-aGVHD.
About the mesoblast
Mesoblast is a world leader in the development of allogeneic (ready-to-use) cellular drugs for the treatment of serious and life-threatening inflammatory diseases. The Company has leveraged its proprietary mesenchymal lineage cell therapy technology platform to establish a broad pipeline of late-stage product candidates that respond to severe inflammation by releasing anti-inflammatory factors that counteract and modulate multiple effector arms of the immune system, leading to a significant reduction in the damaging inflammatory process.
Mesoblast has a strong and extensive global intellectual property portfolio with protection extending to at least 2041 in all major markets. The company’s proprietary manufacturing processes produce industrial-scale, cryopreserved, ready-to-use cellular drugs. These cell therapies, with defined pharmaceutical release criteria, should be readily available to patients worldwide.
Mesoblast is developing product candidates for distinct indications based on its remestemcel-L and rexlemestrocel-L allogeneic stromal cell technology platforms. Remestemcel-L is being developed for inflammatory diseases in children and adults, including steroid-refractory acute graft-versus-host disease, bioresistant inflammatory bowel disease, and acute respiratory distress syndrome. Rexlemestrocel-L is being developed for advanced chronic heart failure and chronic low back pain. Two products have been marketed in Japan and Europe by Mesoblast licensees, and the Company has established commercial partnerships in Europe and China for certain Phase 3 assets.
Mesoblast has offices in Australia, the United States and Singapore and is listed on the Australian Securities Exchange (MSB) and Nasdaq (MESO). For more information, please visit www.mesoblast.com, LinkedIn: Mesoblast Limited and Twitter: @Mesoblast
References / Footnotes
- Berger M, Pessolano R, Carraro F, Saglio F, Vassallo E, Fagioli F. Acute steroid-refractory graft-versus-host disease grade III-IV in pediatric patients. A mono-institutional experience with long-term follow-up. Pediatric transplant. 2020; 24(7):e13806
- Rashidi A, DeFor T, Holtan S, Blazar B, Weisdorf D, MacMillan M. Outcomes and predictors of response in acute steroid-refractory graft-versus-host disease. Biol Blood Marrow Transplant. 25 (2019) 2297-2302
- Biavasco F, Ihorst G, Wasch R, Wehr C, Bertz H, Finke J, Zeiser R. Therapeutic response of refractory acute GVHD to lower intestinal tract glucocorticoids. A bone marrow transplant. 2022
- Niederwieser D, Baldomero H, Szer J. (2016) Hematopoietic stem cell transplantation activity worldwide in 2012 and a SWOT analysis of the global network for blood and marrow transplantation group including the global survey.
- HRSA Transplant Activity Report, CIBMTR, 2019
- Westin, J., Saliba, RM., Lima, M. (2011) Acute steroid-refractory GVHD: predictors and outcomes. Advances in hematology.
- Axt L, Naumann A, Toennies J (2019) Single-center retrospective analysis of outcomes, risk factors, and therapy in steroid-refractory graft-versus-host disease after allogeneic hematopoietic cell transplantation. A bone marrow transplant.
This press release contains forward-looking statements relating to future events or our future financial performance and involve known and unknown risks, uncertainties and other factors that could cause our results, levels of activity, performance or actual achievements differ materially from any future results. , levels of activity, performance or achievements expressed or implied by such forward-looking statements. We make these 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 construed as guarantees of future performance or results, and actual results may differ from the results anticipated in such forward-looking statements, and the differences may be material and adverse. Forward-looking statements include, but are not limited to, statements regarding: the initiation, timing, progress and results of preclinical and clinical studies of Mesoblast, as well as Mesoblast’s research and development programs; Mesoblast’s ability to advance product candidates, enroll in and complete clinical studies, including multinational clinical trials; Mesoblast’s ability to advance its manufacturing capabilities; the timing or likelihood of regulatory filings and approvals (including resubmission BLA), manufacturing activities and product marketing activities, if any; the commercialization of Mesoblast’s product candidates, if approved; regulatory or public perceptions and market acceptance surrounding the use of stem cell therapies; the possibility that Mesoblast’s product candidates, if approved, may be withdrawn from the market due to adverse events or patient 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 in its product candidates and Mesoblast’s ability to successfully defend against alleged infringement; the scope of protection that Mesoblast is able to establish and maintain for the intellectual property rights covering its product candidates and technology; estimates of Mesoblast’s expenses, future revenues, capital requirements and additional financing requirements; the financial performance of Mesoblast; developments regarding Mesoblast’s competitors and industry; and pricing and reimbursement of Mesoblast’s product candidates, if approved. You should read this press release, together with our risk factors, in our latest reports filed 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 that may be expressed or implied by such statements, and accordingly, you should not place undue reliance on such forward-looking statements . We undertake no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future developments or otherwise.
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