Mesoblast provides update following meeting with FDA OTAT



Key points:

  • Meeting with the United States Food and Drug Administration (FDA) Office of Tissues and Advanced Therapies (OTAT) to discuss potency testing and chemical, manufacturing and control (CMC) elements identified in the letter complete response (CRL) for remestemcel-L in the treatment of acute steroid-refractory graft-versus-host disease (SR-aGVHD) in children
  • OTAT has indicated that Mesoblast’s approach to addressing outstanding CMC items is reasonable
  • OTAT indicated that the in vitro immunomodulatory activity Mesoblast intends to measure potency is a reasonable critical quality attribute (CQA) for the product, and the relevance of this activity to clinical outcomes needs to be established.
  • Mesoblast has now generated substantial new data which he believes establishes the relevance of the project in vitro immunomodulatory activity of remestemcel-L at in vivo clinical effect of the product in the phase 3 trial in children with SR-aGVHD, including survival and biomarkers of in vivo activity
  • Mesoblast will provide this new data to OTAT and process other outstanding items as required for the resubmission of the Biologics License Application (BLA).

NEW YORK, December 30, 2021 (GLOBE NEWSWIRE) – Mesoblast Limited (Nasdaq: MESO; ASX: MSB), a global leader in allogeneic cellular drugs for inflammatory diseases, today provided a regulatory update on remestemcel-L for the acute treatment of steroid-refractory graft-versus-host disease in children following its recent meeting with the FDA OTAT. Mesoblast requested the meeting to consider the suitability of an activity assay related to the immunomodulatory mechanism of action proposed by remestemcel-L as well as the approach of the outstanding CMC elements identified in CSF.

OTAT has indicated that Mesoblast’s approach to dealing with the outstanding CMC items is reasonable, that the in vitro The immunomodulatory activity of remestemcel-L proposed by Mesoblast as a measure of its potency is a reasonable CQA for the product in the treatment of children with SR-aGVHD, and the relevance of this immunomodulatory activity to clinical results has to be established.

Mesoblast has now generated substantial new data which it believes establishes the relevance of the proposal in vitro the immunomodulatory activity of remestemcel-L to the clinical effect of the product in the phase 3 trial completed in pediatric SR-aGVHD, including survival results and product biomarkers in vivo activity. Mesoblast will provide this new data to OTAT and process the other remaining CRL elements as required for the BLA resubmission.

By demonstrating the relevance of the in vitro activity test to clinical outcomes, Mesoblast believes it will be able to show that the remestemcel-L product used in the phase 3 trial in pediatric SR-aGVHD has been standardized as to identity, strength, quality, purity, and dosage form, and that it will meet OTAT’s recommendation for further adequate and well-controlled study.

Mesoblast continues to be in a well-established process with the FDA’s Center for Biologics Evaluation and Research (CBER), and if the re-submission is accepted, CBER will review the adequacy of the clinical data in the context of CMC issues. mentioned above.

About acute steroid refractory graft versus host disease (SR-aGVHD)
GVHD is a severe inflammation of the bloodstream caused by complications from bone marrow transplants. The disease occurs in up to 50% of the 30,000 patients who receive an allogeneic bone marrow transplant each year, mainly during treatment for blood cancers. In patients with the most severe form of GVHD, mortality can reach 90%. There are no approved therapies for the treatment of SR-aGVHD in children under 12 years of age.

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 conditions. The Company has leveraged its proprietary mesenchymal lineage cell therapy technology platform to establish a broad portfolio 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, resulting in 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 cryopreserved and ready-to-use cellular drugs on an industrial scale. These cell therapies, with defined pharmaceutical release criteria, should be readily available to patients around the world.

Mesoblast is developing product candidates for distinct indications based on its remestemcel-L and rexlemestrocel-L stromal cell technology platforms. Remestemcel-L is being developed for inflammatory diseases in children and adults, including acute steroid refractory graft versus host disease and moderate to severe acute respiratory distress syndrome. Rexlemestrocel-L is in development for advanced chronic heart failure and chronic low back pain. Two products have been marketed in Japan and Europe by the licensees of Mesoblast, 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 the Nasdaq (MESO). For more information, please visit www.mesoblast.com, LinkedIn: Mesoblast Limited and Twitter: @Mesoblast

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 results, levels of activity, actual performance or achievements differ materially from any future results. , activity levels, performances or achievements expressed or implied by these forward-looking statements. We make such forward-looking statements in accordance with the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. Forward-looking statements should not be interpreted as a guarantee 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, without limitation, statements regarding: 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, enroll and complete clinical studies, including multinational clinical trials; Mesoblast’s ability to advance its manufacturing capabilities; timing or likelihood of regulatory filings and approvals (including re-submission of BLA), manufacturing activities and product marketing activities, if applicable; marketing of Mesoblast’s product candidates, if approved; regulatory or public perceptions and market acceptance regarding the use of stem cell therapy; the possibility that Mesoblast’s product candidates, if any, will 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 them in cases of suspected infringement; the extent of protection Mesoblast is able to establish and maintain for intellectual property rights covering its product candidates and technology; Mesoblast’s expense estimates, future revenues, capital requirements, and its additional funding requirements; the financial performance of Mesoblast; developments relating to Mesoblast’s competitors and industry; and pricing and reimbursement of Mesoblast product candidates, if approved. You should read this press release, along with our risk factors, in our latest reports filed with the SEC or on our website. The uncertainties and risks which may cause Mesoblast’s actual results, performance or achievements to differ materially from those which may be expressed or implied by such statements, and therefore, you should not place undue reliance on such statements prospective. We assume 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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