Annex 4C Quarterly Activity Report
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NEW YORK, Jan. 30, 2022 (GLOBE NEWSWIRE) — Mesoblast Limited (Nasdaq: MESO; ASX: MSB), the world leader in allogeneic cell-based medicines for inflammatory diseases, today released an operational and financial activity report for the second quarter ended December 31, 2021.
Main operational highlights of the quarter
Activities supporting the potential resubmission of the Biologics License Application (BLA) for remestemcel-L for the treatment of children with steroid-refractory acute graft-vs-host disease (SR-aGVHD):
- Meeting held with the United States Food and Drug Administration (FDA) Office of Tissues and Advanced Therapies (OTAT) to address potency and Chemistry, Manufacturing and Controls (CMC) testing items identified in the letter of complete response (CRL) for remestemcel-L for the treatment of SR-aGVHD in children
- OTAT indicated that the in vitro the immunomodulatory activity Mesoblast intends to measure for product potency is reasonable and the relevance of this activity to clinical outcomes needs to be established
- Results of an investigator-initiated study, published in the peer-reviewed journal A bone marrow transplant1, in children with SR-aGVHD stratified by baseline levels of inflammatory biomarkers, showed that treatment with remestemcel-L was associated with 64% survival in children with biomarker levels most predictive of mortality. high, against only 10% survival (p = 0.01) in matched children. controls treated with other available therapies, including ruxolitinib or other biologics
- These data provide strong evidence that the proposed anti-inflammatory and immunomodulatory mechanism of action of remestemcel-L is responsible for improving survival in patients with SR-aGVHD.
- Mesoblast has now generated substantial new data which it claims establishes the relevance of the proposed potency test measuring remestemcel-L in vitro anti-inflammatory and immunomodulatory activity of live clinical effect of the product in the phase 3 trial in children with SR-aGVHD, including survival and biomarkers of live activity
- Mesoblast will provide this new data to OTAT and process all other outstanding items as required for the new BLA submission
- Mesoblast continues to be in a well-established process with the FDA’s Center for Biologics Evaluation and Research (CBER), and if the new submission is accepted, CBER will review the adequacy of the clinical data in the context of the related CMC issues mentioned. above.
Activities related to the phase 3 programs of rexlemestrocel-L in chronic low back pain (CLBP) and chronic heart failure (CHF):
- Received comments from FDA OTAT indicating agreement with Mesoblast’s proposal for pain reduction at 12 months as the primary endpoint of a pivotal trial to confirm pain reduction pain observed with rexlemestrocel-L in the first phase 3 trial in patients with CLBP due to degenerative disc disease (DDD) refractory to available therapies, including opioids
- Received FDA OTAT feedback confirming that reduction of major adverse cardiovascular events (MACE) in cardiovascular mortality or irreversible morbidity (non-fatal heart attack or stroke) is a clinically meaningful endpoint acceptable for determining the therapeutic benefit of rexlemestrocel-L for patients with chronic heart failure and low ejection fraction (HFrEF)
- Provided OTAT with the first results of new analyzes in pre-specified high-risk groups in the Phase 3 DREAM-HF trial of rexlemestrocel-L in HFrEF, which showed that the greatest benefit of treatment is in patients with diabetes and/or myocardial ischemia (72% of the total population treated), a target population at very high risk of mortality and irreversible morbidity due to micro- and macro-vascular disease despite obtaining treatment optimal standards2
- Preparation for formal FDA submission of detailed analyzes of outcomes in high-risk HFrEF patients with diabetes and/or myocardial ischemia to agree on a potential pathway for approval
Main financial highlights of the quarter
- Revenue was US$3.5 million, including US$2.3 million from TEMCELL® HS Inj.3 royalties on SR-aGVHD sales in Japan, representing a 7% increase in royalties TEMCELL in Comparative Quarter of Last Year
- Total operating activities resulted in a net cash usage of US$19.8 million during the quarter, a 38% reduction from the comparative quarter last year. Nearly half of this expenditure was for regulatory and manufacturing activities for remestemcel-L
- Mesoblast has completed the refinancing of its senior secured credit facility with a new five-year US$90 million facility provided by funds managed by Oaktree Capital Management, LP
- Cash at the end of the quarter was US$94.8 million, with up to an additional US$40 million available to be drawn on existing funding facilities subject to certain milestones.
A copy of Schedule 4C – Quarterly Cash Flow Report for the second quarter of fiscal year 2022 is available on the Investors page of the Company’s website www.mesoblast.com.
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 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, 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 stromal cell technology platforms. Remestemcel-L is being developed for inflammatory diseases in children and adults, including steroid-refractory acute graft-versus-host disease and moderate to severe acute respiratory distress syndrome. Rexlemestrocel-L is being developed for advanced chronic heart failure and chronic low back pain. Two products have been commercialized 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
- Kasikis S., et al. Mesenchymal stromal cell therapy induces high responses and survival in children with steroid-refractory, low-risk GVHD. A bone marrow transplant 2021; https://doi.org/10.1038/s41409-021-01442-3
- Dunlay SM, et al. Traffic. 2019;140:e294–e324
- TEMCELL® HS Inj. is a registered trademark of JCR Pharmaceuticals Co. Ltd.
Forward-looking statementsThis 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. , the 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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Source: Mesoblast Limited