Appendix 4C Quarterly Activity Report
Mesoblast Limited (NASDAQ: MESO) has provided an operational and financial report for Q2 FY2022, highlighting steps towards the resubmission of its Biologics License Application (BLA) for remestemcel-L to treat steroid-refractory acute graft versus host disease (SR-aGVHD) in children. Key operational achievements include FDA meetings to clarify potency and manufacturing controls. Financially, the company reported $3.5 million in revenues, with significant cash reserves of $94.8 million. The Q2 cash usage was reduced by 38% compared to the previous year's quarter, indicating improved financial management.
- FDA engagement for remestemcel-L resubmission demonstrating regulatory progress.
- New data showing 64% survival rate in high-risk children with SR-aGVHD.
- Increased revenues of $3.5 million, including a 7% rise in TEMCELL royalties.
- Reduction in cash usage by 38% indicates improved cost management.
- Strong cash position of $94.8 million with additional financing options available.
- None.
Mesoblast Prepares for Resubmission of Biologics License Application
NEW YORK, Jan. 30, 2022 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today provided an operational and financial activity report for the second quarter ended December 31, 2021.
Key operational highlights for the quarter
Activities supporting potential resubmission of the Biologics License Application (BLA) for remestemcel-L in the treatment of children with steroid-refractory acute graft versus host disease (SR-aGVHD):
- Meeting held with the US Food and Drug Administration’s (FDA) Office of Tissues and Advanced Therapies (OTAT) to address potency assay and chemistry, manufacturing and controls (CMC) items identified in the complete response letter (CRL) for remestemcel-L in the treatment of SR-aGVHD in children
- OTAT indicated that the in vitro immunomodulatory activity Mesoblast intends to measure for potency of the product is reasonable and that the relevance of this activity to clinical outcomes should be established
- Results from an investigator-initiated study, published in the peer-reviewed journal Bone Marrow Transplantation1, in children with SR-aGVHD stratified by baseline levels of inflammatory biomarkers, showed that remestemcel-L treatment was associated with
64% survival in children with biomarker levels predictive for highest mortality compared with only10% survival (p=0.01) in matched controls treated with other available therapies, including ruxolitinib or other biologics - These data provide strong supporting evidence that the proposed anti-inflammatory and immunomodulatory mechanism of action of remestemcel-L is responsible for the improved survival in patients with SR-aGVHD
- Mesoblast has now generated substantial new data that it believes establish the relevance of the proposed potency assay measuring remestemcel-L’s in vitro anti-inflammatory and immunomodulatory activity to the 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 these new data to OTAT and address all other outstanding items as required for resubmission of the BLA
- Mesoblast continues to be in a well-established process with FDA’s Center for Biologics Evaluation and Research (CBER), and if the resubmission is accepted, CBER will consider the adequacy of the clinical data in the context of the related CMC issues noted above.
Activities regarding the rexlemestrocel-L Phase 3 programs in chronic low back pain (CLBP) and chronic heart failure (CHF):
- Received feedback from FDA’s OTAT that it agrees with Mesoblast’s proposal for pain reduction at 12 months as the primary endpoint for a pivotal trial to confirm the observed pain reduction 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 feedback from FDA’s OTAT confirming that reduction in major adverse cardiovascular events (MACE) of cardiovascular mortality or irreversible morbidity (non-fatal heart attack or stroke) is an acceptable clinically meaningful endpoint for determining the treatment benefit of rexlemestrocel-L for patients with chronic heart failure and low ejection fraction (HFrEF)
- Provided OTAT with top-line results of new analyses in pre-specified high-risk groups in the DREAM-HF Phase 3 trial of rexlemestrocel-L in HFrEF which showed that the greatest treatment benefit is in patients with diabetes and/or myocardial ischemia (
72% of total treated population), a target population at very high risk for mortality and irreversible morbidity due to micro- and macro-vascular disease despite receiving optimal standard of care therapies2
- Preparing formal submission to FDA of the detailed analyses of outcomes in high-risk HFrEF patients with diabetes and/or myocardial ischemia to agree on a potential pathway to approval
Key financial highlights for the quarter
- Revenues were US
$3.5 million , including US$2.3 million from TEMCELL® HS Inj.3 royalties on sales for SR-aGVHD in Japan, an increase of7% on TEMCELL royalties in the comparative quarter last year - Total Operating Activities resulted in net cash usage of US
$19.8 million in the quarter, a reduction of38% on the comparative quarter last year. Almost half of this spend was on remestemcel-L regulatory and manufacturing activities - Mesoblast completed a refinancing of its senior secured debt facility with a new US
$90 million five-year facility provided by funds managed by Oaktree Capital Management, L.P.
- Cash on hand at the end of the quarter was US
$94.8 million , with up to an additional US$40 million available to be drawn down from existing financing facilities subject to certain milestones
A copy of the Appendix 4C – Quarterly Cash Flow Report for the second quarter FY2022 is available on the investor page of the company’s website www.mesoblast.com.
About Mesoblast
Mesoblast is a world leader in developing allogeneic (off-the-shelf) cellular medicines for the treatment of severe 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 which 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 has a strong and extensive global intellectual property portfolio with protection extending through to at least 2041 in all major markets. 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 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 in development for advanced chronic heart failure and chronic low back pain. Two products have been commercialized in Japan and Europe by Mesoblast’s 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 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 GVHD and poor risk. Bone Marrow Transplantation 2021; https://doi.org/10.1038/s41409-021-01442-3
- Dunlay SM., et al. Circulation. 2019;140:e294–e324
- TEMCELL® HS Inj. is a registered trademark of JCR Pharmaceuticals Co. Ltd.
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 (including BLA resubmission), 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 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 | Media |
| Paul Hughes | Sumit Media |
| T: +61 3 9639 6036 | Grant Titmus |
| E: investors@mesoblast.com | T: +61 419 388 161 |
| E: grant@sumitmedia.com.au | |
| Rubenstein | |
| Nadine Woloshin | |
| T: +1 917-699-9456 | |
| E: nwoloshin@rubenstein.com |
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