Financial Highlights
- Revenue from royalties on sales of TEMCELL® HS Inj.1 sold in
Japan by our licensee for the quarter increased 36% toUS$1.9 million fromUS$1.4 million for the quarter endedSeptember 30, 2022 . - Net cash usage for operating activities in the quarter was
US$16.5 million ; this represented a reduction ofUS$1.7 million , or 9%, on the comparative quarter in FY2022, and a reduction ofUS$14.1 million , or 46%, on the comparative quarter in FY2021.2
- In
December 2022 we announced that funds managed byOaktree Capital Management, L.P. (“Oaktree”) extended to Mesoblast the availability of up to an additionalUS$30 million of itsUS$90 million five-year facility subject to achieving certain milestones on or beforeSeptember 30, 2023 . - Cash on hand at the end of the quarter was
US$67.6 million , with up to an additionalUS$40 million available to be drawn down from existing financing facilities subject to certain milestones.
Operational Highlights
- The Biologics License Application (BLA) resubmission documents for remestemcel-L in the treatment of children with steroid-refractory graft versus host disease (SR-aGVHD) are complete and expected to be filed with FDA shortly.
- Survival outcomes have not improved over the past two decades for children or adults with the most severe forms of SR-aGVHD.3-5 The lack of any approved treatments for children under 12 means that there is an urgent need for a therapy that improves the dismal survival outcomes in children.
- Long-term survival results were received for remestemcel-L from Mesoblast’s pivotal Phase 3 clinical trial (GVHD-001) in children with SR-aGVHD. The results showed durable survival through 4 years of follow-up. These new long-term survival data are a key component of the Company’s BLA resubmission to the FDA.
The study was performed by theCenter for International Blood and Marrow Transplant Research (CIBMTR) on 51 evaluable children with SR-aGVHD who were enrolled in the phase 3 trial across 20 centers in the US.
Overall survival in the remestemcel-L cohort was 63% at 1 year, 51% at 2 years, and 49% at 4 years, with median survival of 2 to 3 years. In recently published studies of children or adults with SR-aGVHD who received best available therapy (BAT) or the only FDA-approved agent for adults, ruxolitinib, 1 year survival was 40-49% and 2 year survival was 25%-38%.3,6-8
- Mesoblast has previously gained alignment with the FDA on key metrics for a pivotal Phase 3 study of rexlemestrocel-L in patients with chronic low back pain (CLBP) with degenerative disc disease which seeks to replicate the significant reduction in pain seen in the first Phase 3 trial.
- FDA has confirmed that 12-month reduction in pain is an approvable indication, with key secondary measures of improvement in function and reduction in opioid usage.
- Preparation underway to commence the pivotal Phase 3 clinical trial by mid-CY2023.
- Results from three randomized controlled trials of rexlemestrocel-L in class II/III heart failure with reduced ejection fraction (HFrEF) and in end-stage HFrEF with left ventricular assist devices (LVADs) support the idea of a common mechanism of action (MOA) by which rexlemestrocel-L reverses inflammation-related endothelial dysfunction and reduces adverse clinical outcomes across the spectrum of HFrEF patients.
- Improvement in left ventricular ejection fraction (LVEF) at 12 months in patients with HFrEF may be an appropriate early surrogate endpoint for long term reduction in major adverse cardiovascular events (MACE).
- Mesoblast plans to meet with FDA under its existing regenerative medicine advanced therapy (RMAT) designation to discuss data and the evidence of a common MOA across the broader HFrEF spectrum, including LVAD patients.
Other
Salary payments to full-time Executive Directors were
A copy of the Appendix 4C – Quarterly Cash Flow Report for the second quarter FY2023 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 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, biologic-resistant inflammatory bowel disease, and 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
Mesoblast has locations in
References / Footnotes
- TEMCELL® HS Inj. is a registered trademark of JCR Pharmaceuticals Co. Ltd.
- The Appendix 4C for the quarter ended
December 31, 2021 reported net cash usage for operating activities ofUS$19.8 million which was subsequently revised toUS$18.2 million , and the quarter endedDecember 31, 2020 reported net cash usage for operating activities ofUS$32.0 million which was subsequently revised toUS$30.6 million . These revisions were due to a change in accounting policy adopted atDecember 31, 2021 . - Rashidi A et al. Outcomes and predictors of response in steroid-refractory acute graft-versus-host disease: single-center results from a cohort of 203 patients. Biol Blood Bone Marrow Transplant 2019; 25(11):2297-2302.
- Berger M, Pessolano R, Carraro F, Saglio F, Vassallo E, Fagioli F. Steroid-refractory acute graft-versus-host disease graded III-IV in pediatric patients. A mono-institutional experience with a long-term follow-up. Pediatric Transplantation. 2020; 24(7):e13806
- Biavasco F, Ihorst G, Wasch R, Wehr C, Bertz H, Finke J, Zeiser R. Therapy response of glucocorticoid-refractory acute GVHD of the lower intestinal tract. Bone Marrow Transplantation. 2022
- MacMillan ML et al. Pediatric acute GVHD: clinical phenotype and response to upfront steroids. Bone Marrow Transplant 2020; 55(1): 165-171
- Zeiser R et al. Ruxolitinib for Glucocorticoid-Refractory Acute Graft-versus-Host Disease. N Engl J Med 2020;382:1800-10.
- Jagasia M et al. Ruxolitinib for the treatment of steroid-refractory acute GVHD (REACH1): a multicenter, open-label phase 2 trial. Blood. 2020 May 14; 135(20): 1739–1749.
- As required by ASX listing rule 4.7 and reported in Item 6 of the Appendix 4C, reported are the aggregated total payments to related parties being Executive Directors and Non-Executive Directors
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
Release authorized by the Chief Executive.
For more information, please contact:
Corporate Communications / Investors | Media |
BlueDot Media | |
T: +61 3 9639 6036 | |
E: investors@mesoblast.com | T: +61 419 880 486 |
E: steve@bluedot.net.au | |
Rubenstein | |
E: tmackay@rubenstein.com |
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