Mesoblast Chief Executive
“Our second Phase 3 back pain trial with rexlemestrocel-L, aiming to confirm the durable pain reduction that was seen in the first Phase 3 trial, is underway. Finally, we were very pleased to have received a Rare Pediatric Disease (RPD) Designation from FDA for our cardiovascular product Revascor® in children with life-threatening congenital heart disease, and plan to discuss the trial results in the context of a regulatory approval pathway.”
ACTIVITY REPORT
Graft versus Host Disease – Pediatric and Adult Phase 3 Programs
- Mesoblast has requested a meeting with FDA this quarter to provide additional potency and characterization data for its product RYONCIL which it believes demonstrate that the product used as second-line after corticosteroids in the pivotal Phase 3 trial GVHD001 in children with SR-aGVHD, which successfully met its primary endpoint of Day 28 Overall Response, was made to a standard supporting the trial as being adequate and well controlled.
- The new potency assay data show that the RYONCIL product made with the current manufacturing process that has undergone successful inspection by FDA, demonstrates greater potency than the earlier generation product, providing context to its greater impact on survival.
- Showing that the product used in the completed pediatric Phase 3 trial was standardized as to potency and characterization could provide support for approval of the pediatric indication given the absence of any approved therapies for children.
- Survival in adults with SR-aGVHD who have failed at least one additional agent, such as ruxolitinib, remains as low as 20-30% by 100 days.1,2 In contrast, 100-day survival was 63% after remestemcel-L treatment was used under expanded access in 71 patients aged 12 and older with SR-aGVHD who failed to respond to at least one additional agent, such as ruxolitinib.
- The Blood and Marrow Transplant Clinical Trials Network (BMT CTN) in
the United States , a body that is funded by theNational Institutes of Health (NIH) and is responsible for approximately 80% of all US allogeneic BMTs, has agreed to develop and execute a pivotal trial of RYONCIL in adults who are refractory to both corticosteroids and a second line agent such as ruxolitinib. - Mesoblast will provide the Phase 3 trial protocol to FDA ahead of the upcoming meeting this quarter.
Cardiovascular – Program in Pediatric Congenital Heart Disease, Adult Phase 3 Program in Chronic Heart Failure with Reduced Ejection Fraction (HFrEF)
- This month FDA granted Mesoblast a Rare Pediatric Disease (RPD) Designation for Revascor® (rexlemestrocel-L) following submission of results from the randomized controlled trial in children with hypoplastic left heart syndrome (HLHS), a potentially life-threatening congenital heart condition.
- The results from the blinded, randomized, placebo-controlled prospective trial of REVASCOR conducted in
the United States in children with HLHS were published in theDecember 2023 issue of the peer reviewed theJournal of Thoracic and Cardiovascular Surgery Open (JTCVS Open).3 In the HLHS trial, a single intramyocardial administration of REVASCOR at the time of staged surgery resulted in the desired outcome of significantly increased left ventricular (LV) end-systolic and end-diastolic volumes over 12 months compared with controls as measured by 3D echocardiography, (p=0.009 & p=0.020 respectively), facilitating life-saving biventricular surgery to be achievable in 100% of REVASCOR-treated children vs only 57% of controls. - RPD Designation is granted by the FDA for certain serious or life-threatening diseases which primarily affect children.
- On FDA approval of a Biologics Licensing Application (BLA) for REVASCOR for the treatment of HLHS, Mesoblast may be eligible to receive a Priority Review Voucher (PRV) that can be redeemed for any subsequent marketing application or may be sold or transferred to a third party.
- REVASCOR® has shown the potential to reduce major adverse cardiac events such as heart attack and cardiovascular death in high risk patients with HFrRF. Mesoblast will meet with FDA this quarter to address potential pathways to approval for REVASCOR under our Regenerative Medicine Advanced Therapies (RMAT) designation.
Chronic Low Back Pain – Phase 3 Program
- Second Phase 3 trial underway for rexlemestrocel-L in the treatment of chronic low back pain (CLBP) due to inflammatory disc degeneration– a condition affecting at least seven million people in both the US and
Europe alone. - Phase 3 trial activities, investigators and trial sites across
the United States are being managed by a leading contract research organisation (CRO) specializing in pain trials. - The trial’s primary endpoint is reduction in pain at 12 months after a single intra-discal injection of rexlemestrocel-L.
- First Phase 3 trial showed significant pain reduction at 12 and 24 months, and confirmation of these results will provide FDA with a clinical data package that may result in product approval.
FIANANCIAL REPORT
Strengthened Balance Sheet
Institutional Placement and Entitlement Offer completed raising
Cash balance at the end of the quarter was
Cost containment strategy on-track
Cost containment strategies and payroll reductions have been enacted by management and the Board enabling continuation of Phase 3 programs for SR-aGVHD and CLBP in the quarter whilst still achieving reductions in net operating cash spend:
- Net operating cash spend of
US$12.3 million for the quarter. - 25% reduction in net operating cash spend from the comparative quarter in FY2023.
- 32% reduction in net operating cash spend from the comparative quarter in FY2022.
- On target to achieve a 23% (
$15m ) reduction in net operating spend in FY2024 compared to FY2023 which will be partially offset by investment in our Phase 3 programs for SR-aGVHD and CLBP.
We will maintain our focus on cutting costs and preserving cash in the remainder of the year whilst complimenting that with initiatives currently underway to increase cash inflows which would by design enable us to prudently invest in our Phase 3 programs for SR-aGVHD and CLBP. In this regard, we are working on corporate initiatives to strengthen our balance sheet, including royalty monetization and strategic partnerships to both access existing commercial distribution channels and supplement costs of development.
Revenues
Revenue from royalties on sales of TEMCELL® HS Inj.5 sold in
Other
Fees to Non-Executive Directors were nil, consulting payments to Non-Executive Directors were
A copy of the Appendix 4C – Quarterly Cash Flow Report for the second quarter FY2024 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
- 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.
- Abedin S, et al. Ruxolitinib resistance or intolerance in steroid-refractory acute graft versus-host disease — a real-world outcomes analysis.
British Journal of Haematology , 2021;195:429–43. - Wittenberg RE, Gauvreau K, Leighton J,
Moleon-Shea M , Borow KM, Marx GR, Emani SM, Prospective randomized controlled trial of the safety and feasibility of a novel mesenchymal precursor cell therapy in hypoplastic left heart syndrome, JTCVS Open Volume 16,Dec 2023 , doi: https://doi.org/10.1016/j.xjon.2023.09.031 - Using
Reserve Bank of Australia (RBA) published exchange rate fromDecember 31, 2023 of 1A$:0.6840US$ . - TEMCELL® HS Inj. is a registered trademark of JCR Pharmaceuticals Co. Ltd.
- TEMCELL sales by our Licensee are recorded in Japanese Yen before being translated into USD for the purposes of calculating the royalty paid to Mesoblast. Results have been adjusted for the movement of the USD to Japanese Yen exchange rate from 1USD:
133.70 Yen for the 6 months endedDecember 31, 2022 to 1USD:142.82 Yen for the 6 months ended December, 2023. - 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, 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.
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