Appendix 4C Quarterly Activity Report for Quarter Ended December 31, 2024

Rhea-AI Summary

Mesoblast (MESO) reported key developments for Q2 FY2025 ended December 31, 2024. The company achieved a significant milestone with Ryoncil® becoming the first FDA-approved MSC therapy for steroid-refractory acute GvHD in children. A distribution network has been established with Cencora for efficient product delivery.

The company successfully completed a global private placement raising A$260 million (US$161 million). Net operating cash spend was US$10.1 million, reduced by 18% compared to the previous quarter. Cash on hand was US$38 million, with pro-forma cash after January's raise of approximately US$200 million.

The Phase 3 trial of rexlemestrocel-L for chronic low back pain is actively enrolling patients. Under RMAT designation, Mesoblast plans to discuss accelerated approval filing for end-stage heart failure patients with FDA.

Positive

• FDA approval of Ryoncil® as first MSC therapy for SR-aGvHD treatment
• Successful completion of A$260M (US$161M) private placement
• 18% reduction in net operating cash spend vs previous quarter
• Commercial inventory manufactured and distribution network established

Negative

• Continued cash burn of US$10.1M in quarterly operating expenses

NEW YORK, Jan. 30, 2025 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today provided highlights of its recent activities for the second quarter ended December 31, 2024.

ACTIVITY REPORT         

• On December 18, 2024, Ryoncil^® (remestemcel-L) became the first mesenchymal stromal cell (MSC) therapy approved by U.S. FDA for any indication.
• FDA approved Ryoncil^® as the first and only therapy for children aged 2 months and older, including adolescents and teenagers, with steroid-refractory acute graft versus host disease (SR-aGvHD), a life-threatening condition with high mortality rates.
• Commercial inventory has been manufactured and a distribution network has been established using Cencora, a leader in specialty pharmaceutical services and distribution. Cencora will leverage its cryogenic logistics capabilities and state-of-the art cryogenic storage infrastructure to enable the efficient and secure delivery of cryopreserved product to U.S. treatment centers.
• The confirmatory Phase 3 trial of rexlemestrocel-L in patients with chronic low back pain (CLBP) due to inflammatory degenerative disc disease (DDD) of less than five years duration is actively enrolling and treating patients at multiple sites across the United States; the capital raise concluded this month will facilitate expansion of sites enrolling in the trial and acceleration of patient accrual.
• Under its Regenerative Medicine Advanced Therapy (RMAT) designation Mesoblast intends to meet with FDA to discuss data presentation, timing and FDA expectations for an accelerated approval filing in end-stage heart failure patients.
• In November 2024 a publication in the prestigious peer-reviewed European Journal of Heart Failure (EJHF), reported that a single intramyocardial injection of Revascor^® (rexlemestrocel-L), Mesoblast’s second generation allogeneic, STRO3-immunoselected, and industrially manufactured stromal cell therapy, results in improved survival in high-risk NYHA Class II/III patients with ischemic heart failure and inflammation.¹ This identifies the HFrEF population that is responsive to REVASCOR and will be the target of a confirmatory trial after accelerated approval, if received.
• FDA granted REVASCOR RMAT designation following submission of results from the randomized controlled trial in children with hypoplastic left heart syndrome (HLHS), a potentially life-threatening congenital heart condition.
  

FINANCIAL REPORT

Mesoblast successfully completed a global private placement primarily to existing major US, UK, and Australian shareholders raising A$260 million (US$161 million).

Results for Second Quarter ended December 31, 2024

• Net operating cash spend was US$10.1 million.
• Net operating cash spend for the quarter was reduced by 18% (US$2.2 million) versus the prior comparative quarter in FY2024.
• Cash on hand at the end of the quarter was US$38 million (A$61 million) with pro-forma cash after proceeds raised in January of approximately US$200 million (A$322 million).
  

Other

Fees to Non-Executive Directors were US$295,803, consulting payments to Non-Executive Directors were US$80,602 and salary payments to full-time Executive Directors were US$231,552, detailed in Item 6 of the Appendix 4C cash flow report for the quarter.² In the quarter our Non-Executive Directors received the payment of 50% of directors fees owing since 1 August 2023 for which payment was deferred, contingent on FDA approval. From August 2023 to July 2025, our Non-Executive Directors and Executive Directors (our Chief Executive and Chief Medical Officers) have voluntarily reduced cash payment of their fees by 50% and their base salaries by 30% in lieu of accepting equity-based incentives, respectively.

A copy of the Appendix 4C – Quarterly Cash Flow Report for the second quarter FY2025 is available on the investor page of the company’s website www.mesoblast.com.

About Mesoblast
Mesoblast (the Company) is a world leader in developing allogeneic (off-the-shelf) cellular medicines for the treatment of severe and life-threatening inflammatory conditions. The therapies from the Company’s proprietary mesenchymal lineage cell therapy technology platform 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’s RYONCIL^® (remestemcel-L) for the treatment of steroid-refractory acute graft versus host disease (SR-aGvHD) in children 2 months and older is the first FDA-approved mesenchymal stromal cell (MSC) therapy. Please see the full Prescribing Information at www.ryoncil.com.

Mesoblast is committed to developing additional cell therapies for distinct indications based on its remestemcel-L and rexlemestrocel-L allogeneic stromal cell technology platforms. RYONCIL is being developed for additional inflammatory diseases including SR-aGvHD in adults and biologic-resistant inflammatory bowel disease. Rexlemestrocel-L is being developed for heart failure and chronic low back pain. The Company has established commercial partnerships in Japan, Europe and China.

About Mesoblast intellectual property: Mesoblast has a strong and extensive global intellectual property portfolio, with over 1,000 granted patents or patent applications covering mesenchymal stromal cell compositions of matter, methods of manufacturing and indications. These granted patents and patent applications provide commercial protection extending through to at least 2041 in all major markets.

About Mesoblast manufacturing: 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 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

1. Perin EC. Et al. Mesenchymal precursor cells reduce mortality and major morbidity in ischaemic heart failure with inflammation: DREAM-HF. Eur J Heart Fail 2024. https://doi.org/10.1002/ejhf.3522
2. 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 RYONCIL for pediatric SR-aGVHD and any other 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 – Global
Paul Hughes	Allison Worldwide
T: +61 3 9639 6036	Emma Neal
E: investors@mesoblast.com	T: +1 603 545 4843
	E: emma.neal@allisonworldwide.com
	Media – Australia
	BlueDot Media
	Steve Dabkowski
	T: +61 419 880 486
	E: steve@bluedot.net.au

FAQ

What was Mesoblast's (MESO) major FDA approval in December 2024?

In December 2024, Mesoblast's Ryoncil® became the first FDA-approved mesenchymal stromal cell therapy for treating steroid-refractory acute graft versus host disease in children aged 2 months and older.

How much capital did MESO raise in their latest private placement?

Mesoblast raised A$260 million (US$161 million) through a global private placement primarily from existing major US, UK, and Australian shareholders.

What was MESO's cash position at the end of December 2024?

Mesoblast had US$38 million (A$61 million) cash on hand at the end of December 2024, with pro-forma cash after January's raise of approximately US$200 million (A$322 million).

How much did MESO reduce its operating cash spend in Q2 FY2025?

Mesoblast reduced its net operating cash spend by 18% (US$2.2 million) compared to the prior comparative quarter in FY2024.

Which company is handling Ryoncil's distribution for MESO in the US?

Cencora, a leader in specialty pharmaceutical services, will handle Ryoncil's distribution using its cryogenic logistics capabilities and storage infrastructure.