Appendix 4C Quarterly Activity Report

NEW YORK, Jan. 31, 2023 (GLOBE NEWSWIRE) — Mesoblast Limited (Nasdaq:MESO; ASX:MSB), a global leader in allogeneic cell medicines for inflammatory diseases, today announced the We have provided two quarterly activity reports. .

financial highlights

• Royalty income from Temcell sales
  
  ^®
  
  HS injection
  
  ¹
  
  Licensee sales in Japan for the quarter increased 36% to $1.9 million from $1.4 million for the quarter ended September 30, 2022.

• Net cash used from operations in the fourth quarter was $16.5 million. This represents a reduction of USD 1.7 million (9%) compared to the 2022 comparative quarter and represents a reduction of USD 14.1 million (46%) over the 2021 comparative quarter.
  
  ²
  

• In December 2022, a fund managed by Oaktree Capital Management, LP (“Oaktree”) will be awarded up to a $90 million five-year facility, subject to the achievement of certain milestones by September. Announced US$30 million in additional funding for Mesoblast. March 30, 2023.

• Cash on hand at the end of the quarter was US$67.6 million, subject to certain milestones, and up to an additional US$40 million can be drawn from existing lines of credit.

Operational highlights

• The Biologics License Application (BLA) resubmission dossier for remestemcel-L in the treatment of children with steroid-resistant graft-versus-host disease (SR-aGVHD) has been completed and will be submitted to the FDA soon.

• Survival outcomes for children or adults with the most severe forms of SR-aGVHD have not improved over the past two decades.
  
  ^3-5
  
  The lack of approved treatments for children under the age of 12 means that there is an urgent need for treatments that improve children’s dismal survival outcomes.

• Mesoblast’s pivotal Phase 3 clinical trial (GVHD-001) in children with SR-aGVHD provided long-term survival results for remestemcel-L. Results showed durable survival through 4 years of follow-up. These new long-term survival data are an important factor in our resubmission of her BLA to the FDA.

  This study was conducted by the International Center for Blood and Bone Marrow Transplant Research (CIBMTR) in 51 evaluable children with SR-aGVHD enrolled in a Phase 3 trial across 20 US centers.

  Overall survival in the remestemcel-L cohort was 63% at 1 year, 51% at 2 years, and 49% at 4 years, with a median survival of 2-3 years. A recently published study of children or adults with SR-aGVHD who received best available therapy (BAT) or the only FDA-approved drug for adults, ruxolitinib, had a one-year survival rate of 40 ~49%, with a 2-year survival rate of 25% to 38%. .
  
  ^3,6-8
  

• Mesoblast previously collaborated with the FDA on key endpoints of a pivotal Phase 3 trial of rexlemestrocel-L in patients with chronic low back pain with degenerative disc disease (CLBP). trial.

• The FDA has confirmed that pain relief for 12 months is an approvable indication, with important secondary measures of improved function and reduced opioid use.

• Preparations are underway to initiate a pivotal Phase 3 clinical trial by mid-2023.

• Results from three randomized controlled trials of rexlemestrocel-L in class II/III heart failure with reduced ejection fraction (HFrEF) and end-stage HFrEF with left ventricular assist device (LVAD) demonstrated a common mechanism of action (MOA). support the idea of Thereby, rexlemestrocel-L reverses inflammation-associated endothelial dysfunction and reduces adverse clinical outcomes across a spectrum of HFrEF patients.

• Improvement in left ventricular ejection fraction (LVEF) at 12 months in HFrEF patients may be a suitable initial surrogate endpoint for long-term reduction in major adverse cardiovascular events (MACE) .

• Mesoblast plans to meet with the FDA under its existing Regenerative Medicine Advanced Treatment (RMAT) designation to discuss data and evidence of common MOA across the broader HFrEF spectrum, including patients with LVAD.

other


Full-time executive director salaries were US$324,678 and non-executive director compensation was US$192,164 and are detailed in item 6 of the Appendix 4C Cash Flow Report for this quarter.

⁹

Appendix 4C – A copy of the Quarterly Cash Flow Report for the second quarter of fiscal year 2023 is available on the investor page of the company’s website, www.mesoblast.com.

About mesoblast


Mesoblast is a global leader in the development of allogeneic (off-the-shelf) cell medicines for the treatment of severe and life-threatening inflammatory conditions. The Company leverages its proprietary mesenchymal cell therapy technology platform to develop 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. Established a broad portfolio, resulting in significant declines. of harmful inflammatory processes.

Mesoblast has a strong and extensive global intellectual property portfolio protected until at least 2041 in all major markets. Our proprietary manufacturing process produces industrial-scale cryopreserved off-the-shelf cell medicines. These cell therapies have defined drug release standards and are expected to be readily available to patients worldwide.

Mesoblast is developing product candidates for defined indications based on its remestemcel-L and rexlemestrocel-L allogeneic stromal cell technology platforms. Remestemcel-L is being developed for pediatric and adult inflammatory diseases, including steroid-refractory acute graft-versus-host disease, biologic-resistant inflammatory bowel disease, and acute respiratory distress syndrome. Rexlemestrocel-L is being developed for progressive chronic heart failure and chronic low back pain. Two products have been commercialized in Japan and Europe by his 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, visit www.mesoblast.com, LinkedIn: Mesoblast Limited, and Twitter: @Mesoblast.

References / Footnotes

1. Temcell® HS Injection is a registered trademark of JCR Pharmaceuticals Co., Ltd.

2. Net cash used from operations in Appendix 4C for the quarter ended December 31, 2021 was $19.8 million, subsequently restated to $18.2 million Net cash used was reported at $32 million. It has since been revised to $30.6 million. These adjustments are due to changes in accounting policies adopted on December 31, 2021.

3. Rashidi A et al. Outcomes and predictors of response in steroid-resistant acute graft-versus-host disease: single-center outcomes from a cohort of 203 patients.
   
   Biol Blood bone marrow transplant
   
   2019; 25(11):2297-2302.

4. Berger M, Pessolano R, Carraro F, Saglio F, Vassallo E, Fagioli F. Steroid-resistant acute graft-versus-host disease grade III-IV in pediatric patients. Single-center experience with long-term follow-up.
   
   Pediatric transplantation
   
   2020; 24(7):e13806

5. Biavasco F, Ihorst G, Washr R, Wehr C, Bertz H, Finke J, Zeiser R. Treatment response in glucocorticoid-refractory acute GVHD of the lower intestinal tract.
   
   Bone marrow transplantation.
   
   2022

6. Macmillan ML et al. Pediatric acute GVHD: clinical phenotype and response to antecedent steroids.
   
   Bone marrow transplantation
   
   2020; 55(1):165-171

7. Zeiser R et al. Ruxolitinib for glucocorticoid-resistant acute graft-versus-host disease.
   
   N Engl J Med
   
   2020;382:1800-10.

8. Jagasia M et al. Ruxolitinib for the treatment of steroid-resistant acute GVHD (REACH1): a multicenter, open-label, phase 2 trial.
   
   blood.
   
   14 May 2020; 135(20): 1739–1749.

9. Reported are gross payments to related parties who are executive and non-executive directors, as required by ASX Listing Rule 4.7 and reported in Item 6 of Appendix 4C.

Forward-Looking Statements


This press release contains forward-looking statements relating to future events or our future financial performance, which involve known and unknown risks, uncertainties and our actual results, levels of activity, There are other factors that could cause performance or results to differ materially from future results. , the level of activity, performance or achievements expressed or implied by these 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 are not guarantees of future performance or results. Actual results may differ from those anticipated in these forward-looking statements, and such differences may be material and adverse. Forward-looking statements include, but are not limited to, statements regarding the following: Mesoblast’s ability to advance, enroll and successfully complete product candidates in clinical trials, including multinational clinical trials. Ability to improve Mesoblast’s manufacturing capabilities. The timing or likelihood of regulatory submissions and approvals (including resubmission of BLAs), manufacturing activities, and product marketing activities (if any); Commercialization of Mesoblast’s product candidates, if approved. Regulatory or public awareness and market acceptance of the use of stem cell-based therapies. Mesoblast’s product candidate, if approved, may be withdrawn from the market due to patient adverse events or death. The potential benefits of strategic alliance agreements and Mesoblast’s ability to enter into and maintain established strategic alliances. Mesoblast’s ability to establish and maintain intellectual property in product candidates, and Mesoblast’s ability to successfully defend them against alleged infringement. The extent of protection Mesoblast may establish and maintain for its intellectual property rights covering its product candidates and technology. Estimates of Mesoblast’s costs, future earnings, capital requirements and need for additional funding. Mesoblast financial performance. Mesoblast’s competitors and industry developments. Pricing and refunds for Mesoblast’s product candidates if approved. This press release should be read in conjunction with our Risk Factors in our recent filings with the SEC or on our website. Uncertainties and risks that may cause Mesoblast’s actual results, performance and achievements to differ materially from those expressed or implied by such 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.

Release approved by the CEO.

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