Press Release: Appendix 4C Quarterly Activity Report for Quarter Ended September 30, 2024

Appendix 4C Quarterly Activity Report for Quarter Ended September 30, 2024

NEW YORK, Oct. 30, 2024 (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 first quarter ended September 30, 2024.

Mesoblast Chief Executive Silviu Itescu said: "We have had an extremely busy and productive quarter starting right from the outset with the filing of our Biologics License Application $(BLA.AU)$ with the United States Food and Drug Administration (FDA) for approval of Ryoncil$(R)$ (remestemcel-L) in the treatment of children with steroid-refractory acute graft versus host disease (SR-aGvHD). We continue to be engaged in active and ongoing interactions with the agency as part of the review process. We are anticipating a decision prior to or on the FDA's Prescription Drug User Fee Act (PDUFA) goal date of January 7, 2025."

"During the period we put in place a strategic financing to ensure that the Company is well capitalized for a commercial launch of RYONCIL. This has been structured as a convertible note subscription agreement with our largest shareholder for issue, at Mesoblast's sole discretion, up to US$50.0 million convertible notes following approval of RYONCIL by FDA. At the same time, we have maintained a strong focus on our cost control with net operating spend for the period of US$10.5 million, down 26% on the prior corresponding quarter."

"I look forward to providing an update at our Annual General Meeting $(AGM)$ on November 15(th) 12.00 noon AEDT (November 14(th) 8.00pm EST)."

KEY HIGHLIGHTS

Ryoncil(R) (Remestemcel-L) for Steroid-Refractory Acute Graft Versus Host Disease -- Potential FDA Approval

   -- There are no approved treatments for children under 12 with 
      steroid-refractory acute GvHD, making approval of a safe and effective 
      treatment for this vulnerable population the most urgent need. 
 
   -- Mesoblast resubmitted its BLA to FDA for approval of RYONCIL on July 8, 
      2024 and anticipates a decision prior to or on the FDA's Prescription 
      Drug User Fee Act (PDUFA) goal date of January 7, 2025. 
 
   -- FDA has already conducted the Pre-License Inspection (PLI) of the 
      manufacturing process for RYONCIL in May 2023 and this did not result in 
      the issuance of any Form 483. 
 
   -- Inventory has been manufactured and there is an established supply chain 
      to ensure cryopreserved product is available for delivery to meet the 
      needs of each site immediately post approval, with ability to scale up as 
      necessary going forward. 
 
   -- We have been working diligently to lay the groundwork for a successful 
      launch of RYONCIL, including hiring select senior positions to implement 
      a targeted commercial strategy since 50% of pediatric transplants are 
      performed at just 15 centers. 
 
   -- Post approval implementation will initially target those centers with 
      greatest experience using the RYONCIL product and highest volume, with 
      staged rollout beyond. 

Revascor(R) (Rexlemestrocel-L) for Pediatric Congenital Heart Disease - Hypoplastic Left Heart Syndrome

   -- Earlier this year, FDA granted Mesoblast's second generation allogeneic, 
      STRO3-immunoselected, and industrially manufactured stromal cell product 
      REVASCOR both Rare Pediatric Disease Designation (RPDD) and Orphan-Drug 
      Designation $(ODD)$ for treatment of children with hypoplastic left heart 
      syndrome (HLHS), a potentially life-threatening congenital heart 
      condition. 
   -- Results from a blinded, randomized, placebo-controlled prospective trial 
      of REVASCOR conducted in the United States in children with HLHS were 
      published in the December 2023 issue of the peer reviewed The Journal of 
      Thoracic and Cardiovascular Surgery Open (JTCVS Open).1 
 
          -- A single intramyocardial administration of REVASCOR at the time of 
             staged surgery resulted in the desired outcome of significantly 
             larger increases in 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). 
 
          -- These changes are indicative of clinically important growth of the 
             small left ventricle, facilitating the ability to have a 
             successful surgical correction, known as full biventricular (BiV) 
             conversion, which allows for a normal two ventricle circulation. 
 
          -- Without full BiV conversion the right heart chamber is under 
             excessive strain with increased risk of heart failure, liver 
             cirrhosis, and death. 
 
          -- RPDD demonstrates that the disease is serious or life-threatening 
             and the manifestations primarily affect individuals aged from 
             birth to 18 years, including age groups often called neonates, 
             infants, children, and adolescents, and that the disease is a rare 
             disease or condition. 
 
          -- On FDA approval of a BLA for REVASCOR for the treatment of HLHS, 
             Mesoblast may be eligible to receive a Priority Review Voucher 
             $(PRV.UK)$ that can be redeemed for any subsequent marketing 
             application or may be sold or transferred to a third party. 
 
          -- Mesoblast plans to meet with FDA to discuss the clinical data to 
             support regulatory approval for REVASCOR in children with this 
             life-threatening condition. 

REVASCOR for Chronic Heart Failure with Reduced Ejection Fraction (HFrEF) and Persistent Inflammation

   -- In March FDA informed Mesoblast that it supports an accelerated approval 
      pathway for its second generation allogeneic, STRO3-immunoselected, and 
      industrially manufactured stromal cell product REVASCOR, for patients 
      with end-stage ischemic HFrEF kept alive with a left ventricular assist 
      device (LVAD). 
 
   -- This followed presentation to FDA of the results of two complementary 
      randomized controlled trials of REVASCOR, one in patients with end-stage 
      HFrEF and LVADs and a second in advanced NYHA class II/III HFrEF 
      patients. 
 
   -- Mesoblast has received RMAT designation for rexlemestrocel-L in the 
      treatment of end-stage heart failure in LVAD patients and intends to meet 
      with FDA, after the meeting on HLHS, to discuss data presentation, timing 
      and FDA expectations for an accelerated approval filing in these 
      patients. 

Rexlemestrocel-L for Chronic Low Back Pain associated with Degenerative Disc Disease -- Phase 3 Program

   -- The confirmatory Phase 3 trial of Mesoblast's second generation 
      allogeneic, STRO3-immunoselected, and industrially manufactured stromal 
      cell product rexlemestrocel-L in patients with chronic low back pain 
      (CLBP) due to inflammatory degenerative disc disease $(DDD)$ of less than 
      five years duration has commenced enrollment at multiple sites across the 
      United States. 
 
          -- FDA has previously agreed on the design of this 300-patient 
             randomized, placebo-controlled confirmatory Phase 3 trial, and the 
             12-month primary endpoint of pain reduction as an approvable 
             indication. 
 
          -- This endpoint was successfully met in Mesoblast's first Phase 3 
             trial. 
 
          -- Key secondary measures include improvement in quality of life and 
             function. 
 
          -- A particular focus is on treatment of patients on opioids, since 
             discogenic back pain accounts for approximately 50% of 
             prescription opioid usage in the US. 
 
          -- Significant pain reduction and opioid cessation were observed in 
             Mesoblast's first Phase 3 trial. 
 
          -- FDA has designated rexlemestrocel-L a Regenerative Medicine 
             Advanced Therapy (RMAT) for the treatment of chronic low back 
             pain. RMAT designation provides all the benefits of Breakthrough 
             and Fast Track designations, including rolling review and 
             eligibility for priority review on filing of a BLA. 

FINANCIAL REPORT

We have continued our disciplined financial management strategy focused on ensuring that we are well-positioned to execute our clinical, manufacturing, and commercialization plans while maintaining conservative fiscal practices. The successful implementation of our cost containment plan over the past 12 months and the re-prioritization of projects has enabled us to reduce cash expenditure whilst still making significant strides forward on key programs as outlined above.

To ensure that the Company is well capitalized for a commercial launch of RYONCIL, we recently entered into a convertible note subscription agreement with our largest shareholder for issue, at Mesoblast's sole discretion, up to US$50.0 million convertible notes following approval of RYONCIL by FDA.

First Quarter Results

   -- Cash balance at September 30, 2024 is US$51.1 million, with additional 
      US$60.0 million available from existing financing facilities on RYONCIL 
      approval. 
 
   -- Net operating cash spend of US$10.5 million for the first quarter FY2025. 
 
          -- 26% (US$3.7 million) reduction in net operating cash spend for the 
             first quarter FY2025 versus the prior comparative quarter in 
             FY2024. 

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