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) with the United States Food and Drug Administration (FDA) for approval of Ryoncil® (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 15th 12.00 noon AEDT (November 14th 8.00pm EST)."
KEY HIGHLIGHTS
Ryoncil® (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® (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 ...
