Mesoblast Limited (NASDAQ: MESO, ASX:MSB) is awaiting a decision from the U.S. Food and Drug Administration (FDA) regarding its Biologics License Application (BLA) for Ryoncil® (remestemcel-L) for the treatment of children with steroid-refractory acute graft versus host disease (SR-aGVHD). The FDA's Prescription Drug User Fee Act (PDUFA) goal date is set for January 7, 2025.
Ryoncil for Steroid-Refractory Acute Graft Versus Host Disease
There are currently no approved treatments for children under 12 years old with SR-aGVHD, making the potential approval of Ryoncil a critical unmet need. Mesoblast resubmitted its BLA to the FDA on July 8, 2024. The FDA has already conducted a Pre-License Inspection (PLI) of the manufacturing process for Ryoncil in May 2023, which did not result in any Form 483 observations.
Mesoblast has manufactured inventory and established a supply chain to ensure cryopreserved product is readily available for delivery to treatment centers immediately following potential approval. The company is focusing its initial commercialization efforts on the 15 centers that perform 50% of pediatric transplants, with a staged rollout planned thereafter.
Revascor for Pediatric Congenital Heart Disease
Mesoblast's second-generation allogeneic stromal cell product, Revascor® (rexlemestrocel-L), has been granted both Rare Pediatric Disease Designation (RPDD) and Orphan-Drug Designation (ODD) by the FDA for the treatment of children with hypoplastic left heart syndrome (HLHS), a life-threatening congenital heart condition. A blinded, randomized, placebo-controlled trial of Revascor in children with HLHS showed statistically significant increases in left ventricular (LV) end-systolic and end-diastolic volumes over 12 months compared to controls (p=0.009 & p=0.020, respectively), as measured by 3D echocardiography. These changes indicate clinically important growth of the small left ventricle, potentially facilitating successful surgical correction and normal two-ventricle circulation.
Revascor for Chronic Heart Failure
The FDA supports an accelerated approval pathway for Revascor in patients with end-stage ischemic heart failure with reduced ejection fraction (HFrEF) kept alive with a left ventricular assist device (LVAD). This decision followed the presentation of results from two randomized controlled trials of Revascor, one in patients with end-stage HFrEF and LVADs and another in advanced NYHA class II/III HFrEF patients. Mesoblast has received Regenerative Medicine Advanced Therapy (RMAT) designation for rexlemestrocel-L in this indication and plans to meet with the FDA to discuss data presentation, timing, and expectations for an accelerated approval filing.
Rexlemestrocel-L for Chronic Low Back Pain
A confirmatory Phase 3 trial of rexlemestrocel-L has commenced enrollment at multiple sites across the United States for patients with chronic low back pain (CLBP) due to inflammatory degenerative disc disease (DDD) of less than five years duration. The FDA has agreed on the design of this 300-patient randomized, placebo-controlled trial, with the 12-month primary endpoint of pain reduction. The trial will also focus on treatment of patients on opioids, given that discogenic back pain accounts for approximately 50% of prescription opioid usage in the US. Rexlemestrocel-L has been granted RMAT designation for this indication, providing benefits such as rolling review and eligibility for priority review upon BLA filing.
Financial Status
Mesoblast reported a cash balance of US$51.1 million as of September 30, 2024, with an additional US$60.0 million available from existing financing facilities upon Ryoncil approval. Net operating cash spend for the first quarter of FY2025 was US$10.5 million, a 26% reduction compared to the prior corresponding quarter.
