Mesoblast Limited (Nasdaq:MESO; ASX:MSB) is awaiting a decision from the United States Food and Drug Administration (FDA) regarding the approval of 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
The potential approval of Ryoncil addresses a critical unmet need, as there are currently no approved treatments for children under 12 years of age with SR-aGvHD. This vulnerable population urgently requires safe and effective therapeutic options. 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 the issuance of any Form 483. Mesoblast has manufactured sufficient inventory and established a supply chain to ensure immediate availability of cryopreserved product upon approval, with the capacity to scale up production as needed.
To prepare for a successful launch, Mesoblast has been strategically hiring senior personnel to implement a targeted commercial strategy. The initial focus will be on centers with the most experience using RYONCIL and the highest patient volume, followed by a staged rollout to other centers.
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.
Published results from a blinded, randomized, placebo-controlled trial in the Journal of Thoracic and Cardiovascular Surgery Open demonstrated that a single intramyocardial administration of REVASCOR at the time of staged surgery led to significantly larger 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.
Mesoblast plans to meet with the FDA to discuss clinical data supporting regulatory approval for REVASCOR in children with HLHS.
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 in patients with end-stage HFrEF and LVADs, and in advanced NYHA class II/III HFrEF patients.
Mesoblast has received Regenerative Medicine Advanced Therapy (RMAT) designation for rexlemestrocel-L in the treatment of end-stage heart failure in LVAD patients. The company intends to meet with the FDA to discuss data presentation, timing, and FDA expectations for an accelerated approval filing.
Rexlemestrocel-L for Chronic Low Back Pain
A 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 has commenced enrollment across multiple sites in the United States. The FDA has agreed on the design of this 300-patient randomized, placebo-controlled trial, with a 12-month primary endpoint of pain reduction.
Financial Position
Mesoblast's cash balance as of September 30, 2024, was US$51.1 million, 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.
