Mesoblast Ltd. Q4 FY2024 earnings call

• Made significant progress with FDA on three lead products, positioning for first potential product approval. - Clinical pipeline details: RYONCIL BLA resubmitted with ongoing FDA interactions, rexlemestrocel has Phase 3 trials in back pain and heart failure. - Financial highlights: Reduced net operating cash burn, cash balance of $63.3M, and payroll cost containment through salary reductions and deferrals of STI and director fees. - Cost control: CEO, CMO, and management voluntarily reduced base salaries, with non-cash LTIs offered as alternatives, and STI and director fees deferred until FDA BLA approval.

Mesoblast has three key product segments. The lead product RYONCIL is for pediatric steroid-refractory acute graft-versus-host disease, with its BLA resubmitted and a PDUFA date of January 7, 2025. Rexlemestrocel has programs in chronic low back pain (with one completed Phase 3 trial and a second confirmatory Phase 3 trial actively enrolling) and heart failure (REVASCOR for pediatric congenital heart disease, adult end stage heart failure, and adult ischemic heart failure). Financially, cash balance was US$63.3 million at June 30, net operating cash usage reduced by 23% in FY 2024 compared to FY 2023, and payroll cost containment initiatives were implemented.

• RYONCIL has a PDUFA date of January 7, 2025, and plans to initiate a study in adult patients for label extension post-approval. - Rexlemestrocel's chronic low back pain Phase 3 trial is actively enrolling. - REVASCOR has pathways to accelerated approval for pediatric congenital heart disease and adult heart failure, with meetings planned with FDA to discuss filings and confirmatory studies.

• Potential FDA approval delays for RYONCIL or other products. - Uncertainty in clinical trial outcomes for Phase 3 trials. - Challenges in reimbursement for products despite positive clinical data.

Q: Can you discuss any potential partnerships or collaboration that could accelerate your commercialization effort? And how are you planning on tackling reimbursement challenges, assuming you get approval for RYONCIL?

A: For back pain product, has a partner in Europe (Grunenthal) for market access, pricing, and distribution. In US, will seek similar commercialization partner. Reimbursement for RYONCIL is based on precedents from CAR T therapies and gene therapy, considering the orphan population size and curative outcomes.

Q: When should we expect an update with respect to REVASCOR and kind of the regulatory filing plans. And how long do you think the Phase 3 for the spinal product will take?

A: Spinal product Phase 3 expected to enroll over approximately 12 months. For REVASCOR, will meet with FDA to discuss pediatric indication based on rare disease voucher designation and then discuss adult approval pathway based on totality of data from trials.