Mesoblast (MSB) investor relations material

Pipeline overview and program updates

• Two leading platform technologies: remestemcel-L (RYONCIL) and rexlemestrocel-L target rare and blockbuster indications, including SR-aGvHD, chronic low back pain (CLBP), heart failure, and Duchenne muscular dystrophy (DMD).

• RYONCIL is FDA-approved for pediatric SR-aGvHD, with label extension programs underway for adults and DMD.

• Rexlemestrocel-L is advancing in Phase III trials for CLBP and heart failure, targeting multi-billion dollar markets.

• Next-generation pipeline includes CAR-MSCs and OV-MSCs for autoimmune, neuroinflammatory, and oncology indications, with IND-enabling studies ongoing.

• Active label extension and new indication programs in Duchenne muscular dystrophy and chronic GvHD.

Clinical trial data and development milestones

• RYONCIL pivotal pediatric GvHD trial showed 84% survival in severe cases; strong efficacy in adults post-ruxolitinib failure; adult Phase III trial initiated with BMT CTN partnership.

• Rexlemestrocel-L Phase III CLBP trial completes enrollment this month; top-line readout expected mid-next year, BLA filing planned for Q3 next year, potential FDA approval and launch in Q2 2028.

• DREAM heart failure trial showed up to 88% reduction in MI/stroke and 52% reduction in 3-point MACE in high-inflammation subgroups.

• LVAD program demonstrated significant reduction in GI bleeding and right heart failure; orphan drug designation obtained.

• Duchenne muscular dystrophy program received IND clearance for a registrational trial in children aged 5-9, leveraging strong preclinical and safety data.

R&D strategy and innovation priorities

• Focus on multimodal anti-inflammatory mechanisms and leveraging first-in-class MSC platform for multiple high-value indications.

• Investment in next-gen gene-modified MSCs: CAR MSCs for targeted immunomodulation and oncolytic virus delivery for cancer.

• Manufacturing innovation includes automation, bioreactors, and proprietary media to increase yield and reduce COGS.

• Strategic collaborations with Mayo Clinic and Baylor for rapid, cost-effective development of advanced cell therapies.