Mesoblast (MSB) Maxim Group’s 2024 Healthcare Virtual Summit summary

Industry context and regulatory environment

• Regenerative medicine and cell therapy are at a pivotal moment, with significant attention on upcoming regulatory decisions.

• The FDA has shown increased flexibility and maturity in evaluating first-in-class therapies, especially for life-threatening conditions.

• Recent FDA meetings led to acceptance of pediatric data for filing without requiring an additional trial, reflecting evolving regulatory standards.

• The field is moving toward harmonized standards similar to those for small molecules and biologics.

• Companies with robust clinical data and safety profiles are now better positioned for approval.

Clinical development and trial outcomes

• Allogeneic cell therapy targets severe inflammatory diseases, focusing on rapid inflammation control to enable tissue repair.

• Phase III trial in pediatric GVHD met primary and secondary endpoints, with a 9-1 FDA advisory panel vote supporting efficacy.

• Expanded access data in adults refractory to ruxolitinib show 60%-70% survival at day 100, compared to 25% with standard care.

• Pediatric GVHD remains an area of complete unmet need, with no approved therapies and high mortality rates.

• Consistency and potency assays were key to regulatory progress, ensuring commercial product matches clinical trial results.

Near-term and future regulatory milestones

• PDUFA date for pediatric GVHD is set for January 7th, with commercial launch preparations underway.

• Post-approval plans include expanding to adult GVHD, where a significant unmet need persists.

• Accelerated approval filings in heart failure are planned, supported by strong signals in inflamed subgroups.

• Pediatric congenital heart disease program aims for FDA meetings and potential filing, leveraging orphan drug status.

• A second confirmatory phase III trial in inflammatory back pain is ongoing, with readout expected after enrollment completes in about two years.