Mesoblast (MSB) Piper Sandler 37th Annual Healthcare Conference summary

Commercial performance and market adoption

• Ryoncil launched for pediatric steroid-refractory acute graft-versus-host disease, achieving $30 million net sales in the first two quarters and expecting similar or higher sales in the current quarter.

• Achieved broad insurance coverage, including commercial, Medicaid, and CMS, with a J-code secured in October, facilitating reimbursement and repeat usage at treatment centers.

• Current market penetration is about 10%, with a target of 30% within 12 months.

• Ryoncil is the only approved product for children under 12, with early use linked to higher remission and survival rates.

Clinical development and regulatory strategy

• Planning a randomized controlled Phase III trial in adults with severe steroid-refractory acute GVHD, combining Ryoncil with ruxolitinib, targeting a market three times larger than pediatrics.

• Trial protocol is finalized, awaiting FDA sign-off, with enrollment across 80 US sites; aiming for a 2027 filing and potential label expansion.

• Parallel regulatory filings planned in Europe using FDA documentation for EMA approval.

Pipeline expansion and new indications

• Exploring Ryoncil's potential in inflammatory bowel disease and rare pediatric inflammatory diseases, leveraging strong safety data.

• Considering both investigator-initiated and company-sponsored trials, with a focus on innovation and collaboration with physicians.