Mesoblast (US:MESO)2025-02-28 16:38Financial Data and Key Metrics Changes - The cash balance at December 31, 2024, was USD 38 million, with pro forma cash of approximately USD 200 million after a global private placement that raised USD 161 million [8] - Net operating cash spend was USD 20.7 million for the first half of FY 2025, representing a 22% reduction compared to the first half of FY 2024 [8] - The company reported a loss after tax of USD 47.9 million for the half year, with total operating cash flows of USD 20.7 million, a reduction of USD 5.9 million from the previous half year [13] Business Line Data and Key Metrics Changes - RYONCIL, the first FDA-approved mesenchymal stromal cell therapy, is now available for children with steroid-refractory acute graft-versus-host disease [15][25] - The FDA approval led to a reversal of a $23 million provision against inventory, now recognized as an inventory asset on the balance sheet [9][12] - The overall response rate for RYONCIL in the Phase 3 trial was 70% at day 28, significantly higher than other therapies for the same condition [17] Market Data and Key Metrics Changes - The addressable market in the US for RYONCIL is approximately 375 new children per year with life-threatening steroid-refractory acute graft-versus-host disease [16] - The cost of treating a child who dies within 12 months of a transplant due to steroid-refractory GVHD is approximately $2.5 million, which is $1.8 million higher than for those who survive [19] - The company is targeting 45 key transplant centers that account for 80% of patients [25][60] Company Strategy and Development Direction - The company aims to expand RYONCIL's label to include inflammatory bowel disease, targeting both children and adults [27] - There is a strategy to establish RYONCIL for adult patients with GVHD, particularly those who fail ruxolitinib, which has no approved therapies [31] - The company is preparing for accelerated approval for Revascor in adults with heart failure with low ejection fraction [49] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the rapid delivery of RYONCIL to children in need and emphasized the extensive efforts across all company levels to ensure a successful launch [86] - The management is confident in the positive feedback from payers regarding RYONCIL's pricing and its clinical efficacy [80] - Future meetings with the FDA are anticipated to clarify the pathway for accelerated approval for heart failure treatments [82] Other Important Information - The company has a scalable manufacturing capability that has been FDA inspected, allowing it to meet global commercial needs [3] - The company has over 1,000 patents and patent applications supporting its products [2] Q&A Session Summary Question: How large is the sales force for RYONCIL and how many centers are trained? - The sales force consists of nine key account managers focusing on 45 key transplant centers, which represent 80% of the potential market. Onboarding has already started [60][61] Question: How far along is the enrollment for the chronic lower back pain trial? - Currently, there are approximately 15 centers onboard, with plans to increase to about 40. The company expects to ramp up enrollment significantly in the coming weeks [68] Question: What feedback has been received from payers regarding RYONCIL's pricing? - Feedback has been positive, with an appreciation for the low number of affected children and the long-term survival benefits RYONCIL offers [80]