MSB to dispute FDA finding in Type A meeting, page-74

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Cantor Fitzgerald Equity Research October 1, 2020 Mesoblast Ltd. ADR (MESO - $18.59, Overweight, Target: $23.00)

Still Hopeful For Accelerated Approval Post CRL

Quick Take

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Takeaways

Today (10/1) MESO announced and held a conference call to discuss the U.S. FDA's Complete Response Letter (CRL) to its Biologics License Application (BLA) for remestemcel-L for the treatment of pediatric steroid-refractory acute graft versus host disease (SR-aGVHD). Since there are no currently approved treatments for this life-threatening condition in children under 12, MESO will urgently request a Type A meeting with the FDA, expected within 30 days (~early November), to discuss a potential accelerated approval with a post-approval condition for an additional study. According to MESO there are precedent examples of the FDA agreeing to an accelerated approval under similar circumstances. While the Oncologic Drugs Advisory Committee (ODAC) of the FDA voted 9:1 that the available data support the efficacy of remestemcel-L in pediatric patients with SR-aGVHD, the FDA recommended that MESO conduct at least one additional randomized, controlled study in adults and/or children to provide further evidence of the effectiveness of remestemcel-L for SR-aGVHD. MESO noted that it is looking at an additional adult trial, since it would be hard to do a randomized study in children. Physicians are reluctant to not give study drug to children. On a positive note, MESO is currently conducting a randomized, controlled Phase 3 trial evaluating remestemcel-L in up to 300 ventilator-dependent adults with moderate to severe acute respiratory distress syndrome (ARDS) due to COVID-19. A second interim analysis by the trial’s independent Data Safety Monitoring Board is expected in early November, with completion of patient enrollment expected in December. COVID-19 ARDS is an inflammatory disease with a similar profile of damaging inflammatory cytokines as is seen in children with SR-aGVHD, and is the primary cause of death in COVID-19 infection. The trial’s primary endpoint is reduction of all-cause mortality within 30 days of randomization. Finally, we are still expecting MESO to potentially report positive data from its chronic low back pain trial and its heart failure trial before year-`end.