Mesoblast Provides Remestemcel-L Update and Quarterly Activity Report
NEW YORK, July 30, 2020 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today provided an update on upcoming milestones for its lead product candidate remestemcel-L, and an activity report for the fourth quarter ended June 30, 2020.
Mesoblast Chief Executive Dr Silviu Itescu stated: “Remestemcel-L has two imminent major milestones, the interim analysis in the ongoing Phase 3 trial of remestemcel-L in COVID-19 patients with acute respiratory distress syndrome and the FDA advisory committee panel review of our submission for potential approval of RYONCIL™ (remestemcel-L) in children with steroid-refractory acute graft versus host disease. Together with the upcoming Phase 3 read-outs in chronic heart failure and back pain, these key milestones will take the Company into the most significant period in its history.”
Remestemcel-L Phase 3 Trial for COVID-19 Acute Respiratory Distress Syndrome
The independent Data Safety Monitoring Board (DSMB) has set a date for early September to complete the first interim analysis of the Phase 3 trial of remestemcel-L in ventilator-dependent COVID-19 patients with moderate to severe acute respiratory distress syndrome (ARDS). The trial’s first 90 patients will have completed 30 days of follow up during August, after which the DSMB will perform an interim analysis review of the safety and efficacy data. The DSMB will then inform Mesoblast on whether the trial should proceed as planned, or should stop early.
There are currently no approved treatments for COVID-19 ARDS, the primary cause of death in patients infected with COVID-19.
The clinical protocol evaluating remestemcel-L in patients in the Phase 3 trial was based on results from a pilot study using remestemcel-L under emergency compassionate care at Mt Sinai Hospital in New York, with 75% (nine of 12) of patients with moderate to severe ARDS successfully taken off a ventilator and discharged from hospital within a median of 10 days.
The placebo-controlled, double-blinded trial commenced enrollment in the reporting quarter of ventilator-dependent patients in intensive care units with moderate to severe COVID-19 ARDS randomized (1:1) to receive either two intravenous infusions of remestemcel-L three to five days apart or placebo on top of maximal care. The primary endpoint is all-cause mortality within 30 days of randomization, with the key secondary endpoint being the number of days off mechanical ventilator support.
Up to 30 leading medical centers across the United States are expected to participate in the trial, which is expected to complete recruitment in September 2020.
Remestemcel-L for Steroid-Refractory Acute Graft Versus Host Disease
The Oncologic Drugs Advisory Committee (ODAC) of the United States Food and Drug Administration (FDA) has scheduled a meeting for August 13, 2020 to review data supporting the Company’s Biologics License Application (BLA) for approval of RYONCIL in the treatment of steroid-refractory acute graft versus host disease (SR-aGVHD) in children.
There are currently no FDA-approved treatments in the United States for children under 12 with SR-aGVHD, a potentially life-threatening complication of an allogeneic bone marrow transplant for blood cancer.
Although the FDA will consider the recommendation of the panel, the final decision regarding the approval of the product is made by the FDA solely, and the recommendations by the panel are non-binding.
RYONCIL has been accepted for Priority Review by the FDA with an action date of September 30, 2020, under the Prescription Drug User Fee Act (PDUFA). If approved, RYONCIL is planned for launch in the United States in 2020 with product inventory in place.
Cash Flow Report for the Fourth Quarter FY2020
Cash on hand at the end of the quarter was US$129.3million (A$188.4 million). Mesoblast completed a US$90 million (A$138 million) capital raise from global institutional investors in May 2020. Over the next 12 months, Mesoblast may have access to an additional US$67.5 million through existing financing facilities and strategic partnerships.
Total Operating Activities resulted in net cash usage of US$19.6 million in the quarter ended June 30, 2020, as the Company continues to prepare for the potential approval and commercial launch of RYONCIL in the United States.
A copy of the Appendix 4C – Quarterly Cash Flow Report for the fourth quarter FY2020 is available on the investor page of www.mesoblast.com.
Mesoblast’s Biologics License Application to seek approval of its product candidate RYONCIL™ (remestemcel-L) for pediatric steroid-refractory acute graft versus host disease has been accepted for priority review by the United States Food and Drug Administration (FDA), and if approved, product launch in the United States is expected in 2020. Remestemcel-L is also being developed for other inflammatory diseases in children and adults including moderate to severe acute respiratory distress syndrome (ARDS). Following positive pilot trial results under compassionate care use, remestemcel-L is being evaluated in a Phase 3 randomized controlled trial in 300 patients with COVID-19 ARDS in the United States. Mesoblast is completing Phase 3 trials for its product candidates for advanced heart failure and chronic low back pain. Two products have been commercialized in Japan and Europe by Mesoblast’s licensees, and the Company has established commercial partnerships in Europe and China for certain Phase 3 assets.
Mesoblast has locations in Australia, the United States and Singapore and is listed on the Australian Securities Exchange (MSB) and on the Nasdaq (MESO). For more information, please see www.mesoblast.com, LinkedIn: Mesoblast Limited and Twitter: @Mesoblast
1.TEMCELL®1 HS Inj. is a registered trademark of JCR Pharmaceuticals Co. Ltd.
Release authorized by the Chief Executive.
For further information, please contact:
Keynotes - Lincoln Ballroom
Protecting Critical Infrastructure with Epic Security