Operational and Financial Results for the Period Ended September 30, 2020
NEW YORK, Nov. 19, 2020 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today reported operational and financial results for the first quarter ended September 30, 2020 (FY2021).
Mesoblast has entered into an exclusive worldwide license and collaboration agreement with Novartis for the development, manufacture and commercialization of its lead mesenchymal stromal cell (MSC) product candidate, remestemcel-L, with an initial focus on the treatment of acute respiratory distress syndrome (ARDS), including that associated with COVID-19. Details of this transaction have been lodged in a separate announcement with the ASX and Nasdaq today.
As foreshadowed following the capital raising in May 2020, Mesoblast increased its investment in both clinical development and manufacturing to facilitate the potential availability of remestemcel-L for patients with COVID-19. These activities led to the strategic collaboration with Novartis which will open new opportunities in respiratory indications. This collaboration will also provide the commercial and manufacturing strength to bring this important cellular medicine to the many patients with COVID-19 and its life-threatening complication of ARDS.
Cash on hand at September 30, 2020 was US$108.1 million (A$152.1 million)1 and, on transaction closing,2 pro-forma cash on hand is US$158.1 million (A$222.4 million).
Remestemcel-L for COVID-19 Complications in Adults and Children
As cases of COVID-19 surge in the United States and globally, deaths continue to increase from ARDS in ventilator-dependent patients as a result of an overactive immune response in the lungs to COVID-19. Remestemcel-L is being evaluated for its potential to reduce 30-day mortality on top of maximal care in a Phase 3 randomized controlled trial of up to 300 ventilator-dependent adults with moderate or severe COVID-19 ARDS. The key secondary endpoint is the number of days off mechanical ventilator support.
After two prior interim analyses reviewing safety and efficacy data, the trial’s independent Data Safety Monitoring Board (DSMB) recommended trial continuation as planned. Trial enrollment has now surpassed 180 patients.
The trial aims to confirm pilot data where nine of 12 (75%) ventilator-dependent adult patients with COVID-19 ARDS who received two doses of remestemcel-L under emergency compassionate use at New York’s Mt Sinai Hospital were successfully discharged within a median of 10 days.
Children hospitalized with COVID-19 infection are at risk of a life-threatening inflammatory condition called multi-system inflammatory syndrome (MIS-C) which involves multiple critical organs and their vasculature, is associated with COVID-19 antibodies, and is thought to be a post-viral autoimmune process. In approximately 50% of cases this inflammation is associated with significant cardiovascular complications resulting in decreased heart function and dilation of coronary arteries.3-5
Mesoblast’s existing Investigational New Drug (IND) application provides physicians with access to use remestemcel-L in COVID-19 infected children aged between two months and 17 years with MIS-C. 6 Two COVID-19 infected children with MIS-C who received remestemcel-L for severe heart failure fully recovered heart function and were discharged within 30 hours of the second dose.
Remestemcel-L for Steroid-Refractory Acute Graft Versus Host Disease
As part of the broad license and collaboration agreement with Novartis for remestemcel-L, Mesoblast will retain full rights and economics for graft versus host disease (GVHD).
On August 13, 2020, results from 309 children with steroid-refractory acute graft versus host disease (SR-aGVHD) treated with remestemcel-L were presented to the Oncologic Drugs Advisory Committee (ODAC) of the United States Food and Drug Administration (FDA). The ODAC panel voted 9:1 that the available data support the efficacy of remestemcel-L in pediatric patients with SR-aGVHD7. Despite the overwhelming ODAC vote, on September 30, the FDA provided Mesoblast with a Complete Response Letter.
On November 17, a Type A meeting was held with the FDA to discuss the review of the Biologics License Application for remestemcel-L and a potential pathway for accelerated approval with a post-approval requirement to conduct an additional randomized controlled study in patients 12 years and older. At the current time it appears that the FDA review team will not agree to accelerated approval. However, the definitive outcome of the Type A meeting will not be known until Mesoblast receives the formal minutes which are expected within 30 days of the meeting. If the current review team does not agree to accelerated approval, Mesoblast will request a further Type A meeting to initiate the well-established FDA dispute resolution pathway.
Under the terms of the license and collaboration agreement, Novartis has an option to become the commercial distributor for remestemcel-L in SR-aGVHD outside of Japan.
Remestemcel-L for Inflammatory Bowel Disease
A randomized, controlled study of remestemcel-L delivered by an endoscope directly to the areas of inflammation and tissue injury in up to 48 patients with medically refractory Crohn’s or ulcerative colitis has commenced at Cleveland Clinic. Mesoblast’s objective is to confirm the potential for remestemcel-L to induce luminal healing and early remission in a wider spectrum of diseases with severe inflammation of the gut, in addition to SR-aGVHD.
Despite recent advances in the treatment of inflammatory bowel disease, approximately 30% of patients are primarily unresponsive to anti-TNFa agents and even among responders, up to 10% will lose their response to the drug every year. Up to 80% of patients with medically-refractory Crohn’s disease eventually require surgical treatment of their disease,8 which can have a devastating impact on quality of life. This investigator-initiated study is the first in humans using local cell delivery directly into the gut, and will enable Mesoblast to compare clinical outcomes using this delivery method with results from an ongoing randomized, placebo-controlled trial in patients with biologic-refractory Crohn’s disease where remestemcel-L was administered intravenously.
Rexlemestrocel (REVASCOR®) for Advanced Chronic Heart Failure
In the United States alone, of more than 6.5 million patients with chronic heart failure, there are more than 1.3 million patients with advanced stage of the disease.9 The objective of treatment with Mesoblast’s allogeneic cellular product candidate REVASCOR® is to reduce or reverse the severe inflammatory process in the damaged heart of these patients, and thereby prevent or delay further progression of heart failure or death.
Mesoblast’s randomized placebo-controlled Phase 3 trial in 566 patients with advanced forms of New York Heart Association Class II or Class III disease has completed patient follow-up and all events have been independently adjudicated. While the COVID-19 pandemic has delayed completion of data quality review at the study sites, the Phase 3 trial data readout is expected in the current quarter.
In an earlier randomized placebo-controlled 60-patient Phase 2 trial, a single intra-myocardial injection of REVASCOR at the dose administered in the subsequent Phase 3 trial prevented any hospitalizations or deaths over three years of follow-up in patients with advanced chronic heart failure.
Rexlemestrocel (MPC-06-ID) for Chronic Low Back Pain
Mesoblast’s MPC-06-ID development program targets over 3.2 million patients in the United States and 4 million in the E.U.5 with chronic low back pain due to moderate to severe inflammatory disc degeneration.10 There is a significant need for a safe, efficacious and durable treatment in patients with chronic low back pain due to severely inflamed degenerative disc disease.
While the COVID-19 pandemic has delayed completion of data quality review at the study sites, data readout for the 2:1 randomized placebo-controlled US Phase 3 trial in 404 patients is expected in the current quarter.
Mesoblast continues to collaborate closely with Grünenthal on the clinical protocol for a confirmatory Phase 3 trial in Europe for MPC-06-ID in chronic low back pain due to degenerative disc disease, with the results of this and the United States Phase 3 trial expected to support both FDA and European Medicines Agency regulatory approvals.
Financial Results for the First Quarter Ended September 30, 2020 (FY2021)
As a result of the above, loss after tax for the first quarter FY2021 was US$24.5 million compared to US$5.5 million for first quarter FY2020. The net loss attributable to ordinary shareholders was 4.21 US cents per share for first quarter FY2021, compared with 1.10 US cents per share for first quarter FY2020.
Conference Call Details
The archived webcast will be available on the Investor page of the Company’s website: www.mesoblast.com
Remestemcel-L is being developed for inflammatory diseases in children and adults including steroid-refractory acute graft versus host disease and moderate to severe acute respiratory distress syndrome. 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
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