SINGLE INTERVENTION WITH REXLEMESTROCEL-L IMPROVES LEFT VENTRICULAR EJECTION FRACTION AT 12 MONTHS, PRECEDING LONG-TERM REDUCTION IN MAJOR ADVERSE CARDIOVASCULAR EVENTS 

Mesoblast Limited today announced that treatment of HFrEF patients with rexlemestrocel-L, its allogeneic “off-the-shelf” product candidate for the treatment of chronic heart failure with reduced ejection fraction, resulted in greater improvement in the pre-specified analysis of left ventricular ejection fraction at 12 months relative to controls in the DREAM-HF Phase 3 trial. Improvement in LVEF was most pronounced in the setting of inflammation and preceded long-term reduction in the 3-point MACE of cardiovascular death, non-fatal heart attack or stroke. These results were recently highlighted in a heart failure panel discussion titled “Late-Stage Advancements in Heart Failure Therapeutics and Management.”

Rexlemestrocel-L is an immunomodulatory therapy developed to target the high degree of inflammation and resultant endothelial dysfunction present across the spectrum of HFrEF, from NYHA class II through end-stage CHF on LVADs. This MOA is postulated to improve systolic function and LVEF in HFrEF patients and reduce the high rate of major cardiovascular events and complications, notably 3-point MACE in NYHA class II/III patients and gastrointestinal (GI) tract ischemia, abnormal GI blood vessels, and life-threatening GI bleeding events in LVAD patients. Rexlemestrocel-L has already been granted FDA RMAT and Orphan Drug designations for treatment of chronic heart failure with left ventricular systolic dysfunction in patients with an LVAD.

Results from two large placebo-controlled randomized studies in patients with HFrEF, a disease associated with inflammation, a 565-patient trial in NYHA class II/III patients (DREAM-HF) and a 159patient trial in end-stage heart failure patients implanted with an LVAD, as well as in an earlier 30patient trial in LVAD patients, provide support for a common MOA for rexlemestrocel-L across the spectrum of HFrEF. New data from the DREAM-HF trial shows that a single intervention with rexlemestrocel-L resulted in improvement from baseline to 12 months in LVEF, which preceded and correlated with long-term reduction in MACE across a mean follow up of 30 months. This suggests that early improvement in LV systolic function, as measured by LVEF change from baseline to 12 months, could be an appropriate surrogate endpoint predictive of adverse long-term clinical outcomes in this patient population. 

MSB has lifted this morning on the back of the announcement, trading at 94.5 cents (up 10.53%).

Novartis has cancelled its agreement with Mesoblast. The agreement was for a planned investment and commercialisation deal that Mesoblast had said could be worth as much as $US505 million.

The share price of Mesoblast has hit a low of $1.335 this morning, which is the lowest price MSB has been since the COVID market correction of March 2020.

UPDATE ON NOVARTIS AGREEMENT

Melbourne, Australia; December 14, and New York, USA; December 13, 2021:

Mesoblast Limited (ASX:MSB; Nasdaq:MESO), global leader in allogeneic cellular medicines for inflammatory diseases, was notified today by Novartis that it has chosen to terminate the agreement with Mesoblast prior to closing. Mesoblast remains highly focused on executing on our short term objective to bring remestemcel-L to market for patients with acute respiratory distress syndrome (ARDS) due to COVID19.

The observed mortality reduction with remestemcel-L in patients aged under 65 in the completed COVID ARDS trial, despite having missed the primary endpoint, is considered by Mesoblast to be a sufficiently strong signal to support pursuing an emergency use authorization (EUA), the most direct path to market. Mesoblast is preparing to initiate a pivotal Phase 3 trial that may support a COVID ARDS EUA.

COVID-19 is likely to remain a serious global problem and to provide a major commercial opportunity for Mesoblast, with a steady state of intensive care unit (ICU) ARDS patients irrespective of vaccines and anti-viral treatments. Variants including Omicron present a growing threat due to increased infectivity and immune evasion from vaccines and monoclonal antibodies, increasing the urgent need for therapeutics to prevent the likely high mortality of those progressing to ICU and ARDS.

Well this announcement will just add to MSB's yearly interest bill. As @Noicewon11 wrote recently: I'm of the belief that MSB will never make a profit. They've been operating as a listed company for circa 17 years and haven't managed to make a cent of earnings in that time.

With so much debts owing, is this borrowing more to kick the can further down the road?

Mesoblast Limited (ASX:MSB; Nasdaq:MESO), global leader in allogeneic cellular medicines for inflammatory diseases, today announced that it has successfully refinanced its existing senior debt facility with a new US$90 million five year facility provided by funds managed by Oaktree Capital Management, L.P. (“Oaktree”).

Mesoblast drew the first tranche of US$60 million on closing, with proceeds being used to repay the outstanding balance of the existing senior debt facility with Hercules Capital, Inc. Up to an additional US$30 million may be drawn on or before December 31, 2022, subject to certain milestones. The facility has a three-year interest only period, at a rate of 9.75% per annum, after which time 40% of the principal amortizes over two years and a final payment due November 2026. Oaktree will also receive warrants to purchase 1,769,669 American Depositary Shares (ADSs)1 at US$7.26 per ADS, a 15% premium to the 30-day VWAP. The warrants may be exercised within 7 years of issuance.

“We are pleased to have leading global investment management firm Oaktree as our new financing partner as we focus on bringing our first product to the US market. Oaktree has a demonstrated partnership approach to innovative companies, making it an excellent fit to support Mesoblast’s commercial growth strategy over the next five years,” said Silviu Itescu, Chief Executive of Mesoblast.

Aman Kumar, Co-Portfolio Manager of Life Sciences Lending at Oaktree said, “We are delighted to partner with Mesoblast at this point in its development. We recognize the quality of the portfolio and the significant near-term milestones that could help the company successfully commercialize its first product in the US.” Cantor Fitzgerald & Co. acted as exclusive arranger and financial advisor to Mesoblast in this transaction.

An exciting announcement from Mesoblast, with a single dose of rexlemestrocel-L on top of standard of care, reducing the incidence of heart attacks or strokes by 79% compared with standard of care alone, p<0.0011.

In a release by the American Heart Association, Dr Perin said: “Cell therapy has the potential to change how we treat heart failure. This study addresses the inflammatory aspects of heart failure, which go mostly untreated, despite significant pharmaceutical and device therapy development.”

MSB shares have risen over 12% on the back of this news.

LATE BREAKING PRESENTATION AT AMERICAN HEART ASSOCIATION ANNUAL MEETING OF LANDMARK PHASE 3 TRIAL OF REXLEMESTROCEL-L IN CHRONIC HEART FAILURE 

Trial Results Highlighted Reduction in Cardiovascular Mortality, Heart Attacks and Strokes, with Greatest Effect Seen in Setting of Inflammation

Key findings of pre-specified outcomes were:

• A single dose of rexlemestrocel-L on top of standard of care reduced the incidence of heart attacks or strokes by 65% across all 537 NYHA class II or class III treated patients compared with standard of care alone, p=0.0011
• Across 301 NYHA class II and class III treated patients with high levels of inflammation (hsCRP ? 2mg/L), rexlemestrocel-L reduced the incidence of heart attacks or strokes by 79% compared with standard of care alone, p<0.0011
• In NYHA class II patients with high levels of inflammation (hs-CRP ? 2mg/L), a single dose of rexlemestrocel-L reduced the incidence of cardiovascular death by 80% compared with standard of care alone, p=0.0051
• Compared with standard of care alone, a single dose of rexlemestrocel-L on top of standard of care reduced the incidence of cardiovascular death, heart attacks or strokes by 33% across all 537 NYHA class II or class III patients, p=0.021, and by 45% in the 301 patients with high levels of inflammation (hs-CRP ? 2mg/L), p=0.0121
• Compared with standard of care alone, addition of rexlemestrocel-L did not further reduce the frequency of hospitalization for worsening HF symptoms as previously reported

A good result for Mesoblast, although it was a very small cohort for the study. We now wait for Mesoblast's upcoming scheduled meeting with FDA’s Office of Tissue and Advanced Therapies (OTAT).

REMESTEMCEL-L IMPROVES SURVIVAL OF CHILDREN WITH BIOMARKERS FOR HIGHEST MORTALITY IN STEROID REFRACTORY ACUTE GVHD

Mesoblast Limited (ASX:MSB; Nasdaq:MESO), global leader in allogeneic cellular medicines for inflammatory diseases, today announced that results published in the latest issue of the peer-reviewed journal Bone Marrow Transplantation1 showed that children with steroid-refractory acute graft versus host disease (SRaGVHD) and biomarkers predictive for highest mortality had 64% survival when treated with remestemcel-L compared with only 10% survival when treated with other available therapies, including ruxolitinib or other biologics.

The study’s senior author and expert in the predictive biomarkers, Dr. James Ferrara, Ward-Coleman Chair in Cancer Medicine, Professor and Director Hematologic Malignancies Translational Research Center at The Icahn School of Medicine at Mount Sinai Hospital, said: “The findings support and extend recent studies that children with severe, SR acute GVHD benefit from remestemcel-L therapy.”

These data provide further support for the proposed anti-inflammatory mechanism of action of remestemcel-L and its immunomodulatory activity in patients with SR-aGVHD, resulting in improved survival outcomes. At its upcoming scheduled meeting with FDA’s Office of Tissue and Advanced Therapies (OTAT), Mesoblast will address the appropriateness of potency assays related to remestemcel-L’s proposed anti-inflammatory mechanism of action as well as the outstanding chemistry, manufacturing and controls (CMC) items which could support a resubmission of the current Biologics License Application (BLA) for remestemcel-L in the treatment of SR-aGVHD in children

Mesoblast Operational and Financial Highlights for Quarter Ended June 30, 2021

Melbourne, Australia; July 30 and New York, USA; July 29, 2021: Mesoblast Limited (ASX:MSB; Nasdaq:MESO), global leader in allogeneic cellular medicines for inflammatory diseases, today provided an update on its pipeline of late-stage product candidates, and an activity report for the fourth quarter ended June 30, 2021. “During the quarter we made significant progress in both regulatory and clinical outcomes for our lead product candidate, remestemcel-L. FDA’s CBER has recently recommended the next steps in the potential approval pathway for remestemcel-L in the treatment of steroid-refractory acute graft versus host disease in children” said Silviu Itescu, Chief Executive of Mesoblast. “Additionally, as COVID infections continue to surge, the 90-day survival outcomes from the remestemcel-L trial in adults with COVID ARDS demonstrated the potential for durable benefit of this therapy in certain segments experiencing the most extreme complication of this disease.”

Key operational highlights:

• Mesoblast met with the United States Food & Drug Administration (FDA) on potential pathways to regulatory approval of its lead technology platform product remestemcel-L for steroid refractory acute graft versus host disease (SR-aGVHD) in children and acute respiratory distress syndrome (ARDS) in adults with COVID-19
• Regarding SR-aGVHD, FDA’s Center for Biologics Evaluation and Research (CBER) recommended that Mesoblast as a next step discuss with CBER’s review team at the Office of Tissue and Advanced Therapies (OTAT) our approach to address certain outstanding chemistry, manufacturing and controls (CMC) items, including potency assays, which could support a resubmission of the current Biologic License Application (BLA) with a six-month review period
• Regarding COVID ARDS, Mesoblast met with the FDA this week to determine the registration pathway for approval of remestemcel-L in this indication, with formal minutes expected in coming weeks
• Results from the randomized controlled trial of remestemcel-L in 222 ventilator-dependent COVID-19 patients with moderate/severe acute respiratory distress syndrome (ARDS) were recently highlighted at the biennial Stem Cells, Cell Therapies, and Bioengineering in Lung Biology and Diseases conference hosted by the University of Vermont, Burlington, VT, and at the International Society for Cell & Gene Therapy (ISCT) Scientific Signatures Series event on Cell and Gene-Based Therapies in Lung Diseases and Critical Illnesses
• The presented data included improved respiratory function in patients treated with remestemcel-L, as well as 90-day survival outcomes showing remestemcel-L significantly reduced mortality by 48% at 90 days compared to controls in a pre-specified analysis of 123 treated patients under 65 years old
• Mesoblast has entered into a license and collaboration agreement with Novartis for the development, manufacture, and commercialization of remestemcel-L, with an initial focus on the treatment of acute respiratory distress syndrome (ARDS), including that associated with COVID-19. The agreement remains subject to certain closing conditions, including time to analyze the results from this COVID-19 ARDS trial
• Mesoblast filed requests and expects to hold meetings with the FDA during the next two quarters to discuss the pathways to US regulatory approvals for its second technology platform rexlemestrocel-L following the recently completed Phase 3 trials in patients with chronic heart failure and chronic inflammatory back pain due to degenerative disc disease
• Mesoblast and its partner in Europe and Latin America, Grünenthal, amended their collaboration agreement in line with a strategy to achieve regulatory harmonization, cost efficiencies and streamlined timelines aiming to leverage the results from a planned US trial in support of potential product approvals in both US and EU

MESOBLAST PROVIDES TOPLINE RESULTS FROM PHASE 3 TRIAL OF REXLEMESTROCEL-L FOR ADVANCED CHRONIC HEART FAILURE

Melbourne, Australia; December 15, and New York, USA; December 14, 2020: Mesoblast Limited (ASX:MSB; Nasdaq:MESO), global leader in allogeneic cellular medicines for inflammatory diseases, today announced top-line results from the landmark DREAM-HF Phase 3 randomized controlled trial of its allogeneic cell therapy rexlemestrocel-L (REVASCOR®) in 537 patients with advanced chronic heart failure1.

Over a mean 30 months of follow-up, patients with advanced chronic heart failure who received a single endomyocardial treatment with rexlemestrocel-L on top of maximal therapies had 60% reduction in incidence of heart attacks or strokes and 60% reduction in death from cardiac causes when treated at an earlier stage in the progressive disease process. Despite significant reduction in the pre-specified endpoint of cardiac death, there was no reduction in recurrent non-fatal decompensated heart failure events, which was the trial’s primary endpoint. This suggests that rexlemestrocel-L reduces mortality by mechanisms that are distinct from those of existing drugs that reduce hospitalization rates but do not significantly impact cardiac mortality.

“There is an urgent need for new therapies that can reduce the high death rates in heart failure patients by different modes of action from existing drugs which reduce hospitalization rates but have not significantly reduced mortality rates,” said Mesoblast Chief Executive Dr Silviu Itescu. “The reduction in mortality seen with rexlemestrocel-L in advanced chronic heart failure underlines the power of this technology and the commitment of Mesoblast to address diseases in patients with high unmet need which are refractory to existing therapies.”

Key highlights were that a single injection of rexlemestrocel-L, on top of maximal therapy, resulted in the following pre-specified outcomes over a 30-month mean follow-up period:

• Significant reduction in the incidence of non-fatal ischemic major adverse cardiac events (MACE) due to a heart attack (myocardial infarction, MI) or stroke (cerebrovascular accident, CVA) by 60% relative to controls in the total population of 537 patients (p=0.002); reduction in MACE was seen consistently across both New York Heart Association (NYHA) class II or III populations and irrespective of whether the underlying cause of heart failure was ischemic or non-ischemic
• Significant reduction in death from all cardiac causes (CV death) in the 206 heart failure patients with NYHA class II disease by 60% relative to controls (p=0.037), which was evident in both ischemic and non-ischemic subgroups
• Prevention of NYHA class II patients progressing to CV death rates of NYHA class III patients (p=0.004); in contrast, NYHA class II patients on maximal therapy in the control group progressed to CV death rates of NYHA class III patients after a mean period of 20 months of disease stability
• Significant reduction in the composite of the pre-specified CV death or ischemic MACE outcomes in heart failure patients with NYHA class II disease by 55% relative to controls (p=0.009)

“The trial results show that rexlemestrocel-L significantly reduces cardiovascular mortality when used early in heart failure patients at risk of disease progression, and provides durable protection from heart attacks or strokes in these vulnerable patients,” said the trial’s co-principal investigator Dr Emerson Perin, Director of the Center for Clinical Research, Medical Director of Texas Heart Institute, and Clinical Professor, Baylor College of Medicine. “New therapies have not materially reduced the high death rates from cardiovascular disease which is why these data have the potential to change the treatment paradigm for patients with advanced chronic heart failure.”

Mesoblast Chief Medical Officer Dr Fred Grossman said: “We expect the mortality benefit observed in this seminal Phase 3 trial will support a potential path for approval of rexlemestrocel-L in patients with advanced chronic heart failure. We are planning to meet and discuss potential pathways to approval based on mortality reduction with the United States Food and Drug Administration.”

MESOBLAST ENTERS GLOBAL COLLABORATION FOR DEVELOPMENT, MANUFACTURE AND COMMERCIALIZATION OF REMESTEMCEL-L

Key transaction terms:

• Novartis will make a US$50 million upfront payment including US$25 million in equity.
• From the initiation of a Phase 3 trial in all-cause ARDS, Novartis will fully fund global clinical development for all-cause ARDS and potentially other respiratory indications.
• Mesoblast may receive a total of US$505 million pending achievement of precommercialization milestones for ARDS indications.
• Mesoblast may receive additional payments post-commercialization of up to US$750 million based on achieving certain sales milestones and tiered double-digit royalties on product sales.
• Mesoblast will retain full rights and economics for remestemcel-L for graft versus host disease (GVHD), and Novartis has an option to, if exercised, become the commercial distributor outside of Japan.
• For most non-respiratory indications, the parties may co-fund development and commercialization on a 50:50 profit-share basis.
• Mesoblast will be responsible for clinical and commercial manufacturing and Novartis will purchase commercial product under agreed pricing terms. Novartis will reimburse Mesoblast up to US$50 million on the achievement of certain milestones related to the successful implementation of its next-generation manufacturing processes using its proprietary media and three-dimensional bioreactors aimed at delivering substantial manufacturing efficiencies. Novartis will be responsible for any capital expenditure required to meet increased capacity requirements for manufacture of remestemcel-L.

MESOBLAST WINS 2020 FIERCE BIOTECH INNOVATION OF THE YEAR AWARD FOR REMESTEMCEL-L

Melbourne, Australia; September 15 and New York; USA; September 14, 2020: Mesoblast Limited (ASX:MSB; Nasdaq:MESO), global leader in allogeneic cellular medicines for inflammatory diseases, today announced that its lead product candidate remestemcel-L has been selected as the winner of the Fierce Innovation Awards - Life Sciences Edition 2020 for Biotech Innovation. The Fierce Innovation Awards is a peer-reviewed program from the publisher of FierceBiotech and FiercePharma.

Mesoblast Chief Executive Dr Silviu Itescu stated: “This important award is recognition of Mesoblast’s leadership as an innovator in the cell therapy industry, and of the potential for remestemcel-L to profoundly impact the lives of children suffering with steroid-refractory acute graft versus host disease (SR-aGVHD).”

Remestemcel-L is under priority review by the United States Food and Drug Administration (FDA) for pediatric SR-aGVHD and, if approved, product launch in the United States is expected in 2020. The FDA has set a Prescription Drug User Fee Act (PDUFA) action date of September 30, 2020.

Remestemcel-L is an investigational therapy comprising culture-expanded mesenchymal stem cells derived from the bone marrow of an unrelated donor. It is thought to have immunomodulatory properties to counteract the cytokine storms that are implicated in various inflammatory conditions by down-regulating the production of pro-inflammatory cytokines, increasing production of antiinflammatory cytokines, and enabling recruitment of naturally occurring anti-inflammatory cells to involved tissues.

Given the extensive inflammatory response in COVID-19 infection, remestemcel-L is also being evaluated in a randomized, controlled Phase 3 trial in up to 300 ventilator-dependent adults with moderate to severe acute respiratory distress syndrome (ARDS), the primary cause of mortality in COVID-19 patients. The trial aims to confirm results from a pilot study at New York’s Mt Sinai hospital which showed that nine of 12 patients (75%) were successfully discharged from hospital a median of 10 days after receiving two intravenous doses of remestemcel-L within five days. The trial’s independent Data Safety Monitoring Board (DSMB) recently completed an interim analysis of the trial’s first 30% enrolled patients and recommended that the trial should continue as planned after reviewing all safety data and results for the trial’s primary endpoint of all-cause mortality within 30 days of randomization. The DSMB will perform a second interim analysis when 45% of the enrollment target has completed 30 days of follow-up.

MESOBLAST REPORTS SUBSTANTIAL OPERATIONAL PROGRESS AND FINANCIAL RESULTS FOR THE YEAR ENDED JUNE 30, 2020

Financial Highlights

• 92% increase in revenues to US$32.2 million for FY2020, compared with US$16.7 million for FY2019. o 127% increase in milestone revenue from strategic partnerships, to US$25.0 million for FY2020. o 32% increase in royalty revenue on TEMCELL® HS. Inj.1 sales in Japan by licensee JCR Pharmaceuticals (JCR), to US$6.6 million for FY2020.
• 13% reduction in loss after tax (US$77.9 million for FY2020 compared with US$89.8 million for FY2019), even after US$13.8 million increased investment in commercial readiness for the potential US launch of RYONCIL (US$8.8 million for commercial manufacturing activities and US$5.0 million for sales/marketing). 
• US$129.3 million (A$188.4 million)2 cash on hand at June 30, 2020, after US$90 million (A$138 million)3 capital raise from global institutional investors in May 2020.
• May have access to up to an additional US$67.5 million over the next 12 months through existing financing facilities and strategic partnerships.

U.S. FDA ADVISORY COMMITTEE VOTES NINE TO ONE IN FAVOR OF REMESTEMCEL-L (RYONCIL™) FOR EFFICACY IN CHILDREN WITH STEROIDREFRACTORY ACUTE GRAFT VERSUS HOST DISEASE

Mesoblast Limited (ASX:MSB; Nasdaq: MESO), global leader in cellular medicines for inflammatory diseases, today announced that the Oncologic Drugs Advisory Committee (ODAC) of the United States Food and Drug Administration (FDA) voted overwhelmingly in favor that the available data support the efficacy of remestemcel-L (RYONCIL™) in pediatric patients with steroid-refractory acute graft versus host disease (SR-aGVHD).

Mesoblast Chief Medical Officer Dr Fred Grossman said: “Steroid-refractory acute graft versus host disease is an area of extreme need, especially in vulnerable children under 12 years old where there is no approved therapy. We are very encouraged by today’s outcome and are committed to working closely with the FDA as they complete their review of our submission regarding approval of RYONCIL for this life-threatening complication of an allogeneic bone marrow transplant.”

The ODAC is an independent panel of experts that evaluates efficacy and safety of data and makes appropriate recommendations to the FDA. Although the FDA will consider the recommendation of the panel, the final decision regarding the approval of the product is made solely by the FDA, 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 by the PDUFA date, Mesoblast plans to launch RYONCIL in the United States in 2020.

Pediatric transplant physician Dr Joanne Kurtzberg, the Jerome Harris Distinguished Professor of Pediatrics and Professor of Pathology, and Director, Pediatric Blood and Marrow Transplant Program at Duke University Medical Center, said: “This devastating condition has an extremely poor prognosis and there are no FDA-approved options for children under the age of 12. The clinical studies I have directed have demonstrated the potential for this treatment to fill a significant unmet medical need.”

Request for trading halt for Mesoblast Limited (ASX: MSB)

Pursuant to ASX Listing Rule 17.1, Mesoblast Limited ACN 109 431 870 (ASX: MSB; NASDAQ: MESO) (the Company) requests a trading halt in its securities effective immediately pending an announcement by the Company in relation to the upcoming meeting of the Oncologic Drugs Advisory Committee of the United States Food and Drug Administration.

For the purposes of Listing Rule 17.1, the Company provides the following information:

(a) the trading halt is requested pending an announcement in relation to the matters above;

(b) the Company requests that the trading halt continues until it makes an announcement regarding the matters above which is expected to be on Friday, 14 August 2020; and

(c) the Company is not aware of any reason why the trading halt should not be granted or of any other information necessary to inform the market about the trading halt.

UPDATE ON SCHEDULED FDA ADVISORY COMMITTEE MEETING

Melbourne, Australia; August 11, 2020; and New York, USA; August 10, 2020: Mesoblast Limited (ASX:MSB; Nasdaq:MESO) today provided an update on the scheduled meeting of the Oncologic Drugs Advisory Committee (ODAC) of the United States Food and Drug Administration (FDA) which will review data supporting the Company’s Biologics License Application (BLA) for approval of RYONCIL™ (remestemcel-L) 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.

The meeting is scheduled to take place on August 13, 2020 from 8am to 5pm ET. The ODAC will vote in the afternoon session on whether the available data support the efficacy of remestemcel-L in pediatric patients with SR-aGVHD. This session will discuss the Phase 3 trial results and supporting clinical data included in the BLA. The morning session will be non-voting and will discuss issues related to the characterization and critical quality attributes of remestemcel-L.

Mesoblast has extensively prepared for this meeting and has provided a publicly available briefing book. Briefing materials and webcast information have been made publicly available and can be found on the FDA website at: https://www.fda.gov/advisory-committees/advisory-committeecalendar/august-13-2020-meeting-oncologic-drugs-advisory-committee-meeting-announcement08132020-08132020#event-materials

The ODAC is an independent panel of experts that provides advice and appropriate recommendations to the FDA based on potential issues highlighted by the FDA during their review of the efficacy and safety of marketed and investigational products for use in the treatment of cancer. Although the FDA will consider the recommendation of the advisory committee, 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 is under Priority Review by the FDA with an action date of September 30, 2020, under the Prescription Drug User Fee Act (PDUFA).

FDA ADVISORY COMMITTEE SETS REVIEW DATE FOR MESOBLAST’S REMESTEMCEL-L IN PEDIATRIC STEROID-REFRACTORY ACUTE GRAFT VERSUS HOST DISEASE

Mesoblast Limited (ASX:MSB; Nasdaq:MESO) today announced that the Oncologic Drugs Advisory Committee (ODAC) of the United States Food and Drug Administration (FDA) has scheduled a meeting on August 13, 2020 to review data supporting the Company’s Biologics License Application (BLA) for approval of RYONCIL™ (remestemcel-L) for 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 SRaGVHD, a potentially life-threatening complication of an allogeneic bone marrow transplant for blood cancer. RYONCIL is under Priority Review by the FDA with an action date of September 30, 2020, under the Prescription Drug User Fee Act (PDUFA).

The ODAC is an independent panel of experts that provides advice and appropriate recommendations to the FDA based on potential issues highlighted by the FDA during their review of the efficacy and safety of marketed and investigational products for use in the treatment of cancer. The ODAC review will comprise two separate sessions: a morning session which will discuss issues related to the characterization and critical quality attributes of remestemcel-L as they relate to clinical effectiveness, and an afternoon session which will discuss results from clinical trials included in the BLA.

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 nonbinding.