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MESOBLAST OUTLINES POTENTIAL PATHWAY TO ACCELERATED MARKET ENTRY FOR MPC-150-IM IN ADVANCED CHRONIC HEART FAILURE
New York, USA; and Melbourne, Australia; August 23, 2017: Mesoblast Limited (ASX:MSB; Nasdaq: MESO) today announced plans to achieve an accelerated market entry of the Company's proprietary allogeneic mesenchymal precursor cell (MPC) product candidate MPC-150-IM in the treatment of patients with the most advanced stages of chronic heart failure (CHF), defined as New York Heart Association (NYHA) stages Class III and Class IV.
Patients with NYHA Class III/Class IV experience high mortality rates, recurrent hospitalizations, and incur substantial cost of care despite maximal existing therapies. We believe that under new regulatory frameworks that recognize the serious and life-threatening nature of advanced CHF, positive results from our ongoing Phase 2b/3 trials in these patients could support accelerated approval for MPC-150-IM and an opportunity to create a paradigm shift in this potential multi-billion dollar market.
MPC-150-IM is being evaluated in two ongoing randomized placebo-controlled Phase 2b/3 trials in patients with either severe or end-stage advanced CHF. The mechanism of action (MOA) by which MPC-150-IM is thought to exert its effects in these patient populations is through immunomodulation and cardiac repair. Positive clinical signals supporting a common underlying MOA have been previously published in Phase 2 trials of Mesoblast's allogeneic MPC therapy in moderate/severe and end-stage heart failure.[1,2]
Specifically, the ongoing Phase 2b/3 trials in advanced CHF are:
The primary efficacy endpoint of the study is the number of temporary weans from LVAD tolerated over the 6 months post-randomization, indicating strengthening of the native heart muscle. Additional efficacy endpoints include patient survival, adverse events and rehospitalization rates over 12 months.
This trial is expected to complete enrollment shortly, with top-line results for the trial's primary endpoint expected in Q1 2018.
More than 400 of the anticipated approximately 600 NYHA Class II/III CHF patients have been randomized to date. The trial's primary efficacy endpoint is a comparison of recurrent non-fatal HF-related major adverse cardiac events (HF-MACE) between either MPC-treated patients or sham-treated controls.
In April 2017, Mesoblast announced that a pre-specified interim futility analysis of the efficacy endpoint in this Phase 3 trial was successful in the trial's first 270 patients. After notifying the Company of the interim analysis results, the trial's Independent Data Monitoring Committee stated that it had no safety concerns relating to MPC-150-IM and recommended that the trial should continue as planned. We believe that positive results from this Phase 3 trial in advanced CHF patients would serve to confirm results with MPC-150-IM obtained in end-stage heart failure patients.
About Advanced Chronic Heart Failure (CHF)
CHF is a progressive disease and is classified in relation to the severity of the symptoms experienced by the patient. The most commonly used classification system was established by the NYHA and ranges from Class I-II (mild to moderate) to Class III/IV (severe to end-stage). In 2016, more than 15 million patients in the seven major global pharmaceutical markets were estimated to have been diagnosed with CHF.[3] Prevalence is expected to grow 46% by 2030 in the United States alone, affecting more than 8 million Americans.[4] Approximately half of people who develop heart failure die within 5 years of diagnosis.[5][6] Patients with advanced CHF (NYHA Class III or Class IV) have the highest burden of disease, recurrent hospitalizations and mortality. In the United States alone, the NYHA Class III patient population is estimated at 1.3 million patients and the NYHA class IV population at 250,000 patients. There are approximately 50,000 patients with end-stage class IV heart failure who, despite optimal medical therapy, have a one-year mortality exceeding 50%.[7] The only options to increase survival in these patients are the use of LVADs or of heart transplants, the latter limited by donor availability to less than 3000 patients annually.[8] CHF causes severe economic, social, and personal costs. In the United States, it is estimated that CHF results in direct costs of $60.2 billion annually when identified as a primary diagnosis and $115 billion as part of a disease milieu.9
1 Perin EC, Borow KM, Silva GV, et al. A phase II dose-escalation study of allogeneic mesenchymal precursor cells in patients with ischemic or non-ischemic heart failure. Circulation Research. July 2015.
2 Ascheim DD, Gelijns AC, Goldstein D, et al. Mesenchymal Precursor Cells as adjunctive therapy in recipients of contemporary LVADs. Circulation. 2014;129:2287-2296.
3 PharmaPoint: Heart Failure - Global Drug Forecast and Market Analysis to 2025.
4 GlobalData-PharmaPoint (2016): Heart Failure-Global Drug Forecast and Market Analysis to 2025.
5AHA Statistical Update - Heart Disease and Stroke Statistics-(2017). Circulation. 2017;131.
6 A Re-Evaluation of the Costs of Heart Failure and its Implications for Allocation of Health Resources in the United States. Voigt J. Clinl.Cardiol. 37, 5, 312-321 (2014).
7Gustafsson G, Rogers J. (2017) Left ventricular assist device therapy in advanced heart failure: patient selection and outcomes. European Journal of Heart Failure 19, 595-602.
8Agency for Healthcare Research and Quality: HCUPnet: ICD-9 principal procedure code 27.51 2014.
9A Re-Evaluation of the Costs of Heart Failure and its Implications for Allocation of Health Resources in the United States. Voigt J. Clinl.Cardiol. 37, 5, 312-321 (2014).
Mesoblast Limited (ASX:MSB; Nasdaq:MESO) is a global leader in developing innovative cell-based medicines. The Company has leveraged its proprietary technology platform, which is based on specialized cells known as mesenchymal lineage adult stem cells, to establish a broad portfolio of late-stage product candidates. Mesoblast's allogeneic, off-the-shelf' cell product candidates target advanced stages of diseases with high, unmet medical needs including cardiovascular conditions, orthopedic disorders, immunologic and inflammatory disorders and oncologic/hematologic conditions.
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