United States Congress Legislates New Accelerated Approval Pathway for Regenerative Advanced Therapies

NEW YORK and MELBOURNE, Australia, Dec. 09, 2016 -- Mesoblast Limited (Nasdaq:MESO) (ASX:MSB) today welcomed the passage of a Bill approving the 21^st Century Cures Act by the United States Congress which provides an accelerated approval pathway for cell-based medicines designated as regenerative advanced therapies.

Chief Executive Silviu Itescu said that Mesoblast believes a number of its cell therapy product candidates meet the Act’s criteria for regenerative advanced therapies based on their intended use to treat, modify, reverse, or cure a serious or life-threatening disease or condition.

"We will work closely with the U.S. Food and Drug Administration on the appropriate regulatory pathways for our product candidates that could meet the Act’s criteria for the new regenerative advanced therapy designation. 

“We believe our regenerative medicine technology platform may be particularly effective in the most serious segments of major diseases, including the life-threatening complications of chronic advanced heart failure. Over 300 patients have now been enrolled in our Phase 3 trial of MPC-150-IM in chronic advanced heart failure. We look forward to the upcoming interim analysis of the Phase 3 trial's primary endpoint," Dr Itescu added.

Designated regenerative advanced therapies will be eligible for priority review and accelerated approval through, as appropriate, surrogate or intermediate endpoints reasonably likely to predict long term clinical benefit, or reliance upon data obtained from a meaningful number of sites.

For regenerative advanced products receiving accelerated approval, the post approval requirements may include the submission of clinical evidence, clinical studies, patient registries, or other sources of real world evidence, such as electronic health records, or the collection of larger confirmatory data sets.

About Mesoblast
Mesoblast Limited (Nasdaq:MESO) (ASX:MSB) 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 diseases, immune-mediated and inflammatory disorders, orthopedic disorders, and oncologic/hematologic conditions. 
           
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For further information, please contact:

Julie Meldrum
Corporate Communications, Mesoblast 
T: +61 3 9639 6036
E: [email protected]

Schond Greenway
Investor Relations, Mesoblast 
T: +1 212 880 2060
E: [email protected]