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20-F
MESOBLAST LTD filed this Form 20-F on 08/31/2018
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Our Tier 1 Phase 3 clinical trial evaluating MPC-150-IM for moderate to advanced chronic heart failure is actively recruiting across North America. Our Tier 1 Phase 3 clinical trial evaluating MPC-06-ID for chronic low back pain completed enrollment in March 2018. The Tier 1 Phase 3 trial of MSC-100-IV for acute graft versus host disease in children has successfully met the Day 28 primary endpoint after completing enrollment in December 2017.

Tier 1 Programs

MSC-100-IV for the Treatment of acute Graft versus Host Disease (aGVHD)

Overview

MSC-100-IV is our intravenously delivered product candidate for the treatment of acute steroid-refractory graft versus host disease, or SR-aGVHD, following allogeneic bone marrow transplant. Available data from clinical dose ranging studies identified an effective dose to be 2 million MSCs/kg, body weight, to be administered repeatedly for at least four weeks after diagnosis of aGVHD. For the U.S. market, the unit packaging is 25 million cells per vial for intravenous infusion.

In a bone marrow transplant, donor cells can attack the recipient, causing aGVHD. The donor T-cell mediated inflammatory response involves secretion of TNF-alpha and IFN-gamma, resulting in activation of pro-inflammatory T-cells and tissue damage in the skin, gut and liver which can be fatal.

MSC-100-IV was developed to counteract the inflammatory processes by down-regulating the production of pro-inflammatory cytokines, increasing production of anti-inflammatory cytokines, and enabling recruitment of endogenous anti-inflammatory cells to involved tissues.

Currently there are no approved therapies for patients with acute graft versus host disease (SR-aGVHD) in the U.S.

MSC-100-IV has been used for the treatment of aGVHD in children in the U.S., Canada and several European countries under an expanded access program. This program enrolled more than 240 patients suffering from SR-aGVHD.

Market Opportunity

According to the Center for International Blood and Marrow Transplant Research, there are approximately 30,000 allogeneic BMTs globally per year for diseases including hematological cancers, with ~20% of all cases in the pediatric population. Nearly 50% of all allogeneic BMT patients develop aGVHD. Liver or gastrointestinal involvement occur in up to 40% of all patients with aGVHD and are associated with the greatest risk of death, with mortality rates of up to 85%.

The aGVHD market requires a small, targeted commercial footprint. The target market for aGVHD will primarily be board-certified physicians in hematology/oncology who perform hematopoietic stem cell transplants. In the U.S., there are approximately 75 centers that perform pediatric transplants, with 50% of all transplants occurring at approximately 15 centers. Similarly, there are approximately 110 centers that perform adult transplants with half of those transplants occurring at approximately 20 centers.

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