Mesoblast Ltd. (MESO:NASDAQ; MSB:ASX), which concentrates its efforts on inflammatory diseases, today announced that "the Oncologic Drugs Advisory Committee (ODAC) of the U.S. 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)."
The company's Chief Medical Officer Dr. Fred Grossman commented, "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 firm explained that the ODAC is an independent scientific advisory panel that evaluates drug safety and efficacy data and then makes non-binding recommendations to the FDA based upon its findings. The company noted that "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), and if approved by that date, Mesoblast plans to launch RYONCIL in the U.S. in 2020."
"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," stated Dr. Joanne Kurtzberg, pediatric transplant physician, the Jerome Harris Distinguished Professor of Pediatrics and professor of pathology, and director of the Pediatric Blood and Marrow Transplant Program at Duke University Medical Center.
The company stated that aGVHD occurs in about 50% of patients receiving allogeneic bone marrow transplants (BMT). Each year more than 30,000 patients worldwide undergo an allogeneic BMT primarily during blood cancer treatments. The firm indicated that in the most severe cases of aGVHD mortality rates are often as high as 90%. Mesoblast pointed out that presently there are no U.S. FDA-approved treatments for children under 12 years of age with SR-aGVHD, which all too often can be a life-threatening complication of an allogeneic BMT for blood cancer.
The company described its lead product candidate RYONCIL (remestemcel-L) as "an investigational therapy comprising culture-expanded mesenchymal stem cells derived from the bone marrow of an unrelated donor that is administered to patients in a series of intravenous infusions." The firm additionally stated that "RYONCIL is believed to have immunomodulatory properties to counteract the inflammatory processes that are implicated in steroid-refractory acute graft versus host disease by down-regulating the production of pro-inflammatory cytokines, increasing production of anti-inflammatory cytokines, and enabling recruitment of naturally occurring anti-inflammatory cells to involved tissues."
Mesoblast Ltd. is headquartered in Melbourne, Australia, and is a developer of industrial-scale, cryopreserved, off-the-shelf allogeneic cellular medicines. The company utilizes its proprietary mesenchymal lineage cell therapy technology platform in order to create a wide portfolio of commercial products and late-stage product candidates. The firm indicated that "the Biologics License Application to seek approval of its product candidate RYONCIL™ (remestemcel-L) for steroid-refractory acute graft versus host disease has been accepted for priority review by the U.S. Food and Drug Administration." The company is also investigating and developing Remestemcel-L for uses in other inflammatory diseases in children and adults including moderate to severe acute respiratory distress syndrome. The firm is now in the process of completing Phase 3 trials for advanced heart failure and chronic low back pain product candidates. The company has offices located in Australia, the U.S. and Singapore.
Mesoblast began the day with a market capitalization of around $1.4 billion with approximately 115.5 million shares outstanding and a short interest of about 1.4%. MESO ADR shares opened nearly 56% higher today at $18.41 (+$6.60, +55.88%) over yesterday's $11.81 closing price. The stock has traded today between $16.25 and $19.60 per share and is currently trading at $17.81 (+$6.00, +50.80%).
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