Mesoblast (ASX:MSB; NASDAQ:MESO) is rallying today after the company announced yesterday after market close that the FDA approved RYONCIL (remestemcel-L), an allogeneic (donor) bone marrow-derived mesenchymal stromal cell (MSC) therapy indicated for the treatment of steroid-refractory acute graft-versus-host disease (SR-aGVHD) in pediatric patients. RYONCIL is now the only MSC therapy approved in the U.S. for any indication, and the only approved therapy for (SR-aGvHD in children 2 months and older, including adolescents and teenagers.
As of writing, Mesoblast’s stock is up 27% to $15.63, and has traded as high as $19.15.
In a statement, Mesoblast CEO, Dr. Silviu Itsecu, said, “We are very pleased that the FDA has granted approval of RYONCIL and are proud of the company’s commitment to the GVHD community in bringing this important new treatment to children and families with no other acceptable options.”
“With RYONCIL approval by FDA, Mesoblast has demonstrated the ability to bring the
first MSC product to market. We will continue to work closely with FDA to obtain approval of our other late-stage products, including REVASCOR® for cardiovascular diseases and rexlemestrocel-L for inflammatory pain indications, as well as expanding the indications for RYONCIL in both children and adults with inflammatory conditions,” Dr. Itsecu added.
Approved stem cell therapies like RYONCIL are limited. In addition to Mesoblast, other prominent companies in the space, including BioRestorative Therapies (NASDAQ:BRTX) and Cell Technologies (Private), are working to add to the list.
BioRestorative’s lead cell therapy candidate, BRTX-100, is a product formulated from autologous (or a person’s own) cultured mesenchymal stem cells collected from the patient’s bone marrow. The product is intended to be used for the non-surgical treatment of painful lumbosacral disc disorders or as a complementary therapeutic to a surgical procedure. The company recently reported positive preliminary 26–52 week blinded data from the first 10 patients with chronic lumbar disc disease (cLDD) enrolled in the ongoing Phase 2 clinical trial of BRTX-100. In a press release announcing the preliminary data, BioRestorative indicated that, if the positive trends continue, it is confident that the Phase 2 trial will meet its Primary and Secondary endpoints.
Platform ChondroStem stem cells for osteoarthritis are Cell Technologies’ core offering. The company is preparing for the launch of a pivotal phase clinical trial of CLL-001, its autologous stem cell therapeutic product candidate. If the trial is successful, Cell Technologies is potentially eyeing the first FDA approved stem cells in the U.S .for osteoarthritis. The company also has plans to advance to clinical trials of its next-generation CLL-002, its allogeneic stem cell program in osteoarthritis.
