Welcome to your Update from the Clinic for the month of March. Undoubtedly the big news in March came from Vericel, which saw its stock double after reporting positive topline results from its Phase 2b study in heart failure. There were also some first dosings and clinical study starts.
A study of Vericel’s (VCEL) ixmyelocel-T met its primary endpoint demonstrating a significant decrease in patient deaths and cardiovascular hospitalizations in patients with acute heart failure, relative to placebo. Vericel, formerly Aastrom, has been at it for a while now with limited success in clinical development. Despite being formed in 1989, Vericel didn’t have a marketed product until its acquisition of Sanofi’s regenerative medicine business in 2014. So, some positive results are a breath of fresh air for investors. Although, VCEL stock did correct itself early in April after a number of secondary endpoints in the Phase 2b study were disclosed, including duration of time to first event, the size of the left ventricle cavity, and the distance a patient could walk, which were not significantly different to placebo. Based on the primary endpoints, however, this is still a product that should continue to be investigated for an indication with plenty of unmet need. Vericel also announced FDA acceptance for filing of a biologics license application (BLA) for its MACI product in cartilage defects of the knee.
Argos Therapeutics (ARGS) initiated a Phase 2 clinical study of AGS-003 for patients with non-small cell lung cancer. AGS-003 is composed of dendritic cells loaded with patient-specific tumour RNA. Aduro Biotech (ADRO) dosed its first patient in a Phase 1/2 study investigating CRS-207 in combination with epacadostat in patients with ovarian cancer (the company’s SEASCAPE study can be found here). The company also launched an immunotherapeutics and vaccine research collaboration with UC Berkeley that will investigate novel treatments for oncology, infectious disease, and autoimmune disease. As part of the collaboration, Aduro will provide Berkeley $7.5 million in funding over three years and access to its platforms, including LADD, STING-pathway inhibitors, and B cell monoclonal antibodies.
Some advances were made on the diabetes front. Caladrius Biosciences (CLBS) announced treatment of the first patient in its study investigating CLBSO3 in early-onset type 1 diabetes in adolescents. CLBSO3 is an autologous therapy composed of a patient’s own T regulatory cells. The study will enroll 111 patients, between the ages of 12 and 17, in two cohorts and is being conducted in collaboration with Sanford Health. Mesoblast (MBS) exclusively licensed technology, from Harvard Medical School, that can be used to modify mesenchymal lineage adult stem cells (MLCs) and improve their homing to areas of inflammation. Preclinical studies showed that administration of fucosylated MLCs, in mouse models of diabetes, dramatically reduced inflammation of the pancreas.
T regulatory cells are emerging as a promising new paradigm to slow progression of early-onset type 1 diabetes. By curbing destruction of pancreatic islets early in a patient’s life, it may be possible to stabilize the disease and prevent further destruction of insulin producing cells.
Disclaimer: “Update from the Clinic” is a blog post generated by news flow from public regenerative medicine (RM) companies around the globe. As CCRM has public RM companies in its industry consortium, and the number of such companies is relatively limited on a global scale, Mark Curtis will sometimes include CCRM consortium members in his review. This blog post is provided for general information only and nothing contained in the material constitutes a recommendation for the purchase or sale of any security. The author is not a shareholder of any public RM company. To see a list of CCRM’s industry consortium members, please visithttp://ccrm.ca/industry-consortium