Welcome to your Update from the Clinic for the month of November. There were a number of mid-stage clinical trials launched by cell-based immunotherapy companies this past month, furthering a robust global pipeline of CAR/TCR products. We also saw some efficacy data from StemCells, which reported positive interim results from its Phase 2 spinal cord injury trial and Celyad, which reported initial safety data from the first few patients in its Phase 1 CAR-NKG2D trial.
Kite Pharma (KITE) launched the ZUMA-1 and ZUMA-2 Phase 2 clinical studies, both of which will evaluate Kite’s lead product KTE-C19 in oncology indications. ZUMA-1 will investigate KTE-C19 in patients with aggressive, refractory non-Hodgkin’s lymphoma (NHL) and will enrol 112 patients in total. Topline results from the Phase 1 portion of the study were presented at the American Society for Hematology (ASH) meeting this month. Interim results from the Phase 2 portion are expected in 2016. Kite is on track to commercialize its first product for NHL some time in 2017. ZUMA-2 will investigate KTE-C19 in patients with relapsed/refractory mantle cell lymphoma (MCL) and will enrol 70 patients. Patients included in the study will be those who have failed previous anthracycline or bendamustine-containing chemotherapy, or anti-CD20 antibody.
Breast cancer patients who have tumour-infiltrating lymphocytes (TILs) typically have a better prognosis. Many immunotherapies, which are in development, aim to increase tumour immunogenicity and influx of immune cells. OncoSec Medical (ONCS) enrolled its first patient in a pilot biomarker study investigating ImmunoPulse IL-12 in patients with triple negative breast cancer (TNBC). The objective of the study will be to evaluate whether intra-tumoural IL-12 plasmid electroporation changes the spectrum of lymphocyte subsets within patient tumours. By increasing the number of TILs inside a tumour, OncoSec believes ImmunoPulse IL-12 will improve the efficacy of checkpoint inhibitors like anti-PD-1/PD-L1 when administered in combination. Adaptimmune (ADAP) also launched on a solid tumour indication with a Phase 1/2 study of a TCR product targeting NY-ESO-1 in patients with non-small cell lung cancer (NSCLC).
Patients in StemCells’ (STEM) Phase 2 PATHWAY study are one and two years post-injury. Despite this, many showed improvements in both muscle strength and function following transplant, a first in clinical studies of spinal cord injury. It is rare for patients to exhibit improvements in spinal cord function following injury much beyond a year. Clinicians observed patients performing motor tasks that they were previously unable to do, such as opening a jar, picking up coins and grasping a key. It’s exciting to see a true regenerative medicine technology making its way into mid-stage clinical studies, for an application where there is an incredible amount of unmet need.
Finally, Celyad (CYAD) reported on the first few patients in its Phase 1 study investigating CAR-NKG2D in patients with AML and multiple myeloma. Celyad is a unique company for two reasons: first, instead of using an antibody-based CAR it’s using a receptor from NK cells; and second, the company is using gene editing technology, like Cellectis, to inhibit TCR function so that T cells are cloaked from the host immune system and persist in vivo. The first three patients in the study showed no adverse events 30 days post-infusion. Data for the remaining nine patients in the study are expected in 2016. While it is very early days, these initial safety results, for an innovative CAR technology, are promising.
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