Welcome to your update from the Clinic for the month of July. Adaptimmune moves a second candidate into the clinic. Ziopharm receives an Orphan Drug Designation for its cutting edge IL-12 technology. Canadian cell therapy company, Hemostemix, keeps moving forward with enrolment in its clinical trial for ACP-01 in critical limb ischemia.
Oxford-based Adaptimmune (ADAP) announced that the FDA has accepted its investigational new drug (IND) for MAGE-A10 T, a T cell receptor (TCR) technology being developed for locally advanced and metastatic lung cancer. The company will initiate a Phase 1/2 study and begin enrolment in 2015. MAGE-A10 T targets cancer/testis tumor-associated antigens inside the cancer cell, which are up-regulated in approximately 30% of lung and bladder cancer, and skin melanomas. This is the second TCR candidate that ADAP has moved into the clinic, making it the current leader in clinical development of TCR products.
Hemostemix (HEM) is rapidly enrolling patients in its international Phase 2 clinical study investigating ACP-01 for the treatment of critical limb ischemia (CLI), which is being completed at six sites between Canada and South Africa. ACP-01 is an autologous cell therapy derived from a patient’s own blood. Once blood is removed from a patient it is fractionated to isolate a population of cells Hemostemix refers to as a synergetic cell population. This population of cells is then differentiated towards the identity of an anglogenic precursor, placed into syringes and injected back into diseased tissue to support growth of new blood vessels. The FDA recently cleared HEM’s IND, which will allow the company to begin enrolling patients at sites in the U.S. and bring it closer to its goal of enrolling 100 patients in total. HEM is going head-to-head with Pluristem, Vericel, and ReNeuron in CLI.
Ziopharm (ZIOP) received an Orphan Drug Designation from the FDA for Ad-RTS-hIL-12 in the treatment of malignant glioma. The candidate is currently in Phase 1 development for glioma and Phase 2 development for breast cancer. ZIOP uses an adenovirus to deliver the interleukin-12 (IL-12) gene to the site of the cancer through intratumoral injection. In order to regulate the amount of IL-12 protein created, the IL-12 gene is placed under the control of an inducible promoter (RheoSwitch® provided by Intrexon). The inducible promoter is turned on with veledimex, which can be taken orally by the patient. After translation, IL-12 facilitates an anti-tumor T cell response against the cancer cell it was created in. See OncoSec Medical’s platform for another approach to intratumoral IL-12 therapy, which utilizes injection and electroporation.
IL-12 was originally deployed into humans for the treatment of cancer in bolus form (injected with a needle at high concentration); however, this approach led to cytokine storms in some patients (systemic cascades of immune signaling that are very difficult to thwart once initiated), which can be fatal. As a result, what was once thought to be a promising approach to treating cancer was shelved and left to collect dust for many years. With the advent of synthetic biology and genetic engineering, IL-12 is now undergoing a revival. ZIOP’s solution to the previous safety issues is quite ingenious.
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 visit http://ccrm.ca/industry-consortium