I have recently joined the business development group at CCRM, as an analyst, and believe it would be useful to start compiling some recaps on clinical developments in the regenerative medicine (RM) and cell therapy industries to complement the other science-related and commercialization content that appears on this blog. I also intend to put together some commentary on partnering and licensing in these spaces.
My first post, ‘Update from the Clinic,’ focuses on the progress of RM products through the clinic, with an emphasis on cells and biomaterials. The second piece, ‘Regen Med Deal Review,’ will focus on notable partnering, licensing, financing, and M&A transactions that occur within industry. I will strive to keep posts as regular as possible so the flow of information remains reasonably fresh.
With that being said, I’ll launch into my first recap on the clinic.
An exciting development this month comes from Sernova (TSXV:SVA), which has been developing its platform Cell Pouch™ technology. The pouch, which becomes vascularized by the patient’s body following transplantation, is designed with narrow reservoirs to house therapeutic cells. Once inside, the therapeutic cells help manage a patient’s disease.
On September 26th, Sernova announced a world-first, reporting positive safety and biocompatibility data from a Phase 1 clinical study for the treatment of diabetes using the Cell Pouch™ and islet cells. Therapeutic cells survived and produced insulin in the first two patients in the study. No immune-mediated destruction of islet cells was observed, suggesting the cell pouch provides protection from the host’s immune system.
While Sernova’s current focus is T1 diabetes, the CEO, Dr. Philip Toleikis, has plans to investigate other therapeutic cell types to combine with the Cell Pouch™. Sernova recently signed an MTA with German-based Medicyte to investigate the effects of using Medicyte’s upcyte® cells in the Cell Pouch™ for the treatment of hemophilia A.
Another significant clinical development came from New Zealand-based Living Cell Technologies (ASX:LCT), which announced a first-in-man implant in a Phase 1 study of NTCELL® (cells derived from the choroid plexus region of the brain) for patients with Parkinson’s disease. LCT has been pioneering the field of cell encapsulation for transplantation over the last several years.
There was positive news for Dendreon (NASDAQ:DNDN) this past month as it received marketing approval for its product PROVENGETM (activated autologous blood mononuclear cells) in Europe, for use in patients with metastatic castrate-resistant prostate cancer. The company was given the green light to market the product following three multicentre Phase 3 studies that enrolled 738 patients. The therapy was shown to increase median survival by 4.1 months.
Verastem (NASDAQ:VSTM) has announced a number of clinical studies for its lead candidate defactinib: a Phase 2 study for KRAS-mutated non-small cell lung cancer, a Phase 2 study in patients with malignant pleural mesothelioma (a rare form of cancer that is commonly caused by exposure to asbestos), and a Phase 1 study in Japanese patients with advanced solid tumours. Defactinib was granted orphan drug designation by the FDA, in July of this year, for use in patients with mesothelioma.
Along with Stem Cell Therapeutics (TSXV:SSS), Verastem is one of a small handful of companies with a mandate to commercialize therapeutics that specifically target cancer stem cells. The former recently announced that its CD47 antagonist program is moving into the IND-enabling phase, while tigecycline, a cancer stem cell-targeting therapeutic being developed by Stem Cell Therapeutics in conjunction with Toronto’s MaRS Innovation, is currently being evaluated in a Phase 1 study for relapsed or refractory acute myeloid leukemia.
Aastrom Biosciences (NASDAQ:ASTM) has received approval from Health Canada to initiate a Phase 2 study of ixmyelocel-T (patient’s own expanded bone marrow cells) in patients with advanced heart failure, while south of the border the FDA gave Pluristem (NASDAQ:PSTI) the go-ahead to continue its Phase 2 clinical study of PLX-PAD cells (placenta-derived). The study, being evaluated for treating muscle pain in patients with ischemia and peripheral leg disease, was halted in June of this year following a severe allergic incident in one patient.
Currently, what really stands out to me in the RM space is Sernova’s Cell Pouch™ product. If the company can find a pluripotent source of mature, functional islet cells to produce them in commercial quantities, this combination technology has the potential to be a game-changer for T1 diabetes patients. Stay tuned for the first Regen Med Deal Review, which should be posted in the next week or two.
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