Welcome to your Update from the Clinic for the month of May. StemCells announced positive topline results from a Phase I/II study in spinal cord injury. Kite Pharma treated its first patient in a Phase I/II study investigating an anti-CD19 CAR T construct in Non-Hodgkin’s Lymphoma. Following some promising interim results, NewLink Genetics decided to stay the course with its Phase III study investigating Algenpantucel-L for the treatment of pancreatic cancer.
In an international, open-label Phase 2 study investigating StemCells’ (STEM) HuCNS-SC product (human neural stem cells) in patients with thoracic spinal cord injury, 12 patients were given transplants. Seven of these had the most severe classification of injury (AIS A), indicating that they had neither motor function nor sensory function below the site of injury, while the remaining patients were classified AIS B and had some, limited, sensory function below the site of injury. Preliminary efficacy data was positive. Several patients began to exhibit improvements in sensory function around three months post-transplant. Improvements were so positive in two of the AIS A patients that the severity of their injury was downgraded to AIS B.
One of the key players in the CD19 race, Kite Pharma (KITE), treated its first patient in a Phase I/II study investigating KTE-C19, a chimeric antigen receptor (CAR) T-cell product, in patients with refractory diffuse large B cell lymphoma (DLBCL). The company is going head-to-head with Juno, Novartis, Cellectis and a number of other companies (Takara, Bellicum, Cellular Biomedicine) that all have CAR technologies targeting the same antigen. Once the Phase 1 portion of the study is complete, KITE will launch the Phase 2 segment, which will include 112 patients.
Recessive dystrophic epidermolysis bullosa (RDEB) is a progressive, congenital disorder that is caused by a mutation in the gene encoding collagen VII, which is required to anchor layers of skin together. Without the protein, skin layers begin to separate and blister, causing extreme pain. Ultimately, the disease leads to death. Signals has a post about EB here. For obvious reasons, the FDA is expediting the licensing of Fibrocell’s (FCSC) FCX-007, a genetically modified fibroblast product that is designed to produce collagen VII. The product, which has already received an Orphan Designation, was recently given a Rare Pediatric Disease Designation by the FDA to further support its development. The new designation will allow Fibrocell to apply to the FDA for a Rare Pediatric Disease Priority Review Voucher.
Pluristem (PSTM), which is developing placenta-derived therapeutic cells, received notification from Japan’s Pharmaceutical and Medical Devices Agency (PMDA) that it has approved the manufacturing methods used to scale up its PLX-PAD cells for use in clinical studies. As a result, the company is now pursuing the launch of a Phase 1/2 study through Japan’s Accelerated Approval Pathway, which, remarkably, provides regenerative medicine companies a means to not only have their technologies licensed, but also reimbursed, on nothing more than preliminary safety and efficacy data. Now that’s progressive policy!
There are a handful of companies in the cell-based immunotherapy space that have technologies that have surpassed the Phase 2 stage: one of these is NewLink Genetics (NLNK), which is certainly a unique company amongst its peers. Recognizing an immediate market opportunity, the company forged a deal with Merck, late in 2014, to begin development of a vaccine against Ebola. In addition to this, NLNK’s pipeline is rich with cell-based immunotherapy assets targeting oncology indications of high unmet need, including pancreatic cancer, advanced lung cancer and metastatic melanoma. After positive results from its Phase 2 study investigating Algenpantucel-L in resectable (lesion can be removed) pancreatic cancer, the company successfully enrolled 722 patients in a Phase 3 study (IMPRESS). The study is being conducted under a Special Protocol Assessment (SPA) with the FDA.
NLNK is now among a small group of companies that is harnessing whole tumor cells to prime patients’ immune systems to fight malignant cells. The company has created a number of generic tumor cell lines that express a specific, immunogenic, protein that catalyzes an immune response against the tumor cell it is expressed on. The result is a strong, targeted response against the cancer cell line. This approach provides several benefits, including reduced manufacturing costs by way of bulk manufacturing, fewer logistical challenges (no need to harvest cells from patients and fly them to manufacturing centres), and more robust quality control (QC) through standardized QC assays. Both Cellectis and Celyad also have allogeneic immunotherapies in development (CAR-T). The trend towards “universal” products is likely to continue, as we discover new ways to genetically alter cells to circumvent immune rejection.
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