.
Welcome to your Update from the Clinic for the month of March. Investors weren’t so keen on Neuralstem’s topline results in amyotropic lateral sclerosis (ALS). NeoStem’s follow-up data from its PreSERVE study in acute myocardial infarction (AMI) was somewhat lacklustre. Northwest Biotherapeutics continues to report positive data on its DCVax technology, breathing hope into what could shortly be the world’s first cell-based immunotherapy for brain cancer.
Since the release of its Phase 2 topline data of NSI-566 in ALS patients, Neuralstem (CUR) has lost almost 50% of its market capitalization. While the therapy was proven safe, only seven of 15 patients responded following transplant of the neural stem cells (“response” was defined as a “near-zero” decline, or improvement in motor function). Understandably, this didn’t provide investors the confidence they needed with respect to the future prospects of the therapy. There is the possibility that CUR could focus enrollment on responders, moving forward, but this would necessitate a battery of tests capable of identifying this subset.
NeoStem (NBS) announced 12-month follow-up data from its Phase 2 PreSERVE study investigating NBS10 (CD34+ cells) in patients with AMI. Given the high standard-of-care and low mortality in this indication, it is not surprising it failed to demonstrate a significant mortality benefit. As an exploratory efficacy endpoint, the company measured the size in reduction of patients’ infarcts. Patients receiving the therapy experienced a 41% reduction in infarct size vs. the control group, which showed a 25% reduction, suggesting the cells have a healing effect.
Vericel (VCEL, formerly Aastrom) also announced follow-up data, providing details of its Phase 3 SUMMIT extension study investigating MACI (matrix-applied characterized autologous cultured chondrocytes) in patients with articular cartilage defects. Results were consistent with those observed at the two-year mark. MACI was found to be statistically significantly better than the standard-of-care – microfracture. The extension study included 128 patients that were tracked for an additional year following the original SUMMIT study.
The good news keeps rolling in for Northwest Biotherapeutics (NWBT), which announced positive data from an information arm of 51 patients who were administered DCVax for the treatment of Glioblastoma multiforme (a type of brain tumour). A number of the patients who exhibited a positive therapeutic effect were independently categorized as rapid progressors by a third party imaging group. In this subset, the cancer is so aggressive it begins to grow back immediately following chemoradiation therapy after resection (surgical removal) of the tumour. Median overall survival for the arm was 18.3 months. Approximately 30% of patients lived longer than two years, the majority of whom are still alive. Overall survival in rapid progressors was 15.3 months – about 5-7 months more than expected when comparing to the results in published literature. NWBT recently launched two new clinical sites for its Phase 3 study in Canada.
After completing a battery of preclinical studies on its placenta-derived PLX-RAD cells, Pluristem (PSTM) is solidifying plans to bring the technology forward into the clinic in at least two hematopoietic indications. Mechanistic studies point to the revival of platelet production, in addition to stimulation of red and white blood cell production, as the mode in which the cells are capable of restoring function of the bone marrow compartment. It is anticipated the technology can be used for bone marrow failure and improving the speed of bone marrow engraftment. Pluristem will continue working with the NIH on the use of the therapy for acute radiation syndrome.
The complexity of CNS indications has always presented challenges to industry. While Neuralstem may not have success with ALS, it’s taking several shots on goal. In addition to ALS, the company is deploying NSI-566 to spinal cord injury (Phase 1), ischemic stroke (Phase 1/2), and multiple sclerosis, Alzheimer’s, traumatic brain injury, Parkinson’s, Huntington’s, and cerebral palsy (all preclinical). It is also developing a number of drug treatments for CNS indications. Providing it can find funding for these programs, the diversification will help drive commercial outputs.
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
Mark Curtis
Latest posts by Mark Curtis (see all)
- Update from the Clinic: May - June 30, 2016
- Cell Therapy Deal Review: May - June 23, 2016
- Update from the Clinic: March - April 28, 2016
Thanks for catching that Bob.
You posted the symbol for Northwest incorrectly, its NWBO