StemCells, Inc. is a top biotech company developing stem cell-based therapies. They have a deep pipeline that includes already ongoing trials for a variety of diseases. I invited company leadership to do an interview and they graciously accepted.
Below is the interview with CFO Greg Schiffman (picture at left from LinkedIn) including what I thought were some tough questions from me and very detailed answers from Shiffman. Thank you, Greg.
1. Where does the company stand today in terms of product development, capitalization, and such? What are its strengths? What are potential areas for improvement or development?
We currently have three active programs underway two clinical and one pre-clinical. The two clinical programs are in the spine, spinal cord injury (SCI), and in the eye, dry age related macular degeneration (AMD), and the pre-clinical program is in the brain focused on Alzheimer’s disease. Both clinical programs have completed enrollment of the Phase I/II clinical trials and we have plans to initiate controlled proof of concept Phase II studies in both indications in the second half of this year. Both indications have large unmet medical needs. We have released interim results for both of the Phase I/II programs. We have not seen any safety issues associated with the cells. In addition, we have seen preliminary signs of efficacy in both programs. Our pre-clinical efforts are focused on filing an IND for Alzheimer’s disease in 2016 following which we could move this program into the clinic.
We just completed a $20 million gross equity financing. If you include the proceeds from the equity financing and warrant exercises that occurred in the month of July, the company had approximately $37.8 million dollars of cash and marketable securities at the end of June. This provides us with a strong balance sheet to move our programs forward.
We believe that we have a unique platform utilizing our proprietary HuCNS-SC® human neural stem cells that have the potential to address numerous indications affecting the CNS. This would include white matter brain disorders such as leukodystrophies, multiple sclerosis, cerebral palsy and transverse myelitis. Other potential indications could include Alzheimer’s, spinal cord injury, dry age-related macular degeneration and centrally mediated lysosomal storage disorders. We have performed pre-clinical work in many of these indications and we have selected one indication, where there is a large unmet medical need, in each major organ of the CNS the brain, eye and spine to pursue clinically. The cells have shown a very favorable safety profile and all of the pre-clinical and clinical findings have shown signs of efficacy. This is a very exciting time for the Company as we are now rapidly generating a significant amount of clinical data which will help to provide much greater insight into the potential clinical benefit we can bring to patients.
2. What unique challenges does the stem cell/cell therapy/regenerative medicine biotech sector face overall say compared to a pharma company producing a chemical-based drug and how is StemCells, Inc. approaching those hurdles?
Anytime you are pursuing a new therapeutic paradigm you encounter additional regulatory hurdles and scientific scrutiny. StemCells, Inc. has very methodically pursued the science. We have published all of our work so that others can critique the findings. It has taken a significant amount of time and resources pursuing the science to understand the potential of the platform we are building. However, it enabled us to move forward with our clinical programs on a very strong scientific foundation. In addition, given the nature of our cells which engraft into the host, replicate, migrate within the host and differentiate into the cell types associated with the CNS, our early clinical trials faced significant safety hurdles as this was the first time that these cells would be transplanted in humans. The first indications had to be fatal diseases and the subjects were in the most advanced stage of the disease. In addition, we were limited to one surgeon and a single site for patient recruitment. We would treat a patient following which safety data was generated and submitted to the FDA for review. After review we were able to begin looking for the next patient to be transplanted with the cells. The clinical trials moved forward very slowly given these hurdles. However, given the favorable safety profile we have shown in both the spine and eye, as well as the favorable safety profile demonstrated in our previous clinical work in the brain for both Batten disease and Pelizaeus-Merzbacher disease (PMD), we are now able to proceed forward at a much more rapid pace, consistent with other biologic clinical trials, with our Phase II studies. In 2015 we plan to have between 10-15 sites enrolling patients for each of our clinical programs and expect to be able to complete the enrollment in about one year. As we continue to move forward with the clinical efforts, and hopefully to an approved therapeutic, we expect that there will be additional hurdles to overcome including physician and patient education on this new platform of stem cells. This is an exciting opportunity; and we are comfortable in our role as pioneers breaking new ground. Please continue reading on Page 2!