StemCells, Inc. (STEM) announced on Thursday interim results from a phase 1/2 trial of its HuCNS-SC cells in dry age-related macular degeneration (AMD). The results are encouraging in a field that has long demonstrated promising results in animal models that ultimately fail to translate in the clinic, and safety has been a non-issue thus far in the human trial. The approach attempts to fundamentally affect the underlying cause of the disease. STEM is up 22% in morning trading.
For some perspective, STEM has been around for a long, long time as a publicly traded biotech company. The company IPO’d in 1992, changed its name and focus to stem cell therapeutics in 2000, and the stock currently trades near the absolute bottom of its 20-year history, not exactly confidence-inspiring for potential investors.
The phase 1/2 trial evaluates the safety and early efficacy of HuCNS-SC cells in dry AMD, and patients had to have evidence of GA for inclusion. Patients with BCVA (best-corrected visual acuity) of less than or equal to 20/400 in the study eye were enrolled in Cohort I; patients with less severe BCVA of 20/320 to 20/100 in the study eye are enrolled in Cohort II.
In cohort 1, four subjects were transplanted with 200,000 stem cells each. Another four subjects were transplanted with 1 million cells. Cohort II, patients with less severe BCVA, will consist of eight subjects who will undergo transplantation with 1 million cells.
Key takeaways from Cohort I include:
- A 70 % reduction in the rate of geographic atrophy (GA)when compared to the control eye, and a 65% reduction in the rate of GA in the study eye compared to the natural history of the disease. Atrophy is the progressive loss of photoreceptors and the retinal pigmented epithelium, the underlying cause of vision loss in dry AMD.
- The company also reported improvements in visual function (the ability to distinguish shades of light versus dark, or “contrast sensitivity”), in 4 of 7 (57%) patients.
The data were presented on Wednesday at the 12th annual meeting of the International Society for Stem Cell Research (ISSCR). StemCells hopes to begin a randomized proof of concept trial in the second half of this year.
Advanced Cell Technology (ACTC) also tests its own stem cell therapeutic in dry AMD.
