Asterias Biotherapeutics continues to add to their respectable data set, announcing on Tuesday that patients being treated in its SCiStar trial using AST-OPC1 cells have continued to produce meaningful motor function improvement from a previous 10-million cell injection. The updated results stem from observation during the 6-month and 9-month follow-up period. The AST-OPC1 trial is treating patients that have complete cervical spinal cord injuries.
What AST Has Seen
Patients in the study have continued to demonstrate improvement in motor skill, with recovery being observed in upper extremity motor function in five of five patients, representing a 100% response rate. AST stated that they believe the results to be meaningful, and that the increases in motor function can lead to a dramatically increased quality of life and may also provide patient’s with relative independence in regard to personal care, allowing them to recapture their ability to perform necessary activities that require the ability to use fingers, hands, and arms.
Steve Cartt, CEO of AST, said, “The results to date in the 10 million cell cohort treated with AST-OPC1 cells show that the improvements in arm, hand and finger function observed very early in the study have been maintained and in most patients have even been further enhanced over time,” He added, “These results to date are quite encouraging, and we look forward to initiating discussions with the FDA in mid-2017 to begin to determine the most appropriate clinical and regulatory path forward for this innovative therapy. In addition, we anticipate reporting 12-month efficacy and safety data for this cohort, as well as 6-month efficacy and safety data for the currently-enrolling AIS-A 20 million cell and AIS-B 10 million cell cohorts, during the third quarter of 2017.”
With AST being a relatively under-the-radar stock, the results were not met with much hype or hysteria. However, investors may very well want to keep AST on their shopping list, as the company is not only performing extremely well in its clinical trials, but has also positioned itself well for potential partnerships in the coming months. Although early in the process, these “meaningful” results can offer an attractive springboard to interested parties outside of the United States, where encouragement of stem cell trials are more pronounced.
The data release on Tuesday showed that additional motor function improvement is apparent at both the 6-month and 9-month follow-up intervals. The increases in motor function is being seen in both classifications of injury, AIS-A and AIS-B, which distinguish between levels of both sensory and motor function.
AST expects to enroll five cohorts, with the first two already completed. The next three cohorts are expected to treat both AIS-A and AIS-B patients with a 20-million cell dose of the AST-OPC-1 cells. Initial data is expected to be presented beginning in the middle of 2017 and continuing with quarterly patient follow-up results progressing through 2018.
AST further expects to begin dialogue with the FDA in mid-2017 to not only only discuss current trial results, but to implement a strategic trial design that may enable an expedited approval process, taking advantage of trial pathways that are intended to deliver breakthrough treatments to market.
While results are early, reliable indications from the SCiStar trial demonstrate that the efficacy being earned by the treatment is well beyond natural occurrence, quieting the argument that spontaneous improvement is the driving mechanism in patient response. Although the patient and trial size is small with regard to current participants, the science behind the cell therapy is well documented, with positive results being consistently demonstrated in pre-clinical, animal, and human studies.
A key takeaway from the Tuesday call was that AST released initial data from two of the patients being treated in the 20-million cell cohort. During this early stage of the 20-million cell study, each patient has shown at least one motor level improvement; but, it’s important to note that one of the patients has already achieved a two motor level improvement in motor function. And, again, there has been no sign of adverse reaction to the treatment.
With AST emerging as the predominant leader in the space to treat SCI, especially with Geron’s setbacks, the company is ripe to grow into analyst price targets that are in excess of $10.00 per share during the next twelve month period.
Although some investors may advise caution based on the patient trial size believing that results can be easily skewed to the upside, others beg to differ. While true to point that the patient enrollment is low, what can’t be argued as mere coincidence is that AST has provided a 100% positive response rate in increasing both patient sensory and motor function. For that reason, I’ll side with the bulls.
Disclosure: This article was written by Kenny Soulstring, and it reflects my own opinions and unique articulation. This article is not intended to offer investing advice, guarantee 100% accurate predictions or to be interpreted as providing a personal recommendation. What I can guarantee, though, is accurate research, thoughtful analysis and an enthusiasm about any stock that I cover.
While I seek to uncover emerging companies that I feel have true value and potential, it’s important that investors assign an appropriate time horizon to each of their investments, understanding that emerging companies need time to mature.
I wrote this article myself and it includes my own research and expresses my own opinions. I am not receiving compensation for it (other than from CNA Finance). I have no business relationship with any company whose stock is mentioned in this article.
Additional Disclosure: I am long AST and may purchase additional shares within the next 72 hours.
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