Athersys, Inc. (NASDAQ:ATHX) announced positive results from the analysis of one-year follow-up data from its Phase 2 clinical study of the intravenous administration of MultiStem® cell therapy to treat patients who have suffered an ischemic stroke. Dr. David Hess, lead clinical investigator of this study and a stroke specialist and Chairman of the Department of Neurology at the Medical College of Georgia, Augusta University, presented the summary results today at the 2016 International Stroke Conference in Los Angeles. The one-year data demonstrates that MultiStem-treated subjects on average continued to improve through one year and had a significantly higher rate of “Excellent Outcome” (defined clinically as attaining mRS 0-1, NIHSS 0-1 and BI ≥95) compared to placebo subjects at one year when evaluating all subjects enrolled in the study (p=0.02), i.e., the intent-to-treat population. The relative improvement in Excellent Outcomes was even more pronounced in the patients who received MultiStem treatment within 36 hours of the stroke (p < 0.01).
“We are particularly excited by the one-year follow-up results because they show that MultiStem treatment can significantly increase the number of patients who have an Excellent Outcome, meaning complete or nearly full recovery, over the standard of care when considering all subjects in the trial,” commented Dr. Gil Van Bokkelen, Chairman & CEO at Athersys. “The one-year data continues to confirm that MultiStem treatment is well tolerated and is associated with continued improvement of other measures of function through one year. As we saw in the 90-day interim analysis results announced last April, patients who received MultiStem treatment within 36 hours of the stroke did substantially better than placebo patients and later treatment MultiStem subjects. As a result, we will continue to focus our ongoing clinical development on treatment within 36 hours of the stroke.”
Data highlights from the 365-day follow-up data analysis include:
- MultiStem treatment continued to be well tolerated through 365 days;
- Among all subjects who received MultiStem treatment (n=65), 23.1% of patients achieved an Excellent Outcome at 365 days, compared to 8.2% of patients who received placebo (n=61), and the 14.9% difference was statistically significant (p=0.02) and compared favorably to the 8.8% difference at 90 days;
- Among patients who received MultiStem treatment within 36 hours following the stroke, 29.0% achieved Excellent Outcomes (n=31), and compared to all placebo subjects (n=61), the 20.8% difference was significant (p < 0.01) and also greater than the 9.5% difference at 90 days;
|Proportion of Subjects with Excellent Outcome at Day 90 and Over One Year|
|Subjects||Day 90||Day 365|
|All MultiStem (n=65)||15.4||%||23.1||%|
|All Placebo (n=61)||6.6||%||8.2||%|
|Difference with all placebo||8.8||%||14.9%*|
|Early Treatment with MultiStem (n=31)||16.1||%||29.0||%|
|Difference with all placebo||9.5||%||20.8%**|
*p = 0.02, **p < 0.01
- Substantial improvements were also observed in the Barthel Index, which is the clinical scale used to assess the ability of patients to live independently. Among all subjects (65 MultiStem, 61 placebo), 61.5% of MultiStem patients had an excellent outcome in the Barthel Index (≥95), compared to 44.3% of placebo patients (p=0.05); furthermore, 67.7% of the subset of MultiStem patients who had treatment within 36 hours (n=31) achieved an excellent Barthel outcome, representing a 23.4% difference with the incidence for all placebo patients (p=0.03); and
- Among MultiStem patients who did not achieve an Excellent Outcome at 365 days, there appears to be meaningful benefit from the treatment relative to standard of care, with reductions in average initial hospitalization days, mortality, life threatening adverse events and infections. For example, comparing all such MultiStem and placebo subjects, MultiStem-treated patients had 1.6 fewer average hospitalization days, and an 11% lower proportion of patients with death or life threatening adverse events. In addition, when comparing subjects receiving early treatment with MultiStem against all placebo subjects, MultiStem patients had an average of 2.9 fewer hospitalization days, and an 11.4% lower incidence of death or life threatening adverse events. Further, such MultiStem patients appear to have better functional improvement than these placebo patients over one year, as evidenced by a higher proportion of excellent Barthel Index outcomes (≥95), 50% for MultiStem subjects (and 55% for early treatment MultiStem), compared to 39% for placebo subjects.
“Achievement of an Excellent Outcome is important because it means that a patient has substantially improved in each of the three clinical rating scales used to assess patient improvement and has regained the ability to live and function independently with a high quality of life,” continued Van Bokkelen. “Furthermore, when evaluating patients that either received no reperfusion therapy, treatment with tPA alone, or mechanical reperfusion alone, we observed a greater than five-fold increase in the proportion of patients that achieved an Excellent Outcome at one year when comparing subjects that received MultiStem treatment within 36 hours versus placebo.”
Phase 2 Clinical Study Design
The randomized, double-blind, placebo-controlled Phase 2 clinical trial was conducted at sites in the United States and the United Kingdom. The study was conducted in two parts – a small dose selection phase involving 16 patients in two cohorts, followed by larger efficacy phase of 118 patients. The evaluable patient population included 8 patients from cohort 2 and the cohort 3 patients, which all received a high dose of treatment or placebo.
The study enrolled subjects who received intravenously either MultiStem treatment or placebo one to two days following the stroke. Functional and neurological deficit and recovery following the ischemic stroke were evaluated using three standard methods: the modified Rankin Score (mRS), a scale from 0-6 directed to assessing disability; the NIH Stroke Scale (NIHSS), a scale from 0-42 for evaluating neurological deficit; and the Barthel Index, assessing performance related to activities of daily living on a 100 point scale. See www.strokecenter.org/professionals/stroke-diagnosis/stroke-assessment-scales/ for additional information on these assessment scales. Additionally, other clinical, safety and biomarker data was collected over the assessment period. Of the patients evaluated in the study, 65 patients were in the MultiStem treatment group and 61 patients were in the placebo group, and among the MultiStem subjects, 31 received MultiStem treatment within 36 hours following the stroke. (Original Source)
Shares of Athersys are up nearly 19% in after-hours trading. ATHX has a 1-year high of $3.43 and a 1-year low of $0.90. The stock’s 50-day moving average is $1.16 and its 200-day moving average is $1.12.
On the ratings front, Athersys has been the subject of a number of recent research reports. In a report issued on January 8, WBB analyst Stephen Brozak maintained a Buy rating on ATHX, with a price target of $9, which implies an upside of 520.7% from current levels. Separately, on December 22, Maxim Group’s Jason Kolbert maintained a Buy rating on the stock and has a price target of $5.
According to TipRanks.com, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Stephen Brozak and Jason Kolbert have a total average return of 5.9% and -21.4% respectively. Brozak has a success rate of 40.9% and is ranked #812 out of 3610 analysts, while Kolbert has a success rate of 24.3% and is ranked #3607.
Athersys Inc is a biopharmaceutical company developing regenerative medicine. It is engaged in the discovery and development of therapies designed to extend and enhance human life.