Monday, 28 April 2014

Athersys stem cell trial for Ulcerative colitis fails to "show meaningful benefit"

athersys bowel disease
Athersys, Inc. (Nasdaq:ATHX) today announced interim results from the Phase 2 clinical study of the administration of Athersys' MultiStem® cell therapy to treat refractory ulcerative colitis (UC) patients being conducted by Pfizer, Inc. The study results demonstrate favorable safety and tolerability for MultiStem through 8 weeks following treatment.

 However, the cell therapy failed to show meaningful benefit, following a single administration, in patients suffering from chronic, moderate-to-severe UC who have failed other therapies. These results reflect patient data 8 weeks following cell therapy or placebo administration and include the primary efficacy endpoints for the study. Additional 16-week results, including data about the impact from a second round of dosing for a subset of patients, longer term secondary clinical endpoints, and biomarker evaluation will be available after additional analysis has been completed.

Data highlights from the 8-week interim analysis include:
  • MultiStem cell therapy demonstrated favorable tolerability and safety profile through 8 weeks following treatment;
  • In this chronic, advanced UC patient population, a single IV administration of MultiStem did not show a statistically significant improvement compared to placebo in the primary efficacy endpoints - change in endoscopic score from baseline as measured by modified Baron score at 8 weeks and change in Mayo rectal bleeding subscore from baseline at 4 and 8 weeks; and
  • At 4 weeks, the proportion of responders on MultiStem was significantly greater than placebo but the benefit was offset by declines in a minority of MultiStem-treated patients such that overall benefit at all time points measured was not significant. Multiple additional secondary endpoints at week 4 and week 8 were also explored and there were no significant differences observed between the two groups including clinical remission and clinical response.

"These results confirm the consistent safety profile Athersys has seen in previous clinical studies involving MultiStem. This study was focused on a challenging patient population with chronic disease - ulcerative colitis patients who have become resistant, intolerant or unresponsive to other therapies.

These interim results tell us a single administration of MultiStem in this patient group, while safe, was not sufficient to have a meaningful clinical effect. Obviously, we are disappointed by the efficacy results. We anticipate additional data from the study over time from patients who have received further treatment, which may provide more insight into the factors at work and the potential relevance for MultiStem in this area. 
In the meantime, we continue to advance our other clinical and preclinical programs. We have a strong balance sheet, and are pursuing multiple exciting opportunities where we remain confident MultiStem and our other technologies will have an important impact." commented Dr. Gil Van Bokkelen, Chairman & CEO at Athersys. 

Phase 2 Clinical Study Design
The randomized, double-blind, placebo-controlled Phase 2 clinical trial is being conducted by Pfizer under a collaboration and license agreement with Athersys at sites in the United States, Canada and Europe. The study was conducted in two parts - a small dose selection phase involving 18 patients, followed by larger efficacy phase of 88 enrolled patients powered for the primary endpoints. Eligible patients had moderate-to-severe active UC with a Mayo score of 6 to 12 points and endoscopic score of at least 2 (as measured by modified Baron score) despite prior treatment with corticosteroids, immunosuppressants, or anti-TNF agents.

Subjects enrolled in the efficacy stage of the study received either MultiStem treatment or placebo initially, followed by a second round of treatment with MultiStem therapy or placebo at eight weeks. The primary endpoints for the study include incidence and severity of adverse events over 16 weeks, change in endoscopic score (as measured by modified Baron score) at week eight, and changes in the Mayo rectal bleeding sub-score at weeks four and eight. Additionally, there are multiple secondary and exploratory endpoints evaluating disease indicators and markers over 16 weeks and through the entire study period.

Of the 88 patients enrolled in the larger cohort, 48 patients were enrolled in to the MultiStem treatment group and 40 patients were enrolled in the placebo group. The mean disease duration for patients enrolled in the efficacy stage of the study was 10 years and the other baseline characteristics confirm a patient population with advanced ulcerative colitis.

Ulcerative colitis is a form of inflammatory bowel disease (IBD). Ulcerative colitis is a form of colitis, a disease of the colon (the largest portion of the large intestine), that includes characteristic ulcers, or open sores. The main symptom of active disease is usually constant diarrhea mixed with blood, of gradual onset. IBD is often confused with irritable bowel syndrome (IBS).


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