Since Celyad failed to reach its primary endpoints in a stem cell therapy test in heart patients back in June, CardioCell has felt the pressure to deliver and over the weekend posted broadly positive data. But questions, and the need for more clinical data, remain.
The San Diego, CA-based biotech has published new top-line data from the ESC Congress in Rome that used its allogeneic mesenchymal stem cells, delivered through an IV, to heart failure patients with nonischemic cardiomyopathy.
The biotech used a number of endpoints to assess efficacy, including a 6-minute walk test and the KCCQ Clinical Summary score, both of which hit statistical significance, although exact figures were not released.
There was however only a “trend to improvement” in KCCQ Functional Status score, with the data also showing the stem cell therapy can suppress inflammation, something the biotech stresses is “noteworthy because inflammation is believed to importantly contribute to [heart failure] HF progression.” There was in addition a statistically significant reduction in natural killer cells.
But overall, there was no difference in cardiac structure and function and the company had to dig deep with some “exploratory data analysis” to show that there was a statistically significant improvement in the left ventricular end systolic and diastolic volumes in the stem cell-treated groups--something it said that was not seen in the placebo-treated group.
The single-blind, placebo-controlled, crossover study gave the treatment to just 22 enrolled patients with nonischemic cardiomyopathy.
The test was set up to randomize patients, with both CardioCell's treatment and the placebo given intravenously. At 90 days after the first injection, the control group received CardioCell's treatment, and the original treatment group received the placebo solution.
Dr. Mihai Gheorghiade, a protocol co-designer of this study and a member of CardioCell's scientific advisory board, said these data showed that its therapy “improved well-being of the patients that have already been on evidence-based HF therapy.” He added: “This is very innovative and marks a new beginning in HF management, especially in light of negative results of many drug-intervention trials.”
There have also been other stem cell therapy failures in heart failure, including over the summer when Celyad missed the primary endpoints in its CHART-1 study in certain heart patients. It did however find that a “positive trend was clearly identifiable,” with its C-Cure treatment, meaning it could still try for an EMA application using the subgroup analyses.
CardioCell said future studies will necessarily be needed in larger studies with a focus on clinical endpoints to assess just how well its treatment works, and most importantly whether it can boost how well the heart works in these patients.
“Based on these promising clinical-trial findings, CardioCell is excited to be developing larger studies based on IV delivery of our [therapy] for heart failure indications, including both non-ischemic and ischemic cardiomyopathy,” says Sergey Sikora, CEO of CardioCell and co-author of the study.
“We are very encouraged by the degree of statistically significant improvement in patient health status, and we look forward to exploring whether multiple IV treatments will lead to further improvement, including cardiac function.”
CardioCell gets its therapy from parent company Stemedica, which are derived from bone-marrow.
This is a similar approach used by San Carlos, CA-based BioCardia, which earlier this year dropped plans for a $50 million IPO, which also uses a bone marrow-derived therapy. In a previous Phase II study with 30 patients its treatment CardiAMP “supported efficacy relative to patient quality of life and functional capacity, which was shown to be both statistically and clinically significant.”