Cell Therapy Well-Tolerated in Severe COVID-19
The same therapy by Capricor Therapeutics has demonstrated efficacy in Duchenne muscular dystrophy.
The cell therapy CAP-1002 was well-tolerated in hospitalized patients with severe COVID-19, according to new data from the phase 2 INSPIRE trial (NCT04623671) announced by Capricor Therapeutics.1
“We were pleased that CAP-1002 was shown to be safe and well-tolerated in this group of patients that were profoundly ill with symptoms of COVID-19 which bodes well for the general safety and tolerability of infused CAP-1002 even in this heterogeneous and severely sick population of patients,” Linda Marbán, PhD, chief executive officer, Capricor, said in a statement.1
The double-blind INSPIRE trial enrolled 62 randomized patients, 32% of which were admitted to the ICU prior to intravenous treatment with CAP-1002. Most patients (82%) met the World Health Organization ordinal scale criteria of severe disease (range, 0-8; median, 5). Data from the trial demonstrate that the cell therapy was well-tolerated and consistent with other evaluations of the therapy and the trial met its primary endpoint of safety.
Investigators also assessedexploratory efficacy of CAP-1002 but did not find significant differences. Six deaths occurred in the placebo group and 5 occurred in the CAP-1002 group, for an overall mortality rate of 20% (n =11).
READ MORE: COVID-19 Cell Therapy Trial Completes Dosing
“In light of the effectiveness and widespread use of available vaccines, the reduction in mortality and the decline in COVID-19 hospitalization rates in the United States, we will continue to evaluate the next steps for this program. Further, as we continue to analyze this dataset in more detail, we will remain open to discussing potential partnering opportunities should they become available,” Marbán added.1
CAP-1002 is a cardiosphere-derived cell therapy that Capricor is exploring in multiple indications. The therapy has been evaluated in a phase 1/2 trial for the treatment of Duchenne muscular dystrophy (DMD)-associated cardiomyopathy (HOPE trial, NCT02485938) as well as a phase 2 trial for the treatment of DMD (HOPE-2 trial, NCT03406780).
Data on CAP-1002's efficacy in DMD was published in The Lancet in March 2022.2 Evaluable patients treated with CAP-1002 had a 2.6 point-mean difference in Performance of Upper Limb (PUL) score change at 12 months from baseline compared to placebo (percentile difference 36.2 [92% CI, 12·7–59·7]; P = .014). Investigators also observed a trend of improved cardiac function in patients treated with CAP-1002, however this was not statistically significant.
The therapy was well-tolerated, with 3 patients experiencing infusion-related hypersensitivity reactions without long-term sequelae and 1 patient discontinuing therapy due to a severe allergic reaction. There were no other serious adverse events.
“Robust research is imperative to truly usher in a new class of treatments for these patients in dire need and historically Duchenne trials have not focused of the most severely affected non-ambulatory patients with the greatest disease burden,” first author and principal investigator Craig McDonald, MD, professor and chair, Department of Physical Medicine and Rehabilitation, University of California – Davis, said in an earlier statement.3 “We believe that the publication of these Phase 2 results in an eminent publication such as The Lancet, represents a major achievement for the development of CAP-1002 and further validates the power of this cell therapy for all patients with DMD regardless of their stage of disease.”
