Capricor hits primary endpoint in Duchenne extension study, readying its FDA pitch
It has been a very bumpy ride for Capricor Therapeutics over the past several years, including a halted J&J partnership and venture into penny stock status. However, the latest news from the San Diego-based biotech shows that its experimental Duchenne drug may be a durable, long-term treatment.
The company posted the one-year results from its open-label extension study for their candidate dubbed CAP-1002. The extension from its Phase II study, named HOPE-2, was conducted in boys and young men with later-stage Duchenne muscular dystrophy who paused and then resumed treatment after a year. Researchers measured patients in the one-year follow-up against the original study’s treatment arm effect and in two subsets of the original placebo group.
Capricor’s drug hit the primary endpoint on an upper limb muscle function scale when compared to all three groups. When pitted against the treatment arm, CAP-1002 induced a statistically significant difference in the scale, good for a p-value of p=0.023. Against the HOPE-2 placebo group, the extension trial recorded a p-value of p=0.015. Against the group of placebo patients who remained off-treatment, CAP-1002 posted a value of p=0.006.
The biotech said CAP-1002 was made available to all 20 patients originally enrolled in the HOPE-2 study. Of those, 13 entered the extension and 12 completed the first year of follow-up. As in HOPE-2, CAP-1002 was administered quarterly and the current results are from the one-year analysis.
“The open-label extension phase of the HOPE-2 study is fairly unique in its design in that all patients were off CAP-1002 or placebo for a mean of approximately one year before resumption or initiation of therapy. These data suggest patients on CAP-1002 accumulate benefit over time where their skeletal muscle function is better preserved which may indicate a long-term potential benefit of CAP-1002,” said Linda Marbán, Capricor’s CEO, in a statement.
Capricor’s candidate consists of allogeneic cardiosphere-derived cells, or CDCs, a type of progenitor cell to exert potent immunomodulatory activity and is being investigated for its potential to modify the immune system’s activity to encourage cellular regeneration. The biotech is charging ahead with the candidate, planning to present these data to the FDA.
In March, the results from the Phase II were posted in The Lancet, favoring CAP-1002 over placebo in the muscle function scale with a value of p=0.014. Cardiac MRI assessments showed improvements in heart function and structure with CAP-1002 treatment.
In January, the company signed a collaboration with Japanese pharma company Nippon Shinyaku. Capricor saw a $30 million upfront payment to run the Phase III trial for CAP-1002 while leaving the door open for $705 million in potential milestones.
The latest news out of Capricor has seemed to bring investors some positivity as the company $CAPR has seen its stock rise 41% since the start of the year.
But Capricor has not been without setbacks. In 2018, the biotech noted that it had voluntarily halted a clinical trial of a stem cell therapy the company previously hoped would tamp down on the damaging inflammation associated with DMD. The halt was called following a “severe allergic reaction” that occurred during infusion.
And in 2019, the company said it would explore strategic alternatives for one or more of its products and cut 21 jobs to keep financially afloat, but had resumed dosing in its DMD trial.