HOPE-3 cardiac data: the primary result, the missed secondary, and the small imaging set
Capricor release, 9 November 2026. Deramiocel in Duchenne muscular dystrophy. PDUFA date 22 November 2026.
Primary endpoint
Met
Upper limb function
106 patients, 12 months
Key secondary
Missed, p=0.09
LVEF, all patients
+1.8 points vs placebo
Supportive / status
Nominal p-values
LGE on 9 vs 13
PDUFA 22 November 2026
Deramiocel is investigational and not approved. Natural-history comparisons are post hoc and non-randomized.
Figures as reported by Capricor on 9 November 2026. Deramiocel is investigational. Subgroup and LGE p-values are nominal.
Key LVEF secondary missed: p=0.09.
Subgroup and scar data are nominal.
On 9 November Capricor reported that HOPE-3 cardiac data for Deramiocel in Duchenne muscular dystrophy won an AHA award, per its company release. The key secondary endpoint, LVEF in all patients, did not reach significance (p=0.09).
What was reported
HOPE-3 randomized 106 patients with DMD to Deramiocel or placebo for 12 months. Capricor says the trial met its primary endpoint of upper limb function. The key secondary endpoint was change in LVEF in the intent-to-treat population.
Versus placebo, LVEF in all patients was 1.8 percentage points higher (p=0.09), which did not reach statistical significance. In the cardiomyopathy subgroup, described as prespecified, LVEF was 2.8 percentage points higher (p=0.02, nominal).
On cardiac MRI late gadolinium enhancement (LGE), Deramiocel patients had 3.0 fewer new scarred segments than placebo (p=0.02, nominal). That comparison included 9 Deramiocel and 13 placebo patients.
Capricor also showed a comparison with matched patients from a natural history dataset (DMDCCC). That comparison is post hoc and non-randomized.
One-year HOPE-3 results were published in The Lancet in July 2026. The Biologics License Application is under FDA review with a PDUFA target action date of 22 November 2026. Deramiocel is investigational and not approved.
What this does and does not show
The key secondary endpoint missed. That is the headline fact for cardiac function, and an award does not change it.
The cardiomyopathy subgroup and LGE p-values are nominal, meaning not adjusted for multiple comparisons. The LGE comparison rests on 9 versus 13 patients, so the estimate rests on few patients.
The natural history comparison is post hoc and non-randomized, with 9 Deramiocel versus 62 comparator patients for LGE. Its p-values are also nominal. It can generate a hypothesis but cannot replace the randomized result.
What GTC would ask
Which cardiac claims, if any, is the sponsor asking FDA to rely on, and which analyses were prespecified? The release does not say how the cardiac data feed the BLA review.
The placebo comparison lists 39 versus 44 patients for LVEF in all patients, fewer than the 106 randomized. Ask why, and how missing data were handled.
For investors: the PDUFA date is 22 November, so the regulatory outcome will arrive within weeks. Judge the cardiac package on the missed key secondary and the small LGE sample, not on the award.
Practical takeaway
When a key secondary misses, check whether the supportive imaging is nominal and small before it carries weight in your thesis. Then list which avoidable hurdles in the package the sponsor can still close.
Related GTC reading
Frequently asked questions
Did HOPE-3 meet its key secondary cardiac endpoint?
The key secondary endpoint, change in LVEF in all patients versus placebo, was 1.8 percentage points higher with Deramiocel. It did not reach statistical significance (p=0.09), according to Capricor.
What did the cardiomyopathy subgroup show?
LVEF was 2.8 percentage points higher with Deramiocel than placebo (p=0.02, nominal) in the cardiomyopathy subgroup. The p-value is not adjusted for multiple comparisons.
How reliable are the LGE scar findings?
LGE showed 3.0 fewer new scarred segments versus placebo (p=0.02, nominal), but the comparison included only 9 Deramiocel and 13 placebo patients. Nominal p-values are not adjusted for multiple comparisons.
What is the natural history comparison?
Capricor compared treated patients with matched patients from the DMDCCC natural history dataset. The comparison is post hoc and non-randomized, so it is supportive context rather than randomized evidence.
Is Deramiocel approved?
No. Deramiocel is investigational and not approved. Capricor says its BLA is under FDA review with a PDUFA target action date of 22 November 2026.
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