CAP-1002 Shows Benefits in Arm Function in DMD After 18 Months

New HOPE-2 trial data also show treatment safe, well-tolerated

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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After 18 months, treatment with the experimental cell therapy CAP-1002 continues to show beneficial effects on upper limb function in people with Duchenne muscular dystrophy (DMD), according to new data from the open-label extension phase of the HOPE-2 study.

The new trial data also suggests a consistent safety profile, with most patients tolerating CAP-1002 well, the therapy’s developer Capricor Therapeutics, noted in a company press release.

“We are extremely pleased by the robust and consistent results observed to date, which, together with the favorable safety/tolerability profile, suggest that CAP-1002 holds promise as a potential anchor therapy for DMD patients,” said Linda Marbán, PhD, Capricor’s CEO.

Capricor last year launched a Phase 3 trial called HOPE-3 ( NCT05126758), which is testing CAP-1002 against a placebo in males with DMD ages 10 and up. The study is still recruiting participants at 10 centers in the U.S.

“We thank the patients, their families, caregivers, and the broader Duchenne community for working with us on this promising therapy,” Marbán said.

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Testing CAP-1002 in the HOPE studies

The original Phase 2 HOPE-2 study (NCT03406780) enrolled 20 boys and young men with relatively advanced DMD, most of whom were unable to walk. The participants were randomly assigned to receive CAP-1002 or a placebo, administered by infusion into the bloodstream (intravenously, or into the vein), every three months for one year.

Results showed that, when compared with the placebo, CAP-1002 significantly improved arm and hand function, as measured with the Performance of the Upper Limb (PUL) scale.

Participants who completed the original HOPE-2 study had the option to enroll in its open-label extension (OLE), where all are being treated with CAP-1002 and monitored for long-term safety and efficacy outcomes. There was a gap in treatment of about one year between the end of the original trial and the start of the OLE.

Of 13 participants who entered into the OLE, 12 have completed 18 months of follow-up in the study. Data show that participants continually on CAP-1002 still have better PUL scores — meaning better arm function — than those patients given the placebo during the original trial.

“Taken together, these data build on the impressive results from HOPE-2 and suggest that patients accumulate benefit over time with steady preservation of skeletal muscle functions, which underscore the potential long-term benefit of CAP-1002,” said Craig McDonald, MD, a professor at the University of California, Davis, and principal investigator on the study.

Additionally, disease progression has slowed for all patients in the OLE, regardless of whether they were on placebo or active treatment in the original study, according to Capricor.

The long-term efficacy and potential disease modification effect will augment our clinical package as we continue on our regulatory pathway towards potential approval of CAP-1002 for treatment of patients with DMD.

Safety data from the 18-month OLE were consistent with earlier findings; CAP-1002 has overall been well-tolerated according to Capricor.

“In the OLE phase of the HOPE-2 study, we observed statistically significant slowing of the progression of disease across patients treated with CAP-1002,” Marbán said.

That data will be used by Capricor in seeking regulatory approval of the cell therapy.

“The long-term efficacy and potential disease modification effect will augment our clinical package as we continue on our regulatory pathway towards potential approval of CAP-1002 for treatment of patients with DMD,” Marbán said.

McDonald noted that the HOPE-2 extension study is ongoing.

“We are encouraged by the improvements in upper limb function in the treatment group at 18-months and will continue to treat and follow these patients to further evaluate the disease modifying potential of CAP-1002,” he said.

“DMD is a progressive disease associated with loss of function over time and there is a critical need for treatment options to slow the rate of decline and preserve upper limb function to enable key everyday activities,” McDonald added.