Capricor Therapeutics (NASDAQ:CAPR) reported 18-month results from its ongoing HOPE-2 open label extension (OLE) study in patients with later-stage Duchenne muscular dystrophy (DMD).
DMD is genetic disorder characterized by progressive muscle degeneration and weakness due to the alterations in a protein called dystrophin.
The company said that data from the OLE study continues to showed disease modification with statistically significant differences in the Performance of the Upper Limb (PUL version 2.0) scale in the CAP-1002 original treatment group, compared to the original placebo group from HOPE-2.
This showed long-term benefit in skeletal muscle function, as per the company.
In addition, disease progression was reduced equally in both groups once patients began treatment in the OLE, according to the company.
Capricor noted that the study had met the main goal at the one-year timepoint and continues to show statistically significant improvements on the PUL scale for patients on CAP-1002 at 18-months.
In the study, CAP-1002 was made available to the original 20 patients enrolled in HOPE-2. Of those, 13 entered and 12 completed 18-months of study follow-up.
“Further, 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,” said Capricor CEO Linda Marbán.
CAP-1002 continued to show a consistent safety profile and was well-tolerated in the study, Capricor added.
Capricor said it is currently conducting a phase 3 trial called HOPE-3.
CAPR -7.68% to $4.21 premarket Jan. 25