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Capricor Therapeutics (NASDAQ:CAPR) reported 18-month outcomes from its ongoing HOPE-2 open label extension (OLE) research in sufferers with later-stage Duchenne muscular dystrophy (DMD).
DMD is genetic dysfunction characterised by progressive muscle degeneration and weak point as a result of alterations in a protein referred to as dystrophin.
The corporate stated that information from the OLE research continues to confirmed illness modification with statistically vital variations within the Efficiency of the Higher Limb (PUL model 2.0) scale within the CAP-1002 authentic therapy group, in comparison with the unique placebo group from HOPE-2.
This confirmed long-term profit in skeletal muscle operate, as per the corporate.
As well as, illness development was diminished equally in each teams as soon as sufferers started therapy within the OLE, in accordance with the corporate.
Capricor famous that the research had met the principle objective on the one-year timepoint and continues to point out statistically vital enhancements on the PUL scale for sufferers on CAP-1002 at 18-months.
Within the research, CAP-1002 was made accessible to the unique 20 sufferers enrolled in HOPE-2. Of these, 13 entered and 12 accomplished 18-months of research follow-up.
“Additional, the long-term efficacy and potential illness modification impact will increase our medical bundle as we proceed on our regulatory pathway in the direction of potential approval of CAP-1002 for therapy of sufferers with DMD,” stated Capricor CEO Linda Marbán.
CAP-1002 continued to point out a constant security profile and was well-tolerated within the research, Capricor added.
Capricor stated it’s at present conducting a section 3 trial referred to as HOPE-3.
CAPR -7.68% to $4.21 premarket Jan. 25
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