NIPPON SHINYAKU CO

Nippon Shinyaku received rare pediatric disease designation for NS-050/NCNP-03, developed for Duchenne muscular dystrophy. NS-050/NCNP-03, an antisense oligonucleotide, aims to treat patients with gene mutations amenable to exon 50 skipping therapy, producing a functional dystrophin protein to suppress muscle function deterioration. Clinical trials are being prepared in Japan and the U.S.

Capricor Therapeutics reports 3-year HOPE-2 OLE study results showing deramiocel (CAP-1002) improves skeletal muscle and cardiac function in Duchenne muscular dystrophy patients. Significant benefits in upper limb function and cardiac measures observed. Pre-BLA meeting with FDA planned for Q3 2024 to expedite BLA filing.

Capricor Therapeutics announced a Pre-BLA meeting with the FDA in Q3 2024 for deramiocel (CAP-1002) to treat Duchenne Muscular Dystrophy (DMD), aiming to finalize BLA filing plans. The company also plans to present HOPE-2 OLE 36-month data at the PPMD Annual Conference. DMD, a severe genetic disorder, lacks a cure, highlighting the need for innovative treatments like deramiocel.

CAP-1002, an investigational cell therapy by Capricor Therapeutics, aims to improve muscle strength and heart health in Duchenne muscular dystrophy (DMD) patients through intravenous infusion. It uses cardiosphere-derived cells to promote muscle repair, reduce inflammation, and inhibit scarring. Currently in Phase 3 trials, CAP-1002 has shown promise in slowing disease progression and improving cardiac function, with ongoing studies to confirm its efficacy and safety.

Capricor Therapeutics reported positive 3-year results from the HOPE-2 study of CAP-1002 for Duchenne Muscular Dystrophy, showing benefits in skeletal muscle function and cardiac function stabilization. Results were shared with the FDA, supporting CAP-1002's long-term impact and safety profile.

Capricor Therapeutics' CAP-1002 trial for Duchenne muscular dystrophy continues after positive DSMB review. Plans to discuss expedited FDA approval in 2024. CAP-1002, derived from heart cells, aims to improve arm and heart function in DMD patients. Recruitment ongoing; top-line data expected late 2024.