Express significantly relieves the symptoms of advanced DMD patients, and the Phase 2 clinical results of cell therapy are positive

▎Editor of WuXi AppTec's content team On September 24, 2021, Capricor Therapeutics announced the final results of a phase 2 clinical trial of cell therapy CAP-1002 for the treatment of patients with advanced Duchenne muscular dystrophy (DMD)
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The trial reached the primary efficacy endpoint of slowing down the patient's lower limb function.
Improvements in other endpoints related to skeletal muscle and heart function also showed a slowdown in disease progression
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Therefore, the company plans to advance this therapy into a pivotal Phase 3 clinical trial
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Duchenne muscular dystrophy is a rare and fatal genetic disease
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The patient has a mutation in the gene encoding dystrophin on the X chromosome, resulting in a deficiency of functional dystrophin
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The lack or defect of dystrophin causes chronic damage and inflammation during muscle contraction, and affects muscle regeneration
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CAP-1002 is an allogeneic cell therapy.
They are composed of heart-derived cells (CDCs).
CDCs are a special cell group containing cardiac progenitor cells
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They release exosomes containing microRNA, non-coding RNA and protein to improve muscle scarring or fibrosis and cardiac function in patients with DMD
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CAP-1002 also shows effective immunomodulatory activity and may promote cell regeneration
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▲Introduction to CAP-1002 (picture source: Capricor Therapeutics company official website) This trial was conducted in patients in the late stage of DMD.
About 80% of patients were unable to walk, and all patients received stable steroid therapy
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The final data analysis showed that after receiving 4 CAP-1002 treatments for 1 year, the index for testing upper limb function (PUL v1.
2) reached the primary efficacy endpoint, the patient’s upper limb function decline rate was slowed by 71%, and other skeletal muscle and heart measurement indexes There has also been improvement
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The specific results of the trial are shown in the table below: Image source: References [1] Professor Craig McDonald, the principal investigator of this clinical trial, commented: “The results of this ground-breaking trial are exciting and we see that CAP-1002 is capable This results in statistically significant changes in patients' skeletal muscle and heart function
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For these patients with limited treatment options, our data supports that CAP-1002 becomes an important treatment option and may slow the progression of Duchenne muscular dystrophy
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"Reference: [1] Capricor Therapeutics Announces Positive Final Data From its Phase 2 HOPE-2 Trial in Patients with Duchenne Muscular Dystrophy Treated with CAP-1002.
Retrieved September 24, 2021, from https:// news-release/2021/09/24/2302866/0/en/Capricor-Therapeutics-Announces-Positive-Final-Data-From-its-Phase-2-HOPE-2-Trial-in-Patients-with-Duchenne- Muscular-Dystrophy-Treated-with-CAP-1002.
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