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The FDA's Center for Biologics Evaluation and Research director Vinay Prasad unilaterally canceled an advisory committee meeting for Capricor Therapeutics' Duchenne muscular dystrophy cell therapy due to skepticism about the treatment.

Beam Therapeutics received FDA orphan drug designation for its sickle cell disease treatment, providing regulatory incentives for this rare genetic disorder affecting hemoglobin production.

• Capricor Therapeutics achieves significant milestone as FDA grants priority review for Deramiocel, potentially accelerating approval timeline for Duchenne muscular dystrophy treatment. • The priority review designation underscores the urgent unmet medical need in DMD treatment, with a target action date expected within 6 months rather than the standard 10-month review period. • If approved, Deramiocel would represent a novel therapeutic approach for DMD patients, offering a potential new treatment option in the limited landscape of available therapies.

Capricor Therapeutics has completed its Biologics License Application (BLA) submission to the FDA for deramiocel to treat Duchenne muscular dystrophy (DMD) cardiomyopathy.

Capricor Therapeutics' deramiocel has been granted Orphan Drug and Advanced Therapy Medicinal Product (ATMP) designations by the EMA for treating Duchenne muscular dystrophy (DMD).

• Capricor Therapeutics' deramiocel has been granted Orphan Drug and Advanced Therapy Medicinal Product (ATMP) designations by the EMA for Duchenne muscular dystrophy (DMD). • These designations offer benefits such as market exclusivity and reduced regulatory fees, potentially accelerating deramiocel's development and market entry in Europe. • Deramiocel, comprising allogeneic cardiosphere-derived cells, has shown immunomodulatory, antifibrotic, and regenerative properties in clinical studies for DMD treatment. • Capricor is also advancing a rolling Biologics License Application (BLA) with the FDA for deramiocel, targeting full approval for DMD-cardiomyopathy by the end of the year.

• The European Medicines Agency (EMA) has granted Orphan Drug Designation to deramiocel for treating Duchenne muscular dystrophy (DMD), providing market exclusivity and reduced regulatory fees. • Deramiocel also received Advanced Therapy Medicinal Product (ATMP) designation from the EMA, streamlining development and offering regulatory support for this cell-based therapy. • These EMA designations, along with FDA Orphan Drug and RMAT designations, aim to secure market exclusivity for deramiocel in key global markets. • Capricor Therapeutics is advancing deramiocel through Phase 3 clinical development and has initiated a rolling BLA submission with the U.S. FDA.

Capricor Therapeutics is pursuing accelerated FDA approval for its cell therapy to improve heart function in Duchenne muscular dystrophy patients.

• Wave Life Sciences reported positive interim results for WVE-N531, an exon-skipping oligonucleotide, showing promising dystrophin expression in patients with Duchenne muscular dystrophy. • Sarepta Therapeutics expanded the label for its gene therapy Elevidys to include all patients aged four and older with mutations in the dystrophin gene. • Italfarmaco's Duvyzat (givinostat), the first nonsteroidal treatment for all genetic variants of Duchenne, received FDA approval based on phase 3 trial results. • Avidity Biosciences' delpacibart zotadirsen (del-zota) demonstrated a significant increase in dystrophin production and reduction of creatine kinase levels in a phase 1/2 trial.

• Capricor Therapeutics plans to file a Biologics License Application (BLA) with the FDA for deramiocel to treat Duchenne muscular dystrophy (DMD) cardiomyopathy. • The BLA will be supported by cardiac data from Phase 2 HOPE-2 and HOPE-2 OLE trials, compared with natural history data. • Capricor has initiated its rolling submission process with the FDA for deramiocel, with completion expected by the end of 2024. • Deramiocel has shown immunomodulatory, antifibrotic, and regenerative actions in dystrophinopathy and heart failure in clinical studies.