NS Pharma, Inc.

FDA Grants Orphan Drug Designation to NS-229 for Rare Vasculitis Condition

a month ago

• The FDA has granted Orphan Drug Designation to NS-229, a selective JAK1 inhibitor being developed by NS Pharma for eosinophilic granulomatosis with polyangiitis (EGPA). • NS-229 targets the JAK1 enzyme to regulate immune cell function and prevent tissue damage in EGPA, a rare autoimmune disease affecting between 5,600 and 14,500 Americans. • The designation provides NS Pharma with seven years of market exclusivity, supporting the ongoing Phase 2 global clinical trial of the investigational therapy.

REGENXBIO and Nippon Shinyaku Partner to Advance Gene Therapies for MPS I and MPS II

4 months ago

• REGENXBIO and Nippon Shinyaku have entered an exclusive partnership to develop and commercialize RGX-121 for MPS II and RGX-111 for MPS I. • Nippon Shinyaku will lead commercialization in the U.S. and Asia, while REGENXBIO will continue to lead clinical development and manufacturing. • The agreement includes an upfront payment of $110 million to REGENXBIO, with potential milestone payments reaching up to $700 million. • RGX-121 and RGX-111 are AAV-based gene therapies designed to address the underlying enzyme deficiencies in MPS II and MPS I, respectively.

Capricor Therapeutics Completes FDA Submission for Deramiocel in DMD Cardiomyopathy

5 months ago

• Capricor Therapeutics has completed its Biologics License Application (BLA) submission to the FDA for deramiocel to treat Duchenne muscular dystrophy (DMD) cardiomyopathy. • The BLA is supported by data from Phase 2 HOPE-2 and HOPE-2 Open Label Extension (OLE) trials, showing attenuation of cardiac implications of DMD. • The FDA has been requested to grant priority review, potentially reducing the review period to six months from the standard ten months. • The BLA submission triggers a $10 million milestone payment to Capricor from its distribution partner, Nippon Shinyaku.

Capricor's Deramiocel Receives Orphan Drug and ATMP Status from EMA for Duchenne Muscular Dystrophy

6 months ago

• Capricor Therapeutics' deramiocel has been granted Orphan Drug and Advanced Therapy Medicinal Product (ATMP) designations by the EMA for treating Duchenne muscular dystrophy (DMD). • These designations provide Capricor with benefits such as market exclusivity and regulatory support, potentially reducing time to market for deramiocel in Europe. • Deramiocel is currently under review by the U.S. FDA, with a Biologics License Application (BLA) submitted for treating DMD-cardiomyopathy. • Deramiocel has demonstrated immunomodulatory, antifibrotic, and regenerative actions in preclinical and clinical studies, targeting the underlying pathology of DMD.

EMA Grants Orphan Drug and ATMP Status to Capricor's Deramiocel for Duchenne Muscular Dystrophy

6 months ago

• 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 Advances Deramiocel for Duchenne Muscular Dystrophy Cardiomyopathy

8 months ago

• 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.

Capricor's Deramiocel Shows Sustained Benefits in Duchenne Muscular Dystrophy

a year ago

• Capricor Therapeutics' deramiocel demonstrated a 47% reduction in disease progression in skeletal muscle over three years in DMD patients. • The therapy also showed improvements in cardiac function, particularly in patients with higher ejection fractions at baseline. • Capricor plans to engage with the FDA in Q3 2024 to discuss the Biologics License Application (BLA) filing for deramiocel. • A Phase III pivotal trial is ongoing, with topline data expected in Q4 2024, potentially reshaping the DMD treatment landscape.

Capricor's CAP-1002 Shows Sustained Benefits in Duchenne Muscular Dystrophy After 3 Years

a year ago

• Capricor Therapeutics' CAP-1002 demonstrates sustained benefits in Duchenne muscular dystrophy (DMD) patients after three years of treatment in the HOPE-2 open-label extension study. • The study showed a statistically significant reduction in the decline of upper limb function (PUL 2.0) and stabilization of left ventricular ejection fraction (LVEF) compared to an external comparator. • CAP-1002 continues to exhibit a consistently well-tolerated safety profile throughout the extension study, supporting its potential as a long-term treatment for DMD. • Topline results from the Phase 3 HOPE-3 pivotal trial are expected in the fourth quarter of 2024, with potential for BLA submission to the FDA.