NIPPON SHINYAKU CO

NS Pharma's parent company Nippon Shinyaku has finalized a strategic partnership with Boston Children's Hospital to develop innovative therapies for rare diseases.

Four innovative therapies are advancing through clinical trials to address critical unmet needs in MPS I treatment, particularly targeting central nervous system complications that current therapy Aldurazyme cannot reach.

• REGENXBIO has closed a non-dilutive royalty bond agreement with Healthcare Royalty for up to $250 million, receiving $150 million upfront that extends its cash runway into early 2027. • The deal monetizes select royalties from ZOLGENSMA for SMA and payments from gene therapies for MPS disorders, while REGENXBIO retains other funding opportunities including a potential Priority Review Voucher. • This strategic financing supports REGENXBIO's late-stage pipeline development, including RGX-121 for MPS II, RGX-202 for Duchenne muscular dystrophy, and ABBV-RGX-314 for wet AMD.

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).

Durham-based Atsena Therapeutics has raised $150 million in an oversubscribed Series C financing led by Bain Capital to advance gene therapies for inherited blindness conditions.

REGENXBIO and Nippon Shinyaku have entered an exclusive partnership to develop and commercialize RGX-121 for MPS II and RGX-111 for MPS I.

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 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 FDA has approved obecabtagene autoleucel (obe-cel), a CD19-directed CAR-T therapy, for adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (r/r B-cell ALL). • Approval was based on the Phase 1b/2 FELIX trial, which demonstrated overall complete remission rates above 60% and durable remissions exceeding 12 months. • Clinical holds on CARsgen's BCMA-directed CAR-T therapy zevorcabtagene autoleucel and Claudin18.2-directed CAR-T satricabtagene autoleucel have been removed by the FDA.

Atsena Therapeutics and Nippon Shinyaku have entered an exclusive license agreement to commercialize ATSN-101 in the U.S. and Japan, a gene therapy for Leber congenital amaurosis (LCA1).