NIPPON SHINYAKU CO
🇯🇵Japan
- Country
- 🇯🇵Japan
- Ownership
- Public
- Established
- 1919-09-23
- Employees
- 2.2K
- Market Cap
- $1.6B
REGENXBIO Secures $250 Million Royalty Monetization Deal to Advance Gene Therapy Pipeline
• 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.
FDA Grants Orphan Drug Designation to NS-229 for Rare Vasculitis Condition
• 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.
Atsena Therapeutics Secures $150M Series C Funding to Advance Ocular Gene Therapies for Inherited Blindness
• 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.
• The funding will primarily support ATSN-201 for X-linked retinoschisis (XLRS), while also advancing the company's preclinical pipeline and expanding its novel AAV gene therapy platform technology.
• Atsena has recently reported positive preliminary data from its ATSN-201 clinical trial and secured a partnership with Nippon Shinyaku to advance ATSN-101 for Leber Congenital Amaurosis type 1 (LCA1).
Wave Life Sciences to Seek FDA Approval for Duchenne Muscular Dystrophy Drug Following Promising Phase 2 Results
• Wave Life Sciences will pursue FDA accelerated approval for WVE-N531, an exon 53 skipping therapy for Duchenne muscular dystrophy, following positive Phase 2 trial results showing increased dystrophin production.
• The experimental treatment demonstrated substantial improvements in muscle health with a statistically significant improvement in "time to rise" functional tests compared to historical controls through 48 weeks of treatment.
• If approved, WVE-N531 could offer a potential monthly dosing regimen and become a new therapeutic option for approximately 8-10% of Duchenne patients with specific genetic mutations amenable to exon 53 skipping.
Global Clinical Trials for Myelodysplastic Syndrome Show Significant Industry Investment in 2025
• A comprehensive review of global Myelodysplastic Syndrome clinical trials reveals extensive research activity across G7 and E7 countries, with major pharmaceutical companies leading development efforts.
• The analysis highlights significant participation from industry leaders including Bristol-Myers Squibb, Novartis, and Sanofi, demonstrating strong commercial interest in MDS therapeutics.
• The report indicates evolving clinical trial landscapes across multiple regions, with detailed tracking of trial phases, enrollment trends, and success rates over the past five years.
REGENXBIO and Nippon Shinyaku Partner to Advance Gene Therapies for MPS I and MPS II
• 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.
Arvinas Advances Vepdegestrant into Phase 3 Trials for Breast Cancer and Updates Pipeline Milestones
• Arvinas plans to initiate two Phase 3 trials in 2025 for vepdegestrant in ER+/HER2- metastatic breast cancer, one in the first-line setting with atirmociclib and another in the second-line setting with a CDK4/6 inhibitor.
• Topline data from the Phase 3 VERITAC-2 monotherapy trial of vepdegestrant in second-line-plus ER+/HER2- metastatic breast cancer is anticipated in the first quarter of 2025.
• Arvinas is set to present initial data from the Phase 1 trial of ARV-393 in B-cell lymphomas and file an IND application for a novel PROTAC KRAS G12D degrader in 2025.
• Phase 1 trial with PROTAC LRRK2 degrader ARV-102 in patients with Parkinson’s disease has been initiated, with data expected to be presented in the first half of 2025.
Dyne Therapeutics' DYNE-101 Shows Promise in DM1 Trial, FDA Grants Fast Track Designation
• Dyne Therapeutics' DYNE-101 demonstrates compelling results in Phase 1/2 ACHIEVE trial for myotonic dystrophy type 1 (DM1).
• The FDA grants Fast Track designation to DYNE-101, expediting its development and regulatory review process.
• Dyne plans to initiate a global Registrational Expansion Cohort, aiming for U.S. Accelerated Approval submission in H1 2026.
• DYNE-251 for Duchenne muscular dystrophy (DMD) is also advancing, with potential regulatory submissions expected in early 2026.
Sarepta Seeks Accelerated Approval for DMD Gene Therapy SRP-9001
• Sarepta Therapeutics has submitted SRP-9001 (delandistrogene moxeparvovec) to the FDA for accelerated approval to treat ambulatory Duchenne muscular dystrophy (DMD) patients.
• The filing is based on positive data from early-stage studies, showing improvements in clinical function and a consistent safety profile, while awaiting Phase 3 EMBARK results.
• SRP-9001, a one-time gene therapy, delivers a shortened dystrophin gene via an AAV vector, addressing the underlying genetic defect in DMD patients.
• If approved, SRP-9001 would offer a one-time treatment option for DMD, contrasting with Sarepta's existing chronic exon-skipping therapies.
Capricor Therapeutics Completes FDA Submission for Deramiocel in DMD Cardiomyopathy
• 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.
Atsena Therapeutics Advances ATSN-201 Gene Therapy for X-Linked Retinoschisis with Phase I/II Trial
• Atsena Therapeutics has completed dosing in Part A of its Phase I/II LIGHTHOUSE study, which evaluates ATSN-201 for X-linked retinoschisis (XLRS).
• Part A of the study demonstrated structural and functional benefits in patients across all dose levels of ATSN-201, with no serious adverse events reported.
• The company has initiated Part B of the LIGHTHOUSE study, which will enroll both adult and pediatric participants to further assess safety and efficacy.
• ATSN-201 leverages AAV.SPR, a novel spreading capsid, to achieve therapeutic gene expression in photoreceptors, offering a potential treatment for XLRS.
Capricor Therapeutics Advances Deramiocel for Duchenne Muscular Dystrophy Cardiomyopathy
• 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.
CytomX Therapeutics Reports Promising Interim Phase 1 Data for CX-2051 Antibody-Drug Conjugate
• CytomX Therapeutics has announced encouraging interim Phase 1 data for CX-2051, their novel antibody-drug conjugate targeting EpCAM-expressing solid tumors, showing early signs of clinical activity.
• The dose-escalation study demonstrated a manageable safety profile with dose-limiting toxicities observed only at higher dose levels, supporting continued development of the therapeutic candidate.
• Preliminary efficacy signals were observed across multiple tumor types, with several patients achieving stable disease, suggesting potential clinical benefit in difficult-to-treat EpCAM-positive malignancies.
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