相关临床试验
2
2 进行中
药物批准
0
批准总数
监管机构
0
监管机构数
成立时间
N/A
尚未招募
2
100.0%
暂无批准数据
- Over 25 pharmaceutical companies are actively developing 30+ TGF-β inhibitors to address significant unmet needs in oncology and fibrosis where existing therapies show limited effectiveness. - Key pipeline advances include Keros Therapeutics initiating Phase III trials for elritercept in myelodysplastic syndromes and EpicentRx receiving FDA Fast Track designation for AdAPT-001 in soft tissue sarcoma. - Recent clinical milestones demonstrate promising efficacy across multiple indications, with companies like Isarna Therapeutics reporting positive Phase II results for retinal fibrosis treatment ISTH0036.
- Isarna Therapeutics' antisense therapy ISTH0036, which blocks TGF-β2 production, demonstrated positive results in treating retinal fibrosis in wet AMD and DME patients at the ARVO 2025 Annual Meeting. - Patients receiving intravitreal ISTH0036 injections every eight weeks experienced stable or improved visual acuity, reduced central retinal thickness, and significant decreases in fibrosis-associated hyperreflective material volume compared to standard anti-VEGF therapy. - Following these promising results, Isarna plans to discuss development strategy with US and EU regulators to advance ISTH0036 into Phase 2b/Phase 3 pivotal clinical studies as a potential first-in-class antifibrotic agent.
• Ionis Pharmaceuticals, a pioneer in antisense oligonucleotide (ASO) drugs, priced a $500 million IPO to fund clinical programs and commercial launches. • Isarna Therapeutics' lead candidate, ISTH0036, targeting TGF-β, shows promise in Phase 2 trials for wet AMD and DME, major ophthalmic conditions. • Regulus Therapeutics is advancing RGLS8429, an ASO targeting microRNA for autosomal dominant polycystic kidney disease (ADPKD), with positive Phase 1b data. • Sarepta Therapeutics, with three approved PPMO therapies for Duchenne muscular dystrophy (DMD), continues to expand its RNA-based therapeutic pipeline.
