Record mula sa primary source
Decision-gradeTopline na resulta
SLDB: interim na datos ng INSPIRE DUCHENNE; nakaplanong pulong sa FDA sa H1 2026
Ang 8-K ay nag-uulat ng positibong interim Phase 1/2 data mula sa 23 kalahok na nagpapakita ng mean 58% microdystrophin expression at malakas na ugnayan sa DAPC restoration at muscle-protection biomarkers. Ang kumpanya ay inuna ang kanilang planadong FDA meeting sa H1 2026 upang talakayin ang mga potensyal na registrational pathways, kasama ang accelerated approval, pagkatapos mag-dose ng 30 kalahok at simulan ang IMPACT DUCHENNE Phase 3 ex-U.S.
Mahahalagang fact
- Na-file
- Nob 3, 2025, 10:15 PM
- Kaganapan
- Topline na resulta
- Direksyon
- Positibo
- Kumpanya
SLDBSolid Biosciences- Asset
- SGT-003
- Pinagmulan
- SEC 8-K
- Kumpiyansa
- May source
- nabigyan ng dosis
- 23 kalahok hanggang Oct 31 2025
- nNOS IF
- katamtamang 26%
- Takbo ng LVEF
- normal na saklaw (60-69%)
- Pagbawas ng CK
- 34% sa Araw 90, 42% sa Araw 360
- Target na pagbibigay ng dosis
- 30 kalahok pagsapit ng unang bahagi ng 2026
- Bilang ng kopya ng vector
- 13 bawat nukleus
- Pagbawas ng cTnI
- 31% sa Araw 90, 70% sa Araw 360
- Microdystrophin IF
- katamtamang 51%
- Microdystrophin MS
- katamtamang 58%
- Microdystrophin WB
- katamtamang 58%
- Beta sarcoglycan IF
- katamtamang 50%
Sipi mula sa source
EX-99.1 2 d87050dex991.htm EX-99.1 EX-99.1 Exhibit 99.1 Solid Biosciences Reports Third Quarter 2025 Financial Results and Provides Update on INSPIRE DUCHENNE Clinical Trial Progress and Planned Regulatory Discussions - Duchenne (SGT-003): 23 participants have been dosed in the INSPIRE DUCHENNE trial as of October 31, 2025; Solid expects to have dosed 30 participants in total by early 2026, then plans to meet with the FDA to discuss potential registrational pathways in H1 2026 - - SGT-003 has been generally well tolerated using a steroid-only prophylactic immunomodulation regimen; cardiac safety monitoring continued to show reduction in cardiac injury and early signals of cardiac function normalization - - Day 90 biopsy data from 10 treated participants (ages 5-10) showed all participa