Duyuru • Mar 12

Solid Biosciences Inc. Provides Interim Positive Clinical Update on Phase 1/2 Inspire Duchenne Trial

Solid Biosciences Inc. reported updated positive interim data from the ongoing Phase 1/2 INSPIRE DUCHENNE clinical trial of SGT-003. The new data, including additional muscle biopsy, serum biomarker, cardiac function, and safety analyses, add to a growing dataset that suggests the potential of the biological activity of SGT-003 microdystrophin therapy. INSPIRE DUCHENNE is a Phase 1/2 first-in-human, open-label, single-dose, multicenter trial designed to evaluate the safety, tolerability and efficacy of SGT-003 in pediatric participants with Duchenne at a dose level of 1E14vg/kg. SGT-003 is administered as a one-time intravenous infusion. The interim clinical data reported in this release are as of a February 23, 2026, data cutoff date. SGT-003 has been generally well tolerated in the 40 participants dosed as of March 11, 2026. The safety and tolerability profile observed in the INSPIRE DUCHENNE trial continued to be promising; SGT-003 is administered using a low-burden, steroid-only prophylactic immunomodulation regimen. The trial is being conducted at 15 clinical sites across the US, Canada, Italy and the United Kingdom and participant dosing remains ongoing. Microdystrophin transduction and expression levels, beta-sarcoglycan localization and nNOS activity were evaluated by biopsy in 20 participants (ages 1-10 years) at Day 90 and in 3 participants at Day 360. Results demonstrated robust mean vector copies per nucleus and microdystrophin expression as well as properly localized and restored beta-sarcoglycan-positive fibers and nNOS activity-positive fibers. Beta-sarcoglycan and nNOS are critical components of the dystrophin-associated protein complex (DAPC). In Duchenne, the absence of dystrophin destabilizes the DAPC, triggering a cascade of structural, signaling and metabolic defects that impair muscle integrity. Reconstituting critical components of the DAPC, including beta-sarcoglycan and nNOS, could suggest biologic correlation of SGT-003’s treatment effect. Solid’s microdystrophin construct is the only microdystrophin gene therapy, approved or investigational, that contains the R16/R17 binding domain, which uniquely localizes nNOS to the muscle. Table 1: 90- and 360-day biopsy results: Day 90 (n=20 unless noted), Day 360 (n=3). Mean vector copies per nucleus: 11 (Day 90), 12 (Day 360). Mean microdystrophin expression by western blot (%): 60% (n=19) (Day 90), 91% (Day 360). Mean microdystrophin expression by mass spectroscopy (%): 52% (n=17) (Day 90), 86% (Day 360). Mean microdystrophin-positive fibers by immunofluorescence (%): 63% (Day 90), 69% (Day 360). Properly localized and restored beta-sarcoglycan-positive fibers (%): 60% (Day 90), 69% (Day 360). nNOS activity-positive fibers (%): 35% (Day 90), 33% (Day 360). Western blot and mass spectrometry baselines were 0% mean normal dystrophin, microdystrophin-positive fibers by immunofluorescence was based on a manual count, as are beta-sarcoglycan and nNOS-positive fibers. These assays are conducted by multiple external vendors; at the time of analysis, one western blot sample and three mass spectrometry samples had not been received. Additionally, Solid has identified an extensive biomarker panel to comprehensively evaluate treatment effect on muscle integrity. Collectively, these observed biomarker improvements at Day 90 and Day 360 suggest improved muscle fiber health and stability, reduced ongoing muscle damage, and an interruption of the chronic degeneration/regeneration cycle that is characteristic of Duchenne. In particular, embryonic myosin heavy chain (eMHC) is an informative predictor of disease progression. eMHC is typically expressed during fetal development but is also expressed when muscle satellite cells differentiate into muscle fibers, which in Duchenne occurs in response to muscle fiber damage. In the absence of functional dystrophin, newly generated muscle fibers also fail, leading to continued but ultimately futile satellite cell activation. A mean 44% observed reduction in eMHC positive fibers seen at Day 90 (n=20) suggests that SGT-003 treatment has potentially disrupted this chronic and futile degeneration-regeneration cycle, stabilizing muscle fibers and preserving the reservoir of satellite cells. Table 2: Improvements in multiple biomarkers of muscle integrity observed at Day 90 and Day 360. Serum Biomarkers: Day 90 Mean Reductions (n=24 unless noted), Day 360 Mean Reductions (n=7 unless noted). Serum creatine kinase (CK): 38% (Day 90), 37% (Day 360). Serum alanine transaminase (ALT): 43% (Day 90), 27% (Day 360). Serum aspartate aminotransferase (AST): 30% (Day 90), 32% (Day 360). Serum lactate dehydrogenase (LDH): 46% (n=21) (Day 90), 38% (n=6) (Day 360). Serum titin: 22% (n=11) (Day 90), 25% (n=2) (Day 360). Certain data from a subset of participants were not available at the time of analysis. While cardiac assessments were initially included as safety evaluations, stabilization-to-improvement in systolic function continues to be observed as of the data cutoff date, as measured by left ventricular ejection fraction (LVEF). Observed improvements were driven largely by participants with low-normal baseline LVEF (defined as = 60%). Cardiomyopathy is a leading cause of death in Duchenne, with 25% of individuals displaying evidence of cardiomyopathy by six years of age, increasing to 59% by 10 years of age. SGT-003 is an investigational gene therapy containing a differentiated microdystrophin construct and a proprietary, next-generation capsid, POLARIS-101™ (formerly known as AAV-SLB101), which was rationally designed to target integrin receptors, and has shown enhanced cardiac and skeletal muscle transduction with decreased liver targeting in data from the Phase 1/2 INSPIRE DUCHENNE clinical trial and in nonclinical studies. SGT-003’s microdystrophin construct uniquely includes the R16/17 domains, which localize nNOS to the muscle. Nonclinical studies have shown that nNOS can improve blood flow to the muscle thereby reducing muscle breakdown from ischemia and muscle fatigue. Together, these design features suggest that SGT-003 could be a potential best-in-class investigational gene therapy for the treatment of Duchenne. INSPIRE DUCHENNE is a first-in-human, open-label, single-dose, multicenter Phase 1/2 clinical trial to evaluate the safety, tolerability and efficacy of SGT-003 in pediatric participants with a genetically confirmed Duchenne diagnosis with a documented dystrophin gene mutation. INSPIRE DUCHENNE is a multinational trial designed to enroll participants in the United States, Canada, the United Kingdom and Italy. IMPACT DUCHENNE is a Phase 3 randomized, double-blind, placebo-controlled trial to evaluate the efficacy of a single dose of SGT-003 in ambulatory participants aged 7 to less than 12 with a genetically confirmed Duchenne diagnosis. IMPACT DUCHENNE is a multinational trial intended to support potential regulatory authorizations.

Duyuru • Feb 18

Solid Biosciences Reports Positive Initial Clinical Data from Next-Generation Duchenne Gene Therapy Candidate SGT-003

Solid Biosciences Inc. announced positive initial data from the Phase 1/2 INSPIRE DUCHENNE trial evaluating SGT-003, a next-generation gene therapy product candidate intended for the treatment of Duchenne muscular dystrophy (Duchenne). Interim 90-day biopsy data reported in the first three participants showed an average microdystrophin expression of 110%, as measured by western blot, and improvements in multiple biomarkers that are indicators of muscle health and resilience. SGT-003 was well-tolerated in the first six participants dosed as of the data cutoff date of February 11, 2025. As of the cutoff date, all six participants have reached at least 20 days post SGT-003 treatment. Adverse events (AEs) observed after SGT-003 treatment were typical of those observed in AAV gene therapy, including nausea, vomiting, fever and transient declines in platelets in some participants. No serious adverse events (SAEs) or suspected unexpected serious adverse reactions (SUSARs) were observed, and there was no evidence of thrombotic microangiopathy (TMA), atypical hemolytic uremic syndrome (aHUS), or hemolysis. Importantly, none of the AEs that were observed required the use of additional immunomodulatory agents such as eculizumab, sirolimus or rituximab. The INSPIRE DUCHENNE trial is a Phase 1/2 first in human, open-label, single-dose, multicenter trial designed to evaluate the safety, tolerability and efficacy of SGT-003 in pediatric patients with Duchenne at a dose of 1E14vg/kg. SGT-003 is administered as a one-time intravenous infusion. As of the data cutoff date of February 11, 2025, a total of six participants have been dosed in the INSPIRE DUCHENNE trial. Enrollment in the INSPIRE DUCHENNE trial is ongoing, with at least 10 total participants anticipated to be dosed by early in the second quarter of 2025 and approximately 20 total participants anticipated to be dosed by the fourth quarter of 2025. INSPIRE DUCHENNE currently has a total of six active clinical sites in the United States and Canada and approved clinical trial applications (CTAs) in the United Kingdom and Italy. Solid expects to activate additional trial sites by the end of 2025. 90-Day Initial Data: The 90-day data reported as of the data cutoff date of February 11, 2025, includes: microdystrophin expression, measures of restoration and activation of key elements of the dystrophin-associated protein complex, key muscle integrity biomarker evaluation, in each case, from the first three participants dosed in the INSPIRE DUCHENNE trial, and interim safety findings from the first six participants dosed in the INSPIRE DUCHENNE trial. The first three participants are two 5-year-old boys and one 7-year-old boy at the time of dosing. The second three participants are a 6-year-old boy and two 7-year-old boys at the time of dosing. Muscle Integrity Biomarker Evaluation at Day 90 (N=3): Mean reductions observed in markers of muscle injury and stress: Serum creatine kinase (CK) (IU/L): -57%. Serum aspartate aminotransferase (AST) (IU/L): -45%Serum alanine transaminase (ALT) (IU/L): -54%. Serum lactate dehydrogenase (LDH) (IU/L): -60%. Mean reductions observed in markers of muscle breakdown and dystrophic regeneration: Serum titin (pmol/L): -42%. Embryonic myosin heavy chain (eMHC) positive fibers: -59%. Measure of Potential Cardiac Benefit: At Day 180, mean cardiac function increased by 8% (N=2) from baseline as measured by left ventricular ejection fraction. The third participant had not reached Day 180 follow up as of the data cutoff date of February 11, 2025. Reduction in serum cardiac hs-troponin I (hs-cTnI) of -36% observed at Day 90 in one participant who entered the trial with elevated hs-cTnI levels. Two of the first three participants entered the study with normal baseline cTnI levels. Two participants in total (N=6) had elevated troponin at baseline that reduced below initial baseline values post-dose. Safety Update for the First Six Participants Dosed: SGT-003 was well-tolerated. No SAEs observed. No SUSARs observed. No hospitalizations reported. No evidence of TMA or aHUS observed. All treatment-related AEs resolved with no sequelae. None of the AEs required the use of additional immunomodulatory agents such as eculizumab, sirolimus or rituximab. No AEs of hepatic transaminitis observed, including no elevated gamma-glutamyl transferase (GGT) levels. One adverse event of special interest (AESI) was observed. Mild, transient hs-troponin I elevation observed (CTCAE Grade 1) that resolved without intervention. No clinical evidence of myocarditis observed. No EKG or echocardiographic changes observed. Most common AEs observed: Nausea/vomiting. Transient thrombocytopenia. One CTCAE Grade 3 episode that resolved within days without intervention. No evidence of hemolysis observed. Infusion related hypersensitivity reaction. One CTCAE Grade 3 episode of prolonged fever that resolved within days without intervention. Fever.

Faiz karşılama oranı	n/a
Nakit	US$380.68m
Eşitlik	US$374.13m
Toplam yükümlülükler	US$48.76m
Toplam varlıklar	US$422.89m

Veri	Son Güncelleme (UTC saati)
Şirket Analizi	2026/05/20 01:17
Gün Sonu Hisse Fiyatı	2026/05/20 00:00
Kazançlar	2026/03/31
Yıllık Kazançlar	2025/12/31

Paket	Veri	Zaman Çerçevesi	Örnek ABD Kaynağı ^*
Şirket Finansalları	10 yıl	Gelir tablosu Nakit akış tablosu Bilanço	SEC Form 10-K SEC Form 10-Q
Analist Konsensüs Tahminleri	+3 yıl	Finansal tahminler Analist fiyat hedefleri	Analist Araştırma Raporları Blue Matrix
Piyasa Fiyatları	30 yıl	Hisse senedi fiyatları Temettüler, Bölünmeler ve Eylemler	ICE Piyasa Verileri SEC Form S-1
Sahiplik	10 yıl	En büyük hissedarlar İçeriden öğrenenlerin ticareti	SEC Form 4 SEC Form 13D
Yönetim	10 yıl	Liderlik ekibi Yönetim Kurulu	SEC Form 10-K SEC Form DEF 14A
Önemli Gelişmeler	10 yıl	Şirket duyuruları	SEC Form 8-K

Analist	Kurum
Timothy Chiang	BTIG
Kristen Kluska	Cantor Fitzgerald & Co.
Charles Duncan	Cantor Fitzgerald & Co.

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Recent updates

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