Solid Biosciences Highlights FDA Path for SGT-003 DMD Study, Shares Early Friedreich’s Ataxia Update

Defense World Staff on Feb 17th, 2026

Friedreich’s ataxia: dual route administration and first patient dosed Turning to FA, Cumbo said the program uses a dual route of administration designed to reach the dentate nucleus of the cerebellum while also targeting the spinal column and heart. He described MRI-guided stereotactic delivery into both sides of the dentate nucleus, led by neurosurgeon Dr. Lonser at Ohio State University, followed by intravenous dosing after a rest period. 

Cumbo said Solid dosed its first FA patient, a 27-year-old who was “very, very sick,” and referenced an mFARS score of 93 (noting the patient had been in the high 80s). Roughly 40 days after dosing, he said the patient experienced a headache that resolved with Tylenol and that the company had not seen other issues. He added that the team was “already hearing things” suggestive of efficacy, but emphasized it did not yet have proof. 

For planned data disclosure, Cumbo said Solid expects to dose three to six FA patients over the course of the year and anticipates reading out results from the first three patients in the second half of the year, while continuing dosing in a second cohort. On expectations for clinical benefit, he cited 2.14 as the mFARS bar associated with the regulatory approval of SKYCLARYS.

Targeting frataxin deficiency in DRG neurons and fibroblasts: omaveloxolone restores metabolic and iron balance to reduce ferroptosis

Portillo-Carrasquer M, Sanz-Alcázar A, Sánchez-López B, Delaspre F, Pazos-Gil M, Oliveira-Jorge L, Castells-Roca L, Tamarit J, Ros J, Cabiscol E. Targeting frataxin deficiency in DRG neurons and fibroblasts: omaveloxolone restores metabolic and iron balance to reduce ferroptosis. Biomed Pharmacother. 2026 Feb;195:119031. doi: 10.1016/j.biopha.2026.119031. Epub 2026 Jan 23. PMID: 41579709.

Here we used frataxin-deficient DRG neurons to better understand the drug's role in these sensory neurons. Omaveloxolone improved most of the analyzed parameters, including frataxin levels, cell survival, mitochondrial respiratory activity, iron homeostasis, oxidative stress, transferrin receptor 1 and glutathione peroxidase 4 levels, as well as the GSH/GSSG ratio.

Domain Specific Placebo Response in the Modified Friedreich's Ataxia Rating Scale

Rummey C, Farmer JM, Lynch DR. Domain Specific Placebo Response in the Modified Friedreich's Ataxia Rating Scale. Ann Clin Transl Neurol. 2026 Feb;13(2):393-398. doi: 10.1002/acn3.70239. Epub 2025 Nov 26. PMID: 41293985; PMCID: PMC12883693. 

The placebo response in clinical trials in ataxias complicates outcome interpretation and potentially obscures genuine treatment effects. We analyzed placebo group data from past trials in Friedreich Ataxia and observed notable responses in appendicular items, in contrast to minimal changes in axial function, as measured by respective subscores of the modified Friedreich Ataxia Rating Scale (mFARS). The effect increased with the number of consecutive tests, shorter testing intervals, and older group ages. This has implications for trial design and endpoint selection, thus strengthening the utility of the Upright Stability Score (USS), a sub‐score of mFARS, as an independent measure.

Syndromic and Metabolic Cardiomyopathies: Rare Variants for Adults (Glycogen Storage Diseases, Friedreich’s Ataxia)

Turk Kardiyol Dern Ars 2024;52(Suppl 1):S1-S206. In Friedreich’s Ataxia patients, concentric left ventricular hypertrophy is common; asymmetric hypertrophy is very rare. Diastolic wall thickness is usually less than 15 mm, the ejection fraction is preserved, and outflow tract obstruction is typically absent.

Clinical manifestations of Friedreich ataxia in patient with neurofibromatosis seen at Daoud Charity Clinic Sudan: A case report

Abbasher Hussein, Mohajer Ismael, Mohamed Baraka, Abdaleliah Abobker, Wadah Altoom, Osama Suliman, Clinical manifestations of Friedreich ataxia in patient with neurofibromatosis seen at Daoud Charity Clinic Sudan: A case report, Journal of the Neurological Sciences, Volume 480, Supplement, 2025, 125530, ISSN 0022-510X, doi:10.1016/j.jns.2025.125530. 

 The case of Fredrich's ataxia and neurofibromatosis had been reported. Despite this, there was no clear association or overlapping relationship between the two diseases.

Mitochondrial iron overload is associated with lysosomal dysfunction-mediated mitophagy impairment in the heart of Friedreich’s ataxia

Eunbin Jee, Maisha Medha, Hwayoung Baek, Jonghan Kim, Yuho Kim, Mitochondrial iron overload is associated with lysosomal dysfunction-mediated mitophagy impairment in the heart of Friedreich’s ataxia, Mitochondrion, 2026, 102120, ISSN 1567-7249, doi:10.1016/j.mito.2026.102120. 

Collectively, our study provides mechanistic insight into the role of mitochondrial iron aggregates in the pathogenesis of FRDA-related cardiomyopathy and suggests a potential contribution of lysosomal dysfunction to impaired mitochondrial quality control in the context of cardiac frataxin deficiency.

La omaveloxolona recibe luz verde en España como primer tratamiento específico para la Ataxia de Friedreich

Comisión Interministerial de Precios de los Medicamentos 28 de enero de 2026. Skyclarys H* (omaveloxolona): Tratamiento de la ataxia de Friedreich en adultos y adolescentes a partir de los 16 años de edad.

Larimar’s Pediatric Friedreich’s Ataxia Trial Termination: What Investors Should Know

TipRanks Clinical-Trials-Jan 28, 2026 

"The study was first submitted in November 2024, signaling the formal start of the regulatory process for this pediatric program. The most recent update to the record was filed on January 26, 2026, showing that key information on the trial has been reviewed and refreshed. Notably, the trial status is listed as “terminated,” indicating that the study was stopped early and will not reach a planned primary or final completion date; reasons for termination are not detailed in the registry entry."

California grants $7.4 million to advance gene-edited stem cell therapy for Friedreich’ s ataxia

Gen 13, 2026. The California Institute for Regenerative Medicine (CIRM) has awarded $7.4 million to support a University of California San Diego team developing a first-of-its-kind stem cell-based gene therapy for Friedreich’s ataxia, a rare inherited neurodegenerative disease that causes progressive loss of coordination, muscle strength, heart function and overall mobility. The new funding will help the research team complete the final steps required by federal regulators before they can apply to begin a first-in-human clinical trial.

Orphan Drug Designation for SGT-212 Dual-Route Gene Therapy for the Treatment of Friedreich’s Ataxia

CHARLESTOWN, Mass., Jan. 12, 2026 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. today announced that U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to SGT-212 for the treatment of Friedreich’s ataxia (FA). Additionally, earlier today, the Company reported that the first participant has been dosed in FALCON, a Phase 1b, first-in-human clinical trial evaluating SGT-212 for the treatment of FA.