Drugs / SGT-001
Regulatory milestones approvals, filings & regulatory actions · 2 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Clinical hold | US (FDA) | — | DMD-related muscular dystrophy | 2019-11-12 | “FDA) has notified the company that IGNITE DMD, its Phase I/II study of SGT-001, has been placed on clinical hold.” solidbio.com ↗ “Company received FDA clinical hold letter and is working to respond and determine a path to resume dosing” solidbio.com ↗ |
| Clinical hold | US (FDA) | — | DMD-related muscular dystrophy | 2018-03-14 | “IGNITE DMD, its Phase I/II clinical trial for SGT-001 microdystrophin gene transfer in Duchenne muscular dystrophy (DMD), has been placed on Clinical Hold.” solidbio.com ↗ “In November 2019, Solid announced that the SGT-001 IGNITE DMD trial was placed on clinical hold by the U.S. Food and Drug Administration (FDA).” solidbio.com ↗ “Submitted a response to the FDA regarding the previously announced partial clinical hold on the high dose of SGT-001 in IGNITE DMD” solidbio.com ↗ |
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT03368742 | Dec 2017 → Oct 2026 expected | Duchenne muscular dystrophy | Solid Biosciences Inc. | Active not recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 22
| Date | Issuer | Release |
|---|---|---|
| 2022-10-13 | Solid Biosciences Inc. | Results Solid Biosciences Presents New SGT-001 IGNITE DMD Study Results at World Muscle Society 2022 Congress Demonstrating Improvements in Ambulatory Function solidbio.com ↗
In the IGNITE DMD clinical trial, patients receiving SGT-001 improved from baseline in SV95C at one year, whereas a control patient included in the study and natural history data both demonstrated declines over the same period. |
| 2022-04-27 | Solid Biosciences Inc. | Results Solid Biosciences Announces Updated Corporate Strategy to Develop SGT-001 and SGT-003 Pipeline Programs for Patients with Duchenne Muscular Dystrophy solidbio.com ↗
Recent data suggest that our lead program, SGT-001, holds meaningful promise as a potential treatment option. |
| 2022-03-14 | Solid Biosciences Inc. | Results Solid Biosciences Reports Fourth Quarter and Full-Year 2021 Financial Results and 2-Year Efficacy and Safety Data from the Ongoing Phase I/II IGNITE DMD Clinical Trial of SGT-001 solidbio.com ↗
The company reported positive two-year efficacy and safety data from the first three patients treated in the high dose (2E14 vg/kg) cohort of the ongoing Phase I/II IGNITE DMD clinical trial. |
| 2021-09-29 | Solid Biosciences Inc. | Results Solid Biosciences Reports Additional Pulmonary Function Results from the Ongoing IGNITE DMD Phase I/II Clinical Trial of SGT-001 solidbio.com ↗
Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company focused on advancing meaningful therapies for Duchenne muscular dystrophy (Duchenne), today reported additional positive pulmonary function data from the first six patients in the ongoing IGNITE DMD Phase I/II clinical trial of SGT-001. |
| 2021-09-23 | Solid Biosciences Inc. | Results Solid Biosciences Reports 1.5-Year Data from Patients in the Ongoing IGNITE DMD Phase I/II Clinical Trial of SGT-001 solidbio.com ↗ |
| 2021-05-14 | Solid Biosciences Inc. | Results Solid Biosciences Provides First Quarter 2021 Business Update and Financial Results solidbio.com ↗
Long-term biopsy data generated from the ongoing IGNITE DMD trial demonstrate durable microdystrophin expression as measured up to two years post dosing of SGT-001. |
| 2021-03-15 | Solid Biosciences Inc. | Results Solid Biosciences Reports Efficacy and Safety Data from the Ongoing IGNITE DMD Clinical Trial and Resumption of Patient Dosing in the 2E14 vg/kg Cohort solidbio.com ↗
Interim data from six patients provide evidence of a potential benefit of SGT-001 in functional endpoints of North Star Ambulatory Assessments (NSAA), 6-minute walk test (6MWT), pulmonary function tests (PFTs), and clinically validated patient reported outcome measures (PROMs) |
| 2020-10-01 | Solid Biosciences Inc. | Regulatory Solid Biosciences Announces FDA Lifts Clinical Hold on IGNITE DMD Clinical Trial solidbio.com ↗
Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company focused on advancing meaningful therapies for Duchenne muscular dystrophy (Duchenne), today announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold placed on the Company’s IGNITE DMD Phase I/II clinical trial. |
| 2020-07-24 | Solid Biosciences Inc. | Regulatory Solid Biosciences Provides Update Regarding the IGNITE DMD Phase I/II Clinical Hold solidbio.com ↗
Today, the FDA responded by maintaining the clinical hold and requesting further manufacturing information, updated safety and efficacy data for all patients dosed, and providing direction on total viral load to be administered per patient. |
| 2020-05-14 | Solid Biosciences Inc. | Results Solid Biosciences Presents Data from Duchenne Muscular Dystrophy Gene Therapy Program at ASGCT 23rd Annual Meeting solidbio.com ↗
SGT-001 administration results in dose-dependent, muscle wide microdystrophin expression in muscle biopsies collected 90 days post-SGT-001 administration. |
| 2020-05-07 | Solid Biosciences Inc. | Regulatory Solid Biosciences Provides Update regarding SGT-001 Phase I/II Clinical Hold on IGNITE DMD solidbio.com ↗
The FDA has responded by maintaining the clinical hold and requesting further data and analyses relating to this manufacturing process. |
| 2020-03-12 | Solid Biosciences Inc. | Results Solid Biosciences Reports Fourth Quarter and Full Year 2019 Financial Results and Provides Business Update solidbio.com ↗
Using immunofluorescence assays, 50%-70% of the muscle fibers were determined to express SGT-001 microdystrophin. |
| 2019-12-18 | Solid Biosciences Inc. | Results Solid Biosciences Provides Data Update from SGT-001 Development Program solidbio.com ↗
The data from these patients show SGT-001 microdystrophin expression and associated neuronal nitric oxide synthase (nNOS) function, providing evidence that SGT-001 has the potential to result in therapeutic benefit for patients with Duchenne. |
| 2019-11-12 | Solid Biosciences Inc. | Regulatory Solid Biosciences Provides SGT-001 Program Update solidbio.com ↗
The FDA has notified the company that the study has been placed on clinical hold. |
| 2019-05-13 | Solid Biosciences Inc. | Results Solid Biosciences Reports First Quarter 2019 Financial Results and Provides Business Update solidbio.com ↗ |
| 2019-02-07 | Solid Biosciences Inc. | Results Solid Biosciences Announces Preliminary SGT-001 Data and Intention to Dose Escalate in IGNITE DMD Clinical Trial for Duchenne Muscular Dystrophy solidbio.com ↗
Solid Biosciences (Nasdaq: SLDB) announced today preliminary findings from IGNITE DMD, the Company’s Phase I/II dose-ascending clinical trial evaluating the safety and efficacy of SGT-001 microdystrophin gene transfer for the treatment of Duchenne muscular dystrophy (DMD). |
| 2018-06-18 | Solid Biosciences Inc. | Regulatory Solid Biosciences Announces FDA Removes Clinical Hold on SGT-001 solidbio.com ↗
Solid Biosciences Inc. (NASDAQ:SLDB) today announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold on IGNITE DMD, the Company’s Phase I/II clinical trial for its investigational microdystrophin gene transfer, SGT-001, for the treatment of Duchenne muscular dystrophy (DMD). |
| 2018-05-18 | Solid Biosciences Inc. | Results Solid Biosciences Announces New Preclinical Data at the American Society of Gene and Cell Therapy Annual Meeting solidbio.com ↗
New data for SGT-001, the Company’s lead microdystrophin gene transfer candidate, further demonstrate its potential to produce long-term and body-wide microdystrophin expression that correlates with significant improvements in muscle function. |
| 2018-04-18 | Solid Biosciences Inc. | Regulatory Solid Biosciences Provides Update On SGT-001 Clinical Development Program For Duchenne Muscular Dystrophy solidbio.com ↗
The FDA also informed Solid that the Company has satisfactorily addressed manufacturing-related questions that led to the previously-announced partial clinical hold on the high dose of SGT-001 in IGNITE DMD. |
| 2018-03-14 | Solid Biosciences Inc. | Regulatory Solid Biosciences Announces Clinical Hold On SGT-001 Phase I/II Clinical Trial For Duchenne Muscular Dystrophy solidbio.com ↗
The FDA informed the company that the clinical hold was due to the event. |
| 2017-05-11 | Solid Biosciences Inc. | Results New Preclinical Data Support SGT-001 As A Novel Treatment Approach For Duchenne Muscular Dystrophy solidbio.com ↗
Solid Biosciences announced today that new data from two preclinical studies reinforce the potential of its investigational microdystrophin gene therapy, SGT-001, to be an effective treatment approach for Duchenne muscular dystrophy (DMD). |
| 2016-10-24 | Solid Biosciences Inc. | Regulatory Solid Biosciences Granted U.S. and E.U. Orphan Drug Designations for Lead Gene Therapy Candidate for Duchenne Muscular Dystrophy solidbio.com ↗
Solid Biosciences and its subsidiary, Solid GT, announced today that the U.S. Food and Drug Administration and the European Commission have granted Orphan Drug designations for the company’s gene therapy candidate, SGT-001, for the treatment of patients with Duchenne muscular dystrophy (DMD). |
All press releases naming this drug 25 releases
Evidence & citations 3 cited values
Every value below carries the sentence it was read from. 1 source stands behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | SGT-001 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT03368742 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “AAV9 vector containing muscle-specific promoter and microdystrophin construct” NCT03368742 ↗ |
| Route | Intravenous | “single intravenous (IV) infusion of SGT-001” NCT03368742 ↗ |