DMD candidate SGT-003 receives Rare Pediatric Disease Designation

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The planned phase 1/2 INSPIRE Duchenne trial (NCT06138639) is a first in-human, open-label, multicenter trial to evaluate tolerability and safety of SGT-003.

DMD candidate SGT-003 receives Rare Pediatric Disease Designation | Image Credit: © Araki - © Araki - stock.adobe.com.

DMD candidate SGT-003 receives Rare Pediatric Disease Designation | Image Credit: © Araki - © Araki - stock.adobe.com.

Takeaways:

  • Solid Bioscience's gene therapy candidate SGT-003 for Duchenne muscular dystrophy (DMD) has received Rare Pediatric Disease Designation from the FDA.
  • The planned phase 1/2 INSPIRE Duchenne trial will evaluate the tolerability and safety of SGT-003 in pediatric DMD patients, with 2 cohorts.
  • Inclusion criteria for the trial include ambulatory ability, negative AAV antibodies, and stable prednisone or deflazacort dose for a specified duration.
  • Exclusion criteria encompass previous treatment with dystrophin-modifying drugs, gene transfer drugs, or recent exposure to investigational drugs.
  • SGT-003 utilizes a proprietary capsid to deliver a DNA sequence encoding a shortened form of the dystrophin protein and is currently recruiting participants at select locations.

Solid Bioscience's Duchenne muscular dystrophy (DMD) gene therapy candidate, SGT-003, has received Rare Pediatric Disease Designation from the FDA, the company announced in a press release.1

“Preclinical data suggests that SGT-003 has potential to significantly improve on existing treatments for Duchenne by using a muscle tropic proprietary capsid to deliver a DNA sequence encoding a shortened form of the dystrophin protein which, importantly, includes the nNOS binding domain," said Gabriel Brooks, MD, chief medical officer, Solid Biosciences.1

The planned phase 1/2 INSPIRE Duchenne trial (NCT06138639) is a first in-human, open-label, multicenter trial to evaluate tolerability and safety of SGT-003 at a dose of 1E14vg/kg in pediatric DMD patients.1,2

The trial will feature 2 cohorts. Cohort 1 will include male patients aged 4 to younger than 6 years, while cohort 2 will feature male patients aged 6 to younger than 8 years.2

The candidate will be administered as a 1-time intravenous infusion to the patients in both cohorts (minimum of 3 patients each), with the potential for cohort expansion.1

Inclusion criteria for the planned trial is2:

  • Ambulatory defined as "being able to walk without the use of an assistive device."
  • AAV antibody negative
  • Patients on a stable dose of at least 0.5 mg / kg / day of oral daily prednisone or 0.75 mg / kg / day deflazacort for 12 weeks or longer prior to study entry
  • Cohort 1 < 18 kg body weight
  • Cohort 2 < 30 kg body weight

Exclusion criteria includes, but is not limited to2:

  • Current or previous treatment with approved or investigational dystrophin modifying drugs such as eteplirsen, golodirsen, casimersen, and viltolarsen
  • Current or prior treatment with an approved or investigational gene transfer drug
  • Exposure to another investigational drug within 3 months prior to screening or 5 half-lives since last administration, whichever is longer
  • Established clinical diagnosis of DMD that is associated with any deletion mutation in exons 1 to 11 or 42 to 45, inclusive, in the DMD gene as documented by a genetic report and confirmed by Sponsor genetic testing

"We look forward to rapidly bringing SGT-003 to the clinic and hope to all Duchenne patients in need," added Brooks.1

DMD, the genetic muscle-wasting disease that generally affects males, has symptoms that usually appear between ages 3 and 5 years. The progressive and irreversible disease impacts approximately 1 in every 3500 to 5000 live male births in the United States, which has an estimated prevalence of 5000 to 15,000 cases.1

According to Solid Biosciences, "SGT-003 uses a proprietary, rationally designed capsid (AAV-SLB101) to deliver a DNA sequence encoding a shortened form of the dystrophin protein (microdystrophin), containing the R16-R17 nNOS binding domain."1

Locations that are currently recruiting, according to clinicaltrials.gov, are University of California, Los Angeles Medical Center and Nationwide Children's Hospital, Columbus, Ohio.2

Click here for contact information regarding recruiting clinical trial locations.2

More DMD coverage: FDA approves givinostat for Duchenne muscular dystrophy in patients 6 years and up

The decision makes givinostat the first nonsteroidal drug approved to treat patients with all genetic variants of DMD.

Read the full FDA approval article, here.

References:

1. Solid Biosciences receives Rare Pediatric Disease Designation from the FDA for Duchenne muscular dystrophy gene therapy candidate SGT-003. Solid Biosciences. Press release. April 1, 2024. Accessed April 2, 2024. https://investors.solidbio.com/news-releases/news-release-details/solid-biosciences-receives-rare-pediatric-disease-designation

2. A study of SGT-003 gene therapy in Duchenne muscular dystrophy (INSPIRE DUCHENNE). ClinicalTrials.gov. Updated March 29, 2024. Accessed April 2, 2024. https://classic.clinicaltrials.gov/ct2/show/NCT06138639

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