Solid Biosciences Reduces Workforce, Focuses on Advancing DMD Pipeline

Solid Biosciences, a company focused on advancing gene therapies for Duchenne muscular dystrophy, said it is reducing its workforce by 35 percent to prioritize the development of lead candidates SGT-001 and SGT-003 to treat the rare genetic and fatal muscle wasting disease.

Photo: Ilan Ganot, CEO, president, and co-founder of Solid Biosciences

As part of streamlining operations, Solid Bio is shifting to a commercially scaled transient transfection-based manufacturing process, which it believes may provide improvements to manufacturability as well as additional organizational efficiencies.

The company also said Joel Schneider, Solid’s chief operating officer, will be leaving the company at the end of May to accept a role as the CEO for a privately held novel, viral-based gene therapy platform company.

In March, Solid Biosciences reported encouraging interim functional and biomarker data, and patient reported outcome measures from six patients after treatment in the ongoing IGNITE DMD phase 1/2 clinical trial of its lead gene therapy candidate SGT-001, demonstrating that patients showed sustained benefit compared with natural history trajectories two years after treatment with SGT-001 across functional, pulmonary, and patient reported outcome measures.

The positive news was welcomed as the clinical program had been plagued by problems with the U.S. Food and Drug Administration placing a clinical hold on the program in November 2019 after a seven-year-old boy dosed in the study experienced a serious adverse event deemed related to the study drug. The hold was lifted in October 2020 after Solid changed its manufacturing process to allow target dosing to be achieved with fewer viral particles thus reducing the total viral load.

Solid’s SGT-001 is a novel AAV-mediated gene transfer therapy designed to address the underlying genetic cause of Duchenne—mutations in the dystrophin gene that result in the absence or near absence of dystrophin protein. SGT-001 is systemically administered to deliver a synthetic dystrophin gene, called microdystrophin, to the body that encodes for a functional protein surrogate that is expressed in muscles and stabilizes essential associated proteins, including neuronal nitric oxide synthase. Data from Solid’s clinical program suggests that SGT-001 has the potential to slow or stop the progression of Duchenne, regardless of genetic mutation or disease stage.

“Today, we are sharing changes to our corporate strategy to better align our resources behind Solid’s core values of innovation and patient centricity, with a focus on bringing our differentiated micro-dystrophin gene therapy to more patients,” said Ilan Ganot, CEO, president, and co-founder of Solid Biosciences. “We will be transitioning SGT-001 to a commercially scaled manufacturing process, advancing SGT-003 toward an anticipated IND submission in early 2023, and aligning our organization behind these two programs.”

Besides SGT-001, Solid shared new preclinical data, which suggest that Solid’s novel capsid may offer enhanced muscle tropism compared with AAV9, and has the potential to benefit patients with Duchenne as well as the broader gene therapy landscape for muscle-related disorders.

Along with prioritizing the advancement of SGT-001 and SGT-003, Solid anticipates that the use of transfection-based manufacturing processes for both SGT-001 and SGT-003 will allow it to focus its operating structure and better leverage external manufacturing expertise. In addition, the company plans to narrow R&D activities to those related to SGT-001, SGT-003 and next generation capsids.

The reorganization will result in a reduction in planned corporate expenditures which is anticipated to extend funding of operations through important clinical milestones and into the second quarter of 2024.

Author: Rare Daily Staff