Solid Biosciences Cuts Workforce, Restructures to Focus on SGT-001 Development

Joana Carvalho, PhD avatar

by Joana Carvalho, PhD |

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Solid Biosciences announced it will reduce its workforce by approximately one third to cut expenses and focus on advancing the development of SGT-001, its gene therapy candidate for the treatment of Duchenne muscular dystrophy (DMD).

SGT-001 uses a viral vector to deliver a gene producing a short artificial form of dystrophin — called microdystrophin — into muscle cells. Microdystrophin has all the key components of the normal protein lacking in those with DMD.

The company is currently working closely with the U.S. Food and Drug Administration (FDA) to lift the second clinical hold on the IGNITE DMD trial (NCT03368742). Placed last November, this hold was due to a serious adverse event (side effect), including decreased number of platelets and red blood cells, and transient kidney problems, deemed related to treatment of a 7-year-old boy.

In December 2019, Solid Biosciences announced new data from two boys who received the highest dose of SGT-001 in IGNITE DMD. Muscle biopsies performed three months after treatment revealed the presence of microdystrophin in muscle fibers of both boys. This was accompanied by a reduction in the levels of creatine kinase, a marker of muscle damage, over the course of the study.

As part of its restructuring, the company will focus on determining how to address this clinical hold and resume dosing. Other ongoing development programs will be reduced.

“We believe SGT-001 holds great potential for the treatment of Duchenne, and in order to effectively evaluate its potential for patients, we made some difficult choices to focus our resources and help extend our cash runway,” Ilan Ganot, Solid’s CEO, president and co-founder, said in a press release.

“We are grateful for the efforts and contributions of our employees who have worked tirelessly to advance our mission to improve the lives of patients with Duchenne. We are committed to supporting all employees during this transition,” Ganot said.

In addition, Alvaro Amorrortu, CEO, and Jorge Quiroz, MD, chief medical officer, will leave the company and become external advisors.

In turn, Cathryn Clary, MD, and Jeffry Lawrence, MD, two industry veterans who have been working with Solid Biosciences for the past few months, will assume leadership positions to guide the company’s clinical and regulatory activities.

“I would like to thank Alvaro and Jorge for their contributions to Solid over the last several years. Their leadership and dedication to advancing meaningful new therapies for patients with Duchenne has been commendable and we wish them the best in their future endeavor,” Ganot said.

“While Solid will look a little different as we move into 2020, our unwavering commitment to the Duchenne community remains strong. We are focused on the work ahead to determine the best approach to resume dosing of SGT-001,” he said.

According to the company, these organizational changes will reduce the company’s corporate expenses and are expected extend the company’s financial resources into 2021.