Solid Bio Restructures to Get Halted Gene Therapy Study Back on Track

Xconomy Boston — 

Solid Biosciences is slashing its workforce, including two top executives, in order to devote the company’s remaining resources to its experimental gene therapy for Duchenne muscular dystrophy.

The corporate restructuring announced Thursday comes two months after the FDA placed a hold on the study after safety problems emerged that were linked to the gene therapy, SGT-001. Cambridge, MA-based Solid Bio (NASDAQ: SLDB) says going forward it will focus on how to address the clinical hold and resume testing. With the corporate changes, Solid Bio says it has enough cash to last into next year. At the end of the third quarter of 2019, the company reported cash and other holdings totaling $105.7 million.

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Following the announcement, Solid Bio’s stock price slid more than 17 percent to $3.66 per share in pre-market trading.

Solid Bio has been developing SGT-001 as a way to potentially address the genetic defect underpinning Duchenne. Patients who have the inherited disease don’t make enough of the muscle protein dystrophin. The Solid Bio gene therapy uses an engineered virus to deliver genetic material intended to restore dystrophin production. But the company had also previously disclosed there’s a chance that the dosing requirements of the gene therapy could increase the risk of side effects related to the virus used in the treatment.

The complications reported in the November clinical hold included an immune system reaction, a decrease in red blood cells, kidney injury, and blood circulation difficulties. Those problems are similar to ones cited in the FDA’s 2018 clinical hold on tests of SGT-001. Months later, the agency allowed the study to resume but with additional safety measures.

Solid Bio’s board approved the corporate restructuring on Tuesday, according to a securities filing. In the first quarter of this year, the company expects to record a $2.1 million charge related to the layoffs, which will cut about one third of its workforce. Last year’s annual report states that the company had 111 full-time employees as of Dec. 31, 2018. Those leaving Solid Bio include Alvaro Amorrortu, the company’s chief operating officer, and Jorge Quiroz, its chief medical officer. But both will continue to advise Solid Bio under consulting agreements.

Photo by Flickr user reynermedia via a Creative Commons license