- Solid Biosciences is laying off about 37 of its 111 employees, including two top executives, as it tries to persuade the Food and Drug Administration to allow resumption of dosing in its clinical trial of its Duchenne muscular dystrophy gene therapy.
- The decision will help Solid extend its financing into 2021, but will curtail development of a second Duchenne drug as well as an assistive device.
- Shares in the Cambridge, Massachusetts-based firm fell 13% to $3.86 apiece in morning trading. The company has lost 88% of its valuation in the past 12 months.
Without taking this action, Solid was in danger of running out of cash around mid-year. It had $106 million in cash and liquid securities as of Sept. 30, 2019, and was burning about $23 million per quarter during 2019.
Its suppressed valuation meant another big share offering, like its $60 million private placement last July or its $134 million public offering in January 2018, isn't an alternative until it can return to investors' favor with positive data from its gene therapy, called SGT-001. And as long as the clinical hold due to an adverse event is effective, that won't happen.
Moreover, what data has been generated from the clinical trial has been underwhelming, failing to match a rather impressive benchmark set by DMD gene therapy leader Sarepta Therapeutics.
As part of the restructuring, Chief Operating Officer Alvaro Amorrortu and Chief Medical Officer Jorge Quiroz will be leaving the company. Former Novartis patient affairs chief Cathryn Clary and former Ipsen executive Jeffry Lawrence, both of whom work for management consulting firm SSI Strategy, will serve as advisers on clinical and regulatory activities.
Now with the restructuring in place, Solid's sole focus is on getting SGT-001 off the clinical hold and enrolling new patients. When it disclosed early data from a three-patient dosing cohort in December, CEO Ilan Ganot said the single patient hospitalized for an immune-related response had been discharged.
In a letter to DMD patients and their families posted Thursday, Ganot said the company will "work every day with resolve and urgency to determine the best path forward to resume dosing of SGT-001."
Every day the clinical hold persists, Solid falls further behind Sarepta, which has completed dosing in a placebo-controlled trial and plans to begin a trial of its commercial supply in mid-2020. Sarepta signed a $1.2 billion deal with Roche for rights to its gene therapy, dubbed SRP-9001, outside the United States, in a further sign of its lead in the DMD gene therapy space.
Meanwhile, the company will be putting on the back burner a preclinical drug for DMD called anti-LTBP4, which aimed to inhibit a protein called TGF-beta that mediates fibrosis, as well as a wearable suit aimed at helping DMD patients with mobility.