Solid Biosciences has sacrificed a third of its staff as it desperately tries to cling to its troubled Duchenne muscular dystrophy (DMD) programme.
The ‘leaner’ workforce will redirect all of Solid Bio’s remaining resources to advancing its lead DMD candidate SGT-001 – a programme which was put on clinical hold last November due to safety concerns.
The FDA halted the IGNITE DMD trial following a report of a serious adverse event (SAE) was deemed to be related to the drug.
To Solid Bio’s dismay, shares in the company dropped close to 71% following the announcement of the SAE and subsequent clinical hold.
According to Solid Bio, to date six patients have been dosed with SGT-001 – this includes three patients in the first cohort at a 5E13 vg/kg dose who are well and continue to be followed up per study protocol.
However, one out of the three patients subsequently dosed in the second cohort at 2E14vg/kg experienced complications characterised by complement activation, thrombocytopenia, a decrease in red blood cell count, acute kidney injury and cardio-pulmonary insufficiency.
Solid Bio then reported the event to the FDA and the study’s Data Safety Monitoring Board (DSMB), to which the FDA responded by issuing a clinical hold.
“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,” said Ilan Ganot, CEO, president and co-founder of Solid Bio.
“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,” he added.
Following the restructuring efforts, chief operating officer Alvaro Amorrortu and chief medical officer Jorge Quiroz will depart from the company.
Solid Bio will now have to direct all its efforts on restarting the SGT-001 programme, and with increasing rival therapies entering the market it may prove to be a tough sell to the FDA on taking the trial off clinical hold.
Sarepta Therapeutics was granted a surprise FDA approval for its follow-up DMD drug Vyondys 53 – a drug which was also plagued with safety concerns and was initially rejected by the regulatory agency.
Solid Bio now has enough cash to last it through to 2021, which will hopefully be enough time to convince the FDA that its DMD candidate has enough benefit for an approval.