Santhera axes half its employees and sees CMO depart after PhIII DMD fail
Less than a month after showing its lead Duchenne muscular dystrophy program out the door, Santhera Pharmaceuticals is slashing half its staff.
The Swiss biotech announced the move Monday morning as it continues its restructuring around a second DMD candidate, vamorolone. Among the employees departing is Santhera’s now-former CMO Kristina Sjöblom Nygren, though the company noted that she left for “family reasons and to pursue other opportunities,” per a statement.
Santhera added that the reductions will save roughly $10.9 million in annual costs, following a one-time charge of about $3.26 million. In total, Santhera is cutting more than 50 positions, bringing its total workforce to 47 full-time equivalent employees.
Vamorolone became the company’s primary focus after Santhera reported that the other DMD program, idebenone, failed a Phase III interim analysis last month. The next data, a 6-month readout in a pivotal Phase IIb trial, is due sometime in the second quarter of 2021. If all goes well, Santhera said, an NDA could come as soon as next year’s fourth quarter.
That trial is measuring improvement in time to stand over baseline after 24 weeks, enrolling DMD patients that are not taking steroid treatments. Santhera previously noted that vamorolone has the potential to be an alternative to such therapies altogether by slowing disease progress without steroidal side effects. The drug itself is a steroid designed to have similar effects as the corticosteroids Duchenne patients currently take.
Vamorolone’s most recent data comes from a Phase IIa back in August 2019, which hit the primary endpoint in showing statistically significant and proportional improvements from baseline on time to stand from supine.
Santhera said it expects to be the first to market with a dissociative steroid in the US in 2022, acquiring global rights to the experimental drug in September after first signing on two years ago. Originally developed by ReveraGen, vamorolone was acquired following an option agreement with Actelion, now known as Idorsia, in 2016. Idorsia then sold the option to Santhera in 2018.
Idebenone had previously been held up as one of Santhera’s more promising programs, but it had often run into trouble before being axed last month. Designed to improve lung function in patients by energizing weakened muscle cells, idebenone was originally submitted for FDA approval back in 2016, but regulators were not impressed. The agency asked for a confirmatory trial to prove efficacy — the trial that ultimately flopped in October.
But idebenone also ran into trouble in Europe when the EMA shot down a label expansion into DMD in 2017 and an appeal in 2018. Approved on the continent as Raxone to treat Leber’s hereditary optic neuropathy, the drug also failed an NIH-backed multiple sclerosis trial in March 2018 after showing no difference from a placebo.
In addition to the restructuring around vamorolone, Santhera is also developing lonodelestat for cystic fibrosis and other lung diseases.