PTC Therapeutics announced this week that it is halting its preclinical and early research in gene therapy as part of “a strategic portfolio prioritization.”
Instead, the biopharma will pour most of its attention into Upstaza, a gene therapy for aromatic L-amino acid decarboxylase (AADC) deficiency.
The company is also axing its preclinical stage programs in Friedreich ataxia, a nerve disorder, as well as Angelman syndrome, another genetic disorder that affects the nervous system. Additionally, PTC will stop its programs in rare CNS and ophthalmological disorders.
Along with those halts, PTC is chopping down its staff size by about 8%, according to a Securities and Exchange Commission filing. Among those leaving the organization as part of the restructuring is chief financial officer Emily Hill.
In a statement Tuesday, PTC Therapeutics noted that it expects to see a 15% reduction in residual operating expenses in 2023 as a result of the reprioritization effort.
The company said the decision was based on the desire to focus on parts of its drug pipeline that are most likely to have the highest significant return on investment for patients with high unmet medical needs.
“We believe that the decision to discontinue our pipeline gene therapy programs enables PTC to focus R&D efforts on our other innovative and differentiated scientific platforms and strongly positions us for long-term growth and success,” PTC CEO Matthew Klein said in a statement. “Where possible, we will work to ensure that the discontinued gene therapy programs can be developed by other parties so that the therapies have the potential to benefit patients.”
The news comes shortly after PTC released the topline results from its MOVE-FA trial in Friedreich ataxia (FA). Vatiquinone, the drug in question, did not reach its primary endpoint of statistically significant change in the mFARS score.
Still, the program for vatiquinone is one the biopharma will continue to bet on – Klein cited the “encouraging” findings of meaningful impact on certain aspects of disease progression – and plans to push toward eventual approval.
“Given… the unmet medical need for pediatric patients with FA, we look forward to discussing a potential path to registration with regulatory authorities,” Klein said in a statement.
The shift will also allow PTC to focus on global commercialization of Upstaza, which is currently authorized in the European Union.
According to the European Medicines Agency, Upstaza was authorized under “exceptional circumstances,” meaning that PTC is required to provide additional data and continuing studies to ensure long-term safety and efficacy of the drug. In November, Upstaza also received authorization in the U.K.
