PTC Therapeutics trims pipeline, staff

May 24, 2023

Rare disease-focused biotech PTC Therapeutics announced this week that it would be restructuring its workforce and discontinuing preclinical and early development programs in order to prioritize its approved brain gene therapy.

In the statement shared, PTC revealed that it estimates reductions of approximately 15% in residual 2023 operating expenses, which will involve reducing its workforce and trimming its gene therapy pipeline, in order to focus on areas that could deliver significant return on investment such as its brain gene therapy, Upstaza. 

Included in the workforce reduction is Chief Financial Officer, Emily Hill, who PTC said has been "relieved of her responsibilities."

The biotech will discontinue its gene therapy programs for Friedreich ataxia, Angelman syndrome, and other rare CNS and ophthalmological disorders at different stages of preclinical development. 

Upstaza, which is the first-ever gene therapy approved to be administered in the brain, was approved last year in the EU to treat patients aged 18 months and older with aromatic L-amino acid decarboxylase (AADC) deficiency. AADC is an inherited disorder that affects the way signals are passed between certain cells in the nervous system and is characterized in infants by symptoms such as severe developmental delay, stiffness and difficulty moving. 

Upstaza has yet to grab FDA approval, however. Back in April, PTC reported that the FDA had requested additional bioanalytical data in support of comparability analyses between the clinical and commercial drug product. PTC is working with the agency to provide the necessary data prior to submitting a BLA, which is expected in the third quarter of 2023.