NEW YORK – PTC Therapeutics on Thursday announced it will lay off around one-quarter of its staff as part of a strategic reprioritization effort, under which it is moving away from developing gene therapies.
The workforce reduction primarily affected employees involved in early-stage research programs and those at a gene therapy manufacturing facility in Hopewell, New Jersey. The company unveiled plans in May to discontinue preclinical and early research in multiple gene therapy programs including for Friedreich ataxia and Angelman syndrome.
The reprioritization effort and associated layoffs are expected to reduce PTC's annualized operating expenses by about 20 percent compared to its 2023 operating expenses guidance.
In August, PTC reported $213.8 million in revenue for the second quarter, up 29 percent compared to $165.5 million in Q2 2022, though its net loss grew year over year to $198.9 million from $152.1 million. As of June 30, the firm held $337.9 million in cash, cash equivalents, and marketable securities.
In reporting its Q2 financials, the South Plainfield, New Jersey-based firm projected total revenues in 2023 in the range of $940 million to $1 billion, and non-GAAP R&D and selling, general, and administrative expense for the full year between $810 million and $860 million, excluding around $120 million in non-cash stock-based compensation. The firm also said it expects to record a one-time expense of $62 million during the year if it achieves clinical and regulatory milestones from prior acquisitions and expenses under a rights exchange agreement.
PTC is still developing medicines for rare disorders within neurology, metabolism, and oncology. The firm is testing PTC518, an oral medication designed to degrade huntingtin messenger RNA in patients with genetically confirmed Huntington's disease with a CAG repeat length from 40 to 50 in an ongoing Phase II study, for example.
The company on Wednesday also said it will ask the European Medicines Agency's Committee for Medicinal Products for Human Use to reexamine its opinion on Translarna (ataluren), which is designed to enable cells to produce functional dystrophin for the around 15 percent of Duchenne muscular dystrophy patients with nonsense mutations.
CHMP earlier this month recommended against converting the drug's conditional marketing authorization to a full authorization after determining there wasn't enough evidence to confirm Translarna's clinical benefit. In a study involving DMD patients whose ability to walk was progressively declining, at 18 months of treatment, there was no significant difference between Translarna-treated patients and those in the placebo arm when comparing the distance they could walk in six minutes.
"We remain confident that we have the data to address the concerns raised by CHMP in its negative opinion," PTC CEO Matthew Klein said in a statement.
Revenues from PTC's DMD franchise grew 21 percent in Q2 2023 to $162 million compared to the year-ago period; Translarna sales accounted for $96 million of that revenue.