Skip to main content
Premium Trial:

Request an Annual Quote

PTC Therapeutics Cuts Multiple Gene Therapy Programs

NEW YORK – PTC Therapeutics is discontinuing preclinical and early research in multiple gene therapy programs as part of a strategic portfolio prioritization, the rare disease treatment company said Tuesday.

PTC is deprioritizing early-stage gene therapy development to focus on development programs "likely to deliver significant return on investment and transformative therapies for patients with high unmet medical need," the company said in a statement. The decision includes discontinuing preclinical-stage programs in Friedreich ataxia and Angelman syndrome.

The reprioritization effort, which will include layoffs, is expected to reduce PTC's residual 2023 operating expenses by 15 percent, according to the company.

"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 company plans to continue development of other therapies for central nervous system and metabolic disorders. PTC will continue development and commercialization efforts for its gene therapy to treat aromatic L-amino acid decarboxylase deficiency, Upstaza (eladocagene exuparvovec), which was granted marketing authorization in the EU and UK last year.

Klein, previously the company's chief operating officer, took the helm as CEO after founding CEO Stuart Peltz said he would retire in March. Peltz at the time said he would continue to serve as a senior consultant to the firm and join its scientific advisory board.

On Tuesday, PTC also said company CFO Emily Hill has been "relieved of her responsibilities" and will leave the company.

PTC recently posted $220.4 million in Q1 revenue, up 48 percent from $148.7 million in the year-ago quarter. The company reported a net loss of $139 million, or $1.88 per share, compared to a net loss of $126.7 million, or $1.78 per share, in last year's Q1. As of March 31, the firm held $286.3 million in cash, cash equivalents, and marketable securities.