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Stuart Peltz, founder of PTC, retires after 25 years as CEO

After 25 years as CEO of PTC Therapeutics, Stuart Peltz is stepping down from his role as head of the rare disease drugmaker.

The company announced Peltz’s retirement on Friday, naming its chief operating officer Matthew Klein as his successor, effective immediately. Peltz will continue to serve as a senior adviser to PTC through April 2025 as well as join its scientific advisory board.

On an introductory conference call Friday morning, Klein said the transition had been planned “for quite some time,” and noted the announcement was timed to the 25th anniversary of PTC’s founding by Peltz and another member of the company’s board.

“We talk a lot internally about the importance of succession planning and my appointment to COO in 2022 was very much an intentional move to prepare me for this transition,” said Klein, who has led recent investor events, including presenting for the company at the J.P. Morgan Healthcare Conference in January.

Danielle Brill, an analyst at Raymond James, noted the transition was not a surprise to her team. “Stu had been increasingly taking a backseat role … and Matt in many ways, had already become the face of the company,” she wrote in a client note Friday.

Klein’s succession is a notable change in leadership for a company that’s been led by the same person for its entire corporate history. But, on Friday’s call, Klein emphasized PTC’s goals and vision would remain the same, and reiterated the company’s forecast of up to $1 billion in revenue this year.

While planning to stay the course, Klein did indicate he would work with the rest of PTC’s leadership to review the company’s strategy.

“Taking over the CEO helm puts me in a position to maybe have a slightly different perspective on this,” he said. “My plan is to continue to work with our leadership team … to revisit our pipeline, review our portfolio and review our strategy as a natural thing that we do as part of responsible and thoughtful corporate leadership.”

Under Peltz, PTC has brought to market several therapies, bought into rights of others and, along the way, steered the company through several ups and downs. The company developed a Duchenne muscular dystrophy treatment called Translarna that is sold in Europe. But PTC has tried unsuccessfully for years to win approval in the U.S.

PTC also acquired a second Duchenne medicine, the steroid Emflaza, through a then-controversial deal with Marathon Pharmaceuticals. Emflaza is now approved in the U.S.

The company also holds commercial rights to two rare disease therapies in Latin America. Most recently, it won European approval for a gene therapy for a condition known as AADC deficiency.

Arguably its greatest drug development success, however, has been Evrysdi, an oral treatment for spinal muscular atrophy that’s sold by Roche in the U.S. The drug generated about $1.2 billion in global sales in 2022, from which PTC earned about $114 million in royalties.