From surgeon to CEO
As a surgeon working in reconstructive burn surgery, Matt Klein ran an intensive care unit treating severely injured children and adults.
He sees the experience as comparable to his current role as CEO at PTC Therapeutics, a global biopharmaceutical company that delivers transformative therapies for people living with rare diseases.
Both involve leading multidisciplinary teams and making important decisions based on limited data, he points out.
And life at PTC is not so different, he says, from “working in a high-pressure environment like an operating room, where literally, life and death hangs in the balance, but getting people to not be deterred by that anxiety and that challenge.”
Klein took over at PTC in 2023. At that point the company already had almost 25 years under its belt, having launched as a pioneer in RNA (ribonucleic acid) therapies.
Today the company’s range has widened to embrace multiple treatments and platforms, but the original vision remains, he emphasizes: “We're growing, we're evolving, but we're never going to get away from that patient-focused mission that we started with over a quarter century ago.”
Partnerships foster programs – and profit
PTC was the first company to develop a drug for Duchenne muscular dystrophy. Its RNA expertise then led to the launch of a small molecule splicing platform.
“The first product of our splicing efforts is a drug called Evrysdi, which we developed along with Roche, and is now the leading therapy for spinal muscular atrophy worldwide,” Klein says.
This project is one of several partnerships forged by PTC: “We've been very thoughtful in terms of combining both home-grown science and business development,” he adds.
Besides advancing therapy programs, these partnerships are bolstering Klein’s priority of moving the company to a break-even point and ultimate profitability.
For example, a deal with Novartis saw the pharma gain rights to PTC518, which PTC hopes will become the first oral disease-modifying therapy for Huntington’s disease. The arrangement includes $1 billion upfront, milestones of up to $1.9 billion, a 40% U.S. profit share and double-digit tiered royalties on ex-U.S. sales.
“With that money coming in, we closed Q1 of 2025 with over $2 billion in cash,” Klein says. “We're managing our expenses and now have sufficient capital to do all that we plan to do, including potential commercial launches for several programs, and get to cashflow break-even without needing to raise additional capital.”
“We now have sufficient capital to do all that we plan to do, including potential commercial launches for several programs, and get to cashflow break-even without needing to raise additional capital.”
Matt Klein, CEO, PTC Therapeutics
Poised for commercialization
In the short term, PTC is awaiting some key decisions on programs. Sephience, its treatment for the rare metabolic disease phenylketonuria (PKU), has earned approval from European regulators and a verdict from the FDA is expected soon.
Vatiquinone is the subject of another pending decision. “If vatiquinone is approved by the FDA, it’ll be the first-ever therapy for kids with Friedrich’s ataxia, and that's really important, given how aggressive and debilitating Friedrich’s ataxia can be for pediatric patients,” says Klein.
The latter drug is a product of PTC’s platform investigating ferroptosis, an important pathway for the way cells die in inflammatory diseases. The company now has a suite of programs targeting ferroptosis for neurological disorders and other therapeutic areas.
The company’s platforms allow it to speed up development of successive drugs, says Klein. “Through continuous learning, we’ve now developed something we call PTSeek, a high-throughput screening engine that has cut down in years the time it takes to identify a target for splicing and small molecules that can affect that target,” he says.
“We focus on the things that we know we can do that others can't. And we have a long view: we understand that it may take several efforts. if you don't succeed at first, that's really a learning opportunity to go back and do be better the second time.”
“We focus on the things that we know we can do that others can’t, and we have a long view.”
Matt Klein, CEO, PTC Therapeutics
