Hope for DMD Patients

Each night, 30 million people in the US go to bed knowing that a cure for their disease does not exist. Experts define a rare disease as a condition that affects fewer than 200,000 people. Only about 500 out of 7,000 known rare diseases have FDA-approved treatments.

In 1983, Congress passed legislation designed to incentivize the development of drugs, therapies, and devices that target rare diseases. In response, many pharmaceutical companies rushed to form specialized drug development teams. While these groups include epidemiologists, clinical research scientists, lab techs, and biochemists, the most effective teams work in step with their business counterparts to bring new products to market.

Lawyers develop IP strategies, government affairs specialists monitor regulatory issues, and communications managers draft marketing materials. Ryan Wong is a certified public accountant with more than fifteen years of experience in pharma and biotech. He joined Sarepta Therapeutics in 2021 to lead a finance transformation and help the global biotechnology company prepare for the release of a breakthrough therapy that treats Duchenne muscular dystrophy (DMD).

DMD is a genetic neuromuscular disease caused by a gene mutation that damages and weakens the muscles. Most patients must use wheelchairs before their teenage years and will eventually die when their heart and lung muscles deteriorate. DMD impacts about 1 in every 5,000 individuals worldwide. While the majority of patients are male, a small percentage of patients are female.

When Wong came to Sarepta, the company’s novel gene therapy for DMD patients was in clinical trials. “I joined this company to help develop the first gene therapy for DMD and bring this treatment to a patient population that’s been devastated by a fatal disease that doesn’t have a cure,” he says.

“We believed this therapy, if approved, would be in high demand and bring a period of robust revenue growth and cash flow,” he says. Wong was tasked with preparing the company to transition from a loss-making company to a profitable biotech with Wall Street expectations.

Wong’s unique experience equipped him to navigate the challenge before him. The CPA began his career in public accounting with Deloitte before joining Allergan. He’s spent the last fifteen years building his expertise in pharma and biotech finance.

Sarepta already had three approved therapies to treat DMD. However, those RNA therapies only reach about 30 percent of the DMD population. The adeno-associated virus vector-based gene therapy known as Elevidys reaches a much larger percentage of patients. According to a Sarepta press release, the product was designed “to address the underlying genetic cause of Duchenne muscular dystrophy—mutations or changes in the DMD gene that result in the lack of dystrophin protein—through the delivery of a transgene that codes for the targeted production of Elevidys micro-dystrophin in skeletal muscle.”

In July 2021, Wong developed a plan to “level-up” Sarepta’s internal finance team to properly support the business. Together, they introduced the financial education and discipline that would help guide strategy and enable outcomes as Elevidys wound its way through the development and approval process.

In 2023, an FDA advisory committee was held to discuss the Biologics License Application (BLA) for Elevidys and the data supporting the treatment. The panel of experts composed of chemists, pharmacologists, statisticians, and physicians asked questions and gave Sarepta the chance to answer, and offered a forum for public comment.

Wong and many of his colleagues gathered to watch a broadcast of the event and await the panel’s decision. The advisory committee voted to recommend approval. When the approval news came a few weeks later, it brought a mixed reaction. The FDA approved Elevidys but restricted its label for the treatment of patients ages four and five. The agency requested more data to consider a broader application. “Getting initial FDA approval for Elevidys was an incredible win for Sarepta, but we knew our work was not done,” says Wong.

In the months that followed, research and development teams worked overtime to complete additional trials and submit a supplement to Sarepta’s FDA application. In June of 2024, the company announced the FDA-approved expansion of Elevidys to treat Duchenne patients aged four and above. Today, the treatment can now reach the majority of patients suffering from DMD.

As Wong and his colleagues work behind the scenes to bring these therapies to patients, they often receive letters or visits from patients and their families. These stories motivate Wong to continue his work. “We are on an urgent mission to help these patients,” he says. “They inspire us and make this work so rewarding and meaningful.”