Imagine a revolutionary medical therapy that can subtly tweak your body at the genetic level, change how your DNA is read, and potentially change the course of a life-threatening disease with no known cure. On September 19, 2016, the Food and Drug Administration approved exactly that.
Developed by research-based biopharmaceutical company Sarepta Therapeutics, Exondys 51 (eteplirsen) is a personalized drug treatment for patients suffering from Duchenne muscular dystrophy, a progressive disease that occurs in one of every 3,500–5,000 male children worldwide. “The drug acts like a molecular bandage at the RNA level,” Sarepta’s Chris Verni says. “My dad has a science background, and he was just amazed. He said it sounds like science fiction—something one could not even imagine twenty or thirty years ago.”
As chief intellectual property counsel for Sarepta, Verni is stationed at the front line of the company’s legal IP function. With the support of his group, Verni’s role is to obtain and to defend the patents that ensure the business can continue to innovate with products such as Exondys 51 and develop additional pioneering treatments for Duchenne that improve patients’ lives. “Intellectual property is the lifeblood of a biotech company,” Verni says. “At the heart of the patent system is the need to protect what companies are innovating to provide a window of exclusivity to fuel future investment. In our case, the patents that we obtain today allow us to deliver our drugs to patients in the future.” Providing a window of exclusivity is pivotal and beneficial to patients and business alike because it allows for both consumer access and affordability for current and future therapies.
Verni was uniquely primed for his role after earning a bachelor’s degree in chemistry from the University of Rochester and working as a bench chemist for ArQule, a drug discovery company that services major pharmaceutical companies. “It was really in that context that I had a close view of an entrepreneurial biotech business that I thought was really interesting,” Verni says. However, he knew that he didn’t want to be a chemist forever. “I wanted to continue being involved in science but also wanted to become involved with biotech businesses to help them grow,” he says.
Stemming from a prescient observation that the world was beginning to evolve from a manufacturing-based climate to one centered on innovation, Verni decided to pursue patent law, which would allow him to have impact on the frontier of biotechnology. “I thought it would be great to be at the forefront of innovation and to work with companies as a patent attorney to help protect the technology and integrate the IP strategies that advance the business,” he says.
Fresh from law school, he went on to private practice at a national law firm and then took an in-house position at Fortune 100 pharmaceutical company Pfizer. “It was really an amazing experience to practice pharmaceutical-based patent law 24-7 at a company that’s very sophisticated,” he says. “Pfizer is named on a lot of important patent cases that have really shaped the law for pharmaceuticals.” From there, he went to Genzyme to hone his legal experience in the deal-making arena to help support the company’s emerging specialty transplant and oncology business.
“Patients don’t want to be denied treatment due to a patent matter.”
With Sarepta Therapeutics, Verni says he’s found the role he always wanted, and he applies industry experience to his position as the company’s first patent attorney in its thirty-five-year history. “The technologies underlying Exondys 51 are unique,” he says. “It’s one of three in a broad class of antisense oligonucleotides that have ever been approved by the FDA. We’ve had to be flexible in our thinking as a legal team and challenge our creativity in order to achieve our IP objectives. We haven’t fallen back on how people did things twenty years ago.”
Verni’s scientific background has not only given him the depth of knowledge to craft the company’s patents, but it has also allowed him to effectively highlight the chemical and molecular nuances of Exondys 51 in litigation to fact-finders and US Patent and Trademark Office (USPTO) examiners. This led the team to succeed in their legal arguments and obtaining sought-after patents for drug candidates. “As we were developing and filing for FDA approval of our lead drug candidate, a competitor, BioMarin, had also filed a new drug application at the FDA,” Verni says. “During the same time, our patent estates were competing in parallel at the USPTO.”
Although the drug candidates were both aimed at treating Duchenne muscular dystrophy through a process called exon skipping, Verni was able to show that Sarepta’s drug differed significantly from BioMarin’s. “We were in a horse race at both the FDA and the patent office, and the FDA ended up not approving BioMarin’s application,” he says.
At the same time, BioMarin was also seeking to lay claim to certain aspects of Sarepta’s drug treatment in the patent sphere, an effort that the USPTO ultimately ruled in Sarepta’s favor on the substantive issues. The rulings are currently on appeal. Because of this, Verni explains that one of his objectives is to be sensitive to the needs of the patient community. “The patients don’t want to be denied treatment due to a patent matter,” he says. “At the end of the day, we’re a drug company and our mission is to provide drugs to patients who need them. My role is to do whatever it takes in accordance with the law to accomplish that in the most effective way possible.”
The law constantly changes, and according to Verni, it tends to follow advances in technology rather than define them. “What we, as IP attorneys, need to do is manage that change and, frankly, to advocate for change where it’s needed,” Verni explains. “The high-tech, pharmaceutical, and biotechnology industries don’t always side the same way on issues. The best thing we can do is to be mindful of that and raise our voices as issues come up in legislation.”
Of course, the reason for all of this is to have the most positive impact on the lives of patients. “During the FDA approval process for Exondys 51, there was an advisory meeting that was open to the public, where, for over three hours, we heard from fifty-two different people and groups speaking about our drug candidate,” Verni recalls. “This was a combination of patients, families, and experts who had dedicated their lives to the drug hunt for Duchenne. It was really humanizing to hear their stories. It’s very gratifying to be part of a company that is doing something so meaningful for patients and their families.”