It took less than a decade for a small start-up to transform into a billion-dollar global enterprise. And now, that biopharmaceutical company is undergoing its latest metamorphosis to seek major gains in narrow spaces. Founded in 2008 with a handful of employees and no office, the precursor to Horizon Pharma began with a big idea: accessible medicines for unmet patient needs. Today, with more than one thousand employees, that means treating rare diseases. And thanks to Joe Whalen, patients on the fringe of medical knowledge take center stage.
“Unless you know someone impacted, you may not have heard about these rare diseases, so we really see the benefit in helping these patients,” Whalen says. “As we grew, we could see that rare diseases had the highest unmet treatment need.”
Less than 5 percent of about seven thousand rare diseases receive one FDA-approved treatment, and Whalen says that those rare diseases, which affect less than 200,000 people, also map the high-growth trajectory for Horizon Pharma. Since its founding in 2008 as a small start-up, Horizon Pharma is continuing to grow as it aims to expand its pipeline of rare disease medicines with the help of Whalen, senior vice president of business development and alliance management.
But before coming to Horizon Pharma, Whalen was earning his BBA in accounting from the University of Notre Dame before earning his MBA in finance at the University of Illinois Urbana-Champaign. He started his career at Searle as a business analyst in 1994 and later supported the strategy for pharmaceutical products in various Europe and US markets. In 2001, he joined Baxter Healthcare as a senior manager of transactions analysis prior to his promotion to director of business development two years later.
He left Baxter to consult for Horizon Therapeutics during its merger with Nitec Pharma in 2010. “We were in survival mode,” Whalen recalls. Three months later, Whalen arrived at the renamed Horizon Pharma and helped take it public a little more than a year later. Specializing in medications for arthritis, pain, and inflammatory diseases, its net sales comprised only two drugs, DUEXIS and RAYOS, by 2013. The risk of stagnation in a crowded market loomed.
So, Horizon Pharma’s leadership team, led by CEO Tim Walbert, envisioned a strategic pivot to not only evolve the company, but also to better serve a new demographic of patients in untapped markets. These diseases include everything from severe fungal infections, such as chronic granulomatous disease (CGD) to urea cycle disorders and more. “We know that drug development is risky, so we feel we need a number of shots on goal to be successful,” Whalen says.
Whalen helps lead advances in this new direction through key acquisitions. In the last five years, Horizon Pharma landed eight groundbreaking deals to expand its product pipeline: VIMOVO from AstraZeneca in 2013; Vidara Therapeutics and PENNSAID 2% from Nuvo Research in 2014; Hyperion Therapeutics in 2015; Crealta, Raptor, and the global rights to Imukin in 2016; and River Vision Development Corp. last year.
“There are a lot of companies that start with one or two products like we did, but then realize the better option for them is to sell and put it in the hands of people like us who have the late-stage clinical experience to get it approved and the commercial experience to get it to patients,” Whalen explains. “And if we have drugs that can benefit patients outside the United States, then we look for partners to distribute those drugs there to maximize the potential for our products to reach patients.”
The results have been staggering, including a 93 percent compound annual growth rate from 2013 to 2017 based on consensus. The first nine months of 2017 comprised eleven medicines, six of which treat rare diseases. According to Whalen, Horizon Pharma will continue to invest in its orphan business unit with the cash flow from its more conventional primary care division. The stability of its osteoarthritis treatments, for example, is supporting Horizon Pharma’s move into uncharted territory.
Dennis Bennett, partner at Global Patent Group, works hand in hand with Whalen when it comes to patent due diligence, and has seen Whalen’s effective leadership firsthand. “Joe is a wizard at leading the disparate group of experts to do the ‘deep dive’ due diligence,” Bennett says.
Nephrology, or kidney health, is one such promising space. About 25–50 percent of patients with chronic kidney disease also suffer from gout—painful inflammation of the joints. Yet mainstream therapies are limited when it comes to treating both diseases together, as needed for about 100,000 patients in the United States. Horizon Pharma’s answer: KRYSTEXXA, the first and only biologic treatment for gout patients.
Another avenue of expansion for the biopharmaceutical company is endocrinology, the field of hormone-related diseases. When Horizon Pharma acquired teprotumumab last year, it also potentially gained the first FDA-approved therapy for thyroid eye disease (TED) as its first acquisition of a development-stage medicine for rare diseases.
“This is a painful condition,” Whalen says. “The only treatments right now are steroids, which have limited efficacy, or surgery for severe cases.” This move marked the company’s aim to serve more than 15,000 TED patients in the United States as well as a stake in the annual sales potential of more than $250 million.
One product in particular hits close to home for Whalen. ACTIMMUNE, which treats CGD, is also being tested in three clinical trials to study its potential as an immune booster for cancer patients. “My mom is a breast cancer survivor,” he says. “She wouldn’t be here without the US healthcare system.”
Horizon Pharma partners with the Moffitt Cancer Center and Research Institute, Fox Chase Cancer Center, and National Cancer Institute. Whalen adds that the company also invites patients, within or outside the company, to speak at the office. “People leave those meetings with tears in their eyes,” he says. “It’s motivating to continue to work to find the next solutions for patients.”
With each new acquisition and research breakthrough, Horizon Pharma is proving its capacity for high growth in narrow segments. Looking ahead, Whalen says he will continue building long-term value though a collegial approach for Horizon Pharma as it delves further into the identification of undiagnosed patients and unique treatments.
“The pace is fast, and it’s always changing. But we’re making a difference in people’s lives,” Whalen says. “When I look back and see how far we’ve come, it’s been an amazing ride.”
Photo by Sandy Frias