BIO: In conversation with Emil Kakkis, Ultragenyx CEO

Emil Kakkis, M.D., Ph.D., went from clinical practice to industry when he couldn't find a partner to help him advance a rare disease treatment he had developed. (Amirah Al Idrus)

PHILADELPHIA—When Emil Kakkis, M.D., Ph.D., started Ultragenyx Pharmaceutical, “it was me and my secretary on April 22, 2010.” Nine years later, it’s grown to more than 700 employees and two marketed products, with three clinical-stage programs and a slew of preclinical prospects right behind them. 

An often-touted edge that small biotechs have over their Big Pharma counterparts is a mix of passion, urgency and the nimbleness required to not just innovate, but to follow through and deliver new medicines to patients. It’s not news, but bureaucracy is most often fingered as the killer of innovation at larger companies and is something many small companies are leery of. 

“A lot of early-stage companies, including ourselves, don’t want bureaucracy. They don’t want controls,” Kakkis told FierceBiotech. “But that all changes when you become a commercial company … As we grow, the truth is, we have to implement controls and rules because of laws that exist and our responsibility as a company.” 

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In short, the rules and regulations are coming. The question is, said Kakkis, is whether a company can hold onto its entrepreneurial spirit and keep “finding new products in its development engine” as it progresses from clinical- to commercial-stage. 

To keep its innovation engine going, Ultragenyx has set up a translational research team that manages the early-stage pipeline. Any employee can come up with an idea, pitch it to the group and get it funded from a separate budget. Like a small biotech raising venture funding, a group working on such a project can grow and eventually become part of Ultragenyx’s portfolio. 

“We purposely allow them to do kind of crazy things and make mistakes. We assume that’s what will happen because the budget is relatively small and tight, and they can get a lot of things done.” 

“We are incredibly proud of this kind of environment; it keeps a small-company feel,” Kakkis said.  

Though a rare disease company wasn’t a tough sell in 2010, securing capital was hard during the “hangover” from the financial crisis. Ultragenyx ended up raising $45 million in series A funding in 2011, a sum that may be dwarfed by the ever-increasing VC rounds of today but would have been considered sizable up until a few years ago. 

RELATED: Biotech's top 10 money raisers of 2018 

Kakkis had aimed for $30 million, which he figured would fund two programs through phase 2. The key thing, he said, was having enough money to hire the right people to take those programs forward.

“What we are seeing now is that whole idea supersized. That means not just getting good talent; it means getting a super team,” he said. “The truth is that a larger amount of money can become self-fulfilling. A high-quality team gives a company the flexibility to do what it needs to do to win.” 

Because plans change and drugs fail, “it’s not about the initial plan being successful. It's about having a team that can find their way to a final plan that does succeed.” 

As for how the rare disease field has changed since he founded Ultragenyx, Kakkis is pleased about the different treatment methods that have emerged, “opening the door to diseases that have never been treated before.” 

“It is the golden age of rare disease treatment these days. But as more and more companies come into the space, we need to make sure they are understanding the full responsibility of their participation in this space,” he said. 

“If you develop a rare disease drug, you are now the most important thing to those patients’ lives. A high level of responsibility comes with the privilege to take care of those patients.”

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