Ultragenyx - 2013 Fierce 15

A global game for rare disease

Based: Novato, CA
Founded: 2010
CEO: Emil Kakkis
Clinical focus: Rare diseases
Website: www.ultragenyx.com

The scoop: Most three-year-old biotech toddlers would be happy at that stage of the game to find some big partners to work with as they figure out how to balance a load of R&D work on a slim budget. At Ultragenyx, the executive team was still adding new programs to an already ambitious slate of projects.

What makes Ultragenyx fierce: The most recent addition to the pipeline is KRN23, an antibody designed to treat rare cases of X-linked hypophosphatemia that is now wrapping a Phase I/II study at Kyowa Hakko Kirin. At the time KRN23 showed up on Ultragenyx's radar, it was the only rare-disease program at the Japanese pharma company, says CEO Emil Kakkis. When the deal was done just days ago, it was Ultragenyx's fourth midstage therapy.

For a biotech to achieve that kind of growth while banking $105 million in commitments from some top venture capital backers is extraordinary. Kakkis and his team, though, are in a hurry to build a contender in one of the hottest arenas in biotech, carving out a place under the orphan-drug sun that is intended to win the hearts of investors for an IPO that's already on the drawing boards.

"We couldn't possibly do any more programs," says Kakkis, noting that the biotech now has 5 programs in clinical development. "This is the limit of our capacity," he adds, noting that the plan was to have three programs in the clinic at any one time. The KHK opportunity was too good to pass up, though. So now Ultragenyx will build a dedicated research team to spur development.

That's been Kakkis' plan from the start. When he talked to FierceBiotech in early September from a conference in Barcelona, the biotech had a roster of 45 employees. About another dozen will be brought in to fill the remaining spots in the lineup. And they will be the original core group split into teams dedicated to building what Kakkis--a veteran investigator at BioMarin--envisions as a global rare-disease company, complete with marketing operations in the U.S. and key regions around the world.

"The truth is, companies in rare diseases that restrict themselves to the U.S. give up too much value too early," says Kakkis. The U.S. may have fewer reimbursement issues, but why give up South American markets that can deliver more patients than the U.S. for the drugs you plan to sell? That's why he wants to create a worldwide organization.

Ultragenyx, which operates out of a base north of San Francisco, is not in the business of building new drugs for other companies. As the CEO puts it: "I don't want to be a CRO."

Kakkis is also changing the rules on patient recruitment. His VP of commercial planning goes out and recruits patients for their studies rather than waiting for the sites to draw them in. And that way the company can accelerate a timeline--Kakkis calls it changing the pathway in a regulatory-compliant way--that now has the potential to read out key midstage data on four programs next year. In the fast-paced world of rare diseases, Kakkis is determined to shave time off the record.

He also believes he has a good story to tell investors.

"We're planning to go into the public market next year," says the CEO. That's how you build a company for the long run, he adds, giving you a much better shot at recruiting the best and the brightest people in the business.

Says Kakkis: "I want people passionately interested in getting drugs approved, not flipping them. I want to build a company that does that better than anybody else. I can't prevent a buyout after going public, but I can keep the value so high it would force overpaying to get us."

That way, he can be left alone to make sure Ultragenyx reaches its full potential.

Investors: Adage Capital Partners, T. Rowe Price Associates. Jennison Associates brought in clients. Funds and accounts under management by subsidiaries of BlackRock, Sanofi-Genzyme BioVentures 1, Shire and additional public market funds have also chipped in. Founding investors are TPG Biotech, Fidelity Biosciences, HealthCap and Pappas Ventures.

For more:
Startup Ultragenyx snags PhII program for growing rare-disease pipeline
Ultragenyx snags another candidate for its rare disease drug pipeline
Investors gamble $75M on Ultragenyx's plan to jump-start rare-disease player

-- John Carroll (email)

Ultragenyx - 2013 Fierce 15