What’s Driving the Marin County Ultranyx CEO to Pursue Rare Disease Treatments

Emil Kakkis has spent most of his career developing drugs for diseases most people have never heard of.

The 61-year-old is the CEO and founder of Ultragenyx, a Marin County-based biopharmaceutical company focused on finding treatments for patients with rare genetic diseases.

Kakkis began his career in academia in Southern California, but has been in North Bay since 1998. He was at BioMarin Pharmaceutical in San Rafael before starting his own company in 2010. Today, Ultragenyx has over 1 100 employees worldwide.

Ultragenyx’s philosophy is to be “patient-centered”.

Kakkis said the traditional approach for drug companies is to determine the value of a drug, take a position on price, iron out discounts to get deals, and then for insurance companies “to try to figure out how to restrict access to the drug”. .”

He added: “No one is looking at how many people with this disease are going to get (the drug).”

Although Ultragenyx brought FDA-approved drugs to market, it did not make a profit. The company focuses on research and development as it grows.

The company went public in 2014. Its fourth-quarter and full-year earnings call will be on February 10. At the end of 2021, the company’s cash balance was approximately $1 billion.

Preliminary 2021 revenue totals for the drug Crysvita, which treats a condition where the body does not retain enough phosphorus, are expected to be around $193 million, with projections for 2022 to reach at least $250 million. For the company’s drug Dojolvi, the only FDA-approved treatment for children and adults with a rare metabolic disease called long-chain fatty acid oxidation disorders, the 2021 revenue forecast is at least $38 million, and 2022 should see numbers closer to $55 million. .

Kakkis reveals more about the company and himself in the following Q&A with the Business Journal.

Ultragenyx specializes in finding cures for rare diseases. How do you decide which diseases to focus on?

We focus on finding powerful treatments for bad genetic diseases and try to limit the influence of other considerations such as patents and established market sizes that often force other companies to overlook real opportunities.

We like to be opportunistic more than strategic and will sometimes pursue new diseases and fashions if a special opportunity presents itself, as happened with the Angelman Syndrome program with our partner (a biotech start-up) GeneTx.

The truth is, great science doesn’t just hang around. Most great projects are hard to find. Sometimes science comes from a random place and it may not fit your perfect strategy. The pressure is the biology of the disease and being able to treat it.

You worked for BioMarin for over a decade before launching Ultragenyx in 2010. Why did you want to start your own business?

I had known many patients and families (while trying to develop treatments for their ultra-rare diseases) and so I knew there was still a need for another rare disease society with a broader view of what which was possible.

Launching Ultragenyx was a chance to influence and shape a rare disease company early on. I could change the way we approached development, regulation, early access, patient discovery, parameter development and commercialization in an innovative way that is simply not possible with a large existing company . Fortunately, this dream came true.

What trends affecting your industry are keeping you up at night?

Of course, the global pandemic has kept many of us awake at night for the past two years, for both personal and professional reasons. Even with the ongoing pandemic, as a rare disease company, there is an urgent need to bring these new treatments to patients amid even more hurdles, including travel restrictions, clinic closures, stay-at-home orders, supply chain issues, or regulatory process delays.

Where will your business and industry be in the next five years?

Over the next five years, Ultragenyx will continue to bring new rare disease drugs to more patients around the world, and I hope our gene therapy platform will be used by Ultragenyx and our partners to advance our understanding. how to correct the underlying genetic causes. of certain diseases.

I believe that in that time frame, Ultragenyx will be one of the leading rare disease companies in the world with more types of treatments and diseases treated than any other company. And our approach to marketing focused on expanding accessibility and moderating pricing will become the norm for others.

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