Ultragenyx Pharmaceutical Inc and Takeda Pharmaceutical Company enter into a Collaboration to Develop Rare Genetic Diseases


Ultragenyx Pharmaceutical Inc. a biopharmaceutical company focused on the development of novel productsfor rare and ultra-rare genetic diseases, and Takeda Pharmaceutical Company Limited announced a strategic partnership to develop and commercialize therapies to treat rare genetic diseases.

Ultragenyx will initially receive an exclusive license to one preclinical Takeda product candidate in a pre-determined field of use, and will have an exclusive option to co-develop and co-commercialize the product candidate in additional therapeutic areas. The companies have also established a five-year research collaboration in which Ultragenyx will have the option to license up to five additional Takeda product candidates for rare diseases after the parties agree on and conduct initial validation activities under the purview of a Joint Research Committee.

“This broad collaboration provides Ultragenyx with a product opportunity that is approaching clinical-stage development as well as a potential continued source of new product candidates that will help us achieve our goal of bringing a new therapy into the clinic every one to two years,” said Emil D. Kakkis, MD, PhD, Chief Executive Officer of Ultragenyx. “Takeda has an impressive early pipeline of therapies with potential across a number of rare genetic diseases, and we are pleased that Takeda has chosen to partner with us to bring these therapies to patients with rare diseases that have few or no treatment options.”

"Ultragenyx is a rapidly emerging rare disease company, led by a highly experienced and successful management team," said Andrew Plump, M.D., Ph.D., Chief Medical and Scientific Officer of Takeda. “This partnership provides Takeda access to Ultragenyx’s strong patient-centric development and regulatory capabilities in the rare disease space, and could create significant value for both companies by delivering important new therapies to patients.”

Takeda will receive an exclusive option to commercialize any licensed products resulting from the collaboration in Asia, including Japan. In addition, Takeda receives an option to exclusively license one Ultragenyx pipeline product in Japan. Each company will receive potential development and sales milestone payments and royalties on net sales of licensed products by the other party.

Takeda will invest up to $65 million in Ultragenyx in two tranches, the first of which will comprise a $25million stock purchase along with a $15 million cash premium at closing. This will be followed at Ultragenyx’s option, within 12 months, by a second equity purchase of $25million with no additional premium. A potential third equity investment by Takeda is contingent upon Ultragenyx achieving a specific development milestone on a second asset. No additional financial details were disclosed.

The completion of the transactions are subject to Hart-Scott-Rodino Antitrust Improvements Act of 1976 (HSR) review and the satisfaction of other customary closing conditions.

About Ultragenyx
Ultragenyx is a clinical-stage biopharmaceutical company committed to bringing to market novel products for the treatment of rare and ultra-rare diseases, with a focus on serious, debilitating genetic diseases. Founded in 2010, the company has rapidly built a diverse portfolio of product candidates with the potential to address diseases for which the unmet medical need is high, the biology for treatment is clear, and for which there are no approved therapies.

The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency.

For more information on Ultragenyx, please visit the company’s website at

About Takeda
Takeda Pharmaceutical Company Limited (TSE: 4502) is a global, R&D-driven pharmaceutical company committed to bringing better health and a brighter future to patients by translating science into life-changing medicines. Takeda focuses its research efforts on oncology, gastroenterology and central nervous system therapeutic areas. It also has specific development programs in specialty cardiovascular diseases as well as late-stage candidates for vaccines. Takeda conducts R&D both internally and with partners to stay at the leading edge of innovation. New innovative products, especially in oncology, central nervous system and gastroenterology, as well as its presence in emerging markets, fuel the growth of Takeda. More than 30,000 Takeda employees are committed to improving quality of life for patients, working with our partners in health care in more than 70 countries. For more information, visit

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