Ultragenyx Pharmaceutical, a biopharmaceutical company focused on the development of novel products for rare and ultra-rare genetic diseases, and Takeda Pharmaceutical, have formed a 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 Kakkis, chief executive officer, 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, chief medical and scientific officer, 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 $25 million 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 $25 million with no additional premium. A potential third equity investment by Takeda is contingent upon Ultragenyx achieving a specific development milestone on a second asset.