Ultragenyx and Arcturus Expand Existing Research Collaboration and License Agreement to Develop Additional Nucleic Acid Therapies for Rare Diseases

Ultragenyx Pharmaceutical, Inc. and Arcturus Therapeutics Holdings Inc. announced that they have expanded their collaboration to discover and develop mRNA, DNA and siRNA therapeutics for up to 12 rare disease targets.

“This expanded collaboration further solidifies our mRNA platform by adding additional targets and expanding our ability to potentially treat more diseases,” said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. “We are pleased with the progress of our ongoing collaboration. Our most advanced mRNA program, UX053 for the treatment of Glycogen Storage Disease Type III, is expected to move into the clinic next year, and we look forward to further building upon the initial success of this partnership.”

“The expansion of the collaboration with Ultragenyx underscores the partnership’s early successes and ongoing commitment,” said Joseph Payne, President and Chief Executive Officer of Arcturus. “Our expertise in the discovery, early development and manufacturing of RNA medicines aligns well with Ultragenyx’s proven clinical development and commercial experience in rare diseases.” He further added, “We look forward to utilizing Ultragenyx’s seasoned experience and presence on our Board of Directors to advance mRNA therapeutics as a new class of genetic medicines, and build Arcturus into a leading, independent biopharmaceutical company that delivers value to patients and shareholders.”

In connection with the amendment to the license agreement, Ultragenyx made a $6 million cash upfront payment to Arcturus. Ultragenyx is also purchasing 2,400,000 shares of Arcturus’ common stock at a stated value of $10 per share. Ultragenyx has an option to purchase an additional 600,000 shares of Arcturus’ common stock at $16 per share. Ultragenyx will become Arcturus’ largest shareholder with Karah Parschauer, J.D., General Counsel of Ultragenyx, joining Arcturus’ Board of Directors and Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx, joining as an Arcturus Board Observer.

Arcturus is entitled to preclinical, clinical, regulatory, and sales milestone payments for each product developed under the collaboration. Under the amended license agreement, certain early-stage milestone payments are reduced and the total potential milestone payments are increased due to the expanded number of targets. Arcturus is also entitled to reimbursement of related research expenses and royalties on commercial sales.

The original collaboration and license agreement between Ultragenyx and Arcturus was signed in October 2015. The two companies have been working together to develop mRNA therapeutic candidates for certain rare disease targets. The first disclosed indication under the collaboration is Glycogen Storage Disease Type III, and an Investigational New Drug (IND) application for this mRNA therapeutic program, UX053, is expected to be filed in 2020.