Ultragenyx, Arcturus sign agreement to develop mRNA therapeutics to certain rare diseases

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, and Arcturus Therapeutics, Inc., a leading RNA medicines company, today announced that they have entered into a research collaboration and license agreement to discover and develop messenger RNA (mRNA) therapeutics to certain rare disease targets using Arcturus' UNA Oligomer™ chemistry and LUNAR™ nanoparticle delivery platform.

Under the terms of the agreement, Ultragenyx will make an upfront payment of $10 million to Arcturus. During the initial phase of the collaboration, Arcturus will design and optimize mRNA therapeutics for two selected rare disease targets. Ultragenyx has the option to add up to eight additional rare disease targets during the collaborative research period. Ultragenyx will be responsible for the development and commercialization of all products under the collaboration. Arcturus will be entitled to preclinical, clinical, regulatory, and sales milestone payments of up to $156 million for each target, as well as reimbursement of all research expenses and mid-single to low double-digit royalties on commercial sales.

"This collaboration with Arcturus will help us address a wider range of rare diseases than is possible with currently available approaches," said Emil D. Kakkis, MD, PhD, Chief Executive Officer of Ultragenyx. "The joint program combines Arcturus' technology platform, which we believe solves some of the key issues associated with mRNA therapeutics, with Ultragenyx's expertise in rare disease drug development."

"The Ultragenyx-Arcturus partnership is an ideal combination to leverage the potential of mRNA therapeutics to provide important new treatments for rare disease patients," said Joseph E. Payne, President and CEO of Arcturus Therapeutics. "Arcturus is thrilled to work with Ultragenyx, one of the premier rare disease companies, to identify treatments for challenging rare disease targets using our messenger RNA therapeutics platform."