Rare disease biotech Ultragenyx Pharmaceutical and mRNA therapeutics company Arcturus Therapeutics are expanding their collaboration to discover and develop mRNA, DNA and siRNA therapeutics for up to 12 rare disease targets using Arcturus’ RNA therapeutics platforms.
Photo: Joseph Payne, president and CEO of Arcturus
Arcturus platform can be applied toward multiple types of nucleic acid medicines including small interfering RNA, messenger RNA, replicon RNA, antisense RNA, microRNA, DNA, and gene editing therapeutics.
As part of the expanded agreement, Arcturus will receive a $6 million upfront payment. Ultragenyx will also make a $24 million equity investment in Arcturus, with an option to purchase additional shares at a premium, making it the largest shareholder of the San Diego-based company. Karah Parschauer, general counsel of Ultragenyx, will join Arcturus’ board of directors and Emil Kakkis, president and CEO of Ultragenyx, will join as an Arcturus board observer.
Arcturus is also entitled to preclinical, clinical, regulatory, and sales milestone payments for each product developed under the collaboration, and royalties on commercial sales. 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. Ultragenyx will also reimburse Arcturus for related research expenses.
“The expansion of the collaboration with Ultragenyx underscores the partnership’s early successes and ongoing commitment,” said Joseph Payne, president and CEO 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.”
The original collaboration and license agreement between the two companies was signed in October 2015 and they 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 (GSDIII), or Cori disease, an inherited autosomal recessive disorder caused by mutations in the AGL gene that lead to the buildup of glycogen in the body’s cells that impair the function of certain organs and tissues, especially the liver and muscles. Symptoms typically begin in infancy and may include hypoglycemia, hyperlipidemia (excess of fats in the blood), and elevated blood levels of liver enzymes; later symptoms may include hepatomegaly, chronic liver disease (cirrhosis) and liver failure later in life. Currently there are no approved treatments for GSDIII and symptoms are managed through diet.
Author: Rare Daily Staff
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