Ionis Pharmaceuticals has earned a $5 million milestone payment associated with initiation and enrollment in a newly initiated phase 1 clinical study of IONIS-JBI1-2.5Rx, an orally delivered antisense drug designed to treat patients with a gastrointestinal (GI) immune disorder by reducing the production of an undisclosed target locally in the GI tract. Janssen Biotech, Inc. (Janssen), which is responsible for global development, regulatory and commercialization activities, is conducting the phase 1 study.

"This study represents an important milestone for us and our antisense technology because IONIS-JBI1-2.5Rx is our first orally delivered antisense drug for the treatment of GI disorders to advance into the clinic. We are pleased with the success of our collaboration with Janssen. In under two years, we have advanced a drug into clinical trials and identified and initiated IND-enabling studies for a second drug development candidate, IONIS-JBI2-2.5Rx. Our collaboration with Janssen combines Ionis' broad applicability of our antisense technology with their expertise in GI autoimmune disorders and therapeutic formulation," said C. Frank Bennett, senior vice president of research at Ionis Pharmaceuticals.

Under its global collaboration agreement with Janssen, Ionis is eligible to receive nearly $585 million in development, regulatory and sales milestone payments and license fees. In addition, Ionis will receive tiered royalties that on average are double-digits on sales from any product that is successfully commercialized.