December 28, 2017
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Ionis begins clinical study of oral antisense GI drug

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Ionis Pharmaceuticals announced it has begun enrollment in a phase 1 clinical study of its oral antisense drug in patients with a gastrointestinal autoimmune disorder, earning a $5 million milestone payment from its partner Janssen Biotech.

IONIS-JBI1-2.5Rx is an orally administered 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,” according to a press release.

“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,” C. Frank Bennett, senior vice president of research at Ionis Pharmaceuticals, said in the press release. “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 [investigational new drug-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.”

Ionis could receive nearly $585 million in milestone payments and license fees under its global collaboration with Janssen, which will lead the phase 1 study, and global development, regulatory and commercialization activities, per the press release. Ionis will also receive tiered royalties on sales from any successfully commercialized products.

Disclosures: Bennett is employed by Ionis.