Purdue Pharma L.P. and Exicure, Inc. have announced a strategic research collaboration, option and license agreement to discover and develop a treatment for psoriasis, and other diseases amenable to a gene regulation approach utilizing Exicure’s Spherical Nucleic Acid (SNA)™ technology. Purdue Pharma, upon exercise of the options, will obtain full worldwide development and commercial rights to AST-005, and rights to three additional collaboration targets with associated intellectual property. The lead compound, AST-005, is an investigational, topically-applied SNA that targets tumor necrosis factor (TNF), a pro-inflammatory cytokine shown to be a key mediator of psoriasis. In a recently completed Phase 1 trial, fifteen patient volunteers with chronic plaque psoriasis were treated with AST-005 for ten days. The first trial with AST-005, based on Exicure’s proprietary SNA technology, met the safety and tolerability requirements in patients with mild to moderate psoriasis. In addition to the safety data typically collected from the first Phase 1 trial, pharmacodynamic assessments were performed from the treated plaque area.
Psoriasis is an inflammatory disease that results in red, scaly patches on the skin. Current treatments for psoriasis include biologics, orally administered drugs and a variety of non-targeted topical treatments. The unique ability of SNA constructs allows AST-005 to penetrate the skin when applied topically and knock down the TNF gene of interest.
Under the terms of agreement, Exicure received an upfront payment, will receive an equity investment and could potentially receive development, regulatory and commercial milestone compensation, which together with the upfront payment and equity investment, could total up to $790 million, if all programs are fully developed, as well as royalties on potential sales of the products.
“This long-term strategic collaboration agreement represents a significant expansion of Purdue’s pipeline, and provides us with the potential to further our mission of developing novel therapies for patients with chronic conditions,” said Mark Timney, President and Chief Executive Officer, Purdue Pharma L.P. “We look forward to combining Exicure’s SNA technology and deep expertise in this field with Purdue’s resources and expertise in clinical studies, regulatory affairs and commercialization for the benefit of patients.”
“Given the broad potential of SNA technology, we want to forge long-term strategic relationships with best-in-class companies. As we were looking for a global leader with a proven track record in the development and global commercialization of treatments for patients with chronic conditions, we were very impressed with the Purdue Pharma’s clinical and commercial capabilities,” said Dr. David Giljohann, Chief Executive Officer, Exicure.