Purdue Pharma will use Exicure’s Spherical Nucleic Acid (SNA)™ technology to discover and develop treatments for psoriasis and other diseases amenable to a gene-regulation approach, through a collaboration that could generate up to $790 million-plus for Exicure.

The companies have inked a strategic research collaboration, option, and license agreement that, upon exercise of options, gives Purdue Pharma full worldwide development and commercial rights to Exicure’s lead compound—the Phase II-bound AST-005—as well as rights to three additional collaboration targets with associated intellectual property.

AST-005 is a topically-applied SNA that targets tumor necrosis factor (TNF), a pro-inflammatory cytokine shown to be a key mediator of psoriasis. SNAs are nanoscale, spherical arrangements of densely packed and radially oriented nucleic acids, whose structure is designed to enable the safe and effective delivery into cells and tissues of therapeutic importance, without the need for additional physical or chemical methods or components.

On October 11, Exicure announced positive data from a completed Phase I trial in which 15 patient volunteers with chronic plaque psoriasis were treated with AST-005 for 10 days. The first trial with AST-005, based on Exicure’s proprietary SNA technology, met safety and tolerability requirements in patients with mild to moderate psoriasis.

In addition to the safety data typically collected from the first Phase I trial, pharmacodynamic assessments were performed from the treated plaque area. Treatment with the highest dosing strength of AST-005 gel resulted in a statistically significant decrease in TNF mRNA expression in the psoriatic skin, Exicure said.

According to Exicure, AST-005 can penetrate the skin when applied topically and knockout the TNF gene of interest due to the unique ability of SNA constructs.

Under the companies’ agreement, Exicure received an upfront payment, will receive an equity investment, and could potentially receive payments tied to achieving development, regulatory, and commercial milestones—all potentially totaling up to $790 million if all programs are fully developed, plus 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,” Purdue Pharma president and CEO Mark Timney said in a statement.

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