Purdue Pharma and Exicure have announced a strategic research alliance and licensing agreement for AST-005, a potential treatment for psoriasis and other diseases amenable to a gene regulation approach by Exicure called spherical nucleic acid (SNA) technology.
Purdue will own full global rights to AST-005, among other collaboration targets.
Together with an upfront payment and equity investment, Exicure could be paid as much as $790 million.
SNAs are immune-modulatory agents that allow for highly targeted gene regulation. The technology is based on the spherical, dense and radial architecture of these specific nucleic acids, which help overcome one of the most difficult obstacles to nucleic acid-based therapies: delivering therapeutics into cells and tissues safely and effectively, without the need for additional physical or chemical aids.
AST-005 is an investigational SNA that targets tumor necrosis factor (TNF), a pro-inflammatory cytokine shown to play a key role in the development of psoriasis. The treatment is applied as a topical gel.
In preclinical studies, AST-005 led to a significant decrease in TNF in viable human psoriatic skin samples.
In a recently completed Phase 1 trial, launched last April, 15 volunteers with chronic plaque psoriasis received AST-005 for ten days. It was the first trial with AST-005, based on Exicure’s SNA.
The Phase 1 trial evaluated the safety and tolerability of AST-005, and all requirements were met. Findings showed that treatment with the highest dosing strength of AST-005 resulted in a statistically significant decrease in TNF expression in psoriatic skin.
“We believe these results provide the proof that SNA technology can potentially be used to regulate gene expression in a variety of local applications, including those in the skin, eye, lung and GI tract,” said David Giljohann, PhD, chief executive officer of Exicure.
Exicure received an upfront payment, will receive an equity investment and could receive development, regulatory and commercial milestone compensation.
“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,” Mark Timmey, president and chief executive officer of Purdue Pharma, said in a press release.
