BioCryst Pharmaceuticals, Inc. and Clearside Biomedical, Inc. announced the entry into a license agreement enabling BioCryst to develop its investigational plasma kallikrein inhibitor, avoralstat, with Clearside's SCS Microinjector to deliver avoralstat directly to the back of the eye through the suprachoroidal space to treat patients with diabetic macular edema (DME). Avoralstat has high potency and low solubility, which are two characteristics important to achieving potential efficacy with reduced dosing frequency in the eye for DME patients. Delivering avoralstat directly into the suprachoroid space could allow avoralstat to inhibit plasma kallikrein at the sites of edema formation in DME disease, the retinal and choroidal vascular endothelium.

Avoralstat was previously evaluated in an oral formulation in a Phase 3 clinical trial in patients withhereditary angioedema (HAE). In the HAE clinical trial program in 276 individuals, oral avoralstat was safe and well tolerated with an adverse event profile similar to placebo. Under the terms of the agreement, Clearside will receive a $5 million upfront license fee from BioCryst.

Clearside is eligible to receive up to an additional $30 million in clinical and regulatory milestone payments, and up to a total of $47.5 million in three post-approval sales-based milestone payments as annual global net product sales progress to $2 billion. BioCryst will pay Clearside tiered mid-single digit royalties on annual global net product sales, at three tiers, including a top tier of > $1.5 billion. DME is the most common cause of vision loss in individuals with diabetes and at least one-third of patients have persistent DME despite treatment with anti-VEGF therapies, which are administered via monthly injection.

Data have shown that elevated kallikrein may be a cause of non-response to anti-VEGF therapy. BioCryst will host a Research and Development (R&D) Day at 1:00 pm ET at its Discovery Center of Excellence in Birmingham, AL. At the R&D Day, BioCryst plans to describe its drug discovery process and introduce additional therapies from its pipeline.

The live webcast and replay of the R&D Day will be available online in the investors section of the BioCryst website at .