Woman in a lab coat and safety glasses operates a laboratory machine, placing a tray of samples into the device.

Pipeline

MAKING “EXTRA- ORDINARY” POSSIBLE STARTS HERE

A pipeline for hereditary angioedema (HAE) and other rare diseases

We are committed to discovering and developing oral small-molecule and protein therapeutics that not only have the potential to move the needle, but move mountains, for patients—restoring a sense of freedom for them and their loved ones.

PhaseAsset	Program	Lead Optimization	Pre-Clinical	Proof of Concept^†	Pivotal^‡	Approved / Commercial
ORLADEYO® (berotralstat) Oral Plasma Kallikrein Inhibitor ORLADEYO is a plasma kallikrein inhibitor indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older.	Hereditary Angioedema (HAE)
ORLADEYO® (berotralstat) Oral Plasma Kallikrein Inhibitor in Pediatrics ORLADEYO is a plasma kallikrein inhibitor being investigated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in pediatric patients 2 to <12 years of age.	Hereditary Angioedema (HAE)
BCX17725 Protein Therapeutic BCX17725 is a KLK5 inhibitor protein therapeutic for the potential treatment of Netherton syndrome.	Netherton Syndrome
Avoralstat Ocular Plasma Kallikrein Inhibitor Avoralstat is a plasma kallikrein inhibitor for the potential treatment of diabetic macular edema (DME)	Diabetic Macular Edema (DME)
Oral C5 Inhibitor BioCryst’s discovery team is designing oral drug candidates that inhibit the C5 protein, which drives downstream terminal functions.	Complement-Mediated Diseases
Oral C2 Inhibitor BioCryst’s discovery team is designing oral drug candidates that inhibit the classical and lectin pathways of the complement system including downstream terminal functions.	Complement-Mediated Diseases
Bifunctional Complement Inhibitor BioCryst’s discovery team is designing protein therapeutic candidates that can simultaneously address multiple pathways in the complement system.	Complement-Mediated Diseases

*ORLADEYO for pediatric patients, BCX17725, and avoralstat are investigational and have not been deemed safe and effective by the FDA.

^†Proof of Concept is typically Phase 1 or 2.

^‡Pivotal is typically Phase 3.

This page may contain forward-looking statements, including statements regarding future results, unaudited and forward-looking financial information and company performance or achievements. These statements are subject to known and unknown risks and uncertainties which may cause our actual results, performance or achievements to be materially different from any future results or performances expressed or implied on this webpage. You should not place undue reliance on the forward-looking statements. For additional information, including important risk factors, please refer to BioCryst’s documents filed with the SEC and located at https://ir.biocryst.com/financial-information/sec-filings.

Close-up of various labeled laboratory sample vials containing different liquids and substances, arranged on a white paper towel on a lab bench.

Medicines that meet the needs of patients

For someone living with a rare disease, the concept of being able to go about life without a constant reminder of their condition would represent a world of difference. Restoring a new sense of freedom for patients and their loved ones is our goal. This is why we blend cutting-edge technology and complex science to develop first-in-class or best-in-class oral small-molecule and protein therapeutics. Learn more >>