[2023.][NASDAQ:BCRX]BioCryst Begins Enrollment in Proof-of-Concept Trial to Confirm Safe, Effective, Once-daily Dose of Oral Factor D Inhibitor, BCX10013

 [2023.][NASDAQ:BCRX]BioCryst Begins Enrollment in Proof-of-Concept Trial to Confirm Safe, Effective, Once-daily Dose of Oral Factor D Inhibitor, BCX10013

In a groundbreaking development, BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) has initiated a proof-of-concept clinical trial for BCX10013, a potential game-changing oral Factor D inhibitor designed to treat complement-mediated diseases. The trial aims to evaluate the preliminary efficacy and safety profile of once-daily dosing with BCX10013.

Dr. Ryan Arnold, BioCryst's Chief Medical Officer, expressed the company's commitment to developing first-in-class or best-in-class medicines. BCX10013, with its potential for once-daily dosing and remarkable safety and efficacy, could revolutionize the treatment landscape for patients suffering from complement-mediated diseases like paroxysmal nocturnal hemoglobinuria (P**).

The trial, which is open-label and multicenter, will include up to 15 adult P** patients in countries where standard-of-care complement inhibitor therapy is unavailable. It will involve intra-subject dose escalation at four-week intervals, with treatment options lasting up to 24 weeks. The primary endpoints are focused on safety and include monitoring for treatment-emergent adverse events and laboratory abnormalities. Key secondary endpoints will assess efficacy through various biomarkers, such as lactate dehydrogenase (LDH) levels and hemoglobin levels.

BCX10013 is just one of BioCryst's initiatives in the field of complement system targeting. The company is actively developing medicines targeting different pathways within the complement system to address a range of complement-mediated diseases.

For more information about this groundbreaking trial, please visit ClinicalTrials.gov and search for NCT number NCT06100900.

About BioCryst Pharmaceuticals: BioCryst Pharmaceuticals specializes in discovering novel, oral, small-molecule medicines to treat rare diseases where significant unmet medical needs exist. Their flagship product, ORLADEYO® (berotralstat), is approved in the United States and several global markets. BioCryst continues to advance its complement program with BCX10013, an oral Factor D inhibitor in clinical development. Additionally, they have RAPIVAB® (peramivir injection) approved in the U.S. and multiple global markets.

Forward-Looking Statements: This press release includes forward-looking statements regarding BioCryst's complement program and future results. These statements are subject to various risks and uncertainties, including those related to the ongoing COVID-19 pandemic, delays in clinical development, regulatory challenges, and market acceptance of their products. For more information on these factors, please refer to BioCryst's recent filings with the Securities and Exchange Commission.

For further inquiries, please contact: John Bluth +1 919 859 7910 jbluth@biocryst.com