BioCryst Pharmaceuticals Inc. is dedicated to developing innovative therapeutics for rare diseases, particularly hereditary angioedema (HAE). The company employs a novel, structure-guided approach that integrates traditional biology and medicinal chemistry with advanced technologies to create first-in-class or best-in-class oral small-molecule and protein therapeutics. Their pipeline includes several investigational drugs, such as ORLADEYO® (berotralstat), a plasma kallikrein inhibitor designed for the prophylaxis of HAE attacks in both adults and pediatric patients. Additionally, BioCryst is exploring other therapeutic candidates targeting various conditions, including Netherton syndrome and diabetic macular edema, through a combination of oral and protein therapeutic strategies.

The company emphasizes a patient-centric approach, focusing on understanding the needs of patients and their families to ensure that the right treatments are accessible to those who need them most. BioCryst's commitment to transparency and collaboration with advocacy organizations underpins their mission to restore a sense of freedom for patients living with rare diseases. By blending cutting-edge technology with a deep understanding of patient needs, BioCryst aims to not only advance the science of drug development but also to make a meaningful impact on the lives of patients worldwide.

United States

BioCryst Pharmaceuticals is a global biotechnology company with a deep commitment to improving the lives of people living with hereditary angioedema (HAE) and other rare diseases. We leverage our expertise in structure-guided drug design to develop first-in-class or best-in-class oral small-molecule and protein therapeutics to target difficult-to-treat diseases.

We are a commercial-stage biotech company with US headquarters in Durham, North Carolina. Our European headquarters is in Dublin, Ireland, and our Discovery Center of Excellence is rooted in Birmingham, Alabama, with many other regional offices around the world. As world leaders in structure-guided drug design, we draw on the strengths and expertise of all our colleagues—from those of us in the field or behind the scenes in our labs, to others based in home offices and around the globe.

To view our LinkedIn community guidelines, please visit bit.ly/BCRXGuidelines.

Cardiovascular Diseases

Eye Diseases

Genetic Diseases

Infectious Diseases

Metabolic Diseases

Rare diseases

BioCryst Pharmaceuticals Inc.

Hereditary angioedema

Rare genetic disease

Berotralstat is a plasma kallikrein inhibitor that binds to plasma kallikrein and inhibits its proteolytic activity. Plasma kallikrein is a protease that cleaves high-molecular-weight-kininogen (HMWK) to generate cleaved HMWK (cHMWK) and bradykinin, a potent vasodilator that increases vascular permeability resulting in swelling and pain associated with HAE. In patients with HAE due to C1- inhibitor (C1-INH) deficiency or dysfunction, normal regulation of plasma kallikrein activity is not present, which leads to uncontrolled increases in plasma kallikrein activity and results in angioedema attacks. Berotralstat decreases plasma kallikrein activity to control excess bradykinin generation in patients with HAE.
<a href="https://www.biocryst.com/wp-content/uploads/2020/12/ORLADEYO_PI_V1_2020.pdf">Source</a>

Orladeyo (Berotralstat)

Acute lymphoblastic leukemia

Influenza

Leukemia

Peramivir is an inhibitor of influenza virus neuraminidase, an enzyme that releases viral particles from the plasma membrane of infected cells. The median neuraminidase inhibitory activity (IC50 values) of peramivir in biochemical assays against influenza A/H1N1 virus, influenza A/H3N2 virus, and influenza B virus clinical isolates were 0.16 nM (n=44; range 0.01- 1.77 nM), 0.13 nM (n=32; range 0.05-11 nM), and 0.99 nM (n=39; range 0.04-54.2 nM), respectively, in a neuraminidase assay with a fluorescently labeled MUNANA substrate (<a href="https://www.accessdata.fda.gov/drugsatfda_docs/label/2014/206426lbl.pdf">Learn more HERE</a>).&nbsp;

Rapivab (Peramivir) 

Netherton syndrome

BCX17725 is a protein therapeutic that is designed to treat the underlying protein deficiency that causes Netherton syndrome by inhibiting KLK5, a serine protease in the skin that is unregulated in people with Netherton syndrome.

BCX17725

COVID-19

Galidesivir is an <a href="https://pubchem.ncbi.nlm.nih.gov/compound/adenosine" rel="follow">adenosine</a> analogue that has been investigated for use against Zaire Ebolavirus. In animal studies, galidesivir was effective in increasing the survival rates from infections caused by various pathogens, including Ebola, Marburg, Yellow Fever and Zika viruses. In vitro, it displayed broad-spectrum antiviral activity against various negative- and positive-sense RNA viruses, including coronaviruses, filoviruses, and arenaviruses. Phase 1 clinical trials have begun to determine the safety of this drug in humans. Because of its activity against other coronaviruses, it may be studied as a potential therapy for COVID-19. (<a href="https://pubchem.ncbi.nlm.nih.gov/compound/Galidesivir">Learn more - National Library of Medicine</a>)

Galidesivir (BCX4430)

Paroxysmal nocturnal hemoglobinuria

The complement system comprises biological cascades of amplifying enzyme &nbsp;cleavages involving more than 30 proteins and protein fragments and may &nbsp;be activated through three pathways: the classical pathway (initiated &nbsp;by antibody-antigen complexes), the lectin pathway (initiated by lectin &nbsp;binding) and the alternative pathway (initiated by microbial surfaces). &nbsp;The alternative pathway also provides a critical amplification loop for &nbsp;all three pathways, regardless of the initiating mechanism. Factor D is &nbsp;an essential enzyme in the alternative pathway, thus making Factor D an &nbsp;attractive target to address complement-mediated diseases.
<a href="https://www.biocryst.com/our-pipeline/complement-program/">Source&nbsp;</a>

BCX9930 

Fibrodysplasia ossificans progressiva

Discovered by BioCryst, BCX9250 is a novel, oral, inhibitor of the ALK-2 &nbsp;enzyme. The ALK-2 enzyme is a part of the normal signaling pathway for &nbsp;bone formation and responds to binding its specific ligands (bone &nbsp;morphogenic proteins, BMPs) by stimulating normal bone growth and &nbsp;renewal in healthy children and adults. Specific activating mutations of &nbsp;the ALK-2 gene are seen in all cases of FOP. An activating mutation in &nbsp;ALK-2 is necessary for the disease to occur, making the ALK-2 enzyme an &nbsp;ideal drug target for treatment of FOP.
<a href="https://ir.biocryst.com/news-releases/news-release-details/biocryst-announces-positive-phase-1-results-bcx9250-oral-alk-2">Source&nbsp;</a>

BioCryst Pharmaceuticals Inc.

Company Details