BioCryst, BCRX, Company Profile, Stock Summary
BioCryst is a commercial-stage biotech company that is committed to delivering extraordinary medicines that help patients live ordinary lives. BioCryst is passionate about advancing novel therapeutics for patients with rare and serious diseases.
BioCryst’s US headquarters is located in Durham, North Carolina, our European headquarters in Dublin, Ireland and our Discovery Center of Excellence in Birmingham, Alabama. With expertise in drug discovery, clinical development, and regulatory affairs, we are advancing clinical programs and generating new compounds from own discovery engine.
BioCryst Pharmaceuticals discovers novel, oral, small-molecule medicines that treat rare diseases in which significant unmet medical needs exist and an enzyme plays a key role in the biological pathway of the disease. Oral, once-daily ORLADEYO® (berotralstat) is approved in the United States, the European Union, Japan, the United Kingdom and the United Arab Emirates.
Insiders Buying vs Selling Ratio 2:1
We have noticed a bullish behavior from insiders, a number of insiders bought the stock in the past 12 months, the ratio was about 2 to 1.
European Medicines Agency grants access to PRIME
European Medicines Agency (EMA) has granted access to the Priority Medicines (PRIME) scheme for BCX9250, a novel, oral activin receptor-like kinase-2 (ALK-2) inhibitor discovered and developed by BioCryst for the treatment of fibrodysplasia ossificans progressiva (FOP).
PRIME is a program launched by the EMA to enhance support for the development of medicines that target an unmet medical need. This voluntary program is based on enhanced interaction and early dialogue with developers of promising medicines and is designed to optimize development plans and speed up evaluation so these medicines can potentially reach patients earlier. According to the EMA, developers of medicines that are eligible for PRIME can expect additional opportunities for scientific advice and be eligible for accelerated assessment at the time of application for a marketing authorization.
“Promising results from non-clinical data and the first-in-human Phase 1 safety, tolerability and pharmacokinetics study in healthy subjects formed the basis of the application for PRIME eligibility. We are pleased with the EMA’s decision to grant PRIME eligibility to BCX9250 – the first investigational drug for this indication to receive this designation – based on the early evidence of the potential of BCX9250 to address the unmet need for patients living with FOP. We look forward to applying the benefits available to us through PRIME as we continue to advance our ALK-2 inhibitor program,” said Dr. Helen Thackray, chief research and development officer of BioCryst.
FOP is an ultra-rare, severely disabling genetic disorder characterized by the irregular formation of bone outside the normal skeleton, also known as heterotopic ossification (HO). HO can occur in muscles, tendons, ligaments and other connective tissues. Patients with FOP become bound by this irregular ossification over time, with restricted movement and fused joints, resulting in deformities, restricted mobility and premature mortality.
BCX9250 is designed to inhibit the ALK-2 enzyme, which is a part of the normal signaling pathway for bone formation and responds to binding its specific ligands (bone morphogenic proteins, BMPs) by stimulating normal bone growth and renewal in healthy children and adults. Specific activating mutations of the ALK-2 gene are seen in all cases of FOP. An activating mutation in ALK-2 is necessary for the disease to occur, making the ALK-2 enzyme an ideal drug target for treatment of FOP.
The Company has several ongoing trails at various stages
BioCryst has several ongoing development programs including BCX9930,
an oral Factor D inhibitor for the treatment of complement-mediated diseases, BCX9250, an ALK-2 inhibitor for the treatment of fibrodysplasia
ossificans progressiva, and galidesivir, a potential treatment for Marburg virus disease and Yellow Fever.
RAPIVAB® (peramivir injection) has received regulatory approval in the U.S., Canada, Australia, Japan, Taiwan and Korea.
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