Date: 02/13/2024

Who We Are At BioCryst

At BioCryst Pharmaceuticals, we work tirelessly, not just for, but with patient communities.
At the heart of who we are and what we do is a dedication to understanding and elevating patient community voices through meaningful engagement and collaborative partnerships.

BioCryst is dedicated to advancing novel therapeutics for patients with complement-mediated and rare diseases, including Netherton syndrome. We are committed to a people-focused approach as we develop and bring first-in-class or best-in-class therapeutics to patients around the globe. Our team of researchers developed the first and only oral, once-daily therapy designed specifically to prevent hereditary angioedema (HAE) attacks (a rare, potentially life-threatening genetic condition that causes spontaneous episodes of swelling) in adult and pediatric patients 12 years and older. The therapy is approved and available in the United States and many other countries around the world. We work together across all parts of our organization - from those of us in the field or behind the bench in our labs, to those in our offices and around the globe - to bring medicines to patients who are eagerly waiting for solutions to help restore a sense of freedom for them and their loved ones.

Our research and development team based at our Discovery Center of Excellence in Birmingham, Alabama, uses a unique structure-guided approach to discover and develop potential therapies, including five new programs announced during our Research & Development Day in November 2023. These include investigational therapies that could potentially treat Netherton syndrome, diabetic macular edema (DME), and multiple different complement-mediated diseases, in addition to expanding our therapy for HAE to pediatric patients under the age of 12. Our investigational therapy being studied for Netherton syndrome, called BCX17725, is currently in the pre-clinical phase and has shown rapid distribution to the epidermis (outer layer) of the skin when administered in nonclinical studies. This year, we plan to begin evaluating BCX17725 in healthy individuals. Our hope is to achieve ‘proof of concept’ in patients (early-stage clinical trials providing evidence of the expected response in patients) with Netherton syndrome in 2025 and share our findings from this study in 2026. Our goal is to create an injectable medication that can be taken every two weeks or less frequently in both pediatric and adult patients.

BioCryst’s Global Patient Advocacy and Clinical Development teams collaborate closely with patient communities we serve to incorporate patient and care partner voices into our drug development processes. We listen to and understand the needs of patients and their communities, incorporating their perspectives into every step of the drug development process. From every decision we make to every action we take. We partner with patient organizations at every opportunity to learn from them – including convening advisory boards and listening sessions, spreading awareness about these conditions, attending conferences, and so much more! Now, we are looking to the FIRST Foundation patient, care partner, and healthcare provider community to collaborate with to ensure that we are meeting the true needs of the Netherton syndrome patient community while achieving the extraordinary. ?

We look forward to meeting you in person at the 2024 FIRST National Conference in Albuquerque, NM. Please stop by our table to say "hello" during the Welcome Reception                                                                                                                                                                                on Thursday, June 27 from 6:30pm - 8:30pm.

Learn more about our commitment to patient communities!

Website: https://www.biocryst.com/patient-advocacy/

Email: PatientAdvocacy@biocryst.com