BioCryst Starts Phase 1 Trial of BCX17725 for Netherton Syndrome

BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) has initiated a Phase 1 clinical trial for BCX17725, a potent and selective investigational inhibitor of kallikrein 5 (KLK5), aimed at treating Netherton syndrome. The first participant has been enrolled in this trial, marking a significant step for the company, which is eager to bring a novel protein therapeutic solution to patients suffering from this rare genetic disorder.

Netherton syndrome is a severe, lifelong genetic condition that affects the skin, hair, and immune system. It is caused by the dysfunction of a natural inhibitor of KLK5, leading to symptoms such as red, scaly, and inflamed skin, fragile hair, and a higher susceptibility to skin infections, allergies, asthma, and eczema. This condition can be life-threatening, particularly in infancy due to the risk of dehydration and recurrent infections. Currently, there are no approved treatments for Netherton syndrome, leaving a significant gap in effective therapies for patients.

Dr. Helen Thackray, chief research and development officer at BioCryst, highlighted the difficulties faced by those living with Netherton syndrome. She pointed out that the only current treatment options are topical treatments and other supportive medications, which do not address the disease's underlying causes. According to Dr. Thackray, BCX17725 has shown promising data in nonclinical studies, suggesting that it could offer the potency, specificity, and convenient dosing required to potentially become a best-in-class therapy for this ultra-rare disease.

The Phase 1 clinical trial will assess the safety, tolerability, pharmacokinetics, and immunogenicity of BCX17725. This will be done through single and multiple doses administered to healthy adult participants (Parts 1 and 2) and multiple doses given to participants with Netherton syndrome in an open-label design (Part 3). BioCryst aims to release initial data from this trial by the end of 2025.

BCX17725 represents a pioneering approach to treating Netherton syndrome by targeting the underlying protein deficiency. It functions by inhibiting KLK5, a serine protease in the skin that is overly active in individuals with this condition. This targeted strategy seeks to provide a disease-modifying treatment, potentially transforming the therapeutic landscape for patients who currently have very limited options.

BioCryst Pharmaceuticals is a global biotechnology firm dedicated to improving the lives of people affected by complement-mediated and other rare diseases. The company's expertise lies in structure-guided drug design, enabling the development of both first-in-class and best-in-class oral small-molecule and protein therapeutics aimed at challenging diseases. BioCryst has successfully commercialized ORLADEYO® (berotralstat), the first oral, once-daily plasma kallikrein inhibitor, and continues to advance a diverse pipeline of promising therapies.

For detailed information about the trial, interested parties can search the NCT number NCT06539507 on ClinicalTrials.gov.

BioCryst's ongoing commitment to rare disease treatment innovation is evident in its pursuit of effective therapies like BCX17725. The company's work holds promise for significantly enhancing the quality of life for those living with Netherton syndrome and potentially other rare conditions.