New clinical trial looking at a potential new drug for treating Netherton syndrome

BioCryst Pharmaceuticals has provided the following update about their BCX17725 program. The Ichthyosis Support Group (ISG) will continue to provide updates as we learn more about this program. 

Update from BioCryst Pharmaceuticals About BCX17725 Program 

"A Phase 1 clinical trial looking at a potential new drug for treating Netherton syndrome has just begun in Australia. The drug is called BCX17725. It is a protein therapeutic (type of biologic drug) that is designed to function similarly to a protein found in skin that is either missing or defective in people with Netherton syndrome. The drug is injected into the body. BCX17725 was discovered and is being developed by researchers at BioCryst Pharmaceuticals, which is headquartered in the USA.

This clinical trial is the first time that BCX17725 has been given to human participants. The trial is designed to assess the safety, tolerability, pharmacokinetics (how the body interacts with a drug) and immunogenicity (the ability of a drug to cause the body to produce an immune response) of BCX17725.

The trial is being conducted in three parts. The first part will evaluate the drug when given in increasing single doses to adult participants who do not have Netherton syndrome. In the second part, the drug will be given in increasing multiple doses also to adult participants who do not have Netherton syndrome, which is typical in a Phase 1 trial. The third part will evaluate the drug when given in multiple doses to people with Netherton syndrome. The first two parts are conducted in a blinded fashion, meaning neither the study investigators nor the participants enrolled in the trial will know if they are receiving BCX17725 or placebo. In the third part, all participants will receive the active drug.

Prior to being studied in humans (clinical study), BCX17725 was evaluated in nonclinical studies (non-human studies) that showed the investigational therapy has the potential to achieve the potency (the intensity of effect produced for a given drug dose), specificity (the capacity of a drug to cause a particular effect) and convenient dosing needed to become an important therapy for people with Netherton syndrome.

Initial results from the trial are expected by the end of 2025. There is currently one active trial site located in Queensland, Australia. For more information about this trial, please visit ClinicalTrials.gov and search for the study NCT (NCT06539507)." BioCryst Pharmaceuticals

Further information

The Ichthyosis Support Group (ISG) hope this clinical trial will be expanded but has no further information at this time.

For information about active ichthyosis and related skin type clinical trials please visit ClinicalTrials.gov