Mikaela Vanik

Mikaela Vanik asked for clarification on whether Netherton syndrome Part 3 and 4 data would be released simultaneously. She also inquired about the variability of disease severity at baseline, the difficulty of enrolling uniform patients, allowed background treatments, and patient progression based on phenotype.

Answer

President and CEO Charlie Gayer confirmed plans to release all Netherton syndrome data together, noting only 2-3 patients in Part 3 and prioritizing Part 4. Chief Development Officer Dr. Bill Sheridan discussed the spectrum of disease severity, the underdiagnosis of Netherton syndrome, and the high enthusiasm from investigators for patient recruitment, emphasizing selection of patients with obvious illness.

Mikaela Vanik asked for clarification on the data release timing for Netherton syndrome Parts 3 and 4, and discussed disease severity variability at baseline. She also inquired about the difficulty of enrolling uniform patients, allowed background treatments, and patient phenotype considerations.

Answer

President and CEO Charlie Gayer stated the plan is to release all Netherton syndrome data together, prioritizing Part 4 (3-month dosing) with only 2-3 patients in Part 3. Chief Development Officer Dr. Bill Sheridan explained that while there's a spectrum of severity, investigators are enthusiastic and have identified patients with obvious illness, making recruitment less of a concern. He confirmed patients with clear signs of illness are being selected to evaluate drug benefit.