Gospel Enyindah-Asonye

Gospel Enyindah-Asonye, on behalf of Vikram Purohit at Morgan Stanley, asked for guidance on how best to interpret the upcoming functional data for the RGX-202 Duchenne program.

Answer

Dr. Steve Pakola, Chief Medical Officer, explained that the company will assess the totality of the data, including standard timed function tests like the 10-meter walk/run, NSAA scores, and caregiver-reported outcomes. CEO Curran Simpson added that their analysis will be supported by comparisons to external controls and substantial natural history databases.

On behalf of Vikram Purohit, Gospel Enyindah-Asonye from Morgan Stanley asked about the ideal partnership structure and potential timing for Kymera's oncology assets, KT-253 and KT-333.

Answer

CEO Nello Mainolfi stated that an ideal partner would have a strong clinical and commercial franchise in hematology, where the assets have shown promising activity. This would maximize patient value while allowing Kymera to focus its internal resources on its immunology pipeline. He did not comment on specific timing or ongoing discussions.

Gospel Enyindah-Asonye of Morgan Stanley asked about the efficacy bar for VTX958 in Crohn's disease that would justify continued development, based on the data expected in the second half of the year.

Answer

Executive Martin Auster explained that the primary endpoint is the change in CDAI score. He noted that a meaningful response, competitive with approved biologics, would be in the upper double-digits to low hundreds. For the secondary endpoint of endoscopic response, a competitive result would be in the high teens to low 20s delta versus placebo.