Joshua Fleishman

Joshua Fleishman asked if patient compliance was also the primary factor for enabling at-home self-dosing for KB803 (ocular DEB), and what differentiated the three-times-a-week dosing schedule for KB803 from the once-daily schedule for KB801. He also inquired if the shift in Italian launch guidance from mid-2026 to the second half was due to rescheduling or pushback on VYJUVEK's efficacy.

Answer

Suma Krishnan, President of Research and Development, Krystal Biotech, confirmed that home administration was a key deciding factor for KB803, influenced by lessons learned from VYJUVEK and patient feedback requesting flexibility for multiple weekly administrations. She stated that the three-times-a-week regimen for KB803 provides this flexibility, with confidence from prior data that it will not impact efficacy. Krish Krishnan, Chairman and CEO, Krystal Biotech, clarified that the Italian launch timeline is not considered a 'delay,' but rather a natural variation in predicting specific months within a half-year, with no change to the overall timeline.

Joshua Fleishman, on behalf of Ritu Baral, asked if patient compliance was the primary factor for enabling at-home self-dosing for KB803 (ocular DEB), and what differentiated the three-times-a-week dosing for KB803 from the once-daily schedule for KB801. He also questioned if the shift in Italy's launch guidance from mid-2026 to the second half indicated a delay or pushback on VYJUVEK efficacy.

Answer

Suma Krishnan, President of Research and Development, confirmed that home administration was a key factor, informed by VYJUVEK patient feedback for flexibility. The three-times-a-week regimen for KB803 allows patients to administer a volume multiple times weekly, optimizing convenience without impacting efficacy based on prior data. Krish Krishnan, Chairman and CEO, clarified that the Italian launch timeframe is not considered a 'delay,' as predicting exact months is difficult, and the guidance remains consistent with past expectations.

Joshua Fleishman of TD Cowen asked how patient drug holidays might affect the goal of 720 U.S. patients initiating VYJUVEC and questioned the pricing strategy for the European market.

Answer

Chairman & CEO Krish Krishnan stated they are on track for the 720-patient number but are a quarter or two behind schedule, now expecting it early next year. SVP & GM for Europe Laurent Goux explained that Germany allows free pricing for the first 12 months, and HTA discussions are proceeding as planned in other key countries.

Joshua Fleishman from Cowen and Company, LLC asked for Sangamo's perspective on how their Nav1.7-targeted therapy, ST-503, will compare to recent small molecule Nav1.8 inhibitors and whether recent trial outcomes for Nav1.8 have affected their conviction in Nav1.7 as a target.

Answer

CEO Sandy Macrae responded that the company is now even more convinced that Nav1.7 is the correct target. He cited evidence from spontaneous human mutations where Nav1.7 mutations lead to a complete lack of pain, a stronger signal than that seen with Nav1.8 mutations. Macrae expressed confidence in their approach and looks forward to generating data from the dose-ranging study.

Joshua Fleishman of TD Cowen posed a multi-part question covering the timeline for the new U.S. manufacturing process, pipeline expansion priorities, Pombility/OpFolda launch expectations as more patients on Nexviazyme reach the two-year reevaluation mark, and the current reimbursement status.

Answer

Chief Business Officer Sébastien Martel noted progress on the U.S. drug product facility with Sharp but did not provide specific timelines. President and CEO Bradley Campbell added that reimbursement is proceeding well, with preferred status in key markets like the Netherlands. He confirmed patient switches from Nexviazyme are occurring relative to market share and that future pipeline expansion will focus on late-stage, de-risked assets similar to DMX200.