Lili Nsongo

Lili Nsongo asked about the rationale for stopping at the 15mg dose for the CF program given no safety signals, and if there's flexibility to increase dosing. She also inquired about the expectation of a bifurcated regulatory path for pediatric and adult OTC deficiency patient populations.

Answer

Joseph Payne, President and CEO, reiterated that 10mg showed early efficacy, and the 12-week study extends that duration, with flexibility to increase to 15mg if needed, also considering cost of goods. He confirmed a likely bifurcated regulatory path for OTC due to differing severity. Alan H. Cohen, Chief Medical Officer, added that the FDA has shown willingness to offer more flexibility for ultra-rare pediatric populations.

Lili Nsongo, on behalf of Mani Faroohar at Leerink Partners, questioned the adoption and growth expectations for the new predictive toxicology feature and asked to quantify the potential for increased usage among existing customers.

Answer

CEO Ramy Farid highlighted that the recently launched beta version of the predictive toxicology feature is seeing significant excitement and demand, aligning with the FDA's push to reduce animal testing. CFO Richie Jain added that there is substantial room for growth within existing customers, with a key strategy being the migration of smaller and medium-tier accounts to larger contracts.