Josh Schimmer

Josh Schimmer asked if BridgeBio has discussed with payers what might happen to Attruby's formulary positioning when Tafamidis generics enter, and if they've discussed combination therapy with payers given its cost and lack of data.

Answer

Neil Kumar, CEO and Founder, BridgeBio Pharma, stated that BridgeBio has not had discussions with payers regarding Tafamidis generics or combination therapy. He believes payers will eventually exert more control over the category but not within the next 6 to 12 months. Matt Outten, COO, BridgeBio Pharma, emphasized their focus on ensuring Attruby's availability to patients and competing on data in the doctor's office rather than in the payer space.

Josh Schimmer asked if BridgeBio has had discussions with payers regarding Atrobi's formulary positioning when Tafamidis generics enter the market, and if they've discussed combination therapy with payers given its cost and lack of supporting data.

Answer

Neil Kumar, CEO and Founder, BridgeBio Pharma, stated that BridgeBio has not had discussions with payers regarding Tafamidis generics or combination therapy. He believes payers will eventually exert more control over the category but not within the next 6 to 12 months. Matt Outten, Chief Commercial Officer, BridgeBio Pharma, emphasized focusing on ensuring Atrobi's availability and leveraging its data in discussions with physicians.

Josh Schimmer of Cantor Fitzgerald asked for an update on the experience with LFT elevations seen with adamelin, given prior scrutiny, and inquired about the plan for future updates on the drug's liver safety profile.

Answer

Founder and CEO R. Scott Struthers stated the company is very comfortable with the emerging and growing safety experience with adamelin. He indicated that updates will be provided as the data matures and becomes meaningful, suggesting that as the program expands into Phase III and open-label extensions, the growing experience will provide more comfort and that "no news is good news."

Josh Schimmer of Cantor Fitzgerald asked if any patients eligible for the EXPAND study are already receiving LIVMARLI through compassionate use or other programs.

Answer

Chief Medical Officer Joanne Quan explained that while the EXPAND study was initiated due to numerous compassionate use requests, the trial itself is enrolling treatment-naive patients to properly assess response. This ensures the integrity of the study data.

Josh Schimmer from Cantor Fitzgerald sought to clarify if the guided Q3 revenue decline was U.S.-only and asked about the heavy weighting of R&D expenses towards the oncology program.

Answer

Chairman & CEO Krish Krishnan confirmed the Q3 revenue decline commentary was specific to the U.S. market. President of R&D Suma Krishnan explained the high oncology R&D cost is due to the expensive combination trial with Keytruda and preparations for a potential future controlled study.

Josh Schimmer from Evercore ISI asked what specific data to expect from the DUBLIN-3 trial presentation at ESMO, particularly regarding subset analyses for Western and PD-1/PD-L1 exposed patients. He also questioned if the trial's ex-U.S. patient majority would be sufficient for FDA approval and how that uncertainty impacts potential partnership discussions.

Answer

Co-Founder, Chairman and CEO Lan Huang confirmed that the ESMO presentation would include full ITT population data and the eagerly awaited subset analyses. Dr. Huang addressed the FDA approval concern by noting that 27% of FDA oncology approvals have used less than 10% U.S. data, and the topic would be discussed with the FDA in a planned pre-NDA meeting. She declined to provide details on ongoing partnership discussions.

Josh Schimmer asked what data from pediatric patients would inform the Phase 2 Danon trial design and if the IMO patient who passed away had unique features. He followed up by asking if younger pediatric patients would have baseline abnormalities amenable to improvement.

Answer

CEO Gaurav Shah said they need to see confirmation of benefit in pediatrics and will use natural history data to inform the Phase 2 design. CMO Jonathan Schwartz noted that while the IMO patient had no specific high-risk identifiers, chronic lung compromise is common in the condition. Shah clarified that pediatric patients enrolled in the trial will have existing signs of cardiac disease (Class 2 heart failure or higher).

Josh Schimmer of Evercore ISI inquired about the status of discussions with the FDA regarding a potential accelerated approval for Molgradex in aPAP based on the IMPALA data. He also asked about non-microbiological outcomes in the NTM study, such as weight loss, and the balance between efficacy and the risk of exacerbations in that indication.

Answer

Chief Medical Officer Badrul Chowdhury explained that the company made a strategic decision to conduct a second Phase 3 study (IMPALA 2) rather than seek accelerated approval, citing internal priorities and shortcomings in the initial IMPALA data. Chief Operating Officer Taneli Jouhikainen added that for the NTM study, there were no clear average improvements in secondary endpoints like weight loss, and it was difficult to assess exacerbation rates in the open-label study, though individual cases would be reviewed.