Cassie Chen

Cassie asked for Beam Therapeutics' read on GSK returning the rights for A1AT, and for the BEAM-302 pivotal trial, inquired about the FDA's minimum requirements for representative U.S. enrollments, given the ex-U.S. nature of the Phase I/II study.

Answer

CEO John Evans declined to comment on GSK's situation, reiterating Beam's belief in a one-and-done A1AT therapy and BEAM-302's best-in-class data. Regarding U.S. enrollment, he stated it's premature to discuss specific ratios but confirmed an open IND, active U.S. presence, and commitment to meeting all U.S. approval requirements.

Cassie asked for Beam's perspective on GSK returning the rights for A1AT, contrasting RNA editing with Beam's DNA editing approach. She also inquired about the FDA's minimum requirements for representative U.S. enrollment in the BEAM-302 pivotal trial, given the current Phase I/II is ex-U.S.

Answer

CEO John Evans declined to comment on GSK's situation but reiterated Beam's belief that a one-and-done DNA editing therapy for A1AT, like BEAM-302, offers a preferable product profile and has shown best-in-class data for A1AT levels. Regarding U.S. enrollment, he stated it's premature to discuss specific ratios but confirmed active U.S. participation in the trial, ensuring all requirements for U.S. approval will be met.

Cassie inquired about the potential pricing strategy for ARO-INHBE and ARO-ALK7, considering the competitive landscape and recent pricing announcements from other companies like Lilly.

Answer

James Hamilton, Chief Medical Officer and Head of R&D, stated that it is too early to speculate on potential pricing for these drug candidates. He emphasized that the company is still in the process of understanding the biology and how these drugs could work in various patient populations.

Cassie (on behalf of Luca Issi) asked about the potential pricing strategy for ARO-INHBE and ARO-ALK7, considering the competitive landscape with drugs like Zepbound and Wegovy. She also inquired about the anticipated costs for these obesity candidates.

Answer

James Hamilton, Chief Medical Officer and Head of R&D, stated that it is too early to speculate on potential pricing for ARO-INHBE and ARO-ALK7, as the company is still evaluating the biology and how these candidates will work in various patient populations.

Cassie, on for Luca Issi, asked about the TTR cardiomyopathy market, questioning if Ionis was surprised by a competitor not lowering price after a label expansion and whether WAINUA would compete on price.

Answer

Chief Global Product Strategy Officer Kyle Jenne responded that he was not surprised by the competitor's pricing strategy, suggesting it was likely to balance their existing patient mix. He clarified that the pricing strategy for WAINUA is ultimately partner AstraZeneca's decision and will be based on market dynamics and the drug's value proposition.