Dina Ramadane

Dina Ramadane asked about the 'bull case' for OPUS-1 data, specifically if it involves statistical superiority on certain high-priority strains, and inquired about the development status and potential timeline for VAX-XL to enter the clinic.

Answer

Grant Pickering, CEO of Vaxcyte, emphasized that coverage is paramount in the PCV class, and VAX-31's advantage lies in expanded coverage combined with improved immune responses. He noted that VAX-XL, a third-generation broader spectrum vaccine, is part of a lifecycle management strategy, with clinical advancement planned for an appropriate time when serotype replacement warrants it.

Dina Ramadane asked about the 'bull case' for OPUS-1 data beyond regulatory approval, focusing on statistical superiority for high-priority strains, and the development status of the VAX-XL program.

Answer

Grant Pickering, CEO of Vaxcyte, emphasized that 'coverage is king' in the PCV class, citing Capvaxive's market share gains. He highlighted VAX-31's potential for both broad coverage and improved immune responses, noting 18 of 20 common serotypes were directionally higher and 7 statistically significantly higher than Prevnar 20 in Phase 2. For VAX-XL, Pickering described it as a third-generation life cycle management strategy, currently focused on preparedness for future serotype replacement rather than immediate clinical entry.

Dina Ramadene asked about how future deal structures in the China market might differ from historical deals and any added diligence hurdles, and for Royalty Pharma's view on the importance of a 15%-20% treatment effect size for the Lp(a) lowering class readouts to the commercial peak sales opportunity.

Answer

Pablo Legorreta, CEO and Chairman of the Board, stated that deal structures and diligence processes for China deals are not expected to change, as royalties would be paid by U.S. or European marketers, and emphasized the need for local presence. Marshall Urist, EVP, Head of Research and Investments, affirmed that the effect size in the Novartis Lp(a) trial does matter, and discussions will focus on the range, patient benefits, and subgroups to determine commercial impact.

Dina Ramadene asked how future deal structures in the China market might differ from historical deals and if the diligence process presents any added hurdles impacting efficiency. She also inquired about the importance of a treatment effect size (above or below 15%-20% risk reduction) in the upcoming Lp(a) lowering class readouts for the commercial peak sales opportunity.

Answer

Pablo Legorreta, CEO and Chairman of the Board, stated that they do not foresee changes in deal structure or diligence processes for China deals, as the payer of the royalty would typically be a U.S. or European company. He emphasized the need for local presence to be effective in the market. Marshall Urist, EVP and Head of Research and Investments, confirmed that the effect size in the Novartis Lp(a) trial does matter, and there will be significant discussion around the specific risk reduction and patient subgroups that benefit most, highlighting the incredible potential of their development-stage portfolio.

Dina Ramadane, on for Jason Gerberry, asked where seizure freedom fits within the statistical hierarchy for X-TOLE2 and about assumptions for this endpoint. She also inquired about the lead indication selection for XEN1120 and the desired pharmacology profile from its Phase I trial.

Answer

President and CEO Ian Mortimer stated that for XEN1120, the Phase I trial will assess PK and safety, with the goal of advancing into a proof-of-concept study in pain. Chief Medical Officer Dr. Chris Kenney clarified that seizure freedom is not in the statistical hierarchy for the double-blind portion of the study, as it's an endpoint best measured over a longer term, making the open-label extension data the primary focus for that outcome.

Dina Ramadan from Bank of America asked about the broader market opportunity for FILSPARI in FSGS, including whether it requires market building and the potential challenges for a new therapy in a space with no approved treatments.

Answer

Chief Commercial Officer Peter Heerma estimated the addressable U.S. market at 15,000 to 30,000 patients, noting it's the same prescriber base as IgAN, which should lead to rapid uptake. Chief Medical Officer Dr. Jula Inrig added that there is a high unmet need for effective, non-immunosuppressive therapies. President and CEO Dr. Eric Dube concluded that they expect to become the leader and new standard of care.

Asked about the drivers for the expected acceleration in LYFGENIA patient starts in the second half of the year and the competitive dynamics influencing prescriber choice between sickle cell gene therapies.

Answer

The LYFGENIA acceleration was always anticipated for the second half of the year due to the longer patient journey involving payer approvals and clinical readiness; it is a timing issue, not a demand issue. The company believes it holds a competitive lead due to its larger QTC network, higher U.S. patient start numbers, and favorable market research showing physician preference for LYFGENIA.