Rick Bienkowski

Rick Bienkowski asked for more detail on the value proposition for NTLA-2002 in HAE and the expected pricing flexibility, considering the competitive landscape and potential cost offsets for payers.

Answer

CEO John Leonard emphasized that NTLA-2002's unique ability to get the vast majority of patients to a state of zero attacks without any other therapy is a key differentiator. He noted the significant cost of current chronic HAE therapies (often over $250k annually) provides a substantial window for pricing that offers value to patients, payers, and shareholders without setting new records for high-priced drugs.

Rick Bienkowski asked about the anticipated size of the sales force for NTLA-2002, other necessary commercial preparations, and sought confirmation that the majority of this spending would occur in 2027.

Answer

CFO Edward Dulac stated that the HAE market is well-defined, requiring a 'relatively tiny and neat' commercial footprint, and declined to give a specific sales force size. He confirmed that senior commercial leadership hiring began in 2024 and will continue in 2025, with the broader marketing and sales force to be hired in 2026 ahead of the 2027 launch.

Rick Bienkowski inquired about the enrollment pace for the MAGNITUDE-1 study, noting the primary completion date listed on clinicaltrials.gov, and asked for clarification on the underlying assumptions.

Answer

CEO John Leonard responded that the HAELO study serves as a good proxy. He confirmed the MAGNITUDE-1 study has 765 patients with global sites being activated and that enrollment is currently tracking ahead of internal projections. He indicated a potential for future updates on the timeline.

Rick Bienkowski asked about the potential impact of U.S. tariff policies on Canada, Mexico, and China on CARVYKTI's manufacturing costs or supply chain.

Answer

Executive Jessie Yeung stated that under the company's current assessment, there is immaterial exposure to tariffs in those countries. She noted they will continue to monitor the situation and reminded that the product comes from the U.S. and Europe, with plans to supply Europe from the Belgium facilities.

Rick Bienkowski asked for commentary on the FDA potentially reconsidering CAR-T black box warnings and which pipeline programs might see clinical updates in 2025.

Answer

CMO Mythili Koneru stated they have not had specific discussions with the FDA on the black box warning. She identified the Phase 1 trials for DLL3 (with Novartis) and Claudin 18.2 as being well-enrolled and likely to have data updates next year. CEO Ying Huang added that literature suggests no increased rate of secondary malignancies in CAR-T patients versus standard of care.

Rick Bienkowski from Cantor Fitzgerald requested more detail on the expected pace of patient dosing in the BEACON trial, given that 35 patients were enrolled but only 8 had been dosed to date.

Answer

CEO John Evans stated that enrollment has exceeded expectations and that the pace of dosing is set to increase significantly. He explained that the lengthy manufacturing process was a gating factor, but doses are now 'rolling off the line fairly regularly,' which will lead to a 'big uptick' in dosing soon. He also noted that with 35 patients enrolled, the clock has started on the follow-up period for a potential registrational filing.

Rick Bienkowski of Cantor Fitzgerald requested more detail on the expected pace of dosing in the BEACON trial and the potential timeline for dosing the 27 patients who have been enrolled but not yet treated.

Answer

CEO John Evans expressed satisfaction with the enrollment pace, which has exceeded expectations with 35 patients enrolled. He noted that with manufactured doses now becoming available regularly, there will be a significant uptick in dosing in the near future, putting them well on their way toward the trial's goal of 45 dosed patients for a potential registration package.

Rick Bienkowski from Cantor Fitzgerald requested more detail on the expected pace of dosing in the BEACON trial and the timeline to dose all enrolled patients, noting 35 were enrolled but only 8 dosed.

Answer

CEO John Evans explained that enrollment has exceeded expectations and that manufacturing the cell therapy product is the rate-limiting step. He stated that doses are now 'rolling off the line fairly regularly' and a 'big uptick in doses' is expected soon. He also highlighted that the trial is well on its way to the 45 total dosed patients planned for the potentially registrational study.