Troy Langford

Troy Langford from TD Cowen inquired about the expected level of disease progression on functional measures in the ATTR-CM patient population, to provide context for the upcoming Phase 1 data update.

Answer

EVP & CMO David Lebwohl explained that historically, placebo patients in similar trials show worsening trends on measures like proBNP and the six-minute walk test. In contrast, Intellia's Phase 1 data has shown stabilization or improvement following a deep TTR reduction. He noted the event rate in their high-risk Phase 1 cohort has been very low relative to historical data, and the next update will provide more extended follow-up.

Troy Langford asked for more color on physician feedback to the NTLA-2001 (Nex-z) Phase I data presented last November, wanting to know which data points resonated most with them.

Answer

Chief Medical Officer David Lebwohl highlighted that physicians are most impressed by the rapid and deep TTR reduction (90% reduction in one month), which contrasts sharply with the slower, less profound reduction seen with silencers. He added that the favorable data on clinical measures like proBNP, 6-minute walk, and time to first event are also key differentiators driving investigator enthusiasm and trial enrollment.

Troy Langford from TD Cowen asked if there is any reason to expect a substantially different level of benefit for Sunosi in the Phase III pediatric ADHD study compared to the adult trial. He also inquired about the anticipated duration of the pediatric study.

Answer

Herriot Tabuteau, CEO, noted that there has historically been a correlation between ADHD efficacy in adults and pediatrics, making the adult data encouraging. However, he emphasized the need to conduct the pediatric study and stated that it is premature to provide a timeline for its completion.

Troy Langford from TD Cowen asked about the potential market size for Ziihera (zanidatamab) in breast cancer, specifically seeking to quantify the number of patients who have progressed on or are intolerant to Enhertu.

Answer

Renée Galá, President and COO, contextualized the opportunity by stating the overall zanidatamab peak sales potential is over $2 billion across its targeted indications, with the breast cancer patient population exceeding 150,000 in key geographies. Robert Iannone, EVP and Global Head of R&D, added that Ziihera aims to be the treatment of choice after Enhertu, potentially capturing a high proportion of patients in the second- or third-line metastatic setting.

Troy Langford of TD Cowen asked for an estimate of the patient population for Ziihera in breast cancer, specifically those who have progressed on or are intolerant to Enhertu.

Answer

President & COO Renée Galá framed the overall zanidatamab opportunity as over $2 billion, noting the breast cancer market is significantly larger than GEA or BTC, with over 150,000 patients. EVP, Global Head of R&D Rob Iannone added that Ziihera could become the treatment of choice post-Enhertu, capturing a high proportion of these patients.

Troy Langford asked about the timeline for ALKS 2680, specifically how quickly Alkermes could advance to pivotal studies following positive Phase II data and if a Phase III trial could begin before the end of 2025.

Answer

CEO Richard Pops explained that much of the Phase II infrastructure was built with Phase III in mind. The plan is to get top-line data, schedule an end-of-Phase II meeting with the FDA, and potentially file for breakthrough designation, with the goal of starting Phase III as quickly as possible after the regulatory meeting.