Ben Burnett

Ben Burnett asked about physician feedback and appetite for using Brensupri in more moderate patients or those with only one recent exacerbation, beyond the initial focus on severe patients.

Answer

Will Lewis, Chairman and CEO, noted that it's still early, but physicians are currently focused on patients matching the ASPEN study criteria (two or more exacerbations), which represents a moderate to severe profile. He acknowledged the broader opportunity in other populations, including COPD and asthma comorbid patients, but emphasized a 'one step at a time' approach as physicians gain experience.

Ben Burnett asked about physician feedback and appetite for using BRINSUPRI in more moderate patients or those with only one recent exacerbation, beyond the initial focus on severe patients.

Answer

Will Lewis, Chairman and CEO of Insmed, stated that it's still early, but physicians' initial focus aligns with the ASPEN study criteria (two or more exacerbations, moderate-to-severe profile). He noted the broad label and potential for comorbid COPD/asthma patients, emphasizing that physician experience will shape broader use over time.

Ben Burnett asked about the phase three scenarios for Elixerextant, specifically if two phase three studies are needed and if an NT2 study combined with an NT1 phase three would suffice for approval in both indications.

Answer

Richard Pops, CEO, confirmed that the current assumption is two phase three studies (standalone for NT1 and a similar study for NT2) to seek labeling for narcolepsy, pending confirmation from the FDA.

Carolina Ibanez-Ventoso on for Ben Burnett asked for the specific timing of the MRD conversion measurement for the mid-2025 interim analysis of the cema-cel ALPHA3 trial.

Answer

EVP of R&D and CMO Dr. Zachary Roberts responded that the company has not disclosed the exact timing of the MRD sample draw. He also noted that the lymphodepletion decision will be based on an assessment of multiple elements, not just the MRD conversion data.

On behalf of Ben Burnett from Stifel, an analyst asked what the ex-vivo data from the SLE patient sample implies about the necessary FT819 dose and expansion required to achieve deep B-cell depletion in vivo.

Answer

Dr. Bob Valamehr, Chief Research and Development Officer, explained that the data showed effective B-cell elimination at a 2:1 effector-to-target ratio. Given an estimated disease burden of 100-300 million B-cells in an SLE patient, the current dose of 360 million cells is well-positioned to match the effective dose seen in vitro, providing confidence in its potential for in-vivo efficacy.

Ben Burnett inquired about the expected duration of SDMA and SAM level reduction required to observe a clinical response and asked if the company plans to continue dose escalation until an MTD is reached.

Answer

Chief Scientific Officer Michael White explained that the time to response could vary by indication but requires continuous, strong target suppression. CEO & President Yujiro Hata added that they believe they are in the efficacious range at Cohort 6 and could start expansion there while continuing to escalate in parallel to formally define the MTD.