James Condulis

James Condulis from Stifel Nicolaus asked about the upcoming HFpEF data for ulicamten, what constitutes a successful outcome, and the importance of the nHCM trial results for confirming its mechanism.

Answer

Stuart Kupfer, SVP & Chief Medical Officer, outlined that a 'win' for the Phase 2 AMBER HFpEF trial would include symptomatic improvement (KCCQ, NYHA), better biomarkers, and improved diastolic function. He and Fady Malik, EVP of R&D, confirmed that positive data from nHCM studies inform the potential benefit in HFpEF due to the shared pathophysiology.

James Condulis of Stifel asked if the FDA's request for a REMS came after the mid-cycle review and requested more color on that meeting.

Answer

Robert I. Blum, President and CEO, confirmed the timing was around the mid-cycle review, as indicated in a prior 8-K filing. He stated that the company has provided all the detail it can on the matter and will maintain consistency with its previous disclosures.

James Condulis of Stifel asked about the potential for the REMS program for mavacamten (Camzyos) to be eased and how that might impact the competitive differentiation for aficamten's own potential REMS.

Answer

President & CEO Robert I. Blum acknowledged BMS's application to ease the Camzyos REMS and stated that market research indicates several meaningful points of differentiation for aficamten beyond echo frequency. EVP & CCO Andrew Callos added that if the entire CMI category sees relaxed monitoring, it would be a positive for market growth, as it could encourage more physicians to treat patients. Blum reiterated the goal is to expand CMI use and expressed confidence in aficamten's differentiated profile.

James Condulis of Stifel Nicolaus asked if the initial patient starts were driven by normal physician visit cadences or by proactive, off-cycle patient requests, and also inquired about the primary rate-limiting factor for uptake going forward.

Answer

CCO Meredith Manning responded that they are seeing both proactive patients scheduling early and patients coming in during normal cycles. She noted that many clinics are now establishing dedicated PWS clinic days, which should help create a steadier patient flow and manage the high demand, which appears to be the main logistical factor.

James Condulis of Stifel Nicolaus asked whether the initial patient start forms were driven by routine physician visits or by proactive patients seeking treatment off-cycle. He also inquired about the primary rate-limiting factor for the launch going forward.

Answer

Chief Commercial Officer Meredith Manning explained that they are seeing both proactive patients and those coming in during normal cycles. She noted a positive trend of clinics establishing dedicated PWS clinic days, which should help create a steadier patient flow. She did not identify a specific rate-limiter, stating there is significant opportunity ahead.

James Condulis asked about the typical conversion rate from patient start forms to paid prescriptions in rare disease launches and whether the pace of new start forms is expected to accelerate.

Answer

CCO Meredith Manning responded that while it is too early to provide a specific percentage, they expect the conversion rate to be comparable to other strong rare disease launches. CFO James MacKaness added that the significant unmet need for a PWS treatment should support a conversion rate that is at least on par with industry benchmarks.

James Condulis asked about the typical conversion rate from patient start forms to paid prescriptions in rare disease launches and whether the pace of new start forms is expected to accelerate.

Answer

CCO Meredith Manning responded that it is too early to provide a specific conversion percentage but that the company expects results comparable to other strong rare disease launches. Executive James MacKaness added that the significant unmet need in PWS should support a conversion rate that is at least comparable to peers.