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CEO Vishwas Seshadri and CTO Brian Kevany addressed the CMC question, stating they aligned with the FDA in a Type A meeting and are confident in the resubmission package, with identity and Stargardt testing being the main new data points for FDA review. CCO Madhav Vasanthavada explained the commercial strategy, noting that market research shows high patient and physician interest in pz-cel due to its durable wound healing profile, and payers have not raised concerns about its use relative to other available therapies.

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CEO Vishwas Seshadri stated that no additional work was requested by the FDA, only input on the statistical approach for validation protocols. Chief Commercial Officer Madhav Vasanthavada explained the 3-month post-approval period is necessary for site-specific activities like P&T committee reviews and charge master updates, which require the final product label. Vasanthavada also reported that payer discussions have been positive, with payers recognizing pz-cel's distinct profile for large wounds and not raising major objections to its use alongside other treatments.

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Executive Alexander Sapir and Chief Scientific Officer Iain Fraser clarified that the healthy volunteer studies were always part of the routine development plan to assess formulations and drug interactions, and their timing was coincidental to the Oxbryta withdrawal. Regarding African sites, an executive (likely retiring CMO Pat Horn) stated the FDA has a long history of accepting ex-U.S. data that meets GCP standards, expressing confidence in the selected sites, many of which have prior regulatory audit experience.

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Executive Iain Fraser clarified that the powering for REACH was based specifically on the FSHD type 1 patient population from the ReDUX4 study. He explained that the over-enrollment of type 1 patients (from 210 to 242) is what increased the study's power from 93% to 96%. He noted that FSHD type 2 patients were stratified but not included in this primary power calculation.

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President and COO Peter Radovich stated the guidance increase is driven by continued strong Alagille syndrome growth and the robust PFIC launch in the U.S. Chief Medical Officer Joanne M. Quan confirmed the EXPAND study is on track with its previously guided 18-month enrollment timeline. CEO Christopher Peetz noted that while other indications for PDE4D may exist, the current focus is on achieving proof-of-concept in Fragile X.

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CMO David Lebwohl stated the trial will not include patients with major liver disease, as they are best treated by also reducing the mutant protein, which NTLA-3001 does not do. CEO John Leonard added that Intellia is developing a separate gene writing program to address both the lung and liver aspects of the disease.

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President and CEO Bradley Campbell clarified that the strategy is to first leverage their self-sustaining infrastructure to add de-risked products for top-line growth, with increased pipeline investment to follow as cash flow strengthens. Chief Development Officer Dr. Jeff Castelli confirmed the internal programs use refined and improved versions of the constructs from the Caritas work, with a primary focus on Fabry disease.

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President and CEO Bradley Campbell and Chief Development Officer Dr. Jeff Castelli confirmed this is a key focus. Their strategy is to shift physician expectations from disease stability to the possibility of improvement. Dr. Castelli added that Amicus reminds physicians that their clinical data showed patients improved after switching from prior ERTs, suggesting that 'stable' may not be the best possible outcome and that regaining function is a potential benefit of Pombiliti and Opfolda.

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Chairman, CEO and President David Meeker said there are no current plans to share the RM-718 Phase I data but would consider it, and he was unsure about the extent of clinical trial site overlap. Chief Financial Officer Hunter Smith addressed the strategic question, stating it's too early to speculate on the trade-off but emphasized the company is dilution-sensitive while aiming to maximize long-term value.

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CEO Emil Kakkis stated setrusumab is manufactured at a 3,000-liter scale at an approved and licensed contract manufacturer in Germany that they already use for Mepsevii. CCO Erik Harris and Kakkis confirmed that despite the Crysvita royalty transition, they have retained key commercial leadership and a field team of 16, providing a strong foundation to scale up for a successful launch.

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CEO John Evans noted that some patient-to-patient variability is expected. He explained that for patients 3 and 4, their last blood transfusion was closer to the one-month measurement point, which can temporarily affect the measurement of endogenously produced hemoglobin. He advised that longer-term follow-up is needed to properly assess the trend.

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CEO John Evans noted that some patient variability is expected and that for patients 3 and 4, their last blood transfusion was closer to the month 1 time point, which can temporarily dilute the measurement of endogenously produced hemoglobin. He advised that longer follow-up is needed to fully assess the trajectory and stabilization of hemoglobin levels.

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Christine Wilson, SVP and Head of U.S. Sales and Marketing, stated that success is built on balanced growth across both COEs and the community, noting that 70% of Q3 prescribers were new. President of R&D Suma Krishnan confirmed the year-end AATD update will include data from Cohorts 1 and 2. She added that if expression is sufficient in Cohort 2, they will stop there, but they have the option to proceed to Cohort 3 as no dose-limiting toxicities have been observed.

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