Maxwell Skor • H.C. Wainwright & Co.

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President & CEO Jan Møller Mikkelsen did not directly confirm if one trial would suffice but highlighted the unprecedented growth velocity observed. He discussed a potential treatment paradigm where patients could receive the combination therapy for 1-2 year 'boosts' and then maintain on CNP monotherapy, suggesting a focus on defining the optimal clinical use rather than just the regulatory path.

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Chief Medical & Development Officer Dana Pizzuti explained that due to the ambitious endpoint, which requires addressing both A4 levels and reducing glucocorticoids to physiologic levels, the company does not expect a high placebo response rate. She stated it would be very difficult for a placebo patient to achieve this endpoint, regardless of their position on the CAH spectrum.

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Dr. R. Struthers, CEO, stated that their interactions with the FDA (CDER) have not shown any significant changes and that business is proceeding as normal across all their programs, most of which are for rare diseases. He sees no differences at this point.

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CEO William Lewis reported 'so far so good' but emphasized a highly cautious 'belt and suspenders' approach. He explained the key differentiation is that intrathecal delivery avoids the first-pass liver effect, allowing for a lower viral dose while achieving remarkable tissue transduction, which could improve safety and efficacy. CMO Martina Flammer added that another important distinction is that the study does not use weight-based dosing.

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CMO Martina Flammer explained that PVR is measured at baseline and end-of-study to assess the reduction over the treatment period. She stated that the recognized registrational endpoint for a Phase III trial in PAH is the 6-minute walk distance, which they anticipate using as the primary endpoint.

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CMO Martina Flammer explained that PVR is measured at baseline and end-of-study to assess reduction over the treatment period. She stated that the recognized registrational endpoint for Phase III is 6-minute walk distance, which is what they anticipate using as the primary endpoint, though clinical worsening would also be considered.

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Howard Horn, CFO, confirmed that PRVs from three programs are factored into their financial planning. Emil Kakkis, CEO, added that even if the PRV program is not reauthorized (affecting the OI voucher), the other two would proceed, and their likely higher value would result in a similar overall cash impact, maintaining their financial trajectory.

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President and CEO Bradley Campbell reiterated the company's high confidence in the strength and longevity of its Galafold IP. He stated that the recent settlement with Teva reinforces their case against remaining litigants and noted that statistically, such cases often lead to settlement, especially after one party has already settled. He refrained from commenting on specific litigation details.

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President and CEO Bradley Campbell expressed hope for regulatory modernization, including greater use of real-world evidence and more flexibility on placebo controls in rare diseases. Chief Development Officer Dr. Jeff Castelli identified other rare kidney diseases with unaddressed inflammatory components, like proliferative lupus nephritis, as potential future indications for DMX-200. Campbell concluded that achieving profitability will provide the resources to pursue such portfolio expansions.

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Robert I. Blum, President and CEO, described the situation as fluid and avoided speculating on a hypothetical earlier submission. He confirmed that Cytokinetics has been closely monitoring all developments, including the Camzyos REMS modification, and has submitted a REMS they believe will secure a distinct and differentiated profile for aficamten.

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Chief Commercial Officer John Vavricka explained that government-supported programs like Medicaid and Medicare represent the majority of the payer mix for HCV. He clarified that both branded Epclusa and its authorized copy are utilized, with the choice dictated by individual payer financial models. CEO and Founder Dr. Jean-Pierre Sommadossi added that true generics for current treatments are not anticipated until at least 2036 due to existing intellectual property.

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