Maxwell Skor

Maxwell Skor of Morgan Stanley inquired about how proceeds from Priority Review Vouchers (PRVs) are being factored into the company's path to GAAP profitability by 2027 and the level of confidence in receiving all three anticipated PRVs.

Answer

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.

Maxwell Skor asked for a characterization of Brensupri's performance to date relative to the provided launch analogs and expectations for the next two and four quarters.

Answer

Will Lewis, Chairman and CEO, clarified that the company has not provided formal guidance but has used benchmarks of strong respiratory launches (high double-digit millions in first two full quarters combined) as ambitions. He cautioned against over-interpreting the first six weeks of data due to inventory build, initial trials, and early adopter effects, stating that Q4 data will provide clearer insights into the launch trajectory.

Maxwell Skor asked how BRINSUPRI's performance to date compares to launch analogs and about expectations for the next two full quarters and the following four quarters.

Answer

Will Lewis, Chairman and CEO of Insmed, clarified that no formal guidance has been provided, only benchmarks from strong respiratory launches (high double-digit millions in the first two full quarters combined). He reiterated caution due to the very early data (six weeks), inventory build, and initial trials, emphasizing that Q4 will provide clearer insights into the launch trajectory.

Maxwell Skor asked for early insights from the first DMD patient dosed in the ASCEND trial and how Insmed's intrathecal delivery approach is differentiated from other gene therapies.

Answer

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.

Maxwell Skor asked about the TPIP readout in PAH, questioning the rationale for measuring PVR versus baseline and what the primary endpoint would be for a potential Phase III trial.

Answer

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.

Maxwell Skor asked for the rationale for measuring PVR versus baseline in the TPIP trial, expectations for placebo rates, and what the primary endpoint for a potential Phase III trial might be.

Answer

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.

An analyst on behalf of Maxwell Skor from Morgan Stanley asked about expectations for placebo response in the global Phase III study for CAH and any potential impacts from different geographies.

Answer

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.

Maxwell Skor asked for commentary on the evolving regulatory environment and any potential implications for the rare disease space, acknowledging Crinetics' interactions are not with CDER for paltusotine.

Answer

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.

Maxwell Skor of Morgan Stanley asked if a single pivotal trial for the TransCon CNP and growth hormone combination could be sufficient for approval, given the impressive growth velocity seen in the COACH trial.

Answer

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.

Maxwell Skor of Morgan Stanley asked for an update on Amicus's confidence in its Galafold intellectual property position and the potential for a settlement with Aurobindo, particularly after reviewing Aurobindo's brief for summary judgment.

Answer

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.

Maxwell Skor of Morgan Stanley inquired about other potential indications for DMX-200's mechanism and asked for insights on potential FDA changes to improve rare disease drug development.

Answer

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.

Maxwell Skor of Morgan Stanley asked if the recently submitted REMS for aficamten differs from what might have been submitted initially, considering the recent Camzyos REMS update.

Answer

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.

Maxwell Skor of Morgan Stanley inquired about the status of the treatment Emergency Use Authorization (EUA) for PEMGARDA, including any feedback from the FDA, and asked what factors would restore the company's confidence in providing revenue guidance.

Answer

Marc Elia, Chairman of the Board, stated that the treatment EUA application is being updated and they are awaiting feedback from the FDA. Regarding guidance, he explained that confidence is tied to recent performance trends and that the company feels it has moved past the recent period of market confusion. Chief Commercial Officer Timothy Lee added that he is pleased with current growth trends and ongoing commercial initiatives, which support a positive outlook.

Inquired about the timeline and potential market size for a second Emergency Use Authorization (EUA) for treatment, and asked about the accuracy and expansion of the PEMGARDA infusion site locator, particularly regarding large academic centers.

Answer

The company stated that while a year-end timeline for the treatment EUA seems reasonable, the decision is ultimately up to the FDA. The opportunity is seen as potentially large and additive to the prevention rollout. The infusion site locator is being updated in real-time and does not yet fully reflect all available sites. A dedicated team is actively working to onboard major academic centers and private infusion sites to improve access.

Maxwell Skor of H.C. Wainwright & Co. asked for details on Invivyd's marketing strategy for PEMGARDA, including direct-to-consumer plans and target physicians, and inquired about the planned compact clinical trial for the treatment indication.

Answer

Marc Elia, Chairman, explained the compact trial is a confirmatory study for the treatment EUA, focusing on safety, pharmacokinetics, and virology based on existing data. Jeremy Gowler, Interim CEO, stated the initial marketing focus is on healthcare provider (HCP) awareness, with direct-to-consumer (DTC) campaigns being explored for the future, noting strong organic interest from the highly-engaged patient community.

Maxwell Skor inquired about the payer dynamics for Hepatitis C, specifically asking what percentage of chronic HCV patients are on Medicaid and if they are typically prescribed generic Epclusa.

Answer

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.