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President and CEO Ian Mortimer explained that human PK data is needed to confirm dosing frequency but that an IV formulation is a future possibility. For the proof-of-concept study, he indicated that the company is evaluating postoperative pain models, such as bunionectomy and abdominoplasty, with more details to be shared at an upcoming R&D webinar.

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Chief Medical Officer Dr. Chris Kenney explained that the fMRI endpoint was chosen by the academic investigators to mechanistically explore the drug's effect on the brain's reward circuit, following up on a prior study. He acknowledged the fMRI endpoint was negative but stressed that Xenon's focus was on the clinical scales (MADRS and SHAPS), which showed consistent and positive signals, reinforcing the drug's potential antidepressant effects.

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President and CEO Ian Mortimer stated that while early proof-of-concept studies would likely be in traditional acute pain models like bunionectomy, he believes the program has broad potential in both acute and chronic pain. He confirmed that trial design elements, including the potential use of an active comparator, are currently under consideration, with more details to be shared at a future R&D-focused event.

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Chief Commercial Officer Isabel Kalofonos stated that paltusotine will use a closed distribution system. She confirmed that the company does not plan to make the data available and that it will be blocked from services like IQVIA to allow Crinetics to track the launch and uptake across different segments internally.

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Dr. R. Struthers, CEO, clarified that their trial is designed to enroll a broader patient population than the crinecerfont study. He confirmed the pediatric plan involves ensuring correct doses for different age groups, not just a change in nomenclature. A key differentiator he noted is that their study includes glucocorticoid reduction, which the other trial did not.

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Chief Endocrinologist Dr. Alan Krasner explained that surgery is the main treatment, but many patients do not receive it, and the current medical option only addresses hypercalcemia, not bone or kidney damage. He stated a PTH antagonist could treat all aspects of the disease. CEO R. Struthers added that it could transform the treatment paradigm for non-surgical candidates and that an R&D Day is planned to provide more detail.

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President & CEO Curran Simpson explained the trial is designed for efficiency, with an interim readout from the Phase 2b portion that allows it to roll into a Phase 3 portion and trigger the start of a second pivotal study. CMO Dr. Steve Pakola confirmed the trials will be double-masked with a sham injection control, a plan aligned with the FDA. He noted an active comparator is not feasible as repeated injections are not the standard of care for this patient population.

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CEO Curran Simpson stated that from their viewpoint, it is 'business as usual' with the FDA, with no significant changes in response times or team stability. Chief Medical Officer Dr. Steve Pakola responded that trial screening shows a broad distribution across ages. He emphasized that RGX-202's key differentiators, especially its strong safety profile with no SAEs or LFT elevations and high microdystrophin expression in older boys, are compelling factors for families across the entire age spectrum.

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CFO Jim Mackaness reiterated cash OpEx guidance of $120M-$130M for 2025, potentially rising toward $140M with EU and lifecycle initiatives. He projected 2026 OpEx to be north of $150M. He explained that with a launch exceeding 'modestly successful' expectations, the company is on a path to near-term cash flow positivity.

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CFO Jim Mackaness reiterated prior guidance of $120M-$130M in cash OpEx for 2025, potentially rising to $140M, and projected over $150M for 2026 depending on the European strategy. He stated that the strong launch puts the company on a path to reach cash flow positivity in the near term, exceeding their 'modestly successful' launch model.

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CFO James MacKaness noted that Vykat XR's weight-based pricing is a key differentiating factor, especially with a potentially younger patient mix. CCO Meredith Manning pointed out that Soleno's sales force was more recently deployed than the SKYCLARYS team was at launch. CEO Anish Bhatnagar emphasized the broad base of 131 unique prescribers as a strong foundation for sustainable growth.

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Executive James MacKaness noted that Vykat XR's weight-based pricing is a key variable, particularly with a potentially younger patient mix. CCO Meredith Manning added that Soleno's sales team was more recently deployed than the SKYCLARYS team was at launch. Executive Anish Bhatnagar emphasized the broad base of 131 prescribers as a strong foundation for sustainable growth.

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President & CEO Marcio Souza confirmed the data was entirely from focal onset seizure patients, with generalized epilepsy data to come later. He explained that a direct safety breakdown by background ASM is difficult because patients were on multiple drugs. However, he noted that analysis of blood levels showed a significant portion of patients were at toxic concentrations of their background ASMs, which likely contributed to side effects and reinforces the strategy of proactively reducing background doses.

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CFO Matt Abernethy clarified that there was a $5 million inventory build in the channel during the quarter, reflecting the product's growth trajectory. He noted that the gross-to-net calculation is currently driven primarily by co-pay assistance and distribution fees, as they have not yet entered into significant payer contracting for the product.

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CEO Kyle Gano responded that their competitor's drug appears to be used primarily as a monotherapy, and he emphasized the difficulty of running adjunctive treatment trials. He stated Neurocrine's current strategy is to focus on the large standalone opportunity for its muscarinic agents in acute psychosis and other indications like bipolar mania.

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CEO Kyle Gano expressed excitement for the program, highlighting its key differentiator: an IV-administered gene therapy using advanced capsids designed to deliver frataxin to both the cardiovascular system and the CNS, addressing cornerstone symptoms of the disease. He confirmed the program is on track to advance from preclinical to clinical development later in the year.

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President and CEO Douglas Ingram stated that the autopsy results are not yet available but are expected in the next month. Head of R&D Dr. Louise Rodino-Klapac explained the label update is a process of finalizing language with the FDA, which has already confirmed general agreement, rather than awaiting a decision, and the agency would simply ask questions if needed.

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CEO Douglas Ingram and Chief Customer Officer Dallan Murray explained that it's not a single bottleneck but a complex, multi-step cadence that takes about four months per patient. Murray summarized the key factor as 'time,' as the process involves robust demand, ample site capacity, and positive reimbursement trends, but requires coordination of ~25 people per institution for each dose. Ingram emphasized that their team's expertise in managing this detailed process gives them confidence in their guidance.

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CEO Doug Ingram clarified the studies are for naive patients with pre-existing antibodies, not redosing. Head of R&D Dr. Louise Rodino-Klapac projected initial safety and expression data in early 2025, which will inform any future redosing strategy. Ingram added that Sarepta has a right of first negotiation for Hansa's next-generation therapies.

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CCO Dallan Murray and CEO Douglas Ingram explained the calculation is not simple due to confounders, primarily the significant and robust PMO sales that occur outside the U.S. Murray stated that they have not seen concurrent commercial use of both therapies to date, though they are aware of some patient interest in that possibility, similar to what has been seen in the SMA space.

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CEO Christopher Peetz acknowledged that the competitor's Phase II proof-of-concept data was a factor but stated Mirum's study start is independent due to their molecule's differentiated profile. Chief Medical Officer Joanne M. Quan emphasized that MRM-3379 is highly selective and has a significantly higher brain-to-plasma ratio, which she described as a key potential advantage for treating a CNS condition like Fragile X.

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An executive, likely Dr. Karen Sims, explained that while a robust set of biomarkers is being collected, there are no clear expectations for significant changes in HBV-specific markers after a short 28-day treatment, as AB-101 is an immune modulator, not a direct-acting antiviral. Chief Scientific Officer Dr. Mike Sofia added that the primary strategic goal is to establish AB-101's monotherapy profile before advancing it into combination therapy with imdusiran.

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Chief Medical Officer Dr. Karen Sims clarified that 25mg is not necessarily a dose cap for AB-101, as the trial design allows for flexibility. She stated she could not comment on unreleased nivolumab data but affirmed the company takes a holistic view of all its trials. Chief Scientific Officer Dr. Michael Sofia responded that the company is aware of a recent abstract on ALPK1 agonism and will evaluate the target more fully after its presentation at EASL.

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The company explained that targeting PD-L1 with a small molecule fits their liver-centric strategy. Nivolumab was chosen for the 202 trial based on collaborator Barinthus's prior positive data. Durvalumab was chosen for the 203 study to specifically inform the future proprietary combination of imdusiran and AB-101 by exploring optimal timing and administration.

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The company has not looked at blinded data. Their confidence in a low placebo rate (and thus being well-powered) comes from the trial's design, which enrolled a highly refractory population that had failed an average of 3.5 drugs. The secondary endpoint, time-to-status cessation, is a continuous variable and considered statistically very robust. They expect a large separation, as ganaxolone-treated patients in Phase 2 stopped seizing in a median of 5 minutes, while placebo patients would likely not stop for hours until receiving another treatment.

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