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CEO Dr. Roger Jeffs attributed competitor issues to a formulation that struggles with lower airway drug delivery. He contrasted this with YUTREPIA's PRINT technology, which enables better tolerability and faster titration to higher doses. Chief Medical Officer Dr. Rajeev Saggar added that ASCENT data shows patients can reach high therapeutic doses in weeks, not months, with a favorable tolerability profile, which is a key differentiator.

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CEO Dr. Roger Jeffs projected a 3.5 to 4-year timeline from first patient enrollment to a regulatory decision for L606, with trial initiation planned for the first half of 2025. He noted the potential to accelerate this timeline due to significant unmet need in ex-U.S. markets where competitor products are not available. Dr. Jeffs emphasized the strategy to establish L606 as a global brand, supported by an expanded license with Pharmosa and a next-generation portable nebulizer.

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Chief Commercial Officer Peter Heerma stated that the REMS program has not been an obstacle to performance, noting that FILSPARI has set 'best-in-class benchmarks' compared to recent rare nephrology launches that did not have a REMS. However, he added that the company looks forward to the potential modification in August to enhance convenience for patients and physicians.

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CCO Peter Heerma stated that while the current REMS has not been a major obstacle to adoption, a modification would have a positive impact. He believes changing from monthly to quarterly monitoring would broaden physicians' perception of which patients are suitable candidates for FILSPARI, particularly those they might preemptively consider non-compliant with a monthly schedule, thereby expanding the addressable patient pool.

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CFO George Elston clarified that the cash runway guidance into 2027 includes all work for DURAVYU in wet AMD and internal preparation for a DME study, but not the cost of the study itself. He described the razuprotafib program as being on 'percolation mode' with a lower priority to conserve cash and maintain focus on wet AMD.

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President and CEO Dr. Pravin Dugel expressed high confidence, referencing a prior U.S. study where 60% of patients went 52 weeks on a single injection, noting that was in a non-enriched population. He believes a significant number of patients in the current, more selected trial population will achieve this endpoint. He positioned this potential outcome as highly valuable for physicians, payers, and patients, and a key factor in securing a best-in-class label.

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President and CEO Dr. Pravin Dugel stated that the regulatory path is very clear, supported by a Special Protocol Agreement for SOL-1 and a Type C written response for SOL-R. He clarified that the company's current assumption, based on historical FDA precedent, is that two positive studies will be required for approval.

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Chief Financial Officer Cecilia Jones explained that the company does not anticipate an initial patient bolus, expecting instead a steady uptake as patients attend their regular physician visits. CEO Brian Goff noted the initial focus will be on 65% of the patient population. For the Gulf region, Jones identified it as the second commercial priority after the U.S., with a launch ramp dynamic similar to Europe, requiring time to secure access and formulary listings post-approval.

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CFO Cecilia Jones stated that capital allocation priorities are the potential launches in thalassemia and sickle cell disease, advancing the current pipeline, and disciplined pipeline expansion. CEO Brian Goff and CMO Dr. Sarah Gheuens confirmed the RISE UP trial's two primary endpoints, hemoglobin response and sickle cell pain crisis reduction, are each powered at over 90%.

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CEO Dr. Cedric Francois stated the data represents a chance for young patients to potentially avoid dialysis or transplant, highlighting a superior profile on proteinuria, eGFR, and C3c staining. CMO Dr. Caroline Baumal, who attended the nephrology meeting, reported that clinicians were 'ecstatic' and 'excited' about the findings, particularly the rapid onset of effect as early as four weeks, which could prevent irreversible kidney damage.

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