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CEO Dr. Matthew Klein stated there was no discussion of delays in the vutiquinone review. He and CBO Eric Pauwels differentiated the potential launch from Skyclaris, highlighting PTC's experienced pediatric neurology team, the different patient dynamics (especially for pediatrics), and the lack of a monitoring requirement for vutiquinone. Dr. Klein added that the FA community generally believes a cocktail of therapies will be optimal, so combination use with Skyclaris is certainly something that could be explored by physicians.

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CEO Dr. Matthew Klein confirmed PTC has earlier-stage programs leveraging its splicing and ferroptosis platforms, including a DHODH inhibitor and an NLRP3 inhibitor. He also affirmed that the company is actively evaluating various business development opportunities, from early-stage assets to commercial licenses, to complement its portfolio.

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CEO Dr. Matthew Klein defended the placebo group as the most appropriate 'gold standard' benchmark for the trial, highlighting the heterogeneity of Huntington's disease. He emphasized that having 12 months of placebo-controlled data is a significant strength and that while they could use natural history to augment longer-term data, the concurrent placebo control is crucial.

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CEO Matthew Klein positioned vatiquinone as acting upstream of SKYCLARYS and stressed the significant unmet need for a therapy in pediatric and adolescent FA patients, also noting potential for combination use. For utreloxastat, he highlighted the strong preclinical data supporting the rationale of targeting ferroptosis, a key pathological element in the complex and aggressive nature of ALS.

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Founder and CEO R. Scott Struthers responded that he views the Crinesity launch as a positive development that raises disease awareness and should actually benefit enrollment, noting that the bulk of Crinetics' trial enrollment is outside the U.S. Chief Medical & Development Officer Dana Pizzuti added that Crinetics is targeting a broader CAH patient population, aiming to address the full spectrum of the disease, which is a key distinction.

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CEO R. Struthers addressed the CAH trial, stating he does not expect the newly approved competitor drug to significantly impact global enrollment, anticipating better recruitment than prior trials. CCO Isabel Kalofonos explained the patient activation strategy involves disease awareness campaigns highlighting unmet needs, educational events, media outreach, and a comprehensive patient support hub.

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CEO R. Struthers explained that for large indications like Graves' disease, the company applies extra scrutiny regarding scalability and physicochemical properties. Biologically, he noted that since TSH receptor activation is the root cause of Graves' and also cross-activates the IGF-1 receptor, blocking the TSH receptor directly should be at least as efficacious as targeting the downstream IGF pathway.

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CEO William Lewis clarified that while they do not anticipate any specific delays, the timing of the readout depends on ensuring the data is cleaned to submission-level quality. Regarding the blinded data, he stressed that it does not confirm efficacy but that the overall pattern of results is consistent with what one would expect to see if the drug were working. He cautioned against over-interpreting this observation.

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CEO William Lewis positioned the dual Phase II and Phase III publications in the NEJM as an extremely rare and positive event. He contextualized the editorial's mention of macrolides as a perspective from rationed healthcare systems, a view not encountered in Insmed's target markets and one that carries significant clinical risks like antibiotic resistance.

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CEO William Lewis confirmed a high-resolution CT scan is required for a definitive bronchiectasis diagnosis. Regarding the editorial, he contextualized it as a perspective from rationed healthcare systems, stating macrolide monotherapy is not a standard of care in target markets and is not encountered in payer discussions. CMO Martina Flammer highlighted that over 60% of views on the NEJM article came from the public, indicating strong patient interest.

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CEO Will Lewis identified market access as the most critical controllable element, emphasizing a strategy for a 'frictionless launch' through verbal physician attestation and minor contracting. CFO Sara Bonstein added that the field access team has been augmented. Regarding the FDA, Lewis expressed confidence, noting that review staff are funded by PDUFA fees and should be insulated from general government staffing issues.

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CEO Will Lewis expressed high confidence in the newly expanded sales force, highlighting their experience and ability to call on every U.S. pulmonologist, comparing the pre-launch strategy to the successful ARIKAYCE launch. CFO Sara Bonstein pointed to successful respiratory launches like DUPIXENT as analogs for the potential revenue ramp, while reiterating the $5 billion-plus peak sales opportunity for brensocatib in bronchiectasis. Will Lewis noted that no final decisions on pricing have been made.

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CEO Will Lewis highlighted the high quality and experience of the 184-rep sales force, noting they can call on every U.S. pulmonologist and are replicating the successful ARIKAYCE launch model. CFO Sara Bonstein pointed to respiratory launch analogs like DUPIXENT, which achieved high double-digit millions in revenue in their first two quarters, underscoring brensocatib's $5 billion-plus peak sales potential. Lewis stated that no final decisions on pricing have been made.

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President and CEO Bradley Campbell confirmed that all necessary Pombility/OpFolda material for 2025 U.S. commercial and clinical use has already been imported, negating any material P&L impact from tariffs this year. He stated the Ireland facility will supply Europe in late 2025 and the U.S. in 2026, with sufficient capacity for forecasted global demand. He also highlighted a diverse supply strategy, including a new U.S. drug product partnership with Sharp.

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President and CEO Bradley Campbell stated the deal aligns with their strategy for late-stage, de-risked assets with synergies to their rare disease capabilities. Confidence was built on strong Phase II data, high unmet need, and recent FDA alignment on proteinuria as a primary endpoint. Chief Development Officer Dr. Jeff Castelli added that DMX-200 is differentiated by its unique mechanism targeting monocyte-driven inflammation, unlike competitors focused on hemodynamics.

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President and CEO Bradley Campbell explained that Q2 had benefited from a new country launch and clinical trial conversions, making Q3 a more normalized quarter. He noted that the gap between prescriptions and treated patients is closing as insurance processes improve. He stated that the 2025 outlook depends on the year-end patient count and the timing of new country launches, which are contingent on reimbursement outcomes.

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President and CEO Bradley Campbell confirmed that the rate of new patient additions is progressing as expected and is higher than last year. He anticipates momentum will build through the second half as new countries launch, but noted the revenue impact will be more significant in 2025. The current focus is maximizing the total number of patients on therapy by year-end to establish a strong revenue run-rate for next year.

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President & CEO Paul Bolno stated that preclinical models indicate they have not yet hit peak saturation of the ADAR enzyme, suggesting ample room for increased protein production with multi-dosing and higher doses. He defined 'best-in-class' for WVE-006 by its use of GalNAc for efficient, subcutaneous delivery without LMPs, combined with proprietary chemistry like PN and N3-uridine modifications that optimize the AIMER editing platform for high hepatocyte uptake and intracellular stability.

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President and CEO Paul Bolno explained that based on platform chemistry, multiple doses of WVE-006 are expected to increase protein production and durability compared to a single dose. For WVE-007, he emphasized that while weight loss will be tracked, the key differentiator from GLP-1s will be demonstrating healthy weight loss through body composition changes, specifically fat loss without muscle reduction, which the initial data will aim to establish.

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CEO Faheem Hasnain explained the extension was to honor commitments to patients and physicians amid high demand, ensuring goodwill for future studies like PH-ILD. He, along with CMO Dr. Richard Aranda, noted that the global patient base includes 'purer' PAH patients with fewer comorbidities, who have historically shown a larger treatment effect. CFO & COO Bryan Giraudo added that the previous TORREY study was more US-focused due to the COVID-19 pandemic.

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President and CEO Peter Greenleaf stated that any tariff impact would be minimal for several years, as the company has multiple years of API stockpiled on U.S. soil and the API represents a small portion of COGS. He noted that changing transfer pricing rules would likely require an act of Congress, not just an executive order. Greenleaf confirmed the company is watching all policy developments closely, noting that Aurinia's payer mix is split evenly between private and government payers.

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President and CEO Peter Greenleaf explained that after five years on the market, the company believes its historical financial results provide sufficient insight, making patient-level metrics unnecessary. Chief Medical Officer Dr. Greg Keenan confirmed that preclinical data for AUR200 suggests high potency, which could potentially allow for lower or less frequent dosing, but that this will be confirmed in human studies.

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CMO Dr. Walid Abi-Saab emphasized the long and critical history of working with patient advocacy groups like CHDI, calling them balanced, scientifically driven, and essential for developing the natural history data used in trials. He affirmed that uniQure's progress would not have been possible without their collaboration, which is well-respected by the FDA.

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CEO Emil Kakkis acknowledged the competitive environment, stating that the most potent and effective drug will likely lead the market. He positioned GTX-102 as the most potent ASO based on existing data and highlighted the company's extensive long-term data from nearly 70 patients in the expanded Phase II study as a key differentiator. He affirmed Ultragenyx's goal to be the long-term leader in Angelman syndrome treatment.

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CEO Emil Kakkis noted that fractures in the Phase II study were not limited to severe OI types, as some Type I patients also fractured. Regarding UX111, CEO Emil Kakkis and CCO Erik Harris stated they will leverage learnings from other gene therapy launches like Zolgensma, focusing on payer engagement and the disease's urgency. The existing commercial team for Dojolvi is already engaged with the target treatment centers.

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CEO Emil Kakkis stated the Orbit study was powered assuming a 50% fracture rate reduction, but noted the Phase 2 data showed a 67% reduction on top of prior bisphosphonate use. He explained this gives them confidence in the design. For the Cosmic study, a similar 50% reduction assumption was used, noting that the higher fracture rate in younger children allows for a smaller study size.

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President and CEO Douglas Ingram (speaking in place of Dallan Murray) described the patient webinar as very meaningful but just one part of a broader educational effort. He stressed the need to communicate the complete evidence package—both the safety profile and the impressive efficacy data, like the muscle MRI results—to the entire patient and physician community to underscore the therapy's value and the urgency of treatment.

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CEO Douglas Ingram disagreed with the characterization of modest growth, highlighting the projected 160% year-over-year growth for ELEVIDYS in 2025. He described the launch as being in a robust growth phase, explaining that the quarterly cadence is dictated by a predictable, roughly four-month process for each patient from start form to infusion, which the company had anticipated and is managing effectively.

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Dr. Louise Rodino-Klapac, Head of R&D, explained that the long-term follow-up from the EMBARK study will track patients over time and compare them to natural history. The study will continue to use MRI/MRS and T2 imaging to assess inflammation and edema, and she confirmed that strain analysis is a component of the MRI results.

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CEO Douglas Ingram asserted that the company has been wholly committed to the Duchenne community for over seven years, successfully launching four transformative therapies. He believes the vast majority of the informed patient and physician communities understand and support Sarepta's efforts, citing the high demand for ELEVIDYS as evidence. He emphasized that the team's track record in serving the community is second to none.

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Cristin Hubbard, Executive Vice President and Chief Commercial Officer, detailed a strategy focused on driving adoption across all age groups, especially the newly-labeled 0-4 population. Key initiatives include increasing awareness to expand the prescriber base, particularly among pediatric endocrinologists, and investing in additional field personnel. She noted the new business unit structure enhances focused execution on these strategies.

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President and CEO Alexander Hardy clarified that the company's IP for Voxzogo extends to the mid-2030s, providing ample time to convert the market to a more efficacious BMN 333. EVP, Worldwide R&D Greg Freiberg added that the development timeline for BMN 390 represents an 'upper error bar' and the company will actively look for opportunities to accelerate it.

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EVP and President, Ascendis U.S. Jay Wu asserted that their patient support hub is a 'well-oiled machine' with extensive experience from the SKYTROFA launch, supported by a strong field reimbursement team, ensuring office burden will not be a bottleneck. On the COACH trial, CEO Jan Mikkelsen expressed high confidence that the combination of TransCon CNP and growth hormone could set a new standard for treatment, improving not only height but also other medical complications of achondroplasia.

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Jan Mikkelsen, President and CEO, stated he expects a much faster and larger uptake for YORVIPATH in the U.S. compared to Europe and does not see the monitoring as a deterrent. Scott Smith, EVP and CFO, explained the SKYTROFA guidance adjustment was due to slightly lower-than-expected Q3 volume and a cautious outlook for Q4.

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Iain Fraser, Head of Development, confirmed that hematological parameters indicating hemolysis, like bilirubin, will be reported. He stated that full 3-month treatment data for all 16 patients will be available, with follow-up data for a subset. Executive Alexander Sapir explained that the high adherence rate is captured using an AI tool called AiCure, which provides more timely and accurate data than traditional pill counts by having patients record themselves taking the medication.

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CEO Alexander Sapir stated that the 10 enrolled patients are from the U.S. and South Africa and tend to be on the more severe side, with high adherence rates above 90%. Executive Iain Fraser added that the withdrawal of Oxbryta has created a new pool of eligible patients. Regarding the DSMB, Fraser clarified its primary focus is on reviewing safety and tolerability data from the 12mg cohort to approve advancement to the 20mg dose.

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CEO Alexander Sapir explained the pociredir delay is due to the long lead times required to activate new academic sites, as previous sites did not have patients matching the new, stricter inclusion criteria. For losmapimod, executive Iain Fraser clarified the goal is to define a 'meaningful score difference' (MSD) for individual patients, and the trial results will likely be analyzed based on the proportion of patients who exceed that MSD.

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CCO Peter Heerma explained that while the fulfillment rate has improved significantly since the early launch, a 100% conversion is never expected, citing an 85-90% pickup rate even for retail nephrology products. CEO Eric Dube added that patient compliance and persistence rates are very high once on therapy, indicating the main focus is on improving the initial conversion from a PSF to a paid prescription.

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CEO Carsten Brunn and CMO Peter Traber explained that liver diseases are a primary target due to being T-cell mediated and the presence of known autoantigens in conditions like PBC. Dr. Traber noted the approach could extend to other autoimmune diseases. Regarding Sobi, Mr. Brunn stated that while he couldn't comment on specific resources, Sobi is highly committed, with a BLA filing planned for H1 2024 and more data to be presented at a future scientific conference.

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CEO Carsten Brunn and CMO Peter Traber explained that liver diseases are a prime target due to their T-cell-mediated nature, the presence of known autoantigens, and preclinical data showing ImmTOR's accumulation in the liver. They view this as a strategic entry point to broader autoimmune applications. Regarding Sobi, Carsten Brunn stated that while he cannot comment on Sobi's specific resource allocation, Sobi is highly committed to the program and is targeting a BLA filing in the first half of 2024, with full data to be presented at a future scientific conference.

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CEO Carsten Brunn and CMO Peter Traber explained that the focus on liver diseases is driven by their T-cell mediated nature and promising preclinical data. Traber noted that success in liver diseases could pave the way for broader autoimmune applications. Regarding Sobi, Brunn confirmed their strong commitment and a planned BLA filing in H1 2024, with full data from the DISSOLVE trials to be presented at a future scientific conference.

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CEO Carsten Brunn and CMO Peter Traber explained that liver diseases are a prime target due to their T-cell-mediated nature and the fact that ImmTOR accumulates in the liver. Traber noted that preclinical data shows additive effects and that findings could extend to other autoimmune diseases. Regarding SEL-212, Brunn confirmed Sobi is highly committed, with a BLA filing planned for H1 2024 and full data to be presented at a future scientific conference.

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President and CEO Carsten Brunn confirmed that interim data for SEL-302, focusing on safety, biomarkers, and neutralizing antibody (Nab) response, is expected toward the end of the year, likely after the first ImmTOR-dosed patient. Regarding ImmTOR-IL, Brunn stated that Selecta's combination approach is a first-in-class Treg-selective therapy. He highlighted that the liver is a strategic focus because ImmTOR accumulates there and has shown hepatoprotective properties in models, with more preclinical data to be shared after the DISSOLVE readout.

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President and CEO Carsten Brunn confirmed that interim data for SEL-302, including safety, biomarkers, and Nab response from the first patient with ImmTOR, is expected toward the end of the year. He differentiated the ImmTOR-IL program by highlighting its first-in-class approach of inducing and expanding Treg-selective cells, and noted the strategic focus on liver diseases where ImmTOR accumulates and has shown hepatoprotective effects in models.

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SVP and Head of Clinical Development Roxana Donisa Dreghici stated that the company is confident in SGT-001's risk-benefit profile, highlighting an optimized eculizumab regimen and close patient monitoring as key parts of the mitigation strategy, which will be reassessed for the new manufacturing material. CEO Ilan Ganot added that engagement with regulators has begun, with the goal of having new material ready to dose patients in early 2023, while the final clinical trial design is still being determined.

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Chief Scientific Officer Carl Morris acknowledged an apparent correlation in the data but cautioned it was from a small sample size (n=3). Acting Chief Medical Officer Dr. Cathryn Clary added that while the results are encouraging for designing a future registration trial, more data is needed. Regarding the SAE, Dr. Clary described it as an expected inflammatory response whose complexity requires thorough analysis before dosing Patient 9, a process being conducted with the DSMB and FDA.

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CTO Joel Schneider confirmed that the company uses multiple orthogonal methods to quantify microdystrophin, including immunofluorescence, western blot, and mass spectrometry. He highlighted the development of a quantitative in vitro expression assay that was instrumental in getting the FDA clinical hold lifted. CEO Ilan Ganot elaborated on strategy, stating that the company is constantly exploring next-generation vectors through both in-house research and collaborations to create more options for patients, viewing the Ultragenyx partnership as a key example of this approach.

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Chief Technology Officer Joel Solomon Schneider confirmed that the company uses multiple orthogonal methods to quantify microdystrophin, including immunofluorescence, western blot, and mass spectrometry. He also highlighted a new quantitative in vitro expression assay that was key in getting the FDA clinical hold lifted. CEO Ilan Ganot elaborated on vector strategy, stating that Solid is continuously exploring next-generation vectors through in-house research and remains open to further collaborations to ensure more therapeutic options for patients.

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President and CEO Aoife Brennan deferred to Chief Business Officer Molly Harper, who explained that low PKU treatment rates are due to the limited efficacy of current therapies for the majority of patients, not a lack of awareness. She stated the community needs a safe, convenient, and broadly effective oral option. David Hava then addressed the Ginkgo collaboration, noting that while SYNB1353 uses the same core platform as other programs, Ginkgo's expertise is in identifying novel molecular components like transporters and enzymes, which accelerates development and improves potency.

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Dr. Ronald Hoffman of Mount Sinai Hospital responded that platelet and leukocyte counts have remained steady during treatment, with no thrombotic events or disease evolution observed. Samuel Saks, Chief Medical Officer, added that the company is currently designing the pivotal study with thought leaders and plans to discuss it with the FDA, with further data updates expected in the second half of the year. President and CEO Dinesh Patel noted that enrollment is strong and the company will share more data as it becomes available.

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