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CEO Dr. Matthew Klein acknowledged watching the CBER review with interest, noting that positive read-throughs are possible and that regulatory agencies increasingly desire alignment between divisions like CBER and CBER on rare diseases. He expressed confidence in PTC's own data package for accelerated approval. CFO Pierre Gravier confirmed that PTC still owes a low single-digit royalty to Sarepta.

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President & CEO Marcio Souza attributed the discontinuation rate partly to the open-label setting and a learning curve for investigators in adjusting background therapies, expecting it to decrease. He agreed the placebo rate in POWER-one could be lower due to the highly refractory patient population and high-quality site management.

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CEO Marcio Souza confirmed that LGS patients will be included in the GGE study and that the Study 2 readout timing is influenced by the Study 1 interim to protect program integrity. He also noted a standard futility analysis is included. Regarding Vormatrigine, he cited strong preclinical evidence for its potential in pain but did not share clinical anecdotes, stating a full plan will be shared in the near future.

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CEO Marcio Souza stated that both studies are slated to read out together, but if Study 1 is resized, their readouts would be separated by a short period due to strong ongoing patient screening. For relutrigine, he noted a 20-30% seizure reduction would be 'phenomenal' given the severe patient population. He confirmed the initial indications are the 'tip of the iceberg,' suggesting a broader opportunity as a backbone therapy across many severe pediatric DEEs currently treated with toxic alternatives.

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CEO Joseph Belanoff clarified that the $3-5 billion revenue projection for the next 3-5 years is driven almost entirely by relacorilant, which is expected to replace Korlym. He noted the Teva patent case is not a factor in this forecast. Sean Maduck, President of Corcept Endocrinology, added that the second pharmacy will be operational in Q4 2025, with further network expansion being considered for the relacorilant launch.

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Sean Maduck, President of Endocrinology, explained the pharmacy issue was related to staffing, which has been remedied. He noted that for relacorilant, which does not have the same restrictions as Korlym, a broader distribution network is being explored. Chief Business Officer Charles Robb responded that the company is immediately seeking guidance from US and European regulators on the next steps for the ALS program.

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Chief Business Officer Gary Robb confirmed the FDA's view, citing published guidance that a single well-controlled study (GRACE) with confirmatory evidence is sufficient for approval. He expressed confidence in their position due to the extensive and consistent data across all studies. While declining to comment on specific FDA interactions, Robb stated there were no impediments to filing the NDA. Chief Development Officer William Guyer confirmed that all hyperglycemia endpoints were statistically significantly different from placebo, favoring relacorilant.

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President & CEO Paul Bolno explained that the company is advancing to cohort three (400 mg) while simultaneously expanding cohort two (240 mg) because cohort two's dose was modeled to align with weight loss seen in preclinical DIO models. He noted that this dose is expected to show more fat loss than GLP-1s for equivalent weight reduction. Bolno added that the decision was driven by favorable safety and strong preclinical-to-clinical translation, giving them confidence to enrich the dataset at this key dose level.

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President and CEO Dr. Paul Bolno explained that the INHBE data release is tied to an internal cutoff at predefined time points, which remain undisclosed. He confirmed the programs are under the FDA's CDR division and expressed confidence in the accelerated approval pathway, citing the comprehensive nature of Wave's data. Dr. Bolno differentiated Wave's allele-selective Huntington's program from pan-silencing approaches, noting Wave's superior mutant huntingtin reduction and the strong correlation observed with caudate atrophy, which he contrasted with competitor findings.

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President and CEO Paul Bolno stated that if mutant huntingtin lowering were accepted as a surrogate endpoint, Wave would be in a position to file for approval based on its existing SELECT-HD data. He explained the larger patient prevalence number reflects the opportunity to treat earlier, pre-symptomatic patients identified through genetic testing, which is a key advantage of their allele-selective approach.

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CEO Paul Bolno explained that the WVE-007 program aims for a differentiated label focused on 'healthy, sustainable weight loss.' He highlighted preclinical data showing fat loss with muscle mass retention, which will be a key measurement in the clinical trial starting in Q1 2025. Regarding the broader DMD pipeline, Dr. Bolno stated that other exon-skipping candidates have been synthesized and tested with encouraging results, but further guidance on their advancement is pending the 48-week data from WVE-N531 and regulatory feedback expected in Q1 2025.

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President & CEO Brian Lian stated that a specific oral maintenance dose has not yet been selected, as the company is awaiting the Phase II oral data to inform that decision. He clarified that while having the oral data first would be ideal, it is not a mandatory prerequisite for initiating the maintenance study.

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Brian Lian, an executive at Viking, explained that the FDA meeting will focus on finalizing the Phase III protocol, including doses and trial size. He confirmed the amylin agonist is a dual amylin and calcitonin receptor agonist (DACRA) and is benchmarked against established compounds like pramlintide and cagrilintide.

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President and Head of R&D Sukumar Nagendran explained that direct measurement of the intranuclear MECP2 protein is not currently feasible, so the rapid and consistent clinical improvements seen in patients serve as the key biomarker. CEO Sean Nolan projected that pivotal trial site initiation could begin soon, with potential dosing in Q4 2025 or Q1 2026, pending the 30-day FDA review.

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CEO Sean Nolan stated definitively that the company will no longer report RSBQ data, as the FDA does not consider it a viable primary or secondary endpoint. He confirmed that the key functional and milestone-based endpoints being discussed for Part B are already being collected in Part A, ensuring data continuity.

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CEO Sean Nolan candidly explained that the initial early data release was driven by the company's precarious financial position at the time. The positive result from the first patient enabled a successful financing. Now in a stronger position, the company is reverting to a more traditional and robust cohort-by-cohort reporting strategy. He defined a "fulsome" update as having data from the majority of the three patients in each high-dose cohort with over six months of follow-up.

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CEO Emil Kakkis firmly refuted this theory, stating that the company's clinical data from Phase II demonstrates that the drug is effective across all major OI types. He explained that while the underlying collagen defects differ, the net result of building more bone mass leads to increased bone strength and reduced fractures for all patients, regardless of their specific collagen mutation.

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CEO Emil Kakkis asserted that going to the final analysis would not imply a weaker effect, attributing a potential delay to high statistical variation in patient fracture rates rather than drug performance. He reiterated confidence that setrusumab will be a superior treatment to bisphosphonates, referencing the consistent 67% fracture reduction seen in Phase II.

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Bruce Cozadd, Chairman and CEO, stated that he did not have that specific information available. He also noted that the definition of 'PD-1 positive' can vary depending on the assay used, but confirmed that the company is measuring the biomarker and can conduct post-hoc analyses to support regulatory filings.

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Bruce Cozadd, Chairman and CEO, declined to provide additional details on either topic. He reiterated that the company is exploring lower doses for JZP-441 in patients and that there is no new information to share regarding the potential for longer exclusivity for Zepzelca at this time.

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CEO Bruce Cozadd declined to provide additional information on either topic. He reiterated that the JZP-441 trial is exploring lower doses in patients and that the company has no further specifics to share on Zepzelca's potential patent extensions at this time.

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President and COO Renée Galá attributed growth to a differentiated product profile, strong commercial execution, momentum in the adult setting, and improved market access. EVP, Global Head of R&D Robert Iannone addressed Japan, stating that despite the trial not meeting its primary endpoint at an interim analysis, they believe the overall data could support registration and plan to engage with Japanese regulators.

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President and COO Renée Galá attributed the 18% year-over-year growth to Epidiolex's differentiated profile, especially data on non-seizure benefits, along with strong commercial execution and momentum in the adult setting. EVP, Global Head of R&D, Rob Iannone, addressed the Japan situation, stating that despite the trial's interim analysis not meeting its primary endpoint, they believe the overall data could support registration and plan to engage with Japanese regulators.

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Mark Jacobson, COO, offered no new color on the FDA meeting, stating the focus is now on compiling the sNDA package. Nick Pizzie, CFO, explained that it is still premature to issue sales guidance for Auvelity due to evolving payer coverage, a recent sales force expansion, and an upcoming DTC campaign. He directed investors to the previously stated peak sales potential of $1 billion to $3 billion for the MDD indication alone.

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CEO Herriot Tabuteau stated that ADVANCE-2 is powered similarly to ADVANCE-1 at 90% to detect a comparable effect size. An executive, likely CCO Ari Maizel, noted it's difficult to compare the impact of the two drivers, but the company is pleased with demand growth post-coverage expansion, which occurred during summer seasonality.

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Executive Michael Kalb addressed the gross-to-net question, stating the 2024 impact from the Inflation Reduction Act (IRA) was minimal (<1%), but is expected to increase to a 3% to 3.5% impact in 2025 and continue rising. CCO Jeffrey Del Carmen responded on dosing, noting the average daily dose has increased by about 2 milligrams since the label expansion and that approximately 20% of patients are currently at or around the 100-milligram dose.

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COO & CSO Steven Miller confirmed the company is actively evaluating new territories for FIRDAPSE expansion. CEO Rich Daly added that opportunities in China are being explored. Regarding FYCOMPA, Executive Michael Kalb explained that the gross-to-net is higher due to new fee structures with distributors and government agencies post-TSA with Eisai, and this level is expected to be the new baseline. Daly reiterated that underlying prescription demand for FYCOMPA remains solid.

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Regarding a European partnership, Executive David Zaccardelli stated that the deal structure would depend on the partner's capabilities and that the company would prioritize the best outcome for shareholders. On reimbursement, Chief Commercial Officer Chris Martin clarified that the issue is not access but patient out-of-pocket costs. He noted that about 80% of claims fall under medical benefit, and for patients with supplemental insurance or who have met their deductible, co-pays are typically low. He also highlighted the availability of patient assistance programs.

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Chief Commercial Officer Chris Martin reported that over 80% of prescriptions are processed through medical benefit with minimal hurdles, and most patients have a co-pay under $10. He confirmed the GOLD guidelines committee is meeting soon, which presents an opportunity for Ohtuvayre's inclusion. He also noted that while use on top of triple therapy is at 50%, the other 50% is encouragingly spread across other patient types, consistent with market research.

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An unnamed executive explained that ensifentrine's potential in non-CF bronchiectasis stems from its dual mechanism targeting neutrophilic inflammation and improving mucociliary function, which could reduce exacerbations and make patients feel better. While there is no specific bronchiectasis animal model data, they cited the wealth of data from COPD on reducing cough and sputum, which involves similar pathophysiology.

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CEO John Leonard explained that gene writing is a key part of their platform, and the choice of tool depends on the specific therapeutic problem. He noted its suitability for certain conditions and mentioned that its first application is expected to be an adjunct to their alpha-1 program, addressing both lung and liver disease.

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