Financial Data and Key Metrics Changes - Total revenue for Q1 2025 was reported at $46 million, with product revenue net at $43.1 million, a decline from $54.4 million in Q4 2024 but an increase from $40.3 million in Q1 2024 [6][21] - Royalty revenue was $2.9 million in Q1 2025, down from $4.7 million in Q4 2024 and $3.5 million in Q1 2024 [7][36] - Net income based on GAAP for Q1 2025 was $3 million or $0.06 per share, compared to $19.3 million or $0.39 per share in Q4 2024 [34][35] Business Line Data and Key Metrics Changes - NERLYNX sales saw a decrease in bottle sales to 2,338 in Q1 2025, down from 2,964 in Q4 2024, with a 6% increase in new prescriptions compared to the previous quarter [8][22] - Demand for NERLYNX declined by 6% quarter over quarter but increased by 2% year over year [25] - The distribution model showed 67% of business through the specialty pharmacy channel and 33% through the specialty distributor channel [20] Market Data and Key Metrics Changes - Enrollment for NERLYNX increased by 6% quarter over quarter but declined by 8% year over year [24] - Total prescriptions decreased by 9% quarter over quarter and 3% year over year [24] - Stronger growth was noted in the specialty distributor channel, driven by increased sales in the GPO segment and 340B purchasing [20] Company Strategy and Development Direction - The company is focused on expanding NERLYNX utilization, particularly among patients at increased risk of recurrence, and has revised marketing strategies accordingly [18][26] - Puma is evaluating several drugs for potential in-licensing or acquisition to diversify its portfolio [17] - The company aims to maintain positive net income and is committed to financial discipline and expense management [43] Management's Comments on Operating Environment and Future Outlook - Management acknowledged the impact of seasonal inventory changes and higher gross to net expenses on revenue [21][36] - The company anticipates net NERLYNX product revenue for fiscal year 2025 to be in the range of $192 million to $198 million [38] - Management expressed optimism regarding the ongoing clinical trials and the potential for new data to be presented in the future [12][16] Other Important Information - The company reported cash burn of approximately $7.8 million in Q1 2025, compared to $4.3 million in Q4 2024 [41] - As of March 31, 2025, the company had approximately $93 million in cash, cash equivalents, and marketable securities [42] - The company is in the process of amending the protocol for the ELYSCA lung study to increase the dose of alisertib [16] Q&A Session Summary Question: Inquiry about Alosertib IP and Manufacturing - Management confirmed that the IP for Alosertib is licensed from Takeda, and manufacturing is primarily done in the U.S. [48][50] Question: Questions on ELYSCA lung cancer trial and NERLYNX Phase one data - Management stated that the protocol amendment for the lung cancer trial is underway, and they expect interim data later this year [58][60] - They noted better activity in certain tumor types with the combination of neratinib and ADCs, but more data is needed for specific expectations [61] Question: Impact of Medicare Part D redesign - Management indicated that co-pays for patients have declined, making NERLYNX more affordable and increasing the percentage of business going through Medicare [64]

Financial Data and Key Metrics Changes - Total revenue for Q1 2025 was reported at $46 million, with product revenue net at $43.1 million, a decline from $54.4 million in Q4 2024 but an increase from $40.3 million in Q1 2024 [5][20][34] - Net income based on GAAP for Q1 2025 was $3 million or $0.06 per share, compared to $19.3 million or $0.39 per share in Q4 2024, which included a non-cash deferred income tax benefit [32][33] - Non-GAAP net income for Q1 2025 was $5 million or $0.10 per share [33] Business Line Data and Key Metrics Changes - NERLYNX sales in Q1 2025 were $43.1 million, down from $54.4 million in Q4 2024, with a significant impact from inventory changes [20][34] - The number of NERLYNX bottles sold in Q1 2025 was 2,338, a decrease of approximately 21% quarter over quarter [20][21] - Royalty revenue was $2.9 million in Q1 2025, down from $4.7 million in Q4 2024 [34] Market Data and Key Metrics Changes - In Q1 2025, new prescriptions (NRx) increased by approximately 6% compared to Q4 2024, while total prescriptions (TRx) decreased by approximately 9% [22][23] - Demand for NERLYNX declined by about 6% quarter over quarter but increased by about 2% year over year [24] Company Strategy and Development Direction - The company is focused on expanding the utilization of NERLYNX, particularly among patients at increased risk of recurrence, and has revised marketing strategies accordingly [17][25] - Puma is evaluating several drugs for potential in-licensing or acquisition to diversify its portfolio [16] Management's Comments on Operating Environment and Future Outlook - Management emphasized the importance of maintaining positive net income and fiscal responsibility, attributing recent financial performance to expense reductions and financial discipline [40] - The company anticipates net NERLYNX product revenue for fiscal year 2025 to be in the range of $192 million to $198 million [35] Other Important Information - The company reported cash burn of approximately $7.8 million in Q1 2025, compared to $4.3 million in Q4 2024 [38] - As of March 31, 2025, the company had approximately $93 million in cash, cash equivalents, and marketable securities [39] Q&A Session Summary Question: Inquiry about Alosertib IP and manufacturing - Management confirmed that the IP for Alosertib is licensed from Takeda, and manufacturing is primarily done in the U.S. [43][44][46] Question: Questions regarding ELYSCA lung cancer trial and NERLYNX Phase one data - Management stated that the protocol amendment for the ELYSCA lung cancer trial is to increase the dose, and they expect interim data later this year [52][53] - For NERLYNX, better activity was observed in certain tumor types, and further data is anticipated [56][57][58] Question: Impact of Medicare Part D redesign - Management noted that co-pays for patients have declined, making NERLYNX more affordable and increasing the percentage of business through Medicare [60]

Core Insights - Viatris Inc. has released positive data from a Phase 3 study on the contraceptive efficacy and safety of XULANE LO, a low-dose weekly dermal patch [1][2] - The company also reported results from a Phase 3 program for a fast-acting formulation of meloxicam, MR-107A-02, for moderate-to-severe acute pain [4][5] Contraceptive Study Findings - XULANE LO demonstrated a Pearl Index (PI) of 4.14, indicating contraceptive efficacy, with a cumulative probability of pregnancy over 13 cycles at 3.7% [2] - The safety profile was favorable, with most treatment-emergent adverse events reported as mild to moderate, and a low rate of unscheduled bleeding and spotting [2][3] - The patch adhesion profile was noted as best-in-class, with only 1.3% of patches completely detaching during the seven-day wear period [3] Pain Management Study Findings - MR-107A-02 showed statistically significant and clinically meaningful results in pain management following herniorrhaphy and bunionectomy surgeries [5][8] - The treatment led to a notable reduction in opioid usage, with 72.6% of patients opioid-free after herniorrhaphy compared to 58.6% in the placebo group, and 56.9% opioid-free after bunionectomy compared to 33.1% in the placebo group [8] Financial Performance - For Q1 2025, Viatris reported total revenues of $3.3 billion, down 11% year-over-year, primarily due to the negative Indore Impact [6][9] - The estimated Indore Impact on total revenues was approximately $140 million, with management projecting a revenue reduction of $500 million for 2025 due to issues at the Indore facility [7][9] - The company reaffirmed its 2025 sales outlook of $13.5 billion to $14 billion, with adjusted EPS guidance raised to $2.16-$2.30 [9][10] Stock Performance - Following the announcements, VTRS stock increased by 10.2%, reaching $9.48 [10]

Group 1: Research and Development - The company has 4 specialized pharmaceutical research institutions and 1 postdoctoral workstation, with a research team of approximately 200 people, accounting for about 20% of total employees. In 2024, R&D investment is expected to be 16.76% of sales revenue [1] - The WYY project is a novel compound with over 20 intellectual property applications globally, demonstrating superior efficacy and safety compared to competitors in animal studies. It is currently in the preclinical research stage, with an IND application planned for submission within the year [2] Group 2: Project Updates - The Wuhan Psychological Rehabilitation Hospital project received its medical institution practice license by the end of December 2024, aiming to operate as a tertiary specialized hospital [4] - The company is advancing the production line for recombinant collagen, with stable production processes meeting industry standards, and is preparing for large-scale production [6] Group 3: Market and Financial Insights - The gross profit margin for the company's main product, "A Le," has remained around 60% despite the impact of centralized procurement policies [8] - The company is considering external licensing for the WYY project based on clinical trial progress and commercialization conditions [3] Group 4: Future Developments - The company is focusing on developing CBD for treating pulmonary arterial hypertension, with a significant unmet market demand, as there are approximately 12 million patients in China [7][8] - VGX-3100, a key project of a major associate company, is undergoing phase III clinical trials for HPV-related cervical lesions, with plans to complete participant enrollment by 2025 [8]

Financial Data and Key Metrics Changes - The company reported overall revenue growth of 18% in Q1 2025, driven by Tyvaso DPI revenues [18][19] - Non-GAAP net income for Q1 2025 was $22 million, representing a 43% increase compared to the same period in 2024 [21] - Net income for Q1 2025 was $13 million, a 24% increase from $11 million in Q1 2024 [20] Business Line Data and Key Metrics Changes - The endocrine business experienced a 20% growth in new prescriptions (NRxs) and a 14% growth in total prescriptions (TRxs) [4] - Tyvaso collaboration generated $30 million in royalty revenue and $29 million in manufacturing revenue for Q1 2025 [5][19] - Afrezza net revenues increased by 3% to $15 million, although impacted by one-time adjustments and shipment timing [19] Market Data and Key Metrics Changes - The orphan lung opportunity, particularly with Tyvaso DPI, is expected to exceed $1 billion by the end of the decade, with a focus on the US and Japan [10] - The company anticipates significant revenue potential from pediatric patients, estimating that a 10% market share could yield $150 million in net revenue [24] Company Strategy and Development Direction - The company is focusing on expanding its pediatric market strategy for Afrezza, which will differ significantly from the adult market approach [66] - The strategy includes leveraging strong data from recent studies and enhancing the sales force's presence [63][64] - The company aims to build shareholder value through various catalysts, including the anticipated approval of pediatric indications and ongoing clinical trials [22][23] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the growth trajectory of Afrezza, particularly with new data and improved sales execution [63] - The company is optimistic about the upcoming pediatric indication approval and its potential impact on market uptake [66] - Management acknowledged the challenges posed by the macroeconomic environment but believes the company can navigate these headwinds effectively [87] Other Important Information - The company has activated 85% of sites for the MannKind 101 study across four countries, with 55 patients randomized and minimal dropouts [12][13] - The company is preparing for several upcoming scientific conferences to present new data and engage with the medical community [26] Q&A Session Summary Question: Can you provide initial thoughts on the Phase two-three trial design for MANKIND-201? - The company is considering a several dose arm trial compared to placebo, with ongoing discussions regarding patient selection [30][31] Question: Are you looking to measure FVC in the IPF study? - The company plans to look at multiple doses and aims to see a delta from placebo, focusing on patient enrollment for future phases [34][36] Question: What is the rationale behind the label update for adults? - The company is seeking to improve dosing conversion for better control and anticipates scaling faster in the adult market [45][46] Question: How are you thinking about the potential impact to supply chain for Afrezza? - The company does not anticipate significant supply chain impacts due to its US manufacturing base and is managing potential tariff issues [47][48] Question: Can you provide insights on the treatment landscape for NTM and the role of 201? - The company believes inhaled natendib could serve as a background therapy for new drugs, focusing on expanding treatment options for patients [52][54]

Financial Data and Key Metrics Changes - The company reported a 18% increase in overall revenues for Q1 2025, driven by strong performance in Tyvaso DPI royalties and collaboration services [17][18] - Non-GAAP net income for Q1 2025 was $22 million, representing a 43% increase compared to the same period in 2024 [20] - The company ended the quarter with cash and investments totaling $198 million, allowing for continued investment in its pipeline [20] Business Line Data and Key Metrics Changes - The endocrine business experienced a 20% growth in new prescriptions (NRxs) and a 14% growth in total prescriptions (TRxs) [4] - Tyvaso collaboration generated $30 million in royalty revenue and $29 million in manufacturing revenue for Q1 2025, marking a 32% and 18% increase respectively compared to the prior year [5][18] - Afrezza net revenues were $15 million, a 3% increase year-over-year, although impacted by one-time adjustments and shipment timing [18][19] Market Data and Key Metrics Changes - The orphan lung opportunity, particularly with Tyvaso DPI, is expected to exceed $1 billion by the end of the decade, with a focus on the US and Japan [10] - The company anticipates significant market potential in the pediatric segment, estimating that a 10% market share could yield approximately $150 million in net revenue [22] Company Strategy and Development Direction - The company is focusing on expanding its pediatric offerings and anticipates filing for pediatric expansion in mid-2025 [4][22] - The strategy includes leveraging strong data from recent studies to enhance market penetration and address the pediatric diabetes market differently than the adult market [65][66] - The company aims to build shareholder value through a series of anticipated catalysts, including the approval of pediatric indications and ongoing clinical trials [21][25] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the growth trajectory of Afrezza, particularly with the positive reception of recent data and the potential for pediatric approval [6][61] - The company is optimistic about navigating economic headwinds, including tariffs, due to its predominantly US-based manufacturing [46][85] - Management highlighted the importance of ongoing clinical trials and the potential for new product launches to drive future growth [52][81] Other Important Information - The company is actively preparing for several upcoming scientific conferences to present new data and engage with the investment community [25] - Management noted that the current supply chain situation is manageable, with key products either exempt from tariffs or positioned to navigate potential impacts [46][47] Q&A Session Summary Question: Can you provide initial thoughts on the Phase two-three trial design for MANKIND-201? - Management indicated that they are still finalizing the trial design and have not provided extensive guidance on FDA feedback yet [29][30] Question: Are you looking to measure FVC in the IPF study? - Management confirmed that they will be looking at multiple doses and measuring delta from placebo, but specifics are still being finalized [34][35] Question: What is the rationale behind the label update for adults? - Management explained that the label change aims to improve dosing and control, with expectations for approval in Q4 [44] Question: How do you see the treatment landscape evolving for NTM and IPF? - Management noted the challenges in treating these diseases and emphasized the potential for their products to provide better tolerability and efficacy [52] Question: What are your thoughts on adherence in pediatric diabetes? - Management expressed optimism about adherence in pediatric patients, noting that caregivers are typically more diligent and that the product's profile may appeal to parents [72][74]

Financial Data and Key Metrics Changes - Acadia Pharmaceuticals reported total revenues of $244.3 million for Q1 2025, representing a 19% increase year-over-year [6][29] - R&D expenses increased to $78.3 million from $59.7 million in Q1 2024, driven by higher spending on clinical stage programs [32] - SG&A expenses rose to $126.4 million from $108 million in Q1 2024, primarily due to ongoing consumer activation campaigns for NUPLAZID and expansion of the Debut team [32] Business Line Data and Key Metrics Changes - Debut sales reached $84.6 million in Q1 2025, an 11% increase from the previous year, with a record number of unique patients receiving shipments [7][29] - NUPLAZID generated $159.7 million in revenue for the quarter, up 23% year-over-year, with 6% of this growth attributed to volume [7][31] Market Data and Key Metrics Changes - The number of unique patients receiving Debut shipments was 954, marking an all-time high for the brand [7][11] - NUPLAZID's gross price increased by over 2% year-over-year, with a gross-to-net adjustment of 24.1% for the quarter [31] Company Strategy and Development Direction - Acadia is focused on accelerating the commercial trajectory of Debut and capitalizing on the momentum of NUPLAZID while expanding its global presence [35] - The company plans to host its first R&D Day to provide insights into its development strategy and pipeline innovations [9][35] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the sustainability of Debut's growth and reiterated full-year sales guidance for both Debut and NUPLAZID [7][35] - The timeline for the COMPASS PWS Phase III study results is now expected by the end of 2025, indicating progress in pipeline development [9][35] Other Important Information - Acadia anticipates EMA approval for Debut in Q1 2026, with a substantial market opportunity in Europe for Rett syndrome patients [14][28] - The company has received orphan drug designation for trofinetide in Japan, which may lead to priority review [28] Q&A Session Summary Question: What does good 101 data look like and how does it relate to FDA discussions? - Management indicated that statistically significant results demonstrating a clinically meaningful impact on hyperphagia would be considered good data [40] Question: How should we model a European price for Debut? - Management expressed confidence in obtaining a strong EU price that reflects the value provided to patients in the Rett community [41] Question: What are the key growth drivers for NUPLAZID and Debut from Q1 to Q2? - Management highlighted patient demand and the impact of expanded customer models as key growth drivers for both brands [44][46] Question: What are the biggest risks to the approval process for trofinetide in the EU? - Management stated confidence in the EMA approval process, noting that they are on track with the regulatory timeline [80] Question: How does Acadia view its positioning against Celeno's product for Prader Willi? - Management believes there is room for multiple therapies in the Prader Willi space, emphasizing the complexity of patient needs [95]

Financial Data and Key Metrics Changes - Curis reported a net loss of $10.6 million or $1.25 per share for Q1 2025, compared to a net loss of $11.9 million or $2.05 per share for the same period in 2024, indicating an improvement in loss per share [12] - Research and development expenses decreased to $8.5 million in Q1 2025 from $9.6 million in Q1 2024, primarily due to lower employee-related costs [12] - General and administrative expenses also decreased to $4 million in Q1 2025 from $4.9 million in Q1 2024, attributed to lower employee-related costs and professional fees [12] - Cash and cash equivalents totaled $20.3 million as of March 31, 2025, with the company expecting this to support operations into Q4 2025 [13] Business Line Data and Key Metrics Changes - The Take Aim Lymphoma study is progressing, evaluating emavucertib in combination with ibrutinib in patients with relapsed/refractory primary CNS lymphoma [7] - As of January 2, 2025, 27 patients have been treated with the emavucertib and ibrutinib combination, showing promising results in tumor burden reduction [8] - In AML, a 38% composite complete response rate was observed in a study of 21 patients with FLT3 mutations treated with emavucertib, which is significantly higher than the 21% rate for gilteritinib [10] Market Data and Key Metrics Changes - The company is focusing on expanding the use of emavucertib beyond primary CNS lymphoma into additional indications such as NHL, AML, and solid tumors [5] - The enrollment for the lymphoma study is ongoing, with 37 sites currently open across the U.S., Europe, and Israel [20] Company Strategy and Development Direction - Curis is prioritizing the PCNSL study while also advancing its AML studies, with a focus on safety and tolerability in the frontline setting [18] - The company aims to enroll 30 to 40 additional patients for NDA submission in the lymphoma study, targeting six to eight responses in that dataset [9] - The addition of Dr. Ahmad Hamdi to the executive team is expected to enhance the company's strategic direction in expanding treatment options [4] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the collaboration with the FDA, indicating no concerns about changes affecting their agreement [28] - The company is optimistic about the potential of emavucertib in combination therapies and is focused on maximizing its utility across various indications [36] Other Important Information - Curis completed a registered direct financing and private placement in March 2025, raising approximately $8.8 million [13] - The company plans to provide additional updates on the Take Aim Lymphoma study at the ASH conference later this year [9] Q&A Session Summary Question: Positioning of lymphoma versus AML - Management is advancing both lymphoma and AML studies simultaneously, with more resources allocated to the PCNSL study due to its advanced stage [18] Question: Enrollment in lymphoma study and FDA conversations - Enrollment is on track with 37 sites open, and management is confident in their collaboration with the FDA [25][28] Question: Mutations affecting responses and AML development steps - It is too early to discuss specific mutations affecting responses; the focus is on the mechanism of action [33] - Future plans for AML development will be discussed after completing the ongoing triplet study [35]

Financial Data and Key Metrics Changes - Neurocrine Biosciences reported $545 million in product sales for INGREZZA in Q1 2025, impacted by one less order week and patient reauthorization processes [9] - The company reaffirmed its 2025 sales guidance range for INGREZZA at $2.5 billion to $2.6 billion, factoring in expected acceleration of new patient additions [10][11] - The company has approximately $1.8 billion in cash to support its commercial and clinical development strategies [12] Business Line Data and Key Metrics Changes - INGREZZA achieved record new patient starts in Q1 2025, indicating strong demand despite market challenges [10][14] - KRONESITY generated $15 million in net revenue in its first full quarter of launch, with 70% of dispensers receiving reimbursement [11][19] - The company expanded formulary coverage in Medicare Part D, increasing patient access for INGREZZA [10][16] Market Data and Key Metrics Changes - The payer environment has been influenced by the Inflation Reduction Act, affecting prior authorization processes for both new and continuing patients [16] - Approximately 40% of patients with tardive dyskinesia (TD) have been diagnosed, with less than 10% receiving standard care treatment [15] Company Strategy and Development Direction - The company aims to evolve from a single blockbuster to a multiple blockbuster neuroscience company, focusing on both near and long-term revenue growth [6] - Neurocrine is advancing its R&D portfolio, with ongoing Phase III studies for osevampatore and MBI-568, and plans to expand its muscarinic portfolio into new Phase II studies [7][23] - The leadership transition includes welcoming Dr. Sanjay Keswani as the new Chief Medical Officer [7][27] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the momentum from record new patient starts and the potential for growth in Q2 and beyond [35][36] - The company is optimistic about the early adoption of KRONESITY and expects continued growth in the pediatric and adolescent segments [19][70] Other Important Information - The company has retired 3.6 million shares over the past two quarters, reflecting a commitment to returning capital to shareholders [12] - The transition of Dr. Eiry Roberts to a strategic advisory role marks a significant change in leadership, with a focus on maintaining the momentum in the company's pipeline [27] Q&A Session Summary Question: What are the expectations for INGREZZA in Q2? - Management noted that Q1 challenges were anticipated, but record new patient starts provide confidence for Q2 growth [34][35] Question: How should KRONESITY's initial numbers be interpreted? - Management indicated that it is too early to define a trend for KRONESITY, but initial adoption has exceeded expectations [37][38] Question: What are the reimbursement dynamics for KRONESITY moving forward? - The company expects some health plans to conduct formal reviews of KRONESITY, but initial reimbursement metrics are favorable [41][44] Question: Can you provide insights on the share split between INGREZZA and Teva? - Management stated that INGREZZA continues to hold the majority share in TD prescriptions, with trends aligning with expectations [48] Question: What is the expected growth for INGREZZA in Q2? - Management anticipates a nice step up in sales due to one additional order week and record new patient starts, despite some gross to net impacts [53][54] Question: What is the timeline for KRONESITY data from ongoing studies? - Management plans to release one-year data on KRONESITY at upcoming medical meetings, with a focus on various clinical endpoints [99]

Financial Data and Key Metrics Changes - The company reported quarterly revenue of $145.5 million, with $134 million coming from Orlodayo, marking a significant increase from previous quarters [6][27] - Annual revenue guidance for Orlodayo has been raised to between $580 million and $600 million, reflecting a growth of 33% to 37% over the previous year [7][28] - Operating profit for Q1 2025 was $21.2 million, with net income slightly positive, and cash at the end of the quarter was $317 million [28][30] Business Line Data and Key Metrics Changes - The U.S. commercial team has successfully transitioned patients from free drug to paid status, achieving an 84% paid rate among commercial insurance patients, up from 73.5% [12][27] - The paid patient rate increased by 10 percentage points in Q1, with two-thirds of this improvement attributed to the Inflation Reduction Act [11][57] - The gross to net ratio improved, with expectations to maintain an average of around 15% for the full year [14][28] Market Data and Key Metrics Changes - The demand for Orlodayo has remained strong, with new prescriptions slightly exceeding the best quarter from the previous year [9][62] - The company anticipates capturing more revenue opportunities as the patient base continues to grow, aiming for $1 billion in global revenue by 2029 [13][62] Company Strategy and Development Direction - The company is focused on advancing its pipeline and has submitted a pediatric NDA for Orlodayo, aiming to introduce an oral granule formulation for patients aged 2 to 11 [16][30] - The company is also progressing with clinical trials for new treatments targeting Netherton syndrome and diabetic macular edema, with initial data expected by the end of the year [17][26] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's financial strength and ability to achieve profitability this year, one year earlier than previously planned [7][30] - The management highlighted the importance of sustainable revenue growth and the ability to pay down debt while investing in the pipeline [8][30] Other Important Information - The company paid down $75 million of its debt in April, reducing outstanding debt to $249 million, which is expected to save approximately $23 million in interest payments over the life of the debt [28][30] - The company is well-positioned in a competitive market, with a focus on providing effective treatments for underserved patient populations [30] Q&A Session Summary Question: What is the potential for pediatric patients on TAKHZYRO? - Management estimates around 500 pediatric patients on TAKHZYRO in the U.S., with at least 200 potentially appropriate for prophylactic therapy [34][35] Question: What factors contributed to the increase in paid patients? - The increase in paid patients was primarily due to improved Medicare coverage and better execution in the commercial segment [46][57] Question: What benchmarks are being looked for in the DME program? - The company is looking for a clear reduction in retinal edema and thickness as a benchmark for moving forward with the DME program [92][93] Question: How does the company plan to handle the pediatric launch? - The company aims to minimize the lag in deployment upon approval and is preparing to promote the pediatric formulation immediately [100][101] Question: What is the expected dosing range for the new treatment? - The initial dosing for the new treatment will be around six milligrams, with adjustments based on patient response [105][106]