Core Insights - The US FDA has approved a higher-dose version of Spinraza, which is expected to sustain Biogen's position in the spinal muscular atrophy (SMA) market, generating over $1.5 billion annually [1][2] Group 1: Product Approval and Market Impact - The high-dose Spinraza regimen includes larger loading doses and higher maintenance levels, potentially addressing the "waning-off effect" in patients and improving response durability [2] - The therapy's rollout in Japan is ahead of expectations, with some patients returning to Spinraza after trying alternatives, and it has also received approvals in Europe and Switzerland [3] - The approval of high-dose Spinraza is seen as a bridge to next-generation therapies, such as salanersen, which may reach late-stage data by 2028 [4] Group 2: Competitive Landscape - Competition in the SMA market is expected to increase, but Spinraza's position remains strong, with data showing better motor function improvement when used with Spinraza compared to Roche's Evrysdi [6] - Roche has discontinued its emugrobart program, indicating challenges for some competing therapies [6] Group 3: Broader Portfolio and Future Growth - Biogen's diverse portfolio, including therapies like Leqembi and Vumerity, is seen as a foundation for potential stock gains from 2026 to 2027, with each therapy having over $1 billion in blockbuster potential [7] - The company aims to have one to two pipeline products approved to drive topline revenue growth, with management actively pursuing assets valued up to $5 billion to $6 billion [8] Group 4: Pricing and Market Performance - While pricing for high-dose Spinraza is not disclosed, improved efficacy could justify a premium during the initial treatment phase [9] - Biogen's shares increased by 2% to $187 following the news [9]

Denali Therapeutics Conference Call Summary Company Overview - **Company**: Denali Therapeutics (NasdaqGS:DNLI) - **Product**: AVLAYAH (tividenofusp alfa-eknm) - **Indication**: Treatment of Hunter syndrome (MPS II) Key Points FDA Approval and Product Significance - Denali announced the FDA approval of AVLAYAH, the first enzyme replacement therapy designed to cross the blood-brain barrier for Hunter syndrome [2][3] - This approval is a historic milestone, marking the first meaningful therapeutic advance for the MPS II community in nearly two decades [7] - AVLAYAH is designed to address both systemic and neurological manifestations of Hunter syndrome, a progressive, life-limiting condition affecting children [6][12] Clinical Data and Efficacy - AVLAYAH demonstrated a significant 91% mean reduction in cerebrospinal fluid (CSF) heparan sulfate levels from baseline, with 93% of patients achieving levels below the upper limit of normal at week 24 [18] - The treatment also normalized urine GAG levels in 68% of patients by week 24, indicating effective clearance of peripheral substrate [18] - Clinical data published in the New England Journal of Medicine showed large and sustained reductions in biomarkers of disease activity in both the CNS and periphery [14][15] Commercialization Strategy - Denali is prepared for the commercial launch of AVLAYAH, with a focus on engaging the MPS II community and ensuring patient access [20][24] - The company aims to capture approximately 75% of the treated U.S. prevalent population, which consists of around 500 patients [22] - Denali's pricing strategy includes a wholesale acquisition cost of $5,200 per 150-mg vial, with annual costs for maintenance dosing ranging from approximately $270,000 to $811,000 depending on patient weight [26] Market Dynamics and Patient Engagement - The MPS II market is characterized by a concentrated prescriber base, with most patients managed by clinical geneticists in about 100 centers of excellence [24] - Denali has established relationships with major MPS II centers and has a sales team with extensive experience in rare diseases [25] - The company has engaged with payers covering approximately 90% of commercial lives, receiving constructive feedback regarding the unmet need for AVLAYAH [23] Future Outlook and Pipeline Expansion - Denali views AVLAYAH as the foundation for a durable multi-program franchise in lysosomal storage disorders, with plans to expand into other diseases such as Sanfilippo syndrome and Alzheimer's disease [32][34] - The approval of AVLAYAH sets a precedent for using CSF heparan sulfate as a surrogate biomarker for future regulatory submissions [56][97] Safety and Monitoring - Safety data indicated that infusion-related reactions were the most common adverse events, with manageable incidence over time [16] - The prescribing information includes guidance on monitoring for hypersensitivity and anemia, which are common concerns with enzyme replacement therapies [18] Conclusion - Denali Therapeutics is positioned for a successful launch of AVLAYAH, addressing a critical unmet need in the treatment of Hunter syndrome and paving the way for future innovations in rare diseases [27][34]

Core Insights - BioLineRx Ltd. is a biopharmaceutical company focused on developing treatments for oncology and rare diseases, currently in the clinical development stage [1] Financial Performance - For the earnings report on March 23, 2026, BioLineRx reported an earnings per share (EPS) of -0.38, matching the estimated EPS, while generating revenue of $514,000, which surpassed the estimated revenue of $492,000 [2][6] - The company has a negative price-to-earnings (P/E) ratio of approximately -3.24 and a negative earnings yield of about -30.83%, indicating current losses [4][6] - The price-to-sales ratio stands at 0.84, suggesting the stock is valued at less than its sales per share, which may attract growth-oriented investors [4][6] Clinical Development - BioLineRx is advancing its clinical pipeline with a Phase 1/2a clinical trial of GLIX1 for glioblastoma (GBM), set to begin by the end of March 2026, with patient enrollment expected shortly thereafter [3][6] Financial Health - The company has a debt-to-equity ratio of about 0.58, indicating a moderate level of debt, and a current ratio of approximately 2.09, suggesting a strong ability to cover short-term liabilities with short-term assets [5]

Financial Data and Key Metrics Changes - Total revenues grew by 15% in Q4 2025 and by 27% for the full year, reaching EUR 376.1 million [2][25] - Operating profit for 2025 was $26 million, a significant improvement from a loss in 2024 [2][28] - Operating cash flow was $55 million in 2025, with cash position at year-end above that of 2024 [3][28] - Adjusted operating profit was EUR 36.4 million, compared to a loss of EUR 8.6 million in 2024 [28] Business Line Data and Key Metrics Changes - RUCONEST revenue grew 26% year-over-year and 9% in Q4 2025, with U.S. volume growth of 20% [3][9] - Joenja revenue increased by 29% year-over-year and 53% in Q4 2025, reaching $19.8 million globally [4][12] - Joenja annual revenues exceeded EUR 50 million for the first time, triggering a EUR 5 million sales milestone payment [25] Market Data and Key Metrics Changes - Joenja's growth was fueled by new patient acceleration in the U.S. and increased demand in international markets, including the U.K. [4][14] - The number of identified APDS patients in the U.S. increased by 40 in 2025, more than double the increase in 2024 [14] Company Strategy and Development Direction - The company aims to maintain strong financial discipline while investing in growth drivers, focusing on becoming a leading rare disease company [6][8] - RUCONEST is positioned as a cornerstone treatment for severe HAE patients, while Joenja is expected to generate a significant portion of future revenues [32][34] - The company is exploring geographic expansion and regulatory approvals for Joenja, with a focus on pediatric indications [15][30] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in continued growth for RUCONEST and Joenja, with expectations for 2026 revenue between EUR 405 million and EUR 425 million [6][29] - The company anticipates operating expenses to increase at a slower pace, despite higher R&D investments [30] - Management highlighted the importance of upcoming clinical data readouts as potential value inflection points [32][34] Other Important Information - The company completed the enrollment of two leniolisib phase II trials and expects top-line data readout in the second half of 2026 [7][19] - The company is committed to optimizing capital allocations to support growth and shareholder value [8][30] Q&A Session Summary Question: Joenja growth expectations from U.S. vs. international markets - Management expects around 70%-75% of Joenja's growth in 2026 to come from the U.S. market [41][43] Question: RUCONEST patient return dynamics - Patients tend to return to RUCONEST quickly after trialing other treatments that do not control their crises effectively [37][38] Question: Timeline for getting patients on paid therapy in international markets - The company plans to submit pricing for reimbursement in Japan shortly, with launch timelines planned for summer [60][61] Question: Variants of uncertain significance (VUS) project timeline - New experiments are being planned, but timelines for results are not yet clear [76] Question: Inventory-related effects on RUCONEST sales - The company expects a higher inventory drawdown in Q1 due to market dynamics settling [78]

Financial Data and Key Metrics Changes - Total revenues grew by 15% in Q4 2025 and by 27% for the full year, reaching EUR 376.1 million [2][25] - Operating profit for 2025 was $26 million, a significant improvement from a loss in 2024 [2] - Operating cash flow was $55 million in 2025, with cash position at year-end above 2024 levels [3][28] - Adjusted operating profit was EUR 36.4 million, compared to a loss of EUR 8.6 million in 2024 [28] Business Line Data and Key Metrics Changes - RUCONEST revenue grew 26% year-over-year and 9% in Q4 2025, with U.S. volume growth of 20% [4][9] - Joenja revenue increased by 29% year-over-year and 53% in Q4 2025, reaching $19.8 million globally [12][25] - Joenja's annual revenues exceeded EUR 50 million for the first time, triggering a EUR 5 million sales milestone payment [25] Market Data and Key Metrics Changes - Joenja's growth was fueled by new patient acceleration in the U.S. and increased demand in international markets, including the U.K. [4][14] - The number of identified APDS patients in the U.S. increased by 40 in 2025, more than double the increase in 2024 [14] Company Strategy and Development Direction - The company aims to maintain strong financial discipline while optimizing capital allocations to growth drivers [6][8] - Pharming is transitioning from a single asset company to a high-growth biotech with two commercial products and a late-stage pipeline [4] - The company expects 2026 revenue between EUR 405 million and EUR 425 million, representing 8%-13% growth [6][29] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in continued growth for RUCONEST and accelerating growth for Joenja, particularly in the U.S. pediatric market [7][30] - The company is focused on addressing FDA feedback regarding the pediatric label expansion for Joenja [22] - Management highlighted the importance of upcoming clinical data readouts as potential value inflection points [33] Other Important Information - The company completed the enrollment of two leniolisib phase 2 trials for lower prevalence PIDs, with data readouts expected in the second half of 2026 [7][19] - The company is committed to exploring M&A opportunities that are value-accretive and complementary to its mission [67] Q&A Session Summary Question: Joenja growth expectations from U.S. vs. international markets - Management expects approximately 70%-75% of Joenja's growth in 2026 to come from the U.S. market [41][43] Question: Dynamics of RUCONEST and patient returns - Patients tend to return to RUCONEST quickly after trialing new treatments that do not adequately control their crises [38] Question: Timeline for getting patients on paid therapy in international markets - The company plans to submit pricing for reimbursement in Japan shortly, with launch timelines planned for summer [60] Question: Variants of uncertain significance (VUS) and their potential reclassification - New experiments are planned to generate data needed for reclassification of VUS, but timelines are still uncertain [76] Question: Inventory-related effects on RUCONEST sales - The company anticipates a higher inventory drawdown in Q1 due to market dynamics settling, which is a common quarterly fluctuation [78]

Financial Data and Key Metrics Changes - Total revenues grew by 15% in Q4 2025 and by 27% for the full year, reaching EUR 376.1 million [3][26] - Operating profit for 2025 was $26 million, a significant improvement from a loss in 2024 [3][29] - Operating cash flow was $55 million in 2025, with cash position at year-end above that of 2024 [4][29] - Adjusted operating profit was EUR 36.4 million, compared to a loss of EUR 8.6 million in 2024 [29] Business Line Data and Key Metrics Changes - RUCONEST revenue grew 26% year-over-year and 9% in Q4 2025, with U.S. volume growth of 20% [4][9] - Joenja experienced a 29% year-over-year growth and a 53% growth in Q4 2025, reaching $19.8 million globally [5][12] - Joenja's annual revenues exceeded EUR 50 million for the first time, triggering a EUR 5 million sales milestone payment [26] Market Data and Key Metrics Changes - Joenja's growth was fueled by new patient acceleration in the U.S. and increased demand in international markets, particularly in the U.K. [5][13] - The number of identified APDS patients in the U.S. increased significantly, reflecting successful educational efforts [13][16] Company Strategy and Development Direction - The company aims to maintain strong financial discipline while optimizing capital allocations to growth drivers [6][8] - Pharming is transitioning from a single asset company to a high-growth biotech with two commercial products and a late-stage pipeline [5][6] - The company is focused on expanding Joenja's market presence and preparing for pediatric approvals in the U.S. [7][15] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in continued growth for RUCONEST and Joenja, with expectations for 2026 revenue between EUR 405 million and EUR 425 million [6][30] - The company anticipates challenges in early 2026 due to inventory drawdowns but expects mid-single-digit growth for RUCONEST [30][31] - Management highlighted the importance of upcoming clinical data readouts as potential value inflection points [33] Other Important Information - The company completed the enrollment of two leniolisib phase 2 trials and expects data readouts in the second half of 2026 [7][19] - The company is actively pursuing regulatory approvals in multiple regions, including Europe and Japan, for its pipeline products [24][25] Q&A Session Summary Question: Joenja growth expectations from U.S. vs. international markets - Management expects approximately 70%-75% of Joenja's growth in 2026 to come from the U.S. market [42][43] Question: Dynamics of RUCONEST and patient returns - Patients tend to return to RUCONEST quickly after trying other treatments that do not adequately control their crises [38][50] Question: Access to paid therapies in international markets - The reimbursement process in international markets is slower initially but becomes efficient once negotiated [54] Question: Timeline for additional trials for Joenja - Management anticipates a phase 3 randomized study following the results of ongoing trials [56] Question: Variants of uncertain significance (VUS) and their potential - New experiments are planned to better understand VUS, but timelines for results are still uncertain [76]

Zevra Therapeutics Conference Call Summary Company Overview - **Company**: Zevra Therapeutics (NasdaqGS: ZVRA) - **Focus**: Commercial-stage organization specializing in rare diseases, particularly Niemann-Pick disease type C (NPC) and vascular Ehlers-Danlos syndrome Key Points MIPLYFFA Performance - **Launch Success**: MIPLYFFA, the only disease-modifying therapy for NPC, launched successfully with a robust commercial performance [5][12] - **Revenue Growth**: Total revenue reached $106.5 million, marking a 30% quarter-over-quarter growth [12] - **Patient Enrollment**: 24 enrollment forms were submitted in Q4, indicating strong demand [12] Patient Demographics and Diagnosis - **Prevalence and Diagnosis**: Approximately 900 patients in the U.S. are estimated to have NPC, with 300-350 diagnosed [10][18] - **New Diagnoses**: Efforts in genetic testing and disease awareness are leading to an increase in newly diagnosed patients [13][18] - **Patient Journey**: The organization is focused on understanding the unique journey of each patient to improve diagnosis rates [20][23] Clinical Profile and Treatment Impact - **Disease Modification**: MIPLYFFA shows the ability to halt disease progression within 12 weeks, with long-term data supporting efficacy over 4-6 years [11][12] - **Safety Profile**: The therapy has an acceptable safety profile, which is crucial for patient acceptance [11] Geographic Expansion - **International Opportunities**: Zevra is exploring geographic expansion beyond the U.S. and Europe, with plans to increase access to MIPLYFFA [6][39] - **European Market**: The company is seeing a pull for the product in Europe, with 92 patients added in Q3, representing about 10% of the diagnosed population [39] Celiprolol Development - **Ongoing Trials**: Celiprolol is currently enrolling patients for vascular Ehlers-Danlos syndrome, with a focus on accelerating clinical development [6][52] - **Market Potential**: The U.S. prevalence for this condition is approximately 7,500 patients, indicating a significant market opportunity [58] Financial Strategy - **Capital Allocation**: The company emphasizes disciplined capital allocation, allowing for independent operation without reliance on capital markets [54][57] - **Investment Focus**: Investments are being made in MIPLYFFA, Celiprolol, and European market expansion [57] Awareness and Education Efforts - **Healthcare Provider Engagement**: Zevra is actively educating healthcare providers about NPC and MIPLYFFA to improve diagnosis and treatment rates [15][30] - **Genetic Testing Initiatives**: Collaborations with organizations like GeneDx are aimed at facilitating earlier diagnosis through genetic testing [15][16] Future Outlook - **Long-term Vision**: The company aims to continue focusing on MIPLYFFA's growth while developing Celiprolol and expanding its market presence [52][57] - **Market Strategy**: A flexible go-to-market strategy is being developed for Europe and other international markets, considering local dynamics and patient needs [40][43] Additional Insights - **Patient Retention**: High persistency rates are observed, with most patients remaining on therapy unless they progress to a point where therapy is no longer needed [26][29] - **Market Share**: Zevra has captured approximately 40%-50% market share in its first full year of MIPLYFFA's launch [24] This summary encapsulates the key insights from the conference call, highlighting Zevra Therapeutics' strategic focus, market performance, and future growth opportunities in the rare disease sector.

Financial Performance - In Q4 2025, total net revenue was $34.1 million, including $26.4 million from MIPLYFFA sales, $400,000 from OLPRUVA, $5.6 million from global EAP reimbursements, and $1.8 million from royalties under the AZSTARYS license [22][23] - For the full year 2025, net revenue reached $106.5 million, with $87.4 million from MIPLYFFA sales, $800,000 from OLPRUVA, $13 million from global EAP reimbursements, and $5 million from royalties [24] - Operating expenses for Q4 were $23 million, a decrease of $1.5 million year-over-year, while full-year operating expenses were $90.4 million, down $6.6 million from 2024 [22][24] - Net income for Q4 was $12.2 million, compared to a net loss of $35.7 million in Q4 2024, and full-year net income was $83.2 million, compared to a net loss of $105.5 million in 2024 [23][24] Business Line Performance - MIPLYFFA generated $87.4 million in revenue for the full year 2025, with strong commercial performance reflected in 52 prescription enrollment forms received in the first year of commercialization [5][6] - In Q4, 24 new prescription enrollment forms were received, contributing to a total of 161 enrollments since launch [6][14] - The company is expanding access to MIPLYFFA through a global expanded access program, with 113 patients enrolled by the end of 2025 [8] Market Data - The estimated U.S. prevalence of Niemann-Pick disease type C (NPC) is approximately 900 individuals, with 300-350 currently diagnosed [7] - In Europe, NPC prevalence is estimated at around 1,100 individuals, with the current EAP supporting nearly 10% of this population [9] - Diagnosis rates in Europe are higher due to the approval of miglustat, leading to stronger physician awareness and higher patient identification [10] Company Strategy and Industry Competition - The company aims to redefine therapies for rare diseases, focusing on patient-centricity, integrity, accountability, innovation, and courage [5] - MIPLYFFA has exclusivity in the U.S. through 2031 under the Orphan Drug Designation, and the company is pursuing a patent term extension [7] - The company is also advancing celiprolol for the treatment of vascular Ehlers-Danlos syndrome, with ongoing Phase III studies [10][11] Management Commentary on Operating Environment and Future Outlook - Management expressed confidence in the growth potential of MIPLYFFA, driven by both previously diagnosed and newly diagnosed patients [15][16] - The company is focused on expanding access and strengthening relationships with key opinion leaders to maximize commercial impact [10] - Management highlighted the importance of ongoing education and awareness campaigns to facilitate early diagnosis and treatment [16][18] Other Important Information - The company moved its headquarters to Boston to enhance collaboration and access to specialized talent [12][13] - The new CFO, Justin Renz, officially started on the call date and will support the company's long-term value creation [4][28] Q&A Session Summary Question: Can you provide details on the split of new diagnoses versus previously identified patients? - Management noted that there has been meaningful growth in newly diagnosed patients, attributed to clinical data strength and disease awareness campaigns [33][34] Question: What is the expected timeline for diagnosis confirmation? - Management indicated that the timeline for diagnosis can vary significantly due to the nature of rare diseases, but efforts are being made to streamline the process [37][40] Question: Can you discuss persistency rates for patients on therapy? - Management stated that it is too early to provide meaningful data on persistency but expressed encouragement from refill rates observed [45][46] Question: How is the initial experience with the Uniphar collaboration? - Management expressed satisfaction with the collaboration, noting that it has facilitated rapid product delivery to patients [47][48] Question: What is the expected growth of EAP revenue? - Management clarified that EAP revenue is based on individual patient shipments and will have variability quarter-over-quarter [54][55] Question: How is the gross to net evolving in the U.S.? - Management noted that gross to net has evolved and is dynamic, making it difficult to provide specific guidance [58] Question: What is the average age of patients on MIPLYFFA? - Management indicated that approximately half of MIPLYFFA patients are adults, reflecting the patient mix seen in the expanded access program [64][66] Question: What are the next steps for the EMA application? - Management confirmed readiness to respond to the EMA's questions and emphasized the robustness of the data package submitted [69]

Core Insights - The treatment landscape for achondroplasia (ACH) is undergoing significant transformation with the introduction of new therapies challenging the existing market leader, Vosoritide [1][4][10] Group 1: Current Market Dynamics - Vosoritide, developed by BioMarin, was the first approved drug for ACH, projected to generate sales of $927 million by 2025, reflecting a 26% year-over-year growth [1][3] - The drug's mechanism involves a C-type natriuretic peptide analog that promotes growth by inhibiting FGFR3 activity, leading to an average height increase of 1.57 cm per year in clinical trials [3][4] - Despite its success, Vosoritide faces competition from several emerging therapies, including Ascendis' TransCon-CNP and BridgeBio's Infigratinib, which are designed to improve efficacy and patient compliance [1][4][10] Group 2: Emerging Competitors - Infigratinib, a small molecule drug from BridgeBio, targets FGFR3 directly and has shown superior clinical results, with an average height increase of 2.1 cm per year, and a more convenient oral administration route [4][7] - The drug's clinical trials reported no serious adverse effects, enhancing its appeal compared to Vosoritide, which requires daily injections [7][9] - Ascendis' TransCon-CNP aims for weekly dosing and has demonstrated an annual height increase of 2.29 cm, indicating a potential edge over Vosoritide [10][11] Group 3: Future Developments - The competitive landscape is intensifying with multiple next-generation therapies in development, including high-selectivity FGFR inhibitors and nucleic acid-based treatments [11][12] - The market is expected to evolve rapidly, with each incremental improvement in efficacy and administration method potentially reshaping the treatment paradigm for ACH [12][13] - The industry is witnessing a shift from overlooked rare diseases to a focus on developing targeted therapies, highlighting the growing recognition of the ACH market's potential [12][13]

Financial Data and Key Metrics Changes - BioCryst Pharmaceuticals reported full-year ORLADEYO revenue of $601.8 million, representing a 38% increase year-on-year and a 43% increase when excluding the European business sold in October [5][20] - Non-GAAP total revenue increased by 45% year-on-year, with non-GAAP ORLADEYO revenues rising by approximately $169 million or 43% year-on-year [20][21] - Non-GAAP operating profit surged to $214 million, marking a 198% increase year-on-year, the highest in the company's history [21][24] Business Line Data and Key Metrics Changes - The HAE portfolio saw significant growth, with ORLADEYO capsules for ages 12 and up and ORLADEYO Pellets for younger children being key products [7][9] - The pivotal trial for navenibart is progressing well, with a mean attack rate reduction of 92% for every three months dosing and 90% for every six months dosing reported [14][15] Market Data and Key Metrics Changes - The company anticipates full-year 2026 ORLADEYO revenues to be between $625 million and $645 million, representing approximately 13% growth over 2025 revenues adjusted for Europe [24][25] - The pediatric market for ORLADEYO is expected to grow significantly, with the potential to double the number of diagnosed children and treatment rates [77] Company Strategy and Development Direction - BioCryst aims to remain a profitable rare disease company focused on commercialization, innovation, and disciplined clinical development [5][6] - The company is committed to capital allocation that drives long-term value creation and plans to terminate programs lacking a compelling path forward [22][24] - The strategy includes maintaining a strong focus on the HAE market, with a portfolio of differentiated products to meet diverse patient needs [6][11] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's ability to achieve blockbuster potential for ORLADEYO and highlighted the importance of patient growth and market dynamics [25][26] - The company is optimistic about the launch of ORLADEYO Pellets for children and the ongoing development of navenibart and BCX-17725 [12][89] Other Important Information - The company ended the year with a strong liquidity position of $337.5 million in cash and investments [24] - BioCryst entered into a $400 million financing facility with Blackstone Life Sciences to support growth initiatives [24] Q&A Session Summary Question: Timing for navenibart regulatory submission - Management confirmed that everything is on track for filing by the end of next year, with approval expected by late 2028 [31][33] Question: Predicting super responders for ORLADEYO - There is no way to predict super responders; patients must try the medication to determine their response [37][39] Question: Impact of new entrants on ORLADEYO - Management noted that new entrants are not expected to impact ORLADEYO negatively, as patients prefer oral prophylaxis over injectables [70][71] Question: ORLADEYO price increase and growth levers - A 9% price increase was implemented, with a focus on net patient growth as the primary KPI for achieving long-term revenue goals [75][76] Question: Netherton syndrome trial data release - Management plans to release data from both parts 3 and 4 of the Netherton syndrome trial together [57][58]