Financial Data and Key Metrics Changes - Total revenues for Q3 2025 were $120.7 million, a significant increase from $2.7 million in the same period last year, primarily driven by $108.1 million in net product revenue from Atrobi [25][26]. - Total operating expenses increased to $259.3 million from $193.9 million year-over-year, with a notable rise in SG&A expenses by $68.8 million [26][27]. - The company ended Q3 with a strong cash position of $645.9 million, providing a solid cash runway for future operations [27]. Business Line Data and Key Metrics Changes - Atrobi generated $108.1 million in net product sales, with 5,259 unique patient prescriptions delivered to 1,355 unique healthcare providers [11][25]. - The company reported strong growth in the ATTR-CM market, with an increase in prescribing from both returning and new physicians [18][21]. Market Data and Key Metrics Changes - The ATTR-CM market continues to expand, with no signs of slowing down, contributing to a sustained growth runway for Atrobi [21]. - The company is seeing increased diagnosis rates for ATTR-CM, with expectations to reach closer to 250,000 diagnosed patients in the U.S. [39]. Company Strategy and Development Direction - The company aims for a market share of 30+% by volume in the coming years, focusing on the continued success of Atrobi and the upcoming launches of Encalirate and BBP-418 [6][11]. - The company is actively building infrastructure for global commercialization to ensure access to its therapies worldwide [24]. Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the ongoing success of Atrobi and the potential for strong market share growth, supported by positive clinical data from recent trials [6][11]. - The company is optimistic about the upcoming readouts for Infogratinib and the continued expansion of its pipeline [22][24]. Other Important Information - The company has a robust late-stage pipeline, with significant progress in R&D, including two recent positive phase 3 trial results [4][6]. - The company is focused on leveraging real-world evidence to support its market position and drive adoption of its therapies [16][78]. Q&A Session Summary Question: What is the percentage of new patient share for Atrobi? - Management estimates that the naive share is well in the 20s, with double-digit growth in overall prescriptions quarter-on-quarter [30][31]. Question: Can you elaborate on ATTR-CM diagnosis rates? - There has been robust growth in diagnosis rates, with ongoing education and excitement in the medical community about ATTR cardiomyopathy [39]. Question: Thoughts on Pfizer's 28-day free trial program? - Management views it positively as it encourages competition and emphasizes the importance of access for patients [44][46]. Question: How does the ex-US opportunity compare to the US market? - The ex-US opportunity is promising, with Bayer successfully commercializing in Europe, although pricing dynamics differ [50][52]. Question: Will there be a head-to-head study against Tafamidis? - Management believes that real-world evidence will be more impactful than a costly head-to-head study, as they already demonstrate superior efficacy [72][78]. Question: What differentiates Infogratinib in achondroplasia? - Infogratinib is expected to be more efficacious, safer, and more convenient due to its oral administration, addressing unmet needs in the market [83][86].

Financial Data and Key Metrics Changes - Total revenues for Q3 2025 were $120.7 million, consisting of $108.1 million from Attruby net product revenue, $4.3 million in royalty revenue, and $8.3 million in license and services revenue, compared to $2.7 million in the same period last year, indicating a significant increase in total revenues driven primarily by Attruby sales [25][26][27] - Operating expenses for Q3 2025 were $259.3 million, up from $193.9 million in the same period last year, with a notable increase in SG&A expenses [26][27] Business Line Data and Key Metrics Changes - Attruby generated $108.1 million in net product sales in Q3 2025, with 5,259 unique patient prescriptions delivered to 1,355 unique healthcare providers, reflecting strong commercial execution [11][25] - The company aims for a market share of 30-35% by volume in the ATTR-CM market, with continued momentum in both scientific and commercial aspects of the Attruby franchise [6][11] Market Data and Key Metrics Changes - The ATTR-CM market continues to expand, with growth coming from all segments, and an increase in prescribing from both returning and new physicians [18][20] - The company has seen a steady rise in first-time prescribers adopting Attruby, indicating growing prescriber confidence and sustained demand [20][21] Company Strategy and Development Direction - The company is focused on launching new products from its late-stage pipeline, including Encalirate and BBP-418, which have shown promising phase III results [4][22] - The strategy includes leveraging the experience gained from the Attruby launch to inform future product launches and expanding disease state education initiatives [22][23] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving a market share of over 30% for Attruby, supported by strong clinical data and ongoing market expansion [6][11] - The company anticipates continued growth in diagnosis rates for ATTR-CM, with expectations to reach approximately 250,000 diagnosed patients in the U.S. [39] Other Important Information - The company ended Q3 2025 with a strong cash position of $645.9 million, providing significant cash runway for future investments [27] - The company is actively building infrastructure for global commercialization to ensure access to its therapies worldwide [24] Q&A Session Summary Question: What is the percentage of new patient share for Attruby? - Management estimates that the naive share is well in the 20s, with double-digit growth in overall prescriptions quarter-on-quarter [30][31] Question: Can you elaborate on ATTR-CM diagnosis rates? - There has been robust growth in diagnosis rates, with ongoing excitement and education in the field, leading to increased identification of new patients [39] Question: Thoughts on Pfizer's 28-day free trial program? - Management views it positively as it encourages competition and emphasizes the importance of access programs [44][46] Question: How does the ex-US opportunity compare to the US? - The ex-US opportunity is promising, with Bayer successfully commercializing in Europe, although pricing dynamics differ [50][52] Question: Discussions with payers regarding formulary positioning of Attruby? - Management has not had discussions about formulary positioning with payers but remains focused on ensuring Attruby's availability [54][56] Question: What is driving the growth in unique prescribers and prescriptions per prescriber? - Growth is driven by both increased penetration within existing accounts and expansion into new centers, with a focus on educating physicians [58][60] Question: What are the differentiating elements for Infigratinib in achondroplasia? - Infigratinib is expected to be more efficacious, safer, and more convenient due to its oral route of administration [84][86]

Financial Data and Key Metrics Changes - Total revenues for Q3 2025 were $120.7 million, a significant increase from $2.7 million in the same period last year, primarily driven by $108.1 million in net product revenue from Atrobi [26][27] - Total operating expenses increased to $259.3 million from $193.9 million year-over-year, with a notable rise in SG&A expenses by $68.8 million [26][27] - The company ended the quarter with a strong cash position of $645.9 million, providing a solid cash runway for future operations [27] Business Line Data and Key Metrics Changes - Atrobi generated $108.1 million in net product sales, with 5,259 unique patient prescriptions delivered to 1,355 unique healthcare providers [11][26] - The company aims for a market share of 30% to 35% by volume in the ATTR-CM market, with continued growth expected [11][19] Market Data and Key Metrics Changes - The ATTR-CM market is expanding, with growth coming from all segments, and an increase in prescribing from both returning and new physicians [19][21] - The company has seen a steady rise in first-time prescribers adopting Atrobi, indicating growing confidence among healthcare providers [19][21] Company Strategy and Development Direction - The company is focused on expanding its late-stage pipeline, with recent positive phase 3 readouts for BBP-418 and Encalirate, which are expected to redefine care for patients [23][25] - The strategy includes leveraging the success of Atrobi to inform future product launches and expanding disease state education initiatives [24][25] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the ongoing success of Atrobi and the potential for significant market share growth, supported by strong clinical data and a competitive pricing strategy [11][19] - The company anticipates continued growth in diagnosis rates for ATTR-CM, which will further expand the patient population eligible for Atrobi [36] Other Important Information - The company is actively building infrastructure for global commercialization to ensure access to its therapies worldwide [25] - Management highlighted the importance of real-world evidence in demonstrating the efficacy of Atrobi compared to competitors [70] Q&A Session Summary Question: What is the percentage of new patient share for Atrobi? - Management estimates that the naive share is now well in the 20s, with double-digit growth in overall prescriptions quarter-on-quarter [31] Question: Can you elaborate on ATTR-CM diagnosis rates? - There has been robust growth in diagnosis rates, with ongoing excitement and education in the field, leading to an expected increase in diagnosed patients [36] Question: Thoughts on Pfizer's 28-day free trial program? - Management views it positively as it encourages competition and emphasizes the importance of access for patients [41][42] Question: How do you see the ex-US opportunity relative to the US? - The ex-US opportunity is promising, with Bayer successfully commercializing in Europe, and management expects similar sales ratios between Europe and the US [48] Question: Have you discussed formulary positioning of Atrobi with payers? - Management has focused on ensuring Atrobi is on an equal playing field with competitors and has not yet had discussions regarding formulary positioning [49][50] Question: What is driving the momentum in unique prescribers and prescriptions per prescriber? - The momentum is driven by both greater penetration within existing accounts and expansion into new centers, with a focus on educating physicians [52] Question: What differentiates Infogratinib in achondroplasia? - Infogratinib is expected to be more efficacious, safer, and more convenient due to its oral administration, addressing unmet needs in the market [73][75] Question: What are the key assumptions for the $2 billion opportunity in achondroplasia and hypochondroplasia? - The estimates are based on capturing share from competitors and growing the market, addressing substantial unmet needs not covered by existing therapies [78]