Financial Data and Key Metrics Changes - Scholar Rock reported a strong start to 2025, with significant progress in bringing up epitigromab for SMA patients globally [6][9] - The company ended the quarter with $364.4 million in cash, and has an additional $100 million available under its debt facility to support the upcoming launch [25][26] Business Line Data and Key Metrics Changes - The phase three SAPPHIRE trial for epitigromab showed a statistically significant improvement in motor function, indicating its potential to reverse SMA progression [7][13] - The company is preparing for a U.S. commercial launch of epitigromab anticipated in Q3 2025, with plans to expand into Europe, Asia Pacific, and Latin America [9][20] Market Data and Key Metrics Changes - Approximately 10,000 SMA patients in the U.S. and around 35,000 globally have received SMN-targeted therapies, highlighting the market potential for epitigromab [21][22] - Market research indicates that 90% of SMA patients are seeking new treatment options that improve muscle strength, emphasizing the demand for epitigromab [20] Company Strategy and Development Direction - Scholar Rock aims to establish itself as a fully integrated global biopharmaceutical company, focusing on the launch of epitigromab and expanding its pipeline into other neuromuscular disorders [9][28] - The company is also exploring the EMBRAZE study to understand its role in treating obesity, indicating a diversification of its research focus [10][11] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the upcoming U.S. launch and the potential for sustainable growth driven by epitigromab and its pipeline [9][28] - The management team is committed to ensuring that no patient with SMA is left behind, reflecting a strong patient-centric approach [29] Other Important Information - The BLA for epitigromab was granted priority review by the FDA, with a PDUFA date set for September 22, 2025 [8][15] - The company is actively engaging with U.S. payers and preparing for reimbursement discussions in Europe, indicating proactive market access strategies [30][35] Q&A Session Summary Question: How receptive are U.S. payers to coverage of combination therapy for SMA patients? - Management indicated that early discussions with U.S. payers have been positive, with a focus on the need for better therapeutic options for SMA patients [32][34] Question: Can you comment on interactions with the FDA regarding the upadacumab review? - Management reported that interactions with the FDA have been routine and constructive, with no issues anticipated for the September 22 PDUFA date [38][39] Question: What are the expectations for the EMBRAZE study data in June? - Management confirmed that the primary focus will be on the 24-week data, with additional follow-up information provided as necessary [42][43] Question: How are you evaluating the potential to secure a commercial partner for Europe? - Management stated that they do not prioritize finding a partner outside the U.S., as they feel well-equipped to serve patients directly [88][89] Question: What are the launch dynamics and early demand expectations for epitigromab? - Management expressed optimism about the launch, citing the 100% newborn screening for SMA in the U.S. and the concentration of patients in centers of excellence [69][70]

Financial Data and Key Metrics Changes - Scholar Rock reported a strong start to 2025, with significant progress in bringing up epitigromab for SMA patients globally [6][9] - The company ended the quarter with $364.4 million in cash, indicating a solid financial position to support upcoming initiatives [25] Business Line Data and Key Metrics Changes - The phase three SAPPHIRE trial for epitigromab showed statistically significant improvement in motor function, with patients treated having a threefold higher chance of a three-point or greater increase in Hammersmith Scale compared to placebo [7][13] - The company is preparing for a U.S. commercial launch of epitigromab anticipated in Q3 2025, with plans to expand into Europe, Asia Pacific, and Latin America [9][22] Market Data and Key Metrics Changes - Approximately 10,000 SMA patients in the U.S. and around 35,000 globally have received SMN-targeted therapies, highlighting the market potential for epitigromab [21] - The company is focusing on the dual modality treatment approach for SMA, combining SMN-targeted therapies with muscle-targeting therapies [20] Company Strategy and Development Direction - Scholar Rock aims to establish itself as a fully integrated global biopharmaceutical company, with a focus on SMA and potential expansion into other neuromuscular disorders [9][27] - The company is also exploring the EMBRAZE study to understand its role in treating obesity, indicating a diversification of its research focus [10][11] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the upcoming U.S. launch of epitigromab and the potential for sustainable growth through the end of the decade [9][27] - The company is committed to ensuring that no patient with SMA is left behind, emphasizing its responsibility to patients and families [28] Other Important Information - The BLA for epitigromab was granted priority review by the FDA, with a PDUFA date set for September 22, 2025 [8][15] - The company has an additional $100 million under its debt facility to support the upcoming launch, extending its financial runway into 2027 [25][26] Q&A Session Summary Question: How have discussions with U.S. payers gone regarding coverage for combination therapy? - Management indicated that early discussions with payers have been positive, with a focus on the need for better therapeutic options for SMA patients [31][34] Question: Can you provide an update on interactions with the FDA regarding the upadacumab review? - Management reported that interactions with the FDA have been routine and constructive, with no issues anticipated for the September 22 PDUFA date [36][37] Question: What are the expectations for the EMBRAZE study data in June? - Management confirmed that the primary focus will be on the 24-week data, with additional follow-up information provided as necessary [41][42] Question: How do you evaluate the potential to secure a commercial partner for Europe? - Management stated that they do not prioritize seeking a partner outside the U.S., feeling confident in their ability to serve patients directly [86] Question: What are the expectations for launch dynamics and early demand post-approval? - Management noted that while they will not provide specific guidance, they are optimistic due to the existing infrastructure and patient population [68][70]

Financial Data and Key Metrics Changes - The company ended the quarter with $364.4 million in cash [25] - The company has an additional $100 million under its debt facility that can be drawn down this year to support the upcoming launch, extending its anticipated runway into 2027 [26] Business Line Data and Key Metrics Changes - The company is focused on the commercial launch of epitigromab for SMA, with a PDUFA date set for September 22, 2025 [6][13] - The company is preparing for a global launch, starting in the US in Q3 2025, and is also evaluating expanding the study of upitigramab into other rare neuromuscular disorders [7][8] Market Data and Key Metrics Changes - Approximately 10,000 patients with SMA are in the US, with about two-thirds having received SMN-targeted therapies [20] - Globally, around 35,000 patients have received SMN-targeted therapies, indicating a significant market opportunity for epitigromab [20] Company Strategy and Development Direction - The company aims to establish itself as a fully integrated global biopharmaceutical company, focusing on SMA and exploring additional indications for its therapies [7][14] - The company is committed to ensuring that any patient with SMA who can benefit from epitigromab has access to it, indicating a patient-centric approach [7] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the upcoming launch and the potential of epitigromab to transform the treatment landscape for SMA [19] - The company is aware of the competitive landscape and the need for dual modalities to address both motor neuron and muscle degeneration in SMA patients [20] Other Important Information - The EMBRAZE proof of concept study is ongoing, focusing on the role of the company in treating obesity and preserving lean mass [9][10] - The company is on track to share top-line results from the EMBRAZE study in June 2025 [10] Q&A Session Summary Question: How have discussions with U.S. payers gone regarding coverage for combination therapy? - Management indicated that early discussions with payers have been positive, and they expect that the budget impact for any single payer will be limited due to the rarity of the disease [31][34] Question: Can you comment on interactions with the FDA regarding the upadacumab review? - Management reported that interactions with the FDA have been routine and constructive, with no issues anticipated for the September 22 PDUFA date [38] Question: What are the expectations for the EMBRAZE study data in June? - Management confirmed that they will provide the 24-week data as the main study outcome and will include any necessary follow-up information [43] Question: How quickly will the company be able to launch after the PDUFA date? - The company is prepared to launch the day after the PDUFA date, with ample supply to meet demand [60] Question: How is the company evaluating the potential to secure a commercial partner for Europe? - Management stated that they do not prioritize finding a partner outside the U.S. and feel well-equipped to serve patients directly [86]