Scholar Rock(US:SRRK)2023-01-11 18:42Apitegromab (Spinal Muscular Atrophy - SMA) - The global SMA treatment market is expected to reach $11.4 billion by 2028 [5] - Apitegromab is a muscle-directed approach aimed at improving motor function in SMA patients, complementing SMN-directed therapies [41] - TOPAZ trial results showed transformative potential in non-ambulatory Types 2 and 3 SMA patients [9] - In a TOPAZ analysis of non-ambulatory Types 2/3 SMA patients aged 2-12 years (n=16) receiving apitegromab at 20 mg/kg, the mean HFMSE change from baseline was +4.4 points, with 81% (13/16) experiencing ≥ 1-point increase and 56% (9/16) experiencing ≥ 3-point increase in HFMSE [68] - Approximately 90% (51/57) of patients remained on apitegromab as of December 31, 2022, in the TOPAZ trial [193] - Scholar Rock is conducting a Phase 3 SAPPHIRE study with data readout expected in 2024, and anticipates a potential launch in 2025 [23, 199] SRK-181 (Immuno-Oncology) - SRK-181 targets TGFβ-1 to overcome CPI resistance, with a potential market of 7.9-10.41 million US patients on CPI therapies [80, 77] - In the DRAGON Part A trial, at 800 mg q3w, 1 partial response (PR) was observed in a patient with anti-PD-1-resistant clear cell renal cell carcinoma (ccRCC) [100] - DRAGON Part A demonstrated the ability to escalate to high doses of SRK-181, exceeding anticipated efficacious drug exposure levels [102] Pipeline and Financials - Scholar Rock has an anticipated cash runway into 2025 [24]