Scholar Rock Holding Corporation

For the second quarter, Scholar Rock reported $108.9 million in operating expenses, including $19.7 million of noncash stock-based compensation; excluding stock-based compensation, operating expenses were $89.2 million. The company ended the quarter with $492 million in cash, cash equivalents and marketable securities, and that balance included $63 million in net proceeds from its ATM program during the quarter. Management did not provide revenue or EPS figures on the call. Looking ahead, the company said apitegromab remains on track for a decision by the September 30 PDUFA date, with U.S. launch readiness in place and Europe still awaiting FDA inspection classification for Catalent Indiana; it also plans to pursue approval and launch preparations in Germany and to engage regulators later this year on the subcutaneous apitegromab program after approval.

David Hallal framed the quarter as a pivotal moment, saying the company is operating “from a position of strength” and is focused on bringing what he called the world’s first muscle-targeted therapy to SMA patients. He stressed urgency, discipline and the company’s “belt and suspenders” approach to the regulatory path, pointing to two fill/finish facilities and ongoing constructive dialogue with FDA and EMA. His tone was confident and patient-focused, repeatedly emphasizing the unmet need in SMA and the company’s readiness to launch quickly once approved.

Vikas Sinha highlighted disciplined spending and balance sheet support for the launch and pipeline. He said Q2 operating expenses were $108.9 million, or $89.2 million excluding $19.7 million of stock-based compensation, and noted those expenses were generally consistent with Q2 2025 despite increased commercial investment. He said cash, cash equivalents and marketable securities totaled $492 million at quarter-end, including $63 million of ATM proceeds, and added that after FDA approval the company would have the option to draw an additional $150 million from its debt facility and plans to monetize a priority review voucher.

Analysts focused heavily on whether the second fill/finish facility alone can support approval and whether any delay at Catalent Indiana could affect the PDUFA timeline. Management said the second site provides a fully independent path, the FDA already has the agreed data package, and they do not expect removing a facility from the application to affect timing. Questions also centered on payer behavior, especially prior authorization and step edits; Keith Woods said prior auth is expected, first patients will likely be early-access and ONYX patients, and the lack of a J-code could make the average time from prescription to infusion more than 60 days in the first six months.