On January 11, 2021 Scholar Rock (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, reported a corporate update and highlighted priorities for 2021 (Press release, Scholar Rock, JAN 11, 2021, View Source [SID1234573843]).

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"2020 was a transformative year for Scholar Rock with significant progress made both clinically and operationally with positive interim data from the TOPAZ trial providing initial proof-of-concept of apitegromab’s potential in SMA and showing the potential benefit of inhibiting the latent forms of growth factors," said Tony Kingsley, President and CEO of Scholar Rock. "In 2021, we will further elucidate the potential of our product candidates through data from our TOPAZ Phase 2 trial in SMA and our DRAGON Phase 1 trial in cancer immunotherapy as well as build upon our scientific platform towards additional indications."

2021 Expected Milestones:

Apitegromab is a highly selective inhibitor of latent myostatin activation being developed as the potential first muscle-directed therapy for the treatment of spinal muscular atrophy (SMA).

Top-line Efficacy and Safety Data from TOPAZ Phase 2 Trial in SMA Anticipated in 2Q21. A total of 58 patients were enrolled across the three cohorts of the TOPAZ clinical trial with one discontinuation to date. Positive six-month interim analysis data were announced in October 2020, demonstrating apitegromab’s potential to improve motor function in patients with Type 2 and Type 3 SMA. The top-line 12-month efficacy and safety data are expected in the second quarter of 2021.
As of January 8, 2021, 56 patients have completed the 12-month study and all 56 have opted into the extension period.

Identification of Second Indication for Apitegromab Planned for 2021. With the demonstration of initial proof-of-concept from the TOPAZ interim analysis, Scholar Rock is actively evaluating multiple other disorders for which the selective inhibition of the activation of myostatin may offer therapeutic benefit.
SRK-181 is a potent and highly selective inhibitor of latent transforming growth factor beta 1 (TGFβ1) activation being developed with an aim of overcoming resistance to and increasing the number of patients who may benefit from, checkpoint inhibitor therapy.

Initial Clinical Response and Safety Data from the DRAGON Trial are Anticipated in the Second Half of 2021. SRK-181 is being evaluated in the two-part DRAGON Phase 1 trial in patients with locally advanced or metastatic solid tumors exhibiting primary resistance to anti-PD-(L)1 therapy. Dose escalation in Part A of the DRAGON trial is progressing with the highest planned dose of 2400 mg now being evaluated in Part A1 (SRK-181 as a single-agent) and the 800 mg dose being currently evaluated in Part A2 (SRK-181 in combination with an approved anti-PD-(L)1 therapy).
The DRAGON trial is anticipated to advance to Part B in the second quarter of 2021, which is expected to encompass multiple cohorts, including urothelial carcinoma, cutaneous melanoma, non-small cell lung cancer, and other solid tumors. Each cohort will enroll up to 40 patients with locally advanced or metastatic solid tumors for which they have been treated with an approved anti-PD-(L)1 therapy and have demonstrated primary resistance. Patients in Part B will be treated with SRK-181 in combination with an approved anti-PD-(L)1 therapy.

"We enter 2021 with positive momentum across our programs. We believe we’ve demonstrated the potential of our novel scientific approach to treating a wide array of disease areas for which growth factors play a critical role in disease progression," said Ted Myles, CFO and Head of Business Operations of Scholar Rock. "Our strong balance sheet, which can fund operations into 2023, provides flexibility as we continue to invest in developing important therapies for patients in need."