Scholar Rock Reports Full Year 2021 Financial Results and Highlights Business Progress

On March 7, 2022 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 financial results for the full year ended December 31, 2021, and highlighted recent progress and upcoming milestones for its pipeline programs (Press release, Scholar Rock, MAR 7, 2022, View Source [SID1234609600]).

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"We are excited by the momentum and engagement around our ongoing Phase 3 SAPPHIRE trial of apitegromab in spinal muscular atrophy, as well as our DRAGON Phase 1 trial of SRK-181 in solid tumors. These programs have significant potential to address unmet patient need in a differentiated way, as well as demonstrate the tremendous potential of Scholar Rock’s platform, which focuses on inhibiting the activation of latent growth factors," said Nagesh Mahanthappa, Interim Chief Executive Officer of Scholar Rock. "While we progress these clinical programs, we also continue to build upon our world-leading expertise in selectively targeting the activation mechanisms of the TGFβ-superfamily growth factors and advance our preclinical pipeline focused on diseases such as fibrosis and regulation of iron metabolism. We look forward to providing more details about these programs at scientific meetings and through publications in the coming months."

Company Updates and Upcoming Milestones

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

Conducting SAPPHIRE Phase 3 Clinical Trial Evaluating Apitegromab in Patients with Non-Ambulatory Type 2 and 3 SMA. The randomized, double-blind, placebo-controlled Phase 3 clinical trial is evaluating apitegromab as add-on therapy for patients on either nusinersen or risdiplam. The study design plans for approximately 156 patients aged 2-12 years old with non-ambulatory Type 2 and 3 SMA to be enrolled in the main efficacy population. Patients will be randomized 1:1:1 to receive for 12 months either apitegromab 20 mg/kg, apitegromab 10 mg/kg, or placebo by intravenous (IV) infusion every 4 weeks in addition to background SMN treatment.
Data from TOPAZ Phase 2 Open Label Extension Trial Expected to be Presented by mid-2022. As of February 28, 2022, 55 of 57 patients remain enrolled in the long-term extension trial of apitegromab in Type 2 and Type 3 SMA.
SRK-181 is a selective inhibitor of latent TGFβ1 activation being developed with the aim of overcoming primary resistance to and increasing the number of patients who may benefit from checkpoint inhibitor therapy.

Advancing Part B of the DRAGON Phase 1 POC Trial for SRK-181. Based on the safety and pharmacokinetic data from Part A of the DRAGON Phase 1 trial, Scholar Rock is conducting the Part B dose expansion portion of the trial, in which SRK-181 is dosed at 1500 mg every three weeks (Q3W) in patients receiving an approved anti-PD-(L)1 therapy dosed Q3W, or 1000 mg every two weeks (Q2W) in patients receiving an approved anti-PD-(L)1 therapy dosed Q2W. Part B consists of multiple proof-of-concept cohorts focused upon evaluating the ability of SRK-181 to overcome primary resistance to anti-PD-(L)1 therapy. Each cohort will enroll up to 40 patients with various solid tumors, including urothelial carcinoma (UC), cutaneous melanoma (MEL), non-small cell lung cancer (NSCLC), clear cell renal cell carcinoma (ccRCC), and other solid tumors. The biomarker strategy in part B of DRAGON will explore early signs of SRK-181 activity, including target engagement and pathway modulation. This will include measuring effects on both circulating and tumor immune contexture, such as CD8+ T cell infiltration and reductions in myeloid-derived suppressor cell (MDSC) populations as well as analysis of TGFβ-related pathway signaling. Early efficacy and safety data are anticipated in 2022.
Full Year 2021 Financial Results

For the full year ended December 31, 2021, net loss was $131.8 million or $3.59 per share compared to a net loss of $86.5.0 million or $2.81 per share for the year ended December 31, 2020.

Revenue was $18.8 million for the year ended December 31, 2021 compared to $15.4 million for the year ended December 31, 2020. Revenue was related to the Gilead fibrosis-focused research collaboration, which concluded in December 2021.
Research and development expense was $108.5 million for the year ended December 31, 2021 compared to $74.1 million for the year ended December 31, 2020. The increase year-over-year was primarily attributable to planned spend associated with apitegromab development, including costs associated with clinical drug supply manufacturing and costs associated with our SAPPHIRE trial, as well as higher personnel-related costs.
General and administrative expense was $40.3 million for the year ended December 31, 2021 compared to $28.2 million for the year ended December 31, 2020. The increase year-over-year was primarily attributable to professional services and higher personnel-related costs, including filling key positions essential to progressing research, development and pre-commercial activities.
As of December 31, 2021, Scholar Rock had cash, cash equivalents, and marketable securities of approximately $253.0 million, which is expected to fund the Company’s operations into mid-2023. "Continued operational excellence is an important priority for us in 2022 as we advance several programs across the continuum of drug development. As we continue to progress our SMA program with our Phase 3 SAPPHIRE trial, we are also encouraged that so many of the patients who participated in the TOPAZ study are still opting to receive apitegromab. We look forward to obtaining further insights into longer-term treatment outcomes and sharing that data in the coming months. In our immuno-oncology program, the DRAGON study has entered Part B and could produce meaningful data later this year and our research colleagues are pushing forward on several fronts as we continue to generate exciting programs from our scientific platform," said Ted Myles, COO and CFO of Scholar Rock.