On March 9, 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 financial results for the full year ended December 31, 2020 and highlighted recent progress and upcoming milestones for its pipeline programs (Press release, Scholar Rock, MAR 9, 2021, View Source [SID1234576325]).
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"2021 is poised to further validate the therapeutic potential of our scientific platform, and we are starting the year with strong momentum as we continue to execute and advance our clinical trials in both SMA and immuno-oncology," said Tony Kingsley, President and CEO of Scholar Rock. "All patients in the TOPAZ trial in SMA have now completed the 12-month treatment period and we are on-track to report top-line results next quarter. We also look forward to clinical read-outs from our SRK-181 DRAGON trial in cancer immunotherapy, along with continued progress with our earlier-stage programs."
Company Updates and Upcoming Milestones
Apitegromab is a highly selective inhibitor of latent myostatin activation being developed as the potential first muscle-directed therapy for the treatment of SMA.
Top-Line Efficacy and Safety Data from the TOPAZ Phase 2 Trial Expected in 2Q21. A total of 58 patients were enrolled across the three cohorts of the TOPAZ clinical trial (NCT03921528). With the exception of one patient who discontinued early from the trial for reasons unrelated to the study drug, all patients completed the 12-month treatment period and have elected to opt into the extension period. Top-line data from the 12-month treatment period will provide additional insights on apitegromab’s potential in improving motor function in patients with Type 2 and Type 3 SMA, including the potential for durability of effect and for further motor function increases observed from the 6-month interim analysis.
U.S. Patent Issued Providing Broad Therapeutic Use of Apitegromab. The United States Patent Office (USPTO) has issued U.S. Patent 10,882,904 with an expiry of September 2036. The issued claims are broadly directed to the use of apitegromab to achieve two or more of the following therapeutic effects: increase muscle mass or function, decrease adipose-to-muscle ratios, decrease intramuscular fat infiltration, and prevent muscle loss or atrophy, without limiting to specific indications.
Identification of Second Indication for Apitegromab Planned for 2021. Scholar Rock is evaluating multiple other diseases 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 TGFβ1 activation being developed with the aim of overcoming resistance to and increasing the number of patients who may benefit from checkpoint inhibitor therapy.
Presented Preclinical Data at the TGFβ for Immuno-Oncology Drug Development Summit. In January 2021, Scholar Rock participated in a panel discussion titled "Debating the Best Approach to Target TGF-β" and presented "Inhibition of TGFβ1 Activation with SRK-181 Overcomes Primary Resistance to Checkpoint Inhibition Therapy" at the TGFβ for Immuno-Oncology Drug Development Summit. The presentation provided an overview of the preclinical data demonstrating that combination treatment with a murine version of SRK-181 and an anti-PD-1 therapy led to tumor regression and an improved preclinical toxicity profile compared to less selective TGFβ inhibition.
DRAGON Phase 1 Trial Anticipated to Advance to Part B Dose Expansion in 2Q21. SRK-181 is being evaluated in the two-part DRAGON trial (NCT04291079) in patients with locally advanced or metastatic solid tumors exhibiting primary resistance to anti-PD-(L)1 therapy. The main goals of Part A of the trial are to evaluate the safety and pharmacokinetics of SRK-181 and to determine the dosing regimen to be evaluated in Part B of the trial. As of March 8, 2021, dose escalation in Part A1 (SRK-181 as a single-agent) has progressed beyond the previously announced highest planned dose of 2400 mg every 3 weeks (Q3W) to 3000 mg Q3W to further characterize the upper bounds of the dose range, as permitted by the protocol. Dose escalation in Part A2 of the trial (SRK-181 in combination with an approved anti-PD-(L)1 therapy) has progressed to 1600 mg Q3W.
The Part B dose expansion portion of the trial will consist of 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.
Initial clinical response and safety data from the DRAGON trial are expected in the second half of 2021. The ongoing COVID-19 pandemic may impact the enrollment and dosing of patients in the trial.
Full Year 2020 Financial Results
For the full year ended December 31, 2020, net loss was $86.5 million or $2.81 per share compared to a net loss of $51.0 million or $1.85 per share for the year ended December 31, 2019.
Revenue was $15.4 million for the year ended December 31, 2020 compared to $20.5 million for the year ended December 31, 2019. Revenue was related to the Gilead fibrosis-focused collaboration that was executed in December 2018.
Research and development expense was $74.1 million for the year ended December 31, 2020 compared to $54.2 million for the year ended December 31, 2019. The increase year-over-year was primarily attributable to costs associated with the apitegromab TOPAZ Phase 2 clinical trial, including clinical drug supply manufacturing, the SRK-181 DRAGON Phase 1 clinical trial, and higher personnel-related costs.
General and administrative expense was $28.2 million for the year ended December 31, 2020 compared to $20.8 million for the year ended December 31, 2019. The increase year-over-year was primarily attributable to higher personnel-related costs and professional services.
As of December 31, 2020, Scholar Rock had cash, cash equivalents, and marketable securities of approximately $341.0 million.