On August 8, 2022 Scholar Rock (NASDAQ: SRRK), a Phase 3 clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, reported financial results and corporate updates for the second quarter ended June 30, 2022 (Press release, Scholar Rock, AUG 8, 2022, View Source [SID1234617816]).
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"We are pleased to have reported important progress with our two clinical programs during the second quarter. Enrollment continues to advance in our pivotal Phase 3 SAPPHIRE trial for apitegromab in SMA. We also presented new data from our Phase 2 TOPAZ trial highlighting sizeable and sustained improvement in Hammersmith Functional Motor Scale-Expanded (HFMSE) scores at 24 months for non-ambulatory patients, reinforcing our belief in the unique potential of apitegromab as a muscle-targeted therapy to treat SMA, and we continue to evaluate these patients in their third year of treatment," said Nagesh Mahanthappa, Founding Chief Executive Officer & President of Scholar Rock. "In our SRK-181 program, enrollment is ongoing in the Phase 1 DRAGON trial Part B dose expansion. We look forward to providing updates on DRAGON as data become available. Further, our focused research continues to make excellent progress as we develop next-generation programs to build out our future pipeline."
Company Highlights and Upcoming Milestones
Apitegromab is a selective inhibitor of myostatin activation being developed as the first muscle-targeted therapy with the potential to treat spinal muscular atrophy (SMA).
Positive 24-Month Phase 2 TOPAZ Extension Data Support Potential Benefit for Patients. Scholar Rock announced positive data at the Cure SMA Research & Clinical Care Meeting in June, demonstrating sizable and sustained motor function improvement at 24 months with apitegromab as measured by Hammersmith Functional Motor Scale-Expanded (HFMSE) scores for non-ambulatory patients with Types 2 and 3 SMA on nusinersen. Results also showed a substantial increase in Revised Upper Limb Module (RULM) scores, with no serious safety risks identified over 24 months of apitegromab treatment. Of the 55 patients who completed the 24-month extension period, 54 opted to continue into the 36-month extension period.
Enrollment Ongoing for Phase 3 SAPPHIRE Clinical Trial Evaluating Apitegromab in Non-Ambulatory Patients with Types 2 and 3 SMA. The randomized, double-blind, placebo-controlled Phase 3 clinical trial is evaluating apitegromab for patients on either nusinersen or risdiplam. Approximately 156 non-ambulatory patients aged 2-12 years old with Types 2 and 3 SMA are planned 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. SAPPHIRE is expected to be conducted across 55 sites in the U.S. and Europe. Scholar Rock presented on the trial design at the 17th International Congress on Neuromuscular Diseases (ICNMD 2022) in July 2022, along with publication of the abstract in the peer-reviewed Journal of Neuromuscular Diseases.
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 Enrollment for Part B of the Phase 1 DRAGON Proof-of-Concept Clinical Trial for SRK-181. Part B of the Phase 1 DRAGON trial consists of multiple proof-of-concept cohorts focused on evaluating the ability of SRK-181 to overcome primary resistance to anti-PD-(L)1 therapy in patients with solid tumors. The biomarker strategy for DRAGON explores early signs of SRK-181 activity, including target engagement and pathway modulation. It includes 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. Initial evidence of drug activity and safety data are anticipated in 2022.
Scholar Rock’s preclinical discovery pipeline includes a highly selective, context-dependent TGFβ1 antibody that inhibits proTGFβ1 activation selectively in the extracellular matrix via targeting the covalent complexes of proTGFβ1 with latent TGFβ binding proteins 1 and 3 ("LTBPs"— the targets are collectively referred to as "LTBP-TGFβ1").
Preclinical Data Supports Selective Targeting of Matrix-Associated TGFβ1 as an Attractive Approach for Anti-Fibrotic Therapy. Scholar Rock recently presented data on its targeted approach to LTBP-TGFβ1 that show reduction of TGFβ1 signaling and reduction of fibrosis in relevant preclinical in vivo models.
Second Quarter 2022 Financial Results
For the quarter ended June 30, 2022, net loss was $44.0 million or $1.06 per share compared to a net loss of $30.7 million or $0.84 per share for the quarter ended June 30, 2021.
Revenue was $0 for the quarter ended June 30, 2022, compared to $4.6 million for the quarter ended June 30, 2021.
Research and development expense was $32.1 million for the quarter ended June 30, 2022, compared to $25.6 million for the quarter ended June 30, 2021. The increase was primarily attributable to increased clinical costs associated with apitegromab and higher personnel costs, including severance expenses associated with the recent restructuring.
General and administrative expense was $11.1 million for the quarter ended June 30, 2022, compared to $9.3 million for the quarter ended June 30, 2021. The increase was primarily due to higher personnel costs, including severance expenses associated with the recent restructuring.
As of June 30, 2022, Scholar Rock had cash, cash equivalents, and marketable securities of approximately $371 million, which is expected to fund the Company’s anticipated operating and capital expenditure requirements into 2025.
"We were thrilled to announce the completion of a $205 million registered direct offering during the quarter. This financing included several high-quality, long-term oriented, fundamentals-based biotechnology investors who saw the promise of our platform and our programs. Importantly, this capital puts us in a strong financial position to fully fund the Phase 3 SAPPHIRE trial, continue advancing Part B of the Phase 1 DRAGON trial for SRK-181, while investing in selected early-stage programs that exemplify the power of our scientific platform," said Ted Myles, Chief Operating Officer and Chief Financial Officer of Scholar Rock.