On March 7, 2023 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 full year ended December 31, 2022 (Press release, Scholar Rock, MAR 7, 2023, View Source [SID1234628255]).

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"2023 is off to a great start as we continue to build upon our momentum from last year. Importantly, we are on track to complete the enrollment of our pivotal SAPPHIRE trial of apitegromab and are looking forward to providing 36-month extension data from the Phase 2 TOPAZ trial of apitegromab in the coming months. Additionally, we look forward to sharing biomarker and clinical updates throughout the year from the SRK-181 Phase 1 DRAGON trial in patients with relapsed and/or refractory cancers resistant to checkpoint inhibitor therapy," said Jay Backstrom, M.D., M.P.H., President & Chief Executive Officer of Scholar Rock.

Company Highlights and Upcoming Milestones

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

Continue to progress and complete enrollment of Phase 3 SAPPHIRE clinical trial. The last patient is expected to be enrolled in SAPPHIRE in 2023, with the top-line data readout expected in 2024. If successful, and if approved, the company expects to initiate a commercial product launch in 2025. SAPPHIRE is a randomized, double-blind, placebo-controlled Phase 3 clinical trial evaluating apitegromab in patients with nonambulatory Types 2 and 3 SMA on either nusinersen or risdiplam. The U.S. Food and Drug Administration (FDA) has granted Fast Track, Orphan Drug, and Rare Pediatric Disease designations, and the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) and Orphan designations to apitegromab for the treatment of SMA.
36-month extension data from Phase 2 TOPAZ trial to be presented in mid-2023. As of December 31, 2022, approximately 90 percent of patients who entered the trial’s long-term extension study (51/57) remained on study. At the Cure SMA Research & Clinical Care Meeting in June 2022, the company presented 24-month extension data from the TOPAZ trial which demonstrated sustained gains in Hammersmith Functional Motor Scale Expanded (HFMSE), increased Revised Upper Limb Module (RULM) scores, and positive trends in quality-of-life data for nonambulatory patients with Types 2 and 3 SMA receiving an SMN therapy.
SRK-181 is an investigational selective inhibitor of latent TGFβ-1 activation and is being developed with the aim of overcoming resistance to checkpoint therapy in patients with advanced cancer.

Continue to progress Phase 1 DRAGON trial. The company expects to provide biomarker and clinical updates from the DRAGON proof-of-concept trial throughout 2023. In November 2022, Scholar Rock presented data from DRAGON at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s (SITC) (Free SITC Whitepaper) Annual Meeting that showed SRK-181 continued to be generally well tolerated with early indications of efficacy (as of the data cut-off date of August 29, 2022).
Encore data from DRAGON will be presented at the European Society for Medical Oncology Targeted Anticancer Therapies (ESMO TAT) Congress in Paris, France on March 7 at 11:05-12:30 p.m. EST. The presentation is titled "Safety and efficacy results of SRK-181 (a latent TGFβ1 inhibitor) from a phase 1 trial (DRAGON trial)."
Corporate

Appointed Tracey M. Sacco as Chief Commercial Officer. In February 2023, Ms. Sacco joined Scholar Rock to build and lead all commercial functions, including sales, marketing, market access, and commercial operations. She will help the company prepare for the potential commercial launch of apitegromab, if approved. Ms. Sacco brings extensive biopharmaceutical industry experience, including commercial strategy and global launch experience of rare disease products.
Full Year 2022 Financial Results

For the full year ended December 31, 2022, net loss was $134.5 million or $2.26 per share compared to a net loss of $131.8 million or $3.59 per share for the full year ended December 31, 2021.

Revenue was $33.2 million for the year ended December 31, 2022, compared to $18.8 million for the year ended December 31, 2021. Revenue recognized during both periods was associated with the Gilead collaboration which ended in 2022.
Research and development expense was $124.4 million for the year ended December 31, 2022, compared to $108.5 million for the year ended December 31, 2021. The increase was primarily attributable to costs associated with running the company’s Phase 3 SAPPHIRE pivotal trial for apitegromab in SMA, including clinical trials costs as well as costs associated with employee compensation and benefits.
General and administrative expense was $43.1 million for the year ended December 31, 2022, compared to $40.3 million for the year ended December 31, 2021. The increase was due to an increase in employee compensation and benefits, primarily associated with equity-based compensation and professional fees.
As of December 31, 2022, Scholar Rock had cash, cash equivalents, and marketable securities of approximately $315 million, which is expected to fund the Company’s anticipated operating and capital expenditure requirements into 2025.
"As Scholar Rock works to develop transformative therapies for patients with our highly differentiated platform, we are entering 2023 with an experienced team, a strong balance sheet, and a clear line of sight to important anticipated milestones, all of which are expected to help advance the next phase of growth for the company," said Ted Myles, Chief Operating Officer and Chief Financial Officer of Scholar Rock.