\nScholar Rock (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today reported financial results for the full year ended December 31, 2020 and highlighted recent progress and upcoming milestones for its pipeline programs.\n<\/p>\n
\n\u201c2021 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,\u201d said Tony Kingsley, President and CEO of Scholar Rock. \u201cAll 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.\u201d\n<\/p>\n
Company Updates and Upcoming Milestones<\/b><\/p>\n
Apitegromab<\/span><\/i><\/b> is a highly selective inhibitor of latent myostatin activation being developed as the potential first muscle-directed therapy for the treatment of SMA.<\/i><\/p>\n SRK-181 <\/span><\/i><\/b>is a potent and highly selective inhibitor of latent TGF\u03b21 activation being developed with the aim of overcoming resistance to and increasing the number of patients who may benefit from checkpoint inhibitor therapy.<\/i><\/p>\n \nThe 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.\n<\/p>\n \nInitial 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.\n<\/p>\n Full Year 2020 Financial Results<\/b><\/p>\n \nFor 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.\n<\/p>\n \nAs of December 31, 2020, Scholar Rock had cash, cash equivalents, and marketable securities of approximately $341.0 million.\n<\/p>\n About Scholar Rock<\/b><\/p>\n Scholar Rock<\/a> is a clinical-stage biopharmaceutical company focused on the discovery and development of innovative medicines for the treatment of serious diseases in which signaling by protein growth factors plays a fundamental role. Scholar Rock is creating a pipeline of novel product candidates with the potential to transform the lives of patients suffering from a wide range of serious diseases, including neuromuscular disorders, cancer, and fibrosis. Scholar Rock\u2019s approach to targeting the molecular mechanisms of growth factor activation enabled it to develop a proprietary platform<\/a> for the discovery and development of monoclonal antibodies that locally and selectively target these signaling proteins at the cellular level. By developing product candidates that act in the disease microenvironment, the Company intends to avoid the historical challenges associated with inhibiting growth factors for therapeutic effect. Scholar Rock believes its focus on biologically validated growth factors may facilitate a more efficient development path. For more information, please visit www.ScholarRock.com<\/a> or follow Scholar Rock on Twitter (@ScholarRock<\/a>) and LinkedIn (https:\/\/www.linkedin.com\/company\/scholar-rock\/<\/a>).\n<\/p>\n \nScholar Rock\u00ae is a registered trademark of Scholar Rock, Inc.\n<\/p>\n Forward-Looking Statements<\/b><\/p>\n \nThis press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding Scholar Rock\u2019s future expectations, plans and prospects, including without limitation, Scholar Rock\u2019s expectations regarding its growth, strategy, progress and timing of its clinical trials for apitegromab, SRK-181, and other product candidates and indication selection and development timing, its cash runway, the ability of any product candidate to perform in humans in a manner consistent with earlier nonclinical, preclinical or clinical trial data, and the potential of its product candidates and proprietary platform.The use of words such as \u201cmay,\u201d \u201cmight,\u201d \u201cwill,\u201d \u201cshould,\u201d \u201cexpect,\u201d \u201cplan,\u201d \u201canticipate,\u201d \u201cbelieve,\u201d \u201cestimate,\u201d \u201cproject,\u201d \u201cintend,\u201d \u201cfuture,\u201d \u201cpotential,\u201d or \u201ccontinue,\u201d and other similar expressions are intended to identify such forward-looking statements. All such forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include Scholar Rock\u2019s ability to provide the financial support, resources and expertise necessary to identify and develop product candidates on the expected timeline, the data generated from Scholar Rock\u2019s nonclinical and preclinical studies and clinical trials, competition from third parties that are developing products for similar uses, Scholar Rock\u2019s ability to obtain, maintain and protect its intellectual property, the success of Scholar Rock\u2019s current and potential future collaborations, including its collaboration with Gilead, Scholar Rock\u2019s dependence on third parties for development and manufacture of product candidates including to supply any clinical trials, Scholar Rock\u2019s ability to manage expenses and to obtain additional funding when needed to support its business activities and establish and maintain strategic business alliances and new business initiatives, and the impacts of public health pandemics such as COVID-19 on business operations and expectations, as well as those risks more fully discussed in the section entitled “Risk Factors” in Scholar Rock\u2019s Annual Report on Form 10-K for the year ended December 31, 2020, as well as discussions of potential risks, uncertainties, and other important factors in Scholar Rock\u2019s subsequent filings with the Securities and Exchange Commission. Any forward-looking statements represent Scholar Rock\u2019s views only as of today and should not be relied upon as representing its views as of any subsequent date. All information in this press release is as of the date of the release, and Scholar Rock undertakes no duty to update this information unless required by law.\n<\/p>\n Years Ended December 31<\/b><\/p>\n<\/td>\n<\/tr>\n 2020<\/b><\/p>\n<\/td>\n 2019<\/b><\/p>\n<\/td>\n<\/tr>\n \n$\n<\/p>\n<\/td>\n \n15,403\n<\/p>\n<\/td>\n \n\u00a0\n<\/p>\n<\/td>\n \n$\n<\/p>\n<\/td>\n \n20,492\n<\/p>\n<\/td>\n \n\u00a0\n<\/p>\n<\/td>\n<\/tr>\n \n\u00a0\n<\/p>\n<\/td>\n \n74,062\n<\/p>\n<\/td>\n \n\u00a0\n<\/p>\n<\/td>\n \n\u00a0\n<\/p>\n<\/td>\n \n54,217\n<\/p>\n<\/td>\n \n\u00a0\n<\/p>\n<\/td>\n<\/tr>\n \n\u00a0\n<\/p>\n<\/td>\n \n28,219\n<\/p>\n<\/td>\n \n\u00a0\n<\/p>\n<\/td>\n \n\u00a0\n<\/p>\n<\/td>\n \n20,817\n<\/p>\n<\/td>\n \n\u00a0\n<\/p>\n<\/td>\n<\/tr>\n \n\u00a0\n<\/p>\n<\/td>\n \n102,281\n<\/p>\n<\/td>\n \n\u00a0\n<\/p>\n<\/td>\n \n\u00a0\n<\/p>\n<\/td>\n \n75,034\n<\/p>\n<\/td>\n \n\u00a0\n<\/p>\n<\/td>\n<\/tr>\n \n\u00a0\n<\/p>\n<\/td>\n \n(86,878\n<\/p>\n<\/td>\n \n)\n<\/p>\n<\/td>\n \n\u00a0\n<\/p>\n<\/td>\n \n(54,542\n<\/p>\n<\/td>\n \n)\n<\/p>\n<\/td>\n<\/tr>\n \n\u00a0\n<\/p>\n<\/td>\n \n395\n<\/p>\n<\/td>\n \n\u00a0\n<\/p>\n<\/td>\n \n\u00a0\n<\/p>\n<\/td>\n \n3,542\n<\/p>\n<\/td>\n \n\u00a0\n<\/p>\n<\/td>\n<\/tr>\n \n$\n<\/p>\n<\/td>\n \n(86,483\n<\/p>\n<\/td>\n \n)\n<\/p>\n<\/td>\n \n$\n<\/p>\n<\/td>\n \n(51,000\n<\/p>\n<\/td>\n \n)\n<\/p>\n<\/td>\n<\/tr>\n \n$\n<\/p>\n<\/td>\n \n(2.81\n<\/p>\n<\/td>\n \n)\n<\/p>\n<\/td>\n \n$\n<\/p>\n<\/td>\n \n(1.85\n<\/p>\n<\/td>\n \n)\n<\/p>\n<\/td>\n<\/tr>\n \n\u00a0\n<\/p>\n<\/td>\n \n30,734,109\n<\/p>\n<\/td>\n \n\u00a0\n<\/p>\n<\/td>\n \n\u00a0\n<\/p>\n<\/td>\n \n27,537,939\n<\/p>\n<\/td>\n \n\u00a0\n<\/p>\n<\/td>\n<\/tr>\n<\/table>\n December 31, 2020<\/b><\/p>\n<\/td>\n December 31, 2019<\/b><\/p>\n<\/td>\n<\/tr>\n \n$\n<\/p>\n<\/td>\n \n341,031\n<\/p>\n<\/td>\n \n$\n<\/p>\n<\/td>\n \n157,448\n<\/p>\n<\/td>\n<\/tr>\n \n\u00a0\n<\/p>\n<\/td>\n \n3,373\n<\/p>\n<\/td>\n \n\u00a0\n<\/p>\n<\/td>\n \n27,719\n<\/p>\n<\/td>\n<\/tr>\n \n\u00a0\n<\/p>\n<\/td>\n \n344,404\n<\/p>\n<\/td>\n \n\u00a0\n<\/p>\n<\/td>\n \n185,167\n<\/p>\n<\/td>\n<\/tr>\n \n\u00a0\n<\/p>\n<\/td>\n \n43,901\n<\/p>\n<\/td>\n \n\u00a0\n<\/p>\n<\/td>\n \n11,214\n<\/p>\n<\/td>\n<\/tr>\n \n$\n<\/p>\n<\/td>\n \n388,305\n<\/p>\n<\/td>\n \n$\n<\/p>\n<\/td>\n \n196,381\n<\/p>\n<\/td>\n<\/tr>\n \n$\n<\/p>\n<\/td>\n \n42,564\n<\/p>\n<\/td>\n \n$\n<\/p>\n<\/td>\n \n32,814\n<\/p>\n<\/td>\n<\/tr>\n \n\u00a0\n<\/p>\n<\/td>\n \n84,971\n<\/p>\n<\/td>\n \n\u00a0\n<\/p>\n<\/td>\n \n50,666\n<\/p>\n<\/td>\n<\/tr>\n \n\u00a0\n<\/p>\n<\/td>\n \n127,535\n<\/p>\n<\/td>\n \n\u00a0\n<\/p>\n<\/td>\n \n83,480\n<\/p>\n<\/td>\n<\/tr>\n \n\u00a0\n<\/p>\n<\/td>\n \n260,770\n<\/p>\n<\/td>\n \n\u00a0\n<\/p>\n<\/td>\n \n112,901\n<\/p>\n<\/td>\n<\/tr>\n \n$\n<\/p>\n<\/td>\n \n388,305\n<\/p>\n<\/td>\n \n$\n<\/p>\n<\/td>\n \n196,381\n<\/p>\n<\/td>\n<\/tr>\n<\/table>\n \n\u00a0\n<\/p>\n View source version on businesswire.com: <\/span>https:\/\/www.businesswire.com\/news\/home\/20210309005683\/en\/<\/a><\/span><\/p>\n Scholar Rock Contacts: Media KEYWORDS:<\/b> United States North America Massachusetts<\/p>\n INDUSTRY KEYWORDS:<\/b> Research Clinical Trials Other Health Biotechnology General Health Pharmaceutical Health Science Oncology Other Science<\/p>\n\n
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\n Scholar Rock Holding Corporation<\/b><\/td>\n<\/tr>\n \n Condensed Consolidated Statements of Operations<\/b><\/td>\n<\/tr>\n \n (unaudited)<\/td>\n<\/tr>\n \n (in thousands, except share and per share data)<\/td>\n<\/tr>\n \n \n \n \n \n \u00a0<\/td>\n<\/tr>\n \n \n \n \n \n \u00a0<\/td>\n<\/tr>\n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \n \u00a0<\/td>\n<\/tr>\n \n Revenue<\/td>\n \n \n \n \n \n \n \n \n \n \n \n \n \n \u00a0<\/td>\n<\/tr>\n \n Operating expenses<\/td>\n \n \n \n <\/tr>\n \n Research and development<\/td>\n \n \n \n \n \n \n \n \n \n General and administrative<\/td>\n \n \n \n \n \n \n \n \n \n Total operating expenses<\/td>\n \n \n \n \n \n \n \n \n \n Loss from operations<\/td>\n \n \n \n \n \n \n \n \n \n Other income (expense), net<\/td>\n \n \n \n \n \n \n \n \n \n Net loss<\/td>\n \n \n \n \n \n \n \n \n \n \n \n \n \n \u00a0<\/td>\n<\/tr>\n \n Net loss per share, basic and diluted<\/td>\n \n \n \n \n \n \n \n \n \n \n \n \n \n \u00a0<\/td>\n<\/tr>\n \n Weighted average common shares outstanding, basic and diluted<\/td>\n \n \n \n \n \n \n \n \n \n
\n \n Scholar Rock Holding Corporation<\/b><\/td>\n<\/tr>\n \n \n Condensed Consolidated Balance Sheets <\/b><\/td>\n<\/tr>\n \n \n (unaudited)<\/td>\n<\/tr>\n \n \n (in thousands)<\/td>\n<\/tr>\n \n \n \n \n \n \n \u00a0<\/td>\n<\/tr>\n \n \n \n \n \n \n \n \n Assets<\/b><\/td>\n \n \n \n <\/tr>\n \n \n Cash, cash equivalents and marketable securities<\/td>\n \n \n \n \n \n \n \n \n Other current assets<\/td>\n \n \n \n \n \n \n \n \n Total current assets<\/td>\n \n \n \n \n \n \n \n \n Other assets<\/td>\n \n \n \n \n \n \n \n Total assets<\/td>\n \n \n \n \n \n \n \n \n \n \n \n \n \u00a0<\/td>\n<\/tr>\n \n Liabilities and Stockholders’ Equity<\/b><\/td>\n \n \n \n <\/tr>\n \n \n Current liabilities<\/td>\n \n \n \n \n \n \n \n \n Long-term liabilities<\/td>\n \n \n \n \n \n \n \n \n Total liabilities<\/td>\n \n \n \n \n \n \n \n \n Total stockholders’ equity<\/td>\n \n \n \n \n \n \n \n Total liabilities and stockholders’ equity<\/td>\n \n \n \n \n \n \n <\/p>\n
\n<\/b>
Investors
\n<\/b>
Catherine Hu
\n
chu@scholarrock.com<\/a><\/p>\n
\n<\/b>
Ariane Lovell
\n
Finn Partners
\n
ariane.lovell@finnpartners.com
\n<\/a>
917-565-2204\n<\/p>\n
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