Scholar Rock Reports Third Quarter 2019 Financial Results and Highlights Business Progress
- Enrollment on track in the TOPAZ Phase 2 clinical trial of SRK-015 in patients with Type 2 and Type 3 Spinal Muscular Atrophy for preliminary PK/PD results by year-end
- Accelerated initiation of Phase 1 dose escalation and proof-of-concept clinical trial for SRK-181 in patients with solid tumors to the first quarter of 2020
“The third quarter of 2019 has been a time of focused execution as we continue to advance our growing pipeline. Notably, we have been able to accelerate the development of SRK-181 and are now planning to initiate a Phase 1 dose escalation and proof-of-concept trial in patients with solid tumors in the first quarter of 2020, a testament to the strength of our team and scientific progress,” said Nagesh Mahanthappa, Ph.D., President and CEO of
Company Highlights and Upcoming Milestones
- Preclinical and Phase 1 SRK-015 Healthy Volunteer Data Presented at
World Muscle Society Congress. In October 2019, Scholar Rockpresented data for SRK-015, a highly selective inhibitor of myostatin activation, that further enforce the therapeutic rationale for the advancement of SRK-015 into the TOPAZ Phase 2 clinical trial. Preclinical studies demonstrated improved muscle strength following the administration of a mouse analog of SRK-015 (muSRK-015P) in mouse models of early and late stage restoration of SMN, the protein that is deficient in the genetic disease SMA. Multi-fold increases in serum latent myostatin levels following treatment with muSRK-015P were shown across animal species in mice, rats, and cynomolgus monkeys. In the Phase 1 clinical trial in adult healthy volunteers, SRK -015 was well-tolerated, had a well-behaved pharmacokinetic (PK) profile, and demonstrated robust and sustained target engagement.
Scholar Rock is on track to report preliminary PK and pharmacodynamic (PD) data for a subset of patients in the Phase 2 TOPAZ trial by the end of 2019, which will provide initial observations on target engagement of latent myostatin in patients with Type 2 and Type 3 SMA. These preliminary PK/PD results will be followed by interim safety and efficacy results for a subset of patients with six months of treatment exposure expected in the first half of 2020 and top-line data for the full 12-month treatment period expected beginning in the fourth quarter of 2020.
- Identification of Second Indication for SRK-015 Planned for 2020.
Scholar Rockcontinues to evaluate multiple potential opportunities for which SRK-015 could offer clinical benefit and is assessing additional potential clinical settings in which the selective inhibition of the activation of myostatin may offer therapeutic benefit.
- Accelerated Initiation of Phase 1 Dose Escalation and Proof-of-Concept Clinical Trial of SRK-181 in Patients with Solid Tumors to the First Quarter of 2020.
Scholar Rockintends to develop SRK-181, an isoform-selective inhibitor of TGFβ1 activation as a cancer immunotherapy in combination with anti-PD(L)1 antibodies. The company now plans to initiate a Phase 1 trial in the first quarter of 2020, in patients with locally advanced or metastatic solid tumors that exhibit primary resistance to anti-PD(L)1 antibodies. The two-part trial will consist of a dose escalation portion as both a single-agent and SRK-181 in combination with an approved anti-PD(L)1 antibody, as well as a dose expansion portion consisting of multiple tumor-specific cohorts evaluating SRK-181 in combination with an approved anti-PD(L)1 antibody.
- Nomination of a Product Candidate from the RGMc Program Planned in 2020. Scholar Rock is evaluating a number of highly specific inhibitors of repulsive guidance molecule C (RGMc) and plans to nominate an antibody as its third product candidate in 2020. RGMc’s known function is localized to hepatocytes and the identification of RGMc selective-antibodies may offer the potential for liver-specific modulation of BMP6 signaling to address iron-restricted anemias.
Third Quarter 2019 Financial Results
For the quarter ended
- Revenue was
$4.8 millionfor the quarter ended September 30, 2019and was related to the Gilead Collaboration Agreement that was executed in December 2018.
- Research and development expense was
$15.7 millionfor the quarter ended September 30, 2019compared to $8.1 millionfor the quarter ended September 30, 2018. The increase year-over-year primarily reflects preclinical and manufacturing costs for SRK-181, clinical development costs associated with SRK-015, and higher personnel-related costs.
- General and administrative expense was
$6.2 millionfor the quarter ended September 30, 2019compared to $3.2 millionfor the quarter ended September 30, 2018. The increase year-over-year was primarily attributable to increased headcount, stock compensation, separation-related expense, and professional services.
About Scholar Rock
Scholar Rock 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, fibrosis and anemia. Scholar Rock’s newly elucidated understanding of the molecular mechanisms of growth factor activation enabled it to develop a proprietary platform 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® is a registered trademark of
This 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’s future expectations, plans and prospects, including without limitation, Scholar Rock’s expectations regarding its growth, strategy, progress and timing of its clinical trials for SRK-015, SRK-181, and other product candidates and indication selection and development timing, and the ability of any product candidate to perform in humans in a manner consistent with nonclinical or preclinical study data. The use of words such as “may,” “might,” “will,” “should,” “expect,” “plan,” “anticipate,” “believe,” “estimate,” “project,” “intend,” “future,” “potential,” or “continue,” 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’s 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’s nonclinical and preclinical studies and clinical trials, competition from third parties that are developing products for similar uses, Scholar Rock’s ability to obtain, maintain and protect its intellectual property, the success of Scholar Rock’s current and potential future collaborations, including its collaboration with Gilead, Scholar Rock’s dependence on third parties for development and manufacture of product candidates including to supply any clinical trials, and Scholar Rock’s 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 as well as those risks more fully discussed in the section entitled "Risk Factors" in Scholar Rock’s Quarterly Report on Form 10-Q for the quarter ended
Scholar Rock Contact:
The Yates Network
|Scholar Rock Holding Corporation|
|Condensed Consolidated Statements of Operations|
|(in thousands, except share and per share data)|
|Three Months Ended September 30,||Nine Months Ended September 30,|
|Research and development||15,699||8,061||40,153||26,185|
|General and administrative||6,181||3,173||14,961||8,947|
|Total operating expenses||21,880||11,234||55,114||35,132|
|Loss from operations||(17,106||)||(11,234||)||(42,195||)||(35,132||)|
|Other income (expense), net||959||472||2,768||772|
|Net loss per share, basic and diluted||$||(0.55||)||$||(0.44||)||$||(1.46||)||$||(2.72||)|
|Weighted average common shares outstanding, basic and diluted||29,232,158||24,310,681||26,929,215||12,647,032|
|Scholar Rock Holding Corporation|
|Condensed Consolidated Balance Sheets|
|September 30, 2019||December 31, 2018|
|Cash, cash equivalents and marketable securities||$||176,082||$||175,645|
|Other current assets||3,157||2,296|
|Total current assets||179,239||177,941|
|Liabilities and Stockholders' Equity|
|Total stockholders' equity||121,790||106,623|
|Total liabilities and stockholders' equity||$||188,542||$||181,336|
Source: Scholar Rock