Scholar Rock Reports First Quarter 2023 Financial Results and Highlights Business Progress
- Enrollment of pivotal Phase 3 SAPPHIRE trial evaluating apitegromab on track for completion in 2023
- Upcoming presentations of 36-month apitegromab data from Phase 2 TOPAZ trial at Cure SMA’s Annual
- Continued advancement of Phase 1 DRAGON trial of SRK-181; biomarker and clinical data update planned in the second half of 2023
- Approximately
“We are executing across our business and leveraging Scholar Rock’s differentiated platform, which selectively targets latent forms of growth factors, such as myostatin and TGFβ1, to develop highly selective and potentially transformative therapies for patients who have high unmet medical need,” said
Recent Company Highlights and Upcoming Milestones
Apitegromab is an investigational antibody inhibiting myostatin activation by selectively binding the pro- and latent forms of myostatin and is being developed as the potential first muscle-targeted therapy for the treatment of spinal muscular atrophy (SMA).
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Continued progress towards completion of enrollment for Phase 3 SAPPHIRE clinical trial. The randomized double-blind, placebo-controlled clinical trial evaluating apitegromab for patients with nonambulatory Types 2 and 3 SMA on either nusinersen or risdiplam, is actively enrolling SMA patients across sites in the
U.S. andEurope . Enrollment completion is expected 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. -
36-month data from Phase 2 TOPAZ trial to be presented at Cure SMA conference in June. The company announced two upcoming oral presentations at the
Cure SMA Research & Clinical Conference , taking placeJune 28-30 inOrlando, Florida . -
24-month data from the TOPAZ trial presented at the 2023
Muscular Dystrophy Association (MDA) Clinical and Scientific Conference in March. The company shared data evaluating outcomes after 24 months of treatment with apitegromab, demonstrating sustained improvements in motor function in patients with nonambulatory Types 2 and 3 SMA.
SRK-181 is an investigational selective inhibitor of latent TGFβ1 activation and is being developed with the aim of overcoming resistance to checkpoint therapy in patients with advanced cancer.
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Advancing Phase 1 DRAGON proof-of-concept trial. In the second half of 2023, the company expects to provide biomarker and clinical updates from Part B of the DRAGON trial. In
March 2023 ,Scholar Rock presented encore data at theEuropean Society for Medical Oncology Targeted Anticancer Therapies (ESMO TAT)Congress , which showed that SRK-181 continued to be generally well tolerated with early indications of efficacy (as of the data cut-off date ofDecember 2, 2022 ).
Corporate
Announced the addition of
First Quarter 2023 Financial Results
For the quarter ended
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Revenue was
$0 for the quarter endedMarch 31, 2023 , compared to$33.2 million for the quarter endedMarch 31, 2022 . The prior year revenue was related to the Gilead fibrosis-focused research collaboration, which was executed inDecember 2018 and concluded inDecember 2021 .
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Research and development expense was
$29.7 million for the quarter endedMarch 31, 2023 , compared to$29.4 million for the quarter endedMarch 31, 2022 . The increase was primarily attributable to costs associated with clinical trials, including the Phase 3 SAPPHIRE pivotal trial for apitegromab in SMA, and the DRAGON trial for SRK-181. These increases were offset by decreases in employee compensation and benefits costs, resulting from the restructuring inMay 2022 .
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General and administrative expense was
$10.8 million for the quarter endedMarch 31, 2023 , compared to$10.8 million for the quarter endedMarch 31, 2022 .
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As of
March 31, 2023 ,Scholar Rock had cash, cash equivalents, and marketable securities of approximately$275 million , which is expected to fund the company’s anticipated operating and capital expenditure requirements into 2025.
“Scholar Rock is in a strong financial position, and we are relentlessly focused on achieving our goal of improving the lives of patients who may potentially benefit from our medicines,” said
About
Availability of Other Information About Scholar Rock
Investors and others should note that we communicate with our investors and the public using our company website www.scholarrock.com, including, but not limited to, company disclosures, investor presentations and FAQs,
Scholar Rock® is a registered trademark of
Forward-Looking Statements
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 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 “may,” “might,” “could,” “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, without limitation, that preclinical and clinical data, including the results from the Phase 2 clinical trial of apitegromab, or Part A of the Phase 1 clinical trial of SRK-181, are not predictive of, may be inconsistent with, or more favorable than, data generated from future clinical trials of the same product candidates, including, without limitation, the Phase 3 clinical trial of apitegromab in SMA or Part B of the Phase 1 clinical trial of SRK-181; 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; information provided or decisions made by regulatory authorities; competition from third parties that are developing products for similar uses; Scholar Rock’s ability to obtain, maintain and protect its intellectual property; Scholar Rock’s dependence on third parties for development and manufacture of product candidates including, without limitation, to supply any clinical trials; 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, 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’s Quarterly Report on Form 10-Q for the quarter ended
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Condensed Consolidated Statements of Operations |
||||||
(unaudited) |
||||||
(in thousands, except share and per share data) |
||||||
Three Months Ended |
||||||
2023 |
2022 |
|||||
Revenue |
$ |
— |
$ |
33,193 |
||
Operating expenses |
||||||
Research and development |
|
29,735 |
|
29,366 |
||
General and administrative |
|
10,774 |
|
10,760 |
||
Total operating expenses |
|
40,509 |
|
40,126 |
||
Loss from operations |
|
(40,509) |
|
(6,933) |
||
Other income (expense), net |
|
1,130 |
|
(1,017) |
||
Net loss |
$ |
(39,379) |
$ |
(7,950) |
||
Net loss per share, basic and diluted |
$ |
(0.49) |
$ |
(0.21) |
||
Weighted average common shares outstanding, basic and diluted |
|
79,610,059 |
|
37,456,574 |
||
Condensed Consolidated Balance Sheets |
||||||
(unaudited) |
||||||
(in thousands) |
||||||
|
|
|||||
Assets |
||||||
Cash, cash equivalents and marketable securities |
$ |
275,284 |
$ |
315,361 |
||
Other current assets |
|
11,874 |
|
12,663 |
||
Total current assets |
|
287,158 |
|
328,024 |
||
Other assets |
|
27,563 |
|
30,144 |
||
Total assets |
$ |
314,721 |
$ |
358,168 |
||
Liabilities and Stockholders' Equity |
||||||
Current liabilities |
$ |
26,208 |
$ |
36,389 |
||
Long-term liabilities |
|
59,860 |
|
61,544 |
||
Total liabilities |
|
86,068 |
|
97,933 |
||
Total stockholders' equity |
|
228,653 |
|
260,235 |
||
Total liabilities and stockholders' equity |
$ |
314,721 |
$ |
358,168 |
||
View source version on businesswire.com: https://www.businesswire.com/news/home/20230509005251/en/
Investors & Media
Rushmie Nofsinger
rnofsinger@scholarrock.com
ir@scholarrock.com
857-259-5573
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