Scholar Rock Reports Second Quarter 2022 Financial Results and Highlights Business Progress
Scholar Rock (NASDAQ: SRRK) reported positive 24-month data from its Phase 2 TOPAZ trial, supporting apitegromab's potential for Spinal Muscular Atrophy (SMA) patients. The company is actively enrolling for its pivotal Phase 3 SAPPHIRE trial. Financially, Scholar Rock completed a $205 million direct offering, strengthening its balance sheet, while net loss widened to $44 million for Q2 2022, with revenues dropping to $0 from $4.6 million a year prior. R&D expenses increased due to higher clinical costs for apitegromab.
- Positive 24-month data from the Phase 2 TOPAZ trial showing sustained motor function improvement in SMA patients.
- Ongoing enrollment in the pivotal Phase 3 SAPPHIRE trial for apitegromab.
- Completion of a $205 million registered direct offering, strengthening the financial position.
- Q2 2022 net loss increased to $44 million from $30.7 million in Q2 2021.
- Revenue fell to $0 in Q2 2022 from $4.6 million in Q2 2021.
- R&D expenses rose to $32.1 million, primarily due to increased clinical costs and severance expenses.
- Positive 24-month data from ongoing Phase 2 TOPAZ trial extension supports potential benefit of apitegromab for patients with Types 2 and 3 Spinal Muscular Atrophy (SMA)
- Actively enrolling in pivotal Phase 3 SAPPHIRE clinical trial for apitegromab
- Discovery pipeline advancements made for LTBP-TGFβ1 target to treat a wide range of fibrotic diseases
- Significantly strengthened balance sheet with the completion of
“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
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).
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Positive 24-Month Phase 2 TOPAZ Extension Data Support Potential Benefit for Patients.
Scholar Rock announced positive data at theCure 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. andEurope .Scholar Rock presented on the trial design at the 17thInternational Congress on Neuromuscular Diseases (ICNMD 2022) inJuly 2022 , along with publication of the abstract in the peer-reviewedJournal 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”).
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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
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Revenue was
for the quarter ended$0 June 30, 2022 , compared to for the quarter ended$4.6 million June 30, 2021 .
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Research and development expense was
for the quarter ended$32.1 million June 30, 2022 , compared to for the quarter ended$25.6 million 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.
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General and administrative expense was
for the quarter ended$11.1 million June 30, 2022 , compared to for the quarter ended$9.3 million June 30, 2021 . The increase was primarily due to higher personnel costs, including severance expenses associated with the recent restructuring.
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As of
June 30, 2022 ,Scholar Rock had cash, cash equivalents, and marketable securities of approximately , which is expected to fund the Company’s anticipated operating and capital expenditure requirements into 2025.$371 million
“We were thrilled to announce the completion of a
About
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 candidate, 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
Condensed Consolidated Statements of Operations | |||||||||||||||
(unaudited) | |||||||||||||||
(in thousands, except share and per share data) | |||||||||||||||
Three Months Ended |
Six Months Ended |
||||||||||||||
2022 |
2021 |
2022 |
2021 |
||||||||||||
Revenue | $ |
— |
|
$ |
4,595 |
|
$ |
33,193 |
|
$ |
9,303 |
|
|||
Operating expenses | |||||||||||||||
Research and development |
|
32,073 |
|
|
25,603 |
|
|
61,439 |
|
|
48,152 |
|
|||
General and administrative |
|
11,074 |
|
|
9,265 |
|
|
21,834 |
|
|
18,631 |
|
|||
Total operating expenses |
|
43,147 |
|
|
34,868 |
|
|
83,273 |
|
|
66,783 |
|
|||
Loss from operations |
|
(43,147 |
) |
|
(30,273 |
) |
|
(50,080 |
) |
|
(57,480 |
) |
|||
Other income (expense), net |
|
(853 |
) |
|
(434 |
) |
|
(1,870 |
) |
|
(898 |
) |
|||
Net loss | $ |
(44,000 |
) |
$ |
(30,707 |
) |
$ |
(51,950 |
) |
$ |
(58,378 |
) |
|||
Net loss per share, basic and diluted | $ |
(1.06 |
) |
$ |
(0.84 |
) |
$ |
(1.31 |
) |
$ |
(1.60 |
) |
|||
Weighted average common shares outstanding, basic and diluted |
|
41,622,392 |
|
|
36,582,708 |
|
|
39,550,991 |
|
|
36,482,132 |
|
Condensed Consolidated Balance Sheets | ||||||
(unaudited) | ||||||
(in thousands) | ||||||
Assets | ||||||
Cash, cash equivalents and marketable securities | $ |
370,688 |
$ |
252,994 |
||
Other current assets |
|
18,451 |
|
12,325 |
||
Total current assets |
|
389,139 |
|
265,319 |
||
Other assets |
|
34,989 |
|
39,126 |
||
Total assets | $ |
424,128 |
$ |
304,445 |
||
Liabilities and Stockholders' Equity | ||||||
Current liabilities | $ |
40,162 |
$ |
64,297 |
||
Long-term liabilities |
|
54,459 |
|
68,074 |
||
Total liabilities |
|
94,621 |
|
132,371 |
||
Total stockholders' equity |
|
329,507 |
|
172,074 |
||
Total liabilities and stockholders' equity | $ |
424,128 |
$ |
304,445 |
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Investors
Rushmie Nofsinger
rnofsinger@scholarrock.com
ir@scholarrock.com
857-259-5573
Media
ariane.lovell@finnpartners.com
media@scholarrock.com
917-565-2204
Source:
FAQ
What were the Q2 2022 financial results for Scholar Rock (SRRK)?
What is the status of the Phase 3 SAPPHIRE trial for apitegromab?
What positive data was reported from the TOPAZ trial for Scholar Rock (SRRK)?