- On track to report topline data from pivotal
Phase 3 SAPPHIRE trial in patients with Spinal Muscular Atrophy in
4Q 2024
- Initiating Phase 2 proof-of-concept trial
evaluating apitegromab in obesity in June 2024
- Updated SRK-181 clinical data selected for
oral presentation at American Society of Clinical Oncology
(ASCO)
- New SRK-439 preclinical data selected for
oral presentation at American Diabetes Association (ADA)
- Cash, cash equivalents and marketable
securities of $238 million as of March 31, 2024; expected to
support runway into 2H 2025
Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical
company focused on advancing innovative treatments for spinal
muscular atrophy (SMA), cardiometabolic disorders, and other
serious diseases where protein growth factors play a fundamental
role, today reported financial results and corporate updates for
the first quarter ended March 31, 2024.
“Scholar Rock is now only two quarters away from reporting
topline data for our pivotal Phase 3 SAPPHIRE trial, which has the
potential to build upon the promising data generated from our prior
Phase 2 trial,” said Jay Backstrom, M.D., MPH, President &
Chief Executive Officer of Scholar Rock. “As we look towards a
potential commercial launch of apitegromab in SMA in 2025, we’re
thrilled to be on the precipice of delivering a new class of
treatment with potential to meaningfully impact people living with
SMA.”
Dr. Backstrom continued, “In parallel, we continue to make
important progress with our cardiometabolic program. As we advance
SRK-439 towards IND, we plan to present new preclinical data at
upcoming conferences that further support SRK-439’s differentiated
product profile. Additionally, we remain on track to open
enrollment in June for our Phase 2 proof-of-concept trial
evaluating apitegromab in combination with GLP-1 agonist treatment
in obesity, which is designed to assess the effect of our selective
approach to blocking myostatin to preserve lean muscle mass as part
of healthy weight management. As a leader in selective myostatin
inhibition, it is a priority for us to apply our expertise in the
cardiometabolic arena, where safe preservation of lean muscle mass
is important.”
Company Highlights and Upcoming Milestones
SMA Program
Apitegromab is an
investigational, fully human monoclonal antibody that inhibits
myostatin activation by selectively binding the pro- and latent
forms of myostatin in skeletal muscle and is being developed as a
potential first muscle-targeted therapy for the treatment of SMA.
Apitegromab is the only muscle-targeted therapy to show clinical
proof-of-concept in SMA.
- On track to report topline data from Phase 3 SAPPHIRE
clinical trial in 4Q 2024. If the trial is successful and
apitegromab is approved, the Company expects to initiate a
commercial product launch in 2025.
- ONYX long-term extension study ongoing for patients from
both the TOPAZ and SAPPHIRE studies. More than 90% of patients
on combination therapy have completed 4 years of apitegromab
treatment and enrolled into ONYX.
Cardiometabolic Program
SRK-439 is a novel,
preclinical, investigational myostatin inhibitor that has high in
vitro affinity for pro- and latent myostatin and maintains
myostatin specificity (i.e., no GDF11 or Activin A binding), and is
initially being developed for the treatment of obesity.
- Initiating a Phase 2 proof-of-concept trial with apitegromab
in combination with a GLP-1 receptor agonist (GLP-1 RA) in obesity
in June. The Phase 2 trial is a randomized, double-blind,
placebo-controlled, multi-center study to evaluate the effect of
apitegromab, a highly selective myostatin inhibitor, to preserve
lean muscle mass as an adjunctive therapy in overweight and obese
adults who are taking a GLP-1 RA. Data is expected in mid-2025 and
will be used to guide clinical development of SRK-439. The Company
plans to file an IND for SRK-439 for the treatment of obesity in
2025.
- Preclinical data from the SRK-439 program selected for an
oral presentation during the American Diabetes Association 84th
Scientific Sessions being held June 21-24, 2024 in Orlando,
Florida. The presentation will include updates on SRK-439 and
will be held on June 23 at 2:45 p.m. ET.
Immuno-Oncology Program
SRK-181 is an
investigational selective inhibitor of latent TGFβ1 activation and
developed with the aim of overcoming resistance to checkpoint
therapy in patients with advanced cancer.
- New SRK-181 data from the Phase 1 DRAGON proof-of-concept
trial selected for an oral presentation at the ASCO Annual Meeting
being held May 31- June 4, 2024 in Chicago. Enrollment of the
DRAGON trial was completed in December 2023, and patients who
remain on the study continue to be treated. The presentation,
“Phase 1 study (DRAGON) of SRK-181 (linavonkibart), a latent TGFβ1
inhibitor, combined with pembrolizumab in anti-PD1 resistant
patients with advanced solid tumors: updated results of expansion
part,” will be presented on June 3 at 1:50 p.m. CT/2:50 p.m. ET.
The Company will hold a webcast to discuss the data from the ASCO
presentation on June 4 at 8:00 a.m. ET.
Corporate
- Company hosting Investor & Analyst Day on May 22, 2024
in New York City. Event to highlight the Company’s myostatin
inhibition programs in SMA and obesity.
First Quarter 2024 Financial Results
For the quarter ended March 31, 2024, net loss was $56.9 million
or $0.59 per share compared to a net loss of $39.4 million or $0.49
per share for the quarter ended March 31, 2023.
- The Company did not record any revenue for the quarter ended
March 31, 2024 or for the quarter ended March 31, 2023.
- Research and development expense was $43.1 million for the
quarter ended March 31, 2024, compared to $29.7 million for the
quarter ended March 31, 2023. The increase was primarily
attributable to clinical trial and research study costs.
- General and administrative expense was $15.3 million for the
quarter ended March 31, 2024, compared to $10.8 million for the
quarter ended March 31, 2023. The increase was due to
employee-related costs.
- As of March 31, 2024, Scholar Rock had cash, cash equivalents,
and marketable securities of approximately $238 million, which is
expected to fund the Company’s anticipated operating and capital
expenditure requirements into the second half of 2025.
“We are acutely focused on execution in 2024 as we prepare for
the pivotal Phase 3 readout of apitegromab in SMA and continue to
progress our cardiometabolic program through clinical development.
Our cash position enables us to reach multiple upcoming key
milestones as we prepare for our next phase of growth,” said Ted
Myles, Chief Operating Officer and Chief Financial Officer of
Scholar Rock.
About the Phase 3 SAPPHIRE Trial
SAPPHIRE is an ongoing randomized, double-blind,
placebo-controlled, Phase 3 clinical trial evaluating the safety
and efficacy of apitegromab in nonambulatory patients with Types 2
and 3 SMA who are receiving SMN-targeted therapy (either nusinersen
or risdiplam). SAPPHIRE targeted enrolling approximately 156
patients aged 2-12 years old in the main efficacy population. These
patients were randomized 1:1:1 to receive for 12 months either
apitegromab 10 mg/kg, apitegromab 20 mg/kg, or placebo by
intravenous (IV) infusion every 4 weeks. An exploratory population
that targeted enrolling up to 48 patients aged 13-21 years old will
also separately be evaluated. These patients were randomized 2:1 to
receive either apitegromab 20 mg/kg or placebo. For more
information about SAPPHIRE, visit www.clinicaltrials.gov.
Apitegromab has not been approved for any use by the US FDA or any
other health authority, and its safety and efficacy have not been
established.
About SRK-439
SRK-439 is a novel, preclinical, investigational myostatin
inhibitor that has high in vitro affinity for pro- and latent
myostatin and maintains myostatin specificity (i.e., no GDF11 or
Activin-A binding), and is initially being developed for the
treatment of obesity. Based on preclinical data, SRK-439 has the
potential to support healthier weight management by preserving lean
mass. The efficacy and safety of SRK-439 have not been established
and SRK-439 has not been approved for any use by the FDA or any
other regulatory agency.
About Scholar Rock
Scholar Rock is a biopharmaceutical company that discovers,
develops, and delivers life-changing therapies for people with
serious diseases that have high unmet need. As a global leader in
the biology of the transforming growth factor beta (TGFβ)
superfamily of cell proteins and named for the visual resemblance
of a scholar rock to protein structures, the clinical-stage company
is focused on advancing innovative treatments where protein growth
factors are fundamental. Over the past decade, Scholar Rock has
created a pipeline with the potential to advance the standard of
care for neuromuscular disease, cardiometabolic disorders, cancer,
and other conditions where growth factor-targeted drugs can play a
transformational role.
Scholar Rock is the only company to show clinical proof of
concept for a muscle-targeted treatment in spinal muscular atrophy
(SMA). This commitment to unlocking fundamentally different
therapeutic approaches is powered by broad application of a
proprietary platform, which has developed novel monoclonal
antibodies to modulate protein growth factors with extraordinary
selectivity. By harnessing cutting-edge science in disease spaces
that are historically under-addressed through traditional
therapies, Scholar Rock works every day to create new possibilities
for patients. Learn more about our approach at ScholarRock.com and
follow @ScholarRock and on LinkedIn.
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, Securities and
Exchange Commission filings, press releases, public conference call
transcripts and webcast transcripts, as well as on Twitter and
LinkedIn. The information that we post on our website or on Twitter
or LinkedIn could be deemed to be material information. As a
result, we encourage investors, the media and others interested to
review the information that we post there on a regular basis. The
contents of our website or social media shall not be deemed
incorporated by reference in any filing under the Securities Act of
1933, as amended.
Scholar Rock® is a registered trademark of Scholar Rock,
Inc.
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 and SRK-181 and its preclinical programs, including
SRK-439, and indication selection and development timing, including
the therapeutic potential, clinical benefits and safety thereof,
expectations regarding timing, success and data announcements of
current ongoing preclinical and clinical trials, its cash runway,
expectations regarding the achievement of important milestones, 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 or Part B 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 or ongoing
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; 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 March 31, 2024, as well
as discussions of potential risks, uncertainties, and other
important factors in Scholar Rock’s subsequent filings with the
Securities and Exchange Commission. Any forward-looking statements
represent Scholar Rock’s 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.
Scholar Rock Holding
Corporation
Condensed Consolidated
Statements of Operations
(unaudited)
(in thousands, except share and
per share data)
Three Months Ended March
31
2024
2023
Operating expenses
Research and development
$
43,094
$
29,735
General and administrative
15,325
10,774
Total operating expenses
58,419
40,509
Loss from operations
(58,419
)
(40,509
)
Other income (expense), net
1,566
1,130
Net loss
$
(56,853
)
$
(39,379
)
Net loss per share, basic and diluted
$
(0.59
)
$
(0.49
)
Weighted average common shares
outstanding, basic and diluted
95,892,601
79,610,059
Scholar Rock Holding
Corporation
Condensed Consolidated Balance
Sheets
(unaudited)
(in thousands)
March 31, 2024
December 31, 2023
Assets
Cash, cash equivalents and marketable
securities
$
238,432
$
279,938
Other current assets
8,501
8,256
Total current assets
246,933
288,194
Other assets
20,352
22,841
Total assets
$
267,285
$
311,035
Liabilities and Stockholders'
Equity
Current liabilities
$
37,506
$
32,741
Long-term liabilities
47,006
53,076
Total liabilities
84,512
85,817
Total stockholders' equity
182,773
225,218
Total liabilities and stockholders'
equity
$
267,285
$
311,035
View source
version on businesswire.com: https://www.businesswire.com/news/home/20240507572680/en/
Scholar Rock:
Investors & Media Rushmie Nofsinger Scholar
Rock rnofsinger@scholarrock.com ir@scholarrock.com 857-259-5573
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