- Remains on track to report topline data from
pivotal Phase 3 SAPPHIRE trial in patients with Spinal Muscular
Atrophy (SMA) in 4Q 2024
- New data from Phase 2 TOPAZ extension study
in patients with nonambulatory SMA showed sustained clinical
benefit over 48 months, a continued favorable safety profile with
no new safety findings; patient retention rate of over 90%
- Phase 2 EMBRAZE proof-of-concept trial
enrolling ahead of schedule, topline data expected in 2Q 2025
- Presented new SRK-439 preclinical data at
American Diabetes Association’s 84th Scientific Sessions (ADA)
supporting the potential to contribute to a favorable body
composition; increased lean mass and reduced fat mass regain
following withdrawal from GLP-1 receptor agonist treatment
- Management to host update call today at 8:15
a.m. ET
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 second quarter ended June 30, 2024.
“Scholar Rock continues to execute across our portfolio of
highly selective myostatin inhibition programs, further cementing
our position as the global leader in harnessing the life-changing
potential of TGF-beta superfamily biology,” said Jay Backstrom,
M.D., MPH, President & Chief Executive Officer of Scholar Rock.
“With the only muscle-targeted program to demonstrate clinical
proof-of-concept in SMA, our confidence in our lead program
apitegromab continues to be supported by the clinical data
generated over the past four years. At 48 months, over 90% of TOPAZ
patients with nonambulatory SMA remained on apitegromab treatment
on top of SMN therapy and we continued to observe sustained
clinical benefit. We look forward to reporting topline data from
the Phase 3 SAPPHIRE trial of apitegromab in SMA in the fourth
quarter of this year.”
Dr. Backstrom continued, “In addition, we are pleased with the
progress of our cardiometabolic program. The enrollment of the
Phase 2 proof-of-concept EMBRAZE study evaluating apitegromab in
obesity has been advancing ahead of schedule and as a result, we
are updating our guidance for topline data to the second quarter of
2025. We also presented new preclinical data supporting SRK-439’s
potential to help patients retain lean muscle mass at our investor
event in May and at the ADA 84th Scientific Sessions in June. The
hallmark of our approach in designing both apitegromab and SRK-439
is the exquisite selectivity for pro- and latent forms of
myostatin. Our data in SMA suggest that this selectivity matters
for patients, and we are excited to show how SRK-439 can become an
integral component of the treatment and management of obesity
helping to preserve lean muscle mass for sustainable and healthy
weight loss management.”
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.
- Reported that long-term apitegromab data continued to show
substantial and sustained motor function improvements over 48
months1. The mean change in Hammersmith Functional Motor Scale
(HFMSE) from baseline in nonambulatory patients (ages 2-21) on
combination therapy (nusinersen and 20 mg/kg of apitegromab) was
5.3 points (95% CI: 1.5, 9.2; n=23), and for patients 2-12 was 6.4
points (95% CI: 1.8, 11.0, n=19). The mean change in RULM for the
2-21 age group was 3.6 points (95% CI: 2.0, 5.3; N=22) and for the
2-12 age group was 4.5 (95% CI: 2.7, 6.3; n=18). Of the 35
participants in the pooled nonambulatory population, 33 remained in
the study over 4 years. The data analysis excluded the scores of 11
patients after undergoing scoliosis surgery, a known confounding
factor for motor function assessment. Additional details will be
discussed on the conference call this morning.
12-Month Data
24-Month Data
36-Month Data
48-Month Data
Age 2-21 Years Mean Change from
Baseline in HFMSE (95% Confidence Interval)
3.6 points
(1.2, 6.0)
n=32
4.2 points
(1.9, 6.6)
n=29
4.0 points
(1.0, 6.9)
n=28
5.3 points
(1.5, 9.2)
n=23
Age 2-12 Years Mean Change from
Baseline in HFMSE (95% Confidence Interval)
4.6 points
(1.8, 7.4)
n=26
5.2 points
(2.3, 8.0)
n=23
4.8 points
(1.3, 8.3)
n=23
6.4 points
(1.8, 11.0)
n=19
Age 2-21 Years Mean Change from
Baseline in RULM (95% Confidence Interval)
1.3 points
(0.2, 2.3)
n=31
2.3 points
(1.2, 3.3)
n=31
2.4 points
(1.1, 3.7)
n=27
3.6 points
(2.0, 5.3)
n=22
Age 2-12 Years Mean Change from
Baseline in RULM (95% Confidence Interval)
1.2 points
(0.1, 2.4)
n=25
2.2 points
(1.0, 3.5)
n=25
2.8 points
(1.4, 4.2)
n=22
4.5 points
(2.7, 6.3)
n=18
- For the 48-month evaluation, an observed case analysis was
conducted using available data by analysis timepoint, censoring any
HFMSE and RULM assessments after the patient received scoliosis
surgery. The analysis population pooled the nonambulatory patients
(Cohorts 2 and 3) and included patients receiving either low dose
(2 mg/kg) or high dose (20 mg/kg) apitegromab (inclusive of
patients in Cohort 3 who switched from 2 mg/kg to 20 mg/kg in Year
2). A total of 11 patients in the population had scoliosis surgery
during the study and their data was excluded from any HFMSE or RULM
assessments at 48 months. Visit windows were applied to utilize
data from unscheduled or early termination visits if the patient
was missing the HFMSE or RULM total score at the scheduled
visit.
- The ONYX open-label, multicenter extension study is
ongoing. The extension study is evaluating the long-term safety
and efficacy of apitegromab in patients with Type 2 and Type 3 SMA
who completed the TOPAZ or SAPPHIRE trials. More than 90 percent of
patients on combination therapy in the TOPAZ study 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.
- Presented preclinical data from the SRK-439 program. In
May, the Company announced new preclinical data comparing SRK-439
and an anti-activin receptor II (anti-ActRII) antibody that
supported SRK-439’s potential as best in class in preserving lean
muscle mass in patients on GLP-1 receptor agonists (GLP-1 RAs). In
June, the company presented new preclinical data at the American
Diabetes Association 84th Scientific Sessions supporting the
potential of SRK-439 to increase lean mass and contribute to a
favorable body composition following withdrawal from GLP-1 RA
treatment.
- Initiated Phase 2 EMBRAZE proof-of-concept trial with
apitegromab in combination with a GLP-1 receptor agonist (GLP-1 RA)
in obesity in May. The Phase 2 trial is a randomized,
double-blind, placebo-controlled, multi-center study to evaluate
the effect of apitegromab, a highly selective investigational
myostatin inhibitor, to preserve muscle mass as an adjunctive
therapy in overweight and obese adults who are taking a GLP-1 RA.
Data are expected in the second quarter of 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.
Other Pipeline Updates
- New SRK-181 data from the Phase 1 DRAGON proof-of-concept
trial presented at the ASCO Annual Meeting in June. SRK-181 is
an investigational selective inhibitor of latent TGFβ-1 activation
and developed with the aim of overcoming resistance to checkpoint
therapy in patients with advanced cancer. Clinical data showed
encouraging responses in heavily pretreated and anti-PD-(L)1
resistant patients across multiple tumor types. Enrollment of the
DRAGON trial was completed in December 2023, and patients who
remain on the study continue to be treated.
- Published data on SRK-373 was featured on the cover of
Science Signaling in July. The article describes the
selectivity of SRK-373 for LTBP-presented TGFβ-1, as well as
efficacy data from two preclinical models that establish the
feasibility of selectively targeting this particular form of TGFβ-1
for the treatment of fibrosis. SRK-373 is an investigational
selective inhibitor of matrix associated TGFβ-1 in development for
the treatment of fibrosis.
Second Quarter 2024 Financial Results
For the quarter ended June 30, 2024, net loss was $58.5 million
or $0.60 per share compared to a net loss of $37.9 million or $0.47
per share for the quarter ended June 30, 2023.
- The Company did not record any revenue for the quarter ended
June 30, 2024 or for the quarter ended June 30, 2023.
- Research and development expense was $42.4 million for the
quarter ended June 30, 2024, compared to $26.9 million for the
quarter ended June 30, 2023. The increase was primarily
attributable to clinical trial and employee compensation
costs.
- General and administrative expense was $17.1 million for the
quarter ended June 30, 2024, compared to $12.2 million for the
quarter ended June 30, 2023. The increase was due to
employee-related costs.
- As of June 30, 2024, Scholar Rock had cash, cash equivalents,
and marketable securities of approximately $190.5 million, which is
expected to fund the Company’s anticipated operating and capital
expenditure requirements into the second half of 2025.
“Our year-to-date progress across our pipeline of
industry-leading myostatin inhibition programs, combined with our
highly experienced and disciplined team, provides us with a robust
foundation for growth as we advance towards multiple milestones and
our potential evolution into a commercial-stage company,” said Ted
Myles, Chief Operating Officer and Chief Financial Officer of
Scholar Rock.
Conference Call Information
Management will provide an update on the Company and discuss
second quarter 2024 results via conference call on Thursday, August
8 at 8:15 am ET. To access the live conference call, participants
may register here. The live audio webcast of the call will be
available under “Events and Presentations” in the Investor
Relations section of the Scholar Rock website at
http://investors.scholarrock.com. To participate via telephone,
please register in advance here. Upon registration, all telephone
participants will receive a confirmation email detailing how to
join the conference call, including the dial-in number along with a
unique passcode and registrant ID that can be used to access the
call. An archived replay of the webcast will be available on the
Company’s website for approximately 90 days.
About Apitegromab
Apitegromab is an investigational fully human monoclonal
antibody inhibiting myostatin activation by selectively binding the
pro- and latent forms of myostatin in the skeletal muscle. It is
the first muscle-targeted treatment candidate to demonstrate
clinical proof-of-concept in spinal muscular atrophy (SMA).
Myostatin, a member of the TGFβ superfamily of growth factors, is
expressed primarily by skeletal muscle cells, and the absence of
its gene is associated with an increase in muscle mass and strength
in multiple animal species, including humans. Scholar Rock believes
that its highly selective targeting of pro- and latent forms of
myostatin with apitegromab may lead to a clinically meaningful
improvement in motor function in patients with SMA. The U.S. Food
and Drug Administration (FDA) has granted Fast Track, Orphan Drug
and Rare Pediatric Disease designations, and the European Medicines
Agency (EMA) has granted Priority Medicines (PRIME) and Orphan
Medicinal Product designations, to apitegromab for the treatment of
SMA. The efficacy and safety of apitegromab have not been
established and apitegromab has not been approved for any use by
the FDA or any other regulatory agency.
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 EMBRAZE
EMBRAZE is a randomized, double-blind, placebo-controlled, Phase
2 proof-of-concept trial evaluating the efficacy, safety and
pharmacokinetics of apitegromab in adults with a body mass index
(BMI) of >27 (overweight) or a BMI of >30 (obese) and taking
a GLP-1 RA (tirzepatide or semaglutide). The target enrollment of
EMBRAZE is 100 subjects aged 18-65 who are overweight or obese
without diabetes. As part of the study design, the treatment period
is 24 weeks, and all subjects will receive a GLP-1 RA. In addition,
all subjects will be randomized 1:1 to receive either apitegromab
or placebo by intravenous (IV) infusion every four weeks during the
24-week treatment period. The primary endpoint is change from
baseline at Week 24 in lean mass assessed by dual-energy X-ray
absorptiometry. Secondary endpoints include additional weight loss
measures, safety and tolerability, and pharmacokinetic outcomes.
Exploratory endpoints at Weeks 24 and 32 include cardiometabolic
parameters (e.g. HbA1c), body composition, and physical
function.
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 cardiometabolic disorders, including obesity. Based on
preclinical data, SRK-439 has the potential to support healthier
weight management by preserving lean mass during weight loss. 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, and our ability to continue as a going
concern; 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 June 30, 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 June
30,
Six Months Ended June
30,
2024
2023
2024
2023
Operating expenses
Research and development
$
42,373
$
26,867
$
85,466
$
56,602
General and administrative
17,125
12,215
32,451
22,989
Total operating expenses
59,498
39,082
117,917
79,591
Loss from operations
(59,498
)
(39,082
)
(117,917
)
(79,591
)
Other income (expense), net
990
1,157
2,556
2,287
Net loss
$
(58,508
)
$
(37,925
)
$
(115,361
)
$
(77,304
)
Net loss per share, basic and diluted
$
(0.60
)
$
(0.47
)
$
(1.20
)
$
(0.97
)
Weighted average common shares
outstanding, basic and diluted
96,813,116
80,117,983
96,352,858
79,865,424
Scholar Rock Holding
Corporation
Condensed Consolidated Balance
Sheets
(unaudited)
(in thousands)
June 30, 2024
December 31, 2023
Assets
Cash, cash equivalents and marketable
securities
$
190,494
$
279,938
Other current assets
8,643
8,256
Total current assets
199,137
288,194
Other assets
27,728
22,841
Total assets
$
226,865
$
311,035
Liabilities and Stockholders'
Equity
Current liabilities
$
32,987
$
32,741
Long-term liabilities
60,258
53,076
Total liabilities
93,245
85,817
Total stockholders' equity
133,620
225,218
Total liabilities and stockholders'
equity
$
226,865
$
311,035
View source
version on businesswire.com: https://www.businesswire.com/news/home/20240808052500/en/
Scholar Rock:
Investors Rushmie Nofsinger Scholar Rock
rnofsinger@scholarrock.com ir@scholarrock.com 857-259-5573
Media Molly MacLeod Scholar Rock mmacleod@scholarrock.com
media@scholarrock.com 802-579-5995
Scholar Rock (NASDAQ:SRRK)
Historical Stock Chart
From Nov 2024 to Dec 2024
Scholar Rock (NASDAQ:SRRK)
Historical Stock Chart
From Dec 2023 to Dec 2024