- Apitegromab shows transformative potential in
spinal muscular atrophy (SMA) with 74% of patients with
non-ambulatory Type 2 and 3 SMA in the TOPAZ Phase 2 trial
attaining a clinical improvement in HFMSE at 12 months
- Mean increase from baseline in HFMSE of 6.2
points (across both evaluated doses) observed in younger
non-ambulatory patients; up to 20-point increases in HFMSE observed
with 35% of patients demonstrating a >10-point increase
- Anticipate initiating apitegromab Phase 3
trial by year-end 2021 in patients with non-ambulatory Type 2 and 3
SMA, representing approximately 2/3 of SMA patient population
- Dose escalation continues to progress in
DRAGON Phase 1 trial evaluating SRK-181 in combination with
anti-PD-(L)1 therapy; initial data anticipated by year-end 2021
Scholar Rock (NASDAQ: SRRK), a clinical-stage biopharmaceutical
company focused on the treatment of serious diseases in which
protein growth factors play a fundamental role, today reported
financial results for the first quarter ended March 31, 2021 and
highlighted recent progress and upcoming milestones for its
pipeline programs.
Company Updates and Upcoming Milestones
Apitegromab is a selective
inhibitor of myostatin activation being developed as the potential
first muscle-directed therapy for the treatment of spinal muscular
atrophy (SMA).
- TOPAZ 12-Month Data Show Transformative Potential in SMA of
Apitegromab as Add-on to SMN Upregulator Therapy. In April
2021, positive top-line data were announced from the TOPAZ Phase 2
trial (NCT03921528) in patients with Type 2 and Type 3 SMA. Both
younger and older non-ambulatory patients treated with apitegromab
and background SMN upregulator therapy over the 12-month period
attained meaningful motor function improvements with 74% (23/31) of
patients demonstrating a clinical improvement of ≥1-point increase
in Hammersmith Functional Motor Scale Expanded (HFMSE).
- In the younger non-ambulatory TOPAZ cohort (ages 2-6 years),
treatment with apitegromab led to a mean increase from baseline in
HFMSE of +6.2 points across both evaluated doses; a mean increase
of +7.1 points for the 20 mg/kg dose and +5.3 points with the 2
mg/kg dose. Increase in HFMSE from baseline across both apitegromab
doses: − 82% (14/17) of patients attained a >1-point increase − 59% (10/17) of patients
attained a >3-point increase − 59%
(10/17) of patients attained a >5-point increase − 35% (6/17) of patients
attained a >10-point increase
- In the older non-ambulatory TOPAZ cohort (age 8-19 years),
treatment with apitegromab 20 mg/kg led to a mean increase from
baseline in HFMSE of +0.6 points by the primary intent-to-treat
analysis and +1.2 points by the prespecified per protocol analysis.
Increase in HFMSE from baseline for this patient population: − 64%
(9/14) of patients attained a >1-point increase − 29% (4/14) of patients
attained a >3-point increase
Patients enrolled in TOPAZ were receiving
chronic maintenance doses of nusinersen; both the younger and older
non-ambulatory cohorts had received more than five mean maintenance
doses of nusinersen (approximately two years of treatment) at
baseline. Clinical data from the CHERISH and SHINE studies of
nusinersen offer background insights into this patient population.1
These studies observed that nusinersen-treated patients primarily
experience stabilization or only slight increases in HFMSE scores
beyond the initial 15-month treatment period.2 In addition, even in
the initial 15-month treatment period, older patients (age 5-12) on
average experience declines in HFMSE and rarely attain a
>3-point increase in HFMSE in a
12-month timeframe.3
The five most frequently reported
treatment-emergent adverse events included headache, pyrexia, upper
respiratory tract infection, cough, and nasopharyngitis. All 57
patients who completed the 12-month treatment period elected to opt
into the extension period.
- Phase 3 Registrational Trial Evaluating Apitegromab in
Patients with Non-Ambulatory Type 2 and 3 Patients Anticipated to
Initiate by Year-End 2021. TOPAZ Phase 2 top-line efficacy and
safety results support the continued evaluation of apitegromab in a
Phase 3 trial. Subject to feedback from regulatory agencies,
Scholar Rock intends to conduct a Phase 3 trial to evaluate
apitegromab with background SMN upregulator therapy in
non-ambulatory Type 2 and Type 3 SMA, the patient population where
apitegromab demonstrated the largest increases in motor function
(HFMSE scores) in TOPAZ. Patients with non-ambulatory Type 2 and
Type 3 SMA represent approximately two-thirds of the overall
prevalent SMA patient population.
- Additional Data and Analyses from the TOPAZ Trial to be
Presented at Upcoming Medical Congresses. Scholar Rock is
conducting additional data analyses, including exploratory analyses
using patient-level data, evaluating additional outcome measures,
and reviewing additional safety data from the TOPAZ Phase 2 trial.
The Company plans to present the TOPAZ top-line results as well as
findings from these analyses at upcoming medical congresses.
- Apitegromab Granted Priority Medicines (PRIME) Designation
by the European Medicines Agency (EMA). In March 2021, the EMA
granted PRIME designation to apitegromab, recognizing its potential
to address unmet medical needs of patients with SMA. With the PRIME
designation, Scholar Rock has greater access and enhanced dialogue
with the regulatory agency to optimize development plans and
evaluations.
- Two U.S. Patents Issued Adding Further Protection for
Inhibitors of Myostatin Activation and Related Methods, Including
Apitegromab. In March 2021, the United States Patent Office
(USPTO) issued U.S. Patent No. 10,946,036 with an expiry of June
2037, protecting both add-on and combination therapy with a
myostatin inhibitor and a neuronal corrector therapy (such as SMN
upregulator therapy) for the treatment of SMA. In April 2021, the
USPTO issued U.S. Patent No. 10,981,981 with an expiry of May 2034,
broadly covering methods for making inhibitors of myostatin (GDF8)
activation based on Scholar Rock’s proprietary platform approach of
targeting the precursor forms of growth factors.
- Additional Potential Indications Identified for
Apitegromab. Scholar Rock has identified multiple diseases for
which the selective inhibition of the activation of myostatin may
offer therapeutic benefit, including additional patient populations
in SMA (such as Type 1 SMA and ambulatory Type 3 SMA) and potential
indications outside of SMA, such as Becker Muscular Dystrophy
(BMD).
SRK-181 is a selective
inhibitor of latent TGFβ1 activation being developed with the aim
of overcoming resistance to and increasing the number of patients
who may benefit from checkpoint inhibitor therapy.
- Published Preclinical Development Data for SRK-181 in
International Journal of Toxicology. In March 2021, Scholar
Rock announced the publication of “Nonclinical Development of
SRK-181: An Anti-latent TGFβ1 Monoclonal Antibody for the Treatment
of Locally Advanced or Metastatic Solid Tumors,” in the
peer-reviewed International Journal of Toxicology. This publication
provided a comprehensive preclinical assessment of the
pharmacology, pharmacokinetics, and safety of SRK-181, which
provided support for the dose selection strategy for the ongoing
DRAGON Phase 1 trial.
- Initial Clinical Response and Safety Data from Part A of the
DRAGON Phase 1 Trial Anticipated by Year-End 2021. SRK-181 is
being evaluated in the two-part DRAGON trial (NCT04291079) in
patients with locally advanced or metastatic solid tumors
exhibiting primary resistance to anti-PD-(L)1 therapy. Dose
escalation continues to progress in Part A of the trial, which
evaluates the safety and pharmacokinetics of SRK-181, and the
Company expects to advance to the Part B dose expansion portion of
the trial in mid-2021. Part B will consist of multiple cohorts,
including urothelial carcinoma, cutaneous melanoma, non-small cell
lung cancer, and other solid tumors. Each cohort will enroll up to
40 patients who have demonstrated primary resistance to
anti-PD-(L)1 therapy and will be treated with SRK-181 in
combination with an approved anti-PD-(L)1 therapy.
“2021 is off to a great start as we are delighted with the
positive 12-month top-line data from our TOPAZ study, which shows
the transformative potential of apitegromab in SMA,” said Tony
Kingsley, President and CEO of Scholar Rock. “Based on these
exciting results and the potential to bring apitegromab to the SMA
community, we will be working with urgency on behalf of patients to
initiate our Phase 3 registrational trial in patients with
non-ambulatory Type 2 and 3 SMA by the end of the year.”
First Quarter 2021 Financial Results
For the quarter ended March 31, 2021, net loss was $27.7 million
or $0.76 per share compared to a net loss of $17.1 million or $0.58
per share for the quarter ended March 31, 2020.
- Revenue was $4.7 million for the quarter ended March 31, 2021
compared to $5.0 million for the quarter ended March 31, 2020 and
was related to the Gilead fibrosis-focused collaboration (the
“Gilead Collaboration Agreement”) that was executed in December
2018.
- Research and development expense was $22.5 million for the
quarter ended March 31, 2021 compared to $16.9 million for the
quarter ended March 31, 2020. The increase year-over-year primarily
reflects manufacturing costs associated with apitegromab and higher
personnel and facility-related costs, partially offset by lower
manufacturing costs associated with SRK-181.
- General and administrative expense was $9.4 million for the
quarter ended March 31, 2021 compared to $5.8 million for the
quarter ended March 31, 2020. The increase year-over-year was
primarily attributed to higher personnel and facility-related
costs.
“We are focused on advancing our portfolio of clinical and
preclinical programs with several near-term milestones, including
the initiation of a Phase 3 trial evaluating apitegromab in SMA,
completion of dose escalation in Part A and initiation of the Part
B dose expansion portion of the DRAGON Phase 1 trial of SRK-181 in
patients with solid tumors,” said Ted Myles, CFO and Head of
Business Operations of Scholar Rock. “We are well positioned to
continue to execute on our plans as we ended the first quarter with
approximately $315 million in cash, which can fund operations into
2023.”
1This information from third-party studies is provided for
background purposes only and is not intended to convey or imply a
comparison to the TOPAZ clinical trial results. 2 Source:
“Longer-term treatment with nusinersen: results in later-onset
spinal muscular atrophy from the SHINE study” P.257, World Muscle
Society Congress 2020 3 Source: Mercuri E, et.al. Nusinersen versus
sham control in later-onset spinal muscular atrophy. N Engl J Med.
2018;378:625-635.
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, and
fibrosis. Scholar Rock’s approach to targeting 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
believes its focus on biologically validated growth factors may
facilitate a more efficient development path. For more information,
please visit www.ScholarRock.com or follow Scholar Rock on Twitter
(@ScholarRock) and LinkedIn
(https://www.linkedin.com/company/scholar-rock/).
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, 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,” “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 preclinical and clinical data, including the
12-month top-line results from the Phase 2 trial of apitegromab,
are not predictive of, are inconsistent with, or more favorable
than, data generated from future clinical trials of the same
product candidate, including the planned Phase 3 trial of
apitegromab in SMA, 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, 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, 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 March
31, 2021, 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
2021
2020
Revenue
$
4,708
$
5,030
Operating expenses Research and development
22,549
16,902
General and administrative
9,366
5,822
Total operating expenses
31,915
22,724
Loss from operations
(27,207
)
(17,694
)
Other income (expense), net
(464
)
624
Net loss
$
(27,671
)
$
(17,070
)
Net loss per share, basic and diluted
$
(0.76
)
$
(0.58
)
Weighted average common shares outstanding, basic and
diluted
36,380,438
29,527,349
Scholar Rock Holding
Corporation
Condensed Consolidated Balance
Sheets
(unaudited)
(in thousands)
March 31, 2021
December 31, 2020
Assets Cash, cash equivalents and
marketable securities
$
314,689
$
341,031
Other current assets
3,803
3,373
Total current assets
318,492
344,404
Other assets
42,094
43,901
Total assets
$
360,586
$
388,305
Liabilities and Stockholders'
Equity Current liabilities
$
69,955
$
42,564
Long-term liabilities
50,091
84,971
Total liabilities
120,046
127,535
Total stockholders' equity
240,540
260,770
Total liabilities and stockholders' equity
$
360,586
$
388,305
View source
version on businesswire.com: https://www.businesswire.com/news/home/20210513005216/en/
Investors Catherine Hu chu@scholarrock.com
Media Ariane Lovell Finn Partners
ariane.lovell@finnpartners.com 917-565-2204
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