Scholar Rock Provides Corporate Update and Outlines Key R&D Priorities for 2020
January 10 2020 - 6:30AM
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 highlighted key accomplishments of the past year and
announced R&D priorities for 2020.
Key Accomplishments:
- SRK-015: highly specific inhibitor of latent
myostatin activation being developed for the treatment of Spinal
Muscular Atrophy (SMA).
- Initiated and completed enrollment of the TOPAZ Phase 2
proof-of-concept trial of SRK-015 in patients with Type 2 and Type
3 SMA.
- Preliminary pharmacokinetic (PK) and pharmacodynamic (PD) data
from 29 patients in the TOPAZ trial are consistent with robust
target engagement in patients with SMA, providing the first
evidence of successful pharmacologic engagement of a latent growth
factor in a human disease setting.
- No clinically significant safety signals had been observed in
TOPAZ as of the data cutoff for the preliminary PK/PD
analysis.
- SRK-181: potent and selective inhibitor of
latent transforming growth factor beta 1 (TGFβ1) activation aimed
at expanding anti-tumor responses from immunotherapy by overcoming
primary resistance to checkpoint inhibitor therapies.
- Accelerated non-clinical development and submitted an
Investigational New Drug (IND) application to the U.S. Food and
Drug Administration (FDA), supporting plans to initiate a Phase 1
proof-of-concept trial in patients with solid tumors in the first
quarter of 2020.
- Demonstrated preclinically that Scholar Rock’s highly selective
inhibitor of latent TGFβ1 activation rendered resistant solid
tumors vulnerable to PD-1 blockade and drove tumor regression in
syngeneic mouse tumor models emulating key features of clinically
observed primary resistance.
- Demonstrated preclinically the potential of SRK-181 for reduced
toxicity that has historically limited drug exposure from
non-selective TGFβ inhibition. In a pilot toxicology study,
treatment of adult rats with SRK-181 up to a weekly dose of
100mg/kg for four weeks had an improved toxicity profile and
avoided the cardiovascular toxicity observed with a non-selective
TGFβ antibody and an ALK5 inhibitor. In addition, no adverse
effects were observed up to the highest evaluated dose of 200 mg/kg
per week in a 4-week GLP rat toxicology study and 300 mg/kg per
week in a 4-week GLP non-human primate toxicology study.
- Preclinical research programs:
- Achieved first milestone and earned a $25 million payment in
strategic collaboration with Gilead Sciences, Inc. by demonstrating
that highly selective inhibitors of latent TGFβ1 activation are
efficacious in relevant preclinical models of fibrotic diseases.
Scholar Rock is advancing multiple collaboration programs toward
product candidate selection.
- Continued to progress preclinical pipeline programs for
neuromuscular disorders, cancers, fibrosis, and anemias.
“The past 12 months were a time of remarkable progress for
Scholar Rock across all of our R&D programs. We rapidly
advanced our SRK-015 program in SMA by completing enrollment in the
TOPAZ trial in just eight months, achieved the initial catalytic
milestone in our fibrosis-focused collaboration with Gilead, and
submitted our IND application for SRK-181 for cancer immunotherapy
at the end of the year,” said Nagesh Mahanthappa, Ph.D., President
and CEO of Scholar Rock. “As we begin 2020 with great momentum, we
look forward to further advancing our robust pipeline in the year
ahead. Importantly, with the recent implication of TGFβ1 activity
as a major checkpoint inhibitor resistance mechanism in patients,
we believe that SRK-181 holds unique potential to meaningfully
broaden clinical responses to PD-(L)1 inhibitors. With our plans to
initiate enrollment of patients with solid tumors in our Phase 1
proof-of-concept trial this quarter, we now expect initial clinical
data for SRK-181 as well as for SRK-015 this year.”
2020 R&D Priorities:
SRK-015 Program for Spinal Muscular
Atrophy:
- Interim Safety and Efficacy Data from TOPAZ Phase 2
Proof-of-Concept Trial Expected in Mid-2020. Scholar
Rock plans to report interim safety and efficacy results at six
months of treatment exposure for all three fully enrolled cohorts
in mid-2020. Top-line data for the full 12-month treatment period
are expected beginning in the fourth quarter of 2020 and into the
first quarter of 2021.
- Identification of Second Indication for SRK-015 Planned
for 2020. Scholar Rock continues to evaluate multiple
potential opportunities beyond SMA, for which the selective
inhibition of the activation of myostatin with SRK-015 may offer
therapeutic benefit.
SRK-181 Program for Immuno-Oncology:
- Initiation of SRK-181 Phase 1 Dose Escalation and Dose
Expansion Clinical Trial in Patients with Solid Tumors Planned for
the First Quarter of 2020. TGFβ signaling has been
implicated in driving immune exclusion, a key culprit of primary
resistance to checkpoint inhibitor therapies as described by
multiple academic and industrial groups. Scholar Rock is developing
SRK-181 as a potential therapy in cancer immunotherapy to broaden
responses to anti-PD-(L)1 therapies. In an aim to reduce toxicities
associated with non-selective TGFβ inhibition that have
historically limited the ability to dose at clinically meaningful
levels, SRK-181 has been designed to selectively block the TGFβ1
isoform. An IND application for SRK-181 has been submitted to the
FDA and Scholar Rock plans to initiate a Phase 1 proof-of-concept
trial in the first quarter of 2020 in patients with locally
advanced or metastatic solid tumors. The two-part trial will
consist of a dose escalation portion for SRK-181 as both a
single-agent and in combination with an approved anti-PD-(L)1
antibody, followed by a dose expansion portion evaluating SRK-181
in combination with an approved anti-PD-(L)1 antibody in multiple
tumor-specific cohorts, such as urothelial carcinoma, melanoma,
non-small cell lung cancer, and other solid tumors. Key objectives
of the study include evaluating the safety and pharmacokinetics of
SRK-181 and the efficacy of SRK-181 in combination with
anti-PD-(L)1 therapy in the treatment of solid tumors exhibiting
primary resistance to anti-PD-(L)1 therapy. Initial clinical data
from the Phase 1 trial are expected in the second half of 2020 with
clinical response and safety data anticipated in 2021.
Strategic Fibrosis Collaboration with Gilead
Sciences:
- Further Advance Collaborative Programs Towards Product
Candidate Selection. Scholar Rock has demonstrated in
preclinical studies that potent and selective inhibitors of TGFβ1
signaling prevent the activation of the growth factor in the
fibrotic matrix and may offer a powerful new approach to
suppressing pro-fibrotic signaling in multiple organs. With the
recent achievement of the first milestone with the demonstration of
efficacy in in vivo proof-of-concept studies, Scholar Rock and
Gilead are advancing the collaboration with the aim of selecting
molecules to be developed as new medicines for patients with
fibrotic diseases. Gilead has exclusive options to license
worldwide rights to product candidates that emerge from three
Scholar Rock TGFβ programs. Scholar Rock is responsible for
antibody discovery and preclinical research through product
candidate nomination, after which, upon exercising the option for a
program, Gilead will be responsible for the program’s preclinical
and clinical development and commercialization. Scholar Rock is
eligible to receive up to an additional $1,425 million in potential
payments from Gilead as well as high single-digit to low
double-digit tiered royalties on sales of potential future products
originating from the collaboration.
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, fibrosis and anemia.
Scholar Rock’s newly elucidated understanding of 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 StatementsThis 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; timing of its clinical trials for SRK-015, SRK-181, and
other product candidates; indication selection and development
timing; the ability of any product candidate to perform in humans
in a manner consistent with nonclinical or preclinical study data;
progress under its collaboration with Gilead. 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
Scholar Rock’s ability to provide the financial support, resources
and expertise necessary to identify and develop product candidates
on the expected timeline; preclinical data and results may not be
predictive of clinical results; 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 ability to support its 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; and
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 September 30, 2019, 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 Contact: Investors/Media Catherine
Hu chu@scholarrock.com 917-601-1649
Media Contact: The Yates Network Kathryn Morris
kathryn@theyatesnetwork.com 914-204-6412
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