resTORbio Announces Initiation of Phase 1b/2a Trial of RTB101 in Parkinson’s Disease
02 Abril 2019 - 9:00AM
resTORbio, Inc. (Nasdaq: TORC), a clinical-stage biopharmaceutical
company developing innovative medicines that target the biology of
aging to prevent or treat age-related diseases, today announced the
initiation of a Phase 1b/2a trial of RTB101, its orally
administered, small molecule, potent inhibitor of target of
rapamycin complex 1 (TORC1) product candidate, alone or in
combination with sirolimus, in Parkinson’s disease (PD).
“We believe that TORC1 may be an important therapeutic target
for several neurodegenerative diseases associated with aging, in
which misfolded proteins aggregate and cause neuronal toxicity. As
such, we are excited to initiate our first clinical trial in PD and
expand our pipeline into neurodegenerative disease,” said Chen
Schor, Co-Founder, President and CEO of resTORbio. “Multiple
preclinical models of PD have demonstrated the potential benefits
of TORC1 inhibition, and intermittent TORC1 inhibition with a
synergistic combination of RTB101 and sirolimus may serve as a
promising approach for the treatment of PD. We look forward to
reporting data from this trial in 2020.”
Selective and broad inhibition of TORC1 has been shown to
ameliorate neurodegenerative disease in several preclinical studies
across multiple species, including models of PD. TORC1 inhibition
with RTB101 in combination with sirolimus, a rapalog, may provide a
therapeutic benefit to PD patients by potentially inducing
autophagy to clear protein aggregates in neurons, increasing
lysosomal biogenesis and decreasing glucosylceramide (GL1)
synthesis.
Phase 1b/2a Trial DesignThe four-week,
multicenter, 2:1 randomized, double-blind, placebo-controlled Phase
1b/2a trial is evaluating the safety and tolerability of RTB101
alone or in combination with sirolimus in PD. The Company plans to
enroll 45 patients with mild PD who are already on standard-of-care
therapy, including those with and without glucocerebrosidase (GBA)
mutations. Patients are expected to be enrolled into five cohorts
and dosed once-weekly with RTB101 300mg alone or in combination
with three dose levels of sirolimus (2 mg, 4 mg and 6 mg). The
planned primary endpoint of the trial is safety and tolerability,
and secondary endpoints will include exposure in blood, plasma and
cerebrospinal fluid (CSF). The planned exploratory endpoints
include biomarkers in plasma and CSF, and various clinical
assessments. Data from this trial is expected in 2020.
About Parkinson’s diseaseParkinson’s disease,
or PD, is a progressive neurodegenerative disease that affects
approximately 7.5 million people worldwide. The incidence of PD
increases rapidly in people 60 years of age and older, with a mean
age at diagnosis of 70.5 years. GBA gene mutations are the most
common of the currently known PD genetic mutations and up to 10
percent of people with PD in the United States carry it.
About resTORbioresTORbio, Inc. is a clinical
stage biopharmaceutical company developing innovative medicines
that target the biology of aging to prevent or treat age-related
diseases. resTORbio’s lead program selectively inhibits TORC1, an
evolutionarily conserved pathway that contributes to the decline in
function of multiple organ systems, including the immune,
cardiovascular and central nervous systems.
Forward-Looking StatementsThis press release
contains forward-looking statements within the meaning of the
Private Securities Litigation Reform Act of 1995, as amended.
Investors are cautioned that statements in this press release which
are not strictly historical statements, including, without
limitation, express or implied statements or guidance
regarding our proposed timing and trial design for our Phase
1b/2a clinical trial of RTB101, alone or in combination with
sirolimus, in Parkinson’s disease (PD), including anticipated
results of this clinical trial, our expectations around the role
and importance of TORC1 in neurodegenerative diseases, our
estimates as to the number of people with PD worldwide and the
United States and rates of diagnosis, our future plans to expand
our pipeline and develop RTB101 alone or in combination with
rapalogs, such as everolimus or sirolimus, including the
therapeutic potential and clinical benefits thereof and the
potential patient populations that may be addressed by our product
candidates, our ongoing and future clinical trials for RTB101,
including the timing of the initiation and anticipated results of
these trials, the continued expansion of our pipeline, and our
ability to replicate results achieved in our clinical trials in any
future trials constitute forward-looking statements identified by
words like “believe,” “expect,” “may,” “will,” “should,” “seek,”
“anticipate,” or “could” and similar expressions.
Such forward-looking statements are subject to a number of risks
and uncertainties that could cause actual results to differ
materially from those anticipated, including, without limitation,
risks associated with: our Phase 1b/2a clinical trial of RTB101,
alone or in combination with sirolimus, in Parkinson’s disease
(PD); our ability to successfully demonstrate the efficacy and
safety of our lead product candidate; the clinical results for our
lead product candidate which may not support further development of
additional indications; uncertainties related to the results of our
clinical trials predictive of future results in connection with
future trials; the timing and outcome of our planned interactions
with regulatory authorities; and obtaining, maintaining and
protecting our intellectual property; as well as those risks more
fully discussed in the section entitled “Risk Factors” in the
Annual Report on Form 10-K filed by resTORbio, Inc. with
the Securities and Exchange Commission, as well as discussions
of potential risks, uncertainties, and other important factors in
our subsequent filings with the Securities and Exchange
Commission. In addition, any forward-looking statements
represent our views only as of today and should not be relied upon
as representing its views as of any subsequent date. resTORbio
explicitly disclaims any obligation to update any forward-looking
statements.
Investor ContactJennifer RobinsonresTORbio,
Inc.857-772-7029jrobinson@restorbio.com
Media ContactAmir KhanBiosector
2212-462-8767Amir.Khan@Syneoshealth.com
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