resTORbio Announces Interim Results for Phase 1b/2a Trial of RTB101 in Patients with Parkinson’s Disease and Provides Corpo...
February 19 2020 - 8:00AM
resTORbio (Nasdaq: TORC) today announced interim results from the
ongoing Phase 1b/2a trial of RTB101, an orally-administered small
molecule potent target of rapamycin complex 1 (TORC1) inhibitor
product candidate, alone or in combination with sirolimus, in
Parkinson’s disease (PD).
“We are pleased to have observed that RTB101 is well tolerated
and crosses the blood brain barrier in Parkinson’s disease patients
at concentrations that have the potential to induce autophagy, the
process by which cells break down toxic misfolded protein
aggregates. Preclinical data suggest that induction of autophagy
has the potential to slow the progression not only of Parkinson’s
disease but also of multiple other neurodegenerative diseases that
are characterized by the accumulation of toxic protein aggregates
in cells such as Huntington’s and Alzheimer’s disease,” said Dr.
Joan Mannick, Co-Founder and Chief Medical Officer of
resTORbio.
Phase 1b/2a Trial of RTB101 alone and in combination
with sirolimus in Parkinson’s disease interim resultsThe
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 escalating doses of sirolimus (2 mg, 4
mg and 6 mg) once weekly for 4 weeks in patients with Parkinson’s
disease. To date, patients have been enrolled in three cohorts and
dosed once weekly with 300 mg of RTB101 alone, 2 mg of sirolimus
alone, or a combination of 300 mg RTB101 and 2 mg of sirolimus.
Results of the interim study analysis indicated that all 3 dosing
regimens were well tolerated and RTB101 300 mg once weekly was
observed to cross the blood brain barrier. The concentrations of
RTB101 in cerebrospinal fluid (CSF) in subjects dosed with RTB101
300 mg once weekly monotherapy were higher than expected and based
on preclinical models, have the potential to induce autophagy in
the brain. Sirolimus at the dose of 2 mg, alone or in combination
with RTB101, was not detected in the CSF. Data from the first three
cohorts in the study suggest that the concentrations of RTB101
observed in the CSF four hours after dosing were highest when
RTB101 was given as a monotherapy. Enrollment of the RTB101 300 mg
in combination with sirolimus 4 mg once weekly cohort is
ongoing.
Corporate
Update
The company has initiated a process to evaluate external
opportunities, such as partnerships, acquisitions, mergers and
other financial and strategic alternatives to maximize shareholder
value. The company has engaged JMP Securities LLC to act as a
strategic advisor for this process. There can be no assurance that
this strategic review process will result in the company pursuing
any transaction or that any transaction, if pursued, will be
completed. The company has not set a timetable for completion of
this strategic review process, and the company does not intend to
comment further unless or until its Board of Directors has approved
a definitive course of action, the review process is concluded, or
it is determined other disclosure is appropriate.
About Parkinson’s DiseaseParkinson’s disease
(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. Inhibition of TORC1 has been
shown in multiple preclinical models to ameliorate
neurodegenerative diseases including Parkinson’s disease. TORC1
inhibition with RTB101 alone or in combination with sirolimus, may
provide a therapeutic benefit to PD patients by ameliorating
levodopa-induced dyskinesia and/or by inducing autophagy which
leads to the breakdown of protein aggregates and improved neuronal
survival.
About RTB101RTB101 is an oral, selective, and
potent TORC1 inhibitor product candidate that inhibits the
phosphorylation of multiple targets downstream of TORC1. Inhibition
of TORC1 has been observed to extend lifespan and healthspan in
aging preclinical species and to improve the function of aging
organ systems, including neurologic function, suggesting potential
benefits in several aging-related diseases.
About resTORbioresTORbio, Inc. is a
clinical-stage biopharmaceutical company developing innovative
medicines that target the biology of aging to treat aging-related
diseases. resTORbio’s lead program selectively inhibits TORC1, an
evolutionarily conserved pathway that contributes to the decline in
function of aging organ systems, including neurologic function.
Learn more about resTORbio, Inc. at www.resTORbio.com.
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, our proposed timing and anticipated results of our
Phase 1b/2a clinical trial of RTB101 alone or in combination with
sirolimus in patients with mild to severe Parkinson’s disease,
including the announcement of interim results; our future plans to
develop RTB101 alone or in combination with rapalogs, such as
everolimus or sirolimus, including the therapeutic potential and
clinical benefits thereof; our expectations on the potential
patient populations that may be addressed by our product
candidates; our ability to replicate results achieved in our
clinical trials in any future trials; and our engagement of JMP
Securities LLC and our plans to explore and evaluate strategic
alternatives and external opportunities, constitute forward-looking
statements identified by words such as, but not limited to,
“believe,” “expect,” “may,” “will,” “should,” “seek,” “anticipate,”
or “could” and similar words or expressions. Any
forward-looking statements in this statement 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 from those anticipated, including,
without limitation, risks associated with: our ongoing Phase 1b/2a
clinical trial of RTB101 alone or in combination with sirolimus in
Parkinson’s disease, including the announcement of interim results;
the timing and anticipated results of our clinical trials; the risk
that the results of our clinical trials will be predictive of
future results in connection with future clinical trials; our
ability to explore and evaluate strategic alternatives and external
opportunities, 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 are
neither historical facts nor assurances of future performance.
Instead, they represent our beliefs, expectations, assumptions and
views only as of today and should not be relied upon as
representing our beliefs, expectations, assumptions and views as of
any subsequent date. resTORbio explicitly disclaims any obligation
to update any forward-looking statements. No representations or
warranties (expressed or implied) are made about the accuracy of
any such forward-looking statements.
Investor ContactLauren StivalStern Investor
Relations, Inc.212-362-1200lauren.stival@sternir.com
Media ContactKaren
SharmaMacDougall781-235-3060ksharma@macbiocom.com
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