Rubius Therapeutics Announces Dosing of First Patient with Relapsed/Refractory Acute Myeloid Leukemia in the Ongoing Phase 1/...
November 05 2020 - 8:00AM
Rubius Therapeutics, Inc. (Nasdaq:RUBY), a clinical-stage
biopharmaceutical company that is genetically engineering red blood
cells to create an entirely new class of cellular medicines called
Red Cell Therapeutics™, today announced that the first patient with
acute myeloid leukemia (AML) has been dosed in its ongoing Phase
1/2 clinical trial of RTX-240, an allogeneic cellular therapy
product candidate that is being evaluated for the treatment of
patients with relapsed/refractory or locally advanced solid tumors
or relapsed/refractory AML. The ongoing clinical study of RTX-240
has two Phase 1 arms, one in all solid tumors and the other in
relapsed/refractory AML. RTX-240 is engineered to stimulate innate
and adaptive immunity by activating natural killer (NK) cells and T
cells inside the patient’s body to generate a potent anti-tumor
immune response.
“A number of studies show that NK cells can exhibit potent
anti-tumor activity against AML, but tumor-associated mechanisms
often suppress the proper function of NK cells leading to disease
progression,” said Christina Coughlin, M.D., Ph.D., chief medical
officer of Rubius Therapeutics. “When NK cells are restored to
their full anti-tumor potential, their cytolytic activity predicts
a better long-term outcome for patients with AML. RTX-240 is
designed to replicate immune system function by activating and
expanding a patient’s own NK and T cells in the body to generate an
immune response against cancer.”
RTX-240 is engineered to simultaneously express hundreds of
thousands of copies of the costimulatory molecule 4-1BB ligand
(4-1BBL) and the cytokine interleukin-15 (IL-15TP) to broadly
stimulate both powerful arms of the immune system to generate
anti-tumor immunity.
“Despite advances in the management of AML, the overall outcome
for patients with relapsed or refractory disease remains quite poor
due to several factors, including lack of disease response to
salvage therapy and the overall toxicity of the therapies,” said
Alexander Spira, M.D., Ph.D., director of the Virginia Cancer
Specialists Research Institute and the Phase I Trial Program.
“RTX-240 potentially offers a novel way of treating
relapsed/refractory AML by using the body’s own immune system to
attack cancer. We look forward to working with Rubius Therapeutics
on this differentiated approach to treating AML.”
About Acute Myeloid Leukemia
(AML)AML is a rare and aggressive cancer of the
blood and bone marrow, and is the most common form of acute
leukemia in adults. AML begins in the bone marrow and most often
moves quickly into the blood or other areas of the body.
Refractory AML occurs when patients have received treatment,
typically two rounds of chemotherapy, but the disease does not go
into remission. Some patients experience remission and then a
return of the leukemia, which is referred to as relapsed AML. The
overall outcome for patients with relapsed/refractory AML remains
poor, with 5-year overall survival of approximately 10
percent. Limited therapeutic options currently exist for these
patients.
About the RTX-240 Clinical TrialRubius
Therapeutics is enrolling patients in a Phase 1/2 open label,
multicenter, multidose, first-in-human dose-escalation and
expansion study of RTX-240. The study contains two Phase 1 dose
escalation arms: one in patients with relapsed/refractory or
locally advanced solid tumors and another in patients with
relapsed/refractory acute myeloid leukemia. These two Phase 1 arms
will determine the safety and tolerability, pharmacokinetics,
maximum tolerated dose and a recommended Phase 2 dose and dosing
regimen of RTX-240 in patients with solid tumors as well as in
patients with relapsed/refractory AML. The trial will also assess
the pharmacodynamics of RTX-240 measured by changes in T and NK
cell number and function relative to baseline and anti-tumor
activity in both patient populations. The study will include an
expansion phase in specified tumor types during the Phase 2 portion
of the solid tumor arm. The extent to which the COVID-19 pandemic
may impact Rubius’ ability to enroll patients in the trial will
depend on future developments.
About RTX-240RTX-240 is an allogeneic cellular
therapy product candidate that is being evaluated for the treatment
of patients with relapsed/refractory or locally advanced solid
tumors or relapsed/refractory acute myeloid leukemia. RTX-240 is
engineered to simultaneously present hundreds of thousands of
copies of the costimulatory molecule 4-1BBL and IL-15TP
(trans-presentation of IL-15 on IL-15Rα) in their native forms and
is designed to stimulate innate and adaptive immunity by activating
NK cells and T cells inside the patient’s body to generate an
anti-tumor immune response.
About Rubius TherapeuticsRubius Therapeutics,
Inc. (Nasdaq:RUBY) is a clinical-stage biopharmaceutical company
that is genetically engineering red blood cells to create an
entirely new class of cellular medicines called Red Cell
Therapeutics™. The Company’s proprietary RED PLATFORM® genetically
engineers and cultures Red Cell Therapeutics to create selective,
potent and off-the-shelf allogeneic cellular therapies for the
potential treatment of cancer and autoimmune diseases. Rubius’
initial product candidates are designed to activate and expand
immune system function to fight cancer and modulate the immune
system to induce tolerance for the treatment of autoimmune
diseases. Rubius Therapeutics’ manufacturing site was recently
named 2020 Top 5 Best Places to Work in Rhode Island among
medium-sized companies by Providence Business News. For more
information, visit www.rubiustx.com, follow us on Twitter or
LinkedIn or like us on Facebook.
Forward Looking StatementsThis
press release contains forward-looking statements within the
meaning of the Private Securities Litigation Reform Act of 1995, as
amended, including, without limitation, statements regarding our
expectations regarding the therapeutic potential of our Red Cell
Therapeutics, including RTX-240 for the treatment of solid tumors
and acute myeloid leukemia, and our strategy, business plans and
focus. The words “may,” “will,” “could,” “would,” “should,”
“expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,”
“predict,” “project,” “potential,” “continue,” “target” and similar
expressions are intended to identify forward-looking statements,
although not all forward-looking statements contain these
identifying words. Any forward-looking statements in this press
release are based on management’s current expectations and beliefs
and are subject to a number of risks, uncertainties and important
factors that may cause actual events or results to differ
materially from those expressed or implied by any forward-looking
statements contained in this press release, including, without
limitation, expectations regarding the therapeutic potential of
RTX-240 and our clinical study of RTX-240, those risks and
uncertainties related to the development of our Red Cell
Therapeutic product candidates and their therapeutic potential and
other risks identified in our SEC filings, including our Quarterly
Report on Form 10-Q for the quarter ended June 30, 2020, and
subsequent filings with the SEC. We caution you not to place undue
reliance on any forward-looking statements, which speak only as of
the date they are made. We disclaim any obligation to publicly
update or revise any such statements to reflect any change in
expectations or in events, conditions or circumstances on which any
such statements may be based, or that may affect the likelihood
that actual results will differ from those set forth in the
forward-looking statements. Any forward-looking statements
contained in this press release represent our views only as of the
date hereof and should not be relied upon as representing its views
as of any subsequent date. We explicitly disclaim any obligation to
update any forward-looking statements.
Contacts:
InvestorsLori Melançon, Vice
President, Corporate Communications and Investor Relations+1 (617)
949-5296lori.melancon@rubiustx.com
Media Marissa Hanify, Director, Corporate
CommunicationsMarissa.hanify@rubiustx.com
Dan Budwick, 1AB+1 (973) 271-6085dan@1abmedia.com
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