Rubius Therapeutics, Inc. (Nasdaq: RUBY), a clinical-stage
biopharmaceutical company that is biologically engineering red
blood cells to create an entirely new class of cellular medicines
called Red Cell Therapeutics™ for the treatment of cancer and
autoimmune diseases, today announced updated clinical data from the
ongoing monotherapy Phase 1 arm of the Phase 1/2 clinical trial of
RTX-240 in patients with advanced solid tumors at the American
Association for Cancer Research Annual Meeting. The Company is
hosting a webcast to discuss these data as well as data from the
monotherapy Phase 1 arm of RTX-240 in patients with acute myeloid
leukemia (AML) today at 1:15 p.m. EDT.
“Recent advances in immuno-oncology have
transformed the treatment of cancer and improved outcomes for
certain patients. Yet, of the estimated 40 percent of patients
eligible for immunotherapy, the majority do not have durable
responses. In addition, combinations of immune checkpoint
inhibitors have higher toxicity, underscoring the need for safer
and more effective immunotherapy combination treatments,” said
Alexander I. Spira, M.D., Ph.D., FACP, director of the Virginia
Cancer Specialists Research Institute and the Phase 1 Trial
Program, and a clinical investigator in all of Rubius’ oncology
clinical trials. “In this Phase 1 trial of heavily pretreated
patients with advanced solid tumors, RTX-240 demonstrated
single-agent activity with encouraging tolerability results,
including in patients whose disease had progressed on prior
PD-1/PD-L1-based treatment regimens. Based on these data, I believe
RTX-240 has the potential to be an ideal candidate to be developed
as a combination therapy with immune checkpoint inhibitors,
especially in earlier lines of therapy.”
“We believe the encouraging results of monotherapy
RTX-240 reported provide clinical support of the RED PLATFORM® and
the development of our entire oncology pipeline of Red Cell
Therapeutics given the programmable nature of our platform,” said
Pablo J. Cagnoni, M.D., president and chief executive officer.
“With multiple data milestones expected over the next 12 months,
Rubius Therapeutics’ pipeline of oncology Red Cell Therapeutics has
the opportunity to show potential benefit in additional types of
cancer.”
Updated Data from the Phase 1 Trial of
RTX-240 in Patients with Advanced Solid Tumors
Nine dose cohorts (n=34) were completed in the
monotherapy solid tumor arm of the trial at the time of the data
cutoff on March 4, 2022, with 34 patients evaluable for safety
(primary outcome measure) and 27 patients evaluable for efficacy
(secondary outcome measure). Enrollment continues in the 5e10 Q3W
dose cohort.
As of the cutoff date, disease control was observed
in 10 patients (1 partial response, 2 unconfirmed partial responses
and 7 with stable disease), 9 of whom had experienced disease
progression on prior anti-PD-1/anti-PD-L1 therapy.
There were three best responses of partial response
(PR) in non-small cell lung cancer (NSCLC), anal cancer and uveal
melanoma patients:
- An unconfirmed PR (uPR) with 41% decrease of all target lesions
and a notable decrease of an external protruding chest wall mass in
a patient with non-small cell lung cancer (NSCLC) whose disease had
progressed on prior anti-PD-L1 therapy;
- A confirmed PR with a 54% reduction in the target lesions in a
patient with metastatic anal cancer whose disease had progressed on
anti-PD-L1 therapy; and
- An uPR with 100% decrease of the target hepatic lesion and
resolution of multiple non-target hepatic lesions in a patient with
metastatic uveal melanoma whose disease had progressed on anti-PD-1
therapy.
Stable disease was observed in 5 patients,
including 3 with metastatic NSCLC and 2 with renal cell carcinoma
(RCC) across the 3e10 cohorts, supporting the Company’s decision to
expand the Phase 1 arm of RTX-240 plus pembrolizumab to NSCLC and
RCC patients. One patient each with NSCLC and RCC remained on
monotherapy treatment with SD greater than 6 months as of the
cutoff date.
As of the cutoff date, RTX-240 has been shown to
have been generally well tolerated with no treatment-related or
investigator-identified immune-related Grade 3/4 adverse events
(AEs) and no dose-limiting toxicities.
Based on the totality of clinical, tolerability and
pharmacodynamic data, a recommended monotherapy Phase 2 dose of
5e10 cells administered every 3 weeks was selected. This dose will
be further explored in the combination expansion cohort of NSCLC
and RCC patients.
“Immune agonists and cytokines have been the focus
of oncology research given their known importance for immune
activation. However, current approaches have been unable to
overcome toxicity challenges, resulting in a narrow therapeutic
index, particularly in combination with checkpoint inhibitors. With
today’s updated clinical data showing that RTX-240 generated
clinical responses with favorable tolerability results in patients
whose tumors had progressed on therapy with anti-PD-1/PD-L1
antibodies, we believe these data support the potential of immune
agonists for the treatment of cancer,” said Larry Turka, M.D.,
chief scientific officer and head of translational medicine at
Rubius Therapeutics. “Given the clinical results observed, we are
expanding the ongoing Phase 1 arm of RTX-240 in combination with
pembrolizumab to patients with NSCLC and RCC who have had fewer
prior treatment regimens. This cohort is expected to inform our
strategy of advancing RTX-240 in combination with pembrolizumab in
a Phase 2 clinical trial.”
Final Phase 1 Clinical Results in
Relapsed/Refractory AML
Rubius also announced final clinical results from
the Phase 1 arm of monotherapy RTX-240 in relapsed/refractory AML.
As of the cutoff date of March 4, 2022, seventeen patients were
enrolled across 4 dose levels. No DLTs were observed and there were
3 treatment-related Grade 3/4 adverse events. There were no
investigator-reported immune-related AEs. Five patients had SD
greater than 3 months, and 1 patient had a significant blast count
reduction (53% to 6%).
“In this study, RTX-240 has shown activation and
expansion of NK and T cells with favorable safety results, which
continues to support the proposed mechanism of action of RTX-240.
Based on these data, we believe RTX-240 could improve outcomes for
AML patients when used as maintenance therapy for patients in
remission following high-dose chemotherapy and stem cell
transplantation,” continued Dr. Turka. “Based on these data, we
believe we have established the necessary foundation to evaluate
RTX-240 in the maintenance setting for the treatment of AML.
However, to focus our resources on advancing RTX-240 in combination
with pembrolizumab in NSCLC and RCC, we do not plan to pursue a
separate clinical trial in AML in the near-term.”
Upcoming Anticipated
Milestones
To evaluate the full potential of RTX-240, Rubius’
other oncology programs and the RED PLATFORM, Rubius plans to
execute several critical milestones within the next 12 months and
has sufficient cash runway into the second half of 2023:
- Report initial Phase 1 clinical results for RTX-321 for the
treatment of HPV 16-positive cancers during the second half of
2022;
- Report initial Phase 1 clinical results for RTX-240 in
combination with pembrolizumab in advanced solid tumors and data
from the additional NSCLC and RCC patients in the second half of
2022;
- Select a clinical candidate for the first autoimmune program in
type 1 diabetes during the second half of 2022; and
- Report initial Phase 1 clinical results for RTX-224 for the
treatment of advanced solid tumors during the first quarter of
2023.
AACR Poster Presentation
Abstract Title: Phase 1 Trial of
RTX-240, Allogeneic Red Blood Cells Engineered to Express 4-1BBL
and Trans-Presented IL-15, in Patients with Advanced Solid
TumorsSession Title: Phase I Clinical Trials
2Session Date and Time: Tuesday, April 12, 2022,
9:00 a.m. – 12:30 p.m. ETLocation: New Orleans
Convention Center, Exhibit Halls D-H, Poster Section
33Poster Board Number: 12Abstract
Number: CT187
Conference Call
The Company will host a conference call and webcast
at 1:15 p.m. EDT to discuss this update. The audio webcast will be
available on the Events and Presentations page within the Investors
and Media section of the Rubius Therapeutics website. The update
may also be accessed by dialing (800) 289-0045 (domestic) or (615)
622-8086 (international) five minutes prior to the start of the
call. The conference ID is 7865329. An archived webcast will be
accessible for 90 days after the event.
About RTX-240
RTX-240, Rubius Therapeutics’ lead oncology
program, is an allogeneic, off-the-shelf cellular therapy product
candidate that is engineered to simultaneously present hundreds of
thousands of copies of the costimulatory molecule 4-1BB ligand
(4-1BBL) and IL-15TP (trans-presentation of IL-15 on IL-15Rα) in
their native forms. RTX-240 is designed to broadly stimulate the
immune system by activating and expanding both NK and memory T
cells to generate a potent anti-tumor response.
About the RTX-240 Clinical
Trial
The Phase 1/2 clinical trial of RTX-240 is an open
label, multicenter, multidose, first-in-human dose-escalation and
expansion study designed to evaluate the safety and tolerability,
pharmacokinetics, maximum tolerated dose, a recommended Phase 2
dose and dosing regimen of RTX-240. The trial is also assessing the
pharmacodynamics of RTX- 240 measured by changes in T and NK cell
number and function relative to baseline and anti-tumor activity.
The trial has three separate Phase 1 arms: a monotherapy dose
escalation arm in adults with relapsed/refractory or locally
advanced solid tumors, a monotherapy dose escalation arm in adults
with relapsed/refractory acute myeloid leukemia, and a combination
therapy dose escalation arm with pembrolizumab in adults with
relapsed/refractory or locally advanced solid tumors.
About Rubius Therapeutics
Rubius Therapeutics is a clinical-stage
biopharmaceutical company developing a new class of medicines
called Red Cell Therapeutics™. The Company’s proprietary RED
PLATFORM® was designed to biologically engineer and culture Red
Cell Therapeutics™ that are selective, potent and off-the-shelf
allogeneic cellular therapies for the potential treatment of
several diseases across multiple therapeutic areas. Rubius’ initial
focus is to advance RCT™ product candidates for the treatment of
cancer and autoimmune diseases by leveraging two distinct
therapeutic modalities — potent cell-cell interaction and tolerance
induction. Rubius Therapeutics was recently named among the
2021 Top Places to Work in Massachusetts by the Boston Globe, and
its manufacturing site was recently named 2021 Best
Places to Work in Rhode Island by Providence Business News.
For more information, visit www.rubiustx.com, follow us on Twitter
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Forward-Looking Statements This
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 with respect to the potential of our pipeline of Red
Cell Therapeutics, our plans to announce results from our RTX-240
trial in patients with solid tumors and AML and to share expected
next steps for the Phase 2 clinical development, our expectations
regarding our timelines for reporting additional clinical data, our
expectations for and beliefs about the initial results from the
RTX-240 trial and its validation of the RED PLATFORM and its impact
on the potential of our entire oncology platform, and the extent to
which the COVID-19 pandemic may impact Rubius’ ability to enroll
patients in the trial will depend on future developments and our
expectations regarding 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, those risks and uncertainties related to the
development of our Red Cell Therapeutic product candidates and
their therapeutic potential, our ability to execute on our plans
and expectations, our analyses of clinical and preclinical data and
other risks identified in our filings with the U.S. Securities
and Exchange Commission (SEC), including our Annual Report on Form
10-K for the year ended December 31, 2021 and subsequent filings
with the SEC, and risks and uncertainties related to the severity
and duration of the impact of COVID-19 on our business and
operations. 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.
Contacts:
InvestorsLori Murray, Chief Corporate Affairs
Officerlori.murray@rubiustx.com
Media Marissa Hanify, Director,
Corporate Communicationsmarissa.hanify@rubiustx.com
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