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 provided an overview of the Company’s
achievements in 2021 and outlined its objectives for 2022. Pablo J.
Cagnoni, M.D., president and chief executive officer, will present
these updates on Wednesday, January 12, 2022, at 8:15 a.m. EST at
the virtual 40th Annual J.P. Morgan Healthcare Conference.
“In 2021, Rubius Therapeutics demonstrated strong execution in
advancing our clinical oncology pipeline and showing preclinical
proof of concept of our tolerance induction approach in
autoimmunity that could extend well beyond type 1 diabetes to other
T cell-mediated diseases,” said Pablo J. Cagnoni, M.D., president
and chief executive officer, of Rubius Therapeutics. “With the
initial clinical results from the Phase 1 clinical trial of RTX-240
in patients with advanced solid tumors reported in March 2021, we
provided clinical validation of the RED PLATFORM® and increased our
likelihood of success across our entire oncology pipeline of Red
Cell Therapeutics. With multiple data milestones on the horizon in
2022, we are on the cusp of potentially further validating the RED
PLATFORM and benefiting an even greater number of patients.”
Anticipated 2022 Catalysts and Operational
Objectives
- Present additional clinical results from the Phase 1 arm of the
RTX-240 Phase 1/2 clinical trial in advanced solid tumors and the
Phase 1 arm in relapsed/refractory acute myeloid leukemia (AML)
during the first quarter of 2022
- Initiate RTX-240 Phase 2 expansion cohorts in select solid
tumor types during the first quarter of 2022
- Report initial clinical results from the Phase 1 clinical trial
of RTX-321 in patients with advanced HPV 16-positive cancers during
the second half of 2022
- Present initial clinical data from the Phase 1 arm of the
RTX-240 clinical trial in combination with pembrolizumab in
patients with advanced solid tumors during the second half of
2022
- Scale to 200L bioreactors by mid-2022 to support potential
pivotal trial and eventual commercialization
2021 Achievements and Operational Updates
RED PLATFORM
- Enabling rapid and repeatable parallel generation of
therapeutic candidates with the programmable RED PLATFORM
- 1,000 different therapeutic proteins biologically engineered
since platform inception, underscoring the highly versatile and
programmable nature of the platform
- The platform creates multiple modalities for the treatment of
cancer and autoimmune disease and the ability to express hundreds
of thousands of copies of therapeutic proteins on or within the
cell to access numerous immune pathways
- Achieved clinical validation of the RED PLATFORM with initial
clinical results from the single-agent RTX-240 Phase 1 clinical
trial in advanced solid tumors, reported in March 2021
- RCTs are well tolerated, induce the desired biological effect
and generate clinical benefit in certain patients with advanced
solid tumors
- Advancing next generation artificial antigen-presenting cells
(aAPCs) with loadable MHC Class I, enabling the presentation of
multiple antigens on a single RCT and broadening the potential
patient population with a library of HLA types
Oncology
Broad Immune Stimulation
RTX-240
RTX-240 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 CD8+ memory T
cells to generate a potent anti-tumor response.
- Established clinical proof of concept of RTX-240 in advanced
solid tumors, based on initial results reported in March 2021,
potentially increasing the likelihood of clinical success across
the oncology pipeline
- Escalated the dose of single-agent RTX-240 in the Phase 1 solid
tumor clinical trial to three doses of 5e10 cells followed by 1e10
cells until disease progression or unacceptable toxicity, based on
no dose-limiting toxicities observed to date, a clear dose response
in the increase of NK cells and other pharmacodynamic effects
- Additional clinical results are expected from this trial and
the Phase 1 arm in relapsed/refractory AML during the first quarter
of 2022
- The Company plans to initiate RTX-240 Phase 2 expansion cohorts
in select solid tumor types during the first quarter of 2022
- Continuing dose escalation in the RTX-240 Phase 1 combination
study with pembrolizumab in patients with advanced solid tumors
with initial clinical data expected during the second half of
2022
RTX-224RTX-224 is an allogeneic, off-the-shelf
cellular therapy product candidate that is engineered to express
hundreds of thousands of copies of 4-1BBL and interleukin-12
(IL-12) on the cell surface. While the lead oncology product
candidate, RTX-240, is designed to broadly stimulate the immune
system by activating and expanding NK and CD8+ memory T cells,
RTX-224 is expected to produce a broad and potent anti-tumor T cell
response, an innate immune response and have anti-tumor activity in
those tumor types with known sensitivity to T cell killing,
including tumor types with high mutational burden, PD-L1 expression
and known responsiveness to checkpoint inhibitors.
- Expect to begin dosing patients in the Phase 1/2 clinical trial
of RTX-224 in selected relapsed/refractory or locally advanced
solid tumors during the first quarter of 2022
- Select cancers include non-small cell lung cancer, cutaneous
melanoma, head and neck squamous cell carcinoma, urothelial
(bladder) carcinoma and triple-negative breast cancer
- Presented preclinical data at the Society for Immunotherapy of
Cancer’s (SITC) 36th Annual Meeting in November 2021, demonstrating
that the mouse surrogate of RTX-224, mRBC-224, generated potent
anti-tumor activity in B16F10 melanoma models, intravenously and
subcutaneously, that was associated with pharmacodynamic changes in
the tumors, including activated CD8 + T cells, NK cells and
macrophages
Antigen-Specific Immune Stimulation
RTX-321 Artificial Antigen-Presenting Cell (aAPC)
Development Program for Human Papillomavirus (HPV) 16-Positive
Cancers
RTX-321 is an allogeneic, off-the-shelf artificial aAPC therapy
product candidate that is engineered to induce a tumor-specific
immune response by expanding antigen-specific T cells. RTX-321
expresses hundreds of thousands of copies of an HPV peptide antigen
bound to major histocompatibility complex class I proteins, the
costimulatory molecule 4-1BBL and the cytokine IL-12 on the cell
surface to mimic human T cell-APC interactions.
- Continuing enrollment in the Phase 1 clinical trial of RTX-321
in patients with advanced HPV 16-positive cancers
- Based on no dose limiting toxicities to date, the Company plans
to dose escalate beyond the current cohort of 1e10 cells
- Plan to report initial clinical results during the second half
of 2022
Autoimmune Diseases and Type 1 Diabetes
Red Cell Therapeutics for the treatment of autoimmune disease
are biologically engineered to express proteins and peptides inside
the cell and are designed to be phagocytized, or ingested, by
dendritic cells or macrophages to induce tolerance, retraining the
immune system to no longer recognize these self-antigens as
foreign.
- Demonstrated tolerance induction and bystander suppression in
stringent type 1 diabetes preclinical models
- Established efficacy in the BDC2.5 adoptive transfer model with
data supporting that repeated dosing extended duration of disease
protection, reversed established inflammation, which is important
for the treatment of existing autoimmunity, and induced two types
of regulatory T cells, resulting in protection against
re-challenge
- Showed efficacy in non-obese diabetes (NOD) preclinical model
- Results at 25 weeks exhibit bystander suppression by delivering
only two antigens, indicating the mouse surrogate of RTX-T1D
prevented or delayed disease caused by many autoantigens
- These findings are potentially translatable beyond type 1
diabetes to multiple autoimmune diseases, including other Rubius’
high priority target indications, including multiple sclerosis and
celiac disease.
Fully Owned Manufacturing
- Increased cells produced per batch by four times in 50L
bioreactors from 2020 to 2021, enabling uninterrupted clinical
supply for three Phase 1 arms of the RTX-240 clinical trial and
Phase 1 RTX-321 trial
- Additional accomplishments include:
- High success rate: greater than 90% lot success rate for
RTX-240 and RTX-321 clinical supply in 2021
- Hundreds of doses administered across three arms of RTX-240
Phase 1 and RTX-321 Phase 1 trials
- High transduction efficiency: greater than 90% of cells are
transduced with therapeutic proteins
- Highly consistent protein expression (dual or triple)
- Introduced frozen drug substance for RTX-321 and RTX-224,
enabling inventory storage of greater than two years
- Developing frozen drug product to further simplify supply chain
with the goal of making our therapies available around the
world
- Bringing all product testing in-house to strengthen supply
chain and reduce time-to-product release
- Continuing to invest in the platform to improve productivity
and efficiency
- Scaling to 200L bioreactors by mid-2022 to support potential
pivotal trial and eventual commercialization
Listen to the Webcast
A live audio webcast of Dr. Cagnoni’s presentation will be
available on January 12, 2022, at 8:15 a.m. EST within the
Investors & Media section of the Rubius Therapeutics website.
An archived replay will be accessible for 30 days following the
event.
About Rubius TherapeuticsRubius 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 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 beliefs about
Rubius’ execution across preclinical and clinical development,
Rubius’ plans and expected timing to present clinical results for
RTX-240 and RTX-321, beliefs that findings from preclinical models
will be translatable to multiple T cell-mediated autoimmune
diseases, plans to advance and expectations for aAPCs, plans and
timing to scale manufacturing, beliefs about the scope and
versatility of the programmable RED PLATFORM and the generation of
therapeutic candidates, expectations regarding the timing for
initiating the RTX-224 trial, expectations regarding the
therapeutic potential and safety profile of our pipeline of Red
Cell Therapeutics, our interpretations of data, including as to the
efficacy of our product candidates with respect to autoimmune
diseases and Type 1 diabetes, as well as beliefs about our
manufacturing accomplishments and goals and expectations for
further manufacturing activities. 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, 2020 and our subsequent filings with the SEC,
including our Quarterly Report on Form 10-Q for the quarter
ended September 30, 2021, 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: InvestorsElhan Webb,
CFA, VP Investor Relationselhan.webb@rubiustx.com
Media Marissa Hanify, Director, Corporate
Communicationsmarissa.hanify@rubiustx.com
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