Rubius Therapeutics to Highlight the Power of its RED PLATFORM®, Significant Advances Across Red Cell Therapeutic Oncology Pipeline and New Type 1 Diabetes Program at Platform and Pipeline Day
December 16 2021 - 8:00AM
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 significant advances from its
RED PLATFORM and across its pipeline of Red Cell Therapeutics
(RCTs) at the Company’s first Pipeline and Platform Day.
“Over the course of our presentations, we will highlight the
incredible scope and versatility of the programmable RED PLATFORM,
enabling targeting of multiple immune pathways via several
different modalities, as well as the progress Rubius Therapeutics
is making in advancing our clinical-stage cancer therapies,
autoimmune programs and manufacturing capabilities,” said Pablo J.
Cagnoni, M.D., “Today, for the first time, we will be unveiling
preclinical proof of concept data demonstrating tolerance induction
with bystander suppression of type 1 diabetes, a T cell-mediated
disease. We expect next year to be data rich with several important
clinical milestones from our oncology pipeline, including
additional clinical results from the single-agent RTX-240 Phase 1
clinical trial in advanced solid tumors and acute myeloid leukemia
and initial clinical results from our RTX-321 Phase 1 clinical
trial in advanced HPV 16-positive cancers, which we anticipate
occurring during the first quarter of 2022.”
Highlights and Progress
RED PLATFORM
- 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 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
- Enabling rapid and repeatable
parallel generation of therapeutic candidates with the programmable
RED 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
Oncology
Broad Immune Stimulation Approach
RTX-240
- 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
- Escalating the dose of single-agent
RTX-240 in the Phase 1 solid tumor clinical trial to three doses of
5e10 followed by one dose 1e10 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
single-agent 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.
RTX-224
- Planning to initiate the Phase 1
clinical trial of RTX-224 in patients with certain advanced solid
tumors during the first quarter of 2022
- Investigational New Drug application
cleared
Antigen-Specific Immune Stimulation
Approach
RTX-321
- Continuing enrollment in Phase 1
clinical trial of RTX-321 in patients with advanced HPV 16-positive
cancers
- Planning to report initial clinical
results during the first quarter of 2022
Autoimmune Diseases and Type 1 Diabetes
- 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, reverses established
inflammation, which is important for the treatment of existing
autoimmunity, and induces 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.
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
- Greater than 200 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
Video Webcast InformationA live video webcast
is scheduled for today from 11:00 a.m. to 2:00 p.m. ET and can be
accessed via the Events and Presentations page within the Investor
and Media section of the Rubius Therapeutics website. The Rubius
management team and Dr. Nepom will host a Q&A session following
the presentations.
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 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 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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