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 reported fourth quarter and full year
2021 financial results.
“Twenty twenty-one was a year of strong execution for Rubius
Therapeutics as we advanced our clinical oncology pipeline,
strengthened our in-house manufacturing capabilities and showed
preclinical proof of concept of our tolerance induction approach in
type 1 diabetes, which has the potential to be extended to other T
cell-mediated autoimmune diseases,” said Pablo J. Cagnoni, M.D.,
president and chief executive officer of Rubius Therapeutics.
“Twenty twenty-two is set to be a catalyst rich year with several
clinical data milestones, including, during the first quarter,
updated results from our single-agent RTX-240 Phase 1 clinical
trial in advanced solid tumors and, during the second half of 2022,
initial clinical data from our RTX-240 Phase 1 arm in combination
with pembrolizumab in advanced solid tumors and from our RTX-321
clinical trial in advanced HPV 16-positive cancers.”
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
Fourth Quarter and Full Year 2021
Highlights
Broad Immune Stimulation
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
- Patients continue to be dosed in the
single-agent RTX-240 Phase 1 solid tumor clinical trial, with no
dose-limiting toxicities observed to date and 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-224
- In January 2022, the first patient was dosed in the Phase 1/2
clinical trial of RTX-224 in selected relapsed/refractory or
locally advanced solid tumors that 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 blood and tumors, including activated
CD4+ and 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
- Continuing enrollment in the Phase 1
clinical trial of RTX-321 in patients with advanced HPV 16-positive
cancers with no dose limiting toxicities observed to date
- Planning to report initial clinical
results during the second half of 2022
Autoimmune Diseases and Type 1 Diabetes
- Demonstrated tolerance induction with 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,
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
- Introduced frozen drug substance for
RTX-321 and RTX-224, potentially enabling inventory storage of
greater than two years
- In process of scaling to 200L
bioreactors by mid-2022 to support potential pivotal trials and
eventual commercialization
Fourth Quarter 2021 Financial Results
Net loss for the fourth quarter of 2021 was $55.0 million or
$0.61 per common share, compared to $40.5 million or $0.50 per
common share in the fourth quarter of 2020.
In the fourth quarter of 2021, Rubius invested $39.8 million in
research and development (R&D) related to its novel RED
PLATFORM® and towards expanding and advancing its product pipeline,
as compared to $25.6 million in the fourth quarter of 2020. This
year-over-year increase was principally due to a $11.0 million
increase in costs incurred for the Company’s lead cancer programs,
RTX-240 and RTX-321, primarily from clinical research organization
(CRO) and internal manufacturing costs incurred in connection with
the three arms of its Phase 1/2 clinical trial of RTX-240, for its
Phase 1 clinical trial of RTX-321 for the treatment of
HPV16-positive cancers and for start-up costs related to its Phase
1 clinical trial of RTX-224. Additionally, personnel-related costs
increased $1.4 million principally for additions to headcount to
support the Company’s expanded operations and stock-based
compensation increased by $1.5 million.
General and administrative (G&A) expenses were $13.9 million
during the fourth quarter of 2021, as compared to $14.1 million for
the fourth quarter of 2020. While there were increases totaling
$1.0 million across professional fees, facility and personnel
costs, they were offset by a decline in stock-based compensation
expense of $1.2 million following the full vesting of large awards
early in the third quarter of 2021.
Full Year 2021 Financial Results
Net loss for the full year 2021 was $196.5 million or $2.23 per
common share, compared to $167.7 million or $2.08 per common share
for the full year 2020.
For the full year 2021, Rubius invested $141.6 million in
R&D related to its novel RED PLATFORM® and towards expanding
and advancing its product pipeline, as compared to $116.1 million
for the full year 2020. The year-over-year increase was driven by
$28.7 million of incremental costs to advance the Company’s lead
cancer programs, including CRO and internal manufacturing costs.
These costs were associated with the three arms of its Phase 1/2
clinical trial of RTX-240, for its Phase 1 clinical trial of
RTX-321 in patients with advanced HPV 16-positive cancers, and for
start-up costs related to its Phase 1 clinical trial of RTX-224.
The increase in cancer program costs was partially offset by a $5.0
million reduction in rare disease program costs following the
deprioritization of the Company’s rare disease pipeline in March
2020. Additionally, platform development, early-stage research and
other unallocated expenses increased by $1.8 million. This
consisted of $4.3 million in additional stock-based compensation
and a $3.1 million increase in personnel and facility related
costs, which were partially offset by reductions in contract
R&D, laboratory supplies and research materials as research
activities shifted to support clinical programs.
G&A expenses were $53.0 million during full year of 2021, as
compared to $50.3 million for the same period in 2020. The higher
costs were driven by a $3.4 million increase in personnel and
facility related costs, as well as a $1.6 million increase in
professional and consultant fees. These increases were offset by a
decrease in stock-based compensation expense following the vesting
of large awards early in the second half of 2021.
Cash Position
As of December 31, 2021, cash and cash equivalents were $225.8
million as compared to $176.3 million in cash, cash equivalents and
investments as of December 31, 2020, providing Rubius with a cash
runway into the second quarter of 2023 and the ability to extend
the runway into the middle of 2023. In connection with its
underwritten public offering completed in March 2021, the Company
received net proceeds of $187.2 million, after deducting
underwriting discounts and commission and other offering costs. In
addition, in June 2021, the Company amended its debt facility,
postponing principal payments by two and a half years, until
mid-2024.
Rubius Therapeutics,
Inc.Condensed Consolidated Statement of
Operations(in thousands, except share and per
share data)(unaudited)
|
For the three months ended December 31, |
|
|
|
For the year ended December
31, |
|
|
2021 |
|
|
2020 |
|
|
2021 |
|
|
2020 |
|
Revenue |
$ |
- |
|
|
$ |
- |
|
|
$ |
- |
|
|
$ |
- |
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Research and development |
|
39,824 |
|
|
|
|
25,616 |
|
|
|
|
141,587 |
|
|
|
|
116,107 |
|
|
General and administrative |
|
13,903 |
|
|
|
|
14,100 |
|
|
|
|
53,029 |
|
|
|
|
50,341 |
|
|
Total operating expenses |
|
53,727 |
|
|
|
|
39,716 |
|
|
|
|
194,616 |
|
|
|
|
166,448 |
|
|
Loss from operations |
|
(53,727 |
) |
|
|
|
(39.716 |
) |
|
|
|
(194,616 |
) |
|
|
|
(166,448 |
) |
|
Other income (expense), net |
|
(1,294 |
) |
|
|
|
(823 |
) |
|
|
|
(1,931 |
) |
|
|
|
(1,283 |
) |
|
Net loss |
$ |
(55,021 |
) |
|
|
$ |
(40,539 |
) |
|
|
$ |
(196,547 |
) |
|
|
$ |
(167,731 |
) |
|
Net loss per share, basic and
diluted |
$ |
(0.61 |
) |
|
|
$ |
(0.50 |
) |
|
|
$ |
(2.23 |
) |
|
|
$ |
(2.08 |
) |
|
Weighted average common shares
outstanding, basic and diluted: |
|
89,918,679 |
|
|
|
|
80,961,343 |
|
|
|
|
87,950,440 |
|
|
|
|
80,624,608 |
|
|
Rubius Therapeutics,
Inc.Condensed Consolidated Balance Sheet
Data(in
thousands)(unaudited)
|
December 31, |
|
December 31, |
|
2021 |
|
2020 |
Cash, cash equivalents and
investments |
$ |
225,848 |
|
$ |
176,287 |
Total assets |
|
318,021 |
|
|
277,794 |
Total liabilities |
|
139,239 |
|
|
136,234 |
Total stockholders'
equity |
|
178,782 |
|
|
141,560 |
About RTX-240RTX-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 RTX-321RTX-321, the Company’s second
oncology program, is an allogeneic, off-the-shelf 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.
About RTX-224 RTX-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 IL-12 on
the cell surface. In contrast to RTX-240, RTX-224 is designed as a
broad immune agonist of both adaptive and innate responses,
activating CD8+ and CD4+ T cells, promoting antigen presentation
and activating and expanding NK cells. It 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 prior activity of
checkpoint inhibitors.About Red Cell Therapeutics in
Autoimmune Diseases 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.
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, 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, including multiple sclerosis and celiac disease, and Type
1 diabetes, as well as beliefs about our manufacturing
accomplishments and storage capabilities, 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 Quarterly Report on Form 10-Q for the quarter
ended September 30, 2021 and subsequent filings with the SEC,
including our Annual Report on Form 10-K for the year ended
December 31, 2021, which will be filed on or about the date hereof,
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
Rubius Therapeutics (NASDAQ:RUBY)
Historical Stock Chart
From Aug 2024 to Sep 2024
Rubius Therapeutics (NASDAQ:RUBY)
Historical Stock Chart
From Sep 2023 to Sep 2024