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 second quarter 2022 financial
results and provided a business update.
“We continue to advance enrollment in the NSCLC and RCC
expansion cohorts of our Phase 1 clinical trial of RTX-240 in
combination with pembrolizumab, with initial clinical results
expected in the second half of 2022, including data in advanced
solid tumors and data from the initial patients with NSCLC and RCC
enrolled in the study. Enrollment also progressed in the Phase 1/2
clinical trial of RTX-224 in select advanced solid tumors, with
initial results expected by year-end or in the first quarter of
2023,” said Pablo J. Cagnoni, M.D., president and chief executive
officer. “Also, during the quarter, we presented new preclinical
data, as part of our type 1 diabetes program, that demonstrated
prevention of diabetes and bystander suppression in the stringent
non-obese diabetes preclinical model. We plan to select a clinical
candidate for our type 1 diabetes program later this year.”
Recent Highlights
Oncology
RTX-240 + Pembrolizumab in Advanced Solid
Tumors
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 an anti-tumor response.
- Advanced enrollment in the expansion
cohorts of the Phase 1 combination arm of RTX-240 plus
pembrolizumab in patients with advanced solid tumors
- Enrolling up to 20 patients who have
experienced disease progression with 1-2 prior treatment regimens
in the metastatic setting in preparation for a future Phase 2
clinical trial of RTX-240 in combination with pembrolizumab in an
earlier line of therapy
- Initial clinical results in advanced
solid tumors and data from the initial enrolled NSCLC and RCC
patients expected in 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 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.
- Continued dose escalation in the Phase 1/2 clinical trial of
RTX-224 in select relapsed/refractory or locally advanced solid
tumors, including non-small cell lung cancer, cutaneous melanoma,
head and neck squamous cell carcinoma, urothelial (bladder)
carcinoma and triple-negative breast cancer
- Initial clinical results are expected by year-end or during the
first quarter of 2023
Autoimmunity
Type 1 Diabetes
RCT product candidates are engineered to express specific
autoantigens that are the target of immune responses which cause
autoimmune disease. This approach takes advantage of the natural
tolerogenic properties of red blood cells. RCTs are designed to
suppress, modulate or eliminate disease-causing cells. Type 1
diabetes is a T-cell driven autoimmune disease with well-defined
antigens, making it an ideal disease indication for this
antigen-specific tolerance approach.
In June, the Company presented new preclinical data for its type
1 diabetes program at the FOCIS 2022 Annual Meeting in San
Francisco:
- Demonstrated tolerance induction and
bystander suppression in stringent type 1 diabetes preclinical
models
- From ongoing experiment, showed new
efficacy data in the NOD preclinical model
- By increasing to 3 doses
administered and optimizing the dosing schedule, bystander
suppression was achieved at 25 weeks by delivering only two
antigens, indicating disease prevention caused by many
autoantigens
- Established efficacy in the BDC2.5
adoptive transfer model with data supporting that repeated dosing
extends 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
- These findings are potentially
translatable beyond type 1 diabetes to multiple autoimmune
diseases, including multiple sclerosis and celiac disease
Leadership Update
In July 2022, Rubius announced that Susanne Schaffert, Ph.D.,
joined its board of directors. Dr. Schaffert most recently served
as President of Novartis Oncology and brings more than 25 years of
experience across clinical development, marketing and sales,
finance and commercialization in the global pharmaceutical and
biotechnology industries, with a focus on oncology, immuno-oncology
and cell therapy.
Anticipated 2022 Catalysts and Operational
Objectives
To evaluate the full potential of RTX-240, Rubius’ other
oncology programs and the RED PLATFORM, Rubius plans to execute
several critical milestones in the near term and, based on its
current cash and cash equivalents, including borrowings under its
credit facility, has sufficient cash runway into the second half of
2023:
- Report initial Phase 1 clinical results for RTX-240 in
combination with pembrolizumab in advanced solid tumors and data
from the initial enrolled 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 by year-end or during the first
quarter of 2023.
Second Quarter Financial Results
Net loss for the second quarter of 2022 was $44.2 million or
$0.49 per common share, compared to $50.2 million or $0.56 per
common share in the second quarter of 2021.
In the second quarter of 2022, Rubius invested $33.0 million in
research and development (R&D) related to its novel RED
PLATFORM® and towards expanding and advancing its product pipeline,
as compared to $36.1 million in the second quarter of 2021. The
year-over-year decrease was principally due to a decrease in direct
costs of $2.9 million related to the deprioritization of RTX-321
and RTX-240 AML and monotherapy studies. We expect these costs to
continue to decrease in future periods. This decrease was partially
offset by an increase in clinical costs related to RTX-224.
Platform development, early-stage research and unallocated expenses
decreased by $0.1 million primarily due to a decrease of $0.8
million in stock-based compensation expense related to a reduction
in the market price of our common stock, resulting in a lower
valuation of stock options granted in 2022, and a decrease of $0.3
million in facility-related and other expenses due to lower spend
on non-capitalized software costs in the current year. These
decreases were partially offset by an increase of $0.4 million in
contract research and development related to drug discovery
activities and platform development. Additionally,
personnel-related costs increased by $0.5 million to support our
prioritization of clinical programs.
G&A expenses were $9.9 million during the second quarter of
2022, as compared to $13.9 million for the second quarter of 2021.
The lower costs were primarily due to a decrease in stock-based
compensation expense of $3.7 million, which was driven by stock
option awards that fully vested during the second half of 2021 and
first half of 2022, as well as a reduction in the market price of
our common stock, resulting in a lower valuation of stock options
granted in 2022.
Six Month Financial Results
Net loss for the first six months of 2022 was $96.7 million or
$1.07 per common share, compared to $92.5 million or $1.08 per
common share in the first six months of 2021.
In the six months ended June 30, 2022, Rubius invested $71.3
million in R&D related to its novel RED PLATFORM® and towards
expanding and advancing its product pipeline, as compared to $63.7
million in the first six months of 2021. The year-over-year
increase was driven primarily by an increase in direct costs of
$2.1 million related to an increase in preclinical and clinical
costs associated with RTX-224 and RTX-240, principally due to
clinical research organization, or CRO, costs and internal
manufacturing costs. This increase was partially offset by a
decrease in clinical costs related to RTX-321 due to start-up
activities in the prior period and current period deprioritization
of clinical development. We expect these costs to decrease in
future periods. Platform development, early-stage research and
unallocated expenses increased by $5.4 million principally due an
increase of $2.9 million in personnel-related costs related to
headcount increases through the second half of 2021 to support
operations. Additionally, increases of $1.7 million in contract
research and development and $1.0 million in laboratory supplies
and research materials were related to drug discovery activities
and platform development. These increases were partially offset by
a decrease of $0.4 million in facility-related and other expenses
due to lower spend on non-capitalized software costs and a
reduction in building operating costs in the current year.
G&A expenses were $22.5 million during the first six months
of 2022, as compared to $27.1 million for the same period in 2021.
The lower costs were due to a decrease in stock-based compensation
expense of $5.0 million, which was driven by stock option awards
that fully vested during the second half of 2021 and first half of
2022, as well as a reduction in the market price of our common
stock, resulting in a lower valuation of stock options granted in
2022. The decrease was partially offset by an increase in
personnel-related expenses of $0.3 million driven by additions to
headcount in our general and administrative function.
Cash Position
As of June 30, 2022, cash, cash equivalents and investments were
$140.7 million (including $75 million in borrowings under its
credit facility), compared to $225.8 million as of December 31,
2021. During the first six months of 2022, the Company used $81.4
million of cash to fund operations, $78.4 million to purchase
investments and $4.2 million to fund capital expenditures,
consisting mostly of renovation costs incurred at our manufacturing
facility.
Rubius Therapeutics,
Inc.Condensed Consolidated Statement of
Operations(in thousands, except share and per
share data)(unaudited)
|
For the three months ended June 30, |
|
|
For the six months ended June
30, |
|
|
2022 |
|
|
2021 |
|
|
2022 |
|
|
2021 |
|
Revenue |
$ |
- |
|
|
$ |
- |
|
|
$ |
- |
|
|
$ |
- |
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Research and development |
|
32,998 |
|
|
|
36,072 |
|
|
|
71,297 |
|
|
|
63,749 |
|
General and administrative |
|
9,908 |
|
|
|
13,851 |
|
|
|
22,471 |
|
|
|
27,091 |
|
Total operating expenses |
|
42,906 |
|
|
|
49,923 |
|
|
|
93,768 |
|
|
|
90,840 |
|
Loss from operations |
|
(42,906 |
) |
|
|
(49,923 |
) |
|
|
(93,768 |
) |
|
|
(90,840 |
) |
Other income (expense), net |
|
(1,335 |
) |
|
|
(257 |
) |
|
|
(2,885 |
) |
|
|
(1,670 |
) |
Net loss |
$ |
(44,241 |
) |
|
$ |
(50,180 |
) |
|
$ |
(96,653 |
) |
|
$ |
(92,510 |
) |
Net loss per share, basic and
diluted |
$ |
(0.49 |
) |
|
$ |
(0.56 |
) |
|
$ |
(1.07 |
) |
|
$ |
(1.08 |
) |
Weighted average common shares outstanding, basic and diluted: |
|
90,257,524 |
|
|
|
89,517,784 |
|
|
|
90,203,586 |
|
|
|
85,936,079 |
|
Rubius Therapeutics,
Inc.Condensed Consolidated Balance Sheet
Data(in
thousands)(unaudited)
|
June 30, |
|
December 31, |
|
2022 |
|
2021 |
Cash, cash equivalents and investments |
$ |
140,713 |
|
$ |
225,848 |
Total assets |
|
229,082 |
|
|
318,021 |
Total liabilities |
|
133,542 |
|
|
139,239 |
Total stockholders’
equity |
|
95,540 |
|
|
178,782 |
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 named among the 2021 Top Places to
Work in Massachusetts by the Boston Globe, and its manufacturing
site was recently named 2022 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-224 and RTX-240, beliefs about the opportunities for and
advantages of our drug candidates, plans and timing to execute on
critical milestones and our ability to fund those activities, our
interpretations of data, including as to the efficacy of our
product candidates, expectations regarding the therapeutic
potential and safety profile of our pipeline candidates, beliefs
about patients’ needs, expectations for our cash position and
runway, as well as beliefs about our manufacturing plans and
accomplishments. 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, risks related to the substantial doubt about our
ability to continue as a going concern given that we currently do
not have adequate financial resources to fund our forecasted
operating costs for at least twelve months and have significant
debt outstanding, our ability to pursue and secure financing to
fund our operations, 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, including our Quarterly Report on Form 10-Q for the
quarter-ended June 30, 2022, 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: InvestorsLori
Murray, Chief Corporate Affairs Officer
lori.murray@rubiustx.com
Media Marissa Hanify, Director, Corporate
Communicationsmarissa.hanify@rubiustx.com
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