Rubius Therapeutics, Inc. (Nasdaq:RUBY), a clinical-stage
biopharmaceutical company that is genetically engineering red blood
cells to create an entirely new class of cellular medicines called
Red Cell Therapeutics™, today reported first quarter 2021 financial
results and provided a business update.
“We had a great start to the year by reporting initial positive
data from our ongoing Phase 1/2 clinical trial of RTX-240 in
patients with advanced solid tumors, which provided evidence of the
broad potential of the RED PLATFORM® across our entire pipeline of
cancer and autoimmune programs,” said Pablo J. Cagnoni, M.D.,
president and chief executive officer of Rubius Therapeutics. “With
several expected clinical milestones from our pipeline, the coming
year is poised to be an exciting one for patients, our employees
and shareholders.”
Enabled by the RED PLATFORM, Red Cell Therapeutics’ (RCTs) are
expected to provide advantages over other therapies by potentially
generating a broad anti-tumor response with limited side effects
and a wide therapeutic window given the biodistribution of RCTs to
the vasculature and spleen. Additionally, RCTs do not have the
complex supply chain and administration logistics of other cell
therapies, as RCTs are designed to be prepared in the pharmacy,
administered in an outpatient setting and do not require
lymphodepletion prior to administration.
First Quarter 2021 Highlights
RTX-240 Phase 1/2 Clinical Program for Advanced Solid
Tumors On March 15, 2021, the Company reported preliminary
safety (n=16) and efficacy (n=15) findings based on RECIST v1.1.,
with a data cutoff of February 28, 2021. The key takeaways from the
initial data were:
- RTX-240 demonstrated favorable emerging safety results across
dose levels:
- There were no treatment-related Grade 3/4 adverse events and no
dose-limiting toxicities. The most common treatment-related Grade
1/2 adverse events were fatigue, chills, nausea, decreased appetite
and arthralgias. There was a single Grade 1 event of liver
toxicity.
- Single-agent activity was observed with two partial responses:
- A confirmed partial response (PR) in a patient with metastatic
anal cancer and an unconfirmed PR in a patient with metastatic
uveal melanoma. Both patients’ disease had progressed on prior
anti-PD-1 therapy.
- Stable disease (SD) was observed in six patients, including
four individual patients with stable disease for at least 12 weeks,
(one each with non-small cell lung cancer, soft tissue sarcoma,
pancreatic cancer and prostate cancer).
- Pharmacodynamic effects observed included the activation and/or
expansion of the key natural killer (NK) and/or T cells types in
all patients (n=16).
- Observed evidence of immune cell trafficking of activated NK
and T cells into the tumor microenvironment in two solid tumor
biopsies and one AML biopsy.
Dose optimization and enrollment continues in the RTX-240 Phase
1/2 advanced solid tumor study. The Company plans to present
additional data from the study this year.
Phase 1 Arm in Ongoing Phase 1/2 RTX-240 Clinical Trial
in Relapsed/Refractory Acute Myeloid Leukemia (AML)
- RTX-240 is currently being evaluated as a single-agent in a
Phase 1 arm of the ongoing Phase 1/2 clinical trial of RTX-240 in
patients with relapsed/refractory AML.
- As of May 10, 2021, Rubius is enrolling patients in the third
and fourth dose cohorts, in accordance with the statistical design
of the study, which allows enrollment of two dose cohorts
simultaneously.
- On March 15, 2021, the Company presented preliminary
trafficking data from the first patient in the trial, indicating
strong accumulation of activated, granzyme B-positive NK and T
cells in the bone marrow, which is the site of disease in AML.
RTX-321 Artificial Antigen-Presenting Cell (aAPC)
Development Program for Human Papillomavirus (HPV) 16-Positive
Cancers
- Dosing additional patients in the Phase 1 clinical trial of
RTX-321 in patients with advanced HPV 16-positive cancers,
including cervical, head and neck and anal cancer.
- RTX-321 has a unique frozen drug substance formulation,
enabling a potential truly off-the-shelf product with a shelf life
of up to several years.
- Following liquid reformulation, RTX-321 has an in-vial shelf
life of approximately 52 days.
Presentations at Medical Conferences
- Preliminary safety and efficacy data from RTX-240 Phase 1/2
Clinical Trial for advanced solid tumors was presented at the
American Association for Cancer Research (AACR) Virtual Annual
Meeting on April 10, 2021.
Anticipated 2021 Catalysts and Operational
Objectives
- Present additional clinical data from the RTX-240 solid tumor
Phase 1 clinical trial;
- Select specific solid tumor types that will be pursued in the
Phase 2 expansion cohort of RTX-240;
- Report initial clinical data from the Phase 1 arm of the
RTX-240 clinical trial in relapsed/refractory AML;
- Initiate the Phase 1 clinical trial of RTX-240 in combination
with anti-PD-1 therapy in advanced solid tumors in the second half
of 2021;
- Report initial Phase 1 clinical data from RTX-321 for the
treatment of HPV 16-positive cancers by the first quarter of 2022;
and
- Submit an Investigational New Drug Application for RTX-224 by
year-end.
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, Rubius Therapeutics’
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.
First Quarter 2021 Financial ResultsNet loss
for the first quarter of 2021 was $42.3 million or $0.51 per common
share, compared to $48.5 million or $0.60 per common share in the
first quarter of 2020.
In the first quarter of 2021, Rubius invested $27.7 million in
research and development (R&D) related to its novel RED
PLATFORM® and towards expanding and advancing its product pipeline,
compared to $36.2 million in the first quarter of 2020. This
year-over-year decrease was driven primarily by a $4.9 million
reduction in program expenses consisting of a $6.7 million
reduction in rare disease program costs, following the
deprioritization of the Company’s rare disease pipeline in March
2020, partially offset by an increase in costs incurred for the
Company’s cancer programs, including, RTX-240 and RTX-321.
Additionally, platform development, early stage research and other
unallocated expenses decreased by $3.6 million due principally to
$2.5 million in reductions in contract R&D, laboratory supplies
and research materials driven primarily by a shift in activities to
support the oncology clinical programs. Personnel-related costs
also decreased as a result of non-recurring costs incurred in the
first quarter of 2020.
G&A expenses were $13.2 million during the first quarter of
2021, compared to $12.7 million for the first quarter of 2020. The
higher costs were primarily driven by an increase in professional
and consultant fees related to increased patent costs and an
increase in facility-related and other expenses due to higher
building operating costs.
During the first quarter of 2021, other income and expenses
decreased by $1.8 million, from net income of $0.4 million in the
first quarter of 2020, to net expense of $1.4 million. The
change was due to a lower average cash balance, lower prevailing
interest rates and an increase in outstanding debt following the
final borrowing under the Company’s debt facility in June 2020.
Cash PositionAs of March 31, 2021, cash, cash
equivalents and investments were $330.7 million, compared to $176.3
million as of December 31, 2020. During the quarter, the Company
received net proceeds, after deducting underwriting discounts and
commission, of $188.0 million in connection with its underwritten
public offering completed in March 2021.
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 genetically 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
2020 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 our
expectations regarding the therapeutic potential of our pipeline of
Red Cell Therapeutics, including RTX-240 for the treatment of
patients with relapsed/refractory or locally advanced solid tumors
and its advantages over other therapies and RTX-321 for the
treatment of HPV 16-positive cancers, our expectations regarding
the timing, enrollment, additional data from and success of the
current and future cohorts and phases of the clinical trial of
RTX-240, our expectations regarding the biological effects of
RTX-240 on innate and adaptive immunity, including activation and
increased numbers of NK cells and T cells in the clinical trial of
RTX-240, and our expectations regarding the full data set from the
Phase 1 clinical trial and its ability to unlock the potential of
the RED PLATFORM across our entire pipeline of Red Cell
Therapeutics for the treatment of cancer, including our
expectations regarding the therapeutic potential of RTX-321,
timelines related to the Phase 1 clinical trial of RTX-321 and our
release of initial data from such trial, our expectations regarding
the timing of an IND for RTX-224, our expectations regarding our
ability to expand manufacturing capabilities, our expectations
regarding the potential shelf life of our frozen drug substance for
RTX-321, our expectations regarding our cash runway and 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 and
other risks identified in our SEC filings, including our Quarterly
Report on Form 10-K for the year ended December 31, 2020, 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. We
explicitly disclaim any obligation to update any forward-looking
statements.
Contacts: InvestorsElhan Webb,
CFA, Vice President of Investor
Relationselhan.webb@rubiustx.com
Media Marissa Hanify, Director, Corporate
Communicationsmarissa.hanify@rubiustx.com
Dan Budwick, 1AB+1 (973) 271-6085dan@1abmedia.com
Rubius Therapeutics,
Inc.Condensed Consolidated Statement of
Operations(in thousands, except share and per
share data)(unaudited)
|
|
For the three monthsended March
31, |
|
|
|
2021 |
|
2020 |
|
Revenue |
|
$ |
|
|
$ |
|
|
Operating expenses: |
|
|
|
|
|
|
|
Research and development |
|
|
27,677 |
|
|
|
36,186 |
|
General and administrative |
|
|
13,240 |
|
|
|
12,664 |
|
Total operating expenses |
|
|
40,917 |
|
|
|
48,850 |
|
Loss from operations |
|
|
(40,917 |
) |
|
|
(48,850 |
) |
Other income (expense), net |
|
|
(1,413 |
) |
|
|
364 |
|
Net loss |
|
$ |
(42,330 |
) |
|
$ |
(48,486 |
) |
Net loss per share, basic and
diluted |
|
$ |
(0.51 |
) |
|
$ |
(0.60 |
) |
Weighted average common shares outstanding, basic and diluted: |
|
|
82,314,577 |
|
|
|
80,271,848 |
|
|
|
|
|
|
|
|
|
Rubius Therapeutics,
Inc.Condensed Consolidated Balance Sheet
Data(in
thousands)(unaudited)
|
March 31, |
|
December 31, |
|
2021 |
|
2020 |
Cash, cash equivalents and investments |
$ |
330,650 |
|
$ |
176,287 |
Total assets |
|
430,230 |
|
|
277,794 |
Total liabilities |
|
129,247 |
|
|
136,234 |
Total stockholders’
equity |
|
300,983 |
|
|
141,560 |
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