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 second quarter 2021
financial results and provided a business update.
“During the quarter, we continued to make excellent progress in
advancing our fully owned oncology pipeline,” said Pablo J.
Cagnoni, M.D., President and Chief Executive Officer. “Given the
favorable emerging safety profile and promising initial clinical
activity reported for RTX-240, we initiated a combination Phase 1
arm with pembrolizumab with the goal of providing benefit to
patients with cancers that have relapsed or are refractory after
treatment with anti-PD-1 or PD-L1 antibodies.”
“Bolstered by the promising clinical activity and safety
reported as part of the initial data results in March 2021, and no
dose-limiting toxicities observed to date, we are continuing to
explore the dose and schedule in new cohorts in the Phase 1 arm of
single-agent RTX-240 in advanced solid tumors,” said Christina
Coughlin, M.D., Ph.D., Chief Medical Officer. “We plan to present a
comprehensive clinical data set from the trial, including data from
the new cohorts and longer follow up for all patients.”
Second Quarter 2021 and Recent Highlights
Strengthened Leadership Team
- Dannielle Appelhans was appointed chief operating officer. She
will oversee corporate strategy and technical operations. Dannielle
brings significant experience in building organizations as they
evolve from early- to late-stage development, with a particular
focus on clinical and commercial manufacturing and scaling supply
chains.
Single-Agent RTX-240 Phase 1/2 Clinical Trial for
Advanced Solid Tumors
- During the second quarter, two additional cohorts were added to
explore the dose of RTX-240 with a more frequent schedule of
administration in the Phase 1 single-agent arm in solid tumors.
Enrollment in these cohorts continues.
- The changes in dose and schedule are supported by initial
clinical activity, safety data and no dose-limiting toxicities
observed to date along with additional emerging data from dose
escalation in the Phase 1 study.
- The Company currently plans to present comprehensive results
from this study at the end of 2021 or during the first quarter of
2022.
- The Company presented preliminary safety and efficacy data from
RTX-240 Phase 1/2 clinical trial for advanced solid tumors at the
American Association for Cancer Research (AACR) Virtual Annual
Meeting on April 10, 2021.
- The initial safety (n=16) and efficacy (n=15) findings, based
on RECIST v1.1., with a data cutoff of February 28, 2021,
demonstrated favorable emerging safety results across dose levels
with no treatment-related Grade 3/4 adverse events or dose-limiting
toxicities.
- Single-agent activity was demonstrated with two partial
responses (1 confirmed and 1 unconfirmed) and six patients with
stable disease.
- RTX-240 stimulated innate and adaptive immunity as demonstrated
by the activation and expansion of NK or memory CD8+ T cells in all
patients, with 9/16 patients showing activation and expansion in
both cell types.
- Additionally, trafficking data was separately reported as part
of the initial data readout on March 15, 2021, which noted that
immune cell trafficking of activated NK and T cells into the tumor
microenvironment (TME) was observed in two solid tumor biopsies and
one acute myeloid leukemia (AML) biopsy.
Phase 1 Arm in Ongoing Phase 1/2 RTX-240 Clinical Trial
in Combination with
KEYTRUDA® (pembrolizumab)
for Advanced Solid Tumors
- The first patient was dosed with RTX-240 in combination with
pembrolizumab for the treatment of patients with
relapsed/refractory or locally advanced solid tumors in June 2021.
- To be eligible for the trial, patients must have disease that
is relapsed or refractory to an anti-PD-1 or PD-L1 therapy. The
study continues to enroll additional patients.
- With its mechanism of action as a broad immune agonist, RTX-240
may have synergy with immune checkpoint inhibition and potentially
overcome resistance to PD-1 inhibition.
- Preliminary data from single-agent RTX-240 Phase 1 study
reported in March 2021, showed early evidence of favorable
immune-permissive changes in the TME, including increased
expression of PD-L1 and/or increased ratio of M1/M2 macrophages
after treatment with RTX-240 in three out of four patient biopsies,
suggesting single-agent RTX-240 is able to induce changes in the
TME that have been associated with response to checkpoint
inhibition.
Phase 1 Arm in Ongoing Phase 1/2 RTX-240 Clinical Trial
in Relapsed/Refractory (R/R) Acute Myeloid Leukemia
- 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 R/R AML.
- Based on the initial clinical and pharmacodynamic data observed
in the single-agent solid tumor Phase 1 arm, the Company
implemented a more frequent dose administration schedule and added
an additional cohort in the R/R AML arm of the trial.
- Dosing has been completed in the first four dose cohorts and
enrollment in an additional dose cohort is expected to begin in the
third quarter of 2021.
- Given the mechanism of RTX-240 in activating and expanding NK
and T cells and its preliminary favorable safety profile seen to
date, RTX-240 could potentially provide benefit as a maintenance
therapy for AML patients in remission following chemotherapy or
stem cell transplantation.
RTX-321 Artificial Antigen-Presenting Cell (aAPC)
Development Program for Human Papillomavirus (HPV) 16-Positive
Cancers
- Enrollment continues in the Phase 1 clinical trial of RTX-321
in patients with advanced HPV 16-positive cancers, including
cervical, head & neck cancers, and anal cancer.
Peer-Reviewed Publications and Poster Presentations at
Medical Conferences
- In July 2021, the manuscript entitled “Anti-Tumor Effects of
RTX-240: an Engineered Red Blood Cell Expressing 4-1BB Ligand and
Interleukin-15” was published in the peer-reviewed
journal Cancer Immunology, Immunotherapy, highlighting
preclinical findings for RTX-240, which demonstrated that RTX-240
activates and expands CD8+ T cells and NK cells in vitro and in
vivo generating potent anti-tumor activity in both a colorectal and
melanoma model.
- In May 2021, the manuscript entitled, “Engineered Red Blood
Cells as an Off-the-Shelf Allogeneic Anti-Tumor Therapeutic” was
published in the peer-reviewed journal Nature Communications
highlighting preclinical findings for RTX-321, which demonstrated
that the surrogate of RTX-321 induced a broad immune response,
epitope spreading and memory formation in preclinical models.
- 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.
Near-Term Catalysts and Operational
Objectives
- Present additional clinical data from the RTX-240 solid tumor
Phase 1 clinical trial at the end of 2021 or during the first
quarter of 2022;
- 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;
- 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.
Second Quarter Financial ResultsNet loss for
the second quarter of 2021 was $50.2 million or $0.56 per common
share, compared to $37.9 million or $0.47 per common share in the
second quarter of 2020.
In the second quarter of 2021, Rubius invested $36.1 million in
research and development (R&D) related to its novel RED
PLATFORM® and towards expanding and advancing its product pipeline,
as compared to $26.1 million in the second quarter of 2020. This
year-over-year increase was principally due to a $9.1 million
increase in costs incurred for the Company’s lead cancer programs,
including, RTX-240 and RTX-321, primarily CRO and internal
manufacturing costs incurred in connection with both arms of its
Phase 1/2 clinical trial of RTX-240 for the treatment of solid
tumors and AML and for its Phase 1 clinical trial of RTX-321 for
the treatment of HPV 16-positive cancers. Additionally,
personnel-related costs increased $1.2 million for additions to
headcount to support the Company’s expanded operations and
stock-based compensation expense increased by $1.0 million.
Increases in oncology program expenses, personnel-related costs and
stock-based compensation expense were offset by a $1.5 million
reduction in contract R&D, laboratory supplies and research
materials driven primarily by a shift in research activities to
support clinical programs.
General and administrative (G&A) expenses were $13.9 million
during the second quarter of 2021, as compared to $11.6 million for
the second quarter of 2020. The higher costs were driven by a $1.0
million increase in personnel-related costs to support rising
headcount in the Company’s general and administrative function,
including recruitment costs, as well as a $0.9 million increase in
professional and consultant fees and facility-related expenses.
Six Month Financial Results Net loss for the
first six months of 2021 was $92.5 million or $1.08 per common
share, compared to $86.3 million or $1.07 per common share in the
first six months of 2020.
In the six months ended June 30, 2021, Rubius invested $63.7
million in R&D related to its novel RED PLATFORM® and towards
expanding and advancing its product pipeline, as compared to $62.3
million in the first six months of 2020. The year-over-year
increase was driven primarily by a $10.9 million increase in costs
incurred for the Company’s lead cancer programs. These costs were
incurred for its Phase 1/2 clinical trial of RTX-240 for the
treatment of solid tumors, including clinical CRO and internal
manufacturing costs, as well as costs incurred for its Phase 1
clinical trial of RTX-321 in patients with advanced HPV 16-positive
cancers. This increase was partially offset by a $6.7 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 decreased by $2.8 million due
principally to reductions in contract R&D, laboratory supplies
and research materials as research activities shifted to support
clinical programs.
G&A expenses were $27.1 million during the first six months
of 2021, as compared to $24.3 million for the same period in 2020.
The higher costs were driven by a $1.0 million increase in
personnel-related costs, including recruitment costs, as well as a
$1.7 million increase in professional and consultant fees and
facility-related expenses.
During the first six months of 2021, other income and expenses
decreased by $1.9 million, from other income, net of $0.2 million
during the first six months of 2020, to other expense, net of $1.7
million. The change was principally due to 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 June 30, 2021, cash, cash
equivalents and investments were $298.2 million, compared to $176.3
million as of December 31, 2020. 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, during the second quarter, 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 monthsended June 30, |
|
|
For the six monthsended June
30, |
|
|
2021 |
|
|
2020 |
|
|
2021 |
|
|
2020 |
|
Revenue |
$ |
|
|
|
$ |
|
|
|
$ |
|
|
|
$ |
|
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Research and development |
|
36,072 |
|
|
|
26,096 |
|
|
|
63,749 |
|
|
|
62,282 |
|
General and administrative |
|
13,851 |
|
|
|
11,601 |
|
|
|
27,091 |
|
|
|
24,265 |
|
Total operating expenses |
|
49,923 |
|
|
|
37,697 |
|
|
|
90,840 |
|
|
|
86,547 |
|
Loss from operations |
|
(49,923 |
) |
|
|
(37,697 |
) |
|
|
(90,840 |
) |
|
|
(86,547 |
) |
Other income (expense), net |
|
(257) |
|
|
|
(157) |
|
|
|
(1,670) |
|
|
|
207 |
|
Net loss |
$ |
(50,180 |
) |
|
$ |
(37,854 |
) |
|
$ |
(92,510 |
) |
|
$ |
(86,340 |
) |
Net loss per share, basic and
diluted |
$ |
(0.56 |
) |
|
$ |
(0.47 |
) |
|
$ |
(1.08 |
) |
|
$ |
(1.07 |
) |
Weighted average common shares
outstanding, basic and diluted: |
|
89,517,784 |
|
|
|
80,481,756 |
|
|
|
85,936,079 |
|
|
|
80,376,830 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Rubius Therapeutics,
Inc.Condensed Consolidated Balance Sheet
Data(in
thousands)(unaudited)
|
June 30, |
|
|
December 31, |
|
|
2021 |
|
|
2020 |
|
Cash, cash equivalents and investments |
$ |
298,242 |
|
|
$ |
176,287 |
|
Total assets |
|
396,084 |
|
|
|
277,794 |
|
Total liabilities |
|
131,970 |
|
|
|
136,234 |
|
Total stockholders’
equity |
|
264,114 |
|
|
|
141,560 |
|
|
|
|
|
|
|
|
|
About Red Cell TherapeuticsEnabled 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.
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 cellular
therapy 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 retaining the ability to
activate and expand 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 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 Top
Places to Work in Massachusetts by the Boston Globe in 2020, and
its manufacturing site was recently named 2021 Top 5 Best Places to
Work in Rhode Island among medium-sized companies 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 and safety profile
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 interpretations of preliminary data, including
indications as to 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, synergies of
RTX-240 with immune checkpoint inhibition, the potential for
RTX-240 to overcome resistance to PD-1 inhibition and that RTX-240
could provide benefit as a maintenance therapy for certain AML
patients, our expectations regarding and plans to present the full
data set from the Phase 1 clinical trial, 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, goals for the RTX-240 Phase 1 arm
study, expectations for our strengthened leadership team, 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-Q for the
quarter ended March 31, 2021, and subsequent filings with the SEC,
including our upcoming Form 10-Q for the quarter ended June 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, 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
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