-- Received Protocol Assistance Feedback from
European Medicines Agency on the Phase 2 Study for RLYB212; Company
to Proceed Forward with Clinical Trial Application Process in
Europe --
-- Phase 2 Dose Confirmation Study for RLYB212
in Pregnant Women at Higher Risk for FNAIT Expected to Initiate in
2H 2024 --
-- $109.9 million Cash, Cash Equivalents and
Marketable Securities as of December 31, 2023; Provides Runway into
Mid-2026 --
Rallybio Corporation (Nasdaq: RLYB), a clinical-stage
biotechnology company committed to identifying and accelerating the
development of life-transforming therapies for patients with severe
and rare diseases, today reported financial results for the fourth
quarter and full year ended December 31, 2023 and provided an
update on recent program and corporate developments.
“We are encouraged by the continued progress we made across our
portfolio throughout 2023. Following our February 2024 announcement
regarding the prioritization of our portfolio and reorganization of
our operations, we believe our existing capital will fund our
revised operating plan into the middle of mid-2026 and intend to
focus our efforts in 2024 on our clinical-stage programs, RLYB212
and RLYB116. We have received feedback from the European Medicines
Agency for RLYB212 and are pleased to now move forward with the
clinical trial application process in Europe where we will conduct
the Phase 2 dose confirmation study for RLYB212 in pregnant women
at higher risk for FNAIT. The Phase 2 study remains on track to
initiate in the second half of 2024,” said Stephen Uden, M.D.,
Chief Executive Officer of Rallybio.
Dr. Uden continued, “In parallel, the manufacturing work for
RLYB116 is ongoing and we are encouraged by the data indicating the
potential to achieve improved tolerability at higher doses. We
expect to complete this work and provide an update on the
development plan for RLYB116 in the second half of 2024. We also
remain committed to advancing our preclinical programs to the next
milestone and will seek alternative options to advance and or
finance these programs with a goal of realizing the value of these
programs.”
Recent Business Highlights and Upcoming Milestones:
Corporate Updates
In February 2024, Rallybio announced a prioritization of its
portfolio and 45% workforce reduction to focus the Company’s
resources on its two Phase 2 ready assets, RLYB212, a novel human
monoclonal anti-HPA-1a antibody in development for the prevention
of fetal and neonatal alloimmune thrombocytopenia (FNAIT), and
RLYB116, a once-weekly, low volume subcutaneously injected
inhibitor of complement component 5 (C5) in development for
patients with complement mediated diseases. With these changes,
Rallybio announced that the Company expects its cash runway to
extend into the middle of 2026.
The workforce reduction included 19 roles and will be
substantially complete by the end of the first quarter of 2024.
Rallybio estimates that the workforce reduction will result in
aggregate charges of approximately $3.3 million primarily for
one-time employee severance and benefit costs, excluding
share-based compensation expense.
Maternal Fetal Blood Disorders
In November 2023, Rallybio announced preliminary data from the
completed multiple dose cohort of the Phase 1 safety and
pharmacokinetics (PK) study for RLYB212. The Phase 1 multiple dose
cohort for RLYB212 was initiated in the first quarter of 2023 to
evaluate the safety and PK of subcutaneous (SC) RLYB212 based on
repeat dosing over 12 weeks. The preliminary data demonstrated that
multiple dose PK were consistent both within and between subjects
and, together with the Company’s clinical pharmacology modeling
predictions, support a once monthly dosing regimen for the planned
Phase 2 study in pregnant women at higher risk for FNAIT.
Consistent with previously reported data, RLYB212 was observed to
be generally well-tolerated with no reports of injection site
reactions or serious adverse events.
Rallybio announced today that the Company has received Protocol
Assistance Feedback from the European Medicines Agency (EMA) on the
RLYB212 Phase 2 study, and the Company is now moving forward with
Clinical Trial Applications to support conduct of the Phase 2 study
in Europe. Rallybio continues to expect to initiate the Phase 2
dose confirmation study in pregnant women at higher risk for FNAIT
in the second half of 2024.
The Company continues to screen in the FNAIT natural history
alloimmunization study, a non-interventional study designed to
provide a contemporary dataset for HPA-1a alloimmunization
frequency in a racially and ethnically diverse population. The
Company expects screening for the natural history study to continue
simultaneously with execution of the Phase 2 dose confirmation
study. As of March 1, 2024, Rallybio has screened approximately
9,400 women in the natural history study, and expects to screen
between 20,000 and 30,000 women in the study.
Complement Dysregulation
In December 2023, Rallybio announced preliminary Phase 1
multiple ascending dose data for RLYB116. Results demonstrated that
a 100 mg low volume once-a-week dose of subcutaneously administered
RLYB116 achieved sustained mean reductions in free C5 of greater
than 93%. In addition, RLYB116 administered as a 100 mg once-a-week
dose was observed to be generally well tolerated with injection
site reaction as the most common adverse event (AE) in the cohort,
occurring in 60% of the participants in the cohort. All AEs with
the 100 mg weekly dose were mild in severity.
While the exposure concentrations of RLYB116 demonstrated in the
Phase 1 study are expected to be suitable for the treatment of
patients with generalized myasthenia gravis, the Company believes
ongoing manufacturing enhancements will enable higher exposure to
RLYB116, supporting the treatment of patients with a broader range
of complement-mediated diseases, including paroxysmal nocturnal
hemoglobinuria and antiphospholipid syndrome. The manufacturing
work is progressing and Rallybio is encouraged by the data
indicating the potential to achieve improved tolerability at higher
doses.
Rallybio continues to expect to complete this manufacturing work
in the second half of 2024 and to provide an update on the
development plan for RLYB116 at that time.
Rallybio, together with its partner EyePoint Pharmaceuticals,
Inc., continue to evaluate sustained delivery of Rallybio’s
inhibitor of C5 using EyePoint’s proprietary technology for
sustained intraocular drug delivery. Rallybio and EyePoint expect
to provide an update on this collaboration in the first half of
2024.
Preclinical Programs
The revised operating plan will fund preclinical program
activities to important 2024 milestones, as described below. Beyond
achievement of these milestones, Rallybio will seek alternative
options to further advance these programs, including additional
partnerships and other forms of non-dilutive financing.
- RLYB331, Matriptase-2 (MTP-2) Inhibitor: Completion of
the ongoing preclinical activities, with data expected in the first
half of 2024.
- ENPP1 Inhibitor, Exscientia Partnership: Lead
AI-designed compounds enter candidate selection process in the
second half of 2024.
- AbCellera Partnership: Advancement of discovery efforts
to the next research milestone in the second half of 2024.
Fourth Quarter and Full Year 2023 Financial Results
- Research & Development (R&D) Expenses: R&D
expenses were $15.9 million for the fourth quarter of 2023,
compared to $10.8 million for the same period in 2022. R&D
expenses for the fourth quarter 2023 increased primarily due to an
increase in costs to advance RLYB212, RLYB116 and RLYB331 as
compared to the prior year. R&D expenses were $53.5 million for
the year ended December 31, 2023 compared to $40.7 million for the
year ended December 31, 2022. R&D expenses for the year ended
December 31, 2023 increased primarily due to an increase in costs
to advance RLYB212, RLYB116 and RLYB331 and additional R&D
related headcount costs, including share-based compensation expense
as compared to the prior year. These costs were offset by a
decrease in R&D asset acquisition costs and R&D costs
related to RLYB114.
- General & Administrative (G&A) Expenses: G&A
expenses were $5.2 million for the fourth quarter of 2023, compared
to $6.3 million for the same period in 2022. G&A expenses for
the fourth quarter of 2023 decreased primarily due to a decrease in
payroll and personnel-related costs, lower director and officer
insurance premiums and lower other general and administrative
related expenses as compared to the prior year. G&A expenses
were $25.4 million for the year ended December 31, 2023 compared to
$27.2 million for the year ended December 31, 2022. The decrease in
G&A expenses for the year ended December 31, 2023 was primarily
due to a decrease in payroll and personnel-related costs and lower
director and officer insurance premiums; offset by increases in
other general and administrative related expenses.
- Net Loss and Net Loss Per Common Share: Net loss was
$20.2 million for the fourth quarter of 2023, or net loss per
common share of $0.50 compared to a net loss of $16.2 million, or
net loss per common share of $0.46 for the fourth quarter of 2022.
Net loss was $74.6 million, or net loss per share of $1.84, for the
year ended December 31, 2023 compared to $66.7 million, or net loss
per share of $2.09, for the year ended December 31, 2022.
- Cash Position: As of December 31, 2023, cash, cash
equivalents, and marketable securities were $109.9 million.
About Rallybio
Rallybio (Nasdaq: RLYB) is a clinical-stage biotechnology
company with a mission to develop and commercialize
life-transforming therapies for patients with severe and rare
diseases. Rallybio has built a broad pipeline of promising product
candidates aimed at addressing diseases with unmet medical need in
areas of maternal fetal health, complement dysregulation,
hematology, and metabolic disorders. The Company has two clinical
stage programs: RLYB212, an anti-HPA-1a antibody for the prevention
of fetal and neonatal alloimmune thrombocytopenia (FNAIT) and
RLYB116, an inhibitor of complement component 5 (C5), with the
potential to treat several diseases of complement dysregulation, as
well as additional programs in preclinical development. Rallybio is
headquartered in New Haven, Connecticut. For more information,
please visit www.rallybio.com and follow us on LinkedIn and
Twitter.
Forward-Looking Statements
This press release contains forward-looking statements that are
based on our management’s beliefs and assumptions and on currently
available information. All statements, other than statements of
historical facts contained in this press release are
forward-looking statements. In some cases, forward-looking
statements can be identified by terms such as “may,” “will,”
“should,” “expect,” “plan,” “anticipate,” “could,” “intend,”
“target,” “project,” “contemplate,” “believe,” “estimate,”
“predict,” “potential” or “continue” or the negative of these terms
or other similar expressions, although not all forward-looking
statements contain these words. Forward-looking statements in this
press release include, but are not limited to, statements
concerning the timing of initiation of the Phase 2 dose
confirmation study for RLYB212, the release of screening numbers of
women in the natural history study, whether the results of the
natural history study and the planned Phase 2 dose confirmation
study will be sufficient to support design and implementation of a
Phase 3 registrational study for RLYB212, whether the manufacturing
work for RLYB116 will be timely completed or successful, our
expectations regarding the usefulness of data from our clinical
studies, the timing of achieving milestones in 2024 for our
preclinical programs, the likelihood that Rallybio will be
successful in developing RLYB212, RLYB116, or any of our other
product candidates, our ability to successfully identify and
implement alternative and acceptable options to further advance our
programs, expected costs related to the workforce reduction and
related charges, including the timing of such charges, the expected
use of operating cost savings associated with the updated operating
plan and the timing, our estimates of our capital requirements and
the sufficiency thereof, our ability to advance our portfolio, and
our cash runway. The forward-looking statements in this press
release are only predictions and are based largely on management’s
current expectations and projections about future events and
financial trends that management believes may affect Rallybio’s
business, financial condition and results of operations. These
forward-looking statements speak only as of the date of this press
release and are subject to a number of known and unknown risks,
uncertainties and assumptions, including, but not limited to, our
ability to successfully initiate and conduct our planned clinical
trials, including the FNAIT natural history study, and the Phase 2
clinical trial for RLYB212, and complete such clinical trial and
obtain results on our expected timelines, or at all, whether our
cash resources will be sufficient to fund our operating expenses
and capital expenditure requirements and whether we will be
successful raising additional capital, our ability to identify new
product candidates and successfully acquire such product candidates
from third parties, our ability to enter into strategic
partnerships or other arrangements, including the development of
RLYB114, competition from other biotechnology and pharmaceutical
companies, and those risks and uncertainties described in
Rallybio’s filings with the U.S. Securities and Exchange Commission
(SEC), including Rallybio’s Quarterly Report on Form 10-Q for the
period ended September 30, 2023, and subsequent filings with the
SEC. The events and circumstances reflected in our forward-looking
statements may not be achieved or occur and actual future results,
levels of activity, performance and events and circumstances could
differ materially from those projected in the forward-looking
statements. Except as required by applicable law, we are not
obligated to publicly update or revise any forward-looking
statements contained in this press release, whether as a result of
any new information, future events, changed circumstances or
otherwise.
Financial Tables
RALLYBIO CORPORATION
SELECTED CONDENSED
CONSOLIDATED FINANCIAL INFORMATION
Condensed Consolidated
Statements of Operations and Comprehensive Loss
(Unaudited)
FOR THE THREE MONTHS ENDED
DECEMBER 31,
FOR THE YEAR ENDED
DECEMBER 31
(in thousands, except share and per
share amounts)
2023
2022
2023
2022
Operating expenses:
Research and development
$
15,924
$
10,793
$
53,544
$
40,689
General and administrative
5,188
6,298
25,388
27,195
Total operating expenses
21,112
17,091
78,932
67,884
Loss from operations
(21,112
)
(17,091
)
(78,932
)
(67,884
)
Other income:
Interest income
1,448
1,071
6,147
1,963
Other income
35
31
262
342
Total other income, net
1,483
1,102
6,409
2,305
Loss before equity in losses of joint
venture
(19,629
)
(15,989
)
(72,523
)
(65,579
)
Loss on investment in joint venture
613
214
2,041
1,075
Net loss
$
(20,242
)
$
(16,203
)
$
(74,564
)
$
(66,654
)
Net loss per common share, basic and
diluted
$
(0.50
)
$
(0.46
)
$
(1.84
)
$
(2.09
)
Weighted-average common shares
outstanding, basic and diluted
40,639,567
35,516,630
40,447,388
31,821,311
Other comprehensive gain (loss):
Net unrealized gain (loss) on marketable
securities
223
220
229
(214
)
Other comprehensive gain (loss)
223
220
229
(214
)
Comprehensive loss
$
(20,019
)
$
(15,983
)
$
(74,335
)
$
(66,868
)
Condensed Consolidated Balance
Sheets
(Unaudited)
(in thousands)
DECEMBER 31,
2023
DECEMBER 31,
2022
Cash, cash equivalents and marketable
securities
$
109,929
$
168,994
Total assets
115,620
180,435
Total liabilities
9,436
11,118
Total stockholders' equity
106,184
169,317
View source
version on businesswire.com: https://www.businesswire.com/news/home/20240311125049/en/
Investor Contacts Ami Bavishi Head of Investor Relations
and Corporate Communications (475) 47-RALLY (Ext. 282)
abavishi@rallybio.com
Hannah Deresiewicz Stern Investor Relations, Inc. (212) 362-1200
hannah.deresiewicz@sternir.com
Media Contact Victoria Reynolds Mission North (760)
579-2134 rallybio@missionnorth.com
Rallybio (NASDAQ:RLYB)
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