-- Phase 2 Study for RLYB212 Expected to
Initiate in 2H 2024 --
-- Company to Provide Update on Phase 2
Discussions with European Medicines Agency for RLYB212 in 1H 2024
--
-- Cash Runway Extended into 3Q 2025 --
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 provided an update on recent
accomplishments and announced expected 2024 milestones. The Company
will present these updates at the 42nd Annual J.P. Morgan Health
Care Conference in San Francisco, California on Wednesday, January
10, 2024, at 8:15 a.m. PT (11:15 a.m. ET).
“Over the past twelve months we made significant progress
towards building a broad and sustainable pipeline of transformative
therapeutics for rare diseases with severe unmet needs,” said
Stephen Uden, M.D., Chief Executive Officer of Rallybio. “In 2023,
we announced encouraging Phase 1 results for our lead program,
RLYB212, for the prevention of fetal and neonatal alloimmune
thrombocytopenia, as well as for RLYB116, our C5 inhibitor. In
parallel, we completed critical work on our earlier stage pipeline
programs while carefully managing our expenses.”
Dr. Uden continued, “We are committed to building on this
momentum in 2024. We remain on track to initiate a Phase 2 study
for RLYB212 in the second half of 2024 to confirm the dose regimen
for RLYB212 in pregnant women at higher risk for FNAIT.
Furthermore, we believe RLYB116 is a promising drug candidate that
has the potential to address an unmet need for patients by
delivering a more convenient once-a-week self-administered therapy,
and our earlier stage programs are expected to reach important
milestones in 2024. We are also pleased that through careful
management of our cash, our current cash runway guidance has been
extended into the third quarter of 2025. We expect to have
additional updates to our plans and cash runway before the end of
the first quarter.”
Recent Portfolio Milestones and Expected Upcoming
Milestones
Maternal Fetal Blood Disorders
Rallybio expects to provide an update on Phase 2 discussions
with the European Medicines Agency (EMA) for RLYB212, a novel human
monoclonal anti-HPA-1a antibody in development for the prevention
of fetal and neonatal alloimmune thrombocytopenia (FNAIT), in the
first half of 2024.
Rallybio expects to initiate its Phase 2 dose confirmation study
for RLYB212 in the second half of 2024. The study is designed to
confirm the dose regimen for RLYB212 in pregnant women at higher
risk for FNAIT, prior to initiation of a Phase 3 registrational
study.
In addition, Rallybio continues to advance its FNAIT natural
history alloimmunization study. The FNAIT natural history study is
a non-interventional study designed to provide a contemporary
dataset for HPA-1a alloimmunization frequency in a racially and
ethnically diverse population that can serve as a control arm for
the planned single-arm Phase 3 registrational study. In addition,
the natural history study is intended to establish the operational
framework for the Phase 2 study and future Phase 3 registrational
study. Screening for the natural history study is expected to
continue simultaneously with execution of the Phase 2 study. Data
from both studies will be used for the end of Phase 2 regulatory
discussions with the U.S. Food and Drug Administration and the EMA
to support design and initiation of the Phase 3 registrational
study.
The natural history study has screened more than 7,600 women to
date and the Company expects to provide an update on screening for
2024 in the first quarter.
Complement Dysregulation
In December 2023, Rallybio announced preliminary Phase 1
multiple ascending dose data for RLYB116. Preliminary results
showed 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 data support advancement of RLYB116 for patients with
certain complement-mediated diseases, including generalized
myasthenia gravis (gMG), the Company is prioritizing near-term
investments in RLYB116 manufacturing and expects that additional
manufacturing work will improve tolerability at higher doses with a
low injection volume and infrequent subcutaneous administration.
The Company believes such enhancements will enable higher exposure
to RLYB116 and potentially increase C5 reduction, which can result
in treating a broader range of complement-mediated diseases,
including paroxysmal nocturnal hemoglobinuria and antiphospholipid
syndrome. Rallybio expects to complete this manufacturing work and
provide an update on the development plan for RLYB116 in the second
half of 2024.
Rallybio, together with its partner EyePoint Pharmaceuticals,
Inc., continue to evaluate sustained delivery of Rallybio’s
inhibitor of C5 using EyePoint’s proprietary Durasert® technology
for sustained intraocular drug delivery, with the initial focus on
geographic atrophy, an advanced form of age-related macular
degeneration that leads to irreversible vision loss. Rallybio and
EyePoint expect to provide an update on this collaboration in the
first half of 2024.
Hematological Disorders
Rallybio continues to advance preclinical activities for
RLYB331, a preclinical, therapeutic monoclonal antibody that
inhibits Matriptase-2 (MTP-2), to support the transition of this
asset into clinical development. Rallybio expects to report
additional animal data from this program in the first half of
2024.
Metabolic Disorders
Rallybio, together with its partner Exscientia, continues to
work toward the selection of a small molecule development candidate
to advance into the clinic targeting ENPP1 for the treatment of
patients with hypophosphatasia (HPP). Significant progress has been
made and proof of mechanism studies are in progress with a leading
global HPP expert. Rallybio and Exscientia plan to provide an
update on the progress of the program in the second half of
2024.
Rallybio, together with its partner AbCellera, are focused on
the discovery, development, and commercialization of novel
antibody-based therapeutics for rare diseases. The partnership’s
first discovery program is focused on identifying a novel treatment
for patients with rare metabolic diseases.
Financial Update
Cash, cash equivalents, and marketable securities were $121.4
million as of September 30, 2023. The Company currently expects its
cash runway to extend into the third quarter of 2025.
Webcast of Presentation at the 42nd Annual J.P. Morgan Health
Care Conference
Rallybio is scheduled to present at the 42nd Annual J.P. Morgan
Health Care Conference on Wednesday, January 10, 2024, at 8:15 a.m.
PT (11:15 a.m. ET). A live webcast of the presentation and
subsequent question and answer session will be accessible through
the Events and Presentations section of Rallybio’s website. An
archived replay of the webcast will be available for 30 days
following the presentation.
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 progress and number of women
screened 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 reporting results from our collaboration
with EyePoint, the timing of reporting results of the animal
studies with RLYB331, the timing of providing an update of our
joint venture with Exscientia, the likelihood that Rallybio will be
successful in developing RLYB212, RLYB116, or any of our other
product candidates, 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 studies, and complete such clinical studies 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 enter into
strategic partnerships or other arrangements, 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.
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version on businesswire.com: https://www.businesswire.com/news/home/20240102849893/en/
Investors Ami Bavishi Head of Investor Relations and
Communications 475-47-RALLY (Ext. 282) abavishi@rallybio.com
Hannah Deresiewicz Stern Investor Relations, Inc. 212-362-1200
hannah.deresiewicz@sternir.com
Media Jorge Gaeta Mission North (516) 430-7659
Rallybio@missionnorth.com
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