-- RLYB212 Demonstrated Dose-Dependent,
Rapid and Complete Elimination of Transfused HPA-1a Positive
Platelets in HPA-1a Negative Subjects --
-- Mean Reduction in Platelet Elimination
Half-Life After Subcutaneous Administration of RLYB212 was ≥90% in
both RLYB212 Dose Groups, Achieving Proof-of-Concept Criteria
--
-- Phase 2 Study in Pregnant Women at Higher
Risk of HPA-1a Alloimmunization to be Initiated in 2H 2024 --
-- Phase 1 Multiple Dose Data and
Maternal-Fetal Toxicology Data On-track for 4Q 2023 --
-- Company to Host Investor and Analyst Meeting
Webcast with Corresponding Slides at 4:00pm ET --
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 data from the Phase 1b
proof-of-concept study of RLYB212, a novel monoclonal anti-HPA-1a
antibody in development for the prevention of fetal and neonatal
alloimmune thrombocytopenia (FNAIT). The data, delivered in an oral
presentation by Christof Geisen, M.D., at the 31st Congress of the
International Society on Thrombosis and Haemostasis (ISTH), showed
that subcutaneous (SC) RLYB212 administration produced a
dose-dependent, rapid and complete elimination of transfused HPA-1a
positive platelets in HPA-1a negative subjects, with both doses
meeting the prespecified proof-of-concept criteria of ≥90%
reduction in mean platelet elimination half-life. Mean platelet
elimination half-life was 5.8 hours (0.09mg) and 1.5 hours (0.29mg)
for RLYB212 compared to 71.7 hours for placebo.
Christof Geisen, M.D., Institute of Transfusion Medicine and
Immunohaematology, German Red Cross Blood Transfusion Service, and
the lead author for the RLYB212 abstract stated, “These
proof-of-concept results provide further understanding as to how
FNAIT may have the potential to be prevented through the rapid and
complete elimination of fetal platelet antigens prior to maternal
alloimmunization. These data are a critical step forward to address
a significant unmet need with no currently approved treatments or
therapies to prevent maternal alloimmunization as well as the
occurrence of FNAIT in babies.”
Phase 1b Proof-of-Concept (POC) Study of RLYB212 in
FNAIT
The Phase 1b single-blind, placebo-controlled proof-of-concept
study was designed to establish the ability of SC RLYB212 to
rapidly eliminate HPA-1a positive platelets transfused to HPA-1a
negative healthy subjects. The study included 11 males aged 18 to
65 years, randomized to RLYB212 0.09mg (n=4), RLYB212 0.29mg (n=5),
or placebo (n=2).
Summary of Proof-of-Concept Results
- RLYB212 demonstrated a dose-dependent, rapid and complete
elimination of transfused HPA-1a positive platelets in HPA-1a
negative subjects.
- Mean platelet elimination half-life was 5.8 hours (0.09mg) and
1.5 hours (0.29mg) for RLYB212 compared to 71.7 hours for placebo,
meeting the study’s prespecified criteria for proof-of-concept in
both dose groups (≥ 90% reduction in mean platelet elimination
half-life).
- The study’s broad range of pharmacodynamic and pharmacokinetic
(PK) data will allow substantive modeling of the RLYB212
concentration-effect relationship and inform the target dose
regimen for the planned future studies.
- Platelet elimination profiles after SC administration of
RLYB212 were consistent with those of Rhesus factor D
(RhD)-positive erythrocytes after intramuscular administration of
anti-RhD agents, which are well-established to safely and
effectively prevent RhD alloimmunization during pregnancy.
- Consistent with previously reported data, RLYB212 was observed
to be well-tolerated with no reports of serious or severe adverse
events.
“The data reported today at ISTH continue to support our belief
in the potential use of subcutaneous RLYB212 as a prophylactic
therapeutic for the prevention of HPA-1a alloimmunization and
FNAIT. We are pleased to see rapid and complete platelet
elimination in our target concentration range, with both dose
groups meeting the proof-of-concept criteria of at least 90%
reduction in mean platelet elimination half-life,” commented Róisín
Armstrong, Ph.D., Rallybio’s RLYB212 Program Lead. “With no
currently approved therapies for the prevention of FNAIT, there
remains a substantial unmet need for a treatment that can
effectively eliminate fetal platelet antigen and, as a result,
significantly decrease the risk of severe bleeding in the fetus and
neonate, including intracranial hemorrhage and its devastating
consequences.”
Dr. Armstrong continued, “We continue to focus our efforts on
completing 12-week repeat dosing in the Phase 1 safety and PK study
of RLYB212, along with the ongoing toxicology program, both of
which are on track for the fourth quarter of 2023. In the second
half of 2024 we plan to commence a Phase 2 study in pregnant women
at higher risk of HPA-1a alloimmunization, designed to confirm the
RLYB212 dose regimen for our Phase 3 registrational study. We are
also planning for discussions with regulators later in 2023 or in
the first half of 2024 in advance of the Phase 2 study.”
Clinical Development Update for RLYB212
Rallybio will host an investor and analyst meeting beginning at
4:00 p.m. ET to discuss clinical development plans for RLYB212.
RLYB212 Catalysts for 2023
- Rallybio remains on track to complete the following RLYB212
milestones in the fourth quarter of 2023:
- Comprehensive toxicology program, including maternal-fetal
toxicology
- Multiple dose cohort of Phase 1 safety and PK study
Phase 2 Dose Confirmation Study
- The Company plans to initiate a Phase 2 dose confirmation study
in the second half of 2024, designed to confirm the RLYB212 dose
regimen in pregnant women at higher risk of FNAIT prior to
initiation of a larger Phase 3 registrational study.
- This study will employ a sentinel, sequenced cohort design to
allow for any required adjustments to the dose regimen, prior to
advancing the confirmed dose regimen into the registrational
study.
Natural History Study
- The Natural History Study is designed to provide a contemporary
dataset for HPA-1a alloimmunization frequency in a racially and
ethnically diverse population that can serve as a control for the
planned single-arm registrational study, along with establishing
the operational framework for seamless initiation of the Phase 2
study and future Phase 3 registrational study.
- The Natural History Study has screened approximately 4,500
women to date and an estimated 7,600 women are planned to be
screened by end of 2023.
- 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 end of Phase 2 regulatory discussions with
the U.S. Food and Drug Administration and the European Medicines
Agency to support design and initiation of the Phase 3
registrational study.
Phase 3 Registrational Study
- Following completion of the Phase 2 dose confirmation study,
Rallybio expects to commence a Phase 3 registrational study, after
consultation with regulatory authorities.
Investor and Analyst Meeting Webcast
Rallybio will host an investor and analyst meeting on Saturday,
June 24, 2023 from 4:00 to 6:00 p.m. Eastern Time. The webcast and
corresponding slides can be accessed through the Events and
Presentations section of Rallybio’s website at
http://www.rallybio.com.
About FNAIT
Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT) is a
potentially life-threatening rare disease that can cause
uncontrolled bleeding in fetuses and newborns. FNAIT can arise
during pregnancy due to an immune incompatibility between an
expectant mother and her fetus in a specific platelet antigen
called human platelet antigen 1, or HPA-1.
There are two predominant forms of HPA-1, known as HPA-1a and
HPA-1b, which are expressed on the surface of platelets.
Individuals who are homozygous for HPA-1b, meaning that they have
two copies of the HPA-1b allele and no copies of the HPA-1a allele,
are also known as HPA-1a negative. Upon exposure to the HPA-1a
antigen, these individuals can develop antibodies to that antigen
in a process known as alloimmunization. In expectant mothers,
alloimmunization can occur upon mixing of fetal blood with maternal
blood. When alloimmunization occurs in an expectant mother, the
anti-HPA-1a antibodies that develop in the mother can cross the
placenta and destroy platelets in the fetus. The destruction of
platelets in the fetus can result in severely low platelet counts,
or thrombocytopenia, and potentially lead to devastating
consequences including miscarriage, stillbirth, death of the
newborn, or severe lifelong neurological disability in those babies
who survive. There is currently no approved therapy for the
prevention or prenatal treatment of FNAIT.
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, a C5 complement inhibitor 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 with an
additional facility at the University of Connecticut’s Technology
Incubation Program in Farmington, 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 results from the RLYB212 Phase 1b proof-of-concept
study, statements concerning the substance, design and timing of
our planned or ongoing studies for RLYB212, including the planned
Phase 2 study and Phase 3 registrational study, the timing of the
availability of data from such studies, our expectations regarding
reporting of data from such studies, our expectations regarding the
usefulness of such data, the success of modeling to inform dosing
for a future registrational study, our ability to advance RLYB212
into future clinical studies, the outcomes of discussions with
healthcare authorities, including the FDA, and the likelihood that
Rallybio will be successful in developing RLYB212 as an approach to
prevent FNAIT. 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, including the FNAIT natural history study, the Phase 1b
clinical study for RLYB212, and our planned Phase 2 and Phase 3
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 March 31, 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/20230622247357/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 Tara DiMilia 908-369-7168 Tara.dimilia@tmstrat.com
Rallybio (NASDAQ:RLYB)
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