-- FNAIT Natural History Study to Inform
Frequency of FNAIT Risk in a Broad and Diverse Population of
Pregnant Women --
-- Study Designed to Provide a Contemporary
Control Dataset to Support a Future Registrational Trial in
Pregnant Women --
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 presented details from the Rallybio Fetal
and Neonatal Alloimmune Thrombocytopenia (FNAIT) natural history
study in a poster presentation at the 65th American Society of
Hematology (ASH) Annual Meeting, in San Diego, California. The
FNAIT natural history study is a prospective, non-interventional,
multinational alloimmunization study to determine the frequency of
expectant mothers of diverse races and ethnicities at higher FNAIT
risk.
“There is a significant unmet need to establish routine maternal
blood screening to identify women at higher risk of FNAIT and
provide a well-tolerated, effective prophylactic treatment.
Screening mothers for FNAIT during pregnancy is not routinely
performed and currently most at-risk pregnancies are not identified
prior to birth,” said Emilie Vander Haar, M.D., Weill Cornell
Medicine, New York-Presbyterian Hospital and co-author. “By
analogy, the introduction of antenatal screening in Rh disease
combined with an effective prophylactic therapy has resulted in the
virtual elimination of that disease and is one of the most
significant medical advances ever achieved. Achieving the same in
FNAIT would have lasting implications for expectant moms and their
babies.”
Previous FNAIT studies primarily conducted in Caucasian
populations suggest that approximately 2% of expectant women are
HPA-1a negative and, therefore, at risk for FNAIT. This FNAIT
natural history study is the first prospective study that seeks to
characterize risk for FNAIT in a racially and ethnically diverse
population of pregnant women.
The study is presently being conducted in the United States and
across multiple European countries including Germany, Netherlands,
Norway, Sweden, and the United Kingdom. Expectant mothers are
screened at gestational weeks 10 to 14, enabling early
identification and follow-up of women at higher risk of
alloimmunization. Rallybio expects to screen up to 30,000 expectant
mothers in the natural history study, with an estimated 7,600 women
screened by the end of 2023.
Screening for the natural history study is expected to continue
in parallel with Rallybio’s Phase 2 study of RLYB212, an
anti-HPA-1a monoclonal antibody for the prevention of FNAIT. The
Phase 2 study is expected to initiate in the second half of 2024.
Data from the natural history study is designed to provide a
contemporary control for the planned single-arm Phase 3
registrational study.
The poster can be viewed on the Publications & Presentations
page of the Company’s website www.rallybio.com.
Additional information about the ASH Annual Meeting is available
at: https://www.hematology.org/meetings/annual-meeting.
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, 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 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 FNAIT natural history study,
expectations regarding the use of such results, the estimated
number of expectant mothers screened in the natural history study,
and whether a prophylactic treatment for FNAIT can be successfully
developed. 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 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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20231205166108/en/
Investor Contacts 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 Contact Jorge Gaeta
Mission North (516) 430-7659 Rallybio@missionnorth.com
Rallybio (NASDAQ:RLYB)
Gráfica de Acción Histórica
De Abr 2024 a May 2024
Rallybio (NASDAQ:RLYB)
Gráfica de Acción Histórica
De May 2023 a May 2024