– Results Indicate that FNAIT Risk is More
Prevalent Than Previously Estimated –
Rallybio Corporation (Nasdaq: RLYB), a clinical-stage
biotechnology company translating scientific advances into
transformative therapies for patients with devastating rare
diseases, announced today that full data from an epidemiological
analysis quantifying the proportion of women across diverse
populations at higher risk of a fetal and neonatal alloimmune
thrombocytopenia (FNAIT)-impacted pregnancy will be presented at
the American Society of Human Genetics (ASHG) 2024 Annual Meeting
to be held November 5 – 9, 2024 in Denver, CO.
“With our Phase 2 trial of RLYB212 in pregnant women at higher
risk for HPA-1a alloimmunization and FNAIT on track to initiate in
the fourth quarter of 2024, we continue our efforts to raise
awareness of FNAIT and broaden the understanding of the population
at risk for this potentially devastating disease,” said Stephen
Uden, MD, Chief Executive Officer of Rallybio. Dr. Uden continued,
“Data from this analysis provide the first clear evidence of the
extent to which ancestries beyond the Caucasian population can
carry a higher risk for FNAIT, underscoring the importance of
screening all pregnant women for FNAIT risk as part of standard
prenatal care. This would ensure that all women at higher risk for
FNAIT could then be offered prophylactic treatment with RLYB212, if
approved.”
The ASHG abstract #7041T, which can be found here,
details the high-level findings of the epidemiological analysis. In
this analysis, women at higher risk for alloimmunization and FNAIT
were defined as those having an HPA-1a negative and HLA-DRB3*01:01
positive genotype. Allele frequencies were obtained from gnomAD v4
for HPA-1a and the U.S. National Marrow Donor Registry (NMDR) for
HLA-DRB3*01:01.
Proportions of women at risk of alloimmunizing were highest in
Caucasian populations, with the highest proportions in the
Ashkenazi Jewish population (2.36% and 0.65% of women at risk and
at higher risk, respectively), followed by non-Finnish Europeans
(2.34% and 0.64%), Middle Eastern (2.25% and 0.62%), Amish (2.25%
and 0.62%), White Hispanic (2.25% and 0.59%) and Finnish (2.03% and
0.56%). Additionally, women in some non-Caucasian population groups
were also found to be at risk including the Caribbean Hispanic
population (1.48% and 0.33%), followed by African / African
American (1.13% and 0.28%). Women of South Asian, East Asian and
Amerindigenous ancestries were found to have lower risk (<1.0%
and <0.1%).
Taken together, these data indicate that the proportion of
pregnant women at higher risk for FNAIT annually has been
significantly underestimated. For example, in key geographies of
North America and major European countries, it is estimated that
more than 30,000 pregnancies each year are at higher risk for
FNAIT, representing a 40% increase from prior estimates and
translating into a commercial opportunity of $1.6 billion.
This analysis was conducted by Rallybio in partnership with
HealthLumen, a leader in epidemiological modeling of rare genetic
diseases. The poster will be available in the Publications &
Presentations section of Rallybio’s website following the
conclusion of the conference.
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 HPA-1a-negative
expectant mothers bearing a HPA-1a-positive fetus, 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. 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 diversity of ancestries that carry the
genetic markers for FNAIT, the increase in the number of
pregnancies estimated to be at high risk of FNAIT each year based
on the epidemiological analysis and our estimates of the number of
pregnancies at higher risk of FNAIT, our ability to identify the
number of pregnant women at higher risk of FNAIT based on the
results of the analysis, our ability to ensure routine prenatal
screening, the timing of initiation of the Phase 2 dose
confirmation study for RLYB212, our expectations regarding the
usefulness of data from our clinical studies, and the timing of
publications relating to FNAIT and RLYB212. 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 trials 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 June
30, 2024, 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/20240920956554/en/
Investor Contacts Samantha Tracy Rallybio Corporation
(475) 47-RALLY (Ext. 282) investors@rallybio.com
Kevin Lui Precision AQ (212) 698-8691
kevin.lui@precisionaq.com
Media Contact Victoria Reynolds Mission North (760)
579-2134 rallybio@missionnorth.com
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