- Data Support HPA-1a Negative Frequency
of More than 2% in Nearly 200,000 Screened Pregnant Women -
- Among HPA-1a Negative Pregnant Women,
Approximately 33% are at Higher Risk for Alloimmunization -
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 announced the presentation of results from
a fetal and neonatal alloimmune thrombocytopenia (FNAIT) systematic
literature review and meta-analysis at the Academy of Managed Care
Pharmacy (AMCP) 2024 Annual Meeting, which is taking place in New
Orleans, LA. The results of this research found that, in a pooled
analysis of 198,062 pregnant women, 2.2% were HPA-1a negative and
32.3% of these women were also HLA-DRB3*01:01 positive and
therefore at ~25-fold higher risk for alloimmunization. These rates
are consistent with Rallybio’s current estimate of annual at-risk
pregnancies and translate to tens of thousands of fetuses and
newborns at risk each year for the potentially devastating
consequences of FNAIT.
“We are pleased to establish a robust foundation of knowledge
documenting the frequency of FNAIT risk as reported from a pooled
analysis of peer-review literature, which is consistent with our
current estimates,” said Stephen Uden, M.D., Chief Executive
Officer of Rallybio. “This information, in combination with data
from our ongoing FNAIT natural history study, will enable us to
create a shared understanding of the number of pregnant women and
babies at higher risk of FNAIT annually, underscoring the
importance of having an effective preventative therapeutic
option.”
Rallybio is developing RLYB212, a novel human monoclonal
anti-HPA-1a antibody designed to prevent alloimmunization in
pregnant women, thereby eliminating the risk of FNAIT and its
potentially devastating consequences in their fetuses and newborns.
Rallybio is on track to initiate a Phase 2 dose confirmation study
for RLYB212 in pregnant women in the second half of 2024. The
company is also conducting an ongoing FNAIT natural history study
that will provide a contemporary dataset for HPA-1a
alloimmunization frequency in a racially and ethnically diverse
population, which is intended to support a future Phase 3
registration study for RLYB212. RLYB212 is the only investigational
therapy currently reported to be in clinical development to address
the needs of pregnant women at higher risk of FNAIT who have not
alloimmunized.
The poster, titled “Fetal and Neonatal Alloimmune
Thrombocytopenia: A Systematic Literature Review and Meta-analysis
of Adverse Pregnancy-Related Outcomes to Support the Development of
a Novel Prophylactic Therapeutic,” was presented by Andrea V.
Margulis of RTI Health Solutions. Specifically, the literature
review found that, of 198,062 screened pregnant women, 2.2% (95%
confidence interval [CI], 2.0%-2.5%) were HPA-1a negative; 32.3%
(28.6%-36.1%) of HPA-1a–negative women were HLA-DRB3*01:01 positive
and therefore at even higher risk for alloimmunization.
Approximately 10% of HPA-1a–negative women were already
alloimmunized to HPA-1a. The meta-analysis is based on 12
observational cohort studies from Europe, Canada, and Egypt
published from 1985 through 2018. A link to the poster is available
here.
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 rates of women who are HPA-1a negative and
HLA-DRB3*01:01 positive, the level of increased higher risk for
alloimmunization among women who are HPA-1a negative and
HLA-DRB3*01:01 positive, the actual rates of annual at-risk
pregnancies for FNAIT and the number of potential pregnancies that
could be at risk, our belief that the literature review establishes
the relevant foundation of knowledge regarding such rates, our
ability to create a shared understanding of the number of pregnant
women and babies at higher risk of FNAIT annually, the timing of
initiation of the Phase 2 dose confirmation study for RLYB212,
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, and the likelihood that Rallybio will be successful in
developing 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
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
Annual Report on Form 10-K for the period ended December 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/20240417889090/en/
Investors Samantha Tracy Rallybio Corporation (475)
47-RALLY (Ext. 282) investors@rallybio.com Hannah Deresiewicz Stern
Investor Relations, Inc. (212) 362-1200
hannah.deresiewicz@sternir.com Media Victoria Reynolds
Mission North (760) 579-2134 rallybio@missionnorth.com
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