Rallybio Presents Natural History Study for Fetal and Neonatal Alloimmune Thrombocytopenia at the 65th American Society of Hematology Annual Meeting
-- 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 --
“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
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
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
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
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