Fetomaternal Transfusion

-- Data Support Potential Use of an Anti-HPA-1a Antibody as Prophylaxis for FNAIT -- -- In March 2023, Rallybio Announced Clinical Proof-of-Concept Achieved for its Lead Product Candidate, RLYB212, a Monoclonal Anti-HPA-1a Antibody, in the First Quarter of 2023 -- NEW HAVEN, Conn.--(BUSINESS WIRE)-- Rallybio Corporation (Nasdaq: RLYB) today announced publication of Phase 1 / 2 proof-of-concept results for RLYB211, a polyclonal anti-HPA-1a antibody derived from human plasma for the prevention of FNAIT. FNAIT is a rare and life-threatening bleeding disorder in which maternal alloantibodies directed against fetal platelets can lead to devastating outcomes for the fetus/neonate. There are currently no approved therapies for the prevention or treatment of FNAIT. The findings were published in the Journal of Thrombosis and Haemostasis, the official journal of the International Society on Thrombosis and Haemostasis. Rallybio’s randomized, single-blind, placebo-controlled, study investigated the ability of a single dose of intravenous RLYB211 to eliminate HPA-1a-positive platelets transfused into HPA-1a-negative healthy subjects. The platelet transfusion in this study was selected based on the precedent of historical clinical trial designs to assess the efficacy of anti-D prophylaxis, in which the transfusion of antigen positive cells is designed to mimic a catastrophic fetal maternal hemorrhage. As has been shown with anti-D prophylactic agents, the ability of RLYB211 to drive the rapid and complete elimination of antigen positive cells is a surrogate indicator of its potential to prevent maternal alloimmunization, and therefore FNAIT. In Cohort 1, subjects received RLYB211 or placebo one hour after transfusion of HPA-1a positive platelets and in Cohort 1B subjects received RLYB211 or placebo followed by platelet transfusion one week later. Proof-of-concept was defined as ≥90% reduction in platelet elimination half-life compared to placebo. The publication stated: RLYB211 accelerated the elimination of HPA-1a positive platelets versus placebo, with all subjects meeting proof-of-concept criteria for greater than 90% reduction in platelet elimination half-life compared to placebo. Rapid elimination of transfused platelets was evident 7 days after RLYB211 administration, simulating prophylactic administration of an anti-HPA-1a antibody prior to a fetal-maternal bleed. RLYB211 was well tolerated and no serious adverse events were reported. “We are pleased that these clinical results were published in the Journal of Thrombosis and Haemostasis, indicating that RLYB211 markedly accelerated the elimination of HPA-1a-positive platelets. We believe that these data demonstrate the prophylactic potential of anti-HPA-1a antibodies to prevent HPA-1a alloimmunization and occurrence of FNAIT,” said Róisín Armstrong, Ph.D., Rallybio’s FNAIT Program Lead. “Our FNAIT program continues to advance and as previously announced, we are pleased that an abstract reporting results from the Phase 1b proof-of-concept study for RLYB212, our monoclonal candidate, has been accepted for presentation at the upcoming 31st Congress of International Society on Thrombosis and Haemostasis in June.” Dr. Christof Geisen, Institute of Transfusion Medicine and Immunohaematology, German Red Cross Blood Transfusion Service, and a key collaborator in the RLYB211 study, stated, “In addition to the rapid elimination of transfused platelets in all subjects, the effect of RLYB211 was also demonstrated when it was administered one week prior to platelet transfusion, suggesting a durable potential treatment effect for HPA-1a-specific antibodies and that FNAIT might safely be prevented by rapidly eliminating platelet antigen from maternal circulation before alloimmunization can occur.” The RLYB211 Phase 1 / 2 results were published in the April issue (Volume 21, Issue 4, Pages 838-849) of the Journal of Thrombosis and Haemostasis, . Given the advantages of small volume subcutaneous dosing of RLYB212 as compared to RLYB211 and the expected manufacturing and supply efficiencies for RLYB212, Rallybio announced in March 2023 that the Company will not continue development of RLYB211. 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 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 and follow us on LinkedIn. Forward-Looking Statements This press release contains forward-looking statements that are based on our management’s beliefs and assumptions and on information currently available to management. 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 ability of RLYB212 and RLYB211, and the prophylactic administration of HPA-1a-specific antibodies, to clear platelets and prevent alloimmunization in pregnant women, and the anticipated threshold effect for rapid and complete elimination of HPA-1a positive platelets, the expected progress, results, and plans for RLYB212 and RLYB211, the initiation, substance, design and timing of our planned or ongoing studies for RLYB212, the timing of the availability of data from such studies, our expectations regarding reporting of data from such studies, and our expectations regarding the usefulness of data from such studies. 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 ongoing and future clinical trials for RLYB212 for the prevention of FNAIT, 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, 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, 2022, 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. Moreover, we operate in an evolving environment. New risks and uncertainties may emerge from time to time, and it is not possible for management to predict all risks and uncertainties. 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.

-- RLYB212 Showed Rapid Elimination of Transfused, HPA-1a Positive Platelets in HPA-1a Negative Subjects -- -- Clinical Findings and Safety Pro with Previously Reported Data; Continue to Support the Potential for RLYB212 as a Prophylactic Treatment for FNAIT -- -- Company Expects to Present Results at a Scientific Conference in 2023 -- NEW HAVEN, Conn.--(BUSINESS WIRE)-- Rallybio Corporation (Nasdaq: RLYB) today announced that clinical proof-of-concept has been achieved in a Phase 1b study for RLYB212, an anti-HPA-1a monoclonal antibody for the prevention of fetal and neonatal alloimmune thrombocytopenia (FNAIT). Results show that one week after a single subcutaneous dose, RLYB212 was able to rapidly and completely eliminate transfused, HPA-1a positive platelets in HPA-1a negative subjects. Additional findings from the study show: The reduction in mean platelet elimination half-life was greater than 90% in both RLYB212 dose groups compared to placebo and was dose related. The broad range of pharmacokinetic and pharmacodynamic data allows substantive modeling to inform dose selection for a future registrational study. RLYB212 was observed to be well-tolerated with no serious adverse events reported. The Company expects to report data from the Phase 1b clinical study of RLYB212 at a scientific conference in 2023. “Our FNAIT development program has consistently demonstrated the effectiveness of anti-HPA-1a antibodies to rapidly eliminate HPA-1a positive platelets from the circulation of HPA-1a negative subjects,” commented Róisín Armstrong, Ph.D., Rallybio’s RLYB212 Program Lead. “We’ve also established in published nonclinical studies the association between rapid platelet elimination and prevention of HPA-1a alloimmunization, which can lead to negative and potentially life-threatening outcomes in FNAIT. Collectively, these data reinforce our belief on the potential for an anti-HPA-1a antibody to be a viable approach for preventing FNAIT and we look forward to continued advancement of the RLYB212 development program.” Rallybio also announced today that testing in the multi-dose cohort of its single-center Phase 1 trial in Europe began in the first quarter of 2023. This portion of the Phase 1 study will evaluate safety and pharmacokinetics of RLYB212 based on repeat dosing over 12 weeks in healthy male and female participants. The Company expects results from this cohort of subjects in the fourth quarter of 2023. Martin Mackay, Ph.D., Chief Executive Officer of Rallybio, stated, “We are very pleased with the progress of our RLYB212 program. Throughout the program, we have carefully laid the groundwork to advance a product candidate that we believe can have a significant impact on the lives of expectant mothers and neonates. RLYB212 exemplifies Rallybio’s enduring commitment to transforming the treatment of rare diseases with little to no therapeutic options.” Given the favorable development pro RLYB212 to date, the data generated to date for RLYB212, and the expected manufacturing and supply efficiencies for RLYB212, the Company also announced today that RLYB211, a plasma-derived polyclonal anti-HPA-1a antibody, will not be advanced further in clinical development. About the RLYB212 Phase 1b Study Rallybio’s Phase 1b study is a single-blind, placebo-controlled proof-of-concept study designed to establish the ability of subcutaneous RLYB212 to rapidly accelerate the elimination of HPA-1a positive platelets transfused to HPA-1a negative healthy male participants. In this single-center, EU-based study, the elimination of transfused platelets serves as a surrogate for assessing the ability of an anti-HPA-1a antibody to drive rapid elimination of HPA-1a positive fetal platelets from an expectant mother’s circulation, thereby potentially preventing HPA-1a maternal alloimmunization and the occurrence of FNAIT in fetuses and newborns. The platelet challenge in this model represents an equivalent fetal maternal hemorrhage of 30 mL, a rare and catastrophic scenario during pregnancy. In August 2022, the Company amended the Phase 1b protocol to include a higher dose of RLYB212 and further broaden the range of pharmacokinetic and pharmacodynamic data for RLYB212, enabling substantive modeling of the concentration-effect relationship that can inform dosing for a future registrational study. The Phase 1b study has been conducted at the Clinical Research department of the Fraunhofer Institute for Translational Medicine and Pharmacology ITMP, in Frankfurt/Main, Germany, in collaboration with the Institute of Transfusion Medicine and Immunohaematology, German Red Cross (Deutsches Rotes Kreuz) Blood Transfusion Service Baden-Württemberg-Hessen gGmbH in Frankfurt/Main, Germany. 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 is a clinical-stage biotechnology company committed to identifying and accelerating the development of life-transforming therapies for patients with severe and rare diseases. Since its launch in January 2018, Rallybio has built a portfolio of promising product candidates, which are now in development to address rare diseases in the areas of hematology, immuno-inflammation, maternal fetal health, and metabolic disorders. The Company’s mission is being advanced by a team of highly experienced biopharma industry leaders with extensive research, development, and rare disease expertise. 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 . 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. 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 substance, design and timing of our planned or ongoing studies for RLYB212, the timing of the availability of data from such studies, our expectations regarding reporting of data from such studies, our expectations regarding the usefulness such data, the success of modeling to inform dosing for a future registrational study, our ability to advance RLYB212 into future clinical studies, 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 trials, including the FNAIT natural history study, and the Phase 1 and 1b clinical trials for RLYB212 and the Phase 1 study for RLYB116, 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 identify new product candidates and successfully acquire such product candidates from third parties, our ability to integrate RLYB331 into our pipeline, 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-Q for the period ended September 30, 2022, 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.

While Gamida Cell is still charging its way toward approval for its therapies with new CEO Abigail Jenkins in place, it’s looking to garner a little more cash. The company announced on Wednesday that it will be offering up over 12 million shares and expecting proceeds of an estimated $20 million. The offer is expected to close on Sept. 30. According to Gamida Cell, it plans to use the cash to help with the potential launch of its omidubicel therapy if it gets approval. Omidubicel has a PDUFA date set for Jan. 30, 2023. If the therapy does get the thumbs-up, the company plans to also use the cash to continue the development of its other candidates and for other corporate purposes. The rare disease biotech RallyBio is getting ready to reveal its initial Phase Ib results for one of its candidates. The company said Wednesday that it has presented preliminary results in its Phase IB proof-of-concept study for its monoclonal antibody, dubbed RLYB212. The candidate aims to prevent fetal and neonatal alloimmune thrombocytopenia (FNAIT). The data reportedly showed that one week after a single dose, levels of transfused HPA-1a positive platelets went down rapidly when put up against a placebo, in a model of a “catastrophic fetal maternal hemorrhage.” Early data also show the potential for dosing less frequently. “These preliminary results continue to support our projected effective target therapeutic concentrations for the prevention of maternal HPA-1a alloimmunization by RLYB212. We are pleased to see the rapid and complete elimination of transfused platelets in all subjects to date, with a greater than 90% reduction of the mean platelet elimination half-life compared to placebo, consistent with our proof-of-concept criteria,” RallyBio CEO Martin Mackay said in a statement . Mackay also stated that the full data will be presented sometime in the first quarter of 2023. Maryland-based biotech MaxCyte is working with a major company to advance its CRISPR program. MaxCyte has signed a licensing deal with Vertex Pharmaceuticals. The deal will have Vertex gain rights to use MaxCyte’s CRISPR/Cas-9-based gene editing platform both commercially and in the clinic, though those rights aren’t exclusive. In the deal, MaxCyte will get licensing fees and other revenue related to the program. However, the financial details of the deal were not revealed. The platform itself, according to MaxCyte, is a gene-edited cell therapy that is meant to treat patients with sickle cell disease.