Rallybio Announces Publication of RLYB212 Phase 1 Proof-of-Concept Study Results in Thrombosis and Haemostasis
Rallybio (Nasdaq: RLYB) has published Phase 1 proof-of-concept study results for RLYB212, a novel monoclonal anti-HPA-1a antibody, in Thrombosis and Haemostasis. The study demonstrated that RLYB212 rapidly clears HPA-1a positive platelets, a important step in preventing alloimmunization and fetal and neonatal alloimmune thrombocytopenia (FNAIT). Both tested doses (0.09 mg and 0.29 mg) met the prespecified criteria of ≥90% reduction in mean platelet elimination half-life compared to placebo. The drug was generally well-tolerated with no serious adverse events reported. Rallybio is on track to initiate a Phase 2 dose confirmation trial in pregnant women at higher risk for FNAIT in Q4 2024.
Rallybio (Nasdaq: RLYB) ha pubblicato i risultati dello studio di fase 1 della prova di concetto per RLYB212, un nuovo anticorpo monoclonale anti-HPA-1a, su Thrombosis and Haemostasis. Lo studio ha dimostrato che RLYB212 elimina rapidamente le piastrine positive per HPA-1a, un passo importante per prevenire l’alloimmunizzazione e la trombocitopenia alloimmune fetale e neonatale (FNAIT). Entrambi i dosaggi testati (0,09 mg e 0,29 mg) hanno soddisfatto i criteri prestabiliti di riduzione ≥90% nella vita media di eliminazione delle piastrine rispetto al placebo. Il farmaco è stato generalmente ben tollerato e non sono stati segnalati eventi avversi gravi. Rallybio è pronta a iniziare un trial di conferma del dosaggio di fase 2 in donne in gravidanza ad alto rischio per FNAIT nel quarto trimestre del 2024.
Rallybio (Nasdaq: RLYB) ha publicado los resultados del estudio de fase 1 de prueba de concepto para RLYB212, un nuevo anticuerpo monoclonal anti-HPA-1a, en Thrombosis and Haemostasis. El estudio demostró que RLYB212 elimina rápidamente las plaquetas positivas para HPA-1a, un paso importante para prevenir la alo-inmunización y la trombocitopenia alo-inmune fetal y neonatal (FNAIT). Las dos dosis probadas (0,09 mg y 0,29 mg) cumplieron con los criterios preespecificados de una reducción del ≥90% en la vida media de eliminación de plaquetas en comparación con el placebo. El fármaco fue generalmente bien tolerado y no se informaron eventos adversos graves. Rallybio está en camino de iniciar un ensayo de confirmación de dosis de fase 2 en mujeres embarazadas con alto riesgo de FNAIT en el cuarto trimestre de 2024.
Rallybio (Nasdaq: RLYB)는 Thrombosis and Haemostasis에 RLYB212의 1상 개념 증명 연구 결과를 발표했습니다. 이 연구는 RLYB212가 HPA-1a 양성 혈소판을 빠르게 제거한다는 것을 보여주었으며, 이는 알로면역화 및 태아 및 신생아 알로면역 혈소판감소증(FNAIT)을 예방하는 중요한 단계입니다. 시험된 두 가지 용량(0.09 mg 및 0.29 mg)은 위약과 비교하여 혈소판 제거 반감기를 평균 90% 이상 감소시키는 사전 규정 기준을 충족했습니다. 이 약물은 일반적으로 잘 견딜 수 있었고, 심각한 부작용이 보고되지 않았습니다. Rallybio는 2024년 4분기까지 FNAIT의 높은 위험으로 임신한 여성들을 위한 2상 용량 확인 시험을 시작할 준비를 하고 있습니다.
Rallybio (Nasdaq: RLYB) a publié les résultats d'une étude de phase 1 sur la preuve de concept pour RLYB212, un nouvel anticorps monoclonal anti-HPA-1a, dans Thrombosis and Haemostasis. L'étude a démontré que RLYB212 élimine rapidement les plaquettes positives pour HPA-1a, étape importante pour prévenir l'allo-immunisation et la thrombocytopénie allo-immune néonatale et fœtale (FNAIT). Les deux doses testées (0,09 mg et 0,29 mg) ont respecté les critères préétablis d'une réduction de ≥90% de la demi-vie d'élimination moyenne des plaquettes par rapport au placebo. Le médicament a généralement été bien toléré et aucun événement indésirable grave n'a été signalé. Rallybio prévoit de lancer un essai de confirmation de dose de phase 2 chez des femmes enceintes à haut risque de FNAIT au quatrième trimestre 2024.
Rallybio (Nasdaq: RLYB) hat die Ergebnisse der Phase-1-Pilotstudie für RLYB212, ein neuartiger monoklonaler Anti-HPA-1a-Antikörper, in Thrombosis and Haemostasis veröffentlicht. Die Studie zeigte, dass RLYB212 schnell HPA-1a-positive Thrombozyten entfernt, was ein wichtiger Schritt zur Verhinderung von Alloimmunisierung und fetaler sowie neonataler alloimmuner Thrombozytopenie (FNAIT) ist. Beide getesteten Dosen (0,09 mg und 0,29 mg) erfüllten die vordefinierten Kriterien einer ≥90%igen Reduktion der mittleren Thrombozyteneliminationshalbwertszeit im Vergleich zu Placebo. Das Medikament wurde im Allgemeinen gut vertragen und es wurden keine schwerwiegenden Nebenwirkungen gemeldet. Rallybio ist auf dem Weg, im vierten Quartal 2024 eine Phase-2-Dosierungsbestätigungsstudie bei schwangeren Frauen mit höherem Risiko für FNAIT zu starten.
- Phase 1 proof-of-concept study for RLYB212 met prespecified criteria
- RLYB212 demonstrated rapid and complete elimination of transfused HPA-1a positive platelets
- Both tested doses (0.09 mg and 0.29 mg) achieved ≥90% reduction in mean platelet elimination half-life vs placebo
- RLYB212 was generally well-tolerated with no reports of serious adverse events
- Phase 2 dose confirmation trial on track to initiate in Q4 2024
- None.
The publication of RLYB212's Phase 1 proof-of-concept study results in Thrombosis and Haemostasis marks a significant milestone for Rallybio. The data demonstrates that RLYB212 effectively clears HPA-1a positive platelets, which is important for preventing alloimmunization and FNAIT. This is a positive development for the company's pipeline.
The study's success in meeting prespecified proof-of-concept criteria with both tested doses (
The comparison to Rhesus factor D (RhD) prevention adds credibility to RLYB212's potential efficacy, as it mimics a well-established and successful prevention strategy. The general tolerability of RLYB212 without serious adverse events is also encouraging for its safety profile.
Rallybio's progress with RLYB212 represents a potential value driver for the company. The positive Phase 1 results and upcoming Phase 2 trial initiation in Q4 2024 provide a clear development pathway, which is important for investor confidence in biotech stocks.
The company's focus on an underserved area of maternal fetal health could translate into a significant market opportunity if RLYB212 continues to show promise. The ongoing FNAIT natural history study also adds value by potentially reducing the cost and complexity of future clinical trials.
However, investors should note that Rallybio is still in early clinical stages and significant hurdles remain before potential commercialization. The company's ability to fund ongoing development and maintain a strong cash position will be critical factors to watch in the coming quarters.
Rallybio's focus on FNAIT prevention addresses an unmet medical need in maternal-fetal health. The market potential for RLYB212 could be substantial, given the lack of approved preventive treatments for FNAIT. The company's strategy to develop a diverse dataset through its natural history study is smart, potentially streamlining the regulatory pathway.
The comparison to RhD prevention is particularly insightful, as it provides a proven model for market adoption. If RLYB212 can achieve similar success, it could become a standard of care in high-risk pregnancies. However, market penetration will depend on factors such as pricing, reimbursement and physician education about FNAIT risks.
Rallybio's progress puts them in a strong position in this niche market, but they should be prepared for potential competition as the unmet need becomes more widely recognized in the pharmaceutical industry.
– Study Demonstrates that RLYB212 Rapidly Clears HPA-1a Positive Platelets, an Essential Step in Preventing Alloimmunization and FNAIT –
– Rallybio On Track to Initiate RLYB212 Phase 2 Dose Confirmation Trial in Pregnant Women at Higher Risk for FNAIT in 4Q 2024 –
“These data were instrumental in our selection of the initial dose for the upcoming Phase 2 trial of RLYB212, which is on track for initiation in the fourth quarter of 2024,” said Stephen Uden, M.D., Chief Executive Officer of Rallybio. “Our primary focus is to advance RLYB212 through development with a goal of bringing a new therapy to a completely underserved area of maternal fetal health.”
Data from the Phase 1 proof-of-concept study demonstrated that subcutaneous administration of RLYB212 produced a dose-dependent, rapid, and complete elimination of transfused HPA-1a positive platelets in HPA-1a negative subjects, with both doses (0.09 mg and 0.29 mg) meeting the prespecified proof-of-concept criteria of ≥
The bolus challenge of HPA-1a positive platelets was designed as a surrogate for a large 30 mL fetal-maternal hemorrhage. Platelet elimination profiles after subcutaneous administration of RLYB212 were consistent with those of Rhesus factor D (RhD)-positive erythrocytes after intramuscular administration of anti-RhD agents, which are well-established to safely and effectively prevent RhD alloimmunization during pregnancy. Consistent with previously reported data, RLYB212 was generally well-tolerated with no reports of serious or severe adverse events.
Rallybio is on track to initiate a Phase 2 dose confirmation trial in pregnant women at higher risk for HPA-1a alloimmunization and FNAIT in the fourth quarter of 2024. The Company also continues to screen pregnant women in its ongoing FNAIT natural history study, which is designed to provide a contemporary dataset for HPA-1a alloimmunization frequency in a racially and ethnically diverse population that can serve as a control arm for a planned Phase 3 trial.
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
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 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 FNAIT natural history study, 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
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Investors
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Rallybio Corporation
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Source: Rallybio Corporation
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