Rallybio Announces Initiation of Dosing in RLYB212 Phase 2 Clinical Trial
Rallybio (RLYB) has initiated dosing in its Phase 2 clinical trial of RLYB212, targeting pregnant women at higher risk for HPA-1a alloimmunization and fetal and neonatal alloimmune thrombocytopenia (FNAIT). The trial will evaluate pharmacokinetics and safety across three stages: a sentinel participant, followed by two cohorts totaling eight participants.
The study involves subcutaneous administration of RLYB212 starting at Gestational Week 16 and continuing every 4 weeks through delivery. Key data readouts are expected in Q2 2025 for second trimester pharmacokinetic and safety data, and Q3 2025 for delivery-time data. The trial will be conducted at European sites.
Rallybio (RLYB) ha avviato la somministrazione nel suo trial clinico di fase 2 per RLYB212, rivolto a donne in gravidanza ad alto rischio di alloimmunizzazione HPA-1a e trombocitopenia alloimmune fetale e neonatale (FNAIT). Lo studio valuterà la farmacocinetica e la sicurezza attraverso tre fasi: un partecipante sentinella, seguito da due coorti che totalizzano otto partecipanti.
Lo studio prevede la somministrazione sottocutanea di RLYB212 a partire dalla Settimana Gestazionale 16 e continuando ogni 4 settimane fino al parto. I dati chiave sono attesi nel Q2 2025 per i dati di farmacocinetica e sicurezza del secondo trimestre, e nel Q3 2025 per i dati al momento del parto. Il trial sarà condotto in siti europei.
Rallybio (RLYB) ha iniciado la dosificación en su ensayo clínico de fase 2 de RLYB212, dirigido a mujeres embarazadas con mayor riesgo de aloimmunización HPA-1a y trombocitopenia aloimune fetal y neonatal (FNAIT). El ensayo evaluará la farmacocinética y la seguridad en tres etapas: un participante centinela, seguido de dos cohortes que totalizan ocho participantes.
El estudio implica la administración subcutánea de RLYB212 comenzando en la Semana Gestacional 16 y continuando cada 4 semanas hasta el parto. Se esperan resultados clave en Q2 2025 para datos de farmacocinética y seguridad del segundo trimestre, y en Q3 2025 para datos en el momento del parto. El ensayo se llevará a cabo en sitios europeos.
Rallybio (RLYB)는 RLYB212의 2상 임상시험에서 조산 위험이 높은 임산부를 대상으로 투약을 시작했습니다. 이 시험은 HPA-1a 알로면역 및 태아 및 신생아 알로면역 혈소판감소증(FNAIT)을 목표로 합니다. 시험은 세 가지 단계로 약동학 및 안전성을 평가하며, 첫 번째는 센티넬 참가자, 이후 두 개의 코호트로 총 8명의 참가자로 진행됩니다.
이 연구는 임신 16주부터 시작하여 출산까지 매 4주마다 RLYB212를 피하 투여하는 방식으로 진행됩니다. 주요 데이터 결과는 2025년 2분기에 두 번째 분기의 약동학 및 안전성 데이터, 2025년 3분기에는 출산 시 데이터가 예상됩니다. 이 임상시험은 유럽의 여러 장소에서 실시됩니다.
Rallybio (RLYB) a lancé la dosification dans son essai clinique de phase 2 portant sur RLYB212, visant les femmes enceintes à risque accru d'alloimmunisation HPA-1a et de thrombocytopénie alloimmune fœtale et néonatale (FNAIT). L'essai évaluera la pharmacocinétique et la sécurité à travers trois étapes : un participant sentinelle, suivi de deux cohortes totalisant huit participants.
L'étude implique l'administration sous-cutanée de RLYB212 à partir de la 16e semaine de grossesse et se poursuivant toutes les 4 semaines jusqu'à l'accouchement. Les résultats clés sont attendus au Q2 2025 pour les données de pharmacocinétique et de sécurité du deuxième trimestre, et au Q3 2025 pour les données au moment de l'accouchement. L'essai sera mené sur des sites en Europe.
Rallybio (RLYB) hat mit der Dosierung in seiner Phase-2-Studie zu RLYB212 begonnen, die sich an schwangere Frauen mit einem höheren Risiko für HPA-1a-Aloimmunisierung und fetale sowie neonataler alloimmune Thrombozytopenie (FNAIT) richtet. Die Studie wird pharmakokinetische und sicherheitsrelevante Daten in drei Phasen bewerten: ein Sentinel-Teilnehmer gefolgt von zwei Kohorten, die insgesamt acht Teilnehmer umfassen.
Die Studie umfasst die subkutane Verabreichung von RLYB212, beginnend in der 16. Schwangerschaftswoche und alle 4 Wochen bis zur Entbindung. Wichtige Daten werden im Q2 2025 für die pharmakokinetischen und sicherheitsrelevanten Daten des zweiten Trimester und im Q3 2025 für die Daten zur Entbindung erwartet. Die Studie wird an europäischen Standorten durchgeführt.
- First participant dosed in Phase 2 trial, marking significant program milestone
- Clear timeline for data readouts in Q2 and Q3 2025
- Structured trial design with staged enrollment allowing for safety monitoring
- Extended timeline for initial data readouts (2025)
- trial size with only 8 total participants
- Geographic limitation to European sites only
Insights
The initiation of RLYB212's Phase 2 trial represents a significant milestone in Rallybio's development pipeline, particularly given the company's $35.2M market capitalization. The trial's design reveals several strategic advantages:
The staged enrollment approach, beginning with a sentinel participant followed by two cohorts, demonstrates a thoughtful risk-mitigation strategy critical for maternal-fetal therapeutics. This design allows for careful safety monitoring and potential dose adjustments before broader participant exposure.
Key aspects warranting investor attention:
- The Q2 and Q3 2025 data readouts will be important catalysts, potentially affecting the company's valuation and strategic position
- The focus on European trial sites suggests a strategic approach to regulatory pathways and market access
- The trial's design to assess both maternal safety and neonatal outcomes addresses comprehensive regulatory requirements for maternal-fetal therapeutics
For a small-cap biotech, success in this program could be transformative. FNAIT represents an underserved market with competition. The potential for RLYB212 to prevent maternal alloimmunization addresses a critical medical need, which could translate into significant market opportunity if the drug demonstrates efficacy and safety.
However, investors should note that with current market capitalization and typical burn rates for clinical-stage biotechs, additional financing may be needed to complete clinical development. The upcoming data readouts will likely be critical decision points for both clinical progression and financial strategy.
– Key Data Readouts from Sentinel Participant Expected in 2Q 2025 and 3Q 2025 –
“Dosing the sentinel participant in our RLYB212 Phase 2 trial marks a significant milestone for our RLYB212 program and for Rallybio,” said Stephen Uden, M.D., Chief Executive Officer of Rallybio. “In addition to the assessment of safety, we are looking for the PK profile in the sentinel participant to demonstrate the ability of RLYB212 to achieve and maintain target concentrations throughout pregnancy. We plan to provide an update in the second quarter, as we continue to advance our mission to deliver a safe and effective therapeutic to prevent maternal alloimmunization and the potentially catastrophic consequences of FNAIT.”
The single-arm Phase 2 dose confirmation trial (2024-512651-20/NCT06435845) is designed to assess the PK and safety of RLYB212 in pregnant women at higher risk for HPA-1a alloimmunization and FNAIT. Secondary objectives include assessments of pregnancy and neonatal/infant outcomes, and the occurrence of emergent HPA-1a alloimmunization. Subcutaneous administration of RLYB212 is to be initiated by Gestational Week 16 and continued every 4 weeks through parturition.
The Phase 2 trial is designed to enroll participants in three stages: first with a sentinel pregnant woman, followed by an initial cohort (Cohort 1) that will include three pregnant women, and then a second cohort (Cohort 2) that will include four pregnant women, for a total target enrollment of eight participants. A data review for participants and infants is planned prior to the initiation of each cohort. The trial will seek to enroll participants at sites in
About RLYB212
RLYB212 is a subcutaneously administered monoclonal anti-HPA-1a antibody in development for the prevention of HPA-1a alloimmunization in pregnant women at higher risk for HPA-1a alloimmunization and FNAIT. RLYB212 is designed to rapidly eliminate HPA-1a positive fetal platelets from a pregnant woman’s circulation, thereby preventing maternal HPA-1a alloimmunization. Prevention of maternal alloimmunization eliminates the risk of FNAIT in the fetus.
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 needs 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 inhibitor 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 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 disclosure of preliminary PK and safety data for the sentinel participant in the RLYB212 Phase 2 trial, and whether RLYB212 will be an effective therapeutic approach for 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 2 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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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
media@rallybio.com
Source: Rallybio Corporation
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