Rallybio Announces Initiation of Phase 2 Clinical Trial of RLYB212
Rallybio (RLYB) has initiated a Phase 2 clinical trial for RLYB212, targeting pregnant women at higher risk for HPA-1a alloimmunization and fetal and neonatal alloimmune thrombocytopenia (FNAIT). The single-arm trial will evaluate pharmacokinetics and safety, with screening now underway for the first sentinel participant. The study will enroll eight participants across three stages: one sentinel pregnant woman, followed by Cohort 1 with three women, and Cohort 2 with four women. RLYB212 will be administered subcutaneously starting at Gestational Week 16 and continuing every 4 weeks until birth. The trial will be conducted across sites in Belgium, the Netherlands, Norway, Sweden, and the United Kingdom.
Rallybio (RLYB) ha avviato uno studio 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 a braccio singolo valuterà la farmacocinetica e la sicurezza, con lo screening già in corso per la prima partecipante sentinella. Lo studio recluterà otto partecipanti in tre fasi: una donna in gravidanza sentinella, seguita dal Coorte 1 con tre donne e dal Coorte 2 con quattro donne. RLYB212 sarà somministrato per via sottocutanea a partire dalla settimana gestazionale 16 e continuando ogni 4 settimane fino al parto. La sperimentazione sarà condotta in siti in Belgio, Paesi Bassi, Norvegia, Svezia e Regno Unito.
Rallybio (RLYB) ha iniciado un ensayo clínico de Fase 2 para RLYB212, enfocado en mujeres embarazadas con alto riesgo de alloimmunización HPA-1a y trombocitopatía alloinmune fetal y neonatal (FNAIT). El ensayo de brazo único evaluará la farmacocinética y la seguridad, con el cribado ya en marcha para la primera participante centinela. El estudio inscribirá a ocho participantes en tres etapas: una mujer embarazada centinela, seguida por el Cohorte 1 con tres mujeres y el Cohorte 2 con cuatro mujeres. RLYB212 se administrará por vía subcutánea comenzando en la Semana Gestacional 16 y continuará cada 4 semanas hasta el parto. El ensayo se llevará a cabo en sitios en Bélgica, Países Bajos, Noruega, Suecia y el Reino Unido.
Rallybio (RLYB)는 HPA-1a 알로면역 및 태아 및 신생아 알로면역 혈소판감소증(FNAIT)의 위험이 높은 임산부를 대상으로 RLYB212에 대한 2상 임상시험을 시작했습니다. 단일 그룹 시험은 약물의 약동학 및 안전성을 평가하며, 현재 첫 번째 센티넬 참가자에 대한 스크리닝이 진행 중입니다. 이 연구는 세 단계에 걸쳐 총 8명의 참가자를 모집합니다: 하나의 센티넬 임산부, 그 다음 3명의 여성이 포함된 코호트 1, 그리고 4명의 여성이 포함된 코호트 2입니다. RLYB212는 임신 16주부터 시작하여 출산까지 4주마다 피하 주사로 투여될 예정입니다. 이 시험은 벨기에, 네덜란드, 노르웨이, 스웨덴, 영국의 여러 장소에서 진행됩니다.
Rallybio (RLYB) a lancé un essai clinique de phase 2 pour RLYB212, visant les femmes enceintes à risque accru d'alloiimmunisation HPA-1a et de thrombocytopénie alloimmune fœtale et néonatale (FNAIT). Cet essai à bras unique évaluera la pharmacocinétique et la sécurité, avec un dépistage en cours pour la première participante sentinelle. L'étude recrute huit participants en trois étapes : une femme enceinte sentinelle, suivie du Cohorte 1 avec trois femmes et du Cohorte 2 avec quatre femmes. RLYB212 sera administré par voie sous-cutanée à partir de la semaine de grossesse 16 et continuera toutes les 4 semaines jusqu'à l'accouchement. L'essai sera mené sur des sites en Belgique, aux Pays-Bas, en Norvège, en Suède et au Royaume-Uni.
Rallybio (RLYB) hat eine klinische Studie der Phase 2 zu RLYB212 gestartet, die sich an schwangere Frauen mit hohem Risiko für HPA-1a-Alloimmunisierung und fetale sowie neonatale Alloimmunthrombozytopenie (FNAIT) richtet. Die Einarmstudie wird die Pharmakokinetik und Sicherheit bewerten, wobei das Screening für die erste Sentinel-Teilnehmerin bereits im Gange ist. Die Studie wird insgesamt acht Teilnehmerinnen in drei Phasen einschließen: Eine sentinel schwangere Frau, gefolgt von Kohorte 1 mit drei Frauen und Kohorte 2 mit vier Frauen. RLYB212 wird subkutan ab der Schwangerschaftswoche 16 verabreicht und alle 4 Wochen bis zur Geburt fortgesetzt. Die Studie wird an Standorten in Belgien, den Niederlanden, Norwegen, Schweden und dem Vereinigten Königreich durchgeführt.
- First-ever potential preventative therapeutic for FNAIT
- Trial progression to Phase 2 stage
- Multi-country trial implementation across five European nations
- Small trial size of only 8 participants indicates initial scope
- Extended trial duration due to three-stage enrollment process with data reviews between cohorts
Insights
The initiation of Phase 2 trials for RLYB212 represents a significant advancement in addressing FNAIT, a rare but serious condition affecting pregnancies. The trial's design is notably conservative and safety-focused, with a careful staged enrollment approach starting with a single sentinel patient followed by two small cohorts. This methodical approach is appropriate given the sensitive patient population.
The multi-country trial structure across five European nations suggests strong investigator interest and should facilitate recruitment, though the small target enrollment of eight participants indicates this is primarily a safety and PK study rather than a full efficacy trial. The 16-week gestational age intervention point is strategically chosen, as it allows for preventive treatment while maintaining an acceptable safety profile.
For investors, while this development is positive, the small trial size and staged approach means revenue impact is still years away. The rare disease focus and preventive nature of the treatment could support premium pricing if successful, but market size will be by the specific patient population.
— Screening is Now Underway to Identify Pregnant Women at Higher Risk for HPA-1a Alloimmunization and FNAIT —
“The initiation of screening in the RLYB212 Phase 2 trial is a significant milestone,” said Stephen Uden, M.D., Chief Executive Officer of Rallybio. “We are thrilled to achieve this critical step towards delivering on our mission to prevent maternal alloimmunization, and FNAIT and its potentially catastrophic consequences. We thank trial participants as well as our dedicated investigators, site staff, and our partners for their commitment to our shared mission. We look forward to providing further updates on the trial’s progress upon dosing of the first participant.”
The single-arm Phase 2 dose confirmation trial (2024-512651-20/NCT06435845) is designed to assess the pharmacokinetics (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 will be initiated by Gestational Week 16 and will continue every 4 weeks through parturition.
“The impact of FNAIT can be devastating for babies, parents, and families, and the need for a safe and effective therapeutic that can prevent maternal alloimmunization and FNAIT is high,” said Dr. Vasilis Sitras, M.D., Head of Department, Fetal Medicine, Oslo University Hospital. “We are excited to participate in the development of RLYB212 as the first ever potential preventative therapeutic and hope that one day, we will see an end to pregnancies impacted by FNAIT.”
The Phase 2 trial is designed to enroll participants in three stages: first with a sentinel pregnant woman, for which screening is now underway, an initial cohort (Cohort 1) that will include three pregnant women, and 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 across
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 screening participants in the RLYB212 Phase 2 trial and the timing of enrolling the first participant, and our expectations regarding the usefulness of data from the Phase 2 trial. 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
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Media Contact
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Source: Rallybio Corporation
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