Rallybio Announces Approval of Clinical Trial Applications for Phase 2 Trial of RLYB212 in Pregnant Women at Higher Risk of Alloimmunization and FNAIT
Rallybio (Nasdaq: RLYB) has received approval for clinical trial applications from the EMA and MHRA for a Phase 2 trial of RLYB212 in pregnant women at higher risk of HPA-1a alloimmunization and FNAIT. The trial, set to begin screening in Q4 2024, will be conducted across five European countries and will evaluate eight pregnant women. The single-arm study will assess pharmacokinetics and safety of RLYB212, a monoclonal anti-HPA-1a antibody, with subcutaneous administration starting at Gestational Week 16 and continuing every 4 weeks through parturition.
Rallybio (Nasdaq: RLYB) ha ricevuto l'approvazione per le domande di sperimentazione clinica da parte dell'EMA e dell'MHRA per un trial di Fase 2 di RLYB212 in donne in gravidanza ad alto rischio di alloimmunizzazione HPA-1a e FNAIT. La sperimentazione, che inizierà a selezionare partecipanti nel quarto trimestre del 2024, si svolgerà in cinque paesi europei e valuterà otto donne incinte. Lo studio a braccio singolo esaminerà la farmacocinetica e la sicurezza di RLYB212, un anticorpo monoclonale anti-HPA-1a, con somministrazione sottocutanea a partire dalla sedicesima settimana di gestazione e proseguendo ogni 4 settimane fino al parto.
Rallybio (Nasdaq: RLYB) ha recibido la aprobación de las solicitudes de ensayos clínicos por parte de la EMA y la MHRA para un ensayo de Fase 2 de RLYB212 en mujeres embarazadas con alto riesgo de aloimmunización HPA-1a y FNAIT. El ensayo, que comenzará con la selección de participantes en el cuarto trimestre de 2024, se llevará a cabo en cinco países europeos y evaluará a ocho mujeres embarazadas. El estudio de brazo único evaluará la farmacocinética y la seguridad de RLYB212, un anticuerpo monoclonal anti-HPA-1a, con administración subcutánea que comenzará en la semana 16 de gestación y continuará cada 4 semanas hasta el parto.
랠리바이오 (Nasdaq: RLYB)는 고위험 HPA-1a 알로면역화 및 FNAIT에 대비한 임산부를 대상으로 하는 2상 시험에 대해 EMA와 MHRA로부터 임상 시험 신청 승인을 받았습니다. 이 시험은 2024년 4분기에 참가자 선별을 시작할 예정이며, 5개 유럽 국가에서 실시되어 8명의 임산부를 평가할 것입니다. 단일 팔 연구는 16주 차부터 시작하여 출산까지 4주마다 진행되는 HPA-1a에 대한 단클론 항체인 RLYB212의 약리학적 작용과 안전성을 평가할 것입니다.
Rallybio (Nasdaq: RLYB) a reçu l'approbation des demandes d'essai clinique de l'EMA et de la MHRA pour un essai de Phase 2 de RLYB212 chez des femmes enceintes à haut risque d'allo-immunisation HPA-1a et de FNAIT. L'essai, qui débutera le dépistage au quatrième trimestre de 2024, sera réalisé dans cinq pays européens et évaluera huit femmes enceintes. L'étude en groupe unique évaluera la pharmacocinétique et la sécurité de RLYB212, un anticorps monoclonal anti-HPA-1a, avec une administration sous-cutanée commençant à la seizième semaine de gestation et se poursuivant toutes les 4 semaines jusqu'à l'accouchement.
Rallybio (Nasdaq: RLYB) hat die Genehmigung für klinische Prüfungsanträge von der EMA und MHRA für eine Phase 2-Studie von RLYB212 bei schwangeren Frauen mit erhöhtem Risiko für HPA-1a-Aloimmunisierung und FNAIT erhalten. Die Studie, die im vierten Quartal 2024 mit dem Screening beginnen wird, wird in fünf europäischen Ländern durchgeführt und acht schwangere Frauen bewerten. Die einarmige Studie wird die Pharmakokinetik und Sicherheit von RLYB212, einem monoklonalen Anti-HPA-1a-Antikörper, untersuchen, wobei die subkutane Verabreichung ab der 16. Schwangerschaftswoche alle 4 Wochen bis zur Entbindung erfolgen wird.
- First-ever clinical trial approved for preventing maternal alloimmunization and FNAIT
- Successful regulatory approvals from both EMA and MHRA
- Trial initiation on track for Q4 2024
- Small trial size of only 8 participants may limit data significance
- Extended treatment duration through entire pregnancy may impact patient compliance
Insights
The approval of CTAs for RLYB212's Phase 2 trial represents a significant milestone in addressing FNAIT prevention. The trial's design targeting pregnant women at higher risk of HPA-1a alloimmunization is particularly noteworthy as it's the first of its kind. The small trial size of 8 participants is appropriate for this rare condition and initial safety assessment.
The study's focus on both PK and safety endpoints, along with secondary outcomes tracking pregnancy and alloimmunization, provides a comprehensive evaluation framework. The Q4 2024 timeline for screening initiation and the multi-country approach across Belgium, Netherlands, Norway, Sweden and UK suggests a well-coordinated effort to access the target patient population.
While this development is promising, investors should note that this is still an early-stage trial and success in Phase 2 will be important for determining the program's future potential.
— First Ever Trial in Pregnant Women at Higher Risk of Alloimmunization and FNAIT —
— Screening in the Phase 2 Clinical Trial On Track to Initiate in 4Q 2024 —
“Securing these CTA approvals from European health authorities to advance RLYB212 into a Phase 2 trial is a significant achievement. These approvals are a testament to the dedication and innovation of our team and our partners as we advance this first ever program to prevent maternal alloimmunization and FNAIT,” said Stephen Uden, M.D., Chief Executive Officer of Rallybio. “We are activating clinical sites and expect to initiate screening this quarter, which will mark another important step toward achieving our mission to prevent FNAIT and its potentially devastating consequences.”
The single-arm Phase 2 dose confirmation trial (2024-512651-20/NCT06435845) will be conducted in
“These approvals recognize Rallybio’s commitment to safely and effectively progress a novel prophylactic approach for preventing alloimmunization in pregnant women at higher FNAIT risk,” said Steven Ryder, M.D., Chief Medical Officer at Rallybio. “We are proud to be leaders in bringing much needed innovation to this area of women’s health, which has gone unaddressed for far too long.”
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 activating sites and initiating screening for the Phase 2 RLYB212 clinical trial, and statements concerning Rallybio’s ability to successfully complete the Phase 2 trial, advance to a Phase 3 trial and obtain marketing approval of 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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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
Victoria Reynolds
Mission North
(760) 579-2134
rallybio@missionnorth.com
Source: Rallybio Corporation
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