Rallybio Announces Promising RLYB212 and RLYB332 Preclinical Data at the 66th American Society of Hematology Annual Meeting
Rallybio (RLYB) presented promising preclinical data for two pipeline candidates at the 66th ASH Annual Meeting. The first study showed that RLYB212 successfully prevented maternal alloimmunization and FNAIT in pregnant mice at doses of 1.01 or 5.05 µg/kg, with pups maintaining normal platelet counts and showing no signs of thrombocytopenia.
The second study demonstrated that RLYB332, a long-acting anti-matriptase-2 antibody, showed rapid and sustained effects on pharmacodynamic parameters including serum iron, UIBC, and transferrin saturation in humanized FcRn mice. The treatment was well-tolerated and showed superior results compared to comparator molecules, positioning it as a potential best-in-class therapeutic for iron overload diseases.
Rallybio (RLYB) ha presentato dati preclinici promettenti per due candidati della pipeline al 66° Congresso Annuale dell'ASH. Il primo studio ha dimostrato che RLYB212 ha prevenuto con successo l'alloimmunizzazione materna e il FNAIT in topi gravidi a dosi di 1.01 o 5.05 µg/kg, con i cuccioli che mantenvano conteggi piastrinici normali e non mostravano segni di trombocitopenia.
Il secondo studio ha dimostrato che RLYB332, un anticorpo anti-matriptasi-2 a lunga durata, ha mostrato effetti rapidi e prolungati su parametri farmacodinamici tra cui ferro sierico, UIBC e saturazione della transferrina in topi FcRn umanizzati. Il trattamento è stato ben tollerato e ha mostrato risultati superiori rispetto a molecole di confronto, posizionandolo come un potenziale terapeutico di riferimento per le malattie da sovraccarico di ferro.
Rallybio (RLYB) presentó datos preclínicos prometedores para dos candidatos de su pipeline en la 66ª Reunión Anual de ASH. El primer estudio mostró que RLYB212 logró prevenir la aloimmunización materna y el FNAIT en ratonas embarazadas a dosis de 1.01 o 5.05 µg/kg, con las crías manteniendo conteos de plaquetas normales y sin signos de trombocitopenia.
El segundo estudio demostró que RLYB332, un anticuerpo anti-matriptasa-2 de acción prolongada, mostró efectos rápidos y sostenidos sobre parámetros farmacodinámicos, incluidos el hierro sérico, UIBC y la saturación de transferrina en ratones FcRn humanizados. El tratamiento fue bien tolerado y mostró resultados superiores en comparación con moléculas de referencia, posicionándolo como un potencial tratamiento de referencia para enfermedades por sobrecarga de hierro.
Rallybio (RLYB)는 제66회 ASH 연례 회의에서 두 개의 파이프라인 후보에 대한 유망한 전임상 데이터를 발표했습니다. 첫 번째 연구에서는 RLYB212가 임신한 쥐에서 1.01 또는 5.05 µg/kg의 용량으로 모체 면역화 및 FNAIT를 성공적으로 예방했으며, 새끼들은 정상적인 혈소판 수치를 유지하고 혈소판 감소증의 징후를 보이지 않았습니다.
두 번째 연구에서는 RLYB332라는 장기 작용하는 항-마트리프타제-2 항체가 인간화된 FcRn 쥐에서 혈청 철, UIBC 및 트랜스페린 포화도와 같은 약리역학적 매개변수에 빠르고 지속적인 영향을 미쳤음을 보여주었습니다. 이 치료는 잘 견디었고 비교할 수 있는 분자들에 비해 우수한 결과를 보여주며, 철분 과잉 질환을 위한 잠재적인 최고의 치료제로 자리 잡고 있습니다.
Rallybio (RLYB) a présenté des données précliniques prometteuses pour deux candidats de son pipeline lors de la 66e réunion annuelle de l'ASH. La première étude a montré que RLYB212 a réussi à prévenir l'allo-immunisation maternelle et le FNAIT chez des souris enceintes à des doses de 1,01 ou 5,05 µg/kg, les petits maintenant des taux de plaquettes normaux et ne montrant aucun signe de thrombocytopénie.
La deuxième étude a démontré que RLYB332, un anticorps anti-matriptase-2 à action prolongée, a montré des effets rapides et durables sur les paramètres pharmacodynamiques, y compris le fer sérique, l'UIBC et la saturation en transferrine chez des souris FcRn humanisées. Le traitement a été bien toléré et a montré de meilleurs résultats par rapport aux molécules témoins, se positionnant comme un traitement potentiel de premier choix pour les maladies liées à une surcharge en fer.
Rallybio (RLYB) hat auf dem 66. Jahrestreffen der ASH vielversprechende präklinische Daten zu zwei Pipeline-Kandidaten vorgestellt. Die erste Studie zeigte, dass RLYB212 erfolgreich eine maternale Alloimmunisierung und FNAIT bei schwangeren Mäusen in Dosen von 1.01 oder 5.05 µg/kg verhinderte, wobei die Nachkommen normale Thrombozytenwerte beibehielten und keine Anzeichen von Thrombozytopenie zeigten.
Die zweite Studie ergab, dass RLYB332, ein langwirksamer Anti-Matriptase-2-Antikörper, schnell und nachhaltig auf pharmakodynamische Parameter wie Serum-Eisen, UIBC und Transferrinsättigung bei humanisierten FcRn-Mäusen wirkte. Die Behandlung wurde gut vertragen und zeigte überlegene Ergebnisse im Vergleich zu Vergleichsmolekülen und positioniert sich als potenzieller Therapeutikum der Spitzenklasse für Eisenüberladungserkrankungen.
- RLYB212 demonstrated efficacy in preventing maternal alloimmunization and FNAIT in preclinical studies
- RLYB212 showed positive safety profile with no thrombocytopenia in pups
- RLYB332 exhibited superior performance compared to competitor molecules
- RLYB332 demonstrated good tolerability in preclinical studies
- Both candidates are still in early stages requiring further clinical validation
- No human efficacy data presented yet
Insights
“With our Phase 2 trial underway evaluating RLYB212 in pregnant women at higher risk of maternal alloimmunization and FNAIT, we are pleased that our collaborators at Versiti continue to add to the preclinical data showing RLYB212 has the potential to be a safe and effective preventative therapeutic using innovative nonclinical models,” said Stephen Uden, M.D., Chief Executive Officer of Rallybio. “Additionally, we are delighted to share, for the first time, preclinical data demonstrating the potential for RLYB332, our long-acting anti-matriptase-2 antibody, to be a best-in-class therapeutic for patients with diseases of iron overload.”
In a poster titled “Prophylactic Administration of HPA-1a–Specific Antibody RLYB212 Safely Prevents Fetal/Neonatal Alloimmune Thrombocytopenia Due to HPA-1a Incompatibility in Pregnant Mice,” preclinical data was presented demonstrating that prophylactic administration of RLYB212 to pregnant mice at doses of 1.01 or 5.05 µg/kg prevented maternal alloimmunization. Further, pups born to RLYB212-treated, but not untreated, pregnant mice had normal platelet counts, demonstrating the ability of RLYB212 to prevent fetal and neonatal alloimmune thrombocytopenia (FNAIT). Data also showed that administration of RLYB212 did not cause thrombocytopenia in pups, supporting its safety as a prophylactic treatment.
In a poster titled “Long-Acting Anti-Matriptase-2 Antibody as a Potentially Best-in-Class Therapy for Iron Overload Diseases,” preclinical data was presented demonstrating that single intravenous injections of RLYB332 to humanized FcRn mice had rapid and sustained effects on pharmacodynamic (PD) parameters, including serum iron, unsaturated iron binding capacity (UIBC), and transferrin saturation (TSAT), and these effects were greater than those produced by comparator molecules. Additionally, treatment with RLYB332 was generally well-tolerated. These findings support RLYB332 as a potentially best-in-class therapeutic for the treatment of diseases of iron overload.
Poster presentation details:
Title: Prophylactic Administration of HPA-1a–Specific Antibody RLYB212 Safely Prevents Fetal/Neonatal Alloimmune Thrombocytopenia in Pregnant Mice
Publication Number: 1185
Session Name: 311. Disorders of Platelet Number or Function: Clinical and Epidemiological: Poster I
Session Date: Saturday, December 7, 2024
Presentation Time: 5:30 p.m. - 7:30 p.m. PT
Title: Long-Acting Anti-Matriptase-2 Antibody as a Potentially Best-in-Class Therapy for Iron Overload Diseases
Publication Number: 3854
Session Name: 102. Iron Homeostasis and Biology: Poster III
Session Date: Monday, December 9, 2024
Presentation Time: 6:00 p.m. - 8:00 p.m. PT
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 our expectations regarding future favorable clinical results based on RLYB212 and RLYB332 preclinical data, including our expectations regarding efficacy, safety, dosing, and administration, whether a monoclonal antibody inhibitor of matriptase-2 will be an effective treatment of diseases of iron overload, and the potential success of RLYB212 and RLYB332 for the diseases that we expect to treat. 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 a 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
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Mission North
(760) 579-2134
rallybio@missionnorth.com
Source: Rallybio Corporation
FAQ
What were the key findings for RLYB212 in the ASH 2024 presentation?
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