Rallybio to Initiate RLYB116 Confirmatory Clinical PK/PD Study in Second Quarter 2025
Rallybio (RLYB) announced new findings for RLYB116, its once-weekly subcutaneous C5 inhibitor for rare diseases. New biomarker analyses revealed that RLYB116 achieved greater complement inhibition in Phase 1 than initially reported, with the original assay overestimating free C5 levels by approximately ten-fold. The company completed manufacturing process enhancements expected to improve RLYB116's tolerability profile. A confirmatory clinical PK/PD study is planned for Q2 2025, involving two cohorts testing weekly doses of 150mg and 225mg over 4 weeks with a 10-week follow-up period.
Rallybio (RLYB) ha annunciato nuovi risultati per RLYB116, il suo inibitore sottocutaneo di C5 somministrato una volta a settimana per malattie rare. Nuove analisi dei biomarker hanno rivelato che RLYB116 ha raggiunto una inibizione del complemento maggiore nella Fase 1 rispetto a quanto inizialmente riportato, con l'assay originale che sovrastimava i livelli di C5 libero di circa dieci volte. L'azienda ha completato miglioramenti nel processo di produzione che si prevede miglioreranno il profilo di tollerabilità di RLYB116. Uno studio clinico PK/PD di conferma è programmato per il secondo trimestre del 2025, coinvolgendo due coorti che testeranno dosi settimanali di 150mg e 225mg per 4 settimane con un periodo di follow-up di 10 settimane.
Rallybio (RLYB) anunció nuevos hallazgos para RLYB116, su inhibidor subcutáneo de C5 administrado una vez por semana para enfermedades raras. Nuevos análisis de biomarcadores revelaron que RLYB116 logró una mayor inhibición del complemento en la Fase 1 de lo que se había informado inicialmente, con el ensayo original sobrestimando los niveles de C5 libre aproximadamente en diez veces. La empresa completó mejoras en el proceso de fabricación que se espera que mejoren el perfil de tolerancia de RLYB116. Un estudio clínico PK/PD de confirmación está planeado para el segundo trimestre de 2025, involucrando dos cohortes que probarán dosis semanales de 150 mg y 225 mg durante 4 semanas con un período de seguimiento de 10 semanas.
Rallybio (RLYB)는 희귀 질환을 위한 주 1회 피하 C5 억제제 RLYB116에 대한 새로운 결과를 발표했습니다. 새로운 바이오마커 분석에 따르면 RLYB116은 1상에서 처음 보고된 것보다 더 큰 보완 억제를 달성했으며, 원래의 분석이 자유 C5 수치를 약 10배 과대평가했다고 합니다. 이 회사는 RLYB116의 내약성 프로필을 개선할 것으로 예상되는 제조 공정 개선을 완료했습니다. 확증 임상 PK/PD 연구는 2025년 2분기로 계획되며, 주 150mg 및 225mg의 용량을 4주 동안 테스트하는 두 집단이 포함되며 10주 추적 관찰 기간이 있습니다.
Rallybio (RLYB) a annoncé de nouvelles découvertes concernant RLYB116, son inhibiteur sous-cutané de C5 administré une fois par semaine pour les maladies rares. De nouvelles analyses de biomarqueurs ont révélé que RLYB116 a atteint une inhibition du complément plus importante en Phase 1 que ce qui avait été initialement rapporté, le test d'origine ayant surestimé les niveaux de C5 libre d'environ dix fois. L'entreprise a complété des améliorations du processus de fabrication qui devraient améliorer le profil de tolérance de RLYB116. Une étude clinique PK/PD de confirmation est prévue pour le deuxième trimestre 2025, impliquant deux cohortes testant des doses hebdomadaires de 150 mg et 225 mg pendant 4 semaines avec une période de suivi de 10 semaines.
Rallybio (RLYB) hat neue Erkenntnisse zu RLYB116, seinem einmal wöchentlich subkutan verabreichten C5-Inhibitor für seltene Krankheiten, bekannt gegeben. Neue Biomarker-Analysen haben gezeigt, dass RLYB116 in Phase 1 eine stärkere Komplementinhibition erreicht hat als ursprünglich berichtet, wobei der ursprüngliche Test die freien C5-Spiegel etwa zehnfach überschätzt hat. Das Unternehmen hat Verbesserungen im Fertigungsprozess abgeschlossen, die voraussichtlich das Verträglichkeitsprofil von RLYB116 verbessern werden. Eine bestätigende klinische PK/PD-Studie ist für das zweite Quartal 2025 geplant, die zwei Kohorten umfassen wird, die wöchentliche Dosen von 150 mg und 225 mg über 4 Wochen mit einer 10-wöchigen Nachbeobachtungszeit testen.
- New biomarker analyses showed RLYB116 achieved better complement inhibition than initially reported
- Manufacturing process enhancements completed to improve drug tolerability
- Clear development timeline with confirmatory PK/PD study planned for Q2 2025
- Delayed timeline with next clinical study not starting until Q2 2025
- Initial Phase 1 study measurements were inaccurate, suggesting potential reliability issues
Insights
The biomarker characterization analyses reveal a significant finding - the original RLYB116 assay overestimated free C5 levels by approximately 10-fold, suggesting much stronger complement inhibition than initially reported. This is a important development that substantially improves the drug's potential efficacy profile.
The manufacturing process improvements completed in Q3 2024 to enhance drug substance purity could address the tolerability concerns from the Phase 1 trial. The planned confirmatory PK/PD study with weekly doses of 150mg and 225mg will be critical in validating both the improved tolerability and sustained complement inhibition.
However, investors should note that with initiation planned for Q2 2025, meaningful clinical data won't be available until late 2025 at the earliest. The company's small market cap of
-- New Biomarker Characterization Analyses Indicate RLYB116 Produced Complete and Sustained Complement Inhibition in Previous Phase 1 MAD Study –
-- Manufacturing Process Enhancements Expected to Further Improve RLYB116 Tolerability --
-- Webcast Today at 8:30 AM Eastern Time --
“Recently completed efforts to better understand the disconnect between the compelling preclinical potency data of RLYB116 and the results of the Phase 1 study indicate that RLYB116 achieved greater complement inhibition in the Phase 1 study than initially reported,” said Stephen Uden, M.D., Chief Executive Officer of Rallybio. “This, together with successful manufacturing process enhancements to further improve the tolerability profile, uniquely positions RLYB116 as a potentially best-in-class C5 inhibitor that can meet patient demand for a once-weekly, small volume, subcutaneous, self-administered therapeutic. We look forward to bringing RLYB116 into a confirmatory clinical PK/PD study in the second quarter of 2025 and expect RLYB116 to demonstrate complete and sustained complement inhibition and an improved tolerability profile.”
Following the completion of the RLYB116 Phase 1 single- and multiple-ascending dose (SAD/MAD) study in the fourth quarter of 2023, Rallybio conducted a series of biomarker characterization analyses. These analyses indicate the RLYB116 assay used to measure free C5 overestimated the levels of free C5 by approximately ten-fold, indicating that RLYB116 produced greater complement inhibition than initially reported.
Additionally, Rallybio completed manufacturing process enhancements in the third quarter of 2024 that are expected to further improve the tolerability of RLYB116. Based on the results of enhanced analytical techniques, including mass spectrometry, process enhancements have successfully further purified the RLYB116 drug substance. As a result, RLYB116 is expected to have a favorable tolerability profile at doses at and above those evaluated in the Phase 1 MAD study.
Rallybio plans to initiate a RLYB116 confirmatory clinical PK/PD study in the second quarter of 2025 to demonstrate improved tolerability as well as complete and sustained complement inhibition. This single-blind multiple ascending dose study will evaluate a 4-week treatment duration that will include two cohorts of 8 participants each. It is planned that Cohort 1 will evaluate weekly dosing of 150 mg and Cohort 2 will evaluate weekly dosing of 225 mg. Follow-up will continue for 10 weeks after the conclusion of treatment.
Webcast Information
The live webcast will be accessible through the Events and Presentations section of Rallybio’s investor relations website at https://investors.rallybio.com. A replay and accompanying slides of the webcast will be available on the Rallybio website for 30 days following the event.
About RLYB116 Phase 1 Study
RLYB116 is an innovative, once-weekly, small volume, subcutaneously injected C5 inhibitor in development for the treatment of patients with complement-mediated diseases. A Phase 1 single- and multiple-ascending dose (SAD/MAD) study was conducted in healthy participants. The MAD portion of the study had an adaptive single-blind design with a 4-week treatment duration and 10-week follow-up period. The study was designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of RLYB116 and included 4 cohorts: Cohort 1 (weekly dosing of 100 mg), Cohort 2 (3 doses of 100 mg the first week followed by weekly dosing), Cohort 3 (150 mg weekly dosing reduced to 125 mg weekly dosing), and Cohort 4 (75 mg twice the first week followed by 100 mg twice per week). The study was completed in the fourth quarter of 2023.
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, 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 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 planned RLYB116 confirmatory clinical PK/PD study, the expected dosing for RLYB116 in future clinical trials and whether RLYB116 will produce complete and sustained inhibition of C5, the frequency of administration of RLYB116, whether RLYB116 will be a best-in-class C5 inhibitor, whether the RLYB116 manufacturing process enhancements will improve tolerability or be successful at the desired doses, and our characterization of the actual level of complement inhibition delivered by RLYB116. 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 RLYB116 confirmatory clinical PK/PD study, 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
FAQ
When will Rallybio (RLYB) start the confirmatory PK/PD study for RLYB116?
What dosing regimens will be tested in Rallybio's (RLYB) RLYB116 confirmatory study?
What were the new findings about RLYB116's complement inhibition in Rallybio's (RLYB) Phase 1 study?
