Database for
Therapeutic Antibodies
Therapeutic Antibodies
| Entry ID | 1047 |
| INN | Birtamimab |
| Status | Clinical |
| Drug code(s) | NEOD001; murine parent is 2A4 |
| Brand name | None |
| mAb sequence source | mAb humanized |
| General Molecular Category | Naked monospecific |
| Format, general category | Full length Ab |
| Format details | None |
| Isotype (Fc) | IgG1 |
| Light chain isotype | kappa |
| Linker | None |
| Ave. DAR | None |
| Conjugated/fused moiety | None |
| Discovery method/technology | None |
| Target(s) | Amyloid light chain |
| Indications of clinical studies | Systemic amyloidosis |
| Primary therapeutic area | Cardiovascular / hemostasis disorders |
| Most advanced stage of development (global) | Phase 3 |
| Status | Active |
| Start of clinical phase (IND filing or first Phase 1) | November 15, 2012 |
| Start of Phase 2 | |
| Start of Phase 3 | December 15, 2014 |
| Date BLA/NDA submitted to FDA | |
| Year of first approval (global) | None |
| Date of first US approval | |
| INN, US product name | None |
| US or EU approved indications | None |
| Company | Onclave Therapeutics Limited |
| Licensee/Partner | Prothena |
| Comments about company or candidate | The VITAL study (NCT02312206) of birtamimab in newly diagnosed AL amyloidosis was discontinued early per futility analysis; results published in Blood June 2023. (https://ashpublications.org/blood/article/doi/10.1182/blood.2022019406/496536/Birtamimab-plus-standard-of-care-in-light-chain) As of June 2022 company presentation: upcoming milestones in 2024 (Confirmatory Phase 3 AFFIRM-AL study results). NCT04973137 Phase 3 started in Aug 2021 recruiting as of last update in Nov 2024. Onclave Therapeutics Limited is a subsidiary of Prothena (formerly Neotope Biosciences, an Elan spin-off co.) Feb. 01, 2021: Prothena Corporation plc announced that following further analyses by the Company and multiple in-depth discussions with the U.S. Food and Drug Administration (FDA) regarding the previous analysis of patients categorized as Mayo Stage IV at baseline in the VITAL study, Prothena is advancing birtamimab into the confirmatory Phase 3 AFFIRM-AL study in this category of patients with AL amyloidosis. This registration-enabling study will be conducted with a primary endpoint of all-cause mortality at p<0.10 under a Special Protocol Assessment (SPA) agreement with FDA. April 18, 2019 I Prothena Corporation plc (NASDAQ:PRTA), a clinical-stage neuroscience company, today reported final results from the Phase 3 VITAL Amyloidosis study of NEOD001 (birtamimab) in newly diagnosed, treatment naïve patients with AL Amyloidosis and cardiac dysfunction (N=260), which was discontinued in 2018. The final hazard ratio (HR) for the composite primary endpoint (time to all-cause mortality or time to cardiac hospitalization more than 90 days after first infusion of study drug) of 0.835 (95% CI: 0.5799, 1.2011; p=0.3300) was consistent with the futility analysis reported in April 2018. Post hoc analyses of all-cause mortality revealed a potential survival benefit favoring NEOD001 in the category of patients at highest risk for early mortality (Mayo Stage IV, n=77) with a HR of 0.544 (95% CI: 0.2738, 1.0826; p=0.0787). This potential survival benefit was more pronounced in Mayo Stage IV patients during the initial 12 months of treatment, with a HR of 0.498 (95% CI: 0.2404, 1.0304; p=0.0556). April 2018: Prothena Corporation announced that the Company is discontinuing development of NEOD001, an investigational antibody that was being evaluated for the treatment of AL amyloidosis. The decision was based on results from the Phase IIb PRONTO study and a futility analysis of the Phase III VITAL study. Based on the results from the Phase 2b PRONTO study, which did not meet its primary or secondary endpoints, the Company asked the independent data monitoring committee (DMC) of the Phase 3 VITAL study to review a futility analysis of the ongoing VITAL study. The DMC recommended discontinuation of the VITAL study for futility. The Company therefore decided to discontinue all development of NEOD001, including the VITAL study as well as the open label extension studies. Two Phase 2 studies recruiting as of Oct 2017; topline results in the Phase 2b PRONTO study (129 patients) expected in the second quarter of 2018. Company press release dated 12/15/2014 indicates Phase 3 VITAL study was recently initiated, but NCT02312206 is listed with a start date of Feb 2015. Fast track designation received in Dec 2014. US orphan designation; Phase 1 study NCT01707264 listed with start date of April 2013 is recruiting as of June 2014 |
| Full address of company | 77 Sir John Rogerson’s Quay, Block C Grand Canal Docklands, Dublin 2, D02 VK60, Ireland Europe Ireland https://www.prothena.com/contact-us/ |
Humanized but given a chimeric name by USAN Council, as per Prothena Biosciences
| Anticipated events | None |
| Factor(s) contributing to discontinuation | None |