Database for
Therapeutic Antibodies
Therapeutic Antibodies
| Entry ID | 1175 |
| INN | Garetosmab |
| Status | Clinical |
| Drug code(s) | REGN2477 |
| Brand name | None |
| mAb sequence source | mAb human |
| General Molecular Category | Naked monospecific |
| Format, general category | Full length Ab |
| Format details | None |
| Isotype (Fc) | IgG4 |
| Light chain isotype | kappa |
| Linker | None |
| Ave. DAR | None |
| Conjugated/fused moiety | None |
| Discovery method/technology | Transgenic mouse (VelocImmune) |
| Target(s) | Activin A |
| Indications of clinical studies | Fibrodysplasia ossificans progressiva |
| Primary therapeutic area | Skeletal disorders |
| Most advanced stage of development (global) | Phase 3 |
| Status | Active |
| Start of clinical phase (IND filing or first Phase 1) | July 15, 2016 |
| Start of Phase 2 | November 15, 2017 |
| Start of Phase 3 | October 01, 2022 |
| 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 | Regeneron Pharmaceuticals |
| Licensee/Partner | None |
| Comments about company or candidate | Listed as Phase 3 in Regeneron pipeline dated Oct 2024. https://investor.regeneron.com/static-files/4ac68b22-7abe-4d1f-a15a-2a9b739cc935 NCT05394116 Phase 3 in FOP started as of Q3 2022 update from Regeneron; BLA is not expected until 2025+ (May 2023 presentation https://investor.regeneron.com/static-files/dbe7aad3-8aff-483f-9366-fa1678d7b738) NCT04577820 Phase 3 study in FOP was withdrawn (Phase 2 Study R2477-FOP-1940 has been withdrawn and the next phase of the development program is being planned); listed as Phase 2 in Regeneron pipeline dated Feb 8, 2022 Aug 2021: Regeneron is announcing that the Phase II LUMINA-1 safety and efficacy trial will be closing and that a new Phase III trial of the drug garetosmab is being planned, with initiation in 2022. The details of that trial, including the inclusion/exclusion criteria and clinical trial sites, are still under development. A Phase III trial is the next step in the drug development and regulatory review process. A decision was made to place the trial on clinical hold based on reports of fatal serious adverse events (SAEs) in the trial during the open-label portion in which all participants took the drug. As shared in their letter on June 4, stopping the study to evaluate safety and efficacy was the top priority for Regeneron. During this process, Regeneron met with the US Food & Drug Administration (FDA) about the safety and efficacy of garetosmab. It is standard practice to pause clinical trials when SAEs occur to fully evaluate their connection or lack of connection to the study drug. https://d3n8a8pro7vhmx.cloudfront.net/ifopa/pages/2537/attachments/original/1628862969/08.13.2021._IFOPA_Letter_on_Closing_LUMINA_1.pdf?1628862969 May 2021: List as possible regulatory submission in 2021; https://investor.regeneron.com/static-files/e90481f9-321f-4a55-804e-1d6ce4557112 NCT04577820 Phase 3 in Fibrodyplasia Ossificans Progressiva due to start in Nov 2020 not recruiting as of May 2021. Asset listed as Phase 2 in Regeneron pipeline dated May 10, 2021. Regeneron announced in Nov 2020 that it is pausing dosing of garetosmab (REGN2477) in the Phase I LUMINA-1 trial. The product was designed to address fibrodysplasia ossificans progressiva (FOP), an ultra-rate genetic disorder in which muscles, tendons and ligaments are progressively replaced by bone. The decision was based on reports of fatal serious adverse events in the trial during the open-label extension. Feb 2020: Regeneron plans regulatory submission in FOP in 2020 (to be discussed with regulators). Listed as Phase 2 in Regeneron pipeline update June 2019. NCT03188666 Phase 2 study in Fibrodysplasia Ossificans Progressiva started in Nov 2017. NCT02870400 Phase 1 in healthy women not of childbearing potential; Fast-track status for FOP. In 2017, the U.S. Food and Drug Administration (FDA) granted Fast Track designation for garetosmab for the prevention of heterotopic ossification in patients with FOP. In the U.S. and European Union (EU), garetosmab has been granted Orphan Designation. |
| Full address of company | Tarrytown, New York, United States North America United States of America https://www.regeneron.com/ |
Human monoclonal antibody that inhibits activin A, but does not bind other TGF-β family members. Nature Communications volume 8, Article number: 15153 (2017). REGN2477 was created by immunizing Regeneron’s VelocImmune mice with mature human activin A. The resulting antibody contains an IgG4 constant region and is a fully human monoclonal specific to activin
| Anticipated events | None |
| Factor(s) contributing to discontinuation | None |