Linvoseltamab

In 2024, FDA issued CRLs for MDMA in PTSD and several cancer drugs due to manufacturing issues. Biosimilars for aflibercept and denosumab were approved, including first interchangeables. Two drugs for Niemann-Pick disease type C and a novel schizophrenia treatment targeting cholinergic receptors were also approved.

The FDA rejected Johnson & Johnson's subcutaneous amivantamab application due to manufacturing issues, not safety or efficacy. The subcutaneous form was non-inferior to the IV version, offering greater convenience for advanced NSCLC patients with EGFR mutations. J&J is working with the FDA to resolve the issue.

Regeneron Pharmaceuticals announced new data from its hematology pipeline at the ASH 2024 Annual Meeting, showcasing advancements in treating blood cancers and disorders. Key highlights include head-to-head results for pozelimab plus cemdisiran vs. ravulizumab in PNH, odronextamab's potential in follicular lymphoma, and progress in DLBCL and MZL treatments.

Q3 2024 revenues up 11% to $3.72 billion; Dupixent global net sales by Sanofi up 23% to $3.82 billion; U.S. net sales for EYLEA HD and EYLEA up 3% to $1.54 billion; Libtayo global net sales up 24% to $289 million; GAAP diluted EPS up 30% to $11.54; FDA approves Dupixent for COPD and eosinophilic phenotype; positive results for Dupixent in CSU and BP trials.

Bispecific antibodies, with two binding domains, offer targeted therapeutics and address co-administration challenges, gaining traction in biopharma. Initially approved for cancer, they now target various diseases, with 12 approved in the U.S. and more in development. Their dual action reduces patient burden and enhances treatment effects, potentially transforming therapeutic paradigms beyond oncology. The market is projected to grow significantly due to rising chronic disease incidence and technological advancements.

Various drugs and their statuses are listed, including IN-001 for anaphylaxis (Fast Track), Finereonone for heart failure (Phase 3), Tirzepatide for heart failure and obesity (Phase 3), TMB-001 for congenital ichthyosis (Phase 3), Bentracimab for ticagrelor reversal (Priority Review), Fabhalta for immunoglobulin A nephropathy (accelerated approval), RLS-0071 for acute graft-vs-host disease (Fast Track and Orphan Drug), CHIKV VLP for chikungunya (Priority Review), Diazoxide choline for Prader-Willi syndrome (Priority Review), Mirdametinib for neurofibromatosis type 1 (Priority Review), Setmelanotide for genetic obesity (Priority Review), AR882 for gout (Fast Track), SPN-830 for Parkinson disease (NDA review), BGB-16673 for chronic lymphocytic leukemia (Fast Track), Cabozantinib for neuroendocrine tumors (NDA review), Linvoseltamab for multiple myeloma (Complete Response Letter), Nivolumab plus ipilimumab for hepatocellular carcinoma (BLA review), Midomafetamine for PTSD (Complete Response Letter), and Tezepelumab-ekko for COPD (Breakthrough Therapy).

Linvoseltamab, a BCMA bispecific antibody, showed efficacy in relapsed/refractory multiple myeloma with an objective response rate of 70.9%, but requires aggressive supportive care to manage high infection risks, especially in communities without clinical trial experience. The importance of supportive care, including intravenous immunoglobulin, and potentially reducing dosing frequency to mitigate infection risks, was emphasized.

FDA issued a Complete Response Letter for Regeneron's linvoseltamab BLA in R/R MM; issue relates to a third-party fill/finish manufacturer. Regeneron working with manufacturer and FDA to resolve and bring linvoseltamab to R/R MM patients. EMA review ongoing. Linvoseltamab is investigational, not yet approved.

At EHA 2024, key studies on MM treatments were presented, including DREAMM-8, LINKER-MM1, and GMMG-HD7. Blenrep showed promising results in RRMM, with a 77% ORR. Linvoseltamab demonstrated efficacy in RRMM, with a 71% ORR. Sarclisa improved treatment responses in newly diagnosed MM, showing higher MRD negativity rates.

Linvoseltamab, a bispecific antibody for relapsed/refractory multiple myeloma, showed promising results in the LINKER-MM1 trial with a 71% ORR and durable responses. FDA decision expected by August 2024. High response rates across subgroups, manageable adverse events, and ongoing Phase 3 trial.