On December 21, 2023 Galapagos NV (Euronext & NASDAQ: GLPG) reported that the first patient has been dosed in PAPILIO-1, the Phase 1/2 study to evaluate the safety, efficacy, and feasibility of our seven-day vein-to-vein, point-of-care manufactured BCMA CAR-T candidate, GLPG5301, in adult patients with relapsed/refractory multiple myeloma (rrMM) (Press release, Galapagos, DEC 21, 2023, View Source [SID1234638748]). This is Galapagos’ third oncology CAR-T program in clinical development.
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GLPG5301 is an autologous, second-generation/4-1BB B-cell maturation antigen (BCMA)-directed CAR-T product candidate, administered as an intravenous infusion of a fresh product in a single fixed dose, at point-of-care.
"Patients living with relapsed/refractory multiple myeloma have a very poor prognosis and a significant high unmet medical need for novel treatment options. CAR-T therapy is one such option. By combining innovative science with breakthrough point-of-care delivery of novel CAR-T therapies, we aim to enhance patient outcomes and improve their quality of life," said Jeevan Shetty, Head of Clinical Development Oncology at Galapagos. "We are very pleased that the first patient with rrMM in PAPILIO-1 has been dosed with our BCMA CAR-T candidate, GLPG5301. This marks another milestone in the roll-out of our point-of-care network and the build-up of our CAR-T portfolio, which now consists of three ongoing clinical programs in severe hemato-oncology indications."
About the PAPILIO-1 Phase 1/2 study (EU CT 2022-500782-27-00)
PAPILIO-1 is a Phase 1/2, open-label, multi-center study to evaluate the feasibility, safety, and efficacy of point-of-care manufactured GLPG5301, our BCMA CAR-T product candidate, in patients with relapsed/refractory multiple myeloma (rrMM) after ≥2 prior lines therapy. The primary objective of the Phase 1 part of the PAPILIO-1 study is to evaluate safety and determine the recommended dose for the Phase 2 part of the study. The primary objective of the Phase 2 part of the study is to evaluate the efficacy of GLPG5301, as measured by the objective response rate (ORR). Secondary objectives for both Phase 1 and Phase 2 include further assessment of the safety of GLPG5301, additional efficacy endpoints, including assessment of minimal residual disease (MRD), as well as the feasibility of point-of-care manufacture of GLPG5301 in rrMM patients. Each enrolled patient will be followed for 24 months.
During Phase 1, up to 3 dose levels will be evaluated and at least 12 patients will be enrolled to establish the recommended Phase 2 dose. Approximately 30 additional patients will be enrolled in the Phase 2 part of the study to confirm the safety and efficacy of GLPG5301.
About Galapagos’ innovative approach to CAR-T manufacturing near the point-of-care
Galapagos’ decentralized, innovative point-of-care CAR-T manufacturing platform consists of an end-to-end xCellit workflow management and monitoring software system, a decentralized, functionally closed, automated manufacturing platform for cell therapies (using Lonza’s Cocoon) and a proprietary quality control (QC) testing and release strategy. The combination of these three core components offers the potential for administration of a fresh product, a median vein-to-vein time of 7 days (i.e. the time between T-cell collection and CAR-T infusion), and greater physicians oversight throughout the process.
About Relapsed/refractory multiple myeloma (rrMM)
Multiple myeloma (MM) is typically characterized by the neoplastic proliferation of plasma cells producing a monoclonal immunoglobulin. The plasma cells proliferate in the bone marrow and may result in extensive skeletal destruction with osteopenia, and osteolytic lesions with or without pathologic fractures. The diagnosis of MM is made when one (or more) of the following clinical presentations are present: bone pain with lytic lesions discovered on routine skeletal films or other imaging modalities, an increased total serum protein concentration with the presence of a monoclonal protein in the urine or serum, and anemia, hypercalcemia or renal failure. The patient may be either symptomatic or their disease may be discovered incidentally.
Despite improvements in treatment, patient with MM ultimately relapse or become refractory to available regiments. Triple-refractory (refractory to CD38 monoclonal antibodies [mAbs], proteasome inhibitor [PI] and immunomodulatory drug [IMiD] or penta-refractory (refractory to CD38 mAbs, 2 Pls and 2 IMiDs) patients have a poor prognosis and are in urgent need of novel treatment options.