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Scientific Publications

Contributing to and advancing the scientific dialogue.

April 2024

Clinical activity and safety of tovorafenib in patients with optic pathway gliomas in FIREFLY-1

Leary SES, de Vos-Kerkhof E, Perreault S, Witt O, Ziegler DS, Hernáiz Driever P, Franson AT, Baxter PA, Whipple NS, Kline C, Segal D, Jabado N, Bailey S, McCowage G, Hansford JR, Khuong-Quang D-A, Gottardo HG, Hassall T, Han JW, Yalon Oren M, Chi SN, McLeod L, Qiu J, McKenna C, Da Costa D, Govinda Raju S, Hargrave D, Kilburn LB, Landi DB, Nysom K. 902. Clinical Activity and Safety of Tovorafenib in Patients with Optic Pathway Gliomas in FIREFLY-1. Poster presentation at: 2024 ASPHO (American Society of Pediatric Hematology/Oncology); April 4 & 5, 2024; Seattle, WA.

Jan 2024

A phase 3, randomized trial of tovorafenib vs. chemotherapy in pediatric and young adult patients with newly diagnosed low-grade glioma harboring an activating RAF alteration

van Tilburg CM, Kilburn LB, Perreault S, Schmidt R, Azizi AA, Cruz-Martínez O, Zápotocký M, Scheinemann K, Schouten-van Meeteren AYN, Sehested A, Opocher E, Hernáiz Driever P, Avula S, Ziegler DS, Capper D, Koch A, Sahm F, Qiu J, Tsao L-P, Blackman SC, Manley P, Milde T, Witt R, Jones DTW, Hargrave D, Witt O. LOGGIC/FIREFLY-2: A phase 3, randomized trial of tovorafenib vs. chemotherapy in pediatric and young adult patients with newly diagnosed low-grade glioma harboring an activating RAF alteration [published online ahead of print January 30, 2024]. BMC Cancer. 2024.

Nov 2023

Clinical activity of RAF inhibitor tovorafenib according to prior MAPK inhibitor treatment in the registrational low-grade glioma arm of the phase 2 FIREFLY-1 (PNOC026) study

Khuong-Quang DA, Nysom K, Landi DB, Ziegler DS, Hernáiz Driever P, Leary SES, Bailey S, van der Lugt J, Perreault S, Waanders AJ, Baxter PA, Witt O, Hargrave D, McCowage G, Hansford JR, Toledano H, Oren L, Tan EEK, Gerber NU, Kang HJ, Larouche V, Abdelbaki MS, Cornelio I, Kim Y, Walter A, Manley P, Kilburn LB. CTNI-37. Clinical activity of RAF inhibitor tovorafenib according to prior MAPK inhibitor treatment in the registrational low-grade glioma arm of the phase 2 FIREFLY-1 (PNOC026) study. Oral presentation at: 28th Annual Meeting and Education Day of the Society for Neuro-Oncology, November 17, 2023, Vancouver, Canada.

Nov 2023

Clinical activity and safety of the RAF inhibitor tovorafenib in patients with optic pathway gliomas in the registrational pediatric low-grade glioma arm of the phase 2 FIREFLY-1 (PNOC026) study

Nysom K, Kilburn LB, Leary SES, Landi DB, de Vos-Kerkhof E, Perreault S, Witt O, Ziegler DS, Hernáiz Driever P, Franson AT, Baxter PA, Whipple NS, Kline C, Segal D, Jabado N, Bailey S, McCowage G, Hansford JR, Khuong-Quang D-A, Gottardo NG, Hassall TE, Han JW, Oren MY, Chi SN, Lee V, McLeod L, Qiu J, Walter A, Manley P, Hargrave D. CTNI-24. Clinical activity and safety of the RAF inhibitor tovorafenib in patients with optic pathway gliomas in the registrational pediatric low-grade glioma arm of the phase 2 FIREFLY-1 (PNOC026) study. Presentation at: 28th Annual Meeting and Education Day of the Society for Neuro-Oncology, November 17, 2023, Vancouver, Canada.

Nov 2023

The type II RAF inhibitor tovorafenib in relapsed/refractory pediatric low-grade glioma: the phase 2 FIREFLY-1 trial

Kilburn LB, Khuong-Quang D-A, Hansford JR, Landi D, van der Lugt J, Leary SES, Hernáiz Driever P, Bailey S, Perreault S, McCowage G, Waanders AJ, Ziegler DS, Witt O, Baxter PA, Kang HJ, Hassall TE, Han JW, Hargrave D, Franson AT, Oren MY, Toledano H, Larouche V, Kline C, Abdelbaki MS, Jabado N, Gottardo NG, Gerber NU, Whipple NS, Segal D, Chi SN, Oren L, Tan EEK, Mueller S, Cornelio I, McLeod L, Zhao X, Walter A, Da Costa D, Manley P, Blackman SC, Packer RJ, Nysom K. The type II RAF inhibitor tovorafenib in relapsed/refractory pediatric low-grade glioma: the phase 2 FIREFLY-1 trial [published online ahead of print November 17, 2023]. Nat Med. 2023.

Nov 2023

Preliminary clinical activity of the type II RAF inhibitor tovorafenib in RAF-fusion-driven recurrent/progressive sarcomas.

van Eijkelenburg Natasha KA, van der Lugt J, Da Costa D, Manley P, Hume S, Kopp LM, Lee J. Abstract 1552798. Preliminary clinical activity of the type II RAF inhibitor tovorafenib in RAF-fusion-driven recurrent/progressive sarcomas. Poster presentation at: The CTOS (Connective Tissue Oncology Society) 2023 Annual Meeting; November 1-4, 2023; Dublin, Ireland

July 2023

Activity of RAF Inhibitor Tovorafenib in a Pediatric Patient with a Recurrent Spindle Cell Sarcoma Harboring a Novel SNX8-BRAF Gene Fusion

Offer K, McGuire MT, Song K, Goldfischer MJ, Davare MA, Corless CL, Beadling C, Neff T, Cox MC, Raju SG, Blackman SC. Activity of Type II RAF Inhibitor Tovorafenib in a Pediatric Patient with a Recurrent Spindle Cell Sarcoma Harboring a Novel SNX8-BRAF Gene Fusion [published online ahead of print July 6, 2023]. JCO Precis Oncol. 2023;7.

June 2023

Clinical activity of pan-RAF inhibitor tovorafenib in the registrational pediatric low-grade glioma arm of the phase 2 FIREFLY-1 (PNOC026) study.

Kilburn L, Khuong-Quang D-A, Nysom K, Landi D, Ziegler DS, Hernáiz Driever P, Leary S, Bailey S, Van der Lugt J, Perreault S, Waanders AJ, Baxter P, Witt O, Hargrave D, McCowage G, Zhao X, Da Costa D, Cox MC, Manley P, Hansford JR. LGG-09. Clinical activity of pan-RAF inhibitor tovorafenib in the registrational pediatric low-grade glioma arm of the phase 2 FIREFLY-1 (PNOC026) study. Neuro-Oncol. 2023(suppl 1);i57. (ABSTRACT CITATION ID: NOAD073.218)

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