• Alcindor T, Kasper B, Gounder M, et al. Tumor volume and T2 hyperintensity changes from DeFi: a phase 3, randomized, controlled trial of nirogacestat in patients with desmoid tumors. Poster discussion at: American Society of Clinical Oncology Annual Meeting; June 2-6, 2023; Chicago, IL. Supplemental materials.
• van der Graaf WT, Gounder M, Ratan R, et al. Impact of nirogacestat on pain, a key symptom in patients with desmoid tumors (DT): results from the phase 3 DeFi study. Poster presented at: American Society of Clinical Oncology Annual Meeting; June 2-6, 2023; Chicago, IL.
• Fernandez MM, Bell T, Tumminello B, et al. Burden of surgery in desmoid tumors. Poster presented at: Professional Society for Health Economics and Outcomes Research Annual Meeting; May 7-10, 2023; Boston, MA.
• Bell T, Fernandez MM, Khan S, et al. Patient burden of illness associated with desmoid tumors. Poster presented at: International Desmoid Tumor Research Workshop; September 23, 2022; virtual.
• Gounder MM, Atkinson TM, Bell T, et al. GOunder/Desmoid Tumor Research Foundation DEsmoid Symptom/Impact Scale (GODDESS©): psychometric properties and clinically meaningful thresholds as assessed in the phase 3 DeFi randomized controlled clinical trial. Poster presented at: International Desmoid Tumor Research Workshop; September 23, 2022; virtual.
• Kasper B, Ratan R, Alcindor T, et al. DeFi: A phase 3 trial of nirogacestat for progressing desmoid tumors (DT). Oral presentation at: Annual Meeting of the European Society for Medical Oncology; September 9-13, 2022; Paris, France.

Desmoid tumor publications

• Bektas M, Bell T, Khan S, et al. Desmoid tumors: a comprehensive review. Adv Ther. 2023 Jul 12. doi:10.1007/s12325-023-02592-0.
• Gounder MM, Atkinson TM, Bell T, et al. GOunder/Desmoid Tumor Research Foundation DEsmoid Symptom/Impact Scale (GODDESS©): psychometric properties and clinically meaningful thresholds as assessed in the Phase 3 DeFi randomized controlled clinical trial. Qual Life Res. 2023 Jun 22. doi: 10.1007/s11136-023-03445-7.
• Fernandez MM, Bell T, Tumminello B, Khan S, Zhou S, Oton AB. Disease and economic burden of surgery in desmoid tumors: a review. Expert Rev Pharmacoecon Outcomes Res. 2023;23(6):607-618.
• Gounder M, Ratan R, Alcindor T, et al. Nirogacestat, a gamma-secretase inhibitor for desmoid tumors. N Engl J Med. 2023;388(10):898-912.
• Reynolds AC, McKenzie LJ. Cancer treatment-related ovarian dysfunction in women of childbearing potential: management and fertility preservation options. J Clin Oncol. 2023;41(12):2281-2292.
• Federman N. Molecular pathogenesis of desmoid tumor and the role of γ-secretase inhibition. NPJ Precis Oncol. 2022;6(1):62.
• Riedel RF, Agulnik M. Evolving strategies for management of desmoid tumor. Cancer. 2022;128(16):3027-3040.
• O’Sullivan Coyne GO, Kummar S, Steinberg SM, et al. Extended progression-free survival and long-term safety of nirogacestat in patients with desmoid tumors. J Clin Oncol. 2022;40(16_suppl):11545-11545.*
• Anneberg M, Svane H, Fryzek J, et al. The epidemiology of desmoid tumors in Denmark. Cancer Epidemiol. 2022;77:102114.
• Takahashi T, Prensner JR, Robson CD, Janeway KA, Weigel BJ. Safety and efficacy of gamma-secretase inhibitor nirogacestat (PF-03084014) in desmoid tumor: report of four pediatric/young adult cases. Pediatr Blood Cancer. 2020;67(10):e28636.**
• Villalobos VM, Hall F, Jimeno A, et al. Long-term follow-up of desmoid fibromatosis treated with PF-03084014, an oral gamma secretase inhibitor. Ann Surg Oncol. 2018;25(3):768-775.
• Kummar S, Coyne GO, Do KT, et al. Clinical activity of the γ-secretase inhibitor PF-03084014 in adults with desmoid tumors (aggressive fibromatosis). J Clin Oncol. 2017;35(14):1561-1569.*
• Messersmith WA, Shapiro GI, Cleary JM, et al. A phase I, dose-finding study in patients with advanced solid malignancies of the oral γ-secretase inhibitor PF-03084014. Clin Cancer Res. 2015;21(1):60-67.

Ovarian granulosa cell tumor conference presentations

• Konstantinopoulos PA, Lewis JA, Shearer T, O’Brien H, Breitbach CJ, Cheng S. Trial in progress: a phase 2 trial of nirogacestat, a γ-secretase inhibitor, in patients with recurrent ovarian granulosa cell tumors (NCT 05348356). Poster presented at: Annual Meeting of the Society of Gynecologic Oncology; March 25-28, 2023; Tampa, FL.

Ovarian granulosa cell tumor publications

• Li J, Bao R, Peng S, Zhang C. The molecular mechanism of ovarian granulosa cell tumors. J Ovarian Res. 2018;11(1):13.
• Irusta G, Pazos MC, Abramovich D, De Zúñiga I, Parborell F, Tesone M. Effects of an inhibitor of the γ-secretase complex on proliferation and apoptotic parameters in a FOXL2-mutated granulosa tumor cell line (KGN). Biol Reprod. 2013;89(1):9.
• Shah SP, Kobel M, Senz J, et al. Mutation of FOXL2 in granulosa-cell tumors of the ovary. N Engl J Med. 2009;360(26):2719-2729.

Multiple myeloma conference presentations

• Callander N, Richardson P, Hus M, et al. Low-dose belantamab mafodotin (belamaf) in combination with nirogacestat vs belamaf monotherapy in patients with relapsed/refractory multiple myeloma (RRMM): phase 1/2 DREAMM-5 platform sub-study 3. Poster presented at: European Hematology Association; June 8-11, 2023; Frankfurt, Germany.
• Offner F, Decaux O, Hulin C, et al. Teclistamab + Nirogacestat in Relapsed/Refractory Multiple Myeloma: The Phase 1b MajesTEC-2 Study. Oral presentation at: European Hematology Association; June 8-11, 2023; Frankfurt, Germany.
• Shearer T, Williams RL, Johnson M, et al. Population pharmacokinetic-pharmacodynamic model of nirogacestat effects on B-cell maturation antigen in healthy subjects. Poster presented at: American Society of Clinical Oncology Annual Meeting; June 2-6, 2023; Chicago, IL. Supplemental materials.
• Landgren O, Lazandijian D, O’Connell A, Finn G, Raje N. MagnetisMM-4: an open label, phase 1b/2 umbrella study of elranatamab in combination with other anti-cancer treatments for patients with multiple myeloma. Poster presented at: American Society of Hematology Annual Meeting; December 10-13, 2022; New Orleans, LA.
• Otero PR, Joseph NS, Kumar SK, et al. Trial in progress: linvoseltamab (REGN5458), a BCMAxCD3 bispecific antibody, in a phase 1b multi-cohort study of combination regimens for patients with relapsed/refractory multiple myeloma. Poster presented at: American Society of Hematology Annual Meeting; December 10-13, 2022; New Orleans, LA.
• Shearer T, Williams Jr. RL, Johnson M, et al. Pharmacodynamic effects of nirogacestat, a gamma secretase inhibitor, on B-cell maturation antigen in healthy participants. Poster presented at: American Society of Hematology Annual Meeting; December 10-13, 2022; New Orleans, LA.
• Lonial S, Grosicki S, Hus M, et al. Synergistic effects of low-dose belantamab mafodotin in combination with a gamma-secretase inhibitor (nirogacestat) in patients with relapsed/refractory multiple myeloma (RRMM): DREAMM-5 study. Poster discussion presented at: American Society of Clinical Oncology Annual Meeting; June 3-7, 2022; Chicago, IL.
• Karwacz K, Hooper AT, Mathur D, et al. BCMAxCD3 bispecific antibody PF-06863135: preclinical rationale for therapeutic combinations. Poster presented at: American Association for Cancer Research Annual Meeting; April 24-29, 2020; virtual.
• Eastman S, Shelton C, Gupta I, et al. Synergistic activity of belantamab mafodotin (anti-BCMA immuno-conjugate) with PF-03084014 (gamma-secretase inhibitor) in BCMA-expressing cancer cell lines. Poster presented at: American Society of Hematology Annual Meeting; December 7-10, 2019; Orlando, FL.

Multiple myeloma publications

• Cowan AJ, Pont MJ, Sather BD, et al. γ-Secretase inhibitor in combination with BCMA chimeric antigen receptor T-cell immunotherapy for individuals with relapsed or refractory multiple myeloma: a phase 1, first-in-human trial. Lancet Oncol. 2023;24(7):811-822.
• Rodriguez C, Weisel K, Baz RC, et al. Dose escalation and expansion of Abbv-383 in combination with anti-cancer regimens in relapsed or refractory multiple myeloma. Blood. 2022;140(Supplement 1):7326-7327.
• Pillarisetti K, Powers G, Luistro L, et al. Teclistamab is an active T cell–redirecting bispecific antibody against B-cell maturation antigen for multiple myeloma. Blood Advances. 2020:4(18):4538-4549.
• Pont MJ, Hill T, Cole GO, et al. γ-Secretase inhibition increases efficacy of BCMA-specific chimeric antigen receptor T cells in multiple myeloma. Blood. 2019;134(19):1585-1597.

• Shearer T, Mizuno T, Vinks AA, Hoang T, Langseth AJ, Weiss BD. Population pharmacokinetics and pharmacodynamics (PK/PD) modeling of mirdametinib in patients with neurofibromatosis type 1-related plexiform neurofibromas. Poster presented at: Children’s Tumor Foundation NF Conference; June 21-24, 2022; Philadelphia, PA.
• Moertel C, Babovic-Vuksanovic D, Gershin T, Hirbe A, for the ReNeu Investigators. Phase 2b trial of the MEK inhibitor mirdametinib in patients with neurofibromatosis type 1-associated plexiform neurofibromas (ReNeu) – NCT03962543 – interim results. Oral presentation at: Children’s Tumor Foundation NF Conference; June 14-16, 2021; virtual.

NF1 publications

• Weiss BD, Wolters PL, Plotkin SR, et al. NF106: a Neurofibromatosis Clinical Trials Consortium phase II trial of the MEK inhibitor mirdametinib (PD-0325901) in adolescents and adults with NF1-related plexiform neurofibromas. J Clin Oncol. 2021;39(7):797-806.*
• Jousma E, Rizvi TA, Wu J, et al. Preclinical assessments of the MEK inhibitor PD-0325901 in a mouse model of neurofibromatosis type 1. Pediatr Blood Cancer. 2015;62(10):1709-1716.*
• Jessen WJ, Miller SJ, Jousma E, et al. MEK inhibition exhibits efficacy in human and mouse neurofibromatosis tumors. J Clin Invest. 2013;123(1):340-347.*

• Solomon B. Safety, pharmacokinetics, and antitumor activity findings from a phase 1b, open-label, dose-escalation and expansion study investigating RAF dimer inhibitor lifirafenib in combination with MEK inhibitor mirdametinib in patients with advanced or refractory solid tumors. Oral presentation at: American Association for Cancer Research Annual Meeting; April 14-19, 2023; Orlando, FL.
• Briker L, Johnson M, Kamal A, Dummer R, Levesque MP, Eichhoff OM. Vertical inhibition of the MAPK pathway as potential treatment strategy in NRAS-mutant melanoma. Poster presented at: EORTC-NCI-AACR Symposium on Molecular Targets and Cancer Therapeutics; Oct 26-28, 2022; Barcelona, Spain.
• Rosen EY, Patel P, Sarapa N, et al. A phase 1b/2a, open-label platform study to evaluate mirdametinib in combination with fulvestrant in ER+ metastatic breast cancers harboring MAPK-activating mutations. Poster presented at San Antonio Breast Cancer Symposium; Dec 7-10, 2021; San Antonio, TX.
• Yuan X, Zhang X, Du R, et al. RAF dimer inhibitor lifirafenib enhances the antitumor activity of MEK inhibitor mirdametinib in RAS mutant tumors. Poster presented at: American Association for Cancer Research Annual Meeting; April 24-29, 2020; virtual.

Other mirdametinib publications

• Vinitsky A, Chiang J, Bag AK, et al. Phase I/II evaluation of single agent mirdametinib (PD-0325901), a brain-penetrant MEK1/2 inhibitor, for the treatment of children, adolescents, and young adults with low-grade glioma (LGG). Neuro Oncol. 2022;24(Supplement_1):i92.
• Yuan X, Tang Z, Du R, et al. RAF dimer inhibition enhances the antitumor activity of MEK inhibitors in K-RAS mutant tumors. Mol Oncol. 2020;14(8):1833-1849.
• LoRusso PM, Krishnamurthi SS, Rinehart JJ, et al. Phase I pharmacokinetic and pharmacodynamic study of the oral MAPK/ERK kinase inhibitor PD-0325901 in patients with advanced cancers. Clin Cancer Res. 2010;16(6):1924-1937.

• Schram AM, Subbiah V, Sullivan R, et al. A first-in-human, phase 1a/1b, open-label, dose-escalation and expansion study to investigate the safety, pharmacokinetics, and antitumor activity of the RAF dimer inhibitor BGB-3245 in patients with advanced or refractory tumors. Oral presentation at: American Association for Cancer Research Annual Meeting; April 14-19, 2023; Orlando, FL.

Brimarafenib publications

• Wang J, Yao Z, Jonsson P, et al. A secondary mutation in BRAF confers resistance to RAF inhibition in a BRAF^V600E-mutant brain tumor. Cancer Discovery. 2018;8(9):1130-1141.
• Dankner M, Rose AAN, Rajkumar S, et al. Classifying BRAF alterations in cancer: new rational therapeutic strategies for actionable mutations. Oncogene. 2018;37(24):3183-3199.
• Yao Z, Torres NW, Tao A, et al. BRAF mutants evade ERK-dependent feedback by different mechanisms that determine their sensitivity to pharmacologic inhibition. Cancer Cell. 2015;28(3):370-383.

• Chen L, Milani de Marval P, Powell K, et al. SW-682: a novel TEAD inhibitor for the treatment of cancers bearing mutations in the Hippo signaling pathway. Poster presented at: American Association for Cancer Research Annual Meeting; April 14-19, 2023; Orlando, FL.
• Kamal A, Candi A, Versele M, et al. Novel antagonists of TEAD palmitoylation inhibit the growth of Hippo-altered cancers in preclinical models. Poster presented at: American Association for Cancer Research Annual Meeting; April 8-13, 2022; New Orleans, LA.