• Ratan R, Kasper B, Schöffski P, et al. Nirogacestat Treatment in Adult Patients with Desmoid Tumors: Long-Term Efficacy and Safety From the Phase 3 DeFi Trial. Oral presentation given at the Connective Tissue Oncology Society Annual Meeting; November 13-16, 2024; San Diego, CA, USA.
• Vincenzi B, Bui NQ, Dileo P, et al. Efficacy of Nirogacestat in Patients with Desmoid Tumors and Poor Prognostic Factors: Patient-Reported Outcomes, Progression-Free Survival, and Objective Response Rate in the Phase 3 DeFi Trial. Oral presentation given at the Connective Tissue Oncology Society Annual Meeting; November 13-16, 2024; San Diego, CA, USA.
• Kasper B, Federman N, Reichardt P, et al. Descriptive Evaluation of Patients With Desmoid Tumor and Co-Occurring Somatic Mutations of CTNNB1 and APC in the Phase 3 DeFi Trial. Poster presented at the Connective Tissue Oncology Society Annual Meeting; November 13-16, 2024; San Diego, CA, USA.
• Lindsay SE, Madison C, Doshi D, et al. Desmoid Tumor Cohort Creation and Characteristics in the Veterans Affairs (VA) National Database. Poster presented at the Connective Tissue Oncology Society Annual Meeting; November 13-16, 2024; San Diego, CA, USA.
• Loggers ET, Chugh R, Hartner L, et al. Monitoring Ovarian Function in Oncology Trials: Results and Insights from the DeFi Phase 3 Trial of Nirogacestat in Desmoid Tumor. Rapid Oral Abstract presented at: American Society of Clinical Oncology Annual Meeting; May 31-June 4, 2024; Chicago, IL.
• Vincenzi B, Bui NQ, Dileo P, et al. Efficacy of Nirogacestat in Patients with Poor Prognostic Factors for Desmoid Tumors: Analyses from the Randomized Phase 3 DeFi Study. Poster presented at: American Society of Clinical Oncology Annual Meeting; May 31-June 4, 2024; Chicago, IL.
• Kasper B, Federman N Reichardt P, et al. Efficacy and Safety of Nirogacestat in Patients with Desmoid Tumor and Adenomatous Polyposis Coli (APC) Mutation: Phase 3 DeFi Analyses. Poster presented at: American Society of Clinical Oncology Annual Meeting; May 31-June 4, 2024; Chicago, IL.
• Stacchiotti S, Chawla S, Chugh R, et al. Impact of nirogacestat on patient-reported outcomes in adults with desmoid tumor with a best overall response of stable disease: Post hoc analysis from the DeFi study. Oral presentation given at the European Society for Medical Oncology Sarcoma and Rare Cancers Annual Congress; March 14–16; 2024; Lugano, Switzerland.
• Mercier K, Hernandez L, Lucas A, Bell T, Tumminello B, Zhou S. Impact of Desmoid Tumors on Patients’ Quality of Life. Desmoid Tumor Research Foundation Natural History Study. Poster presented at the European Society for Medical Oncology Sarcoma and Rare Cancers Annual Congress; March 14–16; 2024; Lugano, Switzerland.
• Mercier K, Hernandez L, Lucas A, Bell T, Oton AB, Zhou S. Treatment Landscape for Desmoid Tumors. Desmoid Tumor Research Foundation Natural History Study. Poster presented at the European Society for Medical Oncology Sarcoma and Rare Cancers Annual Congress; March 14–16; 2024; Lugano, Switzerland.
• Zhou S, Lucas A, Hernandez L, Bell T, Goble S, Mercier K. Characterizing the Patient Journey to Diagnosis of Desmoid Tumor: Desmoid Tumor Research Foundation Natural History Study. Poster presented at: Connective Tissue Oncology Society Annual Meeting; November 1-4, 2023; Dublin, Ireland.
• Kasper B, Gounder MM, Ratan R, et al. Impact of Nirogacestat on Functional Status in Patients with Desmoid Tumors: Results From the Phase 3 DeFi Study. Poster presented at: Connective Tissue Oncology Society Annual Meeting; November 1-4, 2023; Dublin, Ireland.
• 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

• Kasper B, Gounder M, Hernandez L, Baumgarten C, Ratan R. Capturing Patient Voice to Improve Outcomes That Matter to Patients with Desmoid Tumor. Cancer Manag Res. 2024 Jun 7;16:617-628. doi: 10.2147/CMAR.S362694. eCollection 2024.
• Loggers ET, Chugh R, Federman N, Hartner L, Riedel RF, Cho S, Hyslop D, Lim A, Oton AB, Oktay KH. Onset and resolution of ovarian toxicity with nirogacestat treatment in females with desmoid tumors: Updated safety analyses from the DeFi phase 3 study. Cancer. 2024 Aug 15;130(16):2812-2821. doi: 10.1002/cncr.35324.
• 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

• Shearer T, Comstock M, Williams RL, et al. Kinetics of nirogacestat-mediated increases in B-cell maturation antigen on plasma cells inform therapeutic combinations in multiple myeloma. Cancer Res Comm. 2024 Nov 11. Online ahead of print.
• 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.

• Babovic-Vuksanovic D, Hirbe AC, Moertel CL, et al. Health-related quality-of-life (HRQoL) in adults and children with neurofibromatosis type 1-associated plexiform neurofibroma (NF1-PN) treated with mirdametinib: Pivotal, phase 2b ReNeu trial. Oral presentation at 29^th Annual Meeting & Education Day of the Society for Neuro-Oncology; November 21-24, 2024; Houston, TX.
• Gershon T, Moertel CL, McNall-Knapp RY, et al. Pivotal, phase 2b ReNeu trial of mirdametinib in children and adults with neurofibromatosis type 1-associated plexiform neurofibroma (NF1-PN): A spotlight on patients achieving deep response. Poster presented at 29^th Annual Meeting & Education Day of the Society for Neuro-Oncology; November 21-24, 2024; Houston, TX.
• Hirbe AC, Anadkat MJ, Boull C, et al. Addressing skin adverse events (AEs) during mirdametinib treatment in patients with neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN): Guidance from a multidisciplinary group of experts on the management of MEK inhibitor-associated skin AEs. Poster presented at 29^th Annual Meeting & Education Day of the Society for Neuro-Oncology; November 21-24, 2024; Houston, TX.
• Klesse LJ, Bornhorst M, Meade J, et al. Evaluation of the dispersible tablet and capsule formulations of mirdametinib in children with neurofibromatosis type 1 (NF1)-associated plexiform neurofibroma (PN): ReNeu trial. Paper presented at: International Symposium on Pediatric Neuro-Oncology; June 29 to July 2, 2024; Philadelphia, PA.
• Moertel C, Hirbe A, Shuhaiber H, et al. for the ReNeu Study Group. ReNeu: A Pivotal Phase 2b Trial of Mirdametinib in Children and Adults with Neurofibromatosis Type 1 (NF1)-Associated Symptomatic Inoperable Plexiform Neurofibroma (PN). Platform oral presentation at Global NF Conference; June 20-25, 2024
• Babovic-Vuksanovic D, Hirbe A, Moertel C, et al. Patient-Reported Outcomes of Pain Severity and Pain Interference from ReNeu: Pivotal Phase 2b Trial of Mirdametinib in Adults and Children with Neurofibromatosis Type 1-Associated Plexiform Neurofibroma (NF1 PN). Platform oral presentation at Global NF Conference; June 20-25, 2024
• Moertel CL, Hirbe AC, Shuhaiber HH, et al. ReNeu: A Pivotal Phase 2b Trial of Mirdametinib in Adults and Children with Neurofibromatosis Type 1 (NF1)-Associated Symptomatic Inoperable Plexiform Neurofibroma (PN). Rapid Oral Abstract presented at: American Society of Clinical Oncology Annual Meeting; May 31-June 4, 2024; Chicago, IL.
• 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

• Moertel CL, Hirbe AC, Shuhaiber HH, et al. ReNeu: a pivotal, phase IIb trial of mirdametinib in adults and children with symptomatic neurofibromatosis type 1-associated plexiform neurofibroma. J Clin Oncol. 2024;[Epub ahead of print]:JCO.24.01034.
• 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.*

• Robinson GW, Vinitsky A, Bag AK, et al. Results from the Phase 1 and Phase 1 expansion cohorts of SJ901: A Phase 1/2 trial of single-agent mirdametinib (PD-0325901) in children, adolescents, and young adults with low-grade glioma. Oral presentation at 29^th Annual Meeting & Education Day of the Society for Neuro-Oncology; November 21-24, 2024; Houston, TX.
• Robinson GW, Vinitsky A, Bag AK, et al. Results from the phase 1 and phase 1 expansion cohorts of SJ901: A phase 1/2 trial of single-agent mirdametinib (PD-0325901) in children, adolescents, and young adults with low-grade glioma. Paper presented at: International Symposium on Pediatric Neuro-Oncology; June 29 to July 2, 2024; Philadelphia, PA.
• 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.

• Schirmer A, Chen L, Celentano L, et al. Rational combination of pan-TEAD inhibitor SW-682 and MEK inhibitor mirdametinib in head and neck squamous cell carcinomas leads to synergistic response. Poster presented at: EORTC-NCI-AACR 36th Symposium on Molecular Targets and Cancer Therapeutics; October 23 – 25, 2024; Barcelona, Spain.
• Chen L, Jové V, Miliani de Marval P, et al. TEAD inhibition by SW-682 potentiates activity of targeted therapies in NSCLC models. Poster presented at: EORTC-NCI-AACR 36th Symposium on Molecular Targets and Cancer Therapeutics; October 23 – 25, 2024; Barcelona, Spain.
• 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.

Preclinical publications

• Hillen H, Candi A, Venderhoydonck B, et al. A novel irreversible TEAD inhibitor, SWTX-143, blocks Hippo pathway transcriptional output and causes tumor regression in preclinical mesothelioma models. Mol Cancer Ther. 2023 Sep 23. doi: 10.1158/1535-7163.MCT-22-0681. Online ahead of print.