- Federica Di Nicolantonio (Torino, Italy)
- Sandra Demaria (New York, United States of America)
903O - A prospective study of a multi-cancer early detection blood test
- Deborah Schrag (New York, United States of America)
Abstract
Background
A validated blood-based multi-cancer early detection (MCED) test uses cfDNA and machine learning to detect a common cancer signal across >50 cancer types and predict cancer signal origin (CSO). PATHFINDER is a prospective study in a screening population that evaluated the clinical feasibility of MCED testing.
Methods
Participants were aged ≥50 y with or without additional cancer risk factors. Blood samples were collected, cfDNA analyzed, and MCED test results returned (MCED-E, early version). Cancer status was confirmed at 1 year for all participants. The primary outcome was the extent of diagnostic testing required for resolution after cancer signal detected by MCED-E. Those with cancer signal and confirmed cancer are true positive (TP), without confirmed cancer are false positive (FP). Diagnostic evaluation was at the discretion of the treating physician. Key secondary outcomes included test performance and safety. A pre-specified analysis evaluated a refined test version (MCED-Scr) using the participants’ banked specimens without return of results.
Results
The MCED-E test detected cancer signal in 1.4% (92/6621) of participants with analyzable samples. Cancer was confirmed in 38% (35/92). Specificity was 99.1% (6235/6290). See table for primary and secondary outcomes. A total of 73% (24/33) of TPs had diagnostic resolution in <3 months. MCED-Scr performance was similar with MCED-E (Table). Four AEs were reported (0.06%); none were due to confirmatory diagnostic procedures.
MCED-E TP FP Total n=35 n=57b N=92 Extent of diagnostic testing (Primary) >1 Imaging test, % 90.9 93.0 92.2 >1 Invasive procedure, % 81.8 29.8 48.9 Time to resolution, median days (IQR) 57 (33, 143) 162 (44, 248) 79 (37, 219) Test performance (Secondary) PPV 35/92 38.0 (28.8, 48.3) NPV 6235/6321 98.6 (98.3, 98.9) CSO Prediction accuracy 33/34c 97.1 (85.1, 99.8) MCED-Scr PPV 25/58 43.1 (31.2, 55.9) NPV 6216/6311 98.5 (98.2, 98.8) CSO Prediction accuracy 22/25 88.0 (70.0, 95.8) aExcludes 2 TPs who had evaluation started prior to test results. bIncludes 1 participant without resolution who is conservatively assumed to be FP. c1 CSO was indeterminate.
Conclusions
MCED testing was feasible in outpatient practice without significant AEs and with a PPV of approximately 40%. Studies to refine multi-cancer screening techniques are ongoing.
Clinical trial identification
NCT04241796.
Editorial acknowledgement
We acknowledge Jennifer Hepker, PhD and Merrilee Johnstone, PhD (Prescott Medical Communications Group, Chicago, IL) and Neva West, PhD (NeuroWest Solutions, Seattle, WA) for medical writing, editorial, and administrative support that was funded by GRAIL LLC a subsidiary of Illumina Inc. currently held separate from Illumina Inc. under the terms of the Interim Measures Order of the European Commission dated 29 October 2021.
Legal entity responsible for the study
GRAIL LLC a subsidiary of Illumina Inc. currently held separate from Illumina Inc. under the terms of the Interim Measures Order of the European Commission dated 29 October 2021.
Funding
GRAIL LLC a subsidiary of Illumina Inc. currently held separate from Illumina Inc. under the terms of the Interim Measures Order of the European Commission dated 29 October 2021.
Disclosure
D. Schrag: Non-Financial Interests, Institutional, Advisory Role: GRAIL, LLC, a subsidiary of Illumina, Inc., Journal of the American Medical Association; Financial Interests, Institutional, Research Grant: GRAIL, LLC, a subsidiary of Illumina, Inc., Dana-Farber Cancer Institute; Financial Interests, Institutional, Speaker's Bureau: Pfizer. C.H. McDonnell III: Financial Interests, Institutional, Stocks/Shares: Sutter Medical Group. L. Nadauld: Financial Interests, Personal, Stocks/Shares: CitizenCorporation, Clarifi, Guidance Genomics. E.A. Klein: Non-Financial Interests, Institutional, Advisory Role: GRAIL, LLC, a subsidiary of Illumina, Inc., Genome Health; Financial Interests, Institutional, Research Grant: GenomeDx Biosciences. K.C. Chung: Financial Interests, Institutional, Full or part-time Employment: GRAIL, LLC, a subsidiary of Illumina, Inc. M. Lopatin: Financial Interests, Institutional, Full or part-time Employment: GRAIL, LLC, a subsidiary of Illumina, Inc. E.T. Fung: Financial Interests, Institutional, Full or part-time Employment: GRAIL, LLC, a subsidiary of Illumina, Inc. T.M. Beer: Financial Interests, Institutional, Advisory Role: GRAIL, LLC, a subsidiary of Illumina, Inc., AbbVie, Amgen, Astellas Pharma, AstraZeneca, Bayer, Constellation, Janssen, Myovant Sciences, Pfizer, Sanofi, Sapience Therapeutics, Bristol Meyers Squib, Clovis Oncology, Dantari Pharmaceuticals, GlaxoSmithKline, Novartis, Tolero; Financial Interests, Personal and Institutional, Stocks/Shares: Arvinas, Inc.; Financial Interests, Personal, Stocks/Shares: Salarius Pharmaceuticals, LLC; Other, Institutional, Research Grant, Grant paid to Institution: AllianceFoundation Trials, Astellas Pharma, Bayer, Boehringer Ingelheim, Corcept Therapeutics, Endocyte Inc., Exact Sciences Corp., Freenome, GRAIL, LLC, a subsidiary of Illumina, Inc., Harpoon Therapeutics, Janssen Research & Development, Medivation, Inc., Merck, Sotio, Theraclone Sciences/OncoResponse, Zenith Epigenetics. All other authors have declared no conflicts of interest.
1696O - Genomic profiling and molecular targeting of lung cancer brain metastases
- Haiying Cheng (Bronx, United States of America)
Abstract
Background
Approximately 40%-50% of cancer patients who develop brain metastases have lung cancer. There is very limited information on genetic signatures associated with lung cancer brain metastases and prior studies mostly included small cohorts of cases.
Methods
We analyzed a large collection of lung cancer cases (n = 47215 for all NSCLC; 29438 for lung adenocarcinoma LUAD) that underwent comprehensive genomic profiling (FoundationOne), and identified potential key genetic alterations involved in loco-regional lesions (Loco) vs extracranial metastases (EM) vs brain metastases. We then performed preclinical studies to determine their functional roles and downstream pathways.
Results
Compared to Loco, the top 3 most enriched genetic alterations were
Conclusions
Legal entity responsible for the study
The authors.
Funding
ACS, ALA, LCFA/IASLC.
Disclosure
H. Cheng: Financial Interests, Personal, Advisory Role: Janssen; Financial Interests, Personal and Institutional, Research Grant: AstraZeneca; Financial Interests, Institutional, Sponsor/Funding: Genentech. E.S. Sokol: Financial Interests, Personal, Full or part-time Employment, Employee: Foundation Medicine; Financial Interests, Personal, Stocks/Shares, Stocks and stock options: Roche. J.S. Ross: Financial Interests, Personal, Full or part-time Employment, Medical Director: Foundation Medicine; Financial Interests, Personal, Stocks/Shares: Roche Holdings, Tango Therapeutics, Celsius Therapeutics. G.M. Frampton: Financial Interests, Personal, Other, Employee: Foundation Medicine; Financial Interests, Personal, Other, stocks/shareholder: Roche. A. Verma: Financial Interests, Personal, Research Grant: Prelude, BMS, GSK, Incyte, Medpacto, Curis and Eli Lilly; Financial Interests, Personal, Advisory Role: Stelexis, Bakx, Novartis, Acceleron and Celgene, Stelexis and Janssen; Financial Interests, Personal, Other, holds equity: Stelexis, Bakx and Throws Exception. B. Halmos: Financial Interests, Personal, Advisory Role: AstraZeneca Boehringer Ingelheim Veracyte Janssen Takeda Merck BMS Genentech Pfizer Eli-Lilly; Financial Interests, Personal and Institutional, Research Grant: Boehringer Ingelheim, AstraZeneca, Merck, BMS, Advaxis, Amgen, AbbVie, Daiichi Sankyo, Pfizer, GSK, Beigene, Janssen. All other authors have declared no conflicts of interest.
66O - MyPathway: A multiple target, multiple basket study of targeted treatments in tissue-agnostic cohorts of patients (pts) with advanced solid tumors
- Claire F. Friedman (New York, United States of America)
Abstract
Background
Targeted agents have activity against indicated tumors, but data are limited in other tumor types with relevant alterations. MyPathway (NCT02091141) is a tissue-agnostic, non-randomized, phase IIa multiple basket trial assessing targeted agent activity for advanced solid tumors with matched alterations but no FDA indication. We previously reported objective response rates (ORR) from MyPathway in pts with HER2 amplification and/or overexpression (23.3%; pertuzumab + trastuzumab [P+H]), tumor mutational burden ≥16 mut/Mb (TMB-H; 38.1%; atezolizumab), and
Methods
Alterations were identified pre-enrollment in pts aged ≥18 years using routine assays. Primary endpoint was ORR. Safety was also assessed.
Results
MyPathway enrollment was between Apr 2014 and Jul 2020 (N=672). Pts had tumors with diverse alterations (e.g. amplifications, non-synonymous mutations) in HER2 (n=357),
Conclusions
Targeted therapy in a tumor-agnostic setting is active. Routine assays can identify therapies with meaningful activity for pts with limited treatment options and high unmet medical need. TEAE, treatment-emergent adverse eventan=27 treated with V and n=1 with V+C
Hh V600E V600E Non-V600E HER2 positive Mutation, no amplification ORR, n (%) 13 (48.1) 5 (35.7) 1 (3.6) 1 (7.7) 35 (36.8) 4 (6.6) 4 (10.8) Safety, n (%) n=55 (V); n=15 (V+C) n=13 n=95 n=61 n=37
Clinical trial identification
NCT02091141.
Editorial acknowledgement
Medical writing support provided by Ashfield MedComms, an Ashfield Health Company, and funded by F. Hoffmann-La Roche/Genentech.
Legal entity responsible for the study
F. Hoffmann-La Roche/Genentech.
Funding
F. Hoffmann-La Roche/Genentech.
Disclosure
C.F. Friedman: Non-Financial Interests, Advisory Board, Compensation waived: Merck, Genentech; Financial Interests, Other, Personal fees: Arch Oncology, Bristol Myers Squibb; Non-Financial Interests, Principal Investigator: Daiichi, Bristol Myers Squibb, Genentech/Roche, Merck, AstraZeneca. C. Swanton: Financial Interests, Research Grant: AstraZeneca, Boehringer Ingelheim, Bristol Myers Squibb, Pfizer, Roche-Ventana, Invitae (previously Archer Dx Inc - collaboration in minimal residual disease sequencing technologies), Ono Pharmaceutical; Financial Interests, Leadership Role, Chief Clinician: Cancer Research UK; Financial Interests, Principal Investigator, Chief Investigator for the MeRmaiD 1 and 2 clinical trials: AstraZeneca; Financial Interests, Principal Investigator, Chief Investigator of the NHS Galleri trial: National Health Service (NHS); Financial Interests, Advisory Role: Amgen, AstraZeneca, Pfizer, Novartis, GlaxoSmithKline, MSD, Bristol Myers Squibb, Illumina, Genentech, Roche-Ventana, GRAIL, Medicxi, Metabomed, Bicycle Therapeutics, Roche Innovation Centre Shanghai, the Sarah Cannon Research Institute; Financial Interests, Stocks/Shares: Epic Bioscience, Bicycle Therapeutics; Financial Interests, Stocks/Shares, stock and co-founder: Achilles Therapeutics. D.R. Spigel: Financial Interests, Advisory Role: Roche/Genentech, Novartis, Celgene, Bristol Myers Squibb, AstraZeneca, Pfizer, Boehringer Ingelheim, AbbVie, Foundation Medicine, GlaxoSmithKline, Lilly, Merck, Moderna Therapeutics, Nektar, Takeda, Amgen, TRM Oncology, Precision Oncology, Evelo Therapeut, Illumina, PharmaMar, G1 Therapeutics, Neon Therapeutics, Celldex, Clovis Oncology, Daiichi Sankyo, EMD Serono, Acerta Pharma, Oncogenex, Astellas Pharma, GRAIL, Transgene, Aeglea Biotherapeutics, Tesaro, Ipsen, ARMO BioSciences, Amgen, Millennium, Genzyme, Intuitive Surgical, Purdue Pharma, Spectrum Pharmaceuticals, Sysmex. R. Bose: Financial Interests, Other, Consultant: Roche/Genentech; Financial Interests, Advisory Role, Received honoraria: Roche/Genentech, Foundation Medicine, Novartis; Financial Interests, Research Grant, Research funding: Puma Biotechnology. H.A. Burris: Financial Interests, Full or part-time Employment: HCA Healthcare/Sarah Cannon; Financial Interests, Stocks/Shares: HCA Healthcare/Sarah Cannon; Financial Interests, Funding, Research funding: Bristol Myers Squibb, Incyte, AstraZeneca, MedImmune, Macrogenics, Novartis, Boehringer Ingelheim, Lilly, Seattle Genetics, Merck, Agios, Jounce Therapeutics, Moderna Therapeutics, GlaxoSmithKline, Verastem, Tesaro, BioMed Valley Disc, TG Therapeutics, Vertex, eFFECTOR Therapeutics, Janssen, BioAtla, CicloMed, Harpoon Therapeutics, Arch, Arvinas, Revolution Medicines, Array BioPharma, Bayer, Kymab, Pfizer, Takeda/Millennium, Foundation Medicine, EMD Serono, ARMO BioSciences, CALGB, Hengrui Therapeutics, XBiotech, Zymeworks, Coordination Pharmaceuticals, NGM Biopharmaceuticals, Gossamer Bio, Ryvu Therapeutics, BioTheryX, BeiGene, Roche/Genentech, AbbVie; Financial Interests, Other, Consultant (uncompensated): Bayer, GRAIL, Novartis, Vincerx Pharma, AstraZeneca, Bristol Myers Squibb, Boehringer Ingelheim, TH Therapeutics, Incyte. W. Yu: Financial Interests, Full or part-time Employment: Genentech Inc.; Financial Interests, Full or part-time Employment: Genentech; Financial Interests, Stocks/Shares: Roche. W. Yu: Full or part-time Employment: Genentech Inc. J. Malato: Financial Interests, Full or part-time Employment: Genentech; Financial Interests, Stocks/Shares: Roche. R. Price: Financial Interests, Full or part-time Employment: Genentech; Financial Interests, Stocks/Shares: Roche. W. Darbonne: Financial Interests, Full or part-time Employment: Genentech; Financial Interests, Stocks/Shares: Roche. T.M. Szado: Financial Interests, Full or part-time Employment: Genentech; Financial Interests, Stocks/Shares: F. Hoffmann-La Roche. K. Schulze: Financial Interests, Full or part-time Employment: Genentech; Financial Interests, Stocks/Shares: Roche. C.J. Sweeney: Financial Interests, Personal, Advisory Board, Consultancy: Genentech-Roche, Bayer, Astellas, Pfizer, Pfizer, Sanofi, Lilly; Financial Interests, Personal, Other, Consultancy: Janssen; Financial Interests, Personal, Stocks/Shares: Leuchemix; Financial Interests, Institutional, Research Grant: Bayer, Janssen, Astellas, Pfizer, Dendreon, Sanofi. J. Hainsworth: Financial Interests, Institutional, Funding, For conduct of MyPathway, paid to institution: Genentech. F. Meric-Bernstam: Financial Interests, Personal, Other, Consultant: AstraZeneca, DebioPharm, eFFECTOR Therapeutics, F. Hoffman-La Roche Ltd., Tyra Biosciences, Xencor, Zymeworks, OnCusp Therapeutics; Financial Interests, Personal, Advisory Board, Advisory Board/Consultant: Seagen; Financial Interests, Personal, Advisory Board: Silverback Therapeutics, Zentalis, Karyopharm, Biovica, Eisai; Financial Interests, Personal, Other, Consulting: Tallac Therapeutics, Lengo Therapeutics, Loxo-Oncology, Black Diamond, Infinity Pharmaceuticals, AbbVie; Financial Interests, Institutional, Other, Local PI / Research Grant: Aileron Therapeutics, Bayer Healthcare, CytomX Therapeutics Inc., Daiichi Sankyo Co. Ltd., eFFECTOR Therapeutics, Taiho Pharmaceutical Co.; Financial Interests, Institutional, Other, Local PI / Research Grant / Coordinating PI: AstraZeneca; Financial Interests, Institutional, Invited Speaker: Calithera Biosciences, Curis Inc., Debiopharm International, Guardant Health Inc., Klus Pharma, Novartis, PPD Investigator Services; Financial Interests, Institutional, Other, Local PI / Steering Committee Member: Genentech Inc.; Financial Interests, Institutional, Research Grant: Takeda Pharmaceutical Co., Puma Biotechnology Inc., Repare. R. Kurzrock: Financial Interests, Funding, Research funding: Biological Dynamics, Boehringer Ingelheim, Debiopharm, Foundation Medicine, Genentech, Grifols, Guardant, Incyte, Konica Minolta, MedImmune, Merck Serono, Omniseq, Pfizer, Sequenom, Takeda, TopAlliance; Financial Interests, Advisory Role, Consultant and/or speaker fees and/or served on an advisory board: Actuate Therapeutics, AstraZeneca, Bicara Therapeutics, Biological Dynamics, Eisai, EOM Pharmaceuticals, Iylon, Merck, NeoGenomics, Neomed, Pfizer, Prosperdtx, Roche, TD2/Volastra, Turning Point Therapeutics, X-Biotech; Financial Interests, Stocks/Shares: CureMatch Inc., CureMetrix, IDbyDNA; Financial Interests, Other, Board member: CureMatch, CureMetrix; Financial Interests, Other, Co-founder: CureMatch.
Invited Discussant 903O, 1696O and 66O
- Federica Di Nicolantonio (Torino, Italy)
Q&A
1660O - Dual immune checkpoint blockade induces analogous alterations in the intratumoral CD8+ T cell and Treg compartments
- Joleen J. Traets (Amsterdam, Netherlands)
Abstract
Background
Immune checkpoint blockade therapies have shown clinical activity in a range of human cancer types. While the capacity of T cells to recognize tumor antigens appears a critical driver of therapy response, the alterations in T cell state that are associated with response to immune checkpoint blockade are not well understood.
Methods
To dissect the effect of neoadjuvant PD-1 and CTLA4 blockade on the intratumoral T cell population in head and neck squamous cell carcinoma (HNSCC), we performed single cell RNA and TCR sequencing on the immune infiltrates of HNSCC samples derived from treatment-naïve patients that were enrolled in the IMCISION trial treated with neoadjuvant Nivolumab (anti-PD1, 3 mg/kg, weeks 1&3) and Ipilimumab (anti-CTLA4, 1mg/kg, week 1) prior to surgery (week 4). The intratumoral T cell population was analyzed in matched pre- and on-treatment tumor biopsies from 1 partial (PR) and 10 major pathological responding (MPR) patients and 7 non-responding patients. MPR was defined as ≤10% and PR was defined as ≤50% but >10% residual viable cancer cells in the surgical resection specimen.
Results
At baseline, a subset of 4-1BB+ regulatory CD4+ T cells (activated Tregs) was more abundant in responding than non-responding tumors. Furthermore, upon therapy, this activated Treg population showed a profound decrease in responding patients. In an analogous process, intratumoral dysfunctional T cells in the CD8+ T cell compartment transitioned to a state of reduced activity and reduced dysfunction upon therapy. Next to these concurrent changes in the activated Treg and dysfunctional CD8+ compartments, the abundance of a separate transitional CD8+ T cell population with low levels of dysfunction increased substantially upon therapy, presumably because of increased T cell entry.
Conclusions
Together, these data demonstrate that the presence of an activated Treg compartment at baseline may predict response to dual PD-1 and CTLA4 blockade in HNSCC, and that this combination therapy results in a parallel remodeling of both the intratumoral Treg and dysfunctional CD8+ T cell compartments.
Clinical trial identification
Vos et al. Nat Commun. 2021 Dec 22;12(1):7348, NCT04620200.
Legal entity responsible for the study
The Netherlands Cancer Institute (NKI) (Antoni van Leeuwenhoek), Charlotte L. zuur.
Funding
Bristol Myers Squibb (BMS).
Disclosure
C.L. Zuur: Financial Interests, Institutional, Funding: Bristol Myers Squibb (BMS). All other authors have declared no conflicts of interest.
67O - Quantifying poly(ADP-Ribose) polymerase (PARP1) in vivo using a non-invasive PARP1 PET tracer
- Lilie L. Lin (Houston, United States of America)
Abstract
Background
Robust non-invasive biomarker assays are needed to improve patient selection and pharmacodynamic assessments for PARP inhibitors as the currently accepted biomarkers (e.g. BRCA1/2 mutations) results in variability in response and benefit. [18F] Fluorthanatrace (FTT) is a positron emission tomography (PET) tracer that has been previously demonstrated to measure in vivo PARP1 expression in breast and ovarian cancers.
Methods
We conducted a single-arm open-label, non-randomized study of [18F]FTT PET as a method to non-invasively measure regional PARP1 expression in patients with primary or metastatic solid tumors with defects in germline or somatic BRCA1/2 or other mutations along the DNA Damage Response (DDR) pathway. Patients were not previously on oncologic therapy for at least 4 weeks but who had either radiographic evidence of progressive disease or newly diagnosed solid tumors with measurable disease. Patients underwent PET imaging 90 minutes after injection of 5-10 mCi of [18F]FTT. Uptake within the primary disease and/or up-to 5 metastatic sites was quantified by measuring the maximum standardized uptake value (SUVmax). Kruskal-Wallis test was used to determine differences between groups.
Results
A total of 44 patients consented for this study and underwent [18F]FTT PET imaging between 2/2019 and 3/2022 with the following primary sites of disease: ovarian (11), breast (19), pancreas (3), prostate (5), bladder (1), endometrial (1), primary peritoneal (1), kidney carcinoma (1) as well as leiomyosarcoma (1) and melanoma (1). All patients had measurable SUV above background. The median SUVmax in index lesions was 5.7 (range 1.76-16.52) in patients with BRCA1/2 mutations vs median 5.6 (range 2.73-5.66) in patient with other DDR pathway defects (ATM, CHEK2, PALB). No difference in SUVmax was observed between patients with or without prior PARPi (median SUVmax 5.7 [range 2.36-10.41] vs median SUVmax 5.72 [range 1.76-16.52]) respectively, p=0.64.
Conclusions
Measurable [18F]FTT uptake is identified across multiple solid tumors imaged. The range of SUVmax overlapped between those with and without BRCA1/2m.
Clinical trial identification
NCT03604315.
Legal entity responsible for the study
University of Texas, MD Anderson Cancer Center.
Funding
National Cancer Institute.
Disclosure
L.L. Lin: Financial Interests, Institutional, Research Grant: Pfizer, AstraZeneca. T.A. Yap: Financial Interests, Personal, Other, Consultant: Almac, Aduro, AstraZeneca, Atrin, Axiom, Bayer, Bristol Myers Squibb, Clovis, Cybrexa, EMD Serono, Guidepoint, Ignyta, I-Mab, Jansen, Merck, Pfizer, Repare, Roche, Schrodinger, Varian, Zai Labs, AbbVie, Acrivon, Adagene, Amphista, Artios, Athena, Avoro, Baptist Health Systems, Beigene, Boxer, C4 Therapeutics, Calithera, Cancer Research UK, Diffusion, F-Star, Genmab, Glenmark, GLG, Globe Life Sciences, GSK, Idience, ImmuneSensor, Institut Gustave Roussy, Intellisphere, Kyn, MEI Pharma, Mereo, Natera, Nexys, Novocure, OHSU, OncoSec, Ono Pharma, Pegascy, PER, Piper-Sandler, Prolynx, resTORbio, Theragnostics, Versant, Vibliome, Xinthera, ZielBio; Financial Interests, Personal, Other, University of Texas MD Anderson Cancer Center, where I am Medical Director of the Institute for Applied Cancer Science, which has a commercial interest in DDR and other inhibitors (IACS30380/ART0380 was licensed to Artios): MD Anderson Cancer Center, Institute for Applied Cancer Sciences; Financial Interests, Personal, Stocks/Shares: Seagen; Financial Interests, Institutional, Other, Grant/Research support: Bayer, Cyteir, EMD Serono, GlaxoSmithKline, Karyopharm, Pfizer, Repare, Sanofi, Artios, AstraZeneca, Beigene, BioNTech, Blueprint, BMS, Clovis, Constellation, Eli Lilly, Haihe, Forbius, F-Star, Genentech, ImmuneSensor, Ionis, Ipsen, Jounce, KSQ, Kyowa, Merck, Mirati, Novartis, Ribon Therapeutics, Regeneron, Rubius; Financial Interests, Institutional: Scholar Rock. All other authors have declared no conflicts of interest.
Invited Discussant 1660O and 67O
- Sandra Demaria (New York, United States of America)
Q&A
- All Speakers (Lugano, Switzerland)