(P 268) INITIAL RESULTS OF PHASE 2/3 TRIAL OF AL102 FOR TREATMENT OF DESMOID TUMORS (DT)

Author(s)

Mrinal M. Gounder, MD

Associate Attending Physician
Memorial Sloan Kettering Cancer Center and Weill Cornell Medical Center
New York, New York, United States

Co-Author(s)

Robin Lewis L. Jones, MD

MEDICAL ONCOLOGIST
Royal Marsden Hospital and Institute of Cancer Research
London, England, United Kingdom
Rashmi Chugh, MD

Professor of Internal Medicine
University of Michigan
Ann Arbor, Michigan, United States
MA

Mark Agulnik, MD

Clinical Professor
City of Hope Comprehensive Cancer Center
Duarte, California, United States
Arun S. Singh, MD

Associate Professor
University of California at Los Angeles
Santa Monica, California, United States
Brian A. Van Tine, MD, PhD

Professor of Medicine
Washington University School of Medicine
St. Louis, Missouri, United States
Vladimir Andelkovic, MD (he/him/his)

Medical Oncologist
Princess Alexandra Hospital and Icon Cancer Centre,
Brisbane, Queensland, Australia
Edwin Choy, MD, PhD (he/him/his)

Physician / Associate Professor
Massachusetts General Hospital
Boston, Massachusetts, United States
Jeremy Lewin, MBBS

Medical Oncologist
Peter MacCallum Cancer Centre
Melbourne, Victoria, Australia
Ravin Ratan, MD, M.Ed.

Associate Professor
The University of Texas MD Anderson Cancer Center
Houston, Texas, United States
AB

Atrayee Basu Mallick, MD (she/her/hers)

associate Prof
Sidney Kimmel Cancer Center, Thomas Jefferson University
Philadelphia, Pennsylvania, United States
BB

Bruce Brockstein, N/A, MD

NorthShore University HealthSystem
Evanston, Illinois, United States
Nam Bui, MD

Clinical Assistant Professor
Stanford University
Palo Alto, California, United States
Sant P. Chawla, MD

Medical Director
Sarcoma Oncology Research Center
Santa Monica, California, United States
SH

Shadi Haddadin, N/A, MD

Jefferson City Medical Group
Jefferson City, Missouri, United States
HK

Hyo Song Kim, N/A, MD, PhD

Yonsei University College of Medicine
Seoul, Kyonggi-do, Republic of Korea
Alexander Lee, MD, PhD (he/him/his)

Consultant Medical Oncologist
The Christie Hospital
Manchester, England, United Kingdom
Javier Martín-Broto, MD, PhD

Medical Oncologist
Fundacíon Jiménez Díaz University Hospital, ATBSARC lab in General Hospital of Villalba, IIS-FJD, Madrid, Spain
Madrid, Madrid, Spain
CR

Christopher W. Ryan, MD

Oregon Health and Science University Knight Cancer Institute, Portland, OR, USA
Portland, Oregon, United States
Gary K. Schwartz, MD

Professor of Medicine
Columbia University Herbert Irving Comprehensive Cancer Center
New York, New York, United States
Winette T.A van der Graaf, MD, PhD

Professor of Medical Oncology at NKI and ErasmusMC; Head of Department of Medical Oncology at NKI; Groupleader AYA and Sarcoma Research at NKI
Netherlands Cancer Institute (NKI), Amsterdam, The Netherlands
Amsterdam, Noord-Holland, Netherlands
JK

Jason B. Kaplan, MD

Medical Director
Ayala Pharmaceuticals
Northbrook, Illinois, United States
Jonathan Y. Yovell, MD, MBA (he/him/his)

VP Clinical Development
Ayala Pharmaceuticals
Rehovot, HaMerkaz, Israel
Gary Gordon, MD, PhD

Chief Medical Officer
Ayala Pharmaceuticals
Highland Park, Illinois, United States
Bernd Kasper, MD, PhD

Associate Professor
Mannheim University Medical Center
Mannheim, Baden-Wurttemberg, Germany

Objective: AL102 is a potent, orally available, selective gamma secretase inhibitor (GSI) under investigation as an antineoplastic agent. GSIs have antitumor activity against DT, including AL101 in a Phase 1 trial and Expanded Access Program (2 confirmed PRs >3.5 years and >1 year; 1 stable disease for 1 year). There remains an unmet need for effective systemic therapy in DT, associated with durable tolerability, tumor regression, and symptom improvement. RINGSIDE is a Phase 2/3 randomized study in patients with progressing DT. Part A (Phase 2) is an open-label part with safety as the primary endpoint.

Methods: Patients are randomized to three dosing regimens: 1.2 mg QD, 2 mg intermittent BIW (2 days on 5 days off), or 4 mg intermittent BIW. Part B is a Phase 3 double-blind, placebo-controlled study with PFS as primary endpoint. The dose for part B will be determined based on the totality of data collected in part A.

Results: Safety, efficacy, PRO, PK, and biomarker results from Part A of the study will be presented at the meeting.

Conclusion: Conclusions of Part A of the study will be presented at the meeting.