Verastem Oncology to Present Phase 2 PRIMO Study Data Evaluating Duvelisib in Relapsed or Refractory Peripheral T-Cell Lymphoma at the American Society of Hematology 2019 Annual Meeting

Results from the Dose Optimization Portion of the Study to be Presented

PRIMO Expansion Phase To Investigate Duvelisib 75mg Twice Daily for Two Cycles, Followed by 25mg Twice Daily

Four Additional Abstracts Selected for Presentation, Including Results from a Phase 1 Study Investigating Duvelisib in Combination with Venetoclax in Patients with Relapsed or Refractory CLL/SLL

BOSTON--(BUSINESS WIRE)-- Verastem, Inc. (Nasdaq:VSTM) (Verastem Oncology or the Company), a biopharmaceutical company focused on developing and commercializing medicines seeking to improve the survival and quality of life of cancer patients, today announced that five abstracts highlighting data for COPIKTRA™ (duvelisib) have been selected for presentation at the upcoming American Society of Hematology 2019 Annual Meeting taking place December 7-10, 2019, in Orlando.

“We are pleased to share additional data on duvelisib across multiple areas of research that continues to expand our understanding of its potential benefit and utility for patients with certain types of blood cancers,” said Brian Stuglik, Chief Executive Officer of Verastem Oncology. “The results of the dose optimization portion of the PRIMO study provide important guidance to support our ongoing evaluation of duvelisib for the treatment of relapsed or refractory PTCL.”

A key abstract at the meeting will feature clinical data from the dose optimization portion of the registration-directed Phase 2 PRIMO study evaluating duvelisib in patients with relapsed or refractory peripheral T-cell lymphoma (PTCL). This open-label, multicenter trial is currently ongoing and is expected to enroll approximately 120 patients. In the dose optimization portion of the study, patients were randomized to receive duvelisib 25mg twice daily with an option for dose escalation (cohort 1) or duvelisib 75mg twice daily continuously (cohort 2) until disease progression or unacceptable toxicity (cycle=28 days).

The primary endpoint of the dose optimization portion is investigator-assessed overall response rate (ORR). Based on the efficacy and safety data to be reported at the meeting, the investigators have elected to investigate duvelisib starting at 75mg twice daily for two cycles, followed by 25mg twice daily, during the dose expansion portion of the study which is currently ongoing. Updated data from those shown in the abstract will be presented at the meeting. Duvelisib is not approved for the treatment of PTCL.

“Identifying additional options for this aggressive type of T-cell lymphoma is critical. In the initial cohort of this trial, we identified that the dose of 75mg twice daily for two cycles helped to achieve more rapid tumor control in what are often aggressive diseases. We will assess if following the induction by 25mg twice daily dose we are able to maintain longer-term disease control and mitigate the potential for later onset toxicities in patients with relapsed or refractory PTCL,” said Steven Horwitz, MD, Memorial Sloan Kettering Cancer Center, and principal investigator of Phase 2 PRIMO study. “We look forward to sharing updated data from this ongoing study at ASH 2019.”

Other abstracts at the meeting include: Preliminary results from a Phase 1 study investigating duvelisib in combination with venetoclax in patients with relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL); an analysis of cytogenetic and molecular markers associated with improved outcomes in the Phase 3 DUO study; and a description of the [soon to be initiated] TEMPO study investigating an intermittent dosing regimen of duvelisib in patients with indolent non-Hodgkin lymphoma. Preclinical data highlighting the effectiveness of duvelisib in combination with venetoclax in Richter syndrome will also be presented.

Details for the ASH 2019 presentations are as follows:

Poster Presentations

Title: Dose Optimization of Duvelisib in Patients with Relapsed or Refractory Peripheral T-Cell Lymphoma from the Phase 2 PRIMO Trial: Selection of Regimen for the Dose-Expansion Phase Lead author: Steven Horwitz, Memorial Sloan Kettering Cancer Center Poster #: 1567 Session: 624. Hodgkin Lymphoma and T/NK Cell Lymphoma – Clinical Studies: Poster I Date and Time: Saturday, December 7, 2019; 5:30-7:30PM ET Location: Orange County Convention Center, Hall B

Title: A Phase I Study of Duvelisib and Venetoclax in Patients with Relapsed or Refractory CLL / SLL Lead author: Jennifer Crombie, Dana-Farber Cancer Institute Poster #: 1763 Session: 642. CLL: Therapy, excluding Transplantation: Poster I Date and Time: Saturday, December 7, 2019; 5:30-7:30 PM ET Location: Orange County Convention Center, Hall B

Title: Cytogenetic and Molecular Marker Associations to Outcomes with Duvelisib and Ofatumumab Treatment in Patients with Relapsed or Refractory CLL/SLL in the DUO Trial Lead author: Jennifer Brown, Dana-Farber Cancer Institute Poster #: 4312 Session: 642. CLL: Therapy, excluding Transplantation: Poster III Date and Time: Monday, December 9, 2019; 6:00-8:00 PM ET Location: Orange County Convention Center, Hall B

Title: The Dual PI3K-δ/γ Inhibitor Duvelisib in Combination with the Bcl-2 Inhibitor Venetoclax Shows Promising Responses in Richter Syndrome-PDX Models Lead author: Andrea Iannello, Università degli Studi di Torino Poster #: 2862 Session: 625. Lymphoma: Pre-Clinical—Chemotherapy and Biologic Agents: Poster II Date and Time: Sunday, December 8, 6:00-8:00 PM ET Location: Orange County Convention Center, Hall B

Publication Only Presentations

Title: Trial in Progress (TiP): A Phase 2, Randomized, Open-Label, 2-Arm Study Comparing 2 Intermittent Dosing Schedules of Duvelisib in Patients with Indolent Non-Hodgkin Lymphoma (iNHL) (TEMPO) Lead author: Reem Karmali, Lurie Cancer Center, Northwestern University Session: 623. Mantle cell, follicular, and other indolent B Cell Lymphoma – Clinical studies

PDF copies of these poster presentations will be available here after the meeting.

COPIKTRA is indicated for the treatment of adult patients with relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL)_after at least two prior therapies and in relapsed or refractory follicular lymphoma (FL) after at least two prior system therapies. Accelerated approved in FL was based on overall response rate and continued approval may be contingent upon confirmatory trials.

COPIKTRA includes a Boxed Warning for fatal and serious toxicities including infections, diarrhea or colitis, cutaneous reactions and pneumonitis. See full Prescribing Information for complete Boxed Warning and other important safety information.

SELECT IMPORTANT SAFETY INFORMATION

This does not include all information needed to use COPIKTRA™ (duvelisib) safely and effectively. See full Prescribing Information.