On March 9, 2017 Epizyme, Inc. (NASDAQ:EPZM), a clinical-stage biopharmaceutical company creating novel epigenetic therapies, reported financial results for the fourth quarter and full year 2016 (Press release, Epizyme, MAR 9, 2017, View Source [SID1234518042]). In addition, the Company reviewed recent highlights and provided guidance on its clinical and research pipeline in 2017.

“2016 was a year of tremendous progress across all aspects of Epizyme, which set the stage for 2017 to be a transformational year for the company,” said Robert Bazemore, president and chief executive officer of Epizyme. “We have a broad clinical development program with tazemetostat in multiple tumor types and treatment settings. We plan to present data from both our ongoing, global Phase 2 studies in both molecularly defined solid tumors and non-Hodgkin lymphoma in June, and to initiate discussions with regulators to define registration pathways for tazemetostat. We are advancing our ongoing monotherapy and combination studies and plan to announce the next development candidate from our preclinical pipeline this year. As we look ahead, we are on our way to achieving our vision of rewriting cancer treatment through novel epigenetic medicines.”

Recent Achievements

Tazemetostat Combination with Prednisolone Initiated: Epizyme has initiated clinical investigation of tazemetostat in combination with prednisolone in relapsed/refractory patients with DLBCL, based on observed preclinical synergy of the agents. This combination regimen is being conducted as the sixth cohort to the ongoing Phase 2 NHL study. Epizyme also anticipates initiating a new combination study in patients with follicular lymphoma in 2017.
Enrollment Completed in Three of Five NHL Study Cohorts: Epizyme has completed enrollment in three of the five cohorts of its ongoing, global Phase 2 study of tazemetostat in patients with relapsed/refractory non-Hodgkin lymphoma (NHL): the two cohorts enrolling patients with diffuse large B-cell lymphoma (DLBCL) with wild-type EZH2 and a third enrolling patients with follicular lymphoma with wild-type EZH2.
Follicular Lymphoma Enrollment Initiated in U.S.: In January 2017, following a positive written response from the U.S. Food and Drug Administration (FDA) to the company’s written request, Epizyme opened enrollment to patients with follicular lymphoma in the United States as part of the Company’s Phase 2 study in NHL.
Fast Track Designation Granted for DLBCL with EZH2 Mutations: In December 2016, the U.S. FDA granted Fast Track designation to tazemetostat for the treatment of patients with DLBCL whose tumors carry an EZH2 activating mutation. DLBCL is the most common form of NHL. The Fast Track designation provides expedited processes that may reduce development time and costs associated with bringing a drug to market.
Epithelioid Sarcoma Cohort Expanded in Phase 2 Solid Tumor Study: In late 2016, Epizyme expanded the epithelioid sarcoma cohort of its ongoing Phase 2 trial of tazemetostat in adult patients with certain molecularly defined solid tumors, following review by the Independent Data Monitoring Committee. This expansion was based on encouraging early activity, including confirmed objective responses, and surpassing the pre-specified futility hurdle in the cohort. This cohort will enroll up to 60 patients, or double the initial cohort size of 30 patients.
Synovial Sarcoma Cohort Completed Enrollment in Phase 2 Solid Tumor Study: The synovial sarcoma cohort of the Phase 2 trial in patients with molecularly defined solid tumors was fully enrolled in November 2016. This arm surpassed its pre-specified futility hurdle; however, the Company concluded that the activity was insufficient to continue further investigation of tazemetostat as a monotherapy. Epizyme is focusing its efforts on the remaining four cohorts of INI1-negative tumors.
Key 2017 Milestones and Goals

Interim Phase 2 Data Presentations Anticipated in the Second Quarter: Epizyme plans to report efficacy, safety and biomarker data from all five arms of the ongoing Phase 2 study in NHL, and from the study cohorts that have reached their futility analysis in the ongoing Phase 2 study in molecularly defined solid tumors. Pending abstract approval, the Company plans to present interim solid tumor data at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. Pending abstract submission and approval, the Company anticipates presenting interim data from all five arms of the ongoing Phase 2 NHL trial at The International Conference on Malignant Lymphoma (ICML).
Preparing for Regulatory Engagement beginning Mid-Year: Epizyme is preparing for regulatory engagement around its Phase 2 studies, beginning with the United States Food and Drug Administration (FDA) in mid-2017 on its molecularly defined solid tumor program. The Company is also preparing for FDA engagement on its NHL program in the second half of the year. These interactions are intended to determine registration paths for tazemetostat in each disease area.
Combination Studies Progressing: The combination studies of tazemetostat in front-line DLBCL with R-CHOP and in relapsed/refractory DLBCL with atezolizumab (Tecentriq) are continuing to enroll patients globally. Epizyme expects the optimal dose of tazemetostat in these combinations will be established this year, and for enrollment to continue to evaluate the safety and efficacy profile of both combinations.
Mesothelioma Study Progressing: Epizyme is continuing to enroll patients in its global Phase 2 study of tazemetostat in patients with mesothelioma characterized by BAP1 loss-of-function. The Company anticipates completing enrollment in this trial in 2017.
Next Development Candidate to be Named: Epizyme is progressing a pipeline of next-generation small molecules against novel epigenetic targets, and plans to name the next epigenetic development candidate from its pipeline in 2017. This is part of the Company’s goal of bringing three new product candidates into the clinic by 2020.
Fourth Quarter and Full-Year 2016 Financial Results

Cash Position: Cash, cash equivalents and marketable securities were $242.2 million as of December 31, 2016, as compared to $208.3 million as of December 31, 2015.
Revenue: Collaboration revenue was $0.5 million for the fourth quarter of 2016 and $8.0 million for the full year ended December 31, 2016, compared to $0.6 million for the fourth quarter of 2015 and $2.6 million for the full year ended December 31, 2015. The year-over-year increase was driven predominantly by the achievement of a $6.0 million milestone under the Company’s agreement with GSK during 2016.
R&D Expenses: Research and development (R&D) expenses were $28.4 million for the fourth quarter of 2016 and $91.5 million for the full year ended December 31, 2016, compared to $16.8 million for the fourth quarter of 2015 and $111.2 million for the full year ended December 31, 2015. R&D spending on discovery research programs and tazemetostat clinical development increased in both the quarter ended December 31, 2016 and, excluding the impact of the $40.0 million upfront payment to Eisai in the first quarter of 2015 to acquire worldwide rights, excluding Japan, to tazemetostat, in the year ended December 31, 2016.
G&A Expenses: General and administrative (G&A) expenses were $7.6 million for the fourth quarter of 2016 and $28.4 million for the full year ended December 31, 2016, compared to $6.0 million for the fourth quarter of 2015 and $23.9 million for the full year ended December 31, 2015. The increase was primarily due to higher compensation-related expenses associated with expansion of the senior leadership team in the first half of 2016 to support the Company’s operational growth, as well as increased investment in business development activities and tazemetostat-related pre-commercial activities.
Net Loss: Net loss was $35.0 million for the fourth quarter of 2016 and $110.2 million for the full year ended December 31, 2016, compared to $22.2 million for the fourth quarter of 2015 and $132.4 million for the full year ended December 31, 2015.
2017 Guidance
Epizyme believes that its cash, cash equivalents and marketable securities of $242.2 million as of December 31, 2016 will be sufficient to fund the Company’s planned operations into at least the third quarter of 2018.