PharmaEngine Announces Phase 2 PEPCOL Study Presented at the 2015 ASCO GI of PEP02 (MM-398) in Combination with 5-FU/LV in Unresectable Metastatic Colorectal Cancer

TAIPEI, Taiwan

PharmaEngine, Inc. (TWO:4162) today announced that the data from the randomized non-comparative phase 2 PEPCOL study of PEP02 (MM-398, liposome irinotecan injection) in unresectable metastatic colorectal cancer (mCRC) were presented as a poster at the American Society of Clinical Oncology 2015 Gastrointestinal Cancers Symposium (ASCO GI) on January 17, 2015. This PEPCOL study was sponsored by Groupe Coopérateur Multidisciplinaire en Oncologie (GERCOR) and co-sponsored by PharmaEngine and PharmaEngine’s partner, Merrimack Pharmaceuticals, Inc. (NASDAQ: MACK).

The PEPCOL study evaluated the efficacy and safety of PEP02 (MM-398) in combination with 5-FU/LV (FUPEP regimen) or irinotecan plus 5-FU/LV (FOLFIRI regimens: FOLFIRI-1 or modified FOLFIRI-3) as a second-line therapy in patients with mCRC. The primary endpoint was the objective response rate (ORR). Fifty-five patients were randomized (FUPEP, n=28; FOLFIRI, n=27). In the intent to treat population, the ORR of the FUPEP regimen was 14% (4/28), which compared favorably with FOLFIRI-1 (0%, 0/10) and was comparable to the modified FOLFIRI-3 regimen (18%, 3/17). Most common grade 3-4 adverse events reported in the respective FUPEP and the FOLFIRI arms were neutropenia (11% vs. 30%) and diarrhea (21% vs. 33%), which were numerically lower in FUPEP arm than in FOLFIRI arm; other aspects of the safety profiles were similar between the two arms.

“FUPEP regimen, which can be combined with bevacizumab, exhibited promising tumor response as compared to standard FOLFIRI (the standard of care for first- or second-line treatment in mCRC patients), and better safety profile versus either FOLFIRI regimens for oxaliplatin-pretreated patients”, said Prof. Aimery de Gramont, Institut Hospitalier Franco-Britannique, Levallois-Perret, France. “We are grateful to GERCOR, the patients and the investigators in contributing to this important study. Based on the acceptable safety profile of the FUPEP regimen in this PEPCOL study, it was added as the third arm to the phase III metastatic pancreatic cancer (NAPOLI-1) study where this FUPEP regimen (MM-398 + 5-FU/LV arm) met the primary endpoint of a statistically significant improvement in overall survival”, said Grace Yeh, Ph.D., President and Chief Executive Officer, PharmaEngine, Inc.

PEPCOL Study Design

PEPCOL is a randomized non-comparative phase 2 study in patients who were previously treated with oxaliplatin-containing first-line regimen. Patients were randomly assigned to FUPEP (PEP02 80 mg/m² d1, folinic acid (FA) 400 mg/m² d1, 5-FU 2,400 mg/m² d1-2) or FOLFIRI (FOLFIRI-1: irinotecan 180 mg/m² d1, FA 400 mg/m² d1, 5-FU bolus 400 mg/m² d1, 5-FU infusion 2,400 mg/m² d1-2; or modified FOLFIRI-3: irinotecan 90 mg/m² d1 and 3, FA 400 mg/m² d1, 5-FU infusion 2,400 mg/m² d1-2). Bevacizumab q2w (5 mg/kg) was allowed in both arms as of June 2012. The primary endpoint was the objective response rate (ORR). The study was conducted at six sites in France.

About MM-398 (PEP02)

MM-398 (PEP02, liposome irinotecan injection or nal-IRI) is a novel, stable nanotherapeutic encapsulation of the marketed chemotherapy drug irinotecan. In May 2011, PharmaEngine and Merrimack executed an exclusive license agreement. Under the terms of the agreement, PharmaEngine granted back Merrimack the rights to develop, manufacture, and commercialize PEP02 (designated as MM-398 by Merrimack) in Asia and Europe, and retained the same rights in Taiwan. In September 2014, Merrimack licensed the rights to MM-398 outside of the US and Taiwan to Baxter International’s biopharmaceutical business (Baxter, NYSE:BAX). In 2011, MM-398 has separately received orphan drug designation from the US FDA and European Medicines Agency (EMA) for the treatment of pancreatic cancer. In addition, MM-398 received Fast Track Designation for post-gemcitabine metastatic pancreatic cancer from the US FDA in November 2014.

About PharmaEngine, Inc. (TWO: 4162)

PharmaEngine is a biopharmaceutical company established in Taipei, Taiwan in 2003. PharmaEngine focuses on the development of new drugs for the treatment of cancer and Asian prevalent diseases. PharmaEngine has three projects: one in pre-NDA stage (PEP02 / MM-398); one in pivotal trial, phase II/III registration trial (PEP503 / NBTXR3); and one in drug discovery (PEP06). For further information, please visit PharmaEngine’s website at http://www.pharmaengine.com.