PharmaEngine Announces Initiation of Global Pivotal Trial of PEP503 (NBTXR3) in Soft Tissue Sarcoma in Asia Pacific Region

Taipei, Taiwan, May 31, 2016

PharmaEngine’s (TWO: 4162) announced announced today that the first patient has been dosed in a global pivotal phase II/III trial of PEP503 (NBTXR3) in soft tissue sarcoma (STS) at Perpetual Succour Hospital, Cebu, Philippines.

This multi-national, randomized, open-label, two-arm pivotal phase II/III trial, referred to as Study 301 (Act.in.sarc study), is being conducted in partnership with PharmaEngine’s partner, Nanobiotix S.A. (Euronext: NANO). PharmaEngine is the co-sponsor of this Study 301 in the Asia-Pacific Region. The primary objective of Study 301 is to enhance the pathological complete response rate (pCR) by dosing PEP503 through intra-tumor injection and then activated by external beam radiation therapy (EBRT). The efficacy of PEP503 combined with radiotherapy in Study 301 will be compared with that of radiotherapy alone.

The total patient enrollment of Study 301 is around 180 patients to be recruited in Europe and South Africa by Nanobiotix and in the Asia-Pacific region (Australia, Hong Kong, and Philippines) by PharmaEngine. The first patient in this global study was enrolled and dosed in Europe in the first quarter of 2015. The patient enrollment is actively ongoing now. The Global Principal Investigator is Prof. Sylvia Bonvalot, MD, PhD (Institut Curie, Paris, France). An interim analysis will be performed once two-thirds of patients have been recruited to ensure the safety of all patients enrolled in the study, the quality of the data collected and the continued scientific validity of the study design.. For detailed information, please visit www.clinicaltrials.gov. (Identifier: NCT02379845).

“We are pleased to have the first patient enrolled in the Asia-Pacific region as part of the global pivotal STS study.“

“The promising results from Nanobiotix’s phase I/II clinical trials in STS and head & neck cancer encouraged us to accelerate the global clinical development and speed up the regulatory registration process as a medical device.

C. Grace Yeh, Ph.D., President and CEO of PharmaEngine

The first patient receiving the first dose of the pivotal study is one of the development milestones in the Licensing and Collaboration Agreement between PharmaEngine and Nanobiotix. PharmaEngine will pay a milestone payment of US$ 1 million to Nanobiotix.

ABOUT PEP503 (NBTXR3)

NBTXR3, the lead project of the NanoXray pipeline of Nanobiotix, is a nanoparticle formulation of hafnium oxide crystals for the local treatment of tumors to enhance the efficacy of radiotherapy. In August 2012, PharmaEngine licensed the development and commercialization rights of NBTXR3 in the Asia-Pacific region from Nanobiotix.In 2014, Nanobiotix presented the pilot study results for NBTXR3 in patients with soft tissue sarcomas at ASCO, which showed a good safety profile and positive signs of efficacy. Then the preliminary pilot studyresults in head and neck cancerpatients (without receiving chemotherapy) and liver cancer patients presented at ASCO 2017and ASCO GI 2018,respectively, also showedpromisingsigns of anti-tumoreffect.

There are other indications being developed for NBTXR3 by Nanobiotix and PharmaEngine. These includehead and neck cancer in patients receiving chemotherapy (Phase I/II, PharmaEngine), rectal cancer (Phase I/II, PharmaEngine), liver cancer (Phase I/II, Nanobiotix)andprostate cancer (Phase I/II, Nanobiotix). NBTXR3 has been classified as a class III medical device in many European and certain Asian countries.

ABOUT PHARMAENGINE

PharmaEngine, Inc. is a biopharmaceutical company headquartered in Taipei, Taiwan with a wholly owned subsidiary, PharmaEngine Europe Sarl in Paris, France. PharmaEngine focuses on the development of new medications for the treatment of cancer and Asian prevalent diseases. PharmaEngine has three ongoing projects: ONIVYDE® has received regulatory approvals in 37 countries; PEP503 (NBTXR3) is in a global pivotal trial of soft tissue sarcoma, and patient recruitment has been completed; and PEP06 is in preclinical development. Get to know more here.

Contact

Chihsing Chang, Vice President, Finance and Administration
P +886 2 2515 8228, ext. 700
chihsing.chang@pharmaengine.com