Onxeo announces positive DSMB recommendation to continue Livatag® ReLive study in HCC

PARIS & COPENHAGEN, Denmark--(BUSINESS WIRE (http://www.businesswire.com/))--
Regulatory News:

Onxeo S.A. (Paris:ONXEO) (NASDAQ OMX:ONXEO) (Euronext Paris, Nasdaq Copenhagen:
ONXEO), an innovative company specializing in the development of orphan oncology
therapeutics, today announced that it has received a unanimous recommendation
from the Data Safety Monitoring Board (DSMB), the independent European board of
experts that monitors the safety of the Livatag® Phase III trial, “ReLive”, to
continue the study without modification.

As per study protocol, the DSMB meets twice a year to review the safety data of
the ReLive trial and subsequently issues recommendations on the conduct of the
study.

The positive DSMB recommendation granted for the eighth time further confirms
the acceptable safety profile of Livatag®.

ReLive is an ongoing international, randomized Phase III trial designed to
evaluate the efficacy of intravenous (IV) administration of Livatag® in patients
with advanced hepatocellular carcinoma (HCC, primary liver cancer) after failure
or intolerance to sorafenib. The study plans to enroll a total of 400 patients
across approximately 90 sites. To date, more than 65% of the patients have been
randomized in the study.

The DSMB reviews the safety data of the treated patients in the study, totaling
more than 600 infusions of Livatag®.

“This eigth positive recommendation from our independent board of experts once
again confirms Livatag®’s safety profile, based on data collected from a growing
number of patients. We remain very confident in the potential of this innovative
product based on a novel nanoparticle formulation, allowing to overcome tumor
resistance to traditional chemotherapy. It could represent a significant
breakthrough in the treatment of hepatocellular carcinoma, a very severe cancer
with a high unmet medical need,” commented Judith Greciet, CEO of Onxeo.

About hepatocellular carcinoma

Hepatocellular carcinoma (HCC) or hepatocarcinoma is the most common of the
primary liver cancers (85% to 90%). According to Globocan (2012 data), liver
cancer is the sixth most common cancer in terms of incidence (782,000 new cases
worldwide each year, 5.6% of all new cancer cases) with the second highest
mortality rate (746,000 deaths, 9.1% of the total) after lung cancer. The risk
factors are well known: infection by hepatitis viruses (B and C),
overconsumption of alcohol (another major cause of cirrhosis) and metabolic
diseases, especially obesity, a growing cause of cirrhosis and HCC.

About ReLive

ReLive is an international Phase III trial designed to assess Livatag®’s
efficacy on survival in 400 patients with advanced hepatocellular carcinoma
(HCC) following treatment after failure or intolerance to Sorafenib. The trial
is ongoing in 11 countries (Europe, US, MENA region). The recruitment rate is in
line with expected timelines of issuing preliminary outcomes of the Phase III
study by mid 2017.

About Livatag® (doxorubicin Transdrug™)

Livatag® (Doxorubicin Transdrug™) is a doxorubicin formulation in the form of
lyophilized nanoparticles of polyisohexylcyanoacrylate (PIHCA).This new
therapeutic approach allows drug resistance to be avoided by short-circuiting
the mechanisms of multi-drug resistance developed by tumor cells through the
masking of the anticancer agent. Acting as a ‘Trojan horse,’ the nanoparticle
formulation avoids rejection of doxorubicin outside the cell so that it can
exert its cytotoxic action. By specifically targeting tumor cells in the liver
and overcoming resistance to doxorubicin, Livatag® represents a significant
breakthrough in the treatment of this cancer. The first indication of this
product is hepatocellular carcinoma; the sixth most widespread cancer in the
world and the second cause of cancer-related death.Livatag® is also being
evaluated within a comprehensive preclinical evaluation program, to explore
potential combinations with immuno-oncology agents (such as checkpoint
inhibitors), cytotoxic agents and targeted therapies and expand product
potential, with a primary focus on solid tumors

About Onxeo

Onxeo is a leading developer of orphan oncology drugs. The Company is focused on
developing innovative therapeutics for rare cancers, one of the fastest growing
markets in the healthcare industry with high, unmet medical needs. Onxeo’s
comprehensive portfolio features a broad orphan oncology pipeline, with three
independent programs in advanced clinical development, including Onxeo’s first
approved orphan oncology drug, Beleodaq®. In addition, Onxeo has successfully
developed and registered two non-cancer products which are currently being
commercialized in the U.S. and Europe. Onxeo’s vision is to become a global
leader and pioneer in oncology, with a focus on orphan or rare cancers, by
developing advanced, effective, and safe therapeutics designed to improve the
lives of patients. The Company is headquartered in Paris, France and has
approximately 50 employees. Onxeo is listed on Euronext in Paris, France
(Ticker: ONXEO, ISIN Code: FR0010095596) and Nasdaq Copenhagen, Denmark (Ticker:
ONXEO).

Onxeo orphan oncology products at the advanced development stage are:

  · Livatag® (Doxorubicin Transdrug™): Currently being evaluated in a Phase III
trial (ReLive) in patients with hepatocellular carcinoma (primary liver cancer);
and in combination with other cancer agents in first-line HCC
  · Beleodaq® (belinostat): FDA-approved in the U.S. in 2014 under the agency’s
accelerated approval program as a second-line treatment for patients with
peripheral T-cell lymphoma (PTCL) and currently marketed by Onxeo’s partner in
the U.S., Spectrum Pharmaceuticals; belinostat in combination with other cancer
agents is currently in development in first-line treatment for patients with
PTCL (BelCHOP) and in other solid tumors
  · AsiDNA : the first-in-class siDNA (signal interfering DNA) which has
successfully undergone a proof-of-concept Phase 1/2a trial in metastatic
melanoma
  · Validive® (Clonidine Lauriad®): Positive final results from a Phase II trial
in head and neck cancer patients with severe oral mucositis;

Learn more by
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This communication expressly or implicitly contains certain forward-looking
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actual results, financial condition, performance or achievements of Onxeo to be
materially different from any future results, performance or achievements
expressed or implied by such forward-looking statements. Onxeo is providing this
communication as of this date and does not undertake to update any forward
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events or otherwise. For a discussion of risks and uncertainties which could
cause actual results, financial condition, performance or achievements of Onxeo
to differ from those contained in the forward-looking statements, please refer
to the Risk Factors ("Facteurs de Risque") section of the 2014 Reference
Document filed with the AMF on April 14, 2015, which is available on the AMF
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Investor Relations, Onxeo
Nathalie Delair-Trepo, + 33 1 45 58 76 00
investors@onxeo.com
or
Alize RP (France)
Caroline Carmagnol / Florence Portejoie, +33 6 64 18 99 59 / +33 6 47 38 90 04
onxeo@alizerp.com
or
The Ruth Group (U.S.)
Kirsten Thomas / Lee Roth, +1-508-280-6592 / +1-646-536-7012
kthomas@theruthgroup.com / lroth@theruthgroup.com