Onxeo Announces First Outcomes of Livatag® Preclinical Plan

· Livatag® Nanoformulation Leads to Increased Exposure and Preferential
Affinity for Liver, Supporting Current Relive Phase III Study Rationale
  · Demonstrates Enhanced Effect in Combination with Immunotherapy
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 data from 2 in vivo studies of the Livatag®
preclinical plan confirming that the nanoparticle formulation meets the
pharmacological requirements for a hepatocellular carcinoma (HCC) treatment and,
furthermore, that the combination of Livatag® with immunotherapy produces an
enhanced efficacy effect, validating Onxeo’s comprehensive strategy to explore
further potential indications for its key product candidate.

The studies were performed in an orthotopic (implantation of tumor cells into
the liver of mice) HCC model in immune-competent mice. Orthotopic models are
considered relevant to the clinical situation and a good predictor of drug
efficacy. Results demonstrate that Livatag® (doxorubicin TransdrugTM) generates
a 12-fold increase in exposure in the tumor tissue within the liver compared to
free doxorubicin, without increasing the drug’s exposure in the heart or other
vital organs. These findings complete, and go beyond, data from a mechanistic
study of Livatag® previously reported at the 2016 American Association for
Cancer Research (AACR) Annual Meeting that revealed that the distribution of
Livatag® nanoparticles in healthy liver was approximately six times higher
compared to free doxorubicin, which indicate that Livatag® nanoparticles have a
preferential affinity for and are able to target the liver and more
particularly, the liver tumor tissue.

As part of this program, Onxeo has also been exploring the potential of Livatag®
when administered with emerging immuno-oncology agents of various classes, such
as promising PD-1 and CTLA-4 checkpoint inhibitors currently in development. A
current study demonstrates that Livatag® produces an enhanced effect in tumor
response (reduction in tumor volume) when given in combination with immuno
-oncology agents in the orthotopic HCC model. Specifically, Livatag® co
-administration with antibodies is associated with a positive increase in
circulating T-cell populations, which is consistent with the observed reduction
in tumor volume.

Graham Dixon, PhD, Chief Scientific Officer at Onxeo, commented, “These
preclinical findings are important because they reinforce our previous research
showing that Livatag® Transdrug™ technology dramatically improves the drug’s
exposure in liver cancer tissue, demonstrating high affinity for the liver,
without increasing exposure in other organs. These data validate the interest of
Livatag® as a potential new therapeutic option for HCC treatment. Besides, we
are pleased to share the first data demonstrating an enhanced anti-cancer
response generated by Livatag® in combination with immuno-oncology agents in
murine HCC models. Results from this study will enable us to extend the value of
one of our lead assets and add to the growing value of Onxeo’s pipeline. In
addition to sharing results from the Phase III ReLive trial next year, we look
forward to continued evaluation of Livatag® in combination with immunotherapy
agents and delivering a plan to advance Livatag® in the clinic in additional
indications.”

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
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. Onxeo’s comprehensive portfolio
features a broad orphan oncology pipeline, with four independent programs in
various stages of clinical development, including Onxeo’s first approved orphan
oncology drug, Beleodaq®. 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’s orphan oncology products 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 US 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 US, 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 I 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

In addition, Onxeo has successfully developed and registered two non-cancer
products which are currently being commercialized in the U.S. and Europe.

Learn more by
visiting www.onxeo.com (http://cts.businesswire.com/ct/CT?id=smartlink&url=http%
3 
A%2F%2Fwww.onxeo.com&esheet=51417133&newsitemid=0&lan=en
-US&anchor=www.onxeo.com&index=1&md5=03c663d3c531d1a90d60a3b9b6c11876).

To receive our press releases and newsletters, please register
on: http://www.onxeo.com/en/newsletter/ (http://cts.businesswire.com/ct/CT?id=sm
a 
rtlink&url=http%3A%2F%2Fwww.onxeo.com%2Fen%2Fnewsletter%2F&esheet=51417133&newsi
t 
emid=0&lan=en
-US&anchor=http%3A%2F%2Fwww.onxeo.com%2Fen%2Fnewsletter%2F&index=2&md5=d82b933b2
a 
64f090f44b1e5b6017c6c2)
Follow us on Twitter: @Onxeo_

Disclaimer
This communication expressly or implicitly contains certain forward-looking
statements concerning Onxeo and its business. Such statements involve certain
known and unknown risks, uncertainties and other factors, which could cause the
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
-looking statements contained herein as a result of new information, future
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 2015 Reference
Document filed with the AMF on April 29, 2016, which is available on the AMF
website (http://www.amf
-france.org (http://cts.businesswire.com/ct/CT?id=smartlink&url=http%3A%2F%2Fwww
. 
amf-france.org&esheet=51417133&newsitemid=0&lan=en
-US&anchor=http%3A%2F%2Fwww.amf
-france.org&index=3&md5=3fc582666b7246d3b4e434926728866a)) or on the company’s
website
(www.onxeo.com (http://cts.businesswire.com/ct/CT?id=smartlink&url=http%3A%2F%2F
w 
ww.onxeo.com&esheet=51417133&newsitemid=0&lan=en
-US&anchor=www.onxeo.com&index=4&md5=cdd8637bcf7e55f5a1300b16d88cbac5)).
Onxeo S.A.
Judith Greciet, CEO
Nicolas Fellmann, CFO
+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