Onxeo Initiates Comprehensive Strategy to Extend Value of Key Orphan Oncology Assets

  · Currently exploring combinations for synergistic effect to identify new
indications for Livatag® and Beleodaq®
  · Signed two collaborations with Lyon’s University Hospital and Synovo’s
immuno-oncology specialists
PARIS & COPENHAGEN--(BUSINESS WIRE (http://www.businesswire.com/))-- Regulatory
News:

Onxeo S.A. (Euronext Paris, NASDAQ Copenhagen:ONXEO), an innovative company
specializing in the development of orphan oncology drugs, announced the
initiation of a comprehensive strategy to explore further potential indications
for Livatag® and Beleodaq® to enhance the value of each therapy. As part of this
plan, the Company has signed two collaborations, one with the Croix-Rousse
Hospital, Hepato-Oncology Team and the Centre de Recherche en Cancérologie,
Inserm U1052, in Lyon, France and a second with the specialized contract
research organization (CRO) Synovo GmbH based in Tubingen, Germany. Both
collaborations will complement Onxeo’s in-house research efforts already
initiated.

This first step of Onxeo’s strategic plan aims to explore the efficacy of
Livatag® (doxorubicin TransdrugTM) and Beleodaq® (belinostat), in novel
combination therapies with classical cytotoxics, targeted therapies and the new
generation of immuno-oncology agents, to identify potential new indications for
the two Onxeo products.

Livatag® is currently being evaluated in the ReLive study, a global Phase 3
trial for the treatment of advanced hepatocellular carcinoma (HCC), the primary
form of liver cancer, for patients failing sorafenib therapy. The objective of
Onxeo’s strategy is to next assess the synergistic effect of Livatag® in
combination with other drugs, including sorafenib, the current standard of care
in first-line HCC.

As a pan-histone deactylase (HDAC) inhibitor, belinostat’s mechanism of action
affects many cellular processes crucial in the development of cancer. This makes
it an ideal combination partner in a range of cancers, notably solid tumors.

The collaboration with the Research Department at Croix-Rousse Hospital and
Centre de Recherche en Cancérologie de Lyon, led by Professor Philippe Merle,
M.D., Ph.D., the principal investigator of the ReLive study and an
internationally-recognized expert in HCC, will explore the potential synergies
of the two Onxeo products with currently-approved products and those in
development for HCC. Following in-vitro testing, promising synergistic
combinations will be tested in in-vivo models (animal models).

In addition, Onxeo has initiated a collaboration with Synovo, which specializes
in immuno-oncology testing, to explore the potential of belinostat and Livatag®
in a range of cancers in association with emerging immune-oncology agents, such
as promising PD-1 and CTLA-4 checkpoint inhibitors currently in development.

A first set of preclinical data will be obtained in early 2016 and will be used
to inform decisions for future steps and clinical options to further develop
these two products. Clinical development of selected indications could then be
initiated over the next one to two years.

Prof. Merle, M.D., Ph.D., principal investigator of the ReLive Phase 3 study of
Livatag®, commented:“Current treatment options are limited for systemic therapy
of HCC, with only one product approved to date, sorafenib, and unfortunately
more than half of patients lose response to treatment within six months. Other
targeted therapies tested as second-line therapies after sorafenib failure or in
combination to sorafenib, did not show any significant impact on the outcome of
patients. There is a significant need for new therapeutic options in HCC. The
Phase 3 trial of Livatag® is currently ongoing, and its safety profile by IV
infusion has been confirmed by several DSMB decisions. We expect Livatag® to
show strong efficacy to improve the outcome of patients as a potential new
efficient tool for the treatment of HCC, alone or in combination with other
approaches.”

Judith Greciet, CEO of Onxeo, commented, “We are thrilled to be launching these
new preclinical programs with two leading research organizations in the field of
oncology. The decision to explore new avenues of treatment with Beleodaq® and
Livatag® was based on extensive research and high-quality preclinical and
clinical data generated by our in-house research team. We believe there is
strong potential for our products in combination with emerging therapies, which
will create value for our company and our shareholders, and further position
Onxeo as a clear leader in orphan oncology research and development.”

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.

About belinostat (Beleodaq®)

Belinostat is a novel pan-histone deacetylase (HDAC) inhibitor that has anti
-cancer activity associated with the inhibition of cell proliferation, the
induction of apoptosis (programmed cell death), the inhibition of angiogenesis
and the induction of cellular differentiation.

Belinostat is designated as an orphan drug in Europe and the United States. In
July 2014, belinostat (Beleodaq®) was granted accelerated approval in the U.S.
by the Food and Drug Administration (FDA) for the treatment of patients with
relapsed or refractory peripheral T-cell lymphoma (PTCL) in second-line
treatment after failure of standard chemotherapy. Approval was based on results
from the pivotal Phase 2 BELIEF study (O’Connor et al, JCO, 2015) of belinostat
in relapsed or refractory PTCL, which demonstrated durable clinical benefit
(objective response rate of 25.8%) and good tolerability. The initiation of a
Phase III trial in collaboration with Onxeo’s U.S. partner, Spectrum
Pharmaceuticals, Inc. is planned in 2016 to expand the indication from second to
first-line treatment of PTCL.

Beyond PTCL, belinostat’s clinical profile supports further development in new
and promising orphan oncology indications. Onxeo is currently reviewing
potential indications in order to define the optimal development plan for
belinostat.

About Onxeo

Onxeo has the vision to become a global leader and pioneer in oncology, with a
focus on orphan or rare cancers, through developing innovative therapeutic
alternatives designed to “make the difference”. The Onxeo team is determined to
develop innovative medicines that provide patients with hope and significantly
improve their lives.

Key orphan oncology products at the advanced development stage are:
Livatag® (Doxorubicin Transdrug™): Phase III in hepatocellular carcinoma
Validive® (Clonidine Lauriad®): Phase II in severe oral mucositis: Positive
final results
Beleodaq® (belinostat): Registered in the US in 2nd-line treatment of peripheral
T-cell lymphoma
For more information, visit the
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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 2014 Reference
Document filed with the AMF on April 14, 2015, which is available on the AMF
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Contacts

Nathalie Delair-Trepo
Investor Relations, Onxeo
investors@onxeo.com
+33 1 45 58 76 00
or
Caroline Carmagnol / Florence Portejoie – Alize RP (France)
onxeo@alizerp.com
+33 6 64 18 99 59 / +33 1 44 54 36 64
or
Kirsten Thomas / Lee Roth – The Ruth Group (U.S.)
kthomas@theruthgroup.com /lroth@theruthgroup.com
+1 508 280 6592 / +1 646 536 7012