Onxeo Announces Promising Results from Preclinical Study of Beleodaq® Combination with Checkpoint Inhibitors

Combination with checkpoint inhibitors decreased tumor growth rate and prolonged
survival in syngeneic HCC mouse model
PARIS & COPENHAGEN, Denmark--(BUSINESS WIRE (http://www.businesswire.com/))--
Regulatory News:

Onxeo S.A. (Paris:ONXEO) (NASDAQ OMX:ONXEO) (Euronext Paris, Nasdaq Copenhagen:
ONXEO), a biopharmaceutical company specializing in the development of
innovative drugs for the treatment of orphan diseases, in particular in
oncology, today announced promising results from preclinical studies evaluating
the potential of its already PTCL (peripheral T-cell lymphoma)-approved
Beleodaq® (belinostat), in combination with checkpoint inhibitors as a potential
treatment option for other tumor indications. The studies were conducted as the
second step of Onxeo’s ongoing preclinical development program in partnership
with leading European research institution the University of Navarra’s
University Clinic and Center for Applied Medical Research in Spain under the
leadership of Prof. Bruno Sangro and Dr. Pablo Sarobe.

The preclinical studies were performed in an immune-competent mouse syngeneic
HCC (hepatocellular carcinoma) model, in which responses to a combination of
Beleodaq® and checkpoint inhibitors were compared to responses from treatment
with checkpoint inhibitors alone. The studies demonstrated a complete cessation
of tumor growth in all mice (100%) treated with the combination of Beleodaq® and
checkpoint inhibitors. The tumor cessation effect continued for approximately
one week after the final dose of belinostat was administered. By comparison,
checkpoint inhibitors alone inhibited tumor growth in only about 30 percent of
mice which received the treatment for the same period.

In addition, mechanistic studies performed on the spleens of the mice
demonstrated the generation of an underlying immune response that correlated
with the observed therapeutic effect of the combination treatment, showing an
increase in production of interleukines (signaling proteins for regulating
immune responses) by activated T-cells and a decrease in the number of
regulatory T-cells, when compared to mice treated only with checkpoint
inhibitors.

Graham Dixon, PhD, Chief Scientific Officer of Onxeo, commented, “These data
represent promising findings for the continued evaluation of Beleodaq® in
cancers beyond peripheral T-cell lymphoma (PTCL) when used as a combination with
immuno-oncology agents in various tumor indications such as for example HCC.
Approximately 20 percent of cancer patients currently treated with checkpoint
inhibitors alone are responding and thus, if translated into the clinic, the
positive outcomes from the combination therapy would account for a significant
improvement in the number of patients exhibiting high treatment responses. As a
next step in our collaboration with Prof. Bruno Sangro and Dr. Pablo Sarobe, we
are conducting follow-up studies to fully characterize these preclinical
findings demonstrating the potential of Beleodaq® in combination with checkpoint
inhibitors in various tumor indications, and in particular, are evaluating the
immune response in the tumor microenvironment in order to improve the
translation of the response into human patients.”

About Onxeo
Onxeo is a biopharmaceutical company specializing in the development of
innovative drugs for the treatment of orphan diseases, in particular in
oncology, driven by high therapeutic demand in one of the fastest growing
segments of the pharmaceutical industry. Onxeo’s objective is to become a major
international player in the field of rare cancers. Its growth strategy is
founded on the development of innovative, effective, and safe drugs based on
breakthrough technologies that can make a real difference in the treatment of
orphan oncology diseases and considerably improve the quality of life of
patients affected by rare and aggressive cancers. 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
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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 2015 Reference
Document filed with the AMF on April 29, 2016, which is available on the AMF
website (http://www.amf
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Contacts

Onxeo S.A.
Judith Greciet, CEO
Nicolas Fellmann, CFO
investors@onxeo.com
+33 1 45 58 76 00
or
Alize RP (France)
Caroline Carmagnol / Florence Portejoie
onxeo@alizerp.com
+33 6 64 18 99 59 / +33 6 47 38 90 04
or
The Ruth Group (U.S.)
Kirsten Thomas / Lee Roth
kthomas@theruthgroup.com / lroth@theruthgroup.com
+1 508 280 6592 / +1 646 536 7012