Onxeo Announces Development of Beleodaq® Oral Formulation Opening New Opportunities for Its HDAC Inhibitor

The company has obtained the first set of positive results of PK study with new
oral formulation of Beleodaq®
PARIS & COPENHAGEN, Denmark--(BUSINESS WIRE (http://www.businesswire.com/))--
Regulatory News:

Onxeo S.A. (Paris:ONXEO) (NASDAQ OMX:ONXEO), an innovative company specializing
in the development of orphan oncology therapeutics, today announced the first
set of positive results from a preclinical pharmacokinetic (PK) study on the
bioavailability of an oral formulation of belinostat (Beleodaq®) and the next
steps in its development plan for the new formulation. Beleodaq® is a histone
deacetylase inhibitor (HDACi) currently approved in the US for the treatment of
patients with relapsed or refractory peripheral T-cell lymphoma (PTCL).

The current formulation of belinostat is a lyophilized formulation for
intravenous (IV) use. The aim of the company is to develop an oral formulation
of belinostat offering strong benefits for patients and physicians in terms of
compliance, as well as opening new opportunities for indications for which an
oral formulation of belinostat is relevant.

As a key step in the development plan, the company has performed a canine PK
bioavailability study to screen two enabling formulation technologies dedicated
to improving solubility and assessed two prototypes selected based on
dissolution properties, and physical and chemical stability.

Findings from this study showed a good level of bioavailability approaching the
theoretical maximum achievable as indicated by the performance of the oral
solution. Onxeo will then select the best of the two formulation technologies,
to pursue formulation development and obtain a suitable clinical prototype and,
in parallel, fine tune the optimal dosing regimen to then start clinical
development in selected indications.

Judith Greciet, CEO of Onxeo, commented: “The positive results that we have
obtained represent an important milestone towards developing a clinically-ready
oral formulation of belinostat, which will provide multiple benefits to patients
and physicians in terms of convenience, pain-free administration, and need for
assistance from medical staff, especially as the other available PTCL treatments
are injectable forms. Finally, it opens up a range of new opportunities in the
orphan oncology field and beyond, giving Onxeo the opportunity to extend
belinostat’s patent protection, thus reinforcing the interest to evaluate the
compound in combination with other drugs for new indications.”

Graham Dixon, PhD, Chief Scientific Officer of Onxeo, added: “The preclinical
study conducted using the oral formulation of belinostat provides solid proof of
achieving a good level of bioavailability and promising preliminary PK data.
These data strongly support our next steps to conduct preclinical in
vivo efficacy studies to confirm efficacy at achievable exposure levels and
clinical trials with oral belinostat. ”

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/IIa 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%
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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

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