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Poxel Reports Results for the First Half 2017 and Provides Corporate Update

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Poxel will host an investor conference call today to discuss the First
Half 2017 results at 1 pm ET (7 pm CET). To participate in the call,
please use the dial-in numbers: US: +1 877-887-4163 FR: +33 (0)172001510
UK: +44 2030432440

Access Code: 11730055#

POXEL
SA
(Euronext – POXEL - FR0012432516), a biopharmaceutical company
focused on the development of innovative treatments for metabolic
disorders, including type 2 diabetes, announced today its results for
the first half of 2017 ended June 30, 2017, and provided a corporate
update. As of June 30, 2017, cash and cash equivalents were EUR €34.9
million (USD $39.8 million).

"I am pleased to report that we have continued to make significant
clinical and regulatory progress, expanded the depth of our management
team and added a new member to our Board of Directors from Japan during
the first half of 2017," said Thomas Kuhn, CEO of Poxel. "For our lead
program, Imeglimin, we announced robust efficacy results for the
299-patient Phase 2b study for the treatment of type 2 diabetes in
Japan. We also strengthened Imeglimin's profile and presented new
mechanistic data showing its benefit in beta cell protection and
detailed its unique insulin secretion pathway in response to glucose.
Promising data was also presented at this year's American Diabetes
Association meeting demonstrating Imeglimin's potential for
cardiovascular protective properties to treat diabetic cardiomyopathy, a
significant cardiovascular complication with limited treatment options."

"Japan is a key focus and an integral part of our business strategy. We
believe that Imeglimin's unique profile could be very attractive given
the specific needs of this important marketplace and the pathophysiology
of Japanese patients," said Thomas Kuhn, CEO of Poxel. "Due to
Imeglimin's safety and efficacy profile, it has the potential to be used
as first-line therapy for type 2 diabetes in Japan for treatment naïve
patients or in combination with other glucose lowering therapies, as
well as for the elderly and sensitive populations. We are looking
forward to initiating the Phase 3 program for Imeglimin in Japan during
the fourth quarter of this year."

"For our second program, PXL770, we recently initiated a Phase 1b
multiple ascending dose study. We believe that PXL770 could have the
potential to treat several chronic metabolic diseases, including those
that affect the liver as well as diabetes and diabetes-related
complications. We are currently evaluating potential indications for our
clinical proof-of-concept program, which we plan to initiate in 2018,"
continued Thomas Kuhn. "In addition, we are actively working to further
leverage our internal capabilities and are currently assessing
additional development opportunities in the metabolic area."

Poxel First Half 2017 Highlights
Imeglimin
In
preclinical and clinical studies, Imeglimin has demonstrated the
potential to address the mitochondrial dysfunction at the core of type 2
diabetes pathophysiology. Imeglimin is the first orally available drug
candidate that simultaneously targets all three key organs affected by
diabetes: the pancreas, the liver and the muscles. Imeglimin has
completed Phase 1 and Phase 2 development in over 1,200 subjects in the
U.S., EU and Japan. Over the course of the first half of 2017, Imeglimin
successfully completed several important milestones including:

Robust Efficacy Results from the Phase 2b study in
Japan

  • The Phase 2b randomized, double-blind, placebo-controlled study tested
    three doses of Imeglimin (500 mg, 1000 mg and 1500 mg) administered
    twice-daily for 24 weeks in 299 Japanese patients for the treatment of
    type 2 diabetes. The study achieved statistically significant results
    for its primary and secondary endpoints.
  • The primary endpoint achieved statistical significance (p<0.0001) for
    the change from baseline in glycated hemoglobin (HbA1c) versus placebo
    in all treatment groups at 24 weeks. Placebo-adjusted HbA1c reduction
    was 0.52%, 0.94% and 1.00% for the 500 mg, 1000 mg and 1500 mg dose
    twice-daily, respectively. Overall, the study showed that Imeglimin
    was safe and well tolerated and the adverse event profile was
    consistent to what was observed in the U.S. and EU Phase 1 and 2
    programs.

Additional Benefits Beyond Glucose Control

  • Data presented at the 77th American Diabetes Association
    (ADA) Scientific Session demonstrated protective effects for diabetic
    cardiomyopathy, which is a significant cardiovascular complication
    that affects approximately 40 percent of type 2 diabetes patients and
    is associated with an increase in morbidity and mortality.1
    The data demonstrated that Imeglimin may have the potential to reduce
    the burden of this prominent cardiovascular complication in type 2
    diabetes patients.
  • In March 2017, Imeglimin successfully completed a thorough QT/QTc
    (TQT) cardiac safety study in 55 healthy subjects and no evidence of
    QT prolongation was observed. This study assesses a drug's risk of QT
    prolongation and its proarrhythmic potential.
  • Data presented at the 9th Scientific Meeting of the Asian
    Association for the Study of Diabetes included mechanistic data
    showing Imeglimin's benefit on beta cell protection and its unique
    insulin secretion pathway in response to glucose. A comprehensive
    summary of the Phase 1 data in Japanese subjects was also presented
    demonstrating that Imeglimin was safe, well tolerated and exhibited a
    similar pharmacokinetic profile to what was observed in Caucasian
    subjects.

Regulatory Update

  • Recently, Poxel met with the Pharmaceuticals and Medical Devices
    Agency (PMDA) in Japan for the Imeglimin End of Phase 2 meeting to
    discuss its Phase 3 program plans and the data package required for a
    Japanese New Drug Application (JNDA) submission. Based on constructive
    interactions and feedback from the PMDA, Poxel plans to move forward
    with its Phase 3 program during the fourth quarter of 2017.
  • The Phase 3 program in Japan will consist of three pivotal studies,
    which include 1) a monotherapy study of double blind treatment versus
    placebo which is similar to the recently completed Phase 2b study, 2)
    a long-term safety study as a monotherapy and add-on to other oral
    therapies or GLP1 agonists, and 3) a long-term safety study as an
    add-on to insulin. These trials will be performed using the optimal
    dose of 1000 mg twice daily. To further differentiate Imeglimin's
    product profile, Poxel will also confirm Imeglimin's potential in
    sensitive patient populations, such as those with kidney-related
    complications due to type 2 diabetes.

PXL770
PXL770 is a
first-in-class direct adenosine monophosphate-activated protein kinase
(AMPK) activator. AMPK plays a key role as a master regulator of
cellular energy, which turns on pathways that replenish energy and turns
off pathways that consume energy, leading to the control of lipid
metabolism, glucose homeostasis and inflammation. Based on this central
metabolic role, targeting AMPK offers the opportunity to pursue a wide
range of indications to treat chronic metabolic diseases,2
including diseases that affect the liver, such as non-alcoholic
steatohepatisis (NASH), as well as type 2 diabetes and diabetes-related
complications, such as diabetic nephropathy.

  • A Phase 1b multiple ascending dose (MAD) trial for PXL770 is currently
    underway. The MAD trial will include up to 76 subjects and evaluate
    the safety, tolerability and pharmacokinetics of PXL770 in at least
    four dose groups. Completion of the MAD trial is anticipated in early
    2018.
  • Results from the first part of the single ascending dose study
    indicate that PXL770 exhibited a favorable safety and tolerability
    profile with no reported serious adverse events.

Corporate

  • Poxel expanded the depth of its management team during the first half
    of 2017 with the appointments of Anne Renevot as Chief Financial
    Officer and Christophe Arbet-Engels as Chief Medical Officer and
    Executive Vice President, Late Development & Medical Affairs. In
    addition, Kumi Sato was appointed to its Board of Directors. Ms. Sato
    will help support Poxel's corporate strategy for Imeglimin in Japan.
  • In June 2017, ENYO Pharma SA reported that the Phase 1a single and
    multiple ascending dose trial evaluating EYP001 in healthy subjects
    has been completed. The results show that EYP001 was safe and
    well-tolerated at all doses studied in 80 subjects. The first Phase 1
    study was designed to determine the safety, tolerability and
    pharmacokinetics of EYP001 in healthy subjects. Another ongoing Phase
    1 study will evaluate the safety, food effect and PK of EYP001 in
    subjects with chronic HBV infection. EYP001 is an FXR agonist licensed
    to ENYO Pharma by Poxel.

Financial Statements for First Half 2017 (IFRS standards)

Poxel devotes the bulk of its resources to research and development
activities. R&D expenses for the first half of 2017 totalled €6.3
million, mainly reflecting the clinical study costs incurred for the
Company's lead program, Imeglimin and its second compound, PXL770. The
€2.2 million decrease in R&D expenses as compared to the first half of
2016 was mainly driven by completion of the Phase 2b clinical study for
Imeglimin in Japan whose top line results were published in May 2017.
The R&D costs are net of the R&D Tax Credit (CIR) that resulted in
income of €1.6 million for the first half of 2017. The €0.5 million
decrease in general and administrative (G&A) costs was mainly driven by
non-recurrent 2016 costs related to financing activities. The financial
expenses for the first half of 2017 totalled € 0.2 million, mainly
reflecting foreign currency exchange loss. The net result for the
financial period ending June 30, 2017 was a net loss of €9.7 million, as
expected, compared to a net loss of €12.4 million in the corresponding
period in 2016. On June 30, 2017, cash and cash equivalents were €34.9
million compared to €45.6 million on December 31, 2016.

Condensed Income Statement In thousand €

     

30 June 2017

30 June 2016

Turnover - -
Net research and development expenses* (6 259) (8 470)
General and administrative expenses (3 249) (3 720)
Operating loss (9 508) (12 190)
Financial expenses/Financial income (180) (196)
Net loss (9 688) (12 386)
 

*Excludes R&D tax credit

Number of shares and voting rights as of June 30, 2017:

Month   Date  

Total number
of shares
outstanding

 

Total of
theoretical
voting
rights (1)

 

Total of exercisable
voting rights (2)

June 6/30/2017 23,034,228 23,034,228 23,005,807

(1) The total number of theoretical voting rights (or "gross" voting
rights) is used as the basis for calculating the crossing of
shareholding thresholds. In accordance with Article 223-11 of the AMF
General Regulation, this number is calculated on the basis of all shares
to which voting rights are attached, including treasury shares whose
voting rights have been suspended.
(2) The total number of
exercisable voting rights (or "net" voting rights) is calculated without
taking into account the treasury shares with suspended voting rights, in
this case, shares held by the Company in the context of a liquidity
contract agreement with ODDO.

Next financial press release: Q3-turnover and cash position
October 16, 2017.

About Imeglimin
Imeglimin is the first clinical candidate in
a new chemical class of oral agents called the Glimins. Imeglimin has a
unique mechanism of action (MOA) that targets mitochondrial
bioenergetics. Imeglimin acts on the three main target organs involved
in glucose homeostasis: the liver, muscle, and the pancreas. This MOA
has the potential for glucose lowering benefits, as well as the
potential to prevent endothelial and diastolic dysfunction, which can
provide protective effects on micro- and macro-vascular defects induced
by diabetes. The additional protective effect on beta-cell survival and
function may lead to a delay in disease progression. This unique mode of
action compared to existing treatments for type 2 diabetes makes
Imeglimin a prime candidate in all stages of the current anti-diabetic
treatment paradigm, including monotherapy or as an add-on to other
glucose lowering therapies for the treatment of patients with type 2
diabetes.

About PXL770
PXL770 is a first-in-class direct adenosine
monophosphate-activated protein kinase (AMPK) activator. AMPK plays a
key role as a master regulator of cellular energy, which turns on
pathways that replenish energy and turns off pathways that consume
energy, leading to the control of lipid metabolism, glucose homeostasis
and inflammation. Based on this central metabolic role, targeting AMPK
offers the opportunity to pursue a wide range of indications to treat
chronic metabolic diseases, including diseases that affect the liver,
such as non-alcoholic steatohepatisis (NASH), as well as type 2 diabetes
and diabetes-related complications, such as diabetic nephropathy.

About Poxel SA
Poxel uses its development expertise in
metabolism to advance a pipeline of drug candidates focused on the
treatment of metabolic disorders, including type 2 diabetes. We have
successfully completed our Phase 2 clinical program for our
first-in-class lead product, Imeglimin, which targets mitochondrial
dysfunction, in the U.S., EU and Japan. Our second program, PXL770, a
direct AMPK activator, is in Phase 1 development. We intend to generate
further growth through strategic partnerships and pipeline development.
(Euronext: POXEL, www.poxelpharma.com)

1 Fitchett et al. European Journal of Heart Failure (2017)
2
Source: Srivastava, R. A et al., (2012) Journal of Lipids Research 53,
2490- 2514

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