Poxel and Sumitomo Dainippon Pharma Announce Positive Top-Line Results for Imeglimin Phase 3 Trial (TIMES 1) in Japan for the Treatment of Type 2 Diabetes

• Imeglimin Phase 3 trial (TIMES 1) met its primary and main
  secondary endpoints
• Imeglimin Phase 3 TIMES 3 16-week, double-blind, placebo
  controlled, randomized part of the trial is expected to report data
  mid-year and TIMES 2 and full results from the TIMES 3 trials are
  anticipated to report top-line results around the end of 2019
• Imeglimin Japanese New Drug Application (JNDA) targeted for 2020
• The Japanese diabetes market is fast-growing and anticipated to
  reach approximately $6B by 2020¹

LYON, France & OSAKA, Japan–(BUSINESS WIRE)–POXEL
SA (Euronext – POXEL – FR0012432516), a biopharmaceutical company
focused on the development of innovative treatments for metabolic
disorders, including type 2 diabetes and non-alcoholic steatohepatitis
(NASH) and Sumitomo Dainippon Pharma Co., Ltd (Head Office: Osaka,
Japan; Representative Director, President and CEO: Hiroshi Nomura;
Securities Code: 4506, First Section of TSE), announced today positive
top-line Phase 3 data results for the Imeglimin TIMES 1 trial for the
treatment of type 2 diabetes in Japan. Referred to as TIMES (Trials
of IMeglimin for Efficacy and Safety),
the Imeglimin Phase 3 program in Japan includes three pivotal trials to
evaluate Imeglimin’s efficacy and safety in over 1,100 patients.

“I am very excited to contribute to the development of a new and
innovative potential treatment option for Japanese patients with type 2
diabetes,” said Professor Kohjiro Ueki, MD, PhD, Director, Diabetes
Research Center, the National Center for Global Health and Medicine,
Tokyo, Japan. “Imeglimin’s safety profile combined with its unique
mechanism of action that targets very important deficiencies occurring
in diabetes, namely beta-cell function, as well as insulin resistance,
could be helpful for Japanese patients to manage their disease.”

The TIMES 1 randomized, double-blind, placebo-controlled monotherapy
trial orally administered 1,000 mg of Imeglimin twice-daily versus
placebo for 24 weeks in 213 Japanese patients. The TIMES 1 trial
demonstrated robust efficacy and achieved statistical significance
(p<0.0001) for its primary endpoint, defined as a change of glycated
hemoglobin A1c (HbA1c) versus placebo at week 24, with an HbA1c
placebo-corrected mean change from baseline of -0.87%.

For the trial’s main secondary endpoint of a decrease from baseline in
fasting plasma glucose (FPG), Imeglimin achieved statistical
significance (p<0.0001) versus placebo at week 24, with a FPG
placebo-corrected mean change from baseline of -19 mg/dL. Analyses of
data for the additional secondary endpoints are ongoing. In this trial,
the overall safety and tolerability of Imeglimin was similar to placebo
and the adverse event profile was consistent with what was observed in
the Phase 2b trial in Japan and the U.S. and European Phase 1 and 2
programs.

“This is a significant milestone for Poxel and for the development of
our most advanced drug candidate. The TIMES 1 results confirm the robust
efficacy combined with favorable safety observed in the Phase 2b trial
in Japan and the potential benefits that Imeglimin can bring to type 2
diabetes patients globally,” said Thomas Kuhn, CEO of Poxel. “The TIMES
1 data is the first major step towards filing the Japanese New Drug
Application in 2020. Japan represents the second largest single market
for type 2 diabetes and, Asia, in broader terms, is considered the most
important geographic location with regards to treating the diabetes
pandemic in the future.”

The TIMES program is a joint development effort between Poxel and
Sumitomo Dainippon Pharma. The companies entered into a strategic
partnership in October 2017 for the development and commercialization of
Imeglimin in Japan, China, South Korea, Taiwan and nine other Southeast
and East Asian countries.²

“Our commitment to diabetes patients is to continue to innovate and
provide new therapeutic options to help them manage their disease. We
are very pleased with the TIMES 1 data results and to be working closely
with Poxel on the TIMES clinical trials,” said Nobuhiko Tamura, Senior
Executive Officer; Drug Development Division of Sumitomo Dainippon
Pharma. “Diabetes is a significant area for our company in Japan, and we
believe that Imeglimin will be a very important addition to our existing
diabetes franchise.”

Poxel anticipates presenting data results from the Phase 3 (TIMES 1)
trial at an upcoming scientific meeting.

Poxel will host a conference call to discuss the results later today. To
access the information please click this link
or refer to Poxel’s website.

About the TIMES Program
TIMES (Trials of Imeglimin
for Efficacy and Safety), the Phase 3 program
for Imeglimin for the treatment of type 2 diabetes in Japan, consists of
three pivotal trials involving over 1,100 patients. The TIMES program
includes the following three trials that will be performed using the
dose of 1,000 mg twice daily:

TIMES 1: A Phase 3, 24-week, double-blind placebo-controlled,
randomized, monotherapy trial to assess the efficacy, safety and
tolerability of Imeglimin in Japanese patients with type 2 diabetes,
using the change in HbA1c as the primary endpoint. Secondary endpoints
of the trial include fasting plasma glucose, other standard glycemic and
non-glycemic parameters.

TIMES 2: A Phase 3, 52-week, open-label, parallel-group trial to
assess the long-term safety and efficacy of Imeglimin in Japanese
patients with type 2 diabetes. In this trial, Imeglimin will be
administrated orally as a monotherapy or combination therapy with
existing hypoglycemic agents, including a DPP4 inhibitor, SGLT2
inhibitor, biguanide, sulphonylurea and GLP1 receptor agonist.

TIMES 3: A Phase 3, 16-week, double-blind, placebo-controlled,
randomized trial with a 36-week open-label extension period to evaluate
the efficacy and safety of Imeglimin in combination with insulin in
Japanese patients with type 2 diabetes and inadequate glycemic control
on insulin therapy.

About Imeglimin
Imeglimin is the first clinical candidate in
a new chemical class of oral agents called Glimins by the World Health
Organization. Imeglimin has a unique mechanism of action (“MOA”) that
targets mitochondrial bioenergetics. Imeglimin acts on all three key
organs which play an important role in the treatment of type 2 diabetes:
the liver, muscles and the pancreas, and it has demonstrated glucose
lowering benefits by increasing insulin secretion in response to
glucose, improving insulin sensitivity and suppressing gluconeogenesis.
This MOA has the potential to prevent endothelial and diastolic
dysfunction, which can provide protective effects on micro- and
macro-vascular defects induced by diabetes. It also has the potential
for protective effect on beta-cell survival and function. This unique
MOA offers the potential opportunity for Imeglimin to be a candidate for
the treatment of type 2 diabetes in almost all stages of the current
anti-diabetic treatment paradigm, including monotherapy or as an add-on
to other glucose-lowering therapies.

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 and
non-alcoholic steatohepatitis (NASH). We have successfully completed the
Phase 2 clinical program for our first-in-class lead product, Imeglimin,
which targets mitochondrial dysfunction, in the U.S., Europe and Japan.
Together, with our partner Sumitomo Dainippon Pharma, we are conducting
the Phase 3 Trials of IMeglimin for Efficacy and Safety (TIMES) program
for the treatment of type 2 diabetes in Japan. Our partner Roivant
Sciences is responsible for Imeglimin’s development and
commercialization in countries outside of Poxel’s partnership with
Sumitomo Dainippon Pharma, including the U.S. and Europe. PXL770, a
first in class direct adenosine monophosphate-activated protein kinase
(AMPK) activator, is in a Phase 2a proof-of-concept program for the
treatment of NASH. PXL770 could also have the potential to treat
additional metabolic diseases. PXL065 (deuterium-stabilized
R-pioglitazone), a mitochondrial pyruvate carrier (MPC) inhibitor, is in
Phase 1 and being developed for the treatment of NASH. Poxel also has
additional earlier-stage programs, including deuterated drug candidates
for metabolic, specialty and rare diseases. We intend to generate
further growth through strategic partnerships and pipeline development.
(Euronext: POXEL, www.poxelpharma.com)

About Sumitomo Dainippon Pharma
Sumitomo Dainippon Pharma
defines its corporate mission as “to broadly contribute to society
through value creation based on innovative research and development
activities for the betterment of healthcare and fuller lives of people
worldwide”. By pouring all our efforts into the research and development
of new drugs, we aim to provide innovative and effective pharmaceutical
solutions to people not only in Japan but also around the world in order
to realize our corporate mission. Sumitomo Dainippon Pharma aims to
create innovative pharmaceutical products in the Psychiatry & Neurology
area, the Oncology area and Regenerative Medicine & Cell Therapy, which
have been designated as the focus research areas. Sumitomo Dainippon
Pharma has also positioned Psychiatry & Neurology, Diabetes and
Specialty as our focus marketing areas in Japan. For more detail, please
visit our website. (URL: https://www.ds-pharma.com)

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Company’s control that could cause the Company’s actual results or
performance to be materially different from the expected results or
performance expressed or implied by such forward-looking statements.

¹Source: Oppenheimer & Co. estimates.
²including:
Indonesia, Vietnam, Thailand, Malaysia, The Philippines, Singapore,
Republic of the Union of Myanmar, Kingdom of Cambodia and Lao People’s
Democratic Republic.

Contacts

Poxel SA
Jonae R. Barnes
Senior Vice President,
Investor Relations and Public Relations
[email protected]
+1 617 818
2985

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