Poxel : Corporate Profile September 2021

FACTSHEET

September 2021

KEY HIGHLIGHTS

Well diversified mid-to-late stage pipeline with first-in-class drug candidates focused on NASH and rare metabolic diseases

A highly experienced management team with extensive metabolic expertise

Key partnership with type-2- diabetes market leader in Japan for TWYMEEG® (Imeglimin) - approved and launched 2021

Two differentiated programs (PXL770 & PXL065) for NASH and ALD

Cash and cash equivalents were EUR 36.9 million (USD 43.9 million) as of June 30, 2021

CONTACTS

• Corporate headquarters in Lyon, France
• Poxel also has subsidiaries in the Boston, Massachusetts area and Tokyo, Japan

Thomas Kuhn

CEO and Co-founder

Anne Renevot

Chief Financial Officer

Elizabeth Woo

SVP, Investor Relations & Communication

[e-mail]

Aurélie Bozza

Investor Relations & Communication Director

[e-mail]

Catherine David

Investor Relations & Communication Manager

[e-mail]

NewCap

Investor Relations and Public Relations

[e-mail]

ABOUT POXEL

Poxel is a clinical stage biopharmaceutical company developing innovative treatments for chronic serious diseases with metabolic pathophysiology, including non-alcoholic steatohepatitis (NASH) and rare disorders. Poxel has clinical and earlier-stageprograms from its adenosine monophosphate-activatedprotein kinase (AMPK) activator and deuterated thiazolidinedione (TZD) platforms targeting chronic and rare metabolic diseases.

TWYMEEG® (Imeglimin), Poxel's first-in-class lead product that targets mitochondrial dysfunction, is approved and launched for the treatment of type 2 diabetes in Japan. TWYMEEG is Poxel's first product to reach commercialization and Japan is the first country in the world to approve Imeglimin. As part of the license agreement with Sumitomo Dainippon Pharma, Poxel received a milestone payment from Sumitomo Dainippon Pharma upon approval which was paid in July 2021. Additionally, Poxel expects to receive escalating double-digit royalties on net sales (based on Poxel's current forecast) and sales-based payments in accordance with sales goals. Poxel has a strategic partnership with Sumitomo Dainippon Pharma for Imeglimin in Japan, China, South Korea, Taiwan and nine other Southeast Asian countries.

For the treatment of NASH, PXL065 (deuterium-stabilizedR-pioglitazone) is in a streamlined Phase 2 trial (DESTINY-1).PXL770, a first-in-class direct AMPK activator, has successfully completed a Phase 2a proof-of-concept trial for the treatment of NASH, which met its objectives.

In the rare inherited metabolic disorder, X-linkedadrenoleukodystrophy (ALD), Poxel intends to initiate Phase 2a proof-of-concept (POC) studies with PXL065 and PXL770 in patients with adrenomyeloneuropathy (AMN), the most common form of ALD. ALD is a severe orphan neurometabolic disease with no approved therapies. ALD is the most common leukodystrophy with a prevalence similar to hemophilia - up to 1/10,000 individuals in the general population have ALD.

A WELL-DIVERSIFIED METABOLIC PIPELINE

PIPELINE PROGRAMS

NASH: PXL065, deuterium-stabilizedR-pioglitazone, is in a streamlined Phase 2 trial (DESTINY-1). Patient enrollment was completed in September 2021, with topline data anticipated approximately one year later. This Phase 2 trial in noncirrhotic biopsy-proven NASH patients will assess three doses of PXL065 compared to placebo in 123 patients over 36 weeks. The results of this trial will be used to help identify the dose or doses for a Phase 3 registration trial. Initiation of the NASH Phase 2b trial for PXL770, a first-in-class, oral direct AMPK activator, is postponed, pending results from the ongoing PXL065 Phase 2 trial in NASH and both Phase 2a POC biomarker studies in AMN.

Rare Metabolic Diseases: In ALD, Phase 2a clinical POC biomarker studies of PXL065 and PXL770 are planned to initiate in early 2022, with data expected by year end 2022. The initial focus will be on patients with AMN, the largest subtype of ALD. Two identical studies will enroll adult male AMN patients and assess the effect of PXL065 and PXL770 over 12 weeks of treatment on pharmacokinetics, safety, and efficacy using relevant biomarkers, including the impact on elevated very long-chain fatty acids (VLCFA), the hallmark plasma marker of disease.

Diabetes: Approved in Japan, TWYMEEG, is an oral treatment for type 2 diabetes with a unique mechanism of action targeting mitochondrial dysfunction, enabling it to simultaneously target the two key defects that cause diabetes - impaired pancreatic b-cell function and insulin resistance. It has an extensive and consistent data package with 25 clinical studies in over 2,500 subjects and has been shown to have robust efficacy both alone and in combination with other drugs currently on the market. Imeglimin has also been shown to be well tolerated and to have a safety profile similar to placebo.

www.poxel.com	© POXEL

FACTSHEET | September 2021

A STRATEGIC PARTNERSHIP FOR TWYMEEG

Poxel and Sumitomo Dainippon Pharma have a strategic partnership for the development and commercialization of TWYMEEG in Japan, China, South Korea, Taiwan and nine other Southeast Asian countries1.

On June 23, 2021, TWYMEEG was approved for the treatment of Type 2 Diabetes in Japan. Japan is the first country in the world to approve Imeglimin hydrochloride.

• The approval in Japan triggered EUR 13.5 million loan from IPF in June 2021 and a milestone payment of EUR 13.2 million (USD 15.8 million)2 from Sumitomo Dainippon Pharma received in July 2021
• Sumitomo Dainippon Pharma product launch in Japan in September 2021
• Sumitomo Dainippon Pharma #1 diabetes franchise in Japan; FY20 USD 890 million3
• As part of the license agreement with Sumitomo Dainippon Pharma, Poxel expects to receive escalating double-digit royalties on net sales (based on Poxel's current forecast) and sales-based payments of up to EUR200 million (USD230 million4)
• For the US and Europe, various options to advance Imeglimin are being considered, including partnering
• Phase 3 TIMES program successfully completed in Japan observed to show robust efficacy with favorable safety and tolerability profile

1. Including: Indonesia, Vietnam, Thailand, Malaysia, the Philippines, Singapore, Myanmar, Cambodia, and Laos.
2. Converted at the exchange rates at date of approval (June 23, 2021).
3. Sumitomo Dainippon Pharma fiscal year April-March.
4. Converted at the exchange rate at the date of the agreement (Oct 30, 2017).

UPCOMING MILESTONES

PREVALENCE

Diabetes

2021-2022

• TWYMEEG approved in June 2021 and launched in September 2021 in Japan
• Sales-basedpayments and escalating double-digit royalties on net sales (based on Poxel's current forecast)
• Poxel is considering various options to advance Imeglimin in the US & Europe

SHAREHOLDER TOOLS

NASH

Q3 2021

• Patient enrollment completed in

PXL065 Ph 2 (DESTINY-1)

Q3 2022

• Topline Results from PXL065 Ph 2 (DESTINY-1)

Rare Metabolic Diseases

Q1 2022

• Initiate Ph 2a biomarker Proof-of-

Concepts (POC) studies for PXL065 and PXL770 in AMN

Q4 2022

• Topline results from both Ph 2a POC studies in AMN

Prevalence of NASH

About 40 million people are affected by NASH in the US, France, Germany, Italy, Spain, the UK and Japan. (Decision Resources)

Prevalence of ALD

ALD, the most common leukodystrophy, has a prevalence similar to hemophilia - up to 1 in 10,000 individuals in the general population have ALD. (https://rarediseases.org)Adrenomyeloneuropathy (AMN) is the largest subtype of ALD.

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	POXEL ON THE STOCK EXCHANGE	SHAREHOLDER STRUCTURE***
	Market	Euronext Paris
	since February 2015
	Ticker	POXEL
	ISIN	FR0012432516
	Market cap.	EUR 175 million**
	Number of shares	28,670,358*
	Share price	EUR 6.11**
	52-week trading range	EUR 5.72 - EUR 7.85
	*as of July 31, 2021. **as of September 24, 2021 ***At the date of the presentation, based on the Company's knowledge.
www.poxel.com		© POXEL