France - 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), today announced the presentation of clinical data from the company's Phase 1b study of PXL065, which established a dose-proportional pharmacokinetic profile with a substantially altered ratio of R- and S-pioglitazone stereoisomers as predicted from preclinical and Phase 1a results.

Additionally, several preclinical studies supporting the efficacy of PXL770 in NASH and other metabolic diseases were presented. The data were illustrated in four poster presentations at The Liver Meeting Digital Experience 2020, held virtually from November 13-16, 2020, in association with the American Association for the Study of Liver Diseases (AASLD). The posters can be accessed on Poxel's website using the following link: https://www.poxelpharma.com/en_us/product-pipeline/posters.

'We are excited to present data that showcases the potential of our oral, first-in-class clinical candidates for NASH, PXL065 and PXL770, and which are complementary to the results observed to-date from both programs,' said Thomas Kuhn, Chief Executive Officer of Poxel. 'Further analysis from our Phase 1b clinical study highlights dose-equivalence for 15 mg of PXL065 vs. 45 mg of Actos. This observation is particularly meaningful as it suggests that our product candidate could have an improved therapeutic profile compared to Actos, with equivalent or greater efficacy, while reducing or eliminating undesirable PPAR-related side effects, including weight gain.'

'Additionally, as displayed across three PXL770 poster presentations, the preclinical data reveal the potential for direct effects on key components of NASH and demonstrate its potential as both a mono- and combination therapy,' continued Mr. Kuhn. 'On the heels of positive, topline Phase 2a results, these additional data support the continued development of PXL770. We look forward to providing future updates on further analysis from the Phase 2a study and the Phase 2b trial design in biopsy-proven NASH patients.'

PXL065, the novel, proprietary deuterium-stabilized R-pioglitazone stereoisomer is currently being evaluated in DESTINY-I, a Phase 2 study in biopsy-proven NASH patients, which seeks to identify the optimal dose or doses for a Phase 3 registration trial. PXL770, a first-in-class direct adenosine monophosphate-activated protein kinase (AMPK) activator, will be advancing into a Phase 2b study for the treatment of noncirrhotic, biopsy-proven NASH. STAMP-NAFLD, the company's Phase 2a proof-of-concept trial in nonalcoholic fatty liver disease (NAFLD) patients, recently met its primary endpoint and study objectives, demonstrating that PXL770 was observed to be safe, well-tolerated and that it achieved a statistically significant improvement in the relative decrease in liver fat mass as measured by MRI-PDFF at 12 weeks. Additionally, across several clinical parameters and preclinical studies, PXL770 has demonstrated broader potential in other chronic metabolic indications. Poxel is also evaluating earlier stage molecules from its AMPK activator and deuterated thiazolidinedione (TZD) platforms targeting other chronic and rare metabolic diseases.

About NASH

NASH is a metabolic disease with no clear disease origin that is quickly becoming a worldwide epidemic. It is characterized by the accumulation of fat in the liver causing inflammation and fibrosis. The disease can be silent for a long period of time, but once it accelerates, severe damage and liver cirrhosis can occur, which can significantly impact liver function or can even result in liver failure or liver cancer. Typical risk factors for NASH include obesity, elevated levels of blood lipids (such as cholesterol and triglycerides) and type 2 diabetes. Currently no curative or specific therapies are available.

About PXL770

PXL770 is a first-in-class AMPK activator. AMPK is a central regulator of multiple metabolic pathways leading to the control of lipid metabolism, glucose homeostasis and inflammation. Based on its 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 NASH.

About PXL065

PXL065 is a novel, proprietary deuterium-stabilized R-pioglitazone. Although pioglitazone is not approved by the FDA for the treatment of NASH, it is the most extensively studied drug for NASH and has demonstrated 'resolution of NASH without worsening of fibrosis' in a Phase 4 trial1. Pioglitazone is the only drug recommended for biopsy-proven NASH patients by the Practice Guidelines published by the AASLD and the European Association for the Study of the Liver (EASL)2. Pioglitazone's off-label use for NASH, however, has been limited due to the PPAR-related side effects, which include weight gain, bone fractures and fluid retention.

Pioglitazone is a 1:1 mixture of two mirror-image compounds (R- and S-stereoisomers) that interconvert in vivo. Using deuterium, we stabilized each stereoisomer and characterized their different pharmacological properties. In in vitro studies, PXL065 has been shown to target mitochondrial pyruvate carrier (MPC) as an inhibitor. In preclinical animal models, PXL065 exhibits the anti-inflammatory and NASH activity associated with pioglitazone with little or no weight gain or fluid retention, side effects which are associated with the S-stereoisomer. Based upon preclinical and Phase 1 results to date, Poxel believes that PXL065 may have a better therapeutic profile than pioglitazone for NASH.

About Poxel SA

Poxel is a dynamic biopharmaceutical company that uses its extensive expertise in developing innovative drugs for metabolic diseases, with a focus on type 2 diabetes and non-alcoholic steatohepatitis (NASH). In its mid-to-late stage pipeline, the Company is currently advancing three drug candidates as well as earlier-stage opportunities. Imeglimin, Poxel's first-in-class lead product, targets mitochondrial dysfunction. Poxel has a strategic partnership with Sumitomo Dainippon Pharma for Imeglimin in Japan, China, South Korea, Taiwan and nine other Southeast Asian countries. A Japanese new drug application (J-NDA) is under review by the Pharmaceuticals and Medical Devices Agency (PMDA) to request approval for the manufacturing and marketing of Imeglimin for the treatment of type 2 diabetes. Poxel also established a partnership with Roivant Sciences for Imeglimin's development and commercialization in countries outside of the partnership with Sumitomo Dainippon Pharma, including the U.S. and Europe. PXL770, a first-in-class direct adenosine monophosphate-activated protein kinase (AMPK) activator, has successfully completed a Phase 2a proof-of-concept trial for the treatment of NASH. The Phase 2a trial met its primary endpoint and study objectives. PXL770 could also have the potential to treat additional metabolic diseases. PXL065 (deuterium-stabilized R-pioglitazone), a MPC inhibitor, is in a streamlined Phase 2 trial for the treatment of NASH. Poxel also has additional earlier-stage programs from its AMPK activator and deuterated TZD platforms targeting chronic and rare metabolic diseases. The Company intends to generate further growth through strategic partnerships and pipeline development. Listed on Euronext Paris, Poxel is headquartered in Lyon, France, and has subsidiaries in Boston, MA, and Tokyo, Japan.

In the context of the COVID-19 outbreak, which was declared a pandemic by the World Health Organization (WHO) on March 12, 2020, the Company is regularly reviewing the impact of the outbreak on its business.

As of the date of this press release, and based on publicly available information, the Company has not identified the occurrence of any material negative effect on its business due to the COVID-19 pandemic that remains unresolved. However, the Company anticipates that the COVID-19 pandemic could have further material negative impact on its business operations. The worldwide impact of COVID-19 may notably affect the Company's internal organization and efficiency, particularly in countries where it operates and where confinement measures are implemented by the authorities. In addition, COVID-19 may impact market conditions and the Company's ability to seek additional funding or enter into partnerships. Particularly, delays in the supply of drug substance or drug products, in the initiation or the timing of results of preclinical and/or clinical trials, as well as delays linked to the responsiveness of regulatory authorities could occur, which could potentially have an impact on the Company's development programs and partnered programs. The Company will continue to actively monitor the situation.

All statements other than statements of historical fact included in this press release about future events are subject to (i) change without notice and (ii) factors beyond the Company's control. These statements may include, without limitation, any statements preceded by, followed by or including words such as 'target,' 'believe,' 'expect,' 'aim,' 'intend,' 'may,' 'anticipate,' 'estimate,' 'plan,' 'project,' 'will,' 'can have,' 'likely,' 'should,' 'would,' 'could' and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the 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.

Contact:

Tel: +1 617 818 2985

Email: jonae.barnes@poxelpharma.com

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