Ipsen Presents Preliminary Results of Exploratory Proof-of-Concept Study with Tasquinimod in Four Advanced Tumor Types at the ESMO 2014 Congress

Regulatory News:

Ipsen (Euronext: IPN; ADR: IPSEY) today announced the presentation at the ESMO 2014 Congress (26-30 September in Madrid) of the preliminary results of the phase II proof-of-concept clinical trial with tasquinimod in monotherapy, evaluating the compound in four advanced tumor types.

The main objective of the study was to determine the clinical activity of tasquinimod in advanced hepatocellular (HCC), ovarian (OC), renal cell (RCC) and gastric (GC) carcinomas in patients who had progressed after standard anti-tumor therapies. Primary endpoint was the PFS rate at a predefined time for each cohort. Secondary objectives included PFS, response rate, OS, safety, pharmacokinetics and biomarkers.

The data did not support further development of tasquinimod in monotherapy in heavily pretreated patients with advanced OC, RCC and GC. Pharmacokinetic and biomarkers analyses are ongoing. Preliminary results from the futility analysis reported sufficient clinical activity to complete the recruitment of the HCC cohort for which results are expected in 2015.

The safety profile was consistent with the known safety profile of tasquinimod in previous studies.

Data from the HCC cohort was presented (Poster p-171)¹ at the International Liver Cancer Association 8^th Annual Conference (5–7 September 2014, Kyoto, Japan).

About the study

The "Umbrella” trial is a Phase II, multicenter, open-label, proof of concept study of tasquinimod in patients with advanced hepatocellular (HCC), ovarian (OC), renal cell (RCC) and gastric (GC) carcinomas

About tasquinimod

Tasquinimod is a novel small molecule that targets the tumor microenvironment by binding to S100A9 and modulating regulatory myeloid cell functions, exerting immunomodulatory, anti-angiogenic and anti-metastatic properties. Tasquinimod may also suppress the tumor hypoxic response, contributing to its effect on the tumor microenvironment. Today the development of tasquinimod is principally focused on the treatment of prostate cancer, but early clinical studies in other cancer indications are performed.

About Ipsen

Ipsen is a global specialty-driven pharmaceutical company with total sales exceeding €1.2 billion in 2013. Ipsen’s ambition is to become a leader in specialty healthcare solutions for targeted debilitating diseases. Its development strategy is supported by 3 franchises: neurology, endocrinology and uro-oncology. Moreover, the Group has an active policy of partnerships. Ipsen's R&D is focused on its innovative and differentiated technological platforms, peptides and toxins. In 2013, R&D expenditure totaled close to €196 million, representing more than 16% of Group sales. Moreover, Ipsen also has a significant presence in primary care. The Group has close to 4,600 employees worldwide. Ipsen’s shares are traded on segment A of Euronext Paris (stock code: IPN, ISIN code: FR0010259150) and eligible to the "Service de Règlement Différé” ("SRD”). The Group is part of the SBF 120 index. Ipsen has implemented a Sponsored Level I American Depositary Receipt (ADR) program, which trade on the over-the-counter market in the United States under the symbol IPSEY. For more information on Ipsen, visit www.ipsen.com.

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¹ Exploring the activity of tasquinimod, a compound with a novel mechanism of action, in patients with advanced hepatocellular carcinoma (HCC), previously treated with sorafenib