Sequana Medical NV (Euronext Brussels: SEQUA), a commercial stage medical device company focused on the development of innovative treatment solutions for the management of liver disease, heart failure, malignant ascites and other fluid imbalance disorders, today announces that the U.S. Food and Drug Administration (FDA) has unconditionally approved its Investigational Device Exemption (IDE) application for POSEIDON,  the alfapump pivotal study in patients with recurrent or refractory ascites due to liver cirrhosis. The study is intended to support approval and reimbursement of the alfapump in the U.S. & Canada.

“We are delighted with the IDE approval and optimised clinical trial design for POSEIDON and are grateful for the close collaboration with the FDA. As a result, and together with the Breakthrough Device Designation granted in January this year, we have brought forward our plans for U.S. launch of the alfapump to H1 2022”, said Ian Crosbie, CEO of Sequana Medical. “This is good news for patients as the growing prevalence of cirrhosis due to Non-Alcoholic Steatohepatitis (NASH) makes the need for a modern and convenient ascites treatment all the more important. We are also encouraged by the recent announcement from CMS in respect of proposed changes to the new technology add-on payment (NTAP) pathway for breakthrough devices, which we believe is a very promising development for the alfapump.”

“Thanks to the positive interactive review process, we received an unconditional IDE approval in just 30 days”, said Gijs Klarenbeek, CMO of Sequana Medical. “The FDA has provided us with invaluable advice on the design of the POSEIDON study. The final study design allows for a reduced number of study patients to be enrolled and a shorter follow-up time for primary endpoint analysis. We look forward to a continued collaboration with the FDA and are excited to start POSEIDON together with the investigators participating in this study.”

About the pivotal POSEIDON study

This is a single arm, open-label, within subject crossover study of the alfapump in patients with recurrent or refractory ascites due to liver cirrhosis in centres across the U.S. and Canada. Sixty patients will be enrolled in the study cohort to enter the pre-implant observation period, allowing for up to 50 patients to be implanted with the alfapump for primary endpoint analysis. The study allows for up to a further 30 patients to be enrolled in a training cohort, to ensure centres are experienced with the alfapump before the study cohort is enrolled. An application for Investigational Testing Authorisation (ITA) has been submitted to Health Canada as well.

Patients will enter into a 3-month pre-implant observation period in which they will receive standard of care therapy, consisting of paracentesis, before the alfapump is implanted. Eligible patients will be implanted with the alfapump and monitored during a 3-month stabilisation period to adjust the settings of the alfapump as per patients’ needs. After this period of stabilisation, a 3-month post-implant observation period will begin. The primary effectiveness outcomes of the study include the proportion of patients with a 50% reduction in the overall average frequency of therapeutic paracentesis per month in the post-implant observation period (month three to month six after implantation) as compared to the pre-implant observation period and the per-patient ratio of post-implant to pre-implant with respect to average monthly number of therapeutic paracentesis. The primary safety endpoint is the rate of alfapump related re-interventions adjudicated by the Clinical Events Committee. Patients will be followed up for up to two years for analysis of secondary outcome measurements including safety (device and/or procedure-related adverse events), quality of life (assessed by general SF-36 as well as disease-specific Ascites-Q questionnaires), patients’ nutritional status, health economics and overall survival.

About the alfapump in recurrent or refractory ascites due to liver cirrhosis

Ascites, a key complication of liver cirrhosis, is the accumulation of ascitic fluid in the abdomen. Patients may accumulate as much as 10 to 15 litres of ascitic fluid within the abdomen every 15 days. Patients suffering from recurrent or refractory ascites have limited treatment options and often have severely impacted quality of life due to the severe swelling of the abdomen, resulting in pain, difficulty breathing, moving, sleeping and eating, severe nausea and constipation. Existing treatment options carry the risk of significant or life-threatening side effects, provide only short-term symptomatic relief or have very limited availability. The number of patients with refractory liver ascites is forecast to increase dramatically due to the growing prevalence of NASH (Non-alcoholic Steatohepatitis).

Sequana Medical’s alfapump is a fully-implanted, programmable, wireless, CE-marked system that automatically pumps ascites from the peritoneal cavity into the bladder, where the body eliminates the ascites naturally through urination. The potential of the alfapump to address the unmet medical need in patients with recurrent or refractory ascites has been demonstrated in multiple clinical studies showing a significant reduction in the need for large volume paracentesis, which is paracentesis where at least 5 litres of fluid is removed (i.e., the current standard of care), and a significant improvement in patients’ quality of life.

A feasibility study in North America in patients with refractory or recurrent ascites has been completed and results were presented at the AASLD (American Association for the Study of Liver Diseases) annual meetings in October 2017 and November 2018.