Sequana Medical announces positive top-line results from the North American pivotal alfapump® study (POSEIDON)
Sequana Medical NV (Euronext Brussels: SEQUA, the "Company" or "Sequana Medical"), a pioneer in the treatment of drug-resistant fluid overload in liver disease, heart failure and cancer, today announces positive top-line results from the North American pivotal POSEIDON study of the alfapump, a fully implantable, wirelessly charged, breakthrough device for the treatment of recurrent or refractory ascites due to liver cirrhosis. Data from 40 patients implanted with the alfapump in the Pivotal Cohort met all primary effectiveness endpoints of the study with statistical significance and primary safety endpoint data was in line with expectations. These positive data will enable the Company to file a Pre-Market Approval (PMA) application with the FDA, planned for H2 2023, intended to support the approval of the alfapump in the US.
Professor Florence Wong, University of Toronto, Hepatologist at Toronto General Hospital, Ontario, Canada and Principal Investigator for the POSEIDON study, commented: “Ascites imposes a significant health care burden and negative impact on quality of life for this large and growing patient population of patients with liver cirrhosis. These positive top-line results are very encouraging, indicating that the alfapump could provide great benefits to patients with cirrhosis and ascites, and dramatically reduce their visits to the hospital for paracentesis. The safety data regarding the primary safety endpoint are in line with expectations and reassuring for the potential of the alfapump as a long-term treatment in this patient population. I look forward to presenting these data at a scientific congress in due course to discuss this important innovation for a patient population that is in clear need of expanded treatment options.”
Ian Crosbie, Chief Executive Officer at Sequana Medical, added: “We are delighted with these excellent results, and the team is focused on bringing the alfapump to this large and growing patient population. We are grateful to the patients, clinicians and their teams who participated in this landmark study. The third party market assessment highlights the large number of recurrent or refractory liver ascites patients, with strong forecast growth driven in part by the Non-Alcoholic Steatohepatitis (“NASH”) epidemic. We believe that there is a clear need for improved treatment options for this important patient group, and we are preparing to commercialize the alfapump through our focused commercial team.”
Positive top-line data from 40 patients in the Pivotal Cohort
Forty patients with recurrent or refractory ascites due to liver cirrhosis have been implanted with the alfapump in the Pivotal Cohort of the POSEIDON study. Over one third of these patients had NASH or combined NASH etiology.
The primary effectiveness endpoint hypotheses include:
1) median per-patient ratio of post-implant three-month observation period (month four to six) (“Post-Implant Observation Period”) to the pre-implant three-month observation period (“Pre-Implant Observation Period”) with respect to number of therapeutic paracentesis (“TP”) is less than 0.5 (or a median reduction of at least 50%); and
2) at least 50% of patients achieve a 50% reduction in the requirement for TP in the same period.
Data from the Pivotal Cohort patients substantially exceeded the pre-defined thresholds for study success as shown in the table below.
Pivotal Cohort N=40
1) Frequency of TP
2) Proportion of patients with a 50% reduction in number of TP Post- vs Pre-Implant
77% of patients
Of the 40 patients implanted with the alfapump in the Pivotal Cohort, 26 patients completed alfapump therapy through day 180 post-implantation. These 26 patients have a median reduction of 100% (mean reduction of 93%) in frequency of TP in the Post-Implant Observation Period vs Pre-Implant Observation Period and 92% of patients have at least a 50% reduction in number of TP in the same period (iii). Pre-specified imputation methods were used to calculate the primary effectiveness endpoints in the other 14 patients that had exited the study prior to completing the six months post-implantation period. Of these 14 patients, eight were due to reasons such as death or withdrawal due to unrelated adverse event or for liver transplant and six were due to alfapump system, procedure or therapy related reasons.
The primary safety endpoint is the combined rate of i) open surgical re-intervention (requiring general anesthesia or laparotomy) due to pump system related adverse event or to restore pump functionality, ii) pump explant (without replacement) due to pump system related adverse event, or iii) pump system related death from time of pump implant through six months post-implantation as adjudicated by the Clinical Events Committee (CEC). There were six primary safety events in the 40 Pivotal Cohort patients, which is in line with Company expectations. Of the six primary safety events, three were explants due to wound or skin erosion, and three were explants due to patient-reported discomfort (all patient-reported discomfort events were adjudicated by the CEC as moderate severity). At the time of the primary endpoint analysis, no unanticipated adverse device effects (UADE (iv)) occurred during the course of the POSEIDON study.
Large and growing population of patients with recurrent or refractory ascites due to liver cirrhosis in North America
The Company has commissioned a market analysis by an international consultancy group that is experienced in such work to assess the North American market of recurrent or refractory ascites due to liver cirrhosis. Through a detailed analysis of claims from Medicare and multiple commercial payers, as well as a review of published literature, the analysis estimates there to be more than 60,000 patients in North America in 2022 with recurrent or refractory ascites. This population is forecast to grow at six to seven percent annually, reaching over 140,000 patients in 2032, with NASH being a major driver of this growth.
(i) Using pre-specified imputation methods
(ii) As per primary effectiveness endpoint hypotheses. Per protocol, testing conducted using nonparametric methods for data that is not normally distributed.
(iii) These observed patient data are not part of the main primary effectiveness endpoint analysis.
(iv) Unanticipated adverse device effect is any serious adverse effect on health or safety, any life-threatening problem or death caused by, or associated with a device, if that effect, problem, or death was not previously identified in nature, severity, or degree of incidence in the application; or any other unanticipated serious problem associated with a device that relates to the rights, safety, or welfare of subjects (source: www.fda.gov)