Ghent,
Recurrent or refractory ascites is a severe condition characterized by the accumulation of fluid in the abdomen. The current standard treatment involves therapeutic paracentesis, an invasive and burdensome procedure that drains ascites from the abdomen using a large needle over an extended period. If approved by the FDA, the alfapump could become the first active implantable medical device in the US that automatically and continuously removes ascites from the abdomen into the bladder, where it is naturally eliminated through urination.
The PMA filing is based on the successful execution of
Data from the patient preference study and a matched cohort analysis of the NACSELDiv registry with the POSEIDON Pivotal Cohort indicated that US patients have a strong preference for the alfapump vs standard paracentesis procedures and that the safety profile of the alfapump is comparable to standard of care.
The North American market of recurrent and refractory ascites due to liver cirrhosis is forecast to grow 6-7% per year, from 78,000 patients in 2025, reaching a market opportunity for alfapump of over
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Strong and durable clinical alfapump profile demonstrated in pivotal POSEIDON study
Pivotal Cohort patients from the POSEIDON study had a 100% median reduction in therapeutic paracentesis in the 4-6 month post-implant period vs the three month pre-implant period, and this was maintained in the 7-12 month post-implant period. These data show that the alfapump has a sustained effect on controlling ascites, virtually eliminating the need for therapeutic paracentesis.
During the 0-6 month post-implant period, the alfapump was explanted in six Pivotal Cohort patients (three due to wound or skin erosion and three due to patient-reported discomfort of moderate severity) and in two Pivotal Cohort patients during the 7-12 month post-implant period (one due to a urinary tract infection and one due to wound dehiscence). The number of Major Adverse Events (MAEsvi) and serious infections were in line with expectations. Importantly, creatine and eGFRvii levels of alfapump-treated patients over 12-month follow-up indicated a stable renal function. Overall, these safety data indicate that the alfapump has a robust safety profile over long-term follow-up. In addition, a matched cohort analysis of the NACSELD registry with the POSEIDON Pivotal Cohort indicated that the safety profile of the alfapump is in line with expectations and comparable to standard paracentesis procedures.
Patients from the POSEIDON study had a clinically meaningful improvement in quality of life (assessed through the physical component score of SF36, a general health quality of life measure, and the Ascites Q score, a quality of life measure specific for patients with ascites) at 6 months post-implant vs three months pre-implant and maintained this at 12 months post-implant, despite disease progression.
The overall trend in survivalviii in patients implanted with the alfapump in the POSEIDON study remained positive over a longer term, with a Kaplan-Meier estimate indicating over 70% survival probability at 12 and 18 months post-implant. This compares favourably with the published literature reporting a predicted survival probability for refractory ascites patients with a similar MELDix score and receiving paracentesis of approximately 17% at 12 months and 5% at 18 monthsx.
Data from the POSEIDON study will be submitted for publication in a peer-reviewed journal.
Outcome patient preference studyxi
The patient preference study was conducted by RTI Health Solutions using a discrete-choice experiment methodology to elicit patient preference for attributes of an implantable pump as a novel interventional treatment for ascites. In total, 125 US patients with a comparable patient profile as the Pivotal Cohort in the POSEIDON study, completed the survey. Results indicate that US patients have a strong preference for the alfapump vs standard paracentesis procedures, with reduction in paracentesis frequency and additional ascites good health days as important attributes. The study also indicated that the alfapump benefit-risk profile from the POSEIDON Pivotal Cohort is superior to what patients require from a novel implantable pump.
About recurrent and refractory ascites due to liver cirrhosis
Recurrent and refractory ascites is a key complication of liver cirrhosis, characterized by the accumulation of fluid in the abdomen. These patients can have up to 15 liters of extra fluid in their bodies, causing many health issues and severely impacting their daily life. Although diuretics are standard of care, the problem is that in many patients they are no longer effective and / or tolerable, requiring patients to undergo regular paracentesis. Paracentesis is a painful and burdensome procedure that drains ascites from the abdomen using a large needle over an extended period, with only short term benefit for the patients, requiring frequent hospitalizations and severely impacting their quality of life.
The North American market for recurrent or refractory ascites due to liver cirrhosis is estimated at 78,000 patients in 2025, and this number is expected to grow at 6-7% per year, reaching over 170,000 patients in 2035 due to the strong growth of non-alcoholic steatohepatitis (NASH) / metabolic dysfunction-associated steatohepatitis (MASH).
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The Company submitted a Premarket Approval (PMA) application to the
Results of the Company's RED DESERT and SAHARA proof-of-concept studies in heart failure support DSR's mechanism of action as breaking the vicious cycle of cardiorenal syndrome. MOJAVE, a US randomized controlled multi-center Phase 1/2a DSR clinical study is ongoing, seeking to confirm the strong efficacy seen in the RED DESERT and SAHARA studies. All three patients from the non-randomized cohort have been successfully treated and the randomized cohort of up to a further 30 patients will start following DSMB approval, planned for Q1 2024.
Important Regulatory Disclaimers
The alfapump system is currently not approved in
Note: alfapump and DSR are registered trademarks.
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