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PALMA, Spain—The orphan condition calciphylaxis—an extreme form of cardiovascular calcification (CVC)—results in high morbidity and mortality, characterized by extremely painful ischemic/necrotic wounds, and rapid progression of the disease leads to extreme pain, infection and death. There is no approved therapy, creating a significant need for a novel approach.
In general, accelerated CVC in the coronary arteries, heart valves and aorta can cause complications such as the development of cardiac dysfunction and failure, with an increased likelihood of arrhythmia and sudden cardiac death. CVC-related cardiovascular problems are fairly common among dialysis patients.
Enter the calcification disorders-focused biopharmaceutical company Sanifit, which recently announced positive topline results from the CaLIPSO Phase 2b international clinical trial of SNF472, an intravenous formulation of myo-inositol hexaphosphate with a novel mechanism of action for the treatment of hemodialysis patients with cardiovascular diseases linked to calcification.
The results, which were published in the journal Circulation and presented at the 2019 scientific sessions of the American Heart Association (which publishes the journal), revealed that the clinical trial met its primary endpoint, demonstrating significantly reduced progression of coronary artery calcium (CAC) volume in patients treated with SNF472, as compared to placebo.
Sanifit, a spinoff from the University of the Baleric Islands, has developed SNF472 as inhibitor of cardiovascular calcification in skin arterioles affecting mostly end-stage kidney disease patients, stated Dr. Paolo Raggi, principal investigator of the trial, who said, “Patients with end-stage kidney disease requiring dialysis, have a five to 30 times higher risk of death due to cardiovascular causes than the general population. The potential for SNF472 to attenuate cardiovascular calcification, and thereby reduce this risk, is incredibly exciting. I look forward to further investigation of SNF472 in this area.”
“SNF472 inhibits the development and progression of ectopic calcifications by binding to the growth sites of hydroxyapatite (HAP) crystals, the main component of calcification deposits, and in doing so blocks the formation and growth of HAP crystals in blood vessels, preventing CVC,” explained Dr. Alex Gold, chief medical officer of Sanifit .
The CaLIPSO trial, a 52-week, double-blind, randomized, placebo-controlled trial, assessed SNF472, a selective inhibitor of the formation and growth of hydroxyapatite crystals, in patients with end-stage kidney disease (ESKD) on hemodialysis. The trial compared progression of CAC volume score and other measurements of CVC by CT scan during 52 weeks of treatment with SNF472 or placebo, in addition to standard therapy, in adult patients with ESKD receiving hemodialysis.
The primary endpoint was change in CAC volume score from baseline to week 52. The primary analysis combined the SNF472 treatment groups and included all patients who received at least one dose of SNF472 or placebo and had a post-randomization evaluable CT scan. The study was conducted at 65 investigational sites in the United States, Spain and the United Kingdom.
The study concluded that, compared with placebo, 52 weeks of treatment with SNF472 significantly attenuated the progression of coronary artery calcification is patients with ESKD receiving hemodialysis. In addition, progression of aortic valve calcification was reduced, and the reduction in the progression of the thoracic aorta was less pronounced. SNF472 was well tolerated. Patients received contemporary management for ESKD mineral metabolism disorder, including drugs with the potential to at least partially attenuate the progression of CVC. Further studies are needed to determine whether SNF472 will decrease the risk of cardiovascular events in patients receiving hemodialysis.
Joan Perelló, CEO of Sanifit, said, “We are delighted with these positive top-line trial results, which show the clear promise of SNF472 in directly targeting the final common pathway in cardiovascular calcification. We believe this could lead to meaningful improvements in the treatment of patients with end-stage kidney disease. We look forward to reviewing the data further as we continue clinical development of SNF472.”
Launched in 2007, Sanifit expanded its activities in the USA in 2016 with the incorporation of a subsidiary with offices in San Diego. The company is preparing for a Phase 3 pivotal study. Sanifit has raised around $130 million, including a Series D funding of $61.8 million in mid-2019.