Rare Daily Staff
Vifor Pharma is making a big push in chronic kidney disease with the acquisition of Sanifit Therapeutics, a Spanish clinical-stage cardio-renal company focused on treatments for end-stage kidney disease patients with progressive vascular calcification disorders, and Inositec, a Swiss company, developing first-in-class non-dialysis treatments for soft tissue and vascular calcification disorders.
Sanifit’s SNF472 is a novel, first-in-class intravenously administered inhibitor of vascular calcification in development for the potential treatment of peripheral artery disease (PAD) and calcific uremic arteriolopathy (CUA) in hemodialysis, both rare diseases related to progressive cardiovascular calcification (CVC). There are currently no approved medicines indicated for CUA or for PAD specifically in this population. SNF472 has already been granted orphan drug designation for the treatment of CUA and PAD by the U.S. Food and Drug Administration and for CUA by the European Medicines Agency.
Calcific uremic arteriolopathy also known as calciphylaxis (CUA), is a rare but potentially devastating condition most often observed in patients with end-stage renal disease, although it does occasionally develop in patients without renal failure. Calciphylaxis is a severe form of CVC in which the calcium deposits block small blood vessels in skin and fat tissue. These blockages cause the development of intensely painful and debilitating chronic skin lesions. Calciphylaxis is a devastating rare disease that affects 1-4 percent of dialysis patients and has a 1-year mortality rate of 55 percent.
Peripheral artery disease (PAD) is a progressive vascular calcific disorder particularly common in chronic kidney disease patients and characterized by stenosis of arteries in the lower extremities causing reduced blood flow to the legs. Patients present with recurrent fatigue or pain that is known as intermittent claudication, which causes a poor quality of life. Progressive PAD requires vascular surgery to reduce the risk for limb amputation.
“From the very beginning, Sanifit has been a pioneer of new approaches to treat calcification disorders, a huge area of unmet need,” said Joan Perelló, CEO of Sanifit. “Vifor Pharma is the ideal partner to take the development of Sanifit’s calcification franchise forward and bring these novel treatments to patients as quickly as possible.”
Inositec’s novel asset INS-3001, is a once-daily subcutaneous treatment for patients with vascular calcification disorders PAD and aortic valve stenosis (AVS), which are both major contributors to cardiovascular morbidity and mortality in affected patient populations. With INS-3001 daily subcutaneous dosing, patients with earlier stages of vascular calcification can be optimally treated, while end-stage kidney disease patients will benefit from the three times per week dosing regimen of SNF472.
“These acquisitions are a perfect fit for our expanding nephrology pipeline, which now includes vascular calcification inhibition treatments across various stages of non-dialysis CKD and even non-CKD patient populations,” said Abbas Hussain, CEO of Vifor Pharma.
Under the terms of the acquisition, Vifor Pharma will acquire 100 percent of the outstanding shares in Sanifit Therapeutics, receiving full global rights for SNF472, for an upfront payment of $231 million (EUR 205 million), clinical, regulatory and market access milestones for up to $191 million (EUR 170 million), and tiered sales-based milestones. Vifor Pharma will acquire 100 percent of the shares in Inositec, receiving full global rights for the lead asset INS-3001, for an upfront payment of $22.5 million (CHF 20 million) and success based clinical earn-out payments in the low triple digit million range.
Sanifit conducted a phase-2b trial in 274 patients to assess the effect of SNF472 on slowing arterial calcification, a major risk factor for cardiovascular disease in dialysis patients. The trial met its primary endpoint in reducing coronary artery calcium progression in patients treated with SNF472, compared to patients receiving placebo over a 52-week period. SNF472 is currently in phase 3 trials in CUA in patients on dialysis, to measure primary endpoints for wound healing and pain. A phase 3 trial in PAD in patients on dialysis, is planned to commence in 2022.
Inositec’s lead vascular calcification inhibitor, INS-3001, is being developed to treat indications of high unmet medical need and has obtained approval for its phase 1 trial, with the first healthy volunteer to be dosed on November 30, 2021. If successful, the plan is to initiate two phase 2 programs in 2023, one in non-dialysis CKD patients suffering from PAD, and a separate trial in patients suffering from AVS, a serious condition whereby the aortic valves calcify to the extent that surgical intervention is the only available option.
The expected addressable patient population in the U.S. and EU suffering from calcifying non-dialysis and dialysis-associated PAD is estimated to be more than 600,000. CUA is an ultra-orphan disease with approximately 10,000 patients across the U.S. and EU. For AVS Vifor estimates approximately 1.1 million patients in the two geographies eligible to receive treatment.
Photo: Abbas Hussain, CEO of Vifor Pharma