Spain's Sanifit chasing calcium disorder blockbuster

Spanish biotech Sanifit is targeting calcification disorders, and says it could have a potential multi-billion dollar treatment in two kidney failure indications.

Following a 36.6 million euro cash funding round, one of the largest for a Spanish biotech for many years, Sanifit has begun a mid-stage trial of its SNF 472 in calciphylaxis, a devastating rare disease in dialysis patients.

Calciphylaxis is characterised by vascular calcification and thrombosis leading to necrosis of the skin and fatty tissue.

Patients with calciphylaxis experience painful skin ulcers with a high risk of severe infection and a 50% rate of death within the first year after diagnosis.

The company will begin a larger mid-stage trial in cardiovascular disease in end-stage renal disease (ESRD) this year, which the company plans to expand to phase 3.

High calcium levels are thought to be a risk factor for cardiovascular disease in ESRD, caused when blood vessels become hardened by calcium, with a knock-on effect on the performance of the heart.

This can lead to heart failure and arrhythmia, CEO Joan Perello, told pharmaphorum in a telephone interview.

Reducing cardiovascular events in ESRD would be a potential blockbuster indication with market potential of over 2 billion euros.

If approved the drug would be the first to interact directly with the calcium crystals formed in these conditions, as currently approved therapies only aim to lower levels of calcium in the blood stream.

Perello said the phase 2b cardiovascular disease trial could be larger than many phase 3 trials, in 75 hospitals across five countries and enrolling around 400 patients.

By 2019 the company aims to have phase 3 data in calciphylaxis and phase 2b data in the larger cardiovascular indication – at which point it will decide whether to look to license the drug to a big pharma company, or launch the company on the stock exchange to raise cash for further development.

Perello told pharmaphorum: “It is a good option for an IPO or a licensing deal.”

Perello said the company would consider a filing on early stage if results are compelling, but at this time no such move is planned.

The large funding round has also had helped to kick-start investment in Spain, at a time when the country’s economy has rebounded following the economic crisis, Perello said.

“I am happy that Sanifit was a first mover. Some of my investors have already had second or third investments in Spain.”