Variant signs worldwide exclusive license and development agreement with L&F Research

Dec 20 2015

Variant Pharmaceuticals, Inc., an emerging pharmaceutical company focusing on rare diseases and other specialty conditions with limited or sub-optimal therapeutic options, announced today a worldwide exclusive license and development agreement with L&F Research LLC to advance development of Variant's lead product, VAR 200. VAR 200 has potential for treating multiple indications associated with the damaging effects of cellular accumulation of cholesterol and lipids on the kidney, including focal segmental glomerulosclerosis (FSGS), a rare disease affecting up to 133,000 people in the United States. FSGS is a leading cause of chronic kidney failure, and there are currently no targeted drug treatments nor cure.

"VAR 200, if successfully developed and commercialized, will redefine the treatment of FSGS," says Pablo Guzman, MD, FACC, and acting Chief Medical Officer at Variant. "We believe that VAR 200 has potential to minimize or prevent kidney cell damage and improve FSGS prognosis."

About FSGS

FSGS is a progressive form of kidney disease associated with accumulation of cholesterol and lipids in the parts of the kidneys that filter waste out of the blood (glomeruli). Damage to the glomeruli causes protein from the blood to leak into the urine, a condition known as proteinuria. As the level of protein increases in the urine, levels in the blood decrease and patients develop a specific set of symptoms, known as nephrotic syndrome. Symptoms of nephrotic syndrome include swelling, especially in the legs and around the eyes, and difficult to treat high blood pressure, or hypertension. In patients with FSGS, high blood cholesterol levels and an increased tendency to form blood clots are also common. As FSGS progresses kidney function worsens. More than 35% of all FSGS patients develop end stage kidney disease within 10 years, requiring dialysis and ultimately kidney transplant to survive. Approximately 1,000 FSGS patients receive a kidney transplant each year.

"We are excited about the potential for VAR 200 to halt or delay the progression of FSGS, and improve quality of life in patients with FSGS," says Alessia Fornoni, MD, PhD, Co-inventor of the licensed technology, and Professor of Medicine at the University of Miami Miller School of Medicine, Director of the Katz Family Drug Discovery Center at the Miller School, and Chief Scientific Officer of L&F Health LLC, an affiliate of L&F Research. "Studies conducted in our lab using an experimental mouse model for FSGS demonstrated that the active ingredient in VAR 200 completely prevented kidney cell damage when administered subcutaneously."

"We salute Dr. Fornoni's innovative contributions as a physician, a researcher and an advocate of people living with kidney disease," says Marc A. Golden, Co-founder and Chief Executive Officer at L&F Research. "Dr. Fornoni and her University of Miami colleague Dr. Sandra Merscher have done groundbreaking work in exploring potential treatments to ameliorate the damaging effects of cellular accumulation of cholesterol and lipids on the kidney. We appreciate the expertise and contributions of U Innovation, led by Vice Provost for Innovation Dr. Norma Kenyon and Technology Transfer Director Jim O'Connell, with special thanks to Senior Licensing Associate Dan Catron."

About VAR 200

VAR 200 is a preclinical asset intended to minimize or prevent kidney cell damage and maintain adequate kidney function by trapping cellular cholesterol and lipids, enabling them to be properly metabolized and removed from the cell, lowering kidney cell lipids toward normal physiological ranges.

"Acquisition of VAR 200 exemplifies Variant's commitment to address high unmet medical needs through innovative treatments for rare diseases and specialty medicines," stated Stephen C. Glover, Co-founder and Chief Executive Officer at Variant. "If our development efforts are successful, we believe that VAR 200 will be a first-in-class treatment for FSGS, with potential for treating other conditions associated with the damaging effects of cellular accumulation of cholesterol and lipids in the kidney, such as Alport Syndrome, a rare disease affecting 50,000-60,000 in the U.S., and Diabetic Kidney Disease, affecting 12 Million in the U.S."