Retrophin Raises $100 Million to Advance Rare Disease Pipeline

Rare Daily Staff

Retrophin said it raised $100.8 million in a secondary public offering of 6.5 million shares of its common stock at $15.50 per share.

In addition, Retrophin has granted the offering’s underwriters a 30-day option to purchase up to an additional 975,000 shares at the offering price.

The rare disease drugmakers’ lead pipeline candidate sparsentan is an experimental therapy in late-stage development for focal segmental glomerulosclerosis (FSGS) and IgA nephropathy (IgAN), rare disorders characterized by progressive scarring of the kidney that often lead to end-stage renal disease.

Retrophin also has partnerships with patient advocacy groups and government researchers to identify potential therapeutics for NGLY1 deficiency and Alagille syndrome, conditions with no approved treatment options.

Retrophin’s R&D efforts are partially supported by revenues from its commercial products including: Chenodal, a synthetic bile acid used to treat patients will gallstones who can’t have surgery and for the treatment of cerebrotendinous xanthomatosis (CTX), a rare inborn error of cholesterol metabolism that often causes chronic diarrhea in infants and leads to neurodegeneration; Cholbam for the treatment of children and adults with rare bile acid synthesis disorders or peroxisomal disorders; and Thiola and Thiola EC for the treatment of cystinuria, a rare genetic disorder that causes increased cystine levels in urine and leads to recurring cystine kidney stones.