Retrophin (RTRX) Corporate Presentation - Slideshow

Sparsentan Clinical Development - Sparsentan is in Phase 3 development for FSGS and IgAN, rare kidney disorders with no approved medicines, targeting ~40,000 FSGS patients in the U S and EU and ~100,000+ IgAN patients in the U S and EU [58] - Phase 2 DUET study showed Sparsentan more than doubled the reduction of proteinuria vs Irbesartan (44 8% vs 18 5%, p=0 006) [30] - In the DUET study, 28 1% of Sparsentan-treated patients achieved FPRE (UP/C ≤ 1 5 g/g and >40% reduction in UP/C) compared to 9 4% in the Irbesartan group (p=0 040) [33, 35] - eGFR remained stable in Sparsentan-treated patients over 84 weeks in the DUET study [40] - Phase 3 DUPLEX study for FSGS and PROTECT study for IgAN are enrolling, with top-line data expected in 1Q21 and 2H21, respectively [45, 48] Financial Overview - Retrophin reported net sales of $48 4 million in 2Q20 and $175 3 million in FY19 [56] - The company's operating expenses were $71 5 million in 2Q20 and $312 7 million in FY19 [56] - Retrophin had a cash balance of approximately $457 4 million as of June 30, 2020, which is expected to fund operations beyond the planned proteinuria readout of pivotal studies and into 2023 [56, 59] Commercial Portfolio - Retrophin has a diversified product portfolio for rare diseases, including Chenodal, Cholbam, and Thiola [51] - The company estimates an addressable population of 4,000 to 5,000 cystinuria patients for Thiola [51] - Net product sales have grown from $28 million in FY 2014 to $175 million in FY 2019 [53]