Retrophin (RTRX) Misses Q4 EPS by 13c, Says Phase 3 Trial of Sparsentan in FSGS to Initiate in Second Half of 2017

Retrophin (NASDAQ: RTRX) reported Q4 EPS of ($0.39), $0.13 worse than the analyst estimate of ($0.26). Revenue for the quarter came in at $37.3 million versus the consensus estimate of $35.95 million.

Retrophin, Inc. (NASDAQ: RTRX) today provided an update on the regulatory pathway for its late-stage product candidate sparsentan, and announced fourth quarter and full year 2016 financial results. Following guidance received from the U.S. Food and Drug Administration (FDA) during the Company’s End of Phase 2 meeting, the Company plans to initiate a single Phase 3 clinical trial to enable a New Drug Application (NDA) filing for sparsentan for the treatment of focal segmental glomerulosclerosis (FSGS). Notably, the trial will include an interim analysis of proteinuria as a surrogate endpoint to serve as the basis for an NDA filing for Subpart H accelerated approval of sparsentan. The confirmatory endpoint of the study will subsequently compare changes from baseline in estimated glomerular filtration rate, or eGFR. The Company is working with the FDA to finalize the study protocol and expects to initiate the trial in the second half of 2017.

“We are pleased to have received regulatory guidance from the FDA for sparsentan and look forward to working with them to finalize our Phase 3 study protocol and initiate this important trial later this year,” said Stephen Aselage, chief executive officer of Retrophin. “We are particularly encouraged by the agency’s alignment on the use of an interim analysis of proteinuria in this trial, which gives us an opportunity to expedite sparsentan’s path to approval. We are eager to build on the strong data from the Phase 2 DUET study to further demonstrate the significant potential of sparsentan in the treatment of FSGS.”

Program Updates

Sparsentan

• The Company today announced plans to initiate a Phase 3 clinical trial of sparsentan in FSGS during the second half of 2017. The study will include an interim analysis of proteinuria to serve as the basis for an NDA filing for accelerated approval of sparsentan. The confirmatory endpoint of the study will compare changes from baseline in eGFR, which is widely regarded as the best overall measure of kidney function.
• In the fourth quarter of 2016, the Company presented positive data from the Phase 2 DUET study of sparsentan at ASN Kidney Week 2016. These data included an analysis of the secondary endpoint showing that a significantly greater proportion of patients receiving sparsentan achieved modified partial remission (mPR) of proteinuria, compared to irbesartan-treated patients. mPR of proteinuria is defined as proteinuria levels of less than or equal to 1.5 g/g and greater than 40 percent reduction of proteinuria from baseline, and is associated with long-term preservation of renal function in FSGS. In addition, four patients receiving sparsentan achieved complete remission, compared to zero irbesartan-treated patients. The data presented also showed sparsentan was generally safe and well-tolerated in the study.

RE-024

• During the fourth quarter of 2016, the Company reached a SPA agreement with the FDA on the design of the Phase 3 FORT study of RE-024. The agreement indicates concurrence by the FDA that the trial design can adequately support an NDA filing seeking U.S. approval of RE-024 for the treatment of PKAN.
• The Company expects to begin dosing the first PKAN patients in the Phase 3 FORT study of RE-024 in mid-2017.
• The four PKAN patients receiving RE-024 under physician-initiated treatment outside of the U.S. continue on therapy and remain stable.

Liquid ursodeoxycholic acid (L-UDCA)

• Development efforts continue with the intention of making L-UDCA commercially available to the subset of primary biliary cholangitis patients who have difficulty swallowing.

Chenodal® (chenodeoxycholic acid)

• The prevalence study of cerebrotendinous xanthomatosis (CTX) in patient populations diagnosed with early-onset idiopathic bilateral cataracts continues to enroll subjects and serve as a valuable approach to raise awareness of the disorder.

2017 Outlook

The Company expects full year 2017 net product sales to be in the range of $150.0 to $160.0 million. The anticipated double-digit percent increase over 2016 is expected to be primarily driven by growth across all three products.

For earnings history and earnings-related data on Retrophin (RTRX) click here.

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